The investigational therapy has been designed to address the genetic deficiency of the naturally occurring enzyme which is the cause of Farber disease


Aceragen buys Enzyvant’s therapy to treat Farber disease. (Credit: Tumisu from Pixabay)

Aceragen, a US-based biopharmaceutical company, has acquired Enzyvant’s investigational therapy, RVT-801 (now ACG-801), to treat Farber disease.

Farber disease is a lysosomal storage disease with a severe inflammatory phenotype for which there is no disease-specific therapy is available.

As part of the transaction, Enzyvant will secure an upfront payment and development and sales-based milestones up to $226m in addition to tiered royalties on net sales.

Also, Aceragen signed a $35m product financing deal with NovaQuest Capital Management to further support the development of the drug.

The financing will help Aceragen to take the investigational therapy into a potential registrational study.

ACG-801 is an investigational form of recombinant human acid ceramidase. It is designed to address the genetic deficiency of the naturally occurring enzyme which is the cause of Farber disease and spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME).

The drug has been granted rare paediatric disease and fast track designations by the US FDA, as well as orphan drug designations by the FDA and EMA.

Aceragen co-founder John Taylor said: “The Aceragen team is delighted to announce the strategic transaction with Enzyvant and the relationship we have established with NovaQuest to continue the development of ACG-801.

“This programme 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.

“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.”

With the closing of the transaction, NovaQuest managing partner Ron Wooten and vice president Stephen Lesser have joined Aceragen’s board of directors.

Enzyvant CEO Rachelle Jacques said: “Breakthrough therapies for patients battling devastating rare diseases often result from extraordinary collaborations between academic discovery teams and dedicated developers like Enzyvant and Aceragen.

“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.”