RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Conditional marketing approval (European Union)

Structure/Sequence

Molecular FormulaC24H30ClN3O5

InChIKeyFKGKZBBDJSKCIS-UHFFFAOYSA-N

CAS Registry732302-99-7

Clinical Trials associated with Givinostat Hydrochloride

NCT06736223

/ RecruitingPhase 1

A Multicentric, Open-label, Non-randomized Study to Evaluate the Pharmacokinetic, Safety and Tolerability of ITF2357 Given as an Oral Single 50 mg Dose in Participants With Chronic Hepatic Impairment Relative to Matched Participants With Normal Hepatic Function

This is a multicentric, open-label, non-randomized study to evaluate the pharmacokinetic, safety and tolerability of ITF2357 in participants with chronic hepatic impairment relative to matched participants with normal hepatic function.

Start Date28 May 2025

Sponsor / Collaborator

Italfarmaco SpA

[+1]

NCT06769633

/ RecruitingPhase 2

A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old

This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
* Planned screening duration: approximately 4 weeks
* Planned Core Treatment duration: approximately 48 weeks
* Planned Extension Treatment duration: approximately 96 weeks
* Planned Follow Up duration: approximately 4 weeks (? 7 days)
* Total duration of study participation: up to 151 weeks (ie, 37-38 months)

Start Date02 Jan 2025

Sponsor / Collaborator

Italfarmaco SpA

[+1]

NCT06093672

/ RecruitingPhase 3

Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

Start Date26 Mar 2024

Sponsor / Collaborator

Italfarmaco SpA

[+1]

100 Clinical Results associated with Givinostat Hydrochloride

100 Translational Medicine associated with Givinostat Hydrochloride

100 Patents (Medical) associated with Givinostat Hydrochloride

255

Literatures (Medical) associated with Givinostat Hydrochloride

01 Oct 2025·BRAIN & DEVELOPMENT

Duchenne muscular dystrophy: Evolving therapeutic strategies and multidimensional evaluation approaches

Review

Author: Komaki, Hirofumi

Duchenne muscular dystrophy (DMD) is a severe X-linked muscular disorder caused by the absence of dystrophin. Standard therapies prolong survival; however, there is an increasing need for disease-modifying approaches and sensitive evaluation tools. This review presents a summary of the recent advances in DMD therapeutic strategies and multidimensional evaluation approaches, emphasizing their clinical applicability and future perspectives. In this study, a comprehensive review of the current literature was conducted focusing on mutation-specific therapies (exon skipping and gene therapy), non-mutation-specific pharmacological interventions (steroids, vamorolone, and histone deacetylase inhibitors), and emerging modalities such as cell-based therapy. The clinical outcome measures used across the disease stages were also examined, ranging from functional motor tests and imaging biomarkers to digital endpoints and quality of life (QOL) indices. Exon skipping and microdystrophin gene therapies have shown promising results in restoring partial dystrophin expression. Vamorolones and givinostat are alternative drugs with improved safety profiles. Time-based motor tests (such as time to sand, 10 m run) and digital biomarkers (Stride Velocity 95th Centile) enable the earlier detection of disease progression. Natural history data, including large registry-based datasets, enhance the interpretation of clinical trials. Patient-reported outcomes and disease-specific QOL measures, such as the DMD-QoL, are increasingly being incorporated to assess meaningful treatment benefits. The therapeutic landscape of DMD is evolving toward combination and personalized approaches. Multidimensional stage-specific evaluations are essential to capture functional changes and their therapeutic effects. Ongoing research on gene-, pharmacological-, and cell-based therapies, coupled with robust outcome assessments, may further improve patient care and QOL.

01 Oct 2025·BIOMEDICINE & PHARMACOTHERAPY

Givinostat inhibits in vitro differentiation of cardiac fibroadipogenic precursors from a mouse model of arrhythmogenic cardiomyopathy

Article

Author: Licandro, Simonetta Andrea ; Calore, Martina ; Fossati, Gianluca ; Forino, Monica ; Romualdi, Chiara ; Braghetta, Paola ; Vencato, Sara ; Romanato, Chiara ; Steinkuhler, Christian ; Rampazzo, Alessandra ; Velle, Angelo ; Facchinello, Nicola ; Vitiello, Libero

Arrhythmogenic cardiomyopathy (ACM) is a rare genetic cardiac disease and one of the leading causes of sudden cardiac death in young individuals and athletes. Current treatments focus on preventing arrhythmias and sudden cardiac death, but no effective therapy targeting disease progression is available. Myocardial replacement with fibro-fatty tissue, a hallmark of ACM, is primarily driven by the activation of cardiac fibroadipogenic precursors (cFAPs). Here, we evaluate the efficacy of a pan inhibitor of histone deacetylases (givinostat) in reducing cFAP proliferation and differentiation. These cells were found to be enriched in the hearts of a transgenic ACM murine model (Tg-hQ), which overexpresses the DSG2 p.Q558* mutation, compared to wild-type (WT) controls. We observed that givinostat reduced the proliferation of both Tg-hQ and WT isolated cFAPS. Additionally, cFAPS were induced to differentiate into adipocytes or fibroblasts and treated with givinostat. The drug treatment led to a reduction in the number of adipocytes under pro-adipogenic conditions. RNA sequencing analysis revealed a significant downregulation of key regulators of adipogenic differentiation and lipid metabolism (Pparg, Cebpa and Adipoq). In parallel, cells cultured under pro-fibrotic conditions showed decreased expression of genes encoding components of the extracellular matrix (Col1a1, Col6a1 and Postn) upon givinostat treatment. Overall, these results support the potential of givinostat in modulating cFAP proliferation and differentiation in vitro, warranting further in vivo studies to assess its impact on fibro-fatty tissue replacement in ACM.

