Genzyme and Isis Begin Second Phase 3 Trial of Mipomersen
Broad New Patent Granted
Genzyme Corp. (Nasdaq: GENZ) and Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) today announced that they have begun a phase 3 study of mipomersen in patients with heterozygous familial hypercholesterolemia (heFH), a genetic disorder that causes exceptionally high levels of LDL cholesterol. It is the first of four new trials the companies plan to initiate by the end of this year, and the second late-stage study of mipomersen, a novel lipid-lowering drug that utilizes antisense technology.
The companies also announced that the United States Patent and Trademark Office granted the patent entitled “Antisense Modulation of Apolipoprotein B (apoB) Expression,” U.S. Patent No. 7,407,943. The patent broadly covers the use of antisense compounds targeting the apoB messenger RNA except a ribozyme.
“We are making excellent progress in implementing our comprehensive development plan for mipomersen,” stated John P. Butler, president of Genzyme’s renal, endocrine and cardiovascular business units. “The start of the heFH study puts us on track to meet our goal of beginning four new mipomersen studies this year. In addition, the recent patent issuance increases the overall value of mipomersen, which we believe has enormous potential to help patients.”
The new trial will evaluate the safety and efficacy of mipomersen in patients who have heFH and coronary artery disease. It is a randomized, double-blind, placebo-controlled study taking place at approximately 30 sites in the U.S. and Canada, with an anticipated total enrollment of around 100 patients. Patients on a stable dose of other lipid-lowering agents are being randomized 2:1 to receive a 200 mg dose of mipomersen or placebo weekly for 26 weeks. The primary endpoint will be percent reduction in LDL cholesterol, and data are expected to be available in 2010. The trial will add to the body of clinical data on mipomersen and the overall product profile.
“This is an exciting moment for mipomersen, combining the initiation of the phase 3 trial in heFH with a very broad patent allowance that expands the patent coverage for the apoB franchise,” said Stanley Crooke, chairman and chief executive officer of Isis. “This comprehensive patent covers methods of inhibiting apoB by targeting anywhere on the messenger RNA including the site to which mipomersen binds, and all therapeutic uses that might result from lowering apoB.”
Mipomersen Development Plan
The initial indication sought for mipomersen will be for patients with homozygous FH, and enrollment in a phase 3 trial in this patient population is expected to be completed by the end of this year. Data are expected to be available in mid-2009 and a U.S. filing for this indication is anticipated during the second half of 2010.
Genzyme and Isis plan to begin three additional trials evaluating mipomersen’s safety and efficacy in reducing LDL cholesterol in high-risk patients during the second half of 2008. These trials will include: one for apheresis-eligible patients, and two for high-risk, high cholesterol patients. All three have anticipated trial designs that include a 2:1 randomization ratio of a 200 mg dose of mipomersen or placebo weekly for 26 weeks.
These trials will continue to build the body of clinical evidence around mipomersen’s value in managing very high risk patients. Data from the trials will also inform the design of the morbidity and mortality outcome study for potential expansion of mipomersen’s label to include a broader group of at-risk, high cholesterol patients on maximally tolerated, currently available therapies.
Following the finalization in June of the mipomersen license and collaboration agreement between Genzyme and Isis, the mipomersen IND and all regulatory authority has been transferred to Genzyme. Now that this transition has taken place, the companies are looking forward to Genzyme beginning discussions with the FDA and regulatory authorities in Europe, where the development path for mipomersen may differ from that in the U.S.
The patent entitled “Antisense Modulation of Apolipoprotein B (apoB) Expression,” U.S. Patent No. 7,407,943 is the first allowance in a series of broad filings protecting the therapeutic use of targeting apoB for the lowering of all atherogenic lipids, including LDL cholesterol and triglycerides. The patent covers the use of both single-stranded and double-stranded (siRNA) antisense drugs complementary to any site of the mRNA of human apoB regardless of their chemistry or antisense mechanism of action. The allowance provides broad protection of the Isis-Genzyme apoB franchise, including mipomersen and potential future follow-on compounds.
Genzyme Safe Harbor Statement
This press release contains forward-looking statements, including without limitation, statements concerning mipomersen’s safety and benefits for patients with high cholesterol, the development plan for mipomersen and FDA’s requirements for its approval. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These risks and uncertainties include, among others: the timing of further discussions with FDA regarding the approval of mipomersen; the timing and content of submissions to and decisions made by the FDA relating to mipomersen; further analysis of clinical trial data; the results of other studies; the actual efficacy and safety of mipomersen; and the risks and uncertainties described in Genzyme’s SEC reports filed under the Securities Exchange Act of 1934, including the factors discussed under the caption “Risk Factors” in Genzyme’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2008. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of today’s date and Genzyme undertakes no obligation to update or revise the statements.
Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.
Isis Safe Harbor Statement
This press release includes forward-looking statements regarding Isis’ collaboration with Genzyme Corporation, its financial and business development activities, and the development, activity, therapeutic potential and safety of mipomersen in treating patients with high cholesterol. Any statement describing Isis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis’ goals or projections. 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, in developing and commercializing systems to identify infectious organisms that are effective and commercially attractive, and in the endeavor of building a business around such products. Isis’ 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 Isis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis’ programs are described in additional detail in Isis’ annual report on Form 10-K for the year ended December 31, 2007, and its quarterly report on Form 10-Q for the quarter ended March 31, 2008, which are on file with the SEC. Copies of these and other documents are available from the Company.
Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals, Inc. Ibis Biosciences and Ibis T5000 are trademarks of Ibis Biosciences, Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics LLC.
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