Financial Data and Key Metrics Changes - The company is expecting pivotal study readouts for its phase three program in frontotemporal dementia by the end of 2025, which could lead to regulatory submission if results are positive [4][28] - The phase three study is designed to detect a 40% slowing of disease progression compared to placebo, with a minimum detectable effect of 25% [27] Business Line Data and Key Metrics Changes - The company has two late-stage clinical programs: a phase three program for frontotemporal dementia and a phase two program for Alzheimer's disease, with recruitment completed for the latter [4][5] - The phase two study for Alzheimer's disease is a 76-week trial, with results expected by the end of 2026 [31] Market Data and Key Metrics Changes - The company is targeting multiple neurodegenerative diseases, including Alzheimer's and Parkinson's, with a focus on progranulin as a universal risk gene for these conditions [35][41] - The collaboration with GSK includes a 50/50 profit share, with significant milestone payments tied to commercial sales in the US and EU [50][51] Company Strategy and Development Direction - The company aims to develop a franchise of drugs that elevate progranulin levels for various neurodegenerative diseases, leveraging its integrated research organization [3][36] - The company is also developing brain carrier technology to enhance drug delivery to the brain, targeting antibody drugs, enzyme drugs, and nucleic acid drugs [54][56] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming phase three results, highlighting the statistical power of the study and the potential for significant clinical impact [28][29] - The management noted that there is still room for improvement in Alzheimer's treatments, indicating a belief in the complementary role of their drugs alongside existing therapies [48][49] Other Important Information - The company has a runway through the second half of 2027, with over $350 million in resources, ensuring that clinical programs are fully funded [76][77] - The company is exploring the potential for partnerships but currently aims to fully own its preclinical programs [78] Q&A Session Summary Question: What distinguishes ladocinamab from other progranulin-directed agents? - The mechanism of action of ladocinamab is unique, as it effectively increases extracellular levels of progranulin without the issues faced by previous transcriptional activators [20][21] Question: What are the key endpoints to watch in the phase three study? - The primary endpoint is a 40% slowing of disease progression compared to placebo, with a minimum detectable effect of 25% [27] Question: What is the collaboration structure with GSK? - The collaboration is a 50/50 profit share, with significant milestone payments based on commercial sales in the US and EU [50][51] Question: How does the company view the Alzheimer's disease landscape? - The company believes there is significant room for improvement in Alzheimer's treatments and sees their drug as a complementary option to existing therapies [48][49] Question: What are the next steps for the GKS enzyme program? - The GKS enzyme program is expected to enter the clinic next year, with the potential to validate the technology for multiple indications [72]

Core Insights - MBX Biosciences, Inc. is focused on developing novel precision peptide therapies for endocrine and metabolic disorders, with a significant emphasis on post-bariatric hypoglycemia (PBH) treatment [1][6] - The company will present findings on MBX 1416 at the American Diabetes Association's 85th Scientific Sessions, highlighting its potential as a once-weekly treatment for PBH [2][1] - MBX 1416 has shown positive results in Phase 1 clinical trials, with a Phase 2 trial expected to start in the latter half of 2025 [2] Company Overview - MBX Biosciences is based in Carmel, Indiana, and utilizes its proprietary PEP™ platform to develop therapies targeting endocrine and metabolic disorders [6] - The company's pipeline includes MBX 2109 for chronic hypoparathyroidism in Phase 2, MBX 1416 for PBH in Phase 1, and an obesity portfolio with an IND filing anticipated in Q2 2025 [6] Product Details - MBX 1416 is an investigational long-acting GLP-1 receptor antagonist designed to prevent severe hypoglycemia in individuals with PBH [4] - PBH is a serious complication following bariatric surgery, characterized by recurrent hypoglycemic episodes, and currently lacks approved pharmacotherapies [5] Upcoming Presentations - Presentations on MBX 1416 will take place on June 22, 2025, during the General Poster Session at the ADA conference, featuring studies on its safety, pharmacokinetics, and effects on blood glucose levels [7]

* Tonix's product development candidates are investigational new drugs or biologics; their efficacy and safety have not been established and have not been approved for any indication. Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners. This press release and further information about Tonix can be found at www.tonixpharma.com. CHATHAM, N.J., June 10, 2025 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (To ...

Axsome Therapeutics (AXSM) FY Conference Summary Company Overview - **Company**: Axsome Therapeutics - **Focus**: Development of innovative medicines for central nervous system (CNS) disorders, addressing significant unmet needs in psychiatry and neurology [2][3] Industry Context - **CNS Disorders**: Over 150 million people in the U.S. are affected by serious CNS conditions, which have been historically underserved by the biopharma industry [2][3] - **Key Conditions**: Major depressive disorder (MDD), Alzheimer's disease agitation, smoking cessation, ADHD, binge eating disorder, obstructive sleep apnea, migraine, narcolepsy, fibromyalgia, and shift work disorder [3][4] Core Strategies - **Five Pillars of Innovation**: 1. **Novel Mechanisms of Action**: Focus on distinct treatment outcomes in areas with multiple existing products [4] 2. **Multi-Mechanistic Modes of Treatment**: Examples include Ovelity and Cymbravo [5] 3. **Clinical Trial Innovation**: Designing trials to effectively detect signals from active molecules [5] 4. **Molecular Drug Delivery**: Innovative delivery methods for CNS-active molecules [6] 5. **Diversified Pipeline**: A singular neuroscience pipeline with three commercial products and multiple late-stage programs [6][7] Financial Highlights - **Potential Peak Sales**: Total potential peak sales across the pipeline estimated at $16.5 billion, with approved products like Avelity, Synoscience, and Bravo projected to reach $2 billion to $4.5 billion [8] - **Cash Position**: $300 million in cash resources as of the end of Q1, sufficient to reach cash flow positivity [39][40] Product Updates - **Ovelity**: Rapid growth with an annualized run rate of $400 million; over 50% of prescriptions are first-line treatments [11][14] - **Sunosi**: Steady growth with an annualized run rate of $100 million, focusing on potential label expansion [17] - **Simbravo**: Newly approved for acute migraine treatment, launching imminently [12][18] Clinical Trials and Regulatory Updates - **Cymbravo**: Approved earlier in the year, with positive Phase III clinical trial readouts [9] - **AXS-five**: NDA submission for Alzheimer's disease agitation on track for Q3; high unmet need with 7 million adults affected [21][22] - **Salriamfetol**: Exploring additional indications including ADHD and major depressive disorder, with ongoing trials [25][28] - **AXS-twelve**: NDA submission planned for narcolepsy in the second half of the year [33] - **AXS-fourteen**: Focused on fibromyalgia, with plans for a new 12-week fixed-dose trial following FDA feedback [35][36] Intellectual Property - **Patent Portfolio**: Broad coverage for pipeline programs, with settlements reached with multiple first filers [38] Conclusion - **Leadership and Future Outlook**: Strong leadership team and board, with a focus on providing updates throughout the year [40]

Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. COACH Trial Interim Topline Week 26 Data June 9, 2025 Cautionary Note on Forward-Looking Statements 2 Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. For investor communication only. Not for use in product promotion. Not for further distribution. Executive Summary This present ...

Disclaimer This presentation contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identif ...

SANTA CLARA, Calif., May 28, 2025 (GLOBE NEWSWIRE) -- SI-BONE, Inc. (Nasdaq: SIBN), a medical device company dedicated to solving sacropelvic disorders, today announced that the company will be participating in the upcoming 2025 Goldman Sachs 46th Global Healthcare Conference in Miami Beach, FL. Management will be hosting a fireside chat on Tuesday, June 10, 2025, at 8:20 a.m. Pacific Time/11:20 a.m. Eastern Time. Investors interested in listening to the conference call may do so by registering at this link ...

Core Insights - Cognition Therapeutics, Inc. has released its tenth episode of the Conversations video podcast, focusing on the lived experiences of caregivers for patients with Lewy body dementia [1][2] - The podcast features insights from caregivers and physician experts involved in Cognition's Phase 2 'SHIMMER' study, discussing the challenges and symptoms associated with dementia with Lewy bodies (DLB) [2] Company Overview - Cognition Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecule therapeutics for age-related neurodegenerative disorders [8] - The company is currently investigating its lead candidate, zervimesine (CT1812), in clinical programs for DLB and Alzheimer's disease [8][11] SHIMMER Study Details - The SHIMMER study is a Phase 2 clinical trial that enrolled 130 adults with mild-to-moderate DLB, randomized to receive either zervimesine or placebo for six months [6] - The study utilized various assessment tools, including the Neuropsychiatric Inventory and the Montreal Cognitive Assessment, to measure cognitive performance and symptoms [6] - The SHIMMER study received approximately $30 million in funding from the National Institute on Aging and was presented at the International Lewy Body Dementia Conference in January 2025 [7]

Core Insights - Axsome Therapeutics, Inc. is presenting three innovative neuroscience programs at the ASCP 2025 Annual Meeting, focusing on treatments for central nervous system disorders [1][22] Group 1: AUVELITY - AUVELITY is an oral NMDA receptor antagonist approved for treating major depressive disorder (MDD) in adults, containing dextromethorphan (45 mg) and bupropion (105 mg) [3][4] - The mechanism of action for AUVELITY in treating depression is not fully understood, but it has received Breakthrough Therapy designation from the FDA for MDD [3][22] - AUVELITY is not approved for use in children and is specifically indicated for MDD [4][5] Group 2: AXS-05 - AXS-05 is being studied for its efficacy and safety in treating agitation associated with Alzheimer's disease through a Phase 3 randomized-withdrawal double-blind placebo-controlled study [2][22] - The lead author for this study is Dr. Jeffrey Cummings, Vice Chair of Research at UNLV [2] Group 3: Solriamfetol - Solriamfetol is being evaluated for excessive daytime sleepiness in patients with narcolepsy and obstructive sleep apnea, particularly those reporting anxiety and depression [2][22] - The lead author for this study is Dr. Ulf Kallweit, Assistant Professor of Neurology at Witten/Herdecke University, Germany [2]

Core Insights - Neuphoria Therapeutics Inc. is making steady progress in its clinical pipeline, particularly with its lead program BNC210 for Social Anxiety Disorder and partnered asset MK-1167 for Alzheimer's disease [2][6] Clinical Programs - BNC210 is currently enrolling in the AFFIRM-1 Phase 3 study for Social Anxiety Disorder, with topline data expected in Q3 2025 [2][7] - MK-1167 is in a Phase 2 trial led by Merck for Alzheimer's disease, showing continued momentum [2][4] - A Phase 2b trial (SYMPHONY) for BNC210 in PTSD is being planned following a successful End-of-Phase 2 meeting with the FDA [7] Product Details - BNC210 is a first-of-its-kind oral therapy designed to treat social anxiety disorder and PTSD without sedation, cognitive impairment, or addiction potential [4][7] - The drug acts as a selective negative allosteric modulator of the α7 nicotinic acetylcholine receptor, aiming to restore neurotransmitter balance [4] Financial Outlook - The company has a cash runway extending into Q3 2026, supporting ongoing clinical trials and development efforts [6]