Spinal muscular atrophy (SMA) is the leading cause of infantile death worldwide. Ionis CEO Dr. Stanley Crooke spoke with Jim Cramer on Monday, and estimated 30,000 infants around the world are born with SMA.
The drug could potentially be a $2 billion franchise, which is much higher than previous estimates, according to analysts. Ionis could receive royalties from those sales in the mid-teens, as well as $150 million in milestone payments from Biogen if everything goes well. This is in addition to the $75 million license fee that Biogen just paid Ionis.
"You can easily come to the notion of a very large commercial opportunity," Crooke said. "Obviously that's Biogen's area of responsibility, and so I won't be able to comment further, other than we believe this is an enormous commercial opportunity."