On March 8, 2022 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), reported the execution of an asset purchase agreement (the Agreement) for the sale of BridgeBio’s NULIBRY (Fosdenopterin) for Injection (Press release, BridgeBio, MAR 8, 2022, View Source [SID1234609754]). NULIBRY is approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening pediatric genetic disorder. The closing of the asset purchase is subject to customary closing conditions.

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"Sentynl’s focus on meaningful treatments for serious rare diseases is further enhanced by the acquisition of Fosdenopterin. We will leverage our existing platform of ultra-rare pediatric disease initiatives to facilitate early diagnosis and treatment by enhancing awareness, newborn screening, genetic testing and patient support across multiple products and rare diseases. By partnering with BridgeBio, we hope to reach even more patients born with MoCD Type A as quickly as possible with the hope of reducing the risk of mortality and progression of this devastating disease," said Matt Heck, CEO of Sentynl.

Under the Agreement, Sentynl will acquire global rights to NULIBRY and will be responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing and commercializing Fosdenopterin globally. BridgeBio will share development responsibilities for Fosdenopterin through approval of the marketing authorization application already under accelerated assessment with the European Medicines Agency and through approval of its regulatory submission with the Israeli Ministry of Health. Sentynl will provide cash payments upon the achievement of certain regulatory milestones. BridgeBio will be eligible to receive commercial milestone payments as well as tiered royalties on adjusted net sales of NULIBRY.

"Sentynl is an ideal partner given its expertise in the rare pediatric neurodevelopment space and its relationships with physicians who diagnose and treat children with MoCD Type A," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "Focused execution means reducing the scope of our internal activity. We will continue to advance high-quality programs in our pipeline while expanding our reach to patients in need of options."

About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity.1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.2

MoCD Type A is an ultra-rare disease. The incidence and prevalence of MoCD Type A in the United States are not known, but the estimated incidence is 1 per 342,000 to 411,000 live births (0.24 and 0.29 per 100,000).3 Based on these estimates, MoCD Type A is likely to be underdiagnosed, with an estimated 22 to 26 missed diagnoses per year in the United States and European Union.

The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

About NULIBRY (Fosdenopterin) for Injection
NULIBRY (Fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.