An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ?) for Hypophosphatasia (RESTORE)

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

Start Date19 Nov 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT07113158

/ Not yet recruitingPhase 3IIT

Effect of Aspirin and Folic Acid for Sudden Sensorineural Hearing Loss

The annual incidence of sudden sensorineural hearing loss (SSNHL) is 5 to 20 per 100 000 persons. The pathophysiology of SSNHL and acute vestibulo-cochlear syndromes (VCS) is unknown in more than 70% of cases.
Hypothesis : an inner ear microvascular disease represents the key element in the pathogenesis of SSNHL and acute VCS.
Plasma serotonin has among other tissular effect a vasospastic on microcirculation such as the inner ear microvascularisation. Increased plasma homocysteine has a deleterious effect on vascular endothelium. Inner ear microvascularisation sensitized by an increased homocysteine level and the vascular wall would vasoconstrict under serotonin stimulation inducing ischemia of the vestibular and/or cochlear organs.

Start Date01 Nov 2025

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT07013136

/ Not yet recruitingEarly Phase 1

Efficacy and Safety of Folic Acid on Acute Kidney Injury in Adults: A Feasibility Study

The goal of this clinical trial is to evaluate the feasibility of folic acid supplementation as a treatment for adult patients with acute kidney injury (AKI) receiving hospital care.
The main question it aims to answer is: Is it feasible to conduct a fully-powered randomized controlled trial (RCT) to assess the efficacy and safety of folic acid supplementation in AKI management?
Researchers will compare two groups (usual care vs. usual care + 5 mg folic acid) to determine the feasibility of folic acid supplementation and estimate the sample size required for a fully powered RCT.
Participants will:
1. Receive either usual care, or usual care combined with oral folic acid (5 mg daily) until AKI resolution (up to 30 days);
2. Undergo regular monitoring during hospitalization and follow-ups at 3 months and one year post-discharge.

Start Date01 Oct 2025

Sponsor / Collaborator

The University of Hong Kong

[+1]

100 Clinical Results associated with Folic Acid

100 Translational Medicine associated with Folic Acid

100 Patents (Medical) associated with Folic Acid

57,158

Literatures (Medical) associated with Folic Acid

31 Dec 2025·Gut Microbes

Cross-feeding-based rational design of a probiotic combination of Bacterides xylanisolvens and Clostridium butyricum therapy for metabolic diseases

Article

Author: Wang, Tao ; Han, Junjie ; Liu, Chang ; Yang, Lan ; Dai, Huanqin ; Guo, Chun-Jun ; Liu, Shuang-Jiang ; Qiao, Shanshan ; Liu, Hongwei ; Sun, Jingzu ; Du, Mengxuan

The human gut microbiota has gained interest as an environmental factor that contributes to health or disease. The development of next-generation live biotherapeutic products (LBPs) is a promising strategy to modulate the gut microbiota and improve human health. In this study, we identified a novel cross-feeding interaction between Bacteroides xylanisolvens and Clostridium butyricum and developed their combination into a novel LBP for treating metabolic syndrome. Using in-silico analysis and in vitro experiments, we demonstrated that B. xylanisolvens supported the growth and butyrate production of C. butyricum by supplying folate, while C. butyricum reciprocated by providing pABA for folate biosynthesis. Animal gavage experiments showed that the two-strain combination LBP exhibited superior therapeutic efficacy against metabolic disorders in high-fat diet-induced obese (DIO) mice compared to either single-strain treatment. Further omics-based analyses revealed that the single-strain treatments exhibited distinct taxonomic preferences in modulating the gut microbiota, whereas the combination LBP achieved more balanced modulation to preserve taxonomic diversity to a greater extent, thereby enhancing the stability and resilience of the gut microbiome. Moreover, the two-strain combinations more effectively restored gut microbial functions by reducing disease-associated pathways and opportunistic pathogen abundance. This work demonstrates the development of new LBP therapy for metabolic diseases from cross-feeding microbial pairs which exerted better self-stability and robust efficacy in complex intestinal environments compared to conventional single-strain LBPs.

31 Dec 2025·GYNECOLOGICAL ENDOCRINOLOGY

Early gestational diabetes mellitus risk predictor using neural network with NearMiss

Article

Author: Su, Xiaojie ; Huang, Lihong ; Zhao, Min

BACKGROUND:

Gestational diabetes mellitus (GDM) is globally recognized as a significant pregnancy-related condition, contributing to complex complications for both mothers and infants. Traditional glucose tolerance tests lack the ability to identify the risk of GDM in early pregnancy, hindering effective prevention and timely intervention during the initial stages.

OBJECTIVE:

The primary objective of this study is to pinpoint potential risk factors for GDM and develop an early GDM risk prediction model using neural networks to facilitate GDM screening in early pregnancy.

