XyloCor Therapeutics Reports Positive Topline Safety and Efficacy Results from Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina

• No serious adverse events related to drug product were reported

• Patients demonstrated improvements in exercise capacity and reductions in episodes of chest pain

• Cardiac imaging results provide mechanistic evidence supporting the therapeutic potential of XC001 in cardiovascular disease

XyloCor Therapeutics, a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced completion of the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. EXACT met both safety and efficacy objectives. There were no safety issues related to drug product or unexpected serious adverse events related to XC001 administration. Six-month data from 28 patients in the Phase 2 portion of the study showed improvements in several key efficacy measures, including reduction in ischemic burden.

“We are excited to see EXACT completing its 6-month endpoint. The trial met all of its safety and exploratory objectives, showing intriguing benefits in these needy patients across a variety of objective and subjective measures,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “The strong range of mechanistic evidence demonstrate that administration of XC001 is a scientifically-sound approach for achieving a biological effect that has the potential to improve patients’ quality of life.”

XC001 is a one-time gene therapy designed to reduce ischemic burden by creating new blood vessels in the heart. In the Phase 2 portion of the EXACT trial, evidence of the drug’s mechanism of action was demonstrated by the reduction of ischemic burden measured by cardiac positron emission tomography (PET) imaging. The reduction in ischemic burden was accompanied by an improvement in total exercise duration, an important measure of exercise capacity. Prior to treatment, almost all subjects had marked limitations on ordinary physical activity. Six months after treatment, nearly half of all subjects were able to conduct ordinary physical activity without causing angina. The data from the Phase 2 EXACT study are potentially meaningful for patients with refractory angina, which includes more than one million people in the United States, who have no treatment options.

“We are excited to share this positive topline data from the Phase 2 portion of the EXACT trial, reinforcing our confidence in XC001 as a novel therapeutic approach with the potential to address the significant unmet medical needs of people with refractory angina,” said Al Gianchetti, President and CEO of XyloCor. "We now look forward to pursuing key upcoming milestones in XC001’s continued development, including finalizing our pivotal trial design through our ongoing discussions with the FDA and other regulatory authorities.”

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About the EXACT Study

The recently completed Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 1011 vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by a cardiac surgeon.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics is a private, clinical-stage biopharmaceutical company developing potential best-in-class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Corporate and Investor Relations:

A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com
610-541-2056

Media Contact:
Mike Beyer, Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

Priothera announces first patients enrolled in pivotal MO-TRANS global Phase 2b/3 study with mocravimod as an adjunctive and maintenance therapy for patients with AML undergoing allogeneic HCT

• Mocravimod is the only S1PR modulator being developed to treat blood cancers and improve CAR-T therapy

• Phase 1b/2a data has shown mocravimod is safe and well tolerated

Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod today announced that the first patients have been enrolled in the pivotal MO-TRANS global Phase 2b/3 study evaluating mocravimod in AML patients undergoing allogeneic hematopoietic cell transplant (HCT).

Mocravimod, a sphingosine 1 phosphate (S1P) receptor modulator which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allogeneic HCT. Mocravimod has shown a clinically relevant benefit in an early clinical study in patients with hematologic malignancies undergoing allogeneic HCT.

Priothera is initiating the pivotal MO-TRANS global Phase 2b/3 study in Europe, Israel, the US and in additional Asian and Latin American countries, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HCT. The double-blind, placebo-controlled study assesses relapse-free and overall survival of two doses of mocravimod in comparison to placebo. Topline data from this study are expected in 2025.

Marcos de Lima, M.D., is the Principal Investigator for the MO-TRANS global Phase 2b/3 trial. Dr. de Lima is professor of medicine at The Ohio State University College of Medicine and a hematologist-oncologist at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute.

Prof de Lima said: “We are excited to be part of the important MO-TRANS global Phase 2b/3 study to investigate mocravimod, a potential new adjunctive and maintenance therapy for patients with Acute Myeloid Leukemia undergoing allogeneic Hematopoietic Cell Transplant. Maintenance therapy is fast becoming the new frontier in the treatment of AML and we are committed to bringing forth new innovative therapies to AML patients.”

Elisabeth Kueenburg, M.D., Chief Medical Officer at Priothera, commented: “This MO-TRANS global Phase 2b/3 study builds on pre-clinical and clinical proof of concept studies which demonstrated mocravimod’s ability to improve survival outcomes for patients with hematological malignancies requiring allogeneic HCT. The mode of action has been well-established in autoimmune indications, but never in hematology. Mocravimod has the potential to be a first-in-class therapy in maintaining the graft-versus-leukemia effect, while preventing graft-versus-host disease, one of the most serious complications of allogeneic HCT. We expect this trial to deliver important clinical data towards the registration of mocravimod in this indication.”

Florent Gros, Co-Founder and CEO of Priothera, said: “Having successfully enrolled the first AML patients undergoing allogeneic HCT in our MO-TRANS global study represents a significant milestone for Priothera as we believe mocravimod has the potential to address a significant unmet need. Furthermore, we anticipate a strong uptake in patient enrollment with a significant number of patients currently being identified. We look forward to seeing topline results in 2025."

***

About mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CAR-T cell therapy.

Mocravimod is currently being investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic cell transplantation (HCT). Allogeneic HCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages mocravimod’s unique mode of action to maintain the beneficial graft-versus leukemia (GVL) activity, while reducing tissue damage resulting from graft-versus-host disease (GVHD), both a consequence of allogeneic HCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and aims to improve patients' quality of life.

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CAR-T cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Unlike immunosuppressive drugs, mocravimod does not suppress the graft-versus-leukemia (GVL) benefits in patients receiving HCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden), EarlyBird Venture Capital (Berlin, Germany), as well as non-dilutive financing in the form of loans from the European Investment Bank under its Venture Debt Instrument and Bpifrance (Grand Est Bpifrance) in the form of a R&D innovation loan.

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contacts

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting

Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Inotrem Receives Funding from the Crohn’s & Colitis Foundation to help develop new therapeutic approaches in Inflammatory Bowel Disease (IBD)

The funding will support the development of INO-02 a new long-acting therapeutic approach to modulate the TREM-1 pathway.

Inotrem, an advanced clinical stage biotech company specialized in immunotherapies for acute and chronic inflammatory syndromes, announced today it received funding from the Crohn’s & Colitis Foundation’s IBD Ventures under a program to accelerate research and development that aim to improve the quality of life for patients with IBD. The funding will support the development of a new therapeutic approach in IBD focusing on the TREM-1 pathway.

The funding follows a R&D collaboration between Inotrem, and the Crohn’s & Colitis Foundation announced in April 2022. The R&D collaboration seeks to pave the way of a personalized medicine approach in IBD patients based on TREM-1. Under the terms of the agreement, Inotrem has access to data and bio-samples from Foundation’s IBD Plexus®, which is the largest IBD database in the US with data from over 25,000 patients. Last over 18 months, the project will be a cornerstone in the design of Inotrem’s first-in-human clinical study with INO-02.

There are today 10 million people worldwide suffering from IBD, and the need for new therapeutic options remains very strong. Approximately 30% of patients are unresponsive to existing therapies and even among the initial responders, in up to 10% the drugs lose efficacy over time. For a decade, Inotrem has been elucidating the biology of TREM-1 as a regulator of the immune response in both acute as well as chronic inflammatory diseases. TREM-1 is a potential important contributor to IBD pathophysiology and targeting this pathway may offer a new treatment option for IBD patients with immune dysregulation.

“We are grateful for the support of such a well-respected organization as The Crohn’s & Colitis Foundation. This funding will help us to expand our patented technology platform centered around the TREM-1 pathway and dedicated to developing new therapies for chronic inflammatory syndromes next to our existing modalities for acute diseases,” indicates Sven Zimmermann, CEO of Inotrem.

“Beyond the financial support, this is a strong recognition of Inotrem’s scientific leadership regarding the role of the TREM-1 pathway in inflammatory processes as well as our robust therapeutic approaches targeting that biological target,” says Marc Derive, Chief Scientific Officer and Cofounder of Inotrem.

About Inotrem

Inotrem S.A. is an advanced clinical stage biotech company specialized in immunotherapies for acute and chronic inflammatory syndromes. The company has developed a new concept of immunomodulation that targets the TREM-1 pathway to control unbalanced inflammatory responses. Through its proprietary technology platform, Inotrem has developed the first-in-class TREM-1 inhibitor, nangibotide, with potential applications in a number of therapeutic indications such as septic shock and myocardial infarction. In parallel, Inotrem has also launched another program to develop a new therapeutic modality targeting chronic inflammatory diseases. The company was founded in 2013 by Dr Jean-Jacques Garaud, a former head of research and early development at the Roche Group, Prof. Sebastien Gibot and Dr Marc Derive. Inotrem is supported by leading European and North American investors. www.inotrem.com

About the Crohn’s & Colitis Foundation

The Crohn's & Colitis Foundation is the leading non-profit organization focused on both research and patient support for inflammatory bowel disease (IBD), with the mission of curing Crohn's disease and ulcerative colitis, and of improving the quality of life of the millions of Americans living with IBD. The Foundation’s work is dramatically accelerating the research process through investment in research initiatives, while also providing extensive educational and support resources for patients and their families, medical professionals, and the public. For more information, visit crohnscolitisfoundation.org, call 888-694-8872, or email info@crohnscolitisfoundation.org.

