RARE Daily

Aceragen Launches with $35 Million and Experimental Therapy for Farber Disease

May 3, 2021

Rare Daily Staff

Aceragen, which is developing therapies for rare and ultra-rare diseases said it acquired an experimental enzyme replacement therapy for Farber disease from Enzyvant and secured a $35 million product financing agreement with NovaQuest Capital Management to fund development into a potential registrational study of the therapy ACG-801.

Under the terms of the agreement with Enzyvant, a subsidiary of Sumitomo Dainippon Pharma, the company will receive an upfront payment and development and sales-based milestones up to $226 million, as well as tiered royalties on net sales.

Farber disease is caused by mutations in the ASAH1 gene, resulting in a deficiency of acid ceramidase, a naturally occurring lysosomal enzyme. The enzyme normally acts to metabolize ceramide, a highly inflammatory and apoptotic lipid. Lack of adequate acid ceramidase function results in accumulation of ceramide and causes a severe inflammatory disease associated with macrophage-filled subcutaneous granulomas, joint damage and deformity, and life-threatening respiratory complications among other severe symptoms. Patients with the most rapidly progressive form of Farber disease usually do not live beyond the first few years of life. A portion of the patient population also have progressive central nervous system involvement, leading to developmental delay and regression. Acid ceramidase deficiency also causes spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME) and dysregulation of ceramide metabolism has been implicated in other diseases and conditions, including cystic fibrosis.

ACG-801 (RVT-801) is an experimental form of recombinant human acid ceramidase (rhAC) designed to address the genetic deficiency of the naturally occurring enzyme which is the cause of Farber disease and SMA-PME. Preclinical development for rhAC has been completed, including in vivo proof of concept in a mouse model of Farber disease, and IND-enabling GLP toxicology studies. Data in animal models has also been published supporting the potential of rhAC as a treatment for cystic fibrosis. Patients with Farber disease have been involved in the development through a quantitative research study and the natural history study. Data from these studies support the clinical investigation of ACG-801 and significant progress has been made in harmonized regulatory discussions with the U.S. Food and Drug Administration and the European Medicines Agency. ACG-801 has been granted Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the FDA and EMA.

“This program is based on the foundational work of 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,” said John Taylor, co-founder, president and CEO of Aceragen. “Aceragen’s relationship with NovaQuest, an experienced investor in the area of pharmaceutical development, will enable us to advance through the planned clinical study and associated regulatory submissions with the goal of delivering a disease-specific therapy to patients who are in desperate need.”

Concurrent with the closing of the transaction, NovaQuest’s Managing Partner Ron Wooten and Vice President Stephen Lesser have joined Aceragen’s board of directors.

Photo: John Taylor, co-founder, president and CEO of Aceragen

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