METHODS:

Initially, we employed statistical tests and models, including univariate and multivariate logistic regression, to identify 14 potential risk factors. Subsequently, we applied various resampling techniques alongside a multi-layer perceptron (MLP). Finally, we evaluated and compared the classification performances of the constructed models using various metric indicators.

RESULTS:

As a result, we identified several factors in early pregnancy significantly associated with GDM (p < 0.05), including BMI, age of menarche, age, higher education, folic acid supplementation, family history of diabetes mellitus, HGB, WBC, PLT, Scr, HBsAg, ALT, ALB, and TBIL. Employing the multivariate logistic model as the baseline achieved an accuracy and AUC of 0.777. In comparison, the MLP-based model using NearMiss exhibited strong predictive performance, achieving scores of 0.943 in AUC and 0.884 in accuracy.

CONCLUSIONS:

In this study, we proposed an innovative interpretable early GDM risk prediction model based on MLP. This model is designed to offer assistance in estimating the risk of GDM in early pregnancy, enabling proactive prevention and timely intervention.

31 Dec 2025·Journal of the International Society of Sports Nutrition

Survey of nutritional intake status in college baseball players

Article

Author: Aoyama, Yuka ; Tanaka, Mamoru ; Seguchi, Manato ; Iio, Yoko ; Kozai, Hana ; Mori, Yukihiro ; Ito, Morihiro

BACKGROUND:

Diet is closely related to exercise performance. To improve athletes' performance and manage their condition, it is important to get sufficient energy and various nutrients. Thus, it is necessary that athletes understand their nutritional intake status to improve performance and maintain health. This study aimed to explore the nutritional intake status of college baseball players using the Food Frequency Questionnaire (FFQ). Furthermore, the characteristics of their nutritional intake status with respect to athletic performance were evaluated. The result of this studyprovide an opportunity for many under-developed college athletes with irregular lifestyles to recognize and improve their nutritional problems.

METHODS:

In October 2022, a questionnaire survey of 116 male members of a college baseball club was conducted. Of whom, 100 (94.3%) members responded to the survey and 92 (92.0%) provided valid responses. The survey items included basic characteristics such as college grade and type of living arrangement, and information on living conditions, e.g. whether the participant ate breakfast. Nutritional intake was evaluated using the FFQ. Players were divided into the first (regular players in official games), second (bench players in official games), third (players who may join the second or higher team in the future), and fourth teams (players who do not belong to the first to third teams); these categories were used as a marker of performance level. The Kruskal-Wallis test was used to analyze the association between the performance levels of baseball players and the intake of each nutrient and food group obtained by the FFQ. For items that showed a significant association, inter-group comparison was performed using the Dunn-Bonferroni method.

RESULTS:

Carbohydrate intake was greater in the second team compared with the third and fourth teams; saturated and monounsaturated fatty acid intake was higher in the third team compared with the fourth team. Calcium, zinc, copper, manganese, insoluble dietary fiber, iodine, and molybdenum intake was higher in the second team compared with the fourth team. Intake of grains, sugar, dairy, and total energy was significantly higher in the second team compared with the fourth team. However, the protein intake ratio was significantly lower in the second team compared with the fourth team. Overall, energy deficiency and associated deficiencies in protein, fat, and carbohydrate were observed, in addition to dietary fiber and calcium deficiencies. The intake of several food groups appeared inadequate, such as potatoes, beans, vegetables, fruits, eggs, milk, and fats.

CONCLUSIONS:

The study showed deficiencies in the amount of energy and nutrients such as protein, fat, and carbohydrate in college baseball players. Differences in the intake of carbohydrate, calcium, and insoluble dietary fiber among different performance levels were observed, with significantly higher intake of carbohydrate, calcium, and insoluble dietary fiber in the second team. Implementing organized and strategic remedial measures and athletes' identification of nutritional problems are vital to overcome nutritional and energy deficiencies. This study provides useful information for the development of strategies to support nutritional intake in college baseball players.

News (Medical) associated with Folic Acid

12 Aug 2025

·ir.fatetherapeutics.com

Fate Therapeutics Reports Second Quarter 2025 Financial Results and Business Updates

