Amyotrophic lateral sclerosis

Stevens Nation Responds to FDA's Refusal to File Letter, NurOwn Recipients Call for Advisory Committee Meeting

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Tuesday, November 22, 2022
Authorization, Muscle weakness, CDER, Brainstorm, Therapy, Camping, Atrophy, Eating, Refusal, FDA, Amyotrophic lateral sclerosis, Pulmonology, EAPS, Spinal cord, University of California, Irvine, Expanded access, RTF, UC, Advisory Committee, CDC, Patient, Phase, Aerospace, Walking, Motor neuron, Neurology, Brain, Life expectancy, Letter, Food, Exercise, Disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, EAP, Pharmaceutical industry, Medical device, Renewable energy, ALS

WASHINGTON, Nov. 21, 2022 /PRNewswire/ -- Brainstorm Cell Therapeutics is seeking FDA approval of its mesenchymal stem cell therapy called NurOwn for the treatment of ALS. In response to the FDA's Refusal to File letter, and as people who participated in the NurOwn trials and its Expanded Access program ("EAP"), we support Brainstorm's efforts to seek an Advisory Committee ("AdCom") meeting.

Key Points: 
  • In response to the FDA's Refusal to File letter, and as people who participated in the NurOwn trials and its Expanded Access program ("EAP"), we support Brainstorm's efforts to seek an Advisory Committee ("AdCom") meeting.
  • We must insist that the FDA allow testimony about the "clinically meaningful" changes that occurred when we received NurOwn.
  • However, the Phase III trial did meet a pre-specified secondary endpoint in a subgroup of patients with less advanced ALS.
  • The best people to attest to how NurOwn made them "feel and function" are the people who received NurOwn.

BioArctic expands portfolio with alpha-synuclein Brain Transporter project

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Thursday, November 17, 2022
Neurodegeneration, Person, University, Technology, ALS, Alzheimer's disease, Partnership, Safety, Amyotrophic lateral sclerosis, Leg, Depression, Nasdaq, Amyotrophy, CET, Nervous system, Parkinson's disease, CEO, Eisai, Alpha-synuclein, Walking, Brain, Tremor, Vaccine, Pharmaceutical industry, Uppsala University

STOCKHOLM, Nov. 17, 2022 /PRNewswire/ -- BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) today announced that the company is expanding its alpha-synuclein project portfolio.

Key Points: 
  • STOCKHOLM, Nov. 17, 2022 /PRNewswire/ -- BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) today announced that the company is expanding its alpha-synuclein project portfolio.
  • The new project, PD-BT2238, combines a selective alpha-synuclein oligomer targeting antibody with BioArctic's proprietary Brain Transporter technology, to increase exposure of the antibody in the brain, with the aim of increasing efficacy of a potential treatment in Parkinson's disease.
  • The PD-BT2238 project is a second-generation alpha-synuclein antibody, combining an oligomer-selective alpha-synuclein antibody with BioArctic's Brain Transporter technology, designed to improve brain exposure.
  • "BioArctics proprietary Brain Transporter technology has the opportunity to improve brain exposure and thereby the efficacy of biologic treatments for various neurodegenerative diseases.

BioArctic expands portfolio with alpha-synuclein Brain Transporter project

Retrieved on: 
Thursday, November 17, 2022
Neurodegeneration, Person, University, Technology, ALS, Alzheimer's disease, Partnership, Safety, Amyotrophic lateral sclerosis, Leg, Depression, Nasdaq, Amyotrophy, CET, Nervous system, Parkinson's disease, CEO, Eisai, Alpha-synuclein, Walking, Brain, Tremor, Vaccine, Pharmaceutical industry, Uppsala University

STOCKHOLM, Nov. 17, 2022 /PRNewswire/ -- BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) today announced that the company is expanding its alpha-synuclein project portfolio.

Key Points: 
  • STOCKHOLM, Nov. 17, 2022 /PRNewswire/ -- BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) today announced that the company is expanding its alpha-synuclein project portfolio.
  • The new project, PD-BT2238, combines a selective alpha-synuclein oligomer targeting antibody with BioArctic's proprietary Brain Transporter technology, to increase exposure of the antibody in the brain, with the aim of increasing efficacy of a potential treatment in Parkinson's disease.
  • The PD-BT2238 project is a second-generation alpha-synuclein antibody, combining an oligomer-selective alpha-synuclein antibody with BioArctic's Brain Transporter technology, designed to improve brain exposure.
  • "BioArctics proprietary Brain Transporter technology has the opportunity to improve brain exposure and thereby the efficacy of biologic treatments for various neurodegenerative diseases.

NeuroSense Receives Regulatory Approval to Commence Patient Enrollment in Italy for its Phase 2b Trial in ALS

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Wednesday, November 16, 2022
Quality of life, Celecoxib, RNA, ALS, EMA, European Medicines Agency, Massachusetts General Hospital, Forward-looking statement, Ciprofloxacin, Safety, Neurodegeneration, FDA, Paralysis, Hope, Company, Italian Medicines Agency, Therapy, Patent, Death, Patient, Parkinson's disease, NASDAQ, Amyotrophic lateral sclerosis, Inflammation, Nature, SOC, Alzheimer's disease, Diagnosis, NDE, Food, Biomarker, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, EU

The Phase 2b ( NCT05357950 ) double-blind, placebo-controlled, multinational clinical trial aims to assess PrimeC's efficacy, as well as safety and tolerability, in people living with ALS.