Calypso Biotech Announces First Patient with Eosinophilic Esophagitis Dosed in Anti-Interleukin-15 (IL-15) Monoclonal Antibody CALY-002 Phase 1a/b Trial, and Animal POC Data in Atopic Dermatitis

• Healthy volunteers phase completed showing differentiating target engagement biomarker data and favorable safety data

• On track for top-line histology data in 2023 in Celiac Disease and Eosinophilic Esophagitis

• Proof-of-concept data of CALY-002 in humanized Atopic Dermatitis model supports “pipeline-in-a drug” value proposition

Calypso Biotech, a leader in the development of Interleukin-15 (IL-15) targeted therapies, announces today dosing of the first Eosinophilic Esophagitis patient in the multiple dosing part of the Phase 1 clinical trial of CALY-002, a novel humanized - and highly differentiated - monoclonal antibody neutralizing IL-15. The ongoing Phase 1 clinical study of CALY-002 includes a single ascending dose (SAD) in Healthy Volunteers as well as multiple ascending dosing (MAD) in cohorts of patients with Celiac Disease or Eosinophilic Esophagitis, two indications with significant unmet medical need where IL-15 plays a critical role in their pathogenesis [NCT04593251].

The SAD part of the study has been completed. Across the wide range of tested dose levels in Healthy Volunteers, CALY-002 was well tolerated, with a PK profile typical for IgG monoclonal antibodies. Importantly, target engagement (i.e., IL-15 blockade) was demonstrated through the reduction of NK cell numbers in blood, owing to the well-known role of IL-15 as a homeostatic factor for NK cells. CALY-002 is the first IL-15-specific antibody to demonstrate blood NK cell reduction in humans, underpinning its unique and differentiated profile.

The MAD part of the study in patients with Celiac Disease or Eosinophilic Esophagitis is currently progressing into the highest dose cohorts. This phase investigates safety, PK, pharmacology, disease biomarkers and clinical efficacy endpoints including histology of multiple doses of CALY-002 or placebo over either an 8-week treatment period in a gluten-challenge setting (Celiac Disease) or a 12-week period of open label CALY-002 treatment (Eosinophilic Esophagitis). Top-line histology data will be generated in 2023 for both indications.

Further, CALY-002 was demonstrated to be active in a recently developed humanized model of Atopic Dermatitis, results presented at the 51st European Society for Dermatological Research conference (Amsterdam, 28 September – 01 October 2022). These important data further highlight the potential of CALY-002 in multiple auto-immune indications.

Calypso Biotech Chief Medical Officer, Dr. Jos Houbiers, MD, PhD, comments “the demonstrated pharmacological efficacy highlights the potency of CALY-002 and is promising for the clinical histology results in the MAD parts of the study. New and improved treatments are much needed for Celiac Disease and Eosinophilic Esophagitis patients who have limited treatment options. For patients with celiac disease, clinical practice learns that adherence to a gluten-free diet is very difficult and many patients continue to experience symptoms; in asymptomatic patients, histological damage to the small intestine can be present unnoticed, studies show.’’

About Calypso Biotech BV

Calypso Biotech BV is a private biotechnology company focused on the research and development of novel biologics to address unmet medical need in autoimmune and inflammatory diseases, with a unique expertise in IL-15 biology. IL- 15 controls immune pathways critically involved in disease onset and maintenance (‘’disease memory’’), as well as tissue destruction, in addition to its broad effect on inflammation. Calypso’s approach offers significant advantages over traditional cytokine interventional therapies and could provide for unprecedented long lasting disease-modifying effects in multiple autoimmune diseases. Calypso Biotech is a spin-off by the healthcare business of Merck KGaA and is headquartered in Amsterdam, The Netherlands, with offices and laboratories in Geneva, Switzerland.

Inotrem Announces That Its ESSENTIAL Phase II Study for the Treatment of Critically ill COVID-19 Patients Meets Its Primary and Key Secondary Endpoints

• Results demonstrate statistically significant efficacy of drug candidate nangibotide in the treatment of patients with severe forms of COVID-19

• Nangibotide treatment improves patients’ clinical status and shows a relative 43% reduction in mortality

• The company will consult with regulatory authorities to advance a new treatment option to severe COVID-19 patients

• ESSENTIAL was made possible thanks to the strong support from French public authorities and their commitment to fight COVID-19

Inotrem, an advanced clinical stage biotech company specializing in immunotherapies for acute and chronic inflammatory syndromes, announced today at the European Society of Intensive Care Medicine’s Annual Congress, held in Paris, positive results for ESSENTIAL, its Phase II clinical trial in COVID-19 patients hospitalized in critical care units and experiencing acute respiratory distress. The study was funded as part of the Capacity Building call for proposals, financed by the Programme d’Investments d’Avenir (PIA), operated on behalf of the French government by Bpifrance, the French national investment bank.

The ESSENTIAL study was terminated at 220 randomized ICU patients requiring ventilatory support (stage 5 or 6 on a 7-point clinical status ordinal scale) and compared infusion of nangibotide at 1mg/kg/hr for up to 5 days of treatment with standard of care. Despite a lower than anticipated sample size, the study met its primary endpoint of an improvement in clinical status according to 7-point clinical status ordinal scale from baseline to Day 28 (p value = 0.040).

Nangibotide treatment also showed a statistically and clinically meaningful 12% absolute and 43% relative reduction in Day 28 mortality (key secondary endpoint) in the overall trial population (p value = 0.030). Among the subpopulation of patients with levels of the TREM-1 pathway activation marker, soluble TREM-1 (sTREM-1) above the median, the absolute and relative reduction in mortality was even more pronounced, amounting to 20% and 47%, respectively (p value = 0.023).

The study showed that nangibotide has a significant and positive impact on the progression of the disease in patients receiving ventilatory support due to COVID-19, as well as on the severity of the respiratory failure, and length of stay in ICU. The trial showed that sTREM-1 is an effective prognostic marker of outcome in severe COVID-19 and, consistent with the results from the company’s prior ASTONISH study, confirmed that sTREM-1 is a predictive marker of a positive response to the treatment by nangibotide.

Jean-Jacques Garaud, Senior VP Head of scientific and medical affairs at Inotrem said: "This new trial brings compelling evidence that the TREM-1 pathway plays a central role in major life-threatening immune dysregulations caused by severe infections, whether it is severe forms of COVID-19 or septic shock. This study strongly suggests that nangibotide, which targets TREM-1, is pathogen agnostic and has the potential to treat those very severe inflammatory conditions caused by both viral and bacterial infections."

Sven Zimmermann, CEO of Inotrem, added: "We are grateful for the continued financial support and confidence awarded to us by the French public authorities to fight COVID-19. This attests of the relevance of our innovative approach to treat inflammatory diseases. The data we obtained is extremely encouraging, and we plan on quickly consulting regulatory authorities in the US and EU."

Thierry Hercend, Independent Board member at Inotrem, said: "The effect of nangibotide on severe forms of COVID 19 added to the recent announcement of the ASTONISH Phase II data in septic shock confirms that targeting the TREM-1 pathway is beneficial in other conditions, infectious or not, leading to severe immune dysregulation in the critical care setting."

Substantial support from public funding to fight COVID-19

At the start of the pandemic in 2020, Inotrem team set out to build on the similarities between the immune dysregulation in severe forms of COVID-19 with those observed in septic shock patients. Building on its deep scientific and medical understanding of the TREM-1 pathway, and with the strong support of the French government, Inotrem launched ESSENTIAL, its clinical trial to assess the efficacy of its drug lead candidate, nangibotide, for COVID-19 patients in the ICU.

In July 2020, the CoviTREM-1 consortium which includes the Nancy and Limoges university hospitals and Inotrem, obtained a first public funding of 7.5 million euros under a call for projects operated by the Secretary General for Investment and Bpifrance. In December 2020, the trial was declared a "Research National Priority" by the French government. In July 2021, Inotrem was authorized to pursue the clinical development of nangibotide up to registration for COVID-19 patients and can draw on additional public funding of up to 45 million euros from Bpifrance, as part of the Programme d’Investments d’Avenir ("PIA").

About the drug candidate nangibotide

Nangibotide is a TREM-1 inhibitor peptide with the potential to restore appropriate inflammatory response, vascular function, and improve post septic shock survival. Nangibotide is the formulation of the active ingredient LR12, which is a 12 amino-acid peptide prepared by chemical synthesis. LR12 is a specific TREM-1 inhibitor, acting as a decoy receptor and interfering in the binding of TREM-1 and its ligand. In preclinical septic shock models, nangibotide was able to restore appropriate inflammatory response, vascular function, and improved animals’ survival post septic shock. Nangibotide in septic shock has been granted the fast-track status in September 2019 by the FDA and the PRIME status in 2017 by the EMA and has recently reported positive results from a Phase IIb trial (ASTONISH) in septic shock patients.

