The goal of this clinical trial is to learn if adjunctive topical Cyclosporine A eye drops combined with standard of care topical Natamycin treatment improves vision outcomes in patients with fungal keratitis.

Start Date01 Jun 2025

Sponsor / Collaborator

The University of California, San Francisco

[+1]

NCT06752694

/ RecruitingPhase 2IIT

Ruxolitinib Based GVHD Prophylaxis Regimen for Older Adults Receiving Non-ATG Containing Non-Myeloablative Hematopoietic Cell Transplantation for Acquired Aplastic Anemia

This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation [HCT]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.

Start Date16 May 2025

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

[+1]

NCT06898853

/ Not yet recruitingNot ApplicableIIT

The Effect of 0.1% Topical Ciclosporin A for 12-weeks on the Ocular Surface Innate and Adaptive Immune Response in Dry Eye Disease

Dry eye disease (DED) is a common, long-lasting condition that affects the surface of the eye. It happens when there's a problem with tear production or quality, which can lead to inflammation and discomfort. The immune system plays a big role in how DED develops and continues.
Researchers have found that in people with DED, there are more immune cells and inflammatory substances in the tears and on the eye's surface. This includes various types of immune cells, like T cells and dendritic cells, which are part of the body's defense system.
The first treatment for DED is usually artificial tears, but because the condition is chronic and can flare up, clinicians often use anti-inflammatory treatments too. One such treatment is cyclosporine A (CsA), which comes as eye drops. CsA works by reducing inflammation and affects how immune cells behave.
Researchers can study the immune cells on the eye's surface using a special microscopy technique called in vivo confocal microscopy (IVCM). A newer version of this method, called functional IVCM (Fun-IVCM), allows researchers to watch how these cells move and behave over time.
In the current study, researchers want to compare 0.1% CsA with a lubricating eye drop to see how they affect the immune cells on the eye's surface. The researchers will use Fun-IVCM to look at the number, shape, and movement of immune cells of the eye. The researchers will also collect samples from the eye's surface and tears to measure various markers of inflammation.
The goal is to better understand how CsA works in treating DED by directly observing its effects on the immune response in the eye, which is unexplored. This could help improve treatments for people suffering from this condition and expand the use of CsA in DED.

Start Date01 May 2025

Sponsor / Collaborator

The University of New South Wales

[+1]

100 Clinical Results associated with Cyclosporine

100 Translational Medicine associated with Cyclosporine

100 Patents (Medical) associated with Cyclosporine

74,629

Literatures (Medical) associated with Cyclosporine

31 Dec 2025·Gut Microbes

Gut microbes metabolize strawberry phytochemicals and mediate their beneficial effects on vascular inflammation

Article

Author: Della Lucia, Ceres Mattos ; Neilson, Andrew P. ; Zhong, Ying ; Anandh Babu, Pon Velayutham ; Iglesias-Carres, Lisard ; Wankhade, Umesh D. ; Symons, J David ; Petersen, Chrissa ; Satheesh Babu, Adhini Kuppuswamy ; Paz, Henry A. ; Shankar, Kartik ; Jalili, Thunder

Evidence suggests that a healthy gut microbiome is essential for metabolizing dietary phytochemicals. However, the microbiome's role in metabolite production and the influence of gut dysbiosis on this process remain unclear. Further, studies on the relationship among gut microbes, metabolites, and biological activities of phytochemicals are limited. We addressed this knowledge gap using strawberry phytochemicals as a model. C57BL/6J mice were fed a standard diet [C]; strawberry-supplemented diet (~2 human servings) [CS]; strawberry-supplemented diet and treated with antibiotics (to deplete gut microbes) [CSA]; high-fat diet (HFD) [HF]; strawberry-supplemented HFD [HS]; and strawberry-supplemented HFD and treated with antibiotics [HSA] for 12 weeks. First, antibiotic treatment suppressed the production of selected metabolites (CSA vs. CS), and p-coumaric acid was identified as a strawberry-derived microbial metabolite. Second, HFD-induced dysbiosis negatively affected metabolite production (HS vs. HF), and hippuric acid was identified as a microbial metabolite in HFD conditions. Third, dietary strawberries improved HFD-induced vascular inflammation (HS vs. HF). However, antibiotic treatment reduced metabolite production and abolished the vascular effects of strawberries (HSA vs. HS), indicating the importance of gut microbes in mediating the vascular benefits of strawberries via metabolites. Fourth, strawberry supplementation decreased Coprobacillus that was positively associated with vascular inflammation, whereas it increased Lachnospiraceae that was negatively associated with vascular inflammation and positively associated with hippuric acid. Fifth, hippuric acid was negatively associated with vascular inflammation. Our study fills in some pieces of the giant puzzle regarding the influence of gut microbes on the biological activities of phytochemicals. HFD-induced gut dysbiosis negatively impacts metabolite production and a strong association exists among gut microbes, strawberry-derived microbial metabolites, and the vascular benefits of dietary strawberries. Further, our study provides significant proof of concept to warrant future research on the use of strawberries as a nutritional strategy to prevent vascular complications.

