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HUTCHMED Highlights Clinical Data to be Presented at the 2026 ASCO Annual Meeting

On May 21, 2026 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:HCM; HKEX:13) reported that new and updated data from several studies of compounds discovered by HUTCHMED will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting taking place from May 29 to June 2, 2026 in Chicago, USA.

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Results from the pivotal Phase II registration study of savolitinib in gastric cancer or gastroesophageal junction adenocarcinoma patients with MET amplification in China will be presented during a rapid oral session. The study met its primary endpoint of objective response rate ("ORR") per RECIST 1.1, as assessed by the Independent Review Committee ("IRC"). As of the data cut-off of October 8, 2025, the IRC-assessed ORR was 32.3% (95%CI: 21.2%, 45.1%), exceeding the pre-specified efficacy threshold. Secondary endpoints included the IRC-assessed disease control rate (DCR) of 63.1%, median time to response (TTR) of 1.4 months, median duration of response (DoR) of 9.7 (95%CI: 3.7, 18.5) months, and median progression-free survival (PFS) of 4.0 (95%CI: 2.6, 5.0) months, respectively. The data supported the New Drug Application (NDA) submission to the China National Medical Products Administration (NMPA), which was accepted and granted priority review in December 2025.

Additionally, further analyses of the fruquintinib’s FRESCO, FRESCO-2, FRUSICA-1 and FRUSICA-2 studies, as well as investigator-initiated studies of fruquintinib and surufatinib spanning across a diverse range of potential tumor indications will be presented.

Details of the presentations, including links to available abstracts, are as follows:

Abstract title Presenter / Lead Author Presentation details
SPONSORED STUDIES
A phase 2 pivotal study of savolitinib in patients with MET-amplified gastric cancer or gastroesophageal junction adenocarcinomas Zhi Peng, Beijing, China 4011
Rapid Oral Abstract Session: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Monday, June 1, 2026 1:15 PM CDT
Tumor shrinkage and depth of response with fruquintinib in patients with metastatic colorectal cancer: Results from FRESCO and FRESCO-2 Elena Elez, Barcelona, Spain 3555
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
Efficacy of fruquintinib plus sintilimab versus axitinib or everolimus by scores of IMDC risk factors and PD-L1 expression at baseline in previously treated advanced renal cell carcinoma: A subgroup analysis of FRUSICA-2 study Kaiwei Yang, Beijing, China 4531
Poster Session: Genitourinary Cancer
– Kidney and Bladder
Efficacy with fruquintinib plus sintilimab versus axitinib or everolimus in advanced renal cell carcinoma: A post-hoc analysis from FRUSICA-2 trial by baseline tumor burden Yuanyuan Qu, Shanghai, China 4533
Poster Session: Genitourinary Cancer
– Kidney and Bladder
Association of Palmar-plantar erythrodysesthesia syndrome (PPES), hypothyroidism and clinical outcome in previously treated endometrial cancer (EMC) with pMMR status: A subgroup analysis of FRUSICA-1 Xiaotian Han, Shanghai, China e17612
Publication Only: Gynecologic Cancer

