On August 5, 2025 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported that new data will be presented at the IASLC 2025 World Conference on Lung Cancer (WCLC) taking place September 6–9, 2025 in Barcelona, Spain, and the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress Meeting (ESMO) (Free ESMO Whitepaper) October 17–21, 2025 in Berlin, Germany (Press release, Nuvation Bio, AUG 5, 2025, View Source [SID1234654816]). These data include new results from the pivotal Phase 2 TRUST-I and TRUST-II studies on the efficacy and safety of IBTROZI (taletrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC).

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"We’re proud of the meaningful impact IBTROZI, a highly selective, next-generation oral tyrosine kinase inhibitor, has had on the ROS1+ NSCLC community following its recent FDA approval," said David Hung, M.D., Founder, President and Chief Executive Officer of Nuvation Bio. "We look forward to presenting new data from our pivotal trials that further reinforce the established efficacy and safety profiles of IBTROZI across multiple patient groups living with ROS1+ NSCLC and that offer additional preclinical and pharmacologic insights. These findings underscore the value IBTROZI brings as an important treatment option for people with this disease."

Presentations Overview:

WCLC

Title: Updated Efficacy and Safety of Taletrectinib in Patients With ROS1+ Non-Small Cell Lung Cancer: The Global TRUST-II Study

Presenter: Geoffrey Liu, M.D., Princess Margaret Cancer Centre, Temerty School of Medicine, University of Toronto

Session Category: MA02 – New Treatment Strategies in Other Than EGFR-Positive Tumors

Date: Sunday, September 7, 2025

Session Time: 12:00 – 1:15 p.m. CEST

Title: Updated Efficacy and Safety of Taletrectinib in Chinese Patients With ROS1+ Non-Small Cell Lung Cancer: Phase 2 TRUST-I Study

Presenter: Wei Li, M.D., Shanghai East Hospital, Tongji University

Session Category: P3.12 – Metastatic Non-small Cell Lung Cancer – Targeted Therapy

Date: Tuesday, September 9, 2025

Session Time: 10:00 – 11:30 a.m. CEST

Title: TRUST-III: Phase 3 Head-to-Head Study of Taletrectinib vs Crizotinib in Patients with ROS1+ Non-Small Cell Lung Cancer

Presenter: Caicun Zhou, M.D., Ph.D., Shanghai East Hospital, Tongji University

Session Category: P3.18 – Clinical Trials in Progress

Date: Tuesday, September 9, 2025

Session Time: 10:00 – 11:30 a.m. CEST

Title: Clinical Pharmacologic Characteristics of Taletrectinib

Presenter: Maurice Perol, M.D., Department of Medical Oncology, Léon Bérard Cancer Center, Lyon, France

Session Category: P3.12 – Metastatic Non-small Cell Lung Cancer – Targeted Therapy

Date: Tuesday, September 9, 2025

Session Time: 10:00 – 11:30 a.m. CEST

Title: Taletrectinib, a Next Generation Selective ROS1 inhibitor, Exhibits a Differentiated Profile in ROS1 Fusion Models

Presenter: Hitisha Patel, Ph.D., Director of Research, Nuvation Bio

Session Category: P3.12 – Metastatic Non-small Cell Lung Cancer – Targeted Therapy

Date: Tuesday, September 9, 2025

Session Time: 10:00 – 11:30 a.m. CEST

ESMO

Title: TRUST-II Global Study: Efficacy and Safety of Taletrectinib After Prior Entrectinib Exposure in Patients with Advanced ROS1+ Non-Small Cell Lung Cancer

Session Category: Saturday Poster Session

Presenter: Filippo De Braud, M.D., University of Milan, Milan, Italy

Date: Saturday, October 18, 2025

Session Time: 12:00 – 12:45 p.m. CEST

At WCLC, Nuvation Bio will also sponsor a satellite symposium, titled "Under the Microscope: Focusing on ROS1+ NSCLC," on Sunday, September 7 from 1:45 – 2:45 p.m. CEST. Presenters include Charu Aggarwal, M.D., MPH, University of Pennsylvania; Jüergen Wolf, M.D., University Hospital Cologne; and Javier deCastro, M.D., La Paz University Hospital.

