HemoShear Raises $40 Million to Advance Rare Disease Portfolio

Rare Daily Staff

HemoShear Therapeutics said it raised $40 million in a series A financing to advance development of its pipeline of treatments for rare metabolic disorders.

Suvretta Capital led the financing along with Janus Henderson Investors, Adage Capital Management, and other private investors.

“This financing will not only advance our treatment for methylmalonic acidemia (MMA) and propionic acidemia (PA), but also help us expand our pipeline of other programs for rare diseases with high unmet need,” said Jim Powers, chairman and CEO of HemoShear.

MMA and PA are rare genetic disorders caused by the deficiency of certain enzymes required to metabolize amino acids. MMA and PA are diagnosed through newborn screening in the United States, and select countries in Europe, the Middle East, and the rest of the world. The diseases result in the buildup of toxic metabolites that can lead to frequent metabolic decompensations, severe organ damage, seizures, developmental deficits, and premature death. In the United States, about 1 in 70,000 newborns is diagnosed with MMA and 1 in 240,000 is diagnosed with PA. Both diseases are more common in the Middle East and North Africa. There are an estimated 4,000 MMA and PA patients in the U.S. and Europe combined.

The funding will enable the company to complete a phase 2 study of its lead compound, HST5040, for the treatment of MMA and PA. HST5040 is an experimental oral small molecule therapy developed by HemoShear to correct metabolic abnormalities associated with MMA and PA. Because HST5040 is a small molecule, it has the ability to distribute to multiple affected tissues and thus has the potential to be active throughout the body, including the liver, kidney, brain, heart and muscles. HST5040 is designed for convenient daily administration at home as a liquid formulation taken either orally or through a gastric feeding tube.

The U.S. Food and Drug Administration has granted HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations to treat MMA and PA. More information about the HERO study can be found on clinicaltrials.gov.

“With HST5040 about to enter the clinic and successful drug discovery partnerships with Takeda in NASH and Horizon in gout, HemoShear has demonstrated the broad utility of their REVEAL-Tx platform to model human disease and identify novel drug targets,” said David Friedman, managing director at Suvretta Capital.

In conjunction with the financing, Friedman and John Tilton will join the board of HemoShear. Friedman has been a biopharma analyst and investor for 15 years with a focus on rare disease companies. Tilton is currently the chief commercial officer at Biohaven Pharmaceutical and was a founding commercial leader at Alexion Pharmaceuticals. He has more than 25 years of experience successfully commercializing bio-pharmaceutical products, launching start-up biotech companies as well as raising capital in private and public markets.

Photo: Jim Power, chairman and CEO of Hemoshear