RARE Daily

April a Quiet Month for Rare Disease Therapeutics Financing and Dealmaking

May 17, 2023

April was a quiet month for rare disease focused drug developers with few financings and no significant deals with disclosed values announced during the month, according to data from Dealforma and Global Genes.

Year-to-date numbers continued to lag compared to 2022, with total equity and debt financings just shy of $4 billion for rare disease therapeutics developers, down 31 percent compared to the first four months of 2022.

Three companies—Sangamo Biosciences, Freeline Therapeutics, and Aeglea BioTherapeutics reported cuts in staff, while Takeda said it would move away from early stage AAV gene therapy work.

Venture capital is down 29 percent year-to-date compared to last year, and public equity and debt are down 19 percent, with no IPOs so far this year.

Significant financings during April include $105 million raised by Swiss biotech Alentis Therapeutics in a series C financing to advance its lead programs for organ fibrosis and oncology into phase 2 and phase 1 studies; $100 million raised by Protagonist Therapeutics to support a global, pivotal, phase 3 study of rusfertide to treat patients with polycythemia vera; and R275 million raised by Mirum Pharmaceuticals in an offering of convertible senior notes that will primarily be used to repurchase revenue interests from an agreement with an affiliate of Oberland Capital Management. Mirum’s Livmarli is approved in the United States for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and in Europe for the same indication in patients two months of age and older.

Partnering potential deal values focused on rare disease therapeutics are down 13 percent year to date compared to the same period in 2022, whereas partnering potential deal values for all therapeutics was on par with 2022. None of the five announced rare disease collaborations disclosed financials.

M&A continues to remain a relative bright spot for all therapeutics focused companies with deal values year to date ahead of the same period in 2022. In April’s only significant acquisition focused on rare disease, Ginkgo Bioworks acquired StrideBio’s AAV capsid discovery and engineering platform assets to strengthen its end-to-end gene therapy capabilities. Financials were not disclosed.

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