FDA ex­perts turn thumbs down on PTC’s wob­bly case for its Duchenne MD drug

Aaron Kessel­heim

Af­ter a blis­ter­ing de­nun­ci­a­tion from the FDA of the da­ta that PTC Ther­a­peu­tics $PTCT has sub­mit­ted on be­half of its Duchenne mus­cu­lar dy­s­tro­phy drug ataluren, a pan­el of ex­perts vot­ed over­whelm­ing­ly against the drug, con­clud­ing that it’s still too ear­ly to say de­fin­i­tive­ly whether the drug works or not and ask­ing for some hard da­ta from a new study to lay the ques­tion to rest once and for all.

By a vote of 10 to 1, with a pa­tient rep­re­sen­ta­tive of­fer­ing the lone show of sup­port, the pan­el de­cid­ed to leave any fi­nal de­ci­sions on this drug un­til an­oth­er piv­otal tri­al can be wrapped.

“It seems like the ev­i­dence that kept com­ing out was most­ly from post hoc re-ex­am­i­na­tions of ex­ist­ing tri­als af­ter the da­ta had been re­vealed,” not­ed Aaron Kessel­heim of Har­vard Med­ical School. “And I’m con­cerned about the pos­si­bil­i­ty for whether con­scious­ly or sub­con­scious­ly there are ways that re­analy­sis of da­ta in that way can be mis­lead­ing.”

“Even the meta-analy­sis wasn’t as con­vinc­ing as it might have been be­cause of it be­ing based on sub­groups,” not­ed Johns Hop­kins’ Caleb Alexan­der. “So I’m not sure…a num­ber of post hoc analy­ses can re­place the con­fi­dence pro­vid­ed by a well-con­trolled ran­dom­ized tri­al that meets its pri­ma­ry pre­spec­i­fied end­points.”

The FDA’s in­ter­nal re­view that ap­peared pub­licly two days ago slammed PTC for forc­ing a new ex­am­i­na­tion of ataluren, a drug that has failed two stud­ies for DMD as well as an­oth­er piv­otal shot at cys­tic fi­bro­sis — which did noth­ing for its case. The agency has re­fused to even ac­cept PTC’s ap­pli­ca­tion on two ear­li­er oc­ca­sions, say­ing the biotech nev­er of­fered close to enough da­ta to war­rant the time and ef­fort.

Janet Wood­cock

The same com­mit­tee of­fered an­oth­er neg­a­tive, though con­sid­er­ably clos­er, vote against a ri­val drug from Sarep­ta. But de­spite adamant ob­jec­tions from se­nior staffers, Janet Wood­cock weighed in on be­half of eteplirsen and pulled out an ac­cel­er­at­ed OK. The drug, though, has a la­bel that states ex­plic­it­ly that there’s no sol­id ev­i­dence of ef­fi­ca­cy in its fa­vor.

PTC, though, had won an OK in Eu­rope for ataluren with the same da­ta and clear­ly felt that it would have a shot, helped by a long line­up of pa­tients and their par­ents who spoke out on be­half of the drug.

They were wrong.

The FDA doesn’t have to fol­low the pan­el’s guid­ance, but giv­en the in­ter­nal re­view and the neg­a­tive vote — along with Wood­cock’s ab­sence from the pro­ceed­ings to­day — PTC would not seem to stand a chance.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.

Credit: AP Images

Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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