Doron Therapeutics has announced the successful completion of its End-of-Phase 2 and initial Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) for its lead biologic candidate MOTYS™ (PTP-001) in the treatment of knee osteoarthritis (OA). This regulatory milestone provides a clear development roadmap for the company as it prepares to initiate its Phase 3 program in Q3 2025.
The FDA meeting yielded several positive outcomes, including confirmation of the size and scope of the Phase 3 clinical program and associated safety database needed to support the proposed indication in knee osteoarthritis. The agency also confirmed the pivotal study design, including primary and key secondary endpoints, while providing valuable input on the statistical analysis plan. Additionally, there was alignment on the implementation of manufacturing changes and lot release specifications, with emphasis on Doron's proprietary disease-relevant methods for assessing drug potency, purity, and identity.
"We're thrilled by the outcome of this meeting and with the Agency's constructive feedback providing clear and actionable guidance on key elements of our Phase 3 program," said MK Kottke, Head of Regulatory and CMC at Doron Therapeutics.
Phase 3 Trial Design
The upcoming Phase 3 study will be a multi-site, multi-national, placebo-controlled randomized clinical trial involving approximately 300 patients. The trial is designed to evaluate improvements in both pain and function for up to 12 months following a single MOTYS injection.
Alessandra Pavesio, Doron Therapeutics' CEO, expressed confidence in the program: "With confirmation of our Phase 3 study design, including a clinically meaningful and statistically robust primary endpoint, we're preparing for the start of our first Phase 3 study. The strength of our clinical evidence, coupled with regulatory alignment on our streamlined Phase 3 program, positions us to advance MOTYS as a potentially transformative therapy in a space where innovation has been lacking and long dominated by sub-optimal therapies."
The Burden of Osteoarthritis
Osteoarthritis represents the most common joint disorder and cause of chronic disability in the United States. The prevalence of knee OA continues to grow due to an aging population, increasing obesity rates, and greater participation in active sports. The condition affects approximately 14 million Americans ages 25 and older, with nearly eight million under the age of 65.
The economic impact is substantial, with OA accounting for more than $185 billion in annual expenditures in the U.S. healthcare system. With limited therapeutic options available, OA is the main contributor to the rise in joint replacement surgeries.
MOTYS: A Novel Approach to Knee Osteoarthritis
MOTYS (PTP-001) represents a novel approach to treating knee osteoarthritis. It is a terminally sterilized, off-the-shelf, regenerative biologic designed as an intra-articular knee injection for the management of symptomatic knee osteoarthritis. The product's consistently potent secretome, growth factor, and extracellular matrix components are derived from donated placental tissues following healthy at-term births.
The FDA has recognized the potential of this innovative therapy by granting MOTYS both FastTrack and RMAT designations in January 2025. These designations are reserved for therapies that address serious conditions with significant unmet medical needs and demonstrate preliminary clinical evidence indicating the potential for substantial improvement over available therapies.
Regulatory Significance
The RMAT designation, established under the 21st Century Cures Act, is particularly significant as it provides accelerated development and review pathways for regenerative medicine therapies. This designation, along with the successful End-of-Phase 2 meeting, positions Doron Therapeutics to advance MOTYS through the regulatory process more efficiently.
Pavesio added, "We're grateful for the Agency's commitment to support innovative therapies advancing this field and look forward to our continued partnership with regulators and clinical investigators in bringing durable pain relief and restored mobility to the millions of patients who need it."
As Doron Therapeutics moves forward with its Phase 3 program, the company aims to address a significant unmet need in the treatment of knee osteoarthritis, potentially offering patients a regenerative alternative to current therapies and possibly delaying or preventing the need for invasive joint replacement surgeries.
