On October 29, 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 third quarter ended September 30, 2025, and provided business updates.

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Commercial Progress:
As of October 25, 2025, 5,259 unique patient prescriptions have been written by 1,355 unique prescribers since FDA approval in November 2024. The third quarter revenue totaled $120.7 million, comprised of $108.1 million of U.S. Attruby net product revenue, $4.3 million from royalty revenue, and $8.3 million in license and services revenue.

"Attruby’s first year on the market has been remarkable, with continued growth across all market segments and strong physician adoption that reflects both the differentiated clinical profile and the trust we’re earning within the community. We’re seeing meaningful momentum where prescribers are not only initiating more patients on therapy but continuing treatment, underscoring Attruby’s real-world impact," said Matt Outten, Chief Commercial Officer of BridgeBio. "Importantly, this launch has also given us an invaluable blueprint for how we bring best-in-class medicines to patients with genetic diseases. With the promising results seen in BBP-418 for LGMD2I/R9 and encaleret for ADH1, we are building on a proven commercial foundation to ensure these communities, each living with urgent, unmet needs, have timely access to transformative therapies and we will apply the playbook we’ve developed to prepare for our next potential approvals."

Pipeline Overview:

Program Status Next expected milestone
Acoramidis for ATTR-CM Approved in U.S., EU, Japan, and UK New variant data to be shared at AHA Scientific Sessions
BBP-418 for LGMD2I/R9 FORTIFY, Phase 3 study interim analysis topline results released File NDA with FDA in 1H 2026
Encaleret for ADH1 CALIBRATE, Phase 3 study topline results released File NDA with FDA in 1H 2026
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 completed Phase 3 study to be initiated in 2026
Infigratinib for hypochondroplasia ACCEL 2/3, Phase 2 study first participant dosed Enrollment completion for Phase 2 portion by end of 2025

Key Program Updates:

"Attruby’s strong commercial performance continues to validate our model, delivering a potential best-in-class medicine to patients who were historically overlooked and building meaningful momentum across all market segments," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "We are now seeing that same success echoed in our pipeline with home-run data in both ADH1 and LGMD2I/R9, and we continue to advance one of the broadest and fastest-moving portfolios in genetic medicine. With achondroplasia data expected in 2026, enrollment nearing completion for the Phase 2 potion of the hypochondroplasia study, and registrational studies for encaleret in chronic hypoparathyroidism and pediatric ADH1 planned next year, we are not slowing down and continue to be impatient for patients. These milestones reflect our growing scalability and strengthen our conviction that BridgeBio is only beginning to show what’s possible as we evolve into a durable, multi-medicine company built for patients with genetic diseases for decades to come."

Attruby (acoramidis) – First near-complete (≥90%) transthyretin (TTR) stabilizer for treatment of transthyretin amyloid cardiomyopathy (ATTR-CM):

At this year’s Heart Failure Society of America (HFSA) Annual Scientific Meeting, BridgeBio presented data from the ATTRibute-CM study showing that acoramidis reduced cumulative cardiovascular outcomes, including cardiovascular mortality (CVM) or recurrent cardiovascular-related hospitalizations (CVH), within the first month of treatment in patients with ATTR-CM. At Month 30, Acoramidis significantly reduced the cumulative risk of CVM or recurrent CVH versus placebo with a 49% hazard reduction (p<0.0001). Acoramidis also showed that 53 events were avoided per 100 treated participants (95% CI:29–79) at Month 30. These data were presented in a Late Breaking Clinical Trials Oral Presentation at the HFSA Annual Scientific Meeting 2025 and simultaneously published in Journal of the American College of Cardiology (JACC).
Earlier in the year at the European Society of Cardiology Congress, BridgeBio shared a post-hoc analysis of ATTRibute-CM, demonstrating a significant reduction in risk of CVM through 42 months post-randomization, with a 44% hazard reduction, setting a new standard for CVM outcomes for patients with ATTR-CM. Acoramidis also demonstrated a significant 46% hazard reduction in the risk of the composite outcome of CVM or first CVH through 42 months.
More data on Attruby will be shared at the American Heart Association (AHA) Congress in November 2025 and in medical congresses throughout 2026.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):

