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Jazz Pharmaceuticals Announces U.S. FDA Approval of Modeyso™ (dordaviprone) as the First and Only Treatment for Recurrent H3 K27M-mutant Diffuse Midline Glioma

On August 6, 2025 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Modeyso (dordaviprone) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy (Press release, Jazz Pharmaceuticals, AUG 6, 2025, View Source [SID1234654889]). Continued approval for this indication may be contingent upon verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial.

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Modeyso is the first and only treatment option approved by the FDA for this ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, many of whom are children and young adults.2 The disease is characterized by rapid progression and historically has had no effective systemic treatment options.3 To address this urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.

"This is a major turning point in neuro-oncology," said Patrick Wen, M.D., Director, Center for Neuro-Oncology, Dana-Farber Cancer Institute and Professor of Neurology, Harvard Medical School. "For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma. While outcomes remain challenging for many patients, the objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement. This therapy was developed with the underlying biology of the tumor in mind and introduces a new treatment option for a population with historically limited choices."

Modeyso is administered as an oral capsule once weekly. The FDA’s decision was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma, selected from five open-label clinical studies based on prespecified eligibility criteria. The overall response rate (ORR), as assessed by blinded independent central review (BICR) using Response Assessment in Neuro-Oncology (RANO) 2.0 criteria, was 22% (95% CI: 12 to 36), with an additional responder identified by integrated RANO 2.0. Among responders, the median duration of response was 10.3 months (95% CI: 7.3 to 15.2), with 73% maintaining their response for at least six months and 27% for at least 12 months.1

"The FDA approval of Modeyso is a milestone moment for the patients and families who have long needed new options, the clinicians who have tirelessly searched for solutions, and the researchers and advocates who never gave up," said Joshua E. Allen, Ph.D., Chief Scientific Officer, Chimerix, a Jazz Pharmaceuticals Company. "We’re proud to deliver precisely the kind of transformative innovation we strive for, and we congratulate our combined Chimerix and Jazz team, and the community who worked together tirelessly to bring this treatment forward. This approval not only equips clinicians with the first targeted option for this disease but also signals a meaningful shift in what patients and families can expect after diagnosis. We would like to extend our thanks to the patients, advocates, clinicians, principal investigators, scientists, regulators and partner institutions who made this possible."

"This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma," said David F. Arons, President and Chief Executive Officer of the National Brain Tumor Society. "This is a fast-moving, devastating disease that turns families’ lives upside down. For years, this diagnosis has lacked an approved treatment and today, that changes. Families finally have a treatment option, and a reason to believe in more time together to make memories that might not have otherwise been possible."

The safety of Modeyso was evaluated in 376 adult and pediatric patients with glioma across four open-label clinical studies. Serious adverse reactions occurred in 33% of patients. Serious adverse reactions reported in more than 2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%) and muscular weakness (2.1%). The most common adverse reactions in patients who received Modeyso (≥20%) were fatigue, headache, vomiting, nausea and musculoskeletal pain.1 See additional safety information below and full prescribing information: View Source

About the Phase 2 Clinical Trial Program
The efficacy and safety of Modeyso were evaluated in adult and pediatric patients with glioma across five open-label, non-randomized clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). A pre-specified integrated efficacy analysis included 50 patients with recurrent H3 K27M-mutant diffuse midline glioma who had measurable disease per Response Assessment in Neuro-Oncology (RANO) criteria. Tumor response was assessed every eight weeks by blinded independent central review (BICR). The primary efficacy endpoint was objective response rate (ORR). Safety was evaluated across four of the clinical studies.1

More information about Modeyso, the Full Prescribing Information, and Patient Information, is available here.

Investor Webcast on August 27, 2025
The company will host a webcast on August 27, 2025, at 4:30 p.m. ET / 9:30 p.m. IST to provide investors an overview of clinical data, patient need and commercialization strategy for Modeyso. The webcast will include commentary from a leading neuro-oncology expert and the company’s senior management.

A live webcast of the presentation may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast. An archive of the webcast will be available for at least one week following the presentation on the Investors section of the company’s website at www.jazzpharmaceuticals.com.

