CELEBRATION, Fla., Feb. 25, 2026 (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 members of Zevra’s executive leadership team will present at the Citizens Life Sciences Conference in Miami Beach, FL on Wednesday, March 11, 2026, at 9:00 a.m. ET. Additionally, management will be available for one-on-one meetings with registered conference attendees. A link ...

Company Overview - Insmed Incorporated (NASDAQ:INSM) is a biopharmaceutical company focused on developing therapies for rare diseases, known for its innovative approach to addressing unmet medical needs [1] - Competitors include other biotech firms specializing in rare diseases, such as Vertex Pharmaceuticals and BioMarin Pharmaceutical [1] Financial Performance - On February 19, 2026, Stifel Nicolaus set a price target of $205 for INSM, indicating a potential upside of 28.22% from the stock's price of $159.88 at that time, reflecting a strategic reassessment from a previous target of $212 [2] - The stock is currently trading at $161, showing a 6.54% increase with a recent price change of $9.89 [2] - Insmed's Q4 2025 earnings call provided insights into the company's financial health and strategic plans, covering key financial metrics such as revenue and profit margins [3] Stock Performance - The stock has shown significant volatility, with a daily range between $147.26 and $163.14 [4] - Over the past year, INSM has reached a high of $212.75 and a low of $60.40, indicating substantial price fluctuations [4] - The company's market capitalization is approximately $34.34 billion, with a trading volume of 4,921,823 shares, reflecting active investor interest [4]

Core Insights - PTC Therapeutics has experienced a significant increase in investment from Palo Alto Investors, which raised its stake by 456,144 shares, valued at approximately $33.1 million, indicating strong investor confidence in the company's future prospects [1][2][10] Company Overview - PTC Therapeutics specializes in the discovery, development, and commercialization of medicines for rare diseases, supported by a diversified portfolio of marketed products and late-stage pipeline candidates [6] - As of the latest report, PTC's market capitalization stands at $6 billion, with a trailing twelve months (TTM) revenue of $1.8 billion and a net income of $751.7 million, a significant improvement from a net loss of $106.7 million the previous year [4][9] Financial Performance - The company reported $211 million in revenue for the third quarter, alongside a net income of $15.9 million, showcasing a strong turnaround and profitability [7][9] - The stock price of PTC Therapeutics has risen to $74.72, reflecting a 49% increase over the past year, significantly outperforming the S&P 500's 13% gain during the same period [3][4] Investment Insights - Palo Alto Investors now holds 9.8% of its $540.4 million in reportable U.S. equity assets in PTC, making it one of the fund's top holdings, which suggests a belief in the company's sustained momentum despite regulatory uncertainties [3][10] - The launch of Sephience has been identified as a growth catalyst, generating $19.6 million in Q3 sales and showing early traction with 521 start forms in the U.S. and 341 patients on therapy worldwide [9][10] Future Outlook - The key question for long-term investors is whether the late-stage pipeline and Sephience can support sustained revenue growth as legacy products decline, with PTC holding cash reserves of $1.7 billion for flexibility during upcoming regulatory milestones [11]

Core Insights - Tang Capital Management increased its stake in PTC Therapeutics by 400,000 shares, raising its total holdings to 1.5 million shares valued at $92.1 million as of September 30, reflecting a $38.3 million increase in net position [2][11] - PTC Therapeutics has shown strong commercial momentum with the successful launch of Sephience, contributing $19.6 million in revenue during the third quarter, and the company reported a quarterly net profit of $15.9 million, a significant turnaround from a $106.7 million loss a year earlier [10][11] - The stock price of PTC Therapeutics reached $78.50, marking a 51% increase over the past year, significantly outperforming the S&P 500, which rose by 13% in the same period [3][4] Company Overview - PTC Therapeutics is a mid-cap biopharmaceutical company focused on developing therapies for rare genetic disorders, with a market capitalization of $6.3 billion and a revenue of $1.8 billion over the trailing twelve months [4][6] - The company has a diversified portfolio of approved products and a robust pipeline addressing unmet medical needs in the rare disease sector, including therapies like Translarna, Emflaza, Tegsedi, Waylivra, and Evrysdi [9][6] - PTC Therapeutics generates revenue primarily through the sale and licensing of its pharmaceutical products and strategic collaborations with industry partners, targeting healthcare providers and specialty pharmacies across various regions [9][6] Financial Performance - In the third quarter, PTC Therapeutics reported revenue of $211 million, driven by the Sephience launch and increased royalty income from Evrysdi [11] - The company's net income for the trailing twelve months stands at $751.7 million, indicating a strong financial position [4]

