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

Santhera Pharmaceuticals Holding AG

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

Santhera Pharmaceuticals Holding AG

100 Clinical Results associated with Vamorolone

100 Translational Medicine associated with Vamorolone

100 Patents (Medical) associated with Vamorolone

Literatures (Medical) associated with Vamorolone

01 Apr 2025·Neurology-Genetics

Association of DMD Gene Variant Classes With Motor Outcomes in a Drug Registration Clinical Trial Setting

Article

Author: Hoffman, Eric P. ; Dang, Utkarsh J. ; Fang, Yuan ; Illei, Katherine I. ; Clemens, Paula

Background and Objectives:

Duchenne muscular dystrophy (DMD) is caused by pathogenic variants of the DMD gene, leading to the loss of dystrophin. Clinical variability in DMD can complicate interpretation of interventional data in clinical trials. One source of clinical variability is allelic heterogeneity (different pathogenic variants with different effects on dystrophin protein expression). We sought to determine whether gene variant classes in clinical trial participants potentially affect clinical trial data interpretation.

Methods:

We analyzed 186 vamorolone trial participants with DMD (VBP15-002/003; VBP15-004) who were 4 to <7 years old and steroid-naïve at baseline. We stratified participants into gene variant classes by either variant location in the gene affecting different gene promoters (5' [Dp427-only] vs 3' [Dp427+other isoforms]) or residual dystrophin levels (null vs possible non-null [5' gene variants, exon 44 skippable, splice site]). We evaluated associations with baseline motor outcomes and treatment response (prednisone and vamorolone).

Results:

Participants with variants in ex63 and downstream (null for Dp427+Dp140+Dp71 protein isoforms) showed poorer baseline motor outcomes for time to stand from supine velocity than those with variants in ex1-44 (Dp427 only). No significant baseline differences were found between likely null and possible non-null variants. Participants with only Dp427 involvement showed significantly better treatment response for the 6-minute walk distance. Most of the comparisons of baseline motor function and treatment response were similar between variant classes.

Discussion:

The large variation in baseline motor function in young, steroid-naïve patients with DMD is only minimally explained by different gene variant classes. While there is strong literature support for 3' variants leading to a more severe motor and cognitive DMD phenotype, we found this variant class under-represented in our clinical trials. This suggests that they may fail inclusion criteria (failure to follow commands; poor motor function). Subgroup analyses in DMD clinical trials at a young age range based on gene variant class may not reveal significant differences and would be relatively noninformative.

04 Mar 2025·Expert Review of Neurotherapeutics

The latest developments in synthetic approaches to Duchenne muscular dystrophy

Review

Author: Johnson, Lucy M. ; Pulskamp, Tariq G. ; Berlau, Daniel J.

INTRODUCTION:

Duchenne muscular dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene, leading to an almost complete absence of dystrophin, which is essential for muscle cell structure and function. This resulting muscle deterioration and fibrosis, eventually causes respiratory failure and cardiomyopathy. While there is currently no cure, existing therapies aim to prolong survival and alleviate symptoms.

AREAS COVERED:

This paper reviews current and emerging therapies for DMD, focusing on their safety and efficacy. Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options. Additionally, future therapies, including gene therapy, stem cell treatments, and anti-fibrotic agents, show promise for clinical application.

EXPERT OPINION:

Advancements in DMD treatments have expanded patient options. While gene therapy offers potential for correcting the genetic defect and alleviating symptoms, corticosteroids remain the most cost-effective and well-researched treatment. This is partly due to the lack of compelling long-term safety and efficacy data for gene therapies. The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients.

04 Feb 2025·ACS Omega

Quantitative Structure–Property Relationship Modeling with the Prediction of Physicochemical Properties of Some Novel Duchenne Muscular Dystrophy Drugs

Article

Author: Santiago, Roy ; K, Jyothish

Duchenne muscular dystrophy is a critical, progressively worsening, and ultimately deadly illness characterized by the deterioration of skeletal muscles, respiratory failure, and heart disease. The pharmaceutical industries are persistently innovating drug design processes to address the rise of infections and effectively treat emerging syndromes or genetically based disorders with the help of quantitative structure-property relationship models. These models are mathematical tools that correlate molecular structures with their physicochemical properties through structural characteristics. Different models can be generated based on the various structural features of the compounds, and topological indices are one such significant structural feature generated from the molecular graph and are key tools used in these models. This study focuses on creating quantitative structure-property relationship models using degree-based topological indices, which are highly effective in quantitative structure-property relationship analysis to explore the diverse physicochemical properties of Duchenne muscular dystrophy drugs with the prediction of properties of a recently approved drug givinostat. Furthermore, the drug discovery and development activities can be accelerated using the developed models to forecast the possible productiveness of novel Duchenne muscular dystrophy treatment drugs.