About H3 K27M-Mutant Diffuse Midline Glioma
H3 K27M-mutant diffuse midline glioma is a rare and highly aggressive brain tumor that primarily affects the midline structures of the brain and spinal cord.4,5 It is characterized by a specific genetic mutation (H3 K27M) that disrupts epigenetic regulation and drives tumor growth.6 Most commonly diagnosed in children and young adults, patients with this type of glioma often face an extremely poor prognosis, with limited therapeutic options and very low survival rates following recurrence.7 Median survival is approximately one year from diagnosis and less than six months after progressing following frontline therapy.7

About Modeyso (dordaviprone)
Modeyso (dordaviprone) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial caseinolytic protease P (ClpP) and also inhibits dopamine D2 receptor (DRD2). In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.1

Modeyso received accelerated approval based on a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Continued approval may be contingent upon verification and description of clinical benefit in the ongoing Phase 3 ACTION trial (NCT05580562), which is evaluating the safety and clinical benefit of Modeyso in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy.6 Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

Modeyso (dordaviprone) is not approved anywhere else in the world.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Hypersensitivity
MODEYSO can cause severe hypersensitivity reactions.

In the pooled safety population, Grade 3 hypersensitivity reactions occurred in 0.3% of patients receiving MODEYSO. Signs and symptoms of hypersensitivity may include rash, hives, fever, low blood pressure, wheezing, or swelling of the face or throat.

Inform patients about the signs and symptoms of hypersensitivity reactions and instruct them to seek immediate medical attention if symptoms occur.

If clinically significant hypersensitivity or anaphylaxis occur, immediately interrupt MODEYSO and initiate appropriate medical treatment and supportive care. Based on the severity of the adverse reaction, temporarily interrupt or permanently discontinue MODEYSO.

QTc Interval Prolongation
MODEYSO causes concentration-dependent QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (e.g. torsades de pointes) or sudden death.

In patients who received MODEYSO and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 6% and 1.2% of patients, respectively.

Monitor ECGs and electrolytes prior to initiation and periodically during treatment, as clinically indicated. Increase the frequency of monitoring in patients with congenital long QT syndrome, existing QTc prolongation, a history of ventricular arrhythmias, electrolyte abnormalities, heart failure, or who are taking strong or moderate CYP3A4 inhibitors.

Avoid concomitant use with other agents known to prolong the QT interval. If concomitant use cannot be avoided, increase the frequency of monitoring and separate administration of MODEYSO and QT-prolonging product.

Interrupt or reduce the dose of MODEYSO in patients who develop QT prolongation; permanently discontinue in patients with signs of life-threatening arrhythmias.

Embryo-Fetal Toxicity
MODEYSO can cause fetal harm when administered to a pregnant woman.
Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose.

ADVERSE REACTIONS
Serious adverse reactions occurred in 33% of the 376 patients who received MODEYSO. Serious adverse reactions in >2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%), and muscular weakness (2.1%). Fatal adverse reactions occurred in 1% of patients who received MODEYSO, including cardiac arrest (0.5%), intracranial hemorrhage (0.3%), and encephalopathy (0.3%).

The most common adverse reactions (≥20%) reported in clinical trials with MODEYSO were fatigue (34%), headache (32%), vomiting (24%), nausea (24%), and musculoskeletal pain (20%). The most common (≥2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (7%), decreased calcium (2.7%), and increased alanine aminotransferase (2.4%).

DRUG INTERACTIONS
Strong and Moderate CYP3A4 Inhibitors
Avoid concomitant use of MODEYSO with strong and moderate CYP3A4 inhibitors. If concomitant use cannot be avoided, reduce the MODEYSO dose as recommended and monitor for toxicity.

Strong and Moderate CYP3A4 Inducers
Avoid concomitant use of strong and moderate CYP3A4 inducers with MODEYSO.

USE IN SPECIFIC POPULATIONS
Lactation
There are no data on the presence of MODEYSO in human milk because of the potential for serious adverse reactions from MODEYSO in breastfed children, advise women not to breastfeed during treatment with MODEYSO and for 1 week after the last dose.

