Expanded access

Savara Reports First Quarter 2024 Financial Results and Provides Business Update

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星期四, 五月 9, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Research, BLA, Inc., Investment, Chemistry, Expanded access, Chair, Pharmaceutical industry

Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, reported financial results for the first quarter ending March 31, 2024 and provided a business update.

Key Points: 
  • Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, reported financial results for the first quarter ending March 31, 2024 and provided a business update.
  • “The IMPALA-2 trial remains on-track and we look forward to reporting top line results by the end of the second quarter,” said Matt Pauls, Chair and CEO, Savara.
  • “Following that, and assuming positive data, we expect to file a BLA in the first half of 2025.
  • As of March 31, 2024, the Company had cash, cash equivalents and short-term investments of approximately $143.0 million and debt of approximately $26.4 million.

Anavex Life Sciences Reports Fiscal 2024 Second Quarter Financial Results and Provides Business Update

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星期四, 五月 9, 2024
FDA, Fast Track, Research, Rett syndrome, Trial of the century, Partnership, Doctor of Philosophy, Conference call, CNS, Procedure, Frontotemporal dementia, EDGAR, Food, Orphan drug, European Medicines Agency, Brain, RWE, MECP2, Drug development, RNA-Seq, Conference, Publication, Pharmacokinetics, U.S. FDA, Marketing authorisation, DSM-IV codes, Patent, Real, RNA, Human, Therapy, Treatment, EMA, Alzheimer's disease, Central nervous system, Clinical trial, Medicine, Marketing, ADAS, Centralisation, Pharmacology, European, Patient, Expanded access, Caregiver, Webcast, Interim, Bank statement, Pharmaceutical industry

NEW YORK, May 09, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative, neurodevelopmental and neuropsychiatric disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, schizophrenia, and other central nervous system (CNS) diseases, today reported financial results for its fiscal quarter ended March 31, 2024.

Key Points: 
  • Parkinson’s disease: Initiation of ANAVEX®2-73 imaging-focused trial and Phase 2b/3 6 month trial is expected in the second half of 2024.
  • General and administrative expenses for the quarter of $2.8 million compared to $2.9 million for the comparable quarter of fiscal 2023.
  • Research and development expenses for the quarter of $9.7 million compared to $11.3 million for the comparable quarter of fiscal 2023.
  • The financial information for the fiscal quarter ended March 31, 2024, should be read in conjunction with the Company’s condensed consolidated interim financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com .

Tiziana Life Sciences Reports Positive 3-Month Neuroimaging Scores in Multiple Sclerosis Patients Receiving Intranasal Foralumab

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星期四, 四月 25, 2024
American Academy, Brain, White matter, Mass, Expanded access, Hospital, Encephalitis, PET, Research, EDSS, Patient, MFIS, Fatigue, DSM-IV codes, Harvard Medical School, Associate, Disability, PIRA, Medical imaging

White Matter Z-scores are a statistical measure used in neuroimaging studies to assess the integrity or abnormalities in structures of the brain.

Key Points: 
  • White Matter Z-scores are a statistical measure used in neuroimaging studies to assess the integrity or abnormalities in structures of the brain.
  • We calculated White Matter Z-scores to measure the effect of intranasal foralumab on microglial activation at baseline and then after foralumab treatment for three months.
  • I welcome the publication of Dr. Singhal’s [F18]PBR06-PET research paper, highlighting these combined findings in Clinical Nuclear Medicine,” commented Gabriele Cerrone, Chairman, acting CEO and founder of Tiziana Life Sciences.
  • *EA5 showed a worsening in their White Matter Z-Score at three months during a pseudo-exacerbation of the patient’s trigeminal neuralgia.

Tiziana Life Sciences Announces FDA Allowance for Additional Twenty Patients to be Enrolled in the Intranasal Foralumab Multiple Sclerosis Expanded Access Program

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星期二, 四月 23, 2024
FDA, Patient, Mass, Hospital, Safety, Food, PET, Fatigue, Neurology, Harvard Medical School, Expanded access, Pharmaceutical industry

To date, of the 10 participating patients, two patients have been dosed for more than one year and eight additional patients have been dosed for six months, all without serious side effects.

