VKTX X-ALD results coming in the next few months. X-ALD is the most common peroxisomal disorder. In the US it affects about 50,000 people in the United States alone. 50,000 potential patients is not a trivial population. The market cap of ALXN ($40B) largely attributable to eculizumab and ravulizumab, which are treatments for PNH (Total population in US: 5000 patients). Based on the valuation of ALXN, the potential X-ALD indication for VK2014 alone could potentially be worth billions in market cap.
If successful in Phase 1, Phase 2 trials will quickly enroll and, given that there are few treatment options for X-ALD, this actually could be VKTX’s first product to market.