Pediatric Use
The safety and effectiveness of MODEYSO have not been established in patients less than 1 year of age. Dosing has not been established for patients weighing less than 22 pounds (10 kg).

Please refer to the full Prescribing Information, including both Patient Information and Instructions for Use, for complete safety and administration information.

Caris Life Sciences Publishes Study Showing AI Signature-positive Breast Cancer Patients Live Almost Twice as Long as AI-negative Patients When Treated with a Checkpoint Inhibitor

On August 6, 2025 Caris Life Sciences (NASDAQ: CAI), a leading, patient-centric, next-generation AI TechBio company and precision medicine pioneer, reported a new study in Communications Medicine, a Nature portfolio journal, demonstrating that Caris’ AI-based image analysis model has the potential to more accurately predict cancer biomarkers and patient survival than the conventional companion diagnostic (CDx) methods (Press release, Caris Life Sciences, AUG 6, 2025, View Source [SID1234654888]). By analyzing hematoxylin and eosin (H&E) images, the study demonstrated that Caris’ AI model can improve the assessment of critical cancer biomarkers and impact patient survival outcomes in breast and colorectal cancers.

For this study, Caris’ AI model analyzed data from over 35,000 patients in the Caris clinico-genomic database. In breast cancer, the AI model scored PD-L1 positive phenotype status using an H&E image alone and assessed overall survival of patients treated with pembrolizumab, achieving a hazard ratio (HR) for overall survival of 0.511 (p<0.001), compared to an HR of 0.882 (p>0.1) for traditionally scored PD-L1 IHCs, a result consistent with an almost doubling of overall survival for patients treated with pembrolizumab. In colorectal cancer, AI predicted mismatch repair deficiency (MMRd) and microsatellite instability (MSI) equivalent to traditional scoring.

"Traditional PD-L1 testing can undercall positive cases, especially near the 1% threshold," said Matthew Oberley, MD, PhD, SVP, Chief Clinical Officer and Pathologist-in-Chief at Caris. "Caris’ AI model enhances predictive accuracy, integrating features from both staining methods, and exhibits superior prognostic precision compared to current biomarker assessments. Clinical adoption of this tool could improve the precision and efficiency of cancer patient evaluation and aid clinical decision making."

"This study highlights how AI can significantly improve the accuracy and efficiency of tissue sample evaluation, and down the line, this has the potential to guide immunotherapy decisions and enhance patient outcomes," said George W. Sledge, Jr., MD, Caris EVP and Chief Medical Officer.

The publication can be viewed in its entirety on the Caris Life Sciences website.

The U.S. FDA Granted Fast Track Designation to Dizal’s Birelentinib for Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

On August 6, 2025 Dizal (SSE:688192), a biopharmaceutical company committed to developing novel medicines for the treatment of cancer and immunological diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its Birelentinib (DZD8586) for the treatment of adult patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor (Press release, Dizal Pharma, AUG 6, 2025, View Source [SID1234654887]).

Patients with CLL/SLL treated with a BTK inhibitor or a BCL-2 inhibitor often relapse or progress due to two major resistance mechanisms: BTK C481X mutations and BTK-independent activation of BCR signaling pathway. No targeted therapy currently addresses both mechanisms, posing an urgent clinical challenge. Although BTK degraders have shown encouraging anti-tumor activity in early CLL/SLL clinical studies, mutation-mediated resistance has already been reported, and degrader-associated toxicities may limit their long-term clinical application.

Birelentinib is a first-in-class, non-covalent, LYN/BTK dual inhibitor with full blood-brain barrier (BBB) penetration. It has high selectivity against other TEC family kinases (TEC, ITK, TXK and BMX). By targeting both BTK and LYN, it blocks both BTK-dependent and -independent BCR-signaling pathways, effectively inhibiting tumor growth of B-cell non-Hodgkin lymphomas (B-NHLs).

The Fast Track Designation is supported by data from a pooled analysis of two phase I/II studies of birelentinib in CLL/SLL patients previously treated with covalent/non-covalent BTK inhibitors and BTK degraders. The results were presented at 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress and featured in oral presentations at both the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 18th International Conference on Malignant Lymphoma (ICML).