FORTIFY, the Phase 3 clinical trial of BBP-418, successfully achieved all primary and secondary endpoints of its interim analysis.
Findings included a highly statistically significant increase of 1.8x change from baseline (p<0.0001) of glycosylated alpha-dystroglycan (αDG) observed in the BBP-418 group compared to approximately no change in the placebo group from baseline to 3 months, which was sustained at 12 months (p<0.0001).
An average reduction in serum creatine kinase (CK), a marker of muscle breakdown, of 82% change from baseline (p<0.0001) was observed in BBP-418 treated individuals.
Importantly, statistically significant and clinically meaningful improvements in ambulation [100-meter timed test (100MTT) velocity, p<0.0001] and pulmonary function [forced vital capacity (FVC) % predicted, sitting position, p=0.0071] were also observed at 12 months in the BBP-418 group compared with the placebo group.
BBP-418 was well-tolerated with no new or unexpected safety findings observed.
BridgeBio intends to file a New Drug Application (NDA) for approval with the FDA in the first half of 2026.
If successful, BBP-418 could be the first approved therapy for individuals living with LGMD2I/R9, potentially representing the first approval of a therapy for any form of LGMD.
The Company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age and in LGMD2M/2U in the near future.

Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1) and chronic hypoparathyroidism:

CALIBRATE, the Phase 3 clinical trial of oral encaleret in ADH1, a genetic form of hypoparathyroidism, successfully achieved all pre-specified primary and key secondary efficacy endpoints.
The primary endpoint was met with 76% of participants administered encaleret achieving both serum and urine calcium within the respective target ranges at Week 24 compared to 4% when on conventional therapy at Week 4 (p<0.0001).
In a key secondary analysis, 91% of participants administered encaleret achieved intact parathyroid hormone above the lower limit of the reference range at Week 24 compared to 7% of participants when on conventional therapy at Week 4 (p<0.0001).
Encaleret was well-tolerated with no discontinuations related to study drug.
BridgeBio intends to submit a NDA to the FDA in the first half of 2026, and a Marketing Authorization Application to the European Medicines Agency to follow.
If approved, encaleret would be the first therapy indicated for individuals living with ADH1.
The Company plans to initiate a registrational clinical trial of encaleret in pediatric ADH1 in the first quarter of 2026
The Company also plans to initiate a Phase 3 study of encaleret in chronic hypoparathyroidism in 2026.

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 randomized.
BridgeBio expects topline results of PROPEL 3 in early 2026.
BridgeBio has reached regulatory alignment with the FDA on the clinical development plan for infigratinib in infants and toddlers with achondroplasia from birth to less than 3 years old. Based on the discussion, the Company initiated clinical development in this important age range.
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 Phase 2 portion of the study by the end of 2025. The Phase 2 data is expected in the second half of 2026.
Infigratinib demonstrates single-digit nanomolar potency in vitro across causative FGFR3 variants in hypochondroplasia, consistent with its activity in achondroplasia, highlighting robust preclinical efficacy across FGFR3-driven skeletal dysplasias (Demuynck et al., JBMR, 2025).
In achondroplasia, infigratinib has received Breakthrough Designation from the FDA, Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation. To date, in hypochondroplasia, infigratinib has received Fast Track Designation and Orphan Drug Designation from the FDA and EMA.
If successful, infigratinib would be the first approved oral therapy option for children living with achondroplasia and hypochondroplasia.

Financial Updates:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities totaled $645.9 million as of September 30, 2025, compared to cash and cash equivalents of $681.1 million as of December 31, 2024. The $35.2 million decrease is primarily attributable to net cash used in operating activities of $389.5 million for the nine months ended September 30, 2025, the repayment of the Company’s previous term loan under its 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. These outflows were partially offset by net proceeds of $563.0 million from the issuance of the 2031 Notes in February 2025 and net proceeds of $297.0 million from the execution of the Royalty Interest Purchase and Sale Agreement with HealthCare Royalty, a related party, and Blue Owl Capital in June 2025.

Total Revenues, Net

Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
(in thousands)
Net product revenue $ 108,111 $ — $ 216,351 $ —
License and services revenue 8,311 2,732 125,441 216,020
Royalty revenue 4,278 — 6,106 —
Total revenues, net $ 120,700 $ 2,732 $ 347,898 $ 216,020

Total revenues, net for the three months ended September 30, 2025, were $120.7 million compared to $2.7 million for the same period in the prior year. The $118.0 million increase was primarily driven by a $108.1 million increase in net product revenue from the Company’s commercial product, Attruby, a $5.6 million increase in license and services revenue, and a $4.3 million increase in royalty revenue earned on net product sales of BEYONTTRA in the EU and Japan.

Total revenues, net for the nine months ended September 30, 2025, were $347.9 million compared to $216.0 million for the same period in the prior year. The $131.9 million increase was primarily driven by a $216.4 million increase in net product revenue from the Company’s commercial product, Attruby, and a $6.1 million increase in royalty revenue earned on net product sales of BEYONTTRA in the EU and Japan. These increases were partially offset by a $90.6 million decrease in license and services revenue, reflecting the timing of recognition of upfront payments from the Company’s exclusive license agreements with collaboration partners as well as regulatory-related milestones recognized upon the approval of BEYONTTRA in the EU and pricing approval in Japan.