First patient treated with FT819 off-the-shelf CAR T-cell product candidate following fludarabine-free conditioning for severe lupus nephritis demonstrated durability of response with drug-free definition of remission in systemic lupus erythematosus (DORIS) at 12-month follow-up Held initial discussion with FDA under FT819 RMAT designation to seek feedback on registrational pathway in moderate-to-severe Systemic Lupus Erythematosus (SLE) and refractory Lupus Nephritis (LN) First extrarenal SLE patient treated with FT819 in the absence of conditioning, and as add-on to standard-of-care maintenance therapy, achieved Low Lupus Disease Activity State (LLDAS) at 3- and 6-month follow-up IND application allowed by FDA for FT836 MICA/B-targeted off-the-shelf CAR T cell with Sword and Shield™ technology for conditioning-free treatment of broad array of solid tumors Projected operating runway extended through YE27 to enable achievement of key clinical and collaboration milestones with $249 million in cash, cash equivalents, and investments SAN DIEGO , Aug. 12, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, today reported business highlights and financial results for the second quarter ended June 30, 2025 . “We begin the second half of the year with meaningful progress across our clinical programs as we continue our mission to make cell therapies accessible to all. Our priority remains focused on driving patient enrollment to demonstrate both the therapeutic differentiation and unique on-demand availability of FT819 in autoimmune diseases. We remain encouraged by the promising FT819 data in SLE and LN we reported this past quarter, showing significant disease improvement with less-intensive or no conditioning, and have made strides in expanding our trial sites and accelerating enrollment. Building on this momentum, we are also working closely with the FDA under our RMAT designation with the goal of commencing our registrational study for FT819 in SLE and LN in 2026,” said Bob Valamehr, Ph.D., MBA, President and Chief Executive Officer of Fate Therapeutics . “Additionally, we continue to strengthen our broader pipeline programs with an extended partnership with Ono Pharmaceuticals , and advancements in bringing our next-generation, off-the-shelf CAR T cells with Sword and Shield™ technology toward the clinic. Operationally, we have taken proactive steps to optimize our resource allocation and extend our cash runway, positioning us well to continue executing across our pipeline, working to bring transformative off-the-shelf cellular immunotherapies to patients with unmet needs.” FT819 iPSC-derived off-the-shelf CAR T-cell program in autoimmune disease In discussion with the FDA on potential registrational study design in moderate-to-severe SLE and refractory LN. In August, the Company met with the U.S. Food and Drug Administration (FDA) under its Regenerative Medicine Advanced Therapy (RMAT) designation for FT819 to seek preliminary feedback on a proposed registrational study design to support regulatory approval in moderate-to-severe SLE and refractory LN. In April 2025 , the Company was granted RMAT designation by the FDA for FT819 to treat moderate-to-severe SLE, including LN. Established under the 21st Century Cures Act, the RMAT designation program was created to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions. Interim Phase 1 SLE data using fludarabine-free conditioning regimen presented at EULAR congress and patient enrollment ongoing. The Phase 1 clinical trial of FT819 for the treatment of patients with moderate-to-severe SLE, including patients with LN and with extrarenal lupus (NCT06308978), continues enrolling patients at two dose levels – a single dose of 360 million cells and a single dose of 900 million cells. The Company intends to identify a recommended dose for a registration enabling study and continues to expand clinical site activation in the U.S. and entry into European Union and United Kingdom to broaden geographic reach. At the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in June, interim Phase 1 data from patients with moderate-to-severe SLE with or without LN using a fludarabine-free conditioning regimen was presented. Three patients with refractory active LN (median prior therapies = 8 [7-8]) were treated with a single dose of FT819 at 360 million cells following a fludarabine (flu)-free conditioning regimen. As of the data cut-off date of May 15, 2025 , all three patients achieved an objective renal response. The first LN patient achieved DORIS as well as complete renal response at 6 months, which was also noted at 12-month follow up. Additionally, one patient with refractory extrarenal lupus (prior therapies = 6, including cyclophosphamide; SLEDAI-2K = 18) was treated with a single dose of FT819 at 900 million cells and a single dose of cyclophosphamide. The patient was evaluable for 1-month follow-up, demonstrating improvement across multiple disease-specific scores including an 8-point reduction in SLEDAI-2K from baseline and a 1-point reduction in physician’s global assessment (PGA). First SLE patient treated as add-on to standard-of-care maintenance therapy. The Company’s Phase 1 SLE study is also designed to assess the safety, pharmacokinetics, and anti-B cell activity of FT819 as an add-on to maintenance therapy without conditioning chemotherapy. At the EULAR Congress in June, the Company reported that the first patient treated while on maintenance therapy, a stable dose of mycophenolate mofetil and steroids for the treatment of refractory extrarenal lupus, received a single dose of FT819 at 360 million cells as an add-on to maintenance therapy (prior therapies = 5). As of the data cut-off date of May 15, 2025 , the patient achieved low lupus disease activity state (LLDAS) at 3- and 6-months following administration of FT819 in the absence of conditioning. The patient also experienced a reduction in SLEDAI-2K to 2 from 8 at baseline and in PGA to 0.5 from 2 at baseline, with tapering of steroid dose to less than 5 mg / day. Patient enrollment is ongoing with the aim of investigating patient outcome with single- or multiple-doses of FT819 within a treatment cycle. Phase 1 SLE study amended to include additional B cell-mediated autoimmune diseases. The Company has expanded its current Phase 1 clinical trial of FT819 to include clinical investigation of multiple B cell-mediated autoimmune diseases, with plans to initiate independent dose-expansion cohorts in the second half of 2025 for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). FT825 / ONO-8250 iPSC-derived off-the-shelf CAR T-cell Program in Solid Tumors Phase 1 study ongoing for advanced solid tumors. