Latest news with #MIPLYFFA
Yahoo
14-05-2025
- Business
- Yahoo
Zevra Therapeutics Inc (ZVRA) Q1 2025 Earnings Call Highlights: Navigating Growth and Challenges
Net Revenue: $20.4 million for Q1 2025. MIPLYFFA Revenue: $17.1 million. OLPRUVA Revenue: $0.1 million. Operating Expense: $22.8 million for Q1 2025. R&D Expenses: $3.3 million, a decrease from the previous year. SG&A Expenses: $19.5 million, an increase of $9.6 million from the previous year. Net Loss: $3.1 million or $0.06 per share for Q1 2025. Cash, Cash Equivalents, and Investments: $68.7 million as of March 31, 2025. Total Debt: Approximately $60 million. Prescription Enrollment Forms for MIPLYFFA: 122 total, with 13 in Q1 2025. Prescription Enrollment Forms for OLPRUVA: 28 total, with 5 in Q1 2025. Covered Lives for MIPLYFFA: 38% coverage achieved by end of Q1 2025. Covered Lives for OLPRUVA: 78% coverage achieved. Warning! GuruFocus has detected 7 Warning Signs with ZVRA. Release Date: May 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Zevra Therapeutics Inc (NASDAQ:ZVRA) exceeded internal expectations for the MIPLYFFA launch, enrolling over one-third of diagnosed Niemann-Pick disease type C (NPC) patients in the US. The company added $148.3 million of non-dilutive capital to its balance sheet through the monetization of a pediatric rare disease Priority Review Voucher, enhancing financial strength. Zevra Therapeutics Inc (NASDAQ:ZVRA) was recognized on Fast Company's Top 10 Most Innovative Companies list in the medicines, therapeutics, and pharmaceuticals category. The Phase 3 DISCOVER trial for Celiprolol, targeting Vascular Ehlers-Danlos syndrome (VEDS), is progressing with patient enrollments, supported by Orphan Drug and Breakthrough Therapy designations. The company is actively expanding MIPLYFFA's availability outside the US, with plans for a Marketing Authorization Application in Europe, targeting approximately 1,100 people living with NPC. Despite the progress, Zevra Therapeutics Inc (NASDAQ:ZVRA) faces challenges with payer reimbursement, with only 38% of covered lives achieved for MIPLYFFA. The launch of OLPRUVA has been slower than expected, with only 28 prescription enrollment forms received since its availability in July 2023. The company reported a net loss of $3.1 million for the first quarter of 2025, although this was an improvement from the previous year. Zevra Therapeutics Inc (NASDAQ:ZVRA) is facing challenges in enrolling patients for the Phase 3 DISCOVER trial for Celiprolol, with only 32 patients enrolled out of a target of 150. The company has moderated expectations for OLPRUVA's launch pace due to unique dynamics in the UCD commercial landscape, indicating potential hurdles in market penetration. Q: Could you provide details on the number of patients currently on MIPLYFFA and the spread of reimbursed patients? A: We have received 122 enrollment forms since the launch, and while we don't report the exact number of patients actively on the drug, the majority are on MIPLYFFA. Those not yet on the drug are undergoing benefits investigation or receiving Quick Start, which is free drug while benefits are being verified. Q: What are the main reasons for reimbursement denials for MIPLYFFA? A: Initial denials are common with rare disease products due to the need for prior authorization. However, we've been successful in securing coverage through direct formulary status or medical exception processes, thanks to MIPLYFFA's demonstrated differentiation and clinical data. Q: How do you track the source of new MIPLYFFA patients, and what is the expected cadence for bringing them on board? A: We track patients from three cohorts: diagnosed, undiagnosed, and those from our Expanded Access Program (EAP). We continue to see enrollments from diagnosed and undiagnosed cohorts due to our awareness initiatives. Inventory levels are maintained at target levels to support patient needs. Q: Can you provide insights into refill rates, patient retention, and average net price for MIPLYFFA? A: Most patients who have started on MIPLYFFA continue to receive refills, indicating strong retention. The distribution of doses aligns with our EAP experience, with most patients receiving higher doses, which informs the average net price. Q: What does the 38% coverage of lives mean for MIPLYFFA, and how does it impact patient access? A: The 38% coverage indicates that plans covering a significant number of lives have reviewed and made MIPLYFFA available. This does not limit patient access, as we successfully secure reimbursement through medical exception pathways, and we expect coverage to increase as more plans evaluate the drug. Q: Can you update us on the enrollment progress for the celiprolol trial? A: We have enrolled 32 patients in the event-driven trial, which targets 150 patients. We are implementing strategies to accelerate enrollment, including targeting COL3A1 patient centers and using genetic testing to identify eligible patients. Q: How does the suspension of a competitor's trial impact your celiprolol trial timeline? A: The suspension of a competitor's trial highlights the unmet need in the space. While we can't provide a specific timeline for enrollment completion, our strategies are starting to pay off, and we are committed to advancing the trial. Q: Are all EAP patients now receiving MIPLYFFA through the commercial channel? A: The process of transitioning EAP patients to commercial drug involves benefits investigation and can vary in duration. Our team is working diligently to ensure patients receive the drug as soon as possible, with some transitions occurring within 24 hours and others taking longer. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. 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Yahoo
