FDA Grants Orphan Drug Designation to DPTX3186 Therapy in Gastric Cancer

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to DPTX3186, an investigational therapy being developed by Dewpoint Therapeutics for gastric cancer, according to a news release from the company.

This marks the first time an orphan designation has been granted to a condensate-modulating therapeutic, representing a notable milestone in the field of condensate biology. The designation followed the opening of an investigational new drug application for DPTX3186, the news release continued.

The orphan drug designation program supports the development of medicines intended for rare diseases that affect fewer than approximately 200,000 individuals in the United States. Benefits of this designation include eligibility for seven years of market exclusivity upon FDA approval, tax credits for qualified clinical trial costs, FDA fee waivers, and the potential for expedited regulatory review. These incentives are intended to help accelerate the availability of new treatment options for patient communities facing limited therapeutic choices, Dewpoint Therapeutics explained in the release.

“The FDA’s decision to grant orphan drug designation to DPTX3186 is an extraordinary validation of both the promise of condensate biology and the importance of our work in gastric cancer,” said Dr. Isaac Klein, chief scientific officer and head of R&D at Dewpoint Therapeutics. “This recognition reflects the agency’s confidence in our mechanism, our science and our shared goal of bringing new hope to patients facing this devastating disease.”

FDA Recognizes DPTX3186, A First-in-Class Agent, for Gastric Cancer

DPTX3186 is described as a first-in-class, oral small-molecule condensate modulator designed to selectively target oncogenic Wnt/β-catenin signaling in tumor cells. Wnt/β-catenin signaling is known to play a role in cancer cell growth and survival, and previous attempts to inhibit this pathway have faced substantial safety challenges, investigators went on to explain in an earlier press release detailing the use of the agent. DPTX3186 is engineered to redistribute β-catenin into an inactive condensate state within tumor cells. This mechanism aims to reduce cancer-promoting activity in a manner intended to avoid the toxicities associated with earlier efforts to block this pathway.

The development of DPTX3186 is supported by a biomarker strategy designed to measure pharmacodynamic effects directly in patient samples, offering the potential for clear, early biological readouts during clinical testing. Dewpoint’s research team described this mechanism as a differentiated and innovative approach in oncology drug development.

Clinical Trial Plans and Collaboration

Dewpoint expects to begin dosing the first patient in a combined phase 1/2 clinical trial before the end of 2025 at leading cancer centers in the United States. The initial trial plans to evaluate safety, pharmacokinetics and preliminary efficacy of DPTX3186 as a single agent in patients with metastatic gastric cancer. Planned expansion cohorts are intended to include combination therapy regimens and additional tumor types driven by Wnt/β-catenin signaling.

“Dosing our first patient this year will be a defining moment for condensate biology and Dewpoint,” said Klein explained in the second news release. “DPTX3186 is the only molecule of its kind: an oral small-molecule c-mod tackling a historically undruggable pathway with a differentiated safety profile. Its discovery on our platform, the bespoke biomarker strategy, and the caliber of partners supporting this trial reflect the transformative potential of condensate science to rewrite what is possible in drug discovery and development.”

The progress of the DPTX3186 program has been supported through a collaboration with Evotec, a drug discovery and development company that contributed preclinical, regulatory and chemistry manufacturing support.

Dr. Cord Dohrmann, chief scientific officer of Evotec, stated: “Evotec is proud to support Dewpoint in translating an entirely new modality from biology to clinic. The DPTX3186 program exemplifies how next-generation science and disciplined execution must go hand in hand. Dewpoint’s advanced expertise in condensate biology and Evotec’s integrated development platform capabilities have together enabled a smoother, more de-risked path to investigational new drug. This project reflects the kind of collaborative, high-science partnerships that will define the future of innovative drug development.”

DPTX3186 is currently the only oral condensate modulator targeting this pathway in clinical development, the news release concluded.

References

1. “FDA Grants Orphan Drug Designation to Dewpoint Therapeutics’ DPTX3186 for the Treatment of Gastric Cancer,” by Dewpoint Therapeutics. News release; Oct. 29, 2025.
2. “Dewpoint Therapeutics Announces an Open IND for First-in-Class Condensate Modulator DPTX3186 for Wnt-Driven Cancers,” by Dewpoint Therapeutics. News release; Oct. 21, 2025.

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