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplex-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). The study now includes fresh-biopsy testing for HER2 expression to ensure patient stratification and eligibility based on HER2 status. Dose escalation is currently ongoing at the third dose level of 900 million cells, with each patient administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 either as monotherapy or in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy. FT825 / ONO-8250 has demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs) to date. Next-generation iPSC-derived off-the-shelf CAR T-cell Programs with Novel Sword & Shield™ Technology Designed to Reduce or Eliminate the Need for Conditioning Chemotherapy IND allowance by FDA for FT836 MICA/B-targeted CAR T-cell program. In July, the FDA allowed the Company’s investigational new drug (IND) application to initiate Phase 1 clinical testing of FT836, a multiplex-engineered CAR T-cell product candidate uniquely targeting major histocompatibility complex (MHC) proteins A (MICA) and B (MICB) which are expressed on many types of cancer cells with limited detection on healthy tissue. The Phase 1 study is designed to assess the safety and activity of FT836 without administration of conditioning chemotherapy for the treatment of advanced solid tumors. The development of FT836 is supported by a $4 million award from the California Institute of Regenerative Medicine (CIRM). Creation of master iPSC bank for FT839 dual-CAR T-cell program. FT839 is a CD19/CD38 dual CAR T-cell product candidate designed to target an array of aberrant immune cells. At the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in May, the Company presented preclinical data demonstrating robust eradication of aberrant CD19+ B cells, CD38+ plasma cells, and CD38+ activated T cells by FT839 using unmatched peripheral blood mononuclear cells sourced from a patient with autoimmune disease. The Company has generated a master iPSC bank for conduct of further preclinical and IND-enabling studies, and is currently evaluating opportunities for clinical investigation of FT839 in hematological malignancies and autoimmunity, beginning in 2026. Other Corporate Updates Extension of Ono collaboration for second solid tumor CAR T-cell product candidate. Under its collaboration with Ono, the Company is conducting preclinical development of a second iPSC-derived CAR T-cell candidate targeting an undisclosed solid tumor antigen. Based on a review of the preclinical data package for the collaboration candidate in June, the Company and Ono agreed to extend the collaboration’s research term and continue further preclinical development of the candidate. The Company expects to continue to receive co-funding from Ono in connection with its preclinical development activities under the joint research plan through at least June 2026 . Operating runway extension. The Company has implemented a tactical operations plan that is expected to extend funding of its operations through the end of 2027, which is intended to enable the achievement of key clinical and collaboration milestones while maintaining sufficient funds to support ongoing operations beyond those milestones. The cash runway extension includes the pipeline prioritization of its iPSC-derived CAR T-cell programs, a 12% reduction in its current employee headcount, and cost saving measures across the organization. Second Quarter 2025 Financial Results Cash & Investment Position: Cash, cash equivalents, and investments as of June 30, 2025 were $248.9 million . Total Revenue: Revenue was $1.9 million for the second quarter of 2025, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical. Total Operating Expenses: Total operating expenses were $38.9 million for the second quarter of 2025, including research and development expenses of $27.4 million and general and administrative expenses of $11.4 million . Such amount included $7.2 million of non-cash stock-based compensation expense. Shares Outstanding: As of June 30, 2025 , common shares outstanding were 114.7 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares. About Fate Therapeutics’ iPSC Product PlatformHuman induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be administered in combination with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with patient- and donor-sourced cell therapies. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications. About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived T-cell and natural killer (NK) cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com. Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Company’s results of operations, financial condition, anticipated operating expenses and cash runway, and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, including the Company’s plans to complete IND-enabling studies and to submit IND applications for its product candidates, the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials, including in new indications, and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the availability of data from the Company’s clinical trials and the Company’s plans to provide updates on its clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, the Company’s clinical and product development strategy, and the Company’s expectations regarding progress and timelines, the objectives, plans and goals of its collaboration with Ono, and the Company’s expectations regarding the receipt of funding under the collaboration and the expected effects of the Company’s pipeline prioritization plan and reduction in force. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, the risk that the Company may not comply with its obligations under and otherwise maintain its collaboration agreement with Ono, the risk that research funding and milestone payments received by the Company under its collaboration may be less than expected, and the risk that the Company may incur operating expenses in amounts greater than anticipated. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission , including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise. Contact: Christina Tartaglia Precision AQ212.362.1200christina.tartaglia@precisionaq.com Source: Fate Therapeutics, Inc.

