Rat­tle and hum: Roche, PTC post up­date on their SMA drug as a shak­en Bio­gen faces yet an­oth­er fran­chise buster

Right on the heels of No­var­tis’ lat­est da­ta on its SMA gene ther­a­py for spinal mus­cu­lar at­ro­phy, PTC and its part­ners at Roche/Genen­tech post­ed their own pos­i­tive take on the lat­est da­ta for their oral ri­val ris­diplam. And a shaky Bio­gen will have to take it in­to ac­count as well as they as­sess the fu­ture for their block­buster drug Spin­raza, which has led the way on treat­ing this dis­ease for more than 2 years.

Tot­ting up their lat­est scores, the ris­diplam team chalked up:

  • 9 in­fants (53%) were able to main­tain an up­right head po­si­tion (HINE-2, n=17)
  • 10 in­fants (59%) demon­strat­ed rolling (rolling to the side, prone to supine, or supine to prone) (HINE-2, n=17)
  • 7 in­fants (41%) were able to sit with­out sup­port for at least 5 sec­onds (BSID-III, n=17)
  • 1 in­fant (6%) was able to stand (HINE-2, n=17)
  • 85.7% (18/21) ba­bies were event-free over­all

Three pa­tients had died, though. And while the re­searchers were quick to note that none of the in­fants died as a re­sult of ther­a­py, both Spin­raza and No­var­tis’ treat­ment have racked up pro­longed pe­ri­ods with no deaths.

That sets the stage for an FDA ap­pli­ca­tion lat­er this year, as No­var­tis awaits word from the agency any day now on its pitch for a gene ther­a­py.

SVB Leerink’s Mani Foroohar looked it over and pro­nounced him­self sat­is­fied.

Topline ris­diplam da­ta re­leased by Roche pri­or to the pre­sen­ta­tions at AAN lat­er this af­ter­noon con­tin­ue to demon­strate an ef­fi­ca­cy and safe­ty pro­file that ap­pears sup­port­ive of reg­is­tra­tion across spinal mus­cu­lar at­ro­phy (SMA) sub­types.

The ju­ry is still out on how this ri­val­ry will play out, but the prospect of a gene ther­a­py as well as an oral drug hit­ting the mar­ket may well have a pro­found ef­fect on Spin­raza. Bio­gen sells this drug for $750,000 for the first year, and then half that for each year there­after. That gives No­var­tis plen­ty of head­room to look for around $2 mil­lion for a claimed once-and-done ther­a­py. And then Roche along­side PTC could cause se­ri­ous dis­rup­tion with what­ev­er price they pick.

Mani Forhoor


What­ev­er par­ents pre­fer, in­sur­ers will have a lot of say over who gets what.

None of that bodes well for Bio­gen, which can ill af­ford to see its fran­chise eat­en up by ri­vals af­ter its big late-stage Alzheimer’s drug ad­u­canum­ab just crashed and burned.

“Bot­tom line — we could see a 1/3, 1/3, 1/3 split of mar­ket over time, Jef­feries an­a­lysts re­cent­ly said that the three drugs could end up di­vid­ing things even­ly. Based on their dis­cus­sions with physi­cians, gene ther­a­py “could be 30-33% share of new SMA Type I-III pa­tients over time. An es­ti­mat­ed 20-25% could look to switch from Spin­raza to gene ther­a­py (de­pends on weight of pa­tient). 2) Sim­i­lar­ly, docs sug­gest 1/3 share for oral ris­diplam, too, af­ter 3-5 years; 3) Com­men­tary by docs sug­gests an even split of opin­ions on the mar­ket as well — from oral pills ‘very ap­peal­ing’ and ‘game chang­er’ and ‘es­pe­cial­ly at­trac­tive for teens/adults’ to oth­er ther­a­pies need more da­ta and like­ly com­bo.”

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources.