ReNEW: A Phase 3, Double-Masked, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects Who Have Dry Age-Related Macular Degeneration (Dry AMD)

The goal of this clinical trial is to evaluate the efficacy, safety and pharmacokinetics of elamipretide in subjects with dry age-related macular degeneration (AMD). The main questions it aims to answer are: what is the rate of change in the macular area of photoreceptor loss in subjects who receive a daily dose of elamipretide compared with those who receive a look-alike substance that contains no active drug, and what is the safety and tolerability of elamipretide daily subcutaneous injections. Participants will receive either once daily subcutaneous doses of 40mg elamipretide or placebo and the two treatment groups will be compared.

Start Date30 May 2024

Sponsor / Collaborator

Stealth BioTherapeutics, Inc.

NCT05162768

/ Active, not recruitingPhase 3

A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) NuPower

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

Start Date29 Apr 2022

Sponsor / Collaborator

Stealth BioTherapeutics, Inc.

NCT05168774

/ CompletedPhase 1/2IIT

A Pilot Investigator Initiated Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide in the Treatment of Advanced Symptoms of Friedreich Ataxia (FRDA)

To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).

Start Date03 Mar 2022

Sponsor / Collaborator

The Children's Hospital of Philadelphia

[+2]

100 Clinical Results associated with Elamipretide Hydrochloride

100 Translational Medicine associated with Elamipretide Hydrochloride

100 Patents (Medical) associated with Elamipretide Hydrochloride

404

Literatures (Medical) associated with Elamipretide Hydrochloride

01 Jun 2025·BIOMATERIALS

A multifunctional mitochondria-protective gene delivery platform promote intervertebral disc regeneration

Article

Author: Kong, Qingquan ; Zhang, Fanjun ; Wu, Ye ; Guo, Chuan ; Hu, Cheng ; Zhang, Bo ; Zheng, Cheng ; Deng, Mingyan ; Wang, Yu ; Wang, Yunbing

Intervertebral disc degeneration (IDD) is a deleterious condition driven by localized inflammation and the associated disruption of the normal homeostatic balance between anabolism and catabolism, contributing to progressive functional abnormalities within the nucleus pulposus (NP). Despite our prior evidence demonstrating that a miR-21 inhibitor can have regenerative effects that counteract the progression of IDD, its application for IDD treatment remains limited by the inadequacy of current local delivery systems. Here, an injectable tannic acid (TA)-loaded hydrogel gene delivery system was developed and used for the encapsulation of a multifunctional mitochondria-protecting gene nanocarrier (PHs). This engineered platform was designed for the sustained on-demand delivery of both miR-21 inhibitor and ss-31 (mitochondrial-targeted peptide) constructs to the NP. This prepared hydrogel could be implanted into the intervertebral disc using a minimally invasive approach whereupon it was able to rapidly release TA. Sustained PHs release was then achieved as appropriate through a mechanism mediated by the activity of MMP-2. Following the targeted uptake of PHs by degenerated NP cells, the subsequent release of encapsulated miR-21 inhibitor suppressed apoptotic cell death and modulated the metabolism of the extracellular matrix (ECM) by targeting the Spry1 gene. At the same time, ss-31 was able to target damaged mitochondria and alleviate inflammatory activity via the suppression of mitochondrial ROS-NLRP3-IL-1β/Caspase1 pathway activity. Synergistic ECM regeneration and anti-inflammatory effects were sufficient to provide therapeutic benefits in an in vivo model of IDD. Together, these results thus highlight this hydrogel-based gene delivery platform as a promising novel approach to the treatment of IDD.

05 Feb 2025·NEUROREPORT

The potential of mitochondrially-targeted tetrapeptide in protecting against noise-induced hearing impairment

Article

Author: Marouf, Azmi ; Alagramam, Kumar N. ; Stepanyan, Ruben ; Kumar, Niranj A.

