Core Insights - Soleno Therapeutics, Inc. announced preliminary unaudited financial and operational results for the three months ended June 30, 2025, indicating ongoing financial review processes that may alter the reported figures [1][5] Financial Performance - Estimated net revenue from sales of VYKATTM XR for the three months ended June 30, 2025, is projected to be between $31.0 million and $33.0 million, based on specific assumptions regarding Medicare/Medicaid rebates and other factors [5] - As of June 30, 2025, Soleno reported approximately $293.8 million in cash, cash equivalents, and marketable securities [5] - The company had approximately $50.0 million of debt outstanding under its loan and security agreement with Oxford Financing LLC and its affiliates as of June 30, 2025 [5] Product Development - Soleno's first commercial product, VYKATTM XR, is a once-daily oral treatment for hyperphagia in individuals with Prader-Willi syndrome, approved on March 26, 2025 [2] - The company received approximately 646 patient start forms from about 295 unique prescribers between the product's approval date and June 30, 2025 [5]

Core Insights - Telix Pharmaceuticals has received a permanent HCPCS code for its next-generation PSMA PET imaging agent, Gozellix, which will enhance billing and reimbursement processes in the U.S. healthcare system [1][2][4] Group 1: Product Development and Approval - Gozellix is indicated for PET scanning of PSMA positive lesions in men with prostate cancer, particularly those with suspected metastasis or biochemical recurrence [3] - The product features an extended shelf-life and flexible production options, addressing logistical barriers to PSMA-PET imaging access [3][4] - Gozellix has been approved by the U.S. FDA, following the success of Telix's first-generation PSMA-PET imaging agent, Illuccix [5] Group 2: Market Impact and Future Prospects - The HCPCS code assignment is a significant milestone that supports clinical adoption and reimbursement for Gozellix, facilitating its commercial scale-up in the U.S. [2][4] - The CEO of Telix emphasized that this development is crucial for improving access to precision medicine imaging for prostate cancer patients across the U.S. [4] Group 3: Company Overview - Telix Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapeutic and diagnostic radiopharmaceuticals [4] - The company operates internationally, with a presence in the U.S., Brazil, Canada, Europe, and Japan, aiming to address unmet medical needs in oncology and rare diseases [4]

Company Overview - Rhythm Pharmaceuticals, Inc. is a global, commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [1] Proposed Offering - Rhythm announced a proposed public offering of $150 million of its common stock, with an additional option for underwriters to purchase up to $22.5 million of shares [1][2] - The offering is subject to market conditions and customary closing conditions, with no assurance on completion or terms [2] Regulatory Compliance - The offering is made under a shelf registration statement on Form S-3, which became effective on March 2, 2023 [3] - A preliminary prospectus supplement will be filed with the SEC and available on their website [3] Contact Information - Investor relations contact is David Connolly, Head of Investor Relations and Corporate Communications, reachable at 857-264-4280 [6]

Jason Campagna, MD, PhD, joins Inventiva as President of R&D and Chief Medical Officer, succeeding Pierre Broqua, PhD, and Michael Cooreman, MDMartine Zimmermann, PharmD, joins as Executive Vice President of Regulatory Affairs and Quality AssuranceThese key leadership appointments underscore Inventiva’s commitment to long-term growth and operational excellence, with topline results from NATiV3 on track for the second half of 2026 Daix (France), New York City (New York, United States), July 9, 2025 – Invent ...

Core Insights - The Phase 3 Orbit study for UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is on track for final analysis by the end of the year [1][3] - The Data Monitoring Committee has confirmed an acceptable safety profile for UX143, allowing the study to proceed [2] - Ultragenyx and Mereo BioPharma are collaborating on the development of setrusumab, targeting OI sub-types I, III, and IV [5][12] Study Details - The Phase 3 Orbit study has enrolled 159 patients across 45 sites in 11 countries, with a primary efficacy endpoint focused on annualized clinical fracture rate [7] - The Cosmic study, which is also in Phase 3, has enrolled 69 patients aged 2 to <7 years, comparing setrusumab to intravenous bisphosphonates [8] - Both studies will conduct final analyses after patients have been on therapy for at least 18 months, with specific statistical thresholds set for each study [4] Background on Osteogenesis Imperfecta - OI is a genetic disorder affecting bone metabolism, primarily caused by mutations in the COL1A1 or COL1A2 genes, leading to increased bone brittleness and a high rate of fractures [9] - Approximately 60,000 individuals are affected by OI in commercially accessible regions, with no globally approved treatments available [9] Mechanism of Setrusumab - Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, which negatively regulates bone formation, potentially increasing bone mass and strength [10] - Previous studies have shown that anti-sclerostin antibodies can significantly improve bone formation and density in OI models [11] Company Profiles - Ultragenyx is focused on developing therapies for rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [13] - Mereo BioPharma is also dedicated to innovative therapeutics for rare diseases, with setrusumab as one of its key candidates [15]

Presented updated data for bexobrutideg (NX-5948) at EHA2025 and ICML-18, demonstrating a favorable safety profile and deepening responses in patients with r/r chronic lymphocytic leukemia (CLL) and Waldenström macroglobulinemia (WM) Secured $15M license fee as Sanofi extends STAT6 collaboration to target type 2 inflammatory diseases Announced FDA clearance of IND application for novel IRAK4 degrader GS-6791/NX-0479, enabling collaboration partner Gilead to initiate Phase 1 trial Well capitalized with cash ...

Conference Call and Webcast Scheduled for Wednesday, July 30SAN DIEGO, July 9, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) announced today that it has scheduled its second quarter 2025 financial results conference call and webcast for 1:30 p.m. Pacific Time (4:30 p.m. Eastern Time) on July 30, 2025.The schedule for the press release and conference call / webcast is as follows: Q2 2025 Press Release: July 30, 2025 at 1:00 p.m. PT / 4:00 p.m. ET Q2 2025 Conference Call: July 30, 2025 at 1 ...

Core Insights - The RESILIENT trial data demonstrates that TNX-102 SL significantly reduces fibromyalgia pain compared to placebo, confirming previous findings from the RELIEF trial [1][2] - If approved, TNX-102 SL will be the first new drug for fibromyalgia in over 15 years, with a PDUFA target date set for August 15, 2025 [1][2] Company Overview - Tonix Pharmaceuticals Holding Corp. is a clinical-stage biopharmaceutical company focused on developing therapies for pain management and central nervous system disorders [8] - The company has submitted a New Drug Application (NDA) for TNX-102 SL based on two statistically significant Phase 3 studies [2][8] Study Details - The RESILIENT study was a randomized, double-blind, placebo-controlled trial involving 457 adults with fibromyalgia across 33 sites in the U.S. [2][7] - Participants received TNX-102 SL in two dosage phases: 2.8 mg for two weeks followed by 5.6 mg for twelve weeks, with a primary endpoint of pain reduction assessed over fourteen weeks [2][7] Efficacy Results - TNX-102 SL achieved a least-squares mean reduction of 1.8 points on the eleven-point daily pain numeric rating scale, compared to a 1.2-point reduction for placebo, indicating high statistical significance [2][7] - Statistically significant improvements were also noted across all six prespecified key secondary endpoints, including various patient-reported outcomes [2][3] Safety Profile - TNX-102 SL was generally well tolerated, with the most common adverse events being mild and transient, such as oral tingling/numbness and a bitter aftertaste [3] - No serious drug-related adverse events or deaths were reported, highlighting a favorable risk-benefit profile [3] Fibromyalgia Context - Fibromyalgia affects an estimated 6-12 million adults in the U.S., predominantly women, and is characterized by chronic pain, fatigue, and sleep disturbances [4] - Current treatment options are often met with dissatisfaction from both physicians and patients, indicating a significant unmet need in the market [4] Product Information - TNX-102 SL is a patented sublingual formulation of cyclobenzaprine hydrochloride designed for rapid absorption and reduced side effects [5][6] - The drug is also being explored for other indications, including acute stress reaction, Long COVID, and alcohol use disorder [5][8]

Key Takeaways PROK surged 515% on positive Phase II data for rilparencel in diabetic chronic kidney disease patients. Group 1 showed a 78% improvement in kidney decline rate, meeting statistical and clinical benchmarks. ProKidney will seek FDA confirmation of eGFR slope as a surrogate endpoint in its ongoing Phase III trial.Shares of ProKidney Corp. (PROK) skyrocketed 515% yesterday after the company reported encouraging top-line data from the phase II REGEN-007 study evaluating its lead pipeline drug, ri ...

Core Viewpoint - Connect Biopharma's collaborator, Simcere Pharmaceutical, has submitted a New Drug Application for rademikibart to treat atopic dermatitis in China, indicating significant progress in the development of this potential best-in-class therapy for inflammatory diseases [1][2]. Company Overview - Connect Biopharma is a clinical-stage biopharmaceutical company focused on developing innovative therapies for inflammatory diseases, with rademikibart as its lead product candidate targeting IL-4Rα [6]. - Simcere Pharmaceutical is an R&D-driven company focusing on therapeutic areas such as neuroscience, anti-oncology, autoimmune, and anti-infection [5]. Product Development - Rademikibart is a fully human monoclonal antibody that targets IL-4Rα, blocking the Th2 inflammatory pathway, which is relevant for treating atopic dermatitis and asthma [4]. - The AD market in China presents a considerable opportunity, with an estimated 70 million patients suffering from the condition [3]. Collaboration Agreement - In 2023, Connect Biopharma and Simcere entered an exclusive license and collaboration agreement for rademikibart in Greater China, granting Simcere exclusive rights for development, manufacturing, and commercialization [2][3]. - Connect Biopharma is eligible for milestone payments totaling approximately $110 million and tiered royalties on net sales in Greater China [3].