About Inotrem

Inotrem S.A. is a biotechnology company specialized in immunotherapy for acute and chronic inflammatory syndromes. The company has developed a new concept of immunomodulation that targets the TREM-1 pathway to control unbalanced inflammatory responses. Through its proprietary technology platform, Inotrem has developed the first-in-class TREM-1 inhibitor, nangibotide, with potential applications in a number of therapeutic indications such as septic shock, severe forms of COVID-19 and myocardial infarction. In parallel, Inotrem has also launched an antibody-based program to develop a new therapeutic modality targeting chronic inflammatory diseases. The company was founded in 2013 by Dr Jean-Jacques Garaud, a former head of research and early development at the Roche Group, Prof. Sébastien Gibot and Dr Marc Derive. Inotrem is supported by leading European and North American investors Inotrem is part of the French Tech 120, a government program dedicated to support the development of fast-growing startups. www.inotrem.com

Corteria Pharmaceuticals strengthens its Executive Leadership Team, Board of Directors, and Scientific Advisory Board

Corteria Pharmaceuticals, a biotechnology company developing innovative treatments for certain forms of heart failure, announced today the recruitment of Dr. Francesca C. Lawson as Chief Medical Officer. Corteria Pharmaceuticals also expanded its Board of Directors with the addition of Dr. Marc Semigran as an independent Director, as well as its Scientific Advisory Board with the addition of Dr. Jeffrey Testani.

"We are highly pleased to welcome Drs. Lawson, Semigran and Testani to our team to accelerate the development of our programs. Their expertise and in-depth knowledge of these diseases will be invaluable to Corteria," commented Philip Janiak, founder and CEO of Corteria Pharmaceuticals.

Francesca C. Lawson, M.D., based in Philadelphia, USA, is a specialist in cardiometabolic diseases with more than 30 years of experience in drug development, from Phase 1 to Phase 3. Dr. Lawson joins Corteria Pharmaceuticals from Applied Therapeutics, where she spent three years overseeing the development of new drug candidates for the treatment of diabetic cardiomyopathy as Vice President, Clinical and Regulatory Strategy. Prior to Applied Therapeutics, she led the clinical trials of cardiometabolic drug candidates at Sanofi where she successfully developed sotaglifozin in worsening heart failure.

Marc Semigran, M.D., a cardiologist with subspecialty in heart failure based in Cambridge, USA, joins the Board of Directors of Corteria Pharmaceuticals as an independent Director. Dr. Semigran currently serves as Chief Medical Officer at Renovacor, and previously as Head of Clinical Development of MyoKardia, a biopharmaceutical company pioneering new approaches for the treatment of cardiomyopathies. There, he led the development of mavacamten in hypertrophic cardiomyopathy. Dr. Semigran is the author of numerous scientific peer-reviewed publications, for which he has received several prestigious awards. While Medical Director of the Heart Failure and Cardiac Transplant Program at Massachusetts General Hospital and Harvard Medical School, he served as principal investigator in major clinical trials.

Jeffrey Testani, M.D., a cardiologist, associate professor of Medicine and Director of Heart Failure Research at Yale University, USA, is an expert on heart failure and cardiac transplantation. Dr. Testani also runs a large research program focusing on cardio-renal syndrome at the Yale Heart and Vascular Center and has published several seminal papers in peer-reviewed journals.

About Corteria Pharmaceuticals:

Founded in 2021 and based in Paris, France, Corteria Pharmaceuticals is a biotechnology company specialized in the development of first-in-class drugs for the treatment of heart failure subpopulations. Despite advances in the management of this serious disease, the prevalence of heart failure continues to rise, with over 60 million cases worldwide. Corteria Pharmaceuticals' innovative approach is based on a selection of therapeutic targets involved in the worsening of heart failure and in the acute form of the disease in humans, as well as a stratification strategy to identify patients likely to respond best to its treatments.

More information is available on the company's website: www.corteriapharma.com

Inotrem announces the outcome of its Phase II ASTONISH trial in septic shock patients demonstrating efficacy of nangibotide

• ASTONISH confirms the therapeutic potential of nangibotide in septic shock patients with excessive activation of the TREM-1 pathway

• ASTONISH confirms that the soluble TREM-1 biomarker predicts response to nangibotide treatment.

Inotrem, an advanced clinical stage biotech company specializing in immunotherapies for acute and chronic inflammatory syndromes, disclosed for the first time today at the International Sepsis Forum held in Barcelona the results for its Phase IIb ASTONISH clinical trial in septic shock patients.

ASTONISH was designed to show the efficacy of nangibotide in septic shock with a precision medicine approach aiming to identify patients who benefit the most from this innovative treatment. This global study enrolled 361 patients across 41 clinical sites in 6 European countries and the United States and succeeded in demonstrating a therapeutic benefit of nangibotide in patients with high levels of the TREM-1 pathway activation marker, soluble TREM-1 (sTREM-1).

ASTONISH studied as a primary endpoint the improvement of a well-established morbidity score evaluating patient clinical evolution and organ function, the SOFA score. The change of this score at 5 days after initiation of treatment in the nangibotide-treated arms was compared to the placebo arm in all patients and in patients with a high level of sTREM-1. Importantly, and as part of the prespecified analysis for defining the optimal sTREM-1 cut-off, the benefit of nangibotide high dose treatment versus placebo was clinically and statistically significant at higher concentrations of sTREM-1, representing about 50% of the study population.

Consistent with prior preclinical, observational and Phase IIa trial data in this setting, ASTONISH confirmed that excessive TREM-1 activity is associated with severe immune dysregulation, organ dysfunction and ultimately death.

ASTONISH demonstrates that TREM-1 modulation with nangibotide improves respiratory, cardiovascular and renal function. The study also provided evidence that nangibotide meaningfully impacts other relevant clinical parameters, displaying a trend towards improvement in All-Cause Mortality at day 28 and the proportion of patients Alive and free of organ support at day 28.

Jean-Jacques Garaud, Senior VP Head of scientific and medical affairs at Inotrem, said: “The ASTONISH trial was designed as a Phase III enabling trial and it did generate important insights about nangibotide’s therapeutic activity and our precision medicine approach in septic shock. We are enthusiastic about this study and Inotrem’s capacity to bring a first in class product in an area with a major unmet medical need”.

“These results represent a major advancement for patients suffering from septic shock. They provide compelling evidence that Inotrem’s innovative solution targeting the TREM-1 pathway has the potential to become the first causal treatment for this severe and often fatal indication,” said Sven Zimmermann, CEO of Inotrem. “Based on these data, we intend to advance nangibotide towards registration studies in septic shock. We look forward to discussing next steps with regulatory authorities.”

Professor Bruno François, Limoges University Hospital and coordinating investigator added: “We observed that the TREM-1 pathway was activated in severe infections leading to septic shock. Nangibotide is the first TREM-1 inhibitor and ASTONISH confirms its potential as a new therapeutic option for the septic shock patient population. We are looking forward to bringing definitive evidence, in a Phase III clinical study that nangibotide can reduce mortality in these critically ill patients”.

Septic shock is the ultimate complication of sepsis and currently constitutes a high unmet medical need. The incidence of septic shock continuously raises, and mortality remains elevated: it is the 10th leading cause of death in developed countries and the 1st cause of death in intensive care units. There is currently no specific therapy approved for this indication besides antibiotics and symptomatic treatment. Inotrem’s solution has the potential to become the first mechanism-based treatment for septic shock.

About nangibotide

Nangibotide is a TREM-1 inhibitor peptide with the potential to restore appropriate inflammatory response, vascular function, and improve post septic shock survival. Nangibotide is the formulation of the active ingredient LR12, which is a 12 amino-acid peptide prepared by chemical synthesis. LR12 is a specific TREM-1 inhibitor, acting as a decoy receptor and interfering in the binding of TREM-1 and its ligand. In preclinical septic shock models, nangibotide was able to restore appropriate inflammatory response, vascular function, and improved animals’ survival post septic shock. Nangibotide in septic shock has been granted the fast-track status in September 2019 by the FDA and the PRIME status in 2017 by the EMA. Nangibotide has also been tested in a Phase II trial in severe forms of COVID-19 (ESSENTIAL). ESSENTIAL data will be communicated on October 25. 2022 at the next ESICM meeting in Paris.

About ASTONISH Study

The Efficacy, Safety and Tolerability of nangibotide in Patients with Septic Shock (ASTONISH) phase IIb trial is a Randomized, Double-blind, Placebo Controlled Dose Selection Study that was performed in Europe and in the US. The study compared the effect of nangibotide at two different doses (0.3 and 1mg/kg/h continuous i.v. infusion for 3 to 5 days) versus standard of care.

