Presentation Summary
Dr. Eric Green, Senior Vice President of Alnylam Pharmaceuticals started by elucidating that ATTR amyloidosis is a rare progressively debilitating disease caused by misfolded Transthyretin (TTR) protein that accumulates as amyloid deposits in multiple tissues, including the heart, nerves and GI tract.
Both the hereditary and wild-type forms of the disease may present in adults with multisystem involvement; and a high burden of disease is often fatal.
Dr. Green said that Alnylam’s objective is to use RNAi therapeutic to dramatically reduce the production of the disease-causing TTR protein in the liver, preventing continued amyloid deposition and allowing the body to potentially remove existing deposits, ultimately halting or improving the manifestations of the disease.
Alnylam also aims to reach every single patient affected by this disease by providing a growing genetic and imaging testing program.
Dr. Green explained that ONPATTRO (Patisiran) is a RNAi therapeutic administered intravenously once every 3 weeks, that was first approved in the U.S. in August 2018 based on a landmark APOLLO Phase III study.
Currently, this therapeutic is sold globally (EU, Canada, Japan, Switzerland and Brazil) in order to reach the most remote ATTR affected populations.
The most recent data sets from the OLE study, or open label extension, specifically the impact of continued treatment with Patisiran on mNIS+7, a measure of neuropathy impairment, showed continued improvement relative to the original APOLLO study baseline for patients treated with Patisiran for 42 months (that is the 18 months on APOLLO and the additional 24 months in the global OLE study).
Unfortunately, the impairment that placebo patients accumulated during APOLLO, can't be fully overcome, further stressing the importance of an early diagnosis and initiation of a treatment, that has the potential to halt or reverse the polyneuropathy symptoms of hATTR amyloidosis.
Dr. Green emphasized that most importantly, Patisiran continues to show a positive benefit risk profile, consistent with the APOLLO Phase III study, with long-term exposure, with some patients having received over 6 years of continuous Patisiran treatment.
To date, the safety profile of Patisiran in this population remains positive and encouraging.
