ALS Functional Rating Scale - Revised

Landmark Study Finds Pison AI Technology Can Detect Neurological Disorder in ALS Patients

Retrieved on: 
Tuesday, April 18, 2023
Science, Neurology, Other Science, Research, Health, Artificial Intelligence, Technology, Other Technology, Temple University, Electromyography, ENG, Deep learning, National Science Foundation, Clinical trial, DSM-IV codes, Data collection, Temple, Fasciculation, Multiple sclerosis, Disorder, ALS Association, Associate, Harvard Medical School, The, Iran National Science Foundation, HIV disease progression rates, Patient, ALS Functional Rating Scale - Revised, FRS, Algorithm, Diagnosis, Hospital, ALS, Disease, Medical imaging, Pison, Brigham, Medicine

These results demonstrate that Pison’s technology has the potential to become a digital biomarker to detect neurological diseases, monitor progression, and inform treatment.

Key Points: 
  • These results demonstrate that Pison’s technology has the potential to become a digital biomarker to detect neurological diseases, monitor progression, and inform treatment.
  • The study found that Pison’s technology could distinguish between patients diagnosed with ALS and a healthy population.
  • The technology demonstrated that it could detect fasciculations and differentiation between healthy people and people living with ALS in a resting state using deep learning algorithms.
  • “This clinical result is an important step towards receiving regulatory approval for medical applications of Pison technology.

Cytokinetics Announces COURAGE-ALS Met Criteria for Futility at Second Interim Analysis

Retrieved on: 
Friday, March 31, 2023
Microsoft Access, Failure mode and effects analysis, Edaravone, Data monitoring committee, Telephone, Clinical trial, Muscle weakness, Vital capacity, Cytokinetics, ALS Functional Rating Scale - Revised, Patient, SAN, Riluzole, Clutch (eggs), DMC, Caregiver, Pharmaceutical industry, Medical imaging, ALS

SOUTH SAN FRANCISCO, Calif., March 31, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the Data Monitoring Committee (DMC) for COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), recently convened to conduct the second planned interim analysis of this Phase 3 clinical trial.

Key Points: 
  • In addition, Cytokinetics plans to discontinue treatment with reldesemtiv in all patients including those in the open-label extension study, COURAGE-ALS OLE.
  • At the interim analysis, approximately 460 patients had been randomized and over 200 had reached the 24-week assessment of the trial endpoints.
  • The first interim analysis assessed for futility, 12 weeks after approximately one-third or more of the planned sample size were randomized.
  • The second interim analysis assessed for futility with the option for a fixed increase in total enrollment, if it had been deemed necessary, to augment the statistical power of the trial.

Coya Therapeutics, Inc. Announces Positive Results from a Proof-of-Concept Academic Clinical Study for COYA 302 in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Tuesday, March 21, 2023
Biotechnology, Pharmaceutical, Managed Care, General Health, Health, Hospitals, Clinical Trials, Other Health, Serum, Disability, Inflammation, Biomarker, COYA, Oxidative stress, ALS Functional Rating Scale - Revised, HIV disease progression rates, Patient, Revise, Therapy, ALS, Safety, Disease, Vaccine, Silviculture

Following the administration of COYA 302 for 48 weeks, patients were evaluated over an 8-week washout period.

Key Points: 
  • Following the administration of COYA 302 for 48 weeks, patients were evaluated over an 8-week washout period.
  • During the 48-week treatment period, COYA 302 appeared to be well tolerated.
  • Preliminary efficacy of COYA 302 was measured by the ALSFRS-R scale, a validated rating tool for monitoring the progression of disability in patients with ALS.
  • “We plan to file an IND with the FDA in the second half of 2023 and initiate a clinical study soon thereafter,” Dr. Hepner added.

BrainStorm Cell Therapeutics Announces Third Quarter 2022 Financial Results and Provides a Corporate Update

Retrieved on: 
Wednesday, November 23, 2022
College of Medicine, Adult, Doctor of Philosophy, Hospital, Brainstorm, ALS, Neurodegeneration, Neuroinflammation, Massachusetts General Hospital, MSC, Therapy, MS, BLA, NEW, Biomarker, Advanced Cell Therapeutics, Research, Cerebrospinal fluid, ALS Functional Rating Scale - Revised, Conference call, FDA, Neuroprotection, Multiple sclerosis, Harvard Medical School, Multiple Sclerosis Journal, Type, Patient, Webcast, American Society for Pharmacology and Experimental Therapeutics, European Committee of the Regions, NASDAQ, Congress, HIV disease progression rates, Treatment, Food, Method, Regulation of tobacco by the U.S. Food and Drug Administration, Cleveland Clinic, Pharmaceutical industry, Renewable energy, Reinsurance, Conference, MD, Neurology

NEW YORK, Nov. 14, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the third quarter ended September 30, 2022, and provided a corporate update.