The materials will be made available in the Publications section of Nuvation Bio’s website after the presentations. To learn more about Nuvation Bio, visit Booth #201 at WCLC and Booth #4018 at ESMO (Free ESMO Whitepaper).

About ROS1+ NSCLC
Each year, more than one million people globally are diagnosed with non-small cell lung cancer (NSCLC), the most common form of lung cancer. It is estimated that approximately 2% of patients with NSCLC have ROS1+ disease. About 35% of patients newly diagnosed with metastatic ROS1+ NSCLC have tumors that have spread to their brain. The brain is also the most common site of disease progression, with about 50% of previously treated patients developing CNS metastases. Despite recent progress for patients with ROS1+ NSCLC, there remains a need for more effective and tolerable treatment options.

About IBTROZI
IBTROZI is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor therapy approved for the treatment of adult patients with advanced ROS1-positive non-small cell lung cancer. Learn more at IBTROZI.com.

About the TRUST Clinical Program
The TRUST clinical program evaluating IBTROZI for the treatment of adult patients with advanced ROS1+ NSCLC included two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, which enrolled 173 patients, and TRUST-II (NCT04919811), a global study, which enrolled 164 patients. The primary endpoint of these registrational studies is confirmed objective response rate (cORR) as assessed by an independent review committee (IRC). Secondary endpoints include intracranial cORR, duration of response, progression-free survival, and safety.

Indication
IBTROZI is indicated for the treatment of adult patients with locally advanced or metastatic ROS1+ non-small cell lung cancer (NSCLC).

IMPORTANT SAFETY INFORMATION FOR IBTROZITM (taletrectinib)

WARNINGS AND PRECAUTIONS

Hepatotoxicity: Hepatotoxicity, including drug-induced liver injury and fatal adverse reactions, can occur. 88% of patients experienced increased AST, including 10% Grade 3/4. 85% of patients experienced increased ALT, including 13% Grade 3/4. Fatal liver events occurred in 0.6% of patients. Median time to first onset of AST or ALT elevation was 15 days (range: 3 days to 20.8 months).

Increased AST or ALT each led to dose interruption in 7% of patients and dose reduction in 5% and 9% of patients, respectively. Permanent discontinuation was caused by increased AST, ALT, or bilirubin each in 0.3% and by hepatotoxicity in 0.6% of patients.

Concurrent elevations in AST or ALT ≥3 times the ULN and total bilirubin ≥2 times the ULN, with normal alkaline phosphatase, occurred in 0.6% of patients.

Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, or fatal ILD or pneumonitis can occur. ILD/pneumonitis occurred in 2.3% of patients, including 1.1% Grade 3/4. One fatal ILD case occurred at the 400 mg daily dose. Median time to first onset of ILD/pneumonitis was 3.8 months (range: 12 days to 11.8 months).

ILD/pneumonitis led to dose interruption in 1.1% of patients, dose reduction in 0.6% of patients, and permanent discontinuation in 0.6% of patients.

QTc Interval Prolongation: QTc interval prolongation can occur, which can increase the risk for ventricular tachyarrhythmias (e.g., torsades de pointes) or sudden death. IBTROZI prolongs the QTc interval in a concentration-dependent manner.

In patients who received IBTROZI and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 13% and 2.6% of patients, respectively. 3.4% of patients experienced Grade ≥3. Median time from first dose of IBTROZI to onset of ECG QT prolongation was 22 days (range: 1 day to 38.7 months). Dose interruption and dose reduction each occurred in 2.8% of patients.

