NHP studies demonstrate EPI-321’s favorable safety and pharmacokinetic profile
IND and CTA applications planned for 2023
SAN FRANCISCO, Calif. — June 16, 2023 — Epicrispr Biotechnologies, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today presented new non-human primate (NHP) data supporting the clinical advancement of EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The data were presented in a poster at the FSHD International Research Congress being held June 15-16, 2023, in Milan, Italy.
“We are very encouraged by the totality of preclinical data on EPI-321, indicating this therapy can reverse disease pathophysiology in FSHD with a high degree of safety. These results were made possible via Epicrispr Biotechnologies’ unparalleled Gene Expression Modulation System (GEMS) platform, which overcomes limitations of other types of genetic medicines,” said Amber Salzman, Ph.D., chief executive officer of Epicrispr Biotechnologies. “We are working to expeditiously bring this therapy to the clinic to provide a much-needed treatment option for patients with FSHD.”
New data presented at the conference detail the safety, pharmacokinetics, and biodistribution of EPI-321 in NHPs. No adverse events, liver toxicity, or severe immune response were observed within three months post-delivery of EPI-321 at both low and high doses. EPI-321 also showed favorable pharmacokinetics and biodistribution, with tissue-specific mRNA analyses indicating robust and specific expression of the cargo DNA in skeletal muscle.
“Following on the heels of the preclinical data we shared in an oral presentation at ASGCT earlier this year, these additional data on EPI-321 further underscore its potential to be a dramatically new approach to treating FSHD,” said Alexandra Collin de l’Hortet, Ph.D., head of therapeutics at Epicrispr Biotechnologies. “Based on the evidence of EPI-321’s ability to halt muscle cell death, and its favorable safety profile, we are committed to rapidly progressing this candidate to clinical studies.”
Epicrispr Biotechnologies plans to file an IND and CTA for EPI-321 later this year and to initiate a first-in-human clinical trial in 2024. The presentation will be made available under the publications section of the Science page on the Epicrispr Biotechnologies website.
About EPI-321
EPI-321 is an epigenetic therapy in preclinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). It was developed using Epicrispr Biotechnologies’ proprietary Gene Expression Modulation System (GEMS) platform. EPI-321 targets the D4Z4 region, which is hypomethylated in FSHD, leading to toxic expression of the DUX4 gene. EPI-321 is designed to remethylate D4Z4, thereby decreasing expression of DUX4.
About Epicrispr Biotechnologies
Epicrispr Biotechnologies is a leading epigenetic engineering company, leveraging the power of CRISPR without cutting DNA. The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies. Through the company’s library of the most compact Cas DNA-binding proteins to work on human cells, the company is developing in vivo therapies with delivery via a single AAV vector. Epicrispr Biotechnologies has an initial focus on facioscapulohumeral muscular dystrophy (FSHD) and is conducting additional research to address alpha-1 antitrypsin deficiency (A1AD), heterozygous familial hypercholesterolemia (HeFH), as well as other indications. The company is financially backed by Horizons Ventures and other leading investors. Visit www.epicrispr.com for more information or follow us on Twitter and LinkedIn.
Investor Contact
Shawn M. Cox
Epicrispr Biotechnologies
Manager, Investor Relations, and Corporate Communications
shawn.cox@epic-bio.com
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