Did You Ride The Rally In Orphazyme?

Shares of Orphazyme A/S (ORPH) soared as much as over 1,300%, touching an intraday price of $77.77, yesterday, as the company awaits FDA word on its lead drug candidate MIPLYFFA.

MIPLYFFA, known generically as Arimoclomol, is proposed for the treatment of Niemann-Pick type C, a rare inherited lysosomal storage disorder, with the regulatory agency’s decision expected on June 17, 2021.

If approved, MIPLYFFA would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) and the drug could achieve peak global sales of $340 million, according to analysts.

Orphazyme acquired the rights to Arimoclomol from CytRx Corp. (CYTR) in 2011 by paying a one-time, upfront payment of $150,000. CytRx is also entitled to receive up to a total of $120 million in milestone payments upon the achievement of certain pre-specified regulatory and business milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from Arimoclomol.

We had alerted readers to ORPH on May 5, 2021 when it was trading around $9. (Report titled “Orphazyme Betting Big On Arimoclomol”)

The stock closed yesterday’s trading at $21.00, up 302.30%.

FDA decision is a binary event as it is linked to volatility and uncertainty.

Will the FDA approve Orphazyme’s MIPLYFFA for the Niemann-Pick disease Type C indication or will it be sent back to the drawing board? Stay tuned for the update.