Birelentinib demonstrated significant anti-tumor efficacy in heavily pretreated CLL/SLL patients with an objective response rate (ORR) of 84.2%, with a good safety profile. Tumor responses were observed irrespective of prior treatment with covalent/non-covalent BTK inhibitors, BTK degraders, or BCL-2 inhibitors, including in patients harboring classic BTK resistance mutations (C481X) as well as other BTK mutations, such as kinase-dead mutations. Responses were durable, with an estimated 9-month duration of response (DOR) rate of 83.3%.

"The granting of Fast Track Designation underscores the U.S. FDA’s recognition of birelentinib’s potential to address an unmet medical need in patients with CLL/SLL," said Dr. Xiaolin Zhang, CEO of Dizal. "We look forward to working closely with the FDA to accelerate the global clinical development of birelentinib and bring this treatment option to patients as quickly as possible."

Fast Track Designation is an FDA program designed to facilitate the development and expedite the review of drugs for serious conditions that address unmet medical needs. The designation enables for frequent FDA interactions and may allow for rolling revie, priority review, or accelerated approval if criteria are met.

About Birelentinib (DZD8586)

Birelentinib is a first-in-class, non-covalent, LYN/BTK dual inhibitor with full blood-brain barrier (BBB) penetration, designed as a potential treatment option for B-cell non-Hodgkin lymphoma (B-NHL).

While Bruton’s Tyrosine Kinase (BTK) inhibitors have been approved for the treatment of B-NHL, resistance can arise through two major mechanisms: the BTK C481X mutation and BTK-independent BCR signaling pathway activation. Currently, there is no targeted therapy available to address both resistance mechanisms, posing an urgent clinical challenge. Although BTK degraders have shown encouraging efficacy in early clinical studies, mutation-related resistance has been reported, and degrader-related toxicities may affect long-term clinical application.

Birelentinib has high selectivity against other TEC family kinases (TEC, ITK, TXK and BMX). By targeting BTK and LYN, it blocks both BTK-dependent and -independent BCR-signaling pathways, effectively inhibiting tumor growth of B-NHLs in cell lines and in animal models. In clinical studies, birelentinib exhibits favorable PK properties, good central nervous system (CNS) permeability, complete blockade of BCR signaling, and encouraging anti-tumor efficacy with good safety and tolerability in patients with B-NHL.

In August 2025, birelentinib was granted Fast Track Designation by the U.S. FDA for the treatment of adult patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor.

Bio-Techne Releases Fourth Quarter Fiscal 2025 Results

On August 6, 2025 Bio-Techne Corporation (NASDAQ: TECH) reported its financial results for the fourth quarter ending June 30, 2025 (Press release, Bio-Techne, AUG 6, 2025, View Source [SID1234654886]).

Fourth Quarter FY2025 Highlights

Fourth quarter organic revenue increased by 3% (4% reported) to $317.0 million. Full year organic revenue increased 5% (5% reported) to $1.2 billion.
GAAP earnings per share (EPS) was $ (0.11) versus $0.25 one year ago. Delivered adjusted EPS of $0.53 compared to $0.49 one year ago. Full year GAAP EPS was $0.46 versus $1.05 one year ago. Full year adjusted EPS was $1.92 versus $1.77 one year ago.
Strong performance in our proteomic analytic and cell therapy growth pillars drove 4% organic growth (6% reported) in the Protein Sciences segment for the quarter, and 5% organic and reported growth for the full year.
Announced divestiture of the Exosome Diagnostics business including the ExoDx Prostate test (EPI) as the Company emphasizes investment and commercial priorities on non-CLIA based product lines in their growth pillars.
The Company’s financial statements are prepared in accordance with accounting principles generally accepted in the United States (GAAP). Adjusted diluted EPS, adjusted net earnings, adjusted gross margin, adjusted operating income, adjusted tax rate, organic revenue, adjusted operating margin, earnings before interest, taxes, depreciation, and amortization (EBITDA), and adjusted EBITDA are non-GAAP measures that exclude certain items detailed later in this press release under the heading "Use of non-GAAP Adjusted Financial Measures." A reconciliation of GAAP to non-GAAP financial measures is included in this press release.