01 Sep 2025·Molecular Therapy-Nucleic Acids

Epigenetic small molecule screening identifies a new HDACi compound for ameliorating Duchenne muscular dystrophy

Article

Author: Farr, Gist H ; Louie, Ke'ale W ; Maenza, Alyssa ; Paquette, Alison G ; Paguio, Alison ; Hasegawa, Eva H ; Henry, Clarissa A ; Ignacz, Amanda C ; Maves, Lisa

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease. There are currently few effective therapies to treat the disease, although many approaches are being pursued. Certain histone deacetylase inhibitors (HDACi) have been shown to ameliorate DMD phenotypes in mouse and zebrafish models, and the HDACi givinostat has recently gained FDA approval for DMD. Our goal was to identify additional HDACi, or other classes of epigenetic small molecules, that are beneficial for DMD. Using an established animal model for DMD, the zebrafish dmd mutant strain sapje, we screened a library of over 800 epigenetic small molecules. Our screening identified a new HDACi, SR-4370, that ameliorated dmd mutant zebrafish skeletal muscle degeneration, as well as additional HDACi that have previously been shown to improve dmd zebrafish. We find that a single early treatment of HDACi can ameliorate the muscle phenotype and increase lifespan in dmd zebrafish. Furthermore, we find that HDACi treatments that improve dmd muscle also cause increased histone acetylation in zebrafish larvae. Our results add to the growing evidence that HDACi are promising candidates for treating DMD. Our study also provides further support for the effectiveness of small molecule screening in dmd zebrafish.

News (Medical) associated with Givinostat Hydrochloride

07 Oct 2025

·www.globenewswire.com

New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress

Long-term treatment with givinostat in boys with Duchenne muscular dystrophy continues to show a favourable safety and tolerability profile Consistent efficacy demonstrated across age groups MILAN, Italy, October 07, 2025 – Italfarmaco S.p.A. announced today that additional data on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 30th annual International Congress of the World Muscle Society (WMS) held October 7-11 in Vienna, Austria. Poster presentations highlight the long-term safety of givinostat in patients with DMD, explore efficacy by age in a post hoc analysis of the pivotal EPIDYS study, and provide a detailed characterisation of gastrointestinal adverse events observed in this Phase 3 study. “These presentations provide new insights into the long-term safety, efficacy by age, and tolerability of givinostat in DMD,” said Scott Baver, PhD, VP, Head of Global Medical Affairs, Rare Diseases. “We are proud to share this data with global leaders advancing research, treatment, and patient care in DMD.” These data from studies sponsored by Italfarmaco presented at the 2025 WMS Congress include: #651P Long-Term Safety of Givinostat in Patients With Duchenne Muscular Dystrophy: Results From an Open-Label Extension StudyThe ongoing long-term study of givinostat in boys with DMD continues to provide insights into the safety and tolerability of the treatment, with results consistent with previous shorter-duration studies. Some patients have now been taking givinostat for more than 8 years in total, across several studies and extension phases. #684VP Efficacy of Givinostat by Age (6–7 and >7 Years): A Post Hoc Analysis of EPIDYSBaseline assessments in the study provided key insights into physical function in boys with DMD across different age groups. #688VP Characterizing Gastrointestinal Adverse Events of Interest From a Phase 3 Study of Givinostat in Patients With Duchenne Muscular DystrophyThe study continues to monitor gastrointestinal events in participants, with most reported events being mild to moderate in severity and resolving without requiring discontinuation of treatment. These findings provide important context for understanding the overall safety and tolerability of long-term therapy. Abstracts have been published in the Sept 2025 supplement of Neuromuscular Disorders here. About Givinostat Givinostat was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease.1,2 Givinostat is approved as a treatment of DMD in multiple regions, including the US, EU and UK. About ITALFARMACO Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. Media enquiries:Anja Heuer / Adolfo Luna | +49 (0) 151 106 199 05 | italfarmaco@trophic.euOther enquiries:Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com References: Comi G, Bertini E, Vita G, et al. S22.008: Development of the histone deacetylases inhibitor Givinostat in Duchenne Muscular Dystrophy. Poster. Neurology. 2018;90(15 (Supplement)).Licandro SA, Crippa L, Pomarico R, et al. The pan HDAC inhibitor Givinostat improves muscle function and histological parameters in two Duchenne muscular dystrophy murine models expressing different haplotypes of the LTBP4 gene. Skelet Muscle. 2021;11(1):19. https://doi.org/10.1186/s13395-021-00273-6. Attachment New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 World Muscle Society Congress

Phase 3Drug ApprovalClinical Result

26 Sep 2025

·www.prnewswire.com

New Data Presented on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2025 Neuromuscular Study Group Annual Scientific Meeting

