Karyopharm Therapeutics (NASDAQ:KPTI)

Institutional ownership is very high as is management's. Key partnerships with large pharma and joint projects validate products and science. Issues are whether they can expand beyond current expertise and also monetize their products.

Last year Karyopharm received FDA approval for a therapy called selinexor as multiple myeloma therapy in patients who have received at least four prior lines but still had refractory disease.
Multiple myeloma is a cancer of plasma cells found in the bone marrow. It’s estimated that 30,000 people are diagnosed with this form of cancer every year in the U.S. alone. There are multiple approved therapies, but it still kills about 13,000 people per year. The disease is often more managed as a chronic condition than cured, with patients hitchhiking to new drugs as old ones stop working.

Selinexor is still relatively new on the market, so its potential as a fifth-line therapy is substantial. The company estimates this market at 6,000 patients per year.

But Karyopharm is working on clinical trials designed to showcase selinexor’s ability as a second- or third-line therapy, which, at 32,000 patients per year, is a substantially larger market. Moreover, multiple myeloma patients tend to stay on their early lines of therapy for longer, meaning it’s an even larger market than just head count.

Karyopharm received data for selinexor in this group earlier this year that it believes support an additional approval with the FDA. It submitted this Supplemental New Drug Application today, meaning the company could now have access to a much larger market upon approval, something that could happen by early next year.
Moreover, Karyopharm is studying selinexor in multiple clinical trials for additional cancer types and even COVID-19! It has submitted an application for this drug in diffuse large B-cell lymphoma (DLBCL) with the FDA expected to respond by June 23, 2020.

But there are even more big catalysts in this small biotech. Karyopharm also expects Phase 3 trial results for selinexor in liposarcoma by the end of the year, and the company is expected to launch a COVID-19 trial soon.

These are big catalysts in a biotech with a market cap of just $1.3 billion. Best of all, the company has plenty of cash, reporting $385 million in cash and investments as of the end of the last quarter, which should be enough to fund it through these big near-term catalysts.