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Zevra Therapeutics Reports Third Quarter 2024 Financial Results and Corporate Updates

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Zevra Therapeutics reported Q3 2024 financial results, highlighting the FDA approval of MIPLYFFA™ for Niemann-Pick disease type C treatment. The company reported revenue of $3.7 million and a net loss of ($33.2 million). Key developments include receiving a rare pediatric disease Priority Review Voucher and recording 90 prescription enrollment forms for MIPLYFFA, with 30% approved for reimbursement. Cash position stands at $95.5 million, expected to extend runway into 2027. The company completed a public offering raising $64.5 million and is implementing a strategic plan focused on commercial excellence, pipeline innovation, talent, and corporate foundation.

Zevra Therapeutics ha riportato i risultati finanziari per il terzo trimestre del 2024, evidenziando l'approvazione da parte della FDA del MIPLYFFA™ per il trattamento della malattia di Niemann-Pick di tipo C. L'azienda ha registrato ricavi di 3,7 milioni di dollari e una perdita netta di (33,2 milioni di dollari). Tra i principali sviluppi, vi è l'ottenimento di un Voucher di Revisione Prioritaria per malattie pediatriche rare e la registrazione di 90 moduli di iscrizione per prescrizioni di MIPLYFFA, con il 30% approvato per il rimborso. La posizione di cassa ammonta a 95,5 milioni di dollari, con aspettative di estendere il periodo di operatività fino al 2027. L'azienda ha completato un'offerta pubblica raccogliendo 64,5 milioni di dollari e sta implementando un piano strategico incentrato sull'eccellenza commerciale, sull'innovazione della pipeline, sui talenti e sulla fondazione aziendale.

Zevra Therapeutics informó los resultados financieros del tercer trimestre de 2024, destacando la aprobación de la FDA del MIPLYFFA™ para el tratamiento de la enfermedad de Niemann-Pick tipo C. La compañía reportó ingresos de 3.7 millones de dólares y una pérdida neta de (33.2 millones de dólares). Entre los desarrollos clave se incluye la recepción de un Voucher de Revisión Prioritaria para enfermedades pediátricas raras y la grabación de 90 formularios de inscripción de prescripción para MIPLYFFA, con un 30% aprobado para reembolso. La posición de efectivo se sitúa en 95.5 millones de dólares, y se espera que esto extienda el periodo de operación hasta 2027. La compañía completó una oferta pública recaudando 64.5 millones de dólares y está implementando un plan estratégico centrado en la excelencia comercial, la innovación de la pipeline, el talento y los fundamentos corporativos.

Zevra Therapeutics는 2024년 3분기 재무 결과를 발표하며, Niemann-Pick 질병 타입 C 치료를 위한 MIPLYFFA™의 FDA 승인을 강조했습니다. 이 회사는 370만 달러의 수익과 (3,320만 달러)의 순손실을 기록했습니다. 주요 개발 사항으로는 희귀 소아 질병 우선 리뷰 바우처를 받았고, MIPLYFFA를 위한 90개의 처방 등록 양식을 기록했으며, 이 중 30%가 환급 승인을 받았다는 점이 있습니다. 현금 잔액은 9,550만 달러로, 2027년까지 운영 기간을 연장할 것으로 예상되고 있습니다. 이 회사는 6450만 달러를 모금하는 공모를 완료했으며, 상업적 우수성, 파이프라인 혁신, 인재 및 기업 기반에 중점을 둔 전략 계획을 시행하고 있습니다.

Zevra Therapeutics a publié ses résultats financiers pour le troisième trimestre 2024, mettant en avant l'approbation par la FDA de MIPLYFFA™ pour le traitement de la maladie de Niemann-Pick de type C. L'entreprise a rapporté des revenus de 3,7 millions de dollars et une perte nette de (33,2 millions de dollars). Parmi les principaux développements, on note la réception d'un bon de priorité pour examen des maladies pédiatriques rares et l'enregistrement de 90 formulaires d'inscription de prescription pour MIPLYFFA, dont 30 % ont été approuvés pour le remboursement. La position de trésorerie s'élève à 95,5 millions de dollars, et il est prévu d'étendre le temps d'exploitation jusqu'en 2027. L'entreprise a complété une offre publique levant 64,5 millions de dollars et met en œuvre un plan stratégique axé sur l'excellence commerciale, l'innovation de pipeline, les talents et les fondations d'entreprise.

