(CercleFinance.com) – Pharnext on Monday unveiled new long-term safety and efficacy data deemed ‘very encouraging’ in patients with Charcot’s disease.
The main results of these data collected over a total duration of five years of clinical trials show in particular that PXT3003 showed a good tolerance and safety profile throughout the first Phase III clinical program.
The company also mentions encouraging efficacy results with regard to functional motor disability, the best efficacy signal having been observed in patients treated with PXT3003 for five years, whereas the condition of patients treated with the placebo has deteriorated.
According to Dr. Burkhard Blank, Pharnext’s medical director, these data ‘although from an open-label extension study, show a lasting benefit of treatment with high-dose PXT3003’.
The company thus shows the hope that PXT3003 could be the first approved treatment for Charcot-Marie-Tooth disease type 1A (CMT1A), a debilitating neurodegenerative disease that affects approximately 150,000 people in Europe and the United States.
Despite these announcements, the stock lost 12% on the Paris Stock Exchange on Monday, continuing a spectacular sell-off which has seen it fall by more than 90% since the start of the year due to the very dilutive impact of its latest funding sources.
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