On October 17, 2025 SystImmune Inc. (SystImmune), a clinical-stage biotechnology company, and Bristol Myers Squibb (NYSE: BMY) reported the oral presentation of the first disclosure of the safety and efficacy data from the global phase I US-Lung-101 study (NCT05983432) of iza-bren (BL-B01D1), a potentially first-in-class EGFR x HER3 bispecific antibody-drug conjugate (ADC), at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 in Berlin, Germany. Iza-bren is jointly developed by SystImmune and Bristol Myers Squibb under a collaboration and exclusive license agreement in territories outside of Mainland China. In August 2025, iza-bren was granted breakthrough therapy designation by U.S. FDA for patients with previously treated EGFR-mutated NSCLC based on the data from China studies and this global study.

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This study evaluated the safety and efficacy of iza-bren in global patients with heavily pre-treated metastatic or unresectable advanced non-small cell lung cancer (NSCLC) and other solid tumors. At the data cut-off (DCO) of July 23, 2025, iza-bren has demonstrated:

Promising antitumor activity in heavily pre-treated patients across multiple tumor types, including EGFR mutant and wildtype NSCLC
Manageable safety profile, with hematologic adverse events effectively managed by standard medical measures, and no interstitial lung disease was observed
In the study, 107 patients with advanced solid tumors were treated, including non-small cell lung cancer (NSCLC) patients with and without EGFR mutations. Most had received several prior therapies. The most common side effects were blood-related, such as neutropenia. These were generally manageable and rarely led to dose reductions or serious complications. No new safety concerns were identified, and no cases of interstitial lung disease were seen. Mandatory preventive measures for neutropenia have been added in ongoing global studies.

For patients receiving 2.5 mg/kg (Days 1 and 8 every 3 weeks), 55% (11 of 20) showed a confirmed response, with a median progression-free survival of 5.4 months. Confirmed responses were also seen in both the subgroup with EGFR-mutated NSCLC (3 of 10 patients) and those without the mutation (3 of 4 patients).

Global registrational studies of first line metastatic TNBC (IZABRIGHT-Breast01, NCT06926868), second line metastatic EGFRmt NSCLC (IZABRIGHT-Lung01, NCT05983432) and second line metastatic Urothelial Cancer (IZABRIGHT-Bladder01, NCT07106762) are ongoing, with studies in other indications planned.

"The first global presentation of iza-bren builds on the compelling data initially observed in Chinese patients, showing consistent efficacy in a heavily pre-treated global population," said Jonathan Cheng, M.D., Chief Medical Officer, SystImmune. "These results support iza-bren’s potential as a bispecific ADC treatment option across multiple tumor types, and we are excited to continue advancing this important program through our global collaboration with Bristol Myers Squibb."

"We are committed to developing first-in-class and best-in-class medicines that can meaningfully improve outcomes for patients with difficult-to-treat cancers," said Anne Kerber, Senior Vice President, Head of Development, Hematology, Oncology and Cell Therapy, Bristol Myers Squibb. "The encouraging activity observed with iza-bren in this early global study reinforce our confidence in its potential, and we look forward to the ongoing registrational studies across lung, breast, and urothelial cancers."

About the BL-B01D1-LUNG-101 Phase I clinical trial
BL-B01D1-LUNG-101 (NCT05983432) is a global, multi-center, Phase I study to evaluate the safety, tolerability, pharmacokinetics, and initial efficacy of iza-bren in participants with metastatic or unresectable NSCLC and other solid tumors. This study will be conducted in two different dosing schedules (Cohort A and Cohort B) and three parts (dose escalation, dose finding and dose expansion). Cohort A will be dosed on Day 1 and Day 8 of a continuous 21-day treatment cycle. Cohort B will be dosed on Day 1 of a continuous 21-day treatment cycle. The primary endpoint includes safety. Secondary endpoints include objective response rate (ORR) by RECIST 1.1 criteria, duration of response (DoR), disease control rate (DCR), progression-free survival (PFS) and overall survival (OR) and PK analysis.