Additional analyses from the givinostat clinical development program review observations on weight-based flexible dosing, characterization of thrombocytopenia, correlations between pharmacodynamic markers and functional outcomes, and respiratory function CONCORD, Mass., Sept. 26, 2025 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that results on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 2025 Neuromuscular Study Group (NMSG) Annual Scientific Meeting held September 26-28 in Stresa, Italy. Poster presentations include analyses of functional outcomes based on weight-based flexible dosing in the pivotal Phase 3 EPIDYS trial, further characterization of thrombocytopenia, the link between vastus lateralis fat fraction (VLFF) measurement and efficacy outcomes, and observations on respiratory function. "These presentations reflect our focus on generating data that support clinicians in assessing the benefit-risk profile and full potential of givinostat, including the opportunity to consider flexible dosing strategies that may help to optimize tolerability and efficacy for some patients," said Scott Baver, Ph.D., VP, Head of Medical Affairs. "We are pleased that new insights on givinostat are being shared with global leaders in DMD research, treatment, and patient advocacy at the 2025 NMSG Annual Scientific Meeting." These data from studies sponsored by Italfarmaco presented at the 2025 NMSG Annual Scientific Meeting include: #1213 Givinostat Weight-based Flexible Dosing: Rationale and Efficacy at the Different Doses This poster includes an analysis of data on weight-based flexible dosing from the multicenter, randomized, double-blind, placebo-controlled, Phase 3 EPIDYS clinical trial. In the EPIDYS study, patients with DMD received weight-based givinostat dosing plus standard of care (i.e., corticosteroids) at two levels, dose A or B, depending on study protocol. Doses were adjusted (A to B or B to C) based on predefined rules related to tolerability to achieve the highest tolerated dose for each patient. This analysis assesses mean change from baseline in 4-stair climb (4SC) time and total North Star Ambulatory Assessment (NSAA) scores to evaluate efficacy based on the final administered dose at week 72. #1207 Characterizing Thrombocytopenia in Patients with Duchenne Muscular Dystrophy Treated with Givinostat: Results from the Phase 3 EPIDYS Trial Thrombocytopenia is a known adverse event of givinostat. This analysis further characterizes the risk and severity of thrombocytopenia or decreased platelet count based on an assessment of data from patients treated with givinostat in the Phase 3 EPIDYS study. #1211 Vastus Lateralis Fat Fraction is Associated with Functional Efficacy Endpoints in Patients with Duchenne Muscular Dystrophy Treated with Givinostat This poster reports findings from a post-hoc analysis assessing whether changes from baseline in VLFF correlate with measures of disease progression in patients with DMD. The analysis examines VLFF alongside efficacy endpoints observed at the end of the study (month 18), including 4SC, time to rise, 6-minute walking test, and NSAA. #1208 Givinostat Effect on Respiratory Function in Duchenne Muscular Dystrophy Before and After Ambulation Loss: Results from EPIDYS, LTSE, and PRO-DMD-01 Studies This poster, which includes findings previously presented at the 2025 MDA Clinical and Scientific Conference, reports observations on respiratory function in patients who experienced loss of ambulation during follow-up. The analysis indirectly compares forced vital capacity (FVC) %-predicted trajectories from patients treated with givinostat and corticosteroids in the Phase 3 EPIDYS study and its ongoing open-label extension with FVC %-predicted trajectories from patients in a natural history study of DMD disease progression who received corticosteroids only. For additional poster details, see abstract #1207, #1208, #1211, and #1213 published here. "The data presented at NMSG highlight important insights into the patient experience, ranging from the impact of flexible dosing to how thrombocytopenia was observed and characterized in the clinical trial setting," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, Division of Pediatric Neurology at the University of Arkansas for Medical Sciences and Arkansas Children's Hospital. "Together, these findings contribute to a deeper understanding of givinostat's use in the treatment of DMD and may help inform clinical decision-making." ITF Therapeutics is not a sponsor of the 2025 NMSG Annual Scientific Meeting. About DUVYZAT® (givinostat) DUVYZAT was discovered through Italfarmaco's research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). DUVYZAT is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease. For more information visit . About ITF Therapeutics LLC ITF Therapeutics was launched in January 2024 as the U.S. affiliate of Italfarmaco focused on the development and commercialization of products to treat rare diseases. Building on a legacy grounded in collaboration and innovation, ITF Therapeutics strives to partner with leaders from the patient advocacy and treatment communities to ensure that our programs reflect and support their unique needs and goals. The establishment of ITF Therapeutics reflects Italfarmaco's goal to build a world-class team of experts who share a passion to make a positive impact for rare disease communities. For more information visit . About Italfarmaco Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production, and commercialization with proven success in many therapeutic areas including immuno-oncology, gynecology, neurology, cardiovascular disease, and rare diseases. Italfarmaco's rare disease unit includes programs in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis, and polycythemia vera. For more information visit . INDICATION DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. IMPORTANT SAFETY INFORMATION Warnings and Precautions Hematological Changes: DUVYZAT can cause dose-related thrombocytopenia and other signs of myelosuppression. Monitor blood count every 2 weeks for the first 2 months, at month 3, and every 3 months thereafter. Modify the dosage for confirmed thrombocytopenia. Discontinuation may be needed if abnormalities worsen. Increased Triglycerides: DUVYZAT can cause elevations in triglycerides. Monitor triglycerides at 1 month, 3 months, 6 months, and then every 6 months thereafter. Modify the dosage if fasting triglycerides are verified >300 mg/dL. Treatment with DUVYZAT should be discontinued if triglycerides remain elevated despite adequate dietary intervention and dosage adjustment. Gastrointestinal Disturbances: Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain were common adverse reactions in DUVYZAT clinical trials. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Modify the dosage of DUVYZAT in patients with moderate or severe diarrhea and discontinue treatment if significant symptoms persist. QTc Prolongation: DUVYZAT can cause prolongation of the QTc interval. Avoid use of DUVYZAT in patients who are at an increased risk for ventricular arrhythmias (including torsades de pointes), such as those with congenital long QT syndrome, coronary artery disease, electrolyte disturbance or in patients taking concomitant medicinal products known to cause QT prolongation. Obtain ECGs prior to initiating treatment with DUVYZAT in patients with underlying cardiac disease or in patients who are taking concomitant medications that cause QT prolongation. Adverse Reactions The most common adverse reactions reported in >5% of patients treated with DUVYZAT are diarrhea (37%), abdominal pain (34%), thrombocytopenia (33%), nausea/vomiting (32%), hypertriglyceridemia (23%), pyrexia (13%), myalgia (9%), rash (9%), arthralgia (8%), fatigue (8%), constipation (7%), and decreased appetite (7%). Drug Interactions Closely monitor when DUVYZAT is used in combination with an oral CYP3A4 sensitive substrate or a sensitive substrate of the OCT2 transporter, for which a small change in substrate plasma concentrations may lead to serious toxicities. Avoid concomitant use with other drugs that prolong the QTc interval; monitor ECG if concomitant use cannot be avoided. If concomitant use cannot be avoided, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold DUVYZAT if the QTc interval is >500 ms or the change from baseline is >60 ms. To report SUSPECTED ADVERSE REACTIONS, contact ITF Therapeutics LLC at 1-833-582-4312 or FDA at 1-800-FDA-1088 or . Please see full Prescribing Information for additional safety information. DUVYZAT is a registered trademark of Italfarmaco S.p.A. U.S.A. Media inquiries: Kaitlin Gallagher | +1 212 253 8881 | [email protected] Other inquiries: Martha McKennitt | Communications Lead U.S.A. | [email protected] Global Media inquiries: Anja Heuer / Adolfo Luna | +49 (0)151 106 199 05 | [email protected] Other inquiries: Samantha Parker | Patient Advocacy and Communications Lead | [email protected] C-DUV-US-0583 09/2025 SOURCE ITF Therapeutics LLC WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3

