AJ1-11095

Medicines called JAK inhibitors are well known in the pharmaceutical industry. Several are approved to treat inflammatory disease conditions or cancer, and regularly bring in billions of dollars in yearly revenue for drugmakers like AbbVie and Pfizer.But JAK inhibitors are flawed. They dont change the course of a disease, and in the last few years,have been linked to serious safety concerns. Those weaknesses have led some startups to look elsewhere in the ongoing search for better immunological drugs.Ajax Therapeutics, a biotechnology company based in Cambridge, Massachusetts, and New York, has a different idea. With the help of computational drug specialist Schrodinger and research that originated at Memorial Sloan Kettering Cancer Center, Ajaxdesigned a newer type of JAK drug that works differently than others that have come before it. The result, the company says, should be a more precise and potent therapy.Nobody's taking the same approach that we're that we're taking to target JAK [enzymes], said CEO Martin Vogelbaum.Ajax will now get a chance to prove those claims. On Monday, it closed a $95 million series C round and said the Food and Drug Administration has cleared the company to begin its first clinical trial. That study will test a drug, codenamed AJ1-11095, Ajax is developing for the bone marrow cancer myelofibrosis.The key difference with Ajaxs drug is how it binds to its target. Multiple approved therapies for myelofibroris, like Incytes Jakafi or Bristol Myers Squibbs Inrebic,affect one or more JAK enzymes. But none of them specifically target JAK2 while the enzyme is in an inactive state, according to Vogelbaum.Doing so, the company says, should enable the drug to overcome resistance mechanisms that limit the effectiveness of other JAK medicines. Preclinical researchAjax has presented at a medical meeting hinted at such potential.Myelofibrosis is a type of bone marrow cancer that disrupts red blood cell production, leading to symptoms like anemia and fatigue. Though multiple JAK therapies are available, led by Jakafi, they affect symptoms, not the diseases course, and some have shown in testing that they can worsen anemia. Ajax claims its approach could lead to more powerful results, though that hasnt yet been proven in trials.We believe, fundamentally, that current myelofibrosis therapies aren't good enough, said Ross Levine, one of Ajaxs co-founders and a leukemia specialist at Memorial Sloan Kettering. JAK2 is the one that drives these diseases, and we thought there was an opportunity to drug it better.Ajaxs series C round was led by Goldman Sachs Alternatives, and included participation from Eli Lilly, RA Capital Management and Schrodinger, among others. A collaboration between Ajax and Schrodinger the firm that worked with Nimbus Therapeutics to discover a so-called TYK2 inhibitor Takeda bought for $4 billion led to the discovery of its lead molecule.We took the [TYK2] team from Schrodinger and poured it into JAK2, given their expertise, Vogelbaum said.Ajaxs last funding round came in 2021, when it brought in $40 million from a group of venture firms and academic research centers. With clinical trials soon on the way, it was able to raise more than twice that number this time around.Were not a company with five different targets and cancers, Levine said. We're not trying to be a platform company. We really do have a clear focus on a target and a disease, with plans, of course, beyond it. '

Financing led by Goldman Sachs Alternatives with participation by new investors Lilly, Vivo Capital, RA Capital Management, Point72 and existing investors AJ1-11095 will be the first Type II JAK2 Inhibitor to enter the clinic; Phase 1 study in myelofibrosis expected to begin in 2H 2024 NEW YORK & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Ajax Therapeutics, Inc., a biopharmaceutical company developing next generation JAK inhibitors for patients with myeloproliferative neoplasms (MPNs), today announced the closing of an oversubscribed $95 million Series C financing. Proceeds from the financing will be used to support the clinical development of Ajax’s first-in-class Type II JAK2 inhibitor, AJ1‑11095, for the treatment of myelofibrosis, as well as advancing the company’s pipeline of treatments for MPNs. The financing was led by Goldman Sachs Alternatives with participation by Eli Lilly and Company, Vivo Capital, RA Capital Management, Point72 and existing investors EcoR1 Capital, Boxer Capital, Schrödinger, Inc. and Inning One Ventures. Concurrent with the financing, Amit Sinha, Head of Life Sciences Investing and Ming Cheah, PhD, Vice President, within Life Sciences Investing at Goldman