INVESTIGATOR-INITIATED STUDIES
Efficacy and safety of fruquintinib combined with chemotherapy versus bevacizumab combined with chemotherapy as second-line treatment for metastatic colorectal cancer: A prospective, multicenter, randomized controlled trial Jianmin Xu, Shanghai, China LBA3563
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
CONCEPT (combination of cetuximab plus fruquintinib treatment ± immunotherapy): A multicenter, randomized, open-label phase II trial in first-line pMMR RAS/BRAF wild-type unresectable metastatic colorectal cancer Yue Liu, Hangzhou, China TPS3680
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
Fruquintinib in combination with tislelizumab vs trifluridine/tipiracil and bevacizumab in MSS mCRC without active liver metastases: The IKF-080/QUINTIS trial Joseph Tintelnot, Hamburg, Germany TPS3684
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
A phase 2 study of fruquintinib combined with sintilimab and chidamide in refractory MSS metastatic colorectal cancer: Preliminary efficacy and safety Chang Wang, Changchun, China 2631
Poster Session: Developmental Therapeutics – Immunotherapy
Fruquintinib plus FOLFIRI or mFOLFOX6 as second-line therapy for patients with RAS-mutant metastatic colorectal cancer (mCRC): A phase II, multicenter, open-label study Yun Xu, Shanghai, China 3528
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
A randomized phase II trial of fruquintinib plus capecitabine versus capecitabine alone as maintenance therapy following first-line chemotherapy in metastatic colorectal cancer (mCRC) Wenhua Li, Shanghai, China 3534
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
A phase II trial of fruquintinib combined with cadonilimab in refractory MSS/pMMR colorectal cancer with pulmonary metastases Mengzhou Guo, Shanghai, China 3552
Poster Session: Gastrointestinal Cancer – Colorectal and Anal
Biomarker-driven assessment of immunochemotherapy with or without fruquintinib as first-line treatment for advanced gastric/GEJ adenocarcinoma: Initial clinical results and subgroup analysis from the MGC-FLORA study Xiaodong Zhu, Shanghai, China 4063
Poster Session: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Phase II study of utidelone plus fruquintinib for the treatment of platinum-resistant recurrent ovarian cancer (FRUTD trial) Hao Wen, Shanghai, China 5579
Poster Session: Gynecologic Cancer
Fruquintinib alternating with bevacizumab plus capecitabine as maintenance therapy after first-line treatment in metastatic colorectal cancer (mCRC): A multicenter, open-label, phase II study Wangjun Liao, Guangzhou, China e15539
Publication Only: Gastrointestinal Cancer – Colorectal and Anal
Intermittent fruquintinib plus trifluridine/tipiracil in refractory metastatic colorectal cancer (mCRC): A single-center, single-arm phase II study Yifu He/Jiayu Niu, Hefei, China e15560
Publication Only: Gastrointestinal Cancer – Colorectal and Anal
Phase I study of liposomal irinotecan plus fruquintinib as third- or later-line therapy for metastatic colorectal cancer Qian Li, Nanning, China e15571
Publication Only: Gastrointestinal Cancer – Colorectal and Anal
Chidamide combined with serplulimab and regorafenib or fruquintinib as third-line therapy for advanced colorectal cancer (C-ooperate/SCOG-C001): A single-arm, exploratory, multicenter, phase 2 trial Wei Li, Suzhou, China e15583
Publication Only: Gastrointestinal Cancer – Colorectal and Anal
Real-world use of fruquintinib in refractory metastatic colorectal cancer in the United States Vasu Bansal, Kansas City, US e15713
Publication Only: Gastrointestinal Cancer – Colorectal and Anal
Fruquintinib in combination with sintilimab and CAPEOX as first-line treatment for advanced gastric/gastroesophageal junction adenocarcinoma: A single-arm, open-label, multicenter phase Ib/II study (FUNCTION) Beibei Chen, Zhengzhou, China e16033
Publication Only: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Fruquintinib in combination with camrelizumab, paclitaxel liposome, and nedaplatin as first-line treatment for advanced esophageal squamous cell carcinoma (ESCC): Updated results from a single-arm, phase II study Yanhong Gu, Nanjing, China e16070
Publication Only: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Updated results of surufatinib combined with gemcitabine and cisplatin and immune checkpoint inhibitor (ICI) for unresectable locally advanced or metastatic intrahepatic cholangiocarcinoma Xuetao Shi/Jingtao Zhong, Jinan, China 4136
Poster Session: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Surufatinib plus KN046 and chemotherapy as first-line treatment for advanced pancreatic ductal adenocarcinoma: Updated results and biomarker analysis from a phase 1b/2 trial Wenquan Wang, Shanghai, China 4198
Poster Session: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Surufatinib combined with toripalimab for the treatment of recurrent ovarian clear cell carcinoma: Update of a prospective single center, single-arm phase II clinical trial Huijuan Yang, Shanghai, China 5586
Poster Session: Gynecologic Cancer
Surufatinib for advanced or metastatic chemotherapy-refractory thymic epithelial tumor: A single-arm, single-center, phase II study Bei Xu, Shanghai, China 8119
Poster Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers
Surufatinib combined with anti-PD-1/PD-L1 antibody in the second line or monotherapy in third line treatment of advanced hepatocellular carcinoma: A single-arm, open-label, multi-center phase II study Fuxiang Zhou, Wuhan, China e16172
Publication Only: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Efficacy and safety of surufatinib combined with immune checkpoint inhibitors plus chemotherapy in patients with biliary tract cancers: A real-world study Shasha Fan, Changsha, China e16222
Publication Only: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary
Osimertinib plus savolitinib in osimertinib-resistant non-small-cell lung cancer with low level gene copy number MET: A multi-center, open-label, and phase 2 study Xiang Han, Qingdao, China e20079
Publication Only: Lung Cancer –
Non-Small Cell Local-Regional/
Small Cell/Other Thoracic Cancers

