MELBOURNE, Australia and SAN FRANCISCO, June 23, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that David Stamler, M.D., Chief Executive Officer of Alterity, will participate in a Fireside Chat hosted by MST Access on Wednesday, 25 June 2025 in Australia / Tuesday, 24 June 2025 in the United States. Dr. Stamler will provide a co ...

Company Overview - Thiogenesis Therapeutics, Corp. is a clinical-stage biopharmaceutical company focused on developing sulfur-based therapeutics for rare pediatric and inherited mitochondrial disorders [2][10] - The company is publicly traded on the TSX Venture Exchange and OTCQX [10] Clinical Programs - The company is advancing two lead Phase 2 clinical programs for its novel thiol drug, TTI-0102 [3] - A European multicenter trial for Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS), initiated on May 14, 2025 [3] - A U.S.-based trial for the Leigh Syndrome Spectrum (LSS), following FDA clearance of the Investigational New Drug (IND) application on June 11, 2025 [3] Drug Candidate - TTI-0102 is a next-generation thiol-based prodrug designed to enhance intracellular levels of glutathione and taurine, which are critical for mitigating oxidative stress in mitochondrial disorders [3][8] - The drug is engineered to address challenges faced by first-generation thiol-based drugs, including short half-life and adverse side effects [8] Mitochondrial Disorders - MELAS is characterized by symptoms such as seizures, muscle weakness, and can lead to loss of motor skills and intellectual disability, affecting approximately 4.1 in 100,000 of the population [7] - LSS is diagnosed in about 1 in 40,000 births and presents symptoms like impaired feeding, loss of motor skills, and seizures, with no current cure available [6] Conference Participation - The company’s CEO, Dr. Patrice Rioux, will participate in a clinical panel discussion at the United Mitochondrial Disease Foundation's 2025 Mitochondrial Medicine Conference, which gathers over 700 scientists and industry stakeholders [2][3]

Core Insights - Orchestra BioMed Holdings, Inc. presented key clinical insights on AVIM therapy for high-risk hypertension at the CSI 2025 Meeting, emphasizing its potential to address hypertensive heart disease, a significant cardiovascular syndrome affecting an aging population [1][6] Company Overview - Orchestra BioMed is a biomedical innovation company focused on accelerating high-impact technologies through risk-reward sharing partnerships with leading medical device companies [5] - The company’s lead product candidate is AVIM therapy, designed for hypertension, which is a leading risk factor for mortality worldwide [5][8] AVIM Therapy Insights - AVIM therapy is a novel, device-based approach specifically targeting patients with hypertensive heart disease, who are at increased risk for major adverse cardiac events and currently lack sufficient therapeutic options [2][3] - The therapy aims to reduce cardiac preload and modulate autonomic nervous system responses to lower blood pressure and improve cardiovascular function, representing a potential paradigm shift in blood pressure management [3][4] Clinical Data and Studies - Pilot studies have shown that AVIM therapy resulted in net reductions of 8.1 mmHg in 24-hour ambulatory systolic blood pressure and 12.3 mmHg in office systolic blood pressure at six months compared to control patients [10] - The BACKBEAT global pivotal study is currently enrolling patients with uncontrolled hypertension who are indicated for a dual-chamber pacemaker, in collaboration with Medtronic [7][10] Market Opportunity - Hypertensive heart disease affects over 7.7 million patients in the U.S., highlighting a significant market opportunity for AVIM therapy, especially given its recent FDA Breakthrough Device Designation [6][10]

Core Insights - Amgen is presenting full results from the Phase 2 study of MariTide, a peptide-antibody conjugate for obesity and Type 2 diabetes, at the 85th American Diabetes Association Scientific Sessions [1][2] - The company is also sharing new data from the Phase 3 FOURIER study of Repatha® in cardiovascular disease [1] - MariTide shows promising 52-week efficacy, safety, and tolerability data, indicating robust weight loss without a plateau in patients with obesity [3][9] Company Overview - Amgen is focused on innovative medicines for serious diseases, with a strong pipeline that includes treatments for obesity and cardiovascular conditions [20][22] - The company has been recognized as one of the "World's Most Innovative Companies" and is part of the Dow Jones Industrial Average and Nasdaq-100 Index [23] Product Information - MariTide is a bispecific GLP-1 receptor agonist and GIPR antagonist, designed to provide a dual mechanism of action for weight loss and diabetes management [9] - Repatha® is a monoclonal antibody that lowers LDL cholesterol by inhibiting PCSK9, with extensive clinical trial data supporting its efficacy [13][14] Industry Context - The prevalence of obesity has more than doubled globally from 1990 to 2022, with significant health implications including increased risk for Type 2 diabetes and cardiovascular diseases [7][8] - Despite the recognition of obesity as a chronic disease, only 1%-3% of eligible adults in the U.S. receive pharmacologic treatment for weight management [8]

Core Viewpoint - Applied Therapeutics, Inc. has announced key executive appointments, promoting Evan Bailey to Chief Medical Officer and Dottie Caplan to Executive Vice President of Patient Advocacy and Government Affairs, effective June 15, 2025 [1][5]. Group 1: Executive Appointments - Evan Bailey, MD, has been promoted to Chief Medical Officer, succeeding Riccardo Perfetti, MD, PhD, who served since 2018 [5]. - Dottie Caplan has been promoted to Executive Vice President, Patient Advocacy and Government Affairs, expanding her role to include government affairs [5][6]. Group 2: Leadership Contributions - Dr. Bailey has been with Applied Therapeutics for four years and has played a critical role in advancing the company's development programs, particularly in clinical development execution [2][3]. - Ms. Caplan has been instrumental in integrating patient voices into clinical development strategies and will continue to lead advocacy efforts in her new role [2][4]. Group 3: Focus on Rare Diseases - The company is dedicated to developing treatments for rare diseases, with its lead drug candidate, govorestat, targeting conditions such as Classic Galactosemia and CMT-SORD [7]. - Dr. Bailey expressed commitment to advancing the portfolio of supporting data for govorestat to address high unmet medical needs in rare disease patients [2][3].

