Chemomab Secures FDA Alignment for Phase 3 Trial of Nebokitug in Primary Sclerosing Cholangitis
核心洞察
Chemomab Therapeutics (搜索) completed a successful FDA End-of-Phase 2 meeting following positive Phase 2 SPRING trial results for nebokitug in primary sclerosing cholangitis (搜索) (PSC (搜索)).
The FDA aligned with Chemomab on a streamlined pathway to potential regulatory approval based on a single Phase 3 trial with a primary composite endpoint measuring time-to-first clinical event.
Nebokitug, a first-in-class monoclonal antibody targeting CCL24 (搜索), demonstrated numerical improvements in inflammation and fibrosis biomarkers, particularly at the 20 mg/kg dose.
Chemomab Therapeutics (搜索) has achieved a significant regulatory milestone with the FDA's alignment on a clear pathway for nebokitug's potential approval in primary sclerosing cholangitis (搜索) (PSC (搜索)), following positive Phase 2 SPRING trial results that many experts consider the strongest data to date in this rare liver disease.
The clinical-stage biotechnology company announced the successful completion of its End-of-Phase 2 meeting with the U.S. Food and Drug Administration, resulting in agreement on the design of a Phase 3 registration study. The streamlined approach provides clarity on a path to full regulatory approval based on a single pivotal trial, with the primary endpoint measuring time-to-first clinical event encompassing multiple clinical events associated with disease progression.
"Our Phase 2 SPRING trial data, viewed by many experts as the strongest to date in PSC (搜索), set the stage for our positive FDA End-of-Phase 2 meeting, which resulted in alignment on a clear pathway to potential regulatory approval based on a single Phase 3 trial," said Adi Mor, PhD, co-founder and Chief Executive Officer of Chemomab.
Phase 2 SPRING Trial Results
The Phase 2 SPRING trial results, published in the American Journal of Gastroenterology in December 2025, demonstrated that nebokitug was generally safe and well tolerated. Patients treated with nebokitug showed numerical improvements in a range of biomarkers for inflammation and fibrosis, particularly at the 20 mg/kg dose and in the pre-specified subgroup of patients with moderate/advanced fibrosis.
Data from the SPRING trial open label extension, presented at the American Association for the Study of Liver Disease (AASLD) The Liver Meeting 2025, showed continued and consistent positive effects of nebokitug on key inflammatory and fibrotic biomarkers when administered to patients with PSC (搜索) for up to 48 weeks. All three poster presentations at AASLD were designated as "posters of distinction" by conference organizers.
Proteomic analyses of 3,000 circulating proteins in patient samples from the SPRING trial revealed that nebokitug-treated patients exhibited significant and dose-dependent changes in proteins playing key roles in fibrosis, immune cell recruitment and inflammation. These analyses highlighted the broad impact of nebokitug's ability to neutralize CCL24 (搜索), including reductions in a wide array of inflammatory and fibrotic biomarkers in treated patients.
Mechanism of Action and Regulatory Status
Nebokitug is a first-in-class dual activity monoclonal antibody that neutralizes CCL24 (搜索), a soluble protein identified as a key driver of disease processes in fibro-inflammatory diseases such as PSC (搜索). The drug targets the macrophage-related mechanism of action, with clinical data providing additional insights into how it may halt or slow disease progression and improve clinical outcomes.
The therapeutic has received FDA and EMA Orphan Drug designations and FDA Fast Track designation for the treatment of PSC (搜索). Chemomab has also secured two new patents covering the use of nebokitug for liver diseases, including primary sclerosing cholangitis (搜索), issued in China and Russia, expanding protections beyond existing patents in the U.S., Europe, Japan and other key territories.
Manufacturing and Development Progress
The FDA confirmed agreement on Chemomab's Chemistry, Manufacturing, and Controls (CMC) strategy and agreed that additional animal toxicology testing routinely required by FDA may be conducted in parallel with the nebokitug Phase 3 trial and submitted as part of the planned Biologics Licensing Application. This represents a favorable outcome that supports the timely advancement of the Phase 3 program.
The World Health Organization's International Nonproprietary Names program assigned the INN designation nebokitug to the company's lead product candidate CM-101 in February 2025.
Financial Position and Strategic Outlook
Chemomab reported cash, cash equivalents and short-term bank deposits of $10.4 million as of December 31, 2025, with a cash runway expected to extend through the end of the first quarter of 2027. Research and development expenses decreased to $5.8 million for the full year 2025, compared to $11.3 million in 2024, primarily due to completion of the Phase 2 PSC (搜索) trial.
Dr. Mor noted that discussions with potential strategic partners continue to advance as the company progresses activities required for timely initiation of the Phase 3 trial in PSC (搜索). The company is also evaluating additional therapeutic indications where nebokitug has generated robust preclinical efficacy signals, providing a strong mechanistic and translational rationale for future clinical development.
The company strengthened its leadership team with the appointment of David M. Weiner, MD, as Interim Chief Medical Officer and the promotion of Jack Lawler, who oversaw the successful Phase 2 SPRING trial, to Chief Development Officer.