23 Sep 2025

·www.globenewswire.com

Santhera Announces Half Year 2025 Financial Results and Provides Corporate Update

Ad hoc announcement pursuant to Art. 53 LR A conference call will be held on September 23, 2025, at 14:00 CEST / 13:00 BST / 08:00 EDT. Details are at the end of this news release Pratteln, Switzerland, September 23, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the Company’s financial results for the six months ended June 30, 2025, reports on progress with AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) and provides updates on its corporate and financing initiatives. Highlights Total revenue increased 70% to CHF 24 million (H1 2024: CHF 14.1 million) driven by strong sales growth in launched markets in addition to growing royalty and product supply revenuesProduct sales increased 76% to CHF 11.6 million (H1 2024: CHF 6.6 million), led by Germany and Austria with first contributions from the UK following the Q2 launchRoyalties from licensing partners in the U.S. and China of CHF 5.4 million (H1 2024: CHF 0.9 million), already >25% above full year 2024; further acceleration expected in H2 2025Global sales (including partners) of AGAMREE exceed USD 100 million on a four consecutive quarter basis, ahead of expectations, triggering a USD 25 million sales milestone payment due, reflected in cost of sales (COS)Operating expenses (excluding non-cash compensation) of CHF 25 million (H1 2024: CHF 24.1 million), in line with full year CHF 50-55 million guidanceOperating loss of CHF 35.4 million (H1 2024: loss of CHF 17.7 million)Global rollout advancing with new distribution agreements (post period-end) across five Gulf Cooperation Council (GCC) countries, India, and TürkiyeCash and cash equivalents of CHF 18.4 million at June 30, 2025 (Dec 31, 2024: CHF 40.9 million); strengthened by approximately CHF 20 million of growth capital in September 2025Cash-flow break-even guidance maintained for mid-2026Full year 2025 revenue is expected to exceed the previous guidance range of CHF 65-70 million Dario Eklund, CEO of Santhera said: “It has been an exciting period for the business, as the global rollout of AGAMREE has continued at pace. Our partner Catalyst is seeing particularly strong demand in the US, with increasing expectations in China through Sperogenix. Sales in Germany and Austria continue to exceed expectations, and the UK is contributing positively following the Q2 launch. We look forward to the second half of the year, with the publication of our GUARDIAN study on the horizon and the rollout of AGAMREE continuing through direct launches in Europe and via distribution and licensing partners globally. All these efforts are aimed at enabling more patients access to this important treatment around the world.” BUSINESS AND CORPORATE UPDATE(Including post period events) Direct market sales growing strongly with approximately 40% of steroid using DMD patients in Germany now treated with AGAMREE. Austria becomes the first country to have in excess of 50% market share for DMD patients taking corticosteroids Pricing and reimbursement discussions advancing in Spain, Italy and the Nordic regions, with launches expected from Q4 2025 into Q1 2026US partner Catalyst Pharmaceuticals, Inc. (“Catalyst”) posted strong AGAMREE H1 sales of USD 49.4 million, on track to reach its guidance for FY 2025 sales of USD 100-110 million, which would trigger an additional milestone to Santhera of USD 12.5 millionChinese partner Sperogenix Therapeutics Ltd. (“Sperogenix”) commences commercial rollout on a non-reimbursed basis. Forecasted demand in China for 2025 and 2026 has increasedContinued rollout of AGAMREE worldwide with distribution agreements signed for five Gulf Corporation Council (GCC) countries, India and Türkiye post period endCatherine Isted joined Santhera as CFO in February and Dr. Melanie Rolli joined the Santhera Board in May Successful launches drive own market sales in EuropeFollowing successful 2024 launches in Germany and Austria, patient numbers continue to grow, with approximately 450-500 patients having initiated AGAMREE and many of these patients having been on the therapy for well over a year. In Germany, use is broadening beyond the initial pediatric cohort as neurologists increasingly (re)start or switch older DMD patients to AGAMREE. Combination therapy with givinostat is also rising in the country. Roughly 40% of corticosteroid-using DMD patients in Germany have been treated to date with AGAMREE. In Austria, strong uptake has continued into 2025, making it the first country where more than half of steroid-using Duchenne patients are being treated with AGAMREE. In the United Kingdom, after positive final NICE guidance in January 2025, AGAMREE became available nationwide from April. Updated UK guidelines on “Corticosteroids in Duchenne muscular dystrophy” (April 2025) favor daily regimens and recognize AGAMREE as equally effective with fewer side effects than traditional corticosteroids. In mid-Q3, Santhera introduced a home-delivery program to streamline access and reduce administrative burden on centers. Early feedback has been positive and is expected to support ordering momentum into Q4. Advancing the rollout of AGAMREE across EuropeA key focus of the business is the continued rollout across Europe, and the Company is advancing multiple national reimbursement submissions and pricing negotiations across the continent. AGAMREE has been available in Spain through a paid, named-patient program since mid-2024, and the Company has been working to secure full approval and reimbursement so all eligible patients can access the medicine. Santhera’s proposal is scheduled for discussion at the upcoming October CIMP meeting. Subject to a positive outcome, the launch into regional and hospital formularies is expected to begin in Q4 2025 and to continue into 2026 as regional rollout progresses. In Portugal, an early access plan is about to start, with pricing and reimbursement discussions running in parallel. Across the Nordics – Sweden, Denmark, Finland, and Norway – pricing and reimbursement filings are progressing alongside pre-marketing activities conducted by local teams. First sales are anticipated between Q4 2025 and