Zevra Therapeutics, Inc. (NASDAQ:ZVRA) Q4 2024 Earnings Conference Call March 11, 2025 4:30 PM ET Company Participants Nichol Ochsner - VP, IR and Corporate Communications Neil McFarlane - President and CEO LaDuane Clifton - CFO Joshua Schafer - Chief Commercial Officer and EVP, Business Development Adrian Quartel - Chief Medical Officer Conference Call Participants Kristen Kluska - Cantor Jason Butler - Citizens JMP Sumant Kulkarni - Canaccord Oren Livnat - H.C. Wainwright Samantha Corwin - William Blair E ...

Financial Performance - FY 2024 net revenue was $23.6 million, with Q4 2024 product net revenue reaching a record $10.2 million[1] - Net revenue for the year ended December 31, 2024, was $23,612,000, a decrease of 14% from $27,461,000 in 2023[39] - The net loss for FY 2024 was ($105.5) million, or ($2.28) per share, compared to a net loss of ($46.0) million, or ($1.30) per share in FY 2023[9] - Net loss for 2024 was $105,511,000, compared to a net loss of $46,049,000 in 2023, representing a 129% increase in losses[39] Revenue Sources - MIPLYFFA generated $10.1 million in net revenue for Q4 2024, while OLPRUVA contributed $0.1 million[12] Operating Expenses - Operating expenses for FY 2024 totaled $97.0 million, with R&D expenses at $42.1 million, reflecting a $2.3 million increase from FY 2023[9] - Total operating expenses increased to $96,963,000 in 2024, up 31% from $74,120,000 in 2023[39] - Research and development expenses rose to $42,095,000 in 2024, an increase of 5% from $39,806,000 in 2023[39] - Selling, general and administrative expenses surged to $54,868,000 in 2024, up 60% from $34,314,000 in 2023[39] - SG&A expenses for Q4 2024 were $16.1 million, an increase of $1.4 million compared to Q4 2023[12] Cash and Investments - As of Dec. 31, 2024, cash, cash equivalents, and investments were $75.5 million, with a cash runway forecast extending into 2029[9] - Cash and cash equivalents decreased to $33,785,000 as of December 31, 2024, down from $43,049,000 in 2023[41] Assets and Liabilities - Total assets increased to $178,127,000 in 2024, compared to $172,327,000 in 2023[42] - Total liabilities rose to $138,461,000 in 2024, up from $110,463,000 in 2023, indicating a 25% increase[41] Share Information - The weighted average number of shares outstanding increased to 46,251,239 in 2024 from 35,452,460 in 2023[39] Regulatory and Market Activities - Zevra initiated the commercial launch of MIPLYFFA on Nov. 21, 2024, following FDA approval on Sep. 20, 2024[5] - The company plans to file for regulatory approval of MIPLYFFA in the EU in the second half of 2025[5] - The company received 109 total prescription enrollment forms for MIPLYFFA as of Dec. 31, 2024[5] - Zevra entered into an asset purchase agreement for the sale of a Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million[8] Focus on Rare Diseases - The company has received Orphan Drug designation for both Celiprolol and KP1077, indicating a focus on rare disease treatments[31][32]

Core Insights - Zevra Therapeutics reported FY 2024 net revenue of $23.6 million, with Q4 2024 net revenue of $12.0 million, driven by the commercial launch of MIPLYFFA [1][11] - The company entered into an asset purchase agreement for the sale of a Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million [6] - Zevra's strategic plan focuses on four pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation [2] Financial Highlights - Q4 2024 net revenue breakdown: $10.1 million from MIPLYFFA, $0.1 million from OLPRUVA, $1.1 million in net reimbursements from the French EAP for arimoclomol, and $0.7 million in royalties under the AZSTARYS license agreement [11] - Operating expenses for Q4 2024 were $24.5 million, including $4.0 million in non-cash stock compensation [10] - FY 2024 net loss was $105.5 million, or $2.28 per share, compared to a net loss of $46.0 million, or $1.30 per share in FY 2023 [12][37] Commercial Launch and Pipeline - MIPLYFFA was launched immediately after FDA approval on September 20, 2024, with U.S. commercial availability starting on November 21, 2024 [8] - As of December 31, 2024, Zevra received 109 total prescription enrollment forms for MIPLYFFA [8] - The company is targeting regulatory approval in the EU for MIPLYFFA, with a Marketing Authorisation Application filing planned for the second half of 2025 [8] Talent and Culture - In Q4 2024, Zevra consolidated its development and scientific functions under the leadership of its Chief Medical Officer, Adrian Quartel, M.D. [5] Corporate Foundation - The sale of the PRV is expected to provide net proceeds of $148.3 million after fees, enhancing the company's cash position [10][11] - As of December 31, 2024, Zevra had cash, cash equivalents, and investments totaling $75.5 million, extending its cash runway into 2029 [11]

Core Points - Zevra Therapeutics launched a new awareness campaign titled 'Learn NPC, Read Between the Signs' on Rare Disease Day to promote early recognition and diagnosis of Niemann-Pick disease type C (NPC) [1][2][3] - The campaign aims to provide educational resources and testing information to healthcare professionals and treatment teams in the U.S. [2][3] - MIPLYFFA (arimoclomol), Zevra's approved therapy for NPC, was highlighted, which received FDA approval on September 20, 2024, and has shown to halt disease progression in clinical trials [4][5] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases with significant unmet needs [15] - The company emphasizes a data-driven approach to drug development and commercialization to address challenges in bringing new therapies to the market [15] Disease Information - Niemann-Pick disease type C (NPC) is characterized as an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder, leading to cholesterol accumulation in cells, particularly neurons [14] - The disease can affect both children and adults, presenting with varying symptoms and often resulting in irreversible disease progression and early mortality [14]

