Date posted: 16-Sep-2014
Category: Press Releases
NovaBiotics Receives Orphan Drug Status from the US Food and Drug Administration (FDA) for Lynovex® for the Treatment of Cystic Fibrosis
Orphan drug designation coincides with dosing of first patients in PhIIa study
Aberdeen, UK – 16 September 2014 – NovaBiotics Ltd, the Aberdeen-based clinical-stage biotechnology company (“NovaBiotics” or the “Company”), today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for Lynovex® (NM001), the Company’s first-in-class therapeutic candidate for the treatment of Cystic Fibrosis (CF).
CF is a chronic, life-limiting disease that affects the lungs, digestive tract and other organ systems. It is estimated that there are 30,000 children and adults with CF in the US and upwards of 70,000 people affected worldwide. It is believed that Lynovex® has unique multi-functionality; combined properties of breaking down the excessive mucus produced in the airways of CF patients, killing the bacteria responsible for the recurrent respiratory infections in CF and disrupting/preventing the biofilms which these bacteria typically form in the CF airway. By functioning in this way, Lynovex® could improve lung function and halt or prevent long term damage or degeneration of respiratory tissues in CF patients.
Lynovex® is being developed by NovaBiotics for CF as an oral (tablet) form for acute exacerbations and in inhaled (dry powder) form for chronic use and maintenance. Lynovex® is intended for application alongside existing CF treatments and potentiates their antimicrobial effects. A Phase IIa clinical trial for the oral form of Lynovex® is already underway and the first patients will be dosed this week. PhIIb studies of the oral form of Lynovex in the UK and Europe are expected to commence in 2015. Clinical development of the inhaled form is also anticipated to get underway in H2 of 2015.
Dr Deborah O’Neil, CEO of NovaBiotics, said: “The orphan drug designation for Lynovex® in the US is a major regulatory milestone for NovaBiotics and highlights the unmet need for effective and safe treatments for CF patients. Dosing of the first patients in the Aberdeen Phase IIa clinical trial is also a significant clinical milestone in Lynovex’s clinical development.”
She added, “If the encouraging data we have achieved so far continues to translate in clinical use, we believe Lynovex® will offer a breakthrough in the treatment of CF. This is a candidate therapy with the potential to significantly improve the health and quality of life for those affected by CF. Importantly, and unlike other treatments that are specific to particular CF mutations, Lynovex® is intended for use by the entire CF patient population, not just sub-groups.”
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Notes to Editors
About Orphan Drug Designation Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the US. The first NDA applicant to receive FDA approval for a particular active ingredient to treat a particular disease with FDA orphan drug designation is entitled to a seven-year exclusive marketing period in the US for that product and indication. Lynovex® has been designated as an orphan drug in Europe since December 2011.
Cystic Fibrosis Cystic fibrosis is an inherited, chronic, debilitating, life-limiting disease that affects around 70,000 individuals worldwide. It is caused by a mutation in a single gene, but the effects of this mutation impact and lead to symptoms in multiple internal organs with the lungs and the digestive system typically being the most significant tissues and where treatment is focused. The overproduction of mucus by the cells lining the airways and digestive tract lead to impaired lung function, recurrent or chronic respiratory infections and inflammation or degenerative lung damage and an impaired ability to digest food.
The respiratory infections associated with CF have a deleterious impact on lung function, and the presence of particular bacterial or fungal pathogens can also rule out transplantation as an intervention option for CF patients. NovaBiotics’ CF programme is therefore focused on controlling and eradicating the entire spectrum of bacteria (and fungi) that infect or colonise the lung of CF patients. Doing so is paramount in maintaining and improving lung health, function and quality of life overall for CF patients. Antibiotic resistance to conventional or existing therapies is a major issue in CF because of the length of time and doses to which patients are exposed to these agents over the course of their life. Furthermore, a number of the emerging pathogens that are becoming more commonly associated with CF are inherently insensitive to conventional antibiotic therapies. Lynovex® is a potential solution to these increasing challenges in CF management and is a result of a complete re-think in how best to tackle the infectious component of this chronic, debilitating disease in the long-term.
About Lynovex® Lynovex® (NM001) is a unique molecule with a dual antibacterial-mucoactive mode of action which aggressively tackles both of the major clinical features responsible for progressive lung disease in CF. Lynovex also kills bacteria very rapidly and is active against drug resistant bacteria as well as bacteria that are in biofilm form (the way in which they grow in the CF lung to avoid clearance by the immune system and/or antibiotics) and importantly, reverses antibiotic resistance in bacteria to conventional antibiotics when used alongside these drugs.
About NovaBiotics NovaBiotics Ltd is a clinical-stage biotechnology company focused on the design and development of first-in-class anti-infectives for difficult-to-treat, medically unmet diseases.
A leading innovator in the anti-infectives space, the Company’s robust pipeline includes clinical stage antimicrobials that have been derived from NovaBiotics’ proprietary anti-infective peptide platform and a “mini biologic” antibacterial-mucolytic compound (Lynovex®). NovaBiotics lead product, NP213 (Novexatin®) is being developed as a topical treatment for fungal nail infections (onychomycosis), a condition that affects more than 10% of the global population.
NovaBiotics is also developing NM001 (Lynovex®), an orphan drug treatment for cystic fibrosis. NM001 has a unique dual antibacterial-mucoactive mode of action that kills bacteria, disables biofilms and disrupts excess mucous produced in the airways of patients with cystic fibrosis (and other chronic respiratory diseases). NM001 is being evaluated in Phase IIa clinical trials as an oral treatment for cystic fibrosis and is also being formulated for inhalation to enable longer term, more targeted delivery of the drug.
An exciting pipeline of preclinical stage drug candidates is already demonstrating significant promise. These include NP339 (Novamycin®), an antifungal peptide agent against Candida spp and other clinically relevant yeasts and moulds as well as NP432 (Novarifyn®), a peptide antibacterial for difficult to treat multi-drug resistant Gram-negative and Gram-positive infections. Further information is available at www.novabiotics.co.uk.
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