Leerink says FDA remarks likely targeted Regenxbio, not uniQure’s program

Leerink notes that Marty Makary, Commissioner of the FDA appeared on CNBC where he defended the agency’s stance on Rare Disease and made some pointed remarks about a specific program that was rejected. Investors are clearly reading through negatively to uniQure (QURE) as the stock is down about 25%, the firm says. Leerink points out that the comments seem to point to a drug that had been officially reviewed and rejected, and highlights that Makary also mentioned the drug had morbidity associated with it and there was no cure. The firm believes this is referring to Regenxbio’s (RGNX) RGX-121, and not AMT-130. Leerink does think uniQure has likely had their Type A meeting for AMT-130 in Huntington’s disease, and is awaiting an update on the meeting minutes.