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Servier Announces Positive Data from Long-Term Follow-Up Analysis of the Phase 3 AGILE Trial of TIBSOVO® (ivosidenib) in IDH1-mutated Acute Myeloid Leukemia

On July 28, 2025 Servier reported that Blood Advances published long-term data from the Phase 3 AGILE trial evaluating TIBSOVO (ivosidenib) in combination with azacitidine versus placebo-azacitidine in patients with newly diagnosed mutant isocitrate dehydrogenase 1 (mIDH1) acute myeloid leukemia (AML) who were unfit to receive intensive chemotherapy (Press release, Servier, JUL 28, 2025, View Source [SID1234654582]). The post-hoc analysis reports positive long-term follow-up results from the pivotal Phase 3 AGILE trial and continues to demonstrate the sustained survival benefit reported in the previous analysis published in the New England Journal of Medicine (NEJM).

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"The clear and robust clinical benefit demonstrated by these long-term analyses – including prolonged overall survival and hematologic recovery – support TIBSOVO as a standard of care treatment for patients with IDH1-mutated AML," said Susan Pandya, M.D., Vice President Clinical Development and Global Head of Oncology LS/LCM, Servier. "These data are a testament to Servier’s industry-leading research in IDH1-mutated cancers, including AML, and demonstrate our steadfast commitment to improving outcomes for patients."

As of data cutoff in June 2022, median follow-up was 28.6 months. Key findings from the newly published analysis include:

Median overall survival (OS) was significantly longer in patients treated with the TIBSOVO combination (29.3 months; 95% CI, 13.2 – not reached) than placebo-azacitidine (7.9 months; 95% CI, 4.1-11.3; hazard ration [HR]=0.42 [0.27, 0.65]; p<.0001).
Hematologic recovery was generally faster and lasted longer in patients in the TIBSOVO arm compared to placebo-azacitidine. Conversion to transfusion independence was more common with TIBSOVO (53.8%) than placebo-azacitidine (17.1%; p=.0004).
Ten (30.3%) of the 33 molecular measurable-residual disease (MRD)-evaluable patients in the TIBSOVO arm converted to an MRD-negative response by Day 1 of Cycle 14, all of whom had a complete response (CR). Seven (70%) of these patients converted to an MRD-negative response by Day 1 of Cycle 7. Of the 23 patients who remained MRD-positive, 19 had a CR and four had a CR with incomplete hematologic recovery (CRi). Two patients (20%) had an MRD-negative response in the placebo-azacitidine arm.
The long-term safety profile of TIBSOVO with azacitidine was consistent with previously reported data. The most commonly reported Grade ≥3 hematologic adverse events (AEs) were anemia (26.4%), neutropenia (30.6%), and febrile neutropenia (27.8%). The most common Grade ≥3 nonhematologic AEs were electrocardiogram QT prolonged (11.1%), pneumonia (22.2%), nausea, (2.8%), hypokalemia (2.8%), and pyrexia (2.8%). There were no new or unexpected safety signals or additional treatment discontinuations due to AEs compared with the primary analysis.
"Long-term results from the AGILE trial underscore the power of TIBSOVO to improve outcomes for patients with IDH1-mutated AML unfit for intensive chemotherapy, a group who historically has had a poor long-term prognosis," said Hartmut Döhner, MD, Professor of Medicine and Director of the Department of Internal Medicine III at the University Hospital Ulm in Germany and Director of the National Center for Tumor Diseases SouthWest (NCT-SW; Ulm site). "These results continue to emphasize the importance of early systematic genetic testing to guide treatment selection for patients – which may lead to improved overall survival rates and a reduction in the risk of death as seen in this study."

TIBSOVO was approved by the U.S. Food and Drug Administration (FDA) in combination with azacitidine for the treatment of patients with newly diagnosed IDH1-mutated AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, in May 2022 under Priority Review. The supplemental New Drug Application (sNDA) for TIBSOVO was reviewed by the FDA under its Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.

