Ionis Sharing Royalties From Spinraza Sales for $500M Upfront

Agreement with Royalty Pharma allows company to advance in late-stage work

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the company is giving Royalty Pharma a percentage of the payments it receives from sales of Spinraza (nusinersen), an approved therapy for spinal muscular atrophy (SMA).

In exchange, Royalty Pharma agreed to pay Ionis $500 million upfront with other milestone payments possible, according to a company press release.

Ionis initially developed Spinraza and licensed it to Biogen in 2016. While Biogen now owns and markets the therapy, Ionis receives royalty payments based on its sales.

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All royalties from Spinraza sales eventually return to Ionis

Under the agreement, Royalty Pharma will receive 25% of these earnings up to $1.5 billion in annual sales through 2027, which will increase to 45% in 2028.

All royalties will revert back to Ionis when total Spinraza royalty payments reach a specified amount — either $475 million or $550 million — depending on certain events and their timing.

“This transaction provides us with significant capital to reach our strategic objectives, the first of which is to achieve commercial readiness for our late-stage programs,” said Brett P. Monia, PhD, Ionis’ CEO.  A company goal is to “continue to deliver a steady cadence of new genetic medicines to the market.”

The deal also includes a percentage of royalty earnings from pelacarsen, an investigational treatment for cardiovascular disease that Ionis licensed to Novartis in 2019. Royalty Pharma will pay Ionis up to an $625 million if certain developmental and commercial milestones for pelacarsen are met.

“We are thrilled to partner with Ionis, an innovator in genetic medicine. This investment is consistent with our strategy of acquiring royalties on innovative therapies in areas of high unmet patient need,” said Pablo Legorreta, founder and CEO of Royalty Pharma.

“Spinraza has transformed the lives of thousands of SMA patients,” Legorreta added.

Spinraza was the first SMA therapy to be approved and is indicated for children and adults with SMA, including those with types 1,2 or 3. It’s been approved in more than 50 countries worldwide, with more than 13,000 patients treated to date.

A type of treatment called an antisense oligonucleotide (ASO), Spinraza works to boost production of the survival motor neuron (SMN) protein that’s deficient in SMA.

Biogen also acquired an investigational SMA treatment from Ionis called BIIB115, an ASO that works in a similar way to Spinraza but might allow for less frequent dosing. As with Spinraza, Ionis will be entitled to royalty payments based on sales of BIIB115 should it be approved.

Donidalorsen, a potential therapy for people with hereditary angioedema, is among the treatments in late-stage development that Ionis is planning to advance.