Bridgebio Pharma - BBIO STOCK NEWS

BridgeBio Pharma presented positive preliminary data from eleven participants in the CANaspire Phase 1/2 clinical trial of BBP-812, an investigational gene therapy for Canavan disease. The study showed significant motor function improvements and milestone achievements at 12-months post-treatment, contrasting with the natural disease progression observed in their CANinform study.

Key findings include sustained reductions in N-acetylaspartate levels across urine, cerebrospinal fluid, and brain, with urine NAA reduced by 64% in low dose and 73% in high dose cohorts. The therapy demonstrated improved myelination and continued motor function progress, with some children achieving independent sitting and walking. BBP-812 was generally well-tolerated and has received multiple FDA designations including RMAT, Orphan Drug, and Fast Track.

BridgeBio Pharma (Nasdaq: BBIO) announced that outcomes data through 42 months from the open-label extension of ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be presented at the 2024 AHA Scientific Sessions. The featured science oral presentation, titled 'Acoramidis Reduces All-Cause Mortality (ACM) and Cardiovascular-Related Hospitalization (CVH): Initial Outcomes from the ATTRibute-CM Open-Label Extension (OLE) Study', will be delivered by Dr. Daniel Judge on November 18.

Additionally, BridgeBio will present three moderated digital posters on ATTR-CM, covering topics such as healthcare costs, baseline risk evolution, and acoramidis' impact on survival. These presentations will take place on November 17. The presentation and posters will be available on BridgeBio's website after the sessions.

BridgeBio Pharma announced the completion of enrollment for its Phase 3 FORTIFY study evaluating BBP-418 in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). The study exceeded its target enrollment, with topline data expected in 2025. BBP-418 could potentially receive Accelerated Approval in the U.S. based on the biomarker glycosylated alpha-dystroglycan (αDG). If successful, BBP-418 would be the first approved therapy for LGMD2I/R9 in the U.S. The study's primary endpoint is the North Star Assessment for limb-girdle type muscular dystrophies, evaluated at 36 months. BBP-418 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the EMA. The completion announcement coincided with the 10th annual LGMD Awareness Day on September 30, 2024.

BridgeBio Pharma (Nasdaq: BBIO) has announced that the FDA granted Breakthrough Therapy Designation to oral infigratinib for children with achondroplasia. This designation, based on preliminary clinical evidence from the PROPEL 2 trial, is designed to expedite development and regulatory review. In Cohort 5 of PROPEL 2, infigratinib showed a statistically significant increase in annualized height velocity (AHV) and improved body proportionality. The global Phase 3 study, PROPEL 3, is enrolling on schedule. If approved, infigratinib could become the first-in-class oral therapeutic option for children with achondroplasia, potentially offering families greater control and choice in treatment options.

BridgeBio Pharma (Nasdaq: BBIO) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BBP-812, its gene therapy program for Canavan disease. This designation is based on promising clinical evidence from the CANaspire Phase 1/2 trial, which showed functional improvements in all dosed patients. RMAT status offers benefits like faster and more frequent FDA interactions, potentially accelerating the approval process.

Key points:

• BBP-812 is an intravenous AAV9 gene therapy for Canavan disease
• All patients in the trial showed improvements in key functional areas
• The therapy has been well-tolerated with a safety profile consistent with other AAV9 gene therapies
• BBP-812 also has Orphan Drug, Rare Pediatric Disease, and Fast Track Designations

If approved, BBP-812 could be the first treatment option for this fatal neurodevelopmental disorder.

BridgeBio Pharma (Nasdaq: BBIO) announced topline results from its Phase 1/2 ADventure study of BBP-631, an investigational gene therapy for congenital adrenal hyperplasia (CAH). The study showed increased endogenous cortisol production in all patients at higher doses, with a maximum change from baseline of 6.6 μg/dL. The therapy was well-tolerated with no treatment-related serious adverse events. Despite these advancements, BridgeBio decided the data do not warrant additional capital investment. The company is reducing its gene therapy budget by over $50M and seeking partnership opportunities for future development of BBP-631 or next-generation CAH gene therapies.

BridgeBio Pharma (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases, has announced the approval of equity grants for 123 new employees. The grants, approved on September 4, 2024, consist of restricted stock units totaling 359,926 shares of the Company's common stock. The vesting schedule begins with one-fourth of the shares vesting on August 16, 2025, followed by quarterly vesting of one-twelfth of the remaining shares, subject to continued employment. These awards were made under BridgeBio's Amended and Restated 2019 Inducement Equity Plan, in accordance with Nasdaq Listing Rule 5635(c)(4), as inducement material for new employees joining the Company.

BridgeBio Pharma (Nasdaq: BBIO) presented additional data from its Phase 3 ATTRibute-CM and open-label extension study of acoramidis in ATTR-CM at ESC 2024. Key findings include:

1. Participants switching from tafamidis and placebo to acoramidis showed a mean increase in serum TTR of 3.0mg/dL at Month 1 and 3.4mg/dL at Month 6 of the OLE.

2. Increased serum TTR at Day 28 correlated with reduced risk of all-cause mortality, cardiovascular mortality, and cardiovascular-related hospitalization.

3. Acoramidis demonstrated a 42% reduction in composite CVH and ACM events and a 50% reduction in cumulative CVH events at Month 30.

BridgeBio has submitted regulatory applications for acoramidis, with FDA PDUFA date set for November 29, 2024, and EMA decision expected in 2025.

BridgeBio Pharma (Nasdaq: BBIO) announced that additional data from its Phase 3 ATTRibute-CM study of acoramidis in ATTR-CM will be presented at two major conferences. At the ESC Congress 2024 in London (Aug 30 - Sep 2), Dr. Mathew S. Maurer will present a moderated poster on the increase in serum TTR levels observed with acoramidis treatment. At the HFSA Annual Scientific Meeting 2024 in Atlanta (Sep 27-30), Dr. Daniel P. Judge will give an oral presentation on how acoramidis improves clinical outcomes in ATTR-CM patients, based on a post hoc recurrent event analysis. These presentations aim to provide further insights into the efficacy of acoramidis in treating transthyretin amyloid cardiomyopathy.