PTC's ataluren flops in an­oth­er PhI­II and an an­a­lyst says it's clear­ly a dud; kill it

PTC Ther­a­peu­tics CEO Stu­art Peltz

PTC Ther­a­peu­tics has whiffed once again on Translar­na (ataluren).

The biotech re­port­ed that ataluren, a re­peat flop in Duchenne mus­cu­lar dy­s­tro­phy, failed a Phase III for non­sense mu­ta­tion cys­tic fi­bro­sis on both the pri­ma­ry and sec­ondary end­points.

In­ves­ti­ga­tors tracked a slight but in­signif­i­cant ad­van­tage for pa­tients in the drug arm of the  CF study, not­ing a bet­ter num­ber for lung ca­pac­i­ty and the rate of pul­monary ex­ac­er­ba­tions. But none of it stacked up in a way worth tak­ing to reg­u­la­tors, and the biotech says it will scrap its work in CF and jerk its ap­pli­ca­tion for ap­proval in Eu­rope.

The com­pa­ny’s stock $PTCT dropped 20% on the news.

PTC has a long track record of fail­ure in the clin­ic when it comes to ataluren. A flop in Phase IIb as well as Phase III for DMD, the FDA re­fused to ac­cept its ap­pli­ca­tion for re­view in ear­ly 2016, say­ing the biotech’s pitch failed to qual­i­fy for a re­view — a rare slap­down from reg­u­la­tors. PTC is now ap­peal­ing that de­ci­sion for the sec­ond time.

De­spite that, though, Eu­ro­pean of­fi­cials have al­lowed the sale of Translar­na for Duchenne mus­cu­lar dy­s­tro­phy. The Eu­ro­pean Com­mis­sion rat­i­fied an ex­ten­sion of PTC’s mar­ket­ing ap­proval in ear­ly Jan­u­ary as PTC pur­sues an­oth­er study to keep its mar­ket­ing au­tho­riza­tion in place. And the biotech ex­pects to earn more than $100 mil­lion this year on sales out­side the US, backed by a num­ber of pa­tient ad­vo­cates who be­lieve the drug works, de­spite the da­ta.

RBC’s Simos Sime­oni­dis said this af­ter­noon that the string of tri­al fail­ures demon­strate that the drug is clear­ly a dud and ought to be pulled.

Fol­low­ing this com­pound’s fail­ure in the ACT DMD tri­al, which was sup­posed to be the con­fir­ma­to­ry tri­al (which based on post-hoc analy­ses from a Phase II tri­al in DMD that had failed to meet its pri­ma­ry end­point), we no longer be­lieved this com­pound worked in CF, and had thus as­signed 0% prob­a­bil­i­ty of suc­cess for it in ACT CF. We now be­lieve there is am­ple clin­i­cal ev­i­dence from mul­ti­ple ran­dom­ized clin­i­cal tri­als that this is an in­ac­tive com­pound.

“We are dis­ap­point­ed with the out­come of this tri­al as there are no treat­ments that tar­get the un­der­ly­ing cause of non­sense mu­ta­tion cys­tic fi­bro­sis, one of the most dif­fi­cult forms to treat,” said Stu­art W. Peltz, PhD, chief ex­ec­u­tive of­fi­cer of PTC Ther­a­peu­tics in pre­pared text. “We are par­tic­u­lar­ly grate­ful to pa­tients and in­ves­ti­ga­tors who par­tic­i­pat­ed in our tri­als. We re­main com­mit­ted to pa­tients re­ceiv­ing ataluren in oth­er in­di­ca­tions.”

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.

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The Advance 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.

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.

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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.

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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.

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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.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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De­nali un­veils new way of cross­ing blood brain bar­ri­er as the big neu­ro­science bet en­ters its clin­i­cal years

Five years ago, as much of pharma began leaving neuroscience, three big-name scientists from Genentech and some A-list investors, including ARCH and Flagship, made a $217 million bet that new genetic insights and a reliance on biomarkers could bring them success. They called it Denali Therapeutics.

Still, Denali faced the problem that neuroscience developers have faced for decades: How do you get a large molecule across the blood-brain barrier, a natural defense evolved precisely to keep them out? Enzyme replacement therapy, for instance, would be a great candidate to treat several neurological disorders, but enzymes can’t cross the barrier.