RARE Daily

Fulcrum Raises $75 Million in Public Offering to Advance Rare Disease Pipeline

August 16, 2022

Fulcrum Therapeutics, a company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need, raised $75 million in a public offering.

The company sold 9.6 million shares of its common stock at $7.82 per share. In addition, Fulcrum has granted the underwriters a 30-day option to purchase up to an additional 1.4 million of its common stock at the public offering price, less the underwriting discount and commissions.

Fulcrum’s proprietary product engine, FulcrumSeek, identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. Fulcrum’s most advanced programs are losmapimod, which is being studied in a phase 3 clinical trial designed to evaluate its safety and efficacy for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a rare, serious, progressive, and debilitating disease that is caused by the aberrant expression of the DUX4 gene, which leads to skeletal muscle cell death and fat infiltration. Patients with FSHD experience progressive muscle weakness leading to significant impairment in function, including the inability to use their upper limbs, communicate via facial expression, and walk unassisted. The pivotal trial is designed to support the U.S. FDA and European Medicines Agency (EMA) regulatory applications of lospmapimod for the treatment of FSHD. If approved, losmapimod would be the first and only therapy for the disease.

In mid-stage studies, Fulcrum’s second lead candidate FTX-6058 is an experimental a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia.  Results from the multiple ascending dose portion of the trial, reported one year ago, demonstrated proof of biology as evidenced by a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes). At 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively. The increases in F-reticulocytes indicate that the HBG mRNA increases observed with FTX-6058 treatment are translating to HbF protein production.

Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The result of the mutation is less efficient oxygen transport and the formation of red blood cells that have a sickle shape. These sickle shaped cells are much less flexible than healthy cells and can block blood vessels or rupture cells. SCD patients typically suffer from serious clinical consequences, which may include anemia, pain, infections, stroke, heart disease, pulmonary hypertension, kidney failure, liver disease and reduced life expectancy.

Author: Rare Daily Staff

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