Alnylam and Genzyme Announce that Genzyme Opts into ALN-AT3 Hemophilia Program for Development and Commercialization Outside of North America and Western Europe
-Marks First Product Opt-In since Formation of Landmark 2014
This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20151001005526/en/
"Our collaboration with Genzyme is a key part of our strategy to advance
RNAi therapeutics to global markets. Genzyme's proven track record in
developing and commercializing therapies for rare diseases makes them an
ideal partner to advance an innovative medicine for the treatment of
hemophilia. Accordingly, we are very pleased that they have elected to
opt into the ALN-AT3 program, the first product opt-in since formation
of our landmark alliance," said
"ALN-AT3 represents a unique and promising new approach for the
potential treatment of hemophilia," said
In January 2014, Alnylam and Genzyme formed an alliance to accelerate and expand the development and commercialization of RNAi therapeutics across the world. The alliance is structured as a multi-product geographic alliance in the field of rare diseases, i.e., programs in Alnylam's Genetic Medicine Strategic Therapeutic Area. Alnylam retains product rights in North America and Western Europe, while Genzyme obtained the right to access certain programs in Alnylam's current and future Genetic Medicines pipeline in the rest of the world (ROW) through the end of 2019, together with certain broader co-development/co-commercialization rights and global rights for certain products. Alnylam maintains development and commercialization control for all programs in its territory.
ALN-AT3 is the third Alnylam product for which Genzyme has exercised its
opt-in right, the first two occurring at the close of the deal in early
2014 for patisiran and revusiran, investigational RNAi therapeutics for
the treatment of transthyretin-mediated amyloidosis. In the case of
ALN-AT3, Genzyme has elected presently to opt into the program for its
ROW rights. Genzyme retains its future opt-in right to co-develop and
co-promote ALN-AT3 with Alnylam in
Per the 2014 agreement, Alnylam will receive R&D funding for programs
where Genzyme has elected to opt in for development and
commercialization. For "regional" programs where Genzyme will develop
and commercialize in their ROW territory, such as patisiran and ALN-AT3
as currently structured, Genzyme pays 20% of global development costs.
In the case of ALN-AT3, such cost sharing is expected to begin in
About ALN-AT3 for Hemophilia
Hemophilias are hereditary disorders caused by genetic deficiencies of various blood clotting factors, resulting in recurrent bleeds into joints, muscles, and other major internal organs. Standard treatment for people with hemophilia involves replacement of the missing clotting factor either as prophylaxis or "on-demand" therapy. However, as many as one third of people with severe hemophilia A will develop an antibody to their replacement factor. These ‘inhibitor' subjects become refractory to standard replacement factor therapy, and therefore are significantly more complicated to manage and have poorer clinical outcomes.
ALN-AT3 is an investigational, subcutaneously administered RNAi therapeutic for the treatment of hemophilia and other rare bleeding disorders. ALN-AT3 is aimed at correcting coagulation defects by knockdown of antithrombin (AT) - an important endogenous anticoagulant. AT acts as a "brake" on the production of thrombin, a protein essential for the formation of a blood clot. ALN-AT3 is being evaluated in a Phase 1 study in people with moderate-to-severe hemophilia. A pivotal Phase 3 clinical trial is planned to start in mid-2016.
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen, Roche,
About Genzyme, a
Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a
Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, including ALN-AT3 for the treatment of hemophilia and rare bleeding disorders, expectations regarding the reporting of data from clinical studies, including completion of human proof-of-concept for ALN-AS1, its expectations regarding Genzyme's participation in the development and commercialization of RNAi therapeutics, its expectations regarding the receipt of potential R&D payments, development, regulatory and sales milestones and royalties from Genzyme, expectations regarding its STAr pipeline growth strategy, and its plans regarding commercialization of RNAi therapeutics, including ALN-AT3, constitute forward-looking statements for the purposes of the safe harbor provisions under
Sanofi Forward Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although
Vice President, Finance & Treasurer
Sanofi Media Relations
Sanofi Investor Relations
Genzyme Media Relations
News Provided by Acquire Media