Q1 2026 as country launches commence. In Italy, following discussions with the Italian Medicines Agency (AIFA), the Company has decided to update the reimbursement dossier with long-term GUARDIAN study data once available. Approval is anticipated in late Q1 2026, assuming good progress with regulators over the coming quarter. In the meantime, an early access program is being rolled out with the aim of covering the majority of Italian expert centers, supported by strong engagement with key opinion leaders and patient advocacy groups. Reimbursement discussions continue in the Benelux region, with launches expected during 2026. In France, pricing negotiations with CEPS are ongoing. As previously disclosed, Santhera expects to include additional GUARDIAN data and potentially other supportive evidence in the dossier to achieve the appropriate ASMR rating and broader positioning. The Company will update the market on expected launch timing as discussions progress. In Switzerland, pre-marketing activities have begun, with marketing authorization expected in H1 2026, with fully reimbursed launch targeted for the second half of 2026. Strong US partner sales with Catalyst continuesSanthera’s U.S. licensing partner, Catalyst, continued to report strong growth, delivering AGAMREE sales of USD 49.4 million in the first half of 2025, driven by accelerated physician adoption. This strong and sustained uptake underscores AGAMREE’s positioning as a new standard of care for DMD. Based on current projections, Catalyst has guided USD 100-110 million in 2025 sales. Upon achieving at least USD 100 million, Santhera would receive a USD 12.5 million milestone. The robust U.S. performance is also increasing Santhera’s inventory requirements and contributing to higher supply sales and royalties. Sperogenix non-reimbursed commercial rollout commencesIn China, partner Sperogenix commenced a non-reimbursed commercial rollout of AGAMREE in September 2025, in addition to the earlier started Early Access Program. Uptake has been encouraging, with more than 250 patients treated to date. Priorities for the second half of 2025 are to expand sales in the non-reimbursed market. In anticipation of increased demand in 2025-2026, Santhera is bringing forward inventory plans to provide product to this market. Geographical expansion continues successfullyAcross additional territories, Santhera is broadening access through distribution partnerships. In 2024, the Company signed an agreement with GENESIS Pharma SA covering 20 Central and Eastern European markets and established regional distribution agreements for Israel and Qatar. During 2025, Santhera signed further agreements in five Gulf Cooperation Council countries, Türkiye, and India, and continues to engage in various discussions to expand its global footprint and broaden patient access to AGAMREE. New AGAMREE data to be presented in early Q4 2025 Santhera continues to generate additional long-term clinical outcomes in DMD patients with up to seven years on AGAMREE and looks forward to reporting long-term effectiveness and safety results from the GUARDIAN study in early Q4 2025. In parallel, real-world evidence abstracts from both younger and older patient cohorts in Germany – where AGAMREE has been available the longest – have been submitted for presentation at the World Muscle Society Congress in Vienna, October 7–11, 2025. Pipeline development and business development initiatives As previously guided, Santhera does not plan near-term investment in additional indication expansions for AGAMREE, though it retains the option to leverage partner-run studies at a future date. Resources will remain focused on maximizing the DMD opportunity. In addition, Santhera remains actively engaged in looking to expand its product portfolio and leveraging its infrastructure through licensing, distribution agreements, and potential M&A transactions, with updates expected in 2026. Executive and Board changesDuring the first half of 2025, there were leadership changes at both the executive and Board levels. In February, Catherine Isted joined as Chief Financial Officer, succeeding Andrew Smith. She brings more than 25 years of life-sciences leadership and was formerly CFO of BenevolentAI, a Euronext-listed AI-driven drug discovery Company. In May 2025, shareholders elected Dr. Melanie Rolli to the Board at the AGM, replacing Dr. Otto Schwarz, who did not stand for re-election. She is a seasoned biopharma executive with over 20 years of international experience and is currently CEO of Helsinn Healthcare. Financing (further detail given in the financials section)In September 2025, Santhera secured approximately CHF 20 million in additional growth capital to meet increased product demand from partners and to support the acceleration of global launches. The financing comprised USD 13 million from a royalty monetization with R-Bridge, on terms designed to preserve long-term value while providing near-term growth capital. In addition, Highbridge increased its commitment with a CHF 10 million upsizing of its convertible bond, extending maturity and enhancing financial flexibility. Guidance and outlookBased on continued strong growth in both direct and partner markets, FY 2025 revenue is now expected to exceed the previous guidance range of CHF 65-70 million. At the same time the Company has maintained disciplined cost control, with operating expenses (excluding non-cash share-based compensation) for 2025, and on a constant-portfolio basis going forward, expected to remain within the previously guided range of CHF 50-55 million. Santhera reiterates its 2028 revenue guidance of EUR 150 million (including royalties but excluding milestones) and maintains its 2030 guidance of more than EUR 150 million in direct market sales alone (excluding all distributor and licensing market royalties and milestones). FINANCIAL PERFORMANCE Financial highlights (including post period events) Total revenue increased 70% to CHF 24.0 million (H1 2024: CHF 14.1 million)Product sales increased 76% to CHF 11.6 million (H1 2024: CHF 6.6 million)Santhera recognized CHF 12.4 million (H1 2024: CHF 7.6 million) from partners in China and North America, reflecting a 63% increase