CYSTEAMINE
NovaBiotics pharma pipeline is focused on the application of cysteamine in infectious-inflammatory respiratory disease.
NMOO1 and Cystic Fibrosis
Cysteamine in oral and inhaled form in exacerbating and stable cystic fibrosis patients respectively could provide the opportunity for a paradigm shift in standard of care therapy. Importantly, as a cystic fibrosis (CF) candidate therapy, NM001 is not a mutation-specific CFTR-targeting intervention. The unique multi-activity of NM001 in both oral and inhaled forms has been demonstrated in our clinical trials and a range of laboratory experiments.
Oral NM001 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.
Orphan drug designation has been granted in the US and Europe for NM001 in the treatment of CF. NM001 has fast track designation for pulmonary exacerbations of CF in the US. Oral NM001 was also designated as a priority review medicine by the Central European Health Technology Assessment body.
Our proprietary inhaled (dry powder) formulation of NM001 has been optimised and from a platform of very encouraging preclinical efficacy and safety data, NovaBiotics has built a clinical development plan for inhaled NM001 in stable CF patients. Inhaled NM001 (in dry powder form) is intended to reduce the frequency of pulmonary exacerbations. This is particularly important for patients who do not/cannot take CFTR modulator therapies.
NM001 and non-cystic fibrosis brochiectasis
NovaBiotics intends to build on a solid platform of CF data for developing NM001 for pulmonary exacerbations of non-cystic fibrosis bronchiectasis (NCFBE) and for maintenance of ventilatory function in NCFBE patients with oral and inhaled NM001, respectively.
Oral cysteamine and mycobacterial disease
NovaBiotics is collaborating with the Liverpool School of Tropical Medicine [insert PR link] on developing oral cysteamine as a microbially and host-directed treatment for tubercular and non-tubercular mycobacterial disease, adjunct to combination antimicrobial therapy. We have a particular focus on drug resistant mycobacterial infections and improving the effectiveness/reducing therapy duration for all mycobacterial treatments.
NM002 and pneumonia
NM002 is an intravenous (IV) form of cysteamine bitartrate that has been investigated in a phase 3 clinical study, REMAP CAP (A Randomised, Embedded, Multifactorial, Adaptive Platform trial for Community Acquired Pneumonia (CAP). Study subjects were 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 were 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.
A compelling body of preclinical data concerning the immunomodulatory-antimicrobial 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 support the use of NM002 in acute respiratory disease. NM002 is further supported by positive clinical data for oral cysteamine as NM001 in CF patients experiencing a pulmonary exacerbation.