About EGFRmt NSCLC
Non-small cell lung cancer (NSCLC) accounts for approximately 80% of all lung cancer cases, which remains the leading cause of cancer-related death worldwide. Among patients with NSCLC, 10% to 15% in Western populations and up to 50% in Asian populations harbor activating EGFR mutations. These tumors, most commonly of non-squamous histology, initially respond to EGFR TKIs such as osimertinib. However, resistance is nearly universal, often occurring after about 18 months, and treatment options beyond TKIs and platinum-based chemotherapy provide limited clinical benefit with significant toxicities, highlighting the critical need for new, effective therapies.

About iza-bren
SystImmune, in collaboration with BMS outside of China, is developing iza-bren (BL-B01D1), a bispecific antibody-drug conjugate (ADC) that targets both EGFR and HER3, which are highly expressed in various epithelial cancers and are known to be associated with cancer cell proliferation and survival. Iza-bren’s dual mechanism of action blocks EGFR and/or HER3 signals to cancer cells, reducing cancer cell proliferation and survival. In addition, upon antibody mediated internalization, iza-bren’s therapeutic novel Topo1i payload is released causing cytotoxic stress that leads to cancer cell death.

(Press release, Bristol-Myers Squibb, OCT 17, 2025, View Source;and-Bristol-Myers-Squibb-Announce-First-Global-Phase-I-Results-of-Iza-bren-an-EGFR-x-HER3-Bispecific-Antibody-Drug-Conjugate-in-Patients-with-Advanced-Solid-Tumors-at-ESMO-2025/default.aspx [SID1234656729])

On October 17, 2025 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported the expansion of its Phase III clinical development program of the novel, potential first-in-class investigational bispecific antibody, ivonescimab, into colorectal cancer (CRC) with the initiation of the global Phase III HARMONi-GI3 trial.

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Summit is starting a Phase III clinical study, HARMONi-GI3, to evaluate ivonescimab plus chemotherapy compared to bevacizumab plus chemotherapy as first line therapy in patients with unresectable metastatic colorectal cancer (CRC).

Clinical trial sites for HARMONi-GI3 are planned to begin activating in the United States prior to the end of the year. The multiregional study intends to enroll approximately 600 patients. The primary endpoint for this study is progression-free survival.

Each year, approximately 48,000 patients are estimated to be diagnosed with or have unresectable recurrent metastatic MSS CRC (also known as mismatch repair-proficient colorectal cancer, or pMMR CRC).1 There have been limited options approved in the United States in the last 20 years for those first-line patients whose tumors are not positive for certain biomarkers or other activating mutations.

MSS CRC is a setting where monoclonal PD-1 inhibitors such as pembrolizumab and nivolumab have failed to show a clinically meaningful benefit. Anti-VEGF therapy (e.g., bevacizumab) plus chemotherapy is the standard of care for many patients with 1L metastatic MSS CRC.

At the 2024 Annual Congress of the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) (ESMO 2024), Akeso Inc. (Akeso) presented encouraging Phase II data of ivonescimab in combination with FOLFOXIRI chemotherapy demonstrating an objective response rate (ORR) of 81.8% (95% CI: 59.7% – 94.8%) and a disease control rate (DCR) of 100% (95% CI: 84.6% – 100.0%) in 22 patients. This Phase II study, AK112-206, was conducted in China and sponsored by Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso. In the ivonescimab plus FOLFOXIRI chemotherapy arm, there were no treatment emergent adverse events that led to permanent discontinuation of ivonescimab as of the data cutoff from the ESMO (Free ESMO Whitepaper) 2024 presentation.

Subsequently, AK112-206 was expanded to include additional patients from the United States and China to study ivonescimab in combination with FOLFOX chemotherapy. FOLFOX in combination with a monoclonal antibody such as bevacizumab represents a preferred treatment regimen for physicians treating patients in the United States and other western territories. The data from the initial patient cohort presented at ESMO (Free ESMO Whitepaper) 2024 have continued to mature, in addition to the global Phase II data generated in combination with FOLFOX in the United States and China, which support the Phase III HARMONi-GI3 study design using FOLFOX.

