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.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Once re­ject­ed, Kala's dry eye drug now gains en­try to a field where No­var­tis is groom­ing its own block­buster

When the FDA slapped a rejection on Kala Pharma’s dry eye drug last August, the biotech’s execs promised investors that a third Phase III study — they had already started at that point — would reverse their fortune.

Today they made good on that promise, clinching an approval for Eysuvis, an ocular corticosteroid being positioned as a first-line, short term treatment of dry eye disease.

Boasting a technology invented by Bob Langer out of MIT, Eysuvis is a corticosteroid, loteprednol etabonate, delivered by mucus-penetrating particles. It promises to enhance penetration into target tissue on the ocular surface, achieving an effect quicker than systemic corticosteroids and stronger than over-the-counter eye drops.

Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Covid-19 roundup: Sanofi and GSK pledge 200M vac­cine dos­es for a glob­al dis­tri­b­u­tion cam­paign

Sanofi and GSK have agreed to give 200 million doses of their vaccine candidate to the COVAX Facility, which is part of a program set up by CEPI, the WHO and Gavi to equitably distribute vaccines around the world.

The idea behind COVAX is to give all participating countries equal access to vaccines, regardless of income level. As of Oct. 14, more than 180 countries had signed agreements to the COVAX Facility, including France and the UK. China joined earlier this month, pledging to make its vaccines a “global public good.” One country notably off the list is the United States.