Through an agreement with Alexion Pharmaceuticals, BridgeBio Pharma acquired a synthetic enzyme replacement therapy for an ultrarare, fatal genetic disease that causes seizures in infants.
Development and commercialization of ALXN1101, a cyclic pyranopterin monophosphate, will be headed up by a new BridgeBio subsidiary, Origin Biosciences, based in Palo Alto, California.
The disease, molybdenum cofactor deficiency type A, is an autosomal recessive inborn error of metabolism caused by a mutation in the MOCS1 gene. Clinical signs become apparent shortly after birth and progress rapidly, with a median survival of three years and severe central nervous system injuries.
ALXN1101, a synthetic version of the missing protein that causes the disease, received an FDA Breakthrough Therapy designation in 2013. It is being studied in a phase 2/3 open-label study of about five patients, which is expected to collect final data in September.
In previous work with a recombinant formulation, 11 patients with MoCD type A had normalization of biomarkers within two days, while eight patients showed some suppression of seizures, and three patients had near-normal development, according to BridgeBio.
“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,” Michael Henderson, BridgeBio’s senior VP of asset acquisition, said in a statement.
Specific terms of the deal have not been disclosed, though BridgeBio said it has committed “sufficient resources” to support Origin through commercialization. Alexion will receive additional payments based on development and sales milestones for the drug, which it originally acquired from Orphatec for $3 million.
Some of Origin’s leadership carries over from its BridgeBio parent and subsidiaries, including Henderson and Carl Dambkowski, M.D., in business development, and Chief Scientific Officer Uma Sinha, Ph.D. Clinical operations will be led by Ramsey Johnson, who previously served as a senior director at X4 Pharmaceuticals.
Late last year, BridgeBio raised $135 million for research and development in genetic diseases. Using a “hub and spoke” corporate structure, the parent company has launched 10 drug programs under dermatology, oncology, cardiology, neurology and endocrine business units.
“In particular, we work to identify promising, but often overlooked, early-stage innovation that can translate into drugs that matter for patients,” Neil Kumar, Ph.D., BridgeBio’s co-founder and CEO, said at the time.