The LIFT will be conducted at Brigham and Women's Hospital (BWH) of Harvard Medical School, focusing on the effect of Pyridostigmine (Mestinon) and Low-Dose Naltrexone (LDN) in subjects aged 18-65 meeting the Canadian consensus criteria (CCC) for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) as well as having specifically Orthostatic Intolerance.
This double-blind, placebo-controlled study will involve 160 participants randomized into one of four possible groups: Pyridostigmine/LDN (40), Pyridostigmine/Placebo (40), LDN/Placebo (40), Placebo/Placebo (40). The dose of Pyridostigmine will be carefully titrated from 30mg to 60mg three times a day, and the dose of LDN will be titrated from 1.5 mg to 4.5 mg once daily.
The trial includes a scale-back plan, allowing participants to reduce their dosage if they experience intolerance symptoms, with adjustments made during weekly visits. This plan provides a personalized approach to medication tolerance, ensuring participant's safety and comfort throughout the trial.
The time commitment for the participant is approximately three (3) months, and during this time, there will be three (3) in-person visits to BWH and four (4) virtual visits. Study procedures will include two (2) submaximum cardiopulmonary exercise tests, questionnaires (virtually completed), and blood and urine collection. We will be recruiting from the BWH Dyspnea Clinic as well as the Open Medicine Foundation (OMF) StudyME Registry and anticipate the entire trial will take two (2) years to complete.
The LIFT represents a significant endeavor to improve treatment options for ME/CFS patients and contribute to the broader understanding of this debilitating condition.

Start Date10 Sep 2024

Sponsor / Collaborator

The Brigham & Women's Hospital, Inc.

[+1]

NCT05334485

/ RecruitingPhase 2/3IIT

Pyridostigmine Efficacy and Safety for Treatment of Ileus After Colorectal Surgery (PESTI Trial)

A double blind, placebo controlled, randomized control trial studying the safety and efficacy of pyridostigmine as a rescue therapy for postoperative ileus. Patients who undergo elective colorectal resection with or without creation of an ostomy, and subsequently develop postoperative ileus will be eligible for enrollment. Patients will be randomized to receive either pyridostigmine or placebo in addition to the current elements of standard of care. Patients will also complete the pyridostigmine bromide side effects scale (PBSES) upon enrollment and following each administration of either intervention or placebo to monitor treatment safety and evaluate for the development of side effects.

Start Date03 Sep 2024

Sponsor / Collaborator

The Cleveland Clinic Foundation

CTR20242481

/ CompletedNot Applicable

溴吡斯的明片在中国健康受试者中空腹状态下的人体生物等效性试验

[Translation] Human bioequivalence study of pyridostigmine bromide tablets in Chinese healthy subjects under fasting condition

考察中国健康受试者在空腹条件下单剂量口服吉林省博大伟业制药有限公司提供的溴吡斯的明片（受试制剂，T，60mg）与持证商Valeant Pharmaceuticals North America LLC的溴吡斯的明片（参比制剂，R，60mg）后的体内药代动力学特征，进行生物等效性评价。

[Translation]

The in vivo pharmacokinetic characteristics of pyridostigmine bromide tablets (test preparation, T, 60 mg) provided by Jilin Boda Weiye Pharmaceutical Co., Ltd. and pyridostigmine bromide tablets (reference preparation, R, 60 mg) from the licensed manufacturer Valeant Pharmaceuticals North America LLC were investigated in healthy Chinese subjects after a single oral dose under fasting conditions, and bioequivalence evaluation was performed.

Start Date22 Aug 2024

Sponsor / Collaborator

Jilin Boda Weiye Pharmaceutical Co. Ltd.

100 Clinical Results associated with Pyridostigmine Bromide

100 Translational Medicine associated with Pyridostigmine Bromide

100 Patents (Medical) associated with Pyridostigmine Bromide

2,567

Literatures (Medical) associated with Pyridostigmine Bromide

01 Mar 2025·Hypertension

Clinical Correlates of Efficacy of Pyridostigmine in the Treatment of Orthostatic Hypotension

Article

Author: Paranjape, Sachin Y. ; Walsh, Emily ; Peltier, Amanda ; Habermann, Ralf ; Tarpara, Kishan ; Shibao, Cyndya A. ; Muldowney, James A. ; Diedrich, Andre ; Okamoto, Luis E. ; Gamboa, Alfredo ; Black, Bonnie K. ; Biaggioni, Italo

