Already on Fast Track, Aceragen Launches with $35 Million and Farber Disease Drug

Fast Track

Grabbing a fast-tracked novel enzyme replacement therapy for a rare pediatric disease, new biopharma Aceragen launched with $35 million in hand for product financing.  

The drug the new company picked up comes from Enzyvant, one of the five Vants entrepreneur Vivek Ramaswamy sold off to Japan’s Sumitomo Dainippon Pharma in 2019. The company had already done some FDA legwork for the drug, RVT-801, by gaining Rare Pediatric Disease and Fast Track Designations for it, in addition to Orphan Drug designations by both the FDA and EMA. 

RVT-801 is being developed for Farber disease, an ultra-rare condition typically onset during early infancy. Patients with mild form typically live to only 5-7 years of age, even less in rapid progressive cases. Excess amounts of lipids build up to harmful levels in joints, tissues and the CNS causing severe inflammation with life-threatening complications. There is currently no treatment for the inherited metabolic disorder.  

Although the path for faster approval had been mapped, Enzyvant decided to sell the drug rather than enter the clinic in order to get all hands on-deck for its lead candidate, RVT-802. The company was hit with a CRL in 2019 after submitting a Biologics Licensing Application to the FDA for the therapy aimed to treat tissue-based regenerative congenital athymia, another ultra-rare pediatric condition. Just last month the BLA was resubmitted

Aceragen decided to pick up the mantle for RVT-801, now labeled ACG-801, to bring the drug through the clinic for a much-needed treatment option for children. Enzyvant received an upfront payment with sales-based milestones of up to $226 million for the deal.  

Co-founder and CEO John Taylor is also CEO of Vizigen Therapeutics, another biotech focused on metabolic and ocular diseases. He spent five years as CEO for SPYRYX Biosciences, a company focused on therapies for cystic fibrosis. 

“This program is based on the foundational work of Dr. Ed Schuchman at the Icahn School of Medicine at Mount Sinai, establishing the potential to address the underlying pathology of Farber disease, a genetic deficiency of acid ceramidase. In addition to the intellectual property and regulatory designations, Aceragen has also acquired from Enzyvant a robust preclinical package including several completed toxicology studies, a quantitative patient research study and the first ever natural history study in patients with Farber disease that documented and quantified the features, impact and progression of this devastating condition,” Taylor said.  

The drug is designed to address the genetic mutation in the ASAH1 gene that causes a deficiency of acid ceramidase in patients with Farber disease and a form of Spinal Muscular Atrophy. ACG-801 is a recombinant human acid ceramidase (rhAC). Preclinical development has already provided in vivo proof of concept in a mouse model and IND-enabling GLP toxicology studies. There’s strong potential to use the treatment for cystic fibrosis as well. 

“Breakthrough therapies for patients battling devastating rare diseases often result from extraordinary collaborations between academic discovery teams and dedicated developers like Enzyvant and Aceragen,” said Rachelle Jacques, CEO of Enzyvant. “We are delighted that Aceragen, with strong capabilities, a commitment equal to our own and a singular focus, will rapidly advance this important therapy to address the significant unmet needs of Farber disease patients and their families.” 

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