Phase 1Cell TherapyClinical ResultImmunotherapyFinancial Statement

04 Aug 2025

·www.globenewswire.com

New Way to Take Iron: IBSA Launches Innovative Orally Dissolving Film Supplement That’s Gentle on the Stomach and Easy to Take

WATFORD, United Kingdom, Aug. 04, 2025 (GLOBE NEWSWIRE) -- A new generation of vitamin supplementation has arrived in the UK. IBSA, the Swiss-based pharmaceutical company, has launched a groundbreaking Iron Orodispersible Film (ODF), a thin, fast-dissolving supplement strip designed to deliver iron and folic acid without the need for water or swallowing tablets. At a time when up to a third of UK adults report difficulty swallowing pills¹, and many struggle with the gastrointestinal side effects of traditional iron supplements², IBSA Iron ODF offers a welcome solution. Using IBSA’s patented FilmTec® technology, this small, lemon-flavoured strip melts in the mouth quickly, delivering a precise dose of 30 mg iron (as ferric pyrophosphate) and 400 μg folic acid with minimal digestive discomfort.“I’ve been taking Iron supplements for over 10 years and tried so many types, tablets, liquids, you name it. They all made me feel sick with nausea and even vomiting. Since I started using the ODF Iron, I haven’t had any of those side effects, and I still get all the benefits. It’s made a huge difference to how I feel in the morning”. Emma, verified patient, Oxford. Unlike some iron tablets that can cause side effects such as nausea, constipation, or a metallic taste, IBSA Iron ODF uses ferric pyrophosphate, a gentler form of iron, alongside a minimalist ODF formulation. This form bypasses much of the digestive system, helping reduce irritation whilst maintaining comparable absorption to some Iron tablets². It’s also gluten-free, lactose-free, and vegan friendly and contains less than 1 kcal per film.Who is it for?IBSA Iron ODF is particularly suited to: Women during menstruation, pregnancy, or postpartum recoveryAthletes with higher iron needsPeople with gastrointestinal absorption issues (e.g. Crohn’s, coeliac disease)Anyone who dislikes or struggles to swallow tablets IBSA Iron ODF is part of a new range of oral film supplements now available from IBSA Care. The range also includes IBSA Vitamin D3 ODF, a 2000 IU supplement formulated to support bone and immune health in individuals with limited sun exposure, and IBSA Vitamin B12 ODF, designed for vegetarians, older adults, and anyone with reduced B12 absorption. All products are based on IBSA’s patented FilmTec® technology, combined with the company’s pharmaceutical expertise and dedication to innovation, providing convenient, high-quality supplements designed for real life. IBSA's ODF technology has been clinically evaluated for iron and vitamin D3 delivery. Studies demonstrate comparable absorption to market-leading capsule formats2 3, offering reassurance that these new supplements are as effective as they are easy to take.Visit www.ibsacare.co.uk for further information. Helen Pope, HPPR+44 (0)7879 818247, helen@helenpopepr.co.uk References1. Cook et al., 2024 – Dysphagia prevalence and patient preferences across Brazil, UK, China, and Indonesia. Healthcare (Basel), 12(18):1827. 2. Cupone et al., 2023 – Clinical evaluation of iron orodispersible film (ODF). Pharmaceutics, 15(6):1575. 3. Cupone et al., 2020 – Development and stability of vitamin D3 ODF formulation. Molecules, 25:5851. UKODF00003DOP: July 2025 A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/c5e03731-fb0b-409a-afd9-fc5e09a52f63

22 Jul 2025

·www.prnewswire.com

Medunik USA expands Siklos® (hydroxyurea) coverage under Medicaid Programs to 37 states