BACKGROUND:The cholinesterase inhibitor pyridostigmine is used to treat orthostatic hypotension by facilitating cholinergic neurotransmission in autonomic ganglia, thereby harnessing residual sympathetic tone to increase blood pressure (BP) preferentially in the upright posture. We hypothesized that less severe autonomic impairment was associated with greater pressor responses to pyridostigmine.METHODS:To identify predictors of pressor response, linear regression analyses between the effect of pyridostigmine on upright BP and markers of autonomic impairment were retrospectively conducted on 38 patients who had a medication trial with pyridostigmine (60 mg single dose).RESULTS:Pyridostigmine increased upright BP by 4±2/3±2 mm Hg but with a wide range of responses (−20/−15 to 29/27 mm Hg; interquartile range, −6/−4 to 11/8 mm Hg). No differences were found between multiple system atrophy (n=14) and patients with pure autonomic failure (n=24). The upright BP response to pyridostigmine was negatively correlated with supine BP and with the pressure recovery time of the Valsalva maneuver, an index of severity of autonomic impairment. In patients with multiple system atrophy, the systolic blood pressure pressor response to pyridostigmine was also positively correlated with the increase in upright heart rate divided by the fall in systolic blood pressure (∆ heart rate/∆ systolic blood pressure) and with upright plasma norepinephrine, both surrogates of residual autonomic function.CONCLUSIONS:Patients with less severe autonomic impairment are more likely to have a positive pressor response to pyridostigmine. Importantly, in this cohort of patients with severe autonomic failure, pyridostigmine was not effective in those with supine hypertension who would benefit the most from the preferential pressor effect of the drug on upright BP.REGISTRATION: URL: http://www.clinicaltrials.gov ; Unique identifier: NCT00223691.

01 Mar 2025·Journal of Geriatric Psychiatry and Neurology

Pyridostigmine for the Management of Neurogenic Orthostatic Hypotension: A Systemic Review

Review

Author: Dylewski, Angela ; Brown, Jamie N. ; Holder, Amanda C.

Background Pyridostigmine is hypothesized to improve neurogenic orthostatic hypotension (nOH) symptoms without causing or exacerbating supine hypertension. The objective of this review was to evaluate the safety and efficacy of pyridostigmine for management of nOH. Methods A literature search of PubMed, Embase, and CENTRAL was performed in December 2023 for prospective trials with a placebo or active comparator. Results Four randomized and two non-randomized studies were reviewed. Three studies utilizing a single dose, crossover design found significant differences of orthostatics using adjunctive pyridostigmine. Two studies assessing longer-term endpoints demonstrated conflicting efficacy of pyridostigmine with one trial finding significant improvement in orthostatics and symptoms after three months of therapy. Use of pyridostigmine did not lead to supine hypertension with most adverse effects being cholinergic. Conclusion Pyridostigmine may be considered as an adjunctive medication in individuals with nOH refractory to standard treatment options as it carries a favorable safety profile with low risk for supine hypertension.

01 Mar 2025·Current Problems in Cardiology

Pathophysiology and management of postural orthostatic tachycardia syndrome (POTS): A literature review

Review

Author: Briasoulis, Alexandros ; Sajjad, Laiba ; Akkawi, Abdul Rahman ; Oundo, Emmanuel ; Ghazal, Mohamad ; Fancher, Andrew ; Tanzeem, Hammad

Postural Orthostatic Tachycardia Syndrome (POTS) is a form of cardiovascular autonomic disorders characterized by orthostatic intolerance and a symptomatic increase in heart rate upon standing, which can significantly impair patients' quality of life. Its pathophysiology is complex, multifactorial; thus, a variety of treatment approaches have been investigated. Recent studies have identified three primary POTS phenotypes-hyperadrenergic, neuropathic, and hypovolemic-each requiring tailored management strategies. First-line treatment for all patients focuses on lifestyle modifications, including increased fluid and salt intake, compression garment use, physical reconditioning, and postural training. Currently, there are no medications approved by the United States Food and Drug Administration (FDA)for POTS. Pharmacologic therapies are primarily used to manage specific symptoms, though the evidence supporting their efficacy is limited. In hyperadrenergic POTS, excessive norepinephrine production or impaired reuptake leads to sympathetic overactivity, making beta-blockers an effective option. Neuropathic POTS, resulting from impaired vasoconstriction during orthostatic stress, responds to agents that enhance vascular tone, such as pyridostigmine and midodrine. Hypovolemic POTS, often triggered by dehydration and physical deconditioning, respond primarily to volume expansion and exercise. This review article provides a comprehensive overview of the pathophysiology and management strategies for POTS, with a focus on phenotype-based approaches to guide tailored treatment and improve patient outcomes.

News (Medical) associated with Pyridostigmine Bromide

21 Oct 2024

·www.biospace.com

Amneal Receives U.S. FDA Approval of New Drug Application for Pyridostigmine Bromide Extended-Release Tablets

