Pipeline Programs - Setrusumab for Osteogenesis Imperfecta (OI) Phase 3 results were reported around the end of 2025, partnered with Ultragenyx, but neither study achieved the primary endpoint of reduction in AFR compared to placebo or bisphosphonates[8, 11] - Alvelestat for Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD) activities are ongoing to support the initiation of Phase 3, following an agreement in principle on the primary endpoints[8] - Vantictumab for osteopetrosis is out-licensed to āshibio, with EU rights retained, and an IND is planned for H2 2026[8] Financial Status - The company has approximately $41 million in cash and cash equivalents as of December 31, 2025, providing a cash runway into mid-2027[8] Setrusumab Clinical Trial Results - In the Orbit study (Setrusumab vs placebo), 19.5% of patients met rescue criteria at 12 months, primarily due to fractures, with a larger number of placebo patients exiting the study[14] - In the Cosmic study (Setrusumab vs bisphosphonates), there was a 59% reduction in vertebral fractures on setrusumab (p=0.081), despite more severe type III/IV patients on setrusumab (65% setrusumab vs 54% IV-BP)[31] - Setrusumab patients in the Orbit study showed improvements in disease severity (PGIS) in peds/teens, as well as improvements in pain/comfort and sports/activity[20] Market Opportunities - Osteogenesis Imperfecta affects approximately 60,000 patients across the US & Europe[9] - Severe Alpha-1 Antitrypsin Deficiency affects an estimated 50,000 patients in North America and 60,000 in Europe[9] - Osteopetrosis has an incidence of 1 in 20,000 in North America and Europe, with onset typically in late childhood[9]

Core Viewpoint - Ipsen presented 14 abstracts at the TOXINS 2026 conference, showcasing the effectiveness of Dysport® (abobotulinumtoxinA) in treating various neurological conditions, particularly post-stroke spasticity, cervical dystonia, and blepharospasm, while also highlighting new indications under evaluation [1][5]. Group 1: Study Findings - An interim analysis from the EPITOME study revealed that 45.7% of stroke survivors with paresis developed post-stroke spasticity (PSS) within one year, an increase from the previously documented 39.5% [2][3][5]. - The study emphasizes the importance of early identification of PSS through a Post-stroke Spasticity Monitoring Questionnaire, which aids clinicians in detecting early signs of spasticity [2][3]. Group 2: Treatment Gaps - Less than 1% of stroke survivors receive BoNT-A treatment for spasticity in routine practice, indicating a significant care gap in the management of post-stroke spasticity [3][5]. - The existing evidence suggests that remote monitoring can facilitate timely intervention, potentially improving rehabilitation outcomes and quality of life for patients [3]. Group 3: Ipsen's Commitment - Ipsen's Executive Vice President, Chief Medical Officer, emphasized the company's mission to address care gaps for individuals with neurological conditions through comprehensive data presentation and studies like EPITOME [4]. - The company aims to provide standardized follow-up care to ensure stroke survivors receive the necessary treatment [4]. Group 4: Dysport Overview - Dysport® (abobotulinumtoxinA) is an injectable botulinum neurotoxin type A product that has been authorized in approximately 90 countries, with over 30 years of clinical experience and more than 18 million treatment years of patient experience [7].

AlexSecret/iStock via Getty Images Dear Partners, In the fourth quarter, Laughing Water Capital (“LWC”) returned approximately 6.8%, bringing full year returns after all fees and expenses to approximately 3.9%.i In contrast, the SP500TR (SP500) and R2000 (RTY) returned approximately 2.7% and 2.2% in the quarter, and finished the year up 17.9% and 12.8% respectively. As always individual results may vary, so please check your individual statements for the most accurate reading of your performance. As a r ...

— First randomized Phase III trial confirming the efficacy of MET inhibition in patients with advanced NSCLC and acquired MET amplification after progression on prior EGFR-TKI treatment — — Savolitinib and osimertinib combination approved in China in June 2025 — HONG KONG, SHANGHAI and FLORHAM PARK, N.J., Jan. 14, 2026 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM; HKEX:​13) today highlights that results from the SACHI Phase III trial were published in The Lancet. SACHI is a Pha ...

Core Viewpoint - Harmony Biosciences is entering 2026 with significant momentum driven by three key factors: strong performance of WAKIX, a robust late-stage pipeline, and a strong financial profile [3][4]. Group 1: WAKIX Performance - WAKIX has shown five years of consecutive growth and profitability, with guidance for 2026 indicating over $1 billion in net revenue, positioning it to achieve blockbuster status this year [3]. Group 2: Late-Stage Pipeline - The company has five Phase III programs in the clinic targeting five distinct CNS indications, which are expected to provide multiple catalysts in the coming years [4]. Group 3: Financial Profile - Harmony Biosciences is characterized by a strong financial profile, indicating profitability and a solid foundation for future growth [4].

PresentationHello, everybody. I'm Ekaterina Knyazkova from JPMorgan and pleased to be introducing ANI Pharmaceuticals. And from the company, we have Nikhil Lalwani, CEO; and Steve Carey, CFO, who will be doing a presentation, and then we will jump into Q&A. And with that, I will turn it over to Nikhil.Nikhil LalwaniPresident, CEO & Director Good afternoon, and thank you for joining us here for a session on ANI Pharmaceuticals. These are our standard disclaimers regarding forward-looking statements, kind of ...

Core Insights - Liquidia Corporation is focused on developing therapies for pulmonary arterial hypertension and has received a bullish price target of $55 from Jefferies, indicating a potential increase of about 48.57% from its trading price of $37.02 [1][6] Stock Performance - Recently, Liquidia's stock price increased by 9.1%, reaching a high of $34.57 and closing at $34.65, with a trading volume of approximately 1.89 million shares, a 14% increase from the average session volume [2] - The stock is currently priced at $37.74, reflecting an increase of 3.11% or $1.14, with fluctuations between $35.28 and $38.19 during the trading day [4] - Over the past year, the stock has reached a high of $39.16 and a low of $11.26, with a market capitalization of approximately $3.22 billion [5] Financial Performance - Liquidia reported preliminary net sales for Q4 of YUTREPIA at approximately $90.1 million, with full-year sales projected to be around $148.3 million for 2025 [3] - The company achieved a positive cash flow exceeding $30 million in Q4 and has approximately $190.7 million in cash on hand [3][6] Commercial Metrics - Since the launch, Liquidia has recorded over 2,800 unique prescriptions, with more than 2,200 patients starting treatment, resulting in a prescription-to-start conversion rate of approximately 85% [4] - The expanding prescriber base supports expectations for durable revenue growth [4]

SAN DIEGO, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Robbins Geller Rudman & Dowd LLP is investigating potential violations of U.S. federal securities laws involving Travere Therapeutics, Inc. (NASDAQ: TVTX). If you have information that could assist in the Travere investigation or if you are a Travere investor who suffered a loss and would like to learn more, you can provide your information here: https://www.rgrdlaw.com/cases-travere-therapeutics-inc-investigation-tvtx.html You can also contact attorney J.C. Sanc ...

BOSTON, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced the appointment of world-renowned neurologist and epilepsy authority Dr. Orrin Devinsky, M.D., as Head of Clinical Strategy, a newly created leadership role. Dr. Devinsky is widely regarded as one of the most influential physician-scientists of his generation in modern epilepsy and clinical neuros ...

NEW YORK, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Galectin Therapeutics Inc. (“Galectin” or the “Company”) (NASDAQ: GALT).  Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Galectin and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] Dec ...