New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen
- Category: DNA RNA and Cells
- Published on Monday, 10 July 2017 20:41
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Significant benefit observed for both clinical measurements of neurological disease progression and quality of life at 8 and 15 months in patients with FAP
CARLSBAD, CA, USA I July 10, 2017 I Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) presented new top-line data from the Phase 3 NEURO-TTR study of inotersen in patients with familial amyloid polyneuropathy (FAP) at the 2017 Peripheral Nerve Society Meeting. Results from the study demonstrated benefit across both primary endpoints of the study: modified Neuropathy Impairment Score +7 (mNIS+7) and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) at 15 months of treatment. Inotersen-treated patients benefited significantly in quality of life compared to placebo, with a difference in magnitude of 11.68 points in the Norfolk QoL-DN score at 15 months of treatment (mean change from baseline of 0.99 vs. 12.67, p=0.0006). Statistically significant benefit was also observed for both mNIS+7 and Norfolk QoL-DN at eight months of treatment with inotersen. The results continue to support the favorable benefit-risk profile for inotersen in patients with FAP. A detailed review of efficacy and safety from the study is ongoing.
"The magnitude of benefit on quality of life achieved with inotersen treatment has never been observed before in patients with FAP," said Teresa Coelho, MD, neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. "As their disease progresses, FAP patients lose the ability to do even simple activities like feeding themselves, which can limit their independence. They also suffer from debilitating symptoms, like severe extremity pain and diarrhea. The quality of life score assesses almost every aspect of this devastating disease on a patient's life. The nearly 12-point difference observed with inotersen treatment from placebo represents a clinically meaningful benefit and provides hope that inotersen could allow FAP patients to maintain greater independence by alleviating debilitating symptoms and preserving their ability to perform daily activities."
"The clinically meaningful benefit in quality of life bring a new energizing level of hope to the amyloidosis community around the world, a community whose critical needs have not been sufficiently addressed," said Isabelle Lousada, president and chief executive officer of the Amyloidosis Research Consortium (ARC). "Going from Phase 1 to Phase 3, inotersen's development program hallmarks the importance of rapidly advancing valuable, life-changing therapies that can have transformational benefits on patients' lives. We hope for a rapid approval process for inotersen to create access and availability of this important medicine, which has the potential to help patients maintain their independence and provide a brighter future for patients who suffer from this desperate and devastating disease."
The Phase 3 NEURO-TTR study met both its primary endpoints, Norfolk QoL-DN and mNIS+7, with high statistical significance. Statistical significance was observed regardless of TTR mutation (V30M vs. Non-V30M) or disease severity (Stage 1 vs. Stage 2). The Norfolk QoL-DN measures patients' perception of symptoms associated with specific nerve fiber damage. The questionnaire includes questions assessing physical function, disease symptoms, activities of daily living, physical sensation and autonomic neuropathy. The mNIS+7 is a composite score that includes measures of muscle strength, quantitative sensation testing and nerve conduction.
"The new top-line data presented today reaffirm inotersen's effect in patients with FAP. Importantly, we observed statistically significant disease benefit with inotersen treatment at only eight months of treatment, and this benefit was further amplified at the end of study analysis. We are very encouraged by the significant benefit demonstrated in quality of life, and, these results further support a strong relationship between patients' perception of benefit in quality of life and clinical measurements of neurological disease progression," said Brett Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals. "We are continuing to review the full data package from the NEURO-TTR study and prepare the regulatory marketing applications for submission this year. We look forward to sharing detailed results in future publications and medical meetings."
The U.S. Food and Drug Administration has granted Orphan Drug Designation and Fast Track Status to inotersen for the treatment of patients with FAP. The European Medicines Agency has granted Orphan Drug Designation to inotersen for the treatment of patients with TTR amyloidosis.
GSK has the option to license inotersen prior to the submission of regulatory applications.
ABOUT TTR AMYLOIDOSIS – FAP
Patients with hATTR-PN primarily experience nerve damage throughout their body resulting in the progressive loss of motor functions, such as walking. As TTR accumulates in major organs, it progressively impacts organ function and eventually leads to death. Therapeutic options for the treatment of hATTR-PN are very limited and there are currently no drugs approved for the treatment of hATTR-PN in the United States. There are an estimated 10,000 hATTR-PN patients worldwide.
ABOUT THE NEURO-TTR PHASE 3 STUDY
ABOUT IONIS PHARMACEUTICALS, INC.
SOURCE: Ionis Pharmaceuticals