Bone Therapeutics Reports Initial Efficacy Results from Osteoblastic Cell Product Trial

Gosselies, Belgium-based Bone Therapeutics Inc., a bone cell therapy company addressing high unmet medical needs in the field of bone fracture repair, fracture prevention and spinal fusion, today announces the 12-month efficacy results from the first cohort of seven patients treated with Preob in its Phase IIA severe osteoporosis trial. These initial data demonstrate positive effects on pain and osteoporosis blood markers of a single administration of Preob.

Preob is Bone Therapeutics’ first-in-class autologous Osteoblastic Cell Product. Based on promising results of the proof-of-concept Phase II studies, which have shown statistically significant and clinically relevant benefits, Preob is currently in two pivotal trials in Europe: a Phase III trial for the treatment of hip osteonecrosis and a Phase IIB/III trial for the treatment of long bone non-union fractures. For these indications, Preob is administered percutaneously via a minimally invasive approach, avoiding the need for open surgery. Additionally, Preob is being evaluated in a proof-of-concept Phase IIA clinical trial for the treatment of severe osteoporosis, where it is administered intravenously.

Osteoporosis is a condition characterized by an excessive loss of bone mass due to impaired bone turnover. The natural balance between bone formation and bone resorption is disturbed, leading to bone fragility and increased fracture risk. The ongoing Phase IIA trial has been designed to evaluate a single intravenous administration of Preob in patients with severe osteoporosis, defined as those who no longer respond to anti-osteoporotic therapy. In total, 20 patients will be enrolled in the study and followed up over 12 months. The primary endpoints of the study are safety and biodistribution of Preob cells. In addition, effects on clinical symptoms (i.e., pain and general health status) and serum markers of bone turnover are being evaluated.
In this first cohort, patients experienced a pronounced and clinically relevant decrease in pain (of more than 40 percent), reaching a maximum at six months post-treatment. In a similar patient population, six-month daily subcutaneous administration of the bone anabolic agent teriparatide only achieved a 30 percent decrease in pain4. A same positive trend was also observed on the general health status of Preob-treated patients.

Moreover, analysis of the profile of bone markers in the blood shows a surprising dual trend: (i) in an early phase of the 12-month follow-up, a decrease (of more than 25 percent) of bone resorption (bone breakdown) markers5 was observed, while bone formation markers6 were either unaffected or even slightly increased and (ii) in a later phase of the 12-month follow-up, a continuous increase in bone formation markers was observed with a moderate increase in bone resorption markers. The unexpected early decrease in bone resorption markers is especially remarkable, as studies7 have shown that in a comparable population of severe osteoporosis patients, where bone turnover is totally suppressed, antiresorptive drugs have no additional effect on bone resorption.

These preliminary results thus seem to indicate that a single administration of Preob progressively stimulates bone remodelling, with a bone formation-to-resorption ratio more favourable than that generally reported with other anti-osteoporotic agents in the same patient population. Indeed, by comparison, while bone anabolic treatments in general show stronger effects on bone formation markers, their bone formation-to-resorption ratio is less favourable, with bone resorption twice as highly increased as bone formation8. Therefore, this surprising effect of Preob on bone turnover suggests a different—potentially more favorable—mechanism-of-action compared to existing therapies.

“These initial 12-month results are encouraging as they show that a single intravenous administration of Preob, through a different mechanism-of-action, could have beneficial effects on pain and bone turnover in treatment-resistant osteoporosis patients,” said Enrico Bastianelli, CEO of Bone Therapeutics. “We look forward to reporting additional results on this innovative approach.”