Pivotal Phase III liver cancer study enrolling in Europe and Israel RAMAT GAN, Israel, Nov. 18, 2025 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs targeting oncological and inflammatory diseases, announced today that a patient treated with Namodenoson has reached an overall survival of 9 years to date with complete response to treatment. The patient, who suffered from advanced liver cancer, was ...

Core Insights - Moleculin Biotech, Inc. is progressing with its pivotal Phase 2B/3 "MIRACLE" study of Annamycin for treating adult patients with relapsed or refractory acute myeloid leukemia (AML), with 60% of the target enrollment completed as of November 4, 2025 [1][2][4] - The company expects to complete treatment for the first 45 subjects by Q1 2026, with initial unblinded data anticipated shortly thereafter [1][4][5] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, and it is designed to avoid common cardiotoxicity associated with existing anthracyclines [7][9] Enrollment and Study Progress - The MIRACLE study is a global, multi-center, randomized, double-blind, placebo-controlled trial, combining data from its Phase 2B and Phase 3 portions to measure primary efficacy endpoints [3][10] - The first unblinding will involve 30 subjects treated with Annamycin in combination with HiDAC and 15 subjects treated with HiDAC plus placebo [3] - Recruitment is ongoing, with subjects enrolled from five countries, although some European sites are experiencing bed shortages affecting enrollment [5][2] Future Expectations - The company anticipates a second unblinding in the first half of 2026, following the initial unblinding of data [4] - The accelerated timeline for recruitment is attributed to positive feedback from potential investigators [4] - Annamycin is positioned as a potentially safer and more effective treatment option for AML, addressing a significant gap in current therapies [2][4]

Announced U.S. Food and Drug Administration (FDA) Acceptance for Reviewof New Drug Application (NDA) for GTx-104 FDA Established April 23, 2026 as PDUFA Target Date for Review of Submission Seeking Approval for GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Phase 3 STRIVE-ON Safety Trial Data Presented at 2025 Neurocritical Care Annual Meeting Granted Sixth U.S. Patent Covering IV Dosing Regimen for GTx-104 PRINCETON, N.J., Nov. 13, 2025 (GLOBE NEWSWIRE) -- Grace Therape ...

Contact Us Back to the Newsroom Exousia Pro is Excited to Announce that it has Received Orphan Drug Designation from the FDA Tuesday, 28 October 2025 10:20 AM Topic:Â Company Update Exousia Pro is in a unique position, as only 11% of ODAs are awarded at the preclinical stage. ORLANDO, FLORIDA / ACCESS Newswire / October 28, 2025 / Exousia Pro, Inc. (formerly Marijuana, Inc.) (OTCPINK:MAJI), a clinical-stage biotech company using exosomes in the treatment of cancer and other maladies, is pleased to announce ...

Company Overview - Grace Therapeutics, Inc. is a late-stage biopharma company focused on developing GTx-104, a novel injectable formulation of nimodipine for I.V. infusion targeting aneurysmal subarachnoid hemorrhage (aSAH) [6] - The company has received Orphan Drug Designation from the FDA for its lead clinical asset, GTx-104, which provides seven years of marketing exclusivity post-launch in the U.S. [6] Funding and Financials - The company secured approximately $4.0 million in additional funding through the exercise of common warrants, issuing 1,345,464 new shares at an exercise price of $3.003 per share [1] - The remaining 1,190,927 common warrants from the 2023 private placement have expired following the FDA's acceptance for review of the NDA for GTx-104 [1] Product Development - GTx-104 is being developed to address significant unmet medical needs in aSAH patients, with a PDUFA target date set by the FDA for April 23, 2026, for the NDA review [2] - The NDA submission is supported by positive data from the Phase 3 STRIVE-ON safety trial of GTx-104 [2] Medical Context - aSAH is a type of stroke caused by the rupture of an aneurysm, accounting for about 5% of all strokes, with an estimated 42,500 hospital-treated patients in the U.S. annually [3] - GTx-104 aims to provide a convenient I.V. delivery method, potentially eliminating the need for nasogastric tube administration in unconscious or dysphagic patients, and has been administered to over 200 patients and healthy volunteers [5] Technology and Advantages - GTx-104 utilizes unique nanoparticle technology to facilitate the aqueous formulation of insoluble nimodipine for standard peripheral I.V. infusion [4] - The intravenous delivery of GTx-104 may lower food effects, drug-to-drug interactions, and dosing errors, while better managing hypotension in aSAH patients [5]

Core Points - Quoin Pharmaceuticals has received Orphan Drug Designation from the FDA for its product QRX003, aimed at treating Netherton Syndrome, following a similar designation from the EMA in May 2025 [1][2][3] - The FDA's Orphan Drug Designation provides benefits such as tax credits for clinical testing, waiver of FDA application fees, and seven years of market exclusivity upon approval [2] - QRX003 is currently undergoing two late-stage pivotal clinical trials, with enrollment expected to complete in Q1 2026 and top-line data anticipated in the second half of 2026 [3] Company Overview - Quoin Pharmaceuticals is a late clinical-stage specialty pharmaceutical company focused on developing treatments for rare and orphan diseases [4] - The company's pipeline includes products targeting various rare conditions, including Netherton Syndrome, Peeling Skin Syndrome, and others [4]

