Acer Therapeutics, Inc.

WILMETTE, Ill., May 24, 2024 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage radiopharmaceutical company focused on developing innovative treatments for cancer patients, today announced that after a career spanning more than 35 years, Monopar’s Chief Financial Officer, Kim R. Tsuchimoto, will be retiring. Ms. Tsuchimoto will be transitioning over the next month, with her last day being on June 30, 2024. Ms. Tsuchimoto will remain as a member of Monopar’s Board of Directors. Monopar has been working over the past 6-plus months on a consulting basis with Karthik Radhakrishnan. Effective July 1, 2024, Mr. Radhakrishnan will be appointed Monopar’s Chief Financial Officer, Principal Accounting Officer, and Principal Financial Officer. Mr. Radhakrishnan brings over 20 years of financial strategy, investment and public company experience. Mr. Radhakrishnan has served as President and founder of Titania Partners LLC, a management consulting firm focused on the life science sector during which time he served as Chief Financial Officer of Santalis Pharmaceuticals, Inc. and Chief Executive Officer of Asylia Therapeutics, Inc. He has also previously served as Chief Financial Officer of Opexa Therapeutics, Inc. (formerly Nasdaq: OPXA) and before that as Vice President at ING Investment Management in New York. “On behalf of Monopar’s Board of Directors and Management team, I would like to thank Kim for her wonderful contributions and leadership during her tenure at the Company. Kim has laid a strong foundation in financial management and corporate compliance to support Monopar’s future growth and success,” said Chandler Robinson, Monopar’s Chief Executive Officer. “We wish Kim a very enjoyable retirement and are looking forward to working with Karthik, with whom we have already had a positive experience over the past 6 to 7 months.” “I am extremely impressed with Monopar’s innovative technology in developing radiopharmaceutical imaging and therapeutic drugs targeting areas of high unmet medical need such as pancreatic cancer and triple-negative breast cancer among others,” said Mr. Radhakrishnan. “I look forward to contributing and working with entire Monopar team and its board of directors in unlocking our full potential.” About Monopar Therapeutics Inc. Monopar Therapeutics is a clinical-stage radiopharmaceutical company focused on developing innovative treatments for cancer patients, including Phase 1-stage MNPR-101-Zr for imaging advanced cancers and late preclinical-stage MNPR-101 radio-immuno-therapeutic (RIT) for the treatment of advanced cancers, as well as early development programs against solid cancers. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Examples of these forward-looking statements include: that Ms. Tsuchimoto will remain on Monopar’s Board. The forward-looking statements involve risks and uncertainties including, but not limited to: that the CFO transition may not be seamless; that Monopar may expend available funds sooner than anticipated or require additional funding due to change in circumstances or unanticipated events; that future preclinical or clinical data will not be as promising as the data to date; not successfully enrolling the MNPR-101-Zr Phase 1 clinical trial if at all; that MNPR-101-Zr and/or MNPR-101 conjugated to a therapeutic radioisotope may cause unexpected serious adverse effects or fail to image or be effective against the cancer tumors in humans; and the significant general risks and uncertainties surrounding the research, development, regulatory approval, and commercialization of imaging agents and therapeutics. Actual results may differ materially from those expressed or implied by such forward-looking statements. Risks are described more fully in Monopar's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Monopar undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Any forward-looking statements contained in this press release represent Monopar’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. CONTACT: Monopar Therapeutics Inc.Investor RelationsKim R. TsuchimotoChief Financial Officerkimtsu@monopartx.com Follow Monopar on social media for updates: X (formerly Twitter): @MonoparTx LinkedIn: Monopar Therapeutics

Full commercial launch of OLPRUVA at the end of January 2024, with market access growing to ~75% of covered lives as of May 1, 2024 Arimoclomol NDA review underway with PDUFA date of September 21, 2024 Reported positive topline results from KP1077 Phase 2 Trial in idiopathic hypersomnia, with full data to be presented at SLEEP 2024 in early June 2024 Conference call scheduled for today, May 8, 2024, at 8:00 a.m. ET CELEBRATION, Fla., May 08, 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 first quarter ended March 31, 2024. “During the first quarter, we made steady progress in executing on our strategic objectives: to successfully launch OLPRUVA and ensure access for patients, to prepare for the potential launch of arimoclomol, and to advance the KP1077 program in sleep disorders,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “Our recent debt refinancing allowed us to simplify our capital structure while providing additional flexibility to support our strategic priorities,” said R. LaDuane Clifton, Zevra’s Chief Financial Officer, Secretary and Treasurer. “With a solid balance sheet, we are focused on creating long-term value for shareholders by executing against our plan of becoming a leading rare disease company.” Recent Business and Corporate Highlights: OLPRUVA The full commercial launch of OLPRUVA was initiated at the end of January 2024, and the team has engaged with more than 90% of the specialists at the 40 centers of excellence that treat people with UCD. During the quarter, Zevra had four new patient enrollments, defined as a prescription for a patient eligible for either benefits investigation or the Company’s Quick Start program.Zevra has seen a meaningful increase in reimbursement coverage, reaching nearly 75% of covered lives as of May 1, 2024. Arimoclomol During the quarter, the Company announced the FDA’s extension of arimoclomol’s Prescription Drug User Fee Act (PDUFA) date to September 21, 2024, and reaffirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled.The National Niemann-Pick Disease Foundation, along with six other NPC advocacy and research organizations, submitted a petition in support of arimoclomol with nearly 1,000 signatures from NPC patients, caregivers and physicians.In April 2024, the Company presented new long-term, real-world data from the expanded access program (EAP: NCT04316637) for the treatment of NPC at the Society for Inherited Metabolic Disorders.Zevra is preparing for the U.S. commercial launch of arimoclomol, if approved, and intends to leverage the commercial infrastructure that is in place for OLPRUVA to accelerate patient access. KP1077 The Company reported positive topline data from its placebo-controlled, double-blind, proof-of-concept Phase 2 study of KP1077 in patients with IH which showed that it was well tolerated and demonstrated early signs of potentially differentiated and clinically meaningful benefits. The full data package from our completed Phase 2 study will be presented at the upcoming SLEEP 2024 conference in early June.The Company is planning for an end-of-Phase 2 (EOP2) meeting with the FDA in the third quarter. Celiprolol The Company recently restarted recruitment of the celiprolol Phase 3 trial in VEDS, also known as the DiSCOVER trial, to support patients currently enrolled and to preserve the value of the program while conducting our portfolio review. Overview of Q1 2024 Financial Results: Net revenue for Q1 2024 was $3.4 million, compared to net revenue of $3.2 million in Q1 2023. The components of revenue during the first quarter included $2.2 million in net reimbursements from the French EAP for arimoclomol, $1.2 million of royalties and other reimbursements under the AZSTARYS® License Agreement, and de minimis sales of OLPRUVA due to limited shipments to our specialty pharmacy in Q1 2024. Research and development (R&D) expenses were $12.3 million for Q1 2024, compared to $8.7 million in Q1 2023. The increase in R&D expenses was primarily driven by an increase in spending for the KP1077 Phase 2 clinical trial that has since been completed, as well as ongoing support of the arimoclomol NDA. Selling, general and administrative (SG&A) expenses were $9.9 million for Q1 2024, compared to $7.2 million in Q1 2023. The period-over-period increase was primarily related to an increase in personnel costs and professional fees associated with the Company's investments in its commercial infrastructure. Net loss for Q1 2024 was ($16.6) million, or ($0.40) per basic and diluted share, compared to a net loss of ($13.2) million, or ($0.38) per basic and diluted share for Q1 2023. As of March 31, 2024, total shares of common stock outstanding were 41,850,494, and fully diluted common shares outstanding decreased by 1,451,966 to 56,778,630, which included 5,603,729 shares issuable upon exercise of warrants. As of March 31, 2024, total cash, cash equivalents, and investments were $52.7 million, a decrease of $15.0 million compared to $67.7 million as of December 31, 2023. The decrease was driven, in part, by increased third-party R&D costs related to the KP1077 clinical development program, reduction in accounts payable and accrued liabilities associated with the Company’s acquisition of Acer Therapeutics, and increased SG&A expenses during the period as the Company invested in its commercial infrastructure. On April 5, 2024, the Company announced the refinancing of our existing debt with up to $100 million in committed capital, which strengthened our balance sheet, simplified our debt structure, and provided non-dilutive capital flexibility. The refinancing was led by Perceptive Advisors and HealthCare Royalty Partners, premier biotech investors. From the initial draw of $60 million at closing, we have refinanced our existing debt of $43 million and added an incremental $14 million in net cash proceeds to the cash balance after fees and discounts. The facility also provides a second tranche of $20 million which can be drawn at the Company’s discretion until October 5, 2025, and a third tranche of $20 million which may be drawn upon the approval of arimoclomol, with each of the additional tranches subject to terms and conditions. Based on our current operating plan, available cash, cash equivalents and investments are expected to extend our cash runway into 2026, subject to continuing compliance with our debt covenants. The Company’s cash runway forecast includes revenue from the expected sales of OLPRUVA, ongoing reimbursements from the French EAP for arimoclomol, ongoing royalties under the AZSTARYS license agreement, investments into the incremental commercial activities needed to support the launch of arimoclomol, if approved, and completion of the KP1077 development program for IH.The Company’s 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. Conference Call Information Zevra will host a conference call and live audio webcast today at 8 a.m. ET, to discuss its corporate and financial results for Q1 2024. The audio webcast will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the audio webcast will be available for 90 days beginning at approximately 9 a.m. ET, on May 8, 2024. Additionally, interested participants and investors may access the conference call by dialing either: 800-343-5419 (U.S.)