On August 5, 2025 Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company, reported that its product portfolio of FDA-approved brands delivered combined net revenues of $10.8 million during the second quarter of 2025, a 10% increase over the prior year period (Press release, Cumberland Pharmaceuticals, AUG 5, 2025, View Source [SID1234654782]). Year-to-date revenues for the first six months of the year totaled $22.6 million, representing an increase of 23% over the first half of 2024.

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Cumberland ended the quarter with approximately $68 million in total assets, $40 million in liabilities and $28 million of shareholders’ equity.

"We have had a strong first half of the year, especially following the progress of our clinical development programs," said Cumberland Pharmaceuticals CEO A.J. Kazimi. "We look forward to building on this success throughout the remainder of the year as we continue our mission of working together to provide unique products that improve the quality of patient care."

RECENT COMPANY DEVELOPMENTS INCLUDE:
Vibativ 4-Vial Starter Pak Now Available for Vizient Providers
Cumberland recently announced the availability of the Vibativ (telavancin) 4-Vial Starter Pak through a new supply arrangement with Vizient Inc., making it accessible to their health care providers nationwide.
As the country’s largest provider-driven health care performance improvement company, Vizient serves more than 65% of the nation’s acute care providers, including 97% of academic medical centers and 35% of the non-acute market. Through this agreement, Vizient members now have access to Vibativ’s new 4-vial configuration, which supports flexible treatment initiation in both inpatient and outpatient settings for this potentially life-saving therapy.

Pharmacokinetic Analysis Reinforces Vibativ Dosing Strategies

A comprehensive new pharmacokinetic analysis of Vibativ was published in Antimicrobial Agents and Chemotherapy in June 2025. The analysis utilizes data from over 1,200 patients across varied demographics and comorbidity profiles. The findings support optimized dosing strategies for patients with different infection severities and renal function levels, reinforcing Vibativ’s critical role in treating life-threatening gram-positive infections.

Ifetroban Clinical Studies
In June 2025, breakthrough findings from Cumberland’s Phase II FIGHT DMD trial, evaluating its ifetroban product candidate in patients with Duchenne muscular dystrophy ("DMD"), were presented at the Parent Project Muscular Dystrophy Annual Conference. The findings demonstrated that high-dose ifetroban delivered a 5.4% improvement in cardiac function in patients with DMD. The presentation also included additional biomarker data indicating reduced cardiac damage, which correlated with the clinical findings. These results position ifetroban as a potential treatment for DMD cardiomyopathy – the leading cause of death in these patients and a critical unmet medical need affecting 90% of DMD patients.

The top-line FIGHT DMD study findings were also selected for a late-breaking presentation at the Muscular Dystrophy Association’s Clinical & Scientific Conference in March 2025. In June 2025, Cumberland completed the comprehensive analysis of the study results, completed its clinical study report and submitted it to the FDA along with a request for an end-of-Phase II meeting.

Meanwhile, Cumberland has been evaluating its ifetroban product candidate in a Phase II clinical program in patients with Systemic Sclerosis. Enrollment in the study was completed this year, and Cumberland is monitoring the clinical sites in preparation to lock the database and begin evaluating the results.
In addition, Cumberland has a Phase II clinical study, the FIGHTING FIBROSIS trial, underway in patients with Idiopathic Pulmonary Fibrosis, the most common form of progressive fibrosing interstitial lung disease. Patient enrollment is now well underway in medical centers across the U.S. The study design includes both an interim safety analysis, as well as an interim efficacy analysis.

New Study Features Caldolor (ibuprofen injection) for Older Patients
In May 2025, Cumberland announced the publication of its study investigating Caldolor (intravenous ibuprofen) in Clinical Therapeutics, demonstrating the product’s safety and efficacy for managing post-operative pain in patients 60 years of age and older. The analysis, encompassing over 1,000 older patients from comprehensive post-surgical studies, represents the first such evaluation in this vulnerable population, where traditional pain management options such as opioids carry increased risk.