Noise-induced hearing loss (NIHL) constitutes a significant global health issue for which there is no effective treatment. The loss of cochlear hair cells and associated synaptopathy are common causes of hearing impairment. One primary mechanism implicated in NIHL is the accumulation of reactive oxygen species (ROS), which ultimately overwhelms cochlear cells. ROS are detected in the cochlea immediately after noise exposure and persist for at least a week. Within cells, ROS are primarily generated in mitochondria as byproducts of cellular metabolism. Elamipretide is a synthetic tetrapeptide known to concentrate in mitochondria, improving mitochondrial function and reducing ROS production. To test the hypothesis that elamipretide treatment mitigates NIHL, 16-week-old male and female CBA/J mice were exposed to 8–16 kHz octave-band noise (OBN) at 98 dB SPL for 2 hours. Elamipretide was administered intraperitoneally immediately after noise exposure and continued for 2 weeks. Efficacy was evaluated based on auditory brainstem response (ABR) thresholds, wave amplitudes, and wave latencies in treated and control groups. Results showed that OBN-exposed mice exhibited an elevation in ABR thresholds at 16 and 32 kHz and a reduction in ABR wave-I amplitude at 32 kHz, although wave-I latencies were not affected at 16 or 32 kHz. Elamipretide treatment prevented the OBN-induced elevation of ABR thresholds and the attenuation of wave-I amplitude. These findings provide proof of concept that mitochondrial-targeted elamipretide can prevent NIHL in a mammalian model and highlight its potential to protect against NIHL in humans.

01 Feb 2025·JOURNAL OF CONTROLLED RELEASE

Synthetic nanoparticles functionalized with cell membrane-mimicking, bone-targeting, and ROS-controlled release agents for osteoporosis treatment

Article

Author: Xue, Deting ; Zhong, Yuliang ; Zhang, Weijun ; Chen, Hongyu ; Hao, Zhengan ; Zhang, Ye ; Yao, Pengjie ; Wu, Xiaoyong ; Bai, Jinwu

Postmenopausal osteoporosis is a common degenerative disease, with suboptimal clinical outcomes. The targets of current therapeutic agents are both nonspecific and diverse. We synthesized a novel nanoparticle (NP), ALN@BMSCM@PLGA-TK-PEG-SS31. After intravenous injection, the NP evaded immune phagocytosis, targeted bone tissue, and efficiently downregulated bone reactive oxygen species (ROS) generation. The core PLGA-TK-PEG-SS31 NP was ∼100 nm in diameter. The TK chemical bond breaks on exposure to ROS, releasing the novel mitochondrion-targeting peptide SS31. Outer bone marrow mesenchymal stem cell membranes (BMSCMs) were used to coat the NP with surface proteins to ensure membrane functionality. The circulation time was prolonged and immune phagocytosis was evaded. Embedding the DSPE-PEG-ALN lipid within the cell membrane enhanced the bone-targeting ability of the NP. Our results suggest that ALN@BMSCM@PLGA-TK-PEG-SS31 exerted dual effects on bone tissue in vitro, significantly inhibiting RANKL-induced osteoclastogenesis in the presence of H₂O₂ and promoting osteogenic differentiation in BMSCs. In a mouse model of ovariectomy-induced osteoporosis, ALN@BMSCM@PLGA-TK-PEG-SS31 significantly ameliorated oxidative stress and increased bone mass with no notable systemic side effects. These results suggest that ALN@BMSCM@PLGA-TK-PEG-SS31 is a promising treatment for osteoporosis.

News (Medical) associated with Elamipretide Hydrochloride

24 Jan 2025

·www.biopharmadive.com

Mixed results for Keytruda; AbbVie to work with Neomorph

Today, a brief rundown of news involving Merck & Co. and AstraZeneca, as well as updates from AbbVie, Stealth BioTherapeutics and Vigil Neuroscience that you may have missed.A regimen of cancer drugs Keytruda and Lenvima extended progression-free survival over standard chemotherapy in people with a type of gastroesophageal tumor, but did not show an overall survival benefit. The results, announced by Merck & Co. and Eisai Friday, are from a Phase 3 study called LEAP-015 that enrolled hundreds of people with locally advanced unresectable or metastatic HER2-negative gastroesophageal adenocarcinoma. The companies said they are continuing to evaluate the data, which theyll present at a future medical meeting. The findings do not affect Keytruda or Lenvimas current approvals, or other trials in the LEAP program. Ned PagliaruloAstraZeneca will invest $570 million in a new office facility in Ontario that will help grow the companys presence in Canada. The drugmaker already employs more than 2,100 people in Canada many of whom work on clinical trials and last year acquired Hamilton, Ontario-based Fusion Pharmaceuticals. The new facility will add more than 700 positions to its workforce there, across all areas of the business, AstraZeneca said Thursday. Ben FidlerAbbVie will work with San Diego-based biotech startup Neomorph to develop protein-degrading molecular glue drugs for cancer and immune diseases, the companies announced Thursday. Neomorph will get an unspecified upfront payment from AbbVie in the deal and stands to receive up to $1.64 billion more in option fees and milestones. Its the third industry collaboration since last February for Neomorph, which also has partnerships with Biogen and Novo Nordisk. Ben FidlerThe Food and Drug Administration will take another three months to decide whether it will approve Stealth BioTherapeutics drug elamipretide for Barth syndrome. The company on Thursday said the FDA determined it needs more time to review recent information submitted by Stealth following an October meeting of agency advisers. The FDA didnt cite any safety issues, nor did it request more testing, Stealth said. The new decision deadline is April 29. Ned PagliaruloShares in Vigil Neuroscience rose by double digits this week after the biotechnology company reported Phase 1 trial data for an experimental drug its developing for Alzheimers. The data, which detail the drugs safety and distribution through the body, support advancement into a Phase 2 study, Vigil said. Code-named VG-3927, the small molecule drug is designed to boost a neuroprotective response in the brain to accumulations of tau and amyloid, proteins associated with Alzheimers. Ned Pagliarulo '