About Inotrem

Inotrem S.A. is a biotechnology company specialized in immunotherapy for acute and chronic inflammatory syndromes. The company has developed a new concept of immunomodulation that targets the TREM-1 pathway to control unbalanced inflammatory responses. Through its proprietary technology platform, Inotrem has developed the first-in-class TREM-1 inhibitor, nangibotide, with potential applications in a number of therapeutic indications such as septic shock, severe forms of COVID-19 and myocardial infarction. In parallel, Inotrem has also launched an antibody-based program to develop a new therapeutic modality targeting chronic inflammatory diseases. The company was founded in 2013 by Dr Jean-Jacques Garaud, a former head of research and early development at the Roche Group, Prof. Sébastien Gibot and Dr Marc Derive. Inotrem is supported by leading European and North American investors Inotrem is part of the French Tech 120, a government program dedicated to support the development of fast-growing startups. www.inotrem.com

Mainstay Medical Announces Publication of Three-Year Patient Outcomes Data from ReActiv8-B Clinical Trial Demonstrating Long-Term Efficacy of ReActiv8® Restorative Neurostimulation™

Data continue to show compelling efficacy and safety, including improvement on all key measures of pain and disability, as compared to the one-year and two-year study results

Mainstay Medical Holdings plc today announced the publication of the three-year patient outcomes data from its pivotal ReActiv8-B clinical trial. The data, published in the journal of the International Neuromodulation Society, Neuromodulation, further establish the efficacy and safety of ReActiv8 Restorative Neurostimulation, including compelling long-term durability and improvement over time on key outcome measures in the treatment of intractable chronic low back pain.

The three-year data show improvements over results from the patients’ one-year and two-year visits on virtually all key efficacy measures. Of note:

*Percent of patients that were on opioids at baseline: (1-year=31/65), (2-Year= 34/57), (3-Year= 36/51).

Overall, 83% of patients experienced substantial and clinically meaningful improvements in pain or disability, or both, at three years.

Chris Gilligan, Director of the Brigham and Women’s Spine Center at Brigham and Women’s Hospital, assistant professor of Anesthesia at Harvard Medical School (Boston, USA), and Principal Investigator of the study, said: “The recently published data from the ReActiv8-B clinical trial continued to show clinically meaningful improvements in both pain and function for patients with refractory chronic low back pain who received three years of neurostimulation. The long-term trajectory and durability of clinical benefits are consistent with the restoration of neuromuscular control and muscle rehabilitation, which gives us confidence that we are able to treat the underlying cause of chronic low back pain in these patients.”

“Multifidus dysfunction in patients with chronic low back pain has historically been a challenging etiology for the spine surgeon community to properly treat,” said Frank Schwab, Chair of Orthopedic Spine Surgery at Lenox Hill Hospital, and Chief of Orthopedic Spine Surgery for Northwell Health System. “These patients are not indicated for surgery, with existing treatment options being temporary and palliative. ReActiv8 therapy has proven to maintain effectiveness long-term and provides this challenging patient population with a safe and restorative solution.”

Jason Hannon, CEO of Mainstay Medical, said: "These 3-year results further validate ReActiv8’s restorative mechanism of action, which treats a primary underlying cause of mechanical chronic lower back pain, multifidus dysfunction. We are proud to have the only commercially available device with a strong safety profile and long-term, peer-reviewed evidence supporting the rehabilitation of this severely affected patient population, and we look forward to continuing to generate clinical and other research to compel physicians and their patients to further utilize the therapy.”

The full publication can be downloaded free of charge at Three-Year Durability of Restorative Neurostimulation Effectiveness in Patients With Chronic Low Back Pain and Multifidus Muscle Dysfunction - ScienceDirect

About ReActiv8®

ReActiv8 is an implantable medical device designed to treat adults with intractable chronic low back pain (CLBP) associated with multifidus muscle dysfunction. Multifidus muscle dysfunction may be evidenced by imaging or physiological testing in adults who have failed therapy including pain medications and physical therapy, and who are not candidates for spine surgery. ReActiv8 has received regulatory approval in several geographic areas, and is commercially available in the European Economic Area, Australia, the UK, and the US.

About Mainstay Medical

Mainstay Medical is a medical device company focused on commercializing its innovative implantable Restorative Neurostimulation™ system, ReActiv8®, for people with disabling mechanical CLBP. Mainstay Medical is headquartered in Dublin, Ireland, and has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands.

Further information can be found at www.mainstaymedical.com.

United Kingdom National Institute for Health and Care Excellence (NICE) Publishes Interventional Procedure Guidance (IPG) for ReActiv8® Restorative Neurostimulation™

ReActiv8 Neurostimulation therapy recommended to be used in the National Health Service; Special arrangements designation to increase access to ReActiv8 for patients

Mainstay Medical Holdings plc announced today that the National Institute for Health and Care Excellence (NICE), a public body of the Department of Health that carries out evidence-based health technology assessments by independent committees, has issued a recommendation that ReActiv8® Restorative Neurostimulation™ can be used in the National Health Service in the U.K., with special arrangements for clinical governance, consent, and audit or research. ReActiv8 is currently the only technology offering restorative neurostimulation to the NHS for refractory mechanical chronic low back pain (CLBP).

“U.K. physicians have some of the most extensive experience with ReActiv8 globally. We are thankful to NICE for recognizing this experience, as well as the substantial body of global evidence we have developed in support of the efficacy and safety of this ground-breaking therapy, “ said Jason Hannon, CEO of Mainstay Medical. “This new guidance will enable patients to have greater access to the therapy when previous treatment options have not proven successful. We look forward to supporting physicians across the U.K. going forward and to continued improvement in patient outcomes.”

“Historically, mechanical CLBP pain therapies were aimed at only managing the symptoms, which is the pain associated with this pathology,” said Dr. Ganesan Baranidharan, Consultant in Anaesthesia and Pain Medicine, Leeds Teaching Hospitals NHS Trust. “ReActiv8 is the only therapy available I am aware of where patients with many years of CLBP actually reverse and effectively rehabilitate their condition durably over a long period of time. ReActiv8 targets the cause of mechanical CLBP, not just the symptoms. The new NICE guidance is a major victory for patients and clinicians, which further confirms the positive patient outcomes we have seen to date. This new guidance will allow more patients to gain access to ReActiv8 within the U.K. and further afield.”

The NICE publication can be found here: Evidence | Neurostimulation of lumbar muscles for refractory non-specific chronic low back pain | Guidance | NICE

About ReActiv8®

ReActiv8 is an implantable medical device designed to treat adults with intractable chronic low back pain (CLBP) associated with multifidus muscle dysfunction. Multifidus muscle dysfunction may be evidenced by imaging or physiological testing in adults who have failed therapy including pain medications and physical therapy, and who are not candidates for spine surgery. ReActiv8 has received regulatory approval in several geographic areas, and is commercially available in the European Economic Area, Australia, the UK, and the US.

About Mainstay Medical

Mainstay Medical is a medical device company focused on commercializing its innovative implantable Restorative Neurostimulation™ system, ReActiv8®, for people with disabling mechanical CLBP. Mainstay Medical is headquartered in Dublin, Ireland and has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands.

Further information can be found at www.mainstaymedical.com.

Medical Microinstruments Secures $75M to Advance Robotic Microsurgery

Series B financing will accelerate clinical trials and global commercialization of the company’s Symani® Surgical System

Medical Microinstruments, Inc. (MMI), a robotics company dedicated to improving clinical outcomes for patients undergoing microsurgery, today announced it has raised $75 million in Series B financing. Deerfield Management led the round with participation from new investors, RA Capital Management and Biostar Capital, as well as existing investors, Andera Partners, Fountain Healthcare Partners, Panakès Partners and Sambatech. The company also announced the addition of three new members to its board of directors.

In addition, the company announced its corporate redomicile from Italy to the United States. The recently opened Center of Excellence facility in Pisa, Italy, with nearly 100 employees will continue to be the hub of the company’s research and development, manufacturing, and other business activities.

Proceeds from this financing round and the company’s planned U.S. presence will launch MMI into its next stage of growth as it continues its mission to improve the quality of patient care by pushing the boundaries of microsurgery. The company seeks to expand indications and support ongoing commercialization efforts for the Symani® Surgical System in Europe where it received CE mark in 2019. MMI also intends to accelerate plans to commercialize in the U.S. and Asia-Pacific, as well as advance clinical research including securing an Investigational Device Exemption (IDE) from the U.S. Food and Drug Administration to conduct a pivotal study. The Symani System was developed specifically to address the challenges of microsurgery and is the only system that offers NanoWrist® Instruments designed to improve a surgeon’s ability to access and suture small, delicate anatomy.

“This financing round, coupled with our commitment to access the U.S. market and the addition of visionary leaders to our board, is an exciting moment for the surgical robotics space,” said Mark Toland, CEO of MMI. “We’re pleased to have bridged the Atlantic with premier U.S. life science investors, and existing European investors, who share our same vision of bringing microsurgical robotics to the world.”

