Friedreich’s ataxia: dual route administration and first patient dosed
Turning to FA, Cumbo said the program uses a dual route of administration designed to reach the dentate nucleus of the cerebellum while also targeting the spinal column and heart. He described MRI-guided stereotactic delivery into both sides of the dentate nucleus, led by neurosurgeon Dr. Lonser at Ohio State University, followed by intravenous dosing after a rest period.
Cumbo said Solid dosed its first FA patient, a 27-year-old who was “very, very sick,” and referenced an mFARS score of 93 (noting the patient had been in the high 80s). Roughly 40 days after dosing, he said the patient experienced a headache that resolved with Tylenol and that the company had not seen other issues. He added that the team was “already hearing things” suggestive of efficacy, but emphasized it did not yet have proof.
For planned data disclosure, Cumbo said Solid expects to dose three to six FA patients over the course of the year and anticipates reading out results from the first three patients in the second half of the year, while continuing dosing in a second cohort. On expectations for clinical benefit, he cited 2.14 as the mFARS bar associated with the regulatory approval of SKYCLARYS.
