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.”

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.