Celiprolol Hydrochloride

CELEBRATION, Fla., April 29, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, Zevra’s President and Chief Executive Officer, has issued a Letter to Stockholders providing a review of key accomplishments and outlook for 2024. Dear Stockholders, It is hard for me to believe that April 2024 marks six months since the start of my tenure as Zevra’s President and Chief Executive Officer in October 2023. From that time, and even prior to my official start date, I began a listening tour, seeking feedback from many of Zevra’s stakeholders – stockholders, investors, employees, patients and caregivers – with an agenda to understand what we do well, where we can improve and what we should be focusing on to drive value. The overarching conclusion from those conversations is that great promise lies at the intersection of the talent, capabilities, and opportunities that we have brought together here at Zevra. There are also many things that deserve more attention and areas where improvement is needed. Taking that a step further, the question of “What’s best for the patient?” has become the mantra of the Zevra team, guiding our decisions daily. And, I have instilled a second clear directive into the Zevra team, which is, “We must execute.” Driving value for patients can best be accomplished by defining a clear strategy, creating necessary plans to reach our strategic objectives, and then executing those plans. Our Company continues to work through a period of significant growth and transformation with the addition of meaningful new skills and capabilities from the strengths of three companies that have now come together to become One Zevra. Today, I am pleased to share with you a summary of our recent key accomplishments and the reasons for my optimism for 2024 and beyond. We are Building a High-Performance Team Our people are at the heart of everything we do. We are building a team of talented professionals committed to using science and data-driven development approaches to create and deliver new therapies to address unmet needs for those we serve. Since we first announced our strategic objective of becoming a leading rare disease company in the first half of 2022, we have seen significant growth in the size of our team. Through our recruiting efforts, and by virtue of our acquisitions, we have grown from a team of 24 employees to a current team of 81. This includes the buildout of targeted customer-facing commercial and medical affairs teams with decades of experience in rare diseases. We also added Adrian Quartel, M.D. as Zevra’s Chief Medical Officer, who brings more than 20 years of experience with a track record of success in clinical development, pharmacovigilance and medical affairs addressing the needs of the rare disease community. We anticipate there are additional key personnel that will be needed to round out our team as we plan for success and continue our evolution into a high-performing team. Our ability to attract and retain the best talent for our mission will be paramount to reaching our full potential as an organization. Equity participation is a key component of total compensation in the biotech industry, which is why we have proposed certain amendments to our 2014 Equity Incentive Plan (the “2014 Plan”) in our 2024 proxy, including the addition of shares and a three-year extension of the 2014 Plan. The significant growth in our headcount combined with executive management changes in 2023 and prior years has impacted our equity usage rate. However, our compensation philosophy is grounded in a data-driven approach, and we intend to utilize the additional shares judiciously in accordance with industry best practices over the remaining life of the 2014 Plan to provide long-term incentives that are fully aligned with stockholders’ interests in the pursuit of long-term value creation. We’ve Made Significant Progress Advancing our Rare Disease Portfolio During the fourth quarter of 2023, and heading into 2024, we made demonstrable progress in executing our key strategic priorities. First, we completed our acquisition of Acer Therapeutics in November 2023, allowing us to start delivering value to patients by commercializing OLPRUVA®. Second, we resubmitted the New Drug Application (NDA) for arimoclomol. Third, we completed the Phase 2 study of KP1077 in patients with idiopathic hypersomnia (IH) and we are preparing to advance KP1077 into a potential Phase 3 trial. Since the completion of the Acer acquisition in mid-November 2023, we have made meaningful progress towards ensuring that people who suffer from certain urea cycle disorders (UCDs) have access to, and are aware of, the benefits of OLPRUVA. I am proud of the team’s execution to build our rare disease commercial capability. As a