RARE Daily

Aceragen Acquires Arrevus, Expands Late-Stage Rare Disease Pipeline

November 3, 2021

Rare disease therapeutics drug developer Aceragen said it acquired Arrevus, a clinical-stage biopharmaceutical company developing therapies for orphan infectious diseases with high unmet need.

Photo: John Taylor, president and CEO, Aceragen

The transaction adds ARV-1801 (now ACG-721), an oral, small molecule treatment in late-stage development for indications including cystic fibrosis, as well as several preclinical programs with potential application to other rare diseases. Carl Kraus, president and CEO of Arrevus, will join Aceragen as chief medical officer.

“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 CEO, Aceragen.

Aceragen intends to launch advanced clinical studies for both ACG-801 and ACG-721 in rare and orphan indications with overlapping commercial footprints. Specifically, the addition of ACG-721 creates a foundation in the cystic fibrosis field on which to investigate ACG-801’s complimentary mechanism.

ACG-801 is an investigational form of recombinant human acid ceramidase (rhAC) designed to address the enzyme deficiency that is the cause of Farber disease, and for which a registrational trial is planned for the second half of 2022.

ACG-721 is intended for the treatment of cystic fibrosis pulmonary exacerbations and other orphan infectious diseases. The company is planning a phase 2/3 trial for cystic fibrosis pulmonary exacerbations that is expected to begin in the second half of 2022, and a phase 2 trial for an additional orphan indication also expected to begin the first half of 2022.

Aceragen launched in May 2021 with the acquisition of Enzyvant’s enzyme replacement therapy for Farber disease, now ACG-801, for which Enzyvant received an undisclosed upfront payment and was eligible for up to $226 million in development and sales-based milestones, as well as tiered royalties on net sales. At the time, the company also entered into an agreement with NovaQuest Capital Management to fund the development of ACG-801.

Author: Rare Daily Staff

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