Latest news with #VascularEhlers-Danlos
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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Time of India
06-05-2025
- Health
- Time of India
This simple finger test may reveal your hidden risk for lung disease
Symptoms How to know if your joints are super-flexible Stiff joints can signal trouble, but super-flexible ones may even bring worse news. If you are able to bend your pinkie backward 90 degrees or touch your thumb to your forearm, this may not necessarily be a good of a rare genetic condition, uber-flexible joints may indicate your increased risk of respiratory illnesses and complicated recovery, as per experts.A quick flexibility test at home could help you uncover this hidden risk. This test is done to understand if your joints can go beyond the typical range of motion. Even if your joints are super-flexible, for most it isn't a sign of worry, but for some people, it could mean they may be suffering from a worrisome condition called Ehlers-Danlos syndrome Ehlers-Danlos syndrome affects your connective tissues - your skin, joints and blood vessel walls. People suffering from the condition have overly flexible joints and stretchy, fragile skin, according to can become a serious issue if you have a wound that needs stitches, as the skin may not be strong enough to hold them properly. Connective tissue, a complex blend of proteins and other elements, gives your body structure, strength, and flexibility, and when it's compromised, healing becomes much more difficult.A severe form of disorder could cause the walls of your blood vessels, intestines or uterus to rupture. People with the condition may have muscle weakness, including in the muscles that control inhaling and may report respiratory issues, including shortness of breath, difficulty with deep inhalation, sleep apnea, coughing, wheezing that mimics asthma and even chest pain that worsens with breathing in. Breathing pattern disorders are also common. For example, people with EDS may unconsciously adopt a 'bracing' pattern, where they hold their inhaled breath for too long," Jeannie Di Bon, a UK-based hypermobility and EDS specialist, told Bon, who has EDS, explains that people with the condition often adopt altered breathing patterns as a subconscious way to feel stable. However, this can reduce lung movement, particularly in the lower lungs, creating conditions that increase the risk of infections and complications from illnesses like COVID-19 and pneumonia. Vascular Ehlers-Danlos syndrome is marked by distinctive facial features like a thin nose, small earlobes, and prominent eyes, along with fragile, translucent skin that bruises easily. In fair-skinned individuals, veins may be clearly visible. The condition can weaken major arteries, including the aorta, increasing the risk of life-threatening ruptures. It can also affect the uterus and intestines, potentially leading to dangerous pain and dislocations are common in this disorder as the joints that can move far past the normal range of disorder, caused by weakened connective tissue, can make your skin unusually stretchy. You might be able to lift a section of skin away from your body more than normal, but it will quickly snap back into place. The skin may also feel exceptionally soft and velvety to the is also noticed that the skin doesn't heal properly as the stitches used to close a wound often tear out and leave a gaping often use the Beighton score to evaluate joint flexibility, which, when combined with other symptoms and family history, can help suggest the presence of Ehlers-Danlos Syndrome (EDS). The score is based on five simple flexibility tests: bending the pinkie finger backward 90 degrees, touching the thumb to the forearm, hyperextending the elbows and knees by more than 10 degrees, and bending forward to place palms flat on the floor without bending the knees. Each maneuver scores one point per side, except the last, which is a single score.A total score above five in adults (or six in children) indicates hypermobility and could be a reason to consult a medical professional about the possibility of EDS.