Pyridostigmine Bromide Extended Release is a once-daily soman nerve agent pretreatment pyridostigmine product for the U.S. Armed Services and U.S. allies developed utilizing Amneal’s GRANDE ® drug delivery technology Developed with the support of Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) BRIDGEWATER, N.J.--(BUSINESS WIRE)--Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) (“Amneal” or the “Company”), a global pharmaceutical company based in the U.S., today announced that it has received U.S. Food and Drug Administration (FDA) approval of its New Drug Application (NDA) for Pyridostigmine Bromide Extended-Release (PB ER) Tablets USP 105 mg. PB ER 105 mg tablets are a once-daily, orally administered product indicated for pretreatment against the lethal effects of soman nerve agent poisoning in adults developed for the U.S. Armed Services and U.S. allies. This product offers potential for improved compliance and steady 24-hour release of the drug’s therapeutic benefit, which is intended to provide an alternative to the current PB product that is administered every eight hours and avoiding the need for service members to self-administer three times a day. This product was not previously disclosed and was developed with Amneal’s GRANDE ® drug delivery technology, which is an advanced gastric retention system that allows for sustained delivery drug release. This project was funded in part by the U.S. government under Agreement W911QY-21-9-0018. “Amneal is proud to receive our second NDA approval this year, after the recent approval of CREXONT for Parkinson’s disease. This new pyridostigmine therapy speaks to Amneal’s expertise in developing new innovative product formulations and our ongoing work with the U.S. government on new solutions. With the approval of pyridostigmine bromide extended-release tablets, we are proudly supporting the U.S. military,” said Chirag and Chintu Patel, Co-Chief Executive Officers of Amneal. The most frequently reported serious adverse reactions were breathing difficulties, dizziness, and loss of consciousness. Other reported adverse reactions observed included dysmenorrhea, nosebleeds, reduced vision in one eye, and decreased tactile sensitivity. Disclaimer: The views and opinions of authors expressed herein do not necessarily state or reflect those of the U.S. government and shall not be used for advertising or product endorsement purposes. About Amneal Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX), headquartered in Bridgewater, NJ, is a global pharmaceuticals company. We make healthy possible through the development, manufacturing, and distribution of a diverse portfolio of over 280 generic and specialty pharmaceuticals, primarily within the United States. In its Generics segment, the Company is expanding across a broad range of complex product categories and therapeutic areas, including injectables and biosimilars. In its Specialty segment, Amneal has a growing portfolio of branded pharmaceuticals focused primarily on central nervous system and endocrine disorders, with a pipeline focused on unmet needs. Through its AvKARE segment, the Company is a distributor of pharmaceuticals and other products for the U.S. federal government, retail, and institutional markets. For more information, please visit . Disclaimer The views expressed herein are those of Amneal and may not reflect the official policy or position of the Department of Defense (DoD) or the U.S. Government. Reference herein to any specific commercial products, process or service by trade name, trademark, manufacturer, or otherwise, does not constitute or imply its endorsement, recommendation, or favoring by the U.S. government and shall not be used for advertising or product endorsement purposes. Cautionary Statement on Forward-Looking Statements Certain statements contained herein, regarding matters that are not historical facts, may be forward-looking statements (as defined in the U.S. Private Securities Litigation Reform Act of 1995). Such forward-looking statements include statements regarding management’s intentions, plans, beliefs, expectations, financial results, or forecasts for the future, including among other things: discussions of future operations; expected or estimated operating results and financial performance; and statements regarding our positioning, including our ability to drive sustainable long-term growth, and other non-historical statements. Words such as “plans,” “expects,” “will,” “anticipates,” “estimates,” and similar words, or the negatives thereof, are intended to identify estimates and forward-looking statements. The forward-looking statements contained herein are also subject generally to other risks and uncertainties that are described from time to time in the Company’s filings with the Securities and Exchange Commission, including under Item 1A, “Risk Factors” in the Company’s most recent Annual Report on Form 10-K and in its subsequent reports on Forms 10-Q and 8-K. Forward-looking statements included herein speak only as of the date hereof and we undertake no obligation to revise or update such statements to reflect the occurrence of events or circumstances after the date hereof. Contacts Investor Contact Anthony DiMeo VP, Investor Relations anthony.dimeo@amneal.com

NDADrug Approval

15 Oct 2024

·www.globenewswire.com

argenx Highlights Data Showing Patient Impact Across Multiple Immunology Programs at 2024 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting and Myasthenia Gravis Foundation of America Scientific Sessions

