Rakuten Medical’s Anti-PD-L1 Antibody-IR700 Conjugate, RM-0256, Selected for AMED Funding

On June 4, 2025 Rakuten Medical, Inc., a global biotechnology company developing and commercializing Alluminox platform-based photoimmunotherapy, reported that its third drug candidate, RM-0256, has been selected for funding by the Japan Agency for Medical Research and Development (AMED) under its Support Program for Orphan Drug prior to the Designation (Press release, Rakuten Medical, JUN 4, 2025, View Source [SID1234653727]).

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RM-0256 is a novel antibody conjugate composed of IRDye700DX (IR700)—a light-activatable dye for which Rakuten Medical holds exclusive global manufacturing and supply rights—and a proprietary anti-PD-L1 monoclonal antibody. The AMED funding will support the clinical development of RM-0256 photoimmunotherapy for unresectable, advanced, or recurrent malignant epithelial tumors.

In Japan, malignant epithelial tumors affect approximately 22,000 individuals annually1. While systemic therapies, including chemotherapy and immune checkpoint inhibitors, are currently the mainstay of treatment for unresectable advanced or recurrent cases, patients who develop resistance to these treatments face limited treatment options, which underscores the urgent need for novel therapeutic approaches.

RM-0256 targets Programmed Cell Death Ligand 1 (PD-L1), a key immune checkpoint protein that allows tumors to evade immune detection by binding to PD-1 receptors on activated T cells. PD-L1 is widely expressed in various solid tumors—including melanoma, ocular melanoma, lung, urothelial, gastrointestinal, gynecological, breast, and head and neck cancers2—as well as on immunosuppressive cells within the tumor microenvironment.

Pre-clinical studies of PD-L1-targeted photoimmunotherapy have suggested three complementary mechanisms of action3:

1) Direct depletion (necrosis) of PD-L1-expressing tumor cells;
2) Activation of anti-tumor immunity through the elimination of PD-L1–expressing immunosuppressive cells;
3) Checkpoint blockade, by inhibiting PD-L1/PD-1 interaction, potentially enhancing systemic immune responses.

With the support of AMED funding, Rakuten Medical is accelerating the development of RM-0256 photoimmunotherapy as a novel, multimodal cancer therapy that may induce both local and systemic anti-tumor effects.

Preliminary Data from Two Clinical Trials with ADC Candidates were Presented Orally at the 2025 ASCO Annual Meeting

On June 4, 2025 Duality Bio (HKEX: 9606.HK) reported the preliminary data of two clinical trials, HER3 ADC candidate DB-1310 and B7H3 ADC candidate DB-1311/BNT324, which is being jointly developed with BioNTech, in Oral/Rapid Oral presentations at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting taking place in Chicago, US, from May 30 to June 3 (Press release, DualityBio, JUN 4, 2025, View Source [SID1234653726]).

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B7H3 ADC candidate DB-1311/BNT324:

Data from the ongoing Phase 1/2 clinical trial (NCT05914116) in patients with heavily pre-treated castration-resistant prostate cancer (CRPC) were presented during an oral session on BNT324/DB-1311, an B7H3-targeted ADC candidate.

The data indicated early clinical activity and a manageable safety profile with low discontinuation rates. Most common adverse events were gastrointestinal and hematologic toxicities. In the 52 efficacy evaluable patients with heavily pretreated CRPC based on RCIST v1.1, the confirmed objective response rate (cORR) was 30.8%, DCR was 90.4%. Among 68 evaluable patients, the 6-month rPFS rate was 69.8%. Similar outcomes were observed across both dose levels (6 mg/kg or 9 mg/kg). Outcomes appeared better in earlier treatment lines and in patients who received one prior NHT, while antitumor activity was also observed in later lines and regardless of type of prior treatment or metastatic site.

The clinical trial is currently enrolling post Lu-177 CRPC (Cohort 11) and taxane-naïve CRPC (Cohort 12).

As the incidence rate of prostate cancer is increasing[1],1 there is a high unmet need for new effective therapies for patients with heavily pretreated CRPC[1]. The DB-1311/BNT324 program received Fast Track Designation by the U.S. Food & Drug Administration ("FDA") for the treatment of patients with advanced/unresectable or metastatic CRPC that has progressed on or after standard systemic regimens in 2024.

