On August 5, 2025 SynOx Therapeutics Limited ("SynOx"), a late-stage clinical biopharmaceutical company developing emactuzumab for Tenosynovial Giant Cell Tumours (TGCT), reported completion of patient enrolment in the TANGENT study (Press release, SynOx Therapeutics, AUG 5, 2025, View Source [SID1234654799]). TANGENT is the company’s global, multi-centre, randomized, double-blind, placebo-controlled registrational Phase 3 trial of emactuzumab in patients with TGCT who are not amenable to or who would not benefit from surgery. SynOx expects to report top-line data from the study in the first quarter of 2026.

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Emactuzumab is a potentially best-in-class CSF-1 receptor (CSF-1R) inhibiting monoclonal antibody (mAb). Clinical evaluation to date has demonstrated the compound to be a promising, next-generation mAb therapy with the potential to offer a short treatment cycle, rapid onset and long duration of response that differentiates it from chronically administered oral agents. This potentially best-in-class profile helped drive strong interest in the TANGENT trial among patients and clinical investigators across sites in the United States, Canada, Europe and Asia, leading to rapid study enrollment.

"Completion of enrollment in our registrational TANGENT trial marks an important milestone for SynOx and for the TGCT community," said Elyse Seltzer, M.D., Chief Medical Officer of SynOx Therapeutics. "We are deeply grateful to the patients, families, investigators, and clinical sites whose dedication has made this study possible and allowed SynOx to fully enroll the trial significantly ahead of our projected timeline. We remain committed, data permitting, to delivering a much-needed new treatment option for this debilitating condition."

"This is a transformational time for SynOx as we advance emactuzumab through its pivotal Phase 3 program," said Ray Barlow, Chief Executive Officer of SynOx. "We’re excited about the next steps and look forward to delivering top-line data that we hope demonstrate how emactuzumab can transform care for patients with this grievous disease."

About the TANGENT Study

TANGENT (ClinicalTrials.gov Identifier: NCT05417789) is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating the efficacy and safety of emactuzumab in patients with TGCT who are not amenable to or would not benefit from surgery. Patients who consent and meet eligibility criteria are randomized in a 2:1 fashion to receive either emactuzumab (1000 mg every two weeks for five doses) or matched placebo.

The primary endpoint is objective response rate (ORR) as assessed by RECIST criteria at six months post-randomization. Key secondary endpoints include patient-reported outcomes (PROMIS-PF), functional assessments of range of motion, pain, stiffness, durability of response, and safety. Patients are followed for two years from randomization, with those demonstrating disease progression after the six-month assessment eligible to receive open-label emactuzumab during follow-up.

On August 5, 2025 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the second quarter 2025 and associated Company developments (Press release, Supernus, AUG 5, 2025, View Source [SID1234654798]).

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"Our strong operating performance in the first half of the year was driven by continued strong sales growth of Qelbree and GOCOVRI, which combined accounted for 72% of total net sales in the second quarter of 2025," said Jack Khattar, President and CEO of Supernus. "Our focus for the second half of the year remains on the launch of ONAPGO, the successful integration of Sage Therapeutics, and the continued performance of our growth products," said Jack Khattar, President and CEO of Supernus.

Acquisition of Sage Therapeutics, Inc.

The Company completed the acquisition of Sage Therapeutics on July 31, 2025, strengthening its leading presence in neuropsychiatric conditions with an innovative commercial product, ZURZUVAE (zuranolone), and a novel CNS discovery platform. The acquisition is expected to accelerate mid- to long-term revenue and cash flow growth and further diversify the Company’s revenue base.
Commercial Highlights

Total IQVIA prescriptions(2) for Qelbree were 225,254 for the second quarter 2025, an increase of 23% compared to the same period in the prior year. Qelbree continues to expand its base of prescribers, with approximately 36,000 prescribers in the second quarter of 2025, up by 23% compared to the same period last year.

