As orthobiologic technologies continue to advance, the ever-present need for robust clinical data remains in order to demonstrate their effectiveness and for products to receive regulatory, payor and hospital approval. In 3Q18, ORTHOWORLD reported on five orthobiologic products in various phases of clinical trials/studies.
First, we draw your attention to two companies that announced study results for their respective products: Bone Therapeutics’ ALLOB® allogenic bone cell therapy to treat fractures and Histogenics’ NeoCart® to treat knee cartilage defects.
All patients enrolled in the Phase I/IIA study of Bone Therapeutics’ ALLOB allogeneic bone cell therapy met the primary endpoint of statistically significant clinical and radiological improvements in the treatment of delayed-union fractures.
At six months following one percutaneous dose of ALLOB to the fracture site, the 21 patients in the study achieved an increase of at least two points on the radiological Tomographic Union Score or an improvement of at least 25% of the clinical Global Disease Evaluation score vs. baseline. Pain at the fracture site, a secondary endpoint, was also reduced by an average of 61%.
ALLOB can be administered via a minimally invasive procedure and is derived from ex vivo cultured bone marrow cells of healthy adult donors. Bone Therapeutics is also studying ALLOB’s potential use in lumbar spinal fusion.
The company’s next priority is submitting an application for a Phase IIB trial in delayed-union fractures. The multicenter, randomized, controlled study is expected to begin in 2H19.
After more than a decade of development, the Phase III clinical trial of Histogenics’ NeoCart tissue-engineered implant did not meet the primary endpoint of a statistically significant improvement in pain and function vs. microfracture in a dual threshold responder analysis at one year.
NeoCart is an autologous cell therapy for the treatment of knee cartilage defects. An implant is produced using a patient’s own cartilage cells harvested from the non-weight-bearing cartilage surface of the femur.
In the modified Intent to Treat population (mITT, which excludes patients who were randomized but not treated with NeoCart), 74% of NeoCart patients exhibited clinically meaningful improvements in pain and function vs. 62% of microfracture patients. In this mITT population, patients treated with NeoCart achieved a statistically significant improvement in pain and function at six months vs. microfracture.
Analysis of the Phase III NeoCart data has yielded important observations, such as a statistically significant improvement of NeoCart vs. microfracture in lesion sizes of greater than 2cm and patients with higher body mass index. Data from this trial are also consistent with results seen in prior clinical trials of NeoCart, as well as the biomechanical data generated as part of a collaboration with Cornell University.
The company will continue to pursue a Biologics License Application with FDA.
This Phase III trial is believed to be the largest and first prospectively designed, randomized clinical trial in North America to evaluate safety and efficacy of a restorative cell therapy to treat knee cartilage damage. Further, it is also believed to be the only trial with a dual threshold responder analysis endpoint.
We spoke with Histogenics CEO Adam Gridley at AAOS earlier this year, where he discussed opportunities that the company is embracing, as well as the health of the orthobiologics market. You can read Gridley’s comments here.
As previously mentioned, five companies announced trial updates. Three enrolled their first patients: CartiHeal’s Agili-C™ implant and Cerapedics’ P-15L Peptide Enhanced Bone Graft, both in Investigational Device Exemption studies, and Kuros Biosciences’ MagnetOs bone graft substitute in a blinded controlled randomized study.
The time and capital invested in bringing these products to market is immense. However, as we noted in THE ORTHOPAEDIC INDUSTRY ANNUAL REPORT®, “development of novel technologies in this space are expected to come from smaller companies. Once they develop and prove efficacy of their products…larger companies will seek acquisitions or collaboration agreements to scale these offerings.”
*Images courtesy of manufacturers