Amyloidosis

Additional Data Showing Acoramidis Increases Serum Transthyretin Which is Associated with Improved Cardiovascular Outcomes Presented at ISA from BridgeBio Pharma’s Phase 3 ATTRibute-CM Study in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Retrieved on: 
Mittwoch, Mai 29, 2024
ACT, Patient, Prognosis, Survival, Amyloidosis, American Heart Association, Month, Risk, Food, ACC, NDA, European Medicines Agency, Calendar, University, ISA, Safety, Serum, ESC, PDUFA, AHA, Congress, Death, Cancer, Biomarker, Cox, Columbia University, Mortality, CVH, MAA, Vancouver General Hospital, American College, Webcast, CVM, Quality of life, TTR, Pharmaceutical industry

ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally administered, highly potent, small molecule stabilizer of TTR.

Key Points: 
  • ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally administered, highly potent, small molecule stabilizer of TTR.
  • BridgeBio will host an investor call on Wednesday, May 29th at 5:30 pm ET to discuss these results.
  • “The data presented at ISA confirm that improved stabilization as reflected in higher serum TTR levels is directly correlated with improved clinical outcomes.
  • Prior analyses from ATTRibute-CM demonstrated that the near-complete stabilization by acoramidis rapidly and durably increased serum TTR levels.

Fortress Biotech Reports First Quarter 2024 Financial Results and Recent Corporate Highlights

Retrieved on: 
Mittwoch, Mai 15, 2024
Amyloidosis, Doxycycline, HIV, NDA, FDA, Vaccination, CRL, New Drug Application, Bangladesh Technical Education Board, Gout, Infection, Erythema, Mustang, Fortification, Inflammation, Fortress Biotech, Hyperuricemia, AstraZeneca, IGA, Disease, Allopurinol, Food, Red, Dotinurad, Safety, Liver transplantation, Carcinoma, Cytomegalovirus, CMV, Minocycline, CKPT, Patient, Exercise, Radiation, PDUFA, Dermatology, Menkes disease, Rosacea, Mortality, BLA, URAT1, Pharmaceutical industry

MIAMI, May 15, 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 first quarter ended March 31, 2024.

Key Points: 
  • In January 2024, Fortress raised gross proceeds of approximately $11.0 million in a registered direct offering priced at-the-market under Nasdaq rules.
  • Fortress’ consolidated net revenue totaled $13.0 million for the first quarter ended March 31, 2024, all of which was generated from our marketed dermatology products.
  • Consolidated research and development expenses including license acquisitions totaled $24.8 million for the first quarter ended March 31, 2024, compared to $39.5 million for the first quarter ended March 31, 2023.
  • Consolidated selling, general and administrative costs were $17.9 million for the first quarter ended March 31, 2024, compared to $25.3 million for the first quarter ended March 31, 2023.

Innate Pharma Reports First Quarter 2024 Business Update and Financial Results

Retrieved on: 
Dienstag, Mai 14, 2024
Oncology, Health, Research, Pharmaceutical, Science, Biotechnology, EV, Drug, Relapse, PDX, MF, FDA, Form, CD20, Carcinoma, Amyloidosis, Euronext Paris, Acute myeloid leukemia, Randomized controlled trial, B-ALL, NK, AACR, Telephone, ADC, Neoplasm, PD-1, ATM, NHL, CRT, Acute lymphoblastic leukemia, GEMOX, Food, NSCLC, EVP, ADS, IPHA, Cancer, ASCO, Poster, BCMA, Oxaliplatin, AstraZeneca, Takeda, Bone marrow, IPH, Lung cancer, Prospectus, PTCL, Spacecraft, CD123, Abstract, Webcast, Pharmaceutical industry

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today reported its consolidated financial results for the quarter ending March 31, 2024.

Key Points: 
  • Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today reported its consolidated financial results for the quarter ending March 31, 2024.
  • Top-line results in MF patients will be presented at the ASCO Annual Meeting 2024 being held May 31 – June 4 in Chicago.
  • Cash, cash equivalents and financial assets of the Company amounted to €113.9 million as of March 31, 2024.
  • Cash, cash equivalents and financial assets as of March 31, 2024 do not include the €4.0 million payment to be received from Sanofi.

