OPMD

Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b/2a Study

Retrieved on: 
星期四, 四月 18, 2024
FDA, Dysphagia, Mutation, EDT, Nature, COMP, Oculopharyngeal muscular dystrophy, Hope, Food, News, Death, Yogurt, Pharmacotherapy, Observation, PABPN1, Disease, OPMD, Subject, Ptosis, Patient, EMA, Caregiver, Committee, Clinical trial, Trial of the century, Orphan drug, European Medicines Agency, Pharmaceutical industry

HAYWARD, Calif., April 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the study’s first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

Key Points: 
  • “To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing.
  • We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301,” said Jerel A.
  • Videofluoroscopic swallowing studies represent the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.
  • The event replay will be placed on the News & Events tab on the Investor page of the Benitec website.

Benitec Biopharma to Host Virtual R&D Day to Discuss BB-301 Clinical Program in Oculopharyngeal Muscular Dystrophy on April 18, 2024

Retrieved on: 
星期二, 四月 9, 2024
Neurology, University college, Patient, McGill University Health Centre, Head, Doctor of Philosophy, MDCM, OPMD, Otorhinolaryngology, Natural history

To register, click here .

Key Points: 
  • To register, click here .
  • The event will feature two key opinion leaders who will discuss the clinical symptoms and the natural history of oculopharyngeal muscular dystrophy (OPMD), a rare genetic muscle disorder.
  • The key opinion leaders will also review the clinical and radiographic methods employed to evaluate disease progression and discuss the current treatment landscape for patients diagnosed with OPMD.
  • A live question and answer session will follow the formal presentations.

Benitec Biopharma Announces First Subject Dosed in Phase 1b/2a Clinical Trial for Gene Therapy Candidate BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy

Retrieved on: 
星期四, 十一月 30, 2023
Natural history, Dysphagia, Research, Silence, RNA-dependent RNA polymerase, OPMD, Replacement, Patient, Data collection, Disorder, Pharmaceutical industry

BB-301 is the Company’s first gene therapy candidate employing the Silence and Replace approach and is being developed for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia.

Key Points: 
  • BB-301 is the Company’s first gene therapy candidate employing the Silence and Replace approach and is being developed for the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia.
  • Dosing of the first subject marks the beginning of the 52-week follow-up period designed to facilitate conclusive evaluation of the primary and secondary endpoints of the BB-301 Phase 1b/2a Clinical Treatment Study.
  • Currently, there are 19 subjects enrolled into the NH study at the U.S. clinical trial site, with each subject having the potential to roll over into the BB-301 Phase 1b/2a Clinical Treatment Study.
  • Interim safety and efficacy data are expected to become available from the BB-301 Phase 1b/2a Clinical Treatment Study in mid-2024.

Benitec Biopharma Releases First Quarter 2024 Financial Results and Provides Operational Update

Retrieved on: 
星期一, 十一月 13, 2023
Natural history, Clinical, REB, Oropharyngeal dysphagia, Dysphagia, Safety, Research, Dispute board, Principal investigator, IND, FIH, FDA, Oculopharyngeal muscular dystrophy, HIV disease progression rates, RNA, Pharmaceutical industry, Nursing, Medical imaging, Health insurance, Cryptocurrency, OPMD, Benitec Biopharma

Dosing with BB-301: 1-day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study.

Key Points: 
  • Dosing with BB-301: 1-day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study.
  • Total Revenues for the quarter ended September 30, 2023, were $0 compared to $0 for the quarter ended September 30, 2022.
  • Total Expenses for the quarter ended September 30, 2023 were $5.9 million compared to $4.6 million for the quarter ended September 30, 2022.
  • The loss from operations for the quarter ended September 30, 2023, was $5.9 million compared to a loss of $4.6 million for the quarter ended September 30, 2022.

Benitec Biopharma Releases Full Year 2023 Financial Results and Provides Operational Update

Retrieved on: 
星期四, 九月 21, 2023
Oculopharyngeal muscular dystrophy, REB, Dysphagia, Natural history, Oropharyngeal dysphagia, Principal investigator, RNA-dependent RNA polymerase, Form 10-K, Research, RNA, Investigational New Drug, 52 Weeks Make A Year, HIV disease progression rates, FIH, Dispute board, FDA, Annual report, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Clinical, Food, Safety, Nursing, Pharmaceutical industry, Medical imaging, Health insurance, Cryptocurrency, Benitec Biopharma, OPMD

HAYWARD, Calif., Sept. 21, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year ended June 30, 2023. The Company has filed its annual report on Form 10-K for the quarter ended June 30, 2023, with the U.S. Securities and Exchange Commission.

Key Points: 
  • The Company has filed its annual report on Form 10-K for the quarter ended June 30, 2023, with the U.S. Securities and Exchange Commission.
  • Dosing with BB-301: 1-day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study.
  • In December 2022, Benitec began screening OPMD subjects at the lead clinical study site in the United States.
  • In January 2023, Benitec announced the enrollment of the first OPMD subject into the OPMD NH Study in the United States.

Benitec Biopharma Receives FDA Clearance of the IND for BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy

Retrieved on: 
星期一, 六月 26, 2023
Oculopharyngeal muscular dystrophy, Dysphagia, RNA-dependent RNA polymerase, OPMD, Data collection, RNA, Patient, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Benitec Biopharma, IND

Oculopharyngeal Muscular Dystrophy (OPMD) is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel.

