A Randomized, Double-blind, Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Progressive Supranuclear Palsy

This is a randomized, placebo-controlled trial of RT001 in patients with PSP.

开始日期2021-06-23

申办/合作机构

Retrotope, Inc.

RBR-62cftbp

/ Active, not recruiting临床2期IIT

Awake Bruxism Diagnosis and Treatment - Randomized Controlled Trial

开始日期2021-03-20

申办/合作机构-

NCT04762589

/ Unknown status临床2期

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Amyotrophic Lateral Sclerosis

RT001-014 is a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis

开始日期2021-03-10

申办/合作机构

Retrotope, Inc.

100 项与 A型肉毒毒素 (Revance) 相关的临床结果

登录后查看更多信息

100 项与 A型肉毒毒素 (Revance) 相关的转化医学

登录后查看更多信息

100 项与 A型肉毒毒素 (Revance) 相关的专利（医药）

登录后查看更多信息

1,345

项与 A型肉毒毒素 (Revance) 相关的文献（医药）

2025-07-01·Ocular Surface

Impact of botulinum toxin type A on ocular pain with neuropathic features

Article

作者: Felix, Elizabeth R ; Kantor, Nicole B ; Reyes, Nicholas ; Lee, Wendy W ; Locatelli, Elyana V T ; Betz, Jason D ; Huang, Jordan J ; Galor, Anat ; Huang, Jaxon J

PURPOSE:

To investigate the impact of botulinum toxin type A (BoNT-A) on neuropathic/nociplastic ocular pain (NOP) and identify predictors of patient outcomes.

METHODS:

A retrospective study of individuals with NOP symptoms (light sensitivity, wind/air sensitivity, persistent pain despite dry eye treatment) who received ≥1 BoNT-A injection. Primary outcome measures included if (responders vs. non-responders) and to what degree (none vs. mild vs. moderate vs. marked) individuals experienced pain improvement 4-6 weeks post-injection. Demographics and clinical exam information was compared between the groups.

RESULTS:

27 individuals received BoNT-A for NOP symptoms. 74 % (n = 20) reported an improvement in pain and were classified as responders. Among responders, the degree of benefit varied, with 25 % reporting mild, 45 % moderate, and 30 % marked pain improvement. Improvements in light sensitivity (37 %), wind/air sensitivity (33 %), and quality of life (QoL) (59 %) were reported by fewer individuals. 80 % of responders and 0 % of non-responders reported QoL improvements afer BoNT-A. In a multivariable model that examined predictors of response (none to marked, 0-3), the presence of fibromyalgia (FM) (β = 0.50; p = 0.004) portended a better response, while shooting pain (β = -0.47; p = 0.007) portended a worse response to BoNT-A, (full model r² = 0.53; p < 0.001). Degree of pain improvement significantly correlated with improvements in light sensitivity, wind/air sensitivity, and QoL (ρ range: 0.42-0.63; p < 0.05).

CONCLUSION:

After BoNT-A, most individuals reported improved ocular pain and QoL, while fewer noted improved light and wind/air sensitivity. Some systemic and ocular factors predicted treatment response and may thus guide treatment.

2025-07-01·CNS & Neurological Disorders-Drug Targets

Comparison of Guided and Unguided Botulinum Injections for Cervical Dystonia: EMG, Ultrasound, and Anatomic Landmarks

Article

作者: Eichel, Roni ; Faust-Socher, Achinoam ; Yahalom, Gilad ; Livneh, Vered ; Gurevich, Tanya ; Israel, Ilana ; Cohen, Mikhal E. ; Schechter, Yosef

Background::

Botulinum Toxin type A (BonTA) is the preferred treatment for Cervical Dystonia (CD). However, the success rate is often suboptimal. One of the reasons for treatment failure is the in accuracy of injections. Some physicians rely on Anatomical Landmarks (AL) for injections, while others use either Ultrasound (US) or Electromyography guidance (EMGg) to improve accuracy.

Methods::

This retrospective two-center study compared the therapeutic outcomes of AL-based and EMGg injections with USg injections. Demographic and clinical assessments of previous visits and current visits were recorded between 2019 and 2023.