Key Points: 
  • To date, of the 10 participating patients, two patients have been dosed for more than one year and eight additional patients have been dosed for six months, all without serious side effects.
  • All patients have either stabilized or improved on treatment with foralumab, and no patients have declined in key clinical measures.
  • These data are the first to combine PET imaging with a novel ligand, immune-biomarkers, clinical measures and comprehensive safety data endpoints in patients receiving long-term intranasal foralumab.
  • Patients not eligible for the Phase 2a trial may now be considered for this expanded EA program.

Global Clinical Trials Market Analysis, Size, and Forecasts 2023 and 2024-2030 Featuring ICON, Wuxi AppTec, SGS, Syneos Health, PRA Health Sciences, Pfizer, IQVIA, Sanofi, and Medpace - ResearchAndMarkets.com

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星期二, 四月 23, 2024
Health, Clinical Trials, USD, FDA, Phase, Cancer, Pain management, SARS-CoV-2, Food, Viral, Intervention, Personalized medicine, Blockchain, Machine learning, Diabetes, National Cancer Institute, Research, Vaccine, ChromeOS, Therapy, Incidence, Patient, NCI, Clinical trial, Expanded access, Prevalence, Pharmaceutical industry

The global clinical trials market is split into phases: Phase 1, Phase 2, Phase 3, and Phase 4.

Key Points: 
  • The global clinical trials market is split into phases: Phase 1, Phase 2, Phase 3, and Phase 4.
  • By indication, the global clinical trials market is categorized into Autoimmune/Inflammation, Pain Management, Oncology, CNS Condition, Diabetes, Obesity, Cardiovascular, and Others.
  • By study material, the global clinical trials market is fragmented into Interventional, Observational, and Expanded Access.
  • The requirement for clinical trials to improve diagnostic tests and vaccines for viral illnesses such as SARS-CoV-2 has exponentially augmented the demand for clinical trials.

Atossa Therapeutics Provides Five-Year (Z)-Endoxifen Treatment Update on FDA-Approved "Expanded Access" Program for a U.S. Breast Cancer Patient

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星期二, 三月 19, 2024
Diagnosis, Growth, Ki-67, Neoadjuvant therapy, Mammography, Cancer, Expanded access, Woman, Neoplasm, Therapy, Breast cancer, CYP2D6, Safety, Axis, Osteoporosis, Hot, Recurrence, Tamoxifen, FDA, Enzyme inhibitor, PR, Pharmaceutical industry

Atossa is a clinical stage biopharmaceutical company developing innovative medicines in areas of significant unmet medical need in oncology with a focus on breast cancer.

Key Points: 
  • Atossa is a clinical stage biopharmaceutical company developing innovative medicines in areas of significant unmet medical need in oncology with a focus on breast cancer.
  • Over five years of adjuvant treatment, her daily (Z)-endoxifen treatment has been well tolerated, and there were no vasomotor symptoms commonly associated with standard of care adjuvant pharmaceuticals.
  • She remains cancer-free today and we are grateful to have been able to help her with a difficult treatment problem.”
    Diagnosis.
  • Under the FDA Expanded Access IND program, the use of Atossa's proprietary (Z)-endoxifen is restricted to this patient only.

eGenesis Announces World’s First Successful Transplant of Genetically Engineered Porcine Kidney in a Living Patient

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星期四, 三月 21, 2024
Research, Other Health, General Health, Oncology, Hospitals, Genetics, Surgery, Clinical Trials, Science, Biotechnology, FDA, Health, Other Science, Pelvic floor dysfunction, MGH, Kidney disease, Gene, Organization, Survival, Massachusetts General Hospital, Transplant, Inflammation, Immunosuppression, Kidney failure, Mortality, Coagulation, Hope, Doctor of Philosophy, Expanded access, Quality of life, Genome, Transgene, Hospital, Kidney, Dialysis, CD40L, Courage, Kidney transplantation, Medicine, Dentistry

eGenesis , a biotechnology company developing human-compatible engineered organs to address the global organ shortage, today announced the first ever transplantation of a genetically engineered porcine kidney into a living human recipient.

