Halia Therapeutics advances NLRP3 inhibitor to Phase 2 final stage

Halia Therapeutics, a biotechnology company based in Utah, is advancing its leading therapeutic candidate, HT-6184, into the second phase of a mid-stage clinical trial. This decision follows promising initial data in patients suffering from lower-risk myelodysplastic syndromes (MDS), a bone marrow condition that disrupts normal blood cell development. The disorder leads to a range of issues such as anemia, increased susceptibility to infections, and potential progression to acute myeloid leukemia.

HT-6184 is an oral inhibitor targeting NEK7/NLRP3 inflammasomes, and it is being assessed for its effectiveness in this patient group. In the preliminary cohort of 18 participants, who received the drug as a standalone therapy, all patients exhibited a hematological response. Specifically, they experienced improvements in the erythroid response, which indicates an increase in healthy red blood cell production after 16 weeks of treatment. The trial's criteria for moving to the next stage required that at least three participants show a positive response to the therapy.

The second stage of the study is anticipated to conclude in the middle of next year. Key outcomes being monitored include transfusion dependence and changes in hemoglobin levels. Halia Therapeutics aims for HT-6184 to address the inflammatory processes that underlie MDS, potentially offering patients improved treatment outcomes and enhanced quality of life, an area where current therapeutic options are notably limited, according to CEO David Bearss.

The ongoing Phase 2 trial plans to enroll up to 40 patients in India, with the enrollment process expected to finish by February. Beyond MDS, HT-6184 is also being evaluated in another clinical trial to assess its efficacy in reducing inflammation and pain following surgical procedures.

Should HT-6184 receive approval, it will enter a competitive market, challenging Bristol Myers Squibb’s Reblozyl and Geron’s Rytelo. Reblozyl was approved in the previous year for anemia treatment in MDS patients, while Rytelo received FDA approval in June for patients who require regular blood transfusions.