(Press release, Hutchison China MediTech, MAY 21, 2026, View Source [SID1234665995])

Junshi Biosciences Announces Approval of Toripalimab NDA for the 1st-line treatment of HER2 Expressing Urothelial Carcinoma

On May 21, 2026 Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies, reported that the new drug application (NDA) for toripalimab in combination with disitamab vedotin for patients with HER2-expressing (which is defined as achieving a score of 1+, 2+ or 3+ in HER2 immunohistochemistry test), locally advanced or metastatic urothelial carcinoma (UC) was approved by the National Medical Products Administration (NMPA). Disitimab vedotin is an antibody drug conjugate independently developed by RemeGen Co., Ltd. With this latest approval, toripalimab injection now has 13 approved indications in the Chinese Mainland.

UC is among the top ten most prevalent malignant tumors in the world, and in China, its incidence and mortality rates continue rising. According to the latest data from the National Cancer Center, in 2022, the number of new cases of UC in China reached 92,900, and the number of deaths exceeded 40,000. UC is a serious threat to the life and health of patients, and there are huge unmet clinical needs.

In 2021, toripalimab injection was approved for the second-line and above treatment of advanced UC, becoming the first immunotherapy drug approved for non-selective population-based indications of advanced UC in China.

The approval of this new indication is based on results from the RC48-C016 study (NCT05302284). The study is a multi-center, randomized, open-label and controlled phase 3 clinical trial which evaluated the efficacy and safety of toripalimab in combination with disitamab vedotin versus gemcitabine in combination with cisplatin/carboplatin in systemic-treatment-naive patients with HER2 (human epidermal growth factor receptor 2)-expressing (which is defined as HER2 IHC 1+, 2+ or 3+) locally advanced or metastatic UC. The study was conducted in 74 clinical centers across China with Professor Jun GUO from Beijing Cancer Hospital and Professor Aiping ZHOU from the Cancer Hospital of the Chinese Academy of Medical Sciences as the principal investigators.

In October 2025, the study results of RC48-C016 were published in The New England Journal of Medicine (NEJM), and shared in an oral presentation at the Presidential Symposium of the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) annual meeting (#LBA7).

The results were positive for both primary endpoints, progression free survival ("PFS", assessed by blinded independent review) and overall survival ("OS"). Compared with traditional chemotherapy, toripalimab in combination with disitamab vedotin for the first-line treatment of HER2-expressing advanced UC more than doubled the median PFS [13.1 months vs. 6.5 months, hazard ratio (HR)=0.36, 95%CI: 0.28-0.46; p<0.0001], as well as the median OS (31.5 months vs. 16.9 months, HR=0.54, 95%CI: 0.41-0.73; p<0.0001). The objective response rate (ORR) greatly increased (76.1% vs. 50.2%) and the medium duration of response (DoR) almost tripled in comparison (14.6 months vs. 5.6 months). The combination therapy group also demonstrated significant improvements in safety compared to the chemotherapy group.

Dr. Jianjun ZOU, General Manager and CEO of Junshi Biosciences, said, "The approval of toripalimab’s 13th indication is a huge milestone for us all, and sheds light on the importance of our open collaboration R&D strategy. In urologic oncology immunotherapy, toripalimab continues to be a driving force and this approval deepens its impact across the immunotherapy landscape. We are immensely proud to partner with RemeGen. Together, we were able to combine two locally-developed innovations to create a powerful synergistic treatment that significantly improves both PFS and OS. Moving forward, Junshi Biosciences will expand on our Immuno-Oncology (I-O) 2.0 strategy, pursuing the next generation of combination therapies and novel target drugs to fulfill our commitment to enduring innovation, ensuring China’s innovation benefits global patients."

About Toripalimab

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and to induce PD-1 receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells.

