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.”

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

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 at 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.

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.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.