āshibio raises $40M to turn an ex-Gilead antibody into rare disease treatment
Orphan DrugExecutive ChangeGene Therapy
Six years after Gilead Sciences terminated a late-stage gastric cancer study of andecaliximab, the antibody is getting a new lease on life as an experimental treatment for a rare genetic disease.
āshibio, helmed by Gilead’s former oncology therapeutic area head Pankaj Bhargava, emerged from stealth Thursday with $40 million in seed and series A financing to develop andecaliximab for fibrodysplasia ossificans progressiva (FOP), a disorder that causes extra-skeletal bone formation.
The funds will help the biotech launch a Phase II/III trial of andecaliximab in patients with FOP next half. The antibody has been granted an orphan drug designation by both the FDA and European Medicines Agency.
MPM BioImpact led the A round, with Agent Capital, YK Bioventures, and Mirae Asset Venture Investment also participating.
Genetic rationale
While andecaliximab may have failed as a cancer treatment, āshibio believes the antibody has potential for FOP because a mutation in the gene that encodes its target – the matrix metalloproteinase-9 (MMP-9) enzyme – has been tied to an amelioration of the disease’s severe symptoms.
Bhargava co-authored a paper, published February in the Journal of Bone and Mineral Research, that examined a 35-year-old patient with the genetic mutation for FOP, but had near-normal mobility and lacked the heterotopic ossification (HO) associated with the disease. The patient also had an MMP-9 mutation, and the authors theorised that this genetic change may have protected the patient from developing the disease’s characteristic abnormal bone formations.
The āshibio team conducted additional MMP-9 gene knockout and pharmacological studies that suggested the enzyme is an attractive target for FOP, as well as other common forms of HO.
The company will give further updates on its andecaliximab FOP programme at two scientific meetings this month: the FOP Drug Development Forum and the International Conference on Children’s Bone Health.
New hires
Meanwhile, āshibio also announced two new members of its C-suite on Wednesday. Victoria Smith joins as chief scientific officer (CSO), and Deborah Wenkert comes on board as chief medical officer (CMO).
Most recently, Smith was CSO at Amphivena Therapeutics, and earlier she served as senior director of biology at Gilead, where she was a co-inventor of andecaliximab.
A paediatric rheumatologist, Wenkert has previously held the role of CMO at both Inozyme Pharma and PreciThera. She also had a five-year stint as the medical director for clinical research at Amgen.
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