The company’s new board members are Andrew ElBardissi, MD, Tess Cameron and Arturo Baroncelli. Dr. ElBardissi is a partner at Deerfield Management, with extensive experience serving as a board member for innovative healthcare companies. Ms. Cameron serves as principal for RA Capital Management and currently sits on the boards of Avilar Therapeutics and Nodexus Inc. Baroncelli previously worked as a robotics business development manager for Comau and will represent MMI’s founders on the board.

“We are thrilled to add further depth and experience to the MMI Board and look forward to working with our new board members to build the robotic microsurgical space,” said Andrew Cleeland, Chairman of the Board for MMI.

“The MMI technology is one of the most significant transformational advancements in surgical robotics that we have seen,” said Dr. ElBardissi. “Having the world’s smallest wristed instruments opens up the field of ‘micro’ robotics to a completely new level of treatment spanning microsurgery for cancer, trauma, orthopedic, pediatric, and one day, neurosurgery patients. Symani will be the future of how microsurgery is performed worldwide.”

“Robotic microsurgery has enormous potential to both improve the standard of care for patients and help surgeons manage procedures that require delicate precision,” said Ms. Cameron. “I look forward to supporting MMI and its world-class team as it begins this exciting chapter.”

The Symani Surgical System is designed to improve a surgeon’s ability to repair anatomical structures such as veins, arteries, nerves and lymphatic vessels as small as 0.3 mm in diameter. The platform provides motion scaling and tremor reduction to allow precise micromovements. Its NanoWrist technology is the world’s smallest wristed instrumentation and is intended to improve a surgeon’s natural dexterity and range of motion beyond the capability of the human hand.

About Medical Microinstruments, Inc.

Medical Microinstruments, Inc. (MMI) was founded in 2015 near Pisa, Italy to enhance surgical performance through the development of a robotic system that enables surgeons to achieve better outcomes in microsurgery. The Symani Surgical System combines proprietary innovations including the world’s smallest wristed microinstruments as well as tremor-reducing and motion-scaling technologies. Together, these powerful capabilities allow more surgeons to successfully perform microsurgery while expanding the field of supermicrosurgery. MMI is backed by international medtech investors including Andera Partners, BioStar, Deerfield Management, Fountain Healthcare Partners, Panakès Partners, RA Capital and Sambatech.

Media Contact:

Sarah Lundberg
Health+Commerce
sarahlundberg@healthandcommerce.com

Ermium Therapeutics extends its initial €6.3 M funding to €12.3 M to further advance breakthrough auto-immune disease therapeutics

Ermium Therapeutics, a Paris-based biotech company developing innovative health products for auto-immune diseases, announced today the extension of its initial funding of €6.3 M to €12.3 M with Kurma Partners and Fountain Healthcare Partners.

The company is discovering and developing small molecules, orally available, that are aimed at addressing a range of chronic auto-immune diseases, affecting millions of people worldwide, and particularly type I interferon-mediated diseases such as systemic lupus erythematosus (SLE), dermatomyositis, Sjögren syndrome, and monogenic interferonopathies. Ermium proprietary compounds are intended to be first-in-class acting on a well-known G protein-coupled receptor (GPCR), namely the CXCR4 chemokine receptor, via a totally novel mechanism of action. The main property of Ermium CXCR4-targeted functionally selective immunomodulators is to potently abolish the release of type I interferons by immune cells, and also other key inflammatory cytokines.

The €6 M proceeds will be used to reach the milestones of drug candidate selection, extend the non- clinical development package, and drive continued efforts on the pharmacological package in support of the early clinical development program in SLE and other auto-immune diseases.

Fountain Healthcare Partners, a transatlantic life science investor, is joining and widening the Ermium Therapeutics initial investment syndicate composed of Kurma Partners, Eurazeo, Domain Therapeutics, and Erganeo.

“I am thrilled to close this extension funding which reflects the confidence of our historical investors along with the commitment of Fountain Healthcare Partners which we are welcoming with enthusiasm. Altogether, this further validates all the efforts the company team has made since the company inception, and also the promising therapeutic potential of the first in class immunomodulators we are developing; Moreover, it will allow our program to move on up to the selection of a drug candidate, with the goal to address a huge medical need for patients suffering from interferonopathies”, said Joël CROUZET, CEO of Ermium Therapeutics.

“We were very impressed by the work accomplished by the team at Ermium Therapeutics. We were quickly convinced of the significant commercial opportunity that could be realized with this investment and the potential application of Ermium’s lead development candidate across a variety of auto-immune disease indications. We are particularly delighted to join Kurma, Eurazeo, Domain Therapeutics, and Erganeo in advancing Ermium Therapeutics first in class compounds to address the high unmet medical need in interferonopathies such as SLE”, added Aidan KING, Managing Partner of Fountain Healthcare Partners and newly appointed member of Ermium Therapeutics board of directors.

“We are particularly proud and impressed by the advances performed by Ermium Therapeutics since the initial funding, based on breakthrough science and a highly innovative approach for critical medical needs such as SLE. This extension funding will accelerate the company development and allow its exciting program to move on towards the first steps of non-clinical development”, concluded Thierry LAUGEL, Managing Partner of Kurma Partners and member of Ermium Therapeutics board of directors.

About Ermium Therapeutics

Ermium Therapeutics, a discovery stage company, was founded in June 2019 by Dr Jean-Philippe Herbeuval (CNRS – Université Paris Cité), Kurma Partners, Domain Therapeutics, and Erganeo. Ermium Therapeutics breakthrough first in class compounds are CXCR4- targeted functionally selective immunomodulators; those orally available compounds are aimed to be developed for auto-immune disease indications, particularly interferonopathies, including SLE. Such immunomodulators exhibit potent anti-inflammatory properties by efficiently downmodulating the release of type I interferons, and also other key proinflammatory cytokines. The company has obtained in vivo POC in different auto-immune / inflammatory disease models. Ermium Therapeutics is based at the Paris Biotech Santé Incubator – Accelerator located at the Cochin Hospital in Paris. The company has signed a worldwide exclusive license agreement on intellectual property from the CNRS and the Université Paris Cité through an agreement with Erganeo. Ermium Therapeutics has received grants and soft loans from Bpifrance, the French agency for innovation. www.ermium.com

About Fountain Healthcare Partners

Fountain Healthcare Partners is a life science focused venture capital fund with over EUR 300 million under management. Within the life science sector, specific areas of interest to Fountain Healthcare Partners include biotechnology, medical devices, specialty pharma and diagnostics. Fountain invests in entrepreneurs and companies with disruptive technologies or products that have a clear pharmacoeconomic benefit and a defined pathway to commercialisation, value enhancement and exit. Fountain’s main office is in Dublin, Ireland, with a second office in New York. The firm deploys the majority of its capital in Europe, with the balance in the United States. www.fh-partners.com

About Kurma Partners

Kurma Partners is a key European Venture Capital firm specialized in healthcare, with more than €700 million under management, with two dedicated franchises: “Kurma Biofund” focused on venture investments in therapeutics (current active fund KBIII) and “Kurma Diagnostics” focused on venture investment in diagnostics and digital health (current active fund Kurma Dx2). Kurma Partners has launched its first “Growth Opportunity Fund” further to its first closing in early 2022. Kurma Partners is part of the Eurazeo Group. www.kurmapartners.com

Contacts

Ermium Therapeutics
Joël Crouzet, PhD, CEO
contact@ermium.com

Ulysse Communications – Press Relations
Bruno Arabian
barabian@ulysse-communication.com
Tel: +33 (0) 6 87 88 47 26

XyloCor Therapeutics Achieves Target Enrollment in Phase 2 EXACT Study of XC001 Novel Gene Therapy for Ischemic Heart Disease

• Positive Phase 1 results reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) revealed XC001 is well tolerated at all dose levels

• Phase I data support XC001 therapeutic effect and potential dose response

• Topline Phase 2 data readout expected in February 2023 with interim results in the second half of this year

XyloCor Therapeutics, a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced it has achieved enrollment of target number of subjects in the Phase 2 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina. Topline results from the Phase 2 study are expected in February 2023 with interim results in the second half of this year.

“Achievement of this important milestone in the Phase 2 portion of the study is a testament to the clinical need in this patient population and I am eager to see the Phase 2 results as they emerge,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “Patients with refractory angina have no treatment options, and the results from the Phase 1 portion of the EXACT trial suggest a dose response and therapeutic potential which is encouraging for the development of XC001 as a treatment to improve these patients’ quality of life. We are very excited to see this more definitive evaluation of the safety and efficacy of this approach.”

“We are pleased to announce this important milestone in enrollment for our Phase 2 study especially during this unprecedented and challenging time,” said Al Gianchetti, President and CEO of XyloCor. "An estimated one million people suffer from refractory angina in the United States, and we are encouraged that XC001 may address the high unmet need in this patient group. XyloCor also plans to study XC001 in other patient groups as well, including as adjunctive therapy in patients undergoing bypass surgery."

