Harnessing protein acetylation 

for breakthrough therapies in peripheral neuropathy, hemoglobinopathies and beyond

 
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LATEST NEWS
07.19.17 - PRESS RELEASE: Regenacy Pharmaceuticals Presents Data Demonstrating that HDAC1,2 Inhibition Improved Cognitive Function in Mouse Models of Alzheimer's Disease at the Alzheimer’s Association International Conference

 
 

 
 

OUR STORY

Regenacy Pharmaceuticals is a pioneer in developing novel treatments through regeneration of normal protein function using oral, isoform selective histone deacetylase enzyme ("HDAC") inhibitors.

We were founded in December 2016 following the acquisition of Acetylon Pharmaceuticals by Celgene. Regenacy received exclusive rights to the clinical stage, orally bioavailable, HDAC6 selective inhibitor, ricolinostat (ACY-1215), for a range of non-cancer disease indications including peripheral neuropathy, polycystic kidney disease and cholangiocarcinoma. Regenacy also has a unique and proprietary portfolio of selective HDAC1,2 inhibitors with significant potential for treating hemoglobinopathies, cognitive dysfunction and leukemia.

 
 

OUR FOCUS

Histone deacetylases (HDACs) are a family of 18 related enzymes found in all human cells that remove acetyl groups from intracellular proteins. This activity is critical in maintaining normal gene expression and protein function throughout the cell, tissues and body.  Disruption of protein acetylation contributes to a wide range of major medical conditions.  Currently marketed HDAC inhibitors lead to significant side-effects by indiscriminate targeting of multiple HDACs. Regenacy’s selective HDAC inhibitors have a greatly improved safety profile, reduce abnormal function of specific HDAC enzymes and can regenerate normal biological function.

Regenacy’s focus is the development of oral, selective HDAC inhibitor compounds to address major areas of unmet clinical need including diabetic and chemotherapy-induced peripheral neuropathy, polycystic kidney disease, cholangiocarcinoma, sickle cell disease, beta thalassemia, cognitive dysfunction and leukemia.

For more information on our discovery activities, please view our publications and presentations.

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DISEASE AREAS

 

Diabetic Peripheral Neuropathy (DPN)

Diabetic Peripheral Neuropathy (DPN) is one of the most common and serious complications of Type 1 and Type 2 diabetes. DPN causes burning pain, numbness, and tingling in the hands and feet, all of which have a major impact on quality of life. Current treatments address only the pain and are minimally effective, addictive, and/or poorly tolerated and do not restore normal function.

 

Chemotherapy Induced Peripheral Neuropathy (CIPN)

An estimated 500,000 patients suffer from chemotherapy induced peripheral neuropathy (CIPN). CIPN is a common adverse effect of several cancer therapies including taxanes and platinum drugs. Symptoms include decreased sensation and tingling of the hands and feet, severe pain, numbness and muscle weakness, all of which can occur during cancer treatment, and frequently persist after chemotherapy has ended. There are no approved therapies to combat the debilitating symptoms of CIPN.

 

Charcot-Marie-Tooth Disease (CMT)

Charcot-Marie-Tooth disease (CMT) is a progressive and degenerative nerve disease that usually appears in adolescence or early adulthood. Symptoms include muscle weakness, decreased muscle size and loss of feeling in the hands and feet resulting in loss of coordination in the limbs. There are no FDA approved treatments to stop or reverse the loss of nerve function in CMT.

 

Sickle Cell Disease (SCD) and Beta Thalassemia (bT)

Hemoglobinopathies, including sickle cell disease (SCD) and beta thalassemia (bT), make up the largest group of severe genetic diseases worldwide. Many SCD patients are at risk for painful and life-threatening vaso-occlusive (blocked blood vessels) crises, and iron overload and other adverse consequences of repeated blood transfusion (bT), resulting in frequent hospitalization, organ failure and shortened life expectancy. There are few therapeutic options for these patients, who typically receive treatment only during crises (pain, hemorrhage, stroke), and no treatments fully restore normal red blood cell function.

 
 
 

OUR PIPELINE

Our team is poised to start Phase 2 trials in peripheral neuropathy with our selective HDAC6 inhibitor ricolinostat. We are also rapidly working to advance promising lead HDAC1/2 inhibitors through drug candidate selection, preclinical development and into human clinical trials for the treatment of sickle cell disease and beta-thalassemia.

 
 
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HDAC6 INHIBITORS

Opportunities for ricolinostat: Peripheral neuropathy, polycystic kidney disease and cholangiocarcinoma.

