Aceragen bags Enzyvant drug and $35M, plots fast path to market

Enzyvant has sold its preclinical Farber disease therapy to Aceragen. NovaQuest Capital Management is funding the newly founded Aceragen, setting the biotech up to enter the clinic and go on to seek approval.

Enzyvant sketched out a fast path to market for enzyme replacement therapy RVT-801 in 2019. At that time, Enzyvant planned to run a single trial of the recombinant human acid ceramidase and bring the drug to market in 2022. Then, the FDA rejected Enzyvant’s lead candidate—RVT-802—and the pandemic hit. Rather than enter the clinic, Enzyvant began seeking a buyer for RVT-801.

Aceragen answered the call for a buyer. The biotech has secured the rights to RVT-801, now known as ACG-801, in return for an upfront payment and development- and sales-based milestones of up to $226 million.

In return for the outlay, Aceragen has gained a candidate designed to combat the accumulation of ceramide in patients with Farber, a lysosomal storage disease. Farber patients often die in the first few years of life and lack specific treatments. ACG-801 has improved outcomes in a mouse model and completed several toxicology studies. 

Aceragen has entered into a $35 million product financing agreement with NovaQuest to support further development of the drug. The money will equip Aceragen to take ACG-801 into a potential registrational study.

John Taylor, co-founder of Aceragen, will oversee the work as president and CEO. Taylor held the same roles at Spyryx Biosciences, a biotech that worked on inhaled peptides for pulmonary diseases including cystic fibrosis before ceasing operations. 

Taylor is joined at Aceragen by Alexander Solyom, who has taken up the title of senior vice president, rare disease development. Solyom was previously senior medical director, clinical development at Enzyvant, a role he took up when his then employer, Plexcera Therapeutics, teamed up with Roivant to develop the enzyme replacement therapy.