Clinical ResultPhase 1AcquisitionPhase 3Phase 2

24 Jan 2025

·www.pharmaceutical-technology.com

Stealth faces another hurdle as FDA delays decision on Barth syndrome drug

The new PDUFA action date for elamipretide is 29 April 2025. Credit: Fahmi Ruddin Hidayat via Getty Images. The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics ’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome. The Prescription Drug User Fee Act (PDUFA) action date has been extended by three months to 29 April 2025, following the submission of additional information by Stealth. Stealth stated that the FDA required more time to fully review the supplemental data, which was submitted in response to requests following the FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) meeting in October 2024. At that meeting, the advisory panel voted 10-6 in favour of elamipretide’s approval. The FDA classified the new data as a major amendment to the NDA, which triggered the standard three-month extension. Despite the delay, Stealth emphasised that the FDA has not raised any safety concerns or requested additional pre-market studies. In the announcement accompanying the extension, Stealth’s CEO Reenie McCarthy said: “We continue to work closely with the agency as it completes its review of the elamipretide NDA and are actively preparing to support broad access to this therapy for individuals living with Barth syndrome as quickly as possible following potential approval.” It’s not the first time elamipretide’s development has faced regulatory challenges. The FDA rejected the company’s initial NDA in 2021, citing the lack of “an adequate and well-controlled trial that provides evidence of effectiveness”. That application was based on the SPIBA-001 study (NCT04689360), a Phase III trial incorporating data from prior studies and historical controls. The FDA expressed concerns about potential biases in the trial design, including selection bias and confounding bias, and questioned the methods used to analyse the results. Specifically, the trial compared data from a natural history cohort monitored in 2012 and 2019 with data from treated patients, which the FDA argued did not sufficiently address these biases. The October 2024 AdCom meeting also faced difficulties. The panel discussed findings from the SPIBA-201 clinical trial (NCT03098797), which assessed the impact of elamipretide in 12 male patients over 12 weeks of daily treatment. The trial failed to meet the primary endpoint of improving walking distance. Despite not meeting the efficacy endpoint, patients and physicians have strongly advocated for the drug’s approval. During the AdCom meeting, testimonials highlighted the drug’s positive effects on weight gain, strength, and overall quality of life (QoL) for individuals with Barth syndrome. Jacob Wilson, a 24-year-old male with Barth syndrome told the panel the medicine “made him feel like a new person”. Ultimately, the committee decided to vote in favour of the drug, acknowledging “the devastating impact of Barth’s” and “the significant unmet medical need” in a document released in advance of the AdCom meeting. The life-threatening disease – which causes symptoms such as cardiomyopathy and growth delays – has no cure, and no other therapies in late-stage clinical development, according to the Barth syndrome Foundation.

Phase 3NDA

23 Jan 2025

·www.prnewswire.com

70+ Key Companies Advancing in Dry Age-Related Macular Degeneration Clinical Trial Therapeutic Space | DelveInsight

