On February 19, 2025, Chemomab Therapeutics issued a press release announcing plans for a Phase 3 clinical trial for the treatment of PSC. The full press release is available on the Chemomab website. The investigational drug being tested has been named nebokitug, previously studied in PSC in the SPRING Study as CM-101.
PSC Partners is encouraged by the news that a potential drug for PSC is moving forward into the final stages of the drug approval process (Phase 3 clinical trial).Â
Some highlighted excerpts from the press release:
The novel trial design does not require a liver biopsy.
The trial is a randomized placebo-controlled (2:1 active to placebo ratio). Patients in the drug treatment arm will receive 20 mg/kg of nebokitug administered intravenously every three weeks.
The primary endpoint* is the time-to-first clinical event[s] … associated with PSC disease progression,
including more common clinical events such as acute cholangitis, strictures requiring intervention or portal hypertension
as well as cholangiocarcinoma, transplant, or death. See the press release for the full list of specific events.
Enrolled patients will remain in the trial until they experience one of the primary endpoint clinical events. On average, participants are expected to remain in the trial for about two years.
Approximately 350 PSC patients will be enrolled in the trial, and the study population will be enriched** for patients with moderate and advanced disease.
The FDA has agreed that a single Phase 3 trial, if successful, would be sufficient to proceed to regulatory review and potential drug approval.
*Primary endpoint: The single most important outcome that researchers measure to determine whether a new treatment is effective.
**Enriched: The investigators intend to enroll a higher number of participants matching these criteria than may be representative of the overall population of people with PSC.
A Phase 3 trial is the final phase of clinical trials before the Food and Drug Administration (FDA) conducts a review to determine whether to approve the drug for market. An overview of the phases of clinical trials can be found here (source FDA).
PSC Partners will continue to update the community as more information becomes available regarding this study. Please check back for updates, including on the Registry’s Ongoing Clinical Trials page.
For more information regarding CM-101:
Previous news article: Chemomab Announces CM-101 Phase 2 SPRING Trial Results
More results from the open-label extension of the SPRING trial are expected in the first quarter of 2025.
Two scientific publications related to CM-101 are available in the Related Publications page of the Registry’s Clinical Trials section.