Geisinger enrolls first patient in amyloid cardiomyopathy trial

Retrieved on: 
Freitag, Mai 10, 2024
Blood pressure, Patient, B cell, Parent, Amyloidosis, Weight gain, Cardiac amyloidosis, Protein, Heart, Heart failure, Clinical trial, Diagnosis, Travel, Safety, Swelling, Weakness, MD, Blood, Hypoesthesia, Research, Amyloid cardiomyopathy, ATTR, Extremities, Genotype, Quality of life, Carpal tunnel syndrome, TTR

DANVILLE, Pa., May 10, 2024 /PRNewswire/ -- Cardiologist Brendan Carry, MD, and a team of Geisinger physicians have enrolled the first patient in the U.S. into a cardiac amyloidosis trial.

Key Points: 
  • DANVILLE, Pa., May 10, 2024 /PRNewswire/ -- Cardiologist Brendan Carry, MD, and a team of Geisinger physicians have enrolled the first patient in the U.S. into a cardiac amyloidosis trial.
  • "If a patient qualifies for the clinical trial, it will allow us to provide a treatment option when they receive an amyloidosis diagnosis without having to travel outside of the region for care," said Dr.
  • "Geisinger is at the forefront of research and technological advancements that allow for better patient outcomes and improvements in their quality of life."
  • The trial is a phase 3, randomized, double-blind, placebo-controlled study that evaluates the safety and effectiveness of amyloid deplete ALXN2220 in participants with transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).

Intellia Therapeutics Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Donnerstag, Mai 9, 2024
FDA, Bone marrow, CRISPR, Research, Recode, IND, KLKB1, Medicine, Polyneuropathy, NK, AATD, Corporation, Congress, Brain, TTR, Amyloidosis, Food, Gene editing, Conference, Eye, Corporate social responsibility, BLA, HAE, Therapy, Safety, Tissue, Cystic fibrosis, Cardiomyopathy, ATM, DNA, Intellia Therapeutics, Bank, EAACI, Lung, AAT, Social, ATTR, NTLA, Cell, Disease, Regeneron Pharmaceuticals, Patient, Liver, ESG, Pharmaceutical industry

“Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.

Key Points: 
  • “Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.
  • Additionally, Intellia plans to report topline results from the Phase 2 portion in mid-2024 and present full results at a medical meeting in the second half of 2024.
  • Collaboration Revenue: Collaboration revenue was $28.9 million during the first quarter of 2024, compared to $12.6 million during the first quarter of 2023.
  • Net Loss: Net loss was $107.4 million for the first quarter of 2024, compared to $103.1 million during the first quarter of 2023.

Prothena Reports First Quarter 2024 Financial Results and Business Highlights

Retrieved on: 
Mittwoch, Mai 8, 2024
Research, Finance, Clinical Trials, Professional Services, Cardiology, Biotechnology, Pharmaceutical, Health, Science, Oncology, Amyloid, Mortality, FDA, License, Fast Track, TTR, ATTR, Organ dysfunction, Risk, Food, Amyloidosis, Orphan, Epitope, Partnership, IND, Roche, Culture, Therapy, Novo Nordisk, Patient, Acquisition, Failure, VITAL, Bristol Myers Squibb, Clinical trial, European Medicines Agency, Overalls, Pharmaceutical industry

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the first quarter of 2024 and provided business highlights.

Key Points: 
  • Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the first quarter of 2024 and provided business highlights.
  • For the first quarter of 2024, Prothena reported a net loss of $72.2 million, as compared to a net loss of $46.9 million for the first quarter of 2023, respectively.
  • Prothena reported total revenue of $0.1 million for the first quarter of 2024, as compared to total revenue of $2.2 million for the first quarter of 2023.
  • Research and development (R&D) expenses totaled $64.1 million for the first quarter of 2024, as compared to $44.8 million for the first quarter of 2023.

Nexcella, an Immix Biopharma Subsidiary, on Track to Dose NXC-201 Patients in United States

Retrieved on: 
Donnerstag, April 18, 2024
FDA, Clinical trial, MD, Patient, AL amyloidosis, LOS, Amyloidosis, Pharmaceutical industry

LOS ANGELES, April 18, 2024 (GLOBE NEWSWIRE) -- Nexcella, an Immix Biopharma, Inc. subsidiary (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 that it is on track to dose NXC-201 patients in the U.S. with no change in patient enrollment timing.

