RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (China), Breakthrough Therapy (China), Paediatric investigation plan (European Union), Promising Innovative Medicine (United Kingdom), Priority Review (Canada)

Structure/Sequence

Molecular FormulaC22H28O4

InChIKeyZYTXTXAMMDTYDQ-DGEXFFLYSA-N

CAS Registry13209-41-1

Clinical Trials associated with Vamorolone

CTIS2024-512828-12-00

/ RecruitingPhase 4

An open-label study to collect safety and effectiveness information on long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy who have completed prior studies with vamorolone (The GUARDIAN Study) - SNT-IV-VAM-011

Start Date11 Dec 2024

Sponsor / Collaborator

Santhera Pharmaceuticals Holding AG

NCT06713135

/ RecruitingPhase 4

An Open-label Study to Collect Safety and Effectiveness Information on Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Who Have Completed Prior Studies With Vamorolone

This study aims to assess safety and effectivness of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamorolone.

Start Date10 Nov 2024

Sponsor / Collaborator-

NCT06689527

/ CompletedPhase 1

An Open-label, Single-arm Study to Evaluate the CYP3A4 Induction Potential of Vamorolone on the Pharmacokinetics of Midazolam (a Sensitive CYP3A4 Probe) in Healthy Subjects.

An open study to learn whether the study medication (vamorolone) is able to affect certain processes in the liver participating in the breakdown of drugs. This will be done by measuring the blood levels of midazolam alone and in combination with vamorolone in healthy participants.

Start Date13 Aug 2024

Sponsor / Collaborator-

100 Clinical Results associated with Vamorolone

100 Translational Medicine associated with Vamorolone

100 Patents (Medical) associated with Vamorolone

Literatures (Medical) associated with Vamorolone

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·JOURNAL OF PHARMACY TECHNOLOGY

Vamorolone Versus Traditional Glucocorticoids in Duchenne Muscular Dystrophy: A Review and Meta-Analysis of Efficacy and Safety

Review

Author: Jahanzeb, Ahmed ; Javed, Dawood ; Jajja, Faran Ahmed ; Javed, Abdullah

Objective: This meta-analysis evaluates the efficacy and safety of vamorolone, a dissociative glucocorticoid, compared with traditional glucocorticoids in treating Duchenne muscular dystrophy (DMD), aiming to assess its potential as a safer alternative with comparable therapeutic benefits. Data Sources: A systematic search was conducted in PubMed (MEDLINE), Embase, and Web of Science from inception till June 2024. Search terms included (Vamorolone) AND (Corticosteroids OR Glucocorticoids OR Prednisone) AND (Duchenne Muscular Dystrophy OR related terms). Only randomized controlled trials (RCTs) and observational studies were included, with no language restrictions. Study Selection and Data Extraction: From 276 identified reports, 135 were screened after duplicate removal, and 30 underwent full-text review. Five studies (2 RCTs, 2 observational, 1 nonrandomized trial) involving 370 patients were included. Two reviewers independently extracted data on efficacy (TTSTAND, TTRW, TTCLIMB velocities) and safety (height percentile, body mass index-z score, osteocalcin, P1NP) using Microsoft Excel, resolving discrepancies through consensus. Data Synthesis: Vamorolone significantly improved TTSTAND (MD = −0.03, 95% confidence interval [CI] = −0.06 to −0.004, P = .02), TTRW (MD = −0.11, 95% CI = −0.22 to −0.01, P = .04), and TTCLIMB velocities (MD = −0.04, 95% CI = −0.08 to −0.003, P = .03). Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31–26.25, P = .001) and bone biomarkers (osteocalcin: MD = 15.68, P < .00001; P1NP: MD = 158.34, P < .00001), with no significant body mass index-z score difference. Conclusions: Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management.

01 Oct 2025·Neurology-Genetics

Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy

Article

Author: Hoffman, Eric P. ; Clemens, Paula R. ; Johnson, Linda ; Dang, Utkarsh J. ; Tobin, Rebecca A. ; James, Meredith K.

Background and Objectives:

Understanding the reliability of outcomes used in clinical care and trials is important to delineate intervention-induced change from random variability. Reliability, sensitivity, and clinical meaningfulness of change are all key aspects of choosing the most appropriate outcome measure for a clinical trial. Common outcome measures to monitor progression and treatment effect in Duchenne muscular dystrophy (DMD) include measures of strength (myometry) and motor function tests: stand from supine velocity (STANDV), North Star Ambulatory Assessment (NSAA), 6-minute walk distance (6MWD), 10-m run/walk (RWV), and 4-stair climb velocity (CLIMBV). Our objective was to present test-retest reliability of common outcome measures using pretreatment measurements and to provide insights into missing measurements.