HER3 ADC DB-1310:

Data from the first-in-human Phase I/IIa study (NCT05785741) presented by Professor Aaron E. Lisberg of the University of California, Los Angeles (UCLA), demonstrated that DB-1310 showed encouraging efficacy and a manageable safety profile in patients with advanced solid tumors who had failed standard treatments.

Among 123 efficacy evaluable patients, the unconfirmed objective response rate (uORR) was 31%, and the disease control rate (DCR) reached 84%. Notably, efficacy was particularly striking in the key subgroup of patients with EGFR-mutated non-small cell lung cancer (NSCLC) (n=46) where uORR reached 44%, DCR was 91%, median progression-free survival (mPFS) was 7.0 months, and median overall survival (mOS) was 18.9 months. The uORR reached an impressive 66.7% at the 5.5 mg/kg Q3W dose level (n=12).

In addition, DB-1310 was well-tolerated with a manageable safety profile. The most common treatment-related adverse events (TRAEs) were Grade 1-2 hematological and gastrointestinal events with a low treatment-related discontinuation rate of 3.5%.

These positive results support the continued development of DB-1310 in advanced solid tumors, particularly in patients with EGFR-mutated NSCLC. The company is advancing its global development program, including exploring DB-1310 as a monotherapy in additional tumor types, as well as exploring DB-1310 in combination with EGFR TKIs and HER2-targeted therapies.

Veeva and Sarah Cannon Research Institute Form Strategic Collaboration to Advance Oncology Clinical Trials

On June 4, 2025 Veeva Systems (NYSE: VEEV) and Sarah Cannon Research Institute (SCRI) reported a strategic collaboration to drive speed and efficiency in oncology clinical trials across SCRI’s more than 200 research site locations (Press release, Sarah Cannon Research Institute, JUN 4, 2025, View Source [SID1234653725]). SCRI is adopting Veeva Clinical Platform to unify its contract research organization (CRO) and site management organization (SMO) on a single platform for seamless data flow across clinical teams and research sites.

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"We are thrilled to advance our clinical trials by integrating Veeva Clinical Platform into our digital toolkit," said SCRI chief information and digital officer Yazhene Krishnaraj. "This strategic collaboration empowers our clinical teams to deliver groundbreaking therapies to patients with enhanced precision and speed."

Veeva Clinical Platform will enable SCRI to simplify and standardize trial processes and information flow, improving how investigators and clinical teams work together and share data. With a connected foundation for clinical research, SCRI will be able to automate key processes and provide a streamlined experience for its sites.

"We’re excited to work closely with SCRI to drive innovation in oncology research," said Jim Reilly, president of Veeva Development Cloud. "In a first-of-a-kind partnership, Veeva Clinical Platform will serve as SCRI’s clinical trial foundation for its CRO and SMO. By standardizing operations on one platform, we can support SCRI in delivering faster and more cost-effective trials."

Continuity Biosciences Acquires Focal Medical to Advance Targeted Drug Delivery for Pancreatic Cancer

On June 4, 2025 Continuity Biosciences, LLC, a developer of advanced drug delivery technologies, reported the acquisition of Focal Medical, Inc., a North Carolina-based biopharmaceutical company pioneering site-specific chemotherapy using iontophoresis (Press release, Continuity Biosciences, JUN 4, 2025, View Source [SID1234653724]). The acquisition represents a key step in Continuity’s strategy to become a leader in device targeted therapeutics for intractable solid tumors.

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Founded by leading scientists and clinicians, Focal Medical has developed a proprietary platform that delivers chemotherapy directly to tumors using iontophoresis—this minimally-invasive technique utilizes a mild electrical current to drive therapeutic compounds into targeted tissues. This method enhances local drug concentration while minimizing systemic toxicity, a critical need in the treatment of pancreatic and other solid-tissue cancers.

Focal Medical’s lead product candidate, an iontophoresis device directed gemcitabine therapy for pancreatic cancer, has been cleared by the U.S. Food and Drug Administration (FDA) through an Investigational New Drug (IND) application. Phase 1b clinical trials are expected to begin later this year.

