Second patient scheduled for implantation with NVD-003 in June 2023
Pivotal trial is expected to begin in the second half of 2024*
MONT SAINT-GUIBERT, Belgium – Novadip Biosciences SA (“Novadip” or “the Company”), a clinical-stage biopharmaceutical company developing a new class of regenerative tissue products to accelerate healing of large bone defects and injuries in a single treatment, today announced that the first patient has been implanted with its investigational tissue regeneration product, NVD-003, in its Phase 1b/2a clinical trial treating four patients with Congenital Pseudarthrosis of Tibia (CPT). Novadip is developing NVD-003, an autologous therapy derived from adipose stem cells (ASC), as a potential single treatment to save limbs and restore mobility in patients with CPT, a rare pediatric bone condition.
The patient was treated at the Saint-Luc University Hospital in Brussels by Dr. Pierre-Louis Docquier, EU principal investigator for this study. Dr. Docquier has considerable clinical experience using ASC-based therapies generated from Novadip’s 3M3 tissue regeneration platform in clinical and compassionate use settings.
“The potential for this approach to accelerate healthy bone formation, restore mobility and avoid the need for amputation in patients with CPT is tremendous,” said Professor Pierre-Louis Docquier, MD, PhD, a specialist in pediatric surgery at the University Hospital Saint-Luc in Brussels. “I think patients will benefit from this therapy as it does not require invasive iliac crest bone grafting which not only can be very painful for patients but is also associated with co-morbidities such as infection, bleeding and large scar formation.” And as already observed in previous compassionate uses in 4 children.
The Phase 1b/2a trial will enroll four CPT patients between two and eight years of age in the US and EU. The primary endpoint of the study is short-term safety of NVD-003 up to 12 months following grafting surgery (GS). Secondary endpoints include evaluation of long-term safety, tibial length, bone formation, bone remodeling and bone union at 3-, 6-, 12- and 24-month time intervals following GS.
“We are excited by the progress in patient recruitment and treatment to date and look forward to assessing clinical and patient outcomes at near- and longer-time intervals post implantation,” said Denis Dufrane, MD, PhD, CEO, and founder of Novadip Biosciences.
“With our growing research partnerships with the clinical investigators and their peer network in the EU and US, we are confident that we will be able to identify and enroll the CPT patients we will need for our planned pivotal trial, which we expect will begin in 2024,” added Judy Ashworth, MD, Novadip Biosciences’ Chief Medical Officer.
NVD-003 Provides Clinical Proof of Concept for Future Development Programs
The company previously announced positive data from a Phase 1/2 clinical trial evaluating the safety and clinical activity of NVD-003 in patients with severe bone non-union (BNU) of the lower limb following trauma. The results demonstrate NVD-003’s ability to reverse severe bone deterioration and to achieve accelerated ossification, and de-risk the development of the Company’s allogeneic “off-the-shelf” therapeutic, NVD-X3, to provide accelerated, durable bone union in common orthopedic conditions such as spinal fusion and non-healing fractures. Both investigational products are derived from Novadip’s 3M3 platform, a 3-dimensional, extracellular matrix that utilizes adipose-derived stem cells to deliver highly specific growth factors and miRNAs to mimic the physiology of natural tissue healing.
About CPT
Congenital pseudarthrosis of the tibia, or CPT, is a devastating diagnosis. Impacting patients in just .5-3.5 in 150,000[1],[2],[3] live births, the condition is very rare and debilitating. Treatment of CPT is difficult and once a fracture occurs, subsequent fractures are likely. Children with CPT can face impaired mobility, years of corrective surgeries to try to repair and stabilize the bone, it is not uncommon for patients to ultimately undergo amputation of the limb.
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 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. Visit www.novadip.com for more information.
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*A pivotal trial is typically a Phase 3 study which presents the data that the FDA uses to decide whether or not to approve a drug. A pivotal study will generally be well-controlled, randomized, of adequate size, and whenever possible, double-blind.
[1] Khan T, Bone Joint J. 2013, PMID: 23908415
[2] Heikkinen ES, Acta Orthop Scand. 1999, PMID: 10429605
[3] Hefti F, J Pediatr Orthop B. 2000, PMID: 10647103
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