result, we initiated the full commercial launch of OLPRUVA as of the end of January 2024. We have concentrated our initial efforts on approximately 40 metabolic treatment centers of excellence across the United States as the first part of our strategy to build awareness with physicians about the differentiation that OLPRUVA can bring to people living with UCDs that are addressed by the product. In the three months since launch, our team has been able to engage with more than 90% of our customers, which reflects the deep rare disease experience of our team. I am also proud to share that we have seen meaningful growth in reimbursement coverage, which was approximately 55% of U.S. covered lives at the time of acquisition, to now more than 70%. The commercial footprint we have established for OLPRUVA is designed to provide scale and cost synergies to support and accelerate the launch and commercialization of arimoclomol, if approved. Our team has put forth tremendous effort to submit a comprehensive data set to the U.S. Food and Drug Administration (FDA) supporting the resubmission of the NDA for arimoclomol. In March, we announced that the FDA had extended the review period for the NDA, resulting in a revised Prescription Drug User Fee Act (PDUFA) date of September 21, 2024, and re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting. While that meeting has not yet been scheduled, we welcome the opportunity for the data to be shared publicly, and even more importantly, to allow the voice of the patient to be heard by the advisory committee, the FDA and the public. We were overwhelmed by the outpouring of support for arimoclomol which came from the NPC community shortly after we filed the resubmission. The National Niemann-Pick Disease Foundation (NNPDF) spearheaded efforts with six other NPC advocacy and research organizations to compile a petition of nearly 1,000 signatures from NPC patients with direct experience utilizing arimoclomol, as well as caregivers and physicians. The petition can be accessed on the NNPDF website here. I encourage you to read the personal stories of people living with this devastating rare disease. Also in March, we reported positive top-line data from our Phase 2 study of KP1077 in patients with IH. We are encouraged by the results which show that KP1077 is well tolerated and demonstrates early signs of differentiated and meaningful clinical benefits. We will be presenting the results from the study at the upcoming SLEEP 2024 conference taking place June 1-5, 2024. The study successfully fulfilled the objectives of providing key information needed for the design of a potentially pivotal efficacy trial. We plan to request an end-of-Phase 2 (EOP2) meeting with the FDA to seek guidance on the Phase 3 clinical trial design and are optimistic about the potential of KP1077 to address unmet needs in sleep disorders. We have also completed our preliminary evaluation of the celiprolol program for the treatment of vascular Ehler-Danlos Syndrome (vEDS), which is a promising product candidate we added from the Acer acquisition. We recently restarted recruitment of the ongoing Phase 3 trial, which is a long-term event-driven trial design, and believe that celiprolol could address significant unmet needs as there are currently no approved treatments for vEDS in the U.S. We have a Foundation of Financial Strength Our balance sheet remains strong and provides Zevra with capital flexibility to achieve our strategic objectives. We recently announced the refinancing of our existing debt with up to $100 million in committed capital which has further strengthened our balance sheet and provides added capital flexibility to support our mission. This new credit facility, which was led by premier biotech investors including Perceptive Advisors and HealthCare Royalty Partners, has simplified our debt structure with an extended maturity and provides non-dilutive capital flexibility to support our strategic priorities. With the initial draw of $60 million, we have refinanced our existing debt of approximately $43 million and added an incremental $14 million in net cash proceeds to the cash balance after fees and discounts. In addition to a strong balance sheet, we also have a solid base of revenues from which we can build. We expect that reimbursements from the arimoclomol expanded access program in France will continue to provide approximately $2.1 million in net revenue per quarter. We expect these reimbursements to continue for as long as we are eligible for the French ATU program and continue to seek approval of the product in Europe. We also remain encouraged by the ongoing royalty and milestone contributions from the AZSTARYS® product for the treatment of ADHD in patients age six years and up, which is being