Auckland Researchers Launch Clinical Trial to Investigate 5FU Chemotherapy Cardiotoxicity
• University of Auckland and Auckland City Hospital researchers initiate a clinical trial to study severe cardiac side effects of 5-fluorouracil (5FU), a widely-used chemotherapy drug affecting 5% of patients.
• The two-year study, funded by multiple organizations including the Gut Cancer Foundation, aims to identify risk factors and mechanisms behind 5FU-induced cardiotoxicity through comprehensive patient monitoring.
• Researchers will utilize Holter monitors, blood tests, and urine samples from cancer patients to understand drug metabolism patterns and develop predictive markers for adverse cardiac reactions.
Infrequent Zoledronate Infusions Reduce Fracture Risk in Early Postmenopausal Women
• A new study reveals that infrequent intravenous infusions of zoledronate significantly reduce the risk of fractures in early postmenopausal women.
• Women receiving two zoledronate infusions five years apart experienced a 44% reduction in spinal fractures compared to those receiving a placebo.
• The research suggests that zoledronate, due to its generic availability and infrequent administration, offers a cost-effective strategy for early fracture prevention.
• Findings indicate potential for primary fracture prevention in women with any bone mineral density, especially those concerned about future fracture risk.
Tune Therapeutics' TUNE-401 Receives Approval for Phase 1b Trial in Hong Kong for Chronic Hepatitis B
• Tune Therapeutics' TUNE-401, an epigenetic silencing therapy, has been approved for a Phase 1b clinical trial in Hong Kong for chronic hepatitis B (CHB).
• The trial will be led by Professor Man-Fung Yuen, focusing on the high prevalence of HBV in Asian populations and addressing the limitations of current treatments.
• TUNE-401 targets both integrated viral DNA and covalently closed circular DNA (cccDNA) to achieve a functional cure by epigenetically silencing the virus.
• Preclinical data demonstrated TUNE-401's ability to repress both cccDNA and integrated DNA in target cells and animal models, showing promise for a 'one-and-done' treatment.
Smartphone App Shows Promise in Treating Tinnitus in Clinical Trial
• A novel smartphone app delivering cognitive-behavioral techniques, sound therapy, and mindfulness practices has shown promise in treating tinnitus.
• In a 12-week clinical trial, 65% of participants using the app reported improvement in their tinnitus symptoms, with enhanced mental well-being.
• The digital polytherapeutic approach aims to rewire the brain, reducing the prominence and impact of phantom sounds associated with tinnitus.
• Researchers emphasize the need for larger, multi-center trials to confirm the efficacy and scalability of the digital therapeutic intervention.
AviadoBio and Astellas Forge $2.18 Billion Deal for Frontotemporal Dementia Gene Therapy
• AviadoBio and Astellas have partnered in a deal worth up to $2.18 billion to advance AVB-101, a gene therapy for frontotemporal dementia (FTD).
• AVB-101 targets the GRN gene mutation, a significant genetic cause of FTD, and has demonstrated promising results in early studies.
• The gene therapy uses a modified virus to deliver DNA directly to the brain, aiming to rescue cells damaged by the GRN gene loss.
• Clinical trials are underway, with early stages focused on halting the progression of FTD using this novel gene supplement approach.
Astellas and AviadoBio Partner on Gene Therapy for Frontotemporal Dementia in $2.18 Billion Deal
• AviadoBio and Astellas have entered into a partnership for the development of AVB-101, a gene therapy targeting frontotemporal dementia, with Astellas providing an upfront investment of $30 million.
• AVB-101 aims to halt the progression of frontotemporal dementia by addressing the GRN gene mutation, which accounts for 5-10% of frontotemporal dementia cases.
• The gene therapy has demonstrated promising results in preclinical studies and is currently undergoing early human clinical trials, with three patients already treated.
• The collaboration seeks to leverage gene therapy to treat neurological diseases, potentially opening avenues for therapies addressing a range of other neurological conditions.
Vir Biotechnology's Tobevibart and Elebsiran Show Promise in Chronic Hepatitis B Treatment
• Vir Biotechnology's MARCH Phase 2 trial shows that tobevibart and elebsiran, alone or with PEG-IFNα, led to HBsAg loss in chronic hepatitis B patients.
• In patients with low baseline HBsAg, 39% achieved HBsAg loss with tobevibart and elebsiran, while 46% achieved it with the triplet regimen.
• The safety and tolerability profiles of both regimens were consistent with prior studies, with mostly mild to moderate treatment-emergent adverse events.
• Functional cure data from the 24-week follow-up are expected in Q2 2025, which will determine the next steps in clinical development.
Vir Biotechnology's Tobevibart and Elebsiran Show Promise in Chronic Hepatitis B Treatment
• Vir Biotechnology announced positive end-of-treatment data from its Phase 2 MARCH trial, evaluating tobevibart and elebsiran combinations for chronic hepatitis B.
• In participants with low baseline HBsAg, 39% achieved HBsAg loss with tobevibart + elebsiran, and 46% with the triplet regimen including pegylated interferon alfa.
• The safety and tolerability profile of the regimens were consistent with prior studies, with treatment-emergent adverse events generally mild to moderate.
• Key functional cure data from the 24-week follow-up are expected in Q2 2025, which will inform the next steps of clinical development.
CRISPR Therapy NTLA-2002 Shows Promise in Hereditary Angioedema Treatment
• NTLA-2002, a CRISPR-Cas9-based gene editing therapy, demonstrates potential as a one-time treatment for hereditary angioedema (HAE), reducing the need for daily medication.
• Phase 2 study results published in the New England Journal of Medicine showed significant reductions in angioedema attacks and kallikrein levels in patients treated with NTLA-2002.
• The study reported a mean percent change in the monthly attack rate from baseline through week 16 of -78% and -79.5% for 25mg and 50mg doses respectively.
• Common adverse events associated with NTLA-2002 included headache, fatigue, and nasopharyngitis, with researchers noting limitations such as small sample size.
CRISPR Gene Editing Shows Promise as Single-Dose Treatment for Hereditary Angioedema
• A single dose of CRISPR-Cas9 gene editing therapy, NTLA-2002, significantly reduces angioedema attacks in patients with hereditary angioedema (HAE).
• The Phase 2 study demonstrated a substantial and sustained reduction in kallikrein levels, a key mediator of swelling in HAE patients.
• The international trial involved multiple national excellence centers and showed promising results, potentially offering a functional cure for HAE.
• NTLA-2002 is advancing to Phase 3 clinical trials, building on the positive outcomes from Phase 1 and 2 studies.
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