FDA Clears Ajax Therapeutics' IND for AJ1-11095 to Treat Myelofibrosis

Ajax Therapeutics, Inc., a biopharmaceutical company focused on the development of advanced JAK inhibitors for treating myeloproliferative neoplasms (MPNs), has announced that the U.S. Food and Drug Administration (FDA) has approved their Investigational New Drug (IND) application. This approval allows Ajax to initiate a Phase 1 clinical study of their novel Type II JAK2 inhibitor, AJ1‑11095, targeting patients with myelofibrosis.

Martin Vogelbaum, co-founder and CEO of Ajax Therapeutics, expressed his excitement about the FDA's clearance, stating that this milestone marks the company’s first program to enter clinical trials. This is also the first clinical study to evaluate a Type II JAK2 inhibitor in patients. David Steensma, MD, FACP, Chief Medical Officer at Ajax, highlighted the innovative nature of AJ1-11095, emphasizing its potential to offer improved efficacy and disease-modifying effects compared to existing therapies.

AJ1-11095 was developed through a collaboration with Schrödinger, utilizing structure-based drug design and computational methods. This next-generation JAK2 inhibitor aims to selectively bind the Type II conformation of the JAK2 kinase, providing greater efficacy and disease modification than the currently approved Type I JAK2 inhibitors. Preclinical studies have shown that AJ1-11095 can reverse marrow fibrosis, reduce mutant allele burden, and maintain efficacy against MPN cells resistant to chronic Type I JAK2 inhibition.

Myelofibrosis (MF) is a rare blood cancer affecting approximately 20,000 patients in the United States. It is characterized by symptoms such as spleen enlargement, bone marrow scarring (fibrosis), progressive anemia, fatigue, night sweats, itching, and abdominal discomfort. These symptoms significantly impact patients' quality of life. Current treatments for MF primarily involve Type I JAK2 inhibitors, which can reduce spleen size and alleviate symptoms but have limited effect on the underlying disease. Over time, many patients discontinue Type I JAK2 inhibitor therapy due to lack of benefit, adverse events, or disease progression, indicating a significant unmet need for more effective treatments.

Ajax Therapeutics aims to address these needs by developing precisely designed therapies for MPNs, including myelofibrosis. The company's approach combines insights from cancer and structural biology with advanced computational drug discovery and protein structure platforms to create novel treatments.

In conclusion, the FDA’s clearance of the IND application for AJ1-11095 signifies a significant step forward for Ajax Therapeutics in their mission to provide new treatment options for myelofibrosis patients. With the initiation of the Phase 1 dose escalation study expected in the second half of 2024, there is hope for improved therapies that can better manage or potentially modify the course of this challenging disease.