NG-350A is a systemically delivered oncolytic immunotherapy driving intratumoral expression of a CD40 agonist monoclonal antibody

Phase 1b FORTRESS study of NG-350A in mismatch repair-proficient locally advanced rectal cancer patients is actively recruiting

Akamis Bio, a clinical-stage oncology company using a proprietary Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform to deliver novel immunotherapeutic payloads to solid tumors, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NG-350A for the treatment of mismatch repair-proficient (pMMR) locally advanced rectal cancer (LARC).

NG-350A is an intravenously delivered oncolytic immunotherapy designed to drive intratumoral expression of a CD40 agonist monoclonal antibody triggering the activation of antigen-presenting cells (APCs) resident in solid tumors and their draining lymph nodes. Once activated, APCs recruit T cells into the vicinity of the tumor to deliver a potent anti-tumor immune response. NG-350A is currently being evaluated in combination with chemoradiotherapy in the actively recruiting Phase 1b FORTRESS study (NCT06459869) in pMMR LARC patients.

“The NG-350A Fast Track designation from FDA is a recognition of the significant unmet need for new therapies to treat locally advanced rectal cancer (LARC),” said Oliver Rosen, M.D., chief medical officer at Akamis Bio. “The global incidence of LARC continues to rise, with a particularly alarming increase of this cancer among younger populations. Patients with mismatch repair-proficient tumors account for approximately 90% of LARC cases, and this population has the greatest need for evolution in the standard of care to include treatments that may enable patients to avoid surgical interventions.”

The FDA grants Fast Track designation to facilitate the development and expedite the review of new drugs that may fill an unmet medical need for serious or life-threatening conditions. A drug receiving the designation may be eligible for more frequent meetings and communications with the FDA to discuss development plans, ensure the collection of appropriate data needed to support approval, and enable a rolling review of an application for marketing authorization. This may lead to earlier drug approval and access for patients. Drugs receiving Fast Track designation may also be eligible for Accelerated Approval and Priority Review if relevant criteria are met.

About NG-350A

NG-350A is a clinical-stage, intravenously delivered T-SIGn® therapeutic designed to drive intratumoral expression of a CD40 agonist monoclonal antibody triggering the activation of antigen-presenting cells (APCs) resident in solid tumors and their draining lymph nodes. Once activated, APCs recruit T cells into the vicinity of the tumor to deliver a potent anti-tumor immune response. Akamis Bio has evaluated NG-350A’s safety, tolerability, and preliminary efficacy as a monotherapy (FORTITUDE study) and in combination with pembrolizumab (FORTIFY study) in patients with metastatic or advanced epithelial tumors. Across these studies, NG-350A has demonstrated a consistent safety and tolerability profile, as well as strong evidence of tumor-selective delivery, replication and transgene expression.

About the FORTRESS Study

The Phase 1b FORTRESS study (NCT06459869) is an open-label, single-arm, and multicenter trial of NG-350A in combination with chemoradiotherapy (CRT) in adult patients with mismatch repair-proficient (pMMR) locally advanced rectal cancer (LARC) and at least one risk factor for local or distant recurrence or with oligometastatic disease. The FORTRESS study builds upon the Akamis Bio-supported, CEDAR study, which showed a significantly greater complete response rate in LARC patients treated with a combination of Akamis Bio’s first generation oncolytic immunotherapy and chemoradiotherapy (CRT), relative to expected outcomes using standard-of-care CRT alone. The FORTRESS study is planning to enroll approximately 30 patients aged eighteen and older with histologically confirmed adenocarcinoma of the rectum which is locally advanced (clinical stage II-III based on pelvic MRI). During the 12-week active study treatment period, patients will receive NG-350A plus CRT (oral capecitabine plus long-course intensity-modulated radiotherapy). The primary endpoint for the study will be the proportion of patients achieving a clinical complete response (cCR) at week 12. Key secondary endpoints will include the incidence and severity of adverse events, clinical response (CR) outcome, and MRI-based tumor regression grade (mrTRG). Patients recently diagnosed with pMMR LARC interested in learning more about the FORTRESS trial can visit www.FortressStudy.org.

About LARC

Colorectal cancer is the third most common cancer diagnosed in both men and women in the United States with about 145,000 people newly diagnosed each year. Amongst the incident colorectal cancer population, about 45,000 people are diagnosed specifically with rectal cancer of which approximately 60 percent have locally advanced rectal cancer (LARC). LARC is defined by the spread of the rectal cancer to nearby tissues or lymph nodes. In patients with LARC, tumors have either grown through muscle and into the outermost layers of the rectum, or in more severe cases, through the wall of the rectum where they may attach to other organs or structures and/or into the lymph nodes. Approximately 90% of LARC patients have mismatch repair-proficient (pMMR) tumors which have a functional DNA repair system.

About Akamis Bio

Headquartered in Cambridge, Massachusetts, Akamis Bio is a clinical-stage oncology company developing systemically administered immunotherapies to treat solid tumors, initially in patients with locally advanced rectal cancer (LARC). Its proprietary Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform is designed to deliver novel immunotherapeutic proteins, biomolecules and transgene combinations to treat solid tumors, with its lead clinical-stage program, NG-350A, driving intratumoral expression of a CD40 agonist monoclonal antibody. To learn more, please visit www.akamisbio.com.

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