More than forty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Europe and Southeast Asia. Ongoing or completed pivotal clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types, including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney, and skin.

In the Chinese mainland, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI). Currently, there are twelve approved indications for toripalimab in the Chinese mainland:

unresectable or metastatic melanoma after failure of standard systemic therapy;
recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least two lines of prior systemic therapy;
locally advanced or metastatic urothelial carcinoma (UC) that failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy;
in combination with cisplatin and gemcitabine as the first-line treatment for patients with locally recurrent or metastatic NPC;
in combination with paclitaxel and cisplatin in first-line treatment of patients with unresectable locally advanced/recurrent or distant metastatic esophageal squamous cell carcinoma (ESCC);
in combination with pemetrexed and platinum as the first-line treatment in EGFR mutation-negative and ALK mutation-negative, unresectable, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC);
in combination with chemotherapy as perioperative treatment and subsequently with monotherapy as adjuvant therapy for the treatment of adult patients with resectable stage IIIA-IIIB NSCLC;
in combination with axitinib for the first-line treatment of patients with medium to high risk unresectable or metastatic renal cell carcinoma (RCC);
in combination with etoposide plus platinum for the first-line treatment of extensive-stage small cell lung cancer (ES-SCLC);
in combination with paclitaxel for injection (albumin-bound) for the first-line treatment of recurrent or metastatic triple-negative breast cancer (TNBC);
in combination with bevacizumab for the first-line treatment of unresectable or metastatic hepatocellular carcinoma (HCC) patients;
first-line treatment for unresectable or metastatic melanoma;
in combination with disitamab vedotin for the first-line treatment of HER2 expressing UC.

The first 12 indications have been included in the National Reimbursement Drug List (NRDL) (2025 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for the treatment of melanoma, RCC and TNBC. Toripalimab for the treatment of advanced NPC and ESCC was approved in Hong Kong SAR, China.

Internationally, toripalimab has been approved for marketing in more than 40 countries and regions including the United States, the European Union, India, the United Kingdom, Australia and Singapore, and is also under review for marketing in various countries and regions worldwide.

(Press release, Shanghai Junshi Bioscience, MAY 21, 2026, View Source [SID1234665994])

BlossomHill Therapeutics to Present Preliminary Results From Phase 1 Dose Escalation/Backfill Cohorts in Phase 1/2 SOLARA Trial of OMNI-EGFR Inhibitor, BH-30643, in Advanced EGFR-mutant NSCLC at the 2026 ASCO Annual Meeting

On May 21, 2026 BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to design and develop innovative small molecule medicines for the treatment of cancer, reported an upcoming presentation of the preliminary, Phase 1 dose escalation/backfill data from the company’s ongoing Phase 1/2 SOLARA clinical trial of BH-30643 in epidermal growth factor receptor (EGFR)-mutant non-small cell lung cancer (NSCLC). The data will be presented in an oral session at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Tuesday, June 2, 2026.

BH-30643 is a non-covalent, macrocyclic, brain active, mutant-selective OMNI-EGFR inhibitor designed to address a broad spectrum of EGFR mutations, including classical mutations, on-target resistance mutations such as C797S (with or without T790M), atypical mutations and exon 20 insertion mutations.

"BH-30643 represents a potentially important advancement in the evolution of targeted therapies for treating EGFR mutant NSCLC," said Xiuning Le, M.D., Ph.D., Associate Professor in Medical Oncology at the MD Anderson Cancer Center. "Using a novel macrocyclic approach, BH-30643 has the potential to impact multiple clinically relevant EGFR mutation classes while minimizing inhibition of wild-type EGFR. The response data we are presenting at ASCO (Free ASCO Whitepaper) are encouraging – in a heavily pretreated Phase 1 dose escalation/backfill population, in patients with brain metastases, and even in the difficult-to-treat C797S/T790M population. These findings suggest BH-30643’s potential to address a significant unmet need for patients, especially those whose disease has progressed on prior EGFR TKIs."

