HNF conducting meeting for FDA to advance therapies for Charcot-Marie-Tooth

NEW YORK, Sept. 6, 2018 /PRNewswire/ -- Hereditary Neuropathy Foundation (HNF) today announced it is conducting a ground-breaking Externally-led Patient-Focused Drug Development Meeting (PFDD) for Charcot-Marie-Tooth Disease (CMT) and Inherited Neuropathies (IN) on Friday, September 28, 2018 at the College Park Marriott Hotel and Conference Center in Hyattsville, MD.

The meeting will bring together patients, caregivers, industry, healthcare providers, payors, NIH, and FDA, to understand the challenges of patients living with CMT and the unmet medical need where there are no drug treatments available. A live webcast will begin at 8:30am ET.

"This is an unprecedented time for the CMT community. The first CMT clinical trials are now reaching late-stage development, and additional pipeline clinical research is scheduled to take place in 2019. It's vital for all stakeholders to understand the life-altering impact that CMT has on patients and families," said Allison Moore, Founder and CEO of HNF. "This opportunity ensures patient perspectives are considered throughout the drug development continuum. We, as patients, are now steering the direction of our futures and have a responsibility to make our voices heard."

"Providing patients with an opportunity to speak directly to FDA representatives about what it's really like living with CMT and outlining what patients need in terms of new therapies, is critical to advancing much-needed medical breakthroughs," added Courtney Hollett, Executive Director, HNF. "We are grateful to all the patients who will bravely testify to represent our community."

Meeting agenda and information:

Distinguished Speakers at the Meeting:

  • Allison Moore, Founder/CEO, Hereditary Neuropathy Foundation
  • Michael Shy, MD, Director, Division of Neuromuscular Medicine, University of Iowa; Director, Inherited Neuropathy Rare Disease Consortium (INC)
  • Stephan Zuchner, MD, PhD, Co-Director, John P. Hussman Institute for Human Genomics University of Miami Miller School of Medicine
  • Florian Thomas, MD, MA, PhD, MS, Chairman, Neuroscience Institute, Hackensack University Medical Center
  • Lucas Kempf, MD, Acting Associate Director for the Rare Disease Program at the FDA
  • Robert Moore, Co-Founder, True Reply
  • James Valentine, JD, MHS, former FDA official, and current Associate at Hyman, Phelps & McNamara

PFDD Event Sponsors

Pharnext, Acceleron Pharma, Cydan, Flex Pharma, Athena Diagnostics, Ionis Pharmaceuticals, Champlain Valley Dispensary, Cresco Labs, Charcot-Marie-Tooth Association, Muscular Dystrophy Association, and EveryLife Foundation

Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for orphan and common neurodegenerative diseases that currently lack curative and/or disease-modifying treatments. Their lead drug is PXT3003, for the treatment of CMT1A, and benefits from orphan drug status in the United States and Europe. The results of this trial are expected by October 2018.

"We are honored to support and participate in the first FDA Externally-led Patient-Focused Drug Development (PFDD) meeting conducted by HNF dedicated to Charcot-Marie-Tooth disease," said Prof. Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext. "This meeting is a unique opportunity for CMT patients to have their voices heard in terms of their needs and the experience of living with this rare and debilitating disease. As of today, there are no therapeutic options for CMT except palliative care. But with new therapeutic options on the horizon, we believe it is critical for regulatory agencies, like the FDA, to grasp the daily challenges faced by CMT patients," he said.

Acceleron Pharma, Inc., a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, is advancing its lead neuromuscular program, ACE-083 in Phase 2 trials of patients with CMT. ACE-083 is a locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

In July, Acceleron presented positive preliminary results from the dose-escalation, Part 1 of its Phase 2 trial in patients with CMT. Preliminary results of Part 2 of the Phase 2 trial are expected by the end of 2019.

"People suffering from CMT currently have no drug therapy options to address the major consequences of this serious disease, such as foot drop leading to mobility impairment and frequent falls," said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. "We are honored to support this important patient-focused event and hope that ACE-083 will prove to be an important agent to increase lower leg function, improve the ability to walk, and reduce falls."

About Charcot-Marie-Tooth (CMT)

CMT is a progressive neuromuscular disease affecting 1 in 2500 people worldwide, with early signs including high arched feet, curled toes, and claw-like hands. Many of these signs begin subtly and may go undiagnosed for years, leading to legs and arms becoming deformed and difficult to use. Those with CMT often lose the ability to walk and may become dependent upon assistive devices to remain mobile. There is no cure. To date, over 100 mutated genes associated with CMT have been identified, with more being discovered each year.

Voice of the Patient Report

Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), these meetings were designed to more systematically gather patients' perspectives on their condition and available therapies to treat their condition. The Voice of the Patient reports summarize the input provided by patients and patient representatives at each of these public meetings.

"We are thrilled to partner with the Charcot-Marie-Tooth Association and the Muscular Dystrophy Association to bring one voice to the FDA as we prepare the post-meeting Voice of the Patient Report," said Joy Aldrich, Advocacy Director, HNF.

About The Hereditary Neuropathy Foundation (HNF)

HNF, a non-profit 501(c) 3 organization whose mission is to increase awareness and accurate diagnosis of CMT and IN, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures. HNF developed the Therapeutic Research in Accelerated Discovery (TRIAD) program, a collaborative effort with academia, government and industry, to develop treatments for CMT. TRIAD involves many groups that span the drug discovery, drug development and diagnostics continuum.

Media contact: Allison Moore -; (212) 722-8396

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SOURCE Hereditary Neuropathy Foundation


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