Operating Costs and Expenses

Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
(in thousands)
Total cost of revenues $ 6,563 $ 598 $ 12,855 $ 1,794
Research and development 112,874 120,444 335,536 376,111
Selling, general and administrative 137,621 68,819 373,140 194,149
Restructuring, impairment and related charges 8,841 4,621 10,216 10,912
Total operating costs and expenses $ 265,899 $ 194,482 $ 731,747 $ 582,966

Operating costs and expenses for the three months ended September 30, 2025 were $265.9 million compared to $194.5 million for the same period in the prior year. The $71.4 million increase was primarily driven by a $68.8 million increase in selling, general and administrative ("SG&A") expenses largely reflecting the Company’s investments in support of the commercial launch and ongoing activities of Attruby, and a $6.0 million increase in total cost of revenues, primarily due to the cost of Attruby products sold. These increases were partially offset by a $7.6 million decrease in research and development ("R&D") expenses as a result of the Company’s reprioritization of its R&D programs.

Operating costs and expenses for the nine months ended September 30, 2025 were $731.7 million compared to $583.0 million for the same period in the prior year. The $148.7 million increase was primarily driven by a $179.0 million increase in SG&A largely reflecting the Company’s investments to support the commercial launch and ongoing activities of Attruby, and an $11.1 million increase in total cost of revenues, primarily due to the cost of Attruby products sold. The increases were partially offset by a $40.6 million decrease in R&D expenses as a result of the Company’s reprioritization of its R&D programs.

Stock-based compensation expenses included in operating costs and expenses for the three months ended September 30, 2025 were $35.3 million, of which $21.9 million is included in SG&A expenses, $12.3 million is included in R&D expenses, $0.7 million is included in restructuring impairment and related charges, and $0.4 million is included in cost of goods sold. Stock-based compensation expenses included in operating costs and expenses for the same period in 2024 were $27.1 million, of which $15.0 million was included in SG&A expenses and $12.1 million was included in R&D expenses.

Stock-based compensation expenses included in operating costs and expenses for the nine months ended September 30, 2025 were $102.0 million, of which $63.1 million is included in SG&A expenses, $37.5 million is included in R&D expenses, $0.8 million is included in restructuring impairment and related charges, and $0.6 million is included in cost of goods sold. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $77.4 million, of which $47.5 million was included in SG&A expenses and $29.8 million was included in R&D expenses, and $0.1 million was included in restructuring, impairment and related charges.

Total Other Income (Expense), Net

Total other income (expense), net for the three and nine months ended September 30, 2025, was $(41.3) million and $(153.9) million, respectively, compared to $27.5 million and $91.0 million, respectively, for the same periods in the prior year.

The change in total other income (expense), net of $68.8 million for the three months ended September 30, 2025, compared to 2024 was primarily due to a decrease in gain on deconsolidation of subsidiaries of $52.0 million, and an increase in noncash interest expense on deferred royalty obligations of $36.4 million, partially offset by a decrease in interest expense of $11.3 million, and an increase in other income of $7.8 million related to noncash income from the Company’s equity method investment.

The change in total other income (expense), net of $244.9 million for the nine months ended September 30, 2025, compared to 2024 was primarily due to a decrease in gain on deconsolidation of subsidiaries of $178.3 million, an increase in noncash interest expense on deferred royalty obligations of $86.5 million, and an increase in net loss from equity method investments of $37.1 million; partially offset by a decrease in interest expense of $28.0 million, an increase in other income of $11.1 million for the change in fair value of the embedded derivative liability component of the Company’s deferred royalty obligation, an increase in other income of $7.5 million for services provided under the transition service agreements, and an increase in other income of $7.8 million related to noncash income from the Company’s equity method investment.

Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share

For the three and nine months ended September 30, 2025, the Company recorded a net loss attributable to common stockholders of BridgeBio of $182.7 million and $532.1 million, respectively, compared to $162.0 million and $270.7 million, respectively, for the same periods in the prior year.

For the three and nine months ended September 30, 2025, the Company reported a net loss per share of $0.95 and $2.79, respectively, compared to $0.86 and $1.46, respectively, for the same periods in the prior year.