in royalties, milestones and product supplyGlobal sales (including partners) of AGAMREE exceed USD 100 million on a four consecutive quarter basis, triggering a USD 25 million sales milestone payment reflected in COSOperating expenses of CHF 27.3 million (H1 2024: CHF 26.7million)Operating loss of CHF -35.4 million (H1 2024: loss of CHF -17.7 million). Excluding the USD 25 million (CHF 20.3 million) milestone, operating loss was reduced by CHF 2.6 million Cash and cash equivalents at June 30, 2025 of CHF 18.4 million (December 31, 2024: CHF 40.9 million) In September, the Company secured CHF 20 million in royalty and convertible bond financingCash-flow break-even guidance maintained for mid-2026 Net RevenueIn the first half year 2025, Santhera reported revenue from contracts with customers of CHF 24.0 million (H1 2024: CHF 14.1 million) driven by strong sales growth in launched markets in addition to growing royalty and product supply revenue. Net sales amounted to CHF 11.6 million following the continued success of AGAMREE in Germany and Austria with first contributions from the UK following the Q2 launch (H1 2024: CHF 6.6 million). Royalties and net sales to licensing partners increased substantially to CHF 5.4 million and CHF 5.7 million (H1 2024: CHF 0.9 million and CHF 1.2 million), driven by U.S. growth. Milestones in the period reached CHF 0.9 million compared to CHF 5.4 million in H1 2024, reflecting a Chinese milestone received in the prior year. Cost of salesCost of sales increased to CHF 32.1 million (H1 2024: CHF 5.2 million). In addition to increased direct and indirect costs due to growing sales, this figure also reflects the milestone payment of USD 25 million (CHF 20.3 million) to ReveraGen and R-Bridge for the achievement of an AGAMREE sales milestone, which is agreed to be paid in line with cash flow generation. Cost of sales for the six months also included non-cash intangible amortization of CHF 2.5 million (H1 2024: CHF 2.5 million) and royalties payable of CHF 3.7 million (H1 2024: CHF 1.0 million). Operating expenses and resultOperating expenses of CHF 27.3 million (H1 2024: CHF 26.7 million) were consistent year-over-year. These relate to employee expenses as the Company expands activities to support the commercialization of AGAMREE, offset by decreases in development expenses due to non-recurring longer-term study expenses closing in the prior year. Development expenses amounted to CHF 11.7 million (H1 2024: CHF 13.8 million). The decrease of -15% was driven by the completion of longer-term studies and CMC (chemistry, manufacturing, and controls) development activities coming to a close in the prior year. Marketing and sales expenses were CHF 6.8 million (H1 2024: CHF 4.7 million). The increase of 45% was driven by the Company's expansion to support the commercialization of AGAMREE in Europe. General and administrative expenses amounted to CHF 8.8 million (H1 2024: CHF 8.3 million), with additional activities focused on supporting commercial growth. The operating result amounted to a CHF -35.4 million loss (H1 2024: CHF -17.7 million loss). Financial income and expenses Financial income amounted to CHF 8.1 million (H1 2024: CHF 8.6 million). The decrease was predominantly related to a lower gain in fair value of financial instruments and a decrease in interest receivable. Financial expenses were CHF 11.4 million (H1 2024: CHF 6.0 million), primarily due to an increase in realized and unrealized foreign exchange losses. In summary, this resulted in a net financial expense of CHF -3.3 million, compared with a net income of CHF 2.6 million for H1 2024. Net resultThe net result in H1 2025 was a CHF -38.8 million loss, compared to a loss of CHF -15.3 million in H1 2024 mainly driven by the one-time USD 25 million (CHF 20.3 million) milestone payable and the financial expenses. Cash balance and cash flows As of June 30, 2025, the Company had cash and cash equivalents of CHF 18.4 million compared to CHF 40.9 million as of December 31, 2024. This represents a decrease of CHF -22.5 million (H1 2024: decrease of CHF -13.9 million). Net cash flow used in operating activities amounted to CHF -20.6 million (H1 2024: net cash outflow of CHF -15.3 million). Net cash flow used in financing activities was CHF -1.6 million (H1 2024: CHF -0.4 million). Assets and liabilitiesIntangible assets decreased by CHF -2.5 million to CHF 66.4 million, reflecting amortization in the period.Total assets decreased by CHF -21.7 million to CHF 130.8 million. This is a result of a CHF -22.5 million reduction in cash and decrease of trade receivables, offset by an increase in inventory to support sales growth following product commercialization. Total liabilities increased by CHF 8.5 million to CHF 133.3 million driven by the 20.3 million milestone payment owed to ReveraGen and R-Bridge for achievement of sales milestones of AGAMREE, offset by a decrease in the fair value of financial liabilities. Shareholders’ equityTotal consolidated equity as of June 30, 2025, was CHF -2.6 million compared to CHF 27.7 million as of December 31, 2024. Financing activitiesThis morning Santhera announced that it has secured approximately CHF 20 million in additional growth capital to meet increased product demand from partners and to support the acceleration of global launches. R-Bridge royalty monetization agreement (USD 13 million) Santhera has secured a royalty monetization with existing investor R-Bridge. Under the terms of the agreement, R-Bridge will receive 25% of net royalties on AGAMREE from Catalyst (North America) and Sperogenix (China). Upon closing, R-Bridge will pay Santhera USD 13 million (CHF 10.3 million), net of certain fees, upfront. This is in addition to an existing agreement under which R-Bridge is entitled to 75% of future royalty income from these licenses. As with the prior arrangement, payments to R-Bridge are capped; once the agreed ceiling or duration is met, North American & China royalties revert to Santhera. Santhera retains buy-back rights over the royalty stream. Highbridge convertible bond extension (CHF 10 million) Under the agreement, Highbridge will provide an additional CHF 10 million via a new convertible note. The instrument