Key Regulatory Milestone Provides Clear Development Roadmap to Registration for MOTYS
Initiation of Phase 3 Program Remains on Schedule to Begin in Q3 2025
CHAPEL HILL, NORTH CAROLINA / ACCESS Newswire / May 20, 2025 / Doron Therapeutics, a clinical-stage biotechnology company focused on regenerative therapies for musculoskeletal diseases, today announced the successful completion of its initial RMAT and End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for MOTYSTM (PTP-001), the company's lead biologic candidate for the treatment of knee osteoarthritis.
Key meeting outcomes include:
Confirmation of size and scope of Phase 3 clinical program and associated safety database to support proposed indication in knee osteoarthritis
Confirmation of pivotal study design, including primary endpoint and key secondary endpoints, with valuable input provided by FDA on statistical analysis plan
Alignment on plan for implementation of manufacturing changes and lot release specifications, with an emphasis on our 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.
"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. This will be a multi-site, multi-national, placebo-controlled RCT involving approximately 300 patients and designed to evaluate improvements in both pain and function for up to 12 months after a single MOTYS injection," said Alessandra Pavesio, Doron Therapeutics' CEO. "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. 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."
About OA
OA is the most common joint disorder and cause of chronic disability in the U.S. Prevalence of knee OA continues to grow because of an aging population, growing prevalence of obesity, and increased participation in active sports, affecting approximately 14 million Americans ages 25 and older, and nearly eight million under the age of 65. Accounting for more than $185 billion in annual expenditures, OA places a significant burden on the U.S. healthcare system and, with limited therapeutics options available, is the main contributor of the rise in joint replacements.
About MOTYS
MOTYS (PTP-001) is a terminally sterilized, off-the-shelf, regenerative biologic designed as an intra-articular knee injection for the management of symptomatic knee osteoarthritis. MOTYS' rigorously controlled and consistently potent secretome, growth factor, and extracellular matrix components are derived from donated placental tissues, following healthy at-term births. MOTYS was granted FastTrack and RMAT designations by FDA in January 2025.
About Doron Therapeutics
Doron Therapeutics is a clinical-stage biotechnology company harnessing the potent anti-inflammatory, tissue-protective and regenerative capabilities of placental tissue biologics to return patients suffering from degenerative musculoskeletal conditions to active, pain-free lives.
Contact Information
Sarah Caley
Head of Business Development
info@dorontherapeutics.com
SOURCE: Doron Threapeutics Inc
View the original press release on ACCESS Newswire:
https://www.accessnewswire.com/newsroom/en/biotechnology/doron-therapeutics-announces-positive-end-of-phase-2-and-initial-rmat-meeting-with-fd-1029758