Significant QTc interval prolongation may occur when IBTROZI is taken with food, strong and moderate CYP3A inhibitors, and/or drugs with a known potential to prolong QTc. Administer IBTROZI on an empty stomach. Avoid concomitant use with strong and moderate CYP3A inhibitors and/or drugs with a known potential to prolong QTc.​

Hyperuricemia: Hyperuricemia can occur and was reported in 14% of patients, with 16% of these requiring urate-lowering medication without pre-existing gout or hyperuricemia. 0.3% of patients experienced Grade ≥3. Median time to first onset was 2.1 months (range: 7 days to 35.8 months). Dose interruption occurred in 0.3% of patients.

Myalgia with Creatine Phosphokinase (CPK) Elevation: Myalgia with or without CPK elevation can occur. Myalgia occurred in 10% of patients. Median time to first onset was 11 days (range: 2 days to 10 months).

Concurrent myalgia with increased CPK within a 7-day time period occurred in 0.9% of patients. Dose interruption occurred in 0.3% of patients with myalgia and concurrent CPK elevation.

Skeletal Fractures: IBTROZI can increase the risk of fractures. ROS1 inhibitors as a class have been associated with skeletal fractures. 3.4% of patients experienced fractures, including 1.4% Grade 3. Some fractures occurred in the setting of a fall or other predisposing factors. Median time to first onset of fracture was 10.7 months (range: 26 days to 29.1 months). Dose interruption occurred in 0.3% of patients.

Embryo-Fetal Toxicity: Based on literature, animal studies, and its mechanism of action, IBTROZI can cause fetal harm when administered to a pregnant woman.

ADVERSE REACTIONS
Among patients who received IBTROZI, the most frequently reported adverse reactions (≥20%) were diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%). ​

The most frequently reported Grade 3/4 laboratory abnormalities (≥5%) were increased ALT (13%), increased AST (10%), decreased neutrophils (5%), and increased creatine phosphokinase (5%). ​

DRUG INTERACTIONS

Strong and Moderate CYP3A Inhibitors/CYP3A Inducers and Drugs that Prolong the QTc Interval: Avoid concomitant use.
Gastric Acid Reducing Agents: Avoid concomitant use with PPIs and H2 receptor antagonists. If an acid-reducing agent cannot be avoided, administer locally acting antacids at least 2 hours before or 2 hours after taking IBTROZI.
OTHER CONSIDERATIONS

Pregnancy: Please see important information in Warnings and Precautions under Embryo-Fetal Toxicity. ​
Lactation: Advise women not to breastfeed during treatment and for 3 weeks after the last dose.
Effect on Fertility: Based on findings in animals, IBTROZI may impair fertility in males and females. The effects on animal fertility were reversible.
Pediatric Use: The safety and effectiveness of IBTROZI in pediatric patients has not been established.
Photosensitivity: IBTROZI can cause photosensitivity. Advise patients to minimize sun exposure and to use sun protection, including broad-spectrum sunscreen, during treatment and for at least 5 days after discontinuation.
Please see accompanying full Prescribing Information.

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 Recursion (Nasdaq: RXRX), a leading clinical stage TechBio company decoding biology to radically improve lives, reported business updates and financial results for its second quarter ended June 30, 2025 (Press release, Recursion Pharmaceuticals, AUG 5, 2025, View Source [SID1234654795]).

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Recursion will host a (L)earnings Call on August 5, 2025 at 8:00 am ET / 6:00 am MT / 1:00 pm BST from Recursion’s X (formerly Twitter), LinkedIn, and YouTube accounts giving analysts, investors, and the public the opportunity to ask questions of the company by submitting questions here: View Source

"The power of our platform not only allows us to discover and develop potential new medicines, but also gives us insights on patient populations to target that would be challenging using traditional methods," said Chris Gibson, Co-Founder and CEO of Recursion. "In discovery, we’re deploying advanced models like Boltz-2 to rapidly design ligands for high-value targets. State of the art platform capabilities helped us drive our fourth partnered discovery milestone with Sanofi this quarter, reflecting tangible momentum across our joint pipeline. We are leveraging these and other improvements to the Recursion OS to not only accelerate and improve our funnel of new programs, but also execution of later stage programs in our pipeline like RBM39 and CDK7."

