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.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.