Alnylam Announces 2018 Corporate Goals
Jan 07,2018
Patisiran Expected to be First Commercially Available RNAi Therapeutic in Mid-2018
Restructured
Givosiran Interim Phase 3 Data Expected in Mid-2018 and U.S. New Drug Application Filing Anticipated in Late 2018
Company Plans to Advance Three Additional Phase 3 Programs: Fitusiran, Inclisiran, and ALN-TTRsc02
Company Updates Guidance to End 2017 with
"In the year ahead, we look forward to the potential global commercial
launch of patisiran, the first-ever RNAi therapeutic to enter the
market, marking the birth of a whole new class of medicines with the
potential to transform the treatment of diseases with high unmet need.
Together with the advancements made in our other late-stage programs, we
now look forward to our transition from a development-stage company
toward a multi-product, global commercial-stage biopharmaceutical
company with a deep and sustainable pipeline and research engine," said
“The recently restructured alliance with
2018 Corporate Goals
Patisiran, an investigational RNAi therapeutic currently under regulatory review for the treatment of hATTR amyloidosis. Alnylam plans to:
- Present additional data from the APOLLO Phase 3 study in early, mid-, and late 2018;
-
File for regulatory approval in
Japan and other global markets, including a Japanese NDA filling with the Pharmaceuticals andMedical Device Agency (PMDA) in mid-2018; - File for regulatory approvals in additional rest-of-world countries in late 2018;
-
Gain regulatory approvals from the
U.S. Food and Drug Administration and theEuropean Medicines Agency in mid- and late 2018, respectively; and - Assuming regulatory approval, launch patisiran in the U.S. and EU in mid- and late 2018, respectively.
Givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyrias (AHPs). Alnylam plans to:
- Present Phase 1/2 open-label extension (OLE) data in early 2018;
- Report topline interim results from the ENVISION Phase 3 trial in mid-2018;
- Assuming a positive interim readout, file for accelerated regulatory approval with submission of a New Drug Application (NDA) in the U.S. in late 2018; and
- Complete enrollment in the ENVISION Phase 3 study in late 2018.
ALN-TTRsc02, an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis. Alnylam plans to:
- Initiate a Phase 3 pivotal study program in late 2018.
Alnylam also intends to support Sanofi’s efforts in advancing fitusiran,
an investigational RNAi therapeutic in development for the treatment of
hemophilia and rare bleeding disorders. Throughout 2018,
Alnylam also plans to support The
In addition, the Company plans to continue advancement of its earlier-stage clinical pipeline programs with multiple data read-outs expected throughout 2018. Alnylam also plans to file one or more new clinical trial applications (CTA) in 2018, and advance its infectious disease collaboration with Vir Biotechnology.
The Company now expects to end 2017 with greater than $1.7 billion in cash, cash equivalents, marketable securities, and restricted cash and intends to provide financial guidance for 2018 in connection with its year-end 2017 financial results in February.
Alnylam management will present a company overview detailing these goals
and guidance at the 36th Annual J.P. Morgan Healthcare
Conference tomorrow, Monday, January 8, 2018 at 8:00 a.m. PT (
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, 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, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About
Alnylam (Nasdaq:ALNY) is leading the translation of RNA interference
(RNAi) into a whole new class of innovative medicines with the potential
to transform the lives of people afflicted with rare genetic,
cardio-metabolic, and hepatic infectious diseases. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
severe and debilitating diseases. Founded in 2002, Alnylam is delivering
on a bold vision to turn scientific possibility into reality, with a
robust discovery platform and deep pipeline of investigational
medicines, including four product candidates that are in late-stage
development. Looking forward, Alnylam will continue to execute on its
"Alnylam 2020" strategy of building a multi-product, commercial-stage
biopharmaceutical company with a sustainable pipeline of RNAi-based
medicines to address the needs of patients who have limited or
inadequate treatment options. Alnylam employs over 700 people in the
U.S. and
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 patisiran, ALN-TTRsc02, givosiran, fitusiran, and inclisran,
its expectations regarding the potential regulatory approval and
commercial launch of patisiran in
None of Alnylam’s investigational therapeutics have been approved by the
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Source:
Alnylam Pharmaceuticals, Inc.
Investors and Media
Christine
Regan Lindenboom, 617-682-4340
or
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Josh Brodsky,
617-551-8276