169

News (Medical) associated with Vamorolone

08 Apr 2025

·www.prnewswire.com

New Drug Submission for AGAMREE® (vamorolone) Accepted for Priority Review by Health Canada for the Treatment of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a rare, debilitating and life-shortening neuromuscular disease1 Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone follows approvals in the US in 2023, and the European Union in early 2024 This New Drug Submission reflects Kye Pharmaceuticals' commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients MISSISSAUGA, ON, April 8, 2025 /PRNewswire/ - Kye Pharmaceuticals, Inc. ("Kye") announced today it has submitted a New Drug Submission (NDS) to Health Canada for the regulatory review and approval of AGAMREE® (vamorolone). If approved, AGAMREE® would be the first and only therapy approved by Health Canada with an indication for the treatment of Duchenne muscular dystrophy (DMD). Health Canada has granted AGAMREE® a priority review and marketing authorization could be granted before the end of 2025. News of the AGAMREE® priority review was welcomed by national patient organizations. "Defeat Duchenne Canada is thrilled that Health Canada has accepted AGAMREE® for priority review. There are currently no approved treatments available in Canada for young people living with Duchenne muscular dystrophy. We urge Health Canada to approve AGAMREE® to ensure all Canadians for whom this treatment would be beneficial, can access it as soon as possible." Lisa McCoy, Chief Executive Officer, Defeat Duchenne. "Muscular Dystrophy Canada (MDC) is pleased that Health Canada has granted Priority Review for AGAMREE® (vamorolone). While corticosteroids have been a key treatment for decades, they are not currently approved for DMD, and their significant side effects have been a challenge for the Duchenne muscular dystrophy community. If approved, this promising treatment will provide options for the Duchenne muscular dystrophy community." Stacey Lintern, Chief Executive Officer, MDC. The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms.2 Duchenne muscular dystrophy (DMD) is a type of muscular dystrophy that causes weakness and a wasting of the muscle due to the absence of a protein called dystrophin. If the body is lacking dystrophin, muscle cells become damaged and movements that are considered natural such as getting up from the floor and walking become challenging. Boys with DMD lose the ability to walk in early teens and have cardiac and respiratory difficulties. Bone thinning and scoliosis are also common for those with Duchenne.1 "Health Canada granting vamorolone a priority review is a promising first step for patients with Duchenne having access to approved medicines in Canada," said Dr. Jean Mah, Director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary. "If approved, vamorolone will provide Duchenne families access to an approved therapy that will positively impact their disease. We would like to thank the patients and families from DMD communities across Canada who have contributed to advancing vamorolone research." This initial step in the review process is a culmination of years of research, including clinical trials of vamorolone conducted in Canada.3 Kye commends the courage of patients and families who participated in these studies, including five clinical trial sites in Canada, generating the data to support the approval of this novel treatment in countries around the world. "This submission is an important milestone for DMD treatments in Canada and we are grateful for our close partnership with Catalyst Pharmaceuticals," stated John McKendry, President and CEO at Kye Pharmaceuticals. "We look forward to working collaboratively with the DMD community and Health Canada throughout the priority review process." About AGAMREE® (vamorolone) AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid with dissociative properties in maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE® to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. 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. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.4 About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, Catalyst is focused on in-licensing, commercializing, and developing innovative therapies. Guided by its deep commitment to patient care, Catalyst prioritizes accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's fastest-growing companies. For more information, please visit Catalyst's website at . References: National Institute of Neurological Disorders and Stroke. Muscular dystrophy: hope through research. Available at [last accessed March 2021] Hodgkinson et al. The Canadian Neuromuscular Disease Registry 2010–2019, Journal of Neuromuscular Diseases 8 (2021) 53–6 Worsfold, N. (2020, June 10). Defeatduchenne.ca. Available at Defeat Duchenne Canada: $1M Grant Towards Clinical Trial – Defeat Duchenne Canada Dang UJ et al. (2024) Neurology 024;102:e208112. doi.org/10.1212/WNL.0000000000208112 SOURCE Kye Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalPriority Review