In April 2025, the Company launched ONAPGO, the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson’s disease. ONAPGO demand is exceeding the Company’s expectations, with more than 750 enrollment forms submitted by more than 300 prescribers through the end of the second quarter of 2025.
Product Pipeline Update

SPN-817 – Novel first-in-class highly selective AChE inhibitor for epilepsy

The Phase 2b randomized, double-blind, placebo-controlled study of 3mg and 4mg twice daily doses is ongoing with a targeted enrollment of approximately 258 adult patients with treatment resistant focal seizures.
SPN-820 – Novel first-in-class molecule that increases mTORC1 mediated synaptic function for depression

The Company plans to initiate a follow-on Phase 2b multi-center, randomized, double-blind, placebo-controlled trial in approximately 200 adults with major depressive disorder (MDD). The study will examine the safety and tolerability of SPN-820 2400 mg given intermittently (twice weekly) as an adjunctive treatment to the current baseline antidepressant therapy, as well as assess the rapid onset of improvement in depressive symptoms. The Company expects to initiate the Phase 2b study by the end of 2025.
SPN-443 – Novel stimulant for ADHD/CNS

The Company completed a Phase 1 pharmacokinetic study of two oral formulations in healthy adults. Both formulations of SPN-443 showed adequate bioavailability and were well tolerated. The Company expects to disclose a lead indication for the product candidate by the end of 2025.
Financial Highlights

This section includes information on non-GAAP financial measures. See "Non-GAAP Financial Information" section for information on non-GAAP financial measures. In addition, a reconciliation of applicable GAAP to non-GAAP financial information is included at the end of this press release.

Revenues

The following table provides information regarding total revenues (dollars in millions):

Three Months Ended
June 30, Six Months Ended
June 30,
2025 2024 Change % 2025 2024 Change %
(unaudited)
Net product sales
Qelbree $ 77.6 $ 59.4 31 % $ 142.3 $ 104.5 36 %
GOCOVRI 36.7 31.7 16 % 67.4 58.3 16 %
APOKYN 12.8 17.3 (26 )% 27.8 33.9 (18 )%
Trokendi XR 11.2 17.1 (35 )% 24.0 33.1 (27 )%
Oxtellar XR 11.6 29.5 (61 )% 21.8 56.5 (61 )%
ONAPGO 1.6 — 100 % 1.6 — 100 %
Other(3) 6.5 7.5 (13 )% 15.1 14.7 3 %
Total net product sales 158.0 162.5 (3 )% 300.0 301.0 — %
Royalty, licensing and other revenues(4) 7.5 5.8 29 % 15.3 11.0 39 %
Total revenues $ 165.5 $ 168.3 (2 )% $ 315.3 $ 312.0 1 %

Total revenues excluding Trokendi XR and Oxtellar XR net sales (non-GAAP)(1) $ 142.7 $ 121.7 17 % $ 269.5 $ 222.4 21 %

Other Financial Highlights

Operating earnings were $12.1 million and $1.9 million for the three and six months ended June 30, 2025, compared to operating earnings of $22.6 million and $19.4 million for the same periods in 2024. The change was primarily due to higher contingent consideration loss, mainly related to the achievement of ONAPGO-related milestones, and higher selling and marketing expenses.
Adjusted operating earnings (non-GAAP) were $40.9 million and $66.9 million for the three and six months ended June 30, 2025, compared to $45.5 million and $67.7 million for the same periods in 2024.
Net earnings and diluted earnings per share were $22.5 million and $0.40 for the three months and $10.7 million and $0.19 for the six months ended June 30, 2025, respectively, compared to net earnings and diluted earnings per share of $19.9 million and $0.36 for the three months and $20.0 million and $0.36 six months ended June 30, 2024, respectively.
At June 30, 2025, cash, cash equivalents, and current marketable securities were approximately $522.6 million compared to $453.6 million as of December 31, 2024. This increase was primarily due to cash generated from operations.

On August 5, 2025 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage biotechnology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported financial results for the second quarter 2025, and provided a corporate update (Press release, Sensei Biotherapeutics, AUG 5, 2025, View Source [SID1234654797]).

