Vital capacity

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

Retrieved on: 
Thursday, April 4, 2024
Vital capacity, MuSK protein, Glucocorticoid, NMPA, Hormone, GMG, Disease, Chinese, MRS, BCMA, Nervous system, Patient, SAN, Enzyme inhibitor, History, Female, Multiple myeloma, CRS, B cell, Biotechnology, IND, Phenotype, Investigational New Drug, QMG, Safety, Thymectomy, FDA, Food

The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.

Key Points: 
  • The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.
  • The 2 subjects were treated with a single infusion of Eque-cel at the doses of 1.0×106 CAR-T/Kg, respectively.
  • No immunomodulatory therapy other than low dose pyridostigmine (90 mg/day and 60 mg/day, respectively) was used during the follow-up period.
  • Anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies decreased rapidly and maintained at very low levels in both subjects after infusion.

C-Path Launches Clinical Trial Simulator for Duchenne Muscular Dystrophy Research

Retrieved on: 
Thursday, March 28, 2024
Research, Vital capacity, Atrophy, Disease, Data analysis, Letter, Pharming, Female, European Medicines Agency, Duchenne muscular dystrophy, Clinical trial, Drug development, FCP, FDA, DMD, CTS, Food, Pharmaceutical industry

This pioneering Drug Development Tool is set to positively impact the medical research community by significantly optimizing clinical trial design.

Key Points: 
  • This pioneering Drug Development Tool is set to positively impact the medical research community by significantly optimizing clinical trial design.
  • This Clinical Trial Simulator will positively impact DMD research by significantly optimizing clinical trial design.
  • "The launch of the DMD Clinical Trial Simulator represents a significant step forward in our fight against Duchenne muscular dystrophy," said Ramona Belfiore-Oshan, Ph.D., Executive Director for D-RSC.
  • Granting access to the D-RSC Clinical Trial Simulator through RDCA-DAP creates a unique opportunity for innovative trial designs in DMD," said Alexandre Bétourné, Pharm.D., Ph.D., Executive Director, RDCA-DAP.

Data from Brainomix's Collaboration with AstraZeneca Shows its AI-Powered e-Lung Better Identifies Lung Fibrosis Patients at Risk of Decline

Retrieved on: 
Wednesday, March 27, 2024
AJRCCM, Medical research, ILD, AstraZeneca, ATS, Survival, Data science, Critical Care Medicine (journal), Partnership, Risk, Boehringer Ingelheim, Royal Brompton Hospital, Patient, American Journal, Lung, WRVS, Biopharmaceutical, Biomarker, Vital capacity, American Thoracic Society, Idiopathic pulmonary fibrosis, IPF, Clinical trial, Tralokinumab, Idiopathic disease, FVC

The study was an analysis of AstraZeneca's Phase 2 52-Week clinical trial of tralokinumab in patients with Idiopathic Pulmonary Fibrosis (IPF).

Key Points: 
  • The study was an analysis of AstraZeneca's Phase 2 52-Week clinical trial of tralokinumab in patients with Idiopathic Pulmonary Fibrosis (IPF).
  • The patient data from the trial was processed with Brainomix's e-Lung tool.
  • The tool is uniquely powered by the weighted reticulovascular score (WRVS), a novel biomarker that incorporates reticular opacities and vascular structures of the lung.
  • WRVS was also able to predict both short- and long-term outcomes of the patients, including lung function decline and survival.

Data from Brainomix's Collaboration with AstraZeneca Shows its AI-Powered e-Lung Better Identifies Lung Fibrosis Patients at Risk of Decline

Retrieved on: 
Wednesday, March 27, 2024
AJRCCM, Medical research, ILD, AstraZeneca, ATS, Survival, Data science, Critical Care Medicine (journal), Partnership, Risk, Boehringer Ingelheim, Royal Brompton Hospital, Patient, American Journal, Lung, WRVS, Biopharmaceutical, Biomarker, Vital capacity, American Thoracic Society, Idiopathic pulmonary fibrosis, IPF, Clinical trial, Tralokinumab, Idiopathic disease, FVC

The study was an analysis of AstraZeneca's Phase 2 52-Week clinical trial of tralokinumab in patients with Idiopathic Pulmonary Fibrosis (IPF).

Key Points: 
  • The study was an analysis of AstraZeneca's Phase 2 52-Week clinical trial of tralokinumab in patients with Idiopathic Pulmonary Fibrosis (IPF).
  • The patient data from the trial was processed with Brainomix's e-Lung tool.
  • The tool is uniquely powered by the weighted reticulovascular score (WRVS), a novel biomarker that incorporates reticular opacities and vascular structures of the lung.
  • WRVS was also able to predict both short- and long-term outcomes of the patients, including lung function decline and survival.

