Novadip Biosciences SA Announces Positive Results from Phase 1/2 Clinical Trial in Patients with Severe Bone Non-Union of the Lower Limb Following Trauma

Study results demonstrate NVD-003 has a favorable safety profile and convincing efficacy including rapid, durable bone healing in patients following failure of conventional treatment

Clinical data will inform development of the Company’s autologous and allogeneic therapeutics for critical size bone defects and common orthopedic conditions

First patient enrolled in Phase 1b/2a US/EU clinical trial in pediatric patients with congenital pseudarthrosis of the tibia

MONT SAINT-GUIBERT, Belgium–(BUSINESS WIRE)–#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 positive data from its Phase 1/2 clinical trial evaluating the safety and clinical activity of its investigational product, NVD-003, in patients with severe bone non-union (BNU) of the lower limb following trauma.

In this study, NVD-003, an autologous tissue engineered product generated from the patients’ own adipose stem cells, was applied in nine patients with bone non-union of the lower limb who had previously undergone several surgical procedures; in one case, a patient had undergone 14 previous procedures. The US Food and Drug Administration (FDA) defines a non-union as a fracture that is at least nine months old and has not shown any signs of healing for three consecutive months despite surgical intervention.

Highlights from the clinical study results include:

• No safety issues or implant associated complications from using the autologous bone tissue engineered product in patients with severe bone non-healing conditions were observed
  
• Two-year clinical and radiological follow up confirmed NVD-003 achieved stable and irreversible bone healing in patients with recalcitrant bone non-union in the lower limb following failure of conventional surgical and bone engraftment treatments
  
• Rapid bone formation was confirmed at three months post-implantation in all patients
  
• Clinical healing with weight bearing and walking at six months post-implantation
  
• 100% success for the NVD-003’s manufacturing capabilities from autologous adipose stem cells

The results demonstrate NVD-003’s ability to reverse severe bone deterioration and to achieve accelerated ossification. A total of eight patients (89%) presented clinical healing during the two years of follow-up post-GS. The median and mean time to clinical healing were six months and nine months, respectively. All patients achieved total weight bearing at six months and seven patients (78%) were walking normally at 2 years.

“This Phase 1 study has shown that NVD-003 represents a convincing step forward in the development of an optimized bone graft substitute in terms of applicability and outcome,” said Professor Torsten Gerich, MD, PhD, Chief of Trauma Service at the Centre Hospitalier de Luxembourg and principal investigator of the trial. “NVD-003 is a very promising product with the potential to outperform autologous bone harvesting and other less potent bone graft substitutes such as DBM, ceramics and growth factors and prevent their accompanying comorbidities. Further investigations in larger scale clinical studies will be needed to confirm these promising results.”

The study’s primary endpoint was safety of NVD‑003 in adult patients with recalcitrant lower limb non-union. Secondary endpoints included:

• healing efficacy of NVD‑003 by radiographic assessments (via X-ray at discharge, six weeks, three months, six months, 12 months, 24 months and via CT-scan at six months, 12 months, 24 months)
• healing efficacy of NVD‑003 by clinical assessments (weight bearing ability, pain at palpation)
• assessment of patient reported outcomes such as pain, quality of life and overall treatment effect (e.g., brief pain inventory, EuroQol-5D, OTE)
• assessment of local complication rate after graft implantation

“The results are fabulous. This is a very nice study introducing a method that has spectacular results in this very difficult to treat group of patients,” said Professor Gunnar Andersson, MD, PhD, former Professor and Chairman Emeritus at the Department of Orthopedic Surgery at Rush University Medical Center in Chicago and Chairman of Novadip’s Clinical Advisory Board. “It may well be that this treatment alternative can replace current first line treatments.”

Novadip is developing NVD-003, an autologous therapy derived from adipose stem cells as a potential single treatment to save limbs and restore mobility​ in patients with congenital pseudoarthrosis of the tibia (CPT), a rare pediatric bone condition.

Enrollment of First Patient in Phase 1b/2a US/EU Clinical Trial in Pediatric Patients with CPT

Novadip recently enrolled and treated the first patient in a Phase 1b/2a clinical trial to study NVD-003 in CPT patients between two and eight years of age in the US and EU. The trial will enroll a total of four patients.

The results of this study in patients with BNU will be used to inform the clinical and regulatory pathway for NVD-003 in patients with CPT.

“We will share the results of this BNU study with the US FDA as part of IND discussions to support registration for NVD-003 for the treatment of CPT,” said Denis Dufrane, MD, PhD, CEO and Co-founder of Novadip Biosciences.

NVD-003 has received orphan drug and rare pediatric disease designations from the US FDA. If NVD-003 is approved by the FDA, Novadip is eligible to receive a priority review voucher that could provide more than $100 million revenue.

The Company is also developing an 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.

“The results from the BNU study also support and de-risk the development of NVD-X3, which, as an allogeneic therapy, has a favorable cost profile that can allow broad distribution and expanded access for patients undergoing spinal fusion or suffering from recalcitrant non-healing fractures,” Dr. Dufrane added.

The risk of bone non-union following fracture is greater than 50% across all bones due to fracture severity, co-morbidities (diabetes, obesity, smoking and other conditions) and medication use¹. Additionally, more than 600,000 spine fusion procedures are performed annually. Together, these indications represent a peak sales opportunity for Novadip of $2.2 billion.

About Novadip Biosciences

Novadip Biosciences SA is a clinical stage company developing best in class tissue regeneration therapies to elevate the standard of care for critical size bone defects, common orthopedic conditions, and cancer. Its unique 3M³ tissue regeneration technology platform has the potential to generate an array of product candidates to address hard and soft tissue reconstruction for patients with limited or no treatment options. Co-Founded in 2013 by Denis Dufrane, MD, PhD, Novadip is headquartered in Mont Saint-Guibert, Belgium. For more information, visit https://www.novadip.com/.

¹ Zura et al. Epidemiology of Fracture Nonunion in 18 Human Bones. JAMA Surg. 2016;151(11):e162775. November 16, 2016

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