Treadwell Therapeutics, a prominent clinical-stage biotechnology company dedicated to addressing unmet needs in cancer treatment, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for ocifisertib (CFI-400945), a pioneering PLK4 inhibitor designed for acute myeloid leukemia (AML). This special designation is reserved for drugs targeting rare diseases affecting fewer than 200,000 individuals in the U.S., offering significant incentives such as grants, tax credits, waiver of administrative fees, and seven years of market exclusivity post-authorization under the FDA’s Orphan Drug Act.
Ocifisertib is presently under evaluation in a Phase 1b/2 study involving adults with relapsed/refractory AML after standard care therapy. Having previously secured FDA Fast Track designation, Treadwell underscores its commitment to addressing the limited treatment options for AML patients, particularly those with TP53 mutated AML who face poor overall survival rates.
Simultaneously, Treadwell announced the appointment of Brenda Marczi as Senior Vice President and Head of Regulatory Affairs. Bringing over 30 years of regulatory expertise from her prior role as Senior Vice President of Regulatory Affairs at Tracon Pharmaceuticals, Inc., Brenda will play a crucial role in overseeing regulatory functions and filing activities in the U.S. and the UK. Her extensive background, including 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, positions her as a valuable addition to Treadwell.
Acting CEO of Treadwell, Dr. Roger Sidhu, expressed excitement about the FDA’s orphan drug designation and the earlier Fast Track designation for ocifisertib. He emphasized Treadwell’s dedication to addressing the high unmet clinical needs of patients with relapsed and/or refractory AML. Dr. Sidhu welcomed Brenda Marczi, highlighting her strategic leadership and regulatory affairs experience as pivotal for Treadwell’s progress, particularly as ocifisertib advances into potentially pivotal studies in 2025.
Treadwell Therapeutics is focused on advancing its clinical pipeline, including ocifisertib (PLK4 inhibitor), CFI-402257 (TTK/Mps1 inhibitor), and CFI-402411 (HPK1 inhibitor), along with various pre-clinical programs.