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Ionis Pharma CEO: Nusinersen an 'enormous commercial opportunity'

Ionis Pharmaceutical shares jumped 30 percent on Monday on positive news of phase 3 data for its spinal muscular atrophy drug called Nusinersen, on which it partnered with Biogen.

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."





Dr. Stanley Crooke, CEO of Ionis Pharmaceuticals
Source: Stanley Crooke
Dr. Stanley Crooke, CEO of Ionis Pharmaceuticals
"I think that the results that we reported are truly remarkable." -Dr. Stnaley Crooke, Ionis Pharmaceuticals CEO

With positive study results, Crooke expects Ionis to be able to move forward aggressively to bring the study to an end. This will ensure that babies in the control arm can now receive Nusinersen. He said it is very important that Biogen's planned broad access program will allow babies to access Nusinersen earlier than they would if they had to wait to finish the trial.

"I think that the results that we reported are truly remarkable," Crooke said.

Ionis changed its name from Isis Pharmaceuticals late last year, and has pioneered antisense technology, which allows it to create drugs that target cells' RNA. Cramer described RNA as the biological equivalent of middle management: the DNA gives cells orders, and RNA carries those orders to the rest of the cell. Thus, if the message to the RNA is altered, it can fix many problems.

Initially investors were very excited about the 38 drugs in Ionis's pipeline. However, many biotech stocks went out of favor with political worries on drug prices and took Ionis down with them. The stock declined even further in May when GlaxoSmithKline said it would not be moving forward with phase 3 trials on a cardiomyopathy drug it had partnered with Ionis on.

"While the company has made a lot of missteps in the past year, there are a lot of drugs in the pipeline that could potentially pay off," the "Mad Money" host said.

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