Core Viewpoint - The European Medicines Agency (EMA) has granted orphan drug designation to AB8939 for the treatment of acute myeloid leukemia (AML), indicating its potential significant benefit over existing therapies [1][2]. Group 1: Orphan Drug Designation - AB8939 previously received orphan drug designation from the US FDA, and the EU designation is a significant milestone, suggesting that it offers substantial benefits to AML patients [2]. - The criteria for orphan drug designation at EMA are stringent, requiring evidence of significant benefit compared to existing treatments [2][4]. Group 2: Efficacy and Safety Data - Preclinical data from mouse models show that AB8939 provides a significant benefit over current therapies like cytarabine, azacitidine, and venetoclax [3]. - Preliminary efficacy and safety data from Phase 1 trials indicate that AB8939 is effective as a monotherapy with different treatment cycles [3]. - AB8939 demonstrates efficacy against drug-resistant AML cells, with 45% of vincristine-resistant and 66% of cytarabine-resistant cells responding to treatment [4]. Group 3: Mechanism of Action - AB8939 is a synthetic molecule that targets cancer cells by destabilizing microtubules essential for cell division and inhibiting enzymes crucial for cancer stem cell survival [6]. - The chemical name of AB8939 is '1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one' [6]. Group 4: Benefits of Orphan Drug Designation - Orphan drug designation in the EU provides various benefits, including scientific advice on product development, access to centralized marketing authorization, and potential financial incentives [8]. - If approved, AB8939 will enjoy 10 years of marketing exclusivity from the date of registration, provided the orphan designation remains valid [8].

Core Viewpoint - Biodexa Pharmaceuticals PLC reported its preliminary results for the year ended December 31, 2024, highlighting its focus on developing innovative biopharmaceutical products for unmet medical needs, with significant advancements in its clinical pipeline, particularly eRapa, tolimidone, and MTX110 [3][10]. Company Overview - Biodexa Pharmaceuticals PLC is a clinical-stage biopharmaceutical company headquartered in Cardiff, UK, focusing on innovative treatments for diseases with unmet medical needs, including Familial Adenomatous Polyposis (FAP), Non-Muscle Invasive Bladder Cancer (NMIBC), Type 1 Diabetes (T1D), and rare/orphan brain cancers [3][10]. Development Pipeline - The company has transitioned from a drug delivery focus to a therapeutics company, with a pipeline of clinical-stage assets including eRapa, tolimidone, and MTX110, which are expected to improve patient outcomes [11][13]. - eRapa is a proprietary oral formulation of rapamycin, currently in Phase 2 studies for FAP and NMIBC, with plans for a Phase 3 study in FAP expected to recruit approximately 168 patients [18][19]. - Tolimidone, a Lyn kinase activator, is being developed for T1D, with a Phase 2a study planned to confirm dosing in T1D patients [22][23]. - MTX110, designed for direct-to-tumor administration, is in Phase I development for aggressive brain cancers, including glioblastoma and diffuse midline glioma [24][26]. Financial Performance - In 2024, the company reported no gross revenue, a decrease from £0.38 million in 2023, primarily due to the cessation of collaboration agreements [56][58]. - Research and development expenditure increased by 34% to £5.44 million, reflecting investments in clinical-stage assets [60]. - The company experienced a net cash outflow of £4.30 million for the year, compared to an inflow of £3.14 million in 2023, indicating ongoing financial challenges [66][68]. Financing Activities - Biodexa raised a total of $11.1 million from financings in May and July 2024, and secured a $17 million grant from the Cancer Prevention and Research Institute of Texas to support eRapa development [12][49]. - The company entered into an Equity Line of Credit (ELOC) for up to $35 million to support its development programs [72][83]. Regulatory and Compliance - eRapa received FDA Fast Track designation in February 2025, indicating its potential to address significant unmet medical needs in FAP [38]. - The company regained compliance with NASDAQ listing requirements after addressing a previous delisting notification [69].

Core Viewpoint - Scinai Immunotherapeutics Ltd. has filed for a Euro 12 million grant to support the development of its monoclonal antibody PC111, which targets severe dermatological conditions, and has entered into an option agreement to acquire the Italian biotech company Pincell srl [1][2][7]. Company Overview - Scinai Immunotherapeutics Ltd. specializes in inflammation and immunology biological products and offers CDMO services through its Scinai Bioservices unit [1][10]. - Pincell srl focuses on developing first-in-class anti-inflammatory therapies for rare and severe skin diseases, founded by experts in dermatology [11]. Product Development - PC111 is a fully human monoclonal antibody that blocks the activation of apoptosis in skin cells, addressing significant unmet medical needs in skin blistering disorders [3][5]. - The antibody has shown potential in preclinical studies to block blister formation in pemphigus without the use of steroids, indicating its promise as a targeted therapy [5][7]. Grant Application - The grant application submitted by Scinai's Polish subsidiary is part of the European Funds for a Modern Economy (FENG) program, with a decision expected by mid-July to early August [2]. - The funding will primarily be non-dilutive, requiring only Euro 3 million from Scinai's capital, which is one-fifth of the total budget [8]. Market Need - Pemphigus, Stevens-Johnson Syndrome (SJS), and Toxic Epidermal Necrolysis (TEN) are severe conditions with high mortality rates (5-10% for pemphigus and SJS, 30-40% for TEN), highlighting the urgent need for effective treatments [6][7]. Strategic Partnership - The acquisition of Pincell is expected to enhance Scinai's capabilities in developing PC111, leveraging Pincell's expertise in dermatology and research [8][9]. - The management team from Pincell will integrate into Scinai's operations, further strengthening the collaboration [9].

Two Presentations Highlight Data Demonstrating Hypericin Photodynamic Therapy Potential with a Preliminary Comparison to Current TherapyPRINCETON, N.J., March 6, 2025 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that its lead investigators for the cutaneous T-cell lymphoma (CTCL) and psoriasis programs are present ...