On October 27, 2025 OncoC4 reported that China’s National Medical Product Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for gotistobart (BNT316/ONC392) for the treatment of patients with squamous non-small cell lung cancer (sqNSCLC) who have progressed on prior standard immuno-oncology therapies (IO). Gotistobart is a next-generation anti-CTLA-4 antibody candidate which is being jointly developed by BioNTech and OncoC4.

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Squamous non-small cell lung cancer (sqNSCLC) is an aggressive subtype of lung cancer accounting for approximately 25-30% of all NSCLC cases, the most common type of lung cancer1. The 5-year relative survival rate for patients with sqNSCLC is 15%2. While novel first line treatments combining immune-checkpoint inhibition and chemotherapy have improved outcomes of patients with NSCLC, the medical need remains high for novel treatments options, especially for patients with lung cancer that have progressed on first-line therapy and have limited treatment options.

"For the past 5 years, limited advances in treatment outcomes have been reported for EGFR wild-type NSCLC patients who progressed on the first-line IO and chemotherapies. We believe gotistobart has the potential to broaden the reach of CTLA-4-targeting immunotherapy and to improve outcomes for patients living with sqNSCLC." said Pan Zheng, MD, PhD, Co-founder and Chief Medical Officer of OncoC4.

"Gotistobart is a next-gen acid pH-sensitive anti-CTLA4 mAb that conferred an improved therapeutic index compared with approved anti-CTLA-4 antibodies by preserving CTLA-4 on regulatory T cells as demonstrated in preclinical models. The emerging data support clinical proof of concept for this new approach", added Yang Liu, PhD, Co-Founder, Chief Executive Officer, and Chief Scientific Officer of OncoC4.

Gotistobart is currently being evaluated in a registrational two-stage randomized Phase 3 trial, PRESERVE-003 (NCT05671510). The clinical trial assesses the efficacy and safety of gotistobart as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic sqNSCLC that progressed under previous PD-(L)1-inhibitor treatment. The initiation of the Phase 3 trial was based on positive safety and efficacy data from an ongoing Phase 1/2 trial (NCT04140526) with gotistobart alone and in combination with pembrolizumab in patients with advanced solid tumors. The program received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in 2022 and represents an innovative approach which aims to leverage the full potential of CTLA-4-targeting therapies.

Breakthrough Therapy Designation is an NMPA program designed to expedite the development and regulatory review of investigational therapies that are designed to address serious or life-threatening conditions. To receive the designation, the candidate needs to demonstrate preliminary clinical evidence that indicates that it may offer substantial improvement over existing therapies on one or more clinically significant endpoints. The NMPA’s BTD designation is based on the Stage 1 safety and efficacy data of the PRESERVE-003 trial. The detailed data will be reported in an upcoming medical conference in December 2025. With the Breakthrough Therapy designation, the development of gotistobart may benefit from more frequent engagement with the NMPA, which will support the collection of appropriate data needed to accelerate development and may also allow for priority review if the relevant criteria are met.

About PRESERVE-003

The two-stage, open-label, randomized Phase 3 trial, PRESERVE-003 (NCT05671510), will assess the efficacy and safety of gotistobart (BNT316/ONC-392) as monotherapy compared to the standard-of-care chemotherapy (docetaxel) in patients with metastatic NSCLC that progressed under previous PD-(L)1-inhibitor treatment. Stage 1, the non-pivotal dose-confirmation stage, assessed the efficacy and safety of two gotistobart dosing regimens in comparison to docetaxel in patients with squamous and non-squamous non-small cell lung cancer (NSCLC). Stage 2 is the pivotal stage of the trial and will assess the safety and efficacy of gotistobart in squamous cell NSCLC. The primary endpoint measure is overall survival. Secondary endpoints include overall response rate, progression-free survival and adverse event profile. Approximately 600 patients are planned to be enrolled at clinical sites in 12 countries and regions globally, including China, Germany, Italy, South Korea, Türkiye, the United Kingdom, and the United States.

About gotistobart (BNT316/ONC392)

Gotistobart is licensed to BioNTech for commercialization and jointly developed clinically by OncoC4 and BioNTech for oncology indications. There are several ongoing clinical trials in different tumor types, investigating gotistobart either as monotherapy or in combination with other therapeutic agents.

