Regulated Information

Bone Therapeutics announces all patients meet primary endpoint in ALLOB^® Phase I/IIA delayed-union study interim analysis

DSMB recommends concluding the study early based on positive efficacy data

Follows recent strong data for allogeneic product in spinal fusion

Gosselies, Belgium, 20 September 2017, 7am CEST - BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the bone cell therapy company addressing high unmet medical needs in orthopaedics and bone diseases, today announces positive interim efficacy results in the Phase I/IIA delayed-union study of its allogeneic bone cell therapy product ALLOB^® in 16 patients. Based on the evaluation of these interim data post a six-month follow-up, the Data and Safety Monitoring Board (DSMB) has recommended stopping the trial early due to the strong efficacy data. These interim data, combined with recently announced results for ALLOB^® in spinal fusion, establish ALLOB^® as a strong product candidate, representing a significant advance in Bone Therapeutics’ pipeline and building a foundation for future value creation.

The Phase I/IIA study was a six-month open-label trial to evaluate the safety and efficacy of ALLOB^® in the treatment of delayed-union fractures of long bones. 16 patients with a fracture that had failed to consolidate after a minimum of three and a maximum of seven months received a single percutaneous administration of ALLOB^® directly into the fracture site and completed the six-month follow-up. Fracture healing of ALLOB^®-treated patients was assessed using clinical evaluation (e.g. health status and pain) and radiological evaluation (based on CT-scan).
 
At six months post administration, 100% of patients met the primary endpoint, defined as an increase of at least 2 points on the radiological Tomographic Union Score (TUS) or an improvement of at least 25% of the clinical Global Disease Evaluation (GDE) score.
 
From a radiological perspective, the patients improved by on average 4 points on the TUS score (statistically significant), twice the required increase of 2 points. This minimum 2-point increase was achieved by 13 out of 16 patients (81%).
 
From a clinical perspective, the health status of patients, as measured by the GDE score, improved by on average 48% (statistically significant). The minimum 25% improvement was achieved by 12 out of 16 patients (75%). Pain at the fracture site, an important secondary endpoint, was reduced by on average 59% (statistically significant).
 
Overall, ALLOB^® was shown to be well tolerated. In one patient, a hypersensitivity reaction without any established cause was reported 4 weeks after administration, with full recovery. In the context of a clinical trial, an association with ALLOB^® cannot be excluded. As previously described in the literature covering clinical studies with allogeneic mesenchymal stem cells or their derivatives, it was observed that blood samples of about half of the patients contained donor-specific antibodies, either pre-existing or developed after administration.
 
Following the positive recommendation of the Data and Safety Monitoring Board based on the positive efficacy results observed in this study, the Company will stop the study recruitment immediately and prepare for the next clinical phase.

Thomas Lienard, CEO of Bone Therapeutics commented: "Following on from the recently reported strong results for our allogeneic product in spinal fusion, the early conclusion of this study in delayed-union fractures allows us to accelerate the development of our allogeneic platform and brings us a step closer to the commercialisation of a potentially game-changing treatment in these large and growing markets."

Miguel Forte, Chief Medical Officer of Bone Therapeutics, added: “These strong data for ALLOB^® demonstrate a significant improvement in delayed-union fracture patients whose only current option is either to wait and see or to undergo painful surgery with significant disease burden and long recovery times. The fact that the DSMB has recommended stopping this study points to the safety and efficacy of ALLOB^® and to its feasibility in this large and promising indication.”
 
The strong Phase IIA interim results for the allogeneic ALLOB^® study in delayed-union fractures will lead Bone Therapeutics to further develop ALLOB^® in difficult fractures. Given the close relationship between the delayed-union and non-union indications, the Company is stopping the recruitment in the non-union trial with its autologous product PREOB^® to focus resources in the allogeneic platform and provide optimal value for patients.