Our lead program ricolinostat (ACY-1215) is currently positioned to enter Phase 2 clinical trials in peripheral neuropathy with first-in-class potential based on compelling proof-of-concept preclinical studies demonstrating restoration of normal nerve function. Ricolinostat has demonstrated an excellent safety and tolerability profile in prior Phase 1 and 2 clinical trials, particularly when contrasted with the high toxicity of currently marketed pan-HDAC inhibitors (e.g. vorinostat, panobinostat).

Ricolinostat selectively inhibits the intracellular enzyme HDAC6. HDAC6 regulates multiple intracellular processes such as protein degradation, cell motility, and cell-cell interactions. Selective inhibition of HDAC6 as a therapeutic target has the potential to ameliorate several severe chronic diseases and restore normal biological function.

 

HDAC1,2 INHIBITORS

Opportunities: Sickle cell disease, beta-thalassemia, cognitive dysfunction and leukemia (AML/MDS).

Regenacy’s HDAC1,2 inhibitors have demonstrated preclinical proof-of-concept for the treatment of sickle cell disease, beta-thalassemia and cognitive dysfunction. HDAC1,2 inhibition can increase the production of fetal hemoglobin (HbF) protein in primates. Elevated levels of normal HbF can reduce disease severity and restore normal function in patients with sickle cell disease or beta-thalassemia by replacing missing or defective adult hemoglobin. Recent studies in mouse models of Alzheimer’s Disease have shown that HDAC1,2 inhibition improved performance in spatial learning.

Regenacy’s proprietary, isoform selective HDAC1,2 inhibitors are differentiated from pan-Class I inhibitors by superior preclinical safety and tolerability profiles, and they include both blood-brain barrier penetrant and non-penetrant preclinical candidates formulated for oral administration as tablets, with excellent pharmacokinetic properties.

 
 
 

OUR TEAM

Regenacy is supported by a team of experts and industry leaders committed to translating innovation into a diverse portfolio of new therapies for millions of people worldwide. Our advisory team is composed of scientists and clinicians at the forefront of developing novel therapies and technologies.

 
 
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LEADERSHIP

SIMON S. JONES, Ph.D.
President and CEO

President and CEO

Simon S. Jones, Ph.D., is the President and Chief Executive Officer of Regenacy Pharmaceuticals and served as Chief Operating Officer and Senior Vice President of Preclinical Development, from the formation of Regenacy in December 2016. Simon joined Acetylon in September 2009 from EPIX Pharmaceuticals Inc., where he was Vice President of Biology and ADMET. Prior to EPIX, Dr. Jones held senior level positions in drug discovery and preclinical development for leading biotechnology companies including ArQule Inc., Curis Inc., Creative BioMolecules Inc. and at Genetics Institute/Wyeth, now part of Pfizer. Dr. Jones received his B.Sc. Honors Degree and Ph.D. in chemistry from Kings College, University of London, U.K., where he also engaged in post-doctoral research, and later at the Massachusetts Institute of Technology, Cambridge, MA under the Nobel Laureate Prof. H.G. Khorana.

MATTHEW JARPE
Vice President of Research & Development

Vice President of Research & Development

Matthew Jarpe is the Vice President of Research and Development of Regenacy Pharmaceuticals. Previously, Matt served as an Associate Vice President of Biology at Regenacy. He served as Director, then Associate Vice President of Biology at Acetylon Pharmaceuticals where he led the development of highly selective inhibitors for HDAC6 as well as HDAC1 and 2 in several non-oncology disease targets. Prior to joining Acetylon, Matt worked at Biogen as a Senior Scientist and at Surface Logix as an Associate Director of Biology. He received his Ph.D. from Johns Hopkins and completed his postdoctoral training at National Jewish Medical Center.

JOHN ROCHA
Chief Financial Officer

Chief Financial Officer

John Rocha is the Chief Financial Officer of Regenacy Pharmaceuticals. Over his 20+ year career, John has served in a variety of finance and accounting roles for biopharmaceutical and healthcare companies including Zycos, MGI Pharma, Logical Therapeutics, Valeritas, and Artisan Pharma. Most recently, John was Senior Director of Finance & Administration at Acetylon Pharmaceuticals where he led all accounting, finance and administrative activities, and was a key member of the senior management team that sold Acetylon to Celgene. John began his career at Ernst & Young, and is a licensed CPA. He received his B.A. from Boston College and his MBA/MS from Northeastern University.

