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Isis Pharmaceuticals Reports an Update on ISIS-TTR Rx, Including Positive Data from Multiple Clinical Studies, Presented Today at the EC-ATTR Meeting
"Data from our study is encouraging. The study is designed to monitor progression of cardiomyopathy in patients treated with ISIS-TTRRx. The MRI data measured left ventricular mass for the first three patients to complete 12 months on therapy. These data show evidence of disease stabilization when compared to baseline. These observations compare favorably to those from our previously published natural history data. In the natural history study1, using the same measurements, we observed disease progression at 12 months," said
- Patients in Dr. Benson's previously published natural history study with an interventricular septum thickness (IVS) >1.3cm (mean =1.9) at study entry had a mean increase of 14 percent in left ventricular mass as measured by MRI at 12 months.
- Patients entering the Phase 2 ISIS-TTRRx study had baseline IVS > 1.3cm (mean =2.03). The first three patients treated with ISIS-TTRRx had a mean decrease of 1.9 percent in left ventricular mass from baseline as measured by MRI at 12 months.
- No discontinuations and injection site reactions occurring in less than two percent of all injections, which were predominantly mild. The safety and tolerability profile of ISIS-TTRRx supports continued development.
"We are encouraged by the substantial reductions in TTR protein we have observed in patients who have a variety of variants of the TTR gene. We believe that the observed tolerability profile and the convenience of dosing ISIS-TTRRx (one low volume, (1.5 mL) once weekly, subcutaneous injection that enables at home administration) contribute significantly to the high rate of patient retention in our Phase 3 FAP study and the high (100 percent) rate of eligible patients enrolling in the OLE study," said
In a poster presented today titled, 'A phase 3 clinical trial with ISIS-TTRRx, a 2nd-generation antisense oligonucleotide targeting transthyretin (TTR), for the treatment of TTR amyloid cardiomyopathy' (abstract # PM15), GSK provided an overview on the design of the ISIS-TTRRx Phase 3 study in patients with TTR amyloid cardiomyopathy (CARDIO-TTR). This randomized, double-blind, international, multi-center study will evaluate the efficacy and safety of ISIS-TTRRx in approximately 500 TTR amyloid cardiomyopathy patients diagnosed with either FAC or wt-TTR amyloidosis. The primary endpoint will be based on clinical composite outcomes that will include mortality, cardiac transplant and cardiovascular hospitalization.
"We are committed to a broad development program for ISIS-TTRRx to treat all patients who suffer from TTR amyloidosis," said
ABOUT ISIS-TTRRx
ISIS-TTRRx is a gen 2.0+ antisense drug Isis is developing with GSK for the treatment of TTR amyloidosis. ISIS-TTRRx is administered as a single 300 mg injection, once weekly, at home self-administered low-volume subcutaneous injection and is designed to inhibit the production of all forms of TTR protein, including both hereditary and wild-type, offering a unique approach to treat all types of TTR amyloidosis. ISIS-TTRRx has already demonstrated sustained and robust TTR reductions in multiple clinical studies in multiple indications of TTR-related amyloidosis.
ISIS-TTRRx is currently being evaluated in a Phase 3 randomized, double-blind, placebo-controlled, international study in patients with FAP. The study is designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life. For further study information, please visit www.clinicaltrials.gov and search for the identifier number NCT01737398.
ABOUT TTR AMYLOIDOSIS
TTR amyloidosis is a fatal genetic disease in which patients experience TTR build up in major organs, including peripheral nerves, heart, intestinal tract, kidney and bladder.
Patients with FAP experience ongoing debilitating nerve damage throughout their body resulting in the progressive loss of motor functions, such as walking. These patients also accumulate TTR in major organs, which progressively impacts their function and eventually leads to death. Therapeutic options for the treatment of FAP are very limited and there are currently no drugs approved for the treatment of FAP in the United States. There are an estimated 10,000 FAP patients worldwide.
Patients with FAC experience ongoing debilitating heart damage resulting in progressive heart failure. Therapeutic options for the treatment of FAC are very limited and there are currently no drugs approved for the treatment of FAC. There are an estimated 40,000 FAC patients worldwide.
wt-TTR amyloidosis (previously referred to as senile systemic amyloidosis or SSA) is a form of TTR amyloidosis associated with the aging process and is characterized by deposition of amyloid fibrils derived from normal or wild-type TTR protein. Although the hereditary forms of TTR are more likely to misfold and aggregate in various tissues, the normal or wild-type TTR protein can also cause heart damage. Amyloid deposition is mainly seen in the myocardium, resulting in arrhythmia (atrial fibrillation) and/or heart failure. wt-TTR amyloidosis typically affects male patients over 80 years, but is also indicated in younger patients, with an onset around 50 years. There are an estimated 200,000 patients worldwide.
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ABOUT
Isis 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, Isis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Isis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; ISIS-TTRRx, a drug Isis is developing with GSK to treat patients with all forms of TTR amyloidosis; and ISIS-SMNRx, a drug Isis is developing with Biogen to treat infants and children with spinal muscular atrophy. Isis' patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.
This press release includes forward-looking statements regarding
In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to
1Benson MD et al. Am J Cardiol. 2011 Jul 15;108(2):285-9.
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SOURCE
D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741; or Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772