March 24 (Reuters) – Karyopharm Therapeutics said on Tuesday its therapy for patients with a rare blood cancer showed a reduction in spleen size in a late-stage trial, meeting one of the two main goals of the study.
Shares of the company were up nearly 14% in premarket trade.
In the 353-patient trial, half of those given Karyopharm’s drug Xpovio in combination with Incyte’s Jakafi saw their spleen size reduced by at least 35% after 24 weeks. In comparison, only 28% of patients who received only Jakafi saw a similar spleen size reduction.
However, the drug missed the other goal of showing a statically significant improvement in patients’ symptoms between those receiving the combination and those on Jakafi alone.
Karyopharm also said the combination may help patients live longer, with a preliminary analysis suggesting a more than 50% reduction in risk of death compared with Jakafi alone. It plans to continue monitoring survival outcomes.
The company said it will meet the U.S. Food and Drug Administration to discuss the results and a potential application for approval.
Xpovio, which was first greenlit by the FDA in 2019, is approved to treat other blood cancers, such as types of myeloma and lymphoma.
The blood cancer myelofibrosis causes scarring in the bone marrow, which makes it difficult for the tissue to make healthy blood cells, and causes an enlarged spleen and progressive anemia.
According to Karyopharm, myelofibrosis affects about 20,000 people in the United States. Current treatments rely on JAK inhibitors such as Jakafi.
Separately, Karyopharm agreed to a $30 million private placement with RA Capital, with up to $44 million more if accompanying warrants are fully exercised.
The proceeds will support general corporate purposes, including clinical programs.
(Reporting by Puyaan Singh and Sahil Pandey in Bengaluru; Editing by Krishna Chandra Eluri)
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