Zevra Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2006-01-01
- Employees
- 69
- Market Cap
- $379.3M
- Website
- http://zevra.com
- Introduction
Zevra Therapeutics, Inc. is a commercial-stage rare disease company that combines science, data and patient needs to create therapies for diseases with limited or no treatment options. The firm focuses on the treatment of rare neurological and sleep conditions such as certain Urea Cycle Disorders, Niemann Pick Disease Type C and Idiopathic Hypersomnia. The company receives royalties for AZSTARYS from its commercial partner Corium. The company was founded by Christal M. M. Mickle and Travis C. Mickle on October 30, 2006 and is headquartered in Celebration, FL.
U.S. Drug Prices Double as Pharmaceutical Industry Shifts Focus to Rare Diseases
• The median annual list price for newly launched pharmaceuticals in the U.S. has more than doubled to $370,000 in 2024 compared to $180,000 in 2021, according to a Reuters analysis.
• Treatments for rare diseases now account for 72% of new drug launches, up from 51% in 2019, with over 40% of these orphan drug approvals targeting oncology indications.
• The highest-priced new therapy is Orchard Therapeutics' Lenmeldy, a gene therapy for metachromatic leukodystrophy, costing patients over $4 million per year.
XOMA Royalty Expands Portfolio to Over 120 Assets, Reports Financial Results for 2024
• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator.
• The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments.
• Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.
Hypersomnia Pipeline Shows Promise with 10+ Emerging Therapies in Development
• DelveInsight's latest report reveals 8+ companies developing 10+ therapies for hypersomnia, with Avadel's Lumryz, Zevra's KP1077, and Alkermes' ALKS 2680 among the key candidates advancing through clinical trials.
• Recent clinical milestones include Axsome's AXS-12 meeting its Phase III primary endpoint with 72-82% reduction in cataplexy attacks, and Takeda's TAK-861 showing promise as the first treatment targeting the underlying pathophysiology of narcolepsy type 1.
• The hypersomnia market is driven by increased awareness, unmet medical needs, and advances in neuroscience, though challenges remain including lack of biomarkers, high trial costs, and regulatory hurdles.
Congress's Failure to Renew Priority Review Voucher Program Threatens Rare Disease Drug Development
• The FDA's rare pediatric disease Priority Review Voucher program expired in December 2023, despite unanimous congressional support for renewal, leaving biotech companies unable to receive new vouchers worth approximately $150 million.
• Eleven priority review vouchers were awarded in 2023, marking a record year, with beneficiaries including treatments for sickle cell disease, Duchenne muscular dystrophy, and other rare conditions.
• The program's suspension threatens future rare disease drug development, as many companies rely on voucher sales for funding, with existing designations only valid until September 2026 for voucher eligibility.
Praxis's Phase 3 Essential Tremor Drug Trial Faces Futility Concerns, Shares Plunge 40%
• Praxis Precision Medicines faces a major setback as trial monitors recommend stopping Phase 3 study of ulixacaltamide for essential tremor due to likely efficacy endpoint failure.
• Bristol Myers Squibb continues its cost-cutting initiative, announcing 223 additional layoffs in Lawrence Township, N.J., as part of its plan to save $2 billion annually by 2027.
• Eli Lilly partners with Magnet Biomedicine in a potential $1.25 billion deal to develop molecular glue drugs for oncology and immunology applications.
FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months
• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration.
• Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients.
• Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.
Zevra Therapeutics Secures $150M Deal for Rare Pediatric Disease Priority Review Voucher
• Zevra Therapeutics has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, with the transaction expected to close within 30-45 days.
• The PRV was awarded following FDA approval of MIPLYFFA, a treatment for neurological manifestations of Niemann-Pick disease type C in patients aged 2 and older.
• The deal is subject to standard closing conditions, including Hart-Scott Rodino Antitrust Improvements Act waiting period expiration, with Cantor Fitzgerald serving as financial advisor.
FDA Expands Rare Disease Research Support with $27 Million in Grants and New Innovation Hub
• The FDA has awarded $17.2 million in clinical trial grants for seven rare disease treatments, including therapies for Cushing's syndrome, blood disorders, cancer, and an inherited eye disease.
