Core Viewpoint - Pharming Group N.V. reported preliminary, unaudited revenues for 2025, estimating approximately US$376 million, which exceeds previous guidance and indicates a 27% growth compared to 2024 [2][5] Financial Performance - Total revenues for 2025 are estimated at US$376 million, surpassing the revised guidance of US$365 to US$375 million [2] - This revenue growth is attributed to the continued success of RUCONEST® and increased demand for Joenja®, particularly in the U.S. market [2] - Full-year operating expenses are expected to be between US$304 million and US$308 million, reflecting the company's commitment to cost discipline [2] Future Outlook - The company anticipates sustained revenue growth and further advancements in its clinical pipeline for 2026 [2] - A complete financial report for Q4 and full year 2025 is scheduled for release on March 12, 2026 [2] Investor Day - Pharming will host a virtual Investor Day on February 3, 2026, to provide updates on its clinical pipeline and financial guidance for 2026 [1][3] - The agenda will include discussions on leniolisib and KL1333, focusing on their clinical trials and the unmet needs in related diseases [3][4] - Presentations will feature leading clinical experts discussing immune dysregulation and mitochondrial diseases, providing context for Pharming's development programs [4]

RESEARCH TRIANGLE PARK, N.C., Jan. 06, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company plans to present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 12, 2026, at 1:30 p.m. ET. A link to the live audio webcast and replay of the presentation may be accessed in the Investors & Media section of BioCryst’s website at www.biocryst.com. About BioCryst Pharmaceuticals BioCryst is a global biotechnology company focuse ...

Core Insights - Mereo BioPharma announced that neither the Phase 3 ORBIT nor COSMIC studies for setrusumab (UX143) achieved their primary endpoint of reducing annualized clinical fracture rates compared to placebo or bisphosphonates, respectively. However, both studies successfully met their secondary endpoints, showing significant improvements in bone mineral density (BMD) [1][2][3]. Study Results - In the ORBIT study, participants showed statistically significant improvements in BMD compared to placebo, consistent with Phase 2 study results, but did not see a corresponding reduction in fracture rates [3]. - The pediatric COSMIC study revealed that patients had a higher baseline fracture rate compared to those in ORBIT. Although setrusumab-treated patients showed meaningful BMD improvements and a reduction in fracture rates compared to bisphosphonate-treated patients, this reduction did not reach statistical significance [4]. Financial and Strategic Considerations - Mereo's cash balance was reported at $48.7 million at the end of Q3 2025. The company plans to manage cash resources carefully, implementing immediate reductions in pre-commercial and manufacturing activities while continuing to analyze the setrusumab data for future steps [5]. - The company is also advancing discussions for partnerships related to alvelestat, another of its drug candidates [2][9]. Product Development Context - Setrusumab is being developed for pediatric and young adult patients with Osteogenesis Imperfecta (OI) across various sub-types, with the ORBIT and COSMIC studies being pivotal in this development [6][8]. - The ORBIT study involved 159 patients aged 5 to 25 years, while the COSMIC study enrolled 69 patients aged 2 to under 7 years, both randomized to receive either setrusumab or a comparator [7][8]. Company Overview - Mereo BioPharma focuses on developing innovative therapeutics for rare diseases, with setrusumab being one of its key candidates for OI. The company has retained commercial rights in the EU and UK and has partnered with Ultragenyx Pharmaceutical for the development of setrusumab [12].

CORAL GABLES, Fla., Dec. 15, 2025 (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 that Rich Daly, President and CEO of Catalyst, along with other members of Catalyst's management team, will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January ...

Sanofi’s Qfitlia and Cablivi approved in China, expanding care for rare diseases Qfitlia, the first antithrombin-lowering therapy for hemophilia, can offer consistent protection with as few as six injections a yearCablivi, the first Nanobody medicine, targets acquired/immune-mediated thrombotic thrombocytopenic purpura — a rare, life-threatening blood clotting disorder Paris, December 11, 2025. The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rar ...