KENNETH JACOBS
Associate Vice President of Program Management

Associate Vice President of Program Management

Ken Jacobs is the Associate Vice President of Program Management at Regenacy Pharmaceuticals. Prior to this role, Ken served as the Senior Director of Program Management at Acetylon Pharmaceuticals where he was also Director of Program Management. He was formerly Director of Project Management at ImmunoGen and Wyeth Pharmaceuticals after holding senior level positions in discovery research at Wyeth and Genetics Institute. Ken received his Ph.D. in Molecular Biology from Washington University in St. Louis.

TIMOTHY KACHMAR
Associate Vice President of Regulatory Affairs

Associate Vice President of Regulatory Affairs

Tim Kachmar is the Associate Vice President of Regulatory Affairs and Quality Assurance of Regenacy Pharmaceuticals. Previously, Tim joined Acetylon Pharmaceuticals as an Associate Vice President of Regulatory Affairs where he led the regulatory affairs team and provided regulatory leadership in the development of novel HDAC compounds. He also served as Senior Director of Regulatory Affairs and Quality Assurance at Verastem, Radius Health, Inc., and Mersana Therapeutics. Tim received his undergraduate degree and M.S. in Biology from the University of Massachusetts at Lowell.

 
President and CEO Executive Chairman

Marc A. Cohen is the Executive Chairman of Regenacy Pharmaceuticals. He is also a Co-founder and Executive Chairman of C4 Therapeutics, Chairman of Frequency Therapeutics, as well as the Co-founder and Chairman of Acetylon Pharmaceuticals, Inc. and OncoPep, Inc., two biotech companies spun out of Dana-Farber Cancer Institute and Harvard University. Additionally, Marc is the Co-founder and Chief Executive Officer of Bublup, Inc. and COBRO Ventures, Inc. He was the Chief Executive Officer, Chairman and Co-founder of OPNET Technologies, Inc., a world leader in network and application performance management. OPNET was sold to Riverbed Technology in December 2012. He is currently a Trustee of the Dana-Farber Cancer Institute where he is focusing on accelerating translational research and increasing the flow of new therapies to patients. Marc received his undergraduate degree in Engineering Science from Harvard University and an M.S. in Electrical Engineering from Stanford University.

Walter Ogier is a Co-Founder and former President & CEO of Regenacy Pharmaceuticals and the founding President & CEO of Acetylon Pharmaceuticals, sold to Celgene in late 2016 following the Regenacy spin-out. In addition to serving as Director and advisor to Regenacy, Mr. Ogier currently serves as a Director of privately held Biothera Pharmaceuticals (Eagan MN) and Thetis Pharmaceuticals (Branford CT) and as an advisor/mentor to several emerging biopharmaceutical companies. Mr. Ogier has more than 30 years of experience in the biopharmaceutical/biomedical industry, including with Baxter Healthcare Corporation (Deerfield IL, stem cell transplant, immuno-oncology, medical devices) in product management and business development roles, with Aastrom Biosciences (Ann Arbor MI, cell therapy) as VP Marketing leading to a successful IPO, and subsequent President & CEO roles with Eligix (Medford MA, sold to BioTransplant), Genetix/bluebird bio (Cambridge MA, gene therapy), and Arbios Systems (Waltham, MA, liver dialysis). Mr. Ogier received a BA magna cum laude in chemistry from Williams College and an MBA from the Yale School of Management.

Ken is the Kraft Family Professor of Medicine at Harvard Medical School as well as Director of the Jerome Lipper Multiple Myeloma Center and Lebow Institute for Myeloma Therapeutics at Dana-Farber Cancer Institute. His research and impact on patient outcomes has been widely recognized including election to the National Institute of Medicine and the Royal Colleges of Physicians and Pathologists and the 2012 American Cancer Society Medal of Honor. His research has pioneered understanding of the tumor microenvironment in multiple myeloma and he has played an extensive role in the development and approval of numerous new treatments for myeloma patients. He also serves on the Board of Directors for C4 Therapeutics. He received his MD from Johns Hopkins Medical School, where he also trained in internal medicine, and completed hematology, medical oncology and tumor immunology training at Dana-Farber Cancer Institute. He is a member of the Institute of Medicine of the National Academy of Sciences and served as President of the International Myeloma Society.

Bruce is a Partner at NewSpring Capital. Bruce was the Chairman and Chief Executive Officer at Barr Pharmaceuticals Inc. from 1994 to 2008. Prior to Barr, he was a Partner at Winston & Strawn LLP and Bishop, Cook, Purcell, and Reynolds. Bruce has been on the Board of Directors for Cardinal Health and Momenta Pharmaceuticals since 2009. He is also on the Board of Directors for C4 Therapeutics, Abon Pharmaceuticals and OncoPep, Inc. Bruce was the Chairman of Generic Pharmaceutical Association from 2006 to 2007. Mr. Downey received a BS degree from Miami University and a JD from Ohio State University.

