Paradigm submits first investigational new drug application with US FDA

Medical company Paradigm Biopharmaceuticals (ASX: PAR) has set the stage for a speedy entry into the US after filing its first investigational new drug (IND) submission to the US FDA for an “expanded access program”, that could see as many as 10 former NFL players receiving an experimental treatment for osteoarthritis.

In addition to the US, Paradigm says it will be filing a number of submissions to global regulatory authorities before year-end.

The company provided a detailed list of future activities including:

• The filing of an Expanded Access Program (EAP) for 10 patients with the US FDA Q3 CY 2019,
• Filing an initial submission with the TGA for Provisional Approval Application of Zilosul (iPPS) for treatment of osteoarthritis,
• Undergo pre-IND meeting with US FDA for an orphan drug Indication and plan a phase 2/3 clinical trial,
• Undergo a pre-IND meeting with US FDA for an osteoarthritis phase 3 clinical trial.

The first US application announced this morning means that Paradigm could obtain access to 10 US residents, who will be treated with Zilosul – otherwise known as injectable iPPS – an antithrombotic agent that acts as a first-line therapeutic treatment of osteoarthritis and other diseases affecting the musculoskeletal system.

If accepted, one of the first beneficiaries is likely to be retired NFL players who have developed early-onset osteoarthritis.

Also, assuming the IND is cleared by the FDA within 30 days, treatment of retired NFL players could occur in September 2019, according to Paradigm.

Tackling osteoarthritis

Osteoarthritis is a disabling disease leading to chronic pain, disability and a decreased quality of life. It is the most common joint disorder in the developed world, with the lifetime risk of developing symptomatic knee osteoarthritis before 85 years of age estimated to be around 44.7%, according to a US study.

Meanwhile, the FDA confirms that osteoarthritis can be a serious disease with an unmet medical need for therapies that modify the underlying pathophysiology of the disease and potentially change its natural course to prevent long-term disability.

Scientific research shows that sports contribute to 7 million injuries in the US and reports have suggested that the development of osteoarthritis may be more prevalent and occur prematurely in individuals who regularly participate in sports.

Currently, around 31 million people in the US suffer from osteoarthritis, thereby making the therapeutic market a multi-billion-dollar opportunity.

By applying for an expanded access status, Paradigm hopes to gain clearance to treat patients with an investigational drug that has a chance of mitigating serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials.

Positive results

Paradigm’s successful Phase 2b clinical trial indicated that the number of subjects receiving iPPS treatment had a clinically meaningful reduction in the grade of their Bone Marrow Edema Lesions (BML) compared to a placebo. Specifically, the group that received iPPS injections experienced reductions that were “statistically significant” over placebo at 50% versus 27.3%.

Given the existing unmet clinical need in treating osteoarthritis in tandem with Paradigm’s positive phase 2b data, US physicians are requesting access to iPPS where existing treatment methods are ineffective.

Furthermore, Paradigm plans to further its understanding of the potential utility of Zilosul for the treatment of BML-associated pain and joint stiffness in a small group of knee osteoarthritis patients while it prepares applications for a larger phase 3 clinical trial.

In response to this morning’s news, Paradigm shares ticked up almost 5% to $1.43.