31 Dec 2025·RENAL FAILURE

Factors associated with chronic calcineurin inhibitor nephrotoxicity in children with minimal-change disease

Article

Author: Yang, Shicong ; Yue, Zhihui ; Mo, Ying ; Jin, Bei ; Cheng, Cheng ; Lu, Ziji ; Lin, Aihua ; Pei, Yuxin ; Chen, Lizhi ; Jiang, Xiaoyun

BACKGROUND:

Calcineurin inhibitors (CNIs), such as cyclosporine (CsA) and tacrolimus (TAC), are commonly used to treat children with complicated minimal change nephrotic syndrome. However, chronic nephrotoxicity associated with CNIs poses a significant safety concern. This study aimed to identify the risk factors that contribute to chronic nephrotoxicity in these patients.

MATERIAL AND METHODS:

Clinical and pathological data of MCD children treated with CsA or TAC in our center between 1 January 2003 and 31 December 2022, were retrospectively reviewed. Kidney biopsies were performed on 80 patients who received CNI treatment for more than 6 months.

RESULTS:

Chronic CNI nephrotoxicity (striped interstitial fibrosis with tubular atrophy) was observed in 15% (12/80) of patients. Higher CNI culminating amounts were shown in patients who developed nephrotoxicity regardless of CsA or TAC treatment. Risk factors for chronic CNI nephrotoxicity included persistent nephrotic-range proteinuria for more than 30 days during CNI treatment, increased urinary NAG level, and CNI resistance. Multivariate analysis revealed that increased urinary NAG level and CNI resistance were the independent risk factors for chronic CNI nephrotoxicity in children with MCD.

CONCLUSION:

MCD children who developed CNI resistance were susceptible to chronic CNI nephrotoxicity. Urinary NAG might be a valuable biomarker for CNI nephrotoxicity prediction in MCD children.

01 Dec 2025·JOURNAL OF CLINICAL IMMUNOLOGY

Restitutio ad integrum: Rescuing the Alveolar Macrophage Function with HSCT in Pulmonary Alveolar Proteinosis Due to CSF2Rα Deficiency

Article

Author: Pandrowala, Ambreen ; Mishra-Sopori, Varsha ; Khan, Sanaa ; Bodhanwala, Minnie ; Hiwarkar, Prashant ; Chandane, Parmarth ; Khosla, Indu ; Kataria, Darshan ; Sehgal, Kunal ; Prabhudesai, Pralhad

Hereditary pulmonary alveolar proteinosis (hPAP) is a rare lung-related primary immunodeficiency. In hPAP, variants of genes encoding the heterodimeric GM-CSF receptor alpha or beta-chains (CSF2Rα, CSF2Rβ) lead to perturbations in GM-CSF signalling. These perturbations impair the scavenging function of pulmonary alveolar macrophages leading to accumulation of surfactant proteins and lipids within the alveoli. The replacement of defective pulmonary alveolar macrophages can be achieved with allogeneic hematopoietic stem cell transplantation. However, previous reports highlight undesirable pulmonary outcomes associated with this therapeutic approach. We report a 4-year-old developmentally normal girl born of second-degree consanguineous marriage diagnosed with severe form of CSFRα-deficient PAP. She required recurrent whole lung lavage and hence was treated with allogeneic hematopoietic stem cell transplantation. A reduced toxicity treosulfan-based myeloablative regimen with alemtuzumab serotherapy was used for conditioning. Ciclosporin, mycophenolate mofetil and FAM (fluticasone inhaler, azithromycin, montelukast) were used to prevent graft-versus-host disease and immune-related complications of lung. Her post-transplant course was uneventful with full donor chimerism and complete resolution of symptoms. We demonstrate for the first time in a case of severe CSF2Rα-deficient PAP, the successful use of hematopoietic stem cell transplantation as a primary curative treatment, restoring normal lungs both anatomically and functionally. The case report provides evidence for considering allogeneic hematopoietic stem cell transplant in severe forms of CSF2R-deficient PAP.