"These Phase 1 dose escalation/backfill data demonstrated encouraging clinical activity for BH-30643, with a favorable tolerability profile in the setting of high PK exposures, and with responses observed across a broad range of EGFR genotypes," said Geoff Oxnard, M.D., Chief Medical Officer of BlossomHill Therapeutics. "Importantly, we are seeing confirmed responses in patients harboring C797S resistance mutations, a population where current treatment options remain limited and outcomes are poor. We believe these findings support the potential for BH-30643 to address significant unmet need. Phase 1 dose expansion is ongoing to determine the recommended Phase 2 dose and support further clinical development, with an initial focus on patients with C797S-positive resistance."

Key presentation highlights to include:

Preliminary results (March 2, 2026, data cut, with efficacy follow-up through April 29, 2026) from the Phase 1 dose escalation/backfill in Phase 1/2 SOLARA trial in patients with heavily pretreated and heterogeneous EGFR-mutant NSCLC. The Phase 1 dose escalation/backfill portion enrolled 82 patients (median of 3 prior lines of therapy, 66% with history of brain metastases, and 71% ECOG=1) across dose cohorts ranging from 20 mg to 160 mg total daily dose of BH-30643.
High plasma exposures were observed that exceeded target EC90 at candidate doses.
BH-30643 demonstrated a favorable tolerability profile with primarily low-grade EGFR wildtype toxicity as well as asymptomatic Gilbert’s-like bilirubin elevation; Grade ≥2 EGFR-wildtype treatment related adverse events (TRAEs) were reported in 27% of patients. No clinically significant cardiac effects were seen.
Responses and prolonged stable disease were observed across diverse EGFR genotypes in a heavily pretreated Phase 1 cohort, suggesting effective targeting of on-target resistance.
32 patients with C797S mutations were response evaluable across escalation and expansion cohorts; favorable overall response rate (ORR, confirmed or unconfirmed and ongoing) were seen both without prior chemo (50%) and with prior chemo (39%).
Responses were observed with or without concurrent T790M and with or without prior history of brain metastases
Robust C797S and T790M ctDNA clearance was observed in the context of diverse EGFR driver mutations including exon 19 del or indel, L858R, G719X, and exon20ins

Presentation Details
Abstract Title: First-in-human trial of BH-30643, a novel macrocyclic, non-covalent, mutant-selective OMNI-EGFR inhibitor, in EGFR-mutant (EGFRm) NSCLC
Abstract Number: 3014
Session Type/Title: Rapid Oral Abstract Session – Developmental Therapeutics – Molecularly Targeted Agents and Tumor Biology
Date and Time: June 2, 2026, at 9:45 AM – 11:15 AM CDT / 10:45 AM-12:15 PM EDT

About BH-30643
BH-30643 is an investigational, novel, orally bioavailable, non-covalent, macrocyclic, mutant-selective, OMNI-EGFR inhibitor. BH-30643 was designed to overcome the limitations of currently approved EGFR inhibitors, which were discovered over a decade ago without the current, modern understanding of the structure and protein dynamics of mutant EGFRs. In preclinical studies, BH-30643 demonstrated potent inhibitory activity across diverse EGFR mutation categories – classical activating mutations, on-target resistance mutations such as C797S with or without T790M, atypical mutations and exon 20 insertions – while maintaining marked selectivity over wild-type EGFR.

BH-30643 is currently being evaluated in the Phase 1/2 SOLARA clinical trial in adults with locally advanced or metastatic NSCLC harboring EGFR or HER2 mutations. For additional information on SOLARA, including a list of study sites and how to enroll, please visit clinicaltrials.gov (NCT06706076).

(Press release, BlossomHill Therapeutics, MAY 21, 2026, View Source [SID1234665993])

HiberCell to Present Preliminary Results from the Phase 1b Trial of GCN2 Activator HC-7366 when Combined with WELIREG® (belzutifan) for the Treatment of Late-Line Clear Cell Renal Cell Carcinoma (ccRCC) at the Upcoming 2026 ASCO Annual Meeting

On May 21, 2026 HiberCell, Inc., a clinical-stage biotechnology company developing therapeutics targeting the integrated stress response (ISR) to address cancer relapse, metastasis, and resistance, reported that preliminary results from its ongoing Phase 1b study (NCT06234605) of HC-7366 in combination with WELIREG (belzutifan), Merck’s (known as MSD outside of the United States and Canada) oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, will be presented in two poster presentations at the upcoming 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The study is evaluating HC-7366, an activator of the ISR kinase GCN2, in patients with advanced clear cell renal cell carcinoma (ccRCC) whose disease progressed after prior PD-1/PD-L1 checkpoint inhibitor and VEGF-tyrosine kinase inhibitor (VEGF-TKI) therapy.