FINANCIAL RESULTS:
Net Revenue: For the second quarter of 2025, net revenues were $10.8 million and included $2.8 million for Kristalose, $3.1 million for Sancuso, $2.7 million for Vibativ and $1.6 million for Caldolor.
Year-to-date 2025 net revenues were $22.6 million. Year-to-date net revenues by product were $6.2 million for Kristalose, $5.4 million for Sancuso, $4.1 million for Vibativ and $2.9 million for Caldolor.
Operating Expenses: Total operating expenses were $11.6 million for the second quarter of 2025 and $22 million for the first half of the year.
Net Income (loss): Year-to-date net income was approximately $516,000 and the second quarter net loss was approximately $741,000.
Adjusted Earnings: Adjusted earnings for the six months ended 2025 were $2.8 million, or $0.18 per diluted share.
Balance Sheet: At June 30, 2025, Cumberland had approximately $68 million in total assets, including $16 million in cash and cash equivalents. Liabilities totaled $40 million, including $5 million on the company’s credit facility. Total shareholders’ equity was $28 million on June 30, 2025.
EARNINGS REPORT CALL:
A conference call will be held today, August 5, 2025, at 4:30 p.m. Eastern Time to provide a company update and discuss the financial results.
The link to register is View Source
Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.
A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting: View Source

On August 5, 2025 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported financial results for the second quarter ended June 30, 2025 (Press release, Cogent Biosciences, AUG 5, 2025, View Source [SID1234654781]).

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"We were thrilled to announce bezuclastinib’s impressive performance in the SUMMIT trial, demonstrating clinically meaningful and statistically significant results across all primary and key secondary endpoints," said Andrew Robbins, the Company’s President and Chief Executive Officer. "These positive data along with the favorable safety profile give us confidence that bezuclastinib has the potential to become the new standard-of-care for NonAdvSM patients. Supported by our recent upsized public offering, Cogent is advancing our mission from a position of strength as we prepare to report top-line results from two additional pivotal trials in GIST and AdvSM in the second half of this year, submit our first New Drug Application by the end of 2025 and make continued progress toward the anticipated commercial launch of bezuclastinib in 2026."

Recent Business Highlights

•
Announced positive top-line results from the registration-directed Part 2 of the SUMMIT clinical trial in NonAdvanced Systemic Mastocytosis (NonAdvSM) patients.
o
The SUMMIT trial, which was designed to assess the clinical benefit of bezuclastinib versus placebo, achieved its primary endpoint with a highly statistically significant difference in the mean change in Total Symptom Score (TSS) at 24 weeks (p=0.0002). TSS was assessed by the Mastocytosis Symptom Severity Daily Diary (MS2D2). The bezuclastinib arm had a mean reduction of 24.3 points in TSS at 24 weeks, versus the placebo arm which had a mean reduction of 15.4 points in TSS, resulting in a placebo-adjusted TSS improvement of 8.91 points. In addition, the SUMMIT trial demonstrated highly statistically significant benefit across all key secondary endpoints, including reduction of serum tryptase on which 87.4% of bezuclastinib-treated patients had ≥50% reduction, compared to no patients in the control arm (87.4% vs. 0%; p<0.0001).
o
Bezuclastinib demonstrated a favorable safety and tolerability profile in SUMMIT.

▪
The majority of treatment emergent adverse events (TEAEs) (98.3% in bezuclastinib arm vs. 88.3% in placebo arm) were of low grade. The most frequent TEAEs reported on bezuclastinib treatment were hair color change (69.5% bezuclastinib vs. 5.0% placebo), altered taste (23.7% bezuclastinib vs. 0% placebo), nausea (22.0% bezuclastinib vs. 13.3% placebo) and ALT/AST elevations (22.0% bezuclastinib vs. 6.6% placebo; >Gr 3, 5.9% vs. 0%). Serious AEs occurred in 4.2% of patients treated with bezuclastinib, compared to 5.0% of patients treated with placebo. Discontinuations due to treatment-related AEs occurred in 5.9% of patients treated with bezuclastinib, all due to ALT/AST elevations and all patients fully resolved. There were no hepatic AEs reported in any patient other than transient and manageable lab abnormalities.
•
Announced two financial transactions, positioning Cogent with access to over $800 million in capital.
o
In June, secured a debt financing facility of up to $400 million with SLR Capital Partners. An initial tranche of $50 million was drawn at closing in June 2025, with additional tranches available upon achieving key clinical and commercial milestones.
o
In July, successfully closed an upsized underwritten public offering of 25,555,556 shares of common stock at $9.00 per share, including the full exercise of the underwriters’ option to purchase an additional 3,333,333 shares. This offering generated net proceeds of $215.8 million.
Anticipated Upcoming Milestones