Ron has over 35 years of Director and executive management experience, serving as a Director, CEO and CFO for public companies and venture-backed portfolio companies in the technology and life sciences industries. Since 1981, he served as CFO during six IPO's or as a CEO for technology companies, and since 2003 as a Director for several public and private companies. He has held executive financial and management roles with Sucampo Pharmaceuticals, OTG Software, Trusted Information Systems, Charmilles Technology Manufacturing Corporation, Leeco Diagnostics, Inc., Somanetics Corporation, and Photon Sources, Inc. He also serves on the Board of Directors for C4 Therapeutics. Ron is a Principal at Hoffman CFO Consulting, a Certified Board Leadership Fellow of the National Association of Corporate Directors (NACD) and holds dual BA degrees in accounting and multi-disciplinary pre-law from Michigan State University.

Bill is Director of Investments for the Kraft Group LLC. Since joining the Kraft Group in 1992, he has been involved with numerous investing and operating initiatives, including the creation of a dedicated family investment office which he leads today. Bill also serves as Board Member or Observer for C4 Therapeutics, Dune Medical, Torpedo Diagnostics, Spero Therapeutics and Silk Therapeutics. Bill received a BSBA from Northeastern University and a MSIM from Boston University.

Miles is the Founder and President of Access Capital, a 30-year-old New York based multi-strategy investment firm. The Company is a leading national asset-based lender that is also an active investor in early stage companies and in multi-family real estate. Miles has been on the board of two New York Stock Exchange companies: Diagnostek, a pharmacy benefits company of which he was also Chairman of the Audit Committee; and Newkirk, a real estate investment trust. He was formerly the Vice Chairman of the Board and Chairman of the Investment Committee of Horace Mann School. He also serves on the Board of Directors for C4 Therapeutics. He is a graduate of Horace Mann, Yale College (BA), NYU Law School (JD) and Harvard Business School (YPO/CEO Program).

Lori is a council member for Dana Farber Multiple Myeloma group, a supporter of the Multiple Myeloma Research Foundation and a former ambassador for Penn Medicine’s Hematologic Malignancies program. She is also a founder and Director of National Air Cargo Holdings, Inc., a multinational corporation with an integrated global freight forwarding business combined with a US Part 121 Air Carrier, National Airlines. Previously, Lori served on the Board of Directors at Acetylon Pharmaceuticals and served as a patient advocate for its HDAC6 selective inhibitor program in hematologic malignancies.

Dr. Elena Prokupets co-founded Lenel Systems International and was President, CEO and Chairman of the Board until its sale to United Technology Corporation (UTC). Elena also co-founded and led Edicon Systems, a pioneer in electronic images and electronic photography, that is now owned by Kodak. Dr. Prokupets was on the Board of Directors of Acetylon Pharmaceuticals until it was sold to Celgene and currently serves on the Board of Directors for Regenacy Pharmaceuticals, C4 Therapeutics and OncoPep, Inc. She is also a Trustee of the University of Rochester and the Managing Director of the Metropolitan Opera of New York. Elena has an MS in Electrical Engineering and PhD in Computer Science.

    • Chair, Director, Lebow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center of the Dana-Farber Cancer Institute; Kraft Professor of Medicine, Harvard Medical School and Acetylon Scientific Founder
    • Expert in hematology, medical oncology and tumor immunology

      • Assistant Professor at Harvard Medical School and the Center for Systems Biology at Massachusetts General Hospital
      • Expert in medicinal chemistry, chemical biology and HDAC inhibitors

      • Professor at Harvard Medical School and Director of the Center for Autonomic and Peripheral Nerve Disorders in the Department of Neurology Beth Israel Deaconess Medical Center
      • Expert in peripheral neuropathies and autonomic dysfunction

      • Professor at the University of Rochester Medical Center
      • Expert in peripheral neuropathy, chronic pain and osteoarthritis

      • Assistant Professor at Harvard Medical School and physician at Boston Children’s Hospital and the Dana-Farber Cancer Institute
      • Expert in hemoglobinopathies, hemoglobin production and hematopoiesis
 

LATEST NEWS & PUBLICATIONS


HDAC6 INHIBITION EFFECTIVELY REVERSES CHEMOTHERAPY-INDUCED PERIPHERAL NEUROPATHY
READ MORE

ALZHEIMER’S NEWS TODAY: REGENACY’S HDAC BLOCKER IMPROVED COGNITIVE FUNCTION IN MICE WITH ALZHEIMER’S, STUDY SHOWS - READ MORE

 
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