commercialized by our partner, Corium, Inc. After earning the first two net sales milestones which totaled $15 million calendar year 2023 under the AZSTARYS license, we are encouraged by the product’s continuing prescription growth since the beginning of 2024, as well as the potential to reach additional cash milestones at higher net sales thresholds in the next few years. Of course, these are steppingstones towards building a sustainable operating business with reliable cash flows from our own commercialization efforts. Achieving our strategic objectives to successfully commercialize OLPRUVA, and then upon approval, successfully launch arimoclomol, are key to reaching that seminal inflection point of value creation. We’re Committed to Governance Best Practices From the beginning of 2023 through today, the composition of our Board of Directors has changed dramatically, bringing new skills, diversity, perspectives and core competencies needed to support Zevra’s objective of becoming a leading rare disease therapeutics company. The addition of Wendy Dixon, Ph.D., John Bode, Douglas Calder and Corey Watton in the first half of last year began a period of Board refreshment, which was completed following the addition of Thomas Anderson, in August 2023, my appointment to the Board in October 2023, and the recent appointment of Alvin Shih, M.D., MBA, in January 2024. With the goal of leading Zevra into its future as a patient-focused, commercial stage, rare disease company, and under the stable leadership of our Board Chair, Tamara Favorito, we have come together with full alignment behind Zevra’s mission. This positions the Board as a pillar of strength from which we can anchor our efforts and move the Company forward. The newly composed Board immediately moved into action by affirming its commitment to employing governance best practices for a company of our size and stage in the biotech industry. The Board is looking to the future in order to prepare and inform how we improve our governance structure today, and it has already started in February 2024, making updates to the Company’s bylaws which included changes that address the universal proxy rules recently adopted by the U.S. Securities and Exchange Commission, as well as updates to certain disclosure requirements in connection with stockholder nominations and proposals. Committee membership was also adjusted in February 2024, which was made possible by completing the Board’s refreshment process. We believe the new committee assignments build upon the strengths of all our directors. And with those new assignments, each standing committee of the Board is now undertaking a comprehensive review of each committee’s charter, seeking to push forward in our commitment to governance best practices in every area: Audit, Compensation, and Nominating and Corporate Governance. Also, the Board will leverage feedback from its self-evaluation process, which provides an opportunity to reflect on and improve both processes and effectiveness. Our work here is not done. The Board is committed to ongoing review and consideration of other adjustments which may be needed to enhance our ability to accomplish our objectives and to strengthen our governance framework. We are Optimistic about Our Future Bringing it all together, I am confident the opportunities we have in front of us have the potential to deliver meaningful stockholder value. We are driving to make the launch of OLPRUVA a success. We look forward to presenting the full data package from the completed KP1077 Phase 2 trial in IH patients at the upcoming SLEEP 2024 conference in early June. We are thoroughly preparing for the potential FDA advisory committee meeting to discuss the arimoclomol NDA, and we are looking forward to the PDUFA date on September 21, 2024. We intend to accelerate the commercial launch of arimoclomol, if approved, by leveraging our commercial capabilities that are now fully installed and look forward to receiving a Priority Review Voucher (PRV) upon approval. The pace of Zevra’s transformation has been tremendous, and I am proud of how much progress we have made. However, there is much more to do. From the combined strength of three teams with diverse experiences and backgrounds, we have become One Zevra, fully focused and dedicated to our mission of bringing life-changing therapeutics to people living with rare disease. We are grateful for you, our stockholders, and for your support in achieving our mission. We look forward to a bold future full of the potential to impact the lives of those we serve. Sincerely, Neil F. McFarlane President and Chief Executive Officer Zevra Therapeutics, Inc. About Zevra Therapeutics Zevra Therapeutics is a rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy as published on its website at . Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. For more information, please visit or follow us on X (formerly Twitter) and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release 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 statements regarding the potential benefits of our debt facility, our cash balance, our corporate governance objectives, potential revenues from our arimoclomol expanded access program, the potential for royalty and milestone contributions, the presentation of data at conferences, the promise and potential impact of our preclinical or clinical trial data, the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for arimoclomol or any other product candidates for any specific disease indication or at any dosage, the potential benefits of any of our products or product candidates, the potential launch or commercialization of any of product candidates or products, personnel needs and growth, including our plans to build out commercial teams for products or product candidates, and our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.  Additional Information and Where to Find It Zevra has filed with the Securities and Exchange Commission (the “SEC”) a definitive proxy statement on Schedule 14A, with respect to its solicitation of proxies for Zevra’s 2024 Annual Meeting of Stockholders. This communication is not a substitute for any proxy statement or other document that Zevra may the SEC in connection with any solicitation by Zevra. INVESTORS AND SECURITY HOLDERS ARE URGED TO READ THE PROXY STATEMENT (INCLUDING ANY AMENDMENTS OR SUPPLEMENTS THERETO) FILED BY ZEVRA AND ANY OTHER RELEVANT DOCUMENTS FILED WITH THE SEC WHEN THEY BECOME AVAILABLE CAREFULLY AND IN THEIR ENTIRETY BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT ANY SOLICITATION. Investors and security holders may obtain copies of these documents and other documents filed with the SEC by Zevra free of charge through the website maintained by the SEC at . Copies of the documents filed by Zevra are also available free of charge by accessing Zevra’s website at . Participants in the Solicitation This communication is neither a solicitation of a proxy or consent nor a substitute for any proxy statement or other filings that may be made with the SEC. Nonetheless, Zevra, its directors and executive officers and other members of management and employees may be deemed to be participants in the solicitation of proxies with respect to a solicitation by Zevra. Information about Zevra’s executive officers and directors is available in Zevra’s definitive proxy statement for the 2024 Annual Meeting of Stockholders, which was filed with the SEC on April 3, 2024. The definitive proxy statement is available free of charge at the SEC’s website at . Copies of the documents filed by Zevra are also available free of charge by accessing Zevra’s website at . Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph.D. +1 (646) 942-5603 adanna.alexander@russopartnersllc.com Ignacio Guerrero-Ros, Ph.D. +1 (646) 942-5604 ignacio.guerrero-ros@russopartnersllc.com

Launch of OLPRUVA® underway Topline results from KP1077 phase 2 study demonstrate clinically meaningful benefits for key IH symptoms Q4 2023 net revenue of $13.2M, and FY 2023 net revenue of $27.5M Conference call scheduled for today, March 28, 2024, at 4:30 p.m. ET CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023. “We made solid progress on our key priorities in 2023,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “As we look to 2024, our strategic priorities are clear; first, successfully launch OLPRUVA® and ensure access for patients; second, prepare for the launch of arimoclomol; and third, advance the KP1077 development program in sleep disorders. Mr. McFarlane continued, “We are encouraged by the positive data from our Phase 2 study of KP1077 in patients with idiopathic hypersomnia. KP1077 has been well tolerated while demonstrating early signs of differentiated clinical benefits. These data will help inform our registrational study, which we will discuss with FDA at an end of phase 2 meeting.” Recent Business and Corporate Highlights: Completion of the Acquisition of Acer Therapeutics, Inc. (Acer): On November 20, 2023, the Company announced the completion of its acquisition of Acer. The acquisition brought multiple complementary rare disease assets, increased Zevra’s revenue potential, and bolstered Zevra’s commercial and development capabilities. OLPRUVA, an FDA-approved treatment indicated for certain urea cycle disorders (UCDs): To support the commercial launch, the Company accelerated the build-out of a focused and effective customer-facing team with decades of experience in rare diseases. The team includes Rare Disease Sales Specialists, Marketers, Patient