Key Points: 
  • Oculopharyngeal Muscular Dystrophy (OPMD) is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel.
  • OPMD patients lose the ability to swallow liquids and solids, resulting in chronic malnutrition, aspiration, and fatal episodes of aspiration pneumonia.
  • Currently, there are no approved therapeutic agents for the treatment of OPMD.
  • “The FDA’s clearance of our IND for BB-301 is a significant milestone for OPMD patients and for Benitec as a Company,” said Jerel A.

Benitec Biopharma to Present at the OPMD International Conference

Retrieved on: 
星期一, 五月 8, 2023
Oculopharyngeal muscular dystrophy, Natural history study, RNA-dependent RNA polymerase, Child development stages, OPMD, RNA, International Conference on Nuclear Disarmament, Oslo, 2008, Conference, Safety, Pharmaceutical industry, Nursing, Benitec Biopharma

HAYWARD, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced that Jerel A.

Key Points: 
  • HAYWARD, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced that Jerel A.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec will present virtually on the BB-301 Clinical Development Program at the Oculopharyngeal Muscular Dystrophy (OPMD) International Conference in Tel Aviv, Israel, on Tuesday, May 16th, 2023 at 12:50 pm Israel Daylight Time.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec.
  • “Enrollment of OPMD subjects into the Natural History Study at the U.S. clinical trial site is proceeding at a rapid pace, with 9 subjects enrolled to date.

Muscular Dystrophy Association Announces Five Recipient Organizations of Advocacy Collaboration Grants

Retrieved on: 
星期四, 二月 16, 2023
Organization, Medicaid, Public policy, Muscular Dystrophy Association, ALS, MDA, Lambert–Eaton myasthenic syndrome, Neuromuscular disease, Patient, CMD, Muscular dystrophy, LEMS, Policy, DMD, SMA, Duchenne muscular dystrophy, OPMD, Pharmaceutical industry, Nursing

New York, NY, Feb. 16, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced the five organizations receiving $109,065 in grant funding.

Key Points: 
  • New York, NY, Feb. 16, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced the five organizations receiving $109,065 in grant funding.
  • The grants will promote and support MDA’s key public policy and advocacy initiatives in coordination with awardees to empower people living with neuromuscular diseases to live longer, more independent lives.
  • In the spirit of MDA's commitment to collaboration, the organization is utilizing this grant program to facilitate impactful and cooperative advocacy projects and initiatives.
  • Applications for the program were announced in 2022 for MDA to collaborate with organizations focused on non-partisan, non-political advocacy initiatives to achieve legislative and public policy victories for the neuromuscular community at the federal, state, local level.

Benitec Biopharma Enrolls First OPMD Subject into the Clinical Development Program

Retrieved on: 
星期一, 一月 23, 2023
Argonaute, RNA, United, Principal, OPMD, Observation, Oculopharyngeal muscular dystrophy, Child development stages, Benitec Biopharma, Dysphagia, Patient, Oropharyngeal dysphagia, NH, Pharmaceutical industry, Medicine

HAYWARD, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the enrollment of the first oculopharyngeal muscular dystrophy (OPMD) patient into the OPMD natural history phase of the BB-301 clinical development program.

Key Points: 
  • HAYWARD, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the enrollment of the first oculopharyngeal muscular dystrophy (OPMD) patient into the OPMD natural history phase of the BB-301 clinical development program.
  • The OPMD Natural History (NH) Study represents the 6-month pre-treatment observation period for each OPMD subject prior to the administration of BB-301 for the treatment of OPMD-related dysphagia.
  • “Following the initiation of OPMD patient screening at the lead clinical site in the United States in the fourth quarter of 2022, the Principal Investigator of the OPMD NH Study reported high enrollment interest from potential study subjects,” said Jerel A.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.

Benitec Biopharma Releases First Quarter 2023 Financial Results and Provides Operational Update

Retrieved on: 
星期四, 十一月 10, 2022
IND, CTA, Research, Principal investigator, Principal, Company, GMP, Child development stages, Institutional review board, GDPR, IRB, Disclosure, Benitec Biopharma, Dispute board, RNA, General Data Protection Regulation, U.S. Securities and Exchange Commission, Argonaute, Oculopharyngeal muscular dystrophy, Drug discovery, Dysphagia, Oropharyngeal dysphagia, HDL-Atherosclerosis Treatment Study, HIV disease progression rates, Achievement, United, Privacy, CEC, REB, Phenotype, GLOBE, FIH, Organization, Behavior, Muscle, NASDAQ, Safety, Union, Clinical, Validation, Animal, Natural history, Insurance, Pharmaceutical industry, Medicine, Medical imaging, Mineral wool, Medical device, OPMD, EU

Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.

Key Points: 
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.
  • In the Third Calendar Quarter 2022, Benitec submitted responses to the Central Ethics Committee (CEC) related to minor non-clinical queries.
  • BB-301 Phase 1b/2a Regulatory Updates:
    Investigational New Drug (IND) and Clinical Trial Application (CTA) filings are anticipated in the First Calendar Quarter 2023.
  • Total Revenues for the quarter ended September 30, 2022, were $0 compared to $0 for the quarter ended September 30, 2021.