Zevra Therapeutics hat die Finanzzahlen für das dritte Quartal 2024 veröffentlicht und die Genehmigung der FDA für MIPLYFFA™ zur Behandlung der Niemann-Pick-Krankheit Typ C hervorgehoben. Das Unternehmen meldete Einnahmen von 3,7 Millionen Dollar und einen Nettverlust von (33,2 Millionen Dollar). Zu den wichtigsten Entwicklungen zählt der Erhalt eines Priority Review Vouchers für seltene pädiatrische Krankheiten sowie die Erfassung von 90 Rezeptanmeldungen für MIPLYFFA, von denen 30 % für die Erstattung genehmigt wurden. Die Liquiditätsposition liegt bei 95,5 Millionen Dollar, was voraussichtlich ausreicht, um bis 2027 zu operieren. Das Unternehmen hat eine öffentliche Angebotsrunde abgeschlossen, bei der 64,5 Millionen Dollar gesammelt wurden, und implementiert einen Strategieplan, der sich auf kommerzielle Exzellenz, Pipeline-Innovation, Talente und Unternehmensgrundlagen konzentriert.

Positive
  • FDA approval of MIPLYFFA as first NPC drug, with 90 prescription enrollments
  • Strong cash position of $95.5 million with runway into 2027
  • Successful public offering raising $64.5 million
  • Receipt of valuable rare pediatric disease Priority Review Voucher
  • OLPRUVA achieving 76% reimbursement coverage of covered lives
Negative
  • Increased net loss to $33.2 million vs $10.4 million in Q3 2023
  • SG&A expenses nearly tripled to $16.2 million from $5.8 million YoY
  • $2.0 million inventory obsolescence reserve for OLPRUVA
  • Minimal OLPRUVA revenue reported

Insights

A critical quarter for Zevra with mixed financial results. $3.7M in revenue was primarily driven by French EAP reimbursements and AZSTARYS royalties, while OLPRUVA sales remain minimal. The net loss widened to $33.2M ($0.69/share) from $10.4M year-over-year, largely due to increased SG&A expenses from commercial launch activities.

The balance sheet shows strength with $95.5M in cash and investments, bolstered by a $64.5M public offering. The projected cash runway into 2027 provides operational stability. Key revenue drivers include potential MIPLYFFA sales and the monetization of the Priority Review Voucher, which could be worth $100M to $120M based on recent transactions.

The FDA approval of MIPLYFFA marks a significant milestone as the first approved treatment for Niemann-Pick disease type C. Early prescription data shows promising uptake with 90 enrollment forms and 30% ready for reimbursement. The streamlined regulatory pathway for KP1077 requiring only one pivotal study could accelerate development timelines.

The strategic shift to focus on late-stage development and commercialization, while discontinuing early-stage research, aligns with industry trends toward efficiency. The VEDS trial progress with 19 enrolled patients demonstrates momentum in the rare disease pipeline.

FDA approval of MIPLYFFA™ and product launch cap third quarter filled with multiple commercial and clinical development milestones

Zevra received rare pediatric disease Priority Review Voucher

Company ends quarter with cash, cash equivalents and investments of $95.5 million

Company to host conference call and webcast today, Nov. 12, 2024, 4:30 p.m. ET

CELEBRATION, Fla., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today reported its financial results for the third quarter ended Sept. 30, 2024, and provided corporate updates.

“The third quarter was one of the most exciting and transformational periods in Zevra’s journey,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. “After years of tireless effort, our team has achieved a major milestone with FDA approval of MIPLYFFA™ (arimoclomol), and we’re celebrating with the Niemann-Pick disease type C (NPC) community. We intend to achieve our goals with a high-performing team committed to execute, focus and innovate to drive our continued growth and long-term transformation.”

