CORAL GABLES, Fla., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today announced its inclusion in Forbes' esteemed list of America's Most Successful Small-Cap Companies for the year 2026. Catalyst has ranked an impressive 11 out of 100 companies on the list. “We are ho ...

About this content About Angela Harmantas Angela Harmantas is an Editor at Proactive. She has over 15 years of experience covering the equity markets in North America, with a particular focus on junior resource stocks. Angela has reported from numerous countries around the world, including Canada, the US, Australia, Brazil, Ghana, and South Africa for leading trade publications. Previously, she worked in investor relations and led the foreign direct investment program in Canada for the Swedish government ...

CAMBRIDGE, Mass., Jan. 05, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that its management team is scheduled to present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 8:15 a.m. PST / 11:15 a.m. EST. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under th ...

Core Viewpoint - Zevra Therapeutics has entered into an exclusive expanded access distribution agreement with Uniphar to provide Niemann-Pick Disease Type C (NPC) patients access to MIPLYFFA (arimoclomol) in select territories outside of Europe [1][3] Company Overview - Zevra Therapeutics is a commercial-stage company focused on therapies for rare diseases, aiming to address unmet needs in this community [14] - The company has developed MIPLYFFA, the only treatment shown to halt disease progression in NPC, with FDA approval received on September 20, 2024 [4][5] Product Information - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [5] - The drug has demonstrated a durable effect, halting disease progression as measured by the NPC Clinical Severity Scale over a one-year trial [4] - MIPLYFFA has shown long-term clinical outcomes with over 5 years of patient experience across more than 270 NPC patients [4] Distribution Agreement - The agreement with Uniphar aims to expand access to MIPLYFFA, leveraging Uniphar's expertise in global distribution and supply chain management [3][16] - This partnership is expected to initially address a select patient population while Zevra continues to prepare for its U.S. commercial launch and potential EU approval [3] Clinical Significance - MIPLYFFA increases the activation of transcription factors that regulate lysosomal expression and has been shown to reduce unesterified cholesterol in NPC fibroblasts [4] - The pivotal phase 3 trial results indicate significant clinical benefits, although the full clinical significance of these findings is not yet fully understood [4] Regulatory Status - MIPLYFFA is currently approved in the U.S. and is under review by the European Medicines Agency for marketing authorization in Europe [3][4]

Core Viewpoint - BioMarin Pharmaceutical Inc. has announced a definitive agreement to acquire Amicus Therapeutics for $14.50 per share in an all-cash transaction valued at approximately $4.8 billion, which is expected to enhance BioMarin's revenue growth and diversify its product portfolio in the rare disease market [2][3][4]. Group 1: Acquisition Details - The acquisition will add two marketed high-growth products, Galafold and Pombiliti + Opfolda, which generated combined revenues of $599 million over the past four quarters [1][4]. - The transaction has been unanimously approved by the Boards of Directors of both companies and is expected to close in the second quarter of 2026, pending regulatory clearances and stockholder approval [2][3]. - BioMarin intends to finance the acquisition through cash on hand and approximately $3.7 billion of non-convertible debt financing [8]. Group 2: Financial Impact - The acquisition is expected to be accretive to Non-GAAP Diluted Earnings Per Share (EPS) in the first 12 months post-close and substantially accretive beginning in 2027 [1][4][5]. - BioMarin is targeting a gross leverage of less than 2.5x within two years after the close of the transaction, demonstrating a commitment to deleveraging [5][8]. Group 3: Strategic Fit - The combination of BioMarin and Amicus is seen as a strategic fit due to both companies' dedication to transforming care for patients with rare diseases [3][4]. - The acquisition will strengthen BioMarin's commercial portfolio and provide opportunities to expand access to Galafold and Pombiliti + Opfolda across its global footprint [1][4][5]. Group 4: Product Information - Galafold is the first oral treatment for Fabry disease, while Pombiliti + Opfolda is a two-component therapy for Pompe disease, both targeting lysosomal storage disorders [4][15]. - Amicus also holds U.S. rights to DMX-200, an investigational small molecule for treating focal segmental glomerulosclerosis, which is in Phase 3 development [4].