Results::

Fifty-one patients were included. Six patients were injected using AL, 14 patients under EMGg, and 31 patients received USg injections. Pain relief was significantly lower for the AL group (40.0% ± 22.4%) compared to both USg and EMGg (81.2% ± 34.0% and 82.2% ± 10.3%, respectively; p = 0.001). Dysphagia was reported in 7.1% of EMGg and 16% of the USg group and none of those treated with AL (p > 0.05).

Conclusion::

The results of this study demonstrated that the clinical outcomes of USg and EMGg BonTA injections are comparable and both techniques are superior to AL. The main side effect observed was dysphagia, which was more common in the USg group, although without reaching statistical significance.

2025-06-01·TOXICON

Effects of botulinum toxin on pain control mechanisms, muscle structure and cortical reorganization, a mini-review

Review

作者: Mollaei, Zahra ; Mehraban Jahromi, Mostafa ; Grünerová Lippertová, Marcela ; Vlček, Přemysl

Spasticity often results in significant disability, which complicates rehabilitation and daily activities. This review explores the role of botulinum toxin type A (BoNT-A) in the treatment of spasticity, focusing on its effects on muscle structure and activity, function, cortical reorganization, and pain. Our findings indicate that BoNT-A injections improve motor function and gait, particularly in stroke patients, by reducing abnormal muscle activity and enhancing postural control. However, BoNT-A may also induce unwanted biomechanical changes, such as muscle atrophy and alterations in contractile elements, which could impact long-term muscle function. Regarding pain management in spasticity, BoNT-A has shown promise by reducing both peripheral and central sensitization mechanisms. Additionally, BoNT-A influences the central nervous system (CNS) by inducing cortical reorganization, which may further contribute to clinical improvements. Lastly, BoNT-A treatment requires careful consideration of individual patient characteristics to optimize outcomes and minimize side effects. A multidisciplinary approach that combines BoNT-A with physical therapy is essential to maximize functional recovery and improve the quality of life in patients with spasticity.

项与 A型肉毒毒素 (Revance) 相关的新闻（医药）

2023-02-21

·PRNewswire

Amyotrophic Lateral Sclerosis Pipeline Booms as 100+ Therapies Likely to Enter in the Treatment Domain | DelveInsight

The prevalence of amyotrophic lateral sclerosis has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of amyotrophic lateral sclerosis and the growing research and development activities to develop novel therapies to treat amyotrophic lateral sclerosis to drive the market. The companies developing the potential therapies in the last stage of development include Biogen, AB Sciences, Helixmith, and several others. LAS VEGAS, Feb. 21, 2023 /PRNewswire/ -- DelveInsight's ' Amyotrophic Lateral Sclerosis Pipeline Insight – 2023 ' report provides comprehensive global coverage of available, marketed, and pipeline amyotrophic lateral sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the amyotrophic lateral sclerosis pipeline domain. Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report DelveInsight's amyotrophic lateral sclerosis pipeline report depicts a robust space with 100+ active players working to develop 100+ pipeline therapies for amyotrophic lateral sclerosis treatment. Key amyotrophic lateral sclerosis companies such as Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals, and others are evaluating new amyotrophic lateral sclerosis drugs to improve the treatment landscape. Promising amyotrophic lateral sclerosis pipeline therapies in various stages of development include BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101, RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil, SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A, CK 0803, 3K3A-APC, QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501, and others. In February 2023, NeuroSense Therapeutics Ltd. and QuantalX Neuroscience Ltd; the developer of Delphi-MD, a clinically objective neurodiagnostic medical device , announced a collaboration to improve early diagnosis and treatment of neurodegenerative diseases.In addition, the companies agreed that QuantalX's Delphi-MD would be used for early diagnosis and ongoing monitoring of trial participants in NeuroSense's planned future pivotal Phase III efficacy trial of PrimeC in people living with amyotrophic lateral sclerosis (ALS), pending the successful conclusion of its ALS Phase IIb trial. In February 2023, Amylyx Pharmaceuticals, Inc. announced the completion of enrollment in PHOENIX, a global, 48-week, randomized, placebo-controlled Phase III clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) in people living with amyotrophic lateral sclerosis (ALS). Amylyx anticipates topline results in 2024. The study enrolled 664 participants living with ALS. In February 2023, UMass Chan Medical School signed a three-year sponsored research agreement with NeuShen Therapeutics Inc. to investigate a gene therapy treatment for amyotrophic lateral sclerosis, a rare neurological disease. Based in Shanghai and Boston, NeuShen is focused on developing innovative treatments for central nervous system disorders using adeno-associated virus-based gene therapy and small molecule delivery. In January 2023, Inflamed Pharma, part of the incubator and accelerator company Xlife Sciences, partnered with researchers at the University Medical Center Gottingen in Germany to develop its investigational therapy ProcCluster for the treatment of amyotrophic lateral sclerosis (ALS).The collaboration, led by Jan C. Koch, MD, a principal investigator at the university's Department of Neurology, will be focused particularly on investigating ProcCluster in cellular models of ALS. In January 2023, Amylyx Pharmaceuticals and Neopharm signed a license and distribution agreement to commercialize AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine) to treat amyotrophic lateral sclerosis (ALS).Neopharm will commercialize the oral, fixed-dose medication in Israel, West Bank, Gaza, and the Palestinian Authority, subject to regulatory review and clearance.According to the agreement, Amylyx will grant exclusive rights to Neopharm for commercializing the therapy in the covered territory. In January 2023, uniQure N.V. and Apic Bio announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. Request a sample and discover the recent advances in amyotrophic lateral sclerosis drug treatment @ Amyotrophic Lateral Sclerosis Pipeline Report The amyotrophic lateral sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage amyotrophic lateral sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the amyotrophic lateral sclerosis clinical trial landscape. Amyotrophic Lateral Sclerosis Overview Amyotrophic Lateral Sclerosis (ALS) is a rare neurological disease that affects the nerve cells (neurons) that control voluntary muscle movement (those muscles we choose to move). Voluntary muscles are responsible for movements such as chewing, walking, and talking. The disease is progressive, which means that the symptoms worsen over time. There is currently no cure for ALS and no effective treatment to slow or reverse the disease's progression. Muscle weakness or stiffness are common early ALS symptoms. Individuals gradually lose strength and the ability to speak, eat, move, and even breathe as all voluntary muscles are affected. Most people with ALS die from respiratory failure, usually within 3 to 5 years of the onset of symptoms. However, approximately 10% of people with ALS live for ten years or more. There is no single test that can definitively diagnose ALS. A detailed history of the symptoms observed by a physician during physical examination, as well as a review of the individual's full medical history and a series of tests to rule out other diseases, are used to make the ALS diagnosis. Find out more about drugs for amyotrophic lateral sclerosis @ New Amyotrophic Lateral Sclerosis Drugs A snapshot of the Amyotrophic Lateral Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging amyotrophic lateral sclerosis pipeline therapies @ Amyotrophic Lateral Sclerosis Clinical Trials Amyotrophic Lateral Sclerosis Therapeutics Assessment The amyotrophic lateral sclerosis pipeline report proffers an integral view of amyotrophic lateral sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Amyotrophic Lateral Sclerosis 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, Parenteral, Intravenous, Subcutaneous, Topical Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics Assessment By Mechanism of Action: RNA interference, Superoxide dismutase 1 expression inhibitors, Angiogenesis inhibitors, Colony stimulating factor inhibitors, Coronavirus-3C-like-proteinase inhibitors, Endopeptidase Clp inhibitors, Focal adhesion protein-tyrosine kinase inhibitors, Lyn protein-tyrosine kinase inhibitors, Macrophage colony-stimulating factor receptor modulators, Mast cell inhibitors, Platelet-derived growth factor receptor antagonists, Protein tyrosine kinase inhibitors, Proto oncogene protein c-kit inhibitors, Proto-oncogene protein c-fyn modulators, Type 3 fibroblast growth factor receptor antagonists, Angiogenesis inducing agents, Gene transference, Hepatocyte growth factor expression stimulants, G protein-coupled receptor modulators, HSP90 heat-shock protein modulators, Ion channel modulators, Antioxidants, NAD(P)H dehydrogenase (quinone) modulators, CD40 ligand inhibitors, Complement C1 inhibitors, Cyclo-oxygenase 2 inhibitors, DNA gyrase inhibitors Key Amyotrophic Lateral Sclerosis Companies: Biogen, Ionis Pharmaceuticals, Ferrer Internacional S.A., AbbVie, Calico Life Sciences LLC, Genuv Inc., Kadimastem, Corcept Therapeutics, AL-S Pharma, Rapa Therapeutics LLC, Cytokinetics, MediciNova, Retrotope, Inc. Woolsey Pharmaceuticals, Sanofi, PTC Therapeutics, Helixmith Co., Ltd., Annexon, Inc., Denali Therapeutics Inc., Revalesio Corporation, Clene Nanomedicine, Ashvattha Therapeutics, Inc., Apellis Pharmaceuticals, Inc., Procypra Therapeutics, Knopp Biosciences, InFlectis BioScience, AI Therapeutics, Inc., Cellenkos, ZZ Biotech, LLC, QurAlis Corporation, Alector Inc., NeuroSense Therapeutics Ltd., Novartis Pharmaceuticals, Eledon Pharmaceuticals, and others. Key Amyotrophic Lateral Sclerosis Pipeline Therapies: BIIB067, FAB122, ABBV-CLS-7262, Trametinib, BIIB105, AstroRx, Dazucorilant, AP-101, RAPA-501, ION-363, Reldesemtiv, MN-166, RT001, Fasudil, SAR443820, PTC857, ANX005, DNL343, RNS60, BIIB100, CNMAu8, 18F-OP-801, Pegcetacoplan (APL-2), Cu(II)ATSM, KNS-760704, IFB-088, LAM-002A, CK 0803, 3K3A-APC, QRL-201, Dexpramipexole, AL001, PrimeC, BLZ945, AT-1501, and others. Dive deep into rich insights for new drugs for amyotrophic lateral sclerosis treatment; visit @ Amyotrophic Lateral Sclerosis Medications Table of Contents For further information on the amyotrophic lateral sclerosis pipeline therapeutics, reach out @ Amyotrophic Lateral Sclerosis Drug Treatment Related Reports Amyotrophic Lateral Sclerosis Epidemiology Amyotrophic Lateral Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted amyotrophic lateral sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Amyotrophic Lateral Sclerosis Market Amyotrophic Lateral Sclerosis Market Insights, Epidemiology, and Market Forecast – 2032 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 amyotrophic lateral sclerosis companies including Helixmith, Sanofi, Denali Therapeutics, Alector, GSK, NeuroSense Therapeutics, Biogen, among others. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 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 Duchenne muscular dystrophy companies including Edgewise Therapeutics, Pfizer, Daiichi Sankyo, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. Facioscapulohumeral Muscular Dystrophy PIpeline Facioscapulohumeral Muscular Dystrophy Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key facioscapulohumeral muscular dystrophy companies, including miRecule, Arrowhead Pharmaceuticals, Fulcrum Therapeutics, among others. Other Trending Reports Tay-sachs Disease Or Gm2 Gangliosidosis Market | Onycholysis Market | Diagnostic Imaging Equipment Market | Chemotherapy-induced Peripheral Neuropathy Market | Global Electrophysiology Devices Market | Anaphylaxis Market | Atherectomy Devices Market | Helicobacter Pylori Infections Market | Ophthalmic Imaging Equipment Market | Androgenetic Alopecia Market | Allergic Rhinitis Market | Chronic Inflammatory Demyelinating Polyneuropathy Market | Chronic Inflammtory Demyelinating Polyneuropathy Market | Colorectal Cancer Crc Market | Opioid Induced Constipation Market | Vertigo Market | Bone Anchored Hearing Systems Market | Wound Closure Devices Market | Hip Replacement Devices Market | Hemodynamic Monitoring Systems Market | Egfr Non-small Cell Lung Cancer Market | Helicobacter Pylori Infection Market | Hyperkalemia Market | Polycythemia Market Neurostimulation Devices Market | Carpal Tunnel Syndrome Market | Ventilator Market | Cerebral Aneurysm Market | Alpha Antitrypsin Market | Binge Eating Disorder Market | Bunion Market | Concussions Market Size | Exocrine Pancreatic Insufficiency Market | Healthcare Due Diligence Services | Minimal Residual Disease Market | Hypertrophic Scar Market | Lung Fibrosis Market | Anterior Uveitis Market | 22q11.2 deletion syndrome Market | X-Linked Retinitis Pigmentosa (XLRP) Market | Acute Radiation Syndrome Market | Alpha-1 Protease Inhibitor Deficiency Market | Androgenetic Alopecia Market | Hyperlipidemia Market | Cardiotoxicity Market | Hypertrophic Cardiomyopathy Market | Fatty Acid Oxidation Disorders (FAODs) Market | Androgen Insensitivity Syndrome Market | Emphysema Market | Canaloplasty Market | Dravet Syndrome Market | Celiac Disease Market | Chlamydia Infections Market | Syphilis Market | Renal Tubular Acidosis Market | Palmoplantar Pustulosis (PPP) Market | Aplastic Anemia Market | Bacterial Pneumonia Market | B cell Chronic Lymphocytic Leukemia Market | B cell Lymphomas Market | Behcets Disease Market Neoantigen-based Personalized Cancer therapeutic Vaccines Competitive Landscape and Market Forecast—by 2035 | Glioblastoma Market Related Healthcare Services Healthcare Consulting Healthcare Competitive Intelligence Services Healthcare Asset Prioritization Services 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 . Connect with us on LinkedIn |Facebook|Twitter Additionally, get in touch with our business executive to explore @ Healthcare Due Diligence Services Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

临床3期引进/卖出临床2期

2021-10-06

·BioSpace

Retrotope Drug Improves Survival in Ultra-Rare Genetic Childhood Disease

Retrotope, based in Los Altos, California, cited data from its Phase II/III trial of RT001 in patients with infantile neuroaxonal dystrophy (INAD). They also reported on a concurrent natural history study of INAD patients’ disease onset and progression. INAD is an ultra-rare, progressive, fatal, infant genetic neurological disorder. It is on a spectrum of diseases called PLA2G6-associated neurodegeneration. A congenital disability in the PLA2G6 enzyme causes an inability to effectively clear the byproducts of lipid peroxidation (LPO), which are toxic. This results in a “death signal” that leads to rapid neuronal cell death in the brains of developing infants. Alex Fay, Assistant Professor of Neurology, University of California, San Francisco and one of the study’s principal investigators, noted, “INAD is an ultra-rare, relentlessly progressive and fatal disease that currently has no approved treatments. This trial compared a group of children with INAD treated with RT001 with a natural history cohort, and the results indicate that RT001 slows progression and prolongs survival compared to no treatment. The findings are striking and suggest that RT001 has the potential to deliver a meaningful impact on neurological function and survival of children with INAD.” RT001 is an isotopically stabilized, synthetic linoleic acid (LA) that Retrotope discovered and developed using its technology platform, which combats the oxidative stress and cellular degeneration that comes from lipid peroxidation (LPO). Polyunsaturated fatty acids (PUFAs) make up the cell and mitochondrial membranes. The LPO process targets PUFAs. Retrotope’s technology generates stabilized PUFAs, such as RT001, incorporating into all membranes and down-regulating LOP to protect membranes from degeneration. The clinical trial included 19 INAD patients and the concurrent natural history of 36 INAD patients as the control group. There were no significant differences between the baseline characteristics in the two cohorts. Patients in the treatment cohort were dosed with RT001 for a minimum of one year with a 30-day treatment-free follow-up. The patients receiving RT001 demonstrated an improvement of 6.42 rank points on the Modified Ashworth Spasticity Scale compared to the control cohort. Similar treatment benefits were observed for the RT001 cohort compared to controls across all five of the efficacy outcomes scales. Progression free survival (absence of pneumonia or death event) was a pre-specified secondary endpoint. The RT001 group demonstrated a statistically significant 82.5% decrease in morbidity risk compared to the controls. A pre-specified exploratory endpoint of survival showed a statistically significant mortality risk decrease of 88.8%. Confirmed deaths were “dramatically lower” in patients receiving RT001 compared to the control group, 11% versus 31%, respectively. “We are very pleased by these topline results and excited to meet with the FDA to discuss the next steps in our ongoing effort to bring RT001 to the INAD patient community,” said Peter G. Milner, Chief Medical Officer of Retrotope. “With clear evidence of benefit on patient survival, as well as consistent improvements observed in all analyzed efficacy endpoints including those which measured vital function and activities of daily living, we believe these study results support the potential of RT001 to address a critical unmet need in hits orphan disease patient population.” Retrotope has ongoing trials for RT001 in amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP) and Friedreich’s ataxia (FA), three of the four most common orphan neurodegenerative diseases. According to Clinical Trials Arena, the company is targeting a funding round in the first quarter of 2022 to raise $100 million. On August 11, the company indicated that it had completed enrollment for its Phase II study of RT001 for progressive supranuclear palsy (PSP). PSP is a progressive, neurodegenerative disease resulting in impaired balance, problems with eye movement, speech, and swallowing, rigid muscle tone, and ultimately, cognitive impairment. It usually affects people between the ages of 60 and 70. It is often misdiagnosed as Parkinson’s disease and Alzheimer’s disease. On April 21, Retrotope completed enrollment of its Phase II trial of RT001 in ALS (Lou Gehrig’s disease). They exceeded the enrollment target of 40 patients in less than six weeks. They expect data from the trial by the end of the year.