Key Points: 
  • The Phase 2b ( NCT05357950 ) double-blind, placebo-controlled, multinational clinical trial aims to assess PrimeC's efficacy, as well as safety and tolerability, in people living with ALS.
  • The study is enrolling and randomizing 69 people living with ALS in a 2:1 ratio to receive PrimeC or placebo, respectively.
  • Primary endpoints of the study include assessment of ALS-biomarkers, evaluation of clinical efficacy, and improvement in quality of life.
  • Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate.

NeuroSense Receives FDA Clearance of IND for its ALS Phase 2b Study

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Tuesday, November 15, 2022
Quality of life, Celecoxib, RNA, ALS, EMA, European Medicines Agency, Forward-looking statement, Union, Ciprofloxacin, Safety, Neurodegeneration, FDA, Paralysis, Company, Investigational New Drug, Therapy, Patent, Death, Patient, Parkinson's disease, NASDAQ, Amyotrophic lateral sclerosis, Inflammation, Nature, SOC, Clinical trial, Alzheimer's disease, IND, Diagnosis, NDE, Food, Biomarker, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, EU

PARADIGM, ( NCT05357950 ), a Phase 2b double-blind, placebo-controlled, multinational clinical trial, aims to assess PrimeC's efficacy, as well as safety and tolerability, in people living with ALS.

Key Points: 
  • PARADIGM, ( NCT05357950 ), a Phase 2b double-blind, placebo-controlled, multinational clinical trial, aims to assess PrimeC's efficacy, as well as safety and tolerability, in people living with ALS.
  • The study is enrolling and randomizing 69 people living with ALS in a 2:1 ratio to receive PrimeC or placebo, respectively.
  • Primary and secondary endpoints of the study include assessment of ALS-biomarkers, evaluation of clinical efficacy, and improvement in quality of life.
  • NeuroSense expects to enroll and dose the first U.S. patients in the next few weeks at leading ALS centers on both the east and west coast.

Yumanity Therapeutics Announces Effectiveness of Registration Statement on Form S-4 In Connection With Proposed Asset Sale to Janssen and Merger with Kineta, Inc.

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Thursday, November 10, 2022
Food and Drug Administration Amendments Act of 2007, Ix, Risk, Sale, Growth, Therapy, Janssen Pharmaceuticals, Company, Registration statement, Merck & Co., Ownership, Special, Prospectus, Securities Act of 1933, SEC, Compte rendu, Form, U.S. Securities and Exchange Commission, Cancer, Patient, Private Securities Litigation Reform Act, Phrase, Amyotrophic lateral sclerosis, Merck, Proxy statement, FTLD, Johnson & Johnson, Muscle atrophy, Toxicity, Register, PIPE, GLOBE, VISTA, Nature, CBI, Failure, Proxy, ALS, Neurodegeneration, Security (finance), Pharmaceutical industry, Property management, Holding company, Nasdaq, Janssen, Kineta, Shareholder, Yuman–Cochimí languages

Yumanity will mail the definitive proxy statement/prospectus (the Proxy Statement) to stockholders of record as of the close of business on November 4, 2022.

Key Points: 
  • Yumanity will mail the definitive proxy statement/prospectus (the Proxy Statement) to stockholders of record as of the close of business on November 4, 2022.
  • This press release may be deemed to be solicitation material with respect to the proposed transactions between Yumanity and Kineta and between Yumanity and Janssen.
  • In connection with the proposed transactions, on August 29, 2022, Yumanity filed with the U.S. Securities and Exchange Commission (the SEC) a registration statement on Form S-4 (the Initial Registration Statement), as amended by Amendment No.
  • 3 to the Initial Registration Statement filed with the SEC on November 4, 2022 (together with the Initial Registration Statement, the Registration Statement), which contains a preliminary proxy statement and prospectus.

Tiziana Life Sciences Announces Near-Term Strategic Focus on Intranasal Foralumab for Diseases of the Central Nervous System (CNS)

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Thursday, November 10, 2022
GLOBE, Lymphocyte, Amyotrophic lateral sclerosis, Pharmacist, PET, Hospital, Safety, Lesional demyelinations of the central nervous system, Inflammation, Company, ALS, Central nervous system, Patient, EAP, Authorization, SPMS, CNS, FDA, Pharmaceutical industry, Brigham, Foralumab

We aim to become a leading company pioneering intranasal therapies for CNS-based diseases and have sufficient capital to fund our programs until the first quarter of 2024.