Key Points: 
  • Conference call and webcast at 8:00 a.m. Eastern Time today
    NEW YORK, Nov. 14, 2022 /PRNewswire/ --BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the third quarter ended September 30, 2022, and provided a corporate update.
  • Financial Results for the Third Quarter Ended September 30, 2022
    Cash, cash equivalents, and short-term bank deposits were approximately $7.4 million as of September 30, 2022, compared to $12.2 million as of June 30, 2022.
  • Research and development expenses for the three months ended September 30, 2022, and 2021 were approximately $3.8 million and $3.6 million, respectively.
  • Net loss per share for the three months ended September 30, 2022, and 2021 was $0.19 and $0.15, respectively.

Corcept Therapeutics Initiates DAZALS – A Phase 2 Trial in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Tuesday, October 11, 2022
Quality of life, Doctor of Pharmacy, SEC, Liver disease, NASDAQ, Patient, University Medical Center Utrecht, Amyotrophic lateral sclerosis, ALS, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Goal, Probability, ALS Functional Rating Scale - Revised, Neurology, Corcept Therapeutics, Intention, Weight gain, Safety, Neuroinflammation, GLOBE, Intellectual property, Muscle, CORT, COVID-19, Pharmaceutical industry

"We are excited to initiate this important study in collaboration with TRICALS, the leading ALS academic consortium in Europe."

Key Points: 
  • "We are excited to initiate this important study in collaboration with TRICALS, the leading ALS academic consortium in Europe."
  • "I am excited to partner with Corcept to study dazucorilant's potential to significantly improve treatment outcomes for people living with ALS."
  • DAZALS is a 198-patient, randomized, double-blind, placebo-controlled Phase 2 trial of dazucorilant in patients with ALS.
  • We disclaim any intention or duty to update forward-looking statements made in this press release.

Cytokinetics Announces Continuation of COURAGE-ALS Following First Interim Analysis

Retrieved on: 
Monday, October 10, 2022
SAN, LinkedIn, Pharmacology, Skeleton, Patent, Mechanics, YouTube, Twitter, HIV disease progression rates, Facebook, Private Securities Litigation Reform Act, HCM, Heart failure, Incidence, Death, GLOBE, Riluzole, Clinical trial, Vital capacity, Tropomyosin, FDA, DMC, Data monitoring committee, Failure rate, Hospital, Diagnosis, Particle-size distribution, Muscle weakness, Act, Calcium, Edaravone, Security (finance), History, The Act, Safe harbor, U.S. Securities and Exchange Commission, Survival, Neurodegeneration, Intellectual property, Life expectancy, Sale, Authorization, Patient, Amyotrophic lateral sclerosis, ALS Functional Rating Scale - Revised, Safety, Protein, Cytokinetics, FSTA, Pharmaceutical industry, ALS

The first interim analysis was triggered 12 weeks after approximately one-third or more of the intended number of patients were randomized to participate in COURAGE-ALS.

Key Points: 
  • The first interim analysis was triggered 12 weeks after approximately one-third or more of the intended number of patients were randomized to participate in COURAGE-ALS.
  • The first interim analysis assessed for futility, 12 weeks after approximately one-third or more of the planned sample size is randomized.
  • For further information regarding these and other risks related to Cytokinetics' business, investors should consult Cytokinetics' filings with the Securities and Exchange Commission.
  • CYTOKINETICS and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries.

Clene Reports Topline Results Demonstrating Survival Signal for CNM-Au8® in Healey ALS Platform Trial

Retrieved on: 
Monday, October 3, 2022
SVC, Master of Science, Hospital, Review, Nasdaq, Drug Quality and Security Act, Respiration (physiology), Brain, Annual report, EMA, FAAN, Marketing, Population, Neurodegeneration, Research, CEO, Investor, R, CNM, Leadership, Biomarker, Patient, Multiple sclerosis, EAP, ALS, Massachusetts General Hospital, Amyotrophic lateral sclerosis, Healey, MS, Department, SALT, Twitter, ALS Functional Rating Scale - Revised, FDA, Webcast, Lists of diseases, Risk, GLOBE, Ventilation, Securities Exchange Act of 1934, Function, Mortality, Department of Neurology, Xuanwu hospital, LinkedIn, RCT, AMG, VAMP regimen, Death, Harvard Medical School, CAFS, Neurology, Safety, Efficiency, EDT, Securities Act of 1933, Remyelination, Neuroprotection, Pharmaceutical industry, Aluminium, Survival, Facebook

This survival signal is consistent with results previously reported by Clene in the Phase 2 RESCUE-ALS trial with CNM-Au8.

Key Points: 
  • This survival signal is consistent with results previously reported by Clene in the Phase 2 RESCUE-ALS trial with CNM-Au8.
  • Based on these topline findings, Clene has selected the CNM-Au8 30 mg dose for continued development in ALS.
  • Michael Hotchkin, Clenes Chief Development Officer, concluded, We thank the ALS community for its support of the Healey ALS Platform trial.
  • Clene will host a conference call and webcast at 8:30 am EDT to discuss the Healey ALS Platform trial topline results for CNM-Au8.

