Intimate and focused, conferences like IARC 2017 — the International Ataxia Research Conference — are the type of scientific meeting that “all rare disease patient groups should try to do,” Warren Huff, the founder and chief executive officer of Reata Pharmaceuticals, said in an interview with Friedreich’s Ataxia News when asked why his company chose to be the meeting’s chief sponsor.
“Its’ a rich environment in which to exchange ideas, communicate what we’re all doing,” Huff said, with patient groups and researchers from “all over the world” coming together around the latest developments in one specific set of genetic diseases — ataxias — and in a setting that’s “smaller in number, and therefore more interactive.”
Progress, both in terms of airing the work Reata has done and understanding the priorities of the patients its efforts might treat, is virtually assured.
“For companies like ours, trying to translate [basic science] into effective therapies,” IARC is “extremely important,” he said.
“In Friedreich’s ataxia, there’s no question that a lot of the work and progress being made is related to really the fantastic work done by the patient associations,” Huff said in Thursday’s interview with Friedreich’s Ataxia News. “The establishment of endpoints for basic research, the establishment of patient registries and, in the case of FARA … a fabulous set of longitudinal history data on the patients.”
As a U.S. company, based in Texas, Reata naturally works mostly closely with FARA, and Huff credits the group for setting Reata onto the investigational Friedreich’s ataxia (FA) therapy — omaveloxolone (RTA 408) — that is now moving into a possibly pivotal Phase 2 trial. The group brought the potential for the treatment’s target, the protein Nfr2 — whose levels are decreased in FA patients and linked to mitochondrial, and hence neurological, health — to Reata’s attention based on work that FARA likely had sponsored.
“They actually initially proposed the idea,” Huff said. “We’re looking for settings like this, where we have true collaborators in the space. It just makes everything much easier and more effective.”
MOXIe (NCT02255435) is a two-part clinical trial now moving into its second, and possibly decisive, phase.
If successful in showing that omaveloxolone works to significantly improve neurological function in FA patients, it will be “registrational,” Huff said. “We gotten clear guidance from U.S. Food and Drug Administration that the part two, if successful, will support registration for the drug and possible approval.”
Part one determined an optimal dose, 160 mg, and though it wasn’t powered for efficacy as a dose-escalating study, “robust” Nfr2 production and disease suppression were seen.
“We’re very excited about the [part one] results. We saw activation of Nrf2, we saw improvement in markers of mitochondrial function, we saw clinically meaningful improvements in the modified FARS standardized neurological exams in the patients,” Huff said. “What was particularly exciting was that they recovered function; their modified FARS improved from baseline in only 12 weeks of treatment.”
The only brake on what part two, the efficacy part of MOXIe, might achieve is getting the 100 or so Friedreich’s ataxia patients it needs enrolled.
That point brought Huff back to IARC 2017, and its role in furthering research.
Through conference presentations, posters and casual conversations, there is “a lot of people seeing [our] data from around the world, a lot of investigators, patient groups — it’s an excellent venue for people to examine the clinical results,” Huff said.
Not surprisingly, his final word of advice for the conference organizers and attendees: “Just keep doing it, because we’re very supportive. What’s been done here between the patient groups and researchers … is just fabulous. I think it’s one of the best examples of this type of cooperation.”
MOXIe is currently recruiting Friedreich’s ataxia patients with modified FARS (Friedreich’s Ataxia Rating Scale) scores from 20 to 80 at study sites across the U.S., and in Austria, Australia and the U.K. More information, including on enrollment and eligibility, is available on its clinical trials.gov page.