Zevra Therapeutics, Inc. · NASDAQ:ZVRA
Zevra Therapeutics — Kempharm — Merger or Acquisition (May 2022)
18 slides from Zevra Therapeutics's merger presentation, May 16, 2022
Slide 1: Arimoclomol Acquisition Call
Exhibit 99.2 Arimoclomol Acquisition Call May 16, 2022 1 1 Trademarks herein are held by their respective owners.
Slide 2: Cautionary Note Regarding Presentation Information
Cautionary Note Regarding Presentation Information This presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, includin
Slide 3: KemPharm Value Proposition
KEMPHARM VALUE PROPOSITION Innovative Two FDA approved and pharmaceutical Focus on high-value partnered medications, company discovering areas with significant ® AZSTARYS and and developing novel unmet needs with ® APADAZ , validate treatments for rare CNS potential to internally approach and regula
Slide 4: Arimoclomol Acquisition - Expanding Pipeline Targeting Rare Diseases
Arimoclomol Acquisition - Expanding Pipeline Targeting Rare Diseases Definitive agreement with Orphazyme A/S to acquire substantially all assets, including arimoclomol • This acquisition is a significant expansion of KemPharm’s development pipeline targeting rare diseases; allows KemPharm to leverag
Slide 5: Definitive Agreement – Summary of Terms
Definitive Agreement – Summary of Terms • KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, with a cash payment of USD $12.8 million o Cash payment will be financed with a revolving line of credit secured by KemPharm’s balance sheet • KemPharm
Slide 6: Product Overview - Arimoclomol
Product Overview - Arimoclomol Disease State, Market Overview and Regulatory Pathway 6 6
Slide 7: About Niemann-Pick Disease Type C (NPC)
1 About Niemann-Pick Disease Type C (NPC) • Ultra-rare progressive lysosomal storage disorder characterized by an inability of the body to transport cellular cholesterol and lipids o Leads to dysfunction in organs such as the brain, spleen and liver o Disease progression is irreversible in all patie
Slide 8: Arimoclomol – Innovative Product for a High Unmet Need
Arimoclomol – Innovative Product for a High Unmet Need • First-in-class, oral treatment intended for NPC o Capsule formulation designed to be swallowed whole, opened to allow contents to be mixed with soft foods/liquids or delivered through a gastric feeding tube o Nonclinical and clinical evidence
Slide 9: Overview of Regulatory Pathway in the U.S. – NDA Resubmission Process
Overview of Regulatory Pathway in the U.S. – NDA Resubmission Process • Orphazyme received a Complete Response Letter (CRL) from the FDA on Jun 17, 2021, regarding their NDA for arimoclomol for the treatment of NPC o The FDA identified three issues: 1) Additional evidence needed to substantiate vali
Slide 10: FDA Agreed with Proposed Plan to Strengthen Primary Endpoint and Analysis
FDA Agreed with Proposed Plan to Strengthen Primary Endpoint and Analysis 1) Conduct a new analysis of the original 5-domain NPCCSS primary endpoint by removing the cognition domain (ambulation, swallowing, speech and fine motor skills remain) to form a new 4-domain NPCCSS endpoint ü FDA confirmed t
Slide 11: Modification of Primary Endpoint Demonstrates Improved Statistical Significance and Treatment Effect
Modification of Primary Endpoint with FDA Agreement Demonstrates Improved Statistical Significance and Treatment Effect 1 1 Full Population Migulstat Subgroup Analysis 2 2 Treatment Effect p-value Treatment Effect p-value Prespecified 5-domain NPCCSS -1.4 0.0456 -2.06 0.0060 Prespecified 4-domain NP
Slide 12: Bolstering Confirmatory Evidence Addresses Another Key Issue Raised in CRL
Bolstering Confirmatory Evidence Addresses Another Key Issue Raised in CRL • Since the CRL and Type A meeting, additional data has already been generated o Numerous studies and additional analysis has been conducted to address this issue o Primary focus was to confirm or elucidate the mechanism of a
Slide 13: Path to Resubmission and Approval Appears Straightforward
Path to Resubmission and Approval Appears Straightforward • Current plan to address the major issues related to the CRL appear addressable ü Primary endpoint work has concluded ü Additional analyses have been conducted ü Confirmatory evidence has been significantly augmented • KemPharm has significa
Slide 14: Commercial Opportunity Potential to Transform KemPharm's Business Model
Commercial Opportunity Potential to Transform KemPharm’s Business Model 14 14
Slide 15: Arimoclomol Market Opportunity Is Compelling, Even With Ultra-Rare Status
Arimoclomol Market Opportunity Is Compelling, Even With Ultra-Rare Status • Upon approval, KemPharm would currently be eligible to receive a Rare Pediatric Disease Priority Review Voucher o Last voucher sold for $110M, which was within the typical range of approx. $100M/voucher o Program could event
Slide 16: Arimoclomol Provides Opportunity to Commercialize and Retain Full Market Value
Arimoclomol Provides KemPharm the Opportunity to Commercialize and Retain Full Market Value • Arimoclomol is an NDA-stage, revenue-generating product upon which we intend to build commercial capabilities that fit with the goal of allowing KemPharm to create and retain value for the benefit of shareh
Slide 17: Full Pipeline of Product Candidates with Substantial Milestones in 2022 and Beyond
Full Pipeline of Product Candidates with Substantial Milestones in 2022 and Beyond PROGRAM PRECLINICAL CLINICAL DEVELOPMENT NDA NEXT MILESTONE ARIMOCLOMOL Niemann-Pick disease type C Re-file NDA: (NPC) Q1 2023 KP1077 Initiate Phase 2 Idiopathic Hypersomnia (IH) Trial: H2 2022 Narcolepsy Initiate Pha
Slide 18: For additional information please contact
For additional information please contact: Jason Rando jrando@tiberend.com 18 18