Summary of Business Highlights

Portfolio – Internal and Partnered Programs

"REC-1245, our potential first-in-class RBM39 degrader, was identified using phenomap-derived insight, and mimics CDK12 loss to induce replication stress and suppress DDR pathways without CDK12 related toxicities. Early data show strong activity in tumors characterized by replication stress and DNA repair vulnerabilities. Our DAHLIA trial is now enrolling select tumor types to identify responsive populations. For REC-617, our CDK7 inhibitor, we leveraged multi-omic and real world patient data and causal AI modeling to select platinum-resistant ovarian cancer as the first combination cohort," said Najat Khan, PhD, Chief R&D Officer and Chief Commercial Officer of Recursion.

Internal Pipeline Updates:
•REC-1245 (RBM39): Recursion provided updates on the biomarker strategy and patient population currently enrolling in the ongoing Phase 1/2 DAHLIA study.
◦About REC-1245
▪Potential first-in-class oral RBM39 degrader that selectively impairs alternative splicing to silence multiple DDR pathways, leading to high replication stress.
▪Characterized to selectively mimic the phenotype associated with CDK12 loss of function using Recursion’s AI-powered maps of human biology.
◦Update on target patient population
▪Early preclinical data shows REC-1245 reduces viability in tumors characterized by replication stress and DNA repair vulnerabilities (DDR defects) across multiple solid tumor types, including MSI-H/dMMR, HRR altered cancers, and other tumors.
▪Multi‑omic profiling underway to refine the molecular signature of sensitivity.
◦Additional DAHLIA trial details

▪Monotherapy dose-escalation of Phase 1/2 DAHLIA trial in patients with advanced solid tumors ongoing.
▪Early safety and PK data from the Phase 1 dose-escalation portion on track for 1H26.
•REC-617 (CDK7): Recursion initiated a combination dose escalation portion of the ELUCIDATE Phase 1/2 trial in 1H25.
◦About REC-617
▪Orally bioavailable, highly potent, and selective CDK7 inhibitor with best-in-class potential.
▪Precision-designed using Recursion’s generative AI and active learning platform to optimize for non-covalent binding and ADME/PK, potentially delivering a broader therapeutic window, reduced off-target effects, and enhanced absorption.
▪Early Phase 1/2 results demonstrated promising safety and efficacy signals, including a durable partial response in a late-stage metastatic ovarian cancer patient and stable disease across four other patients with solid tumors (e.g. CRC, NSCLC).
◦Update on target patient population
▪Based on early clinical, preclinical, and causal AI modeling data, Recursion selected ovarian cancer as the initial combination dose expansion cohort.
◦Additional ELUCIDATE combination trial details
▪REC-617 in combination with standards of care in 2L+ platinum-resistant ovarian cancer population. Enrollment activities have been initiated.
▪Additional tumor types and therapies for single-arm expansion cohorts under evaluation.
▪Additional data from monotherapy dose-escalation on-track for 2H25.
•REC-102 (ENPP1): Acquired full rights to REC-102, Recursion’s ENPP1 inhibitor for the treatment of hypophosphatasia (HPP), from its joint venture with Rallybio.
◦REC-102 is the first potential oral disease-modifying treatment for HPP, a rare and debilitating genetic disorder with limited treatment options.
◦Additional preclinical data from the REC-102 program will be presented at the 2025 American Society for Bone and Mineral Research (ASMBR), being held in Seattle, WA.
▪A poster titled Amelioration of osteomalacia in late-onset HPP mice via pharmacological inhibition of ENPP1 is scheduled for presentation on September 6, 2025 between 2:00 PM – 3:30 PM PT, during the Basic and Translational session.
◦Phase 1 initiation remains on-track for 2H26.

Upcoming milestones:
•REC-4881 (MEK1/2): Additional data in FAP from TUPELO expected in 2H25.
•REC-617 (CDK7): Additional monotherapy data expected in 2H25.