Services and Market Access professionals, as well as Medical Science Liaisons and Patient Advocates who are in the field engaging with key customers, with our efforts largely focused on approximately 40 centers of excellence across the United States. To raise awareness of the benefits of OLPRUVA, the Company has established Quick Start, which is a 30-day free trial to allow patients and physicians to gain experience with the treatment. Zevra continues to partner with the patient community and UCD treatment centers of excellence to drive brand recognition, while also working with payors to ensure access to OLPRUVA. The Company has seen a meaningful increase in reimbursement coverage since the acquisition, and currently there is more than 70% overall coverage for OLPRUVA with commercial and government payors. The Company expects that OLPRUVA’s commercial operations and capabilities will provide scale and cost synergies to support and accelerate the launch and commercialization of arimoclomol, if approved. Arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC), an ultra-rare, genetic, progressive and fatal neurological disease: During Q4 2023, the Company submitted a comprehensive data set to the U.S. Food and Drug Administration (FDA) supporting its resubmission of the New Drug Application (NDA) for arimoclomol. On March 4, 2024, the Company announced that the FDA had extended the review period for the NDA, resulting in a revised Prescription Drug User Fee Act (PDUFA) date of September 21, 2024. The FDA re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled. If approved, arimoclomol will be a first-in-class, orally delivered treatment for NPC, and the Company will be eligible to receive a Priority Review Voucher. Zevra is preparing for the commercial launch of arimoclomol in the U.S., if approved, and will leverage the commercial infrastructure that is in place for OLPRUVA. The Company plans to publish real-world evidence data from its Expanded Access Program (EAP) and additional data from clinical studies regarding safety and efficacy, to inform physician treatment decision-making and support market access. The Company intends to complete the regulatory filing for arimoclomol in the U.S., and then continue its evaluation of optimal regulatory pathways to approval in the E.U. and other parts of the world. KP1077 (serdexmethylphenidate, or SDX), an investigational therapeutic candidate both for the treatment of idiopathic hypersomnia (IH), a rare sleep disorder characterized by excessive daytime sleepiness, and for the treatment of narcolepsy: On March 26, 2024, the Company reported positive topline data from its placebo-controlled, double-blind, proof-of-concept Phase 2 study of KP1077 in patients with IH. KP1077 was well-tolerated at all dose levels evaluated in the trial, including the highest dose of 320 mg daily, regardless of dosing regimen (once or twice daily), supporting the study’s primary endpoint of safety and tolerability. KP1077 produced clinically meaningful improvement in excessive daytime sleepiness, as assessed by change from baseline in the Epworth Sleepiness Scale. This improvement was maintained during both the five-week open-label titration period and throughout the 2-week double-blind withdrawal period for both dosing regimens. Patients administered KP1077 showed benefits in change from baseline for the IH Severity Scale, Sleep Inertia Visual Analog Scale and Brain Fog severity Scale at the end of the open-label dose titration, and at the end of the double-blind withdrawal period. The study successfully fulfilled the objectives of providing key information for the design of a potentially pivotal efficacy trial, and the results of the secondary efficacy endpoints are supportive of initiating a Phase 3 trial of KP1077. The Company plans to request an end-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration to seek guidance on the Phase 3 clinical trial design. The Company will present new data from a Phase 1 study completed under the narcolepsy IND, and the final results from its Phase 2 study of KP1077 in IH at the upcoming SLEEP 2024 conference. Overview of Q4 2023 and FY 2023 Financial Results: Net revenue for Q4 2023 was $13.2 million, compared to prior year Q4 net revenue of $2.2 million. The components of revenue during the current quarter include ongoing royalties from AZSTARYS®, reimbursements from the French EAP for arimoclomol, and some initial sales of OLPRUVA. Research and development (R&D) expenses were $11.4 million for Q4 2023, compared to $6.5 million in Q4 2022. The increase in R&D expenses was primarily driven by the ongoing Phase 2 clinical study in KP1077, and ongoing work supporting to support the arimoclomol NDA which was resubmitted to the FDA in