NS Pharma, Inc. Shares New VILTEPSO® (Viltolarsen) Data at the MDA Clinical & Scientific Conference 2024

Retrieved on: 
Wednesday, March 6, 2024
Duchenne, Research, MD, Duchenne muscular dystrophy, Trial of the century, Vital capacity, Conference, Annual general meeting, AAN, Muscular Dystrophy Association, Patient, FVC, Poster, MDA, Episcopal conference, American Academy, Pharmaceutical industry

PARAMUS, N.J., March 6, 2024 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6. The company presented a poster entitled "Pulmonary and motor function in ambulatory and non-ambulatory participants with Duchenne muscular dystrophy (Duchenne) treated with viltolarsen (VILTEPSO®)" which covers data from the Galactic53 trial demonstrating that the majority of participants receiving viltolarsen experienced meaningful benefit in pulmonary function, including percent predicted forced vital capacity (FVC%p).

Key Points: 
  • PARAMUS, N.J., March 6, 2024 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma) is excited to announce participation in the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida, March 3 – 6.
  • "Galactic53 is the first trial with VILTEPSO to evaluate pulmonary function in participants with Duchenne," explains NS Pharma Vice President Medical Affairs & Pharmacovigilance Leslie Magnus, MD, who also co-authored the poster.
  • "Our team is encouraged by these results and will continue our research into treatments for rare disease."
  • Additional data from this study will also be presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, April 13 – 18 in Denver, Colorado and online.

Cytokinetics Presents Results From COURAGE-ALS at the 34th International Symposium on ALS/MND

Retrieved on: 
Thursday, December 7, 2023
Patient, Research and development, HHD, Caregiver, Hope, Weakness, Disease, Incidence, Muscle weakness, SAN, Edaravone, Clinical trial, University, Barrow Neurological Institute, FVC, COVID-19, Death, Data monitoring committee, Infection, Weather, Transport, Riluzole, Vital capacity, General Grievous, Cytokinetics, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the full results of COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), were presented at the 34th International Symposium on ALS/MND by Jeremy Shefner, M.D., Ph.D., Lead Investigator of COURAGE-ALS, Professor of Neurology, Barrow Neurological Institute, University of Arizona College of Medicine Phoenix.

Key Points: 
  • At the second interim analysis the Data Monitoring Committee recommended the discontinuation of the clinical trial due to futility.
  • At the time of the discontinuation of COURAGE-ALS, 486 patients had started treatment with reldesemtiv or placebo and 276 had completed dosing through 24 weeks.
  • Patients treated with reldesemtiv declined 5.3 points per month (SD=5.3) while patients treated with placebo declined 4.8 points per month (SD=4.4).
  • The survey results showed that site personnel viewed remote visits favorably when considering whether to participate as a site in COURAGE-ALS.

Avalyn Presents Data from its AP01 and AP02 Inhaled Therapeutics Programs for Pulmonary Fibrosis

Retrieved on: 
Thursday, November 9, 2023
Lazarus, Pulmonary fibrosis, Lake, HRCT, Idiopathic pulmonary fibrosis, IPF, FVC, Summit, OLE, Disease, Caregiver, ATLAS, ILD, Vital capacity, Safety, Patient, PFF, FCCP, Outline, Pharmaceutical industry

SEATTLE, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Avalyn Pharma Inc., a clinical-stage biopharmaceutical company focused on development of targeted inhalation therapies for life-threatening pulmonary diseases, today announced the presentation of preclinical data that supported the completed Phase 1a trial of AP02 (inhaled nintedanib) as well as clinical data for AP01 (inhaled pirfenidone) at the 2023 Pulmonary Fibrosis Foundation (PFF) Summit at the JW Marriott Grande Lakes in Orlando, FL.

Key Points: 
  • “We are excited to engage with the pulmonary fibrosis community at the PFF Summit, including people living with the disease, their caregivers and the expert providers that support them, and to share data for our inhaled treatment candidates AP01 and AP02,” said Howard M. Lazarus, M.D.
  • These data demonstrated that inhaled nintedanib was well-tolerated, was indicative of efficient inhaled alveolar deposition, and delivered oral-superior lung levels with reduced systemic exposure.
  • Avalyn will also review the results from the Phase 1b trial of AP01 (ATLAS) in participants with idiopathic pulmonary fibrosis (IPF).
  • The OLE enrolled ATLAS participants and AP01-naïve pulmonary fibrosis participants, and the data showed a durable and consistent response to AP01, including fewer reported adverse events compared to historical reports of oral pirfenidone.

