The randomised, placebo controlled double blind study follows promising preliminary Phase IIa data for NovaBiotics’ orphan status drug candidate. The current trial aims to assess the benefits of oral Lynovex®, the hard gel capsule form of the drug, as adjunct therapy to standard of care interventions in CF infectious exacerbations. The study will involve 120 adult patients in Europe and the US and will run throughout the winter exacerbation season.
Cystic Fibrosis is a life-limiting, inherited disease caused by a defective gene which results in lungs, the digestive system and other tissues becoming congested with thick mucus. The presence of the mucus in the airways of CF patients leads to numerous health issues, including chronic and recurrent infections of the lungs and consequent inflammation, reduction of lung function and breathing difficulties. Lynovex® in oral form is the first multi-active therapy (with antibacterial, mucolytic, anti-biofilm and antibiotic potentiating properties) developed to alleviate the symptoms and impact on lung function of infectious exacerbations in CF.
Dr Deborah O’Neil, CEO of NovaBiotics, said “Dosing the first patients with Lynovex® in the CARE-CF-1 study is a significant milestone for the business. This trial marks an important step in a product candidate which has shown significant potential to mitigate the effects of, and the symptoms experienced during, CF infectious exacerbations. These episodes can have a permanent impact on the respiratory function of CF patients and, with Lynovex® in tablet and inhaled form, we are part of the fight to lift the life-limiting restrictions that CF places on people with the condition; we are rigorously focused on harnessing our science and developing drugs so that people with CF live longer, better quality lives”
The Lynovex® capsules will be taken for 14 days alongside standard of care medication for exacerbations. The therapy is targeted at patients with exacerbations with Gram-negative infection, FEV1 >30% predicted and weight >40kg. Clinical outcomes and microbiology will be assessed to determine the optional dose and dose regimen of Lynovex® and inform the next pivotal phase of pre-registration studies.
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NovaBiotics Limited (“NovaBiotics” or the “Company”) is a private, UK-based anti-infectives focused biopharmaceutical company. The Company has an advanced product portfolio of antimicrobial assets targeting large and important markets with significant unmet clinical needs. These include Lynovex, a novel multi-active orphan therapy for cystic fibrosis, Novexatin for the treatment of onychomycosis, a potential blockbuster outlicenced to Taro Pharmaceuticals, and Novamycin, a novel rapid acting antifungal for aspergillosis and other life-threatening yeast and mould infections. The Company is also developing earlier stage compounds from its proprietary antimicrobial peptide rational drug design platform.
Lynovex is a novel, highly differentiated therapy for the treatment of CF-associated lung disease. The CF market is estimated at $6 billion and there is a clear need for effective treatment of the symptoms of CF-associated lung disease, the most critical clinical manifestation of this orphan disease. Lynovex addresses the key treatment challenges through its unique triple mode of action: Antibacterial - addressing both current (e.g. Pseudomonas) and emerging/antibiotic insensitive (e.g. NTM and Burkholderia Spp) pathogen; Mucolytic - clear superiority to approved mucolytic therapeutics; Antibiofilm - addressing the key challenge of mucus build up within the airways of CF patients. Efficacy has been demonstrated through in vitro study, ex vivo (sputum) and a clinical phase IIa study. In addition, the Company has demonstrated that Lynovex dramatically enhances the effect of existing anti-infective drugs. To that end, Lynovex is intended for use alongside existing standard of care therapies (SOCT) and to treat all CF disease genotypes. It is not a mutation-specific, CFTR-targeting intervention. NovaBiotics is developing two different formulations of the same active ingredient: Oral for CF exacerbations; now in pre-registration CARE-CF studies in the US and EU 2016 and Inhaled for chronic CF therapy; anticipated to commence a clinical POC study 2017.
Derived from the same aminothiol platform as Lynovex®, Nylexa™ is an antibiotic potentiator/resistance-breaking agent being developed as a parenterally administered adjunct to specific antibiotic classes; extending the utility and scope of the existing antibiotic armoury in multi drug resistant (MDR) and extreme drug resistant (XDR) bacterial infections. Resistance potentially breakers have a faster route to clinic than novel chemical antimicrobial entities (NCAE) and as a repurposed aminothiol, this is expedited further in the case of Nylexa™. In vitro and in vivo experiments have already demonstrated Nylexa’s efficacy in a number of infection models.
This topical therapy is a step-change in the treatment of fungal nail infection (onychomycosis), a $6 billion market and affecting 12% of the global population. Based on the results of studies to date, Novexatin is the first topical agent to deliver high cure rates and rapid cosmetic benefits with only 4 weeks of treatment vs. 48 weeks for the existing topical treatments (including Jublia and Kerydin). Novexatin’s placebo-like safety profile has enabled dosing of all nails, which the Company believes is important in delivering a long lasting clinical benefit. A large phase IIb study is underway and results are expected in 2017. Novexatin is being developed with NovaBiotics’ licensing and co-development partner, Taro pharmaceuticals and is currently being further investigated in a Taro-led large PhIIb clinical study.
Closely related to Novexatin in molecular architecture (and therefore de-risked) but with a distinct spectrum of activity, Novamycin is a first-in-class antifungal peptide being developed to target life threatening yeast and mould infections; predominantly invasive pulmonary aspergillosis, but also candidaemia, oral pharyngeal candidiasis, vulvovaginal candidiasis, cryptococcosis, respiratory fungal infections associated with CF and murcomycoses. The Novamycin invasive pulmonary aspergillosis programme is the most advanced with compelling in vitro and in vivo data to support Novamycin’s application in this poorly served, life-threating condition.
Proprietary Antimicrobial Peptide Rational Drug Design Platform
The Company’s proprietary drug discovery platform (from which Novexatin and Novamycin have been derived) is based upon re-engineering endogenous antimicrobial peptides (“AMP”); naturally occurring antifungal and antibacterial agents that form the body’s first line of defence against infection. This rational drug design platform enables the development of smaller, simpler and therapeutically viable molecules, and is validated by NovaBiotics’ Novexatin and Novamycin drug candidates. The Company’s preclinical antibacterial candidate, Novarifyn®, is the next compound derived from the platform to be developed and is due to enter clinical development in 2018.