Drug Research
Drug Discovery & Development

BridgeBio Pharma to acquire late stage therapy for ultra-rare disorder from Alexion

Published 12 June 2018

BridgeBio Pharma has entered into an agreement with Alexion Pharmaceuticals to acquire cyclic pyranopterin monophosphate (cPMP; ALXN1101), a synthetic enzyme co-factor therapy for patients with the ultra-rare disease caused by molybdenum cofactor deficiency (MoCD) Type A.

In addition, BridgeBio announced that it was launching a new subsidiary, Origin Biosciences, with sufficient capital to support clinical development of ALXN1101 through potential regulatory approval and commercialization.

MoCD is an ultra-rare autosomal recessive inborn error of metabolism. The disease is caused by a mutation in the MOCS1 gene and leads to defective production of cPMP. Clinical signs of MoCD present shortly after birth and progress rapidly.

Newborns with MoCD experience difficulty feeding and intractable seizures yet have no approved available therapies. Patients have a median survival of three years, and those who survive often have severe and irreversible injury to their central nervous system.

BridgeBio asset acquisition senior vice president Dr Michael Henderson said: "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.

BridgeBio's team is committed to continuing the development of ALXN1101 for infants born with MoCD Type A deficiency, their families and caregivers."

ALXN1101 is a synthetic version of cPMP, the missing cofactor causing MoCD Type A. In previous work with a recombinant form of cPMP, 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 US FDA.

BridgeBio CEO Dr Neil Kumar said: "Patients born with MoCD face a bleak future, and we will do all we can to pursue the development of this exciting compound, which has the potential to replace the missing enzyme.”

While specific terms of the deal have not been disclosed, BridgeBio 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.

Source: Company Press Release