• An additional $4.7 million will fund natural history studies for rare conditions, while $5.4 million supports research on rare neurodegenerative diseases including CADASIL and Niemann-Pick disease.
• The agency is launching a rare disease innovation hub co-led by CDER and CBER directors to accelerate treatment development for ultra-rare conditions and create stronger connections between developers and patient communities.
Dual Blood Pressure Medication Strategy Halves Arterial Events in vEDS Patients
• A Phase 3 ARCADE trial demonstrates that combining irbesartan with celiprolol reduces severe arterial events by 50% in vascular Ehlers-Danlos syndrome patients over two years.
• The study showed significant reduction in both symptomatic and asymptomatic arterial lesions, with patients on irbesartan experiencing fewer events (13) compared to placebo (35).
• Irbesartan, an affordable generic medication, demonstrated a favorable safety profile with only minor adjustments needed for blood pressure management in some patients.
Biopharma Industry Faces Pivotal Year: J.P. Morgan Conference Signals Key 2025 Challenges
• The 2024 J.P. Morgan Healthcare Conference kicked off with significant M&A activity, including Johnson & Johnson's $15 billion acquisition of Intra-Cellular Therapies.
• Despite notable deals from GSK and Eli Lilly with biotech companies, industry sentiment remains cautious amid regulatory uncertainty and questions about investment returns.
• Key challenges facing the sector include Trump administration's healthcare policies, investor confidence in biotech, success of immunology and obesity drug investments, and growing Chinese competition.
Aquestive Therapeutics Advances Anaphylm NDA Submission and Alopecia Areata Trial
• Aquestive Therapeutics is on track to submit its NDA for Anaphylm sublingual film in Q1 2025, aiming to provide the first orally delivered epinephrine for severe allergic reactions.
• The company is actively recruiting for the Anaphylm pediatric clinical trial, expanding access to this potential treatment across different age groups.
• Aquestive's AQST-108 topical gel completed a pre-IND meeting with the FDA and is set to begin a Phase 2a trial for alopecia areata in Q2 2025.
FDA Approves Zepbound as First Drug for Obstructive Sleep Apnea in Obese Adults
• The FDA has approved Zepbound (tirzepatide) as the first medication for treating moderate-to-severe obstructive sleep apnea (OSA) in obese adults.
• Clinical trials showed Zepbound significantly reduced breathing disruptions and promoted weight loss compared to placebo, with nearly half of patients experiencing remission of OSA symptoms.
• The drug is intended for use alongside a reduced-calorie diet and increased physical activity, offering a new approach to managing OSA and obesity.
• While Zepbound shows promise, it's not suitable for all OSA patients, and continued weight loss is required for sustained effectiveness; common side effects include gastrointestinal issues.
Landmark Medical Advances of 2024: HIV Prevention, Schizophrenia Treatment, and More
• Lenacapavir offers a twice-yearly injectable option for HIV prevention, demonstrating high efficacy in trials, particularly benefiting populations with adherence challenges.
• Xanomeline-trospium (KarXT) introduces a novel mechanism targeting muscarinic receptors for schizophrenia treatment, reducing symptoms without the common side effects of older antipsychotics.
• Tirzepatide shows promise in weight loss and diabetes prevention, with trials indicating significant reductions in diabetes risk and sustained weight management.
• Osimertinib extends survival for advanced EGFR-positive non-small cell lung cancer patients, significantly delaying disease progression and reducing brain metastases.
Zevra Therapeutics Launches MIPLYFFA, First FDA-Approved Treatment for Niemann-Pick Disease Type C
• Zevra Therapeutics has commercially launched MIPLYFFA™ (arimoclomol), the first FDA-approved treatment for Niemann-Pick Disease Type C (NPC).
• MIPLYFFA is approved for use in combination with miglustat to treat neurological symptoms in adult and pediatric NPC patients aged 2 years and older.
• Zevra's patient support program, AmplifyAssist™, will provide resources to help patients navigate prescription and insurance processes for MIPLYFFA.
• Zevra is also focusing on increasing awareness and diagnosis of NPC, which is often misdiagnosed, to broaden the reach of its new therapy.
Zevra Therapeutics Launches MIPLYFFA, First FDA-Approved Treatment for Niemann-Pick Disease Type C
• Zevra Therapeutics has launched MIPLYFFA (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), now available for patients aged two and older.