will also exchange, at parity, the existing CHF 7 million convertible bond that was previously scheduled to mature on 30 September. The new convertible bond will have a three-year maturity, with a conversion price set at a 10% premium to the closing share price on the date of this announcement. In addition, the company will issue Highbridge approximately 110,000 shares as consideration for Highbridge agreeing to increased flexibility in relation to the CHF 35 million 4-year term loan signed in August 2024. Half Year ReportThe Santhera Half Year Report 2025 (English only) is available for download on the Company’s website at www.santhera.com/financial-reports. Analyst BriefingSanthera’s management team will be hosting a briefing for analysts and investors via a webcast at 14:00 CEST (08:00 ET) on 23 September 2025. Register here: https://www.investormeetcompany.com/santhera-pharmaceuticals-holding-ag/register-investor A recording of the webcast and the results presentation will be made available on the website following the event. For further information please contact: Catherine Isted, Chief Financial Officer: IR@santhera.com ICR Healthcare: Santhera@icrhealthcare.com About SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Commission (EC), in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA), in China by the National Medical Products Administration (NMPA) and Hong Kong by the Department of Health (DoH). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China and certain countries in Southeast Asia to Sperogenix Therapeutics. For further information, please visit www.santhera.com. AGAMREE® is a trademark of Santhera Pharmaceuticals. About AGAMREE® (vamorolone) AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [1-4]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [1-4]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [1, 4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has no restriction of growth [5] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [6]. ▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. References:[1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.[6] Hasham et al., MDA 2022 Poster presentation. Link. Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. # # # Interim Consolidated Balance Sheet In CHF thousands Jun 30, 2025 Dec 31, 2024 (unaudited) (audited) Assets Tangible assets 2,311 2,571 Intangible assets 66,439 68,946 Financial assets long-term 249 245 Noncurrent deferred loss on financial instruments 2,967 4,913 Noncurrent assets 71,966 76,675 Current deferred loss on financial instruments 2,739 3,103 Prepaid expenses 437 373 Inventories 26,030 17,527 Trade and other receivables 11,199 13,885 Cash and cash equivalents 18,397 40,925 Current assets 58,802 75,813 Total assets 130,768 152,488 Equity and liabilities Share capital 1,358 1,343 Capital reserves and share premium 647,812 644,410 Accumulated losses (653,525) (614,693) Employee benefit reserve 2,116 (3,025) Treasury shares (63) (65) Translation differences (261) (272) Total equity (2,563) 27,698 Noncurrent payables 7,745 - Noncurrent term loans 32,123 31,729 Noncurrent royalty purchase agreements 27,294 33,165 Noncurrent derivative financial instruments 1,917 2,216 Noncurrent lease liabilities 1,662 1,940 Noncurrent contract liabilities 1,850 1,925 Pension liabilities 2,959 7,672 Noncurrent liabilities 75,550 78,647 Trade and other payables 24,441 9,224 Accrued expenses 16,583 19,345 Income tax payable 37 144 Current royalty purchase agreements 5,856 3,810 Current lease liabilities 592 553 Current convertible bonds 6,845 6,398 Current contract liabilities 89 56 Current derivative financial instruments 1,408 2,323 Current warrant financial instruments 1,930 4,290 Current liabilities 57,781 46,143 Total liabilities 133,331 124,790 Total equity and liabilities 130,768 152,488 Interim Consolidated Income Statement In CHF thousands (except per share data) Six months ended June 30, 2025 2024 (unaudited) (unaudited) Net sales 11,577 6,563 Revenue from outlicensing transactions 6,548 6,262 Net sales to licensing partners 5,888 1,289 Revenue from contracts with customers 24,013 14,114 Cost of sales (32,123) (5,215) Of which amortization intangible assets (2,491) (2,487) Of which royalties payable (3,728) (962) Of which milestones payable (20,490) - Other operating income - 67 Development (11,693) (13,771) Marketing and sales (6,804) (4,660) General and administrative (8,837) (8,265) Other operating expenses - (11) Operating expenses (27,334) (26,707) Operating result (35,444) (17,741) Financial income 8,116 8,638 Financial expenses (11,429) (5,986) Result before taxes (38,757) (15,089) Income taxes (75) (174) Net result (38,832) (15,263) Basic and diluted net result per share (in CHF) (3.04) (1.35) Interim Consolidated Statement of Cash Flows In CHF thousands Six months ended June 30, 2025 2024 (unaudited) (unaudited) Result before taxes (38,757) (15,089) Depreciation and impairment of tangible assets 331 286 Amortization and impairment of intangible assets 2,507 2,487 Share-based compensation 2,335 2,590 Change in fair value of financial instruments, net (4,052) (4,263) Change in pension liabilities 428 - Reversal of current provisions - (106) Income taxes paid - (91) Change in contract liabilities (42) - Change in net working capital 14,211 (1,887) Financial result net of change in fair value of financial instruments 4,408 1,042 Interest received 355 480 Interest paid (2,322) (732) Net cash flow from/(used in) operating activities (20,598) (15,283) Investments in tangible assets (71) - Investments in intangible assets - (72) Change in financial assets long-term - 82 Net cash flow from/(used in) investing activities (71) 10 Proceeds from exercise of equity rights 103 - Repayments of royalty purchase liability (1,430) - Payment of lease liabilities (290) (358) Net cash flow from/(used in) financing activities (1,617) (358) Effects of exchange rate changes on cash and cash equivalents (242) 1,752 Net increase/(decrease) in cash and cash equivalents (22,528) (13,879) Cash and cash equivalents at January 1 40,925 30,370 Cash and cash equivalents at June 30 18,397 16,491 Attachment 250923 Santhera H1 Interim results_Final_updated