Individuals with refractory angina experience pressure or intense pain in the chest due to insufficient blood flow to the heart muscle. These symptoms can severely impact quality of life and may worsen comorbidities.

XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec) is a locally administered, single-dose gene therapy currently in development as a novel approach to treating patients with refractory angina who have no other medical and surgical options. The treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects. XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial with target enrollment of 27 additional subjects at the highest tolerated dose (1 x 1011 vps, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by an experienced cardiac surgeon at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics is a private, clinical-stage biopharmaceutical company developing potential best-in-class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Contacts

Corporate and Investor Relations:
A. Brian Davis, XyloCor Therapeutics
brian.davis@xylocor.com

610-541-2056

Media Contact:
Mike Beyer, Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

Clinical Mocravimod Data in Hematological Malignancies Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (aHSCT) Presented at the 2022 EHA Congress

Phase 1b/2a data shows that mocravimod is safe and well tolerated

Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, presented first clinical data on mocravimod (also known as KRP203) in hematological malignancies patients undergoing allogeneic hematopoietic stem cell transplant (HSCT) at the European Hematology Association (EHA) 2022 Congress, which was held in Vienna, Austria, June 9-12th, 2022.

The oral presentation, which took place on 12th June, was entitled A two-part, single- and two-arm randomized, open-label study to evaluate the safety, tolerability and pharmacokinetics of KRP203 in subjects with hematological malignancies undergoing allogeneic hematopoietic stem cell transplantation. The study showed that in the Phase 1b/2a study mocravimod was safe and well tolerated with promising overall survival.

Key take aways from the data presentation were:

• The study provided first ever human data of a S1P receptor modulator administered to patients undergoing allogeneic HSCT

• S1PR modulator class effects, such as bradycardia, were of no clinical concern

• Promising overall survival (OS) data

• Limited number of relapses, acute and chronic graft-versus-host disease (GVHD)

• Data supportive for conducting the planned MO-TRANS pivotal study to investigate efficacy (Relapse-free survival and OS) and GVHD in AML patients undergoing allogeneic HSCT

Priothera is initiating the pivotal MO-TRANS global Phase 2b/3 study in Europe, US and Japan, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult AML patients undergoing allogeneic HSCT. The study is expected to start in the second half of 2022 and preliminary data from this study are expected by the end of 2024.

Florent Gros, Co-Founder and CEO of Priothera, said: “We are delighted to have been given the opportunity to present the full first human clinical data on mocravimod at the European Hematology Association Congress this year. Based on these results mocravimod has the potential to be a first-in-class therapy in maintaining graft-versus-leukemia responses while preventing graft-versus-host disease. This novel approach would provide a tremendous benefit to AML patients undergoing an allogeneic hematopoietic stem cell transplant. We are excited about mocravimod which leverages a well-described mode of action in a hematology/oncology setting and has been successfully used in autoimmune indications. Pre-clinical and clinical proof of concept studies have herewith demonstrated mocravimod’s ability to improve survival outcomes for this devastating disease. We look forward to advancing the global pivotal Phase 2/3 trial which is due to start in the second half of 2022.”

Priothera’s abstract is available on EHA2022 Congress Abstract Book, here: https://journals.lww.com/hemasphere/Documents/EHA2022%20Congress%20Abstract%20Book.pdf

About Mocravimod

Mocravimod (also known as KRP203) is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers and the improvement of CART cell therapy.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogeneic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies and for the improvement of CART cell therapies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Not being an immunosuppressant, mocravimod maintains the graft-versus-leukemia (GVL) benefits in patients receiving HSCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden) and EarlyBird Venture Capital (Berlin, Germany).

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Priothera Receives Fast Track Designation for mocravimod in Combination with Allogeneic Hematopoietic Stem Cell Transplant (HSCT) for Post Remission Therapy of Acute Myeloid Leukemia (AML) Patients

Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation (FTD) for mocravimod in combination with allogeneic Hematopoietic Stem Cell Transplant (HSCT) for post remission therapy of Acute Myeloid Leukemia (AML) patients. FDA’s Fast Track designation is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening diseases and that demonstrate the potential to address unmet medical needs.

Priothera is working to initiate the MO-TRANS global Phase 2b/3 study in Europe, US and Japan, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult AML patients undergoing allogeneic HSCT. The MO-TRANS study is expected to start in the second half of 2022 and preliminary data from this study are expected by the end of 2024.

Karen Von Graevenitz, Head of Regulatory Affairs at Priothera, commented: “The Fast Track designation grant for mocravimod in combination with allogeneic HSCT is an important milestone and underlines the significant unmet need in AML patients undergoing HSCT, a serious disease where currently no available therapy exists. The designation means mocravimod will be eligible for expedited review and we will work closely with the US FDA to advance the global Phase 2/3 trial which is due to start in the second half of 2022.”

Florent Gros, Co-Founder and CEO of Priothera, added: “Following being granted orphan drug designations for mocravimod in the US and Europe, we are pleased to have been granted Fast Track designation for this highly promising compound. This important regulatory milestone moves us a step closer to bringing mocravimod to patients with AML and other hematologic malignancies.”

About mocravimod

Mocravimod (also known as KRP203), is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator’s unique mode of action to maintain anti-leukemia activity - graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogeneic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Not being an immunosuppressant, mocravimod maintains the graft-versus-leukemia (GVL) benefits in patients receiving HSCT while inhibiting graft-versus-host-disease (GVHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden) and EarlyBird Venture Capital (Berlin, Germany).

For more information please visit: www.priothera.com or follow Priothera on LinkedIn www.linkedin.com/company/priothera/

Contacts

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900

Inotrem announce the appointment of Luke Beshar as Chairman of Board of Directors

Inotrem, an advanced clinical stage biotech company developing TREM- 1 targeting immunotherapies for acute and chronic inflammatory syndromes, announced today the appointment of Luke Beshar as Chairman of its Board of Directors, effective May 25, 2022. Mr. Beshar succeeds Thierry Hercend who served as Chairman since 2014 and remains a member of the Board of Directors.

“We are pleased to welcome Luke Beshar to the Inotrem Board. Luke's deep experience in strategic development of biotech companies, in both executive and Board level positions of several public and private life science companies will be invaluable to Inotrem as we grow our business and pursue our mission to bring new treatments to patients suffering from inflammatory conditions” said Sven Zimmermann, Chief Executive Officer of Inotrem. “On behalf of the entire Inotrem team, I would like to thank Thierry Hercend for the exceptional work he has accomplished over the years as our Chairman and look very much forward to continue working with him as an Independent Non-executive Board member.”

“I feel privileged to take on this role at such an exciting time for Inotrem,” said Luke Beshar. “I am impressed by the quality of the pioneering work the team has accomplished on the biology of TREM-1 and its clinical translation in severe conditions such as septic shock and severe COVID-19. I’m also really excited about the anti-TREM1 pre-clinical monoclonal antibody programs in chronic inflammation and immuno-oncology. I look forward to working with Sven and the team on Inotrem’s accelerating growth and next stage of development.”

Luke Beshar is a seasoned biotechnology executive and financial expert with more than 35 years of general and financial management experience at various public and private companies. Mr. Beshar currently serves as Chairman of the Board of Protara Therapeutics, Inc., a publicly held company focused on immuno-oncology. Mr. Beshar also serves as director and chair of the audit committee of Omega Therapeutics and previously served as a director at Trillium Therapeutics Inc., a publicly held immuno-oncology company, from 2014 until it was acquired by Pfizer in November 2021 and was chair of its audit and compensation committees. Mr. Beshar previously served as a director and chair of audit committee of REGENXBIO Inc. from 2015 (pre-IPO) to 2021. Prior to this, Mr. Beshar served as Executive Vice President and Chief Financial Officer of NPS Pharmaceuticals, Inc. from November 2007 until it was sold to Takeda (Shire plc) in February 2015 and Executive Vice President and Chief Financial Officer of Cambrex Corporation, from 2002 to 2007. Mr. Beshar holds a B.S. degree in Accounting and Finance from Michigan State University and is a Certified Public Accountant.

About Inotrem

Inotrem S.A. is a biotechnology company specialized in immunotherapy for acute and chronic inflammatory syndromes. The company has developed a new concept of immunomodulation that targets the TREM-1 pathway to control unbalanced inflammatory responses. Through its proprietary technology platform, Inotrem has developed the first-in-class TREM-1 inhibitor, LR12 (nangibotide), with potential applications in a number of therapeutic indications such as septic shock and myocardial infarction. In parallel, Inotrem has also launched another program to develop a new therapeutic modality targeting chronic inflammatory diseases. The company was founded in 2013 by Dr Jean-Jacques Garaud, a former head of research and early development at the Roche Group, Prof. Sébastien Gibot and Dr Marc Derive. Inotrem is supported by leading European and North American investors. www.inotrem.com

Medical Microinstruments Launches New Simulator for Robotic Microsurgery

Symani Simulator to accelerate the expansion and adoption of robotic microsurgery

Medical Microinstruments (MMI) SpA, a robotics company dedicated to improving clinical outcomes for patients undergoing microsurgery, today announced the launch of its Symani Surgical System Simulator developed by VirtaMed. The simulator will improve, expand, and digitize the pathways for Symani training as surgeons prepare to expand their microsurgical skills through robotics.