07-04-2025
- Business
- Yahoo
Zevra Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million
CELEBRATION, Fla., April 07, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. 'We have reached another key milestone with the closing on the sale of our PRV, which we received following the successful approval of MIPLYFFATM (arimoclomol) from the U.S. Food and Drug Administration (FDA),' said LaDuane Clifton, Zevra's Chief Financial Officer. 'The $150 million in gross proceeds is non-dilutive capital that further enhances our financial flexibility to support our strategic priorities, which includes executing on the commercial launches of MIPLYFFA and OLPRUVA®, supporting our ongoing Phase 3 trial for celiprolol, and augmenting our solid balance sheet to support future investments that are in alignment with our strategic plan.' Zevra was granted the PRV in September 2024 by the FDA, in connection with the FDA's approval of MIPLYFFA, the first U.S. approved treatment for the ultra-rare neurodegenerative disease, Niemann-Pick type C. In addition, the Company reported that available unaudited cash, cash equivalents and investments as of Mar. 31, 2025, were $68.7 million. Combined with the cash proceeds of $148.3 million, net of fees, from the sale of the PRV after the end of the quarter, cash, cash equivalents and investments would be $217.0 million. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare 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. For more information, please visit or follow us on X and LinkedIn. Financial Disclosure Advisory The cash, cash equivalents and investments information provided in this press release is based on preliminary unaudited information and management estimates for the quarter ended March 31, 2025, is not a comprehensive statement of the Company's financial results as of and for the fiscal quarter ended March 31, 2025 or any other period, and is subject to completion of the Company's financial closing procedures. The Company's independent registered public accounting firm has not conducted a review of and does not express an opinion or any other form of assurance with respect to this preliminary estimate. 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 any of our products or product candidates for any specific disease or at any dosage; the progress of our clinical trials, our strategic and product development objectives; the benefits of the transaction and its impact on the Company's balance sheet or strategic goals; our financial position, including our cash, cash equivalents and investments and available resources; 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, assumptions, 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. Given these risks and uncertainties, you should not place undue reliance on these 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, 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) 754-2545nochsner@ Media Contact Russo PartnersDavid Schull+1 (858) in to access your portfolio
Yahoo
27-02-2025
- Business
- Yahoo
Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million
CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions. LaDuane Clifton, Zevra's Chief Financial Officer said, 'This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA™ and OLPRUVA®, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.' The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which 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. The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act (HSR). Cantor Fitzgerald acted as Zevra's exclusive financial advisor and Latham & Watkins LLP acted as Zevra's legal advisor for this transaction. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare 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. For more information, please visit 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 potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives; the consummation and benefits of the transaction and its impact on the Company's balance sheet; the outcome of any required filings under the HSR; prescription enrollments; our ability to support patients as they navigate the benefits verification process to obtain either MIPLYFFA™ or OLPRUVA®; availability of and access to MIPLYFFA and OLPRUVA; 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, assumptions, 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. Given these risks and uncertainties, you should not place undue reliance on these 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 and nine months ended September 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) 754-2545nochsner@ Russo Partners Contact David Schull+1 (858) in to access your portfolio
