NAYA Biosciences Begins Phase 1/2a Trial for GPC3-targeted Bispecific Antibody in Liver Cancer

NAYA Biosciences, a company specializing in innovative treatments for oncology, autoimmune diseases, and fertility, has provided an update on its clinical trial plans. The company has received regulatory approval from the Israeli Ministry of Health and subsequent institutional review board clearance to begin enrolling patients in a clinical trial for its drug NY-303. This trial will take place in up to seven academic centers and will evaluate NY-303, a GPC3-targeting NK Engager bispecific antibody, as a monotherapy for hepatocellular carcinoma (HCC) in patients who have not responded to first-line immunotherapy.

Dr. Daniel Teper, President of NAYA Biosciences and CEO of NAYA Therapeutics, emphasized the significance of this milestone for the company. He highlighted their commitment to offering new treatment options for patients who do not respond to initial immunotherapy and expressed optimism that positive Phase I safety and preliminary efficacy data would expedite the clinical development of NY-303 for HCC and potentially other tumors.

The Phase 1 segment of the trial will involve dose-escalation, with patients continuing weekly treatments as long as their disease does not progress. Key endpoints of this phase include safety, pharmacokinetics, activity markers, preliminary clinical efficacy, and time-to-progression. Recruitment is expected to begin in early 2025 at prominent medical centers in Israel, including Hadassah Hospital, Sheba Medical Center, and Sourasky Medical Center.

The Phase 2a part of the trial is planned to expand to the United States and Europe, dependent on further regulatory approvals. This phase will evaluate NY-303 at two dose levels identified in Phase I, with major efficacy endpoints including objective response rate and progression-free survival.

Dr. Dan Chiche, Chief Medical Officer of NAYA Therapeutics, noted the high mortality rate associated with HCC and the urgent need for new therapies. He mentioned that less than 30% of patients treated with the current standard of care respond to immunotherapy, with mean progression-free survival being less than seven months. Dr. Chiche emphasized that NY-303’s dual targeting mechanism has the potential to overcome non-response to current immunotherapies.

Hepatocellular carcinoma is the most common form of primary liver cancer, often occurring in individuals with chronic liver conditions. It is a significant global health issue, with over 900,000 new cases annually, making it the sixth most common cancer worldwide. Early detection is challenging, and the overall survival rates are low. Recent advances in immunotherapy provide hope for HCC patients, but new treatments are still crucial for improving survival and quality of life.

NY-303 is an innovative therapeutic candidate that engages and activates natural killer (NK) cells using a bispecific antibody targeting GPC3 on liver cancer cells and NKp46 on NK cells. This mechanism allows NY-303 to convert the tumor environment from "cold" to "hot," making liver cancer cells more susceptible to immunotherapy. This approach is promising for patients with limited treatment options and poor survival rates, including those with other GPC3-expressing tumors.

Current liver cancer treatments include surgery, liver transplantation, targeted drugs, and immunotherapies like checkpoint inhibitors, but advanced-stage liver cancer still has a grim prognosis. NY-303 aims to harness the body's immune system to specifically target and destroy tumor cells, potentially offering a safer profile with fewer side effects compared to traditional therapies. This targeted approach could significantly improve outcomes for HCC patients, addressing the high recurrence rates and treatment challenges associated with advanced liver cancer. As a next-generation immunotherapy, NY-303 may transform treatment possibilities and provide new hope for those facing this challenging diagnosis.