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New Data Presented at Peripheral Nerve Society Meeting Further Support Potential Benefit of Inotersen

"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.

  • Treatment with inotersen resulted in a statistically significant benefit in Norfolk QoL-DN score compared to placebo of 6.14 points at 8 months of treatment (mean change from baseline of 0.81 vs. 6.95, p=0.032) and 11.68 points at 15 months of treatment (mean change from baseline of 0.99 vs. 12.67, p=0.0006).
  • Treatment with inotersen resulted in a statistically significant benefit in mNIS+7 compared to placebo at eight months of treatment (p=0.0005) and at 15 months of treatment (p=0.00000004).
  • As previously identified, the key safety findings in...