Puma in­vestors fret, as Ner­l­ynx Q1 sales fall un­com­fort­ably short of Wall Street es­ti­mates

“We are not pleased with our first-quar­ter Ner­l­ynx rev­enues,” Puma Biotech­nol­o­gy chief Alan Auer­bach said in a post-earn­ings con­fer­ence call on Thurs­day, cit­ing a spate of dis­con­cert­ing fac­tors, in­clud­ing pa­tient dis­con­tin­u­a­tions. In­vestors con­curred with his as­sess­ment, as the stock $PBYI cratered. The dis­dain con­tin­ued ear­ly Fri­day, as shares fell near­ly 37% to $18.96 be­fore the bell.

Alan Auer­bach

The drug, which was in-li­censed by Puma from Pfiz­er $PFE, was ap­proved by the FDA in Ju­ly 2017, to pre­vent breast can­cer re­lapse, fol­low­ing ther­a­py in pa­tients with HER2-pos­i­tive can­cer. The treat­ment is known to have a poor tol­er­a­bil­i­ty pro­file, pre­dom­i­nant­ly re­lat­ing to di­ar­rhea. The Los An­ge­les-based com­pa­ny post­ed Q1 Ner­l­ynx sales of $45.6 mil­lion — a 25% de­cline quar­ter-over-quar­ter — and well be­low Street con­sen­sus ex­pec­ta­tions of about $67 mil­lion.

“The dis­con­tin­u­a­tions oc­cur more fre­quent­ly in the first month. While most physi­cians pre­scribe an an­ti-di­ar­rheal med­ica­tion with Ner­l­ynx, our re­search shows that some pa­tients may not fill the an­ti-di­ar­rheal pre­scrip­tion. There were al­so some physi­cians not pre­scrib­ing any an­ti-di­ar­rheal pro­phy­lax­is med­ica­tions at all, which we be­lieve was due to the lack of aware­ness of the da­ta from our con­trol tri­al us­ing pro­phy­lac­tic an­ti-di­ar­rheal drugs,” Puma CCO Steven Lo said on the call.

Steven Lo

Oth­er rea­sons for the sales miss were at­trib­uted to: pa­tient dis­con­tin­u­a­tions ow­ing to the pro­gres­sion of the dis­ease, or loss of in­sur­ance; an ab­nor­mal­ly high va­can­cy rate in the sales force, with 18 of 80 sales ter­ri­to­ries en­dur­ing turnover dur­ing the quar­ter; and pa­tients com­plet­ing one year of treat­ment on Ner­l­ynx as in­di­cat­ed on the la­bel.

“The neg­a­tive im­pact from treat­ment dis­con­tin­u­a­tions di­rect­ly re­lat­ed to Ner­l­ynx’s chal­leng­ing tol­er­a­bil­i­ty pro­file, which has seem­ing­ly had a re­laps­ing/re­mit­ting im­pact on quar­ter­ly com­mer­cial re­sults, was ex­plic­it­ly de­scribed as worse than ex­pect­ed – in­creas­ing­ly se­quen­tial­ly…Def­i­nite­ly more dis­con­cert­ing, how­ev­er, was the rev­e­la­tion that at cer­tain pe­ri­ods dur­ing the quar­ter, the com­pa­ny’s “high­er than av­er­age va­can­cy rate in (their) sales force” with 18 of 80, or 22% of sales ter­ri­to­ries ‘not hav­ing a sales rep at some point in the quar­ter.’ While seats have ap­par­ent­ly been most­ly re-filled…this sce­nario rais­es con­cerns re­gard­ing the sus­tain­abil­i­ty of the ap­par­ent re­bound seen in April (2Q to date). How­ev­er, the sub­dued tone with which com­men­tary was de­liv­ered, makes it chal­leng­ing to muster con­fi­dence that com­mer­cial dy­nam­ics can achieve even low­ered full-year 2019 Ner­l­ynx sales guid­ance,” Cowen an­a­lysts wrote in a note.

SVB Leerink an­a­lysts low­ered their 2019 US rev­enue es­ti­mates to $221 mil­lion from $265 mil­lion and tem­pered their peak US sales ex­pec­ta­tions to about $369 mil­lion from $400 mil­lion.

