Oral NM001 for pulmonary exacerbations of CF and NCFBE

NM001 in oral (tablet) form completed an exploratory phase 2b global study (CARE CF 1) in CF patients following an earlier, positive phase 2a trial.  CARE CF 1 was designed to determine which clinical endpoints are the most appropriate to demonstrate the benefits of NM001 in exacerbating (adult) CF patients who took NM001 alongside their other ‘standard’ medicines for 14 days (or placebo).  CARE CF 1 was also designed to determine which dose(s) and treatment regimen(s) of NM001 is/are optimal for efficacy.  The endpoint and dose(s) were successfully identified from CARE CF 1.   We now plan to progress NM001’s development in registrational studies in CF and leverage our clinical data thus far to fast track development for NCFBE patients.

Acute respiratory disease – Pandemic Preparedness

NM002’s unique combination of immunomodulatory (anti-inflammatory) and antimicrobial (antiviral and antibacterial) properties position this therapy candidate as a potential game-changer in inflammatory-infectious respiratory disease.

NM002 is an intravenous (IV) medicinal candidate currently being investigated in a major international platform (phase 3) clinical study, REMAP CAP (A Randomised, Embedded, Multifactorial, Adaptive Platform trial for Community Acquired Pneumonia (CAP). The first patients were enrolled and dosed in December 2021.  Study subjects are those in an intensive care setting with confirmed CAP of all causes (viral as well as bacterial, including COVID-19). Primary outcome measures of the phase 3 trial are all-cause mortality at day 90 and days alive and not receiving organ support in ICU for patients with suspected or proven COVID-19 infection.

Although NM002 is initially being developed in IV form, there is potential to investigate an oral form of NM002 for treatment of less severe CAP and other pneumonias in hospital and community settings.

NovaBiotics extensive research with, and deep understanding of aminothiol biochemistry and biology, cysteamine in particular, supports and de-risks NM002’s application in CAP. A compelling body of preclinical data concerning the immunomodulatory mechanisms of NM002, particularly targeting IL-6 and interferons, its host-directed antiviral effects and the company’s published work on cysteamine’s anti-virulence and antibiotic potentiating modalities are further augmented by positive clinical data for NovaBiotics’ NM001 asset, derived from the same platform as NM002 and in late stage development for pulmonary exacerbations of cystic fibrosis.

NP432 has been derived from a platform of antibacterial peptide therapy candidates.  These bactericidal agents have a number of key benefits over conventional antibiotic therapies and the clear potential to succeed where existing treatments for a number of bacterial infections, including drug resistant MDR and XDR infections are failing.

NP432 and our family of antibacterial peptides are derived from same technology platform as NP339 and so the route to clinic anticipated for this novel antibacterial therapy candidate has already been significantly de-risked.

NP432 is NovaBiotics’ preclinical stage lead antibacterial peptide asset and along with its family of related peptides is broad-spectrum, potently and rapidly bactericidal in vitro against clinically and economically important bacteria. NP432 is active against bacteria identified by the World Health Organisation as Priority Pathogens including Pseudomonas aeruginosa, Clostridium difficile, Acinetobacter baumannii and Klebsiella pnuemoniae. NP432 and its related family of antibacterial peptides are active against drug resistant and multi drug resistant bacteria. NP432 has shown to be well-tolerated and has demonstrated efficacy in in vivo infection models.