Polyneuropathy

Intellia Presents New Interim Data from the Ongoing Phase 1 Study of NTLA-2001 at the 4th International ATTR Amyloidosis Meeting

Retrieved on: 
Thursday, November 2, 2023
Amyloid, FDA, Standard of care, Headache, IND, International, ATTR, Cardiomyopathy, Diarrhea, CRISPR, Intellia Therapeutics, TTR, Disease, Polyneuropathy, Serum, Amyloidosis, Safety, Therapy, Patient, Back pain, NTLA, Toxic leukoencephalopathy, Pharmaceutical industry, Vaccine

Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain.

Key Points: 
  • Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain.
  • “With 65 patients reported from the Phase 1 study, this update represents the largest clinical dataset for an in vivo CRISPR-based investigational therapy.
  • These positive interim results add to the growing body of data that demonstrates deep and durable reductions of serum TTR after a single dose of NTLA-2001.
  • The Phase 1 trial is a two-part study evaluating NTLA-2001 in patients with either ATTR amyloidosis with cardiomyopathy (ATTR-CM) or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).

Alnylam Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Period Activity

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, FDA, ATTR, The New England Journal of Medicine, Science, Therapy, Bachelor of Medicine, Bachelor of Surgery, Cardiomyopathy, Medication, Use, Viral hepatitis, Symantec Endpoint Protection, Conference, American Heart Association, Quarter, Acquisition, Haemophilia, Clinical trial, TTR, Truly Madly Deeply – Ultra Rare Tracks, Growth, Polyneuropathy, Risk, Hypertension, RNA interference, ALN, Roche, Sanofi, Amyloidosis, Patient, APOLLO, ASCVD, Phase 1, Pharmaceutical industry, Video game, Medical device, Aluminium, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, Inclisiran

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2023 and reviewed recent business highlights.
  • Attained over 3,790 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of September 30, 2023.
  • Alnylam announces today that it will present a review of its RNAi platform and pipeline progress at a virtual R&D Day on December 13, 2023.
  • Financial Results for the Quarter Ended September 30, 2023

Intellia Therapeutics to Present Updated Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis and Hold Conference Call to Discuss Third Quarter 2023 Earnings in November

Retrieved on: 
Thursday, October 26, 2023
Amyloidosis, International, ATTR, Cardiomyopathy, CRISPR, Intellia Therapeutics, TTR, Q3, Polyneuropathy, Serum, Safety, Therapy, NTLA, Pharmaceutical industry

ET

Key Points: 
  • ET
    CAMBRIDGE, Mass., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced two upcoming events in November.
  • Intellia will present updated data from the ongoing NTLA-2001 Phase 1 study for the treatment of transthyretin (ATTR) amyloidosis being presented at the 4th International ATTR Amyloidosis Meeting, taking place November 2 – 3 in Madrid, Spain.
  • Intellia will host a webcast to discuss its third quarter 2023 financial results and operational highlights on November 9, 2023, at 8 a.m.
  • The Company will also review the NTLA-2001 data presented at the 4th International ATTR Amyloidosis Meeting.

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Monday, October 9, 2023
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, ATTR, Symantec Endpoint Protection, Cardiomyopathy, Premedication, Food, IRR, OLE, Heart Failure Society of America, Shanda, Nyctalopia, TTR, Risk, Therapy, State, Amyloidosis, RNA interference, Vitamin A deficiency, Abdominal pain, Patisiran, Nausea, Headache, Polyneuropathy, Health status of Asian Americans, Paracetamol, Vitamin A, Periodic breathing, Month, CRL, ALN, ASM, Back pain, Patient, Bachelor of Medicine, Bachelor of Surgery, Medication, New Drug Application, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RDA, Alnylam Pharmaceuticals, APOLLO, EAP, Serum, Quality of life, Pharmaceutical industry, Medical device, Medical imaging, Dietary supplement

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s supplemental New Drug Application (sNDA) for patisiran for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s supplemental New Drug Application (sNDA) for patisiran for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
  • Patisiran is the established name for ONPATTRO®, which is approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • The CRL did not identify any issues with respect to clinical safety, study conduct, drug quality or manufacturing.
  • As a result of the CRL, the Company will no longer pursue an expanded indication for patisiran in the U.S.

Alnylam to Present Additional Data for Patisiran in Patients With the Cardiomyopathy of ATTR Amyloidosis, Including 24-Month Results From the APOLLO-B Phase 3 Study, at the Heart Failure Society of America Annual Scientific Meeting 2023

Retrieved on: 
Friday, October 6, 2023
Health, Other Health, Research, Science, Pharmaceutical, Cardiology, Biotechnology, ATTR, Cardiomyopathy, Premedication, Food, IRR, OLE, Heart Failure Society of America, Shanda, Nyctalopia, Risk, Therapy, Amyloidosis, Safety, RNA interference, Vitamin A deficiency, Abdominal pain, Patisiran, Nausea, Headache, FDA, Health status of Asian Americans, Polyneuropathy, Paracetamol, Vitamin A, Periodic breathing, ASM, Back pain, Patient, Medication, New Drug Application, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RDA, APOLLO, PDUFA, EAP, Serum, Prescription Drug User Fee Act, Quality of life, Pharmaceutical industry, Dietary supplement

Data from an interim analysis of the ongoing open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of ATTR amyloidosis through 24 months will be presented.

