With Resta­sis in its crosshairs, My­lan adds a Botox biosim with Re­vance; Ver­tex starts sec­ond PhI­II triple com­bo CF study

With My­lan $MYL al­ready mov­ing in for the kill with a gener­ic ver­sion of Al­ler­gan’s Resta­sis, the big gener­ics com­pa­ny has set up a new pro­gram that can al­so go af­ter Al­ler­gan’s multi­bil­lion-dol­lar fran­chise for Botox. My­lan to­day an­nounced a deal to part­ner with Re­vance Ther­a­peu­tics $RVNC on a short-act­ing knock­off of Botox, which pro­vid­ed Al­ler­gan with chart-top­ping rev­enue of $3.4 bil­lion last year. Re­vance is al­ready an­gling to grab a share of the Botox mar­ket with a late-stage long-act­ing ri­val, along with com­pe­ti­tion from Evo­lus and Hugel. And there have been mixed re­views of just how suc­cess­ful that might be.

→ Ver­tex $VRTX is start­ing yet an­oth­er Phase III study of VX-659 (a sec­ond-gen­er­a­tion cor­rec­tor can­di­date), along with teza­caftor and iva­caftor as an in­ves­ti­ga­tion­al triple com­bo reg­i­men for peo­ple with cys­tic fi­bro­sis. Specif­i­cal­ly, the com­pa­ny is look­ing at CF pa­tients with two copies of the F508del mu­ta­tion, the most com­mon form of the dis­ease. The study will en­roll 100 pa­tients. This is the sec­ond triple com­bo study in­clud­ing these same ther­a­pies, with Ver­tex an­nounc­ing the first in Feb­ru­ary. That tri­al was en­rolling 360 pa­tients. “The first Phase III study…is de­signed to sup­port ap­proval of the VX-659 triple com­bi­na­tion in pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion who cur­rent­ly have no treat­ment that ad­dress­es the un­der­ly­ing cause of dis­ease,” said Jef­frey Chodake­witz, Ver­tex’s ex­ec­u­tive vice pres­i­dent and CMO, in a state­ment. “This sec­ond study is de­signed to en­able us to broad­en the po­ten­tial la­bel for this reg­i­men to in­clude those with the most com­mon ge­net­ic form of cys­tic fi­bro­sis.”

Al­ler­gan’s $AGN has run in­to a speed bump along the reg­u­la­to­ry road for a uter­ine fi­broids drug. In a very brief note, the Dublin-based phar­ma ac­knowl­edged that the FDA has ex­tend­ed the re­view of its NDA for ulipristal ac­etate, set­ting a PDU­FA tar­get ac­tion date in Au­gust. The com­pa­ny is fair­ly op­ti­mistic about the drug — which is al­ready avail­able in Eu­rope and Cana­da and aced both PhI­II tri­als — fore­cast­ing $500 mil­lion to $1 bil­lion in sales.

→ In the clos­ing chap­ter to an em­bar­rass­ing sto­ry about a failed gov­ern­ment re­search sub­sidy pro­gram, San­ford Burn­ham Pre­bys Med­ical Dis­cov­ery In­sti­tute has agreed to re­turn $12.3 mil­lion to the state of Flori­da for fail­ing to ful­fill re­quire­ments made in an in­cen­tive agree­ment. The state first hand­ed SBP $155 mil­lion in in­cen­tives in 2006 — un­der then-gov­er­nor Jeb Bush — amount­ing to one of the largest in­cen­tive pack­ages in the state’s re­cent his­to­ry, ac­cord­ing to the Or­lan­do Sen­tinel. By 2016, how­ev­er, the in­sti­tute had de­cid­ed to re­lo­cate to Cal­i­for­nia for fi­nan­cial rea­sons, with­out reach­ing the goal of cre­at­ing 303 jobs. SBP is still ne­go­ti­at­ing with lo­cal stake­hold­ers re­gard­ing tran­si­tion of its op­er­a­tions to an­oth­er en­ti­ty, but its of­fi­cials in­sist that the in­sti­tute had con­tributed sig­nif­i­cant­ly to Flori­da’s life sci­ences in­dus­try dur­ing its 10-year pres­ence.

With con­tri­bu­tion by John Car­roll and Brit­tany Meil­ing.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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Pay­back? Sarep­ta stunned as FDA spurns fol­lowup to Ex­ondys 51 for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.