"The magnitude of benefit on quality of life achieved with inotersen treatment has never been observed before in patients with FAP," said
"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
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 the NEURO-TTR study were thrombocytopenia and renal dysfunction. Enhanced platelet and renal monitoring have effectively managed these safety events since implementation in the NEURO-TTR study.
"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
Inotersen (IONIS-TTRRx) is a generation 2.0+ antisense drug Ionis is developing for the treatment of TTR amyloidosis. Inotersen is administered once weekly as a single 300 mg subcutaneous injection. The drug is designed to inhibit the production of all forms of TTR protein, including both the hereditary and wild-type forms, offering a unique approach to treat all types of TTR amyloidosis. Inotersen has demonstrated sustained and robust TTR reductions in clinical studies in different populations of patients with TTR-related amyloidosis.
GSK has the option to license inotersen prior to the submission of regulatory applications.
ABOUT TTR AMYLOIDOSIS – FAP
FAP, now referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), is a progressive, debilitating and fatal genetic disease in which patients experience TTR build up in major organs, including peripheral nerves, heart, intestinal tract, kidney and bladder.
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
ABOUT THE NEURO-TTR PHASE 3 STUDY
Inotersen was evaluated in a Phase 3 randomized (2:1), double-blind, placebo-controlled, international study in 172 patients with hATTR-PN. The study was designed to support an application for marketing approval of inotersen in patients with hATTR-PN. The 15-month study measured the effects of inotersen on neurological dysfunction and on quality-of-life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. For further study information, please visit www.clinicaltrials.gov and search for the identifier number NCT01737398.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) is a drug that has been approved in the U.S. and
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis' alliance with GSK and the therapeutic and commercial potential of inotersen. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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Ionis Pharmaceuticals Investor and Media Contacts: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741, Alissa Santa Maria, Assistant Director, Corporate Development, 760-603-2643, Jennifer Capuzelo, Assistant Director, Corporate Communications and Investor Relations, 760-603-2606