First Patient Receives HT-6184 in Phase 2a Trial for Low-Risk MDS

The first patient in a phase 2a clinical trial has received a dose of HT-6184, a treatment intended for patients with lower-risk myelodysplastic syndromes, according to an announcement from Halia Therapeutics, the drug’s manufacturer.

The novel drug, HT-6184, targets the NEK7 protein, part of the NLR3 inflammasome, which is a multiprotein complex that plays a key role in regulating the immune system and inflammatory signaling, as defined by a study published in the journal Frontiers in Aging Neuroscience.

Myelodysplastic syndromes (MDS) are conditions that may occur when the blood-forming cells become abnormal in the bone marrow. This occurrence results in lower levels of one or more types of blood cells, the American Cancer Society states.

According to a news release, the phase 2a trial aims to analyze the safety and effectiveness of HT-6184 in up to 40 patients with lower-risk MDS.

The primary endpoint (the main result measured at the end of a study to see if treatment worked) in the trial aims to measure the improvements in the blood cells based on transfusion dependency and any changes in the hemoglobin (the protein in red blood cells that exchanges oxygen and carbon dioxide throughout the body).

The news release stated the trial is expected to end around the fourth quarter of 2025.

"The dosing of the first patient in our phase 2 trial of HT-6184 marks a significant milestone for Halia in further evaluating the potential of targeting the NLRP3 inflammasome to treat a wide spectrum of immunological and inflammatory diseases," Dr. Margit M. Janát-Amsbury, chief medical officer of Halia Therapeutics, said in the news release.

READ MORE: Reblozyl is a ‘Better First-Line Therapy’ for Anemia in MDS Subset

The manufacturing company of the drug also established that their phase 1 trial demonstrated that HT-6184 was well-tolerated and safe in eligible patients.

During the phase 1 portion of the clinical trial, in which 64 patients were enrolled, and each cohort included eight patients: six patients received HT-6184 orally and two patients received a placebo orally per cohort. This trial’s primary endpoint was the safety and tolerability of HT-6184.

The secondary endpoint of the phase 1 trial was to determine how much of the dose was necessary to be effective enough for the respective patients.

"The recent positive results of our phase 1 trial investigating HT-6184 are extremely encouraging and highlight the functional activity of HT-6184 in being able to reduce inflammatory cytokines,” Janát-Amsbury said. “As we move into this next stage of clinical testing, we look forward to assessing the potential benefit of our inflammasome inhibitor for MDS patients, as well as for other patients who suffer from inflammation-related diseases."

According to the American Cancer Society, it is not entirely known how many people receive diagnoses for MDS in the United States each year. However, there have been estimates that approximately 10,000 people in the United States receive diagnoses each year, and new cases are expected to increase as the country’s population rises.

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