LAMELLASOME™ products have an excellent safety profile as demonstrated in GLP non-clinical safety and toxicity testing, and in two clinical studies. Preclinical studies found:
• Excellent safety and tolerability profile for oral and ocular tissues for cytotoxicity, genotoxicity, irritation and sensitisation.
• Safe inhalation profile with very high NOAEL of ~4,000mg/day (standardised to a 60kg person) from maximum tolerated and long-term inhalation toxicology studies.
Excellent safety in repeat dosing of LAMELLASOME monotherapy was confirmed in clinical studies of moderate to severe dry eye, as well as radiotherapy-induced xerostomia (dry mouth) in patients with head and neck cancer.
As a proof of concept of the platform, Lamellar has chosen two LAMELLASOME™ nucleic acid products, which it is advancing through preclinical efficacy testing. These products target Idiopathic Pulmonary Fibrosis (LAMELLASOME™ IPF-NA) and Cystic Fibrosis (LAMELLASOME™ CF-NA) which are Orphan Drug Designated conditions with high unmet clinical need.
Lamellar is looking for opportunities to advance these products through out-licensing or joint development agreements.
LAMELLASOME™ IPF-NA is a LAMELLASOME™/microRNA (miR) product that is being developed for the treatment of idiopathic pulmonary fibrosis.
Lamellar has shown that the miR is safely and effectively delivered by LAMELLASOME™ and that it has a significant downstream antifibrotic effect, demonstrated as a marked reduction in IPF biomarkers. All transfected cells demonstrated high transfection, high cell viability and maintenance of cell phenotype.
LAMELLASOME™ CF-NA is a LAMELLASOME™/plasmid DNA treatment targeted at functional CFTR replacement therapy for Cystic Fibrosis.
Lamellar has data to prove that LAMELLASOME™ CF-NA provides safe and effective delivery of functional plasmid to lungs in vivo, while avoiding gene therapy-associated immune response.
For more information on the LAMELLASOME NAT platform and our lead products, please contact us or download the LAMELLASOME factsheet.