443

News (Medical) associated with Cyclosporine

20 Mar 2025

·www.globenewswire.com

Eyenovia Enters into Non-Binding Letter of Intent to Effect Reverse Merger with Betaliq

Combination, if successful, would create a new publicly-listed eye care company combining Betaliq’s EyeSol® water-free drug delivery technology for glaucoma with Eyenovia’s Optejet® device platform The combined company would continue marketing Eyenovia’s FDA-approved products – and plans to in-license additional products to generate near-term revenue NEW YORK, March 20, 2025 (GLOBE NEWSWIRE) -- Eyenovia, Inc. (NASDAQ: EYEN) (“Eyenovia” or the “Company”), an ophthalmic technology company developing and commercializing advanced products leveraging its proprietary Optejet topical ophthalmic medication dispensing platform, today announced that it has entered into a non-binding letter of intent (LOI) contemplating a potential reverse merger transaction with Betaliq, Inc., a clinical stage pharmaceutical company with a therapeutic focus on glaucoma. The proposed merger would create a new ophthalmic company that combines two FDA-approved technologies: Betaliq’s EyeSol® water-free drug delivery technology for use in glaucoma, and Eyenovia’s Optejet topical ophthalmic liquid dispensing platform. The proposed transaction remains subject to completion of mutually satisfactory due diligence, the negotiation and execution of definitive agreements on mutually satisfactory terms, the approval of such definitive documentation by the boards of directors of both Eyenovia and Betaliq, and the completion of necessary financing contingencies. There can be no assurance that any such agreement will be executed or the proposed transaction with Betaliq will be consummated, or as to the timing of the entry of any such agreement or the consummation of such proposed transaction. The proposed transaction assumes a value for Betaliq of approximately $77 million and a value for Eyenovia of approximately $15 million, assuming zero cash (net of liabilities) at merger closing. The exchange ratio is intended to result in Betaliq equity holders owning approximately 83.7% of the combined company, while Eyenovia equity holders would own approximately 16.3% at the closing of the merger, on a fully diluted basis. Betaliq’s EyeSol technology offers increased bioavailability and a drop size of 10 microliters that can be replicated in the Optejet device. The EyeSol technology is currently used and licensed in FDA-approved topical eye care medications, including MEIBO™ (perfluorohexyloctane ophthalmic solution, Bausch + Lomb) and VEVYE™ (cyclosporine ophthalmic solution 0.1%, Harrow) Eyenovia’s Optejet dispensing platform, in addition to providing similar benefits to those provided by EyeSol, also may enhance the performance of products by making it easier to use and comply with therapy. Importantly, EyeSol is compatible with the Optejet. Eyenovia continues to advance development of the user-filled Optejet and remains on track to file for U.S. regulatory approval in the fourth quarter of this year. The user-filled Optejet is designed to work with a variety of topical ophthalmic liquids, including artificial tears and lens rewetting products. “Following a review of strategic alternatives that we initiated in January, I, along with my fellow Board members, concluded that continuing the negotiation of this potential merger with Betaliq is in the best interests of our company, our team members, patients and shareholders,” stated Michael Rowe, Chief Executive Officer of Eyenovia. “Under a combined new company, if the transaction is completed, our existing products could continue to be marketed to ophthalmologists and optometrists, while the combination of the EyeSol and Optejet technologies has the potential to create a platform that could fundamentally improve how topical eye medications and products are administered. We look forward to working with the Betaliq team on the completion of due diligence and the negotiation and potential execution of a definitive merger agreement in the coming weeks.” Barry Butler, Chief Executive Officer of Betaliq, added, “This proposed merger with Eyenovia represents a significant opportunity in the eyecare space. The inherent synergies of the EyeSol and Optejet technologies could bring innovative new treatment options to patients with glaucoma as well as other ocular diseases. By leveraging the existing pipelines of Eyenovia and Betaliq with multiple opportunities for pipeline expansion through established partnerships, we believe we can build a leading ophthalmic company.” Eyenovia does not intend to discuss or disclose further developments regarding these discussions unless and until such definitive agreement is executed or its Board of Directors has otherwise determined that further disclosure is appropriate or required by law. Chardan is acting as advisor to Eyenovia in connection with the proposed transaction. Raymond James is acting as an advisor to Betaliq. About Betaliq, Inc.Betaliq, Inc., is a clinical stage pharmaceutical company with a therapeutic focus on Glaucoma, founded in 2018 through a collaboration with Novaliq GmbH. Betaliq is developing a global portfolio of topical glaucoma treatments based on the unique EyeSol® topical delivery system developed by Novaliq. This unique water-free eye drop technology offers increased residency time and enhanced bioavailability to provide the needed efficacy, while using less total drug. EyeSol® based eye drops are non-preserved not containing any chemical preservatives, like benzalkonium chloride, that can cause damage to the ocular surface, while being dispensed in a traditional multi-dose eye drop bottle. For more information, please visit Betaliq.com. About Eyenovia, Inc.Eyenovia, Inc. is an ophthalmic technology company developing and commercializing advanced products leveraging its proprietary Optejet topical ophthalmic medication dispensing platform. The Optejet is especially useful in chronic front-of-the-eye diseases due to its ease of use, enhanced safety and tolerability, and potential for superior compliance versus standard eye drops. Together, these benefits may combine to produce better treatment options and outcomes for patients and providers. The company’s current commercial portfolio includes clobetasol propionate ophthalmic suspension, 0.05%, for post-surgical pain and inflammation, and Mydcombi® for mydriasis. For more information, please visit Eyenovia.com. Forward Looking StatementsExcept for historical information, all the statements, expectations and assumptions contained in this press release are forward-looking statements. Forward-looking statements include, but are not limited to, statements that express our intentions, beliefs, expectations, strategies, predictions or any other statements relating to the potential transaction with Betaliq, our future activities or other future events or conditions, including those relating to the completion of due diligence on and a definitive transaction agreement with Betaliq, the estimated market opportunities for our platform technology, the timing for sales growth of our approved products, and the outcome of the process to explore strategic alternatives to maximize shareholder value. These statements are based on current expectations, estimates and projections about our business based, in part, on assumptions made by management. These statements are not guarantees of future performance and involve risks, uncertainties and assumptions that are difficult to predict. Therefore, actual outcomes and results may, and in some cases are likely to, differ materially from what is expressed or forecasted in the forward-looking statements due to numerous factors discussed from time to time in documents which we file with the U.S. Securities and Exchange Commission. In addition, such statements could be affected by risks and uncertainties related to, among other things: the risk that the proposed transaction with Betaliq does not proceed; risks of our clinical trials, including, but not limited to, the potential advantages of our products, and platform technology; the rate and degree of market acceptance and clinical utility of our products; our estimates regarding the potential market opportunity for our products; reliance on third parties to develop and commercialize our products; the ability of us and our partners to timely develop, implement and maintain manufacturing, commercialization and marketing capabilities and strategies for our products; intellectual property risks; changes in legal, regulatory, legislative and geopolitical environments in the markets in which we operate and the impact of these changes on our ability to obtain regulatory approval for our products and product candidates; our competitive position; and our ability to raise additional funds and to make payments on our debt obligations as and when necessary. Any forward-looking statements speak only as of the date on which they are made, and except as may be required under applicable securities laws, Eyenovia does not undertake any obligation to update any forward-looking statements. Eyenovia Contact:Eyenovia, Inc.Norbert LoweVice President, Commercial Operationsnlowe@eyenovia.comBetaliq Contact:Betaliq, Inc.Barry ButlerChief Executive Officerbbutler@blgmgmt.com Eyenovia Investor Contact:Eric RibnerLifeSci Advisors, LLCeric@lifesciadvisors.com(646) 751-4363