Methods:

Data were used on outcome measures in steroid-naïve, 4 to <7 years participants with DMD in 2 multisite, multicountry vamorolone trials (VBP15-002 [n = 48 participants] and VBP15-004 [n = 121 participants]). Bland-Altman analysis, the coefficient of variation, and the intraclass correlation coefficient (ICC) were used.

Results:

Based on the ICC, NSAA, RWV, and CLIMBV have good reliability, whereas the 6MWD and STANDV have moderate reliability. Two different techniques were used for myometry in VBP15-002 (CINRG Quantitative Measurement System [CQMS]) and VBP15-004 (MicroFET2 handheld digital muscle dynamometer [HHD]). Reliability of myometry ranged from poor to moderate, and CQMS did not show improved reliability over HHD. The 6MWD and myometry showed the highest rates of missingness. Because of similar age ranges and harmonized outcomes, our findings were compared with the FOR-DMD cohort. Myometry and CLIMBV were not in FOR-DMD; reliability findings were concordant for the 4 outcomes in common. For most outcomes, we found reduced reliability compared with previous studies in older age groups.

Discussion:

Reliable outcome measures, appropriate for the age of patients, are key to improving power in clinical trials, leading to fewer participants needed and reducing patient and site burden. We found NSAA, RWV, and CLIMBV to be the most reliable. This should be considered in the context of sensitivity to drug effect, and clinical meaningfulness of changes observed: all 5 motor outcomes were sensitive to drug effect of vamorolone and prednisone, whereas myometry was not.

182

News (Medical) associated with Vamorolone

29 Aug 2025

·www.globenewswire.com

Santhera Enters into Agreement with Ikris Pharma Network for the Distribution of AGAMREE® (Vamorolone) in India

Pratteln, Switzerland, 29 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Ikris Pharma Network (Ikris) to manage the distribution of AGAMREE® (vamorolone) in India, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older. The agreement between Santhera and Ikris has a 5-year term and Santhera will receive a percentage of net sales, in line with previous distribution agreements. Sales are expected to begin in Q4 2025 on a named patient basis. Today’s agreement follows recent distribution deals secured in Turkey and a range of Gulf Cooperation Council countries. Santhera continues to press ahead with the global rollout of AGAMREE. Dario Eklund, Chief Executive Officer of Santhera, said: “We are pleased to partner with Ikris Pharma Network in India, which is part of our wider strategy to increase global access to AGAMREE for patients with DMD. Ikris has been actively working in the area of DMD and their expertise and dedication to ensuring rare disease drugs reach patients makes them an ideal partner in the region.” Praveen Sikri, Founder & CEO of Ikris Pharma Network said: “This collaboration with Santhera aligns with our focus of addressing unmet medical needs in the rare disease community. We look forward to leveraging our distribution network to help DMD patients in India access this life-changing treatment.” 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. 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 Ikris Pharma Network Ikris Pharma Network is India's biggest named patient supply company, specializing in providing access to rare disease medications. With established expertise in logistics, regulatory compliance, and patient access programs, Ikris has supported physicians and patients in obtaining therapies that are not locally available. The company has been actively working in the area of Duchenne muscular dystrophy (DMD), providing assistance to patients and healthcare professionals in India. Partnership Impact:Through its authorization with Santhera Pharmaceuticals, Ikris Pharma Network will facilitate access to AGAMREE® (vamorolone) for eligible patients with Duchenne Muscular Dystrophy in India. The collaboration aims to provide a regulatory-compliant pathway for access and strengthen existing efforts in patient support and disease awareness in the country. Duchenne Muscular Dystrophy (DMD):DMD is a rare genetic disorder caused by mutations in the dystrophin gene, leading to progressive muscle weakness that usually begins in early childhood. Most patients lose ambulation in adolescence. While there is no cure, available treatments such as corticosteroids and supportive care can help manage symptoms. Expanding access to therapies like AGAMREE is intended to support the ongoing care of patients and families living with DMD in India. For further information please contact: SantheraCatherine Isted, Chief Financial Officer: IR@santhera.com ICR Healthcare: Santhera@icrhealthcare.com Ikris Pharma NetworkPraveen Sikri, Chief Executive Officer: info@ikrispharmanetwork.com Bharat Sikri, Global Project Manager: Raredisease@ikrispharmanetwork.com 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. # # # Attachment 250829 Santhera India Ikris Distribution Agreement -FINAL

