Oto Entrepreneur: Dr. Nikki Johnston, Co-Founder of N-ZYME Biomedical

Written by Nikki Johnston, PhD

In 2021, I was recognized for my role as co-founder and CSO of N-Zyme Biomedical, a company focused on effective medical therapy for Laryngopharyngeal Reflux (LPR), a significant unmet clinical need. I was also selected as a 2021 Falk Medical Research Trust Catalyst Award recipient and received a grant for drug reformulation, a process needed to repurpose Fosamprenavir. Fosamprenavir, the drug used to treat HIV, prevented pepsin-mediated laryngeal injury in an LPR model. Local inhalation treatment was effective at a much lower dose by binding to pepsin and inhibiting its enzymatic activity, thus making it an effective treatment for LPR.

My multifaceted career at MCW, including my role at N-Zyme Biomedical and a professorship in Microbiology & Immunology, exemplify the promotion and advancement of research of reflux therapeutics. It is with much gratitude and appreciation that I lead the commitment to innovative approaches to cutting-edge research within our basic science division!
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N-Zyme Biomedical Inc. aims to provide a new formulation of fosamprenavir as a safe, effective, and efficient treatment for laryngopharyngeal reflux (LPR)

While proton pump inhibitor (PPI) therapy is a mainstay in the treatment of gastro-esophageal reflux disease (GERD), their efficacy for laryngopharyngeal reflux (LPR) is poor. Given the paucity of data supporting a beneficial effect of PPI treatment for extraesophageal symptoms, the American Gastroenterological Association now recommend against their use for LPR in the absence of typical GERD symptoms. Studies using combined multi-channel intraluminal impedance – pH monitoring (MII-pH) have shown that many episodes of LPR are non-acidic and that weakly and non-acidic LPR is associated with persistent symptoms in acid-suppressed patients, that are alleviated by anti-reflux surgery.The gastric enzyme pepsin is present in all refluxate and frequently detected in airway tissue and secretions from patients with LPR, absent in MII-pH-confirmed reflux-free control subjects. Non-acid proximal reflux of pepsin is association with symptoms and endoscopic findings and physiologically relevant concentrations cause laryngeal inflammation and damage in in vitro and in vivo studies, including pathological changes consistent with those observed in LPR patients. A new treatment which specifically targets pepsin could therefore be of great value.

Nikki Johnston, PhD identified select HIV protease inhibitors that bind to the active site of pepsin and inhibit the enzyme, preventing pepsin-mediated laryngeal inflammation and damage in an in vivo mouse LPR model. In support, patients taking HIV inhibitors have a significantly lower incidence of LPR (0.2%) compared to that reported for the general population (10 – 34.4%). The lead candidate, fosamprenavir, targets a foreign virus - not present in most individuals, has a good safety profile, and is well tolerated, making it an ideal drug to repurpose allowing a safer and faster assessment in a phase III trial. FDA (IND 149546) and IRB (PRO00037954) approval have been obtained for a 12-week randomized, double-blind, placebo-controlled, phase III clinical trial to assess the efficacy of systemic fosamprenavir for the treatment of LPR. The long-term goal is to provide local inhaled treatment as the larynx and surrounding tissues affected by LPR are readily accessible, allowing a higher concentration at the disease site and lower systemic concentration (International Patent: PCT/US2021/027758 Aerosolized formulations of HIV protease inhibitors for the treatment of airway reflux). This will likely increase efficacy and decrease side effects. The patent has been exclusively licensed to N-Zyme Biomedical Inc. a startup company for which Nikki Johnston is a co-founder and Chief Scientific Officer. Co-founder and Chief Executive Officer, Franco Vigile, is an entrepreneur with over 10 years’ experience in founding/operating successful companies including HaluGen, a leading genetic-based pre-screening platform, and GENNCO Holdings, a Canadian boutique advisory firm. The phase III trial, supported through Venture Capital investment in N-Zyme Biomedical Inc, will begin enrolment Spring 2022 and run concurrently with IND-enabling studies of the dry powder formulation for local inhaled delivery, supported by a Falk Catalyst Award.

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Nikki Johnston, PhD

Associate Professor, Otolaryngology & Communication Sciences and Microbiology & Immunology