Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it has completed enrollment in its Phase II trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (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.
“Our ATTR program is the lead effort in our ‘Alnylam 5x15’ product development and commercialization strategy, which is focused on advancing RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need. We are very encouraged with the clinical activity, safety, and tolerability seen to date with ALN-TTR02 in our Phase II multi-dose study performed in ATTR patients, and look forward to sharing further data at the upcoming ISFAP meeting in November. As we presented at the Peripheral Nerve Society meeting in June, ALN-TTR02 achieved robust knockdown of up to 93% of circulating wild-type and mutant TTR,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “We are pleased to now be able to provide patients on our Phase II study with the opportunity to continue receiving ALN-TTR02 on our OLE study, which will include clinical endpoint measurements such as NIS. We plan on presenting data from the OLE study about once per year hereafter, with an initial presentation of results in 2014. We also remain on track to initiate our Phase III study of ALN-TTR02 in FAP patients by the end of this year, and have now completed discussions with regulatory authorities regarding our overall development plans to support submissions for product approval.”