As­traZeneca’s PhI­II bid on asth­ma drug tralok­inum­ab flops, and that’s no sur­prise

Maybe the best way to look at As­traZeneca R&D now is by di­vid­ing the re­search group in­to two big buck­ets: can­cer and every­thing else. That “every­thing else” group was just hit with a wide­ly ex­pect­ed Phase III fail­ure that the drug gi­ant can ill af­ford as it strug­gles to make a con­vinc­ing case for a des­per­ate­ly need­ed turn­around.

Sean Bo­hen, As­traZeneca

This time the late-stage fail­ure be­longs, once again, to its IL-13 asth­ma drug tralok­inum­ab. In its first of two Phase III stud­ies, the drug failed to sig­nif­i­cant­ly re­duce an­nu­al ex­ac­er­ba­tion rates. There is a sec­ond Phase III un­der­way that As­traZeneca be­lieves could hold the key to ul­ti­mate suc­cess, say­ing that they will com­bine da­ta from the two in de­ter­min­ing its fu­ture.

In ad­di­tion, re­searchers point­ed to a sub-pop­u­la­tion analy­sis in which pa­tients with an el­e­vat­ed mark­er for IL-13 re­spond­ed bet­ter to the drug and they plan to make that a spe­cial fo­cus of the sec­ond tri­al.

How­ev­er.

This drug al­ready failed a Phase IIb tri­al for asth­ma, rais­ing ques­tions about why the com­pa­ny would go ahead and con­tin­ue a ma­jor late-stage ef­fort need­ed to get an OK in asth­ma. But the re­searchers at the time said they were en­cour­aged by a sub­group analy­sis that point­ed to suc­cess. The com­pa­ny qui­et­ly dumped the id­io­path­ic pul­monary fi­bro­sis pro­gram, but in­sist­ed that there was every rea­son to be­lieve that tralok­inum­ab would work in asth­ma. On top of that, Roche’s drug le­brik­izum­ab, al­so an IL-13, came up with a split de­ci­sion in its two Phase III stud­ies last year that amount­ed to a se­vere set­back on that front.

As­traZeneca post­ed some mixed — though gen­er­al­ly pos­i­tive — da­ta from two big Phase III stud­ies of ben­ral­izum­ab, its IL-5 con­tender in the high­ly com­pet­i­tive mar­ket for se­vere asth­ma, last fall. A high place­bo re­sponse se­ri­ous­ly erod­ed one set of late-stage da­ta.

The phar­ma gi­ant sold off the der­ma­tol­ogy rights for tralok­inum­ab to Leo Phar­ma, which says it gath­ered pos­i­tive IPF da­ta on the drug re­cent­ly. As­traZeneca al­so punt­ed rights to bro­dalum­ab af­ter that drug proved dis­ap­point­ing, though ap­prov­able, in Phase III with ev­i­dence that it trig­gered sui­ci­dal think­ing.

As­traZeneca’s stock was large­ly un­af­fect­ed by the Phase III fail­ure to­day, which couldn’t have sur­prised many an­a­lysts. The big play at As­traZeneca now is fo­cused on MYS­TIC, its com­bi­na­tion study of dur­val­um­ab and treme­li­mum­ab in lung can­cer. As­traZeneca has had some big suc­cess­es in can­cer over the last cou­ple of years, win­ning a first OK for dur­val­um­ab and pro­gress­ing with oth­er drugs like Tagris­so and Lyn­parza. But out­side can­cer the com­pa­ny has had a se­ries of grim flops that con­tin­ue to raise ques­tions about CEO Pas­cal So­ri­ot’s promise of a turn­around.

Re­search chief Sean Bo­hen had this to say in the com­pa­ny’s de­fense to­day:

Se­vere asth­ma is a het­ero­ge­neous dis­ease with sig­nif­i­cant un­met needs and we will now await the STRATOS 2 re­sults in the sec­ond half of 2017 to ex­plore the po­ten­tial to treat a sub-group of un­con­trolled asth­ma pa­tients with tralok­inum­ab.

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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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.

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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.

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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.

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.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.