1stOncology™: Cancer Intelligence Service – Page 145 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

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])

Ficerafusp Alfa Plus Pembrolizumab Demonstrated Differentiated Three-Year Overall Survival and Deep Responses Driven by TGF-β Inhibition in 1L R/M HPV-Negative HNSCC

On May 21, 2026 Bicara Therapeutics Inc. (Nasdaq: BCAX), a clinical-stage biopharmaceutical company committed to bringing transformative bifunctional therapies to patients with solid tumors, reported extended follow-up data out to three years from the Phase 1/1b study of ficerafusp alfa in combination with pembrolizumab in first-line (1L) recurrent/metastatic (R/M) human papillomavirus (HPV)-negative head and neck squamous cell carcinoma (HNSCC). The data, which included the 750mg weekly (QW), 1500mg QW, and 2000mg every-other-week (Q2W) expansion cohorts, demonstrated deep, durable responses observed to be driven by TGF-β inhibition. The data will be presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL.

Ficerafusp alfa is a bifunctional epidermal growth factor receptor (EGFR)-directed monoclonal antibody bound to a human transforming growth factor beta (TGF-β) ligand trap designed to enable increased tumor penetration to drive deep and durable responses and potentially improve survival outcomes. Bicara is currently evaluating ficerafusp alfa at 1500mg QW in combination with pembrolizumab in the Phase 3 portion of the ongoing FORTIFI-HN01 pivotal study. Additionally, the company plans to initiate a study evaluating a 12-week loading dose followed by a 2250mg every-three-weeks maintenance dose regimen in the third quarter of 2026 to support long-term administration.

"Ficerafusp alfa continued to deliver deep, durable responses with up to three years of follow-up in 1L R/M HPV-negative HNSCC, reinforcing its best-in-class potential in this setting. At our pivotal study dose of 1500mg QW, an estimated one in three patients was alive at three years – approximately doubling the survival rate observed in retrospective analysis with standard of care pembrolizumab in HPV-negative patients," said Bill Schelman, M.D., Ph.D., Chief Medical Officer of Bicara Therapeutics. "TGF-β inhibition was observed to be the mechanistic foundation of this clinical benefit: driving tumor penetration, enabling immune cell infiltration, and translating depth of response into durable, long-term survival – a clinical profile no other EGFR-directed therapy in head and neck cancer has demonstrated."

Up to three years of follow-up reinforces ficerafusp alfa’s best-in-class potential in 1L R/M HPV-negative HNSCC

Across all dose cohorts of ficerafusp alfa in combination with pembrolizumab, the data demonstrated deep, durable responses and a generally well-tolerated safety profile. All three dose cohorts also demonstrated clinically meaningful duration of response (DOR), progression-free survival (PFS) and overall survival (OS), representing substantial improvements over standard of care treatment.

Notably, complete response (CR) rates have continued to mature across all three cohorts since prior data presentation – increasing to 13% at 750mg QW, 25% at 1500mg QW, and 30% at 2000mg Q2W as of the March 31, 2026 data snapshot.

Additionally, deep responses of at least 80% tumor shrinkage were observed at doses of ficerafusp alfa that resulted in greater TGF-β inhibition and tumor penetration, with more than three-fourths of responders in the 1500mg QW and 2000mg Q2W cohorts achieving deep responses.

Three-year follow-up from the 1500mg QW dose cohort, as of the March 31, 2026 data snapshot, showed an estimated OS rate of 31%, approximately doubling the survival rate observed in retrospective analysis with standard of care pembrolizumab in HPV-negative patients.1

Table 1. Key Efficacy Results Across Ficerafusp Alfa Dose Cohorts in Combination with Pembrolizumab in 1L R/M HPV-Negative HNSCC

750mg QW
(n=30) 1500mg QW
(n=28) 2000mg Q2W
(n=27)
Confirmed overall response rate 57% 54% 48%
CR rate 13% 25% 30%
Deep (≥80%) responses 47% 80% 77%
Median time to response 1.6 months 1.4 months 1.6 months
Median DOR NR (>16.6 months) 21.7 months NR (>12.8 months)
Median PFS 6.9 months 9.9 months 12.7 months
Median OS NM (>19.4 months) 21.3 months NM (>12.7 months; >23.6 months in patients with > 2-year follow-up)*
Data snapshot as of March 31, 2026. NR = not reached; NM = not mature
*Data maturation reflects a bimodal enrollment distribution: in the initial 15 efficacy evaluable patients (median follow-up: 27 months) median OS is not mature but has surpassed 23.6 months. The remaining 12 efficacy evaluable patients had a median follow-up of 11.7 months.

TGF-β inhibition drove depth and durability of response

Biomarker analyses across all three dose cohorts demonstrated sustained TGF-β inhibition and immune activation with ficerafusp alfa, reinforcing the mechanistic link between intra-tumoral TGF-β inhibition, immune activation, and the deep, durable responses.

Deep responses translated to improved durability and long-term outcomes for patients

Ficerafusp alfa’s depth of response, as demonstrated by CR rates and proportion of deep responders, has been well-established and is clinically differentiating. The updated data further reinforced depth of response as a driver of long-term outcomes in patients with 1L R/M HPV-negative HNSCC. Across a pooled cohort analysis, two-thirds of responders achieved deep responses of greater than 80% tumor shrinkage and experienced more durable disease control, with meaningfully longer DOR, PFS, and OS compared to patients with partial responses of less than 80% tumor shrinkage.