License out/inDrug Approval

21 Aug 2025

·www.globenewswire.com

Santhera Secures Agreement with Uniphar for the Distribution of AGAMREE® (Vamorolone) in five GCC (Gulf Cooperation Council) Countries

19 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE® (vamorolone) in the United Arab Emirates, the Kingdom of Saudi Arabia, Kuwait, Oman and Bahrain, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older. Sales are expected to begin on a named patient basis in Q1 2026, with broader commercial sales anticipated in late 2026 following market authorization and pricing agreement. Santhera will receive a percentage of net sales as payment, consistent with the Company's previous distribution agreements. This agreement will help ensure that patients across these countries have timely access to this innovative therapy. Dario Eklund, Chief Executive Officer of Santhera, said: “We are excited to partner with Uniphar for the distribution of AGAMREE in GCC (Gulf Cooperation Council) countries. Uniphar’s strong regional presence, combined with its proven expertise in early and expanded access programs, will help address the significant unmet need for boys and men living with DMD in these countries. This agreement represents another important step in our global expansion strategy and demonstrates our unwavering commitment to making effective rare disease treatments accessible to patients worldwide.” Santhera remains committed to the global rollout of AGAMREE, which already includes multiple regions across North America, Europe and Asia, bringing this important therapy to patients with DMD who are in urgent need of treatment. Brian O’Shaughnessy, Chief Commercial Officer of Uniphar said: “We are proud to collaborate with Santhera to bring AGAMREE to patients in GCC (Gulf Cooperation Council) countries. Our regional knowledge, commercial experience, and dedication to improving access to innovative therapies align perfectly with Santhera’s mission. Together, we aim to ensure that patients living with DMD receive the treatment they need without delay.” 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]. 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. Santhera 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 Medicines Agency (EMA), 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. Headquartered in Dublin, Ireland, Uniphar is a trusted global partner to pharma, medtech and biotech companies, working to improve patient access to medicines around the world. Uniphar’s team of experts harness the capabilities, infrastructure and expertise of a diversified pharmaceutical service provider with more than 57 years’ experience building success stories with 200+ multinational client across 180 countries worldwide. Uniphar works across the entire pharmaceutical product lifecycle, providing integrated solutions that remove barriers to market entry, increase patient access, and optimize brand value. With a strong presence in Europe, North America, APAC, and MENA, Uniphar offers specialist services spanning early access programs, medical affairs, commercialisation, logistics, global sourcing and distribution. Its end-to-end model enables pharmaceutical companies to scale rapidly in complex markets while maintaining compliance and operational excellence. The Company's vision is to improve patient access to pharmaco-medical products and treatments by enhancing connectivity between manufacturers and healthcare professionals and their patients, representing a strong combination of scale, growth, and profitability. The content above comes from the network. if any infringement, please contact us to modify.

License out/inDrug ApprovalClinical Result

15 Aug 2025

·www.globenewswire.com

Santhera extends Highbridge convertible bond to 30th September 2025

Ad hoc announcement pursuant to Art. 53 LRA Pratteln, Switzerland, 15 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that by mutual agreement the Company and Highbridge Capital Management, LLC (Highbridge) have decided to extend the maturity date of the existing CHF 7 million private convertible bond, that has a strike price of CHF 10.00, to 30th September 2025. 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 Medicines Agency (EMA), 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. For further information please contact: SantheraCatherine Isted, Chief Financial Officer: IR@santhera.com ICR Healthcare Santhera@icrhealthcare.com 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. # # # Attachment 250815 Highbridge Convertible Bond_ENGLISH_FINAL

License out/inDrug Approval

100 Deals associated with Vamorolone

External Link

KEGG	Wiki	ATC	Drug Bank
D11000	Vamorolone	-	DB15114

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	United States	Catalyst Pharmaceuticals, Inc.	26 Oct 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Becker Muscular Dystrophy	Phase 2	United States	ReveraGen BioPharma, Inc.	07 Jul 2022
Becker Muscular Dystrophy	Phase 2	United States	Santhera Pharmaceuticals Holding AG	07 Jul 2022
Becker Muscular Dystrophy	Phase 2	Italy	ReveraGen BioPharma, Inc.	07 Jul 2022
Becker Muscular Dystrophy	Phase 2	Italy	Santhera Pharmaceuticals Holding AG	07 Jul 2022
Pediatric Ulcerative Colitis	Phase 2	-	ReveraGen BioPharma, Inc.	01 Sep 2020
Cystic Fibrosis	Preclinical	United States	ReveraGen BioPharma, Inc.	25 May 2016
Multiple Sclerosis	Preclinical	United States	ReveraGen BioPharma, Inc.	25 May 2016
Inflammatory Bowel Diseases	Preclinical	United States	ReveraGen BioPharma, Inc.	04 May 2016
Rheumatoid Arthritis	Preclinical	United States	ReveraGen BioPharma, Inc.	22 Apr 2016
Asthma	Preclinical	United States	ReveraGen BioPharma, Inc.	10 Apr 2016