合作

2021-10-06

·BioSpace

Retrotope Drug Improves Survival in Ultra-Rare Genetic Childhood Disease

Retrotope cited data from its Phase II/III trial of RT001 in patients with infantile neuroaxonal dystrophy and reported on a natural history study of INAD patients’ disease onset and progression. Retrotope , based in Los Altos, California, cited data from its Phase II/III trial of RT001 in patients with infantile neuroaxonal dystrophy (INAD). They also reported on a concurrent natural history study of INAD patients’ disease onset and progression. INAD is an ultra-rare, progressive, fatal, infant genetic neurological disorder. It is on a spectrum of diseases called PLA2G6-associated neurodegeneration. A congenital disability in the PLA2G6 enzyme causes an inability to effectively clear the byproducts of lipid peroxidation (LPO), which are toxic. This results in a “death signal” that leads to rapid neuronal cell death in the brains of developing infants. Alex Fay, Assistant Professor of Neurology, University of California, San Francisco and one of the study’s principal investigators, noted, “INAD is an ultra-rare, relentlessly progressive and fatal disease that currently has no approved treatments. This trial compared a group of children with INAD treated with RT001 with a natural history cohort, and the results indicate that RT001 slows progression and prolongs survival compared to no treatment. The findings are striking and suggest that RT001 has the potential to deliver a meaningful impact on neurological function and survival of children with INAD.” RT001 is an isotopically stabilized, synthetic linoleic acid (LA) that Retrotope discovered and developed using its technology platform, which combats the oxidative stress and cellular degeneration that comes from lipid peroxidation (LPO). Polyunsaturated fatty acids (PUFAs) make up the cell and mitochondrial membranes. The LPO process targets PUFAs. Retrotope’s technology generates stabilized PUFAs, such as RT001, incorporating into all membranes and down-regulating LOP to protect membranes from degeneration. The clinical trial included 19 INAD patients and the concurrent natural history of 36 INAD patients as the control group. There were no significant differences between the baseline characteristics in the two cohorts. Patients in the treatment cohort were dosed with RT001 for a minimum of one year with a 30-day treatment-free follow-up. The patients receiving RT001 demonstrated an improvement of 6.42 rank points on the Modified Ashworth Spasticity Scale compared to the control cohort. Similar treatment benefits were observed for the RT001 cohort compared to controls across all five of the efficacy outcomes scales. Progression free survival (absence of pneumonia or death event) was a pre-specified secondary endpoint. The RT001 group demonstrated a statistically significant 82.5% decrease in morbidity risk compared to the controls. A pre-specified exploratory endpoint of survival showed a statistically significant mortality risk decrease of 88.8%. Confirmed deaths were “dramatically lower” in patients receiving RT001 compared to the control group, 11% versus 31%, respectively. “We are very pleased by these topline results and excited to meet with the FDA to discuss the next steps in our ongoing effort to bring RT001 to the INAD patient community,” said Peter G. Milner, Chief Medical Officer of Retrotope. “With clear evidence of benefit on patient survival, as well as consistent improvements observed in all analyzed efficacy endpoints including those which measured vital function and activities of daily living, we believe these study results support the potential of RT001 to address a critical unmet need in hits orphan disease patient population.” Retrotope has ongoing trials for RT001 in amyotrophic lateral sclerosis (ALS), progressive supranuclear palsy (PSP) and Friedreich’s ataxia (FA), three of the four most common orphan neurodegenerative diseases. According to Clinical Trials Arena, the company is targeting a funding round in the first quarter of 2022 to raise $100 million. On August 11, the company indicated that it had completed enrollment for its Phase II study of RT001 for progressive supranuclear palsy (PSP). PSP is a progressive, neurodegenerative disease resulting in impaired balance, problems with eye movement, speech, and swallowing, rigid muscle tone, and ultimately, cognitive impairment. It usually affects people between the ages of 60 and 70. It is often misdiagnosed as Parkinson’s disease and Alzheimer’s disease. On April 21, Retrotope completed enrollment of its Phase II trial of RT001 in ALS (Lou Gehrig’s disease). They exceeded the enrollment target of 40 patients in less than six weeks. They expect data from the trial by the end of the year.