H1 and Q2 2025 results

On July 28, 2025 AstraZeneca’s Chief Executive Officer, Pascal Soriot, reported on the results and said (Press release, AstraZeneca, JUL 28, 2025, View Source [SID1234654581]):

"Our strong momentum in revenue growth continued through the first half of the year and the delivery from our broad and diverse pipeline has been excellent, with 12 positive key Phase III trial readouts including for baxdrostat, gefurulimab, and Tagrisso in just the past few weeks.

As we enter our next phase of growth, we have pledged $50 billion to continue to grow in the US, which includes the largest manufacturing investment in AstraZeneca’s history, set for Virginia. This landmark investment reflects not only America’s importance but also our confidence in our innovative medicines to transform global health and power AstraZeneca’s ambition to deliver $80 billion revenue by 2030."

Immutep Announces Abstracts Accepted for Presentation at the European Society for Medical Oncology Congress 2025

On July 28, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported three abstracts for clinical trials evaluating its first-in-class MHC Class II agonist, eftilagimod alfa (efti), have been accepted for presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 taking place 17-21 October in Berlin, Germany (Press release, Immutep, JUL 28, 2025, View Source [SID1234654580]).

A Proffered Paper oral presentation will detail results from EFTISARC-NEO, a Phase II investigator-initiated trial in resectable soft tissue sarcoma, and data from the INSIGHT-003 Phase I investigator-initiated trial in first-line non-small cell lung cancer (1L NSCLC) has been accepted for poster presentation. Additionally, an abstract on the Company’s pivotal TACTI-004 Phase III in 1L NSCLC has been accepted for a Trials in Progress ePoster. Details of the presentations are as follows:

Title: EFTISARC-NEO: A phase II study of neoadjuvant eftilagimod alpha, pembrolizumab and radiotherapy in patients with resectable soft tissue sarcoma
Presenter: Katarzyna Kozak, M.D., Ph.D., Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland
Session Category: Proffered Paper
Session Title: Sarcoma
Presentation #: 2686O
Date and Time: Sunday, 19 October 2025 at 16:30 – 18:00 PM CET

Title: Eftilagimod alpha (soluble LAG-3 protein) combined with 1st line chemo-immunotherapy in metastatic non-squamous non-small cell lung cancer (NSCLC) –Updates from INSIGHT-003 (IKF614)
Presenter: Dr. med. Akin Atmaca, Department of Hematology and Oncology, Krankenhaus Nordwest, UCT-University Cancer Center, Frankfurt, Germany
Session Category: Poster
Session Title: NSCLC, metastatic
Presentation #: 1857P
Date and Time: Saturday, 18 October 2025 at 12:00 – 12:45 PM CET

Title: TACTI-004, a double-blinded, randomised phase 3 trial of eftilagimod alfa plus pembrolizumab (P) + chemotherapy (C) vs placebo + P + C in 1st line advanced/metastatic NSCLC
Presenter: Prof. Dr. med. Hans-Georg Kopp, Robert Bosch Hospital, Stuttgart, Germany
Session Category: ePoster
Session Title: NSCLC, metastatic
Presentation #: 2086eTiP

Proffered Papers at ESMO (Free ESMO Whitepaper) are oral presentations of original data of superior quality, followed by expert discussion and perspectives.

Abstracts will be made available on the ESMO (Free ESMO Whitepaper) website on 13 October 2025 at 00.05 CET. The posters will be available on the Posters & Publications section of Immutep’s website after their presentations.

BeOne Medicines Receives Positive CHMP Opinion for TEVIMBRA® in Neoadjuvant/Adjuvant NSCLC Treatment

On July 28, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of TEVIMBRA (tislelizumab), in combination with platinum-containing chemotherapy as neoadjuvant treatment and then continued as monotherapy as adjuvant treatment, for the treatment of adult patients with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence (Press release, BeOne Medicines, JUL 28, 2025, View Source [SID1234654579]). This recommendation is based on the Phase 3 RATIONALE-315 study (NCT04379635).