Af­ter suc­cess­ful­ly lob­by­ing Eu­ro­pean reg­u­la­tors to have a change of heart and ap­prove Ner­l­ynx last year, Puma palmed off the treat­ment to Pierre Fab­re — its sixth mar­ket­ing part­ner —with the Unit­ed States and Japan as the on­ly re­main­ing un­en­cum­bered ma­jor mar­kets.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

In a boost to Rit­ux­an fran­chise, Roche nabs quick ap­proval for po­latuzum­ab ve­dotin

Roche’s lat­est an­ti­body-drug con­ju­gate has crossed the FDA fin­ish line, gain­ing an ac­cel­er­at­ed ap­proval a full two months ahead of sched­ule.

Po­livy, or po­latuzum­ab ve­dotin, is a first-in-class drug tar­get­ing CD79b — a pro­tein promi­nent in B-cell non-Hodgkin lym­phoma. It will now be mar­ket­ed for dif­fuse large B-cell lym­phoma as part of a reg­i­men that al­so in­cludes the chemother­a­py ben­damus­tine and a ver­sion of rit­ux­imab (Rit­ux­an).

An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Plagued by de­lays, As­traZeneca HQ costs soar to £750M as it edges to­ward 2020 com­ple­tion

In the lat­est up­date on As­traZeneca’s de­lay-prone HQ project, the phar­ma gi­ant re­vealed that the cost of con­struc­tion has swelled to £750 mil­lion ($956 mil­lion) — more than dou­ble the orig­i­nal es­ti­mate in 2013.

The move-in date is still in 2020, a spokesper­son con­firmed, af­ter As­traZeneca pushed pro­ject­ed com­ple­tion from 2016 to 2017, and then to the spring of 2019. While the ini­tial plan called for a £330 mil­lion (then $500 mil­lion) in­vest­ment, the cost bal­looned to £500 mil­lion ($650 mil­lion), and more in the most re­cent up­date.

Fresh analy­sis spot­lights car­dio ben­e­fit of J&J's In­vokana in di­a­betes pa­tients with­out his­to­ry of CV dis­ease

In­vokana sales may be mut­ed, but the di­a­betes drug is set to get some love af­ter its mak­er J&J un­veiled da­ta at the Amer­i­can Di­a­betes As­so­ci­a­tion meet­ing on Tues­day sug­gest­ing the med­i­cine can con­fer a car­dio­vas­cu­lar ben­e­fit in pa­tients who do not have pre­ex­ist­ing CV dis­ease.

Back in April, J&J had re­port­ed that in the late-stage CRE­DENCE study, the SGLT2 drug scored a 30% re­duc­tion in the risk of a com­pos­ite of ail­ments: a pro­gres­sion to the dou­bling of serum cre­a­ti­nine, end-stage kid­ney dis­ease and re­nal or car­dio­vas­cu­lar death. In terms of sec­ondary end­points, the drug was al­so found be heart-pro­tec­tive: low­er­ing the risk of CV death and hos­pi­tal­iza­tion for heart fail­ure by 31%, as well as ma­jor ad­verse CV events by 20%. In March, the com­pa­ny sub­mit­ted an ap­pli­ca­tion to ex­pand In­vokana’s la­bel to re­flect its im­pact on chron­ic kid­ney dis­ease.

Sil­i­con Val­ley's most an­tic­i­pat­ed slide deck just dropped. What does it mean for bio­phar­ma's dig­i­tal teams?

These aren’t the typ­i­cal slides you’d see at End­points — no mol­e­cules, clin­i­cal pro­grams, or p-val­ues. In­stead, we’ll talk dig­i­tal and in­ter­net trends, fac­tors that elite glob­al brands — re­gard­less of in­dus­try — must first mea­sure and un­der­stand be­fore de­ploy­ing prod­ucts in­to the world. That’s a con­cept that most of our Big Phar­ma au­di­ence is in tune with. Dig­i­tal aware­ness is key to suc­cess in the dis­cov­ery, de­vel­op­ment, and mar­ket­ing of new bio­phar­ma­ceu­ti­cals, and most of the ma­jors now have a chief dig­i­tal of­fi­cer: No­var­tis, Sanofi, and Pfiz­er, just to name a few.