Long-term and real-world data of VYVGART® (efgartigimod alfa-fcab) and VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) demonstrate speed of onset, depth of response, and durability of response VYVGART demonstrates consistent, favorable safety profile from follow-up safety data that totals >8,000 patient years; no vaccinations required and no impact on human serum albumin levels Real-world data show more than 50 percent of gMG patients demonstrate substantial and sustained reduction in steroid use following VYVGART initiation argenx continues to expand its reach in neurology through pipeline programs, including empasiprubart advancing in MMN and ARGX-119 in ALS and CMS October 15, 2024 – 7:00am CET Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced the presentation of clinical and real-world data across its growing immunology pipeline at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia Gravis Foundation of America (MGFA) Scientific Sessions in Savannah, GA from October 15-18, 2024. “VYVGART continues to deliver impactful benefits to patients in terms of safety, speed of onset, depth of response, and durability of response,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “The robust data we are showing at AANEM and MGFA continue to confirm VYVGART as the leading innovative biologic with an established ability to reduce steroid usage, drive minimal symptom expression for gMG patients, and reduce CIDP symptoms quickly. In addition to VYVGART, we are excited to highlight our growing neurology pipeline, including empasiprubart and ARGX-119, through which we can advance our mission of delivering transformative outcomes for even more patients.” VYVGART and VYVGART Hytrulo Demonstrate Rapid, Deep and Sustained Responses in gMG and CIDP The data presented at AANEM continue to demonstrate the significant impact of VYVGART (including VYVGART Hytrulo), the first-in-class neonatal Fc receptor (FcRn) blocker for people living with generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART is setting a new treatment standard in gMG and has shown rapid, deep and sustained responses, enabling a majority of patients to achieve minimal symptom expression (MSE) with a consistent and favorable safety profile and more than 8,000 patient years of safety data. Based on real-world data, more than half of patients can reduce steroid use by >5mg/day following VYVGART initiation. In CIDP, a majority of patients in the ADHERE trial responded to VYVGART Hytrulo and experienced reduced risk of relapse versus placebo and improvements in motor function and muscle strength, regardless of prior CIDP treatment. Highlights from VYVGART data presented at AANEM and MGFA: Early Line Use and Meaningful Steroid Reduction: VYVGART demonstrates consistent improvement across gMG patient subtypes, including those on mestinon alone, indicating its efficacy in early line use. Real-world gMG data show that at one-year post VYVGART initiation, 55% of patients reduced corticosteroid use by ≥5 mg/day and 42% of patients had achieved steroid doses of ≤5mg/day.Expansion to Seronegative and Ocular MG: argenx is honoring its long-term commitment to the broader MG community with two Phase 3 studies ongoing in additional MG patient populations, including seronegative (ADAPT-SERON) and ocular MG (ADAPT-OCULUS). Seronegative (AChR-) gMG patients evaluated in VYVGART clinical studies experienced consistent and clinically meaningful MG-ADL improvements, including patients achieving MSE.Sustained Functional Benefit in CIDP: VYVGART Hytrulo showed sustained functional benefit in motor function and muscle strength, regardless of prior treatment, which was maintained through ADHERE and the open-label extension study (through week 24).Consistent, Favorable Safety Profile: VYVGART’s consistent and favorable safety profile has been established across multiple autoimmune diseases with no increase in the incidence of adverse events with increased exposure. The unique safety profile of VYVGART is further supported by no black box warnings, no labs or immunoglobulin (Ig) monitoring, and no vaccination requirements. Advancing Immunology Pipeline Across Two First-in-Class Opportunities to Reach New Patients argenx will also highlight two additional pipeline candidates, including Phase 2 ARDA data of empasiprubart (anti-C2 inhibitor) for the treatment of multifocal motor neuropathy (MMN), and clinical trial designs of ARGX-119 (muscle-specific kinase (MuSK) agonist) for the treatment of congenital myasthenic syndromes (CMS) and amyotrophic lateral sclerosis (ALS). Cohort 1 data from the Phase 2 ARDA study will be presented in a poster, showing treatment with empasiprubart for MMN reduced the risk of IVIg retreatment by 91% (HR: 0.09 [95% CI: 0.02–0.44]) and demonstrated significant improvement in grip strength in both hands as compared to placebo. Data from a Patient Global Impression of Change scale show 94.4% of patients said they improved on empasiprubart from the start of the study, compared to 11.1% of placebo patients. As part of its commitment to the MMN community, argenx initiated the iMMersioN longitudinal study of ~150 patients to collect data on the impact of disease and burden of current treatment options on clinical outcomes and quality of life measures. A Phase 3 study of empasiprubart in MMN will start by the end of 2024. argenx posters included in MGFA Scientific Sessions All MGFA posters to be presented on Tuesday, October 15, 12:00 – 12:45pm ETPosters with an asterisk (*) will also be presented in AANEM scientific program Full TitlePresentation DetailsPatterns of Efgartigimod Dosing in Clinical Practice in the United StatesPoster # MG9Real-World Reduction in Oral Glucocorticoid Utilization at 1-Year Following Efgartigimod Initiation*Poster # MG31 / AANEM Poster # 262Efficacy, Safety, and Pharmacodynamics of Efgartigimod PH20 SC Across Bodyweight Quartiles: A Post hoc Analysis of the ADAPT-SC+ TrialPoster # MG32Fixed Cycle and Every-Other-Week Dosing of Intravenous Efgartigimod for Generalized Myasthenia Gravis: Part A of ADAPT NXT*Poster # MG33 /AANEM Poster # 182Design of a Phase 3 Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Subcutaneous Efgartigimod PH20 Administered by Prefilled Syringe in Adults with Ocular Myasthenia GravisPoster # MG38Phase 