Three Rare Disease Companies Raise Combined $380 Million in Public Offerings

Rare Daily Staff

Beam Therapeutics, Syros Pharmaceuticals, and Fulcrum Therapeutics tapped the public markets for capital to advance their pipelines of genetic medicines.

Beam Therapeutics develops precision genetic medicines through base editing. The company is beefing up its coffers through the sale of 2.8 million shares of its common stock to certain institutional investors in a private placement for gross proceeds of approximately $260 million, before fees and expenses.

The offering price of $93.00 per share represents an approximately 10 percent discount to the 5-day volume weighted average share price. The financing syndicate includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management, among others.

The company says its proprietary base editing technology can make precise edits to single base pairs in DNA and RNA to correct, modify, activate, silence, and edit genes at multiple sites at the same time without detectable translocations.

Beam is addressing a variety of targets using three delivery technologies that have been clinically validated for a specific target: electroporation for hematology and oncology targets, non-viral liquid nano-particles for delivery of RNA therapies targeting liver diseases, and AAV-vector gene therapy delivery for ocular and central nervous system disorders.

Beam’s preclinical pipeline is broad and includes compounds in development for rare diseases such as sickle cell disease, beta thalassemia, alpha 1 antitrypsin deficiency, glycogen storage disorder 1a, Stargardt disease, an inherited form of macular degeneration, and other central nervous system disorders.

Syros Pharmaceuticals develops small molecule therapeutics that control the expression of genes. The company raised $75.6 million in an underwritten public offering of 5.4 million shares of common stock at a public offering price of $14.00 per share, which would result in gross proceeds of approximately $75.6 million, before underwriting discounts and commissions.

The proceeds of the offering will be used to fund the development of Syros’ ongoing clinical and preclinical programs, and for working capital and other general corporate purposes.

Syros is advancing a clinical-stage pipeline, including: SY-1425, a first-in-class oral selective RARα agonist in RARA-positive patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide in patients with acute promyelocytic leukemia; and SY-5609, a highly selective and potent oral CDK7 inhibitor in patients with select solid tumors. Syros also has multiple preclinical and discovery programs in oncology and monogenic diseases.

Fulcrum Therapeutics develops treatments to improve the lives of patients with genetically defined rare diseases. The company raised $44 million in an underwritten public offering of 4 million shares of its common stock at a public offering price of $11.00 per share, before deducting underwriting discounts and commissions. In addition, Fulcrum granted the underwriters a 30-day option to purchase up to 600,000 additional shares of its common stock at the public offering price.

Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and phase 3 for the treatment of COVID-19. Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia, into phase 1 clinical development.