Program Highlights

  • On Sept. 20, the U.S. Food and Drug Administration (FDA) approved MIPLYFFA capsules as an orally delivered treatment for NPC. MIPLYFFA, the first NPC drug approved by the FDA, is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. With the approval of MIPLYFFA, Zevra received a rare pediatric disease Priority Review Voucher (PRV) which the Company could monetize to help fund its growth.
  • As of Oct. 31, Zevra has received 90 prescription enrollment forms for MIPLYFFA of which 30% are approved for reimbursement and are ready for fulfillment upon drug availability, consistent with the Company’s prior guidance of eight to 12 weeks post launch.
  • Zevra has launched AmplifyAssist™, a comprehensive support program for caregivers and those eligible living with NPC or UCDs.
  • OLPRUVA® had three new patient enrollments in the third quarter, and reimbursement coverage is at approximately 76% of covered lives. Improving OLPRUVA’s formulary position is a primary objective to make it the preferred treatment for UCDs.
  • Zevra is refining its commercial strategy for OLPRUVA to focus on specific patient segments where there are fewer access barriers, and, together with The National Urea Cycle Disorders Foundation, launched a targeted patient education campaign highlighting the importance of prompt and accurate testing of blood ammonia levels, which, when elevated, may be a sign of a UCD. Campaign materials are available at www.checkammonia.com.
  • The Company recently completed an End-of-Phase 2 meeting with the FDA for KP1077. The Agency indicated that a single pivotal study with appropriate confirmatory evidence will be sufficient to submit a new drug application (NDA). Zevra is evaluating strategic alternatives to advance clinical development and later, commercialization.
  • The Phase 3 DiSCOVER trial of celiprolol for the treatment of Vascular Ehler-Danlos Syndrome (VEDS) is underway, and the Company has achieved its objective to restart enrollment and to begin dosing new patients, reporting that a total of 19 patients are now in the trial as of the end of the third quarter. Zevra is continuing its efforts to expand its partnerships with patient advocacy groups, treatment centers of excellence, and medical key opinion leaders.

Corporate Highlights and Upcoming Milestones

  • In August, Zevra completed an underwritten public offering of 10,615,385 shares of its common stock at an offering price of $6.50 per share for total net proceeds of approximately $64.5 million, after deducting underwriting discounts and commissions and offering expenses. Cantor and William Blair were joint book-running managers for the offering. Citizens JMP and H.C. Wainwright & Co. were co-managers.
  • The Company completed a thorough portfolio assessment and developed a strategic plan for 2025 that builds from its 2024 accomplishments and furthers Zevra’s transformation towards becoming a leading rare disease company with a five-year vision to create value for patients and shareholders by organizing our priorities on four key pillars: Commercial Excellence, Pipeline and Innovation, Talent and Culture, and Corporate Foundation.

Now focused on late-stage clinical development and commercial opportunities, Zevra has discontinued its in-house drug discovery activities and is closing its laboratory facilities in Iowa and Virginia. Future early research and development activities will be outsourced.

Q3 2024 Financial Highlights

  • Revenue, Net: $3.7 million, comprised of $2.6 million in net reimbursements from the French expanded access program (EAP) for arimoclomol, $1.1 million of royalties and other reimbursements under the AZSTARYS® license agreement, and de minimis OLPRUVA revenue.
  • Cost of Goods Sold: $2.3 million, which includes recognition of $2.0 million in inventory obsolescence reserve expense related to OLPRUVA inventory that is nearing expiration.
  • R&D Expenses: $10.9 million, which represents a decrease of $1.4 million compared to Q3 2023 due to the completion of the KP1077 phase 2 trial.
  • SG&A Expenses: $16.2 million for Q3 2024, compared to $5.8 million in Q3 2023, which reflects the commercial team fully in place and actively engaged in commercial launch activities and patient services initiatives. This includes non-cash stock compensation of $6.1 million during the quarter, of which $2.5 million was performance-based upon the approval of MIPLYFFA.
  • Net Loss: ($33.2) million, or ($0.69) per basic and diluted share for Q3 2024, compared to a net loss of ($10.4) million, or ($0.30) per basic and diluted share in Q3 2023.
  • Cash Position: Cash, cash equivalents and investments were $95.5 million as of Sept. 30, 2024.
  • Cash Runway Forecast: Based on the Company’s current operating plan, available cash, cash equivalents and investments are expected to extend Zevra’s cash runway into 2027, subject to continuing compliance with debt covenants. Cash runway forecast includes anticipated revenue from MIPLYFFA sales, reimbursements from the French EAP for arimoclomol, royalties under the AZSTARYS license agreement, and continued investments into our development pipeline programs. It does not include potential proceeds from a PRV sale.
  • Common and Fully Diluted Shares O/S: As of Sept. 30, 2024, total shares of common stock outstanding were 53,227,364, and fully diluted common shares were 67,698,898, which included 5,483,537 shares issuable upon exercise of warrants.

