BONE THERAPEUTICS, the cell therapy company addressing unmet medical needs in orthopedics and other diseases, announces that the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 did not meet the primary and consequently the key secondary endpoints.
The primary objective of the JTA-004 Phase III study was to demonstrate the efficacy of JTA-004 in reducing osteoarthritic knee pain compared to placebo as measured by the WOMAC® pain subscale three months after treatment. A key secondary objective was the comparison between JTA-004 and comparator Hylan G-F 20 in knee pain relief at month 3. Despite JTA-004’s favorable safety profile, the study did not achieve its main objectives as no statistically significant difference in pain reduction could be observed between any of the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.
A statistically significant difference in favor of JTA-004 and the active comparator versus placebo was seen in a post-hoc analysis in a subset of patients with higher pain scores at entry.
The Company, in collaboration with existing and potential partners, will consider the options for the future of JTA-004 development.
“The execution of the study was flawless and a good safety profile was observed in line with previous results. These JTA-004 efficacy results are disappointing. Knee osteoarthritis studies are recognized across the industry to be challenging to evaluate. They are also frequently complicated by a high placebo effect. We will continue to analyze the data and will consider potential next steps,” said Miguel Forte, Chief Executive Officer of Bone Therapeutics. “We are now fully committed to the clinical development of our advanced MSC allogeneic cell and gene therapy platform. Bone Therapeutics is concentrating on the development of this platform for the large market of orthopedic indications, with ALLOB. The progress with this platform has enabled us to expand it to other indications, including immunomodulation.”
Bone Therapeutics is focused on the development of its core assets, the allogeneic cell therapy platform, including ALLOB. ALLOB is currently being evaluated in a randomized, double-blind, placebo-controlled Phase IIb study in 178 patients with fresh tibial fractures at risk of delayed or non-union. 5% to 10% of complicated long bone fractures evolve to delayed union and non-union. This study will assess the potential for a single percutaneous injection of ALLOB to accelerate fracture healing and prevent late-stage complications in these patients. Recruitment is expected to be completed in the first half of 2022 and topline results by the end of 2022. Should the pandemic continue, Bone Therapeutics may have to re-evaluate these timelines and, in that eventuality, will communicate again to the market.
Bone Therapeutics is intensifying its efforts to expand its preclinical and clinical pipeline with additional indications by enhancing and “professionalizing” the therapeutic capacity of its cell and gene therapy platform. This includes the development of a next generation of genetically engineered mesenchymal stromal cells (MSC) and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells (iPSC).