On September 3, 2024 Obsidian Therapeutics, Inc., a clinical-stage biotechnology company pioneering engineered cell and gene therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OBX-115 for the treatment of patients with unresectable or metastatic melanoma that is resistant to immune checkpoint inhibitor (ICI) therapy (Press release, Obsidian Therapeutics, SEP 3, 2024, View Source [SID1234646331]). OBX-115 is a novel engineered tumor-derived autologous T cell immunotherapy (tumor-infiltrating lymphocyte [TIL] cell therapy) armored with membrane-bound IL15 (mbIL15) that is pharmacologically regulated using FDA-approved small molecule drug acetazolamide. This second FDA designation follows a July 2024 announcement of Fast Track designation for OBX-115.

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"RMAT designation for OBX-115 highlights the unmet need in ICI-resistant melanoma and OBX-115’s potential to provide patient-centric, transformative care in this setting," said Parameswaran Hari, M.D., Chief Development Officer of Obsidian. "In an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, initial Phase 1 data showed that OBX-115 exhibited a favorable safety profile relative to what has been observed historically from IL2-dependent non-engineered TIL cell therapy, encouraging efficacy in patients with heavily pre-treated disease, and compatibility with non-surgical core needle biopsy tumor tissue procurement. We look forward to continued collaborative interaction with the FDA as we advance OBX-115 clinical development in both melanoma and non-small cell lung cancer (NSCLC)."

Established under the 21st Century Cures Act, RMAT designation is a dedicated program that includes the benefits of Fast Track and Breakthrough Therapy designations and is designed to expedite the development and review processes for certain promising drug candidates, including cell therapies. An investigational cell therapy is eligible for RMAT designation if it meets the definition of regenerative medicine therapy, is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. RMAT designation provides early and frequent interactions with the FDA, including intensive guidance on efficient drug development. Candidates receiving RMAT designation may also be eligible for Accelerated Approval and Priority Review.

About OBX-115

Obsidian’s lead investigational cytoTIL15 program, OBX-115, is a novel engineered tumor-derived autologous T cell immunotherapy (tumor-infiltrating lymphocyte [TIL] cell therapy) armored with pharmacologically regulatable membrane-bound IL15 (mbIL15). OBX-115 has the potential to become a meaningful therapeutic option for patients with advanced or metastatic melanoma and other solid tumors by leveraging the expected benefits of mbIL15 and Obsidian’s proprietary, differentiated manufacturing process to enhance persistence, antitumor activity, and clinical safety of TIL cell therapy. Obsidian is investigating OBX-115 in a multicenter trial in patients with advanced melanoma or relapsed / refractory NSCLC (NCT06060613).