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Operations
(in thousands, except shares and per share amounts)

Three Months Ended September 30, Nine Months Ended September 30,
2025 2024 2025 2024
(Unaudited) (Unaudited)
Revenues:
Net product revenue $ 108,111 $ — $ 216,351 $ —
License and services revenue 8,311 2,732 125,441 216,020
Royalty revenue 4,278 — 6,106 —
Total revenues, net 120,700 2,732 347,898 216,020
Operating costs and expenses:
Cost of revenues:
Cost of goods sold 4,028 — 8,910 —
Cost of license, services and royalty revenue 2,535 598 3,945 1,794
Total cost of revenues 6,563 598 12,855 1,794
Research and development 112,874 120,444 335,536 376,111
Selling, general and administrative 137,621 68,819 373,140 194,149
Restructuring, impairment and related charges 8,841 4,621 10,216 10,912
Total operating costs and expenses 265,899 194,482 731,747 582,966
Loss from operations (145,199 ) (191,750 ) (383,849 ) (366,946 )
Other income (expense), net:
Interest income 6,239 3,296 15,522 12,566
Interest expense (11,739 ) (23,061 ) (41,467 ) (69,469 )
Noncash interest expense on deferred royalty obligations (1) (36,410 ) — (86,460 ) —
Gain on deconsolidation of subsidiaries — 52,027 — 178,321
Loss on extinguishments of debt — — (21,155 ) (26,590 )
Net loss from equity method investments (15,834 ) (6,563 ) (51,579 ) (14,488 )
Other income, net 16,461 1,797 31,240 10,648
Total other income (expense), net (41,283 ) 27,496 (153,899 ) 90,988
Loss before income taxes (186,482 ) (164,254 ) (537,748 ) (275,958 )
Provision for (benefit from) income taxes (1,545 ) — 555 —
Net loss (184,937 ) (164,254 ) (538,303 ) (275,958 )
Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,194 2,214 6,235 5,246
Net loss attributable to common stockholders of BridgeBio $ (182,743 ) $ (162,040 ) $ (532,068 ) $ (270,712 )
Net loss per share attributable to common stockholders of BridgeBio, basic and diluted $ (0.95 ) $ (0.86 ) $ (2.79 ) $ (1.46 )
Weighted-average shares used in computing net loss per share attributable to common stockholders of BridgeBio, basic and diluted 191,854,152 188,510,372 190,845,133 184,947,173

(1) Including related party amounts of $(5,383) and $(5,560) for the three and nine months ended September 30, 2025, respectively.

Three Months Ended September 30,
Nine Months Ended September 30,
Stock-based Compensation 2025 2024 2025 2024
(Unaudited) (Unaudited)
Cost of goods sold $ 361 $ — $ 578 $ —
Research and development 12,328 12,124 37,582 29,840
Selling, general and administrative 21,866 14,969 63,077 47,511
Restructuring, impairment and related charges 709 38 755 81
Total stock-based compensation $ 35,264 $ 27,131 $ 101,992 $ 77,432

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)

September 30,
2025 December 31,
2024
(Unaudited) (1)
Assets
Cash, cash equivalents and marketable securities $ 645,942 $ 681,101
Accounts receivable, net 116,518 4,722
Inventories 24,527 —
Prepaid expenses and other current assets 52,395 34,869
Investment in nonconsolidated entities 92,168 143,747
Property and equipment, net 5,830 7,011
Operating lease right-of-use assets 6,553 5,767
Intangible assets, net 28,795 23,926
Other assets 25,522 18,195
Total assets $ 998,250 $ 919,338
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable $ 18,702 $ 9,618
Accrued and other current liabilities (2) 183,517 125,672
Operating lease liabilities 8,721 9,202
Deferred revenue 22,218 31,699
2031 Notes, net 564,087 —
2029 Notes, net 740,380 738,872
2027 Notes, net 546,549 545,173
Term loan, net — 437,337
Deferred royalty obligations, net (3) 836,126 479,091
Other long-term liabilities 679 286
Redeemable convertible noncontrolling interests 23 142
Total BridgeBio stockholders’ deficit (1,933,070 ) (1,467,904 )
Noncontrolling interests 10,318 10,150
Total liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit $ 998,250 $ 919,338

(1) The condensed consolidated financial statements as of and for the year ended December 31, 2024 are derived from the audited consolidated financial statements as of that date.
(2) Including a related party amount of $1,647 as of September 30, 2025.
(3) Including a related party amount of $201,242 as of September 30, 2025.