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"The second quarter was a key inflection point for Sensei, as we now transition from early response-focused readouts to longer-term and commercially relevant efficacy signals with the maturity of the data from our Phase 1/2 study of solnerstotug," said John Celebi, President and CEO of Sensei. "Solnerstotug has demonstrated a favorable safety profile and in combination with cemiplimab has not demonstrated significant additional toxicity relative to what is typically observed with PD-(L)1 monotherapy, which we believe could translate into better patient adherence and more meaningful long-term outcomes with continued treatment, as well as physician preference and payor interest."

"As we look to next steps in the clinical advancement of solnerstotug, we envision multiple Phase 2 studies across PD-(L)1 resistant tumor types, aligned with unmet need and commercial potential. We view these differentiated opportunities as potentially derisking overall, while positioning solnerstotug for multiple indications in high-value immunotherapy segments of the ~$50 billion PD-(L)1 market," added Mr. Celebi. "We continue to work on finalizing the Phase 2 strategy for solnerstotug, which will be strongly influenced by the full dose expansion dataset we plan to present later this year."

Solnerstotug (formerly SNS-101) is a conditionally active antibody designed to selectively target the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation) within the tumor microenvironment. VISTA is implicated in numerous cancer indications and its expression correlates with low survival rates.

Sensei’s ongoing multi-center Phase 1/2 clinical trial is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of solnerstotug as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in patients with advanced solid tumors.

Clinical Program Highlights:

Full dose expansion data, including 6-month progression free survival (PFS), from the Phase 1/2 study continues to be expected by year-end 2025.
Enrollment in the Phase 1/2 dose expansion cohort is complete with a total of 64 patients, including:
10 "cold" MSS CRC patients in the monotherapy arm
54 patients in the cemiplimab combination arm consisting of 10 "cold" MSS CRC patients and 44 "hot" tumor patients. 41/44 patients in the "hot" tumor cohort had received and progressed on a prior PD-(L)1 inhibitor.
On March 27th, Sensei announced favorable preliminary clinical data in PD-(L)1 resistant patients from the dose expansion stage of its ongoing Phase 1/2 trial, demonstrating favorable activity in patients with PD-(L)1 resistant "hot" tumors. Solnerstotug continued to be well tolerated, with no dose-limiting toxicities, and the majority of AEs Grade 1 or 2 in severity.
A replay of the March 2025 webcast related to the preliminary data, featuring study investigator Dr. Shiraj Sen, is available on the Sensei website.
Corporate Updates:

On July 30th, the Company announced that clinical data from the dose expansion cohort of the Phase 1/2 trial of solnerstotug alone and in combination with cemiplimab will be presented in a mini oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025, being held October 17-21, 2025 in Berlin, Germany.
Sensei regains Nasdaq compliance following the 1-for-20 reverse split of its common stock, which became effective at 5:00 p.m. ET on June 16th, and subsequent 10-day bid price remaining above $1.
On April 7th, John Celebi participated in a panel discussion titled "New Radiotherapy and Targeted Therapy Approaches" at the Canaccord Genuity Horizons in Oncology Virtual Conference. The panel focused on emerging innovations in cancer treatment and Sensei’s approach to selectively modulating the tumor microenvironment.
Second Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $28.6 million as of June 30, 2025, as compared to $41.3 million as of December 31, 2024. Sensei expects its current cash balance to fund operations into the second quarter of 2026.

Research and Development (R&D) Expenses: R&D expenses were $2.5 million for the quarter ended June 30, 2025, compared to $4.6 million for the quarter ended June 30, 2024. The decrease in R&D expenses was primarily attributable to lower personnel and facilities costs, and reduced manufacturing cost.

General and Administrative (G&A) Expenses: G&A expenses were $2.7 million for the quarter ended June 30, 2025, compared to $3.2 million for the quarter ended June 30, 2024. The decrease in G&A expense was primarily attributable to lower personnel costs.

