Core Viewpoint - Achieve Life Sciences, Inc. has successfully closed a public offering of 15 million shares of common stock, raising gross proceeds of $45 million to advance the development of cytisinicline for nicotine dependence treatment [1][2]. Company Overview - Achieve Life Sciences is a late-stage specialty pharmaceutical company focused on developing and commercializing cytisinicline as a treatment for nicotine dependence, with a New Drug Application submitted to the FDA in June 2025 [5]. - The company has completed two Phase 3 studies and an open-label safety study for cytisinicline, and has also conducted a Phase 2 study for vaping cessation [5]. Product Information - Cytisinicline is a plant-based alkaloid that interacts with nicotinic acetylcholine receptors in the brain, aimed at reducing nicotine cravings and the satisfaction derived from nicotine products [7]. - Approximately 29 million adults in the U.S. smoke combustible cigarettes, and tobacco use is the leading cause of preventable death globally, with over 8 million deaths annually [6]. - There are currently no FDA-approved treatments specifically for nicotine e-cigarette cessation, highlighting a critical need that cytisinicline aims to address [6]. Financial Details - The public offering included 15 million shares at a price of $3.00 per share, along with warrants to purchase up to 16,766,666 additional shares [1]. - Proceeds from the offering will be used for advancing cytisinicline towards FDA marketing approval and for general corporate purposes [2].

Core Insights - Crinetics Pharmaceuticals is set to present long-term efficacy and safety data for its investigational drug PALSONIFY (paltusotine) in acromegaly, demonstrating both biochemical and symptom control with a well-tolerated safety profile [1][2] - The company will also showcase Phase 2 trial results for atumelnant in congenital adrenal hyperplasia (CAH) and data from early-stage development programs in Graves' hyperthyroidism and orbitopathy at the Endocrine Society's Annual Meeting, ENDO 2025 [1][3] PALSONIFY Presentations - Four abstracts will report results from the PALSONIFY development program, including an oral presentation on long-term efficacy, safety, and symptom control in acromegaly patients switching from injectable somatostatin receptor ligands to once-daily oral PALSONIFY [2][4] - Three poster presentations will cover symptom stability in acromegaly, patient-reported outcomes from PATHFNDR-1 and PATHFNDR-2, and open-label extension data from PATHFNDR-2 [2] Atumelnant Presentations - Three abstracts will focus on atumelnant, including an oral presentation of Phase 2 trial results in CAH, highlighting reductions in adrenal volume and significant decreases in potent 11-oxygenated androgens [3][7] Early-Stage Pipeline - Crinetics will present new data from its early-stage pipeline, including findings related to CRN12755 for Graves' hyperthyroidism and orbitopathy [4][8] Company Overview - Crinetics Pharmaceuticals is a clinical-stage pharmaceutical company focused on developing novel therapeutics for endocrine diseases and related tumors, with PALSONIFY and atumelnant as its lead candidates [11]

Summary of Arcturus Therapeutics KOL Presentation on ARCT-810 Phase II Interim Data for OTC Deficiency Company Overview - **Company**: Arcturus Therapeutics - **Headquarters**: San Diego - **Focus**: mRNA medicines, specifically targeting rare liver diseases like ornithine transcarbamylase (OTC) deficiency [5][6] Industry Context - **Industry**: Biotechnology, specifically in the development of mRNA therapeutics for rare diseases - **Condition**: OTC deficiency is the most common urea cycle disorder with significant unmet medical needs [6][7] Key Points from the Presentation 1. **ARCT-810 Overview**: - ARCT-810 is an mRNA therapeutic designed to replace dysfunctional OTC enzymes, improving urea cycle activity, detoxifying ammonia, and potentially eliminating the need for liver transplants [6][7][8] - It utilizes Arcturus' proprietary lunar delivery platform for effective delivery to hepatocytes [7] 2. **Regulatory Designations**: - ARCT-810 has received multiple designations: orphan drug designation, orphan medicinal product designation, fast track designation, and rare pediatric disease designation [8] 3. **Phase II Study Design**: - Two Phase II studies were conducted: one in the US and one in Europe, focusing on safety, tolerability, and biomarker assessments [11][12] - The US study enrolled patients with more severe disease, while the European study included patients with stable disease [12] 4. **Biomarker Results**: - **Plasma Glutamine**: - In the European study, mean glutamine levels decreased from high to normal during treatment and began to rise again after four weeks post-treatment [13][14] - In the US study, glutamine levels normalized after three doses and remained normal for approximately twenty days [14] - **Ureagenesis Function**: - The new N15 assay showed significant increases in relative ureagenesis function (RUF) post-treatment, with a mean increase of 14.7% [16][17] - Two subjects achieved RUF levels above 50%, indicating clinically meaningful improvements [17] - **Ammonia Levels**: - Ammonia levels remained stable and within normal ranges after treatment, supporting the favorable glutamine and ureagenesis data [18] 5. **Safety Profile**: - The safety database included 40 participants, indicating that ARCT-810 was generally safe and well-tolerated [19] - No serious infusion-related reactions were reported, and adverse events were manageable [19][20] 6. **Next Steps**: - Arcturus plans to complete the ongoing Phase II US study and engage with regulatory agencies for a multi-biomarker driven pivotal trial [68][69] Additional Insights - **Clinical Implications**: - The KOLs emphasized the importance of normalizing diet and reducing the need for ammonia scavengers as key success metrics for OTC therapies [78][80] - mRNA therapies are viewed as a potential alternative to liver transplants, especially for severe cases [100][101] - **Comparison with Other Therapies**: - ARCT-810 is positioned as a more effective solution compared to existing ammonia scavengers, which do not restore urea cycle function [87][88] Conclusion - The interim data for ARCT-810 demonstrates promising results in reducing glutamine levels and improving urea cycle function, with a favorable safety profile. The company is poised to advance its clinical development and regulatory strategy to address the significant unmet needs in OTC deficiency treatment [67][68]

