Crinetics Pharmaceuticals has randomized its first patient into the CAREFNDR trial, a 141‑patient, multicenter, double‑blind, placebo‑controlled study evaluating once‑daily oral paltusotine 80 mg in adults with carcinoid syndrome caused by well‑differentiated neuroendocrine tumors.
The trial employs a 2:1 randomization to paltusotine versus placebo and will assess flushing episodes per day at 12 weeks as the primary endpoint, with bowel movement frequency as a key secondary endpoint. A 104‑week open‑label extension will evaluate long‑term efficacy, safety, and tumor control, positioning paltusotine as a potential oral alternative to the current monthly injectable therapies.
Crinetics’ Q3 2025 financial results provide context for the trial launch. The company reported a net loss of $130.1 million, up from $76.8 million in Q3 2024, while revenue fell to $0.143 million from $0.300 million. Research and development expenses rose to $90.5 million from $61.9 million, reflecting the additional investment required to advance the CAREFNDR trial and expand the preclinical portfolio. General and administrative costs increased as the company added personnel and expanded manufacturing activities to support the new program.
Chief Medical and Development Officer Dr. Dana Pizzuti highlighted the unmet need for a convenient, oral therapy for carcinoid syndrome: "Patients with carcinoid syndrome need a treatment option that can consistently manage symptoms without the burden of painful monthly injections. Based on our encouraging Phase 2 results and data from our open‑label extension, we believe paltusotine has the potential to transform the treatment landscape with a once‑daily, oral therapy that may offer meaningful benefits for patients managing the challenging day‑to‑day impacts of carcinoid syndrome."
Investors responded positively to the announcement, reflecting confidence in Crinetics’ strong cash position—over $1.1 billion as of September 30 2025—and the company’s future growth prospects from the PALSONIFY launch for acromegaly and the CAREFNDR trial.
The CAREFNDR milestone underscores Crinetics’ strategy to diversify its revenue base beyond acromegaly. Successful completion of the trial could open a new market for paltusotine, providing a more convenient treatment option and potentially generating a significant new revenue stream. With a projected cash runway into 2029, the company is positioned to sustain its pipeline development while pursuing commercialization opportunities.
Overall, the randomization of the first patient marks a critical step in Crinetics’ pursuit of expanding paltusotine’s therapeutic reach and demonstrates the company’s commitment to advancing its pipeline while maintaining financial resilience.
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