"This is a transformative milestone for Continuity Biosciences," said Ramakrishna Venugopalan, PhD, MBA, Chief Executive Officer of Continuity Biosciences. "Focal Medical’s approach to localized drug delivery aligns perfectly with our vision to improve bioavailability, reduce systemic exposure, and deliver better outcomes for patients. We are thrilled to bring this promising platform into our portfolio and into the clinic."

Through the acquisition, Continuity Biosciences obtains Focal Medical’s entire patent estate, iontophoresis technology platform and specialized equipment, scientific know-how, and licensing agreements with the University of North Carolina at Chapel Hill. In addition, key scientific staff and leadership from Focal will join Continuity, further enhancing its internal capabilities. A dedicated research and development facility in Cary, NC that supports ongoing platform and pipeline advancement is also part of the acquisition and will be expanded to support other delivery platforms.

Innovent and HUTCHMED Jointly Announce NDA Acceptance in China for Sintilimab Combination with Fruquintinib for the Treatment of Advanced Renal Cell Carcinoma

On June 4, 2025 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, and HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:HCM; HKEX:13), reported that the New Drug Application (NDA) for the combination of sintilimab and fruquintinib for the treatment of patients with locally advanced or metastatic renal cell carcinoma who have failed prior treatment with one tyrosine kinase inhibitor (TKI) has been accepted for review by the China National Medical Products Administration (NMPA) (Press release, Innovent Biologics, JUN 4, 2025, View Source [SID1234653723]).

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The NDA is supported by data from FRUSICA-2, a randomized, open-label, active-controlled registration study evaluating the efficacy and safety of fruquintinib in combination with sintilimab versus axitinib or everolimus monotherapy for the second-line treatment of advanced renal cell carcinoma. The study has met its primary endpoint of progression free survival (PFS), as assessed by blinded independent central review (BICR) according to RECIST 1.1 criteria. The combination also demonstrated improvements in secondary endpoints including objective response rate (ORR) and duration of response (DoR). The safety profile was tolerable and no new safety signals were observed. Data from FRUSICA-2 will be submitted for presentation at an upcoming scientific conference. Additional details may be found at clinicaltrials.gov, using identifier NCT05522231.

Dr Hui Zhou, Senior Vice President of Innovent, stated: "The second NDA acceptance of sintilimab and fruquintinib combination represents a significant step toward providing a more effective treatment option for patients with second line advanced renal cell carcinoma in China. Our PD-1 inhibitor, sintilimab (TYVYT), has solidified its position as a cornerstone of immuno-oncology (IO) therapy with this NDA as its 10th indication, marking a meaningful milestone in lifecycle management and clinical value optimization."

Dr Michael Shi, Head of R&D and Chief Medical Officer of HUTCHMED, stated: "Kidney cancer continues to pose significant challenges in China, with limited treatment options for patients who fail first-line therapies. Submitting this NDA for the fruquintinib and sintilimab combination for advanced renal cell carcinoma marks an important step in our efforts to address this unmet need. We are dedicated to making this combination therapy available to patients with renal cell carcinoma. At the same time, through ongoing research, we remain focused on exploring the full potential of this combination, as well as advancing our broader pipeline across multiple cancer types, to provide more patients with new and effective treatment options."

In December 2024, the combination of sintilimab and fruquintinib received conditional approval from the China NMPA for the treatment of patients with advanced mismatch repair proficient (pMMR) endometrial cancer who have failed prior systemic therapy and are not candidates for curative surgery or radiation, based on data from the FRUSICA-1 study (NCT03903705).

About Kidney Cancer and Renal Cell Carcinoma

It is estimated that approximately 435,000 new patients were diagnosed with kidney cancer worldwide in 2022.[i] In China, an estimated 74,000 new patients were diagnosed with kidney cancer in 2022.[ii] Approximately 90% of kidney tumors are RCC.