"As promised earlier in the year, we sought to expand the ivonescimab clinical development program beyond non-small cell lung cancer in order to continue to explore the potential benefits of its specifically-engineered, novel design and mechanism of action, including its cooperative binding attributes," stated Robert W. Duggan and Dr. Maky Zanganeh, Co-Chief Executive Officers of Summit. "Microsatellite stable colorectal cancer represents a global unmet need whereby ivonescimab has the potential to bring the benefits of immunotherapy to solid tumors where PD-1 monoclonal antibodies have not been able to successfully improve upon existing standards of care. We are thrilled by the potential of ivonescimab to make a significant difference to these patients with few front-line options available today."

Conference Call

Summit Therapeutics Inc. will host a conference call and live webcast to discuss recent updates related to ivonescimab, including data released at ESMO (Free ESMO Whitepaper), on Monday, October 20, 2025, at 8:00am ET. Conference call and webcast information will be accessible through our website www.smmttx.com.

An archived edition of the webcast will be available on our website later in the day on Monday.

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, North America, South America, Europe, the Middle East, Africa, and Japan, and as AK112 in China and Australia, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab displays unique cooperative binding to each of its intended targets with multi-fold higher affinity to PD-1 when in the presence of VEGF.

This could differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Ivonescimab’s specifically engineered tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME (Zhong, et al., SITC (Free SITC Whitepaper), 2023). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of this design, together with a half-life of 6 to 7 days after the first dose (Zhong, et al., SITC (Free SITC Whitepaper), 2023) increasing to approximately 10 days at steady state dosing, is to improve upon previously established efficacy thresholds, in addition to side effects and safety profiles associated with these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently engaged in multiple Phase III clinical trials. Over 3,000 patients have been treated with ivonescimab in clinical studies globally.

Summit began its clinical development of ivonescimab in non-small cell lung cancer (NSCLC), commencing enrollment in 2023 in two multiregional Phase III clinical trials, HARMONi and HARMONi-3. In early 2025, the Company began enrolling patients in the United States for HARMONi-7. Summit intends to open clinical trial sites in the United States for the Phase III study in colorectal cancer (CRC) by the end of 2025.

HARMONi is a Phase III clinical trial which intends to evaluate ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who were previously treated with a 3rd generation EGFR TKI (e.g., osimertinib). Enrollment in HARMONi was completed in the second half of 2024, top-line results were announced in May of 2025, with detailed results provided in September 2025.

HARMONi-3 is a Phase III clinical trial which is intended to evaluate ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic, squamous or non-squamous NSCLC, irrespective of PD-L1 expression.

HARMONi-7 is a Phase III clinical trial which is intended to evaluate ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression.

HARMONi-GI3 is a planned Phase III clinical trial evaluating ivonescimab in combination with chemotherapy compared with bevacizumab plus chemotherapy in patients with first-line unresectable metastatic CRC.

In addition, Akeso has recently had positive read-outs in three single-region (China), randomized Phase III clinical trials for ivonescimab in NSCLC: HARMONi-A, HARMONi-2, and HARMONi-6.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression.

HARMONi-6 is a Phase III clinical trial evaluating ivonescimab in combination with platinum-based chemotherapy compared with tislelizumab, an anti-PD-1 antibody, in combination with platinum-based chemotherapy in patients with locally advanced or metastatic squamous NSCLC, irrespective of PD-L1 expression.

Akeso is actively conducting multiple Phase III clinical studies in settings outside of NSCLC, including biliary tract cancer, colorectal cancer, breast cancer, pancreatic cancer, small cell lung cancer, and head and neck cancer.

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was initially approved for marketing authorization in China in May 2024. Ivonescimab was granted Fast Track designation by the US Food & Drug Administration (FDA) for the HARMONi clinical trial setting.

(Press release, Summit Therapeutics, OCT 17, 2025, View Source [SID1234656762])

On October 17, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Libtayo (cemiplimab) as an adjuvant treatment for adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation. The European Commission is expected to make a final decision on the application in the coming months. Libtayo was approved by the U.S. Food and Drug Administration (FDA) for these patients in the U.S. earlier this month.