Key Points: 
  • We aim to become a leading company pioneering intranasal therapies for CNS-based diseases and have sufficient capital to fund our programs until the first quarter of 2024.
  • To date, Tizianas fully human anti-CD3 monoclonal antibody, foralumab, administered intranasally through Tizianas proprietary drug delivery technology, has shown benefit in several different pre-clinical models of CNS-based inflammatory disease.
  • We embark on this journey with our partners at Brigham and Womens Hospital and look forward to studying intranasal foralumab to address a range of underserved inflammatory CNS conditions.
  • Tiziana expects to announce the result of the six-month PET scan on patient EA2 and the nine-month PET scan on patient EA1 also in the first quarter of 2023.

Passage Bio Reports Third Quarter 2022 Financial Results and Provides Business Updates

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Thursday, November 10, 2022
Investor, GLOBE, Private Securities Litigation Reform Act, PASG, IND, Adult, Investment, Risk, U.S. Securities and Exchange Commission, Workforce, Research and development, Amyotrophic lateral sclerosis, GM1, Partnership, Nasdaq, GM1 gangliosidoses, Webcast, Research, Cohort, Krabbe disease, Infant, R, SEC, Failure, Safety, Company, Program, Passage, Clinical trial, University, Patient, Security (finance), Administration, Central nervous system, CNS, Frontotemporal dementia, Pharmaceutical industry, Medicine

ET

Key Points: 
  • ET
    PHILADELPHIA, Nov. 10, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders,today provided business updates and reported financial results for the third quarter ended September 30, 2022.
  • Passage Bio has tremendous potential to transform the lives of patients with devastating CNS orders for which there is significant unmet clinical need, said William Chou, M.D., chief executive officer of Passage Bio.
  • Cash Position: Cash, cash equivalents and marketable securities were $213.8 million as of September 30, 2022, as compared to $239.3 millionas of June 30, 2022.
  • General and Administrative (G&A) Expenses: G&A expenses were $10.7 million for the third quarter ended September 30, 2022, compared to $15.0 million for the same quarter in 2021.

Novozymes and LinusBio announce collaboration to accelerate scientific discovery in Biohealth

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Thursday, November 10, 2022
Kidney, Probiotic, Health, Method, Enzyme, UNICEF, Gasoline, Public health, Amyotrophic lateral sclerosis, World Health Organization, Lead, Autism, Inflammatory bowel disease, Gastroenterology, Factor X, Medicine, Vol, Data science, Biomarker, Toxicity, Clinical trial, Hypertension, Novozymes, Hair, Conference, World, CEO, Anemia, Injury, CNS, Pharmaceutical industry, Fine chemical, Policy

Novozymes, the world leader in biological solutions, and LinusBio (Linus Biotechnology Inc.), a leader in precision exposome sequencing, today announced the companies are forming a collaboration to test a new, non-invasive technology in a clinical trial.

Key Points: 
  • Novozymes, the world leader in biological solutions, and LinusBio (Linus Biotechnology Inc.), a leader in precision exposome sequencing, today announced the companies are forming a collaboration to test a new, non-invasive technology in a clinical trial.
  • The project will apply LinusBios environmental biodynamics platform, enabling detailed temporal mapping of harmful compound exposure, to a clinical trial of probiotics sponsored by Novozymes human health unit, Novozymes OneHealth.
  • As part of the project, LinusBio will conduct a comprehensive study examining lead and other harmful chemical levels.
  • We are very excited to embark on this groundbreaking venture with Novozymes, says Dr. Manish Arora, co-founder and CEO of LinusBio.

The ALS Association and Paralyzed Veterans of America Call on Congress to Quickly Pass Justice for ALS Veterans Act

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Thursday, November 10, 2022
Health, Survivor, Paralyzed Veterans of America, Workforce, Amyotrophic lateral sclerosis, Justice, Income, Research, Education, Disability, Department, Association, PVA, Parent, Family, Death, Diagnosis, Spinal cord, Brain, District, ALS Association, Caregiver, Sport, United States Department of Veterans Affairs, Life expectancy, ALS, Badminton Association of India, Disease, Dependency, CEO, St. Louis Fed Financial Stress Index, Neurodegeneration, Spouse, Paralysis, Recreation, Congress, Investor, DIC, Hospital, Health insurance, Veteran

WASHINGTON, Nov. 10, 2022 /PRNewswire-PRWeb/ -- As the nation prepares to observe Veterans Day, the ALS Association and Paralyzed Veterans of America call on Congress to pass the Justice for ALS Veterans Act (H.R.5607/S.3483), which allows survivors of veterans who died from service-connected ALS to receive a small boost to the rate of Dependency and Indemnity Compensation (DIC), which is commonly known as the "DIC kicker."

Key Points: 
  • Congress should address the financial needs of spouses and dependent children and parents of veterans who passed because of service-related ALS.
  • The Justice for ALS Veterans Act would waive this eight-year requirement and ensure families of all veterans who die of ALS receive this boosted benefit.
  • "The ALS Association wholeheartedly supports this bill to increase compensation for surviving spouses of veterans who die from amyotrophic lateral sclerosis (ALS)," saidCalaneet Balas, President and CEO, The ALS Association.
  • "We urge Congress to quickly pass the Justice for ALS Veterans Act, which would allow these survivors to receive the additional compensation they earned, and need."