Core Insights - Armistice Capital sold 2,152,000 shares of Travere Therapeutics, reducing its position by approximately $29.3 million in the third quarter [2][7] - Following the sale, Armistice holds 6.7 million shares valued at $160.7 million, representing 2% of its assets under management (AUM) [3][4] - Travere Therapeutics' stock price increased by 100% over the past year, significantly outperforming the S&P 500, which rose nearly 14% in the same period [4] Company Overview - Travere Therapeutics has a market capitalization of $3.2 billion and reported a revenue of $435.8 million for the trailing twelve months (TTM) [5] - The company incurred a net loss of $88.5 million in the TTM [5] - As of the latest market close, Travere's share price was $35.44 [5] Business Focus - Travere Therapeutics specializes in therapies for rare diseases, with marketed products including Chenodal, Cholbam, and Thiola, and is advancing clinical candidates like Sparsentan and TVT-058 [6][9] - The company targets high unmet medical needs in the biotechnology sector, leveraging clinical expertise and strategic partnerships for growth [9] Recent Performance - Travere Therapeutics reported a strong quarter with total revenue reaching $164.9 million, driven by a 155% year-over-year increase in FILSPARI's U.S. sales [10] - The company achieved a GAAP net income of $25.7 million, a significant turnaround from a $54.8 million loss a year earlier [10] Investment Perspective - Despite the sale of shares, Armistice Capital remains heavily invested in Travere, which still accounts for 5% of its AUM and is its second-largest holding [11] - The sale appears to be a disciplined portfolio management decision following a significant share price rebound rather than a change in investment thesis, especially with an upcoming FDA decision in FSGS [11] - Long-term investors may find Travere appealing due to its accelerating commercial uptake, improving profitability, and upcoming regulatory catalysts [12]

Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on rare diseases, with a portfolio that includes six self-marketed products worldwide [4] - The company has a robust R&D pipeline, highlighted by its small molecule splicing platform, which has produced Evrysdi for spinal muscular atrophy (SMA) and Votoplam for Huntington's disease [4] Financial Performance - For the third quarter, PTC Therapeutics reported strong performance with revenues of $211 million, which included contributions from Sephience, their phenylketonuria (PKU) drug [5] - The company maintains a strong financial position with approximately $1.7 billion on its balance sheet [5] Product Launch - The launch of Sephience is a significant event for the company, providing a foundation for future growth [5]

