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Halia Therapeutics is moving its lead asset into the second part of a mid-stage trial following initial
positive data in patients with an early form of blood cancer that affects the bone marrow.
The Utah-based biotech is investigating its oral NEK7/NLRP3 inflammasome inhibitor, dubbed
HT-6184, in patients with lower-risk myelodysplastic syndromes. Patients with MDS have abnormal
and underdeveloped blood cells, which can lead to anemia, a heightened risk of infections, and
progression to acute myeloid leukemia, according to the company.
In the first small cohort of 18 participants taking the inhibitor as a monotherapy, the drug
demonstrated hematological improvement in all of the patients’ erythroid response after 16 weeks of
treatment, meaning they saw an improvement in the number of healthy red blood cells. The
requirement to move HT-6184 into the second stage of the trial was at least three patients responding
to treatment.
The study is expected to finish in the middle of next year. The primary endpoints include transfusion
dependency and changes in hemoglobin levels.
“By targeting the underlying inflammatory signaling driving this condition, HT-6184 aims to transform
treatment outcomes and improve quality of life for patients who currently face limited options,” Halia
CEO David Bearss said in a statement.
The Phase 2 trial will enroll up to 40 patients in India, with enrollment to be completed by February.
The drug is also in another trial investigating its impact on inflammation and pain in patients following
a surgical procedure.
If Halia’s drug is approved, it will compete against Bristol Myers Squibb’s Reblozyl, which was
approved for the treatment of anemia in patients with the disorder last year, and Geron’s Rytelo, which
scored FDA approval in June for patients who are dependent on blood transfusions.