Net Loss: Net loss was $4.9 million for the quarter ended June 30, 2025, compared to $7.1 million for the quarter ended June 30, 2024.

On August 5, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported financial results for the second quarter ended June 30, 2025, including sales of TAVALISSE (fostamatinib disodium hexahydrate), GAVRETO (pralsetinib) and REZLIDHIA (olutasidenib), and recent business progress (Press release, Rigel, AUG 5, 2025, View Source [SID1234654796]).

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"Our strategic and disciplined approach to building our business has generated another strong quarter for the company. In the second quarter, we grew net product sales by 76% year-over-year, generated $59.6 million in net income and increased our cash balance to $108.4 million. This strong performance has enabled us to raise our 2025 financial guidance," said Raul Rodriguez, Rigel’s president and CEO. "We’re also excited by the continued advancement of our hematology and oncology development pipeline. In July, we completed enrollment in the dose escalation part of our ongoing Phase 1b study evaluating R289 in patients with relapsed or refractory lower-risk MDS. Later this year, we plan to share updated data from that study and initiate the dose expansion part of the study."

Second Quarter 2025 Business Update

Commercial

· Net product sales were $58.9 million, an increase of 76% from the same period of 2024.
· Rigel’s partner Kissei Pharmaceutical Co., Ltd. (Kissei) announced that its licensing partner, JW Pharmaceutical Corporation, commercially launched TAVALISSE in South Korea in early July.

Corporate

· In April, Rigel notified Eli Lilly and Company (Lilly) that it would not exercise its opt-in right related to the development and commercialization of ocadusertib (previously R552) for the treatment of non-central nervous system (CNS) diseases. As a result of the notification, Rigel recognized $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability in the second quarter. Per the agreement with Lilly, Rigel will continue to be entitled to receive milestone and tiered royalty payments on future net sales of ocadusertib and the companies’ CNS penetrant program.

Clinical Development

· Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a novel and selective dual interleukin receptor-associated kinases 1 and 4 (IRAK1/4) inhibitor, in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). Enrollment in the dose escalation part of the study was completed in July. The company expects to share updated data from the study later this year and plans to initiate the dose expansion part of the study in the second half of this year.

· Rigel presented 4 posters at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and 3 posters at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress. Presentations included the final data from the GAVRETO (pralsetinib) Phase 1/2 ARROW study in RET fusion-positive non-small cell lung cancer (NSCLC) and other solid tumors, and supportive data for REZLIHDIA (olutasidenib) in patients with mutated isocitrate dehydrogenase-1 (mIDH1) relapsed or refractory (R/R) acute myeloid leukemia (AML).

Key Publications

· A paper titled "Effectiveness of Olutasidenib versus Ivosidenib in Patients With Mutated Isocitrate Dehydrogenase 1 Acute Myeloid Leukemia Who Are Relapsed or Refractory to Venetoclax: The 2102-HEM-101 Trial Versus a US Electronic Health Record-Based External Control Arm," was published in June by Catherine Lai, M.D., MPH, lead author and associate professor of Clinical Medicine, Division of Hematology-Oncology, Department of Medicine at University of Pennsylvania, in Leukemia & Lymphoma.
· A paper titled "Olutasidenib Alone or Combined with Azacitidine in Patients with Mutant IDH1 Myelodysplastic Syndrome," was published in July by Jorge E. Cortes, M.D., lead author and director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, in Blood Advances. This is the first full report of the safety and clinical activity of an mIDH1 inhibitor plus hypomethylating agent (HMA) combination in patients with treatment-naïve and R/R MDS. The paper showed olutasidenib, alone or in combination with azacitidine, induced a high rate of clinically meaningful complete remission and hematologic improvement responses with durable remission duration, accompanied by low rates of serious treatment-emergent-adverse events in patients with intermediate-, high- or very high-risk MDS harboring mIDH1.

