BridgeBio Pharma Surpasses Interim Analysis Enrollment Target and Receives U.S. FDA Rare Pediatric Disease Designation for BBP-418, a Potential Treatment for Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
BridgeBio Pharma has exceeded its targeted enrollment for the interim analysis in its Phase 3 FORTIFY study of BBP-418, aimed at treating LGMD2I/R9. The company expects top-line results in 2025. The study is randomized, double-blind, and placebo-controlled, evaluating the safety and efficacy of BBP-418. The study includes an interim analysis at 12 months, focusing on glycosylated αDG levels, which may be used as a surrogate endpoint for accelerated approval.
Recent FDA interactions affirm the potential for accelerated approval based on these surrogate endpoints. Additionally, the FDA has granted Rare Pediatric Disease Designation for BBP-418, which could qualify BridgeBio for a Priority Review Voucher if BBP-418 is approved. This designation highlights the seriousness of LGMD2I/R9, a disease primarily affecting children. The study continues to enroll participants in the U.S., UK, Europe, and Australia.
- BridgeBio has surpassed the interim analysis enrollment target for the Phase 3 FORTIFY study.
- Top-line results from the interim analysis are expected in 2025.
- FDA interactions support the potential for accelerated approval based on glycosylated αDG levels.
- The FDA granted Rare Pediatric Disease Designation for BBP-418.
- Potential qualification for a Priority Review Voucher if BBP-418 is approved.
- The FORTIFY study is enrolling participants globally, including in the U.S., UK, Europe, and Australia.
- Top-line results from the interim analysis will not be available until 2025, indicating a delay in potential market approval.
- High dependency on surrogate endpoints like glycosylated αDG levels for accelerated approval, which may introduce regulatory risks.
Insights
BridgeBio Pharma has made significant progress in its Phase 3 FORTIFY study for BBP-418, a treatment for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Surpassing the interim analysis enrollment target is a positive milestone and indicates strong participation and interest in the trial. Additionally, the U.S. FDA Rare Pediatric Disease Designation shines a spotlight on the urgency and seriousness of this condition, primarily affecting children.
Notably, the use of a novel, validated glycosylated alpha-dystroglycan (αDG) bioassay as a surrogate endpoint is a important aspect here. These surrogate endpoints are used when there is a need to expedite drug approval processes by using early markers that correlate with clinical outcomes. In this case, measuring glycosylated αDG levels can provide an early indication of the drug's efficacy, potentially allowing for Accelerated Approval.
In the short-term, this news demonstrates that BridgeBio is on track with its clinical trial milestones, which can be seen as a positive signal for investors. The RPDD also opens doors for priority review vouchers, which are valuable assets that can be used or sold, providing financial and strategic flexibility.
However, investors should be prepared for the inherent uncertainties in clinical trials and the regulatory approval process. While the interim results expected in 2025 might be promising, the final outcomes and market approval are still pending.
The announcement highlights several favorable factors for BridgeBio Pharma’s market position. Surpassing the enrollment target for the Phase 3 FORTIFY study demonstrates robust operational execution and patient engagement, which are critical success factors for any clinical-stage biopharmaceutical company.
The Rare Pediatric Disease Designation and the possibility of obtaining a Priority Review Voucher are particularly noteworthy. Priority Review Vouchers can significantly accelerate the drug approval process for future drugs, either within the company’s pipeline or through monetization by selling the voucher to another biopharmaceutical entity. This could provide a significant revenue stream or strategic advantage for BridgeBio.
From a market perspective, this news could enhance investor confidence and potentially drive the company's stock price upward in the short-term. However, it's essential to keep an eye on the top-line interim data expected in 2025, as these results will be pivotal for long-term valuation and market positioning. Given the targeted nature of the therapy in a niche and rare disease market, successful approval and commercialization could position BridgeBio as a leader in the genetic disease space.
Investors should also consider the competitive landscape, as advancements by other companies in similar therapeutic areas could influence market dynamics. The long-term impact will largely depend on the trial outcomes and subsequent regulatory decisions.
BridgeBio’s recent milestones may have significant financial implications. Surpassing the enrollment target early in a Phase 3 trial is typically a positive indicator of clinical feasibility and patient interest, both of which bode well for future developments. Additionally, the Rare Pediatric Disease Designation brings potential financial benefits through the Priority Review Voucher system. Such vouchers are valuable strategic assets, either for expediting the review of another candidate in BridgeBio's pipeline or through monetization by selling the voucher to other firms, which has historically fetched up to
Moreover, the focus on the glycosylated αDG bioassay suggests a well-validated pathway to potential accelerated approval, potentially shortening the time and cost to market. This accelerated path, if successful, could lead to earlier revenue generation compared to a traditional approval process.
