gamma-Aminobutyric acid

Retatrutide Shows Promising Weight Loss Results Compared to Other GLP-1 Agonists

5 months ago

• A recent study shows retatrutide, an investigational triple agonist, led to a 22.1% weight loss over 48 weeks, surpassing existing GLP-1 drugs. • Tirzepatide, commercially available as Zepbound, demonstrated a 17.8% weight loss over 72 weeks, highlighting its effectiveness in obesity management. • Semaglutide (Wegovy) resulted in a 13.9% weight loss over 68 weeks, while liraglutide (Saxenda) showed a 5.8% reduction over 26 weeks. • Oral semaglutide and orforglipron show promise in improving accessibility and adherence due to their non-injectable administration.

Stem Cell-Derived Therapy Trial Enrolls First Michigan Patient for Drug-Resistant Epilepsy

5 months ago

• Wayne State University enrolled the first Michigan patient in a Phase I/II trial testing stem cell-derived interneuron implantation to reduce seizure frequency. • The trial, "A First-In-Human Study of Inhibitory Interneurons (NRTX-1001)," targets drug-resistant unilateral mesial temporal lobe epilepsy (MTLE). • The six-hour procedure involves robotic and stereotactic techniques to deliver GABA-secreting cells into the hippocampus, aiming for a 75% seizure reduction. • The study will assess safety, tolerability, cell viability, inflammation, and effects on epilepsy symptoms over two years, with a 13-year follow-up.

Diamyd Medical Advances Toward Accelerated Approval for Diamyd® in Type 1 Diabetes

5 months ago

• Diamyd Medical received positive feedback from the FDA on its Phase 3 DIAGNODE-3 trial, aligning with requirements for Accelerated Approval based on interim efficacy data. • The FDA concurred on the suitability of the safety dataset and confirmatory evidence, including data from DIAGNODE-3 and previous trials, for potential accelerated approval of Diamyd®. • Agreement was reached with the FDA on evaluating stimulated C-peptide levels and HbA1c as co-primary endpoints at the 24-month final analysis for both accelerated and full approvals. • Breakthrough T1D is providing Diamyd Medical with USD 1.75 million in expanded support for the DIAGNODE-3 Phase 3 trial, facilitating a potential regulatory filing for approval.

4-Phenylbutyrate Shows Promise in Treating Genetic Epilepsy in Children

5 months ago

• Early clinical trial results indicate that 4-phenylbutyrate (PBA) significantly reduces seizure activity in children with genetic epilepsies affecting GABA. • Some children in the trial, who had previously failed multiple anti-seizure medications, experienced complete seizure freedom with PBA treatment. • The research, supported by patient advocacy groups, highlights the potential of PBA in treating epilepsies resulting from SLC6A1 and GABA-A receptor mutations. • Further studies are underway to explore additional strategies for enhancing GABA signaling and restoring normal gene function in these epilepsies.

Scilex Bio's KDS2010 Shows Promise in Phase 2 Trials for Obesity and Alzheimer's

5 months ago

• Scilex Bio's KDS2010, a reversible MAO-B inhibitor, is in Phase 2 trials for obesity, with U.S. patient enrollment planned for 2025, targeting metabolism without affecting appetite. • Phase 2 trials for KDS2010 in Alzheimer's are underway, enrolling 114 patients in South Korea, with plans to expand to the U.S. in 2025. • KDS2010 uniquely targets astrocytic GABA inhibition, potentially improving cognitive function in Alzheimer's and offering advantages over existing GLP-1 obesity treatments. • Phase 1 trials demonstrated KDS2010's favorable safety, tolerability, and pharmacokinetics for once-daily dosing, positioning it for further development in both disease areas.

Diamyd's GAD-Alum Immunotherapy Shows Promise in Type 1 Diabetes Redosing Trial

7 months ago

• A clinical trial evaluating redosing of Diamyd's GAD-Alum immunotherapy in Type 1 Diabetes shows promising results, with sustained insulin production and improved glycemic control. • The DIAGNODE-B pilot trial demonstrated the safety and tolerability of redosing GAD-Alum, with no severe adverse events reported during the 12-month follow-up. • Data analysis reveals Diamyd significantly lowers the number, duration, and amplitude of hyperglycemic excursions, improving glycemic control in patients. • Further analysis of Diamyd's clinical trials supports its potential as a precision medicine approach to slow Type 1 Diabetes progression, especially in HLA DR3-DQ2 positive patients.

Gene Therapy and GPR6 Inverse Agonist Show Promise in Parkinson's Disease Trials

7 months ago

• AAV-GAD gene therapy demonstrated safety and improved motor scores by boosting GABA levels in the subthalamic nucleus in a Parkinson's disease trial. • CVN424, a GPR6 inverse agonist, was safe and well-tolerated, showing a clinically meaningful reduction in daily "off" time at 150 mg/day in a phase II Parkinson's study. • The MIND diet was associated with slower cognitive decline in older adults, with relationships varying based on lifestyle and vascular factors. • Modafinil, cognitive behavioral therapy, and their combination showed similar reductions in multiple sclerosis fatigue at 12 weeks in the COMBO-MS trial.

MeiraGTx's AAV-GAD Gene Therapy Shows Promise in Parkinson's Disease Phase I/II Trial

7 months ago

• MeiraGTx's AAV-GAD gene therapy met its primary endpoint, demonstrating safety and tolerability in a Phase I/II trial for Parkinson's disease. • The high-dose group showed a statistically significant 18-point improvement in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score. • Significant improvements in quality of life were observed in both low- and high-dose groups, as measured by the Parkinson’s Disease Questionnaire (PDQ-39). • MeiraGTx plans to initiate a Phase III study in 2025, following positive discussions with regulatory bodies in the US, Europe, and Japan.

Diamyd Medical Warrants Expire as Phase 3 Trial Readout Nears

8 months ago

• Diamyd Medical's warrants of series TO 3 expired on September 25, 2024, concluding the exercise period that began on September 2, 2024. • Several board members and senior executives, including CEO Ulf Hannelius, intended to exercise their warrants, totaling approximately SEK 5.1 million. • The company anticipates an earlier readout from the DIAGNODE-3 Phase 3 trial around March 2026, focusing on Type 1 Diabetes precision medicine. • Diamyd Medical received SEK 17.5 million from collaborations and the sale of Companion Medical shares, supporting accelerated approval efforts.

Diamyd Medical Pursues Accelerated Approval for Diamyd® in Type 1 Diabetes

8 months ago

• Diamyd Medical is set to pursue an accelerated approval pathway in the U.S. for Diamyd® for Stage 3 Type 1 Diabetes. • The FDA has acknowledged C-peptide levels as a surrogate endpoint, potentially expediting the approval process for Diamyd®. • Interim data from the DIAGNODE-3 Phase 3 trial, expected around March 2026, will form the basis for the BLA submission. • The DIAGNODE-3 trial is enrolling patients with Stage 3 Type 1 Diabetes carrying the HLA DR3-DQ2 genotype across multiple sites.

gamma-Aminobutyric acid

Drug Development Updates