"These preliminary results from the Phase 1b study are encouraging," said Robert Motzer, M.D., lead principal investigator for the study. "In a patient population with limited treatment options following checkpoint inhibitor and VEGF-TKI therapy, the combination of HC-7366 and belzutifan demonstrated a manageable safety profile and early signals of disease control that warrant further investigation."

"The data from our dose escalation and expansion cohorts suggest that HC-7366 in combination with belzutifan is generally well tolerated, with preliminary efficacy findings that are consistent with our preclinical hypotheses," said Nandita Bose, Ph.D., Chief Development Officer of HiberCell. "While these are early results, the observed response rates and pharmacodynamic evidence of pathway engagement provide a reasonable basis for continuing evaluation in our Expansion 2 cohort."

Abstract 4534 – A phase 1b, open-label, safety, tolerability, and efficacy study of HC-7366 in combination with belzutifan in patients with locally advanced (inoperable) or metastatic renal cell carcinoma.

The study enrolled patients with advanced ccRCC previously treated with ≥1 anti-PD-1/PD-L1 and ≥1 VEGF-TKI. As of the later data cutoff date for the poster presentation, April 23, 2026, 69 patients received study treatment: 16 monotherapy and 53 as part of dose escalation/Expansion 1 of HC-7366 in combination with belzutifan (7 at 20 mg, 22 at 40 mg, 24 at 60 mg with 120 mg belzutifan). Expansion 2 in ~20 patients is fully enrolled and follow-up is ongoing.

The median number of prior therapies was 3 (range 1-5) in monotherapy and 2 (range 1-4) in combination. Most TEAEs were Grade 1-2. Grade 3 events were mainly hematological (anemia) and gastrointestinal (nausea and diarrhea), with one DLT (Grade 3 nausea, 40 mg combination).

In efficacy-evaluable patients, the 40 mg combination (median follow-up of 13.3 months) demonstrated a best overall response rate (BORR) of 37%, including a confirmed ORR (cORR) of 26%, disease control rate (DCR) of 89%, and primary progressive disease (PD) rate of 11%. The 60 mg combination (median follow-up of 10.9 months) had a BORR and cORR of 37%, DCR of 84%, and primary PD rate of 16%. In monotherapy, BORR of 15% and DCR of 62% were observed. Early efficacy signals at 40–60 mg align with the preclinical projected optimal efficacious dose range.

Overall, HC-7366, alone or in combination with belzutifan, was generally well tolerated. Preliminary efficacy analyses indicate favorable disease control, characterized by a high DCR and low primary PD for the combination.

Abstract 4535 – Combining HC-7366 with belzutifan in patients with renal cell carcinoma to alter tumor and microenvironment: Pharmacokinetic and pharmacodynamic (PK/PD) analysis of a phase 1b study.

Findings from a robust translational program, including pharmacokinetic and pharmacodynamic analyses from paired tumor biopsies and systemic biomarker assessments, demonstrated clear evidence of HC-7366 pathway engagement and a differential pharmacodynamic biomarker profile distinguishing monotherapy from the combination. Notably, several favorable changes countering ccRCC biology were observed, including sustained inhibition of the HIF-2 target EPO, reduction of HIF-1α, cell cycle inhibition, and modulation of pro- and anti-tumor adipokines. Modulation of angiogenic and immune-related pathways further supports the scientific rationale for evaluating HC-7366/belzutifan in combination with immune checkpoint inhibitors and/or VEGF-TKIs.

WELIREG is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About HC-7366

HC-7366 is a first-in-class, first-in-human, selective, potent, small molecule activator of the general control nonderepressible 2 (GCN2) kinase. GCN2 is one of the kinases of the integrated stress response (ISR) family, which responds to amino acid deprivation and is a key metabolic stress sensor in cells. While cancer cells utilize the ISR for survival, prolonged or hyperactivation of GCN2 with HC-7366 has been shown to have antitumor and immunomodulatory activity as a monotherapy and in combination with various standard-of-care agents in preclinical models of both solid and liquid tumors. HC-7366 is currently under clinical development in Phase 1b studies in ccRCC and acute myeloid leukemia (AML).

