Harmony Biosciences : Development Program for Pitolisant in Patients with Myotonic Dystrophy (DM)

Development Program for Pitolisant in Patients with Myotonic Dystrophy

Analyst Event June 17, 2021

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This presentation includes forward‐looking statements within the meaning of the Private Securities Reform Act of 1995. All statements other than statements of historical facts contained in these materials or elsewhere, including statements regarding Harmony Biosciences Holdings, Inc.'s (the "Company") future financial position, business strategy and plans and objectives of management for future operations, should be considered forward-looking statements. Forward-looking statements use words like "believes," "plans," "expects," "intends," "will," "would," "anticipates," "estimates," and similar words or expressions in discussions of the Company's future operations, financial performance or the Company's strategies. These statements are based on current expectations or objectives that are inherently uncertain, especially in light of the Company's limited operating history. These and other important factors discussed under the caption "Risk Factors" in the Company's Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (the "SEC") on March 25, 2021 and its other filings with the SEC could cause actual results to differ materially and adversely from those indicated by the forward-looking statements made in this presentation. While the Company may elect to update such forward-looking statements at some point in the future, it disclaims any obligation to do so, even if subsequent events cause its views to change.

This presentation includes information related to market opportunity as well as cost and other estimates obtained from internal analyses and external sources. The internal analyses are based upon management's understanding of market and industry conditions and have not been verified by independent sources. Similarly, the externally sourced information has been obtained from sources the Company believes to be reliable, but the accuracy and completeness of such information cannot be assured. Neither the company, nor any of its respective officers, directors, managers, employees, agents, or representatives, (i) make any representations or warranties, express or implied, with respect to any of the information contained herein, including the accuracy or completeness of this presentation or any other written or oral information made available to any interested party or its advisor (and any liability therefore is expressly disclaimed), (ii) have any liability from the use of the information, including with respect to any forward-looking statements, or (iii) undertake to update any of the information contained herein or provide additional information as a result of new information or future events or developments.

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Topics to be Covered

• Life cycle management (LCM) strategy for pitolisant
• Overview of Myotonic Dystrophy (DM)
• Patient insights
• Scientific rationale for investigating pitolisant in patients with DM
• Phase 2 clinical trial of pitolisant in patients with DM1
• Concluding remarks

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Pitolisant: Portfolio in a Product Opportunity

Mechanism-based approach to drug development and initial LCM studies based on:

• Role of histamine in normal physiologic functioning
• Role of histamine in disorders of orexin deficiency
• Location of H3 receptors throughout the
  CNS
• Limited H3 receptor populations outside the CNS
• Proven clinical efficacy of pitolisant for
  EDS

Cerebral cortex:

• • Attention; vigilance
• Executive functioning
• • Cognition

Limbic Circuit:

• Emotional control

• Behavioral processing

• Consolidation of memory

Hypothalamus:

• Sleep-wakeregulation
• • Hunger-satiety

• Activation of the cortex

Brainstem:

• Integration of neural circuitry
  (NE, 5-HT, DA, ACh)
• • REM-SleepCenters
  • Descending control of neuromuscular system

Pitolisant has a unique MOA with potential for multiple additional indications in

rare disease patient populations with unmet medical needs

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Life Cycle Management for Pitolisant

"Portfolio in a Product"

Evolving Beyond Sleep & Wakefulness

	New Indications
	Based on H3R MOA
Label Expansion	in Additional Rare
Disease Patient
in Narcolepsy	Populations

Building industry

leadership in narcolepsy

Excessive daytime sleepiness (EDS) primary endpoint and exploring new clinical endpoints related to attention/vigilance, cognitive function, behavior and fatigue

•	Pediatric Narcolepsy indication	•	Prader-Willi Syndrome (PWS)
•	Pediatric Exclusivity	•	Myotonic Dystrophy (DM)

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