BridgeBio Pharma, Inc. develops and commercializes medicines for genetic conditions through a decentralized biopharmaceutical model. News for BBIO centers on Attruby/acoramidis, its oral transthyretin stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM), including clinical data from ATTRibute-CM, commercial updates, Brazil marketing authorization under the BEYONTTRA name, and licensing or commercialization arrangements.
Company updates also cover late-stage genetic-disease programs such as encaleret for autosomal dominant hypocalcemia type 1, BBP-418 for limb-girdle muscular dystrophy type 2I/R9, and oral infigratinib for achondroplasia. Recurring financial and corporate items include quarterly results, product revenue trends, capital actions, employee equity inducement grants, investor conference participation, and pipeline regulatory disclosures.
BridgeBio Pharma (NASDAQ: BBIO) reported positive interim Phase 2 data for infigratinib, showing a mean increase in annualized height velocity (AHV) of 1.52 cm/year in Cohort 4 children aged 5 and older. The company announced a Phase 3 trial for encaleret in autosomal dominant hypocalcemia type 1 (ADH1) and selected a next-generation KRAS G12C dual inhibitor. Financial results revealed a net loss of $9.9 million for Q2 2022, down from $96.3 million a year prior, with $688.6 million in cash and equivalents. The company continues to show robust pipeline momentum.
BridgeBio Pharma announced positive interim results from its Phase 2 trial, PROPEL 2, for infigratinib in children with achondroplasia. At the highest dose (0.128 mg/kg), the mean annualized height velocity (AHV) increased by 1.52 cm/yr (p=0.02, n=11), with a 64% responder rate among participants aged 5 and older. Infigratinib demonstrated a favorable safety profile, with no serious adverse events reported. The company has initiated dosing in Cohort 5 (0.25 mg/kg) after discussions with regulators and aims to present full results by mid-2023.
The CHMP has recommended the approval of NULIBRY for treating molybdenum cofactor deficiency (MoCD) Type A in the EU. This decision is based on robust efficacy and safety data. If approved by the European Commission later this year, NULIBRY will be the first and only therapy for this ultra-rare genetic disorder, which affects fewer than 150 patients globally, with a median survival age of four years. NULIBRY was previously approved by the FDA in 2021 and has shown an 86% reduction in mortality risk in clinical trials compared to historical controls.
BridgeBio Pharma (Nasdaq: BBIO) has partnered with UC Berkeley's Bakar Labs to foster innovation in genetic disease and cancer treatments. This collaboration will support up to 50 startups, leveraging world-class facilities and expertise. CEO Neil Kumar sees the partnership as a way to strengthen the Bay Area biotech ecosystem and expedite the clinical development of new therapies. Bakar Labs aims to facilitate groundbreaking research and shared resources for entrepreneurs, enhancing progress in potential therapies for genetically-driven conditions.
BridgeBio Pharma (Nasdaq: BBIO) announced a collaboration with Baylor College of Medicine on July 21, 2022, to develop therapies for genetic diseases. This partnership aims to leverage Baylor's research capabilities and BridgeBio's drug development expertise to translate innovative findings into practical treatments for patients with unmet medical needs. The collaboration underscores BridgeBio's commitment to fostering meaningful academic partnerships in the biopharmaceutical industry.
BridgeBio Pharma (BBIO) announced positive Phase 1 data for BBP-711, a novel oral therapy aimed at treating primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formation. Results indicated near complete inhibition of glycolate oxidase, significantly increasing plasma glycolate levels. Following its promising tolerability and efficacy, BridgeBio plans to initiate a pivotal Phase 2/3 study by the end of 2022, as well as a Phase 2 study targeting adult recurrent kidney stone formers, which impacts 1.5 million individuals in the U.S. and EU.
BridgeBio Pharma announced promising results from the Phase 1/2 clinical trial of BBP-812, a gene therapy for Canavan disease. Initial data from two participants revealed significant decreases in N-acetylaspartate (NAA) levels in the brain and urine, indicating the therapy may effectively target disease markers. With no serious adverse events reported, the trial is progressing well. If successful, BBP-812 could be the first treatment option for this ultra-rare, fatal condition affecting approximately 1,000 children in the U.S. and EU.
BridgeBio Pharma announced positive Phase 2b results for encaleret, targeting autosomal dominant hypocalcemia type 1 (ADH1). By day 5 of treatment, participants showed restored normal calcium homeostasis, with 92% achieving normal trough blood calcium levels. The company is scheduling a Phase 3 study to commence in 2022, assessing encaleret against standard care. The results are presented at ENDO 2022, and an investor call is scheduled for June 13, 2022, to discuss findings and future plans.
BridgeBio Pharma (Nasdaq: BBIO) and Venthera, Inc. presented preliminary data from the Phase 1b trial of VT30 topical gel, targeting venous and lymphatic lesions. The trial showed that VT30 was generally well-tolerated across 15 participants, with mild to moderate local rashes as the only notable adverse effect. Importantly, pharmacokinetic data indicated drug concentrations were sufficient within lesions, while no systemic drug was detected in plasma. Venthera plans to advance the 0.6% gel into further studies and is seeking development partners.
BridgeBio Pharma (BBIO) announced that preliminary data from a 24-week Phase 2b study of encaleret in treating autosomal dominant hypocalcemia type 1 (ADH1) will be presented at the Endocrine Society's Annual Conference on June 13, 2022. This data suggests that encaleret restores mineral homeostasis, marking a significant step in clinical research. Alongside this, comprehensive presentations on various programs, including achondroplasia and congenital adrenal hyperplasia, will also be discussed. An investor call is slated for the same day to focus on the Phase 2b data.