Self-proclaimed pioneer of RNA-targeted medicine Ionis Pharmaceuticals says it is looking to develop an “even better medicine” to Spinraza to treat spinal muscular atrophy (SMA).

At its annual meeting of stockholders earlier this month, Ionis Pharmaceuticals spoke about the success of its flagship molecule, Spinraza (nusinersen), an oligonucleotide approved by the US Food and Drug Administration (FDA) in December 2016 for the treatment of SMA.

“Spinraza is the standard-of-care for all forms of the disease SMA. It is a medicine that has transformed the lives of these patients and their families in ways that are inconceivable before Spinraza,” founder and CEO Stanley Crooke said.

Spinraza has recently been joined by Avexis’ Zolgensma as FDA approved treatments for SMA

“In a little more than two years it’s delivered more than $3 billion [€2.65 billion] in revenues and sale and we believe that it will have experienced about a $2 billion plus of sales year in 2019, it’s a blockbuster and it deserves to be.”

Co-developed with Biogen, the product is administered through multiple injections into the fluid surrounding the spinal cord. It has been the only option available to treat the rare and often fatal genetic disease affecting muscle strength and movement, SMA.

RNA vs gene therapy

However, last month the FDA approved Zolgensma (onasemnogene abeparvovec), a gene therapy developed by Novartis acquisition AveXis as a one-off treatment for SMA. Zolgensma has a wholesale acquisition cost (WAC) of $2.125 million but in a press release defending the high price tag, Novartis claimed the total cost for the patient is half the 10-year cost of “current chronic SMA treatment,” without directly naming Spinraza.

Spinraza has a list price of $125,000 per dose, equating to a cost of up to $750,000 for the first year and $375,000 for each year after.

At Bernstein’s 35th Annual Strategic Decisions Brokers Conference days after Zolgensma’s approval, Biogen management skirted around the subject of new competition in the SMA space.

“SMA continue to grow and to remain the standard of care in treating those patients, even if there is more competition that may come and address 5% of the current prevalence,” CEO Michel Vounatsos said (transcript here).

“We are used to competing. And by the way, I believe it’s very good for the patients to have options.”

Spinraza 2.0

Meanwhile, Ionis’ Crooke told his stakeholders his firm and Biogen were far from through in the RNA SMA space.

“We’re not done. We and Biogen are making solid progress in finding an even better medicine, a better Spinraza that may produce even greater benefit, and certainly will be more convenient for these patients to use because it would be administered much less frequently.”

Further details were not divulged.

Ionis pioneer

However, he alluded to where Ionis plays in the oligonucleotide and RNA-therapy field, and how continued innovation would strengthen the firm in the SMA space and others:

“We have pioneered the creation, not just of the new technology for drug discovery, but a new sector in our industry. Today, there are seven RNA-targeted medicines that have been approved for commercial use, five of those by Ionis. There are scores and scores of RNA targeted medicines in development, more than 40 of them are from Ionis,” he said.

“We have led the creation of this technology, we continue to be the leaders in this technology… And most excitingly after 30 years of steady progress, the pace of progress, our advances in the technology has never been faster.

“Innovation is very much alive and well at Ionis. We’ve shown that we can take those innovations and rapidly incorporate them into the medicines in our pipeline and that has led steadily improving performance of our medicines which of course expands the opportunity and allows us to approach ever greater commercial potentials.”