Cellenkos Receives FDA Clearance of Investigational New Drug (IND) Application for CK0803 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Wednesday, September 21, 2022
Neuro, Central nervous system, CAFS, Temperature, Safety, Muscle, Degenerative disease, COVID-19, Platelet transfusion, MD, Food, FDA, Columbia University, Inflammatory bowel disease, Blood, Eleanor, ALS Functional Rating Scale - Revised, Lists of diseases, Speech, IND, Ruxolitinib, Lupus nephritis, Survival, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Travel, Aplastic anemia, Doctor of Philosophy, Amyotrophic lateral sclerosis, ALS, Inflammation, Neuron, Disease, Pharmaceutical industry, Regulatory T cell, Incyte

HOUSTON, Sept. 21, 2022 /PRNewswire/ -- Cellenkos, Inc., a clinical stage biotech company that focuses on developing transformative cellular therapeutics for treatment of inflammatory disorders and autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to initiate a Phase 1 safety study followed by a Phase 1b randomized, double blind, placebo control  trial of CK0803, neurotrophic allogeneic Treg cells, in patients with amyotrophic lateral sclerosis (ALS). 

Key Points: 
  • The primary objective of the upcoming Phase 1 study is to establish safety and tolerability of multiple doses of CK0803 administered intravenously in patients with ALS.
  • "Cellenkos has made an extraordinary commitment to ALS and I am pleased to partner with them on this important therapeutic trial.
  • "We are thrilled to have received IND clearance for our CK0803 program in ALS," said Tara Sadeghi, Chief Operating Officer of Cellenkos.
  • Cellenkos is a clinical-stage biotechnology company dedicated to the development and commercialization of cellular therapeutics for treatment of inflammatory diseases and autoimmune disorders.

BioJiva Reports Results of Pilot Phase 2 Clinical Trial of RT001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, September 15, 2022
ALS Functional Rating Scale - Revised, ALS, Week, GLOBE, Degenerative disease, RT001, Patient, Amyotrophic lateral sclerosis, Pharmaceutical industry

LOS ALTOS, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) -- BioJiva, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today reported data from its completed multicenter, randomized, double-blind, placebo-controlled pilot Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease). The trial’s prespecified primary endpoint was change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) following 24 weeks of treatment. At 24 weeks, data demonstrated that patients treated with RT001 experienced less worsening (a 3.3-point reduction from baseline) in ALSFRS-R score as compared to greater worsening (4.6-point reduction from baseline) for the placebo group. Similarly, patients treated with RT001 experienced less worsening (a 13.3-point increase from baseline) in their score on the 40-item ALS assessment questionnaire (ALSAQ40) as compared to the placebo group (a 17.2-point increase from baseline). While suggesting a signal of directional improvement with RT001 treatment, these findings did not achieve statistical significance due to the small size of the study.

Key Points: 
  • While suggesting a signal of directional improvement with RT001 treatment, these findings did not achieve statistical significance due to the small size of the study.
  • This six-month randomized Phase 2 pilot study ( NCT04762589 ), which was followed by a six-month open-label extension during which all patients received RT001, was conducted at four ALS Centers of Excellence in Europe.
  • Investigators enrolled 43 patients with ALS who had symptom duration of less than three years.
  • BioJiva is not a progression of Retrotope and BioJiva is not the former Retrotope.

New Data from Clene’s RESCUE-ALS Study to be Presented at 2022 ENCALS Meeting

Retrieved on: 
Wednesday, June 1, 2022
OLE, Survival, Caregiver, EDT, Marketing, European Network of Transmission System Operators for Electricity, Securities Exchange Act of 1934, Drug Quality and Security Act, R, LinkedIn, Plasma, Annual report, CEO, Neurodegeneration, Neuroprotection, Amyotrophic lateral sclerosis, Hope, Respiration (physiology), Creatine, Quality of life, Patient, Mortality, GLOBE, Risk, ALS, Review, Lists of diseases, Remyelination, NASDAQ, Data, Twitter, Securities Act of 1933, Gold, CNM, ALS Functional Rating Scale - Revised, L1, SALT, Interim, Safety, Pharmaceutical industry, Silviculture, Holding company, Beer, Facebook

The results from the RESCUE- ALS study provide evidence of safety and suggest efficacy of CNM-Au8 in patients with early ALS.

Key Points: 
  • The results from the RESCUE- ALS study provide evidence of safety and suggest efficacy of CNM-Au8 in patients with early ALS.
  • Furthermore, during the OLE, subjects originally randomized to CNM-Au8 continued to maintain stable ALSSQOL-SF scores through 84 weeks or more of treatment.
  • These emerging data highlight the significantly reduced risk of mortality following early and sustained treatment with CNM-Au8., said Rob Etherington, President and CEO of Clene.
  • We look forward to the next major data readout from the HEALEY ALS platform trial that is expected in the third quarter.