Ajax Secures $95M to Initiate JAK Drug Trials

Medicines known as JAK inhibitors are widely recognized in the pharmaceutical industry, with several approved for treating inflammatory conditions or cancer. These drugs generate billions in annual revenue for companies like AbbVie and Pfizer. However, JAK inhibitors have notable flaws. They don't alter the course of diseases and have recently been associated with serious safety issues. These limitations have prompted some startups to seek alternatives in the quest for improved immunological treatments.

Ajax Therapeutics, a biotech firm based in Cambridge, Massachusetts, and New York, has adopted a novel approach. Utilizing computational drug expertise from Schrodinger and research originating from Memorial Sloan Kettering Cancer Center, Ajax has developed a new type of JAK drug that functions differently from existing treatments. The company claims this innovation will result in more precise and effective therapies.

"No one is taking the same approach to targeting JAK enzymes as we are," stated CEO Martin Vogelbaum. Ajax is now positioned to validate its claims. The company recently secured $95 million in a Series C funding round and announced that the Food and Drug Administration has authorized the commencement of its first clinical trial. This study will evaluate a drug, codenamed AJ1-11095, designed to treat myelofibrosis, a type of bone marrow cancer.

The distinguishing factor of Ajax's drug is its binding mechanism. Existing myelofibrosis therapies, such as Incyte's Jakafi and Bristol Myers Squibb's Inrebic, affect one or more JAK enzymes. However, Vogelbaum notes that none specifically target JAK2 in its inactive state. Ajax believes that by doing so, their drug can bypass resistance mechanisms that reduce the efficacy of other JAK inhibitors. Preclinical research presented by Ajax at a medical conference indicated promising results.

Myelofibrosis disrupts red blood cell production, causing symptoms like anemia and fatigue. While current JAK therapies can alleviate symptoms, they don't alter the disease's progression and may even exacerbate anemia. Ajax asserts that its approach could offer more potent results, though this remains to be substantiated in clinical trials.

"Fundamentally, we believe that current myelofibrosis therapies are insufficient," said Ross Levine, a co-founder of Ajax and a leukemia specialist at Memorial Sloan Kettering. "JAK2 is the key driver of these diseases, and we saw an opportunity to improve its targeting."

Ajax's Series C funding was led by Goldman Sachs Alternatives and included investments from Eli Lilly, RA Capital Management, and Schrodinger, among others. The collaboration with Schrodinger, which previously aided Nimbus Therapeutics in discovering a TYK2 inhibitor acquired by Takeda for $4 billion, was instrumental in identifying Ajax's lead molecule.

"We leveraged the expertise of the TYK2 team from Schrodinger and applied it to JAK2," Vogelbaum explained.

Ajax's last funding round, held in 2021, raised $40 million from venture firms and academic research centers. With clinical trials imminent, the company has now secured more than double that amount.

"We're not a company with multiple targets and cancer types," Levine emphasized. "We have a clear focus on a specific target and disease, with future plans expanding beyond it."