•
Announce top-line results from PEAK in the second half of 2025. PEAK is a global, blinded, randomized Phase 3 clinical trial studying the combination of bezuclastinib and sunitinib versus sunitinib alone in patients with imatinib-resistant gastrointestinal stromal tumors (GIST).
•
Announce top-line results from APEX in the second half of 2025. APEX is a registration-directed, global, open-label trial in patients with advanced systemic mastocytosis (AdvSM).
•
Submit Cogent’s first NDA for bezuclastinib by the end of 2025.

Second Quarter 2025 Financial Results

Cash Position: As of June 30, 2025, Cogent had cash, cash equivalents and marketable securities of $237.8 million. The company expects its existing cash, cash equivalents and marketable securities, together with the net proceeds from the $230 million upsized public offering in July 2025, will be sufficient to fund its operating expenses and capital expenditure requirements into 2027, including through potential FDA approval of bezuclastinib for NonAdvSM and early commercial launch activities.

R&D Expenses: Research and development expenses were $62.2 million for the second quarter of 2025 as compared to $54.3 million for the second quarter of 2024. The increase was primarily due to costs incurred to support our on-going SUMMIT, PEAK and APEX clinical trials and to the continued progression of our early stage, preclinical and discovery programs. R&D expenses include non-cash stock compensation expense of $5.0 million for the second quarter of 2025 as compared to $4.7 million for the second quarter of 2024.

G&A Expenses: General and administrative expenses were $13.4 million for the second quarter of 2025 as compared to $10.1 million for the second quarter of 2024. The increase was primarily due to the growth of the organization. G&A expenses include non-cash stock compensation expense of $4.8 million for the second quarter of 2025 as compared to $5.3 million for the second quarter of 2024.

Net Loss: Net loss was $73.5 million for the second quarter of 2025 as compared to a net loss of $59.0 million for the same period of 2024.

On August 5, 2025 Cerus Corporation (Nasdaq:CERS) reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Cerus, AUG 5, 2025, View Source [SID1234654780]).

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"With our singular focus to advance blood safety and availability around the globe by establishing INTERCEPT as the standard of care, this quarter’s commercial results are evidence of the progress we are making in multiple geographies and across our INTERCEPT product portfolio. The early launch and adoption of our next generation, LED-based INT200 illumination device continues to surpass our expectations around performance and customer experience, and advancements in our product development pipeline are encouraging, including the early resubmission for RBC CE Mark and the recent DoD award for the CRYO-First study," said William "Obi" Greenman, Cerus’ president and chief executive officer.

"In addition, the continued strong commercial and financial execution in Q2 resulted in record product sales, a year-over-year narrowing of net loss attributable to Cerus, and another quarter of positive non-GAAP adjusted EBITDA," continued Greenman. "Given the anticipated sales trajectory, we are raising our full-year 2025 product revenue guidance today."

Additional highlights include:

Second-quarter 2025 total revenue comprised of (in millions, except percentages):
Three Months Ended

Six Months Ended

June 30,

Change

June 30,

Change

2025

2024

$

%

2025

2024

$

%

Product Revenue

$

52.4

$

45.1

$

7.4

16

%

$

95.7

$

83.4

$

12.2

15

%

Government Contract Revenue

7.7

5.4

2.2

41

%

13.3

10.5

2.8

27

%

Total Revenue

$

60.1

$

50.5

$

9.6

19

%

$

109.0

$

93.9

$

15.1

16

%

Numbers may not sum due to rounding. Percentages calculated from unrounded figures.

