Ionis

Metagenomi Reports Business Updates and Full Year 2023 Financial Results

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Mercoledì, Marzo 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.

Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome

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Domenica, Aprile 7, 2024
TGS, AP, Ionis, Incidence, Abdominal pain, IONS, Webcast, Patient, NEJM, Fasting, American College, Ionis Pharmaceuticals, FCS, ACC, Triglyceride, Annual general meeting, Medicine, Pharmaceutical industry

CARLSBAD, Calif., April 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis' lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides (TGs) in patients with genetically validated FCS at six months. In addition, olezarsen demonstrated robust and sustained reductions in TGs and serum apolipoprotein C-III (apoC-III) levels. Importantly, olezarsen reduced the incidence of acute pancreatitis (AP) events over the 12-month treatment period compared to placebo. Olezarsen also demonstrated a favorable safety and tolerability profile. These results were presented in an oral presentation at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia and published simultaneously in The New England Journal of Medicine (NEJM). Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.

Key Points: 
  • Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.
  • In the study, patients were treated with olezarsen 80 mg (n=22), 50 mg (n=21) or placebo (n=23) once every four weeks.
  • Serious AEs occurred in 14% of patients treated with olezarsen 80 mg, 19% treated with olezarsen 50 mg, and 39% treated with placebo.
  • Ionis will host a webcast to discuss the detailed results from the Balance study on Monday, April 8 at 10:00 am ET.

Flamingo Therapeutics Announces First Patient Dosed in Phase II PEMDA-HN Study for Head and Neck Squamous Cell Carcinoma (HNSCC)

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Mercoledì, Febbraio 14, 2024
Head, Merck, CMO, MD, Ionis, Apoptosis, STAT3, Safety, Therapy, Patient, RECIST, Mortality, Trial of the century, PD-L1

The global study is planned to be conducted at study centers in the United States, Korea and the United Kingdom.

Key Points: 
  • The global study is planned to be conducted at study centers in the United States, Korea and the United Kingdom.
  • "We are grateful for the contribution of all participants in this study and for the support of our clinical collaborators at each site."
  • "Our site is very excited about being the first enrollment on this important head and neck trial.
  • I believe the combination of danvatirsen and pembrolizumab has tremendous promise to treat patients with head and neck cancer.

EQS-News: Investor interest returns – dividend yield remains high for 2024

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Mercoledì, Febbraio 14, 2024
Acquisition, Annual general meeting, RSV, USD, Cystic fibrosis, Ionis, FDA, Pain, Patient, Policy, BB Biotech, Alnylam Pharmaceuticals, Depression, Pharmaceutical industry

Release as at February 14, 2024

Key Points: 
  • Release as at February 14, 2024
    After a difficult 2023 stock market year, above all due to higher interest rates, the capital markets are now once again looking at biotech companies with interest.
  • The prospect of falling interest rates has led to a rethink.
  • A dividend of this magnitude is exceptional not just in the Swiss equity market, but also in an international comparison.
  • Experts are anticipating similarly high approval figures for 2024 too.

Eplontersen granted U.S. FDA Fast Track designation for patients with transthyretin-mediated amyloid cardiomyopathy

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Giovedì, Febbraio 8, 2024
Ionis Pharmaceuticals, RNA, Cardiovascular disease, Therapy, Fast Track, Patient, FDA, Orphan, Amyloidosis, IONS, AstraZeneca, Food, Ionis, ATTR, Polyneuropathy, DSM-IV codes, Pharmaceutical industry

CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.

Key Points: 
  • CARLSBAD, Calif., Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
  • The FDA grants development programs Fast Track designation to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serious conditions and fill an unmet medical need.
  • "CARDIO-TTRansform is the largest, most comprehensive study ever conducted in ATTR-CM patients, with results expected as early as next year."
  • WAINUA was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Vect-Horus Enters Exclusive License Agreement with Ionis Pharmaceuticals to Advance Systemic Delivery of RNA-Targeted Medicines for Neurological Diseases

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Martedì, Gennaio 30, 2024
Mental Health, Research, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Oncology, Traffic barrier, Ionis Pharmaceuticals, RNA, Therapy, Patient, Central nervous system, DSM-IV codes, IONS, Ionis, Brain, Medication, Tissue, Vaccine

The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.

Key Points: 
  • The agreement provides Ionis with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology ”VECTrans” for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.
  • Ionis has an option to add additional targets to the license.
  • Vect-Horus retains the rights to use its technology, for these specific targets, for all non-RNA-Targeted Medicines.
  • “This license is aligned with our strategy of using our technology to deliver drug payloads, such as RNA-targeted therapeutics, to the brain and other tissues.