"Bio-Techne delivered a solid fourth quarter that was in-line with our expectations, despite ongoing market uncertainty," said Kim Kelderman, President and Chief Executive Officer of Bio-Techne. "We achieved strong performance in our cell therapy and protein analysis instrumentation growth pillars, supported by a resilient pharma market. Our fiscal year 2025 results reflect the outstanding execution by our team and the meaningful impact our innovative solutions continue to deliver for customers, which include accelerating development timelines and driving scientific breakthroughs."

Kelderman added, "Last night, we announced the divestiture of our Exosome Diagnostics business. This strategic decision will immediately strengthen our profitability and allow us to increase our focus on high-growth areas, particularly tools for research and precision diagnostics."

Kelderman concluded, "While market uncertainties persist, I have full confidence in our team’s ability to navigate the evolving landscape and continue delivering meaningful value to all our stakeholders."

Bio-Techne will host an earnings conference call today, August 6, 2025, at 8:00 a.m. CDT. To listen, please dial 1-800-274-8461 or 1-203-518-9814 (for international callers), and reference conference ID TECHQ4. The earnings call can also be accessed via webcast through the following link View Source

A recorded rebroadcast will be available for interested parties unable to participate in the live conference call by dialing 1-844-512- 2921 or 1-412-317-6671 (for international callers) and referencing Conference ID 11159590. The replay will be available from 11:00 a.m. CDT on Wednesday, August 6, 2025, until 11:00 p.m. CDT on Saturday, September 6, 2025.

Fourth Quarter Fiscal 2025

Revenue

Net sales for the fourth quarter increased 4% to $317.0 million. Organic revenue increased 3% compared to the prior year. Foreign currency exchange had a favorable impact of 2%, and non-recurring prior year revenue from a business held-for-sale had an unfavorable impact of 1%.

GAAP Earnings Results

GAAP EPS was ($0.11) per diluted share, versus $0.25 in the same quarter last year. GAAP operating income for the fourth quarter of fiscal 2025 decreased 152% to ($23.9) million, compared to $45.8 million in the fourth quarter of fiscal 2024. GAAP operating margin was (7.5)%, compared to 15.0% in the fourth quarter of fiscal 2024. Current quarter GAAP operating margin was unfavorably impacted by impairment of the Exosome Diagnostics business.

Non-GAAP Earnings Results

Adjusted EPS increased to $0.53 per diluted share compared to $0.49 in the same quarter last year. Adjusted operating income of $101.3 million in the fourth quarter of fiscal 2025 remained relatively consistent with fourth quarter of fiscal 2024 adjusted operating income of $101.8 million. Adjusted operating margin was 32.0% for the fourth quarter of fiscal 2025 compared to 33.5% in the fourth quarter of fiscal 2024. Adjusted operating margin was impacted by unfavorable product mix, especially within the Diagnostic Solutions segment.

Full Year Fiscal 2025

Revenue

Net sales for the full year fiscal 2025 increased 5% to $1.2 billion. Organic revenue increased 5%. Foreign currency exchange and a business held-for-sale did not have a material impact.

GAAP Earnings Results

GAAP EPS was $0.46 per diluted share, compared to $1.05 last fiscal year. GAAP operating income for full year fiscal 2025 decreased 51% to $102.3 million, compared to $206.7 million in the full year fiscal 2024. GAAP operating margin was 8.4%, compared to 17.8% in the full year fiscal 2024. GAAP operating margin was unfavorably impacted by impairment of assets held-for-sale, restructuring and restructuring-related costs, and a non-recurring arbitration award.

Non-GAAP Earnings Results

Adjusted EPS increased to $1.92 per diluted share, compared to $1.77 last fiscal year. Adjusted operating income for fiscal 2025 increased 4% to $383.6 million, compared to $370.2 million for fiscal 2024. Adjusted operating margin for full year fiscal 2025 decreased to 31.6%, compared to 32.1% in full year fiscal 2024. Adjusted operating margin was impacted by reinstatement of incentive compensation accruals as well as unfavorable product mix.