Sachs Alternatives, joined Ajax’s board of directors. “We’re pleased to have attracted this level of support from such distinguished life sciences investors and biopharmaceutical companies in addition to our existing investor syndicate,” said Martin Vogelbaum, co-founder and CEO of Ajax Therapeutics. “We are now well positioned to bring much needed innovation to the field of JAK inhibitors for the treatment of MPNs and look forward to advancing AJ1‑11095 into the clinic for myelofibrosis later this year.” AJ1-11095 was designed by Ajax, through our collaboration with Schrödinger, using structure-based drug design and computational methods at scale, to selectively bind the Type II conformation of the JAK2 kinase and to provide greater efficacy with disease modification compared to all currently approved JAK2 inhibitors which bind the Type I conformation of JAK2. Additionally, AJ1-11095 has been shown in preclinical studies to maintain efficacy against MPN cells that become resistant to chronic Type I JAK2 inhibition. “Despite significant advances brought by the introduction of JAK inhibitors, patients with MPNs continue to have major unmet needs as current therapies, including Type I JAK2 inhibitors, often fail to provide adequate symptomatic relief and have little effect on the underlying disease,” said Amit Sinha, Head of Life Sciences Investing at Goldman Sachs Alternatives. “We look forward to working with Ajax’s management team to bring novel therapies, such as AJ1-11095, to patients with myelofibrosis.” “JAK2 overactivation is central to the pathogenesis of MPNs and a Type II JAK2 inhibitor has the potential to address MPNs beyond myelofibrosis, including patients with polycythemia vera and essential thrombocythemia” said Ming Cheah, a Vice President in Life Sciences Investing at Goldman Sachs Alternatives. “We are proud to support the Company in delivering a new generation of transformational treatments for MPN patients.” “This financing reinforces the value of Ajax’s approach to inhibiting JAK2 with its Type II inhibitor, AJ1-11095,” said Dr. Ross Levine, Ajax co-founder and Chair of Ajax’s Scientific Advisory Board, Senior Vice President for MH Translational Research and Member of the Human Oncology and Pathogenesis Program at Memorial Sloan Kettering Cancer Center. “My lab has been studying Type II inhibition of JAK2 for over 10 years and we believe AJ1-11095 possesses the unique therapeutic properties and disease modifying effects of a highly selective and potent Type II JAK2 inhibitor and we’re excited to bring it to patients with MF.” About Ajax Therapeutics Ajax Therapeutics, Inc. is pursuing uniquely selective approaches to develop novel next generation therapies for myeloproliferative neoplasms (MPNs), including myelofibrosis. By combining the deep cancer and structural biology insights of our founding scientists with the industry’s most advanced computational drug discovery and protein structure platforms, we aim to discover and develop more precisely designed therapies to address the significant unmet needs for patients with MPNs. Please find more information at . About Goldman Sachs Alternatives Goldman Sachs (NYSE: GS) is one of the leading investors in alternatives globally, with over $450 billion in assets and more than 30 years of experience. The business invests in the full spectrum of alternatives including private equity, growth equity, life sciences, private credit, real estate, infrastructure, hedge funds, and sustainability. Clients access these solutions through direct strategies, customized partnerships, and open-architecture programs. The business is driven by a focus on partnership and shared success with its clients, seeking to deliver long-term investment performance drawing on its global network and deep expertise across industries and markets. The alternative investments platform is part of Goldman Sachs Asset Management, which delivers investment and advisory services across public and private markets for the world’s leading institutions, financial advisors, and individuals. Goldman Sachs has over $2.8 trillion in assets under supervision globally as of March 31, 2024. NOTE: Dr. Ross Levine serves on the board of directors of, has provided advisory services for, and has equity interests in Ajax Therapeutics. Dr. Levine also has intellectual property rights and interests that MSK has licensed to Ajax. MSK has intellectual property rights and other financial interests related to Ajax. View source version on businesswire.com: Contacts Kathryn Morris, The Yates Network LLC 914-204-6412 kathryn@theyatesnetwork.com Source: Ajax Therapeutics, Inc. View this news release online at: