Pain Therapeutics’ Protein-targeting Alzheimer’s Therapy Is Safe, Phase 1 Trial Indicates

Pain Therapeutics’ Protein-targeting Alzheimer’s Therapy Is Safe, Phase 1 Trial Indicates
Pain Therapeutics' Alzheimer's therapy PTI-125 was safe, and patients tolerated it well, a Phase 1 clinical trial shows. The therapy is aimed at countering a faulty version of the filamin A protein. Scientists believe this abnormal version contributes to Alzheimer's by facilitating the buildup of toxic nerve cell plaque and promoting nerve cell inflammation. Researchers said the trial also demonstrated that PTI-125 has a favorable pharmacokinetic profile — that is, it moves through the body in a way that could make it effective. The results prompted Pain Therapeutics to plan additional trials. "The clinical data are encouraging," Remi Barbier, president and chief executive officer of Pain Therapeutics, said in a press release. "Given the absence of dose-limiting effects in healthy adults, an excellent non-clinical safety database, a strong scientific rationale, and multiple peer-reviewed publications and research grant awards, we are eager to move this drug program to the next level of development." The reference to an "absence of dose-limiting effects" indicated there was no evidence that increases in the treatment's dose causes problems. The "non-clinical safety" reference was to the therapy's safety profile before human studies began. Pain Therapeutics presented the trial results at the 10th Annual International Conference on Clinical Trials on Alzheimer's Disease in Boston, Nov. 1-4. The presentation was titled “Single ascending dose phase I clinical trial of PTI-125 in healthy volunteers.” The
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