Core Insights - The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rare hematologic diseases: Qfitlia for hemophilia and Cablivi for acquired thrombotic thrombocytopenic purpura (aTTP) [1] - These approvals represent Sanofi's fourth and fifth approvals in China for the year, following Tzield and Sarclisa [1] Qfitlia Overview - Qfitlia is the first antithrombin-lowering therapy for routine prophylaxis in hemophilia patients aged 12 and older, including those with severe hemophilia A or B [2] - The approval is based on ATLAS phase 3 studies showing significant reductions in annualized bleeding rates (ABR) for patients treated with Qfitlia [3] - Qfitlia utilizes small-interfering RNA technology, allowing for low treatment frequency and subcutaneous administration [3][15] - Hemophilia affects over 40,000 individuals in China, highlighting the need for effective treatment options [3] Cablivi Overview - Cablivi is the first targeted therapy for treating aTTP in adults and adolescents aged 12 or older [4] - Approximately 2,700 patients are diagnosed with aTTP annually in China, with a mortality rate of up to 20% despite standard treatments [5] - Cablivi works by inhibiting the interaction between von Willebrand factor and platelets, thus preventing microthrombi formation [5][18] Market Impact - The approvals of Qfitlia and Cablivi expand Sanofi's rare hematology portfolio in China, addressing critical unmet needs in chronic bleeding disorders and acute clotting emergencies [6] - Sanofi's commitment to innovation is emphasized by these approvals, aiming to improve outcomes for patients with rare diseases [7] Clinical Results - Qfitlia demonstrated a 71% reduction in ABR for patients without inhibitors and a 73% reduction for patients with inhibitors compared to traditional treatments [9] - In the open-label extension study, nearly 80% of participants were on a regimen of six injections per year, with 94% achieving target AT levels [17]

Core Insights - Jupiter Neurosciences, Inc. is focused on treatments for neuroinflammation and rare diseases using its patented resveratrol delivery platform, JOTROL [1] - The company has a clinical pipeline with over $50 billion in combined market opportunities [1] Company Overview - Christer Rosen is the Co-Founder, CEO, and Chairman of Jupiter Neurosciences since January 1, 2016, with a background in founding and leading multiple businesses [2] - Rosen previously founded EffRx Pharmaceuticals, where he developed the osteoporosis drug Binosto, which received FDA and EU approvals [2] - Alison Silva serves as the President and Chief Business Officer of Jupiter Neurosciences, with extensive experience in drug development and regulatory strategy [4] - Silva has held leadership roles in various biotech companies, including Cotinga Pharmaceuticals and Synlogic, and has a strong background in orphan drug strategy [4][5]

Presentation[Audio Gap] My name is Geoff Meacham. I'm the senior biopharma analyst here, and I have Jarwei Fang from my team on here as well. We're thrilled today to have PTC Therapeutics. With us on stage is Matt Klein, CEO. Matt, welcome. Good to see you.So lots of questions on Sephience and on your R&D Day. But maybe just for those on the webcast, just give us a bit of a quick background or overall, and then we can get right into it.Matthew KleinCEO & Director Yes, absolutely. So PTC is a global biopharm ...

