A ma­jor com­peti­tor to Bio­gen’s Spin­raza fran­chise could ar­rive as ear­ly as this com­ing May, as Roche and its biotech part­ner ob­tained a pri­or­i­ty re­view for ris­diplam.

FDA reg­u­la­tors will make a de­ci­sion on the spinal mus­cu­lar at­ro­phy drug by May 24, 2020, ac­cord­ing to PTC Ther­a­peu­tics, who is bag­ging $15 mil­lion in mile­stone pay­ment for the NDA fil­ing ac­cep­tance.

The move comes on the heels of topline da­ta from the Phase III SUN­FISH tri­al, in which ris­diplam met the pri­ma­ry end­point on change from base­line in the Mo­tor Func­tion Mea­sure 32 (MFM-32) scale af­ter one year of treat­ment, com­pared to place­bo. The re­sults con­firmed ear­li­er find­ings from part one of the study.

Levi Gar­raway

SUN­FISH en­rolled 180 pa­tients with type 2 or 3 SMA be­tween 2 and 25 years old. An ear­li­er, open la­bel tri­al dubbed FIRE­FISH cov­ered those with the type 1 vari­a­tion of the neu­ro­mus­cu­lar dis­ease.

“The FIRE­FISH and SUN­FISH tri­als were de­signed to rep­re­sent the re­al world spec­trum of peo­ple liv­ing with SMA and in­clude many peo­ple pre­vi­ous­ly un­der­rep­re­sent­ed in clin­i­cal tri­als,” not­ed Levi Gar­raway, Roche’s new­ly mint­ed head of glob­al prod­uct de­vel­op­ment, in a state­ment.

In PTC’s re­lease, CEO Stu­art Peltz added that the NDA sub­mis­sion in­cludes all three types of SMA pa­tients “demon­strat­ing im­prove­ments in mo­tor func­tions and de­vel­op­men­tal mile­stones, and a com­pelling safe­ty pro­file.”

Un­like Spin­raza, an an­ti­sense oligonu­cleotide ad­min­is­tered in­trathe­cal­ly every four months fol­low­ing ini­tial load­ing dos­es, ris­diplam is an oral med­ica­tion to be tak­en dai­ly. By mod­i­fy­ing how the SMN2 gene is spliced, the drug sup­pos­ed­ly in­creas­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

Stu­art Peltz

The drug is al­ready dec­o­rat­ed with the FDA’s or­phan and fast track des­ig­na­tions.

The fil­ing could lead to a broad­er la­bel than ex­pect­ed, ac­cord­ing to a note from Cred­it Su­isse an­a­lyst Evan Seiger­man, who’s pre­vi­ous­ly warned that ris­diplam spells an even big­ger threat to Bio­gen’s Spin­raza fran­chise than No­var­tis’ gene ther­a­py. Since gain­ing ap­proval in May, Zol­gens­ma has been plagued by re­im­burse­ment is­sues as pay­ers push back against its $2.1 mil­lion price tag (al­though No­var­tis stress­es that the pay­ment is de­liv­ered in a 5-year in­stall­ment plan for a sup­posed once-and-done cure). It’s al­so on­ly ap­proved for in­fants un­der the age of 2.

Al­though Spin­raza has earned Bio­gen more than $1.5 bil­lion in the first three quar­ters of 2019, Seiger­man said he is “in­creas­ing­ly con­cerned” about its dura­bil­i­ty. Hav­ing launched late 2016 (and ac­crued more than $4.1 bil­lion in record­ed sales so far), the fran­chise may be­gin to wane come H2 2020.

And Roche is go­ing all-in for the three-ri­val ri­val­ry. To­geth­er with PTC and the SMA Foun­da­tion, the phar­ma gi­ant is run­ning more clin­i­cal tri­als with pa­tients rang­ing from new­borns to 60 years old, in­clud­ing those pre­vi­ous­ly treat­ed with oth­er SMA ther­a­pies.

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.