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Halia Therapeutics Advances HT-6184 in Phase 2 Trials

Halia Therapeutics, a leading biopharmaceutical firm focused on innovative treatments for chronic inflammatory conditions, has made significant strides with its promising drug candidate, HT-6184. Recently, the company reported favorable topline data emanating from the ongoing Phase 2 clinical trial targeting patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS). This announcement comes as Halia moves into the second stage of the trial, focusing on improving treatment options for this patient population.

Significant Findings from Initial Phase

The initial cohort of 18 patients treated with HT-6184 demonstrated a notable hematological improvement-erythroid (HI-E) response following 16 weeks of monotherapy. Impressively, the treatment exceeded the threshold requirement, documenting at least three responders among the patients, thus marking a critical milestone in the trial's progression. With such validation, Halia plans to expand this trial stage by enrolling an additional 8 to 10 patients to further substantiate HT-6184's therapeutic potential.

Dr. Alan List, a member of Halia's Scientific Advisory Board, emphasized the relevance of the NLRP3 inflammasome and myddosome pathways in driving ineffective hematopoiesis within MDS: "The high frequency of erythroid response following treatment with HT-6184 validates the key importance of the NLRP3 inflammasome… offering the prospect of a safe and effective oral therapeutic for LR-MDS patients."

Addressing Unmet Medical Needs

Dr. David J. Bearss, President and CEO of Halia Therapeutics, commented on the trial's significance in addressing gaps in the current treatment landscape for LR-MDS patients. "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," he stated.

Myelodysplastic syndromes represent a critical area of medical need characterized by dysfunctional bone marrow, which leads to insufficient production of healthy blood cells. Patients often deal with complications including anemia, elevated infection risk, and potential progression to acute myeloid leukemia. Current therapies offer limited effectiveness, underscoring the urgent demand for innovative solutions that can enhance patient outcomes and quality of life.

The Innovative Mechanism of HT-6184

HT-6184 operates through an allosteric mechanism, specifically targeting the NEK7 protein, which plays a pivotal role in the functioning of the NLRP3 inflammasome. By disrupting inflammasome signaling, HT-6184 holds the potential to reduce the inflammatory responses that are detrimental to effective hematopoiesis. This addresses a central complication faced by individuals with MDS. Halia's preclinical models revealed that inhibiting NEK7 not only prevents the formation of the NLRP3 inflammasome but also encourages its disassembly when activated.

The Phase 2 trial employs a Simon’s minimax two-stage design to rigorously assess the safety and efficacy of HT-6184 across multiple clinical sites in India, targeting up to 40 patients. Primary endpoints involve monitoring hematologic improvements such as transfusion dependency and hemoglobin level alterations, while secondary endpoints will explore HT-6184's effects on biomarkers indicative of inflammasome activity and the quantification of somatic gene mutation clones.

Looking Ahead

The conclusion of the Phase 2 trial is anticipated by the end of Q2 2025. Halia Therapeutics aims to gather pivotal data through this investigation to support further clinical development and eventual regulatory submission of HT-6184, fostering hopes of providing a viable oral treatment alternative for LR-MDS patients.

As Halia Therapeutics continues on its mission to discover and develop groundbreaking therapeutics, the momentum gained through HT-6184's progress demonstrates the company's commitment to improving lives affected by chronic inflammatory disorders. To learn more about Halia's innovative approaches and therapeutic pipeline, visit Halia Therapeutics' website.

Additionally, Halia Therapeutics encourages followers to connect on social media platforms such as LinkedIn and Twitter (X) to stay updated on their journey of enhancing patient care in the realm of inflammatory diseases.