Bone Therapeutics started as a spin-off from the ULB in 2006. Prof. Valérie Gangji and Dr. Enrico Bastianelli founded the company to develop cell therapies for application in the field of fracture prevention and fracture repair. Bone Therapeutics takes regular cell therapy one step further by differentiating stem cells derived from bone marrow into bone-forming cells. They strive for a minimally invasive approach, which can be considered as a paradigm shift in orthopaedics. Bone Therapeutics is developing two products for different indications currently being tested in 6 clinical trials. These indications include non-union and delayed union fractures, spinal fusion, osteoporosis and osteonecrosis. The first product, PREOB®, consists of autologous cells and the second product ALLOB® is produced with allogeneic cells.
Avoiding surgery
Bone Therapeutics’ uses stem cells that have been differentiated into osteoblasts. These are then administered with a needle through a small incision in the skin to the affected area in the bone, where they induce bone formation. Wim Goemaere, CFO of Bone Therapeutics, explains: “In the case of osteonecrosis of the hip, for example, the bone tissue inside the femoral head dies. This is a very painful condition that almost always results in a collapse of the hip after about two years. Usually, the only option to give these patients their mobility back is a full hip prosthesis. Osteonecrosis affects mainly young men between 30 and 50 years old. For people in these age groups a hip replacement would have a serious impact on their quality of life, as the materials in these prostheses may need to be replaced within 10 to 15 years.
A third hip replacement is nearly impossible and would leave these patients severely handicapped for the remainder of their lives. A phase III clinical trial with our product PREOB® is currently ongoing for this condition. Treatment of these patients with PREOB® through a single percutaneous injection in the femoral head aims to slow down the progression of the disease and eventually help to avoid a hip replacement.”
Recently, Bone Therapeutics began looking at the treatment of spinal fusion. “People suffering from conditions such as spinal disc herniation require surgery to remove the injured intervertebral disc, after which the two vertebrae are fused together. Over one million such procedures are performed each year in the US and in Europe (combined)” continues Goemaere. “Most often, artificial bone substitutes are placed between the vertebrae in the hope that the body’s natural bone growth process will fuse the adjoining vertebrae. This fusion process can take up to two years and in 25% of the cases the fusion fails, leaving the patient in a large amount of pain. We are now performing clinical trials to investigate whether the administration of ALLOB®-cells can accelerate and increase the success rate of the healing process. In addition, a new clinical trial was started for what we call a rescue spinal fusion. Here, we administer ALLOB® non-invasively to patients where the vertebrae have not fused 15 months after the initial surgery.”
Medtronic has a similar product on the market, Infuse, based on a growth factor for bones, BMP-2. “Indeed,” confirms Goemaere, “this used to be a very popular product in the US with a turnover higher than 1 billion dollars. However, Infuse was shown to have multiple side effects, making patients and doctors reluctant to use the product. As a consequence, the turnover of Infuse dropped by two thirds. Thus far we have seen no side effects with our products. Currently we are awaiting efficacy results for both trials. Efficacy will be measured at 12 months post intervention.”
“Another interesting trial, where the cells are not administered locally but intravenously, is also ongoing to treat severe cases of osteoporosis, in patients who have not responded to any of the existing treatments. The cells are labeled radioactively so we can follow their transport through the body for 72 hours. We see that they migrate to the bones, the hips and the spine. It is too early to comment on clinical benefits for the patients treated so far in this trial.”
The ‘off-the-shelf’ benefits of allogeneic cells
Wim Goemaere explains: “PREOB® cells are autologous and as such are produced from the patient’s own bone marrow. Here production is still a 1 to 1 process. ALLOB®-cells, which are allogeneic, offer considerable benefits in this respect.”
With one bone marrow donation from a healthy person, we can make up to 100 doses to treat patients. This allows us to achieve a more ‘off-the-shelf’-product model, closer to what pharmaceutical companies have today.
With regards to whether these cells could possibly be rejected by patients, Goemaere answers: “The cells do not trigger rejection mechanisms in patients. In addition, patients treated with ALLOB® do not have to take immunosuppressants. ALLOB® could also be useful for patients that are unable to deliver stem cells of sufficient quality.”
Marketing osteoblasts
“We have been lucky to have a loyal group of shareholders who supported Bone Therapeutics from the beginning. The Walloon government has also been funding the company for many years. This support has allowed us to work autonomously,” explains Goemaere. Bone Therapeutics launched an IPO early in 2015. “The IPO allowed us to raise 37 million euro permitting us to plan our financial outlook for about 3 years. It also put us in the spotlight, which provided our staff with extra motivation. The IPO also created extra visibility for the company with possible investors. This is important in the light of establishing partnerships for instance for distribution of our products within the EU. We are also planning to start phase III clinical trials in the US. This will help to increase our visibility overseas and should allow us to attract partners over there as well. Bone Therapeutics’ ambition is to become a leader in cell therapy for orthopaedic applications.”
Wim Goemaere’s career initially began in the petrochemistry. Twenty years ago, he changed fields and he began working in biotech as CFO. First with VIB, where he contributed to the development of the institute. In early 2008, he joined Devgen. When Devgen was sold to Syngenta at the beginning of 2013, he felt challenged by the opportunities of a small biotech company and moved to Bone Therapeutics, where he is currently the CFO. In this position he was instrumental in carrying out the IPO that took place earlier this year.
