BridgeBio's Attruby Sees Strong Early Demand; Phase 3 Trials Fully Enrolled
核心洞察
BridgeBio's Attruby (acoramidis) has demonstrated remarkable early demand, with 430 prescriptions written by 248 unique healthcare providers since FDA approval.
The company has fully enrolled three major market Phase 3 clinical trials: FORTIFY (BBP-418 for LGMD2I/R9 (搜索)), CALIBRATE (encaleret for ADH1 (搜索)), and PROPEL 3 (infigratinib for Achondroplasia (搜索)).
BridgeBio is well-financed to launch Attruby and anticipates $105M in regulatory milestones in the first half of 2025 from potential approvals in Europe and Japan.
BridgeBio Pharma, Inc. (Nasdaq: BBIO) has announced significant commercial progress with its newly approved drug Attruby (acoramidis) and provided updates on its late-stage pipeline programs. The company also shared anticipated milestones for 2025, highlighting a focus on genetic diseases.
Attruby's Promising Start
Following FDA approval on November 22, 2024, Attruby, a near-complete TTR stabilizer, has seen rapid adoption, with 430 prescriptions written by 248 physicians across various healthcare settings. Attruby is indicated for reducing cardiovascular death and cardiovascular-related hospitalization in adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM (搜索)).
Neil Kumar, Ph.D., Founder and CEO of BridgeBio, expressed gratitude for the enthusiasm surrounding Attruby and noted the initial commercial momentum. He also highlighted the completion of enrollment in three major Phase 3 clinical trials.
Pipeline Advancements
BridgeBio's pipeline continues to advance, with key updates on several programs:
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BBP-418 for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9 (搜索)): The FORTIFY Phase 3 trial, evaluating BBP-418, a glycosylation substrate, in 112 patients with LGMD2I/R9, is fully enrolled. Topline results from the interim analysis cohort are expected in the second half of 2025. If successful, BBP-418 could be the first approved therapy for this rare genetic disorder.
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Encaleret for Autosomal Dominant Hypocalcemia Type 1 (ADH1 (搜索)): The CALIBRATE Phase 3 trial, assessing encaleret, a calcium-sensing receptor (CaSR (搜索)) antagonist, in 70 patients with ADH1, is fully enrolled. Topline results are anticipated in the second half of 2025. Encaleret aims to be the first approved therapy for ADH1.
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Infigratinib for Achondroplasia (搜索): The PROPEL 3 Phase 3 trial, investigating infigratinib, an FGFR1 (搜索)-3 inhibitor, in 114 children with achondroplasia, is fully enrolled. The company anticipates the last participant's last visit in the second half of 2025. If successful, infigratinib would be the first approved oral therapy for children with achondroplasia.
Financial Position and 2025 Outlook
BridgeBio is well-positioned financially, with $406 million in cash as of the last quarter. The company received $500 million upon Attruby's FDA approval and anticipates $105 million in regulatory milestones in the first half of 2025 from potential approvals of acoramidis in Europe and Japan.
Attruby Safety Information
Adverse reactions reported in patients treated with Attruby included diarrhea (11.6% vs 7.6% in placebo) and upper abdominal pain (5.5% vs 1.4% in placebo). Most reactions were mild and resolved without discontinuation. Discontinuation rates due to adverse events were similar between Attruby and placebo (9.3% and 8.5%, respectively).