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
(In thousands)

Nine Months Ended September 30,
2025 2024
Operating activities:
Net loss $ (538,303 ) $ (275,958 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation 98,385 65,673
Loss on extinguishments of debt 21,155 26,590
Noncash interest expense on deferred royalty obligations (1) 86,460 —
Amortization of debt discount and issuance costs 4,515 5,399
Depreciation and amortization 3,999 4,708
Noncash lease expense 3,443 3,119
Net loss from equity method investments 51,579 14,488
Change in fair value of the embedded derivative associated with the deferred royalty obligation (11,062 ) —
Noncash income from an equity method investment (7,769 ) —
Gain on deconsolidation of subsidiaries — (178,321 )
Gain from investment in equity securities, net — (8,136 )
Other noncash adjustments, net (1,217 ) (2,059 )
Changes in operating assets and liabilities:
Accounts receivable, net (111,796 ) 1,273
Inventories (23,356 ) —
Prepaid expenses and other current assets (17,527 ) (17,543 )
Other assets 568 (428 )
Accounts payable 9,084 5,257
Accrued compensation and benefits 3,212 5,580
Accrued research and development liabilities 347 15,454
Operating lease liabilities (4,757 ) (4,459 )
Deferred revenue (9,480 ) 20,575
Other liabilities (2) 53,030 (6,612 )
Net cash used in operating activities (389,490 ) (325,400 )
Investing activities:
Purchases of marketable securities (10,876 ) (93,811 )
Maturities of marketable securities 8,000 95,000
Purchases of investments in equity securities — (20,271 )
Proceeds from sales of investments in equity securities — 63,229
Proceeds from special cash dividends received from an investment in equity securities 2,302 25,682
Payment for intangible assets (8,495 ) (4,785 )
Purchases of property and equipment (1,064 ) (886 )
Decrease in cash and cash equivalents resulting from deconsolidation of subsidiaries — (140 )
Net cash provided by (used in) investing activities (10,133 ) 64,018
Financing activities:
Proceeds from issuance of 2031 Notes 575,000 —
Issuance costs and discounts associated with 2031 Notes (12,034 ) —
Repurchase of common stock (48,276 ) —
Proceeds from a royalty obligation under the Royalty Purchase Agreement 300,000 —
Issuance costs associated with a royalty obligation under the Royalty Purchase Agreement (3,010 ) —
Proceeds from term loan under the Amended Financing Agreement — 450,000
Issuance costs and discounts associated with term loan under the Amended Financing Agreement — (15,986 )
Repayment of term loans (459,000 ) (473,417 )
Repayments of deferred royalty obligations (3) (6,896 ) —
Proceeds from issuance of common stock through public offerings, net — 314,741
Proceeds from common stock issuances under ESPP 6,414 4,502
Proceeds from stock option exercises, net of repurchases 14,523 808
Transactions with noncontrolling interests 1,550 —
Repurchase of RSU shares to satisfy tax withholding (6,796 ) (6,122 )
Net cash provided by financing activities 361,475 274,526
Net increase (decrease) in cash, cash equivalents and restricted cash (38,148 ) 13,144
Cash, cash equivalents and restricted cash at beginning of period 683,244 394,732
Cash, cash equivalents and restricted cash at end of period $ 645,096 $ 407,876

(1) Including a related party amount of $5,560 for the nine months ended September 30, 2025.
(2) Including a related party amount of $1,647 for the nine months ended September 30, 2025.
(3) Including a related party amount of $(665) for the nine months ended September 30, 2025.

Nine Months Ended September 30,
2025 2024
Supplemental Disclosure of Cash Flow Information:
Cash paid for interest $ 43,670 $ 78,236
Cash paid for income taxes $ 1,153 $ —
Supplemental Disclosures of Noncash Investing and Financing Information:
Unpaid property and equipment $ 12 $ 274
Transfers to noncontrolling interests $ (4,734 ) $ (4,719 )
Reconciliation of Cash, Cash Equivalents and Restricted Cash:
Cash and cash equivalents $ 642,951 $ 266,324
Restricted cash — Included in "Prepaid expenses and other current assets" 126 139,409
Restricted cash — Included in "Other assets" 2,019 2,143
Total cash, cash equivalents and restricted cash at end of periods shown in the condensed consolidated
statements of cash flows $ 645,096 $ 407,876

Webcast Information
BridgeBio will host its quarterly earnings call and simultaneous webcast on Wednesday, October 29th 2025 at 4:30 pm ET. To access the live webcast of BridgeBio’s presentation, please visit the "Events" page within the Investors section of the BridgeBio website at View Source or register online using the following link, View Source A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event.

(Press release, BridgeBio, OCT 29, 2025, View Source [SID1234657114])

On October 29, 2025 Recursion Pharmaceuticals, Inc. (the "Company" or "Recursion") reported the exercise of the option by Roche (as defined below) for a Microglia Map—a first-of-its-kind whole genome map of the brain’s immune cells—generated by Recursion under the Collaboration and License Agreement with Genentech, Inc. and F. Hoffmann-La Roche Ltd (collectively "Roche") dated December 5, 2021 (the "Agreement"). Pursuant to the terms of the Agreement, Roche will pay to the Company an Acceptance Fee of $30 million.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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(Press release, Recursion Pharmaceuticals, OCT 29, 2025, View Source [SID1234657113])

