Aceragen Inc., a biopharmaceutical company focused on advancing transformational therapeutics for rare and ultra-rare diseases, has acquired of Enzyvant’s RVT-801 (now ACG-801), an investigational enzyme replacement therapy for acid ceramidase deficiency presenting as Farber disease, a lysosomal storage disease with a unique, severe inflammatory phenotype for which no disease-specific therapy exists. Enzyvant will receive an upfront payment and development and sales-based milestones up to $226 million, as well as tiered royalties on net sales. In addition, Aceragen entered into a $35 million product financing agreement with NovaQuest Capital Management to fund the development program into a potential registrational study.
Aceragen co-founder John Taylor will lead the company as president and chief executive officer, having previously held the same roles with Spyryx Biosciences.
“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,” Taylor stated. “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. 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.”
ACG-801 is an investigational form of recombinant human acid ceramidase 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. Patients with Farber disease have been involved in the development through a quantitative research study and the natural history study.
“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.”
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.
“We congratulate John and the Aceragen team for identifying this exceptional rare disease opportunity in Farber disease and for choosing NovaQuest’s Product Finance approach among the alternatives,” said Lesser. “It was a pleasure for all of us at NovaQuest to conclude that at-risk, non-dilutive financing is possible as a company-enabling resource for development funding within the realm of ultra-rare disease. NovaQuest believes that opportunities, like ACG-801, offer an attractive area for investment, combining a significant unmet medical need with a treatment that has the potential to deliver a clinically meaningful benefit and improved quality of life for patients.”
“Our product financing agreement with Aceragen furthers NovaQuest’s core strategy and focus of providing innovative funding for a wide range of biopharma companies and products,” Wooten added. “We are pleased to join the Aceragen board and contribute to the team’s efforts to provide benefits to patients with Farber disease.”
Wedbush Securities acted as strategic financial advisor, Hutchison PLLC was corporate counsel, and Hogan Lovells provided IP counsel to Aceragen. Arnold & Porter was legal counsel to Enzyvant, and Wyrick Robbins Yates & Ponton LLP acted as legal counsel to NovaQuest.