BioWatch Thoughts on BioMarin's Q3-2017 Update

BioPortfolio, 30 Oct 2017 | Biotech Watcher

BMN-290 to Treat Friedreich’s Ataxia (FA)

BioMarin announced that it is advancing BMN-290 into clinical development to treat Friedreich’s Ataxia. This is a single gene disorder involving mutations in the FXN gene. This results in progressive loss of motor functions, as well as sensory impairments (i.e. vision, hearing, speech). The cognitive functions remain intact. The median age of death is around 35.

There are no FDA approved therapies. While there are “nuanced” results, the FA area clearly needs a more efficacious therapeutic in the clinical pipeline. Although BioMarin’s approach appears promising, we will reserve commentary until after the Phase 1 program is completed.

Drugs in Phase II/III Clinical Development, Friedreich’s Ataxia, October 2017

Omaveloxone (Omav), Reata Pharma (RETA), Phase 2, Nrf-2 upregulator & NF-kB inhibitor. Omav finished Part 1 of the MOXIe trial with mixed or nuanced results. 172 patient trial slated for top-line results in Q1-2020 and ongoing results until Q4-2022.

TAK-831, Takeda, Phase 2, 65 patient trial with completion slated for Q1-2019.

EPI-743, BioElectron, Phase 2, Testing patients with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care. This includes FA patients

Milestones:
BMN-290 – Friedreich’s Ataxia
Small study involving chromatin modulation therapy shows promise. BMN-290 provides chromatin modulation therapy which appears more appropriate for long-term, chronic therapy. There is no disease-modifying therapy.

BMN-290 is a selective HDAC3 inhibitor of isoform 3, designed to inhibit histone deactylase enzymes. It helps unfold the chromatin structure to allow increased expression, Phase 1 - Safety Launch Q4-2017

BioWatch Thoughts on BioMarin's Q3-2017 Update