•REC-7735 (PI3Kα H1047R): Preclinical studies ongoing with development candidate expected in 2H25.
•REC-1245 (RBM39): Early Phase 1 safety and PK monotherapy data expected in 1H26.
•REC-3565 (MALT1): Early Phase 1 safety and PK monotherapy data expected in 2H26.
•REC-102 (ENPP1): Phase 1 initiation expected in 2H26.
•Potential for over $100 million in partnership milestones by the end of 2026.
◦Several programs are advancing towards potential development candidate designation over the next 12-15 months.
◦Multiple neuroscience target validation programs advancing by leveraging the RecursionOS.
Partnered Discovery Updates:
•Sanofi: Recursion and Sanofi continue to advance multi-target collaboration for up to 15 best-in-class or first-in-class programs across oncology and immunology, with $130 million in upfront and milestone payments achieved to date. Each program has the potential for over $300 million in milestone payments.
◦In 2Q, achieved a $7 million milestone payment for an immunology program. Under the collaboration, this is the fourth partnered program reaching a significant discovery milestone in 18 months.
◦Sanofi is now leveraging combined RecursionOS 2.0, including phenomics, to identify new program opportunities.
◦Several programs are advancing towards potential development candidate designation over the next 12-15 months.
•Roche and Genentech: Recursion continues to make meaningful progress on both building additional neuromaps and driving target validation and small molecule programs in a single GI oncology indication.
◦Neuro: To date, the collaboration has built a whole-genome knockout phenomap derived from over one trillion iPSC-derived neural cells, alongside around 5,000 transcriptomes representing approximately 171 TB of data.
▪Potential neuroscience targets have been identified for validation from the map, and today multiple novel target validation programs are advancing leveraging the RecursionOS and Genentech’s biological expertise.
▪Building additional neuromaps, including multi-modal maps, combining Roche and Genentech’s expertise in single cell screens with Recursion’s and Genentech’s multi-omic machine learning capabilities.
◦GI-Oncology: To date, Recursion has generated all whole genome scale and small molecule GI-oncology specific phenomaps contemplated in the partnership, from which both novel target and small molecule programs can be surfaced.
◦One optioned program continues to advance toward lead series.
◦Focused on advancing multiple novel target and/or compound programs.
•Bayer: Recursion and Bayer have nominated multiple early discovery precision oncology programs against previously "undruggable" targets. Work is underway to advance multiple programs to lead series milestone decisions.
•Merck KgAa, Darmstadt, Germany: Collaboration ongoing to identify first-in-class and best-in-class targets.

Recursion OS 2.0: The platform is continuing to drive program development with applications across biology, chemistry and clinical development.
•Actively expanding the Virtual Cell to understand and predict cellular behavior across a wider range of biology.
◦Boltz-2 open source model released with MIT and Nvidia to commoditize state of the art performance for binding affinity prediction approaching the accuracy of physics-based free energy perturbation (FEP) calculations while being over 1,000 times faster and less computationally expensive. The open source tool has been downloaded by over 40,000 unique users to date.
◦Incorporating diverse cell types beyond HUVEC and disease areas beyond oncology, to discover more novel biology and new medicines.
•Recursion continues to expand its ClinTech platform, integrating high-quality, linked patient datasets like Tempus, HealthVerity, and Helix to strengthen programs, bolster preclinical and early clinical data to select patients (e.g., for REC-617), and optimize recruitment.

Second Quarter 2025 Financial Results

•Cash Position: Cash, cash equivalents and restricted cash were $533.8 million as of June 30, 2025 compared to $603.0 million as of December 31, 2024. Based on current operating plans, the Company believes that its expected cash runway will extend into the fourth quarter of 2027.
•Revenue: Total revenue, consisting primarily of revenue from collaboration agreements, was $19.2 million for the second quarter of 2025, compared to $14.4 million for the second quarter of 2024.
•Research and Development Expenses: Research and development expenses were $128.6 million for the second quarter of 2025, compared to $73.9 million for the second quarter of 2024. The increase was primarily driven by the Company’s agreement with Tempus as well as its business combination with Exscientia in November 2024. This includes recognition of $22.7 million in non-cash expenses for use of Tempus’ patient-centric multimodal oncology data under the companies’ ongoing collaboration.

