UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period From to Commission File No. 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Jurisdiction of Incorporation or Organization) (I.R.S. Employer Identification No.) FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURI ...

Financial Data and Key Metrics Changes - For the full year 2022, net revenue was $10.5 million, with Q4 net revenue at $2.3 million, primarily driven by reimbursements from the French EAP program [20][34] - The company reported a Q4 net loss of $9 million or $0.26 per share, and a full year net loss of $41.5 million or $1.20 per share, which includes a one-time noncash charge of $17.7 million related to in-process R&D assets [35][36] - Cash, cash equivalents, and investments as of December 31, 2022, were $102.9 million, expected to extend the cash runway into 2026 [20][36] Business Line Data and Key Metrics Changes - The arimoclomol program for Niemann-Pick Disease Type C is on track for NDA resubmission as early as Q3 2023, with positive interim data from a Phase II/III safety study presented at the 2023 WORLDSymposium [21][24] - The KP1077 program for rare sleep disorders is currently enrolling patients in a Phase II clinical study, with interim data expected by Q3 2023 and top-line data by the end of 2023 [27][29] Market Data and Key Metrics Changes - The prescription trend for AZSTARYS has improved since July 2022, with expectations to achieve sales milestones in 2023 [30][31] - Net revenue from the French EAP program is expected to continue at approximately $2 million per quarter throughout the year [37] Company Strategy and Development Direction - The company has shifted its focus exclusively to rare diseases, leveraging its strengths in drug development and regulatory approval to create value for shareholders [7][10] - The acquisition of arimoclomol is a strategic move to build commercial capabilities and retain market value for shareholders [10][15] - The company aims to commercialize its products in the U.S. and explore opportunities in other geographies, including the EU, Japan, and China [26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's strong financial foundation and diverse portfolio, anticipating multiple value-creating milestones in 2023 [18][41] - The ongoing discussions with regulatory agencies are seen as productive, with preparations for NDA resubmission for arimoclomol [39][40] - The company is committed to creating value for shareholders while addressing the needs of patients with rare diseases [41][47] Other Important Information - The company has undergone a rebranding to Zevra Therapeutics to better reflect its commitment to rare disease patients [15][22] - The leadership team has been strengthened with new appointments, enhancing the company's capabilities in R&D and commercialization [21][23] Q&A Session Summary Question: Has there been any specific interactions with the FDA since your last update? - Management indicated that there have not been formal interactions with the FDA regarding the resubmission of the arimoclomol NDA [50] Question: What sales threshold triggers the AZSTARYS milestones? - The company expects to achieve one or two sales milestones likely toward the second half of 2023, but specific thresholds could not be disclosed [51][52] Question: Will the Phase II trial data for idiopathic hypersomnia influence the narcolepsy trial? - Management stated that they will evaluate the Phase II data before deciding on a parallel program for narcolepsy [53] Question: What is the current IP landscape of development assets? - The company has patents for arimoclomol that extend into the 2040s, with a focus on extending exclusivity beyond the orphan period [65][67] Question: What are the necessary hurdles for KP1077 approvability? - Management emphasized that while primary endpoints are crucial for approvability, additional exploratory endpoints will enhance the product's market competitiveness [72]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____ to _____ Commission File No. 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Jurisdiction of I ...

KemPharm, Inc. (KMPH) Q3 2022 Results Conference Call November 9, 2022 5:00 PM ET Company Participants Nichol Ochsner - Vice President of Investor Relations Travis Mickle - President and Chief Executive Officer LaDuane Clifton - Chief Financial Officer Conference Call Participants Sumant Kulkarni - Canaccord Jonathan Aschoff - Roth Capital Partners Operator Good afternoon, and welcome to the KemPharm Third Quarter 2022 Corporate and Financial Results Conference Call. Currently, all callers have been placed ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 Commission File No. 001-36913 KemPharm, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Jurisdiction of Incorporation or Organization) (I.R.S. Employer Identification No.) 1180 Celebration Boulevard, Suite 103, Celebration, FL 3474 ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period From to Commission File No. 001-36913 KemPharm, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Jurisdiction of Inco ...

KemPharm, Inc. (KMPH) Q2 2022 Earnings Conference Call August 11, 2022 5:00 PM ET Company Participants Nichol Ochsner - Vice President-Investor Relations & Corporate Communications Travis Mickle - President & Chief Executive Officer Perry Sternberg - Chief Executive Officer, Corium Inc. LaDuane Clifton - Chief Financial Officer Conference Call Participants Jonathan Aschoff - ROTH Capital Partners Operator Good afternoon, and welcome to the KemPharm Second Quarter 2020 Corporate and Financial Results Confere ...

Company Overview - KemPharm is focused on developing and commercializing treatments for rare CNS, neurodegenerative, and lysosomal storage diseases[7] - The company has a diverse product portfolio with clinical-stage pipeline assets, revenue-generating NDA-stage assets, and commercial assets[7] - KemPharm's strategic focus is on CNS/rare diseases, with potential for internal commercialization[15] Arimoclomol (Niemann-Pick Disease Type C) - KemPharm acquired arimoclomol, an NDA-stage drug candidate for Niemann-Pick disease type C (NPC), for $128 million[24] - The French EAP for arimoclomol is expected to generate revenue exceeding $12 million in FY 2022[22, 24] - The company plans to resubmit the NDA for arimoclomol in NPC as early as Q1 2023[15, 39] KP1077 (Idiopathic Hypersomnia) - KP1077 is being developed for the treatment of Idiopathic Hypersomnia (IH), a high-value opportunity with significant unmet need[16] - A Phase 1 clinical trial assessing the cardiovascular safety of SDX (KP1077) is expected to have topline results in Q3 2022[59] - Approximately 37,000 patients are diagnosed with IH and actively seeking treatment[46, 68] AZSTARYS® (ADHD) - AZSTARYS® is a partnered medication for ADHD, providing ongoing revenue potential from royalties and milestones[7, 17] - Over 110 million commercial and Medicaid lives have access to AZSTARYS as of March 1, 2021[79] Financial Position - As of March 31, 2022, KemPharm had $1191 million in cash, cash equivalents, marketable securities, and long-term investments[17, 84, 90] - The company's cash runway extends beyond 2025[17, 84, 90]

Management Presentation March 2022 11 Trademarks herein are held by their respective owners. 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, including without limitation and can be identified by the use of words such as "may," "will," "expect," "project," "estimate," ...

KemPharm, Inc. (KMPH) Q1 2022 Earnings Conference Call May 12, 2022 5:00 PM ET Company Participants Travis Mickle - Co-Founder, President, CEO & Director LaDuane Clifton - CFO, Secretary & Treasurer Conference Call Participants Jason Rando - Tiberend Strategic Advisors Operator Thank you for standing by, and welcome to the KemPharm First Quarter 2022 Results Conference Call. [Operator Instructions]. I would now like to hand the call over to your host, Jason Rando with Tiberend Strategic Advisors. Please go ...