About Sintilimab

Sintilimab, marketed as TYVYT (sintilimab injection) in China, is a PD-1 immunoglobulin G4 monoclonal antibody co-developed and co-commercialized by Innovent and Eli Lilly and Company, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1 / PD-Ligand 1 (PD-L1) pathway, and reactivates T-cells to kill cancer cells.[iii]

In China, sintilimab has been approved and included in the updated NRDL for seven indications. The updated NRDL reimbursement scope for TYVYT (sintilimab injection) includes:

For the treatment of relapsed or refractory classic Hodgkin’s lymphoma after two lines or later of systemic chemotherapy;
For the first-line treatment of unresectable locally advanced or metastatic non-squamous non-small cell lung cancer lacking EGFR or ALK driver gene mutations;
For the treatment of patients with EGFR-mutated locally advanced or metastatic non-squamous non-small cell lung cancer who progressed after EGFR-TKI therapy;
For the first-line treatment of unresectable locally advanced or metastatic squamous non-small cell lung cancer;
For the first-line treatment of unresectable or metastatic hepatocellular carcinoma with no prior systematic treatment;
For the first-line treatment of unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma;
For the first-line treatment of unresectable locally advanced, recurrent or metastatic gastric or gastroesophageal junction adenocarcinoma.
Furthermore, sintilimab’s eighth indication, in combination with fruquintinib for the treatment of patients with advanced endometrial cancer with pMMR tumors that have failed prior systemic therapy and are not candidates for curative surgery or radiation, was conditional approved by the NMPA in December 2024.

Two NDAs for sintilimab are currently under the NMPA review, including:

In combination with ipilimumab as neoadjuvant treatment for resectable MSI-H/dMMR colon cancer is under the NMPA review and has been granted Priority Review designation;
In combination with fruquintinib for the treatment of patients with locally advanced or metastatic renal cell carcinoma who failed prior treatment with one TKI.
In addition, two clinical studies of sintilimab have met their primary endpoints:

Phase 2 clinical study of sintilimab monotherapy as second-line treatment of esophageal squamous cell carcinoma;
Phase 3 clinical study of sintilimab monotherapy as second-line treatment for squamous non-small cell lung cancer with disease progression following platinum-based chemotherapy.
About Fruquintinib

Fruquintinib is a selective oral inhibitor of all three vascular endothelial growth factor receptors (VEGFR) -1, -2 and -3. VEGFR inhibitors play a pivotal role in inhibiting tumor angiogenesis. Fruquintinib was designed to have enhanced selectivity that limits off-target kinase activity, allowing for drug exposure that achieves sustained target inhibition and flexibility for potential use as part of a combination therapy.[iv]

About Fruquintinib Approvals

Fruquintinib is co-developed and co-commercialized in China by HUTCHMED and Eli Lilly and Company under the brand name ELUNATE. It is approved for the treatment of patients with metastatic colorectal cancer who have previously received fluoropyrimidine, oxaliplatin and irinotecan-based chemotherapy, and those who have previously received or are not suitable to receive anti-VEGF therapy or anti-epidermal growth factor receptor (EGFR) therapy (RAS wild-type) in China. It was included in the China National Reimbursement Drug List (NRDL) in January 2020. Since its launch in China, over 100,000 patients with colorectal cancer have been treated with fruquintinib.

The combination of ELUNATE (fruquintinib) and TYVYT (sintilimab injection) has conditional approval in China for the treatment of patients with advanced pMMR endometrial cancer who have failed prior systemic therapy and are not candidates for curative surgery or radiation.

Takeda holds the exclusive worldwide license to further develop, commercialize, and manufacture fruquintinib outside mainland China, Hong Kong and Macau, marketing it under the brand name FRUZAQLA. Fruquintinib received approval for the treatment of previously treated metastatic colorectal cancer in the US, Europe, Japan and many other countries around the world.

The safety and efficacy of fruquintinib for the following investigational uses have not been established and there is no guarantee that it will receive health authority approval or become commercially available in any country for the uses being investigated:

About Fruquintinib for Second-line Treatment of RCC

Single-agent targeted therapy continues to be one of the primary choices for first-line treatment of advanced RCC in China. Notably, advanced RCC patients who have experienced failure with single-agent targeted therapy previously still indicate an unmet medical need.

Results from a proof-of-concept Phase Ib/II study of fruquintinib plus sintilimab were published in Targeted Oncology in January 2025. The combination demonstrated promising efficacy and a tolerable safety profile in this setting. At the data cutoff of October 9, 2024, all 20 enrolled previously treated patients were evaluable for efficacy, with a median follow-up duration of 45.7 months. The confirmed ORR was 60.0% and DCR was 85.0%. Median DoR was 13.9 months and median PFS was 15.9 months. Overall survival (OS) was not reached, and the 36-month OS rate was 58.3%.