Based in Newton, Massachusetts, global pharmaceutical company Karyopharm Therapeutics (NASDAQ:KPTI) announced on June 22nd that it received approval from the U.S. Food and Drug Administration (FDA) for its XPOVIO (Selinexor) product for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
The regulatory green light makes XPOVIO the only single-agent oral therapy on the market for treating adult patients with DLBCL, an aggressive cancer that originates in white blood cells and typically grows in lymph nodes found in the armpits, groin, neck, and other parts of the body. It is a disease that often continues to progress in patients despite the application of multiple types of chemotherapy and targeted-drug combination therapy.
It also means that XPOVIO becomes the first and only FDA-approved drug that can be used to treat both DLBCL and multiple myeloma, a type of cancer that accumulates in bone marrow.
The clinical profile and tolerability of XPOVIO gives physicians an alternative treatment to traditional intravenous chemotherapy regimens.
Favorable response rate observed under accelerated FDA program
The approval was based on the drug’s favorable response rate under the FDA’s Accelerated Approval Program. The oral XPOVIO treatment showed a clinically meaningful overall response rate (ORR) of 29% including a 13% complete response rate and a 16% partial response rate in a pivotal study that involved multiple subtypes of the disease. At the six-month mark, 38% of the responding patients maintained a response rate which was supportive of the drug’s durability.
Karyopharm’s multi-center, single-arm phase 2b study included 134 patients who were previously given anywhere from one to five other systemic therapies. The patients received a fixed 60mg dose of XPOVIO twice weekly for a period of four weeks.
Founder and President Dr. Sharon Shacham commented, “The accelerated approval of oral XPOVIO in patients with relapsed or refractory DLBCL is a significant milestone for the patients and families who currently have limited treatment options available for their disease.”
Karyopharm develops novel first-in-class drugs for the treatment of cancer and other diseases. Its selective inhibitor of nuclear export (SINE) compounds have also shown biological activity in neurodegeneration, inflammation, autoimmune disease, certain viruses, and wound healing models.
The XPOVIO treatment becomes commercially available immediately in the U.S. for its second oncology indication after previously being approved by the FDA for use in multiple myeloma patients. Next year, the company also plans to submit a Marketing Authorization Application (MAA) for the product’s use in patients with DLBCL to the European Medicines Agency.
Despite the positive news, shares of Karyopharm Therapeutics finished flat on the day of the announcement at $18.98 after climbing as high as $20.99 during morning trading.
The stock is well-liked among sell-side analysts as eight out of nine analysts have ‘buy’ recommendations with a price target range of $25 to $40.
Karyopharm is scheduled to report second quarter financial performance on August 4th. The Street is forecasting a net loss per share of $0.53 which would be an improvement over the $0.71 per share that the company posted in the second quarter of 2019.