Orphan Drug Designation granted to Neem Biotech’s lead drug candidate for the treatment of Pseudomonas aeruginosa lung infections in CF patients

Neem Biotech’s lead candidate NX-AS-401 has recently received Orphan Drug Designation from the United States FDA for treatment of Pseudomonas aeruginosa infections in patients with cystic fibrosis. This is a significant boost for the ongoing development of Neem’s flagship candidate drug substance. The outcome of an application for Orphan Drug Designation from the European Medicine Agency for this candidate is pending.

Taken together, obtaining Orphan Drug Designation status across both of these territories will mean potential access for a large proportion of the world’s cystic fibrosis patients to a novel approach to managing a common respiratory complication in cystic fibrosis.

Neem’s candidate is an alternative to conventional antibiotics and is compatible for use as a co-treatment with antibiotics that are currently used extensively to treat Pseudomonal lung infections in cystic fibrosis. NX-AS-401 works as an antimicrobial resistance breaker in Pseudomonas aeruginosa-caused chronic lung infection by virtue of its Quorum Sensing Inhibition capabilities and by disrupting enzyme function.

Neem is working in partnership with the German Mukoviszidose Institut, a subsidiary of the German Cystic Fibrosis Association. This partnership is aimed at developing and trialing a novel and effective alternative intervention to current standard treatment protocols. In particular, this intervention under development aims to reduce the number of Pseudomonas aeruginosa exacerbations suffered by patients with cystic fibrosis and hence to enhance the quality of life for these patients and their families. The fundamental research which led to this development was performed under the guidance of Prof Michael Givskov and was funded by the Mukoviszidose Institut as part of their support for research into the development of treatment options for patients with cystic fibrosis.

The FDA assigns Orphan Drug Designation to products that demonstrate promise as effective and safe agents for diagnosis and/ or treatment of rare diseases or conditions. It does this as a means of encouraging clinical development and marketing of safe and effective treatment, diagnosis or prevention measures for rare diseases and conditions that would otherwise not be financially feasible within existing large pharmaceutical drug development frameworks.

Criteria for classification as a rare disease is based on numbers of affected patients and varies across territories. Diseases or conditions where there are fewer than 50 000 people affected in the United Kingdom, fewer than 5 in 10 000 people affected in Europe and fewer than 200 000 people with the diagnosis in the USA are eligible for rare disease status.

Cystic fibrosis meets these criteria across all regions. It is a rare and life limiting progressive genetic disease. The number of patients with cystic fibrosis affected in Europe approximates 36 000, with estimates ranging from 1 in 8000 to 1 in 10 000 individuals. There are 33 000 patients with cystic fibrosis in the USA.

Cystic fibrosis is caused by a genetic defect in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. This gene controls the movement of salt and water into and out of cells, resulting in a buildup of mucous in the lungs, digestive system and other endocrine systems. In turn, this manifests as difficulty breathing and increased susceptibility to respiratory infections and other digestive difficulties. The chronic lung infection that is caused by the multidrug resistant Pseudomonas aeruginosa bacteria is the largest cause of death in patients with cystic fibrosis.

A first–in-man clinical trial is envisaged to start in early 2017 to evaluate the efficacy of NX-AS-401 as a co-treatment with existing antibiotics used against Pseudomonas aeruginosa-caused chronic lung infections in adult cystic fibrosis patients.

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