Dry macular degeneration is a common eye disorder among people aged above 50 years. It causes blurred or reduced central vision due to thinning of the macula. The macula is the part of the retina responsible for clear vision in a direct line of sight. The pharmaceutical industry is increasingly focused on developing pathway-based therapies for dry AMD, with promising treatments like ALK-001 and LBS-008 in trials, though a combination approach may be needed to fully inhibit disease progression. LAS VEGAS, Jan. 23, 2025 /PRNewswire/ -- DelveInsight's ' Dry Age-Related Macular Degeneration Pipeline Insight 2025 ' report provides comprehensive global coverage of pipeline dry AMD therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the dry AMD pipeline domain. Key Takeaways from the Dry Age-Related Macular Degeneration Pipeline Report DelveInsight's dry AMD pipeline report depicts a robust space with 70+ active players working to develop 80+ pipeline therapies for dry AMD treatment. Key dry AMD companies such as Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others are evaluating new dry AMD drugs to improve the treatment landscape. Promising dry AMD pipeline therapies such as ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others are under different phases of dry AMD clinical trials. In December 2024, Galimedix Therapeutics announced the initiation of its Phase II eDREAM study to assess the safety and efficacy of GAL-101 eye drops in patients with dry age-related macular degeneration. In December 2024, Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). In November 2024, Ocugen, Inc. announced positive preliminary efficacy and safety data from the Phase I dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and most importantly there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). In October 2024, Eyestem Research, focused on creating scalable cell therapies for incurable diseases, had announced a positive outcome of the Drug Safety Monitoring Board (DSMB) review for the first cohort of its Phase one trial to treat Geographic Atrophy secondary to Dry Age-related Macular Degeneration, marking a milestone for Indian science and medical research. In September 2024, Stealth BioTherapeutics Inc. announced the presentation of new bevemipretide (SBT-272) preclinical data demonstrating topical ocular delivery to the retina with protective effects observed in models of AMD. In June 2024, Stealth BioTherapeutics Inc. announced that it has enrolled and dosed its first patient in the ReNEW trial as part of its Phase III clinical program for elamipretide in patients with dry age-related macular degeneration (dry AMD). The program consists of the two Phase III trials, ReNEW and ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD. In April 2024, Eyestem received approval from India's Central Drugs Standards Control Organisation (CDSCO) to commence human trials for Eyecyte RPE, a potential treatment for geographic atrophy arising from dry age-related macular degeneration (AMD). Request a sample and discover the recent advances in dry AMD treatment drugs @ Dry Age-Related Macular Degeneration Pipeline Report The dry AMD pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage dry AMD drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the dry AMD clinical trial landscape. Dry Age-Related Macular Degeneration Overview Age-related macular degeneration (AMD) is one of the top four causes of blindness in older adults and the leading cause of irreversible blindness in developed countries. Around 170 million people globally are affected by AMD, making it the third most common cause of vision loss worldwide. Dry AMD typically progresses slowly, but wet AMD can cause severe visual impairment within days or weeks of onset. In the early stages of AMD, there are no noticeable symptoms, though an ophthalmologist can detect drusen before symptoms arise. Some individuals experience slight vision changes during the intermediate stage, but many remain symptom-free. Others may notice dark or grey spots in their central vision or have difficulty transitioning from bright to dim lighting. Clinicians currently use various imaging techniques to monitor changes in the retina, retinal pigment epithelium (RPE), and choroid, including fundus exams, color fundus photography, fundus autofluorescence (FAF), optical coherence tomography (OCT), and infrared reflectance imaging. Over the past two decades, OCT has significantly advanced the early detection, monitoring, and treatment assessment of dry AMD. Modern spectral-domain OCT (SD-OCT) systems provide detailed cross-sectional and volumetric views of the retina, enabling clinicians to visualize, measure, and monitor retinal layers, RPE, hyperreflective foci, geographic atrophy (GA), and