Sentiment on biotech stocks overall ebbs and flows, but biotech companies can carve very individual paths. Drug company investors look to product pipelines to spot potential growth, especially for scientifically novel assets that can stand out from the pack.
These three biotech stocks have outperformed the group in the past few months thanks to potential blockbuster drugs.
• Juno Therapeutics (JUNO) is up 68% since its early February all-time low — which was also the IBD Medical-Biomed/Biotech group’s nearly two-year low point — as investors have regained confidence in its cancer-fighting pipeline. Juno is working in the cutting-edge field of cell therapy, in which patients’ own cells are re-engineered to help their immune systems kill cancer cells, in this case the chimeric antigen receptor (CAR) T cells.
Juno’s leading candidate, JCAR015, is in midstage testing for acute lymphoblastic leukemia, for which it showed stunning success rates in earlier trials. Last month, its partner Celgene (CELG) said it would exercise its option to commercialize JCAR015 and two similar candidates outside of North America and China, under a 10-year, $1 billion partnership that the two companies inked last summer.
Also last month, Juno showed off some of its earlier-stage CAR-T candidates at the annual meeting of the American Association for Cancer Research, targeting different receptors in other types of blood cancer. When the abstracts for the meeting were released in March, Leerink analyst Michael Schmidt wrote that Juno’s stock should be supported by “clinical data readouts supporting Juno’s business strategy to develop differentiated, potentially best-in-class CAR-T products that could generate translational insights in hematologic and solid tumor indications.”
• BioMarin Pharmaceutical (BMRN) is up 29% since its early February 20-month low as it recovered from the FDA’s rejection of its muscular-dystrophy drug Kyndrisa in January. At its R&D day last month, BioMarin rolled out strong early data for two pipeline candidates, one for hemophilia and one for achondroplasia, a form of dwarfism.
The hemophilia data brought particular attention, as it was the first-ever evidence on gene therapy for the disease in humans. After the therapy, BMN 270, was delivered once to eight patients, all of their conditions went from severe to mild-to-moderate.
This was far better than expected. In a video recorded for clients a couple of weeks later, Evercore ISI analyst John Scotti described the mood at BioMarin’s event: “When they started presenting (the data), jaws just went to the floor. There was an audible gasp in the room.”
The market for hemophilia A, the most common type, runs at $4 billion a year. Analysts say a successful gene therapy could massively disrupt the market of clotting factors that have to be infused weekly at least.
• Intercept Pharmaceuticals (ICPT) is up 52% from its 28-month low in February, after its lead drug Ocaliva passed a major hurdle to market last month. An FDA advisory committee voted to grant it accelerated approval for a liver condition called primary biliary cholangitis (PBC), which the agency has until May 29 to decide on.
While the PBC market is expected to bring in a healthy revenue stream for Intercept, the potential blockbuster indication is in nonalcoholic steatohepatitis (NASH), a liver-scarring disease that affects millions of Americans but has no treatment. Intercept is conducting a large phase-three trial of Ocaliva in the disease, with a launch not expected for several years. But the FDA panel vote did imply that the experts see the benefits as outweighing the drug’s known safety issues.
Ocaliva’s potential has driven buyout speculation around Intercept stock, which has also helped to push up shares. A particular burst of this came on Feb. 12 when anonymous sources suggested that the company was exploring a sale, though nothing’s yet come of it.