Alnylam Launches "Alnylam 2020" Guidance for Advancement and Commercialization of RNAi Therapeutics
- By End of 2020, Company Expects to Achieve Profile with 3 Marketed Products, 10 RNAi Therapeutic Clinical Programs - Including 4 in Late Stages - Across 3 Strategic Therapeutic Areas (STArs) -
- Provides Goals and Guidance for 2015, Exceeding its "Alnylam 5x15" Guidance Established in 2011 and Further Updated in 2014 -
- Increases Guidance to End 2014 with Approximately
"Our ‘Alnylam 5x15' product strategy put Alnylam on a trajectory to
transition from a platform technology company to a platform-driven
product company. We successfully executed on this strategy, and are now
on track to even exceed our expanded 2014 guidance. ‘Alnylam 2020' is
new guidance that marks our expected transition from a late-stage
clinical development company to a multi-product commercial-stage company
with a sustainable development pipeline - a profile that we believe has
rarely been achieved in the biopharmaceutical industry. Specifically,
‘Alnylam 2020' provides what we believe to be an exciting path forward
for Alnylam with multiple marketed products and a rich pipeline of
programs in clinical development, including in late stages, in our 3
Strategic Therapeutic Areas, or ‘STArs'," said
Alnylam also announced today its goals and guidance for 2015 across its
3 STArs, putting the company on track to exceed its "Alnylam 5x15"
guidance - introduced in
Genetic Medicine STAr, a broad pipeline of investigational RNAi therapeutics for rare diseases.
Patisiran, an RNAi therapeutic targeting transthyretin (TTR) for the
treatment of TTR-mediated amyloidosis (ATTR) in patients with familial
amyloidotic polyneuropathy (FAP).
- Continue enrollment in the APOLLO Phase 3 study.
- Present data in mid-2015 from ongoing Phase 2 open-label extension (OLE) study with patisiran in patients with FAP.
Revusiran, a subcutaneously administered RNAi therapeutic targeting
TTR for the treatment of ATTR in patients with familial amyloidotic
- Continue enrollment in the ENDEAVOUR Phase 3 study.
- Present data in late 2015 from ongoing Phase 2 OLE study in patients with TTR cardiac amyloidosis.
- Present results from natural history study of patients with TTR cardiac amyloidosis in early 2015.
ALN-AT3, a subcutaneously administered RNAi therapeutic targeting
antithrombin (AT) for the treatment of hemophilia and rare bleeding
- Continue enrollment in Phase 1 study.
- Present additional data in early and mid-2015, with complete data in late 2015.
- Start Phase 2 study in late 2015.
ALN-CC5, a subcutaneously administered RNAi therapeutic targeting
complement C5 for the treatment of complement-mediated diseases.
- Start Phase 1/2 study in early 2015.
- Present initial data in mid-2015 and additional data in late 2015.
- Initiate "Part C" of Phase 1/2 study in patients with paroxysmal nocturnal hemoglobinuria (PNH) in late 2015.
ALN-AS1, a subcutaneously administered RNAi therapeutic targeting
aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic
porphyrias, including acute intermittent porphyria (AIP).
- Start Phase 1 study in mid-2015, with initial data expected in early 2016.
ALN-AAT, a subcutaneously administered RNAi therapeutic targeting
alpha-1 antitrypsin (AAT) for the treatment of AAT
deficiency-associated liver disease.
- File Investigational New Drug (IND) application or IND equivalent in mid-2015.
- Start Phase 1 study in late 2015.
ALN-GO1, a subcutaneously administered RNAi therapeutic targeting
glycolate oxidase (GO) for the treatment of primary hyperoxaluria type
- Select Development Candidate in mid-2015 to support IND or IND equivalent filing in 2016.
- Advance additional Genetic Medicine STAr programs to support 2 or more INDs in 2016.
- Across its Genetic Medicine STAr, Alnylam plans on developing and commercializing its products through direct marketing and sales in the U.S. and EU, while leveraging its alliance with Genzyme, a Sanofi company, for product commercialization in the rest-of-world (ROW).
Cardio-Metabolic Disease STAr, a pipeline of investigational RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases.
ALN-PCSsc, a subcutaneously administered RNAi therapeutic targeting
PCSK9 for the treatment of hypercholesterolemia; ALN-PCSsc is
partnered with The Medicines Company.
- Continue enrollment in Phase 1 study.
- Present initial data in mid-2015.
- Advance additional Cardio-Metabolic STAr programs to support at least 1 new IND in 2016.
- The company intends to seek strategic partnership opportunities for programs in its Cardio-Metabolic Disease STAr, while retaining significant product development and commercialization rights in the U.S. and EU.
Hepatic Infectious Disease STAr, a pipeline of investigational RNAi therapeutics that address the major global health challenges of hepatic infectious diseases.
ALN-HBV, a subcutaneously administered RNAi therapeutic targeting the
hepatitis B viral (HBV) genome for the treatment of chronic HBV
- File IND or IND equivalent in late 2015.
- Advance additional Hepatic Infectious Disease STAr programs.
- The company intends to seek strategic partnership opportunities for programs in its Hepatic Infectious Disease STAr, while retaining significant product development and commercialization rights in the U.S. and EU.
The company now expects to end 2014 with approximately
Alnylam management plans to present a company overview detailing these
goals and guidance at the 33rd Annual
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 Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme, a Sanofi company. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell,
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, its expectations regarding its STAr pipeline growth strategy, its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, its expectations for the filing of regulatory documents, the timing of the start of clinical studies and presentation of clinical data, and its expected cash position as of
Vice President, Investor Relations and Corporate Communications
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