Pharnext has announced it has completed patient enrolment in the pivotal Phase III PREMIER trial evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A) meeting its previously announced timeline of Q222. This follows the recently presented positive data from the long-term open-label PLEO-CMT-FU study, which continued to show a sustained benefit for patients after five years of treatment and which has a strong read-across for the PREMIER trial, in our view. Recruitment completion also means that the 15-month study stays on track for a Q423 readout. Success here would make PXT3003, which has orphan drug designation in the United States and Europe, the first drug to be approved for this condition. Pharnext's share price climbed as high as 62% on the news before settling down to close with a modest gain.Den vollständigen Artikel lesen ...
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