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The positive opinion is supported by results from the global Phase 3 C-POST trial investigating adjuvant Libtayo versus placebo in patients with CSCC at high risk of recurrence following surgery and radiation. In the trial, Libtayo reduced the risk of disease recurrence or death by 68% compared to placebo (hazard ratio [HR]: 0.32; 95% confidence interval [CI]: 0.20-0.51; p<0.0001). Fewer patients treated with Libtayo had locoregional or distant recurrence compared with those who received placebo (4% vs. 17% and 5% vs. 13%, respectively). Detailed data were published in the New England Journal of Medicine (NEJM) in May 2025.

The safety profile of Libtayo as adjuvant treatment of patients with CSCC at high risk of recurrence after surgery and radiation is consistent with the known safety profile for Libtayo monotherapy in advanced cancers. In the trial, adverse events (AEs) occurred in 91% of patients receiving Libtayo (n=205) and 89% of patients receiving placebo (n=204). Grade ≥3 AEs occurred in 24% and 14% of patients in the Libtayo arm and the placebo arm, respectively. The most common AEs occurring in at least 10% of patients who received Libtayo were fatigue, pruritus, rash, diarrhea, arthralgia, hypothyroidism and maculo-papular rash. The only grade ≥3 AE that occurred in more than 2% of patients in the Libtayo arm was hypertension. AEs led to permanent discontinuation of treatment in 10% of patients who received Libtayo and 2% of patients who received placebo. Two patients in each arm experienced an AE leading to death.

About the Phase 3 Trial
C-POST was a randomized, placebo-controlled, double-blind, multicenter, global Phase 3 trial investigating Libtayo versus placebo as adjuvant treatment for patients with features associated with a high risk of CSCC recurrence and who had completed surgery and post-operative radiation therapy. Trial participants were at high risk of recurrence due to nodal features (extracapsular extension or ≥3 involved lymph nodes) and/or non-nodal features (in-transit metastases, T4 lesion, perineural invasion, or locally recurrent tumor with ≥1 additional poor prognostic features).

The trial enrolled 415 patients who were randomized to receive either Libtayo (n=209) or placebo (n=206) for up to 48 weeks. For the first 12 weeks, Libtayo 350 mg or placebo was administered intravenously every three weeks, followed by Libtayo 700 mg or placebo administered intravenously every six weeks for 36 weeks.

About CSCC
Cutaneous squamous cell carcinoma (CSCC) is a type of non-melanoma skin cancer (NMSC), and one of the most common cancers in the world. In the EU, the incidence of NMSC overall is expected to increase by 40% by 2040. CSCC can often be treated successfully with surgery, but many patients may have a "high risk" form that is more aggressive, and they face an increased risk of recurrence and disease progression.

(Press release, Regeneron, OCT 17, 2025, View Source [SID1234656777])

On October 17, 2025 Corvus Pharmaceuticals, Inc. (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported that interim data from the Phase 1b/2 clinical trial of ciforadenant for patients with metastatic renal cell cancer (RCC) will be presented today in an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025, which is taking place October 17-21, 2025 in Berlin, Germany. The data will be presented by Katy Beckermann, M.D., Ph.D., Director of Genitourinary Cancer Research at Tennessee Oncology and member of the Kidney Cancer Research Consortium (KCRC), the group that is conducting the trial in collaboration with Corvus.

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"We are encouraged by these results exploring ciforadenant in combination with ipilimumab and nivolumab as a potential front-line treatment for renal cell carcinoma," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "Despite enrolling patients with more unfavorable disease compared to historical trials, the triplet combination demonstrated activity that compares favorably to historical results with the doublet alone. These data support our view that blocking adenosine signaling with ciforadenant may provide meaningful benefit for RCC patients. We appreciate the partnership with the Kidney Cancer Research Consortium and we look forward to continuing to follow the 19 patients who remain on therapy to better understand the potential of this approach."

