Fresh tri­al da­ta for­ti­fy po­si­tion of Roche's oral ther­a­py in spinal mus­cu­lar at­ro­phy bat­tle­ground

With an FDA de­ci­sion date loom­ing, Roche on Wednes­day un­veiled pos­i­tive piv­otal da­ta on its block­buster-bound oral spinal mus­cu­lar at­ro­phy (SMA) drug in pa­tients with the most se­vere form of the mus­cle-wast­ing dis­ease.

The FDA is set to make its de­ci­sion on the ther­a­py, ris­diplam, by May 24. It is ex­pect­ed to com­pete with Bio­gen’s Spin­raza and No­var­tis’ Zol­gens­ma.

Part­nered with PTC Ther­a­peu­tics, the Roche drug was test­ed in 41 pa­tients aged 1-7 months with type 1 SMA, a rare ge­net­ic mus­cle-wast­ing dis­ease. The tri­al, dubbed FIRE­FISH, mea­sured ef­fi­ca­cy via the pro­por­tion of in­fants sit­ting with­out sup­port af­ter 12 months of treat­ment, and longer.

The da­ta fol­lows an­oth­er pos­i­tive tri­al, the piv­otal SUN­FISH tri­al, meet­ing the thresh­old for change from base­line in mo­tor func­tion af­ter one year of treat­ment in pa­tients with type 2 or 3 SMA be­tween 2 and 25 years old.

The new Roche da­ta fur­ther high­lights the threat that Roche and part­ner PTC pose in the SMA space for Bio­gen, which se­cured the first-ever SMA ap­proval and has since been dosed in over 9,300 pa­tients, Cred­it Su­isse’s Evan Seiger­man wrote in a note.

“While the im­pact of No­var­tis’ Zol­gens­ma has been rel­a­tive­ly mut­ed, we think that the like­ly ap­proval and launch of ris­diplam could im­pact Bio­gen’s Spin­raza fran­chise,” he said. “We al­so think that Roche could price ris­diplam be­low Spin­raza to help en­cour­age use – es­pe­cial­ly in T2 or T3 pa­tients.”

Spin­raza, an an­ti­sense oligonu­cleotide, is in­ject­ed in the spine every four months fol­low­ing ini­tial load­ing dos­es. Zol­gens­ma, a gene-ther­a­py, is de­signed to be a one-shot cure, while ris­diplam is a dai­ly oral treat­ment, en­gi­neered to work by tweak­ing how the SMN2 gene is spliced, which rais­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

SMA is rare, af­fect­ing 1 per 8,000 to 10,000 peo­ple glob­al­ly, but rep­re­sents a lu­cra­tive bat­tle­ground for these drug­mak­ers. Spin­raza, launched in late 2016, car­ries a list price of $750,000 for the first year and $375,000 an­nu­al­ly there­after. Zol­gens­ma — on­ly ap­proved for pa­tients un­der the age of 2 — caused stick­er shock with its $2.1 mil­lion price tag and the in­evitable push­back from pay­ers, al­though No­var­tis has em­pha­sized that its five-year in­stall­ment plan and cu­ra­tive po­ten­tial makes it worth it.

With Roche’s plan to make ris­diplam cheap­er than Spin­raza, and the ap­peal of oral ad­min­is­tra­tion could make the drug an even big­ger threat to the Spin­raza fran­chise — which gen­er­at­ed more than $1.5 bil­lion in the first three quar­ters of 2019 — com­pared to the world’s most ex­pen­sive ther­a­py, Zol­gens­ma.

Seiger­man fore­cast $2.21 bil­lion in glob­al Spin­raza sales for 2020, and es­ti­mat­ed that num­ber would shrink to $1.79 bil­lion by 2022. “Spin­raza is not core to our the­sis at this point (yet still im­por­tant), as any rev­enue de­clines seen from Spin­raza could be made up for from ad­u­canum­ab (if ap­proved),” he wrote.

As for ris­diplam, an ap­proval is all but cer­tain. Cowen an­a­lysts in No­vem­ber fore­cast sales of about $2.7 bil­lion in 2026.

So­cial im­age: Roche

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Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

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An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

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