临床2期临床结果

100 项与 A型肉毒毒素 (Revance) 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
-	A型肉毒毒素 (Revance)	-	DB00083

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
Friedreich共济失调	临床3期	美国	Retrotope, Inc.	2019-10-30
神经轴索营养不良	临床3期	美国	Retrotope, Inc.	2018-11-05
斜颈	临床3期	美国	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	奥地利	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	加拿大	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	捷克	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	法国	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	德国	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	波兰	Revance Therapeutics, Inc.	2018-06-20
斜颈	临床3期	西班牙	Revance Therapeutics, Inc.	2018-06-20

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04102501 (CTgov)	临床3期	Friedreich共济失调	65	RT001 (RT001)	簾鹽襯鑰窪遞築餘鏇積(築餘夢齋鹽顧壓鑰選願) = 範憲顧鹽觸糧餘範築願鹽壓鏇醖夢簾構鹽壓網 (築窪遞襯構憲選壓選淵, 0.04) 更多	-	2022-05-27
NCT04102501 (CTgov)	临床3期	Friedreich共济失调	65	Placebo (Placebo)	簾鹽襯鑰窪遞築餘鏇積(築餘夢齋鹽顧壓鑰選願) = 餘襯餘築窪艱廠鏇鏇鏇鹽壓鏇醖夢簾構鹽壓網 (築窪遞襯構憲選壓選淵, 0.04) 更多	-	2022-05-27
2439 (AAN2020)	临床1/2期	亨廷顿舞蹈病	1	RT001 2.9g BID	衊築築鹹淵簾壓積觸憲(鹹齋淵壓鏇鹹選遞鬱壓) = 構醖築醖壓範鑰觸襯範夢壓範觸顧齋糧窪齋鹹 (鏇窪襯積顧襯觸選獵願 )	积极	2020-04-14
RT001 (AAN2019)	N/A	泰-萨克斯病 Lipid peroxidation (LPO)	1	RT001	鏇襯積鹹繭鏇觸遞遞鑰(膚膚餘憲顧醖願顧鏇積) = 顧襯蓋觸糧淵廠網夢齋獵繭構簾積獵蓋衊鹽觸 (襯鏇選選繭醖選齋淵艱 ) 更多	积极	2019-04-09