On January 13, 2025 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a new type of biopharmaceutical company focused on genetic diseases, reported updates on its commercial progress for Attruby (acoramidis), status of late-stage pipeline programs, and anticipated 2025 milestones (Press release, BridgeBio, JAN 13, 2025, View Source [SID1234649722]).
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"With the FDA’s approval of Attruby, we marked an important moment for both our organization and the broader ATTR-CM patient community in need of new treatment options. We’re grateful for the enthusiasm surrounding the product and the associated initial commercial momentum, with 430 prescriptions written by 248 unique physicians, and we look forward to continued progress," said Neil Kumar, Ph.D., Founder and CEO of BridgeBio. "Additionally, we are excited to share that we have completed enrollment of all three of our major market Phase 3 clinical trials. I look forward to continuing to work with this stellar team to serve patients with genetic disease in 2025."
Business Update
On November 22, 2024, the U.S. Food and Drug Administration (FDA) approved Attruby (acoramidis), a near-complete TTR stabilizer (≥90%), to reduce cardiovascular death and cardiovascular-related hospitalization in adult patients with ATTR-CM, a progressive fatal disease presenting as an infiltrative, restrictive cardiomyopathy resulting in heart failure.
Since the approval, BridgeBio has seen remarkable momentum with 430 patient prescriptions written by 248 physicians.
Pipeline Updates
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
FORTIFY is a Phase 3 clinical trial of BBP-418 in LGMD2I/R9, a rare genetic disorder caused by variants in the fukutin‑related protein (FKRP) gene that result in progressive muscle degeneration and damage, and eventual loss of functional independence. The trial is fully enrolled with 112 patients.
The Company expects Last Patient – Last Visit (LPLV) and topline readout of the interim analysis cohort in second half 2025.
If successful, BBP-418 would be the first approved therapy for individuals living with LGMD2I/R9.
Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1):
CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, a rare, genetic form of hypoparathyroidism, is fully enrolled with 70 patients. The trial is designed to evaluate the efficacy and safety of encaleret compared to standard of care in adult patients with ADH1.
The Company expects Last Patient – Last Visit and topline readout in second half 2025.
If successful, encaleret would be the first approved therapy for individuals living with ADH1.
Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
PROPEL 3, the Phase 3 clinical trial of infigratinib in achondroplasia, the most common form of disproportionate short stature, is fully enrolled with 114 participants.
The Company expects Last Participant – Last Visit in second half 2025.
If successful, infigratinib would be the first approved oral therapy for children living with achondroplasia.
2025 Milestones
Program Status Anticipated 2025 Milestone
Acoramidis for ATTR-CM US FDA approval on November 22, 2024 EU and Japan approvals in 1H 2025
BBP-418 for LGMD2I/R9 FORTIFY, Phase 3 study enrollment completed Last Patient – Last Visit and Topline readout in 2H 2025
Encaleret for ADH1 CALIBRATE, Phase 3 study enrollment completed Last Patient – Last Visit and Topline readout in 2H 2025
Infigratinib for achondroplasia PROPEL 3, Phase 3 study enrollment completed Last Participant – Last Visit in 2H 2025
About Attruby (acoramidis)
INDICATION
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.