TTR

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.”

Operata Has Been Awarded Selection for the Innovation Showcase at Enterprise Connect 2024 (EC24) for Advancing Customer Experience With CX Observability

Retrieved on: 
Tuesday, March 26, 2024
Software, Data Analytics, Marketing, Artificial Intelligence, Communications, Professional Services, Technology, Electronic Commerce, Cloud, Organization, Communication, Health, TTR, CX, Enterprise, Insurance, AHT, FCR, Multimedia, CPU, Sales

Operata has been awarded selection for the Innovation Showcase at Enterprise Connect 2024 (EC24), marking a significant achievement in its commitment to enhancing the efficiency and functionality of contact centers through CX Observability.

Key Points: 
  • Operata has been awarded selection for the Innovation Showcase at Enterprise Connect 2024 (EC24), marking a significant achievement in its commitment to enhancing the efficiency and functionality of contact centers through CX Observability.
  • The selection of Operata for the Innovation Showcase at Enterprise Connect 2024 (EC24) recognizes it among cutting-edge technologies enhancing customer experiences.
  • The recognition of Operata at EC24 for its innovation in CX Observability underscores the company's unique approach.
  • In light of its selection for the Innovation Showcase, Operata is offering EC attendees a chance to experience the advantages of CX Observability with a free trial.

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.

BridgeBio Pharma and Bayer Announce European Licensing Agreement for Acoramidis in ATTR-CM

Retrieved on: 
Monday, March 4, 2024
PALO, EMA, Senior, MAA, FDA, Food, PDUFA, TTR, Corporate development, European Medicines Agency, Patient, ATTR, NDA, Partnership, Pharmaceutical industry

In exchange, BridgeBio will receive up to $310 million USD comprising of upfront and near-term milestone payments, as well as additional undisclosed sales milestones.

Key Points: 
  • In exchange, BridgeBio will receive up to $310 million USD comprising of upfront and near-term milestone payments, as well as additional undisclosed sales milestones.
  • BridgeBio will also receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in Europe.
  • “We are excited to have found a like-minded partner in Bayer that shares our belief in the potential of acoramidis to ameliorate the lives of ATTR-CM patients.
  • BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023.

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.

Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights

Retrieved on: 
Thursday, February 15, 2024
Research, FDA, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Therapy, Fast Track, Acquisition, SAD, VITAL, Roche, American Society of Hematology, ASH, Risk, Food, Epitope, AAIC, ID, Novo Nordisk, Exercise, Organ dysfunction, Amyloidosis, Amyloid, Conference, Bristol Myers Squibb, BMS, Society, TTR, European Medicines Agency, Patient, Clinical trial, ATTR, IND, Orphan, Mortality, Failure, Pharmaceutical industry

In addition, the Company provided 2024 financial guidance and business highlights.

Key Points: 
  • In addition, the Company provided 2024 financial guidance and business highlights.
  • “2023 was a year of strong progress for Prothena as we advanced our protein dysregulation portfolio and moved closer to becoming a fully integrated commercial company.
  • Total revenue for the fourth quarter and full year of 2023 included BMS collaboration revenue of $0.3 million and $91.3 million, respectively.
  • As of December 31, 2023, Prothena had $621.0 million in cash, cash equivalents and restricted cash, and no debt.

BridgeBio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Retrieved on: 
Monday, February 5, 2024
Mortality, CVH, ACM, TTR, MD, PALO, Patient, Safety, FDA, Food, Cancer, PDUFA, Doctor of Philosophy, Survival rate, NDA, Kaplan–Meier estimator, Pharmaceutical industry

PALO ALTO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for acoramidis, an investigational drug for the treatment of ATTR-CM. The application was based on positive results from ATTRibute-CM, the Company’s Phase 3 study designed to evaluate the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). The FDA has set an action date of November 29, 2024 under the PDUFA. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • The FDA has set an action date of November 29, 2024 under the PDUFA.
  • The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.
  • There is a need for more treatment options that can help fill the significant unmet need that exists for patients today.
  • The Company also received acceptance of its Marketing Authorization Application with the European Medicines Agency and is preparing for additional global regulatory submissions.

BridgeBio Pharma Announces Publication of Positive Results from Phase 3 ATTRibute-CM Study of Acoramidis for Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in the New England Journal of Medicine

Retrieved on: 
Wednesday, January 10, 2024
ACT, Mortality, Social Security, Serum, American Heart Association, Patient, ACM, Research, Hospital, PALO, TTR, Cancer, Survival rate, Food, NDA, Safety, ATTR, FDA, Cardiology, NEJM, Marketing, New Drug Application, CVH, KCCQ, Amyloidosis, University college, Dietary supplement

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

Key Points: 
  • ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, small molecule stabilizer of transthyretin (TTR).
  • Findings presented in the NEJM support acoramidis as an effective and safe treatment option for patients with ATTR-CM and reinforce the hypothesis that greater stabilization of TTR may be associated with improved clinical outcomes.
  • BridgeBio has also presented additional detailed results from ATTRibute-CM at the European Society of Cardiology Congress 2023 in August and at the American Heart Association Scientific Sessions 2023 in November .
  • The Company submitted a New Drug Application to the U.S. FDA in 2023 and intends to submit additional marketing authorization applications to regulatory bodies in 2024.

Next DLP Takes Security Analysts to the Next Level with XTND AI

Retrieved on: 
Tuesday, January 9, 2024
Risk, TTR, Artificial intelligence, DLP, TTC, Organization, XTND, Software, AI, MITRE, Next, Online shopping

BOSTON, Jan. 9, 2024 /PRNewswire-PRWeb/ -- Next DLP ("Next"), a leader in insider risk and data protection, today announced XTND AI, an artificial intelligence powered assistant, that extends insider risk and data security analysts' expertise, enabling every member of the security team to contribute to the business at a higher level.

Key Points: 
  • Next DLP takes security analysts to the next level with XTND AI.
  • BOSTON, Jan. 9, 2024 /PRNewswire-PRWeb/ -- Next DLP ("Next") , a leader in insider risk and data protection, today announced XTND AI, an artificial intelligence powered assistant, that extends insider risk and data security analysts' expertise, enabling every member of the security team to contribute to the business at a higher level.
  • John Stringer, Head of Product at Next DLP said, "XTND is all about helping analysts efficiently run investigations, and therefore, controlling the costs associated with running an effective insider risk program."
  • "XTND is all about helping analysts efficiently run investigations, and therefore, controlling the costs associated with running an effective insider risk program."

Prothena Provides Updates on PRX012, PRX123, Birtamimab and Portfolio Programs

Retrieved on: 
Monday, January 8, 2024
Biotechnology, Health, Clinical Trials, Biology, Failure, Food, Risk, Hand, FDA, Acquisition, Amyloidosis, Roche, Mortality, Organ dysfunction, Orphan, ATTR, VITAL, Novo Nordisk, Epitope, PD, European Medicines Agency, Fast track (FDA), TTR, Amyloid, Alpha-synuclein, Patient, Bristol Myers Squibb, Therapy, Vaccine

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, provided a business update on portfolio programs.

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, provided a business update on portfolio programs.
  • The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for PRX012 for the treatment of AD.
  • Update on Financial Position for December 31, 2023
    At December 31, 2023, Prothena had cash, cash equivalents, and restricted cash of approximately $621 million.
  • Cash on hand provides sufficient capital which takes Prothena beyond the completion of its ongoing clinical trials.