Financial Data and Key Metrics Changes - Catalyst Pharmaceuticals reported total revenue of $148.4 million for Q3 2025, a 15.3% increase year over year from $128.7 million in Q3 2024 [4][21] - Net income before income taxes for Q3 2025 was $71.0 million, reflecting a 24.2% increase compared to $57.2 million in Q3 2024 [22] - GAAP net income for Q3 2025 was $52.8 million, or $0.42 per diluted share, up 20.3% from $43.9 million, or $0.35 per diluted share in Q3 2024 [22] - Non-GAAP net income for Q3 2025 was $86.1 million, or $0.68 per diluted share, compared to $71.1 million, or $0.57 per diluted share in Q3 2024 [23] - The company ended Q3 2025 with a cash position of $689.9 million and no debt, an increase from $517.6 million at the end of 2024 [5][26] Business Line Data and Key Metrics Changes - Firdapse generated revenue of $92.2 million in Q3 2025, a 16.2% increase year over year [6][14] - AGAMREE achieved net product revenue of $32.4 million in Q3 2025, a 115.2% increase from Q3 2024 [8][18] - FYCOMPA reported revenue of $23.8 million in Q3 2025, a decrease of 25.8% year over year due to generic competition [9][19] Market Data and Key Metrics Changes - The company raised its total revenue guidance for 2025 to between $565 million and $585 million, reflecting strong performance across its product lines [5][20] - Firdapse's full-year net product revenue guidance was maintained at between $355 million and $360 million [8][15] - AGAMREE's full-year net product revenue guidance was raised to between $105 million and $115 million [9][19] Company Strategy and Development Direction - Catalyst Pharmaceuticals is focused on expanding its market presence in both idiopathic and cancer-associated LEMS, with a strong emphasis on patient identification and education [7][16] - The company is actively pursuing business development opportunities and evaluating a broad range of potential acquisitions [12] - Catalyst Pathways, a personalized treatment support program, is a key differentiator in enhancing patient engagement and adherence [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term outlook, citing strong demand for rare disease therapies and operational excellence [4][5] - The company anticipates continued growth momentum into 2026, particularly for Firdapse and AGAMREE [8][17] - Management acknowledged the challenges posed by generic competition but remains optimistic about brand loyalty for FYCOMPA [19][33] Other Important Information - Catalyst announced a new share buyback program to repurchase up to $200 million of its common stock [5] - The company is committed to defending its intellectual property, with ongoing litigation against Hetero USA Incorporated set for trial in March 2026 [13][86] Q&A Session Summary Question: Insights on Firdapse's oncology efforts and Fycompa's brand loyalty - Management indicated that they are in the early stages of penetrating the oncology market for LEMS and will provide updates in future quarters [30][31] - Regarding Fycompa, management noted that brand loyalty remains strong, but they expect some revenue erosion as more generics enter the market [33][34] Question: Adoption of NCCN guidelines and Fycompa's stickiness - Management stated that the adoption of updated NCCN guidelines is more of a 2026 story, with ongoing efforts to educate oncology practices [41][42] - The stickiness of Fycompa is attributed to patient reluctance to switch medications, especially for anti-seizure treatments [38][39] Question: AGAMREE prescriber base and lifecycle management opportunities - Management reported strong adoption of AGAMREE across key treatment centers, with ongoing efforts to deepen prescribing [45][46] - Lifecycle management opportunities for AGAMREE are being evaluated, focusing on rare therapeutic areas [47][48] Question: Firdapse growth drivers and guidance rationale - Management highlighted that growth for Firdapse will come from both idiopathic and cancer-associated LEMS, with strong new patient enrollments [62][63] - The decision to maintain guidance rather than raise it reflects a cautious approach, anticipating potential seasonality in Q4 [67][68]

Core Viewpoint - Zevra Therapeutics, Inc. will report its third quarter 2025 corporate and financial results on November 5, 2025, followed by a conference call at 4:30 p.m. ET [1] Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on developing therapies for rare diseases that have limited or no treatment options [3] - The company's mission is to provide life-changing therapeutics to individuals living with rare diseases through innovative, data-driven development and commercialization strategies [3] Upcoming Events - The third quarter 2025 results will be released via a news release after market close on November 5, 2025 [1] - An audio webcast of the conference call will be available on the "Events & Presentations" page of Zevra's Investor Relations website [2] - A replay of the webcast will be accessible for 90 days starting approximately at 5:30 p.m. ET on the same day [2]

Company Overview - Zevra Therapeutics, Inc. is a commercial-stage company focused on developing therapies for rare diseases with limited or no treatment options, aiming to provide life-changing therapeutics to affected individuals [3]. Upcoming Events - Members of Zevra's executive leadership team will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference in Boston, MA, on August 13, 2025, at 1:00 p.m. ET [1]. - Management will also be available for one-on-one meetings with registered conference attendees [2]. Investor Relations - A live webcast of the fireside chat will be accessible via the "Events & Presentations" section on Zevra's Investor Relations website [2].

Core Insights - Catalyst Pharmaceuticals, Inc. has appointed Dr. Daniel Curran to its Board of Directors, bringing over 25 years of pharmaceutical experience [1][2] - Dr. Curran's expertise includes strategy, business development, and rare disease knowledge, which will support Catalyst's growth strategy [3][4] - The company has a strong foundation in delivering innovative therapies for rare diseases and aims to expand its global presence [7] Company Overview - Catalyst Pharmaceuticals is focused on improving the lives of patients with rare diseases through innovative therapies [7] - The company has been recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list [7] - Catalyst emphasizes patient care and accessibility, providing comprehensive support services to ensure patients receive necessary treatments [7] Leadership Background - Dr. Curran has held significant roles at Takeda Pharmaceutical Company, including Senior Vice President and Head of the Rare Genetics and Hematology Therapeutic Area Unit [3][4] - He has also served in leadership positions at Millennium Pharmaceuticals and DuPont Merck Pharmaceuticals [4][5] - Dr. Curran holds an M.D. from the University of Pennsylvania, an M.B.A. from The Wharton School, and a B.S. in chemistry from King's College [6]