The open-label Phase 1b/2 clinical trial is evaluating ciforadenant, the Company’s adenosine A2a receptor inhibitor, as a potential first line therapy for metastatic RCC in combination with ipilimumab (anti-CTLA-4) and nivolumab (anti-PD-1). The trial enrolled 50 patients (8 in Phase 1b portion, 42 in Phase 2 portion) with newly diagnosed or recurrent stage IV clear cell RCC that had not received any prior systemic therapy. Patients received ciforadenant 100 mg oral, twice-daily in combination with ipilimumab (anti-CTLA-4) 1mg/kg given once every three weeks for twelve weeks (4 doses) and nivolumab (anti-PD-1) 3mg/kg given once every three weeks.

The primary endpoint for the Phase 1b portion is safety, tolerability and anti-tumor response. The primary endpoint for the Phase 2 portion is the percent of patients that achieve a deep response, defined as complete response or depth of partial response of >50% tumor volume reduction. Historical data from the Kidney Cancer Research Consortium has shown that deep responses correlate with prolonged progression free survival and that they occur in approximately 32% of patients receiving ipilimumab and nivolumab. Secondary endpoints for the Phase 2 portion include objective response rate (ORR), progression-free survival (PFS) and treatment-related adverse events.

The interim data being presented at ESMO (Free ESMO Whitepaper) demonstrates that triplet therapy with ciforadenant, ipilimumab and nivolumab is feasible and well tolerated. Key highlights from the presentation (data as of May 2025) include:

Patients in the trial had a median age of 61.5 years (53-70 years range) and had unfavorable disease characteristics, with only 54% having a prior nephrectomy (~75-85% is typical for studies involving similar patients). Nephrectomy is associated with improved outcomes in advanced RCC and is often not performed in patients with poor prognosis. In addition, 82% of patients in the trial had a poor or intermediate prognosis by International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) criteria.
The treatment was well-tolerated, in-line with the safety profile of combination treatment with ipilimumab and nivolumab.
The deep response rate was 34%, demonstrating an improvement compared to historical data for the combination of ipilimumab and nivolumab alone, though not statistically significant at this point in time. 19 patients with stable or responding disease remain on therapy with the potential to achieve deep responses.
The ORR by was 46%, including two complete responses and 21 partial responses. The median PFS is 11.04 months.
Dr. Beckermann commented, "Early results from this trial are encouraging, demonstrating consistent efficacy and favorable safety in a challenging RCC population, and we look forward to data from the 19 patients still on treatment."

(Press release, Corvus Pharmaceuticals, OCT 17, 2025, View Source [SID1234656730])

On October 17, 2025 Merck, a leading science and technology company, reported the presentation of longer-term results from the global Phase 3 MANEUVER trial evaluating pimicotinib, an investigational colony stimulating factor-1 receptor (CSF-1R) inhibitor in development by Abbisko Therapeutics Co., Ltd., for the treatment of patients with tenosynovial giant cell tumor (TGCT). This latest analysis showed that, with a median follow-up of 14.3 months, the objective response rate (ORR) for people treated with pimicotinib from the beginning of the study increased considerably to 76.2% (95% CI: 63.8, 86.0) by blinded independent review committee (BICR) per RECIST v1.1, from 54% at Week 25. The study also showed continued clinically meaningful improvements in key secondary endpoints related to patient outcomes such as pain and function. The safety profile was consistent with previously reported data. The results are being presented today in the Sarcoma mini-oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 (Presentation # 2690MO).

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"With the initial results of the global MANEUVER study presented earlier this year at ASCO (Free ASCO Whitepaper), pimicotinib demonstrated the highest objective response rate seen in a Phase 3 clinical trial of a systemic therapy in TGCT," said Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. "These latest findings build on those impressive results, showing that these tumor responses not only persist but deepen over time. Importantly, we also see continued improvements beyond one year in the patient-reported symptoms and functional outcomes that truly make a difference in patients’ abilities to go about their daily lives. Together, these findings indicate the potential for pimicotinib to be a best-in-class systemic treatment for patients with TGCT."