Regulatory Landscape - Colorado is formally reviewing ibogaine for potential legal availability as a treatment for substance use disorders and mental health conditions [1] - This review could position Colorado as the first to legalize the hallucinogen for treatment purposes [1]

INmune Bio Inc. INMB stock is trading lower on Monday, with a session volume of 23.2 million, compared to the average volume of 1.45 million, as per data from Benzinga Pro. The company announced results from its Phase 2 MINDFuL trial evaluating XPro, a selective soluble TNF inhibitor, in early Alzheimer's Disease with biomarkers of inflammation. Also Read: Europe Approves Eisai-Biogen's Alzheimer's Drug 'Under Strict Conditions' After Initial Negative Opinion Despite showing no effects in the modified inten ...

Core Insights - Vor Bio's shares have surged by 265.5% in the past week, significantly outperforming the industry average increase of 0.8% [1] - The company has entered a licensing agreement with RemeGen to develop and commercialize telitacicept in markets outside of China [1][6] Licensing Agreement Details - The licensing deal is valued at $125 million upfront, which includes $45 million in cash and $80 million in stock warrants, along with potential milestone payments exceeding $4 billion and tiered royalties on future sales [6][7] - Vor Bio will focus on developing telitacicept, a dual-target fusion protein that blocks two proteins, BAFF and APRIL, to treat autoimmune diseases [2][7] Clinical Development - A global phase III study for telitacicept in treating generalized myasthenia gravis (gMG) is currently ongoing in the United States, Europe, and South America, with initial data expected in the first half of 2027 [3] Leadership Changes - Vor Bio has appointed Jean-Paul Kress as the new CEO and chairman, following the resignation of the previous CEO, Robert Ang [3][6] Company Outlook - The licensing agreement is seen as a pivotal move that may prevent Vor Bio from shutting down its operations, which was previously announced due to financial difficulties [8]

The SEISMiC C interim analysis will provide a preliminary assessment of istaroxime safety in treating more severe cardiogenic shock patients who may be receiving standard of care with inotropes and vasopressors SCAI Stage C is a sicker patient population than early cardiogenic shock and has high mortality despite currently used drug treatments WARRINGTON, Pa., June 30, 2025 (GLOBE NEWSWIRE) -- Windtree Therapeutics, Inc. ("Windtree" or the "Company") (NasdaqCM: WINT), a diversified company focused on revenu ...

Core Insights - Autonomix Medical, Inc. is expanding its clinical trials to include additional visceral cancer indications, effectively doubling its potential addressable market beyond pancreatic cancer pain [1][2] - The initiation of the PoC 2 phase follows positive results from the PoC 1 trial, which focused on patients with severe pancreatic cancer pain, indicating a strong foundation for multi-indication growth [2][3] - The company's technology platform has the potential to address a wide range of medical conditions, including cardiology, hypertension, and chronic pain management, with a concentrated focus on interventional cancer pain management applications [3][5] Company Overview - Autonomix Medical, Inc. is dedicated to advancing innovative technologies for diagnosing and treating diseases related to the nervous system [4] - The company's first-in-class platform includes a catheter-based microchip sensing array designed to detect and differentiate neural signals with greater sensitivity than existing technologies [4][5] - The technology is currently investigational and has not yet received marketing clearance in the United States [5]

IRVINE, Calif., June 30, 2025 /PRNewswire/ -- Allergan Aesthetics, an AbbVie company (NYSE: ABBV), today announced that the U.S. Food and Drug Administration (FDA) accepted for review the supplemental premarket approval (sPMA) application for SKINVIVE by JUVÉDERM® to reduce neck lines for the improvement of neck appearance. SKINVIVE by JUVÉDERM® is currently approved in the U.S. to improve skin smoothness of the cheeks in adults over the age of 21. "SKINVIVE by JUVÉDERM® is an injectable version of hyaluron ...

Core Viewpoint - Allarity Therapeutics has received formal acceptance of its patent application for the Drug Response Predictor (DRP) companion diagnostic specific to stenoparib from IP Australia, marking a significant step in its global strategy for intellectual property protection and commercialization of its proprietary technology [1][2][3]. Company Overview - Allarity Therapeutics, Inc. is a Phase 2 clinical-stage pharmaceutical company focused on developing stenoparib, a dual PARP and WNT pathway inhibitor, as a personalized cancer treatment using its proprietary DRP technology [1][6]. - The company is headquartered in the U.S. and has a research facility in Denmark, aiming to address significant unmet medical needs in cancer treatment [6]. Patent and Intellectual Property - The accepted patent covers 40 claims and is a crucial part of Allarity's strategy to protect its DRP platform internationally, with the patent expected to be granted within 20 working days if unopposed [2][3]. - Allarity has previously secured a European patent for the stenoparib DRP and holds 18 granted patents for drug-specific DRPs, including eight in the U.S. [3]. Drug and Technology Details - Stenoparib is an orally available small-molecule inhibitor targeting PARP1/2 and tankyrase 1/2, with potential applications in various cancer types, particularly ovarian cancer [4]. - The DRP technology is designed to select patients likely to benefit from specific drugs based on gene expression signatures, enhancing therapeutic benefit rates [5]. Future Prospects - The acceptance of the Australian patent is seen as a vital achievement in securing international IP protection while advancing stenoparib through Phase 2 trials towards U.S. regulatory approval [3].