Mar­ket slack­er Cel­gene does some­thing good for a change, look­ing to turn the cor­ner on past mishaps

At this point Cel­gene has been so bat­tered over its poor per­for­mance of the past year you have to do a dou­ble take to con­vince your­self when some­thing good hap­pens to it.

But there was some con­sid­er­able good news to be had in its Phase III study of fran­chise drug Revlim­id in com­bi­na­tion with Roche’s Rit­ux­an in two forms — fol­lic­u­lar and mar­gin­al zone — of in­do­lent lym­phoma. 

Ac­cord­ing to re­searchers, they tracked a “high­ly sta­tis­ti­cal­ly sig­nif­i­cant” im­prove­ment in pro­gres­sion-free sur­vival in the study, open­ing the door to some near-term mar­ket­ing ap­pli­ca­tions that at least one promi­nent an­a­lyst spec­u­lates could be worth more than $1 bil­lion in added sales.

Cel­gene’s shares were up 1.6% in ear­ly trad­ing Tues­day.

Ge­of­frey Porges, Leerink

We don’t have the da­ta yet, and rarely do in these re­leas­es as the com­pa­nies hold the num­bers for a sci­en­tif­ic con­fer­ence. And over­all sur­vival re­sults have yet to be reached. But no one missed the im­pli­ca­tions of its boast on sta­tis­ti­cal sig­nif­i­cance. 

Cel­gene’s pipeline is stuffed with so many shots like this, some an­a­lysts say it can’t mess up every­thing. In ad­di­tion, it still has an R&D group filled with some of the best re­searchers in the busi­ness and a pipeline that boasts a group of po­ten­tial break­throughs, like bb2121 or JCAR017. So don’t think for a sec­ond that the com­pa­ny doesn’t have plen­ty of sup­port­ers around the in­dus­try.

Leerink’s Ge­of­frey Porges — who of­fered the up­beat in­cre­men­tal sales es­ti­mate with­out will­ing to change the com­pa­ny’s rev­enue fore­cast just yet — is al­so hap­py to give Cel­gene a thumbs up af­ter its many no­table set­backs. He not­ed:

(T)he pos­i­tive re­sult em­pha­sizes the op­tion­al­i­ty that ex­ists in many parts of the com­pa­ny’s pipeline. De­spite re­cent his­to­ry, not every­thing in the com­pa­ny’s port­fo­lio will fail, nor every op­er­a­tional re­sult dis­ap­point. This pes­simism about the com­pa­ny and its stock sug­gests op­por­tu­ni­ty to us, and the op­tion­al­i­ty of the mid- to late stage pipeline is a sig­nif­i­cant rea­son be­hind our con­tin­ued out­per­form rat­ing for the stock, de­spite the many de­vel­op­ment and op­er­a­tional stum­bles in the last year.

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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As­traZeneca's di­a­betes drug Farx­i­ga helps pa­tients with heart dis­ease and with­out di­a­betes in land­mark tri­al

Months ago, data on J&J’s $JNJ Invokana indicated the diabetes drug conferred cardiovascular (CV) benefit in patients who do and do not have preexisting CV disease. On Tuesday, AstraZeneca’s $AZN rival treatment, Farxiga, was shown to cut the risk of CV death or the worsening of heart failure in patients with heart disease, in a landmark trial.

The treatments, in addition to Jardiance from Eli Lilly $LLY, belong to a class of diabetes drugs called sodium-glucose co-transporter 2 (SGLT2) inhibitors, which work by curbing the absorption of glucose via the kidneys so that surplus glucose is excreted through urination.

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.
SpringWorks

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.