PRINCETON, N.J., July 22, 2025 /PRNewswire/ - Medunik USA, a specialty pharmaceutical company dedicated to providing rare disease patients with access to orphan drugs and part of Duchesnay Pharmaceutical Group (DPG), winner of the 2024 Life Sciences Innovation Award by ADRIQ, is pleased to announce that public reimbursement for Siklos® (hydroxyurea) under Medicaid Programs is now available in 37 states, with 13 states including it on their Preferred Drug List, while 24 states offer it with limited, or no, restrictions. This important milestone for Medunik USA allows broader access to this essential medicine, benefiting patients suffering from sickle cell disease (SCD). Continue Reading Siklos coverage in the USA (CNW Group/Medunik USA) Siklos® has been used worldwide to treat SCD for over 15 years and was approved by the FDA for use in the USA in 2017. This medicine is the only hydroxyurea (HU)-based therapeutic option that offers both flexible dosing and administration, giving patients and caregivers more control over managing their care and shaping a treatment routine that works for them. Patients can choose the best way to take Siklos®, whether by swallowing it whole or split based on the dose, or dissolving it in a small amount of water. Siklos® is the only FDA-approved dissolvable formulation of hydroxyurea that reduces painful crises and blood transfusions for sickle cell patients aged 2 years and older. Siklos® has a BOXED WARNING regarding Low Blood Cell Count and Cancer. The most common side effects of Siklos® in children include: infections and low white blood cells, and in adults include: infections, headache, and dry skin. "We are proud to see increased coverage for this important therapy, which represents a significant step forward in the standard of care for patients with sickle cell disease. Siklos® provides patients with options on how to best take their HU treatment," said Tanya Carro, Executive Vice President, U.S. Entities. "With fewer SCD treatment options currently available, it is even more important to empower patients to stay on therapy in a way that fits their lives. That's what drives long-term adherence and meaningful health outcomes." Sickle cell anemia is the most common inherited blood disorder, resulting in the production of abnormal hemoglobin, known as hemoglobin-S, responsible for the sickling of red blood cells. This disease affects nearly 100,000 Americans, decreases life expectancy by 25 to 30 years, induces significant morbidity and, may therefore, reduce quality of life1,2. Most of those affected are of African ancestry; a minority are of Hispanic or southern European, Middle Eastern or Asian Indian descent3. Committed to improving the lives of people living with rare disorders, Medunik USA strives to remove the logistical barriers and to broaden reimbursement pathways to empower patients with SCD and their caregivers. Medunik USA supports patient access by offering a robust assistance program that includes educational resources and savings programs. For more information please visit: . INDICATION AND IMPORTANT SAFETY INFORMATION SIKLOS is a prescription medicine that is used to reduce the frequency of painful crises and reduce the need for blood transfusions in adults and children, 2 years of age and older, with sickle cell anemia with recurrent moderate to severe painful crises. It is not known if SIKLOS is safe and effective in children less than 2 years of age. IMPORTANT SAFETY INFORMATION BOXED WARNING: LOW BLOOD CELL COUNT and CANCER See Full Prescribing Information for complete Boxed Warning. Low blood cell counts are common with SIKLOS, including low red blood cells, white blood cells, and platelets, and can be severe and life threatening. If your white blood cell count becomes very low, you are at increased risk for infection. Your healthcare provider will check your blood cell counts before and every 2 weeks during treatment with SIKLOS . Your healthcare provider may change your dose or tell you to stop taking SIKLOS if you have low blood cell counts. Tell your healthcare provider right away if you get any of the following symptoms: fever or chills; shortness of breath; body aches; unusual headache; feeling very tired; bleeding or unexplained bruising. Cancer. Some people have developed cancer, such as leukemia and skin cancer, after taking SIKLOS for a long time. Your healthcare provider will check you for cancer. You should protect your skin from the sun using sunblock, hats, and sun-protective clothing. WHAT IS THE MOST IMPORTANT INFORMATION YOU SHOULD KNOW ABOUT SIKLOS? SIKLOS can harm your unborn baby. For females taking SIKLOS who can become pregnant: You should talk with your healthcare provider about the risks of SIKLOS to your unborn baby. You should use effective birth control during treatment with SIKLOS and for at least 6 months after treatment with SIKLOS. Your healthcare provider will perform a pregnancy test before you start treatment with SIKLOS. Tell your healthcare provider right away if you become pregnant or think you may be pregnant. For males taking SIKLOS: SIKLOS can affect your sperm. If you have a female sexual partner who can become pregnant, you should use effective birth control during treatment with SIKLOS and for at least 6 months after treatment. SIKLOS may cause fertility problems in males. Talk to your healthcare provider if this is a concern for you. WHO SHOULD NOT TAKE SIKLOS Do not take SIKLOS if you are allergic to hydroxyurea