Cellular therapies have not covered themselves in glory over the past few years, although considering the extraordinarily high hopes for them disappointment was almost inevitable. Even with today's more tempered expectations, the company that brings a cell-based treatment to market will distinguish itself.
The Belgian company Bone Therapeutics is determined to succeed where others have failed. “We have unique products, differentiated bone-forming cells. This is a key difference with respect to other cell therapy companies, most of which are just producing undifferentiated stem cells,” Enrico Bastianelli, the company’s chief executive, says in a face-to-face interview with EP Vantage.
As its name suggests, the company is focused on just one type of tissue. But this is one of its advantages, Mr Bastianelli says. “Bone is ideally suited for cell therapy as it is a naturally regenerative system. We have true bone-forming cells – a very specialised type of cells.”
Its main technology, called Preob, is produced using patients’ own mesenchymal stem cells, taken from their bone marrow. The cells are extracted, treated to induce their differentiation into osteoblastic cells, and reintroduced into the patient at the site of bone damage. Preob is in phase III for the treatment of osteonecrosis and non-union fractures, and Bone Therapeutics has just begun a Phase IIa trial as a treatment for severe treatment-refractory osteoporosis.
This is an important step, Mr Bastianelli says. “We are more focused today on the orthopaedic indications – bone reconstruction – and this is a totally new area.”
And the potential market for the company’s technology could be very large. “The area we are operating in with the orthopaedic indication is huge. The demand is high, with significant unmet needs and very low competition,” Mr Bastianelli says. “In addition, with the osteoporosis trial we broaden the application field for our product.”
He explains that worldwide more than 30 million people, mainly women, have osteoporosis, and over 40% of patients treated with drugs do not respond. “We think this could change the paradigm of treatment. Preob has multiple mechanisms of action, as opposed to a single pharmacologic agent which has only one mechanism of action. And the treatment we have is not only bone-forming, it also recruits osteoclasts.”
Preob’s most advanced indication is osteonecrosis, a disorder usually occurring in the hip where bone tissue dies leading to the bone collapsing. “We want to ... avoid the evolution towards a fracture by implanting the cells directly into the necrotic zone of the bone,” says Mr Bastianelli. He says phase II in this indication showed that Preob was associated with a statistically significant improvement in pain and function of the hip compared with a bone graft, with an improvement from baseline of 60% at 24 months.
Preob will soon enter a phase III trial for non-union fractures, and though this programme is less advanced than osteonecrosis it could reach market first, Mr Bastianelli says. “The osteonecrosis trial is a 24-month follow-up study. We expect to complete recruitment in 15 months so the total duration of the study will be three and a half years.
“In non-union fractures it’s a shorter trial – 12 months – and recruitment should be completed within 18 months, though we’re increasing the number of centres in the study. Even though osteonecrosis is more advanced, non-union fractures should be completed before.”
He says the registration process would add around a year and a half after completion of the phase III trials. This suggest that Preob could be on the market as a therapy for fractures by 2017. Mr Bastianelli says that it will be priced more cheaply than the current standard treatment.
“Our approach will also increase the safety of the patient because we don’t need open surgery to implant the product – everything is done percutaneously. The advantages are no general anaesthesia and no hospitalisation, while the current standard is three or four hours' intervention, plus four or five days' hospitalisation. The cost of current treatment is very high.”
Bone Therapeutics is also developing another technology, an allogeneic cell product obtained from donors, although this is some way behind Preob.
Break a leg
Launch may be four years away, but it is never too early to start thinking about sales strategy. “For Europe we are building our own commercial production facility, so the idea is more to go alone. Osteonecrosis and non-union fractures are small indications – niche markets, with a limited number of surgeons involved.” With eight to 10 reps Bone Therapeutics could cover the whole of the UK.
Across the Atlantic, though, “we haven’t decided if we’ll go with a partner". Mr Bastianelli says Bone would consider either pharma or medtech companies, as long as they were interested in orthopaedics or rheumatology. He names Merck, Lilly, Amgen, UCB and Pfizer as examples. “In medtech, you have different categories of companies – those selling biomaterials, those selling prostheses, or those selling osteosynthesis materials. Of course some companies do all three, but some are more specialised. And they are all possible partners.”
Bone completed a funding round in January that Mr Bastianelli says will see it through to mid-2014; since its therapies will not yet have reached market, further cash will be needed, hence the importance of partnering.
The company is certainly confident in its technology, but potential collaborators might still be understandably reluctant to get involved in this area of medicine. “Cell therapy has been a little bit disappointing over the past years,” says Mr Bastianelli, “ but we think that Bone Therapeutics has the potential to change it.” Results of those phase III trials will show whether he is right.