Key Points: 
  • “We are on track to dose relapsed/refractory AL Amyloidosis NXC-201 patients at our New York City lead site and other leading U.S. sites in mid-2024.
  • U.S. site clinical trial agreements have been signed, and site initiation visits are being scheduled,” said Ilya Rachman, MD PhD CEO of Immix Biopharma and Executive Chairman of Nexcella.
  • “Building on our existing NXC-201 clinical dataset, our U.S. relapsed/refractory AL Amyloidosis clinical trial design is focused on patients with adequate cardiac function who are most likely to experience the greatest clinical benefit from NXC-201.
  • We are committed to providing additional treatment alternatives for relapsed/refractory AL Amyloidosis patients, where there are no FDA approved drugs today.”

Immix Biopharma on Track to Dose NXC-201 Patients in United States

Retrieved on: 
Donnerstag, April 18, 2024
FDA, Clinical trial, MD, Patient, AL amyloidosis, LOS, Amyloidosis, Pharmaceutical industry

LOS ANGELES, April 18, 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 that Immix Biopharma is on track to dose NXC-201 patients in the U.S. with no change in patient enrollment timing.

Key Points: 
  • On track to dose relapsed/refractory AL Amyloidosis patients with CAR-T NXC-201 at New York City lead site and other leading U.S. sites mid-2024
    LOS ANGELES, April 18, 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 that Immix Biopharma is on track to dose NXC-201 patients in the U.S. with no change in patient enrollment timing.
  • “We are on track to dose relapsed/refractory AL Amyloidosis NXC-201 patients at our New York City lead site and other leading U.S. sites in mid-2024.
  • U.S. site clinical trial agreements have been signed, and site initiation visits are being scheduled,” said Ilya Rachman, MD PhD CEO of Immix Biopharma.
  • We are committed to providing additional treatment alternatives for relapsed/refractory AL Amyloidosis patients, where there are no FDA approved drugs today.”

Immix Biopharma Announces Late-Breaking NXC-201 Clinical Data Abstract Accepted for Oral Presentation at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Montag, April 15, 2024
AL amyloidosis, Amyloidosis, MD, Cell, Patient, Gene, Society, LOS, Hematopoietic stem cell transplantation, Cell therapy, Dentistry

Updated clinical data for next-generation CAR-T NXC-201 in relapsed/refractory AL amyloidosis will be presented in Baltimore May 7-11, 2024

Key Points: 
  • Updated clinical data for next-generation CAR-T NXC-201 in relapsed/refractory AL amyloidosis will be presented in Baltimore May 7-11, 2024
    LOS ANGELES, April 15, 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 that updated NXC-201 clinical data has been selected for presentation at the upcoming 27th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT) to be held in Baltimore May 7-11, 2024.
  • “We are delighted to present continued clinical progress developing what we believe is the only CAR-T in AL Amyloidosis at the upcoming 27th Annual Meeting of The American Society of Gene & Cell Therapy,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator for the NEXICART-1 Phase 1b/2a clinical trial of NXC-201.
  • “A potential one-time treatment such as CAR-T NXC-201 for relapsed/refractory AL amyloidosis patients would be a welcome option for this devastating disease.”
    Event: 27th Annual Meeting of The American Society of Gene and Cell Therapy, Baltimore, MD

Prothena Announces Review on Birtamimab Mechanism of Action and Pharmacological Characteristics Published in Leukemia & Lymphoma

Retrieved on: 
Montag, April 15, 2024
Research, Other Health, Pharmaceutical, Oncology, Other Communications, Publishing, Clinical Trials, Science, Cardiology, Biotechnology, Communications, FDA, Health, Amyloid, Mortality, Risk, Amyloidosis, Survival, Therapy, Bone marrow, Dysfunction, Patient, Failure, VITAL, Pharmaceutical industry

The publication can be found in the latest issue of Leukemia & Lymphoma, an international peer-reviewed journal that publishes research on all aspects of hematological malignancies.

Key Points: 
  • The publication can be found in the latest issue of Leukemia & Lymphoma, an international peer-reviewed journal that publishes research on all aspects of hematological malignancies.
  • Birtamimab binds to a highly conserved cryptic epitope which is only exposed in misfolded kappa and lambda light chain (LC) protein.
  • Birtamimab binds and neutralizes soluble, toxic LC aggregates and binds and clears insoluble AL amyloid deposits, without affecting normally folded LC proteins.
  • This mechanism of action is complementary to the current standard of care therapy for AL amyloidosis which does not target the existing toxic LC aggregates and amyloid deposits.