RAD101 in U.S. Phase 2 clinical trial to distinguish between recurrent disease and treatment effect of brain metastases originating from solid tumors of different origin SYDNEY, June 11, 2025 (GLOBE NEWSWIRE) -- Radiopharm Theranostics (ASX:RAD, "Radiopharm" or the "Company"), a clinical-stage biopharmaceutical company focused on developing innovative oncology radiopharmaceuticals for areas of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track De ...

BOSTON, June 10, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to mavorixafor, an oral CXCR4 antagonist, for the treatment of chronic neutropenia (CN). The company is currently conducting a global, pivotal Phase 3 clinical trial (4WARD) evaluating mavorixafor in certain primary CN conditions. Mavorixafor was ...

Summary of Amylyx Pharmaceuticals (AMLX) Conference Call Company Overview - **Company**: Amylyx Pharmaceuticals (AMLX) - **Focus**: Developing therapies for rare diseases, particularly post bariatric hypoglycemia (PBH) and other neurodegenerative conditions [5][6] Key Points on Lead Asset: Avexatide - **Indication**: Avexatide is a competitive inhibitor of GLP-1, targeting PBH, a rare condition following bariatric surgery characterized by severe hypoglycemic events [5][6] - **Clinical Trials**: - Five past trials demonstrated significant benefits, including a 53% reduction in level two hypoglycemic events and a 66% reduction in level three events [6][21] - Phase III study recruitment expected to complete by the end of 2025, with data anticipated in the first half of 2026 [7][49] - **Market Opportunity**: - PBH affects an estimated 160,000 people in the U.S., with the number expected to rise due to increasing bariatric surgeries (approximately 270,000 annually) [12][13] - Current treatment options are limited, primarily involving medical nutrition therapy [14][15] Regulatory Designation and Pricing - **Breakthrough Therapy Designation**: Received based on prior trial data, facilitating closer collaboration with the FDA [7][20] - **Pricing Strategy**: Pricing has not been finalized; however, the company is considering the orphan drug designation and recent market entries in rare endocrine disorders as benchmarks [17][18] Other Assets and Trials - **AMX35**: - Studied in Wolfram syndrome and progressive supranuclear palsy (PSP) [7][58] - Recent data from Wolfram syndrome trials showed stabilization and improvement in various outcomes over 48 weeks [60][61] - **AMX-114**: An antisense oligonucleotide targeting calpain two, currently in a clinical trial for ALS, with data expected by the end of the year [9] Competitive Landscape - **Current Competition**: No known drugs have shown efficacy in treating PBH, positioning Avexatide uniquely in the market [54] - **GLP-1 Agonists**: While there are concerns about competition from GLP-1 agonists, the company believes that the target population for bariatric surgery is distinct and will continue to grow [55][56] Future Milestones - **Phase III Trial**: Expected to provide pivotal data for FDA approval, with a focus on maintaining consistency with Phase II trial parameters [36][62] - **Market Access**: Preparations for market access strategies are underway, emphasizing the importance of ensuring patient access post-approval [52] Conclusion - Amylyx Pharmaceuticals is advancing its lead asset Avexatide through critical clinical trials aimed at addressing significant unmet needs in rare diseases, particularly PBH. The company is optimistic about its market potential and regulatory pathway, with ongoing efforts to ensure successful trial outcomes and market access strategies.

EMA Orphan Drug Designation (ODD) builds on U.S. Food & Drug Administration (FDA) ODD and Fast Track Designation, underscoring the urgent need for new treatment optionsThe multiple regulatory designations were granted following strong positive results from a global randomized Phase 1b/2 study in first-line HCC demonstrating superior outcomes for amezalpat combination therapy across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to sta ...