Core Insights - Hoth Therapeutics, Inc. has received FDA Orphan Drug Designation for HT-KIT, a precision antisense oligonucleotide targeting KIT mRNA, demonstrating over 80% suppression of KIT expression and significant tumor-volume reduction in systemic mastocytosis and GIST models [1][3][5] - The company has completed GLP-validated bioanalytical methods to support IND-enabling studies, with a Japan Patent extending platform protection to 2039 [1][3] Preclinical Data - HT-KIT achieved over 80% reduction of KIT mRNA/protein in both in vitro and in vivo models, with significant tumor-volume reduction observed by Day 8 in xenograft models [7] - The preclinical studies reported no dose-limiting toxicities, indicating a favorable tolerability profile [7] Mechanism of Action - HT-KIT operates at the transcript level, silencing both mutant and wild-type KIT, which may help bypass resistance pathways and reduce off-target effects compared to small-molecule TKIs [3][5] Next Steps - The company plans to complete GLP toxicology and CMC packages, submit an IND, and initiate a Phase 1/2 dose-escalation/expansion study in advanced systemic mastocytosis and other KIT-driven tumors [6][8] - The study will include translational biomarkers of target engagement and early efficacy readouts [6][8] Company Overview - Hoth Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments to improve patient quality of life, collaborating with scientists and clinicians to advance early-stage pharmaceutical research [9]

Core Viewpoint - The FDA has accepted Sanofi's Tzield for expedited review to delay the progression of stage 3 type 1 diabetes, highlighting its potential to address a significant unmet medical need [1][2]. Regulatory Review - Tzield's supplemental biologics license application (sBLA) is part of the Commissioner's National Priority Voucher pilot program, which aims to reduce the review time from 10-12 months to 1-2 months while ensuring safety and efficacy standards [1]. - Tzield is also under review for accelerated approval, which allows the FDA to evaluate therapies for serious conditions based on surrogate endpoints [4][9]. Clinical Study Results - The sBLA is supported by the PROTECT phase 3 study, which demonstrated significant preservation of beta cell function by slowing the decrease in mean C-peptide levels compared to placebo [2][8]. - The PROTECT study involved 328 participants aged 8-17 years, with a randomization ratio of 2:1 for Tzield versus placebo [7]. Safety Profile - Adverse events in the PROTECT study were consistent with previous studies, with common events including headache, nausea, and gastrointestinal symptoms. 1.8% of participants developed cytokine release syndrome possibly related to Tzield [3]. Market Position - If approved, Tzield would be the first disease-modifying therapy for stage 3 type 1 diabetes in adults and pediatric patients aged eight years and older [9]. - Tzield is already approved in multiple countries, including the US, UK, China, and Canada, for delaying the onset of stage 3 type 1 diabetes in patients diagnosed with stage 2 [5][12]. Company Overview - Sanofi is an R&D-driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [13].

Core Insights - Esperion Therapeutics (ESPR) has nominated ESP-2001, a specific allosteric ATP citrate lyase (ACLY) inhibitor, as a new preclinical development candidate for treating primary sclerosing cholangitis (PSC), a rare autoimmune liver disease with no approved treatments [1][7] - The company plans to initiate IND-enabling studies for ESP-2001 and submit an IND application to the FDA, aiming to start clinical studies in 2026 [2] - ESP-2001 has shown potential in reducing liver and bile duct injury, inflammation, and fibrosis in preclinical studies, indicating its ability to impact PSC progression [3] Company Developments - Esperion's stock rose by 5.2% following the announcement of ESP-2001 [2] - The collaboration with Evotec (EVO) was crucial in discovering ESP-2001, combining Esperion's expertise in ACLY therapy with Evotec's drug discovery platform [2][3] - The nomination of ESP-2001 triggered an undisclosed payment to Evotec [3] Financial Performance - Esperion's net product sales of Nexletol and Nexlizet in the U.S. grew by 42% in the first half of 2025, reaching $75.2 million, driven by increased prescription volumes [10] - Esperion aims to diversify its portfolio beyond cardiovascular diseases into the liver disorder market, targeting a potential market opportunity exceeding $1 billion annually with ESP-2001 [10] Market Position - Esperion currently holds a Zacks Rank of 3 (Hold) [11] - The company has two FDA-approved drugs, Nexletol and Nexlizet, which are marketed under different names in ex-U.S. markets [8][9]

Core Viewpoint - Kiniksa Pharmaceuticals has received Orphan Drug Designation from the FDA for KPL-387, aimed at treating pericarditis, including recurrent cases, which highlights the company's commitment to addressing unmet medical needs in rare diseases [1][2]. Company Overview - Kiniksa Pharmaceuticals is focused on developing and commercializing novel therapies for diseases with unmet needs, particularly in cardiovascular indications [4]. - The company aims to improve the lives of patients suffering from debilitating diseases through innovative treatments [4]. Product Information - KPL-387 is a fully human IgG2 monoclonal antibody that targets human IL-1R1, inhibiting the activity of cytokines IL-1α and IL-1β [5]. - The drug is expected to offer a convenient treatment option for recurrent pericarditis patients, potentially allowing for a single monthly subcutaneous self-injection [2][5]. - Data from the Phase 2 dose-focusing portion of the KPL-387 Phase 2/3 trial is anticipated in the second half of 2026 [2]. Orphan Drug Designation - The FDA's Orphan Drug Designation is granted to products treating rare diseases, which includes those affecting fewer than 200,000 people in the U.S. [3]. - This designation provides financial incentives such as grant funding for clinical trials, tax advantages, and waivers for user fees [3].