Segment Results

Management uses adjusted operating results to monitor and evaluate performance of the Company’s business segments, as highlighted below.

Protein Sciences Segment

The Company’s Protein Sciences segment is one of the world’s leading suppliers of specialized proteins such as cytokines and growth factors, immunoassays, antibodies and reagents, to the biopharma and academic research communities. Additionally, the segment provides multiple platforms useful in various areas of protein analysis. In fiscal year 2024, a business within the Protein Sciences Segment met the criteria of held-for-sale; the held-for-sale business has been excluded from the segment’s operating results. Protein Sciences segment’s fourth quarter fiscal 2025 net sales were $226.5 million, an increase of 6% from $214.0 million for the fourth quarter of fiscal 2024. Organic revenue growth was 4% for the fourth quarter of fiscal 2025, with foreign currency exchange having a favorable impact of 2%. The Protein Sciences segment’s operating margin increased to 43.6% in the fourth quarter of fiscal 2025 compared to 43.0% in the fourth quarter of fiscal 2024. The segment’s operating margin increased primarily due to favorable volume leverage and ongoing profitability initiatives.

Protein Sciences segment’s full year fiscal 2025 net sales were $870.2 million, an increase of 5% from $830.9 million for full year fiscal 2024. Organic revenue for the segment increased 5% for the fiscal year, with foreign currency exchange and a business held-for-sale not having a material impact on revenue. Protein Sciences segment’s operating margin was 42.6% in fiscal 2025 compared to 42.7% in fiscal 2024.

Diagnostics and Spatial Biology Segment

The Company’s Diagnostics and Spatial Biology segment develops and provides spatial biology products, carrier screening and oncology kits, as well as exosome-based diagnostics for various pathologies, including prostate cancer. The Diagnostics and Spatial Biology segment also provides blood chemistry and blood gas quality controls, hematology instrument controls, immunoassays and other bulk and custom reagents for the in vitro diagnostic market. The Exosome Diagnostics business met the held-for-sale criteria at June 30, 2025 and incurred an impairment loss of $83.1 million during the quarter. The Diagnostics and Spatial Biology segment’s fourth quarter fiscal 2025 net sales were $89.7 million, a decrease of 1% from $90.7 million for the fourth quarter of fiscal 2024. Organic revenue decreased 1% for the fourth quarter of fiscal 2025, with foreign exchange not having a material impact. The Diagnostics and Spatial Biology segment’s operating margin of 6.0% in the fourth quarter of fiscal 2025 compared to 12.5% in the fourth quarter of fiscal 2024. The segment’s operating margin was impacted by unfavorable product mix.

The Diagnostics and Spatial Biology segment’s full year fiscal 2025 net sales were $346.3 million, an increase of 6% from $326.4 million for full year fiscal 2024. Organic growth for the segment was 6% with foreign currency exchange not having a material impact on revenue growth. The Diagnostics and Spatial Biology segment’s operating margin was 6.2% in fiscal 2025 compared to 7.5% in fiscal 2024. The segment’s operating margin was impacted by the reinstatement of incentive compensation accruals as well as unfavorable product mix.

Compugen Reports Second Quarter 2025 Results

On August 6, 2025 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in predictive computational target discovery powered by AI/ML, reported financial results for the second quarter of 2025 and provided a corporate update (Press release, Compugen, AUG 6, 2025, View Source [SID1234654885]).

"We continued to advance our immuno-oncology (IO) clinical and early-stage pipeline programs," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "We dosed the first patient in MAIA-ovarian, our global adaptive platform trial evaluating COM701 as a single agent for maintenance therapy in patients with relapsed platinum sensitive ovarian cancer (sub-trial 1). In addition, we are looking forward to presenting a pooled analysis of previously presented data from our three Phase 1 trials evaluating COM701 in heavily pretreated platinum resistant ovarian cancer at ESMO (Free ESMO Whitepaper) 2025 in October. We are also progressing the Phase 1 trial for GS-0321 a potential first-in-class anti-IL18BP antibody licensed to Gilead."