•General and Administrative Expenses: General and administrative expenses were $46.7 million for the second quarter of 2025 compared to $31.8 million for the second quarter of 2024. The increase compared to the prior period was primarily due to the inclusion of G&A expenses from the business combination with Exscientia.
•Net Loss: Net loss was $171.9 million for the second quarter of 2025, compared to a net loss of $97.5 million for the second quarter of 2024.
•Operational cash flows: Net cash used in operating activities was $208.4 million for the six months ended June 30, 2025, compared to net cash used in operating activities of $184.5 million for the six months ended June 30, 2024. The increase in cash used in operating activities was primarily driven by the inclusion of Exscientia’s operations, for which the business combination with Recursion closed in November 2024.This was partially offset by cash inflows from partnerships and operational tax rebates totaling $7.0 million and $28.6 million respectively for the first three and six months of 2025. No cash inflows from partnerships or operational tax rebates were recorded during the six months ended June 30, 2024. In association with the restructuring activities announced in June 2025, the Company expects to incur costs totalling $9.3 million, of which $3.9 million has been paid in the second quarter of 2025. Recursion expects to incur all of these expenses in the year ending December 31, 2025.

On August 5, 2025 Eureka Therapeutics, Inc., a clinical-stage biotechnology company pioneering safer and more effective T-cell therapies, reported the appointment of Samuel So, MBBS, FACS, a globally recognized leader in liver cancer surgery, prevention, and health policy at Stanford University, to its Scientific Advisory Board (Press release, Eureka Therapeutics, AUG 5, 2025, View Source [SID1234654817]). Dr. So will provide strategic guidance as Eureka advances its ARTEMIS CAR-T platform and clinical programs targeting hepatocellular carcinoma (HCC), a cancer with significant unmet medical need and limited treatment options.

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Dr. So is the Lui Hac Minh Professor and Professor of Surgery at Stanford University. He founded the multidisciplinary liver cancer program at Stanford Cancer Center and is the founder and executive director of the Asian Liver Center at Stanford School of Medicine, the first U.S. nonprofit dedicated to addressing disproportionately high rates of chronic hepatitis B and liver cancer in Asian populations. He has led major international health initiatives, published extensively in leading journals, and served as a consultant and committee member of many agencies including the U.S. Food and Drug Administration, Institute of Medicine and the World Health Organization. His leadership in viral hepatitis and liver cancer prevention has earned numerous honors, including the CDC and ATSDR Honor Award for advancing global health, and a White House commendation for leadership in viral hepatitis prevention and treatment.

"Eureka’s innovative approach to CAR-T therapy for HCC has the potential to redefine how we treat this disease," said Dr. So. "I look forward to collaborating with the team to accelerate development and deliver new options for patients worldwide."

"Dr. So’s expertise in liver cancer biology and clinical strategy will be instrumental as we advance our ARTEMIS CAR-T programs for HCC," said Cheng Liu, Ph.D., Founder and CEO of Eureka Therapeutics. "His guidance will help us execute on our development plans and strengthen Eureka’s position as a leader in cell therapy."

Eureka’s proprietary ARTEMIS CAR-T platform is designed to overcome key limitations of conventional CAR-T therapies by reducing toxicity to enable higher doses and repeat infusions, improving T-cell persistence for longer-lasting responses, and enhancing tumor infiltration to achieve more potent anti-tumor activity. Eureka’s pipeline includes ET140203 (ARYA-2) for pediatric patients and ECT204 (ARYA-3) for adults, both in Phase I/II clinical trials for advanced liver cancer.

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.