Core Insights - Applied Therapeutics has entered into a definitive agreement for Cycle Group Holdings Limited to acquire the company, focusing on advancing its lead drug candidate, govorestat, for rare CNS metabolic diseases [1][3][6] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing novel drug candidates targeting rare diseases, with its lead candidate being govorestat, an Aldose Reductase Inhibitor for CNS rare metabolic diseases [2][10] Acquisition Details - Cycle will initiate a tender offer to acquire all outstanding shares of Applied common stock at a price of $0.088 per share in cash, along with a non-transferrable contingent value right (CVR) for potential additional payments [3][4] - The CVR includes payments based on FDA approvals and sales milestones, with potential cash payments of up to $0.10 for FDA approval of new drug applications for specific indications and up to $0.20 for achieving worldwide net sales of $200 million [8] Financial Support - Applied has issued a Promissory Note to Cycle, allowing for loans up to $8.5 million to support its working capital needs, with repayment obligations triggered if the transaction is terminated [5] Board Approval - The merger agreement was unanimously approved by the Applied Board of Directors after evaluating strategic alternatives, determining it to be in the best interest of the company and its stockholders [6]

Core Insights - AstraZeneca (AZN) received FDA approval for the expanded use of its oral selective MEK inhibitor, Koselugo (selumetinib), to treat symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1) in the United States [2][6]. Regulatory Approvals - Koselugo's label has been expanded in the EU, Japan, and other countries, with ongoing regulatory reviews [3][6]. - A granule formulation of Koselugo was also approved by the FDA for young children aged one year and older with NF1 PN [3]. Clinical Study Results - The FDA's approval was supported by data from the global phase III KOMET study, which showed a statistically significant overall response rate (ORR) of 20% for Koselugo compared to 5% for placebo by cycle 16 [4][6]. - Approximately 86% of patients receiving Koselugo achieved a duration of response of at least 6 months [5]. Market Performance - Year to date, AstraZeneca's shares have increased by 35.3%, outperforming the industry growth of 15.8% [5]. Disease Background - NF1 is a rare genetic disorder that can lead to the development of non-cancerous PN, affecting multiple organ systems and potentially causing significant health issues [7]. Collaboration and Financials - AstraZeneca has modified its partnership with Merck regarding Koselugo, assuming full global responsibility for the drug's costs, revenues, and profits [9].

Core Insights - Viridian Therapeutics, Inc. (NASDAQ:VRDN) is a biotechnology company focused on developing treatments for rare diseases, with RBC Capital setting a price target of $45, indicating a potential increase of about 63.64% from its current price of $27.50, reflecting strong confidence in the company's future performance [1][5] Financial Position - Viridian is positioned for profitability in 2026 due to successful financing transactions that have eliminated the need for further share dilution, which is a positive sign for investors [2][5] - The current stock price of VRDN is $27.50, showing a 12.61% increase or $3.08, with a market capitalization of approximately $2.23 billion and a trading volume of 4.86 million shares today [4] Competitive Landscape - The competitive landscape is shifting as Roche's Enspryng (satralizumab) has shown disappointing phase 3 results in treating thyroid eye disease, which reduces competitive pressure on Viridian's veligrotug and VRDN-003, potentially enhancing its market position and increasing market share in this therapeutic area [3][5]

