FDA Grants Orphan Drug Designation to EPI-321, Epic Bio's Novel Genetic Medicine Candidate for Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

South San Francisco, CA 94080

- Potential one-time therapy for the treatment of FSHD includes a novel non-cutting dCas protein delivered via a single AAV -

- EPI-321 is the only therapy designed to target the epigenetic root cause of the disease -

- Company on track for clinical initiation in first half of 2024 -

SOUTH SAN FRANCISCO, Calif., Nov. 16, 2023 (GLOBE NEWSWIRE) -- Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults. EPI-321 is an investigational therapy being developed as a potential single-dose treatment to suppress abnormal expression of the DUX4 gene.

Epic plans to initiate a first-in-human, Phase 1/2 clinical study of EPI-321 in the first half of 2024. The multi-center study will be designed to assess the safety, activity, and preliminary efficacy of EPI-321 in individuals with FSHD.

“We are pleased the FDA has recognized the unmet need of those living with FSHD by granting this Orphan Drug Designation, and we believe EPI-321 could serve as an important new therapeutic option for these patients,” said Weston Miller, M.D., chief medical officer of Epic Bio. “We are working diligently to advance EPI-321 toward the clinic, and we look forward to generating meaningful clinical data to inform its future development as a potential new treatment for FSHD.”

The FDA has the authority to grant Orphan Drug Designation to therapies to prevent, diagnose or treat a rare disease or condition, defined as those affecting fewer than 200,000 people in the United States. The designation qualifies drug developers for certain financial benefits, including tax credits, prescription drug user fee waivers, and a potential period of 7 years of market exclusivity should EPI-321 receive marketing approval in the U.S.

About FSHD

FSHD is a rare genetic muscle disease which affects approximately 4.6 in 100,000 people worldwide, and which currently has no cure. It is caused by a genetic mutation leading to aberrant expression of the DUX4 gene. FSHD affects both females and males; 90 percent of patients' symptoms manifest before the age of 201. The disease varies in severity and can manifest in progressive muscle weakness and tissue atrophy in the face, shoulders, upper arms, and lower legs. Twenty-five percent of people with FSHD will require a wheelchair by the age of 502.

About EPI-321

EPI-321 is an investigational epigenetic therapy that aims to address the underlying molecular mechanisms of FSHD by restoring methylation to the D4Z4 region of chromosome 4 and halting toxic expression of the DUX4 gene. EPI-321 is delivered to muscle tissue within a single AAV vector (AAVrh74) which has been clinically validated for muscle delivery. Preclinical studies on EPI-321 have demonstrated its ability to robustly suppress pathological expression of the DUX4 gene and reduce muscle cell death.

About Epic Bio

Epic Bio is a leading epigenetic editing company, leveraging the power of CRISPR without cutting DNA. The company’s proprietary Gene Expression Modulation System (GEMS) includes the smallest Cas protein known to work in human cells, enabling in vivo or ex vivo delivery via a single viral vector. Epic’s lead program, EPI-321, is in IND-enabling studies for the treatment of facioscapulohumeral muscular dystrophy (FSHD); additional programs seek to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), and other indications. The company is financially backed by Horizons Ventures and other leading investors. Visit www.epic-bio.com for more information or follow us on Twitter and LinkedIn

  1. https://www.mda.org/disease/facioscapulohumeral-muscular-dystrophy
  2. https://www.fshdsociety.org/what-is-fshd/early-onset-fshd/


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