Alnylam Pharma Completes Enrollment Of Phase II Clinical Trial With ALN-TTR02

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) announced that it has completed enrollment in its Phase II trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin or TTR gene for the treatment of TTR-mediated amyloidosis or ATTR. Recent interim results from this Phase II study showed that ALN-TTR02 achieved up to 93% knockdown of TTR - the disease-causing protein in ATTR.

ALN-TTR02 activity was found to be rapid, dose dependent, and durable, with similar levels of TTR knockdown observed toward both wild-type and mutant protein.

In addition, ALN-TTR02 was found to be generally safe and well tolerated in this study. Alnylam also announced today that its open-label extension (OLE) study with ALN-TTR02 is open for enrollment. The OLE study will evaluate the long-term safety and tolerability of ALN-TTR02 and will also measure effects of treatment toward a number of clinical endpoints, including a Neuropathy Impairment Score, or "NIS;" the company intends to report clinical data from this study about once per year, with initial data in 2014.

ATTR is caused by mutations in the TTR gene which cause abnormal TTR amyloid protein deposits to accumulate in various tissues including peripheral nerves and heart, resulting in neuropathy and/or cardiomyopathy.

The company said it has also opened enrollment in an OLE study of ALN-TTR02. Eligible patients treated in the Phase II study can enroll in the study, where they will receive ALN-TTR02 at a dose of 0.3 mg/kg every three weeks for up to two years.

In addition, Alnylam said it intends to start a Phase III pivotal trial for ALN-TTR02 in FAP patients by the end of 2013. The primary endpoint will be the difference in the change from baseline in the mNIS+7 score between patients receiving ALN-TTR02 as compared with those receiving placebo. Alnylam has obtained scientific advice for the ALN-TTR02 Phase III study from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and has completed its End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA).