Bone Therapeutics SA
Medical innovation is central to the advancement of treating diseases like cancer, but in comparison the standard of care in bone medicine can seem outdated, still reliant on invasive surgeries or the insertion of prosthetics with a high risk of complications.
Bone Therapeutics is one of the most advanced companies in its field, focused on bone regenerative therapy and striving to address the unmet needs in bone diseases and orthopaedic conditions via its novel and minimally invasive approach. The Company offers the potential for a paradigm shift in the treatment of orthopaedic conditions, combining bone-forming cell products with a minimally invasive approach. The Company’s world leading pipeline of advanced products offers a solution to the major problems outlined above.
The Company’s world-class products are composed of osteoblastic cells (i.e. differentiated bone-forming) used for the treatment of bone diseases by administration directly into the patient damaged bone site(s) and/or by systemic administration.
Bone Therapeutics’ differentiated cells have already acquired a bone-forming capability (specialization) and are thus more efficient to have beneficial effects in bone disease settings than any other cell type.
Safety is also improved through this specialization. Indeed, acquired function avoids the toxicity risk due to unintended biological activities as well as uncontrolled proliferation, as sometimes observed with undifferentiated cells.
Bone Therapeutics has one of the world’s most advanced pipelines, with two first-in-class products in clinical development, and offers a superior alternative to the current standard of care. PREOB®, a autologous osteoblastic cell product – based on cells from the patient is in Phase III trials for the treatment of osteonecrosis and non-union fractures, and in a Phase I/IIa for severe osteoporosis. ALLOB®, an allogeneic therapy based on cells from a healthy universal donor, is in a Phase I/IIa for the treatment of delayed union fractures.
PREOB® targets debilitating diseases including osteonecrosis, a painful hip disorder which leads ultimately – in less than two years – to femoral head collapse, requires total hip replacement, and non-union fractures. Unlike many bone disorders, osteonecrosis is a condition which typically affects younger patients, so prosthetics are not usually appropriate. The efficacy of the current standard of care, if one exists, in the early stage of the disease is very controversial. New cases of osteonecrosis are growing at about 150,000 per year in the EU and US. PREOB® can also offer a new paradigm in non-union fractures, a condition affecting around 200,000 new cases per year, which are disproportionately younger people. PREOB® has already acquired orphan drug status for the treatment of osteonecrosis in both regions.
In trials, the use of PREOB® in non-union fractures has shown significant improvements over the current standard of care; the Phase IIb trial in osteonecrosis showed a more than 50% improvement in pain and function and an over 40% reduction in fracture risk in a follow-up after 24 months.
PREOB® also has potential in severe osteoporosis, in patients who don’t respond to any treatment at all, and who account for about a third of the 30 million patients affected by osteoporosis in the US and EU. The total osteoporosis market is estimated to be worth up to $8 billion. PREOB® is currently in a Phase IIa open label study for osteoporosis.
ALLOB®, in Phase I/IIa trials, is the first allogeneic differentiated osteoblastic cell therapy product for orthopaedic conditions such as delayed union fractures.
Allogeneic cell therapy involves the harvesting of cells from a healthy universal donor, rather than from the patient. Delayed union fractures, defined by the absence of healing after 3 months, affect between 600,000 and 900,000 new patients a year in Europe and the US. The administration of ALLOB® takes about 20 minutes and can be done at a day-clinic.
ALLOB® also has the potential to be applied to conditions such as osteogenesis imperfecta, a rare genetic bone disease characterized by bone fragility and fractures.
The Company has a scalable, state of the art GMP facility with manufacturing authorizations.
A recent review of literature Dr W. Richter, director of the research centre for experimental orthopaedics at Heidelberg University Hospital, points to the superiority of bone marrow stem cells – such as those used by Bone Therapeutics – over adipose-based stem cells. Bone marrow cells were shown to form new bone naturally, compared with adipose cells which show some potential only when stimulated to do so.
The need for cutting edge bone medicine has never been greater. Of the approximately 4 million orthopaedic procedures conducted every year, roughly half are addressable by cell therapy. Bone Therapeutics’ minimally invasive highly effective approach offers the potential to set a new standard.