+1 203-518-9731 (International)Conference ID: ZVRAQ124 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 therapies, 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 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 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 Celiprolol Celiprolol is an investigational clinical candidate for the treatment of Vascular Ehlers-Danlos Syndrome (VEDS). Celiprolol has been granted Orphan Drug and Breakthrough Therapy Designations by the FDA. Zevra recently restarted enrollment in the Phase 3 trial, known as DiSCOVER trial being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. FDA. Celiprolol’s mechanism of action is designed to reduce the mechanical stress on collagen fibers within the arterial wall through vascular dilation and smooth muscle relaxation. About Vascular Ehlers-Danlos Syndrome Vascular Ehlers-Danlos syndrome is a rare genetic cardiovascular disorder which impairs collagen 3 rich connective tissue and leads to vascular and hollow organ ruptures. It is estimated that approximately 7,500 patients in the United States are currently diagnosed patients with VEDS. There remains an unmet need with no approved treatment in the U.S. and celiprolol is currently the standard of care in Europe. 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 www.zevra.com. 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 www.zevra.com 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, Zevra’s quarterly report on Form 10-Q for the three months ended March 31, 2024, 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 Contact Nichol Ochsner+1 (732) 754-2545nochsner@zevra.com Russo Partners Contacts Adanna G. Alexander, Ph.D.+1 (646) 942-5603adanna.alexander@russopartnersllc.com ZEVRA THERAPEUTICS, INC.UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) Three months ended March31, 2024 2023Revenue, net$3,425 $3,176 Cost of product revenue (excluding $1,528 in intangible assets amortizationfor the three months ended March 31, 2024, shown separately below) 175 125 Intangible asset amortization 1,528 - Operating expenses: Research and development 12,277 8,655 Selling, general and administrative 9,931 7,227 Total operating expenses 22,208 15,882 Loss from operations (20,486) (12,831)Other income (expense): Interest expense (735) (182)Fair value adjustment related to warrant and CVR liability 3,627 (1,545)Fair value adjustment related to investments (27) 196 Interest and other income, net 929 1,042 Total other income (expense) 3,794 (489)Loss before income taxes (16,692) (13,320)Income tax benefit 70 103 Net loss$(16,622) $(13,217) Basic and diluted net loss per share of common stock: Net loss$(0.40) $(0.38)Weighted average number of shares of common stock outstanding: Basic and diluted 41,778,774 34,466,542 ZEVRA THERAPEUTICS, INC.UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS(in thousands, except share and par value amounts) March 31, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents$42,849 $43,049 Securities at fair value 9,868 24,688 Accounts and other receivables 8,305 17,377 Prepaid expenses and other current assets 1,868 1,824 Total current assets 62,890 86,938 Inventories 12,426 9,841 Property and equipment, net 695 736 Operating lease right-of-use assets 1,086 790 Goodwill 4,701 4,701 Intangible assets, net 67,699 69,227 Other long-term assets 1,786 94 Total assets$151,283 $172,327 Liabilities and stockholders' equity Current liabilities: Accounts payable and accrued expenses$22,785 $28,403 Line of credit payable - 37,700 Current portion of operating lease liabilities 583 543 Current portion of discount and rebate liabilities 4,452 4,550 Other current liabilities 2,089 2,524 Total current liabilities 29,909 73,720 Secured promissory note 5,059 5,066 Line of credit payable 37,939 - Warrant liability 11,535 16,100 Operating lease liabilities, less current portion 705 456 Discount and rebate liabilities, less current portion 8,781 7,663 Other long-term liabilities 8,537 7,458 Total liabilities 102,465 110,463 Commitments and contingencies Stockholders' equity: Preferred stock: Undesignated preferred stock, $0.0001 par value, 10,000,000 sharesauthorized, no shares issued or outstanding as of March 31, 2024, orDecember 31, 2023 - - Common stock, $0.0001 par value, 250,000,000 shares authorized, 43,426,186shares issued and 41,850,494 shares outstanding as of March 31, 2024;43,110,360 shares issued and 41,534,668 shares outstanding as of December31, 2023 4 4 Additional paid-in capital 476,056 472,664 Treasury stock, at cost (10,983) (10,983)Accumulated deficit (416,400) (399,778)Accumulated other comprehensive income (loss) 141 (43)Total stockholders' equity 48,818 61,864 Total liabilities and stockholders' equity$151,283 $172,327

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