New Positive Clinical Results for Patisiran at AAN

New Positive Clinical Results for Patisiran at AAN

We presented new positive results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, at the American Academy of Neurology (AAN) 2018 Annual Meeting, being held April 21-27, 2018 in Los Angeles.

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Adams et al. – “Patisiran, an Investigational RNAi Therapeutic for Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis: Results from the Phase 3 APOLLO Study”

Adams et al. – “Evaluation of Quality of Life and Disability in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Following Treatment with Patisiran, an Investigational RNAi Therapeutic: Results from the Phase 3 APOLLO Study”

Mora et al. – “Utility of Genetic Testing to Identify Individuals Suspected of Having Hereditary ATTR (hATTR) Amyloidosis”

Post-Hoc Analysis of Hospitalization and Mortality Events
A new post-hoc, exploratory recurrent event analysis revealed an approximately 50% decrease in the composite rate of all-cause hospitalization and mortality over 18 months in patisiran-treated patients, relative to placebo. A similar finding was observed with the composite rate of cardiac hospitalization and all-cause mortality, showing an approximately 45% decrease with patisiran, relative to placebo.

Quartile Analysis of Baseline Neurologic Impairment Score
Based on a quartile analysis of baseline Neurologic Impairment Score (NIS), patisiran demonstrated halting or improvement in the modified NIS+7 (mNIS+7) primary endpoint in patients regardless of baseline neuropathy severity, in contrast to the progression in mNIS+7 seen in placebo-treated patients. While treatment benefit is observed across all stages of disease, these results support the rationale for early treatment with patisiran to potentially halt or improve neuropathy progression or impairment, respectively.

Quality of Life Results
Patisiran treatment was associated with improvement across all domains of the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire at 9 and 18 months, relative to placebo. Significant improvements in disability, gait speed, autonomic neuropathy symptoms, and overall quality of life, as reported by the patient, were also noted.

Overall, there were 13 deaths in the APOLLO study; none were considered related to study drug and the frequency of deaths was lower in the patisiran group (4.7%) as compared with placebo (7.8%). The most commonly reported adverse events (AEs) that occurred more frequently in patisiran-treated patients were peripheral edema and infusion-related reactions (IRRs) and were generally mild to moderate in severity. AEs leading to treatment discontinuation were lower in patisiran-treated patients (4.7%) compared with placebo-treated patients (14.3%).

Alnylam Act™ Update
We also presented results of an analysis of the utilization of genetic testing through Alnylam Act™, a program created to reduce barriers to genetic testing and counseling to help people make more informed decisions about their health. As of March 2018, Alnylam Act has facilitated testing of approximately 4,600 individuals who may carry gene mutations known to be associated with hATTR amyloidosis. Among these, approximately 350 patients were identified with positive pathogenic TTR mutations, representing approximately 7.5 percent of the patients tested since the Alnylam Act program was initiated in 2014.

We believe these data, along with previously presented APOLLO data showing halting or reversal of neuropathy progression in a majority of patients treated with patisiran, strengthen the body of evidence demonstrating that patisiran, if approved, has the potential to be a transformative treatment for patients with all forms of hereditary ATTR amyloidosis.  We continue to work collaboratively with the FDA and EMA through patisiran’s review process, with the goal of making this medicine available to patients as quickly as possible, upon approval.


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