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Alnylam Submits CTA Application for ALN-KHK, an Investigational RNAi Therapeutic for the Treatment of Type 2 Diabetes

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.


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 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.


Alnylam Pharmaceuticals, Inc.

Christine Regan Lindenboom

(Investors and Media)


Josh Brodsky



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