Acquisition

07 Mar 2025

·endpts.com

Start of 2025 comes with generic drug price decreases, several major increases, report finds

More generic drug prices decreased than increased in January, a new report from 46Brooklyn found. The report said for every generic drug price increase in January, 1.3 generic drugs saw their price decrease. It’s a trend that 46Brooklyn CEO Antonio Ciaccia called “a largely favorable view.” Even still, three generic drugs saw their prices increase significantly, including the nerve pain drug pregabalin ER 165 mg (26% increase), the nausea drug dronabinol 10 mg (21% increase), and the ADHD drug methylphenidate CD 30 mg (18% increase). For branded drugs, Chiesi’s Ferriprox and Harrow’s Verkazia both increased by more than 20%, although brand-name increases are largely restricted by a Medicaid program that forces pharma companies to pay rebate penalties for drug price increases greater than the rate of inflation. There were 963 net brand price increases in January, according to the report, which was largely in line with the 896 net brand price increases in January 2024 and 986 price increases in January 2023. A previous analysis from Ciaccia’s consultancy firm 3 Axis Advisors anticipated price hikes to more than 1,000 medicines. The report from 46Brooklyn also highlights several key brand-name drug increases that could cost Medicaid significantly more than what they were paying in previous years. For instance, J&J’s Stelara saw a nearly 5% list price increase, which works out to about $1.5 billion in gross prior-year Medicaid expenditures. Several biosimilars to Stelara recently launched at prices as low as 90% off the brand-name price. Chiesi raised the list price of its enzyme replacement therapy Revcovi by 4.5% in January. The report noted that the average gross cost per prescription in Medicaid for this drug is roughly $200,000, which means the list price increase “is anticipated to add roughly $9K in gross cost per prescription. This is the highest added gross cost per Rx for the brand drugs that took price increases in January 2025.” Chiesi did not immediately respond to a request for comment. In addition to the 46Brooklyn report, Sanofi released a report earlier this week on its pricing principles , showing the French company in 2024 spent 36% of its gross sales to payers as rebates. And UCB also released its own pricing report this week showing that 54% of its 2024 gross sales were provided to supply chain stakeholders, including private and public payers as rebates, discounts and fees.

