Amyloidosis

Orphan designation: Human IgG1 monoclonal antibody targeting amyloid transthyretin Treatment of ATTR amyloidosis, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, Amyloidosis, IRIS, EMA, COMP, European Commission, Marketing, ATTR, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of ATTR amyloidosis on 19 February 2024.
  • Orphan designation does not mean the medicine is available or authorised for use.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent Treatment of AL amyloidosis, 19/02/2024 Positive

Retrieved on: 
Thursday, April 18, 2024
Patient, Committee, Autotransplantation, Amyloidosis, IRIS, EMA, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of AL amyloidosis on 19 February 2024.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • For information on ongoing clinical trials in the EU, see:
    Key facts
    - Active substance
    - Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent
    - Intended use
    - Treatment of AL amyloidosis
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/24/2900
    - Date of designation
    - Sponsor
    Raremoon Consulting Esp S.L.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Fortress Biotech Reports 2023 Financial Results and Recent Corporate Highlights

Retrieved on: 
Thursday, March 28, 2024
AstraZeneca, Exercise, Fortification, New Drug Application, Patient, GBM, CRL, NDA, Menkes, Minocycline, Therapy, Marketing, Radiation, CKPT, Rosacea, Investigational New Drug, Erythema, Safety, BLA, Fortress Biotech, Food, Red, Amyloidosis, Dermatology, Carcinoma, FDA, Hyperhidrosis, Mustang Bio, Menkes disease, PDUFA, Pharmaceutical industry

MIAMI, March 28, 2024 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, today announced financial results and recent corporate highlights for the full-year ended December 31, 2023.

Key Points: 
  • Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, “In 2023, we built a significant amount of momentum to position our Company to achieve multiple milestones in 2024.
  • In March 2024, the FDA accepted the NDA and has set a PDUFA goal date of November 4, 2024.
  • In December 2023, the FDA issued a complete response letter (“CRL”) for the cosibelimab BLA.
  • Fortress’ consolidated net revenue totaled $84.5 million for the full year ended December 31, 2023, which included $59.7 million in net revenue generated from our marketed dermatology products.

Metagenomi Reports Business Updates and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 27, 2024
Research, Dicarboxylic acid, AAV, Enzyme, Development, Gene editing, CAST, Cardiovascular disease, Haemophilia, Vivisection, Patient, Primate, RNA, DNA, Wave 1, Sale, Metagenomics, G&A, IPO, Ultra, Doctor of Philosophy, Protein, MBA, Genome, SMART, Factor VIII, CRISPR, Amyloidosis, MGX, AGT, Liver, LNP, Ionis, PAM, Vaccine

EMERYVILLE, Calif., March 27, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today reported financial results for the full year ended December 31, 2023, and additional business updates.

Key Points: 
  • The aggregate gross proceeds to Metagenomi from the offering were approximately $93.75 million, before deducting underwriting discounts and commissions and offering expenses.
  • In addition, cash used to fund our operations was $91.4 million for the year ended December 31, 2023.
  • R&D Expenses: Research and development (R&D) expenses were $94.4 million for the full year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $28.8 million for the full year ended December 31, 2023.

Immix Biopharma Announces Major Comprehensive Cancer Center as Lead Site for NXC-201 AL Amyloidosis Clinical Trial

Retrieved on: 
Wednesday, March 20, 2024
MD, Amyloidosis, Patient, AL amyloidosis, Pharmaceutical industry

LOS ANGELES, CA, March 20, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and other autoimmune diseases, today announced Memorial Sloan Kettering Cancer Center as lead clinical site for its NXC-201 relapsed/refractory AL Amyloidosis multi-site clinical trial.

Key Points: 
  • LOS ANGELES, CA, March 20, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and other autoimmune diseases, today announced Memorial Sloan Kettering Cancer Center as lead clinical site for its NXC-201 relapsed/refractory AL Amyloidosis multi-site clinical trial.
  • “We are looking forward to this important multi-site clinical trial with NXC-201 CAR-T for patients with AL Amyloidosis,” said Heather Landau, MD, Memorial Sloan Kettering Cancer Center Amyloidosis Program Director.
  • “NXC-201 represents a novel direction in the treatment landscape for relapsed or refractory AL Amyloidosis patients.”

Intellia Therapeutics Announces First Patient Dosed in the Phase 3 MAGNITUDE Study of NTLA-2001 as a Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with Cardiomyopathy

Retrieved on: 
Monday, March 18, 2024
Pharmacology, Royal Free Hospital, NTLA, Disease, TTR, Richmond Pharmacology, Patient, Amyloidosis, Cardiomyopathy, Intellia Therapeutics, Heart failure, Therapy, CRISPR, Safety, Phase 1, ATTR, Pharmaceutical industry

NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.