drusen. While SD-OCT remains the standard of care, emerging technologies like polarization-sensitive OCT (PS-OCT), which offers tissue-selective imaging, may revolutionize AMD research and clinical practices. Currently, no approved treatments exist for dry AMD. Management involves regular monitoring, timely detection of visual deterioration, appropriate rehabilitation, and early identification of choroidal neovascularization (CNV). Research is underway to explore therapeutic strategies to prevent AMD, slow its progression, or restore vision. Potential treatments include lifestyle and behavioral changes, age-related eye disease study (AREDS) supplements, visual cycle modulators, anti-inflammatory agents, complement inhibitors, gene therapy, cell-based therapies, and approaches to reduce toxic by-products. Find out more about dry AMD treatment drugs @ Drugs for Dry Age-Related Macular Degeneration Treatment A snapshot of the Dry Age-Related Macular Degeneration Pipeline Drugs mentioned in the report: Learn more about the emerging dry AMD pipeline therapies @ Dry Age-Related Macular Degeneration Clinical Trials Dry Age-Related Macular Degeneration Therapeutics Assessment The dry AMD pipeline report proffers an integral view of the dry AMD emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Dry Age-Related Macular Degeneration Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Dimerisation inhibitors, Vitamin A replacements, RBP4 protein inhibitors, Sigma-2 receptor antagonists, Complement factor H stimulants, Macrophage inhibitors, Cell replacements, RNA interference, Key Dry Age-Related Macular Degeneration Companies: Alkeus Pharmaceuticals, Dobecure, Belite Bio, Cognition Therapeutics, Aviceda Therapeutics, Stealth BioTherapeutics, Allergo Ophthalmics, Annexon, Inc., Johnson & Johnson, InflammX Therapeutics, Lineage Cell Therapeutics, Ionis Pharmaceuticals, Evergreen Therapeutics, Inc., Alexion, Luxa Biotechnology, Astellas Pharma, OliX Pharmaceuticals, Hoffmann-La Roche, Boehringer Ingelheim, Eyevensys and others Key Dry Age-Related Macular Degeneration Pipeline Therapies: ALK-001, Etamsylate, Tinlarebant (LBS-008), CT1812, AVD-104, Elamipretide, Risuteganib, ANX 007, JNJ 81201887, Xiflam, OpRegen, IONIS-FB-LRx, EG-301, ALXN2040, RPESC-RPE-4W, MA09-hRPE, OLX301A, RO7303359, BI 771716, EYS 611 and others. Dive deep into rich insights for new drugs for dry AMD treatment, visit @ Dry Age-Related Macular Degeneration Drugs Table of Contents For further information on the dry AMD pipeline therapeutics, reach out @ Dry Age-Related Macular Degeneration Treatment Drugs Related Reports Dry Age-Related Macular Degeneration Market Dry Age-Related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key dry AMD companies including Alkeus Pharmaceuticals, Belite Bio, Aviceda Therapeutics, Johnson & Johnson Innovative Medicine, Allegro Ophthalmics, Lineage Cell Therapeutics (CellCure Neurosciences), Roche, Cognition Therapeutics, Stealth BioTherapeutics, Evergreen Therapeutics, Annexon Biosciences, NGM Biopharmaceuticals, AstraZeneca, Alexion Pharmaceuticals, Ionis Pharmaceuticals, Novartis, Regenerative Patch Technologies, among others. Dry Age-related Macular Degeneration Epidemiology Forecast Dry Age-related Macular Degeneration Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted dry AMD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Age-related Macular Degeneration Market Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key AMD companies including Regeneron Pharmaceuticals, Novartis, Roche, Opthea Limited, Kodiak Sciences Inc., REGENXBIO, Alkahest Inc, Graybug Vision, Ribomic USA Inc, Outlook Therapeutics, Inc., Unity Biotechnology, Inc, PanOptica, Inc., Clearside Biomedical, Alexion Pharmaceuticals, AstraZeneca, Evergreen Therapeutics, Alkeus Pharmaceuticals, Stealth BioTherapeutics, CellCure Neurosciences, Regenerative Patch Technologies, Allegro Ophthalmics, Annexon Biosciences, NGM Biopharmaceuticals, Ionis Pharmaceuticals, Apellis Pharmaceuticals, Iveric Bio, Gyroscope Therapeutics, Luxa Biotechnology, Gemini Therapeutics , among others. Wet Age-related Macular Degeneration Market Wet Age-related Macular Degeneration Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wet Age-related Macular Degeneration companies including EyePoint Pharmaceuticals, Inc., AbbVie, Caregen Co. Ltd., Exegenesis Bio, Shanghai Henlius Biotech, Skyline Therapeutics, 4D Molecular Therapeutics, Ocugenix Corporation, Adverum Biotechnologies, Inc., Ashvattha Therapeutics, Inc., AiViva BioPharma, Inc., Ocular Therapeutix, Inc., Clearside Biomedical, Inc., Hoffmann-La Roche, Kyowa Kirin, Inc., Opthea Limited, AffaMed Therapeutics Limited, EyeBiotech Ltd., Novartis , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Phase 3Phase 2

