An experimental Alzheimer’s disease therapy designed by Alzheeon to reduce the deposits of the amyloid plate in the brain failed to overcome a placebo in a phase 3 study, the last blow to a medicine whose history is marked by the clinical trial steps.
The pill twice a day, the valiltramiprosate, did not meet the main objective of the clinical trial to decelerate the cognitive deterioration measured at 78 weeks, Alzheon announced on Thursday. Neverberness, the framingham -based company, based in Massachusetts, said brain volume measures that show a slowdown in brain atrophy, which suggest potential neuroprotective benefits of the drug.
Alzheon also pointed out cognitive benefits nominally statistically significant and significant clinical functional effects in a prepecific subgroup, patients in the early stages of the disease. That is a subgroup of the subgroup of genetically defined patient that Alzheeon expected it to benefit from the drug.
Alzheon’s medicine, known in previous development stages as Alz-801, was licensed by Bellus Health, based in Quebec, in 2013. That the small biotech molecule, called Tramiprosate, was designed to join Beta Amiloid. The hope was that this approach could prevent the aggregation of the amyloid plate in the brain of patients with Alzheimer’s. But the results of two phase 3 studies carried out by the Canadian company showed that the medicine did not lead to a statistically significant improvement in cognitive function.
Alzheon’s analysis of the tramiprosate data found the variability in the blood levels of the drug in the study participants. Valletramiprosate is a profármaco or tramiprosate, which Meeaans becomes tramiprosado in the body. Alzheon changes in the Bellus medication include the improvement in how the compound is absorbed in the body with the aim of reducing the variability observed in the previous tests. Alzheon’s analysis of previous trial data also found signs of improvement in patients carrying the APOE4 gene, a variant that increases the risk of developing Alzheimer’s. Alzheon’s phase 3 test enrolled 325 patients carrying two copies of APOE4.
The new Alzheimer’s medicines approved by the FDA in the last two years, Leqembi de Eisai and Kisunla de Eli Lilly, are antibodies, drugs of large molecules administered by infusion. Alzheon expected their oral drug to sacrifice patients the advantage of a easier dosing formulation.
There was also potential for a security advantage. Anti-amyloid antibody drugs introduce the risk of cerebral inflammation and bleeding called amyloid-related image abnormalities (ARIA). APOE4 carriers have a substantial risk of ARIA in development, so Alzheimer’s patients who carry this genetic variant could have a medication that offers a different approach. While Leqembi and Kisunla can be used to treat these patients under their FDA approval, the labels of these products carry black cash warnings that mark this greater risk of complication. This risk also has a point of conflict for efforts to ensure the regulatory approach in Europe and Australia.
Alzheon did not report serious advertiser reactions or deaths in phase 3 test of the valiltramiprosate. The company also said there was no greater risk of ARIA. Alzheon plans to publish more detailed phase 3 results in a publication reviewed by pairs. Despite losing the main objective of the fundamental test, Alzheon is not abandoning the drug. In the announcement of the company of the preliminary results of the study, the medical director Susan Abushakra said that patients who have two copies of APOE4 “have a desperate need for adorned treatment options.”
“A precision medicine approach is key to addressing the needs of Alzheimer’s patients who have the APOE4/4 genotype, and we are committed to this population of patients,” he said.
A long -term extension study continues to evaluate patients who complete phase 3 or valiltramiprosate test. This study, in progress in the United States, the United Kingdom and Canada, follows patients during an addition to 52 weeks. Last June, the private Alzheeon raised $ 100 million in financing of the E series to support the completion of the fundamental study and prepare for the potential marketing of his Alzheimer’s medicine. The Phase 3 program also has the support of $ 51 million in subsidies funds of the National Aging Institute of the National Health Institutes.
Photo: Yuichiro Chinimage, Getty Images