Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes
December 21 2022 - 8:00AM
Business Wire
– Company Expects to Initiate a Phase 1/2 Study
for ALN-KHK in Early 2023, with Initial Results Expected in Late
2023 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
(RNA interference) therapeutics company, announced today that the
Company has submitted a Clinical Trial Authorization (CTA)
application to Health Canada to initiate a Phase 1/2 study of
ALN-KHK, an investigational RNAi therapeutic targeting
ketohexokinase (KHK) for the treatment of Type 2 diabetes mellitus
(T2DM). Pending regulatory authority and ethic committee approvals,
the Company plans to begin enrollment in a Phase 1/2 study in
healthy overweight to obese volunteers and obese patients with T2DM
in early 2023, and expects to report initial human data in late
2023.
“We are excited to advance ALN-KHK to the clinic as our first
RNAi therapeutic to address the unmet medical need in Type 2
diabetes mellitus and potentially other metabolic disorders,” said
Neil Wallace, Program Leader for the ALN-KHK program at Alnylam.
“Increased dietary fructose consumption, largely resulting from use
of high-fructose corn syrup as a sweetener, is believed to
contribute to the growing pandemic of obesity. Obesity is a
significant risk factor for both T2DM and fatty liver disease.
Fructose itself predisposes to fatty liver disease and thus, in the
context of T2DM, aggravates insulin resistance. We believe
targeting ketohexokinase, also known as fructokinase, the first
enzyme in the pathway of fructose metabolism, is a novel
therapeutic approach to reduce fatty acid synthesis and insulin
resistance, with the potential to improve overall glycemic control
in individuals with T2DM.”
ALN-KHK is a wholly-owned asset in the Alnylam clinical
development portfolio.
About ALN-KHK ALN-KHK is an investigational,
subcutaneously administered RNAi therapeutic targeting
ketohexokinase for the treatment of Type 2 diabetes mellitus
(T2DM). ALN-KHK utilizes Alnylam's Enhanced Stabilization Chemistry
Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous
dosing with increased selectivity and a wide therapeutic index. The
safety and efficacy of ALN-KHK have not been evaluated by the FDA,
EMA, Health Canada, or any other health authority.
About Type 2 Diabetes Mellitus Type 2 Diabetes Mellitus
(T2DM) is a global disease affecting more than 6% of the world’s
population. Its incidence and prevalence rates are believed to be
increasing worldwide with estimates of more than 450 million
affected. The prevalence of diabetes is expected to reach 55
million within the US by 2030. Management of T2DM is aimed at
achieving a target hemoglobin A1c levels (a measure of blood sugar)
of less than 7%. Typically diet and exercise are recommended with
the aim of achieving weight loss; this is rarely successful.
Despite a number of treatment options, many patients do not reach
adequate blood sugar control. There is an unmet need for a
treatment that can produce sufficient glycemic control while
providing simpler dosing administration and longer dosing intervals
that could improve treatment compliance, thereby reducing disease
complications and overall mortality.
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 new class of medicines, known as RNAi
therapeutics, is now a reality. 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 or disease pathway 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 Pharmaceuticals Alnylam Pharmaceuticals
(Nasdaq: ALNY) has led 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 and prevalent
diseases with unmet need. Based on Nobel Prize-winning science,
RNAi therapeutics represent a powerful, clinically validated
approach yielding transformative medicines. Since its founding 20
years ago, Alnylam has led the RNAi Revolution and continues to
deliver on a bold vision to turn scientific possibility into
reality. Alnylam’s commercial RNAi therapeutic products are
ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran),
AMVUTTRA® (vutrisiran) and Leqvio® (inclisiran), which is being
developed and commercialized by Alnylam’s partner, Novartis.
Alnylam has a deep pipeline of investigational medicines, including
multiple product candidates that are in late-stage development.
Alnylam is executing on its “Alnylam P5x25” strategy to deliver
transformative medicines in both rare and common diseases
benefiting patients around the world through sustainable innovation
and exceptional financial performance, resulting in a leading
biotech profile. Alnylam is headquartered in Cambridge, MA. For
more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at @Alnylam, on
LinkedIn, or on Instagram.
Alnylam Forward Looking Statements This press release
contains forward-looking statements within the meaning of Section
27A of the Securities Act of 1933 and Section 21E of the Securities
Exchange Act of 1934. All statements other than historical
statements of fact regarding Alnylam’s expectations beliefs, goals,
plans or prospects, including, without limitation, expectations
regarding Alnylam’s aspiration to become a leading biotech company
and the planned achievement of its “Alnylam P5x25” strategy, the
potential for Alnylam to identify new potential drug development
candidates and advance its research and development programs,
Alnylam’s ability to obtain approval for new commercial products or
additional indications for its existing products, and Alnylam’s
projected commercial and financial performance, should be
considered forward-looking statements. Actual results and future
plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation: the
direct or indirect impact of the COVID-19 global pandemic or any
future pandemic on Alnylam’s business, results of operations and
financial condition and the effectiveness or timeliness of
Alnylam’s efforts to mitigate the impact of the pandemic; the
potential impact of the January 2022 leadership transition on
Alnylam’s ability to attract and retain talent and to successfully
execute on its “Alnylam P5x25” strategy; Alnylam's ability to
discover and develop novel drug candidates and delivery approaches,
including using Alnylam’s IKARIA and GEMINI platforms, and
successfully demonstrate the efficacy and safety of its product
candidates, including ALN-KHK; the pre-clinical and clinical
results for its product candidates, including ALN-KHK; actions or
advice of regulatory agencies and Alnylam’s ability to obtain and
maintain regulatory approval for its product candidates, including
ALN-KHK, as well as favorable pricing and reimbursement;
successfully launching, marketing and selling its approved products
globally; delays, interruptions or failures in the manufacture and
supply of its product candidates or its marketed products;
obtaining, maintaining and protecting intellectual property;
Alnylam’s ability to successfully expand the indication for
ONPATTRO or AMVUTTRA in the future; Alnylam's ability to manage its
growth and operating expenses through disciplined investment in
operations and its ability to achieve a self-sustainable financial
profile in the future without the need for future equity financing;
Alnylam’s ability to maintain strategic business collaborations;
Alnylam's dependence on third parties for the development and
commercialization of certain products, including Novartis, Sanofi,
Regeneron and Vir; the outcome of litigation; the potential impact
of a current government investigation and the risk of future
government investigations; and unexpected expenditures; as well as
those risks more fully discussed in the “Risk Factors” filed with
Alnylam's most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) and in its other SEC
filings. In addition, any forward-looking statements represent
Alnylam's views only as of today and should not be relied upon as
representing its views as of any subsequent date. Alnylam
explicitly disclaims any obligation, except to the extent required
by law, to update any forward-looking statements.
This press release discusses investigational RNAi therapeutics
in development and is not intended to convey conclusions about
efficacy or safety as to those investigational therapeutics or
uses. There is no guarantee that any investigational therapeutics
will successfully complete clinical development or gain health
authority approval.
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Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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