The company is currently evaluating ALLOB, its investigational cell therapy product for bone regeneration.
Bone Therapeutics is turning their attention to their allogeneic cell therapy platform after disappointing results were observed in their phase 3 study (NCT04333160) of JTA-004 for the treatment of osteoarthritic knee pain.
The company, whose pipeline of products currently focus on addressing unmet needs within orthopedic indications, called the results “disappointing,” especially in light of good study execution and a good observed safety profile.
“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, MD, PhD, chief executive officer, Bone Therapeutics, in a statement.
JTA-004 is a next-generation intra-articular injectable that consists of a unique mix of hyaluronic acid, plasma proteins, and a fast-acting analgesic. The therapy was designed to provide added lubrication and protection to arthritic joint cartilage and to alleviate pain.
The study’s secondary end point was a statistically significant difference in pain reduction at month 3 between JTA-004 and Hylan G-F 20, which also failed. A post-hoc analysis showed a statistically significant difference in favor of JTA-004 and Hylan G-F 30 compared to placebo in a subanalysis of patients with higher pain scores at entry.
“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,” Forte added to the statement.
Bone’s allogeneic cell and gene therapy platform employs differentiated mesenchymal stromal cells sourced from bone marrow to address a range of indications. ALLOB is an off-the-shelf, investigational product for bone regeneration currently being evaluated for the potential treatment of delayed-union fractures and spinal fusion procedures, as well as osteotomy, maxillofacial, and dental.
The phase 2b study (NCT04432389) for delayed union fractures has so far enrolled 178 patients with fresh tibial fractures. ALLOB is expected to accelerate fracture healing and prevent late-stage complications in this indication. The trial will complete recruitment in the first half of 2022 and topline results are expected by the end of 2022.
ALLOB is also being evaluated in a phase 2a proof-of-concept trial in spinal fusion procedures (NCT02205138). This trial will assess primary outcomes such as lumbar fusion progression as assessed by CT scan, functional disability using Oswestry Disability Index, and adverse events, all at 12 months post-treatment. Bone Therapeutics expects to release 24-month results in the second half of 2022.
In their statement, Bone also revealed plans for next generation genetically engineered MSCs, including their investigational candidate BT-20, which is currently in preclinical development for inflammatory indications, and induced pluripotent stem cells.