Bone Therapeutics Reports First Results of Its Phase IIA Trial for PREOB® in Severe Osteoporosis

BONE THERAPEUTICS (Brussels:BOTHE) (Paris:BOTHE) (Euronext Brussels and Paris: BOTHE) the bone cell therapy company addressing high unmet medical needs in the field of bone fracture repair and bone fracture prevention, today announces the first results of its Phase IIA PREOB® osteoporosis trial.

Out of the eight patients enrolled, seven were treated in the first part of this Phase IIA trial, which aims to evaluate PREOB® in the treatment of osteoporosis patients who do not respond to anti-osteoporotic therapy1. Primary endpoints of the study are safety and biodistribution of PREOB® cells administered intravenously: homing of cells to the bones most prone to osteoporotic fractures is indeed the first condition for the therapy to have effect.

The biodistribution of radioactively-labelled PREOB® cells was followed during 72 hours using SPECT/CT2 scan to determine their location in the body after intravenous administration. Follow-up of this first group of patients showed a progressive accumulation of PREOB® cells into the axial skeleton (i.e., vertebrae and pelvis) after injection. This outcome, which is necessary to support future efficacy results, could lead to clinical benefits for the patient. Importantly, no serious adverse events related to the treatment were reported.

In the study, patients with severe osteoporosis aged 40 to 85 years are treated by intravenous infusion of PREOB® to determine the fate of the osteoblastic cells after administration and their effect on clinical symptoms, on the occurrence of fractures and on bone turnover.

Osteoporosis is a condition characterized by an excessive loss of bone mass, leading to bone fragility and increased fracture risk. Osteoporosis is considered a serious public health concern and ageing demographics continue to increase the affected population. Current treatments for osteoporosis predominantly inhibit bone resorption, but do not actively stimulate bone formation, and up to one third of patients under treatment are still losing bone mass or experiencing fractures. The potential market of patients who do not respond adequately to treatment is around 10 million patients, or about a third of this $8 bn drug market3 (growing at a 4-5% rate per year). With PREOB®, Bone Therapeutics aims to offer a new mechanism of action based on the administration of differentiated bone-forming cells that provide osteogenic, osteoclastic and angiogenic properties4.

These positive safety data and biodistribution readings show progression towards the Company’s goals in osteoporosis as outlined at the time of its Initial Public Offering in February.

Enrico Bastianelli, CEO of Bone Therapeutics, commented: “In accordance with the clinical pipeline strategy outlined at the time of the IPO, we are delighted to share the first results from the PREOB® osteoporosis trial. Importantly, no treatment-related safety concerns have been reported for the first patient cohort, and the fact that we have shown that our intravenously administered osteoblast are reaching their target seems promising for the upcoming results.”

1 Eight patients were enrolled in the study, of whom one did not meet the eligibility (i.e., inclusion & exclusion) criteria for participating into the study and was therefore not treated.
2 A SPECT (single photon emission computed tomography) scan visualizes the radioactive label attached to the cells in order to determine their location in the body. This is combined with a CT scan, that visualizes anatomical structures using x-rays, to allow specification of the exact location.
3 Transparency Market Research, Osteoporosis Drugs Market
4 Osteoblastic = stimulating bone formation ; osteoclastic = stimulating bone resorption ; angiogenic = stimulating blood vessel formation