Doron Therapeutics begins Phase 3 study of treatment for osteoarthritis of the knee

Sooner or later, about one in 10 Americans will experience knee pain, swelling and stiffness, according to a recent scientific paper. The most common diagnosis is osteoarthritis (OA) of the knee, a complex condition caused by multiple factors, including inflammation and wear and tear on these hard-working joints. The cartilage “cushion” that allows the knee to function smoothly erodes, leaving painful bone-on-bone contact. 

There is no cure yet for OA of the knee. Short-term measures such as oral pain medications, physical therapy or steroid injections into the knee can provide short-term relief. There are only a handful of longer-term alternatives to surgery, especially as the condition worsens.

Age and obesity are risk factors. OA is projected to increase quickly over the next 15 years, due to an older, heavier global population, said another study in the Journal of Orthopaedic Surgery and Research. Related economic effects are huge and the need for more cost-effective, non-surgical solutions is urgent. “OA represents a major source of healthcare expenditure, with an estimated global prevalence of approximately 364.6 million cases of KOA in 2019,” the study noted.

The booming field of biologics—biologically based products—seeks to provide that solution. Doron Therapeutics, a clinical-stage biotechnology company based in Chapel Hill, is developing novel biologics for musculoskeletal diseases.

The company, based in Chapel Hill, announced Oct. 18 that initial patients have been dosed in the Phase 3 clinical trial of MOTYS (PTP-001), the company’s lead biologic candidate for the treatment of knee osteoarthritis. The U.S. Food and Drug Administration has granted the biologic FastTrack and Regenerative Medicine Advanced Therapy. The Phase 3 study of this investigational therapy follows the successful completion of an End-of-Phase 2 meeting held with the FDA in May.

“The initiation of our Phase 3 study represents a critical inflection point for Doron Therapeutics,” said Chief Executive Officer Alessandra Pavesio. “We are advancing a highly differentiated therapy with the potential to address one of the largest areas of unmet need in medicine.” 

"Osteoarthritis remains the leading cause of pain and disability worldwide, yet treatment options for people living with this disease are woefully limited,” said the Phase 3 Principal Investigator David Hunter. Hunter also chairs the Department of Rheumatology at the University of Sydney and the Royal North Shore Hospital, Australia.

“This trial is an important step toward a biologic therapy that could transform care and bring real hope to millions of patients."

The Phase 3 study is a randomized, placebo-controlled trial enrolling approximately 300 patients across multiple sites and countries, with the goal of evaluating improvements in both pain and function for up to 12 months following a single MOTYS injection.