Phase IIa clinical study results for NovaBiotics’ Lynovex® presented at the 38th Annual European Cystic Fibrosis Symposium
Clinical data supports the application of Lynovex® as a novel treatment for the infectious exacerbations associated with cystic fibrosis
Aberdeen, UK – [12th June 2015] – NovaBiotics Ltd, the Aberdeen-based specialty biotechnology company (“NovaBiotics” or the “Company”), today announces the presentation of data from a Phase II a clinical trial of its orphan candidate cystic fibrosis (CF) treatment, Lynovex® (NM001) at ECFS.
Lynovex® (NM001) is being developed by NovaBiotics in oral (capsule) form as the first therapeutic intervention specifically intended to address the infectious exacerbations associated with CF. In parallel, NovaBiotics are developing an inhaled form of Lynovex® for chronic use in CF. In both oral and inhaled form, Lynovex® is intended as an adjunct to standard of care antibiotic therapy.
The data presented at this years’ ECFS are from a 10 patient Phase IIa clinical study conducted by Aberdeen University’s Professor Graham Devereux and his clinical team at Aberdeen Royal Infirmary. In this study, stable adult CF patients from the Aberdeen CF centre cohort received a range of (increasing) Lynovex® doses over the course of 5 weeks alongside their standard of care therapy regimens. The primary objectives of the study were to assess whether the active component of Lynovex® (cysteamine) was safe and well tolerated by the patients, whether it was absorbed into the bloodstream and from there, reached the lung tissue. The study also monitored any initial evidence of clinical benefit from Lynovex®.
The optimal dose at which the drug was safe and well tolerated and absorbed into the blood stream was determined from the trial, as was confirmation that Lynovex® entered the bronchial secretions of the CF patients. Furthermore, the data generated by Professor Devereux suggests that Lynovex® had positive impacts on a number of clinical parameters measured (sputum bacterial load, sputum viscosity, etc.).
The acute, infectious exacerbations that CF patients can suffer multiple times each year have an irreversible, deleterious impact on lung function and quality of life for CF patients. There is a clear, unmet medical and market need for treatments that are designed specifically to not only target the causative bacteria, but also to alleviate the symptoms which patients suffer during these episodes. In laboratory tests Lynovex® (NM001) has already demonstrated unique multiple properties ideally suited to minimising the impact of exacerbation events. The clinical data generated by the study conducted by Professor Devereux appear to confirm these early findings and go further to support Lynovex’s utility as a CF therapeutic in acute exacerbations. The next/final phase of clinical development of this first-in-class CF exacerbation therapy is anticipated to commence within the next 12 months.
Professor Devereux said: “These results are very promising. We were pleasantly surprised because some of the patients improved considerably. The CF team and the CF patients in Aberdeen are very keen to contribute to the development of Lynovex®. The experiences gained by actually giving the drug to people with CF will prove invaluable in the development of Lynovex®”.
Dr Deborah O’Neil, CEO of NovaBiotics, said: “This encouraging data is a key step in the development of Lynovex® as a much needed therapy in CF exacerbations. At the optimal dose, the drug was not only well tolerated and absorbed by the patients, but reached the lungs in the concentration ranges at which we know this molecules’ potent mucolytic and antibacterial effects can be elicited.” She adds, “This trial was only the first study of Lynovex® in adult CF patients, but a very carefully designed one by Prof Devereux that generated meaningful, and in some cases striking findings on which we are already building the final clinical trials required for Lynovex® to be marketed for CF in Europe, the US and elsewhere. From my perspective, this data goes further to support the potential breakthrough that Lynovex® may offer in CF”.
About cystic fibrosis
CF is a chronic, life-limiting disease that affects the lungs, digestive tract and other organ systems. It is an inherited, chronic, debilitating, life-limiting disease that affects around 70,000 individuals worldwide and is caused by any one of >1,500 mutations in a single gene (the CFTR gene). 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.
Lynovex® (NM001) has unique multi-modality as a potential novel CF therapy. It is antibacterial (rapidly bactericidal), an antibiofilm agent, mucoactive and it synergises with standard of care antibiotics to potentiate their effects on the bacterial pathogens associated with CF. With these properties, Lynovex® (NM001) has the potential to tackle the major clinical features responsible for progressive lung disease in CF; breaking down excessive, dehydrated mucus and killing the bacteria that infect and colonise the CF airways. In this respect, Lynovex® is not only active against drug resistant bacteria in CF but reverses antibiotic resistance in bacteria to conventional antibiotics when used as an adjunct/co-therapy.
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. In 2014, NP213 will undergo final clinical evaluation with the Company’s co-development partner, Taro Pharmaceuticals. 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 (CF) and other chronic respiratory diseases. NM001 is being developed as an oral treatment for CF exacerbations and is also being formulated for inhalation for longer term, more targeted delivery of the drug. An exciting pipeline of preclinical stage drug candidates is already demonstrating significant promise. These include Novamycin® (NP339, NP525), an antifungal peptide/lipopeptide agent against Candida spp, Aspergillus 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.
For further information please contact:
Dr. Deborah O’Neil - CEO & CSO
0044 (0)1224 711377