Biosimilar

26 Feb 2025

·ir.madrigalpharma.com

Madrigal Announces New Two-Year Data from the Compensated MASH Cirrhosis Arm of the MAESTRO-NAFLD-1 Trial Demonstrating Potential Benefit of Rezdiffra™ (resmetirom) in Patients with Compensated MASH Cirrhosis

Patients achieved a mean 6.7 kPa reduction in liver stiffness as measured by vibration-controlled transient elastography (VCTE); this represents the largest reduction in liver stiffness reported in a compensated MASH cirrhosis patient population 51% of patients achieved a ≥25% reduction in liver stiffness; a reduction of this magnitude has been associated with reduced progression to end-stage liver disease Company to review topline data during its fourth-quarter and full-year 2024 financial results conference call today, February 26, 2025, at 8 a.m. EST CONSHOHOCKEN, Pa., Feb. 26, 2025 (GLOBE NEWSWIRE) -- Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a biopharmaceutical company focused on delivering novel therapeutics for metabolic dysfunction-associated steatohepatitis (MASH), today reported positive two-year results from the open-label compensated MASH cirrhosis (F4c) arm of the Phase 3 MAESTRO-NAFLD-1 trial of Rezdiffra (resmetirom). Patients treated with Rezdiffra achieved marked reductions in liver stiffness, a surrogate for fibrosis, measured by vibration-controlled transient elastography (VCTE). The Phase 3 MAESTRO-NAFLD-1 trial of Rezdiffra included an open-label active treatment arm of patients with compensated MASH cirrhosis. Madrigal previously reported one-year results from this cohort. Following two years of treatment, 101 patients had VCTE results for analysis. Mean liver stiffness at baseline was 25 kPa and patients achieved a mean 6.7 kPa reduction in liver stiffness at two years (6.1 kPa at one year), which was statistically significant compared to baseline. This represents the largest reduction in liver stiffness reported to date in an F4c MASH population. Mazen Noureddin, M.D., M.H.Sc., Professor of Medicine, Director Houston Research Institute and C.S.O. Summit Clinical Research, stated, “These data demonstrating patients with compensated MASH cirrhosis achieved marked reductions in VCTE are highly encouraging. We use the ‘Baveno rule of 5 kPa’ to stratify risk of liver-related events in patients with MASH, so a mean 6.7 kPa reduction suggests that many patients are moving into a lower risk category. The results are particularly meaningful in light of recently published, multi-center, longitudinal studies1,2 demonstrating VCTE is a strong predictor of clinical outcomes and may be more predictive of clinical outcomes than fibrosis stage assessed by liver biopsy.” In a responder analysis examining ≥25% improvement or worsening of liver stiffness, 51% of patients achieved improvement. An improvement of this magnitude has been associated with reduced progression to end-stage liver disease.1 The safety and tolerability profile of Rezdiffra in the compensated MASH cohort of MAESTRO-NAFLD-1 was consistent with other Rezdiffra clinical trials, with a low rate of discontinuations due to adverse events. Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, “Patients with MASH who progress to cirrhosis face a 42 times higher risk of liver-related mortality, so there is an urgent need to improve care for this underserved population with no approved treatment options. The new two-year data from MAESTRO-NAFLD-1 reinforce our confidence in the ongoing, fully enrolled MAESTRO-NASH OUTCOMES trial of Rezdiffra in patients with compensated MASH cirrhosis. If our OUTCOMES trial is successful, Rezdiffra has the potential to become the first therapy for F2-F4c MASH. It is important to remember that Rezdiffra should not be used for the treatment of patients with compensated MASH cirrhosis until safety and efficacy are established in our Phase 3 trial and the medication receives approval for this indication from regulatory authorities.” Draft FDA guidance recommends that Phase 3 clinical trials in MASH cirrhosis use outcomes as an endpoint, as opposed to biopsy-based surrogate endpoints. This guidance, along with data from the open-label compensated MASH cirrhosis arm of MAESTRO-NAFLD-1, informed the design of the ongoing Phase 3 MAESTRO-NASH OUTCOMES trial. Madrigal plans to present additional results from the compensated MASH cirrhosis arm of the MAESTRO-NAFLD-1 trial at a future medical conference. Rezdiffra is indicated in conjunction with diet and exercise for the treatment of adults with noncirrhotic MASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). Continued approval for this indication may be contingent upon verification and description of clinical benefit in ongoing confirmatory trials. Rezdiffra is not approved for the treatment of patients with MASH cirrhosis. About MASH Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH), is a serious liver disease that can progress to cirrhosis, liver failure, liver cancer, need for liver transplantation, and premature mortality. MASH is expected to become the leading cause of liver transplantation in the U.S. and is already the leading cause of liver transplantation among women. Once patients progress to MASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis), the risk of adverse liver outcomes increases dramatically: these patients have a 10-17 times higher risk of liver-related mortality as compared to patients without fibrosis. Those who progress to cirrhosis face a 42 times higher risk of liver-related mortality, underscoring the need to treat MASH before complications of cirrhosis develop. MASH is also an independent driver of cardiovascular disease, the leading cause of mortality for patients. An estimated 1.5 million patients have been diagnosed with MASH in the U.S., and Madrigal is focused on reaching approximately 315,000 patients with moderate to advanced fibrosis who are under the care of liver specialists. As MASH disease awareness improves and disease prevalence increases, the number of diagnosed patients with MASH with moderate to advanced fibrosis is expected to grow. About Rezdiffra What is Rezdiffra? Rezdiffra is a prescribed medicine used along with diet and exercise to treat adults with nonalcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis), but not with cirrhosis of the liver. It is not known if Rezdiffra is safe and effective in children (under 18 years old). This indication is approved based on improvement of NASH and liver scarring (fibrosis). There are ongoing studies to confirm the clinical benefit of Rezdiffra. Before you take Rezdiffra, tell your healthcare provider about all of your medical conditions, including if you: have any liver problems other than NASH. have gallbladder problems or have been told you have gallbladder problems, including gallstones. are pregnant or plan to become pregnant. It is not known if Rezdiffra will harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if Rezdiffra passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take Rezdiffra. