Tekmira Pharmaceuticals, a specialist in RNA interference (RNAi) therapeutics announced that Alnylam Pharmaceuticals has reported final results from its Phase I clinical study of an RNAi therapeutic for transthyretin (TTR) called ALN-TTR01 that uses Tekmira’s lipid nanoparticle delivery technology to treat TTR-mediated amyloidosis and is produced by Tekmira.
Alnylam Pharmaceuticals presented the latest results at the XIII International Symposium on Amyloidosis conducted in Groningen, The Netherlands. The company had reported interim results in late 2011. According to the results, ALN-TTR01 administration significantly lowered the serum levels of both mutant and wild-type TTR proteins in patients with ATTR. For a single dose, knockdown of the disease-causing TTR protein was long-lasting, quick and based on the dosage. The results demonstrated the safety and tolerability of ALN-TTR01.
Alnylam Pharmaceuticals is now involved in the development of a second drug candidate dubbed as ALN-TTR02, which also uses Tekmira's lipid nanoparticle delivery technology and is produced by Tekmira. Alnylam Pharmaceuticals has started a Phase I clinical study on ALN-TTR02 to assess the clinical activity, tolerability and safety of ALN-TTR02. The company anticipates presenting the results from this study during Q3 of this year.
Mark J. Murray, President and Chief Executive Officer at Tekmira Pharmaceuticals, stated that the company is happy that the latest results from the Phase 1 human clinical study of ALN-TTR01 exemplify the tolerability of Tekmira's lipid nanoparticle delivery technology and its significance in the advancement of RNAi products. The company continues its efforts to advance its lipid nanoparticle delivery platform to facilitate RNAi therapeutics for various clinical indications.