3 Trial Investigating Impact of Intravenous Efgartigimod in Anti-Acetylcholine Receptor Antibody Negative Generalized Myasthenia Gravis*Poster # MG39 / AANEM Poster # 178Observed Efficacy of Efgartigimod in Generalized Myasthenia Gravis Across Patient Subgroups in the ADAPT-SC+ StudyPoster # MG68Exploring the Impact of Non-Steroidal Immunosuppressive Drugs and Steroids on the Development of Comorbidities in Patients with Myasthenia Gravis in the National Veterans Affairs Health NetworkPoster # MG86Quality of Life of Patients with Symptomatic Ocular MG: Comparison with the General PopulationPoster # MG87Steroid Use, Toxicity, and Monitoring in Patients With Generalized Myasthenia Gravis: A Survey Of Neurologists In The United States*Poster # MG89 / AANEM Poster # 235Comparative Risk-Benefit Profiles of Immunomodulatory Therapies for Patients with Generalized Myasthenia GravisPoster # MG98 argenx posters included in AANEM Scientific Program * Session Times: Session I: Wednesday, October 16, 6:15 - 6:45pm ETSession II: Thursday, October 17, 9:30 - 10am ETSession III: Thursday, October 17, 2:45 - 3:15pm ET Full TitlePresentation Details*Long-Term Safety and Efficacy of Efgartigimod PH20 SC in Generalized Myasthenia Gravis: Interim Analysis of Anti-Acetylcholine Receptor Antibody Seronegative Participants in ADAPT-SC+Poster # 144Session I & Session IICombined Analyses of Participants With Anti-Acetylcholine Receptor Seronegative Generalized Myasthenia Gravis Treated With Efgartigimod Across Clinical StudiesPoster # 146Session I & Session IISafety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Participants With DOK7 Congenital Myasthenic Syndromes: Phase 1b Study in ProgressPoster # 165Session I & Session IIIEfficacy and Safety of Subcutaneous Efgartigimod PH20SC in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE Trial Subgroup AnalysisPoster # 176Session I & Session IIEfficacy and Safety of Subcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE/ADHERE+ TrialPoster # 177Session I & Session IIISafety Profile of Intravenous Efgartigimod From Clinical Trials in Immunoglobulin G–Mediated Autoimmune DiseasesPoster # 180Session I & Session IISafety Profile of Subcutaneous Efgartigimod PH20 From Clinical Trials in Immunoglobulin G–Mediated Autoimmune DiseasesPoster # 181Session I & Session IIIEmpasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Initial Safety and Efficacy Data of the Phase 2 ARDA StudyPoster # 198Session I & Session IILong-Term Safety, Tolerability, and Efficacy of Subcutaneous Efgartigimod PH20 in Participants With Generalized Myasthenia Gravis: Interim Results of the ADAPT-SC+ StudyPoster # 212Session I & Session IIISafety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Patients with Amyotrophic Lateral Sclerosis: A Phase 2a Study in ProgressPoster # 237Session I & Session IIRisk of Serious Infections and Malignancies in Adult Myasthenia Gravis Patients: A US Claims Database StudyPoster # 251Session I & Session IIChronic Steroid Toxicity in Adults With Myasthenia Gravis in the United States Based on Electronic Health RecordsPoster # 263Session I & Session IISubcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: Key Secondary Outcomes from the ADHERE TrialPoster # 280Session I & Session IIIEmpasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Baseline Characteristics and MMN Confirmation Committee Outcome of the Phase ARDA Study Cohort 1Poster # 293Session I & Session IICOVID-19 Vaccination Response in Participants Receiving Efgartigimod IV or Efgartigimod PH20 SC in ADAPT+ or ADAPT-SC+Poster # 298Session I & Session IIISteroid Use, Toxicity, and Monitoring in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: A Survey of Neurologists in The United StatesPoster # 306Session I & Session IIIClinical Outcomes, Disease Course, and Quality of Life in Patients With Multifocal Motor Neuropathy: iMMersioN, Study in ProgressPoster # 307Session I & Session II More information on the data presented at the 2024 AANEM Annual Meeting can be found here. See FDA-approved Important Safety Information below, full Prescribing Information for VYVGART and full Prescribing Information for VYVGART Hytrulo for additional information. Important Safety Information What is VYVGART® (efgartigimod alfa-fcab)?VYVGART is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive). IMPORTANT SAFETY INFORMATIONDo not use VYVGART if you have a serious allergy to efgartigimod alfa or any of the other ingredients in VYVGART. VYVGART can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART may cause serious side effects, including: Infection. VYVGART may increase the risk of infection. The most common infections were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain.Allergic Reactions (hypersensitivity reactions). VYVGART can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with VYVGART.Infusion-Related Reactions. VYVGART can cause infusion-related reactions. The most frequent symptoms and signs reported with VYVGART were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines,have received or are scheduled to receive a vaccine (immunization), orhave any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART?The most common side effects of VYVGART are respiratory tract infection, headache, and urinary tract infection. These are not all the possible side effects of VYVGART. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART and talk to your doctor. What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)?VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).VYVGART HYTRULO is a prescription medicine used for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) IMPORTANT SAFETY INFORMATIONDo not use VYVGART HYTRULO if you have a serious allergy to efgartigimod alfa, hyaluronidase, or any of the other ingredients in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic reactions and a decrease in blood pressure leading to fainting. VYVGART HYTRULO may cause serious side effects, including: Infection. VYVGART HYTRULO may increase the risk of infection. The most common infections for efgartigimod alfa-fcab-treated patients were urinary tract and respiratory tract infections. Signs or symptoms of an infection may include fever, chills, frequent and/or painful urination, cough, pain and blockage of nasal passages/sinus, wheezing, shortness of breath, fatigue, sore throat, excess phlegm, nasal discharge, back pain, and/or chest pain.Allergic Reactions (hypersensitivity reactions). VYVGART HYTRULO can cause allergic reactions such as rashes, swelling under the skin, and shortness of breath. Hives were also observed in patients treated with VYVGART HYTRULO. Serious allergic reactions, such as trouble breathing and decrease in blood pressure leading to fainting have been reported with efgartigimod alfa-fcab.Infusion-Related Reactions. VYVGART HYTRULO can cause infusion-related reactions. The most frequent symptoms and signs reported with efgartigimod alfa-fcab were high blood pressure, chills, shivering, and chest, abdominal, and back pain. Tell your doctor if you have signs or symptoms of an infection, allergic reaction, or infusion-related reaction. These can happen while you are receiving your VYVGART HYTRULO treatment or afterward. Your doctor may need to pause or stop your treatment. Contact your doctor immediately if you have signs or symptoms of a serious allergic reaction. Before taking VYVGART HYTRULO, tell your doctor if you: take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines,have received or are scheduled to receive a vaccine (immunization), orhave any allergies or medical conditions, including if you are pregnant or planning to become pregnant, or are breastfeeding. What are the common side effects of VYVGART HYTRULO?The most common side effects in efgartigimod-alfa-fcab-treated patients were respiratory tract infection, headache, and urinary tract infection. Additional common side effects with VYVGART HYTRULO are injection site reactions, including rash, redness of the skin, itching sensation, bruising, pain, and hives. These are not all the possible side effects of VYVGART HYTRULO. Call your doctor for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088. Please see the full Prescribing Information for VYVGART HYTRULO and talk to your doctor. About VYVGARTVYVGART is a human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. It is the first approved FcRn blocker in the United States, EU, China and Canada for the treatment of adults with generalized myasthenia gravis (gMG) who are anti- acetylcholine receptor (AChR) antibody positive and in Japan for the treatment of adults with gMG who do not have sufficient response to steroids or non-steroidal immunosuppressive therapies (ISTs). About VYVGART® HytruloVYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART®, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection. VYVGART Hytrulo is the proprietary name in the U.S. for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It may be marketed under different proprietary names following approval in other regions. About Generalized Myasthenia Gravis (gMG)Generalized myasthenia gravis (gMG) is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months,1 where muscles throughout the body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population. About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair. About EmpasiprubartEmpasiprubart (ARGX-117) is a first-in-class humanized monoclonal antibody that binds C2 and blocks activation of both the classical and lectin pathways of the complement cascade, leaving the alternative pathway intact for its antimicrobial properties. By blocking complement activity upstream of C3 and C5, empasiprubart has the potential to reduce tissue inflammation and cellular damage, representing a broad pipeline opportunity across multiple severe autoimmune indications. In addition to multifocal motor neuropathy, argenx is evaluating empasiprubart in delayed graft function following kidney transplant, dermatomyositis and chronic inflammatory demyelinating polyneuropathy. About Multifocal Motor NeuropathyMultifocal motor neuropathy (MMN) is a rare, severe, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with the presence of anti-GM1 IgM autoantibodies, leading to activation of the classical complement pathway, driving subsequent axon damage. High-dose IVIg is the only approved treatment for MMN and patients typically experience disease progression despite therapy, indicating an unmet need for efficacious and better tolerated therapeutic options. About ARGX-119ARGX-119 is a humanized agonistic monoclonal antibody (mAb) that targets and activates muscle-specific kinase (MuSK) to promote maturation and stabilization of the neuromuscular junction (NMJ). MuSK is a receptor kinase that has a critical role in the structure and function of the NMJ. ARGX-119 is being developed as a potential therapy for patients with neuromuscular disease. About Congenital Myasthenic Syndromes (CMS) Congenital Myasthenic Syndromes (CMS) are a heterogenous group of rare genetic disorders of the neuromuscular junction (NMJ) that lead to muscle weakness. CMS cases are classified into subtypes depending on the underlying genetic mutation. While clinical features vary widely across and within subtypes, the predominant manifestation of CMS is fatigable weakness. Age of onset varies widely; while many patients are diagnosed in infancy or early childhood, patients with milder phenotypes may not present or be diagnosed until adulthood. About Amyotrophic Lateral Sclerosis (ALS) ALS is a neurodegenerative disorder of the brain and spinal cord leading to deteriorating muscle function, weakness and atrophy. The multisystemic nature can have impacts throughout the body with specific signs and symptoms associated with lower motor neuron and upper motor neuron loss. Life expectancy is typically 2.5-5 years from diagnosis. About argenxargenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK, Canada and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube. ContactsMedia:Ben Petokbpetok@argenx.com Investors:Alexandra Roy (US)aroy@argenx.com Lynn Elton (EU)lelton@argenx.com Forward-Looking Statements The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “aim,” and “will,” and include statements argenx makes regarding the anticipated timing of the initiation of the Phase 3 clinical trial for empasiprubart in MMN and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third-party suppliers, service providers and manufacturers; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law. ###