“It was a priority for our company to offer our surgeons a simulation solution so they can practice robotic microsurgery in a more convenient way,” said Mark Toland, CEO of MMI SpA. “By training on the simulator, surgeons will be even more prepared for their first Symani patient or for a challenging case.”

“We were looking for a way to provide surgeons with a Symani experience without needing to transport the system,” said Jamie Milas, VP of Marketing at MMI SpA. “We selected VirtaMed as our trusted partner because of their shared passion for improving patient care and because we knew that they could develop a fully customized simulator that would emulate our device and showcase the advantages of Symani microsurgery.”

VirtaMed was incorporated in 2007 and is a world leader in surgical training solutions and data-driven medical education. VirtaMed develops world-class and educationally relevant virtual reality simulators for various medical disciplines such as orthopedics, obstetrics, gynecology, urology and laparoscopy. The VirtaMed LaparoSTM offers an experience that looks and feels real to the users, including correct physical behavior of internal organs and true to life interactions of surgical tools with those organs.

“We’re thrilled to partner with MMI to address this new frontier of robotic surgery,” said Stefan Tuchschmid, Co- CEO at VirtaMed. “MMI’s technology provides tremendous value for the surgical community and the patients they serve. We’re proud that our collaboration has led to a simulator that’s so realistic that surgeons can immediately begin suturing on Symani after just a few minutes of practice on the simulator.”

Incorporating simulation will also accelerate MMI’s product development process by enabling new solutions to be tested in a virtual environment for efficacy and usability.

MMI’s Symani Surgical System is the only robot dedicated to microsurgery that offers wristed instruments designed to improve a surgeon’s ability to access and suture small, delicate anatomy. Its platform provides motion scaling and tremor reduction to allow surgeons to make precise micro-movements. With Symani, surgeons can perform suturing, ligation, anastomoses and coaptations.

MMI will exhibit at the European Association of Plastic Surgeons, May 26-28 in Naples, Italy and at the World Society for Reconstructive Microsurgery Congress, June 1-4, in Cancun, Mexico. Demonstrations of the Symani Simulator and Symani Surgical System will be available at the MMI booth.

About MMI SpA

Medical Microinstruments S.p.A. (MMI) was founded in 2015 near Pisa, Italy to enhance surgical performance through the development of a robotic system that enables surgeons to achieve better outcomes in microsurgery. The Symani Surgical System combines proprietary innovations including the world’s smallest wristed microinstruments as well as tremor-reducing and motion-scaling technologies. Together, these powerful capabilities allow more surgeons to successfully perform microsurgery while expanding the field of supermicrosurgery. MMI is backed by international medtech investors including Andera Partners, Panakes Partners, Fountain Healthcare Partners and Sambatech.

About VirtaMed

VirtaMed believes medical education is powerfully delivered through data-driven simulation solutions. Since 2007, we have developed the leading solutions for training outside the operating room because we believe healthcare professionals should never have to perform a procedure for the first time on a patient. VirtaMed’s simulators provide the most realistic and cost-effective training available for laparoscopic surgeons.

Media Contact:

Sarah Lundberg
Health+Commerce
sarahlundberg@healthandcommerce.com

Media Contact VirtaMed:

Alex Gunderson
Padilla
Alex.Gunderson@padillaco.com

Medical Microinstruments Announces Major Expansion in Pisa, Italy to Support Growth

New facility will expedite the development and production of MMI’s Symani® robotic microsurgical system

Medical Microinstruments (MMI) SpA, a robotics company dedicated to improving clinical outcomes for patients undergoing microsurgery, today announced the grand opening of its new facility in Pisa, Italy. MMI consolidated locations throughout the Pisa area into a 3,000m2 singular site designed to accommodate company growth. The state-of-the-art facility will serve as the global ‘Center of Excellence’ for microsurgical surgical robotics, encompassing development, manufacturing, and administration.

“Our new facility in Pisa marks the beginning of an exciting new era for MMI,” said Mark Toland, CEO of MMI. “This new space reflects our focus on innovation, collaboration and growth. The expanded manufacturing center will ensure that we are prepared to address new markets and reach more patients with our Symani System and NanoWrist® Instruments.”

The building was fully renovated over 12 months and will serve as the primary office for employees, including a large team of R&D professionals with access to five new advanced R&D labs.

“Working all together in the new facility improves communication and disruptive innovation which is ideal for our research, engineering and clinical efforts,” said Massimiliano Simi, VP of R&D at MMI. “With our new laboratories, we are poised to continue the rapid development and high-quality product design our surgeon’s have come to expect.”

The space was designed to promote teamwork and accommodate the company’s fast growth with 40 additional hires expected by the end of 2022. The 900m2 state-of-the-art manufacturing center includes a clean room and with the capacity to meet the global demand for at least the next 5 years.

“Our cutting-edge facility touts the most advanced manufacturing technologies and production processes, many of which we have patented,” commented Giancarlo Testaverde, VP of Operations at MMI. “We are equipped and ready to meet the increased demands as we expand our installed base and address new markets.”

The Symani System is the only robotic surgical system that offers wristed microinstruments designed to improve a surgeon’s ability to access and suture small, delicate anatomy. The platform provides motion scaling and tremor reduction to allow surgeons to perform precise micro-movements. With Symani, surgeons can perform suturing, anastomoses and coaptations which are required during many complex microsurgical procedures.

"I'm pleased to welcome MMI, an innovative company that fully embodies the entrepreneurial spirit of Tuscany, to its new office in Pisa," said Eugenio Giani, President of Tuscany. "The life sciences industry is growing in our region, and we're eager to support the future growth of the company, especially through our ‘Invest In Tuscany’ office."

About MMI SpA

Medical Microinstruments S.p.A. (MMI) was founded in 2015 near Pisa, Italy to enhance surgical performance through the development of a robotic system that enables surgeons to achieve better outcomes in microsurgery. The Symani Surgical System combines proprietary innovations including the world’s smallest wristed microinstruments as well as tremor-reducing and motion-scaling technologies. Together, these powerful capabilities allow more surgeons to successfully perform microsurgery while expanding the field of supermicrosurgery. MMI is backed by international medtech investors including Andera Partners, Panakes Partners, Fountain Healthcare Partners and Sambatech.

Media Contact:
Sarah Lundberg
Health+Commerce
sarahlundberg@healthandcommerce.com

XyloCor Therapeutics Announces Presentation of Preliminary Clinical Data from Phase 1 Portion of the EXACT Phase 1/2 Study of XC001 Novel Gene Therapy for Refractory Angina at AATS and ASGCT

• Data from the Phase 1 dose‑escalation portion of the Phase 1/2 EXACT study demonstrate XC001 was well‑tolerated at all dose levels tested; highest dose level evaluated selected for ongoing Phase 2 portion of the study

• Preliminary efficacy data highlight XC001 potential for patients with refractory angina with no other treatment options

• Treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects

• Completion of Phase 2 enrollment is expected by the end of May 2022

XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced presentation of initial clinical data from the Phase 1 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina at the American Association for Thoracic Surgery (AATS) Annual Meeting on May 15, 2022, and at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 18, 2022.

XC001: Locally administered, single‑dose gene therapy candidate to address the unmet need in refractory angina

XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec), is under development as a novel approach to treating patients with refractory angina who have exhausted other medical and surgical options. This investigational gene therapy is designed to activate naturally occurring biological pathways by creating new vessels to improve blood flow to areas of the heart not receiving adequate blood supply. This restored blood supply could potentially improve patients’ quality of life by enabling them to resume physical activities and it could reduce episodes of chest pain associated with refractory angina.

In the Phase 1 portion of the EXACT study, 12 subjects with Canadian Cardiovascular Society (CCS) angina class 2‑4 without revascularization options were divided into four escalating dose groups with three subjects each. Each subject received 15 epicardial injections of XC001 at one of the four dosage levels. Safety, efficacy and tolerability evaluations were measured as adverse events (AEs), serious adverse events (SAEs) and change from baseline from three to six months post‑treatment in exercise capacity, ischemic burden by positron emission tomography (PET) imaging, and patient‑reported symptomatology.

No drug‑related SAEs, bleeding complications or ventricular arrhythmias were observed in this Phase 1 dose‑escalation study. Over a six‑month follow up there were a total of 17 SAEs in seven subjects. Eleven SAEs were related to the underlying disease process or other causes. The other six SAEs which occurred in four subjects were judged to be related to the administration procedure, with none of those being unexpected nor resulting in patient death.