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Rezdiffra and other medicines may affect each other, causing side effects. Rezdiffra may affect the way other medicines work, and other medicines may affect how Rezdiffra works. Especially tell your healthcare provider if you take medicines that contain gemfibrozil to help lower your triglycerides, or cyclosporine to suppress your immune system, because Rezdiffra is not recommended in patients taking these medicines. Tell your healthcare provider if you are taking medicines such as clopidogrel to thin your blood or statin medicines to help lower your cholesterol. Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. What are the possible side effects of Rezdiffra? Rezdiffra may cause serious side effects, including: liver injury (hepatotoxicity). Stop taking Rezdiffra and call your healthcare provider right away if you develop the following signs or symptoms of hepatotoxicity: tiredness, nausea, vomiting, fever, rash, your skin or the white part of your eyes turns yellow (jaundice), pain or tenderness in the upper middle or upper right area of your stomach (abdomen). gallbladder problems. Gallbladder problems such as gallstones, inflammation of the gallbladder, or inflammation of the pancreas from gallstones can occur with NASH and may occur if you take Rezdiffra. Call your healthcare provider right away if you develop any signs or symptoms of these conditions including nausea, vomiting, fever, or pain in your stomach area (abdomen) that is severe and will not go away. The pain may be felt going from your abdomen to your back and the pain may happen with or without vomiting. The most common side effects of Rezdiffra include: diarrhea, nausea, itching, stomach (abdominal) pain, vomiting, dizziness, constipation. These are not all the possible side effects of Rezdiffra. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Madrigal at 1-800-905-0324. Please see the full Prescribing Information, including Patient Information, for Rezdiffra. About the Rezdiffra Phase 3 Program Madrigal is currently conducting multiple Phase 3 clinical trials to evaluate the safety and efficacy of Rezdiffra for the treatment of moderate to advanced fibrosis (consistent with fibrosis stages F2-F3) and compensated MASH cirrhosis (consistent with F4c): Data from the 52-week portion of MAESTRO-NASH, together with data from MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE, Phase 2 and Phase 1 data, including safety parameters, formed the basis for accelerated approval of Rezdiffra for treatment of MASH with moderate to advanced liver fibrosis. About Madrigal Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a biopharmaceutical company focused on delivering novel therapeutics for metabolic dysfunction-associated steatohepatitis (MASH), a liver disease with high unmet medical need. Madrigal’s medication, Rezdiffra (resmetirom), is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of MASH. Rezdiffra is the first and only medication approved by the FDA for the treatment of MASH with moderate to advanced fibrosis (consistent with stages F2 to F3). An ongoing Phase 3 outcomes trial is evaluating Rezdiffra for the treatment of compensated MASH cirrhosis (consistent with stage F4c). For more information, visit www.madrigalpharma.com. Forward Looking Statements This press release includes “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended, including Rezdiffra’s potential ability to treat patients with compensated MASH cirrhosis, Madrigals intentions to present data from the MAESTRO-NAFLD-1 trial at a medical conference and expectations regarding the MAESTRO NASH OUTCOMES trial. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to: the assumptions underlying the forward-looking statements; risks of obtaining and maintaining regulatory approvals, including, but not limited to, potential regulatory delays or rejections; the challenges with the commercial launch of a new product, particularly for a company that does not have commercial experience; our history of operating losses and the possibility that we may never achieve or maintain profitability; risks associated with meeting the objectives of Madrigal’s clinical studies, including, but not limited to Madrigal’s ability to achieve enrollment objectives concerning patient numbers (including an adequate safety database), outcomes objectives and/or timing objectives for Madrigal’s studies; any delays or failures in enrollment, and the occurrence of adverse safety events; risks related to the effects of Rezdiffra’s (resmetirom’s) mechanism of action; enrollment and trial conclusion uncertainties; market demand for and acceptance of Rezdiffra; the potential inability to raise sufficient capital to fund ongoing operations as currently planned or to obtain financing on acceptable terms; the ability to service indebtedness and otherwise comply with debt covenants; outcomes or trends from competitive studies; future topline data timing or results; our ability to prevent and/or mitigate cyber-attacks; the timing and outcomes of clinical studies of Rezdiffra (resmetirom); the uncertainties inherent in clinical testing; uncertainties concerning analyses or assessments outside of a controlled clinical trial; and changes in laws and regulations applicable to our business and our ability to comply with such laws and regulations. Undue reliance should not be placed on forward looking statements, which speak only as of the date they are made. Madrigal undertakes no obligation to update any forward-looking statements to reflect new information, events, or circumstances after the date they are made, or to reflect the occurrence of unanticipated events. Please refer to Madrigal’s submissions filed with the U.S. Securities and Exchange Commission, or SEC, for more detailed information regarding these risks and uncertainties and other factors that may cause actual results to differ materially from those expressed or implied. Madrigal specifically discusses these risks and uncertainties in greater detail in the sections appearing in Part I, Item 1A of its Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on February 28, 2024, and Part II, Item 1A of its Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on October 31, 2024, and as updated from time to time by Madrigal’s other filings with the SEC. 1. Lin H, Lee HW, Yip TC, et al. Vibration-Controlled Transient Elastography Scores to Predict Liver-Related Events in Steatotic Liver Disease. JAMA. 2024;331(15):1287–1297. 2. Gawrieh, S, et al. Increases and Decreases in Liver Stiffness Measurement are independently associated with the risk of liver-related events in NAFLD. Journal of Hepatology. 2024;81(4):600–608. Investor Contact Tina Ventura, IR@madrigalpharma.com Media Contact Christopher Frates, media@madrigalpharma.com Source: Madrigal Pharmaceuticals, Inc.