Financial Data and Key Metrics Changes - PTC Therapeutics reported a successful year in 2025, highlighted by the US and EU approval of Sapphiens, which is expected to lead the company to cash flow break-even and beyond in the near future [5][9] - The company is seeing strong momentum in the fourth quarter following the launch of Sapphiens, with early launch numbers being strong and not solely driven by switches from existing therapies [9][26] Business Line Data and Key Metrics Changes - Sapphiens is positioned as a foundational product for PTC, addressing a significant unmet need in the PKU market, which consists of approximately 17,000 patients in the US [7][8] - The company has reported that the average time from patient services form (PSF) to getting on the drug is currently two to four weeks, indicating a seamless execution of the launch [10][11] Market Data and Key Metrics Changes - The launch of Sapphiens has seen uptake across all patient segments, including therapy-naive patients and those switching from Kuvan and Palynziq, indicating a broad market acceptance [24][25] - The company is preparing for a global launch, with expected approvals in Japan and Brazil, and has mapped out strategies for pricing and reimbursement negotiations in various markets [35][40] Company Strategy and Development Direction - PTC is focused on the successful launch of Sapphiens while also advancing its R&D pipeline, particularly in small molecule splicing programs, which hold potential for future therapies [58][62] - The company is committed to managing expenses and achieving cash flow break-even, with plans to reduce operating expenses in 2026 relative to 2025 [68][69] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for Sapphiens and the ability to meet that demand through expanded manufacturing capabilities [46] - The company is actively engaging with regulatory bodies to navigate the complexities of launching in different markets, emphasizing the strength of its data package for Sapphiens [47][48] Other Important Information - PTC has a robust commercial team experienced in rare disease drug launches, which has contributed to the early success of Sapphiens [8][9] - The company is also exploring strategic partnerships for its small molecule RNA platform, which could lead to innovative therapies in oncology and neurodegenerative diseases [62][63] Q&A Session Summary Question: What are the adoption patterns for Sapphiens across different patient segments? - Management noted that early uptake is seen across all segments, including switches from Kuvan and therapy-naive patients, indicating broad market penetration [24][25] Question: How is the company preparing for global launch? - The company has planned for a global launch, with infrastructure in place for expected approvals in Japan and Brazil, and is considering pricing strategies in various markets [35][40] Question: What is the company's strategy for managing expenses? - Management emphasized a commitment to managing expenses while focusing on the success of Sapphiens to drive top-line growth, with plans to reduce operating expenses in 2026 [68][69]

Financial Data and Key Metrics Changes - The company reported a strong cash position of $689.9 million with no funded debt, indicating financial stability and capacity for acquisitions [63] - The company anticipates a mid-term growth rate of 15%-20% for Firdapse as it continues to optimize patient adherence and retention strategies [58][59] Business Line Data and Key Metrics Changes - Firdapse has been on the market since 2019 and is focused on Lambert-Eaton myasthenic syndrome (LEMS), with a significant opportunity for growth in both idiopathic and cancer-associated segments [2][6] - Agamree, targeting Duchenne muscular dystrophy, is expected to penetrate the market further, with a total addressable market estimated at $1.4 billion [17][51] - Fycompa, while experiencing revenue decline due to generic competition, has overperformed expectations despite losing patent protection [3][5] Market Data and Key Metrics Changes - The company has achieved 95% penetration in the top 100 Centers of Excellence (COEs) for DMD, indicating strong market adoption [13] - The cancer-associated LEMS market remains largely untapped, with 90% of patients currently undiagnosed, presenting a significant growth opportunity [28] Company Strategy and Development Direction - The company is focused on a buy-and-build strategy, seeking to acquire de-risked products that are either on the market or near market [4] - The management emphasizes the importance of education and changing care pathways to increase diagnosis rates for LEMS, particularly in oncology settings [24][30] - The company is considering moving further back into the pipeline for future development opportunities as it seeks to accelerate growth [65] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in overcoming the decline of Fycompa through strong patient adherence programs and optimizing existing products [55] - The company is optimistic about the potential of Agamree, especially if it can demonstrate superiority over standard steroids in various health outcomes [17][19] - The management acknowledged the challenging market environment but remains focused on executing its growth strategy [65] Other Important Information - The company has settled three out of four patent challenges, providing a clearer path for market stability through February 2035 [52][53] - The management highlighted the importance of leveraging its strong infrastructure to support rare disease treatments and patient retention [62] Q&A Session Summary Question: What is the peak market opportunity for Agamree? - The company estimates a $1.4 billion opportunity for Agamree, with potential for a 25% market share if it can demonstrate superiority over competitors [17][51] Question: How is the education and diagnosis process for cancer-associated LEMS progressing? - The company is actively educating oncologists and has seen a significant increase in VGCC antibody tests, indicating progress in diagnosis [34][28] Question: What is the strategy for Firdapse moving forward? - The company plans to continue optimizing patient adherence and retention, aiming for a mid-term growth rate of 15%-20% [58][59] Question: What is the company's approach to business development? - The ideal business development opportunity focuses on rare diseases with peak sales potential around $500 million, allowing for strategic acquisitions [60][62] Question: How does the company view its financial health and acquisition capacity? - The company is comfortable with its cash position and can consider acquisitions exceeding $1 billion, maintaining a strong balance sheet [63]