Key Points: 
  • eGenesis , a biotechnology company developing human-compatible engineered organs to address the global organ shortage, today announced the first ever transplantation of a genetically engineered porcine kidney into a living human recipient.
  • Following the procedure, the patient is in good condition and recovering well at MGH.
  • Yet the demand for organs far outpaces supply, with more than 90,000 individuals on the kidney waitlist and approximately 25,000 kidney transplants performed each year.
  • Decades of progress in cross-species transplantation, accelerated by the advancement of modern genome editing tools and next-generation sequencing, have enabled eGenesis to progress genetically engineered organs to the clinical setting.

KalVista Pharmaceuticals Awarded UK Promising Innovative Medicine (PIM) Designation for Sebetralstat

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星期二, 三月 12, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, EAMS, Patient, HAE, National Health Service, Health care, NHS, American Academy, KALV, Expanded access, Marketing, MHRA, PIM, Hereditary angioedema, Safety, AAAAI, Pharmaceutical industry

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced the UK Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Promising Innovative Medicine (PIM) designation for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).

Key Points: 
  • KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced the UK Medicines and Healthcare products Regulatory Agency (MHRA) has awarded the Promising Innovative Medicine (PIM) designation for sebetralstat, an investigational novel, oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).
  • The PIM is the first step in the Early Access to Medicines Scheme (EAMS) which would allow KalVista to treat patients with sebetralstat prior to receiving a Marketing Authorisation.
  • “We are proud to have sebetralstat designated as a Promising Innovative Medicine by the MHRA, which is similar to Expanded Access in the US,” said Ben Palleiko, Chief Executive Officer of KalVista.
  • KalVista recently provided phase 3 data for sebetralstat at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, which displayed clinically and statistically significant results across all endpoints, and an excellent safety and tolerability profile.

Xplore Surpasses Commitments with Fibre and Fixed Wireless Expansion in Prince Edward Island

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星期三, 三月 27, 2024
PEI, Confederation Bridge, Communication, Federation, Internet, Expanded access, Confederation, Investment, Data-rate units, Community, Telecommunications, Cable television

WOODSTOCK, NB, March 27, 2024 /CNW/ - Xplore Inc. is announcing significant progress on its broadband expansion project in Prince Edward Island, highlighted by the company connecting more homes to fibre and fixed wireless Internet than originally planned.

Key Points: 
  • WOODSTOCK, NB, March 27, 2024 /CNW/ - Xplore Inc. is announcing significant progress on its broadband expansion project in Prince Edward Island, highlighted by the company connecting more homes to fibre and fixed wireless Internet than originally planned.
  • Key achievements of the project include:
    Exceeding Fibre Connectivity Commitments: Xplore has connected nearly 3,400 premises to gigabit-speed fibre Internet, ensuring more residents have access to the most advanced Internet technology.
  • It will connect Xplore's Prince Edward Island fibre network to its fibre network across the Maritimes, enhancing Internet connectivity for residents and businesses and fostering potential future technological advancements reliant on robust communication infrastructure.
  • "This is meaningful progress with a purpose," said Rizwan Jamal, President and CEO, Xplore Inc. "By surpassing our commitments for fibre and fixed wireless connectivity, we are ensuring that more residents across Prince Edward Island have access to the benefits of high-speed Internet."

aTyr Pharma Announces Expanded Access Program (EAP) for EFZO-FIT™ Clinical Trial Participants

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星期三, 二月 21, 2024
SAN, Sarcoidosis, Scar, Small RNA, Pi, ATyr Pharma, FDA, Inflammation, Food, Scleroderma, Risk, Myelocyte, Health, Safety, Principal investigator, Expanded access, Neuropilin, Systemic scleroderma, ILD, OLE, Patient, Fibrosis, Clinical trial, EAP, Pharmaceutical industry

SAN DIEGO, Feb. 21, 2024 (GLOBE NEWSWIRE) --  aTyr Pharma, Inc. (Nasdaq: LIFE) (aTyr or the Company), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced plans to initiate an Individual Patient Expanded Access Program (EAP) for its lead therapeutic candidate, efzofitimod, for patients with pulmonary sarcoidosis. The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.

Key Points: 
  • Individual Patient EAP allows access to efzofitimod for patients who complete the Phase 3 EFZO-FIT™ study in pulmonary sarcoidosis.
  • Company initiating program based on blinded EFZO-FIT™ study investigator and patient participant feedback.
  • The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.
  • “We are pleased to make efzofitimod available to patients beyond the duration of the EFZO-FIT™ clinical trial through this Individual Patient EAP,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.