A large pharma company wanted to buy Ajax Therapeutics when it was raising a $40 million Series B in 2021. But the startup thought it could do better on its own.
“We thought that the compound at the time, which was part of the abstract in our 2022 ASH presentation, was a progenitor of what we’ve developed today. So we’ve actually made it better,” CEO Martin Vogelbaum told Endpoints News. “And we felt we were the team that could make it better and get to the best compound that we could take into the clinic.”
The New York biotech has now raised a $95 million oversubscribed Series C to get into the clinic, the five-year-old startup disclosed Monday morning. Goldman Sachs Alternatives led the raise, which included support from Eli Lilly, Vivo Capital, RA Capital Management and others. Ajax had previously raised close to $50 million, Vogelbaum said in an interview.
Ajax will enter Phase 1/2 testing later this year with AJ1-11095. The company believes it is the first type II JAK2 inhibitor, and its scientific founders hope the drug will be more selective than the existing class of JAK2 medicines, first approved in 2011.
“Although it was really gratifying to see the early efforts lead to ruxolitinib, fedratinib, momelotinib, pacritinib, they don’t achieve what we need for patients,” Ross Levine, an Ajax co-founder and SVP for translational research at Memorial Sloan Kettering Cancer Center, said in an interview. “They don’t achieve molecular or pathologic regression. We don’t get patients where we want them to get where we do with some other tyrosine kinase inhibitors.”
That yearning for a better medicine led Levine and a group of other JAK researchers and clinicians to come together around 2017.
“Ross and four of the other A-list celebrity JAK experts in the world pulled this academic consortium together. They were affiliated with Memorial Sloan Kettering, Dana-Farber, NYU and a couple of institutions in Europe and it was called ‘Attack JAK,'” Vogelbaum said.
When Vogelbaum heard about the work, he approached the CEO of Schrödinger, the decades-old computational chemistry biotech that’s now building its own pipeline, as well. Schrödinger helped create AJ1-11095 using its structure-based design capabilities and owned 6.3% of Ajax as of Dec. 31, according to an SEC filing.
Ajax will first investigate its oral treatment in patients who don’t benefit from current JAK2 inhibitors, Levine said. The company also thinks it can eventually move into a first-line setting, the two said in a joint interview.
“Based on data we have, by having a more JAK2 dialed-in drug, it should do what you want and do less of what you don’t want,” Levine said. “We do think that we’ll get better efficacy and that some of the off-target liabilities will be mitigated because of the effort we put in to develop a more selective compound.”
Deals, deals, deals
Ajax hopes to make its initial clinical data splash at the annual American Society of Hematology confab in December 2025, Vogelbaum said. That data could potentially once again spur pharma companies to circle the biotech.
There’s precedent. Drugmakers have made a series of billion-dollar or higher acquisitions for biotechs in the myelofibrosis field, Vogelbaum said.
Novartis is currently working to buy MorphoSys for $2.9 billion to get its myelofibrosis candidate pelabresib, which is expected to be filed for US and European regulatory approval this year. Meanwhile, GSK received FDA approval last September for momelotinib, the drug now marketed as Ojjaara that it got via a $1.9 billion acquisition of Sierra Oncology in 2022. Sobi acquired CTI BioPharma for $1.7 billion last year.
And Merck has made two deals of its own: Its $11.5 billion Acceleron purchase, while largely focused on cardiovascular, also included work in myelofibrosis, and the New Jersey pharma also paid $1.35 billion for Imago BioSciences in 2022, getting access to a candidate being tested in myelofibrosis, among other indications.
Ajax will be “open-minded” about what path it chooses, Vogelbaum said.