Corporate Overview

December 2020

Forward-Looking Statements

THE STATEMENTS IN THIS PRESENTATION MAY INCLUDE FORWARD-LOOKING STATEMENTS WITHIN THE MEANING OF THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995. THESE STATEMENTS, AMONG OTHER THINGS RELATE TO THE FUTURE OPERATIONS, OPPORTUNITIES OR FINANCIAL PERFORMANCE OF ZYNERBA PHARMACEUTICALS, INC. WE MAY, IN SOME CASES, USE TERMS SUCH AS "PREDICTS," "BELIEVES," "POTENTIAL," "PROPOSED," "CONTINUE," "ESTIMATES," "ANTICIPATES," "EXPECTS," "PLANS," "INTENDS," "MAY," "COULD," "MIGHT," "WILL," "SHOULD" OR OTHER WORDS THAT CONVEY UNCERTAINTY OF FUTURE EVENTS OR OUTCOMES TO IDENTIFY THESE FORWARD-LOOKING STATEMENTS. SUCH STATEMENTS ARE SUBJECT TO NUMEROUS IMPORTANT FACTORS, RISKS AND UNCERTAINTIES THAT MAY CAUSE ACTUAL EVENTS OR RESULTS TO DIFFER MATERIALLY FROM THE COMPANY'S CURRENT EXPECTATIONS, INCLUDING THE FOLLOWING: THE COMPANY'S CASH AND CASH EQUIVALENTS MAY NOT BE SUFFICIENT TO SUPPORT ITS OPERATING PLAN FOR AS LONG AS ANTICIPATED; THE RESULTS, COST AND TIMING OF THE COMPANY'S CLINICAL DEVELOPMENT PROGRAMS, INCLUDING ANY DELAYS TO SUCH CLINICAL TRIALS RELATING TO ENROLLMENT OR SITE INITIATION; CLINICAL RESULTS FOR THE COMPANY'S PRODUCT CANDIDATES MAY NOT BE REPLICATED OR CONTINUE TO OCCUR IN ADDITIONAL TRIALS AND MAY NOT OTHERWISE SUPPORT FURTHER DEVELOPMENT IN A SPECIFIED INDICATION OR AT ALL; ACTIONS OR ADVICE OF THE U.S. FOOD AND DRUG ADMINISTRATION AND FOREIGN REGULATORY AGENCIES MAY AFFECT THE DESIGN, INITIATION, TIMING, CONTINUATION AND/OR PROGRESS OF CLINICAL TRIALS OR RESULT IN THE NEED FOR ADDITIONAL CLINICAL TRIALS; THE COMPANY'S ABILITY TO OBTAIN AND MAINTAIN REGULATORY APPROVAL FOR ITS PRODUCT CANDIDATES, AND THE LABELING UNDER ANY SUCH APPROVAL; AND THE COMPANY'S EXPECTATIONS REGARDING ITS ABILITY TO OBTAIN AND ADEQUATELY MAINTAIN SUFFICIENT INTELLECTUAL PROPERTY PROTECTION FOR ITS PRODUCT CANDIDATES. THESE AND OTHER RISKS ARE DESCRIBED IN OUR FILINGS WITH THE SECURITIES AND EXCHANGE COMMISSION, AVAILABLE AT WWW.SEC.GOV. ANY FORWARD-LOOKING STATEMENTS THAT THE COMPANY MAKES IN THIS PRESENTATION SPEAK ONLY AS OF THE DATE OF THIS PRESENTATION. THE COMPANY ASSUMES NO OBLIGATION TO UPDATE FORWARD-LOOKING STATEMENTS WHETHER AS A RESULT OF NEW INFORMATION, FUTURE EVENTS OR OTHERWISE, AFTER THE DATE OF THIS PRESENTATION.

© 2020 Zynerba Pharmaceuticals, Inc. All rights reserved. Zynerba and Zygel are trademarks of Zynerba Pharmaceuticals, Inc. All other trademarks and registered trademarks are property of their respective owners.

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Zynerba Pharmaceuticals (NASDAQ: ZYNE)

A Rare/Near-Rare Neuropsychiatric Company

  • Deep pipeline targeting high unmet medical needs; translating into multi-billion dollar market opportunity with Zygel™ (Cannabidiol Gel)
  • Focused on completing development of Zygel in Fragile X Syndrome (FXS)
    • Pre-plannedad hoc analyses of CONNECT-FX data identified patient population who appear to benefit from Zygel
    • As a result of meeting with FDA in 4Q2020, Zynerba plans to conduct a trial in patients with a highly methylated FMR1 gene to confirm positive results seen in CONNECT-FX responders
  • Continuing to pursue three additional neuropsychiatric indications:
    • 22q11.2 deletion syndrome (22q) - Phase 2 ongoing
    • Autism spectrum disorder (ASD) - Phase 2 complete
    • Developmental and epileptic encephalopathies (DEE) - Phase 2 complete
  • Experienced team with development and commercial expertise in transdermal delivery, orphan diseases, neurology, and psychiatry
  • Cash runway expected to be sufficient to fund operations into 2023

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Deep Clinical Pipeline & Near-term Milestones

Zygel

Cannabidiol Gel

Expected Milestones

Review trial design and protocol

with FDA in 1H21; initiate

confirmatory pivotal trial before the

end of 2021

Completion of enrollment once COVID-19 restrictions in Australia are eased

Discuss Phase 2 results and

regulatory path forward with FDA

in 1H2021

Finalize target syndrome selection

in 2021

*Orphan Drug and Fast Track designation **Orphan Drug designation

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Zynerba Pharmaceuticals Inc. published this content on 17 December 2020 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 02 January 2021 16:17:06 UTC