Four Biotechs Raise Almost $1 Billion to Advance Rare Disease Pipelines

Rare Daily Staff

Four public companies with a rare disease therapeutics focus tapped the market for almost $1 billion of combined capital to advance their pipelines.

Avidity Biosciences, which is focused on rare musculoskeletal dystrophies, raised $401 million in a public offering of 10.6 million shares of its common stock at $38.00 per share, before deducting underwriting expenses. In addition, Avidity granted the underwriters a 30-day option to purchase up to an additional 1.6 million shares of common stock.

Avidity recently reported positive initial phase 1/2 data from its experimental antisense oligonucleotide conjugate AOC 1020 to treat the rare muscle disease facioscapulohumeral muscular dystrophy (FSHD), which demonstrated reductions of greater than 50 percent in DUX4 regulated genes that drive the condition, trends of functional improvement, and favorable safety and tolerability.

Ultragenyx Pharmaceutical raised $350 million in an underwritten public offering of 7.4 million shares of its common stock at $39.00 per share, and in lieu of issuing common stock to certain investors, the company offered pre-funded warrants to purchase 1.5 million shares of its common stock at a purchase price of $38.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant. In addition, the company granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1.3 million shares of the company’s common stock at the public offering price, less the underwriting discount.

Ultragenyx just announced that it will file a BLA for accelerated approval of its experimental gene therapy UX111 for the treatment of Sanfilippo syndrome (MPSIIIA) after reaching agreement with the U.S. Food and Drug Administration that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint.

UX111 is a novel in vivo gene therapy in phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). UX111 is designed to be dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of heparan sulfate, a glycosaminoglycan, in the brain that results in progressive cell damage and neurodegeneration.

Disc Medicine, a company focused on treatments for serious hematologic diseases, raised $178 million in a private investment in public equity offering of 4.9 million shares of its common stock at $36.00 per share, before deducting underwriting discounts and commissions and other offering expenses.

Frazier Life Sciences and Logos Capital led the PIPE financing with participation from new and existing institutional investors, including Access Biotechnology, Adage Capital Partners, Atlas Venture, Janus Henderson Investors, OrbiMed, Paradigm BioCapital, TCGX, Wellington Management, a leading mutual fund, and others.

Disc’s lead experimental candidate, bitopertin, is a heme synthesis modulator in phase 2 development for the treatment of erythropoietic protoporphyria (EPP), a rare genetic metabolic disorder caused by a deficiency of the enzyme ferrochelatase (FECH), which results from mutations in the FECH gene. Due to abnormally low levels of this enzyme, excessive amounts of protoporphyrin accumulate in the bone marrow, blood plasma, and red blood cells. Some patients with symptoms of EPP have a genetic change in a different gene called ALAS2. When a patient has a genetic change in this gene, the condition is referred to as X-linked protoporphyria (XLP). Patients with these disorders experience severe pain on exposure to sunlight and some types of artificial light.

Finally, Rezolute raised $60 million in an underwritten public offering of 11.3 million shares of its common stock at $4.00 per share, and, to certain investors in lieu of common stock, pre-funded warrants to purchase up to 3.8 million shares of common stock at $3.999 per pre-funded warrant, which represents the per share offering price for the common stock less the $0.001 per share exercise price for each pre-funded warrant.

Rezolute’s lead antibody therapy RZ358 is in late-stage development to treat individuals with hypoglycemia caused by hyperinsulinism (HI). It is designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use for the treatment of congenital hyperinsulinism, a genetic disorder in which the insulin cells of the pancreas, secrete too much insulin, which causes low plasma sugar (hypoglycemia) or low blood sugar.