Clinical ResultPhase 3Accelerated ApprovalPhase 2Drug Approval

100 Deals associated with Cyclosporine

External Link

KEGG	Wiki	ATC	Drug Bank
D00184	Cyclosporine	L04AD01 S01XA18	DB00091

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Conjunctivitis, Allergic	China	Santen Oy	29 Apr 2022
Conjunctivitis, Allergic	China	Santen Pharmaceutical (China) Co. Ltd.	29 Apr 2022
Keratoconjunctivitis	Canada	Santen, Inc.	24 Dec 2018
Vernal Keratoconjunctivitis	European Union	Santen Oy	06 Jul 2018
Vernal Keratoconjunctivitis	Iceland	Santen Oy	06 Jul 2018
Vernal Keratoconjunctivitis	Liechtenstein	Santen Oy	06 Jul 2018
Vernal Keratoconjunctivitis	Norway	Santen Oy	06 Jul 2018
Dry Eye Syndromes	European Union	Santen Oy	19 Mar 2015
Dry Eye Syndromes	Iceland	Santen Oy	19 Mar 2015
Dry Eye Syndromes	Liechtenstein	Santen Oy	19 Mar 2015
Dry Eye Syndromes	Norway	Santen Oy	19 Mar 2015
Keratitis	European Union	Santen Oy	19 Mar 2015
Keratitis	Iceland	Santen Oy	19 Mar 2015
Keratitis	Liechtenstein	Santen Oy	19 Mar 2015
Keratitis	Norway	Santen Oy	19 Mar 2015
Xerophthalmia	Japan	Santen Pharmaceutical Co., Ltd.	11 Oct 2005
Kerato conjunctivitis sicca	United States	AbbVie, Inc.	23 Dec 2002
Ocular inflammation	United States	AbbVie, Inc.	23 Dec 2002
Dermatitis, Atopic	Japan	Novartis Pharmaceuticals KK	14 Mar 2000
Immunosuppression	Japan	Novartis Pharmaceuticals KK	14 Mar 2000

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Eye Diseases	NDA/BLA	Canada	Apotex, Inc.	01 Jan 2025
Liver transplant rejection	Phase 3	United States	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Australia	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Belgium	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Canada	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Denmark	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	France	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Germany	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Hungary	Novartis Pharmaceuticals Corp.	01 Oct 2012
Liver transplant rejection	Phase 3	Italy	Novartis Pharmaceuticals Corp.	01 Oct 2012

