BridgeBio Pharma Acquires Late-Stage Therapy for Ultra-Rare Disorder from Alexion

Rare Daily Staff

BridgeBio Pharma said it has entered into an agreement with Alexion Pharmaceuticals to acquire ALXN1101, a synthetic enzyme co-factor therapy for patients with an ultra-rare and deadly metabolic condition molybdenum cofactor deficiency Type A.

BridgeBio said it will launch a new subsidiary, Origin Biosciences, to support clinical development of ALXN1101 through potential regulatory approval and commercialization.

Signs of MoCD become visible shortly after birth and progress rapidly. Newborns with MoCD have difficulty feeding and intractable seizures. Patients have a median survival of three years, and those who survive often have severe and irreversible injury to their central nervous system. There are no approved therapies for the condition.

ALXN1101 is a synthetic version of cyclic pyranopterin monophosphate, or cPMP, the missing cofactor causing MoCD Type A. In previous work with a recombinant form of the cofactor, 11 patients with MoCD Type A had normalization of biomarkers within two days, eight patients showed some suppression of seizures, and three patients had near-normal development. ALXN1101 has received Breakthrough Therapy designation from the U.S. Food and Drug Administration.

“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, senior vice president of asset acquisition at BridgeBio.

BridgeBio did not disclose terms of the deal. BridgeBio said it has committed sufficient resources to Origin Biosciences to enable clinical development, regulatory approval, and to support commercialization of ALXN1101. Alexion will receive additional payments upon the realization of development and sales milestones.

June 11, 2018
Photo: Michael Henderson, senior vice president of asset acquisition at BridgeBio