"Patients with resectable, early-stage NSCLC face an urgent challenge – despite surgery and current therapies, recurrence rates remain alarmingly high," said Dr. Mariano Provencio, Head of the Medical Oncology Department at Hospital Universitario Puerta de Hierro and Professor at the Faculty of Medicine of Universidad Autonoma de Madrid in Spain. "The significant clinical benefit observed in the RATIONALE-315 study has important implications for patients. If approved, perioperative tislelizumab will offer oncologists a powerful new option to improve outcomes and potentially alter the course of this difficult-to-treat disease."

RATIONALE-315 is a double-blind, placebo-controlled, multicenter, Phase 3 study that randomized 453 patients with resectable NSCLC 1:1 to receive either TEVIMBRA plus platinum-based doublet chemotherapy as neoadjuvant treatment followed by TEVIMBRA as adjuvant treatment or placebo plus platinum-based doublet chemotherapy as neoadjuvant treatment followed by placebo as adjuvant treatment. As previously reported at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress Virtual Plenary in February 20241, the dual primary endpoints of event-free survival (EFS) and major pathologic response (MPR) were met at the interim analyses of the RATIONALE-315 study. Results include:

Statistically significant and clinically meaningful improvement in MPR and pathological complete response (pCR) rates:
56.2% of NSCLC patients treated with TEVIMBRA in combination with chemotherapy before surgery achieved MPR compared to 15.0% of patients treated with chemotherapy in combination with placebo (difference: 41.1%; 95% CI: 33.2-49.1, p<0.0001)
40.7% of patients on the TEVIMBRA-based regimen achieved pCR, compared to 5.7% of patients treated with chemotherapy in combination with placebo (difference: 35.0%; 95% CI: 27.9-42.1, p<0.0001)
Statistically significant EFS (HR [95% CI], 0.56 [0.40–0.79]; 1-sided P=0.0003) and trend for overall survival (OS) (HR [95% CI], 0.62 [0.39–0.98]; 1-sided P=0.0193) benefits favoring TEVIMBRA in early data.
Consistent safety profile of the TEVIMBRA arm with that of individual therapies, with 72.1% of patients in the TEVIMBRA arm (vs. 66.4% in the placebo arm) experiencing grade ≥3 treatment-related adverse events (TRAEs) and 15.5% of patients in the TEVIMBRA arm (vs. 8.0% in the placebo arm) experiencing serious TRAEs. There were no new safety signals identified with this regimen, and the most common Grade 3 or 4 TRAEs (≥ 10%) in the TEVIMBRA arm were decreased neutrophil count and decreased white blood cell count.
No impact on the feasibility and completeness of surgery, a key concern around neoadjuvant treatment.
Updated EFS and OS data from the pre-planned final analysis of RATIONALE-315 will be submitted for presentation at an upcoming medical conference.

"TEVIMBRA is already approved in the EU across multiple settings in NSCLC, the most common form of lung cancer, and this positive CHMP opinion expands its potential to help patients earlier in their treatment journey," said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne. "As the foundational asset of our solid tumor portfolio, TEVIMBRA continues to demonstrate its strength and versatility across the continuum of care, bringing us closer to our goal of delivering more comprehensive and effective cancer treatment to more patients."

In lung cancer, TEVIMBRA is already approved in the EU for the first-line treatment of patients with squamous NSCLC, for the first-line treatment of patients with non-squamous NSCLC with PD-L1 high expression, for the treatment of patients with locally advanced or metastatic NSCLC after prior platinum-based therapy, and as a first-line treatment for extensive-stage small cell lung cancer (ES-SCLC). It is also approved as a first-line treatment for patients with gastric or gastroesophageal junction (G/GEJ) adenocarcinoma, as a first-line treatment for unresectable esophageal squamous cell carcinoma (ESCC), as a second-line treatment in ESCC after prior platinum-based chemotherapy, and as a first-line treatment for patients with nasopharyngeal carcinoma (NPC).

