Orphazyme A/S (ORPH) stock prices plummeted by 27.77% after the market opened on May 7th, 2021, bringing the price per share tumbling down to USD$6.12 after the commencing of the trading day.
ORAIALS-01 Ends in Failure
The company announced on May 7th, 2021 that the ORAIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints. While the trial did not report any significant safety signals, topline data will be disclosed in an ENCALS virtual meeting from May 12th to 14th 2021. Complete data from the study is scheduled to be published later in 2021.
Making the Most of the Situation
The disheartening conclusion of arimoclomol failing to benefit the demographic gripped by ALS was not in vain, however. As one of the longest-running clinical studies in its category, the 18 months of evaluation have yielded data that the company hopes will meaningfully contribute to the scientific dialogue on and around ALS. ORPH will also apply the invaluable insights gained from its previous studies to further develop its pipeline as the company continues to facilitate the actualization of the full potential of the heat shock protein response.
Details of the Trial
The third phase of the trial was randomized and placebo-controlled, with 245 patients spread out across 29 sites over 12 countries in North America and Europe. The administration of either arimoclomol or a placebo for 76 weeks was determined randomly, with one patient being given a placebo for every two patients that got the drug.
Details of Endpoints
The primary endpoint was to establish the effectiveness of consistent treatment with the drug in participants with ALS as assessed by the combined assessment of function and survival (CAFS), with the placebo control group serving as a baseline from which results were hoped to vary significantly. This endpoint served to emphasize the net treatment effect based on survival and changes in the ALS Functional Rating Scale-Revised score. Secondary endpoints included these bases, as well as slow vital capacity (SVC).
Future Outlook for 2021
The news of the ORAIALS-01 pivotal trial’s failure to meet endpoints has spelled apparent doom for ORPH stock prices. As the prices continue to spiral, the company must prove prospects of profitability elsewhere in is pipeline and product portfolio.