The Phase II trial of HT-6184 will enroll around 50 patients with MDS who are or are on the borderline of being transfusion-dependent.

Halia Therapeutics is looking to start a Phase II trial of its pipeline candidate HT-6184 in myelodysplastic syndrome (MDS) early next year.

CEO Dr Dave Bearss told the Clinical Trials Arena that the US-based company will be testing the candidate in MDS patients for the first time, with the first dosing due to take place in the US.

HT-6184 targets the protein NEK7 by blocking its ability to bind to NLRP3, which reduces the inflammatory response. The activation of NLRP3 drives the onset and progression of various conditions, including neurological and rheumatological diseases.

Phase II trial plans

The Phase II trial will measure endpoints including the level of creation of white and red blood cells. Patients will also be evaluated with a bone marrow biopsy.

The trial will enrol around 50 patients with MDS who are or are on the borderline of being transfusion-dependent.

Investigators will be looking for improvement in haemoglobin and whether there is improvement in transfusion dependency, with Bearss adding that the goal would be for patients to no longer be dependent, which he described as a meaningful endpoint. The planned trial is taking place internationally with screening for eligible patients ongoing. Site selection and initiation will take place in the US before the company expands to multiple countries, with plans for parts of Asia and Europe.