Bone Therapeutics : 12/11/17 - Bone Therapeutics and Cellthera Pharm LLC announce intention to collaborate and Bone Therapeutics provides overview of its pipeline

Gosselies, Belgium, 11 December 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, and Cellthera Pharm LLC., the cell therapy subsidiary of Pharmstandard, JSC, a leading Russian pharmaceutical group, today announce that they have the intention to collaborate in the field of cell-based therapies for orthopaedics and bone diseases.
The companies will explore the opportunity of a potential licensing of Bone Therapeutics' cell therapy platforms, PREOB in non-traumatic osteonecrosis of the hip and ALLOB for use in delayed union fractures to Cellthera Pharm LLC for the Russian Federation and CIS countries. The project between the companies remains subject to further discussions and due diligence and, at this stage, it cannot be guaranteed the proposed collaboration will occur.
Furthermore, Pharmstandard International S.A., a fully owned subsidiary of Pharmstandard, JSC - Russia's leading pharmaceutical producer, has expressed an intention to participate in a future fundraising of Bone Therapeutics, if and when such a fundraising was to occur.

PIPELINE OVERVIEW
The Company has recently announced a series of positive clinical data further validating its unique bone cell therapy pipeline and today provides a pipeline overview following a previously announced review and streamlining of its portfolio priorities in the last year.
Ongoing clinical trials
Bone Therapeutics currently has two cell therapy products under clinical development (autologous PREOB and allogeneic ALLOB) for three indications in the field of orthopaedics and bone diseases.
ALLOB is currently being evaluated in two Phase II studies:

• Delayed-union fractures: In September, the Company reported positive interim data for its Phase I/IIA study, leading to the early conclusion of the study for strong efficacy. A Phase IIB is currently in preparation.
• Lumbar spinal fusion: In September, the Company also reported positive interim data for its Phase IIA study. The recruitment is expected to be finalized in early 2018.

PREOB is currently being evaluated in a Phase III trial in osteonecrosis of the hip. The Company is expecting interim analysis results in the second half of 2018. Moreover, as an important validation of its cell therapy technology, Bone Therapeutics has recently signed an exclusive license agreement with Asahi Kasei Corporation for the development and commercialization of PREOB in Japan.
Discontinued clinical trials
As previously announced, Bone Therapeutics has reprioritized its portfolio to ensure the best use of resources and to maximize value creation, whilst also taking into account indication specific hurdles discovered during the development process. Based on the clinical benefits, scalability, cost-effectiveness and ability to address large markets, Bone Therapeutics is focusing primarily on the further development of its allogeneic platform ALLOB. Several steps have already been taken as part of this strategy, which are reiterated below:

Despite reporting positive data, the Company opted to discontinue the clinical development of its bone cell therapy approach in severe osteoporosis without a partner, due to the complexity of the disease and the significant R&D investment required to continue clinical evaluation.

• Rescue lumbar spinal fusion

Given the positive interim Phase IIA results for lumbar spinal fusion, the Company decided to focus its resources on delivering a body of well controlled data on the application of ALLOB in lumbar spinal fusion, before developing other spine indications. Therefore, and given the investigators' recommendation to optimise the specific procedure used to administer ALLOB in rescue spinal fusion patients, the development of the indication of rescue spinal fusion has been discontinued and the recruitment of its Phase IIA trial halted. ALLOB was well tolerated but the limited number of patients enrolled in this study will not allow the Company to draw any relevant conclusion for this indication.

Following the announcement of strong interim data and the resulting early conclusion for the Phase IIA study for delayed union fractures, the Company decided to focus its efforts on ALLOB in the market of difficult fractures, and prioritize the indication of delayed union fractures (or patients at risk of non-union) with ALLOB, over the indication of non-union with PREOB. Bone Therapeutics therefore decided to discontinue recruitment and stop the Phase IIB/III study for non-union fractures with autologous PREOB once the follow-up of the currently enrolled patients has been completed. PREOB was well tolerated, but the limited number of patients enrolled in the non-union study due to slow recruitment, will not allow the Company to draw any relevant conclusions in the indication. There is currently no development of ALLOB or PREOB in the indication of non-union and if the Company were to revisit the indication, a new study would need to be initiated.
Bone Therapeutics believes that the clinical programmes it has chosen to focus on, supported by the recent positive interim data with ALLOB, offer strong commercial potential, the best value to patients and physicians, as well as short and medium-long term value creation for the Company.
Bone Therapeutics' current pipeline can be viewed on its website: http://www.bonetherapeutics.com/en/pipeline