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00719888 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Lymphoplasmacytic Lymphoma \| Chronic phase chronic myeloid leukemia ... View more	135	TBI+Fludarabine+Cyclophosphamide (Regimen A: High-dose TBI + Fludarabine + Cyclophosphamide; Single-cord Transplant)	amtpyulwgt = vnbfydrvvv xcfdsqsdcx (sautbaauwr, vluiduyfyw - clebegyokk) View more	-	07 Mar 2025
				TBI+Fludarabine+Cyclophosphamide+Thiotepa (Regimen B: Middle-intensity TBI + Fludarabine + Cyclophosphamide + Thiotepa; Single-cord Transplant)	amtpyulwgt = wmdykgqrxv xcfdsqsdcx (sautbaauwr, oqzqzbkiik - oackixttux) View more
NCT04575779 (Pubmed \| Eur J Drug Metab Pharmacokinet)	Not Applicable	Hematopoietic stem cell transplantation	31	Cyclosporin A 2-h, twice-daily intravenous infusion	dcyiojwclp(zppbvtyenz) = fhgbjklsgj owqskdigeg (vzsbyssrrr )	Positive	27 Nov 2024
				Cyclosporin A 22-h continuous infusion every 24 h	dcyiojwclp(zppbvtyenz) = vftsfonaqz owqskdigeg (vzsbyssrrr )
UEGW2024	Not Applicable	Colitis, Ulcerative	-	Cyclosporine IV	uebukfpjtw(zenmgtqfpe) = AEs occurred in 53.3% of patients, 57.2% of them required a dose reduction or treatment discontinuation. AEs were mainly reversible, but 1 patient died of opportunistic infections (acute tuberculous pneumonia). The occurrence of AEs was not associated with the use of concomitant immunomodulators (p=0.92). Malignancy was observed in 5 patients during follow-up, mainly melanoma and non-melanoma skin cancer. hbbedqgxda (shdrtvykqy ) View more	-	13 Oct 2024
				Oral Cyclosporine
NCT05745701 (CTgov)	Phase 1	Obesity	16	Lotiglipron (Active Comparator: Period 1: Lotiglipron)	ugbmljyync(koekrjuudk) = jiickzpttt yyvxmnouje (hbbhhxefgv, 49) View more	-	23 Sep 2024
				Cyclosporine+Lotiglipron (Experimental: Period 2: Lotiglipron + Cyclosporine)	ugbmljyync(koekrjuudk) = kjsvibdnya yyvxmnouje (hbbhhxefgv, 62) View more
EHA2024	Not Applicable	-	63	Traditional cyclosporine prophylaxis	mddgzggmqv(mbheezumpl) = wmeadelrss ruezasppuy (nynbkdclya ) View more	Positive	14 May 2024
				Delayed oral cyclosporine regimen	mddgzggmqv(mbheezumpl) = kjbwdpfbci ruezasppuy (nynbkdclya ) View more
Thai Lymphoma Study Group (EHA2024)	Not Applicable	Subcutaneous Panniculitis-Like T-Cell Lymphoma serum LDH \| germline HAVCR2 mutations	93	Cyclosporin-based regimen	jiurupauvp(pciecwyndt) = owbfezdsyv mqofeexxml (wesbcujetq ) View more	Positive	14 May 2024
				Chemotherapy	jiurupauvp(pciecwyndt) = yffnukanyh mqofeexxml (wesbcujetq ) View more
NCT00520130 (Pubmed \| Blood Adv) Manual	Phase 1/2	Graft vs Host Disease	83	High-dose Alemtuzumab/Cyclosporine	etoejhtbzd(njkfdbsctp) = pzczlgvxgg xsbzlqbkuk (smimflcsom ) View more	Positive	26 Apr 2024
				Tacrolimus/Methotrexate/Sirolimus	etoejhtbzd(njkfdbsctp) = liimqopopp xsbzlqbkuk (smimflcsom ) View more
NCT03237936 (FAST, CTgov)	Phase 4	Dry Eye Syndromes \| Keratitis	17	1mg/mL ciclosporin	butpcpgrtz = netrysogpx dieyrlkrpj (qgwwtvodzw, yrhgzlqgnc - nshkevknqx) View more	-	18 Apr 2024
ISN WCN2024	Not Applicable	-	148	Cyclosporine	gpoiylvfqw(koapbtcuft) = mlzircuvac mqabmrzzwe (baantylhzy ) View more	Positive	01 Apr 2024
				Tacrolimus	gpoiylvfqw(koapbtcuft) = rcuuoawvwm mqabmrzzwe (baantylhzy ) View more
ISN WCN2024	Not Applicable	-	148	Cyclosporine	yjliblunux(acusxvcdoe) = wuwcednvfi jinltqtgdo (ipawrszhew ) View more	Positive	01 Apr 2024
				Tacrolimus	yjliblunux(acusxvcdoe) = kmnotxoutg jinltqtgdo (ipawrszhew ) View more

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