Core Viewpoint - Zevra Therapeutics has launched a new awareness campaign, 'Learn NPC, Read Between the Signs,' on Rare Disease Day to promote early recognition and diagnosis of Niemann-Pick disease type C (NPC), an ultra-rare neurodegenerative disorder [2][3][4] Company Initiatives - The campaign aims to provide educational resources and testing information to healthcare professionals and treatment teams in the U.S. to improve the diagnosis of NPC [3][4] - Zevra's Chief Commercial Officer emphasized the company's commitment to addressing unmet needs in rare diseases and supporting the NPC community [3] Product Information - MIPLYFFA (arimoclomol) is Zevra's approved therapy for NPC, which received FDA approval on September 20, 2024 [5] - The drug works by activating transcription factors that regulate lysosomal expression and has shown to halt disease progression in a pivotal phase 3 trial [5] - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [6] Disease Overview - Niemann-Pick disease type C is characterized by the body's inability to transport cholesterol and lipids, leading to their accumulation in cells, particularly neurons [15] - The disease presents with varying symptoms and can lead to significant physical and cognitive limitations, often resulting in early mortality [15] Company Background - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to create transformational therapies through data-driven strategies [16]

Core Insights - Zevra Therapeutics, Inc. has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, expected to close within 30 to 45 days, subject to customary closing conditions [1][2] Financial Impact - The sale of the PRV will provide non-dilutive capital, strengthening the company's balance sheet with an addition of $150 million in gross cash proceeds [2] - This capital will support continued investment in strategic priorities, including the commercial launches of MIPLYFFA™ and OLPRUVA®, as well as advancing the pipeline of product candidates for rare diseases [2] Product and Regulatory Context - The PRV was granted to Zevra in September 2024 following the FDA approval of MIPLYFFA (arimoclomol), which is indicated for treating neurological manifestations of Niemann-Pick disease type C (NPC) in patients aged 2 years and older [2] - The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act (HSR) [2]

Core Viewpoint - Zevra Therapeutics, Inc. will report its corporate and financial results for Q4 and full year 2024 on March 11, 2025, and will host a conference call at 4:30 p.m. ET on the same day [1]. Group 1 - The company focuses on providing therapies for individuals with rare diseases, aiming to create transformational therapies where limited or no treatment options exist [3]. - Zevra employs unique, data-driven development and commercialization strategies to address complex drug development challenges [3]. - A link to the audio webcast of the conference call will be available on the "Events & Presentations" page of Zevra's website [2]. Group 2 - A replay of the conference call will be accessible for 90 days starting approximately at 5:30 p.m. ET on the same day [2]. - For further information, Zevra's website and social media platforms can be visited [4]. - Contact information for inquiries is provided, including a dedicated conference ID for the call [6].

Core Insights - Zevra Therapeutics, Inc. is focused on developing therapies for rare diseases with limited treatment options, aiming to provide life-changing therapeutics to affected individuals [2]. Company Events - Zevra's executive leadership team will participate in the TD Cowen 45th Annual Health Care Conference on March 5, 2025, at 10:30 a.m. ET, and the 37th Annual ROTH Conference on March 17, 2025, at 1:00 p.m. PT [1][5]. - Management will be available for one-on-one meetings with registered attendees at each conference [1]. Company Overview - Zevra combines science, data, and patient needs to create transformational therapies, overcoming complex drug development challenges [2]. - The company utilizes unique, data-driven development and commercialization strategies to make new therapies available to the rare disease community [2]. Communication Channels - For more information, Zevra can be followed on X (formerly Twitter) and LinkedIn, and additional details are available on their website [3].

Core Points - Zevra Therapeutics will receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium, recognizing advancements in treating lysosomal diseases [2][3] - Eight abstracts related to MIPLYFFA have been accepted for presentation, including one selected for oral presentation by Dr. Eugen Mengel [1][4] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on rare disease therapeutics, aiming to develop transformational therapies for diseases with limited treatment options [21] - The company emphasizes a data-driven approach to overcome drug development challenges and meet patient needs [21] Product Information - MIPLYFFA (arimoclomol) is indicated for treating neurological manifestations of Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat [10] - The drug has received multiple designations from the FDA, including Breakthrough Therapy and Orphan Drug designations, highlighting its significance in treating NPC [9] Clinical Research and Presentations - The pivotal phase 3 trial of MIPLYFFA demonstrated that it halted disease progression compared to placebo over a one-year period, as measured by the NPC Clinical Severity Scale [9] - The WORLDSymposium will feature various presentations on MIPLYFFA, including efficacy results from a 12-month double-blind randomized trial [4][5][6] Event Participation - Zevra will participate in the WORLDSymposium taking place from February 3-7, 2025, in San Diego, CA, where attendees can visit their booth [7][8]

Core Points - Zevra Therapeutics, Inc. will be added to the Nasdaq Biotechnology Index effective December 23, 2024, which is part of the annual reconstitution of the Nasdaq indexes [1] - The addition to the Nasdaq Biotechnology Index is expected to enhance Zevra's visibility among biotech funds and portfolio managers, reflecting a transformational year for the company [2] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases, aiming to address unmet medical needs [5] - The company employs unique, data-driven strategies for drug development and commercialization to overcome challenges in bringing new therapies to the rare disease community [5] Index Information - The Nasdaq Biotechnology Index tracks the performance of securities classified as biotechnology or pharmaceutical, calculated using a modified capitalization-weighted methodology [3] - Companies must meet specific eligibility criteria, including minimum market capitalization and average daily trading volume, to be included in the index [3]