“The administration of XC001 appears to have been well‑tolerated at all tested doses,” said Nahush Mokadam, M.D., presenting author at AATS, Division Director, Cardiac Surgery, The Ohio State University Wexner Medical Center and Associate Director of the Heart and Vascular Center and site Principal Investigator for this study. “Objective criteria, including results from exercise tolerance tests and PET scans, suggest therapeutic potential.”

Although the Phase 1 portion of the study was primarily focused on safety and Phase 2 dose selection, initial clinical efficacy data appeared promising. Notably, the data showed positive trends in total exercise duration and reductions in patient symptoms and ischemic burden. Although patient numbers are small, preliminary data suggest that response may be correlated to administered dose.

“The preliminary efficacy evaluation suggests a dose response which is encouraging for the development of XC001 as a therapeutic strategy,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “We anticipate that the Phase 2 expansion portion of this study, which is testing the highest and most efficacious dose from Phase I, will complete enrollment this month. We are incredibly excited by the potential for this investigational therapy to improve the quality of life for these cardiac patients.”

“In many other gene therapy trials, safety concerns arose due to systemic administration of high viral particle loads,” added Dr. Povsic. “In contrast, because we can inject XC001 directly into the heart, we can dramatically reduce overall viral particle loads and systemic exposure while increasing efficacy.”

EXACT Phase 1/2 Study Data Presentations at AATS and ASGCT

Lead author, Dr. Mokadam presented three‑month data from the Phase 1 study in the presentation, Dose Escalation Study of Encoberminogene Rezmadenovec (Adenoviral Vector with Multiple Isoforms of Vascular Endothelial Growth Factor) in Refractory Angina: Phase 1 Results at the AATS Annual Meeting.

Dr. Povsic will present six‑month data from the Phase 1 study in the presentation, Preliminary Safety, Tolerability and Efficacy of Direct Epicardial Administration of Encoberminogene Rezmadenovec to Ischemic Myocardium in Patients with Refractory Angina: Six Month Phase 1 Data at the ASGCT 25th Annual Meeting.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, to be followed by an expansion phase of the trial with 27 additional subjects at the highest tolerated dose. The trial is designed to assess the preliminary safety and efficacy of XC001. The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by an experienced cardiac surgeon. The EXACT trial is being conducted at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain. An estimated one million people suffer from refractory angina in the United States.

About XyloCor

XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for which there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Media Contact

Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

Vivasure Medical Announces Series D Financing to Advance Portfolio of PerQseal Vessel Closure Devices

Funds will support clinical development and regulatory approval of fully absorbable percutaneous closure devices for large-bore vessels

Vivasure Medical® (“Vivasure” or the “Company”), a company pioneering novel fully absorbable technology for percutaneous vessel closure, today announced the closing of the first tranche of €22 million ($23M) as part of its Series D financing round that could reach up to €52 million ($54M) in total. Led by a multi-national strategic corporation, the financing includes an option to buy the Company upon certain milestones. Other participants in this Series D financing round include a second strategic corporate investor as well as existing investors, Fountain Healthcare Partners, Orchestra BioMed, LSP Health Economics Fund managed by the EQT Life Sciences team, Panakès Partners and Evonik Venture Capital.

The financing will support the U.S. and European clinical development and regulatory approval of the Company’s portfolio of fully absorbable, patch-based large-bore percutaneous vessel closure devices for transcatheter endovascular and cardiovascular procedures, including PerQseal® and PerQseal+ for arterial closure and PerQseal Blue for venous closure. Vivasure’s innovative PerQseal technology consists of a proprietary bioabsorbable intravascular patch that seals the vessel from the inside, returning the artery or vein to its natural state without leaving behind the remains of any materials such as collagen, metal implants or sutures commonly used in other closure technologies.

Vivasure Medical’s PerQseal device is the first sutureless and fully absorbable synthetic implant for large-bore arterial vessel punctures and is available to physicians in Europe for use in transcatheter endovascular procedures, including transcatheter aortic valve replacement (TAVR), thoracic endovascular aneurysm repair (TEVAR) and endovascular abdominal aneurysm repair (EVAR). The Company’s next-generation PerQseal+ device has an enhanced bioabsorbable patch designed to address more complex patient anatomies and is currently under clinical evaluation in Europe and the U.S. Vivasure is also developing PerQseal Blue, designed exclusively for sutureless and fully absorbable large-bore venous vessel closure following percutaneous cardiovascular procedures, such as transcatheter mitral valve repair or replacement (TMVR), transcatheter tricuspid valve repair or replacement (TTVR) and leadless pacemaker implants. Currently, there are no sutureless options available for vessel closure following large-bore venous procedures.

“As minimally invasive approaches have become the standard of care for cardiovascular procedures, conventional vessel closure techniques have proven to prolong recovery and lead to bleeding complications for patients. This funding represents an important milestone for our company that will help to further advance our portfolio of novel PerQseal sutureless and fully absorbable vessel closure devices in the U.S. and Europe,” said Andrew Glass, chief executive officer of Vivasure Medical. “We are encouraged by early clinical progress from leading heart centers participating in studies currently underway for PerQseal+ and PerQseal Blue, and we look forward to initiating a U.S. pivotal study for PerQseal+ later this year that will support our submission to the FDA.”

“While tremendous progress has been made for minimally invasive structural heart procedures, vascular issues related to the closure of the procedure remain the most common complication of these interventions,” said Azeem Latib, M.D., section head and director of interventional cardiology and director of structural heart interventions at Montefiore Health System. “The novel PerQseal technology is designed to address these shortcomings and has tremendous potential to improve patient outcomes and enhance procedure efficiency.”

About Vivasure Medical

Based in Galway, Ireland, Vivasure is a medical device company developing advanced polymer implants and delivery systems, primarily focused on minimally invasive vessel closure in cardiology, interventional radiology and vascular surgery. Vivasure operates a fully integrated R&D and ISO 13485 certified manufacturing facility and is backed by leading international medtech investors. For more information, please visit www.vivasuremedical.com.

PerQseal®, PerQseal®+ and PerQseal® Blue are not available for sale in the United States.

Contacts

Sierra Smith
408-540-4296
sierra@healthandcommerce.com

Priothera Receives FDA clearance of IND to start Phase 2b/3 study with mocravimod in Acute Myeloid Leukemia (AML) Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant (HSCT)

Global Phase 2b/3 trial (MO-TRANS) assessing the efficacy and safety of mocravimod, a novel S1P receptor modulator, as an adjunctive and maintenance therapy in AML patients undergoing allogeneic HSCT, planned to start in H2 2022

Priothera Ltd, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, today announces that the U.S. Food and Drug Administration (FDA) has provided clearance to proceed with the Company's Investigational New Drug (IND) application to begin its pivotal Phase 2b/3 study of mocravimod (named MO-TRANS).

Priothera will initiate the MO-TRANS global Phase 2b/3 study in Europe, US and Japan, assessing the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult Acute Myeloid Leukemia (AML) patients undergoing allogenic hematopoietic stem cell transplant (HSCT). The MO-TRANS study is expected to start in the second half of 2022 and preliminary data from this study are expected by the end of 2024.

Allogenic stem cell transplantation is the only potentially curative approach for AML patients, however current treatment options are still associated with a high number of side effects, and high mortality rates.

Florent Gros, Co-Founder and CEO of Priothera, commented "The FDA IND clearance to initiate the MO-TRANS study assessing mocravimod in AML patients undergoing allogeneic HSCT is another major milestone for Priothera. We are on track to initiate this pivotal Phase 2b/3 clinical trial and are looking forward to working alongside a large team of enthusiastic investigators across the US, Europe and Asia, who share our goal of bringing mocravimod to patients as an adjunctive and maintenance treatment for AML and potentially other hematologic malignancies."

About mocravimod

Mocravimod (also known as KRP203), is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogenic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator's unique mode of action to maintain anti-leukemia activity - graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogenic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

About Priothera

Priothera is leading the way in developing orally applied sphingosine-1-phosphate (S1P) receptor modulators for the treatment of hematological malignancies. S1P receptor modulators are known to largely reduce egress of T cells from lymphatic tissues. Not being an immunosuppressant, mocravimod maintains the graft-versus-leukemia (GVL) benefits in patients receiving HSCT while inhibiting graft-versus-host-disease (GvHD).

Priothera was founded in 2020 by an experienced team of drug development experts and is headquartered in Dublin, Ireland, and with a subsidiary in Saint-Louis, France. The Company is backed by international founding investors Fountain Healthcare Partners (Dublin, Ireland), funds managed by Tekla Capital Management, LLC (Boston, Massachusetts), HealthCap (Stockholm, Sweden) and EarlyBird Venture Capital (Berlin, Germany).

For more information please visit: www.priothera.com

Contacts

Priothera
Florent Gros, CEO
E: info@priothera.com

MEDiSTRAVA Consulting
Sylvie Berrebi, Sandi Greenwood, Frazer Hall
E: priothera@medistrava.com
T: +44 (0) 203 928 6900