Shares of a company developing a drug for sickle cell disease dropped by more than 50 percent premarket Monday morning following the announcement that a closely watched Phase III study in SCD had failed.
Rockville, Maryland-based GlycoMimetics said Friday evening that according to top-line results, the RESET study of rivipansel in SCD met neither its primary nor key secondary endpoints, though it did not provide further details of the results. RESET, which was being sponsored by New York-based Pfizer, measured as its primary endpoint the time until patients were ready for discharge from the clinic for patients receiving the drug versus those receiving placebo. It enrolled 341 patients aged 6 and older who had bee hospitalized for vaso-occlusive crisis, a form of sickle-cell pain that can cause irreversible organ damage.
Shares of GlycoMimetics were down nearly 60 percent on the Nasdaq after markets opened Monday morning, trading at less than $4 per share. They had closed at $9.13 per share Friday afternoon. Shares of Pfizer were down 1 percent on the New York Stock Exchange.
“We are disappointed with the results, as we have been working in close partnership with the SCD community to advance rivipansel as a potential treatment for acute VOC,” said Brenda Cooperstone, senior vice president for rare diseases at Pfizer Global Product Development, in a statement.
Ahead of the trial results, Cowen analyst Ritu Baral wrote in a note to investors that the bank saw potential for major upside given the low expectations but strong prior Phase II data.
Subsequent to the Monday announcement, Baral wrote that it is impossible to ascribe any value to rivipansel at present due to the lack of visibility on the data and statistics or further details about when those will be released, despite a conversation with the company’s management. “A best case scenario, where data showing consistent trends in all points was presented at ASH in December, could provide some remaining upside from the program,” she wrote.
“We are both surprised and deeply disappointed by this outcome, as we had strongly hoped that rivipansel would have a positive benefit for people living with sickle cell disease,” GlycoMimetics CEO Rachel King said in a statement. “We are grateful to the many people who supported and advanced this program over the years of clinical study, especially to sickle cell patients and their families.”
Other than rivipansel, GlycoMimetics is also running a Phase III study of uproleselan in relapsed and refractory acute myeloid leukemia, results of which are expected near the end of next year.
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