• MIPLYFFA, prescribed with miglustat, has shown promise in halting disease progression over one year, as measured by the NPC Clinical Severity Scale in a Phase 3 trial.
• The drug works by enhancing lysosomal function, aiding in the clearance of accumulated lipids from cells, and has received multiple designations from the FDA and EMA.
• Zevra's AmplifyAssist program supports patients with insurance, copay assistance, and educational resources to ensure consistent access to MIPLYFFA treatment.
Zevra Therapeutics Receives Buy Rating Following FDA Approval of Miplyffa for Niemann-Pick Disease Type C
• Zevra Therapeutics maintains a Buy rating due to its strong financial position, including $95.5 million in cash reserves expected to support operations until 2027.
• The FDA's approval of Miplyffa for Niemann-Pick disease type C (NPC) positions Zevra to capitalize on a lucrative market, further boosted by a Priority Review Voucher.
• Miplyffa's positive reception and the ongoing launch of Olpruva, with expanding market presence and patient enrollments, contribute to the positive outlook.
• Progress in Zevra's KP1077 and celiprolol programs indicates promising future growth prospects, reinforcing the Buy recommendation.
Zevra Therapeutics' Celiprolol Under Evaluation in Phase 3 Trial for vEDS
• Zevra Therapeutics is conducting a Phase 3 clinical trial (DiSCOVER) to assess celiprolol's efficacy in reducing medical emergencies in vascular Ehlers-Danlos syndrome (vEDS) patients.
• The DiSCOVER trial, enrolling 150 vEDS patients with COL3A1 mutations, compares celiprolol to placebo in reducing the occurrence of blood vessel or organ rupture and unexplained sudden death.
• Celiprolol, a beta blocker, aims to relax and widen blood vessels, potentially lowering pressure and reducing rupture risk in vEDS patients, who currently have limited treatment options.
• The FDA has granted celiprolol breakthrough therapy and orphan drug status for vEDS, acknowledging its potential benefit over existing treatments and providing development incentives.
PTC Therapeutics Receives Buy Ratings Following FDA Approval of Gene Therapy Kebilidi
• PTC Therapeutics received a Buy rating from William Blair analyst Sami Corwin due to the FDA approval of Kebilidi, a gene therapy for AADC deficiency.
• Kebilidi is the first gene therapy administered directly to the brain, holding a broad label for treating a wide range of patients with AADC deficiency.
• The company's proactive commercial launch preparations, coupled with a priority review voucher, provide financial advantages and growth prospects.
• Robert W. Baird also assigned a Buy rating to PTC Therapeutics stock, with a price target of $48.00, reinforcing positive market sentiment.
Zevra Therapeutics Reports Positive Q3 2024 Results Driven by MIPLYFFA Approval
• Zevra Therapeutics' Q3 2024 was highlighted by FDA approval of MIPLYFFA for Niemann-Pick disease type C (NPC), marking a significant milestone for the company.
• The company has received 90 prescription enrollment forms for MIPLYFFA, with 30% approved for reimbursement, indicating strong initial uptake post-launch.
• Zevra is refining its commercial strategy for OLPRUVA to focus on specific patient segments and has launched a patient education campaign for Urea Cycle Disorders (UCDs).
• With $95.5 million in cash, cash equivalents, and investments, Zevra's financial runway extends into 2027, supporting continued growth and pipeline development.
Zevra Therapeutics Highlights FDA Approval of MIPLYFFA and Outlines Strategic Vision for Growth
• Zevra Therapeutics celebrated FDA approval of MIPLYFFA (arimoclomol) for Niemann-Pick disease type C (NPC), marking a pivotal milestone and receipt of a rare pediatric disease Priority Review Voucher.
• The company reported 90 prescription enrollment forms for MIPLYFFA, with 30% approved for reimbursement, aligning with expectations for product availability post-launch.
• Zevra is refining its commercial strategy for OLPRUVA, focusing on specific patient segments and launching a targeted patient education campaign for Urea Cycle Disorders (UCDs).
• With a strong cash position of $95.5 million, Zevra is strategically focused on commercial excellence, pipeline innovation, talent development, and corporate foundation to drive long-term value.
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