Financial Data and Key Metrics Changes - The company reported a 15% top-line growth year-to-date and an 11% growth for the quarter, indicating strong momentum in its R&D pipeline and business development activities [4][20]. - The guidance for top-line growth for 2025 has been narrowed to a range of 8-11%, with corresponding adjustments to operating profit growth [20]. Business Line Data and Key Metrics Changes - The diabetes segment experienced a 10% growth, while obesity care saw a remarkable 41% growth, with 83% of this growth coming from International Operations (IO) and 24% from the US [10]. - Rare disease products returned to normal production levels, growing 13% in a balanced manner between IO and the US [10]. Market Data and Key Metrics Changes - The US market for anti-obesity medications continues to expand, with a significant increase in out-of-pocket expenses for patients, rising from 4% to over 10% in just nine months [11]. - The company is addressing a market of approximately 100 million patients in the US for obesity treatment, with only 3-4 million currently being treated [11]. Company Strategy and Development Direction - The company is sharpening its strategy to focus on treating patients with obesity and diabetes, emphasizing the overlap with comorbidities such as MASH (Metabolic Dysfunction-Associated Steatotic Liver Disease) [6][7]. - Recent acquisitions, including that of Akero, are aimed at deepening the company's focus on core therapy areas and addressing unmet needs in comorbidities associated with obesity and diabetes [7][15]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth potential in the obesity market, highlighting the significant unmet needs and the company's strategic focus on expanding its product portfolio [11][13]. - The company acknowledged challenges in the US market, including pricing pressures and competition, but remains committed to leveraging its pipeline and product offerings to drive future growth [20][49]. Other Important Information - The company is preparing for the regulatory submission of new products, including the oral semaglutide and MyMADE for rare diseases, which are expected to contribute to future revenue growth [19][50]. - Management emphasized the importance of understanding patient behaviors and needs in the consumer market, particularly in relation to comorbidities and treatment formats [35][100]. Q&A Session Summary Question: What do you expect to learn from the amycretin trials? - Management stated that they will not progress to phase three without clear differentiation in efficacy, safety, or scalability, and they are awaiting confirmation from phase two data [23]. Question: What are the pricing trends for GLP-1 medications? - Management indicated that pricing for Ozempic is expected to remain stable, with a year-on-year decrease of about 10-15%, while Wegovy's pricing has seen a decline in Q3 [26][27]. Question: How are you addressing the consumerization of the sales force? - The company is focusing on understanding patient needs and behaviors, emphasizing the importance of product formats and direct purchasing channels [30][35]. Question: What is the outlook for the UK market? - Management noted that the UK market is competitive and price-sensitive, but they are seeing strong performance from their products despite pricing pressures [43]. Question: Can you discuss the Metsera acquisition and potential FTC challenges? - Management expressed confidence in the acquisition's merits and their readiness to discuss it with the FTC, emphasizing the quality of the portfolio [52][56]. Question: What is the expected impact of the oral semaglutide launch? - Management believes that the oral market represents a significant opportunity, with expectations of capturing a substantial share due to its unique profile [95][100].

Core Insights - Amicus Therapeutics (FOLD) reported adjusted earnings of 17 cents per share for Q3 2025, surpassing the Zacks Consensus Estimate of 12 cents and up from 10 cents in the same quarter last year [1][6] - Revenues for Q3 totaled $169.1 million, reflecting a 19% year-over-year increase and exceeding the Zacks Consensus Estimate of $165 million [2][6] - The revenue growth was driven by sales of Galafold and the newly approved combination therapy Pombiliti + Opfolda, which is aimed at treating late-onset Pompe disease [2][3] Financial Performance - Galafold sales reached $138.3 million, a 12% increase year-over-year at constant exchange rates, although it fell short of the Zacks Consensus Estimate of $150 million [4][6] - Sales of Pombiliti + Opfolda amounted to $30.7 million, surpassing both the Zacks Consensus Estimate of $29.7 million and the internal estimate of $29.4 million [7] - Adjusted operating expenses rose by 15% to $95.4 million in Q3 2025, while cash and marketable securities increased to $263.8 million from $231 million as of June 30, 2025 [7] Guidance and Future Outlook - Amicus reaffirmed its 2025 guidance, projecting total revenue growth of 15-22% at constant exchange rates, with Galafold expected to grow by 10-15% and Pombiliti + Opfolda by 50-65% [8] - The company anticipates achieving positive GAAP net income in the second half of 2025 and aims to exceed $1 billion in total sales by 2028 [9] Recent Developments - Amicus licensed exclusive rights for Dimerix's phase III program, DMX-200, in the U.S., targeting FSGS, a rare kidney disease, with the pivotal ACTION3 study on track for full enrollment by the end of 2025 [10]