100 Deals associated with Elamipretide Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D10925	-	-	DB11981

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Barth Syndrome	NDA/BLA	US	Stealth BioTherapeutics, Inc.	08 Apr 2024
dry age-related macular degeneration	Phase 3	US	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	CZ	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	DE	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	HU	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	IT	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	NZ	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	ES	Stealth BioTherapeutics, Inc.	30 May 2024
dry age-related macular degeneration	Phase 3	GB	Stealth BioTherapeutics, Inc.	30 May 2024
Mitochondrial Complex I Deficiency	Phase 3	US	Stealth BioTherapeutics, Inc.	29 Apr 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03891875 (ReCLAIM-2, Pubmed \| Ophthalmol Sci)	Phase 2	Age Related Macular Degeneration \| Geographic Atrophy \| Visual Acuity	176	Elamipretide 40 mg	iomeeymgnc(klfababjhq) = 86% of those receiving elamipretide and 71% of the placebo group with the most common events being injection site reactions (e.g., pruritus, injection site pain, bruising, and erythema) kzsjtoljzt (ekxvzfsihy ) View more	Positive	01 Jan 2025
				Placebo
NCT03323749 (MMPOWER-3, Pubmed \| Orphanet J Rare Dis)	Phase 3	Mitochondrial Myopathies mtDNA pathogenic variants \| MT-TL1 pathogenic variants \| POLG ... View more	-	Elamipretide	sngupbicdq(oscjfammvz) = paorwmzjqy cnsilgkaii (wagccdiicl, 8.7)	Positive	21 Nov 2024
				Placebo	sngupbicdq(oscjfammvz) = dsehmyruhe cnsilgkaii (wagccdiicl, 8.6)
NCT03891875 (ReCLAIM-2, EURETINA2024) Manual	Phase 2	dry age-related macular degeneration	-	Elamipretide (lowest quartile group)	ngcncuwopn(ymvhymyasc) = ziyrgqskck vjontmouee (tbkgipdmvy ) View more	Positive	19 Sep 2024
				Placebo (lowest quartile group)	ngcncuwopn(ymvhymyasc) = fyleshrrfr vjontmouee (tbkgipdmvy ) View more
NCT03891875 (RECLAIM-2, CTgov)	Phase 2	Age Related Macular Degeneration \| Geographic Atrophy \| Glycogen Storage Disease Type II	176	Subcutaneous elamipretide through the elamipretide delivery system (Elamipretide)	lrqfgnkzgq(wloxaqizll) = zkqteexgrp qbpqffkxyx (szcpepuxqh, gxaszkldnh - eoswedjqks) View more	-	17 Oct 2023
				Subcutaneos placebo through the elamipretide delivery system (Placebo)	lrqfgnkzgq(wloxaqizll) = vsoaijufvw qbpqffkxyx (szcpepuxqh, yvsnxpdaao - dvmortxdjd) View more
NCT03323749 (MMPOWER-3, Pubmed)	Phase 3	Mitochondrial Myopathies	218	Elamipretide 40mg/day	pzapuhuira(rkuecotkty) = uxtwlzmsuf bdgysoidor (iasrewvarf )	Negative	02 Jun 2023
NCT03891875 (ReCLAIM-2, ARVO2023)	Phase 2	Geographic Atrophy	117	Elamipretide 40 mg once daily	kdrjcpfivc(cbrmnehdeb) = ulmpcfigzi faxistolcy (zyxkkceumz ) View more	-	23 Apr 2023
				Placebo	kdrjcpfivc(cbrmnehdeb) = wavbwjhqbg faxistolcy (zyxkkceumz ) View more
NCT03891875 (RECLAIM-2, PRNewswire) Manual	Phase 2	Geographic Atrophy	176	ELAMIPRETIDE	bqspnzffjl(irkvwpmmgq) = yjkfocugks fmmwzyyxao (kzwucwfbkl )	Positive	02 May 2022
				Placebo	-
ReCLAIM-1 (AAO2021)	Phase 1	Geographic Atrophy	-	Elamipretide 40mg	fcnevrzimy(gummnyiawb) = aaglcqgfpu xuvsejdghu (rqdrbnkbeb ) View more	-	13 Nov 2021
NCT03098797 (Pubmed \| Genet Med) Manual	Phase 2/3	Barth Syndrome	12	elamipretide	lowiyolwlq(zvwjyyzlgo) = iavslffozg oxmqexgotl (qmsssewviz ) View more	Positive	01 Mar 2021
NCT01755858 (EVOLVE, CTgov)	Phase 1/2	Hypertension, Renovascular \| Renal Artery Obstruction \| Reperfusion Injury	16	Bendavia (Bendavia)	uojoovfwuk(ldrbkrbrdz) = auqxjehdgi mowgpkrhtp (vhqnjavtll, wzfobzuphe - stgfykfvww) View more	-	11 Aug 2020
				Placebo (Placebo)	uojoovfwuk(ldrbkrbrdz) = bunoicnfsa mowgpkrhtp (vhqnjavtll, fgsfbldmpd - ezhwtxpgiy) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis