On August 5, 2025 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a new type of biopharmaceutical company focused on genetic diseases, reported its financial results for the second quarter ended June 30, 2025, and provided business updates (Press release, BridgeBio, AUG 5, 2025, View Source [SID1234654779]).
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Commercial Progress:
As of August 1, 2025, 3,751 unique patient prescriptions for Attruby have been written by 1,074 unique healthcare providers since FDA approval in November 2024. The second quarter revenue totaled $110.6 million, comprised of $71.5 million of U.S. Attruby net product revenue, $1.6 million from royalty revenue, and $37.5 million in license and services revenue.
"Attruby’s latest results showcase the power of pairing breakthrough scientific excellence with disciplined commercial execution," said Matt Outten, Chief Commercial Officer of BridgeBio. "Product revenue nearly doubled this quarter, driven by growing adoption across centers of excellence and community physicians. With increasing demand and best-in-class patient access programs, we are confident Attruby will become the standard of care for ATTR-CM, setting the foundation for three additional rare disease launches in 2026 and 2027."
Pipeline Overview:
Program
Status
Next expected milestone
Acoramidis for ATTR-CM
Approved in U.S., EU, Japan, and UK
New rapidity of response data at ESC Congress in August 2025
BBP-418 for LGMD2I/R9
FORTIFY, Phase 3 study enrollment completed
Topline results in fall 2025
Encaleret for ADH1
CALIBRATE, Phase 3 study enrollment completed
Topline results in fall 2025
Infigratinib for achondroplasia
PROPEL 3, Phase 3 study enrollment completed
Topline results in early 2026
Encaleret for chronic hypoparathyroidism
Phase 2 proof-of-principle study ongoing
Late-stage clinical study to be initiated in 2026
Infigratinib for hypochondroplasia
ACCEL 2/3, Phase 2 study first participant dosed
Enrollment completion for Phase 2 portion in 2H 2025
Key Program Updates:
"The launch of Attruby continues to accelerate, increasing the number of patients’ lives we are able to touch. We remain grateful for the physicians and patients who are partnering with us on both treatment and on new clinical research," said Neil Kumar, Ph.D., CEO and Founder of BridgeBio. "The next six months will be transformative with Phase 3 readouts across ADH1, LGMD2I/R9, and achondroplasia. We hope these programs will build on Attruby’s success to allow us to become a leading diversified genetic disease company."
Attruby (acoramidis) – First near-complete (≥90%) transthyretin (TTR) stabilizer for treatment of transthyretin amyloid cardiomyopathy (ATTR-CM):
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At this year’s Annual Congress of the Heart Failure Association of the European Society of Cardiology, BridgeBio shared a post-hoc analysis of ATTRibute-CM, showing acoramidis reduced the annual frequency of cardiovascular hospitalization due to atrial fibrillation (AF)/atrial flutter (AFL) by 43% compared to placebo and reduced the incidence of new-onset AF/AFL by 17% in the subgroup with no prior history of AF compared to placebo in the overall ATTR-CM population.
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Findings were published in the Journal of the American College of Cardiology (JACC), showing for each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever better levels of stabilization achieved by treatment with acoramidis lead to ever better clinical outcomes.
ATTRibute-CM is the only study to demonstrate a direct association between a prompt, sustained increase in serum TTR and survival in patients with ATTR-CM.
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In May 2025, the first asymptomatic participant with a known pathogenic TTR variant, that may lead to transthyretin amyloid disease (either cardiomyopathy, ATTR-CM, polyneuropathy, ATTR-PN, or both) was dosed in ACT-EARLY with acoramidis. ACT-EARLY is the first ever primary prevention study for ATTR, testing the hypothesis that prophylactic treatment of asymptomatic carriers of a pathogenic TTR variant with the near-complete TTR stabilizer, acoramidis, could delay the onset or prevent the development of variant ATTR (ATTRv), also known as hereditary ATTR (hATTR).
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More data on Attruby will be shared at the European Society of Cardiology (ESC) Congress in August 2025 and at additional medical meetings in the second half of 2025.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
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FORTIFY, the 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 results in progressive muscle degeneration and damage, and eventual loss of functional independence, is fully enrolled with 112 participants. The trial is the largest prospective interventional study to ever be conducted in LGMD2I/R9.
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The study includes a planned interim analysis at 12 months focused on assessing a surrogate endpoint biomarker (glycosylated alpha-dystroglycan) to support a potential Accelerated Approval in the U.S.
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Last participant last visit has been achieved, and the topline results of the interim analysis cohort are expected in fall 2025.
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An open-label Phase 2 clinical trial of BBP-418 in LGMD2I/R9 resulted in an approximate doubling of glycosylated alpha-dystroglycan (αDG) levels, a sustained decrease of ≥70% in serum creatine kinase (CK), and stabilization of ambulatory measures, in contrast to the progressive decline observed in natural history.
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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) and chronic hypoparathyroidism:
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CALIBRATE, the Phase 3 clinical trial of oral encaleret in ADH1, a genetic form of hypoparathyroidism, is fully enrolled with 71 participants. The registrational study is the largest prospective interventional study to ever be conducted in ADH1.
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BridgeBio expects topline results of CALIBRATE in the fall 2025.
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If successful, encaleret would be the first approved therapy for individuals living with ADH1.
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Dosing completed in a Phase 2 proof-of-principle clinical trial of encaleret in participants with hypoparathyroidism, which resulted in 80% of N=10 study participants achieving concomitant normal blood and urine calcium within 5 days. The Company intends to advance development of encaleret to enable registration in chronic hypoparathyroidism.
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Newly published findings from analyses of academic biobanks confirm previously cited estimates of ADH1 prevalence to be approximately 1 in 25,000. (source: American Journal of Human Genetics)
Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
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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 randomized.
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BridgeBio expects topline results of PROPEL 3 in early 2026.
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BridgeBio has reached regulatory alignment with the FDA on the clinical development plan for infigratinib in children with achondroplasia from birth to less than 3 years old. Based on the discussion, the Company expects to initiate clinical development in this important age range by the end of the year.
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The first participant in the Phase 2 portion of ACCEL 2/3 in hypochondroplasia was dosed in April 2025 and the Company expects to fully enroll the study for the Phase 2 portion in the second half of 2025.
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In achondroplasia, infigratinib has received Breakthrough Designation from the U.S. Food and Drug Administration, Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation. To date, in hypochondroplasia, infigratinib has received Orphan Drug Designation from the U.S. Food and Drug Administration and Fast Track Designation.
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If successful, infigratinib would be the first approved oral therapy option for children living with achondroplasia and hypochondroplasia.
Corporate Updates:
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BridgeBio received $300 million from the partial and capped sale of a portion of royalties due to the Company on sales of BEYONTTRA in Europe to HealthCare Royalty (HCRx) and funds managed by Blue Owl Capital (Blue Owl).
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BridgeBio received a regulatory-related milestone cash payment of $30 million from Alexion for the Japan approval of BEYONTTRA.
Financial Updates:
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities totaled $756.9 million as of June 30, 2025, compared to cash and cash equivalents of $681.1 million as of December 31, 2024. The $75.8 million net increase is primarily attributable to net proceeds of $563.0 million received from the issuance of the 2031 Notes in February 2025 and net proceeds of $297.0 million received from the execution of the Royalty Interest Purchase and Sale Agreement with HCRx and Blue Owl in June 2025. These increases were partially offset by net cash used in operating activities of $279.9 million for the first half of 2025, repayment of the Company’s previous term loan under the credit facility (including prepayment fees) of $459.0 million in February 2025, and the repurchase of common stock of $48.3 million using proceeds from the 2031 Notes in February 2025.