Gosselies, Belgium, 17 October 2018, 6 pm CEST – BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the bone cell therapy company addressing high unmet medical needs in orthopaedics and bone diseases, today announces results of the first efficacy study of the Company’s enhanced viscosupplement, JTA-004, in patients with moderate symptomatic knee osteoarthritis, supporting future clinical development of the product.
JTA-004 is a patented, non-cellular viscosupplement product for the treatment of knee osteoarthritis (KOA), which Bone Therapeutics has been developing in parallel with its core cell therapy pipeline and addressing its mission of bringing innovative solutions to orthopaedic conditions.
The Company believes the favourable safety and efficacy profile of JTA-004 observed in this first efficacy study supports the move to registration studies and will begin dialogue with the regulatory authorities to determine next steps.
About the trial
The trial was a prospective, multicentre, randomised, double-blind, controlled study including three JTA-004 strengths and one reference product, hylan G-F 20, the global market leader in viscosupplements(1). The main objective of the study was to demonstrate the superiority of a single intra-articular JTA-004 injection to the reference product.
164 patients were randomly assigned to the reference group or one of the three JTA-004 groups. The primary endpoint of the study was the mean change in WOMAC® VA 3.1 pain subscale score (ranging between 0 and 100 mm) between baseline and 6 months after treatment.
The single intra-articular injection of JTA-004 was generally well tolerated. At six months, patients in the three JTA-004 groups showed an improvement in pain vs. baseline ranging from 23.6 mm to 25.9 mm, while patients in the reference group only showed a 14.3 mm improvement. Due to high variability in primary endpoint at six months, statistically significant differences between the individual JTA-004 groups and the reference group were not achieved.
Analysis of the results revealed that the three JTA-004 strengths had a similar efficacy. Therefore, a post hoc exploratory analysis was subsequently performed between the reference group and all pooled JTA-004 treated patients. The exploratory analysis showed a 26.1 mm improvement for the pooled JTA-004 group vs. 15.6 mm(2) for the reference group at month 6, demonstrating a statistically significant superiority of the pooled JTA-004 group compared to the leading viscosupplement on the market.
Professor Jean-Francois Kaux, Head of the department of Physical and Rehabilitation Medicine at University Hospital CHU, Liège, Belgium, commented: “Globally, millions of patients are suffering from knee osteoarthritis which is a leading cause of chronic pain and disability. The management of this condition represents a continuing challenge for clinicians and patients, who are seeking stronger and longer-lasting pain relief alternatives for this painful disease. The strong safety and efficacy profile demonstrated by JTA-004 in this clinical study, in comparison with the most scientifically validated reference product, suggests potential for it to become one of the preferred treatments for patients suffering from knee osteoarthritis. I look forward to the future development of this unique product.”
Osteoarthritis is a progressive disease of the joints and is the most prevalent joint disorder globally. It is characterized by joint pain, tenderness, limitation of movement and stiffness. Knee osteoarthritis, the most common form, affects approximately 250 million people worldwide(3). The prevalence of KOA is expected to increase at a rate of 8% per year in the coming years due to increasingly aging and obese population. The worldwide sales of viscosupplements, one of the commonly used treatments for KOA, had an estimated value of $2.1B in 2016(1).
Viscosupplements are injectable solutions containing hyaluronic acid (HA), a main component of knee joint’s synovial fluid and aim to provide added lubrication and protection to the cartilage of the arthritic joint. JTA-004, which consists of hyaluronic acid, an analgesic and anti-inflammatory agent and an enriched protein solution, showed distinct advantages in preclinical studies over other viscosupplements due to its anti-inflammatory and lubrication properties.
Thomas Lienard, Chief Executive Officer of Bone Therapeutics, added: “We’re delighted with the data from this first trial of JTA-004 in patients with moderate symptomatic knee osteoarthritis, which shows the potential of this enhanced viscosupplement. Based on the favourable efficacy and safety profile observed in this trial, JTA-004 can offer advantages over currently available viscosupplements on the market and we plan to continue the development of this promising product. JTA-004 is a promising addition to our existing pipeline of bone cell therapy clinical programmes, and highly complementary to our broader goal of bringing to market innovative, best-in-class solutions to orthopaedic conditions and bone diseases.”