or any of the ingredients in SIKLOS. See the Medication Guide for a list of the ingredients in SIKLOS. WHAT SHOULD YOU TELL YOUR HEALTH CARE PROVIDER BEFORE TAKING SIKLOS? Tell your healthcare provider about all of your medical conditions, including if you: have kidney problems or are receiving hemodialysis have liver problems have human immunodeficiency virus (HIV) or take HIV medicines. Taking SIKLOS with certain HIV medicines can cause serious reactions and may lead to death. have increased levels of uric acid in your blood (hyperuricemia) have a history of receiving interferon therapy or are currently receiving interferon therapy have leg wounds or ulcers plan to receive any vaccinations. You should not receive "live vaccines" during treatment with SIKLOS. are pregnant or plan to become pregnant. See "What is the most important information I should know about SIKLOS?" are breastfeeding or plan to breastfeed. It is not known if SIKLOS can pass into your breast milk. Do not breastfeed during treatment with SIKLOS. are using a continuous glucose monitor (CGM) to test your blood glucose. Talk to the healthcare provider that prescribed your CGM about whether it is safe to use while you are taking SIKLOS. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. WHAT ARE THE POSSIBLE SIDE EFFECTS OF SIKLOS? SIKLOS may cause serious side effects, including: See "What is the most important information I should know about SIKLOS?" Skin ulcers, including leg ulcers, and death of skin tissue (gangrene) have happened in people who take SIKLOS. This has happened most often in people who receive interferon therapy or have a history of interferon therapy. Your healthcare provider will decrease your dose or stop treatment with SIKLOS if you develop any skin ulcers. Enlarged red blood cells (macrocytosis). Macrocytosis is common in people who take SIKLOS and can make it difficult to detect a decrease of folic acid. Your healthcare provider may prescribe a folic acid supplement for you. Hemolytic Anemia, the fast breakdown of red blood cells, has happened in people who take SIKLOS. Tell your healthcare provider I you develop yellowing of your skin (jaundice) or blood in your urine. Your healthcare provider may do blood tests if you have persistent or worsening anemia not related to sickle cell anemia. The most common side effects of SIKLOS in children include: infections and low white blood cells. The most common side effects of SIKLOS in adults include: infections, headache, and dry skin. These are not all the possible side effects of SIKLOS. You are encouraged to report negative side effects of prescription drugs to the FDA at , or 1-800-FDA-1088. Please read the Full Prescribing Information, including Boxed Warning, Medication Guide and Instructions for Use, at . ABOUT MEDUNIK USA Based in Princeton, New Jersey, Medunik USA is part of Duchesnay Pharmaceutical Group and works to improve the health and quality of life of Americans living with rare diseases by making orphan drug therapies available in the United States. Through its strategic partnerships, Medunik USA develops and provides Americans suffering from rare disease with access to orphan drugs that are not currently available in the U.S. Medunik USA makes critical medications to treat rare diseases available to American patients who might not otherwise have access to these medications. For more information about Medunik USA, please visit . Follow us on LinkedIn. ABOUT DUCHESNAY PHARMACEUTICAL GROUP Duchesnay Pharmaceutical Group (DPG), with its affiliated companies, is headquartered in Blainville, Quebec. The Group consists of six pharmaceutical companies to meet the needs of patients in Canada, the U.S. and abroad. The companies are Duchesnay (Canada) and Duchesnay USA, both dedicated to women's health; Medunik Canada and Medunik USA, which provide treatments for rare and debilitating diseases; and Analog Pharma Canada and Analog Pharma, specializing in orphan generic medications. From its state-of-the-art manufacturing plant, DPG exports its innovative treatments to more than 50 countries. DPG is one of the eight companies across the country chosen to participate in the Government of Canada's Global Hypergrowth Project. This appointment offers exclusive and personalized support for at least two years, in order to accelerate its growth to become an anchor firm in the Canadian economy. DPG is the winner of the 2024 Life Sciences Innovation Award by ADRIQ, the Association for the Development of Research and Innovation of Quebec, which recognizes DPG's healthy workplace culture and commitment to pharmaceutical innovation, while DPG president Éric Gervais is the recipient of the 2024 Bernard-Landry Award by ADRIQ which acknowledges his impactful leadership on Quebec's research and innovation ecosystem. DPG, through its proprietary research and development, and through exclusive partnerships, offers innovative treatments for a variety of medical conditions in women's health, urology, oncology and for rare diseases, plus lower-cost generic medications. DPG recognizes the dedication and professionalism of its employees and promotes a positive culture and flexible work environment. It is deeply committed to environmental responsibility and to giving back to the community through the support of various charitable organizations. For more information, please visit . Follow us on LinkedIn. SOURCE Medunik USA WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalOrphan Drug