(Press release, HiberCell, MAY 21, 2026, View Source [SID1234665992])

Rgenta Therapeutics Presents Positive Preliminary Data from Ongoing Phase 1a/b Clinical Trial of RGT-61159 in Patients with Adenoid Cystic Carcinoma and Colorectal Cancer at the 2026 ASCO Meeting

On May 21, 2026 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported positive preliminary data from its ongoing Phase 1a/b clinical trial of RGT-61159, an oral small molecule targeting MYB, in patients with advanced, relapsed or refractory adenoid cystic carcinoma (ACC) or colorectal cancer (CRC) at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting being held this week in Chicago. The early data supports promising and durable anti-tumor activity in advanced ACC, demonstrated MYB target engagement and an attractive, well-tolerated safety profile at RP2Ds for once-daily oral administration of RGT-61159.

"While still early, these data are encouraging and supportive of the potential of RGT-61159 as a promising anti-tumor agent with a favorable safety profile in a particularly aggressive form of cancer, ACC," said Dr. Ho, M.D., Chief of Head and Neck Medical Oncology at Memorial Sloan Kettering Cancer Center. "Further, these data demonstrate that RGT-61159 knockdown of MYB protein, an oncogene that drives cancer progression, is a promising approach for treating cancers that over-express MYB. I look forward to the final results from this study and seeing the continued maturity of efficacy, safety and durability data."

Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta added, "These are the first clinical data generated for RGT-61159 and we are pleased with the early results showing an impressive disease control rate and anti-tumor activity in ACC that deepens over time. In addition, RGT-61159 has demonstrated a favorable, well-tolerated safety profile. We look forward to initially advancing RGT-61159 into further development in ACC, an indication with a high unmet need, and plan to expand into other MYB-driven cancers in the future."

Rgenta’s Phase 1a/b clinical trial of RGT-61159 is a multi-center, open-label dose escalation and expansion study in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is evaluating safety, tolerability, pharmacokinetics, target engagement and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).

Preliminary data from the initial patients in the trial showed clinically meaningful disease control of 84.6% in 39 evaluable patients, with 3 patients achieving partial response according to RECIST criteria (2 confirmed). Responses continued to deepen with longer treatment duration. Patients with partial responses have remained on study for a mean of 8.2 months, while all ongoing patients across the trial have a mean on-study duration of 7.3 months. RGT-61159 was well tolerated with the most common adverse events being fatigue, anemia, diarrhea and nausea.

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of oncogenic MYB protein production, which has the potential to inhibit proliferation or induce cell death of cancer cells that overexpress MYB protein. MYB acts as a master regulator of cell proliferation differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemias (AML), T-cell acute lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer.

About Adenoid Cystic Carcinoma (ACC)
It is estimated that approximately 200,000 people are living with ACC throughout the world including 11,000 in the US. While it is a rare cancer, ACC is the second most common cancer type arising in the salivary gland and is an aggressive malignancy with a tendency to infiltrate surrounding nerves and metastasize to distant sites. Overactivation of the MYB oncogene has been described as a hallmark of ACC and is noted in over 90% of ACC. Treatment for ACC is extremely challenging and may include surgery and/or radiation, which often fails to control local tumor recurrence and distant metastases. There are no effective targeted therapies available for patients with recurrent and/or metastatic disease. There is thus an unmet medical need for new therapeutic targets and treatment strategies for patients with this fatal cancer.

About Colorectal Cancer (CRC)
CRC is the third most prevalent cancer and the second leading cause of cancer-related mortality worldwide. According to the World Health Organization, in 2022, more than 1.9 million cases of CRC were diagnosed. Despite the improved early detection of CRC and the recent success of targeted therapeutics, approximately 15%-30% of patients present with metastases and 20%-50% of patients with initially localized disease will develop metastases. Patients with relapsed or refractory CRC who have exhausted all the available standard of care therapy options, have a very poor prognosis. MYB is significantly overexpressed in 80-85% of CRC and has been frequently found to be a predictive biomarker of tumor aggressiveness and poor prognosis. Developing novel therapies to treat patients with metastatic CRC remains a major unmet medical need.

(Press release, Rgenta Therapeutics, MAY 21, 2026, View Source [SID1234665991])