Customer demand for Pathogen Reduced, Cryoprecipitated Fibrinogen Complex (commonly referred to as INTERCEPT Fibrinogen Complex, or IFC) continues to increase with second quarter IFC revenue of $5.6 million compared to $2.0 million in the prior year period.
The European regulatory review of our CE Mark application for the INTERCEPT Blood System for Red Blood Cells (RBCs) continues. TÜV-SÜD, the Notified Body, has completed their review of several modules of the submission, including the clinical module, and transferred the dossier to the State Institute for Drug Control (SÚKL), our Competent Authority, in the Czech Republic, for consultation.
Awarded an additional $7.2 million in funding from the U.S. Department of Defense Industrial Base Analysis and Sustainment (IBAS) program for the development of lyophilized IFC.
Second-quarter net loss attributable to Cerus Corporation was $5.7 million. Second-quarter non-GAAP adjusted EBITDA of $0.9 million, marking another quarter of positive non-GAAP adjusted EBITDA.
Cash, cash equivalents, and short-term investments were $78.0 million at June 30, 2025.
Revenue

Product revenue during the second quarter of 2025 was $52.4 million, compared to $45.1 million during the prior year period. This year-over-year increase of 16% was led by growth in IFC and global platelet sales. Second-quarter product revenue included U.S. sales of IFC, which were $5.6 million, up from $2.0 million during the prior year period.

Second-quarter 2025 government contract revenue was $7.7 million, compared to $5.4 million during the prior year period. Our government contract revenue was comprised of funding associated with research and development (R&D) activities related to the INTERCEPT RBC program as well as efforts related to the development of next-generation pathogen reduction technology to treat whole-blood and development of lyophilized IFC. The year-over-year increase was primarily driven by increasing enrollment in the Phase 3 RedeS trial for the INTERCEPT RBC system covered under the Company’s 2016 agreement with BARDA and the activities for the advancement of the INTERCEPT RBC system covered under the Company’s 2024 BARDA contract.

Product Gross Profit and Margin

Product gross profit for the second quarter of 2025 was $29.0 million, increasing by 17% over the prior year period. Product gross margin for the second quarter of 2025 was 55.2% compared to 54.7% for the second quarter of 2024. A number of offsetting factors resulted in relatively stable product gross margin in the second quarter of 2025.

Operating Expenses

Total operating expenses for the second quarter of 2025 were $40.1 million, compared to $33.9 million for the same period of the prior year, reflecting a year-over-year increase of 18%. Both R&D and selling, general, and administrative (SG&A) expenses increased year-over-year, reflecting investments in our business to drive future revenue growth.

R&D expenses for the second quarter of 2025 were $18.9 million, compared to $15.0 million for the second quarter of 2024. The primary drivers for the increase in R&D expenses were related to development costs on INT200, the new LED-based illumination device, higher government contract costs incurred to support the higher government contract revenue and higher employee compensation expenses tied to cost-of-living adjustments which became effective earlier in the year.

SG&A expenses totaled $21.2 million for the second quarter of 2025, compared to $19.0 million for the second quarter of 2024. The primary driver for the increase in SG&A expenses was higher employee compensation expenses tied to cost-of-living adjustments which became effective earlier in the year.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the second quarter of 2025 was $5.7 million, or $0.03 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $5.8 million, or $0.03 per basic and diluted share, for the same period of the prior year. Net loss attributable to Cerus Corporation for the first half of 2025 was $13.4 million compared to net loss attributable to Cerus Corporation of $15.5 million for the first half of 2024.

Non-GAAP Adjusted EBITDA

Non-GAAP adjusted EBITDA for the second quarter of 2025 was positive $0.9 million, compared to non-GAAP adjusted EBITDA of positive $0.8 million for the same period of the prior year. Non-GAAP adjusted EBITDA for the first half of 2025 was a positive $1.1 million compared to non-GAAP adjusted EBITDA of negative $1.9 million for the first half of 2024.

The Company remains committed to its goal of achieving positive, full-year 2025 non-GAAP adjusted EBITDA. For additional information, please see definitions and the reconciliation of this non-GAAP measure to net loss attributable to Cerus Corporation accompanying this release.

