HPK1

With a Focus on Potential Pivotal Studies in 2025, Treadwell has Strengthened its Leadership Team by Appointing Brenda Marczi, PharmD, MBA as SVP and Head of Regulatory Affairs TORONTO & SAN FRANCISCO--(BUSINESS WIRE)-- Treadwell Therapeutics, a privately held clinical-stage biotechnology company pioneering and advancing novel first-in-class medicines for unmet needs in cancer, today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ocifisertib (CFI-400945), a first-in-class, investigational PLK4 inhibitor for the treatment of acute myeloid leukemia (AML). Ocifisertib is currently being evaluated in a Phase 1b/2 study in adults with relapsed/refractory AML following standard of care therapy. Orphan drug designation is granted by the FDA to drugs intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation. Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, grants, tax credits and waiver of certain administrative fees as well as seven years of market exclusivity following marketing authorization. Treadwell also announced today the appointment of Brenda Marczi, as SVP and Head of Regulatory Affairs. Brenda comes to Treadwell with over 30 years of regulatory experience in the pharmaceutical and biotechnology industries. She most recently served as Senior Vice President, Regulatory Affairs at Tracon Pharmaceuticals, Inc., where she oversaw their regulatory function and led all of their strategies and filing activities in the US and UK. Brenda holds a Master of Science in Pharmaceutical Sciences from Rutgers University, a PharmD from the University of Maryland, and an MBA from Wharton, University of Pennsylvania. “The FDA’s decision to grant orphan drug designation, along with the previous FDA Fast Track designation for ocifisertib underscores Treadwell’s dedication to addressing this patient population with few treatment options. Patients with relapsed and/or refractory AML, in particular TP53 mutated disease suffer poor overall survival and represents a high unmet clinical need,” said Roger Sidhu, M.D., Acting CEO of Treadwell. “We look forward to advancing ocifisertib in partnership with investigators, regulators, patients and their families for those with limited treatment options in tough to treat AML. In addition, we are thrilled to have Brenda join us to lead Regulatory Affairs at Treadwell. She is a seasoned strategic leader with a broad experience in leading regulatory strategy from early development through approval. Her leadership will be invaluable to Treadwell as we advance ocifisertib into potentially pivotal studies in 2025.” About Treadwell Therapeutics Treadwell Therapeutics is a clinical-stage oncology company developing novel medicines to address unmet needs in patients with cancer. The Company's robust, internally developed clinical pipeline includes ocifisertib (PLK4 inhibitor), CFI-402257 (TTK/Mps1 inhibitor) and CFI-402411 (HPK1 inhibitor). Treadwell also has a broad pre-clinical pipeline with multiple biologic and next generation TCR based autologous cell therapy programs. For more information, please visit .

- SIK and cGAS are key autoimmune regulators well suited for Nimbus’ structure-based drug design approach - - Company to present pipeline update at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 7:30 am PT - BOSTON--(BUSINESS WIRE)--Nimbus Therapeutics, LLC (“Nimbus Therapeutics” or “Nimbus”), a biotechnology company that designs and develops breakthrough medicines through its powerful computational drug discovery engine, announced the advancement and expansion of its pipeline with the addition of discovery programs targeting innate immunity pathways. These programs, targeting the salt-inducible kinase (SIK) family and cyclic GMP-AMP synthase (cGAS), represent promising opportunities to leverage Nimbus’ industry-leading computational and structure-based drug design expertise to develop highly selective, potent medicines addressing areas of significant unmet need. "Building on the success of our TYK2 program, we are broadening our drug discovery engine to unlock new difficult-to-drug targets with compelling biology,” said Peter Tummino, Ph.D., Chief Scientific Officer of Nimbus. “SIK and cGAS are critical targets in highly prevalent diseases that are well suited to Nimbus’ structure-based drug design approach. We look forward to advancing our discovery and development programs across oncology, immunology, and metabolism to deliver transformative medicines to patients.” Nimbus is advancing its Phase 1/2 trial (NCT05128487) of NDI-101150, a small molecule inhibitor of hematopoietic progenitor kinase 1 (HPK1), and has reported positive dose escalation data showing potential monotherapy clinical benefit for patients with solid tumors. Furthermore, the company expects to initiate IND-enabling activities this year for its oncology program targeting Werner syndrome helicase (WRN). In collaboration with Eli Lilly and Company, Nimbus continues to progress the development of novel targeted therapies that activate AMPK to potentially treat a broad range of metabolic disorders. Nimbus continues to expand its platform capabilities with ongoing investments in cutting-edge technology for drug discovery. Alongside proprietary computational tools which the company has developed in-house, Nimbus’ platform leverages state-of-the-art technology through collaborations such as its recently announced partnership with Anagenex, a leader in generative AI for drug design. Jeb Keiper, M.S., MBA, Nimbus’ Chief Executive Officer, will provide an overview of the company’s progress and pipeline and anticipated milestones for 2024 and beyond at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 7:30 am PT. About Nimbus Therapeutics Nimbus Therapeutics is a clinical-stage, structure-based drug discovery company developing novel small molecule medicines designed to act against well-validated but difficult-to-drug targets implicated in multiple human diseases. Nimbus combines leading-edge computational technologies with a tailored array of machine learning-based predictive modeling approaches. Nimbus’ pipeline includes a clinical-stage HPK1 inhibitor for the treatment of cancer (NCT05128487), as well as a diverse portfolio of preclinical programs focused on cancer, autoimmune conditions, and metabolic diseases. Nimbus is headquartered in Boston, Mass. To learn more about Nimbus, please visit .

According to the deal terms, Anagenex will generate billions of biochemistry data points for each disease target nominated by Nimbus. Anagenex will then use these data to train AI models that can design 100 million new molecules that bind to the targets, and select the best candidates for developing into drug candidates. The therapeutic areas of interest were undisclosed. In return, Nimbus will pay Anagenex an undisclosed amount upfront in addition to option and developmental milestone payments for each program in the collaboration. “We are thrilled to be partnering with Nimbus, one of the original and most successful computationally aided drug discovery and development companies,” said Nicolas Tilmans, CEO of Anagenex, in a public release. “We’re very excited to join forces with them in attacking new targets beyond oncology a few hundred million compounds at a time.” The vast majority of possible protein targets lack drug candidates because they don’t fit conventional understanding about how proteins can be targeted by small molecules, says Anagenex’s website. It is therefore difficult to find a “chemical starting point” for designing new drugs for these targets, and it can take years of trial and error to turn the starting point into a drug candidate, adds the website. To solve this bottleneck, Anagenex uses a mixture of machine learning and tools such as DNA Encoded Libraries (DELs) and Affinity Selected Mass Spectrometry (ASMS) to design drug candidates more quickly and efficiently than traditional methods. To finance the development of its technology and build a treatment pipeline, the firm raised a Series A round worth $30 million in 2022. googletag.cmd.push(function () {