Core Viewpoint - Processa Pharmaceuticals has entered into a binding term sheet with Intact Therapeutics for the exclusive option to license PCS12852, a 5-HT4 receptor agonist aimed at treating gastroparesis and other gastrointestinal motility disorders [1][3]. Financial Terms - Processa is set to receive a $2.5 million option exercise fee, up to $20 million in development and regulatory milestone payments, and over $432.5 million in commercial milestone payments based on net product sales [2][8]. - Intact will pay Processa a double-digit royalty on worldwide net sales of licensed products, excluding South Korea, and provide an equity stake of 3.5% in Intact upon closing [2][8]. Clinical Significance - PCS12852 has shown a favorable safety and efficacy profile in clinical studies, particularly in a Phase 2a trial for diabetic gastroparesis, a condition with limited treatment options [3][4]. - The drug is designed to restore normal gastric emptying without the cardiovascular and central nervous system side effects associated with older agents in this class [3]. Strategic Partnership - The partnership is expected to unlock the value of Processa's non-oncology assets while focusing on developing next-generation cancer therapies [3]. - Intact Therapeutics aims to address significant unmet clinical needs in gastrointestinal diseases, enhancing patient quality of life through innovative therapies [3][5]. Company Background - Intact Therapeutics is a clinical-stage biopharmaceutical company focused on next-generation therapies for gastrointestinal diseases, leveraging proprietary thermal hydrogel technology [5]. - Processa Pharmaceuticals specializes in developing next-generation cancer drugs with improved safety and efficacy, modifying existing FDA-approved oncology therapies [7].

Vivoryon Therapeutics N.V. Q1 2025 Financial Results and Operational Progress Continued progress in advancing varoglutamstat in kidney disease based on encouraging data and expansion of IP portfolioVaroglutamstat’s pre-clinical dataset showing synergistic effect in combination with an SGLT-2 inhibitor in different treatment regimensNovel composition of matter patent for varoglutamstat granted after accelerated review in the U.S.; patent term to provide exclusivity through 2044 with subsequent opportunity fo ...

Core Insights - Roche (RHHBY) is advancing its pipeline candidate prasinezumab into phase III development for early-stage Parkinson's disease based on data from phase IIb PADOVA study and ongoing open-label extensions [1][7] - The candidate is a potential first-in-class anti-alpha-synuclein antibody targeting a known biological driver of Parkinson's disease progression [3] - Year-to-date, Roche's shares have increased by 20.9%, outperforming the industry growth of 4% [1] Development Details - The PADOVA study evaluated prasinezumab's safety and efficacy in 586 randomized patients, although it missed its primary endpoint of time to confirmed motor progression [4][5] - Despite missing statistical significance, positive trends toward reduced motor progression were observed at 104 weeks, with expectations of sustained effects based on additional open-label extension data [5][6] - The ongoing PASADENA and PADOVA open-label studies are assessing the long-term safety and efficacy of prasinezumab in over 750 individuals with early-stage Parkinson's disease [3] Licensing and Financials - Roche holds exclusive rights to prasinezumab under a licensing agreement with Prothena (PRTA) established in December 2013, which includes paying double-digit teen royalties on net sales [7][9] - Prothena has earned $135 million to date, with potential for up to $620 million in additional milestone payments [9] Industry Context - Developing treatments for Parkinson's disease is challenging due to its chronic and progressive nature, with both motor and non-motor symptoms [10] - Other companies, such as UCB and Novartis, are also working on treatments, with UCB's recent investigational drug failing to meet clinical endpoints [11]

Crispr Technology & Applications - Crispr 基因编辑技术能够对患者的 DNA 进行精确编辑，从而识别新的治疗靶点并纠正某些疾病，扩大可治疗疾病的范围 [2] - Crispr 技术与 AI 结合，加速识别靶点和疗法，从而加快新疗法上市的速度 [3] - 癌症，尤其是无法以其他方式治疗的儿童疾病和罕见癌症，可能是 Crispr 技术首先产生影响的领域 [5] Regulatory Landscape - FDA 对新的治疗方法和技术平台持支持态度，并倾向于快速批准 [5] - 美国卫生与公众服务部 (HHS) 部长承诺支持细胞和基因治疗领域，这对行业来说是一个巨大的推动力 [7] - 众议院拨款委员会也表达了对细胞和基因治疗领域的支持，释放了积极信号 [7] Manufacturing & Supply Chain - 该公司主要在美国本土进行生产，对进口尤其是来自中国的依赖程度不高 [9] - 公司在马萨诸塞州进行生产，并在匹兹堡进行扩张 [9] - 匹兹堡的团队致力于支持当地学校的 STEM (科学、技术、工程和数学) 项目 [9]

Vera Therapeutics (VERA) FY 2025 Conference June 11, 2025 03:15 PM ET Speaker0 Okay. Good afternoon. We'll continue with the next session. I'm Paul Choi, and I cover the SpidCap biotech sector here at Goldman Sachs. It's my pleasure to have Vera Therapeutics here on stage with us. To my immediate left is CEO, Marshall Fortis, and to my far left, Chief Medical Officer Rob Brenner. Maybe what we'll do is let Marshall kick it off with maybe some high level comments or an introduction of Avera, just kind of wha ...