December 2023. General and administrative (G&A) expenses were $14.7 million for Q4 2023, compared to $4.7 million in Q4 2022. The period-over-period increase was primarily related to an increase in personnel costs and professional fees associated with our commercial and business development activities. Net loss for Q4 2023 was ($19.6) million, or ($0.51) per basic and diluted share, compared to a net loss of ($3.0) million, or ($0.09) per basic and diluted share for the same period in 2022. Net revenue for FY 2023 was $27.5 million compared to prior year net revenue of $10.2 million. The period-over-period increase was primarily attributed to an increase of $18.5 in royalties and milestones received under the AZSTARYS License Agreement, which includes $15.0 million in one-time net sales milestone payments earned during FY 2023, an increase in sales of arimoclomol of $3.3 million, partially offset by a decrease in consulting revenue of $4.5 million. R&D expenses were $39.8 million for FY 2023, compared to $19.8 million for FY 2022. The increase in R&D expenses was primarily driven by the ongoing KP1077 Phase 2 clinical study in IH, along with work to prepare the arimoclomol NDA for resubmission. G&A expenses were $34.3 million for FY 2023, compared to $15.0 million in FY 2022. The period-over-period increase was primarily related to an increase in personnel costs and professional fees associated with our commercial and business development activities. Net loss for FY 2023 was ($46.0) million, or ($1.30) per basic and diluted share, which includes the non-cash impact of the change in fair value adjustment for the warrant liability of ($1.4) million, or ($0.04) per basic and diluted share. Net loss for FY 2022 was ($26.8) million, or ($0.78) per basic and diluted share. As of December 31, 2023, total cash, cash equivalents, and cash investments were $67.7 million, a decrease of $15.7 million compared to $83.4 million as of September 30, 2023. The decrease was driven, in part, by increased third-party R&D costs related to the KP1077 clinical development program, the arimoclomol program, and increased G&A expenses during the period as the Company invested in its commercial infrastructure. Based on our current operating forecast and available resources, our cash runway is expected to extend into 2026. Our cash runway forecast includes revenue from the expected sales of OLPRUVA, ongoing reimbursements from the French EAP for arimoclomol, and investments into the incremental commercial activities needed to support the launch of arimoclomol, if approved, and completion of the KP1077 development program for IH. Our cash runway forecast does not include any commercial revenue from arimoclomol which could follow a potential FDA approval, or the potential sale of the Priority Review Voucher which would be received upon approval. On November 17, 2023, Zevra completed the acquisition of Acer. Pursuant to the Merger Agreement, Acer continues as a wholly owned subsidiary of Zevra. The Merger included the acquisition of OLPRUVA® (sodium phenylbutyrate) for oral suspension, which was approved by the FDA on December 27, 2022, for the treatment of urea cycle disorders. Acer also has a pipeline of investigational product candidates, including celiprolol for the treatment of vascular Ehlers-Danlos syndrome, patients with a confirmed type III collagen (COL3A1) mutation. At the effective time of the Merger (the "Effective Time"), each share of common stock of Acer, par value $0.0001 per share, issued and outstanding immediately prior to the Effective Time (excluding cancelled shares and any shares held by holders who have exercised their appraisal rights) were converted into the right to receive (i) 0.1210 fully paid and non-assessable shares of common stock of Zevra, par value $0.0001 per share, and (ii) one non-transferable contingent value right (“CVR”) issued by Zevra, which represents the right to receive one or more contingent payments up to an additional $76.0 million upon the achievement, if any, of certain commercial and regulatory milestones for Acer’s OLPRUVA and celiprolol products within specified time periods. Certain additional cash payments are also possible pursuant to the CVRs with respect to milestones involving Acer’s early-stage program ACER-2820 (emetine). As of December 31, 2023, total shares of common stock outstanding were 41,534,668, and fully diluted common shares outstanding were 58,230,596, which included 5,603,729 shares issuable upon exercise of warrants. On March 25, 2024, the Audit Committee (the “Audit Committee”) of the Company’s Board of Directors, after discussion with senior management and the Company’s independent registered public accountants, concluded that the Company’s previously issued audited consolidated