08 Apr 2025

·ir.catalystpharma.com

Catalyst Pharmaceuticals Announces Health Canada’s Acceptance of AGAMREE® New Drug Submission with Priority Review for Sub-Licensee Kye Pharmaceuticals

CORAL GABLES, Fla., April 08, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today reported that its sub-licensee in Canada, Kye Pharmaceuticals, Inc. (Kye), has announced that Health Canada has accepted the New Drug Submission (NDS) for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), for review. The submission has been granted Priority Review, expediting the regulatory process with the potential for marketing authorization before the end of 2025. If approved, AGAMREE would be the first and only treatment option indicated for patients diagnosed with DMD in Canada. “We are pleased that Health Canada has accepted the New Drug Submission for AGAMREE with a Priority Review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada,” stated Richard J. Daly, President and CEO of Catalyst. “In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.” AGAMREE (40 mg/mL oral suspension) received U.S. Food and Drug Administration (FDA) approval on October 26, 2023, for the treatment of DMD in patients aged two years and older and became commercially available in the U.S. on March 13, 2024. Kye currently markets FIRDAPSE®, Catalyst’s flagship product for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), in Canada. LEMS is a rare neuromuscular disorder characterized by debilitating and progressive muscle weakness and fatigue. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects males, with symptoms typically appearing in early childhood, around ages three to five. DMD is caused by mutations in the gene that encodes dystrophin, a protein that plays a crucial role in maintaining the structure and function of muscle fibers. Without dystrophin, muscle cells become fragile and easily damaged, leading to progressive muscle degeneration. Symptoms of DMD usually begin with difficulty in walking, frequent falls, and muscle weakness, particularly in the legs and pelvis. As the disease progresses, individuals may experience difficulty standing, climbing stairs, and eventually, complete loss of mobility. Other complications can include respiratory and cardiac issues due to muscle weakness. About AGAMREE® (vamorolone) AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid designed to achieve dissociative properties while maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. 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. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit www.kyepharma.com About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking Statements This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Health Canada will approve AGAMREE either in the anticipated timeline or at all, (ii) if approved, whether Kye can successfully commercialize AGAMREE in Canada, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2024 and its subsequent filings with the U.S. Securities and Exchange Commission (“SEC”), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. Source: Catalyst Pharmaceuticals, Inc. Source: Catalyst Pharmaceuticals, Inc.

Drug ApprovalPriority ReviewClinical Result

27 Mar 2025

·www.globenewswire.com

Santhera proposes Dr. Melanie Rolli as new member of Board of Directors

Ad hoc announcement pursuant to Art. 53 LR Pratteln, Switzerland, March 27, 2025 – Santhera Pharmaceuticals (SIX: SANN) proposes to elect Dr. Melanie Rolli as an Independent Director at the upcoming Annual General Meeting to be held on May 20, 2025. Current Board member Dr. Otto Schwarz will not stand for re-election. Thomas Meier, Chairman of the Board of Directors of Santhera, said: "We are delighted Dr. Rolli has agreed to be nominated to join the Board; where we believe she would bring extensive strategic and operational expertise across drug development and commercialization. Her experience would be invaluable as we continue the rollout of AGAMREE® in new markets and evaluate opportunities to further expand our portfolio. Dr. Rolli’s nomination follows the recent appointment of Ms. Isted as CFO, reinforcing our commitment to strengthening and diversifying the Company’s Board and leadership team. On behalf of the Board and management I would additionally like to thank Dr. Schwarz for his great dedication and strong and valuable contribution to Santhera’s strategic development during his time as Board member. His guidance and expertise in the early phases of the AGAMREE® product launch was very helpful in setting the path for the Company’s commercial success.” Dr. Melanie Rolli is a seasoned biopharma executive with over 20 years international experience. She has held several executive and non-executive roles at various pharmaceutical and biotechnology companies and has a proven track record of product development and global commercialization. She is currently the Group CEO of Helsinn Healthcare, a global pharmaceutical company commercializing a diverse portfolio of products in cancer supportive care and onco-dermatology. Prior to this she was CEO of AELIX Therapeutics and PIQUR Therapeutics, where she led the strategic refocus and completed successful fundraising campaigns. She also spent 14 years at Novartis, holding increasingly senior roles in global development, medical affairs and patient safety. 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: public-relations@santhera.com orCatherine Isted, Chief Financial OfficerCatherine.Isted@Sanathera.com Disclaimer / Forward-looking 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. # # #

