The Progeria Research Foundation Inc

Progeria drug trial: the progeria research foundation and boston children's hospital continue their partnership for conducting clinical drug trials for children with progeria.the clinical trial team: a team of professionals at boston children's hospital and brigham & women's hospital is working with children with progeria from around the globe.(see schedule o for continuation) the progeria 2-drug, phase i/ii trialin 2015, prf obtained all necessary approvals for its third type of clinical trial, which will assess a two-drug combination of lonafarnib and everolimus. Everolimus is a form of the drug rapamycin, but everolimus can be more easily given to children with progeria. Everolimus targets a different pathway than lonafarnib, and the trial is designed to determine if the combination provides a better treatment than the fda-approved lonafarnib on its own. The progress of this trial is detailed below.in 2016, phase i began. The purpose of a phase i trial is to determine the safest maximum dosage of a drug. Phase i was completed in mid-2017. In july 2017, following the successful completion of phase i, the clinical trial moved onto phase ii. Phase ii of the trial tests the effectiveness of the 2-drug combination. From 2016 through 2020, 60 children and young adults with progeria from 26 countries speaking 16 languages were enrolled and traveled to boston for their trial visits at boston children's hospital, where they underwent a series of tests and received new drug supply. In 2023, the 20+-member trial team continues to evaluate the 2-drug trial data to determine whether the 2-drug combination of everolimus plus lonafarnib is a more effective treatment for progeria than lonafarnib alone.in 2024, the team continues to gather data from the ongoing clinical trial, while children and young adults who lack access to lonafarnib (the fda approved treatment) in their home countries continue to travel to boston for evaluation and to receive this life-extending treatment.gain on sale of priority review vouchers: in january 2021, pursuant to the collaboration and supply agreement entered into by the organization, the organization and an unrelated party reached an agreement with a third party for the sale of u.s. Food and drug administration priority review voucher. The organization's share of the proceeds was $47,442,250. The organization recognized a gain related to this sale during the year ended december 31, 2021 totaling $46,493,405, which represents their share of the proceeds less certain selling costs of $948,845.

Future trial efforts: prf engages members in the scientific community with expertise in various drugs and/or genetic therapies for the treatment and potential cure for progeria. The investigation and collaborative evaluation of drug therapies is pursued to identify drug candidates/genetic therapies that can lead to future clinical trials.