Across the pooled analysis comparing deep responders to partial responders of less than 80% tumor shrinkage, deep responders demonstrated:

A median DOR of 31.6 months;
A median PFS of 36.9 months, with a 65% reduction in the risk of disease progression or death; and
A median OS that has not been reached, with a 63% reduction in the risk of death.
This updated dataset provides compelling evidence for depth of response as a clinical surrogate for differentiated long-term outcomes with ficerafusp alfa treatment.

ASCO 2026 Poster Presentations
Annual Meeting | Chicago, IL | May 30 – June 3, 2026

Poster 1 (Wong et al.): Sustained Depth and Durability of Response with TGF-β Trapping in 1L R/M HPV-Negative HNSCC
Sustained depth and durability of response with TGF-β trapping in recurrent or metastatic (R/M) HPV-negative head and neck squamous cell carcinoma (HNSCC): Long-term results from two expansion cohorts of a phase 1/1b study of ficerafusp alfa plus pembrolizumab
Authors: Wong DJ, et al. | Abstract #6040 | Poster Board: 497 | May 30, 2026, 1:30-4:30 p.m. CDT

Poster 2 (Kaczmar et al.): Impact of Depth of Response on Long-Term Clinical Outcomes
The impact of depth of response on long-term clinical outcomes: Exploratory analyses from multiple expansion cohorts of a phase 1/1b study of ficerafusp alfa plus pembrolizumab in first-line recurrent/metastatic (R/M) HPV-negative head and neck squamous cell carcinoma (HNSCC)
Authors: Kaczmar J, et al. | Abstract #6058| Poster Board: 515 | May 30, 2026, 1:30-4:30 p.m. CDT

Poster 3 (Ferrarotto et al.): FORTIFI-HN01 Trial in Progress
A multicenter, randomized, double-blind, phase 2/3 study of ficerafusp alfa (BCA101) or placebo in combination with pembrolizumab for first-line treatment of PD-L1-positive, recurrent or metastatic head and neck squamous cell carcinoma: FORTIFI-HN01
Authors: Ferrarotto R, et al. | Abstract #TPS6129| Poster Board: 584A | May 30, 2026, 1:30-4:30 p.m. CDT

1. Based on a retrospective analysis of Supplementary Figure 1C, Vasiliadou, Ifigenia, et al. International Journal of Cancer 155.5 (2024): 883-893. No head-to-head studies have been conducted, and cross-trial comparisons differences in molecule composition, trial design, and patient population and characteristics.

Conference Call Information
Bicara will host a live conference call and webcast on Friday, May 22, 2026 at 8:30 a.m. ET. Individuals may register for the conference call by clicking the link here. Once registered, participants will receive dial-in details and a unique PIN that will allow them to access the call. An audio webcast will be accessible through the Investor Relations section of Bicara’s website under Events and Presentations. An archived replay will also be available for 30 days following the event.

About Head and Neck Squamous Cell Carcinoma
Head and neck squamous cell carcinomas (HNSCCs) develop from the mucosal epithelium in the oral cavity, pharynx and larynx and are the most common malignancies that arise in the head and neck. HNSCC is one of the most common cancers in the United States and globally with a rising incidence anticipated to reach one million new global cases annually by 2030. Ten percent of HNSCC patients are diagnosed with metastatic disease and up to 30% develop a recurrence or metastases over time after receiving initial treatment for advanced HNSCC. Most cases of HNSCC are thought to result from accumulated mutations caused by carcinogenic exposures such as tobacco smoke or HPV infection. Approximately 80% of patients with R/M HNSCC are HPV-negative. These HPV-negative tumors often exhibit a recurrence pattern that is primarily local and are associated with severe morbidities, including fatal tumor bleeding, intense pain, difficulty swallowing, significant weight loss, and cachexia. This highlights a critical unmet need for therapies that have the potential to deliver durable anti-tumor responses, ultimately leading to meaningful improvements in patients’ quality of life.

About Ficerafusp Alfa
Ficerafusp alfa is a first-in-class bifunctional antibody designed to drive tumor penetration by breaking barriers in the tumor microenvironment that have challenged the treatment of multiple solid tumor cancers. Specifically, ficerafusp alfa combines two clinically validated targets: an epidermal growth factor receptor (EGFR) directed monoclonal antibody with a domain that binds to human transforming growth factor beta (TGF-β). Through this targeted mechanism, ficerafusp alfa reverses the fibrotic and immune-excluded tumor microenvironment driven by TGF-β signaling to enable tumor penetration that drives deep and durable responses. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ficerafusp alfa in combination with pembrolizumab for the first line (1L) treatment of patients with metastatic or with unresectable, recurrent (R/M) head and neck squamous cell carcinoma (HNSCC) whose tumors express programmed death-ligand 1 with combined positive score (CPS) ≥1, excluding human papillomavirus (HPV)-positive oropharyngeal squamous cell carcinoma. Ficerafusp alfa is currently being evaluated in FORTIFI-HN01, a pivotal Phase 2/3 clinical trial in patients with 1L R/M HNSCC.

(Press release, Bicara Therapeutics, MAY 21, 2026, View Source [SID1234665990])