Key Points: 
  • Data from an interim analysis of the ongoing open-label extension (OLE) period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of ATTR amyloidosis through 24 months will be presented.
  • Patisiran is the established name for ONPATTRO®, which is approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients.
  • Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Alnylam Stock Trading Halted Today; FDA Advisory Committee to Review Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Wednesday, September 13, 2023
FDA, Health, Genetics, Pharmaceutical, Cardiology, Biotechnology, Prescription Drug User Fee Act, Nyctalopia, Therapy, Premedication, Serum, Advisory Committee, New Drug Application, Vitamin A deficiency, Vitamin A, IRR, Abdominal pain, Medication, Amyloidosis, Back pain, ATTR, Oncology Drug Advisory Committee, Patient, Risk, Paracetamol, Patisiran, Nausea, Periodic breathing, RNA interference, Cardiomyopathy, Polyneuropathy, Headache, RDA, Food, Pharmaceutical industry, Dietary supplement

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that NASDAQ has halted trading of the Company’s common stock.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that NASDAQ has halted trading of the Company’s common stock.
  • The U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee is meeting today to review the supplemental New Drug Application for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
  • As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.
  • Patisiran is the established name for ONPATTRO®, which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting on Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Wednesday, September 13, 2023
Health, FDA, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Heart, Prescription Drug User Fee Act, Disease, Nyctalopia, Therapy, Heart failure, Parallel Walk Test, Premedication, Serum, Health status of Asian Americans, New Drug Application, Patisiran, Vitamin A deficiency, Quality of life, Vitamin A, IRR, Abdominal pain, Medication, Amyloidosis, APOLLO, TTR, Back pain, Shanda, Protein, ATTR, Patient, Physician, Risk, Paracetamol, Alnylam Pharmaceuticals, Nausea, Periodic breathing, RNA interference, Cardiomyopathy, Polyneuropathy, Headache, RDA, Food, Safety, Pharmaceutical industry, Medical device, Dietary supplement

The CRDAC voted 9:3 that the benefits of patisiran outweigh its risks for the treatment of the cardiomyopathy of ATTR amyloidosis.

Key Points: 
  • The CRDAC voted 9:3 that the benefits of patisiran outweigh its risks for the treatment of the cardiomyopathy of ATTR amyloidosis.
  • The CRDAC’s vote, while not binding, will be considered by the FDA when making its decision regarding the potential expanded indication for patisiran.
  • Patisiran is the established name for ONPATTRO®, which is approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Alnylam Announces Date of Planned FDA Advisory Committee Meeting for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Friday, June 30, 2023
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Prescription Drug User Fee Act, Nyctalopia, Therapy, Register, Premedication, Serum, New Drug Application, Vitamin A deficiency, Vitamin A, Federal, IRR, Abdominal pain, Medication, Amyloidosis, Back pain, ATTR, Patient, Risk, Paracetamol, Circulatory system, Nausea, Periodic breathing, RNA interference, Cardiomyopathy, Polyneuropathy, Headache, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RDA, Food, Dietary supplement

As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.

Key Points: 
  • As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.
  • An advanced display of the public notice is available in the Federal Register here .
  • Patisiran is the established name for ONPATTRO®, which is currently approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Alnylam Pharmaceuticals Reports First Quarter 2023 Financial Results and Highlights Recent Period Activity

Retrieved on: 
Thursday, May 4, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, HTRS, CHDI, Bachelor of Medicine, Bachelor of Surgery, Patisiran, PEG, Haemophilia, APOLLO, Acquisition, Patient, RNA interference, Cardiomyopathy, Brazilian Health Regulatory Agency, Polyneuropathy, The Lancet, Tissue, Therapy, NASH, Quarter, Corporate social responsibility, Brain, Medication, Randomness, Corporation, Liver, Growth, Innovation, Lancet, Viral hepatitis, Conference, Eos, Safety, ANVISA, Workplace, AHP, Hepatitis B virus, Truly Madly Deeply – Ultra Rare Tracks, Gene silencing, Shanda, Sanofi, HBV, Central nervous system, CER, Regeneron Pharmaceuticals, RTE, SAD, CNS, Peripheral nervous system, TTR, ATTR, Amyloidosis, Hemostasis, Pharmaceutical industry, Vaccine, Management, Cryptocurrency, Inclisiran, Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2023 and reviewed recent business highlights.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2023 and reviewed recent business highlights.
  • Attained over 3,160 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of March 31, 2023.
  • Alnylam announces today that it intends to present 18-month results from the APOLLO-B Phase 3 study of patisiran at ESC-HF 2023, being held May 20-23, 2023 in Prague.
  • Financial Results for the Quarter Ended March 31, 2023

EQS-News: BB BIOTECH AG: Lackluster first quarter for biotech stocks

Retrieved on: 
Friday, April 21, 2023
Well, Pressure, BB Biotech, Sage, Biotechnology, IRA, Messenger, Ionis, Depression, Inflation, RSV, DSM-IV codes, Patient, Investment, Sumitomo Pharma, NBI, MCC, Alnylam Pharmaceuticals, Growth, Inflation Reduction Act of 2022, Phase, FDA, Polyneuropathy, CHF, Pembrolizumab, ALS, News, BB, Clothing, Fine chemical, Pharmaceutical industry, Vaccine, USD, EUR

Despite the difficult start to the year, BB Biotech looks to the future with confidence in view of the fundamental progress that innovative large, mid- and small-cap biotech firms are making.

Key Points: 
  • Despite the difficult start to the year, BB Biotech looks to the future with confidence in view of the fundamental progress that innovative large, mid- and small-cap biotech firms are making.
  • For BB Biotech shares, this meant a total return for the first quarter 2023 of -4.4% in CHF and -6.8% in EUR (including the dividend payment of CHF 2.85 per share).
  • At the end of the quarter, BB Biotech’s portfolio comprised 28 positions – concentrated further on profitable companies which now represent more than one third of BB Biotech’s equity.
  • The quarterly report of BB Biotech AG as at March 31, 2023, can be downloaded at report.bbbiotech.ch/Q123 or www.bbbiotech.com .