In short
Balinatunfib is an investigational oral small molecule that inhibits tumour necrosis factor alpha (TNFα), a protein involved in inflammation. It is being studied in ulcerative colitis and Crohn's disease, two forms of inflammatory bowel disease. Three Phase 2 trials are listed in the registries, including one completed trial with 568 participants in Crohn's disease and two authorised trials enrolling 408 and 371 participants.
Key points
- Balinatunfib is an oral small molecule TNFα inhibitor developed by Sanofi.
- It is being studied in ulcerative colitis and Crohn's disease.
- All three registered trials are Phase 2 studies.
- The largest trial enrolled 568 participants with Crohn's disease and is completed.
- One ongoing trial measures long-term safety and tolerability in both conditions.
- Balinatunfib has no marketing authorisation and is investigational.
What is balinatunfib?
Balinatunfib is an investigational oral small molecule that inhibits tumour necrosis factor alpha (TNFα), a cytokine that drives inflammation in autoimmune diseases. It is a chemically synthesised substance developed by Sanofi Aventis Recherche et Développement. The substance is classified in the therapeutic area of digestive system diseases in the registry records. Balinatunfib has no marketing authorisation in the product records and remains an investigational substance. Unlike biological TNFα inhibitors, which are given by injection or infusion, balinatunfib is designed as a small molecule that can be taken by mouth.
How does balinatunfib work?
TNFα is a key cytokine involved in inflammatory and autoimmune diseases, including inflammatory bowel disease. Biological TNFα inhibitors have transformed treatment, but they have drawbacks such as lack of blood–brain barrier penetration, the need for parenteral administration, and immunogenicity. Balinatunfib is a small molecule TNFα inhibitor intended to address some of these limitations by offering an oral route of administration. The published description identifies it as a clinical oral small molecule TNFα inhibitor.
Which conditions is balinatunfib studied for?
The registered trials investigate balinatunfib in two conditions:
- Ulcerative colitis — a chronic inflammatory bowel disease affecting the large intestine. One authorised Phase 2 trial is studying balinatunfib in 408 participants with moderate-to-severe ulcerative colitis.
- Crohn's disease — a chronic inflammatory bowel disease that can affect any part of the digestive tract. A completed Phase 2 trial enrolled 568 participants, and a long-term safety trial includes participants with Crohn's disease.
A supplementary source also lists psoriasis and rheumatoid arthritis as indications associated with balinatunfib in earlier research.
How is balinatunfib given?
Balinatunfib is given orally as a film-coated tablet. The registry records list the pharmaceutical form as film-coated tablet and the route of administration as oral use. The trial records do not report the specific dose strengths used in the Phase 2 studies.
What do the clinical trials show?
What are the designs of the trials?
Three Phase 2 trials are registered for balinatunfib. The largest is a completed trial in Crohn's disease with 568 participants, which assessed efficacy and dose response of different doses on endoscopic response at the end of induction treatment. A second authorised trial is studying 408 participants with moderate-to-severe ulcerative colitis, measuring clinical remission at Week 12 using the modified Mayo Score. A third authorised trial is investigating long-term safety, tolerability and efficacy in 371 participants with either Crohn's disease or ulcerative colitis.
What are the trials measuring?
The ulcerative colitis trial measures the proportion of participants achieving clinical remission at Week 12 by modified Mayo Score (mMS), a standard scale for assessing ulcerative colitis activity. The Crohn's disease trial measures the proportion of participants achieving endoscopic response, which means visible improvement of the intestinal lining during an endoscopy. The long-term trial counts treatment-emergent adverse events, serious adverse events and adverse events of special interest in both conditions.
Who can take part in these trials?
The ulcerative colitis trial includes participants with moderate-to-severe disease. The completed Crohn's disease trial also enrolled participants with moderate-to-severe Crohn's disease. The long-term safety trial is open to participants with Crohn's disease or ulcerative colitis who are continuing treatment. A separate Phase 1 study evaluated balinatunfib in healthy adult participants, focusing on cardiac repolarisation.
What side effects are known?
The published literature describes TNFα inhibitors as a class associated with inflammatory and autoimmune disease treatment, but the available sources do not provide a specific side effect profile for balinatunfib. The ongoing long-term safety trial is specifically designed to collect data on treatment-emergent adverse events, serious adverse events and adverse events of special interest in participants with Crohn's disease and ulcerative colitis. A completed Phase 1 study evaluated the effect of a single dose of balinatunfib on cardiac repolarisation in healthy adults, which is a standard safety assessment for new oral medicines.