However, investors should be mindful of the risks inherent in drug development. The interim analysis in 2025 will be a significant catalyst and any negative data could adversely affect the stock price. Accordingly, while the news is promising, the inherent volatility in biotech investments should be taken into account.
- BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025
- Recent Type C interactions with U.S. Food and Drug Administration (FDA) focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio's belief that there is potential to pursue Accelerated Approval for BBP-418
- Rare Pediatric Disease Designation for BBP-418 highlights that LGMD2I/R9 is a rare disease with serious manifestations, which primarily impacts children, and if BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher
PALO ALTO, Calif., June 18, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025.
FORTIFY is a randomized, double-blind, placebo-controlled Phase 3 study evaluating the safety and efficacy of BBP-418, an investigational oral therapy in development for the treatment of individuals living with LGMD2I/R9. The study includes a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to support Accelerated Approval. The primary endpoint to be evaluated at 36 months is the North Star Assessment (NSAD) for limb-girdle type muscular dystrophies and is designed to provide confirmatory clinical data. BridgeBio is continuing to enroll FORTIFY in the U.S., UK, Europe, and Australia. More information about the ongoing Phase 3 clinical trial of BBP-418 (NCT05775848) can be found here on clinicaltrials.gov.
Multiple encouraging discussions with the FDA in 2024, focused on the validated glycosylated αDG bioassay and our interim analysis plans, continue to support the Company’s plan to pursue Accelerated Approval for BBP-418 based on the use of glycosylated αDG levels as a surrogate endpoint in LGMD2I/R9. Furthermore, the Agency indicated that the Company’s approach to measure glycosylated αDG levels via a novel, validated muscle tissue-based bioassay appears reasonable. A peer-reviewed manuscript providing details on the novel, multiplexed, fluorescence-based Western Blot assay was recently published in the Journal of Muscle Research and Cell Motility.
“Reduced glycosylated αDG is the primary molecular driver of LGMD2I/R9, a serious, progressively debilitating disease that weakens the muscles causing many affected people to become fully dependent on a caregiver, while also threatening their cardiac and respiratory function,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions, a BridgeBio affiliate that is focused on developing BBP-418 for LGMD2I/R9. “We hope to continue to serve individuals through expeditious enrollment of the Phase 3 FORTIFY study and look forward to continuing to partner with the FDA to accelerate development of a potential new therapeutic option for people living with LGMD2I/R9.”
The Company also announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) for BBP-418 in the treatment of LGMD2I/R9, recognizing the rarity of this disease is characterized by serious manifestations primarily affecting children. If BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher based on receipt of the RPDD. A Priority Review Voucher can be applied to another therapy in the Company’s pipeline for a shorter timeline during the review process of a New Drug Application or can be sold and transferred to another company looking to receive priority review for one of its applications.
About Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
LGMD2I/R9 is a monogenic autosomal recessive disease caused by partial loss of function mutations in the fukutin-related protein (FKRP) gene, and FKRP mutations impair glycosylation of alpha-dystroglycan (αDG), a protein associated with stabilizing muscle cells. Clinical manifestations typically present as a skeletal myopathy affecting the lower and then upper limbs, which is commonly later accompanied by respiratory muscle and cardiac muscle involvement. Individuals who harbor a homozygous L276I genotype typically develop disease manifestations during late childhood with progression to loss of independent ambulation (
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements BridgeBio makes in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic and market potential of BridgeBio’s programs and product candidates, including BBP-418 for the treatment of LGMD2I/R9, the drug design and the potential benefits of BBP-418, including its potential to address serious unmet need for patients with LGMD2I/R9, the potential and the opportunity to pursue Accelerated Approval Pathway for BBP-418 in LGMD2I/R9 in the U.S., the potential BridgeBio may qualify for a Priority Review Voucher based on receipt of the RPDD, the expected timeline of announcing the topline data from the interim analysis of FORTIFY in individuals with LGMD2I/R9 in 2025, and the statements regarding the potential benefit of our clinical trial or of our product candidate in the quotes of Dr. Sproule; the progress, timeline and success of BridgeBio’s ongoing and planned clinical trials of BBP-418, the timeline of evaluation of the NSAD and secondary endpoints at 36 months, and the expectation of providing confirmatory clinical data from such results, the plans of engaging with regulatory authorities, including the collaboration and interaction with the FDA to address the challenges associated with LGMD2I/R9 drug development, among others, reflect BridgeBio’s current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio’s ability to continue and complete its ongoing and planned clinical trials of BBP-418 for the treatment of LGMD2I/R9, initial and ongoing data from its clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, difficulties with enrollment in our clinical trials, adverse events that may be encountered in our clinical trials, the FDA or other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, potential adverse impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on our business operations and expectations as well as those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K, and BridgeBio’s other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
BridgeBio Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220
FAQ
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