RARE Daily

Āshibio Raises $40 Million to Advance Therapies for Bone and Connective Tissue Disorders

June 20, 2024

Rare Daily Staff

Āshibio emerged from stealth mode with $40 million in seed and series A financing to advance therapies for bone and connective tissue disorders.

The company was founded in 2022 by CEO Pankaj Bhargava, and the team at MPM BioImpact, where Bhargava is also an entrepreneur partner.

MPM BioImpact led the series A round, with contributing funds from Agent Capital, YK Bioventures, and Mirae Asset Venture Investment. In conjunction with the financing, Agent Capital Partner and Co-Founder Preston Noon has joined the Board of Directors.

The funding will allow Āshibio to continue to advance development of several investigative therapies including a potential treatment for fibrodysplasia ossificans progressiva (FOP), a rare genetic disease characterized by severe and progressive heterotopic ossification, a pathological condition that causes abnormal bone formation in muscles, tendons, ligaments and other soft tissues. FOP typically starts in early childhood, is characterized by episodic and painful flare-ups, and leads to severe debilitation and shortened life span.

“We are building a pipeline of innovative therapeutics that leverage emerging biology and novel mechanisms to impact bone and connective tissue disorders that have no approved treatments or where current treatment options are inadequate,” said Bhargava.

The company’s lead program is based on the discovery that the matrix metalloproteinase-9 (MMP-9) enzyme could serve as a novel target for FOP.

Research, published in the Journal of Bone and Mineral Research in February 2024, described the case of a unique 35-year-old patient who carries the classic genetic mutation of FOP, ACVR1 R206H, which is present in over 95 percent of patients, yet has extreme lack of heterotopic ossification (HO) and near normal mobility.

The authors found that the patient has an MMP-9 mutation in addition to the FOP mutation that appears to protect him from flare-ups and the abnormal bone formation typically seen in individuals with FOP. Further studies with MMP-9 gene knockout and pharmacological experiments confirmed that MMP-9 may serve as a novel target in FOP and other more common forms of HO.

Āshibio licensed andecaliximab, a humanized antibody that specifically inhibits MMP-9, from Gilead Sciences. Andecaliximab has been administered to approximately 1,000 patients in previous clinical trials.

The company plans to initiate a phase 2/3 trial of andecaliximab in the second half of 2024 in patients with FOP. The U.S. Food and Drug Administration designated andecaliximab an Orphan Drug for the treatment of FOP in March 2024, one month after the European Medicines Agency issued its own Orphan Drug designation for the therapy. The FDA also cleared Āshibio’s Investigational New Drug application for andecaliximab in March 2024.

Prior to joining MPM BioImpact, Bhargava was the Oncology Therapeutic Area head at Gilead Sciences. He has also held executive positions at multiple biotech companies during his career, including Sanofi and Dicerna Pharmaceuticals, where he led development programs for oncology and rare genetic diseases.

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