Second Quarter 2025 and Year-to-Date Financial Update

For the second quarter ended June 30, 2025, total revenues were $101.7 million, consisting of $58.9 million in net product sales and $42.7 million in contract revenues from collaborations. Net product sales grew 76% compared to $33.5 million in the same period of 2024. TAVALISSE net product sales were $40.1 million, growth of 52% compared to $26.4 million in the same period of 2024. GAVRETO net product sales were $11.8 million compared to $1.9 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $7.0 million, growth of 36% compared to $5.2 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $2.0 million of revenue from Grifols S.A. (Grifols) related to delivery of drug supplies and earned royalties, $0.4 million of revenue from Kissei related to the delivery of drug supplies, and $0.2 million of revenue from Medison Pharma (Medison) related to delivery of drug supplies and earned royalties.

Total costs and expenses were $40.6 million compared to $36.4 million for the same period of 2024. The increase in costs and expenses was mainly due to higher cost of product sales, increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289, and higher personnel-related costs and stock-based compensation expense.

Rigel reported net income of $59.6 million, or $3.33 basic and $3.28 diluted per share, compared to a net loss of $1.0 million, or $0.06 basic and diluted per share, for the same period of 2024.

For the six months ended June 30, 2025, total revenues were $155.0 million, consisting of $102.5 million in net product sales and $52.5 million in contract revenues from collaborations. Net product sales grew 72% compared to $59.5 million in the same period of 2024. TAVALISSE net product sales were $68.5 million, growth of 44% compared to $47.5 million in the same period of 2024. GAVRETO net product sales were $20.8 million compared to $1.9 million in the same period of 2024. GAVRETO became commercially available from Rigel in late June 2024. REZLIDHIA net product sales were $13.1 million, growth of 31% compared to $10.0 million in the same period of 2024. Contract revenues from collaborations primarily consisted of $40.0 million in non-cash revenue resulting from the release of the remaining cost share liability related to the agreement with Lilly for the development and commercialization of ocadusertib, $6.7 million of revenue from Grifols related to delivery of drug supplies and earned royalties, $5.1 million of revenue from Kissei related to a milestone payment and delivery of drug supplies and $0.6 million of revenue from Medison related to delivery of drug supplies and earned royalties.

Total costs and expenses were $81.1 million compared to $72.9 million for the same period of 2024. The increase in costs and expenses was mainly due to higher cost of product sales, increased research and development costs driven by the timing of clinical activities related to olutasidenib and R289, and higher personnel-related costs. These increases were partially offset by decreased stock-based compensation expense primarily from performance-based stock awards.

Rigel reported net income of $71.1 million, or $3.98 basic and $3.91 diluted per share, compared to a net loss of $9.3 million, or $0.53 basic and diluted per share, for the same period of 2024.

Cash, cash equivalents and short-term investments as of June 30, 2025 was $108.4 million, compared to $77.3 million as of December 31, 2024.

2025 Outlook

Rigel is updating its 2025 total revenue guidance to approximately $270 to $280 million, an increase from the previous range of approximately $200 to $210 million, which includes:

· Net product sales of approximately $210 to $220 million, an increase from the previous range of approximately $185 to $192 million.
· Contract revenues from collaborations of approximately $60 million, an increase from the previous range of approximately $15 to $18 million.

The company anticipates it will report positive net income for the full year 2025, while funding existing and new clinical development programs.

The above total revenues and contract revenues from collaborations are inclusive of $40 million in non-cash contract revenue related to Rigel’s agreement with Lilly.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time

Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP

In patients with immune thrombocytopenia (ITP), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. Patients suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About NSCLC

It is estimated that over 226,000 adults in the U.S. will be diagnosed with lung cancer in 2025. Lung cancer is the leading cause of cancer death in the U.S, with non-small cell lung cancer (NSCLC) being the most common type accounting for 85-90% of all lung cancer diagnoses.2 RET fusions are implicated in approximately 1-2% of patients with NSCLC.3

About AML

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that there will be about 22,010 new cases in the United States, most in adults, in 2025.4

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.5,6 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.7 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About TAVALISSE

TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE.

About GAVRETO

GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).

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.