Orsini selected as the exclusive specialty pharmacy for WAINUATM (eplontersen) for adults with hereditary transthyretin-mediated amyloid polyneuropathy

Retrieved on: 
Giovedì, Gennaio 4, 2024
Vitamin A deficiency, ELK, AstraZeneca, IDN, FDA, Disease, Ionis, Orsini, Serum, Polyneuropathy, Internationalized domain name, Vitamin A, Pharmacy, Patient, Diagnosis, Disability studies, Amyloidosis

ELK GROVE VILLAGE, Ill., Jan. 4, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy ("Orsini") has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN).

Key Points: 
  • ELK GROVE VILLAGE, Ill., Jan. 4, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy ("Orsini") has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUA™ (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN).
  • "Orsini is proud to be the exclusive specialty pharmacy offering access to WAINUA as a new treatment option for patients living with ATTRv-PN," Orsini CEO Brandon Tom said.
  • Indication: WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
  • Note: While Orsini is the exclusive specialty pharmacy partner for WAINUA, it may also be available for use by integrated delivery networks (IDNs) within qualifying IDN-owned specialty pharmacies.

WAINUA™ (eplontersen) Granted First-Ever Regulatory Approval in the US for the Treatment of Adults With Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

Retrieved on: 
Venerdì, Dicembre 22, 2023
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Quality of life, Tissue, Heart, Heredity, Serum, Polyneuropathy, Death, Inotersen, Ageing, TTR, JAMA, Vitamin A, Diagnosis, Metabolism, Amyloidosis, Pancreas, Disease, Safety, Journal, Heart failure, Disability studies, Sympathetic nervous system, Vitamin A deficiency, Food, Biopharmaceutical, AstraZeneca, Caregiver, ATTR, Patient, Medicine, LICA, Ionis, American Medical Association, Growth, Central nervous system, Pharmaceutical industry

It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.

Key Points: 
  • It is also a pivotal moment for Ionis as WAINUA will be the first in a steady cadence of potential commercial launches for the company.
  • Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels.
  • Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
  • Please see link to US Full Prescribing Information for WAINUA.

Bicycle Therapeutics Provides Data Updates for Three Clinical Programs and Strategy Overview at First R&D Day

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Giovedì, Dicembre 14, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Webcast, MT1-MMP, Peripheral neuropathy, Brain, Safety, Immune system, Cervical cancer, Food, Therapy, Toxicity, Head, Diabetic neuropathy, Nobel Prize, Patient, CD137, Bicycle, FDA, Non-small-cell lung cancer, Pembrolizumab, Neoplasm, Ionis, Investigational New Drug, Brentuximab vedotin, MUC, ORR, Ovary, Anal cancer, Cancer, Ovarian cancer, Toxic leukoencephalopathy, Pain, R, Drug combination, EV, Central nervous system, TNBC, NSCLC, BRC, Pharmaceutical industry, Vaccine, Birth control, Medical imaging, Thromboxane A2

“In totality, the data support the emerging differentiated profile of our Bicycle® molecules, paving the way to deliver best-in-class or first-in-class therapies for many cancers.

Key Points: 
  • “In totality, the data support the emerging differentiated profile of our Bicycle® molecules, paving the way to deliver best-in-class or first-in-class therapies for many cancers.
  • BT8009 is a Nectin-4 Bicycle toxin conjugate (BTC®) designed to overcome the significant toxicity associated with other toxin conjugate approaches.
  • Over 2024, Bicycle Therapeutics intends to generate early human imaging data from its wholly owned BRC pipeline.
  • Bicycle Therapeutics will continue to develop Bicycle® molecules to address disease outside of oncology through innovative partnerships.

Ionis announces the appointment of Michael Yang to Board of Directors

Retrieved on: 
Giovedì, Dicembre 14, 2023
Janssen, Growth, Vertex, Patient, IONS, Acadia Pharmaceuticals, Ionis Pharmaceuticals, Science, Management, Ionis

CARLSBAD, Calif., Dec. 14, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the appointment of Michael Yang, an experienced biopharmaceutical executive, to the Ionis Board of Directors.

Key Points: 
  • CARLSBAD, Calif., Dec. 14, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the appointment of Michael Yang, an experienced biopharmaceutical executive, to the Ionis Board of Directors.
  • Mr. Yang's appointment expands the total number of Ionis Board members to 10.
  • "We are thrilled for Michael to join the Board as he adds deep biopharmaceutical leadership experience, particularly important as Ionis is poised to enter its next phase of growth," said Joseph Loscalzo, M.D., Ph.D., chairman of the Ionis Board of Directors.
  • "We are thrilled to have him as part of the Board and the Ionis team looks forward to working with him."