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06649409 (CTgov)	Phase 1	-	30	Fludrocortisone+Eplerenone	wtzfpnnrvt(cdjwmqgulo) = bwqksbasco gxiykpyyfd (alcilvycum, 1.290309) View more	-	11 Sep 2025
LIONHEART (GlobeNewswire) Manual	Not Applicable	-	30	AGAMREE	pcjbbdonkk(kodretqvwm) = It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. siepvefpda (bvvvgnsrkg )	Positive	01 Oct 2024
				placebo
NCT03439670 (VISION-DMD \| VBP15-004, Literature) Manual	Phase 2	Muscular Dystrophy, Duchenne	121	vamorolone 2 mg/kg/d	cdnmkcaowt(wpbwdlvhit) = bmrsnjkyul llsenhdvkm (fukjeibeyh, 0.0135) View more	Positive	12 Mar 2024
				vamorolone 6 mg/kg/d	cdnmkcaowt(wpbwdlvhit) = esdwvdaede llsenhdvkm (fukjeibeyh, 0.0138) View more
NCT03439670 (VISION-DMD, MDA2024) Manual	Phase 2	Muscular Dystrophy, Duchenne	118	Vamorolone 2 mg/kg/day	ksckekhlzt(daptikpowv) = vtqytnzbrc dnprhbavcu (zzarknezkc ) View more	Negative	03 Mar 2024
				Prednisone 0.75 mg/kg/day	ksckekhlzt(daptikpowv) = yhqrxwibkl dnprhbavcu (zzarknezkc ) View more
NCT03439670 (FDA) Manual	Phase 2	Muscular Dystrophy, Duchenne	90	Placebo	yqmlfwvkmo(jbdutvmxmy) = qztwbzelqb xjpmbfalrt (thkftvacng ) View more	Positive	26 Oct 2023
				AGAMREE 2 mg/kg/d	yqmlfwvkmo(jbdutvmxmy) = rswncaufeh xjpmbfalrt (thkftvacng ) View more
NCT03439670 (Pubmed \| JAMA Neurol)	Phase 2	Muscular Dystrophy, Duchenne	133	Vamorolone 2 mg/kg per day	fojkvnjbqb(rxtbwwijji) = Bone turnover markers declined with prednisone but not with vamorolone alostqqjbl (zhwpfqhggs ) View more	Positive	29 Aug 2022
NCT03439670 (VISION-DMD \| VBP15-004 \| VBP15-004, CTgov)	Phase 2	Muscular Dystrophy, Duchenne	121	Vamorolone (Treatment Group 1)	wyxsxycdmf(ggpenjxdtu) = pfxzhkblwa tbntopmzhj (cksuhwogqb, 0.0628) View more	-	13 Jul 2022
				Vamorolone (Treatment Group 2)	wyxsxycdmf(ggpenjxdtu) = nqqeogefsh tbntopmzhj (cksuhwogqb, 0.0666) View more
NCT03439670 (VBP15-004, MDA2022)	Phase 2	Muscular Dystrophy, Duchenne	121	Vamorolone 60 mg/kg/day	upxzrkqziy(cfaymfeihs) = rpzbxeuwpw wwfapzsgng (yknfvssdin )	Positive	13 Mar 2022
NCT03038399 (Pubmed \| JAMA Netw Open)	Phase 2	Muscular Dystrophy, Duchenne	46	Vamorolone	ltcpnprkzi(akqghaatur) = wprzjgyfmh xvbjvrgsyj (fjdukowipm, -4.48 to 4.04) View more	Positive	04 Jan 2022
NCT03038399 (VBP15-LTE, CTgov)	Phase 2	Muscular Dystrophy, Duchenne	46	Vamorolone (Dose Level Group 1)	zdwzoztekg = aahpndmbwh srrgflzsxw (vpauzsogem, rokegitjvv - tadefaajxl) View more	-	20 May 2021
				Vamorolone (Dose Level Group 2)	zdwzoztekg = argqttsnoz srrgflzsxw (vpauzsogem, jknkfemkgw - pppjvftgdg) View more

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