About NSCLC

Lung cancer is the most commonly diagnosed type of cancer and the leading cause of cancer-related death worldwide.2 In Europe, lung cancer is the third most frequent cancer with 484,306 new cases diagnosed in 2022.3 NSCLC accounts for 80–90% of all lung cancers4, of which resectable NSCLC patients at diagnosis represent around 25–30%5.

About TEVIMBRA (tislelizumab)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

TEVIMBRA is the foundational asset of BeOne’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 countries and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 46 countries, and more than 1.5 million patients have been treated globally.

Important Safety Information

The current European Summary of Product Characteristics (SmPC) for TEVIMBRA is available from the European Medicines Agency.

This information is intended for a global audience. Product indications vary by region.

Arrowhead Pharmaceuticals Earns $100 Million Milestone from Sarepta Therapeutics

On July 28, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported that it has earned a $100 million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT) (Press release, Arrowhead Pharmaceuticals, JUL 28, 2025, View Source [SID1234654578]). The milestone was triggered when Arrowhead reached the first of two prespecified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy. Arrowhead currently expects to achieve the second enrollment target by the end of 2025, which would trigger an additional $200 million milestone payment from Sarepta.

In accordance with the license and collaboration agreement, Arrowhead expects to receive this payment within 60 days.

About the Arrowhead-Sarepta Agreement

Arrowhead and Sarepta signed a global licensing and collaboration agreement in November 2024, which closed in February 2025, whereby Sarepta received rights to multiple investigational treatments that leverage Arrowhead’s leading Targeted RNAi Molecule (TRiMTM) platform. The agreement covers multiple clinical and preclinical programs in rare, genetic diseases of the muscle, central nervous system, and the lungs, and also allows Sarepta to select up to six new targets for Arrowhead to conduct discovery and preclinical development activities in areas complementary to Sarepta’s leadership in precision genetic medicine for rare diseases.

Summary Financial Terms

Upon closing, Arrowhead received a $500 million upfront payment and $325 million through the purchase by Sarepta of Arrowhead common stock priced at $27.25 per share, representing a 35% premium to the 30-day volume weighted average price (VWAP) when the agreement was signed. Arrowhead will also receive $250 million to be paid in annual installments of $50 million over five years, and has the potential to receive an additional $300 million in near-term milestone payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study of ARO-DM1, $100 million of which has now been earned.

Arrowhead is also eligible to receive further development milestone payments of between $110 million and $180 million per program, sales milestone payments of between $500 million and $700 million per program, and tiered royalties on commercial sales up to the low double digits.

Summary of Programs Under the License and Collaboration Agreement

Clinical Stage

ARO-DUX4, which is designed to target the gene that encodes the DUX4 protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy type 1, currently dosing patients in a Phase 1/2 clinical study.
ARO-DM1, which is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK), gene in skeletal muscle as a potential treatment for patients with type 1 myotonic dystrophy, currently dosing patients in a Phase 1/2 clinical study.
ARO-MMP7, which is designed to reduce expression of matrix metalloproteinase 7 (MMP7) in the lung as a potential treatment for idiopathic pulmonary fibrosis, currently dosing patients in a Phase 1/2 clinical study.
ARO-ATXN2, which is designed to silence expression of the toxic ATXN2 protein in the CNS as a potential treatment for spinocerebellar ataxia 2 (SCA2), currently in a Phase 1/2 study that is open for enrollment.
Preclinical Stage

ARO-HTT for patients with Huntington’s disease, expected to be CTA-ready in 2025
ARO-ATXN1 for patients with spinocerebellar ataxia 1 (SCA1) expected to be CTA-ready in 2026
ARO-ATXN3 for patients with spinocerebellar ataxia 3 (SCA3) expected to be CTA-ready in 2026
Discovery

During the five-year term, Sarepta can propose up to six new CNS or muscle targets for which Arrowhead will perform discovery and preclinical development. Sarepta would then receive an exclusive license to those programs and would be responsible for subsequent clinical development and commercialization.

Drug Manufacturing

Under the terms of the agreement, Arrowhead will manufacture clinical drug supply for all programs arising out of the license and collaboration, and commercial drug product for the four programs currently in clinical trials.