- California-based BridgeBio Pharma has snapped up ALXN1101 from Alexion Pharmaceuticals and built a new subsidiary, Origin Biosciences, around the synthetic enzyme cofactor.
- ALXN1101, also known as cyclic pyranopterin monophosphate, is in Phase 2/3 development for molybdenum cofactor deficiency Type A, an ultra-rare autosomal recessive inborn error of metabolism.
- Details of the deal have not been disclosed, but BridgeBio has committed to supply Origin with sufficient capital to support clinical development of ALXN1101 through potential regulatory approval and commercialization. Alexion will receive development and sales milestone payments.
There are no approved therapies for molybdenum cofactor deficiency (MoCD) Type A, caused by defective production of cyclic pyranopterin monophosphate (cPMP). Symptoms begin around birth, with feeding issues and seizures, and treatment is generally palliative, with death at around three years. ALXN1101 is a synthetic form of cPMP, and received Breakthrough Therapy Designation from the Food and Drug Administration in 2013.
"Historically, replacing missing or defective proteins has proven highly efficacious for treating loss of function monogenic conditions – in the case of MoCD type A, we are replacing the missing or defective cPMP, providing children with much needed MoCD activity," said Michael Henderson, SVP of asset acquisition at BridgeBio, in a statement.
In a streamlining of its R&D focus around core therapeutic area in July 2017, Alexion announced that it would out-license ALXN1101, as well as end collaborations with three partners.
BridgeBio's focus is on serious genetic disorders, creating a new subsidiary for each of its leads. These are supported by shared resources, creating what the company describes as an "efficient, decentralized corporate structure."
As well as the newly-created Origin Biosciences, BridgeBio's subsidiaries include: Eidos Therapeutics, with AG10 in Phase 1 for TTR amyloidoisis (ATTR); Navire Pharma, targeting SHP2 in rare and difficult-to-treat cancers; QED Therapeutics, with Novartis' infigratinib in Phase 2-for FGFR driven disorders including cancer; PellePharm, creating a topical treatment in Phase 2/3 for Gorlin syndrome and basal cell carcinoma; and Phoenix Tissue Repair, working on a protein replacement therapy for dystrophic epidermolysis bullosa.