100 Deals associated with Folic Acid

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KEGG	Wiki	ATC	Drug Bank
D00070	Folic Acid	V04CX02 B03BB01	DB00158

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Anemia, Megaloblastic	China	Tianjin Lisheng Pharmaceutical Co., Ltd.	01 Jan 1981
Folic Acid Deficiency	Japan	Fuji Pharma Co., Ltd.	01 Aug 1950
Neural Tube Defects	United States	Eli Lilly & Co.	02 Jun 1947

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hyperhomocysteinemia	Phase 2	China	Beijing Silian Pharmaceutical Co., Ltd.	08 Jul 2025
Non-squamous non-small cell lung cancer	Phase 2	Australia	Eli Lilly & Co.	01 Feb 2008
Non-squamous non-small cell lung cancer	Phase 2	Italy	Eli Lilly & Co.	01 Feb 2008
Non-squamous non-small cell lung cancer	Phase 2	Mexico	Eli Lilly & Co.	01 Feb 2008
Non-squamous non-small cell lung cancer	Phase 2	Spain	Eli Lilly & Co.	01 Feb 2008
Contraception	Phase 1	-	Johnson & Johnson Pharmaceutical Research & Development LLC	01 Jan 2005
Ischemic stroke	IND Approval	China	Beijing Silian Pharmaceutical Co., Ltd.	24 Sep 2024
Homocysteinemia	IND Approval	China	Beijing Silian Pharmaceutical Co., Ltd.	09 Nov 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04354428 (CTgov)	Phase 2/3	COVID-19	289	Ascorbic Acid+Folic Acid (Ascorbic Acid and Folic Acid)	jgutnpsuip = wnhcifsokq gsztpeceue (izyquujmgu, raveotcgnz - fsivjjsduc) View more	-	08 Aug 2022
				Hydroxychloroquine Sulfate+Folic Acid (Hydroxychloroquine and Folic Acid)	jgutnpsuip = dgvxqoychj gsztpeceue (izyquujmgu, kamtljhpwn - hsqchbpsva) View more
NCT03994731 (MIRROR RCT \| MIRROR, CTgov)	Phase 4	Gout	152	gout flare prophylaxis regimen+acetaminophen+methylprednisolone+fexofenadine+folic acid+methotrexate+Pegloticase (Pegloticase + MTX)	oqhxsilvwz = zbefhsyhfz ogqqnixave (ynwbtsxtpm, bzceupkjjt - svxhznohng) View more	-	16 May 2022
				placebo+acetaminophen+methylprednisolone+fexofenadine+folic acid+methotrexate+Pegloticase (Pegloticase + Placebo)	oqhxsilvwz = svkfdorpyb ogqqnixave (ynwbtsxtpm, guwtcvvigy - srirsinzlz) View more
NCT00950989 (CTgov)	Phase 2	Rheumatoid Arthritis	252	Placebo+folic acid+Methotrexate (Placebo)	plgschydht = lbyjksract mcfrqipfvh (gktbxcgojs, rjesylwgva - jasytkuvea) View more	-	21 Dec 2021
				Brodalumab+folic acid+Methotrexate (Brodalumab 70 mg)	plgschydht = sfdnksbdwm mcfrqipfvh (gktbxcgojs, yrwvyqqghw - aiaohgpekv) View more
NCT02930343 (CTgov)	Phase 3	Rheumatoid Arthritis	136	Hydroxychloroquine+Prednisolone+Folic Acid+Methotrexate+Leflunomide (Group 1- MTX+LEF+HCQ)	vgjxwmnhkl = lmtwezjpwt gflrvyzwle (lxokcrjtjd, bxtmgifmeb - pvxonguhqe) View more	-	10 Jun 2021
				Hydroxychloroquine+Prednisolone+Folic Acid+Methotrexate+Sulfasalazine (Group 2- MTX+SSZ+HCQ)	vgjxwmnhkl = rkedzgjyaz gflrvyzwle (lxokcrjtjd, haanrtpdki - cqjsctjoaa) View more
NCT03172325 (CTgov)	Phase 3	Rheumatoid Arthritis	136	Adalimumab+Prednisolone+Folic Acid+Methotrexate (CinnaGen Adalimumab)	hcodjjocyg = tobcpbpslm hfmaoouidi (zvigslakjw, hdrxdfibaq - kafdjasdib) View more	-	02 Feb 2021
				Adalimumab+Prednisolone+Folic Acid+Methotrexate (AbbVie Adalimumab)	hcodjjocyg = ausaxbmfhg hfmaoouidi (zvigslakjw, lduijtcaij - kggcoegfwb) View more
NCT03635957 (MIRROR OL \| MIRROR-OL \| MIRROR, CTgov)	Phase 4	Gout \| Hyperuricemia	14	Infusion Reaction (IR) Prophylaxis+Folic Acid+Methotrexate (MTX)+Pegloticase	rszupwlmmi = dalocdwfbw sbmcdcaaud (jlpjvgkfki, jwftrzwxoi - ojwzyimvoz) View more	-	18 Nov 2020
NCT00655980 (VINO, CTgov)	Not Applicable	Coronary Artery Disease	687	placebo+nitrous oxide (Comparator)	qravllomfd = pejptrzwea deqfyfyunx (vnwbzvqnhj, telxthfqkj - ezveqyzwjz) View more	-	01 Oct 2020
				standard of care (Standard of Care)	qravllomfd = vnwleihixf deqfyfyunx (vnwbzvqnhj, lksuqhacmj - lyacodbmdh) View more
NCT01355159 (FACT, CTgov)	Phase 3	Pre-Eclampsia	2,464	Folic Acid 4 mg (Folic Acid 4 mg)	ilpgpygvwr = vytfzgczam rrumcigipv (xfmrwqnuyp, bjeydkhuxv - lulfqqyblz) View more	-	07 Jul 2020
				Placebo (Placebo)	ilpgpygvwr = cjfzhmskul rrumcigipv (xfmrwqnuyp, gqeisvpzod - qossvpnmrn) View more
NCT01355159 (Pubmed \| J Matern Fetal Neonatal Med)	Phase 3	Pre-Eclampsia	428	High-dose folic acid	ckvrvmiqqp(jhkbjvbtnq) = urkjuyqpfr rwxipuastd (owrlwnlffc )	-	18 Feb 2020
				high-dose folic acid supplementation (Placebo)	ckvrvmiqqp(jhkbjvbtnq) = dmcsuxnhxs rwxipuastd (owrlwnlffc )
NCT00272324 (Pubmed \| Am J Clin Nutr)	Phase 2/3	Colorectal Cancer	1,021	Folic acid 1 mg/d	ufnqkqzmkt(dpplsbscnx): RR = 1.94 (95% CI, 1.02 - 3.68), P-Value = 0.04 View more	-	01 Oct 2019
				Folic acid (Placebo)

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