RESTORING NERVE FUNCTION

to treat diabetic and other peripheral neuropathies

 
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LATEST NEWS
8.16.18 - PRESS RELEASE: REGENACY PHARMACEUTICALS ANNOUNCES PATENT ISSUANCE FOR RICOLINOSTAT TO TREAT DIABETIC PERIPHERAL NEUROPATHY

 
 

 
 

OUR STORY

Regenacy Pharmaceuticals is developing a novel, disease-modifying approach to treating peripheral neuropathies that goes beyond pain and symptom management to restore peripheral nerve function.

Our lead compound ricolinostat (ACY-1215) is an oral, selective inhibitor of the microtubule modifying enzyme HDAC6 with first-in-class potential, currently positioned to enter Phase 2 clinical trials. Our investigation of ricolinostat for diabetic, chemotherapy-induced and inherited peripheral neuropathies is based on compelling proof-of-concept preclinical studies demonstrating restoration of normal nerve function. 

We were founded in December 2016 following the acquisition of Acetylon Pharmaceuticals by Celgene. Regenacy received exclusive rights to ricolinostat, a clinical stage, orally bioavailable, HDAC6 selective inhibitor, for a range of non-cancer disease indications. We also have a unique and proprietary portfolio of selective HDAC1,2 inhibitors with potential for treating cancer, hemoglobinopathies, and cognitive dysfunction.

 
 
 

OUR FOCUS

We are developing ricolinostat to treat diabetic, chemotherapy-induced, HIV related and inherited peripheral neuropathies. Ricolinostat, an oral, selective histone deacetylase 6 (HDAC6) inhibitor, has previously been clinically evaluated in over 200 patients with hematologic cancer and has demonstrated an excellent safety profile.

Inhibition of HDAC6 is a novel approach to restoring normal nerve function for the potential treatment of peripheral neuropathies by reestablishing  the transport function of microtubules. Neurons conducting signals to and from the spinal cord into the feet and hands, are the longest cells in the body.
 

These neurons rely on an internal microtubule transport network to supply energy and nutrients to maintain the ends of the nerves in the skin and muscles. When this transport is disrupted by disease or neurotoxic drugs like chemotherapy, nerve cells can malfunction and send random signals (pain, tingling, muscle spasms) or no signal at all (numbness, paralysis). Preclinical studies in multiple models of peripheral neuropathy provide compelling evidence that HDAC6 inhibition may offer neuroprotection and/or normalize the function of this neural transport network.

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

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SYMPTOMS OF PERIPHERAL NEUROPATHY

Peripheral neuropathy is a disease that affects three types of nerves outside of the brain and spinal cord. Dysfunction of these nerves can cause spontaneous, inappropriate signals or loss of signals.

 
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Pain: Spontaneous signals from pain nerves cause burning or electric shock sensations that are difficult to relieve with standard analgesics.

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Motor Function: Spontaneous firing of motor nerves cause muscle spasms, while loss of signals in these nerves causes paralysis.

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Sensitivity: Spontaneous firing of sensory nerves causes tingling, while loss of signals in these nerves causes numbness.

 
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No treatment has been approved for peripheral neuropathy. Temporary, symptomatic treatments for neuropathic pain are poorly tolerated, minimally effective and possibly addictive. Ricolinostat aims to provide lasting relief of all symptoms of peripheral neuropathy.

Click here for an educational video on the symptoms of peripheral neuropathy from The Foundation of  Peripheral Neuropathy. 

 
 
 

OUR PIPELINE

Our team is poised to start Phase 2 trials with our selective HDAC6 inhibitor ricolinostat in a range of peripheral neuropathies, including diabetic and chemotherapy induced peripheral neuropathy, and Charcot-Marie-Tooth disease. 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).

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.

HDAC6 regulates multiple intracellular processes such as protein degradation, cell motility, and cell-cell interactions. HDAC6 is a microtubule-associated deacetylase also involved in the regulation of α-tubulin–dependent intracellular mitochondrial transport, a critical internal transport network that supplies energy and nutrients to maintain the ends of the nerves in the skin and muscles. Damage or dysfunction in this network results in the collection of symptoms knowns as peripheral neuropathy.

 
 
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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 leading to increased risk of limb amputation. Current treatments, like gabapentin, address only the pain and are minimally effective, addictive, and/or poorly tolerated and do not restore normal function. Ricolinostat decreases pain responses in diabetic rats, similar to gabapentin.

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Unlike gabapentin, the effect of ricolinostat treatment persists for days after the end of dosing, suggesting a disease modifying effect. Click here for a summary of our preclinical data on ricolinostat in DPN.

 
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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. Inhibition of HDAC6 has been shown to reverse multiple symptoms of CIPN in rodent models, including pain, numbness and loss of nerve fibers in the skin, as shown in this recent publication.

Ricolinostat reverses the increased pain neuron firing rate in rats with taxol induced neuropathy. Unlike gabapentin, however, ricolinostat has no effect on normal peripheral nerve firing rates. Click here for a summary of our preclinical data on ricolinostat in CIPN.

 
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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.

Ricolinostat is effective in a mouse model of CMT2F, a rare inherited sensory-motor neuropathy. Treatment with ricolinostat for 3 weeks increases nerve conduction in the periphery and leads to re-enervation of muscles.

Click here to learn more about the types of peripheral neuropathy, risk factors and other statistics.

Additional opportunities for ricolinostat include polycystic kidney disease and cholangiocarcinoma.


HDAC 1,2 Inhibitors

Regenacy also has a unique and proprietary portfolio of selective HDAC1,2 inhibitors with potential for treating cancer, hemoglobinopathies, and cognitive dysfunction. We are positioned to rapidly advance our HDAC1,2 inhibitors through drug candidate selection and preclinical development. For more information, please see the following link.

 
 
 

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.

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.

Dr. Chin is CMO at Frequency Therapeutics and currently Bertarelli Professor of Translational Medical Science and Professor of Medicine Emeritus at Harvard Medical School. He previously served as CMO and Executive Vice President at PhRMA and Executive Dean for Research at Harvard Medical School, following a 10-year career at Eli Lilly, where he was most recently Senior Vice President for Discovery Research and Clinical Investigation. Prior to Lilly, he was Professor of Medicine and a Professor of Obstetrics, Gynecology, and Reproductive Biology at Harvard Medical School and Chief of the Division of Genetics and Senior Physician at Brigham and Women’s Hospital. Dr. Chin is a world-renowned molecular endocrinologist who has received many awards for his research on the molecular mechanisms of nuclear receptor action on thyroid, estrogen, and other hormones. He serves on several scientific boards, including the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institute of Child Health and Human Development (NICHD), Agency for Science, Technology and Research in Singapore, Motus Therapeutics, Rhythm Pharmaceuticals, Catabasis Pharmaceuticals and Takeda Pharmaceuticals. He received an AB in Chemistry from Columbia College and M.D. from Harvard Medical School.

    • 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
    • Bertarelli Professor of Translational Medical Science and Professor of Medicine of Emeritus at Havard Medical School
    • Expert in endocrinology
 

LATEST NEWS & PUBLICATIONS


INHIBITION OF HISTONE DEACETYLASE 6 (HDAC6) PROTECTS AGAINST VINCRISTINE-INDUCED PERIPHERAL NEUROPATHIES -READ MORE

PRESS RELEASE: REGENACY PHARMACEUTICALS ANNOUNCES PATENT ISSUANCE FOR RICOLINOSTAT TO TREAT DIABETIC PERIPHERAL NEUROPATHY -READ MORE

 
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