On October 29, 2025 PDS Biotechnology Corporation (Nasdaq: PDSB) ("PDS Biotech" or the "Company"), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers, reported that after recent review of the final VERSATILE-002 data, the Company has requested a meeting with the Food and Drug Administration ("FDA") to explore an expedited approval pathway for PDS0101 in HPV16-positive Head and Neck Cancer. The FDA meeting request is based on the final results from its VERSATILE-002 trial and a proposed amendment to the VERSATILE-003 Phase 3 trial to reduce the number of patients, while maintaining statistical power, and to add progression free survival (PFS) as an earlier primary endpoint in addition to median overall survival (mOS). If the PFS endpoint is met, it would allow for an accelerated approval submission to the FDA. While the Company’s trial amendment is undergoing review by the FDA, the VERSATILE-003 trial will be temporarily paused.

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"The final survival results and durable clinical responses from our VERSATILE-002 trial are very exciting. It’s the first time that a trial in the recurrent/metastatic HNSCC population has reported a mOS of almost 40 months," said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. "The fact that our Phase 3 study has mOS as its primary endpoint and PFS as a secondary endpoint inherently lengthens the duration of the trial. To shorten the trial duration, we believe a meeting with the FDA to discuss changes to the current trial protocol to include PFS as an earlier primary endpoint independent of mOS is warranted. We look forward to keeping all PDS Biotech stakeholders informed as our discussions with the FDA progress."

Dr. Kirk Shepard, MD, Chief Medical Officer added, "Based on the increasing incidence of HPV16-positive head and neck cancer, our goal is to seek the fastest and most cost-effective regulatory pathway to approval. Our goal is to be efficient in providing a well-tolerated treatment without chemotherapy as an option for patients who currently have no effective therapies for this deadly disease. Treatment with PDS0101 for currently enrolled patients in our VERSATILE-003 Phase 3 trial will continue during the temporary pause of the trial."

About the VERSATILE-002 Trial
VERSATILE-002 (NCT04260126) is an open-label, multi-center Phase 2 clinical trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted immunotherapy, in combination with pembrolizumab for unresectable, recurrent or metastatic HPV16-positive HNSCC. The trial is designed to assess the combination therapy’s impact on patients who are either naive to or refractory to immune checkpoint inhibitors. The full data set from VERSATILE-002 is mature and expected to be submitted for publication later this year.

(Press release, PDS Biotechnology, OCT 29, 2025, View Source [SID1234657111])

On October 29, 2025 ModeX Therapeutics Inc., an OPKO Health company (NASDAQ: OPK), reported that it has entered into a license and collaboration agreement with Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) to discover and develop multispecific antibodies for several therapeutic indications of mutual interest. The collaboration will apply ModeX’s MSTAR platform and Regeneron’s proprietary binders to develop multispecific antibody candidates that target multiple distinct biological pathways in a single molecule.

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Under the terms of the agreement, ModeX will receive an upfront payment of USD $7 million, and potential future product selection payments, clinical and regulatory milestone payments, and commercial milestone payments exceeding USD $200 million per selected molecule. The overall value of the collaboration potentially exceeds $1 billion if multiple products from the collaboration are successful. In addition, ModeX is eligible to receive tiered global net sales royalties, up to low double digits at the highest tier. Regeneron will fund and lead any preclinical, clinical development and commercialization activities for any products it elects to advance.

"This agreement allows us to work closely with Regeneron, world leaders in the development of antibody medicines, to explore the promise of multispecific antibodies in new indications, including immunology, oncology and metabolic diseases. ModeX’s mission is to develop innovative antibodies that recognize multiple targets in a single molecule to treat life-threatening diseases," said Gary Nabel, M.D., Ph.D., President and Chief Executive Officer of ModeX and Chief Innovation Officer of OPKO Health.

"We have dramatically expanded the therapeutic potential of antibodies through our MSTAR platform. Our antibody candidates are already in clinical trials, providing unique disease target combinations and reliable manufacturing," said Elias Zerhouni, M.D., President and Vice Chairman of OPKO. "We believe this collaboration, with Regeneron’s outstanding team, will advance the development of multispecific antibodies across a spectrum of chosen indications in several disease areas of high interest to both parties."

"Like Regeneron, ModeX has a team of scientist entrepreneurs who think creatively about science and technology," said George D. Yancopoluos, M.D., Ph.D., Board Co-Chair, President and Chief Scientific Officer of Regeneron. "By pairing Regeneron’s expertise in drug development with ModeX’s platform for multispecific antibodies, we are building on Regeneron’s longstanding work in bi-and multi-specific antibodies and increasing our shots on goal by identifying more candidates faster to potentially help patients across multiple disease categories."