Upcoming Events

  • Guggenheim Securities Healthcare Innovation Conference, with fireside chat on Wednesday, Nov. 13, 2024, at 4:00 p.m. ET. Management will be available for one-on-one meetings with registered attendees.

Conference Call Information - UPDATED

Zevra will host a conference call and audio webcast today at 4:30 p.m. ET to discuss its corporate update and financial results for the third quarter of 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 ninety (90) days beginning at approximately 5:30 p.m. ET on Nov. 12, 2024. 

Additionally, interested participants and investors may access the conference call by dialing either:

  • (785) 838-9345 (United States) -- updated dial-in
  • +1 (203) 518-9814 (International)
  • Conference ID: ZVRAQ324

About MIPLYFFA

MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. MIPLYFFA was granted Breakthrough Therapy designation, Rare Pediatric Disease designation, Orphan Drug designation, and Fast Track designation by the FDA for the treatment of NPC. MIPLYFFA was further granted Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.

INDICATIONS AND USAGE

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions:

Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve.

Embryofetal Toxicity:

MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.

Increased Creatinine without Affecting Glomerular Function:

Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function.

During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.

The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.

Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema.

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch.

Before prescribing MIPLYFFA, please read the full Prescribing Information, including Instructions for Use.

Drug Interaction(s):

Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.

Use in Females and Males of Reproductive Potential:

Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights.

Renal Impairment:

The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function.

MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg.

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). OLPRUVA is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For more information, please visit www.OLPRUVA.com.

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 KP1077

Serdexmethylphenidate (SDX) is Zevra’s proprietary prodrug of d-methylphenidate (d-MPH) and is the sole active pharmaceutical ingredient (API) of KP1077. KP1077 has been granted Orphan Drug designation by the FDA, and by the European Commission, for the treatment of Idiopathic Hypersomnia, or IH. The U.S. Drug Enforcement Agency (DEA) has classified SDX 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. In addition, KP1077 has intellectual property protection through 2037.

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 the DiSCOVER trial being conducted under a Special Protocol Assessment (SPA) agreement with the 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 Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a commercial-stage 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, Inc. and its affiliates and are subject to the Company's Expanded Access Program (EAP) policy, as published on its website. 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 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 any products or product candidates for any specific disease indication or at any dosage; the potential benefits of any of our products or product candidates for any specific disease or at any dosage; the impact of meetings or communications with the FDA or any advisory committee; statements or decisions by the FDA or any other entity for any products or product candidates; our ability to monetize any PRV; future research and development activities; our strategic and product development objectives, including with respect to becoming a leading, commercially focused rare disease company; the potential benefits of our debt facility; our financial position, including our cash balance; our corporate governance objectives; potential revenues from our arimoclomol expanded access program in France; the potential for royalty and milestone contributions, the presentation of data at conferences; and the timing of any of the foregoing. 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 Dec. 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three months ended Sept. 30, 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.

