IND approval from the FDA for regenerative bone product NVD-003

Mar 4, 2021 | Press Releases

Novadip Biosciences receives IND approval from the FDA for regenerative bone product NVD-003

Authorization to start clinical trial in the US granted for rare paediatric bone disease

Mont-Saint-Guibert, Mar 4, 2021 (GlobeNewswire) – Novadip Biosciences (“Novadip”), a clinical stage company developing treatments to regenerate impaired tissues in patients with significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s Investigational New Drug (IND) application for its autologous cell therapy product NVD 003 for the treatment of Congenital Pseudarthrosis of the Tibia (“CPT”). CPT is an orphan disease, that primarily affects children with devastating long-term functional disabilities.

IND approval has been granted by the FDA allowing Novadip to conduct a pilot study with NVD-003 in the US. The trial will investigate the potential of the Company’s cell-based osteogenic product in young children, between two and eight years of age, suffering from CPT.

NVD-003 is a novel autologous cell-based osteogenic (bone healing) product that has been generated from Novadip’s proprietary tissue regeneration platform. Made from adipose stem cells, NVD-003 has a 3-dimensional structure and works by promoting bone formation. Novadip previously reported positive interim results from a Phase I/IIa study in adults with non-healing fracture of the lower limb. The results showed a clinically and radiologically meaningful improvement in patients with recurrent bone non-union after severe trauma and with no therapeutic alternatives, highlighting the ability of NVD-003 to restore natural bone healing processes. In the frame of compassionate use, young children suffering from CPT have also been treated with this innovative product and it was shown that the need for amputation could be avoided and normal quality of life (e.g. walking, cycling and horse riding) could be significantly improved.

The NVD-003 autologous bone program was granted Orphan Drug designation and Rare Paediatric Disease designation by the US Food and Drug Administration (FDA) in 2020. Following both designations and the successful IND approval, Novadip will be eligible to obtain a priority review voucher when the product reaches the market.

Dr. Denis Dufrane (MD, PhD), Chief Executive Officer and Founder, said: “Receiving IND approval for NVD-003 is a major milestone for Novadip and we are excited by the potential impact this novel therapy could have on patients with few alternative treatment options. The results that we previously reported demonstrate that Novadip’s technology is able to stimulate bone formation and we believe that NVD-003 has the potential to become a new standard of care for people that require critical size bone defect reconstruction. In addition, this data provides further validation of our proprietary platform, supporting the future success of our next generation off-the-shelf products.”

Notes to editors

About Novadip Biosciences

Novadip Biosciences is a clinical stage biopharmaceutical company leveraging its proprietary tissue regeneration technology platform 3M³ to generate multiple product candidates to address hard and soft tissue reconstruction for patients who have limited or no treatment options. The 3M3 platform involves use of 3-dimensional extracellular matrix and adipose-derived stem cells to deliver highly specific growth factors and miRNAs to mimic the physiology of natural healing to create a range of products that address specific challenges in tissue regeneration. Novadip’s initial focus is on reconstruction of critical size bone defects. The company is also applying its 3M3 platform to develop truly novel off-the-shelf/allogeneic therapies addressing more prevalent tissue defects and miRNA/exosome products for broader indications.

About NVD-003

NVD-003, Novadip’s lead product candidate, is a clinical-stage investigational cell-based therapy and a new paradigm in regenerative medicine. Using its proprietary 3M³ technology, stem cells from the patient obtained from only a few millilitres of fatty tissue are cultured in vitro to become a biomaterial consisting of bone forming cells embedded in their self-secreted extracellular matrix together with added hydroxyapatite particles, a mineral naturally present in bone to confer initial strength. For the physician, the product is in the form of a mouldable putty in quantities large enough to fill small as well as large bone defects (>20cm³) using classical or minimally invasive surgery techniques without further complexities. Upon implantation, the bone-forming cells protected in their self-secreted matrix from the harsh pathological environment in the bone defect continue to mature and progressively transform the putty into normal, healthy bone. NVD-003 was granted Orphan Drug designation and Rare Paediatric Disease designation by the US Food and Drug Administration (FDA) in December 2020.

About CPT

Congenital Pseudarthrosis of the Tibia (CPT) is a shin bone fracture that fails to heal properly on its own that is present at birth or manifests in early childhood when starting to walk. The underlying cause of CPT is not completely understood but it leads to abnormal structure of the bone tissue in the tibia, and sometimes fibula, combined with abnormal vascularization of the affected tissue. The natural history of the disease is extremely unfavourable. Despite various medical techniques to restore bone union and vasculature, the final prognosis of CPT remains poor and associated with a significant risk of amputation.

For further information:
Novadip Biosciences
Denis Dufrane, Chief Executive Officer
+32 (10) 779 220

For media enquiries:
Consilium Strategic Communications
Chris Gardner, Matthew Neal, Angela Gray
+44 (0) 20 3709 5700