Three Rare Disease Drug Developers Raise a Combined $430 Million

Two rare disease drug developers tapped the public markets for fresh capital and one went the private offering route to raise a combined $430 million to advance their therapeutic programs.

Forma Therapeutics, a clinical-stage biopharma focused on rare hematologic diseases and cancers, raised $240 million in an underwritten public offering of 5.3 million shares of its common stock at $45.25 per share. In addition, Forma granted the underwriters a 30-day option to purchase up to an additional 795,000 shares of common stock, less underwriting discounts and commissions.

Forma tapped the public markets after presenting phase 1 clinical proof-of-concept data supporting the potential of its novel experimental PKR activator FT4202 that showed it raised hemoglobin levels in patients with sickle cell disease.

Protagonist Therapeutics, a company focused on hematological diseases and inflammatory disorders, raised $100 million in an underwritten public offering of 4.8 million shares of its common stock at $21 per share. In addition, Protagonist granted the underwriters a 30-day option to purchase up to an additional 714,285 shares of common stock, less underwriting discounts and commissions. 

Protagonist’s offering comes on the heels of updated data supporting the long term efficacy of its lead compound PTG-300, an injectable synthetic peptide mimetic of the natural hormone hepcidin that is being studied in a phase 2 trial for the treatment of polycythemia vera, a rare myeloproliferative disease characterized by the excessive production of red blood cells.

Finally, Solid Biosciences, a clinical-stage company focused on treatments for Duchenne muscular dystrophy (DMD), entered into a securities purchase agreement with a select group of institutional and accredited investors for a $90 million private placement.

In October, Solid Biosciences entered into a strategic collaboration and license agreement with Ultragenyx Pharmaceutical focused on the development and commercialization of new gene therapies for DMD, a rare genetic muscle wasting disease, for which it received $40 million upfront. Around the same time, the U.S. Food and Drug Administration lifted a clinical hold on Solid’s phase 1/2 study of an experimental AAV gene therapy to treat the underlying cause of DMD.