FDA Grants Regenerative Medicine Advanced Therapy Designation for OBX-115

The FDA has granted the regenerative medicine advanced therapy (RMAT) designation to OBX-115 (Obsidian Therapeutics, Inc.) to treat individuals with unresectable or metastatic melanoma that is resistant to immune checkpoint inhibitor (ICI) therapy. According to the release, 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.” The acceptance follows an FDA fast track designation that was granted in July of this year.¹

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According to the FDA, melanoma is a type of skin cancer that is caused through exposure to ultraviolet light like sunlight or indoor tanning. The disease results in a significant number of cancer-related deaths.²

“Melanoma is a life-threatening cancer that can cause devastating impacts to affected individuals, with a significant risk of metastasizing and spreading to other areas in the body,” said Nicole Verdun, MD, director of the Office of Therapeutic Products in CBER, in a news release.²

If not found in the early stages, melanoma can spread to other parts of the body and become metastatic. Once in the unresectable or metastatic form of the disease, individuals are often treated with immunotherapy using PD-1 inhibitors.²

“Unresectable or metastatic melanoma is an aggressive form of cancer that can be fatal,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER), in a news release.²

The study authors noted that OBX-115 could offer further treatment options for individuals with advanced or metastatic melanoma by enhancing persistence, antitumor activity, and clinical safety of TIL cell therapy.¹

“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, MD, Chief Development Officer of Obsidian, in a news release.¹

The RMAT designation was granted based off findings from an initial phase 1 open-label study that assessed the safety of efficacy of OBX-115, presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. The results displayed that OBX-115 demonstrated a favorable safety profile, comparative to what was reported from IL2-dependent non-engineered TIL cell therapy, according to study authors.¹

Obsidian Therapeutics provided that they are continuing research for OBX-115 in a phase 1/2, open-label study (NCT06060613).that is aimed to investigate the safety and efficacy of OBX-115 among individuals with advanced solid tumors. The study authors noted that the primary objective for the phase 1 study is to assess the safety and tolerability of OBX-115 regimen, as the phase 2 primary objective is to evaluate the efficacy of OBX-115 regimen, using objective response rate per Response Evaluation Criteria in Solid Tumors (RECIST).³

“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),” said Hari, in a news release.¹

REFERENCES

1. Obsidian Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for OBX-115 for the Treatment of Advanced Melanoma. Business Wire. News release. September 3, 2024. Accessed September 4, 2024. https://www.businesswire.com/news/home/20240903955214/en.

2. FDA Approves First Cellular Therapy to Treat Patients with Unresectable or Metastatic Melanoma. FDA. News release. February 16, 2024. Accessed September 4, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-unresectable-or-metastatic-melanoma.

3. Safety and Efficacy of OBX-115 in Advanced Solid Tumors. ClinicalTrials.gov. June 6, 2024. Accessed September 4, 2024. https://clinicaltrials.gov/study/NCT06060613.