PALO ALTO, Calif. - BridgeBio Pharma, Inc. (NASDAQ: NASDAQ:BBIO), a biopharmaceutical company focusing on genetic diseases, announced the completion of enrollment for its Phase 3 FORTIFY study of BBP-418, targeting the treatment of Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). The study reached its enrollment target on the 10th annual LGMD Awareness Day, with a topline data readout from the interim analysis expected in 2025.
LGMD2I/R9 is caused by mutations in the fukutin-related protein (FKRP) gene, leading to progressive muscle wasting and issues with mobility, respiratory function, and cardiac health. Currently, no approved treatments exist for LGMD2I/R9, leaving individuals with few options to manage their condition.
BBP-418, an investigational oral therapy, has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA, along with Orphan Drug Designation from the European Medicines Agency (EMA). The FORTIFY study is a randomized, double-blind, placebo-controlled trial that includes a 12-month interim analysis focusing on glycosylated alpha-dystroglycan (αDG) as a surrogate endpoint, which could potentially support an application for Accelerated Approval in the U.S.
Douglas Sproule, M.D., M.Sc., Chief Medical Officer of ML Bio Solutions, a BridgeBio company, expressed optimism regarding discussions with the FDA about the opportunity to pursue Accelerated Approval. Kelly Brazzo, CEO of the CureLGMD2i Foundation, highlighted the rapid enrollment as a sign of the significant need for a therapeutic option.
If approved, BBP-418 could become the first disease-modifying therapy for LGMD2I/R9 in the U.S. BridgeBio may also qualify for a Priority Review Voucher based on the Rare Pediatric Designation from the FDA.
This announcement is based on a press release statement and does not suggest any endorsement of the claims. The success of BBP-418 and its approval potential are subject to the outcomes of ongoing clinical trials and regulatory review processes.
In other recent news, BridgeBio Pharma reported noteworthy updates in its drug development pipeline. The company received Breakthrough Therapy Designation from the FDA for infigratinib, an oral drug candidate for children with achondroplasia, based on the PROPEL 2 clinical trial results. In parallel, the company announced the discontinuation of its BBP-631 gene therapy program, an action projected to save over $50 million in research and development.
Simultaneously, BridgeBio's gene therapy BBP-812, developed for Canavan disease, was granted Regenerative Medicine Advanced Therapy designation by the FDA, following positive results from the CANaspire Phase 1/2 trial. Furthermore, the company formed a joint venture named GondolaBio, backed by a $300 million investment from a consortium of investors, aiming to expedite the development of new therapies.
Analyst firms, including BMO Capital, H.C. Wainwright, Piper Sandler, Oppenheimer, Mizuho, and TD Cowen, have maintained positive ratings for BridgeBio. These endorsements follow recent developments in the company's investigational drug, acoramidis, and the expected release of phase 3 data for encaleret and infigratinib.
The company is also seeking approval for Acoramidis, another treatment for ATTR-CM, with the FDA potentially convening an Advisory Committee to deliberate on this drug. These recent developments highlight BridgeBio Pharma's active engagement in drug development and regulatory processes.
InvestingPro Insights
BridgeBio Pharma's (NASDAQ: BBIO) recent milestone in its FORTIFY study for LGMD2I/R9 treatment aligns with some interesting financial insights from InvestingPro. The company's market capitalization stands at $4.69 billion, reflecting investor confidence in its potential.
InvestingPro data shows that BridgeBio's revenue for the last twelve months as of Q2 2023 was $219.12 million, with an impressive revenue growth of 3761.22% over the same period. This substantial growth could be attributed to the company's progress in its drug development pipeline, including BBP-418.
However, it's important to note that BridgeBio is not yet profitable, with an operating income of -$502.99 million for the last twelve months. This is not unusual for biopharmaceutical companies in the development stage, as they often incur significant research and development expenses before bringing products to market.
InvestingPro Tips highlight that analysts anticipate sales growth in the current year, which aligns with the company's advancement in clinical trials. Additionally, BridgeBio's liquid assets exceed short-term obligations, providing financial flexibility as it progresses through costly clinical trials.
For investors seeking a deeper understanding of BridgeBio's financial position and growth prospects, InvestingPro offers 5 additional tips that could provide valuable insights into the company's potential.
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