Key Points: 
  • NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.
  • The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy.
  • We look forward to evaluating the efficacy and safety of NTLA-2001 in patients with cardiomyopathy in the pivotal Phase 3 trial.
  • There is remarkable interest from the ATTR amyloidosis patient community for a potential single-dose treatment, and we look forward to contributing to the advancement of novel treatment approaches.”

Alnylam Launches Hereditary ATTR (hATTR) Amyloidosis Campaign to Help Shorten Time to Diagnosis for Inherited and Rapidly Progressive Disease

Retrieved on: 
Wednesday, March 13, 2024
Science, Other Science, Research, Pharmaceutical, General Health, Health, Genetics, Other Health, Diagnosis, Alnylam Pharmaceuticals, Dizziness, Medication, Learning, Weakness, Genetic testing, Disease, TTR, Risk, Human, Patient, Common, Knowledge, Constipation, Diarrhea, Child, Amyloidosis, MBA, MPH, Heart, RNA interference, Carpal tunnel syndrome, Therapy, Multimedia, Family, Pharmaceutical industry

“Family history is considered one of the most important risk factors for health problems.

Key Points: 
  • “Family history is considered one of the most important risk factors for health problems.
  • “It took seeing nine doctors over seven years for me to finally receive a diagnosis of hATTR amyloidosis,” said Christine, a Family Health History Road Trip participant who is living with hATTR amyloidosis.
  • If I had, I could have potentially been diagnosed and started on a disease management plan much earlier.”
    With an inherited disease like hATTR amyloidosis that progresses rapidly, early diagnosis is crucial.
  • A genetic test can determine whether a person carries one of the more than 120 variants in the TTR gene associated with hATTR amyloidosis.

Public Comment Period Opens for the “Workup of Amyloidosis” Guideline

Retrieved on: 
Wednesday, March 13, 2024
Education, Managed Care, Health, General Health, Pharmaceutical, Oncology, Other Science, Training, Science, Other Education, Clinical Trials, Amyloid, College, Quality of life, Laboratory, Marie Cassidy, Diagnosis, Patient, Cardiology, Nursing, Disease, CAP, Amyloidosis

—The draft statements are available for public comment through April 3, 2024 on cap.org .

Key Points: 
  • —The draft statements are available for public comment through April 3, 2024 on cap.org .
  • “This process ensures tailored care, early detection, and ongoing optimization of treatment strategies for individuals with amyloidosis,” adds co-chair Billie S. Fyfe-Kirschner, MD, FCAP.
  • Following the open comment period, the guideline authors will consider all feedback to finalize the statements.
  • The final statements will appear in the guideline manuscript and will be available at no cost.

Immix Biopharma Announces “Be Proactive in AL” AL Amyloidosis Awareness Initiative

Retrieved on: 
Tuesday, March 5, 2024
Spleen, Liver, Bangladesh Technical Education Board, Autoimmune disease, NIH, Physician, Extrapyramidal system, Weight loss, Dizziness, AL amyloidosis, Amyloidosis, Diagnosis, Joint, Hypoesthesia, Fatigue, Patient, Heart, Tissue, Mortality

LOS ANGELES, CA, March 05, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, today announced its new AL Amyloidosis awareness campaign Be Proactive in AL™.

Key Points: 
  • LOS ANGELES, CA, March 05, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, today announced its new AL Amyloidosis awareness campaign Be Proactive in AL™.
  • The campaign seeks to increase awareness about the critical need to recognize and diagnose AL Amyloidosis early, while also educating AL Amyloidosis patients about available treatment options.
  • AL amyloidosis is a life-threatening immunological disorder in which an abnormal protein called amyloid builds up in tissues and organs.
  • “Today, it is common for AL Amyloidosis patients to see 3 or 4 different physicians and wait months to years before being definitively diagnosed with AL amyloidosis,” said Dena Heath, Amyloidosis Research Consortium Board Secretary and Facilitator, Northern California Amyloidosis Support Group.

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, February 22, 2024
Research, Intellia Therapeutics, Regeneron Pharmaceuticals, Fatigue, Haemophilia, NK, CTA, Liver, Cystic fibrosis, ATM, A1AT, Amyloidosis, HAE, Therapy, European Medicines Agency, ATTR, Haemophilia B, DNA, European Commission, Hereditary angioedema, Recode, Conference, Cardiomyopathy, AATD, Civil service commission, Clinical trial, Facial muscles, CRISPR, Gene editing, Food, NTLA, KLKB1, Lung, NEJM, TTR, Patient, Medicine, Disease, Tissue, Pharmaceutical industry

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.

Key Points: 
  • Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.
  • R&D Expenses: Research and development expenses increased by $9.0 million to $109.0 million during the fourth quarter of 2023, compared to $100.0 million during the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses increased by $5.4 million to $29.0 million during the fourth quarter of 2023, compared to $23.6 million during the fourth quarter of 2022.
  • Net Loss: The Company’s net loss was $132.2 million for the fourth quarter of 2023, compared to $113.4 million during the fourth quarter of 2022.