Continued clinical and manufacturing progress
The positive JTA-004 data complement recent positive clinical and manufacturing developments at Bone Therapeutics which support the continued progress of its pipeline and the increased focus on preparing for commercialisation. Notable recent progress includes:
·In September, Bone Therapeutics announced a positive final readout in the Phase I/IIA delayed-union study of allogeneic bone cell therapy product, ALLOB, adding to a growing body of clinical efficacy and safety data.
·Also in September, the Company presented preclinical in vitro and in vivo results at the 26th Annual Meeting of the European Orthopaedic Research Society (EORS) in which the scientific community acknowledged the potent bone-forming properties of its allogeneic platform.
·Bone Therapeutics also recently announced an optimized production process for ALLOB which allows the Company to achieve a consistent, easy to scale up and economical (around 100,000 doses per donor) manufacturing process and to offer an easy to use, cryopreserved product, all critical parameters required for future commercialisation. The Company plans to implement this optimized process for all future clinical development programmes. Bone Therapeutics received positive feedback on the quality control programme and non-clinical strategy for ALLOB from a Regulatory Agency for the optimization of the manufacturing process.
·The Company’s focus over the next few years is on progressing the clinical development of ALLOB in delayed union fractures and lumbar spinal fusion, subsequently leading to Phase III clinical trials in EU and USA.
·In addition, Bone Therapeutics expects to present the conclusions of the interim analysis after a one-year follow-up of the Phase III study of PREOB, its autologous cell therapy product, in osteonecrosis of the hip in Q4 2018.
(1) Viscosupplementation: Global Analysis and Market Forecasts, April 2017, Global Data
(2) The difference in the mean improvement of the reference group at Month 6 between the two analyses was a consequence of the statistical adjustments for both sample size and sample variation.
(3) Vos et al., A systematic analysis for the Global Burden of Disease Study 2010. Lancet 2012; 380:2163-96
About Bone Therapeutics
Bone Therapeutics is a leading cell therapy company addressing high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy products in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation.
Bone Therapeutics’ technology is based on a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery.
The Company’s primary clinical focus is ALLOB, an allogeneic “off-the-shelf” cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. In addition, the Company also has an autologous bone cell therapy product, PREOB, obtained from patient’s own bone marrow and currently in Phase III development for osteonecrosis of the hip, and JTA-004, a viscosupplement in development for the treatment of knee osteoarthritis.
Bone Therapeutics’ cell therapy products are manufactured to the highest GMP standards and are protected by a rich IP estate covering nine patent families. Further information is available at: www.bonetherapeutics.com.
About Knee Osteoarthritis
Osteoarthritis (OA), also known as degenerative joint disease, is the most common chronic joint condition in which the protective cartilage in the joints progressively break down resulting in joint pain, swelling, stiffness and limited range of motion. The knee is one of the joints that are mostly affected by osteoarthritis, with an estimated 250M cases worldwide.
The prevalence of knee osteoarthritis (KOA) is expected to increase in the coming years due to increasingly aging and obese population. Currently, there is no cure for KOA and treatments focus on relieving and controlling pain and symptoms, preventing disease progression, minimizing disability, and improving quality of life. Most drugs prescribed to KOA patients are topical or oral analgesics and anti-inflammatory drugs. Ultimately, severe KOA lead to highly invasive surgical interventions such as total knee replacement.
About WOMAC® Index
The Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC®) is a widely used, proprietary set of standardized questionnaires used by health professionals to evaluate the condition of patients with osteoarthritis of the knee and hip, including pain, stiffness, and physical functioning of the joints. It can be self-administered and was developed at Western Ontario and McMaster Universities in 1982.
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