Zevra Contact

Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com

Russo Partners Contact

David Schull
+1 (858) 717-2310
david.schull@russopartnersllc.com


ZEVRA THERAPEUTICS, INC. 
UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
 
  Three months ended September 30,  Nine months ended September 30, 
  2024  2023  2024  2023 
Revenue, net $3,695  $2,895  $11,569  $14,541 
Cost of product revenue (excluding $1,545 and $4,619 in intangible asset amortization for the three and nine months ended September 30, 2024, respectively shown separately below)  2,303   144   6,051   946 
Intangible asset amortization  1,545      4,619    
Operating expenses:                
Research and development  10,945   12,297   33,743   28,385 
Selling, general and administrative  16,208   5,818   38,743   19,657 
Total operating expenses  27,153   18,115   72,486   48,042 
Loss from operations  (27,306)  (15,364)  (71,587)  (34,447)
Other (expense) income:                
Interest expense  (2,312)  (366)  (5,157)  (745)
Fair value adjustment related to warrant and CVR liability  (4,746)  3,678   4,660   4,253 
Fair value adjustment related to investments  90   124   64   451 
Interest and other income (expense), net  1,049   1,738   2,248   4,331 
Total other (expense) income  (5,919)  5,174   1,815   8,290 
Loss before income taxes  (33,225)  (10,190)  (69,772)  (26,157)
Income tax expense     (177)      
Net loss $(33,225) $(10,367) $(69,772) $(26,157)
                 
Basic and diluted net loss per share of common stock:                
Net loss $(0.69) $(0.30) $(1.59) $(0.76)
                 
Weighted average number of shares of common stock outstanding:                
Basic and diluted  47,808,817   34,724,614   43,843,851   34,364,075 


ZEVRA THERAPEUTICS, INC. 
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS 
(in thousands, except share and par value amounts)
 
  September 30,  December 31, 
  2024  2023 
Assets        
Current assets:        
Cash and cash equivalents $54,039  $43,049 
Securities at fair value, current  35,337   24,688 
Accounts and other receivables  7,790   17,377 
Prepaid expenses and other current assets  2,276   1,824 
Total current assets  99,442   86,938 
Inventories  8,756   9,841 
Securities at fair value, long-term  6,105    
Property and equipment, net  607   736 
Operating lease right-of-use assets  820   790 
Goodwill  4,701   4,701 
Intangible assets, net  70,608   69,227 
Other long-term assets  512   94 
Total assets $191,551  $172,327 
         
Liabilities and stockholders' equity        
Current liabilities:        
Accounts payable and accrued expenses $24,464  $28,403 
Line of credit payable     37,700 
Current portion of operating lease liabilities  540   543 
Current portion of discount and rebate liabilities  8,547   4,550 
Other current liabilities  930   2,524 
Total current liabilities  34,481   73,720 
Long-term debt  58,904   5,066 
Warrant liability  13,902   16,100 
Operating lease liabilities, less current portion  483   456 
Discount and rebate liabilities, less current portion  8,490   7,663 
Other long-term liabilities  5,521   7,458 
Total liabilities  121,781   110,463 
         
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 September 30, 2024, or December 31, 2023      
Common stock, $0.0001 par value, 250,000,000 shares authorized, 54,803,056 shares issued and 53,227,364 shares outstanding as of September 30, 2024; 43,110,360 shares issued and 41,534,668 shares outstanding as of December 31, 2023  5   4 
Additional paid-in capital  550,413   472,664 
Treasury stock, at cost  (10,983)  (10,983)
Accumulated deficit  (469,550)  (399,778)
Accumulated other comprehensive income (loss)  (115)  (43)
Total stockholders' equity  69,770   61,864 
Total liabilities and stockholders' equity $191,551  $172,327 

FAQ

What was Zevra Therapeutics (ZVRA) revenue in Q3 2024?

Zevra Therapeutics reported revenue of $3.7 million in Q3 2024, consisting of $2.6 million from arimoclomol EAP reimbursements, $1.1 million in AZSTARYS royalties, and minimal OLPRUVA revenue.

When did FDA approve MIPLYFFA for Zevra Therapeutics (ZVRA)?

The FDA approved MIPLYFFA on September 20, 2024, as the first FDA-approved treatment for Niemann-Pick disease type C.

What is Zevra Therapeutics (ZVRA) cash position as of Q3 2024?

Zevra reported cash, cash equivalents and investments of $95.5 million as of September 30, 2024, with an expected runway into 2027.

How many MIPLYFFA prescriptions has Zevra (ZVRA) received since approval?

As of October 31, 2024, Zevra received 90 prescription enrollment forms for MIPLYFFA, with 30% approved for reimbursement and ready for fulfillment.

Zevra Therapeutics, Inc.

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Biotechnology
Pharmaceutical Preparations
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United States of America
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