Biotech Firm Inspirna Shuts Down After 10-Year Journey in microRNA Cancer Research

NoahAI News ·
Biotech Firm Inspirna Shuts Down After 10-Year Journey in microRNA Cancer Research

Inspirna, a New York-based biotech company focused on developing innovative cancer treatments, has announced its closure after a decade-long pursuit of microRNA-based therapies. The news comes as part of a broader trend of biotechnology firms facing financial challenges in the current economic climate.

Inspirna's Winding Down and Industry Impact

Usman "Oz" Azam, M.D., CEO of Inspirna, made the announcement via LinkedIn on March 31, 2025, expressing sadness over the company's demise. The closure affects an undisclosed number of employees, with Azam offering to provide a talent book to recruiters, highlighting the high-caliber professionals now seeking new opportunities in the life sciences sector.

Inspirna, which rebranded from RGENIX in 2021, had successfully raised over $140 million through various investment rounds, attracting notable investors such as Novo Holdings and Sofinnova Partners. The company's most recent funding, a $50 million Series D round in 2022, had seemed to position it for continued growth and development of its pipeline.

Scientific Foundation and Key Programs

Founded on research from Rockefeller University, Inspirna's approach centered on targeting crucial pathways in cancer progression using a proprietary microRNA-based target discovery platform called RNA-DRIVEr. The company's lead programs included:

  1. RGX-202 (ompenaclid): A CKB/SLC6A8 pathway-targeted therapy for gastrointestinal (GI) cancers, particularly those with RAS mutations.
  2. RGX-104: An LXR agonist aimed at treating solid tumors.

Ompenaclid showed promise in clinical trials, with data presented at the 2023 European Society for Medical Oncology annual meeting revealing a 37% objective response rate in 30 evaluable colorectal cancer patients. The drug also demonstrated a median progression-free survival of 10.2 months in a slightly larger cohort of 41 patients.

Recent Partnership and Uncertain Future

In a significant development last year, Merck KGaA entered into a $45 million upfront licensing agreement with Inspirna for the ex-U.S. rights to ompenaclid. This deal highlighted the potential of Inspirna's lead program and seemed to validate the company's approach to cancer treatment.

However, Azam's announcement did not provide details on the fate of ompenaclid or the status of the Merck KGaA partnership in light of Inspirna's closure. The future of these promising drug candidates and ongoing collaborations remains uncertain, raising questions about the potential impact on patients who might have benefited from these therapies.

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