Stoke Therapeutics : Corporate Presentation – October 2021

Published: 2021-10-13 16:22:34 ET STOK

Stoke Therapeutics

NASDAQ: STOK

October 2021

Disclaimer

This presentation has been prepared by Stoke Therapeutics, Inc. ("Stoke" or "our") for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or Stoke or any officer, director, employee, agent or advisor of Stoke. This presentation does not purport to be all-inclusive or to contain all of the information you may desire. Information provided in this presentation speaks only as of the date hereof. Stoke assumes no obligation to publicly update any information or forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments, subsequent events, or circumstances after the date hereof, or to reflect the occurrence of unanticipated events.

This presentation contains "forward-looking" statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of our TANGO platform to design medicines to increase protein production and the expected benefits thereof; the ability of STK-001 to treat the underlying causes of Dravet syndrome and reduce seizures; the preclinical data and study results regarding OPA1; our future operating results, financial position and liquidity; our expectation about timing and execution of anticipated milestones, responses to regulatory authorities, expected nomination of future product candidates and timing thereof. These forward-looking statements

may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will,"

"would," and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such statements, including: our ability to develop, obtain regulatory approval for and commercialize STK-001 and future product candidates, including any product candidate we may select to treat ADOA; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; the direct and indirect impact of COVID-19 on our business, financial condition and operations, including on our expenses, supply chain, strategic partners, research and development costs, clinical trials and employees; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this presentation. We do not undertake any obligation to publicly update any forward-looking statements.

By attending or receiving this presentation you acknowledge that you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made; you will be solely responsible for your own assessment of the market and our market position; and that you will conduct your own analysis and be solely responsible for forming your own view of the potential future performance of Stoke.

STOKE THERAPEUTICS

Boldly Restoring Genetic Health

Addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.

A Differentiated Platform for the Discovery and Development of Novel RNA-based Medicines

Proprietary RNA therapeutics platform

Targets pre-mRNA splicing to restore target protein to near normal levels

STOKE

THERAPEUTICS

HIGHLIGHTS

Broad therapeutic potential

~1,200 monogenic disease genes and ~6,500 additional genes with RNA target signatures

Disease-modifying approach

Clinical stage with emerging pipeline

Our compounds address the

STK-001 is being evaluated in two Phase

underlying cause of severe

1/2a studies for Dravet syndrome (DS).

genetic diseases

OPA1 is a preclinical target for autosomal

dominant optic atrophy (ADOA)

Targeted

Augmentation of

Nuclear

Gene

Output

Our compounds aim to restore protein levels by increasing protein production from the functional copy of a gene and:

Selectively boost expression only in tissues where the protein is normally expressed

Offer one drug for diseases caused by many different mutations

Apply to genes of diverse size: can be used to address small or large gene targets

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Disclaimer

Stoke Therapeutics Inc. published this content on 13 October 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 October 2021 20:21:06 UTC.