financial statements as of and for the fiscal years ended December 31, 2022 and December 31, 2021, included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, (collectively, the “Prior Financial Statements”) should no longer be relied upon. In connection with the preparation of the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 (the “2023 Form 10-K”), the Audit Committee concluded that, in prior years it had not appropriately accounted for certain common stock warrants as liabilities. These errors led to understatements of derivative and warrant liability and additional paid-in capital and fluctuations in fair value adjustment related to derivative and warrant liability during the impacted periods. In addition, in connection with the restatements, the Company has concluded that the previously disclosed errors led to misstatements of fair value adjustment related to derivative and warrant liability, derivative and warrant liability, additional paid-in capital, and retained earnings/(accumulated deficit) that were previously disclosed in the unaudited condensed consolidated balance sheets and statements of operations included in the Company’s Quarterly Reports on Form 10-Q for the quarterly periods ended March 31, 2022, June 30, 2022, September 30, 2022, March 31, 2023, June 30, 2023 and September 30, 2023 (collectively, the “Prior Interim Financial Statements”). On March 25, 2024, the Audit Committee, after discussion with senior management and the Company’s independent registered public accountants, concluded that the Prior Interim Financial Statements should no longer be relied upon. The errors and corrective adjustments identified by the Company are non-cash in nature; and they do not impact results of operations or key metrics used by the Company in managing operations, such as revenue, operating expenses, and loss from operations. Conference Call Information Zevra will host a conference call and live audio webcast today at 4:30 p.m. ET, to discuss its corporate and financial results for Q4 and FY 2023. The audio webcast will be accessible via the Investor Relations section of the Company’s website, . An archive of the audio webcast will be available for 90 days beginning at approximately 5:30 p.m. ET, on March 28, 2024. Additionally, interested participants and investors may access the conference call by dialing either: (800) 245-3047 (U.S.) +1 (203) 518- 9765 (International) Conference ID: ZVRAQ423 About Urea Cycle Disorders UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments if ammonia levels are not controlled.i Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels. About OLPRUVA® OLPRUVA (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA®. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Please see Important Safety Information and full Prescribing Information, including Patient Information. Important Safety Information Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child take, especially if you or your child take corticosteroids, valproic acid, haloperidol, and/or probenecid. OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you. The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. About Niemann-Pick Disease Type C (NPC) Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol Arimoclomol, Zevra’s orally delivered, first-in-class investigational product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee goal date (PDUFA date) of September 21, 2024. About Idiopathic Hypersomnia (IH) Idiopathic hypersomnia (IH) is a rare sleep disorder characterized by excessive daytime sleepiness (EDS). Patients with IH experience daytime lapses into sleep, or an irrepressible need to sleep that persists even with adequate or prolonged nighttime sleep. Additionally, those with IH have extreme difficulty waking, otherwise known as sleep inertia, severe brain fog, and often fall asleep unintentionally or at inappropriate times. These symptoms of IH often lead to further, even more debilitating problems such as memory lapses, difficulty maintaining focus, and depression. It is estimated, based on claims data, that approximately 37,000 patients in the United States are currently diagnosed with IH, although the total patient population may be much larger due to some patients who have not yet been diagnosed, have been misdiagnosed, or are not currently seeking treatment. About KP1077 KP1077 (serdexmethylphenidate or SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and its sole active pharmaceutical ingredient (API). KP1077 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of IH, and the U.S. Drug Enforcement Agency (DEA) has classified SDX, the sole API in KP1077, as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. About Zevra Therapeutics Zevra Therapeutics is a rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. Expanded access programs are made available by Zevra Therapeutics and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy as published on its website at . Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion. For more information, please visit or follow us on X (formerly Twitter) and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release 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 statements regarding the promise and potential impact of our preclinical or clinical trial data, the initiation, timing and results of any clinical trials or readouts, the content, information used for, timing or results of any NDA submissions or resubmissions for arimoclomol or any other product candidates for any specific disease indication or at any dosage, the potential launch or commercialization of any of product candidates or products, the Company’s estimated extent of and impacts of the restatements and the timing of the filing of the 2023 Form 10-K, the sufficiency of our cash, cash equivalents and investments to fund our operating activities for any specific period of time, our plans to build out commercial teams for products or product candidates, and our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2022, as updated in Zevra’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, and Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. __________________________ i Ah Mew N, et al. Urea cycle disorders overview [updated June 22, 2017]. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet]. University of Washington; 1993-2022. Accessed March 20, 2022. ZEVRA THERAPEUTICS, INC. STATEMENTS OF OPERATIONS (In thousands, except share and per share amounts) (Unaudited) Year Ended December 31, 2023 2022 (As Restated) Revenue, net $ 27,461 $ 10,161 Operating expenses: Cost of revenue 2,945 222 Research and development 39,806 19,803 Selling, general and administrative 34,314 15,038 Acquired in-process research and development - 17,663 Total operating expenses 77,065 52,726 Loss from operations (49,604 ) (42,565 ) Other (expense) income: Interest expense (1,501 ) (335 ) Fair value adjustment related to derivative and warrant liability (98 ) 15,159 Fair value adjustment related to investments 613 (577 ) Interest and other income, net 4,541 1,513 Total other income 3,555 15,760 Loss before income taxes (46,049 ) (26,805 ) Income tax (expense) benefit - 33 Net loss $ (46,049 ) $ (26,772 ) Basic and diluted net loss per share of common stock: Net loss $ (1.30 ) $ (0.78 ) Weighted average number of shares of common stock outstanding: Basic and diluted 35,452,460 34,488,800 ZEVRA THERAPEUTICS, INC. BALANCE SHEETS (In thousands, except share and par value amounts) (Unaudited) December 31, 2023 2022 (As Restated) Assets Current assets: Cash and cash equivalents $ 43,049 $ 65,466 Securities at fair value 24,688 16,900 Short-term investments - other - 481 Accounts and other receivables 17,377 8,299 Prepaid expenses and other current assets 1,824 1,688 Total current assets 86,938 92,834 Inventories 9,841 671 Property and equipment, net 736 794 Operating lease right-of-use assets 790 988 Goodwill 4,701 - Long-term investments - other - 20,000 Intangible assets, net 69,227 - Other long-term assets 94 53 Total assets $ 172,327 $ 115,340 Liabilities and stockholders’ equity Current liabilities: Accounts payable and accrued expenses $ 28,403 $ 6,169 Line of credit payable 37,700 12,800 Current portion of operating lease liabilities 543 480 Current portion of discount and rebate liabilities 4,550 4,655 Other current liabilities 2,524 719 Total current liabilities 73,720 24,823 Secured promissory note 5,066 - Derivative and warrant liability 16,100 10,202 Operating lease liabilities, less current portion 456 843 Discount and rebate liabilities, less current portion 7,663 4,327 Other long-term liabilities 7,458 25 Total liabilities 110,463 40,220 Commitments and contingencies Stockholders' equity: Preferred stock: Undesignated preferred stock, $0.0001 par value, 10,000,000 shares authorized, no shares issued or outstanding as of December 31, 2023 or December 31, 2022 - - Common stock, $0.0001 par value, 250,000,000 shares authorized, 43,110,360 shares issued and 41,534,668 shares outstanding as of December 31, 2023; 35,450,257 shares issued and 34,540,304 shares outstanding as of December 31, 2022 4 3 Additional paid-in capital 472,664 436,269 Treasury stock, at cost (10,983 ) (7,536 ) Accumulated deficit (399,778 ) (353,729 ) Accumulated other comprehensive income (43 ) 113 Total stockholders' equity 61,864 75,120 Total liabilities and stockholders' equity $ 172,327 $ 115,340 Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph.D. +1 (646) 942-5603 adanna.alexander@russopartnersllc.com