(Press release, OncoC4, OCT 27, 2025, View Source [SID1234657045])

On October 27, 2025 Kazia Therapeutics Limited ("Kazia" or the "Company") reported its intention to request and hold a follow-up Type C meeting with the U.S. Food & Drug Administration (FDA) to discuss overall survival (OS) findings in newly diagnosed glioblastoma (GBM) patients treated with paxalisib and to seek agency feedback on a potential regulatory pathway aligned with the FDA Oncology Center of Excellence’s Project FrontRunner initiative.

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"GBM remains one of the most lethal cancers with limited therapeutic options. In line with the FDA Oncology Center of Excellence’s Project FrontRunner initiative, we intend to engage the Agency to discuss whether the overall survival data generated in newly diagnosed GBM patients treated with paxalisib may be adequate to support a conditional approval pathway," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "Consistent with this framework, Kazia will propose initiation of the post-approval, randomized Phase 3 confirmatory study prior to submission of the NDA, ensuring that our regulatory strategy fully reflects the FDA’s renewed emphasis on overall survival as the most meaningful endpoint for patients and clinicians."

In its recently issued draft guidance, the FDA stated that overall survival is the "gold standard" endpoint in oncology and "should be prioritized as the primary endpoint when feasible," particularly in diseases with a short natural history where survival can be reliably assessed. Kazia believes GBM is precisely such a setting and intends to present survival analyses, supporting clinical safety, and planned confirmatory trial design for FDA discussion.

Project FrontRunner is an FDA Oncology Center of Excellence initiative encouraging sponsors to consider when it may be appropriate to seek approval of cancer drugs for advanced or metastatic disease in an earlier clinical setting, rather than the traditional approach of developing therapies only for patients who have exhausted available treatment options.

As announced in July 2024, in the prespecified secondary analysis in newly diagnosed (up-front) unmethylated GBM patients, median OS was 15.54 months in the paxalisib arm (n = 54) versus 11.89 months for concurrent standard of care (SOC) (n = 46). Kazia intends to reference Project FrontRunner principles in its Type C briefing package, including an OS-driven confirmatory study plan in newly diagnosed GBM.

"We are moving decisively to bring paxalisib forward in GBM using the endpoints that matter most to patients and physicians," added Dr. Friend. "Our objective is to work collaboratively with the FDA under the guiding principles of Project FrontRunner to pursue a conditional approval in the front-line treatment setting of glioblastoma. In parallel, Kazia will initiate the post-approval, randomized Phase 3 study prior to filing the NDA, ensuring that our development plan fully aligns with the Agency’s modernized, patient-focused framework."

Kazia also notes that leading oncology companies have begun publicly referencing Project FrontRunner in successful FDA actions, underscoring the initiative’s growing relevance for sponsors developing first-line or earlier-setting therapies.

(Press release, Kazia Therapeutics, OCT 27, 2025, View Source [SID1234657044])

On October 27, 2025 Hengrui Pharma (600276.SH; 01276.HK) reported revenue of RMB23.20 billion for the first three quarters of 2025, up 14.85% year-on-year. Net profit attributable to shareholders was RMB5.75 billion, increased by 24.50% compared with the same period last year.

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One NME was approved in China, while eight NDA submissions were made and 48 clinical trial approvals were granted during the period, with four programs admitted to breakthrough-therapy designation lists. The GLP-1/GIP dual agonist HRS9531 reported positive Phase III topline results in weight management, advancing one of the company’s key late-stage metabolic assets.

At ESMO (Free ESMO Whitepaper) 2025 in Berlin, Hengrui presented 46 oncology studies across 14 innovative programs — including nine oral presentations, one of which was published in The Lancet — marking its strongest presence to date at a major global cancer congress. In parallel, Hengrui signed three licensing deals with multinational partners in the third quarter, collectively exceeding US$10 billion in potential transaction value.

Hengrui will remain focused on advancing its homegrown R&D innovation and strengthening global execution through clinical development and strategic partnerships to deliver more cutting-edge therapies to patients worldwide.

(Press release, Hengrui Pharmaceuticals, OCT 27, 2025, View Source [SID1234657043])

On October 27, 2025 Diakonos Oncology Corp., a clinical-stage biotechnology company developing a new generation of immunotherapies to treat challenging and aggressive cancers, reported a poster presentation at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 40th Annual Meeting, being held November 5-9 in National Harbor, MD.