Core Viewpoint - Innovent Biologics has presented promising Phase 1 study results for IBI343, a novel anti-CLDN18.2 ADC, for advanced pancreatic cancer, indicating its potential as a breakthrough therapy in a challenging-to-treat malignancy [2][4]. Company Overview - Innovent Biologics is a leading biopharmaceutical company founded in 2011, focused on developing high-quality medicines for various diseases, including oncology, cardiovascular, and autoimmune disorders [10]. - The company has launched 15 products and has multiple assets in various stages of clinical trials, partnering with over 30 global healthcare companies [10]. Clinical Study Results - The Phase 1 study of IBI343 showed a confirmed overall objective response rate (cORR) of 22.7% and a disease control rate (DCR) of 81.8% in patients with CLDN18.2 expression treated at a 6mg/kg dose [6]. - The median progression-free survival (mPFS) was reported at 5.4 months, and the median overall survival (mOS) was 9.1 months, with variations based on prior treatments [6]. - The safety profile of IBI343 was favorable, with a low rate of gastrointestinal toxicity and no new safety signals reported [6]. Market Context - Pancreatic cancer is one of the most aggressive cancers, with a global 5-year survival rate of less than 10% and approximately 510,000 new cases and 467,000 deaths annually [3][4]. - The clinical options for second-line treatment of advanced pancreatic cancer are limited, highlighting the urgent need for effective therapies [4]. Regulatory Designations - IBI343 has received Breakthrough Therapy Designation (BTD) from China's National Medical Products Administration (NMPA) and Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) [2][9].

Core Viewpoint - Teva Pharmaceutical Industries has received Fast Track designation from the FDA for its investigational anti-IL-15 antibody, TEV-53408, aimed at treating celiac disease, highlighting the urgent need for effective treatments in this area [1][3][7]. Company Overview - Teva Pharmaceutical Industries Ltd. is a global biopharmaceutical leader with over 120 years of experience, focusing on innovation and the development of medicines, including generics and biologics, to improve health outcomes [6]. Product Development - TEV-53408 is currently undergoing a Phase 2a trial to evaluate its efficacy and safety in adults with celiac disease, which affects approximately 1% of the global population [1][4][5]. - The drug works by inhibiting interleukin-15 (IL-15), which is responsible for the immune response to gluten, thereby aiming to reduce intestinal inflammation and damage in celiac disease patients [3][4]. Market Need - Celiac disease is a chronic autoimmune disorder that significantly impacts quality of life, with the only current treatment being a strict gluten-free diet, which many patients struggle to adhere to [5]. - There is a significant unmet medical need for new treatment options, as many patients continue to experience debilitating symptoms even on a gluten-free diet [3][5].

-- First patient dosed in SIGNAL-AA Phase 2a Part B; topline data readout on-track for 1H'26 ---- First patient dosed in SIGNAL-AA Part A open-label extension (OLE) ---- Fast Track designation (FTD) granted to bempikibart for the treatment of alopecia areata (AA); SIGNAL-AA Part A results presented as a late-breaking oral presentation at the 2025 American Academy of Dermatology (AAD) Annual Meeting ---- Cash and cash equivalents of $65.5 million as of March 31, 2025 expected to provide financial runway into ...

– Fast Track Designation highlights potential of ATH434 to address high unmet need for individuals with MSA – MELBOURNE, Australia and SAN FRANCISCO, May 05, 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 the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATH434 for the treatment of Multiple S ...

LONDON and NEW YORK, May 01, 2025 (GLOBE NEWSWIRE) -- OKYO Pharma Limited (NASDAQ: OKYO), an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod (formerly called OK-101) to treat NCP, an ocular condition associated with chronic and often severe pain but without an FDA-approved therapy, announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to urcosimod for the treatment of neuropathic corneal pain (NCP). Fast Track designation by the FDA is g ...

Core Insights - Perspective Therapeutics, Inc. has initiated dosing for the first patient in a new cohort of a Phase 1/2a trial for [212Pb]VMT01, a targeted alpha-particle therapy for melanoma patients with positive MC1R imaging scans [1][2] Company Overview - Perspective Therapeutics is focused on developing advanced radiopharmaceutical treatments for cancer, utilizing the alpha-emitting isotope 212Pb to target cancer cells specifically [6] - The company is also working on complementary imaging diagnostics to personalize treatment and optimize patient outcomes through a "theranostic" approach [6] Clinical Development - The current trial involves administering [212Pb]VMT01 at a dose of 1.5 mCi as monotherapy, with earlier cohorts showing promising initial results [2][3] - The FDA granted Fast Track Designation for [212Pb]VMT01 for treating unresectable or metastatic melanoma with MC1R tumor expression, aimed at expediting its development [4] Melanoma Context - Melanoma is a serious skin cancer with approximately 100,000 new diagnoses and 8,300 deaths annually in the U.S. Metastatic melanoma has a poor prognosis, with a 50% survival rate at one year and 29%-35% at five years [5] - There is a significant unmet need for effective treatments, especially for patients who are refractory to existing therapies, as current second-line therapies offer limited progression-free survival of 2-5 months [5]