Dr. Cohen-Dayag continued, "We are excited to see the progress our partner AstraZeneca is making with its rilvegostomig program, with ten active Phase 3 trials. Rilvegostomig is an Fc reduced PD-1/TIGIT bispecific antibody, the TIGIT component of which is derived from our COM902, and which AstraZeneca has specifically designed and engineered with a unique mechanism of action to harness co-operative binding of both PD-1 and TIGIT to drive enhanced immune responses. At ASCO (Free ASCO Whitepaper) in June this year, AstraZeneca presented encouraging early data from trials evaluating rilvegostomig in combination with TROP2 ADC, Datroway, in NSCLC and in combination with chemotherapy in hepatobiliary cancer. The totality of this data along with data presented in 2024 highlight rilvegostomig as a potential IO backbone for future drug combinations. At the upcoming ESMO (Free ESMO Whitepaper) 2025 conference, AstraZeneca plans to share follow up data from ARTEMIDE-01 in NSCLC as a poster presentation and first data from TROPION-PanTumor03 in bladder cancer as a mini oral session. AstraZeneca’s broad development strategy for rilvegostomig to replace existing PD(L)-1 inhibitors represents a significant potential revenue source for Compugen as we are eligible for both future milestone payments and mid-single digit tiered royalties on future sales."

Dr. Cohen-Dayag added, "Our solid financial position with a cash runway expected to fund operations into 2027 allows us to advance our pipeline of differentiated IO therapies and to leverage Unigen – our validated AI/ML-powered computational target discovery platform to discover novel mechanisms to activate the immune system against cancer. I look forward to transitioning leadership to Dr. Eran Ophir in September and the opportunity of stepping into the newly established role of Executive Chair. With this enhanced leadership expansion, a strategically differentiated pipeline and operational focus, Compugen is well positioned for growth."

Next Planned Milestones

ESMO 2025: poster presentation of a pooled analysis of three Phase 1 trials from previously presented data evaluating COM701 in heavily pretreated platinum resistant ovarian cancer
ESMO 2025: Compugen’s partner, AstraZeneca, plans to present:
updated data from Phase 2 ARTEMIDE-01 evaluating rilvegostomig in metastatic NSCLC as a poster presentation
first data from TROPION-PanTumor03 evaluating rilvegostomig in combination with TROP 2 ADC Datroway in bladder cancer as a mini oral session
H2 2026: data from projected interim analysis of single agent COM701 sub-trial 1 as maintenance therapy in relapsed platinum sensitive ovarian cancer
Second Quarter 2025 Financial Highlights

Cash: As of June 30, 2025, Compugen had approximately $93.9 million in cash, cash equivalents, short-term bank deposits, and investment in marketable securities.

Compugen expects that its cash and cash-related balances will be sufficient to fund its operating plans into 2027. This does not include any cash inflows. The Company has no debt.

Revenue: Compugen reported approximately $1.3 million in revenues for the second quarter ended June 30, 2025, compared to approximately $6.7 million in revenues for the comparable period in 2024. The revenues reported in the second quarter of 2025 reflect recognition of a portion of both the upfront payment and the IND milestone payment from the license agreement with Gilead. The revenues reported in the second quarter of 2024 reflect recognition of portions of the upfront payment from the license agreement with Gilead and the clinical milestone from the license agreement with AstraZeneca.

R&D expenses for the second quarter of 2025 were approximately $5.6 million compared to approximately $6.2 million for the comparable period in 2024.

G&A expenses were approximately $2.2 million for the second quarters of 2025 and 2024.

Net loss for the second quarter of 2025 was approximately $7.3 million, or $0.08 per basic and diluted share, compared with a net loss of approximately $2.1 million, or $0.02 per basic and diluted share, in the second quarter of 2024.

Full financial tables are included below

Conference Call and Webcast Information

The Company will hold a conference call today, August 6, 2025, at 8:30 AM ET to review its second quarter 2025 results. To access the conference call by telephone, please dial 1-866-744-5399 from the United States, or +972-3-918-0644 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.