Balance Sheet and Cash Flows

At June 30, 2025, the Company had cash, cash equivalents, and short-term investments of $78.0 million, compared to $80.5 million at December 31, 2024. The Company’s revolving line of credit allows for an additional $15.0 million as of June 30, 2025, which is dependent on eligible assets supporting the borrowing base.

For the second quarter of 2025, cash used from operations totaled $2.4 million compared to $0.4 million generated during the same period of the prior year. Cash use from operations in the second quarter of 2025 was tied to an increase in working capital, namely inventory in support of the expected growth.

Increasing Full-Year 2025 Product Revenue Guidance

Given the strong performance during the first half of 2025, coupled with increasing conviction about expected second half growth, the Company now expects full-year 2025 product revenue will be in the range of $200 million to $203 million. These revisions include increased IFC guidance, which the Company now expects to be between $16 million to $18 million for 2025. Previously, the Company’s 2025 product revenue guidance range was $194 million to $200 million, including 2025 IFC revenue guidance between $12 million and $15 million.

Quarterly conference Call

The Company will host a conference call at 4:30 P.M. ET this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source

A replay will be available on Cerus’ website approximately three hours after the call through August 26, 2025.

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):

•
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.
•
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.
•
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).
•
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):

•
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.
•
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.
•
Last participant last visit has been achieved, and the topline results of the interim analysis cohort are expected in fall 2025.
•
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.
•
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:

•
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.
•
BridgeBio expects topline results of CALIBRATE in the fall 2025.
•
If successful, encaleret would be the first approved therapy for individuals living with ADH1.
•
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.
•
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:

•
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 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.
•
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.
•
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.
•
If successful, infigratinib would be the first approved oral therapy option for children living with achondroplasia and hypochondroplasia.

Corporate Updates:

•
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).
•
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.

On August 5, 2025 Boundless Bio (Nasdaq: BOLD), a clinical-stage oncology company interrogating extrachromosomal DNA (ecDNA) biology to deliver transformative therapies to patients with previously intractable oncogene amplified cancers, reported financial results and business highlights for the fiscal quarter ended June 30, 2025 (Press release, Boundless Bio, AUG 5, 2025, View Source [SID1234654778]).

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"We are executing with sharpened focus on programs that we believe have the strongest scientific rationale and greatest potential to impact patients with oncogene-amplified cancers," said Zachary Hornby, President and CEO of Boundless Bio. "We are excited to advance our BBI-355/BBI-825 combination in the clinic and to progress BBI-940, our development candidate in our novel kinesin program, toward IND submission, as we work to make a meaningful impact for both patients and shareholders."

Research and Development Highlights and Upcoming Milestones

POTENTIATE clinical trial

•
The Company believes recent preclinical data provide a strong mechanistic rationale to combine BBI-355, its novel, selective, oral CHK1 inhibitor, with BBI-825, its novel, selective, oral RNR inhibitor, for synergistic anti-tumor activity without overlapping toxicity, and with a dosing regimen that does not require continuous administration.
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The BBI-355/BBI-825 combination arm of the POTENTIATE trial is open for enrollment. The Company expects to deliver initial proof-of-concept clinical data within its existing cash runway timeline.

Novel Kinesin program targeting ecDNA segregation and inheritance

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Boundless selected BBI-940 as its development candidate for its novel program targeting a previously undrugged kinesin.
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Boundless expects to submit an investigational new drug (IND) application for BBI-940 in the first half of 2026 and to deliver initial proof-of-concept clinical data within its existing cash runway timeline.

Second Quarter 2025 Financial Results

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Cash Position: Cash, cash equivalents, and short-term investments totaled $127.1 million as of June 30, 2025.
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Research and Development (R&D) Expenses: R&D expenses were $12.2 million for the second quarter of 2025, compared to $14.7 million for the same period in 2024.
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General and Administrative (G&A) Expenses: G&A expenses were $4.8 million for the second quarter of 2025, compared to $4.7 million for the same period in 2024.
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Net Loss: Net loss totaled $15.7 million for the second quarter of 2025, compared to $17.0 for the same period in 2024.