"TGCT causes pain, stiffness, and loss of range of motion, affecting patients’ ability to participate in activities of daily living and with their families or communities. Ultimately, this affects everyone and takes a mental and physical toll," said Sydney Stern, PhD, MS, TGCT Support, a Program of the Life Raft Group. "Patients benefit from more options that address their symptoms and shrink the disease. Importantly, addressing their symptoms enables patients to be the parents, partners, carers, and people they want to be without wondering when their TGCT will take over their life again."

The latest analysis of the global Phase 3 MANEUVER trial includes results from 63 patients who received pimicotinib for 24 weeks in Part 1 and then continued on pimicotinib in the open-label phase of the trial. With a median follow-up of 14.3 months, tumor responses continued to improve:

ORR per BIRC based on RECIST v1.1 increased to 76.2% (95% CI: 63.8, 86.0), from 54% at Week 25.
ORR per BIRC based on tumor volume score (TVS), an endpoint designed specifically for TGCT, increased to 74.6% (95% CI: 62.1, 84.7), from 61.9% at Week 25.
At the time of the data cutoff, the median duration of response was not reached (range: 0.03-19.81 months), with 93.7% of pimicotinib-treated patients experiencing a reduction in tumor size by BIRC per RECIST v1.1 at longer-term follow up.
Pimicotinib also demonstrated clinically meaningful improvements with longer-term follow-up up to week 73 in key patient-reported measures including range of motion, pain, stiffness and physical function that significantly impact people living with TGCT:

For relative range of motion, pimicotinib showed a mean change from baseline of 23.9% (increased from 15.6% at week 25).
Mean change from baseline continued to show improvements in physical function as measured by the patient-reported PROMIS-PF scale, and reductions in stiffness and pain as measured by the Worst Stiffness Numeric Scale Rating and Brief Pain Inventory worst pain rating, respectively.
The analysis also includes results for patients who were initially randomized to receive placebo in Part 1, then switched to pimicotinib in the open label part of the study (n=31). These patients experienced a clear benefit from pimicotinib treatment, with an ORR of 64.5% both by BICR per RECIST v1.1 and by TVS with a median follow-up of 8.5 months after switching to pimicotinib.

At longer-term follow-up, in patients who had received pimicotinib throughout the study, there were no new safety signals, and no evidence of cholestatic hepatotoxicity, drug-induced liver injury or hair/skin hypopigmentation. Most treatment-emergent adverse events remained mild in severity and were manageable.

"These longer-term results highlight the potential of pimicotinib to transform care by providing a systemic therapy that delivers meaningful, lasting benefit not only in terms of reducing tumor burden but in helping patients regain function and live with reduced pain," said Victoria Zazulina, M.D., Head of Development Unit, Oncology, Healthcare business of Merck. "Guided by this robust global study—conducted across North America, Europe and China—we are working with regulatory authorities as we seek to make this treatment available to patients as quickly as possible."

An application for marketing authorization of pimicotinib as a Class 1 innovative drug for adult patients with TGCT has been accepted for review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA). Additional applications are planned in the U.S. and other markets around the world.

About MANEUVER

The pivotal global Phase 3 MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT who require systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted by Abbisko Therapeutics in China (n=45), Europe (n=28), and the U.S. and Canada (n=21).

In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint was objective response rate (ORR) at week 25, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review (BICR) in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score (TVS), relative range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS-PF).

After the double-blind Part 1, eligible patients could continue to the open-label Part 2 for up to 24 weeks of further treatment. Patients who completed Part 2 could then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up.

About Pimicotinib (ABSK021)

Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib has been granted breakthrough therapy designation (BTD) for the treatment of inoperable TGCT by China National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA), and priority medicine (PRIME) designation from the European Medicines Agency (EMA). Merck holds worldwide commercialization rights for pimicotinib.

Advancing the Future of Cancer Care

At Merck, we strive every day to improve the futures of people living with cancer. Building on our 350-year global heritage as pharma pioneers, we are focusing our most promising science to target cancer’s deepest vulnerabilities, pursuing differentiated molecules to strike cancer at its core. By developing new therapies that can help advance cancer care, we are determined to create a world where more cancer patients will become cancer survivors. Learn more at www.merckgroup.com.

(Press release, Merck & Co, OCT 17, 2025, View Source [SID1234656763])