Alnylam Pharmaceuticals Inc. (ALNY) recently announced that its candidate ALN-TTR02 has fared well in a phase I study (n=17). The candidate is being developed for the treatment of transthyretin (:TTR)-mediated amyloidosis (:ATTR). The data was presented at a seminar in Boston University School of Medicine.
The randomized, single-blind, placebo-controlled and single-ascending dose study assessed the safety and tolerability profile of ALN-TTR02. The candidate was found to be safe and well tolerated with an impressive pharmacologic profile. Results from the study revealed that patients treated with ALN-TTR02 experienced reduction of up to 94% of serum TTR following a solitary dose.
We remind investors that last month the US Food and Drug Administration (:FDA) granted orphan drug status to ALN-TTR02. The candidate, ALN-TTR02, received orphan drug status to treat patients suffering from familial amyloidotic polyneuropathy (:FAP). The disorder refers to one of the predominant clinical manifestations of TTR- ATTR.
We note that the orphan drug designation is granted in the US only to drugs that treat a disease affecting less than 200,000 people in the country. Moreover, the orphan drug status for ALN-TTR02 provides Alnylam with seven years of marketing exclusivity (from the day ALN-TTR02 is cleared by the FDA) in the US for the indication.
The company also initiated a phase II trial to evaluate clinical activity, safety and tolerability of multiple once-monthly doses of ALN-TTR02 in patients suffering from ATTR. Subject to positive results, the company plans to initiate pivotal trial in 2013.
Apart from Alnylam, Pfizer (PFE) is also developing its candidate, Vyndaqel (tafamidis), to treat TTR-FAP. We note that TTR-FAP received a complete response letter (CRL) from the FDA in June 2012. The drug is approved in the EU.
Apart from ALN-TTR02, Alnylam is also developing ALN-TTRsc, for the utilization of GalNAc-conjugate delivery technology and subcutaneous dose administration, into clinical development. The company intends to file a New Drug Application (IND) for ALN-TTRsc in the second half of 2012 with data expected in the first half of 2013. Furthermore, the company is developing ALN-RSV01 for the treatment of respiratory syncytial virus infection; ALN-VSP for the treatment of liver cancer.