Beyond bispecifics: ModeX Synergistic Targeting of Antibody Receptors (MSTAR)

Multispecific antibody therapeutics can help generate medicines of the future by targeting three or more disease pathways simultaneously. Many untreatable or complex conditions arise from multiple disease pathways; yet, most medicines only act on a single target. ModeX overcomes these challenges by combining natural protein structures using a platform known as the ModeX Synergistic Targeting of Antibody Receptors (MSTAR) to create unique multispecific medicines. The platform generates multispecific antibodies from modular building blocks intended to modulate potency and maximize specificity to address multiple disease pathways simultaneously.

(Press release, Opko Health, OCT 29, 2025, View Source [SID1234657109])

On October 29, 2025 OPKO Health, Inc. (NASDAQ: OPK) (OPKO) reported business highlights and financial results for the three and nine months ended September 30, 2025.

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Highlights from the third quarter of 2025 and recent weeks include the following:

Entered into a research collaboration with Regeneron Pharmaceuticals to develop multispecific antibodies. This new partnership leverages ModeX’s MSTAR technology platform with Regeneron’s proprietary binders to develop single molecule candidates that target multiple distinct biological pathways in several indications. ModeX is entitled to receive an upfront payment and potential milestone payments exceeding $200 million for each program. The overall value of the collaboration potentially exceeds $1 billion if multiple products from the collaboration are successful. In addition, ModeX is eligible to receive tiered royalties on global net sales, up to low double digits at the highest tier. Regeneron is responsible for funding all preclinical and clinical development, as well as all commercialization activities.
Completed the sale of BioReference Health (BioReference) oncology and related clinical assets to Labcorp for $225 million. The purchase included $192.5 million that was paid at closing and up to $32.5 million in a performance-based earnout. BioReference will continue to offer its core clinical testing services in the New York and New Jersey region and its 4Kscore Test franchise, which represented approximately $300 million in revenue for 2024. This transaction streamlines BioReference’s laboratory services business and better positions the company to optimize its test menu and achieve sustained profitability. OPKO intends to utilize a portion of the proceeds to fund its share repurchase program.
Merck advanced the Phase 1 Epstein-Barr virus vaccine trial (NCT06655324). This investigational vaccine candidate is being developed in collaboration with Merck and evaluates safety and tolerability in up to 200 healthy adults. Enrollment in this ongoing trial is progressing well and the safety and immunogenicity data obtained from this trial will inform the Phase 2 trial design.
First patient dosed in MDX2004 Phase 1/2a study for the treatment of advanced cancers (NCT07110584). This study is designed to evaluate the safety, tolerability and biologic activity of MDX2004, a first-in-class trispecific antibody-fusion protein, as an immunotherapy for advanced cancers. Preclinical proof-of-concept data, as well as clinical dose selection analyses to support MDX2004 development will be showcased in two poster presentations at the 40th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), being held November 5-9, 2025.
Abstract for MDX-2001 CMet-Trop2/CD3-CD28, a first-in-class tetraspecific T-cell engager, was presented at ESMO (Free ESMO Whitepaper) 2025. In October, the abstract titled "A phase I/IIa, multicenter, first-in-human, open-label clinical trial evaluating MDX2001, a tetraspecific T cell engager-expander in patients with advanced solid tumors" was presented at ESMO (Free ESMO Whitepaper) Congress 2025, the annual meeting of the European Society for Medical Oncology. The MDX2001 CMet-Trop2/CD3-CD28 tetraspecific antibody has advanced to the fifth dose level in its Phase 1 clinical trial, with Phase 1b studies in select solid tumors expected to begin in early 2026.
Abstract for first-in-class dual GLP-1/glucagon tablet candidate was presented at the ENDO 2025 annual meeting. In July, the abstract titled "First-in-Class Oral Dual GLP-1/Glucagon Agonist for Patients with Obesity and Metabolic Disorders: In Vivo Pharmacokinetic and Pharmacodynamic Results" highlighting preclinical animal data was presented at ENDO 2025, the annual meeting of the Endocrine Society. Oral OPK-88006 is being developed pursuant to a collaboration and license agreement between OPKO and Entera Bio (Entera) whereby the companies are advancing a proprietary novel dual agonist GLP-1/glucagon peptide as a once-daily tablet treatment with OPK-88006 and Entera’s proprietary N-Tab technology.
Abstract on the pharmacokinetics/pharmacodynamics of oral GLP-2 tablet for the treatment of short bowel syndrome was presented at the 2025 ESPEN Congress. In September, the abstract "A First-in-Class Oral GLP-2 Analog for Treatment of Short Bowel Syndrome" highlighting in vivo animal data was presented at the 47th European Society for Clinical Nutrition & Metabolism (ESPEN) Congress. Pursuant to a research collaboration agreement with Entera, the companies are developing an oral GLP-2 tablet that combines a proprietary long-acting GLP-2 agonist developed by OPKO with Entera’s proprietary N-Tab technology, for patients suffering from short bowel syndrome and additional disorders involving gastrointestinal mucosal inflammation and nutrient malabsorption.
FDA approved the supplemental application for the 4Kscore Test regarding the availability of digital rectal examination information. In July, the U.S. Food and Drug Administration (FDA) approved OPKO’s supplemental application enabling the performance of the 4Kscore Test without digital rectal examination (DRE) information. The 4Kscore Test is indicated for the assessment of the likelihood of aggressive prostate cancer in men age 45 and older who have age-specific elevated/abnormal screening PSA results. Two prospective controlled clinical studies (n=937) concluded that the 4Kscore Test is a reliable (>96% sensitivity and accuracy) blood test to assess the probability of aggressive prostate cancer, before biopsy decisions. In the U.S., over 90% of PSA screening tests are ordered by primary care providers, potential users of the 4Kscore Test who don’t routinely perform a DRE.
Third Quarter Financial Results

