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]

MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C, is available at Zevra’s specialty pharmacy for dispense AmplifyAssist™, Zevra’s comprehensive patient support program, in place to address access barriers CELEBRATION, Fla., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that MIPLYFFA™ (MY-PLY-FAH) (arimoclomol), the first treatment approved by the U.S. Food an ...

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, 2024 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 Number: 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other J ...

Zevra Therapeutics, Inc. (NASDAQ:ZVRA) Q3 2024 Earnings Conference Call November 12, 2024 4:30 PM ET Company Participants Nichol Ochsner - VP, IR & Corporate Communications Neil McFarlane - President & CEO LaDuane Clifton - CFO Joshua Schafer - Chief Commercial Officer & EVP, Business Development Conference Call Participants Jason Butler - JMP Securities Louise Chen - Cantor Fitzgerald Samantha Corwin - William Blair Eddie Hickman - Guggenheim Securities Sumant Kulkarni - Canaccord Genuity Oren Livnat - H.C ...