Raleigh’s Aceragen, Arrevus Combine Forces to Tackle Rare Diseases

Raleigh-based Aceragen, a biopharmaceutical company developing therapeutics for rare and orphan diseases, has acquired Arrevus, another Raleigh company with a similar focus.

The combination gives Aceragen a portfolio of potential therapies for Farber disease, cystic fibrosis and orphan infectious diseases.

“The acquisition of Arrevus represents a major step forward for Aceragen in executing on its stated goal of building a multi-product rare disease company that aspires to commercialize its own products,” said John Taylor, president and chief executive officer of Aceragen. “Adding Arrevus’ clinical and regulatory capabilities, along with its lead program in late-stage development, advances our transition to a diversified, rare-disease-focused biotech company with an amazing growth trajectory.”

The transaction gives Aceragen ownership of Arrevus’ lead drug candidate, an oral, small-molecule drug in late-stage development for indications including cystic fibrosis, as well as several preclinical programs with potential application to other rare diseases. 

“I’m really proud of Arrevus’ record of achievement in bringing forward our lead candidate and in establishing development efforts and collaborations for our earlier programs,” said Carl Kraus, M.D., president and chief executive officer of Arrevus. “My team is excited to join Aceragen. Together we can realize the full potential of the combined portfolio for maximum impact on patient needs.”

Kraus will join Aceragen as chief medical officer.

Multiple trials planned

Aceragen intends to launch advanced clinical studies for the newly acquired cystic fibrosis drug, called ACG-721, as well as its own potential treatment for Farber disease, an enzyme replacement therapy called ACG-801. 

A phase 2/3 trial of ACG-721 for cystic fibrosis pulmonary exacerbations is expected to begin in the second half of 2022, and a phase 2 trial for an additional orphan disease indication is expected to begin the first half of the year.

Cystic fibrosis is a genetically inherited disorder that damages the lungs, digestive system and other organs. 

A registrational trial of ACG-801 for Farber disease is planned for the second half of 2022. ACG-801 is an investigational form of recombinant human acid ceramidase designed to address the enzyme deficiency that causes Farber disease.

Farber disease is a genetically inherited lipid-storage disorder in which harmful levels of fat accumulate in joints, tissues and the central nervous system. 

In advance of the trials, Aceragen has established a treatment safety database of 600 patients.

About the companies

Aceragen was founded earlier this year when it acquired worldwide rights to ACG-801 from Enzyvant, a biopharmaceutical firm focused on therapeutics for rare and ultra-rare diseases. Enzyvant is a subsidiary of Sumitovant Biopharma, which is owned by Sumitomo Dainippon Pharma Co. of Japan. 

Aceragen entered into a $35 million product financing agreement with NovaQuest Capital Management to fund the development of ACG-801.

Prior to co-founding Aceragen, Taylor co-founded and was president and CEO of Spyryx Biosciences, a company he spun out of the University of North Carolina at Chapel Hill to develop inhaled peptides for cystic fibrosis. 

Arrevus was founded in 2015 by Kraus. The company received a $250,000 Small Business Research Loan from the North Carolina Biotechnology Center in 2019 to support the development of its cystic fibrosis drug. It acquired the drug, sodium fusidate, from Melinta Therapeutics of New Haven, Conn., in 2019.