Executive ChangeLicense out/inFinancial StatementDrug Approval

100 Deals associated with Givinostat Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
-	Givinostat Hydrochloride	-	DB12645

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	United States	Italfarmaco SpA	21 Mar 2024

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Polycythemia Vera	Phase 3	United States	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Austria	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Bulgaria	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Croatia	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	France	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Germany	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Hungary	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Ireland	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Israel	Italfarmaco SpA	26 Mar 2024
Polycythemia Vera	Phase 3	Italy	Italfarmaco SpA	26 Mar 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03373968 (EPIDYS, Pubmed \| Ann Clin Transl Neurol)	Phase 3	Muscular Dystrophy, Duchenne	194	Givinostat oral suspension + Givinostat oral suspension	ellftjqqgw(abhbvodhdn) = vfydswtvrw dfesfbycrn (ekrnpoqsdi )	Positive	19 Aug 2025
NCT02851797 (EPIDYS, GlobeNewswire) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat	htvntpqanp(eieacqgxdb) = Post hoc analysis from the completed Phase 3 EPIDYS trial showed less decline in cardiac function throughout the study, compared to those who received placebo. ydsqqzucbk (fusxlixzsx ) View more	Positive	11 Jun 2025
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	56	Givinostat in addition to steroids	tghjoqxxpb(jadgavkrat) = kodmjyyhuz aqiffphkdg (wizgffhhnl, 2.2) View more	Positive	16 Mar 2025
				Steroids only (PRO-DMD-01)	tghjoqxxpb(xkbbarazyf) = yhwickfpmw jkfdjuwjgf (mmkpupyzip, 2.2) View more
NCT03373968 (MDA2025)	Phase 2/3	Muscular Dystrophy, Duchenne	194	Givinostat	mulmivjbie(epgquffazw) = vozjlhoqce ufvsporldb (sorxjgdsxp ) View more	Positive	16 Mar 2025
				Givinostat (Prior Placebo)	mulmivjbie(epgquffazw) = yeqdnkmskl ufvsporldb (sorxjgdsxp ) View more
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	179	Givinostat (off-target group)	oqooipcwqu(yiuxdcdzoq) = gavtwkznma wpkzpsvtst (bptclyakww, 7.9)	Positive	16 Mar 2025
				Placebo (off-target group)	oqooipcwqu(yiuxdcdzoq) = avyldgwyri wpkzpsvtst (bptclyakww, 13.3)
NCT02851797 (EPIDYS, MDA2025)	Phase 3	Muscular Dystrophy, Duchenne	284	Givinostat	rbemhqjjlt(fsrsvvhiam) = pcvmphaxon xulkqhvnjg (owoamowamo ) View more	Positive	16 Mar 2025
				Steroids	rbemhqjjlt(fsrsvvhiam) = prsnyzfvhy xulkqhvnjg (owoamowamo ) View more
NCT05860114 (CTgov)	Phase 1	Medication interactions	8	Givinostat (Givinostat)	dlqtgautur(ztylcxqrxn) = bboyshkegt pecsmjsomr (uuyfgcpiem, 25.8) View more	-	24 Jan 2025
				Givinostat (ITF2374)	dlqtgautur(ztylcxqrxn) = ogquhkcdnb pecsmjsomr (uuyfgcpiem, 45.7) View more
NCT03238235 (CTgov)	Phase 2	Becker Muscular Dystrophy	51	Givinostat (Givinostat)	qrpebudbdx(echrqdcces) = bnsyqyovaw zfmeemslgn (nyqfrfflij, geezpzqaom - crfhsehtbl) View more	-	18 Nov 2024
				Placebo (Placebo)	qrpebudbdx(echrqdcces) = jmrbtloumt zfmeemslgn (nyqfrfflij, doxqewjynv - davbrmhmtu) View more
NCT02851797 (EPIDYS, Literature \| Pubmed \| Lancet Neurol) Manual	Phase 3	Muscular Dystrophy, Duchenne	120	givinostat	kivvtydstr(qbwbazuasp) = zwzludpcmy brujgorbxx (mbgdmwwsce, 1.17 - 1.37)	Positive	01 Apr 2024
				Placebo	kivvtydstr(qbwbazuasp) = jkfwrzexus brujgorbxx (mbgdmwwsce, 1.17 - 1.37)
NCT02851797 (FDA_CDER) Manual	Phase 3	Muscular Dystrophy, Duchenne	179	DUVYZAT	zlhhmlopqj(tecyaedfly) = gnuzllsiei yvzsgcohyb (krdglcabui )	Positive	21 Mar 2024

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