Executive ChangeLicense 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.

to view more data

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

Developing

10 top R&D records.

to view more data

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

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


LIONHEART (GlobeNewswire) Manual	Not Applicable	-	30	AGAMREE	uwveiwjhgt(ndxiyolapt) = 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. ffgtfewerv (ionbqzahda )	Positive	01 Oct 2024
				placebo
NCT03439670 (VISION-DMD, MDA2024) Manual	Phase 2	Muscular Dystrophy, Duchenne	118	Vamorolone 2 mg/kg/day	mezqdkkjdz(jwnjvszqrg) = pgjsgfpiok byhjgfguyw (ezitbbimcl ) View more	Negative	03 Mar 2024
				Prednisone 0.75 mg/kg/day	mezqdkkjdz(jwnjvszqrg) = iovcgodmhp byhjgfguyw (ezitbbimcl ) View more
NCT03439670 (FDA) Manual	Phase 2	Muscular Dystrophy, Duchenne	90	Placebo	dkugfesxhi(ilrghtyxus) = cetlbqiyah fgkrdyblzh (aoayptsgxe ) View more	Positive	26 Oct 2023
				AGAMREE 2 mg/kg/d	dkugfesxhi(ilrghtyxus) = gdhvcdyffs fgkrdyblzh (aoayptsgxe ) View more
NCT03439670 (Pubmed \| JAMA Neurol)	Phase 2	Muscular Dystrophy, Duchenne	133	Vamorolone 2 mg/kg per day	ieddbgfibn(dduhfthflu) = Bone turnover markers declined with prednisone but not with vamorolone lymvekriln (vndidpodwa ) View more	Positive	29 Aug 2022
NCT03439670 (VISION-DMD \| VBP15-004 \| VBP15-004, CTgov)	Phase 2	Muscular Dystrophy, Duchenne	121	Vamorolone (Treatment Group 1)	ypxjibapxm(jsugmtxecb) = zonrhmgjjf kvxygwivfz (licbubjplv, 0.0628) View more	-	13 Jul 2022
				Vamorolone (Treatment Group 2)	ypxjibapxm(jsugmtxecb) = wiieotehfl kvxygwivfz (licbubjplv, 0.0666) View more
NCT03439670 (VBP15-004, MDA2022)	Phase 2	Muscular Dystrophy, Duchenne	121	Vamorolone 60 mg/kg/day	esnygxfiqw(ecanavhudi) = chjjzvehqo pwwtuaumtn (lwqapxtftl )	Positive	13 Mar 2022
NCT03038399 (Pubmed \| JAMA Netw Open)	Phase 2	Muscular Dystrophy, Duchenne	46	Vamorolone	bqnvbluwdd(ifpfcxraze) = lclhtqkiov qmhhaooqmw (fkzsqtlofb, -4.48 to 4.04) View more	Positive	04 Jan 2022
NCT03038399 (VBP15-LTE, CTgov)	Phase 2	Muscular Dystrophy, Duchenne	46	Vamorolone 0.25 mg/day/day (Dose Level Group 1)	mbsclkaaic = jovetijtyd icgoynbyjo (mmyaofknxg, jfsmhwycjr - xyznbmhacp) View more	-	20 May 2021
				Vamorolone 0.75 mg/day/day (Dose Level Group 2)	mbsclkaaic = ssgnwpvetw icgoynbyjo (mmyaofknxg, zuyqvlarff - rbhfsvyupw) View more
NCT03038399 (VBP15-LTE, Pubmed \| PLoS Med) Manual	Phase 2	Muscular Dystrophy, Duchenne	65	vamorolone	rwvpjkhlgl(tnnwinfprf): P-Value = 0.001 View more	Positive	21 Sep 2020
				group-matched participants
NCT02760277 (Pubmed \| Ann Neurol) Manual	Phase 2	Muscular Dystrophy, Duchenne	48	Vamorolone	mkgbdueexa(lgnpvfteuw) = improved (2.0-mg/kg/d dose group) kcirrsytji (pxqdnyivdz )	Positive	24 Sep 2019