Clinical ResultPhase 2Phase 3Phase 1

04 Apr 2024

·www.prnewswire.com

IASO Bio Announces U.S. FDA Approval of Investigational New Drug Application for BCMA CAR-T Equecabtagene Autoleucel for Generalized Myasthenia Gravis

SHANGHAI, NANJING, China and SAN JOSE, Calif., April 4, 2024 /PRNewswire/ -- IASO Biotechnology ("IASO Bio"), a biopharmaceutical company dedicated to discovering, developing, manufacturing and commercialising innovative cell therapy and antibody products, is pleased to announce that the Investigational New Drug (IND) application for its BCMA CAR-T (Equecabtagene Autoleucel) has been approved by the U.S. Food and Drug Administration (FDA) for use in U.S. clinical trials for generalised myasthenia gravis (gMG). The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year. Two subjects with refractory MG were enrolled in an investigator-initiated open-label study to evaluate the safety and efficacy of Eque-cel for the treatment of relapsed/refractory antibody-mediated idiopathic inflammatory disorders of the nervous system (NCT04561557 One was a 33-year-old female, AChR-IgG and Titin-IgG positive, who had been treated with thymectomy 21 months prior to enrollment and had not achieved clinical remission after treatment with cholinesterase inhibitors, glucocorticoids, immunosuppressants, and anti-CD20 monoclonal antibody. The other subject was a 60-year-old female, MuSK-IgG4 positive, with a 20-year history of the disease and failed the previous treatment with hormones, immunosuppressants, and anti-CD20 monoclonal antibody. The 2 subjects were treated with a single infusion of Eque-cel at the doses of 1.0×106 CAR-T/Kg, respectively. Safety: Of the 2 subjects, only 1 subject developed grade 1 Cytokine Release Syndrome (CRS), and no Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS). All grade ≥3 hemocytopenia recovered within 4 weeks post infusion. No new safety risk was identified, and the safety profile was superior compared to that in multiple myeloma indication. Eff icacy : Clinical symptoms continued to improve over 18 months in the 2 subjects. From 3 months after the infusion, patients showed significant improvement in limb strength and vital capacity, and sustained improvement in Myasthenia Gravis-Activities of Daily Living Score (MG-ADL), Quantitative Myasthenia Gravis Score (QMG), Myasthenia Gravis-Quality of Life Score (MG-QOL), and Modified Rankin Score (mRS). No immunomodulatory therapy other than low dose pyridostigmine (90 mg/day and 60 mg/day, respectively) was used during the follow-up period. PK/PD: Eque-cel expanded well after infusion. Anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies decreased rapidly and maintained at very low levels in both subjects after infusion. B cells and plasma cells decreased to undetectable levels in both subjects within 2 months after infusion and then recovered gradually. At 18 months post-infusion, the B cells of both subjects had returned to normal levels, with approximately 80% of them being naïve B cells, while plasma cells remained at low levels. This result suggests that the long-term efficacy of CAR-T cell therapy may be related to the reconstitution of B cells with a predominantly naïve phenotype and the continued clearance of plasma cells. About MG Myasthenia gravis (MG) is an autoantibody-mediated neuromuscular junction disorder. The main clinical manifestation of MG is a decrease in muscle strength in local or systemic muscles, which can affect eye, respiratory and limb muscles and other important muscle groups, which has a huge negative impact on the patient's quality of life, and dysphagia or breathing difficulty caused by a myasthenic crisis can be life-threatening. The main pathogenic antibodies in MG include AChR, MuSK and LRP4 antibodies. A very small number of patients have no detectable antibodies in their serum1. MG can occur at any age and is slightly more common in women than in men 2. According to relevant studies, the incidence rate of MG in the United States is 3.1/100,000 people and the prevalence rate is 37.0/100,000 people3. It is estimated that the number of people with MG incidence in the United States in 2023 was more than 10,000, and the number of patients with MG was about 124,000. Ms. Jinhua Zhang, Founder, Chairman, and Chief Executive Officer of IASO Bio, stated, "We are very pleased that Equecabtagene Autoleucel has been approved for clinical trial for myasthenia gravis in the United States. This is the result of the unremitting efforts of our team and is an important milestone in our efforts to improve the quality of life of patients with autoimmune diseases around the world. We will actively obtain more data on the safety and efficacy of Equecabtagene Autoleucel in the treatment of MG patients, and better explore and define the significance of this innovative cell therapy in the treatment of immune diseases. We hope Equecabtagene Autoleucel will provide a more efficient and long-lasting therapeutic option for myasthenia gravis and potentially other autoimmune diseases." About IASO Bio IASO Bio is a biopharmaceutical company engaged in the discovery and development of novel cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercial production. The pipeline in the company includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received New Drug Application (NDA) approval from China's National Medical Products Administration (NMPA) and U.S. FDA IND approval for the treatment of R/RMM. Leveraging its strong management team, innovative product pipeline, GMP production, as well as integrated manufactural and clinical capabilities, IASO aims to deliver transformative, curable, and affordable therapies that fulfil unmet medical needs to patients in China as well as around the world. For more information, please visit or . SOURCE IASO Bio

