Therapeutic products based on RNA interference (RNAi) represent a significant opportunity for furthering healthcare but, as with many biologics, stability is an issue. Having tested its formulations MDRNA believes they have adequate shelf-lives.
Di-alkylated amino acid (DiLA2) formulations were stored frozen or in refrigerated conditions, at temperatures ranging from -80ºC to 4ºC, and tested for potency after 12 months.
MDRNA found that the formulations maintained in vivo knockdown activity with full potency, after either freezing or refrigeration, and particle characteristics, such as size and charge, were unchanged.
Long-term stability of DiLA2 will be important as MDRNA’s internal and partnered therapeutic candidates move into clinical trials and perhaps ultimately commercial distribution.
"The ability of our novel DiLA2 formulation to be stored frozen or at refrigerated conditions is unique", stated Barry Polisky, chief scientific officer at MDRNA. Polisky added that MDRNA has “the flexibility to tailor storage conditions” to the requirements of each candidate.
DiLA2 is a platform for creating novel liposomal delivery systems. Using the platform MDRNA can alter the charge, acyl chain characteristics and linker length to improve the targeting of specific tissues.
In March 2009 Novartis licensed the delivery platform for an upfront payment of $7.3m (€5.3m). The cash injection was a welcome boost for MDRNA which, in response to the economic environment, chose last year to focus resources on liver cancer treatments.
NASDAQ recently extended MDRNA’s grace period until May 5 2010. To regain compliance the company must have a market value of listed securities (MVLS) of at least $50m for a minimum of ten consecutive days.