Consolidated: Consolidated total revenues for the third quarter of 2025 were $151.7 million compared with $173.6 million for the comparable period of 2024. Operating income for the third quarter of 2025 was $48.1 million compared with $14.2 million for the 2024 quarter. Net income for the third quarter of 2025 included a gain of $101.6 million from the sale of the BioReference oncology assets. The prior-year period included a gain of $121.5 million from the sale of certain BioReference clinical assets and income of $45.9 million related to the investment in GeneDx shares. Net income for the third quarter of 2025 was $21.6 million, or $0.03 per diluted share, compared with $24.9 million, or $0.03 per diluted share, for the 2024 quarter.
Pharmaceuticals: Revenue from products in the third quarter of 2025 was $37.7 million compared with $39.1 million in the third quarter of 2024, reflecting lower sales volumes in certain international operations primarily due to the timing of customer orders and product mix, partially offset by an increase in Rayaldee sales. Revenue from sales of Rayaldee was $7.5 million compared with $5.8 million in the comparable prior-year period. Revenue from the transfer of intellectual property and other was $18.8 million in the third quarter of 2025 compared with $13.2 million in the 2024 period. The increase was driven by higher revenue from the BARDA contract and higher gross profit share payments for NGENLA, which totaled $8.8 million in the 2025 period compared with $7.0 million in the 2024 period. Total costs and expenses decreased to $80.6 million in the third quarter of 2025 from $84.6 million in the prior-year period, primarily due to lower cost of revenue related to lower sales volume and reduced employee-related expense, partially offset by higher research and development expenses driven by progress in the BARDA collaboration and advancements in early-stage programs. Operating loss was $24.2 million in the third quarter of 2025 compared with $32.2 million in the third quarter of 2024, with both periods including $18.0 million of depreciation and amortization expense.
Diagnostics: Revenue from services in the third quarter of 2025 was $95.2 million compared with $121.3 million in the prior-year period, with the decrease primarily due to lower clinical test volume principally as a result of the sale of certain BioReference assets in 2024, partially offset by higher clinical test reimbursement rates. Total costs and expenses, net of the gain on the sale of assets in both periods, were $13.6 million in the third quarter of 2025 compared with $62.7 million in the third quarter of 2024. The decrease was primarily attributable to the assets sold and continued cost-reduction initiatives at BioReference. Operating income was $81.6 million in the third quarter of 2025, which included $4.7 million of depreciation and amortization expense, compared with $58.5 million in the 2024 period, which included $6.1 million of depreciation and amortization expense. The third quarter of 2025 included revenue of $19.5 million and costs and expenses of $25.2 million from the oncology assets that were sold to Labcorp on September 15, 2025.
Cash, cash equivalents, marketable securities and restricted cash: Cash, cash equivalents and restricted cash were $428.9 million as of September 30, 2025. In September 2025, OPKO received $173.3 million in cash consideration and an escrow of $19.2 million subject to any outstanding indemnity claims upon closing of the Labcorp transaction. As of September 30, 2025, approximately $73.8 million of OPKO’s common stock had been repurchased under the program since its authorization in July 2024, and approximately $126.2 million remains authorized and available for future repurchases.
Conference Call and Webcast Information

OPKO’s senior management will provide a business update, discuss third quarter financial results, provide financial guidance and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call here. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here.

A telephone replay will be available until November 5, 2025, by dialing 877-344-7529 (U.S.) or 412-317-0088 (International) and providing the passcode 8678248. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here.

(Press release, Opko Health, OCT 29, 2025, View Source [SID1234657108])