Bristol Myers Squibb Announces U.S. FDA Breakthrough Therapy Designation for Investigational LPA1 Antagonist for Progressive Pulmonary Fibrosis

Retrieved on: 
Tuesday, October 24, 2023
FDA, Health, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, LPA1, BMY, Bristol Myers Squibb, ERS, Second International Congress on Education of the Deaf, LPAR1, Pulmonary fibrosis, Respiratory failure, NYSE, Food, Breakthrough, Head, MD, PPF, Idiopathic pulmonary fibrosis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IPF, Circulatory system, Quality of life, Vital capacity, Safety, European Respiratory Society, Death, Pharmaceutical industry, Immunology

Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for BMS-986278, a potential first-in-class, oral, lysophosphatidic acid receptor 1 (LPA1) antagonist, for the treatment of progressive pulmonary fibrosis (PPF), a devastating, life-threatening illness.

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for BMS-986278, a potential first-in-class, oral, lysophosphatidic acid receptor 1 (LPA1) antagonist, for the treatment of progressive pulmonary fibrosis (PPF), a devastating, life-threatening illness.
  • The Breakthrough Therapy Designation is based on results from the global, randomized Phase 2 study that assessed the safety and efficacy of BMS-986278 treatment versus placebo in people living with idiopathic pulmonary fibrosis (IPF) and PPF.
  • “The FDA’s Breakthrough Therapy Designation underscores the potential of BMS-986278 as an innovative, first-in-class treatment that may redefine the standard of care for progressive pulmonary fibrosis.”
    In addition to this Breakthrough Therapy Designation for PPF, the U.S. FDA has also previously granted BMS-986278 fast-track designation and orphan drug designation for the treatment of IPF.
  • Bristol Myers Squibb is continuing the development of BMS-986278 with the global Phase 3 ALOFT program for PPF ( NCT06025578 ) and IPF ( NCT06003426 ).

PureTech Presents Data from LYT-100 (Deupirfenidone) Trial in Healthy Older Adults at CHEST Annual Meeting

Retrieved on: 
Wednesday, October 11, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, PureTech Health, Keck School of Medicine of USC, Incidence, Central nervous system, Aes, IPF, University, Survival, University of Southern California, Vital capacity, FDA, Idiopathic pulmonary fibrosis, PRTC, LSE, TID, CNS, Patient, Poster, Pharmaceutical industry, Medicine

PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, presented clinical data supporting the differentiated profile of LYT-100 (deupirfenidone) at the CHEST Annual Meeting in Honolulu, Hawaii.

Key Points: 
  • PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, presented clinical data supporting the differentiated profile of LYT-100 (deupirfenidone) at the CHEST Annual Meeting in Honolulu, Hawaii.
  • The presentation expands on data from a completed trial of LYT-100 in healthy older adults, which informed the two doses selected for the ongoing, global Phase 2b dose-ranging trial of LYT-100 (ELEVATE IPF) in patients with idiopathic pulmonary fibrosis (IPF).
  • The trial showed that a 550 mg dose of LYT-100 given three times daily (TID) provided bioequivalent drug exposure to the FDA-approved dose of pirfenidone, 801 mg TID.
  • LYT-100 also demonstrated a 24% lower peak drug concentration than pirfenidone, which is a key factor generally associated with tolerability.

Avalyn Raises $175 Million in Oversubscribed Series C Financing to Advance Inhaled Pulmonary Fibrosis Programs into Later Stage Clinical Studies

Retrieved on: 
Wednesday, September 27, 2023
Citadel, SR, Pivotal, Norwest Venture Partners, Survival, Weight loss, Rash, Pulmonary fibrosis, Vital capacity, Novo Holdings A/S, Patient, Series, Wellington Management Company, Mortality, Nausea, Photosensitivity, IPF, Idiopathic pulmonary fibrosis, Fatigue, Financial services, Pharmaceutical industry

SEATTLE, Sept. 27, 2023 (GLOBE NEWSWIRE) -- Avalyn Pharma Inc., a clinical-stage biopharmaceutical company focused on development of targeted inhalation therapies for life-threatening pulmonary diseases, announced the closing of an oversubscribed $175 million Series C financing. Avalyn plans to use the funds to continue development of its portfolio of inhalation therapies for interstitial lung disease that improve upon currently approved medications, and advance lead clinical assets, AP01 (inhaled pirfenidone) and AP02 (inhaled nintedanib), into mid-stage clinical trials.

Key Points: 
  • Current investors, including Novo Holdings A/S, Norwest Venture Partners, F-Prime Capital, Pivotal bioVenture Partners, and RiverVest Venture Partners, also participated.
  • In conjunction with the financing, Gianna Hoffman-Luca, Ph.D., of Perceptive Xontogeny Venture Funds, and Jill Carroll of SR One, joined Avalyn’s Board of Directors .
  • In clinical trials to date, inhaled AP01 has been well-tolerated and demonstrated enhanced efficacy , when compared to published results of oral pirfenidone.
  • Avalyn plans to advance AP01 into a Phase 2b trial and AP02 into a Phase 2a trial for patients with pulmonary fibrosis.