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"DOC1021 represents a novel and highly targeted approach to activating the patient’s own immune system against cancer cells," said Jay Hartenbach, President and COO of Diakonos Oncology. "Building on promising results in glioblastoma, we are now excited to share preliminary data in pancreatic cancer. Based on this early data, we believe this patient-derived immunotherapy holds great potential for pancreatic cancer and other indications with a high unmet need."

Presentation Details
Title: Phase I Trial of DOC1021 Cell-Based Immunotherapy for Resectable or Borderline Resectable Pancreatic Ductal Adenocarcinoma
Presenting Author: William Decker, PhD, Baylor College of Medicine, Houston, TX
Abstract Number: 540
Session Type: Poster Presentation Session
Session Date: Saturday, November 8, 2025
Location: Prince George ABC Exhibit Halls, Gaylord National Resort and Convention Center

About DOC1021
DOC1021 is a first-in-class, patient-derived double-loaded dendritic cell therapy that uniquely combines tumor lysate and amplified tumor-derived mRNA. The immunotherapy is made with a patient’s dendritic cells combined with mRNA and proteins prepared from freshly obtained patient tumor specimens.

The unique double-loading approach, which mimics a viral infection, unlocks a synergistic and exponentially more powerful tumor killing TH1 response driven by dual protein and RNA antigen sourcing, and it allows targeting of the complete cancer antigen pool. Moreover, the approach does not require any molecular modification of the patient’s immune cells for manufacturing, and does not require preconditioning chemotherapy or high dose IL-2 for administration. DOC1021 allows for a simple administration in the outpatient setting and broad reach via community cancer centers.

In addition to the Phase I pancreatic cancer study (NCT04157127), Diakonos recently opened a Phase 2 glioblastoma (GBM) study with DOC1021 (NCT06805305). Diakonos has received Fast Track designations from the FDA for both the GBM and pancreatic cancer programs, in October 2023 and May 2024, respectively. The company has also received Orphan Drug Designation for the GBM program in January 2024.

(Press release, Diakonos Oncology, OCT 27, 2025, View Source [SID1234657042])

On October 27, 2025 Onward Therapeutics SA, a clinical stage oncology company, reported that its French subsidiary, Onward Therapeutics France, has exercised its exclusive option and entered into a worldwide license agreement with Institut du Cancer de Montpellier (ICM), France, to further advance a breakthrough program in onco-metabolism.

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This license agreement builds upon the exclusive collaboration and option agreement signed between Onward Therapeutics France and ICM, Inserm and Université de Montpellier in June 2021, under which the company provided research funding to support the discovery program at ICM. The successful progress achieved through this collaboration has led to the identification of promising compounds and the transition of the drug discovery program into Onward Therapeutics’ development pipeline. The program is currently in lead optimization, with the goal of nominating IND-enabling candidates next year.

The program, designated OT-S00X by Onward Therapeutics, is an innovative onco-metabolism project aimed at expanding therapeutic possibilities in cancer treatment. Proof-of-principle studies have demonstrated strong preclinical efficacy, and promising candidates from two distinct chemical series have already been identified, and compounds with improved physico-chemical properties will be generated through artificial intelligence approaches.

"OT-S00X represents a breakthrough in targeting tumor onco-metabolism. Preclinical studies have shown that the efficacy of these compounds strongly correlates with a predictive biomarker of response across multiple tumor types, providing a clear rationale for this novel mechanism of action." said Dr. Armand de Gramont, Chief Scientific Officer of Onward Therapeutics."OT-S00X introduces a differentiated metabolic strategy that broadens our pipeline and primarily provides an innovative way to tackle cancer resistance," added Dr Alain Herrera, President of Onward Therapeutics France and Chief Medical Officer of Onward Therapeutics.

"We are delighted to strengthen our collaboration with Onward Therapeutics," said Prof. Marc Ychou, Directeur Général of ICM. "The innovative OT-S00X program, initially developed by our research teams together with Inserm and Université de Montpellier, demonstrates the potential of translational science to generate novel therapeutic strategies in oncology. It also highlights the international reach of ICM and the strength of the Montpellier MedVallée ecosystem in driving cancer innovation."

(Press release, Onward Therapeutics, OCT 27, 2025, View Source [SID1234657041])