Cell TherapyImmunotherapyDrug ApprovalClinical ResultClinical Study

100 Deals associated with Pyridostigmine Bromide

External Link

KEGG	Wiki	ATC	Drug Bank
D00487	Pyridostigmine Bromide	N07AA02	DBSALT000190

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Lambert-Eaton Myasthenic Syndrome	China	Shanghai Zhongxi Sunve Pharmaceutical Co., Ltd.	01 Jan 1995
Myasthenia Gravis	United States	Bausch Health US LLC	06 Apr 1955

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myasthenia Gravis	Phase 1	-	Amneal Intermediate, Inc.	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


DDW2024	Not Applicable	Persian Gulf Syndrome	-	Pyridostigmine Bromide	iekmhivben(sivdvbajvo) = kaqwkocvrq atwuvsnbkf (zoeqzjejmk, 1.18) View more	-	19 May 2024
NCT02941328 \| EUCTR2011-004369-34-NL (Pubmed)	Phase 2	Spinal Muscular Atrophy	35	Pyridostigmine	kvftcvgvar(qzygyqkgtb) = xughuolyed vhaiwenxuv (zctjxyiszt, 0.00 - 1.49) View more	-	01 Jan 2023
NCT04343963 (Pubmed) Manual	Phase 2/3	COVID-19	188	SOC+Pyridostigmine Bromide	mdsjsgeaxg(rwxtvxserr) = fwfgbmsxzq wlbzhbxgfw (dquqcgnnae ) View more	Positive	08 Nov 2022
				SOC+Placebo	mdsjsgeaxg(rwxtvxserr) = xajdkjchuv wlbzhbxgfw (dquqcgnnae ) View more
NCT03674541 (CTgov)	Phase 2	Postural Orthostatic Tachycardia Syndrome \| Fatigue Syndrome, Chronic \| Hypotension, Orthostatic \| Ventricular Dysfunction \| Fibromyalgia	45	Pyridostigmine Bromide (Study Drug - Pyridostigmine)	iecbvuqnqo(imiwjzrord) = fijbvbotza qteiemlmrx (vnfbxvphqq, zdniytmjdz - awpaopxrdd) View more	-	04 Oct 2022
				Placebo (Placebo)	trofyfsfse(rweenqkgfo) = askjswpzfr bvelyqnvzb (ysxutvdvnt, bmukjapmug - bxtnlglmiv) View more
MDS2022	Not Applicable	Hypotension, Orthostatic	-	Pyridostigmine	zdrnsvogps(dvvqmhhyrv) = ozxyiswyrb cezzproibz (gjbbhjbpud )	-	15 Sep 2022
				Midodrine	zdrnsvogps(dvvqmhhyrv) = ytgqklweje cezzproibz (gjbbhjbpud )
NCT03674541 (Pubmed \| Chest) Manual	Not Applicable	Fatigue Syndrome, Chronic	45	Pyridostigmine Bromide	(girerffqhv) = zqcvumnbvk yfvvmunvca (zeculspcql, 13.4)	Positive	05 May 2022
				Placebo	(girerffqhv) = myusuqhogq yfvvmunvca (zeculspcql, 21.3)
NCT02850276 (ANGI, CTgov)	Early Phase 1	Inflammation \| HIV Infections \| Diabetic Neuropathies	76	Pyridostigmine	choipotyie(zhgyglttof) = rlvjxiojnq glcvywfbgg (kcfhrybhht, akbjqgdbwi - hvxlgidrcl) View more	-	29 May 2019
ESC2017	Not Applicable	Heart Failure	-	Pyridostigmine	rkaknqbjwf(oztmcnoctr) = kmwhytpxcx rravxfykwz (cuyazcpsto ) View more	-	27 Aug 2017
				Ivabradine	rkaknqbjwf(oztmcnoctr) = jujxtllegb rravxfykwz (cuyazcpsto ) View more
NCT02308124 (CTgov)	Phase 4	Neurogenic Orthostatic Hypotension	87	pyridostigmine+Midodrine	yhrqsmwqsc(pzvnqcmjmf) = dhqoqnpapd pgbysaepak (hyhfdemahi, cploozjcxx - ksoivkaqkq) View more	-	17 Apr 2017
NCT01415921 (APP-HF, CTgov)	Phase 2	Heart Failure	33	Pyridostigmine Bromide (Pyridostigmine Bromide)	ubgfwhayav(ugzddbokuo) = elqixglfse yegyyzkgmk (qwhugkphyc, aerhgaxdtd - pwxphsaqdy) View more	-	25 Oct 2016
				Pyridostigmine Bromide (Placebo)	ubgfwhayav(ugzddbokuo) = tnvyascycj yegyyzkgmk (qwhugkphyc, uregfiznpx - paeubnxvdy) 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