No­var­tis to spin off Al­con eye care unit, buy back $5B in stock; Ri­val CAR-Ts from No­var­tis and Gilead get thumbs up in Eu­rope

→ Hav­ing mulled over it for more than a year, No­var­tis $NVS is fi­nal­ly spin­ning off Al­con, the eye care unit that for­mer CEO Daniel Vasel­la pur­chased for a to­tal of $51.6 bil­lion. His tenure marked an era when phar­ma­ceu­ti­cal com­pa­nies were try­ing to di­ver­si­fy their busi­ness in face of gener­ic com­pe­ti­tion to brand­ed drugs. But No­var­tis is a dif­fer­ent com­pa­ny now, led by for­mer R&D chief Vas Narasimhan, who told Bloomberg of the de­ci­sion: “We want to be able to fo­cus our cap­i­tal al­lo­ca­tion to our core, and we be­lieve our core is go­ing to be nov­el plat­forms to de­vel­op in­no­v­a­tive med­i­cines and to in­vest in da­ta and dig­i­tal tech­nolo­gies.” The move, which fol­lows Narasimhan’s de­ci­sion to sell No­var­tis’ stake in a con­sumer health­care joint ven­ture to part­ner GSK for $13 bil­lion, comes at a time Al­con — long con­sid­ered an un­der­per­former — is bounc­ing back on rev­enue. That might help with the val­u­a­tion, which ex-CEO Joe Jimenez said could range from $25 bil­lion to $35 bil­lion. Cur­rent Al­con CEO Mike Ball will be­come chair­man af­ter the spin­off, with COO David En­di­cott slat­ed to be­come the new chief — as­sum­ing the mo­tion goes through at No­var­tis’ an­nu­al gen­er­al meet­ing next year. Af­ter the spin-off wraps up in the first half of 2019, No­var­tis is plan­ning to buy back up to $5 bil­lion in stock.

→ Two ri­val CAR-T drugs both got nods from the Eu­ro­pean Med­i­cines Agency pan­el, a sign that their paths to the Eu­ro­pean mar­ket is clear. The Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use (CHMP) rec­om­mend­ed No­var­tis$NVS Kym­ri­ah for the treat­ment of B cell acute lym­phoblas­tic leukemia and dif­fuse large B cell lym­phoma (DL­B­CL). At the same time, the CHMP rec­om­mend­ed Gilead’s $GILD Yescar­ta for DL­B­CL and pri­ma­ry me­di­asti­nal B cell lym­phoma and trans­formed fol­lic­u­lar lym­phoma. Eu­rope’ reg­u­la­to­ry agency usu­al­ly fol­lows the com­mit­tee’s ad­vice, which means both CAR-Ts could soon go head-to-head in DL­B­CL in Eu­rope.

→ Ul­tragenyx $RARE al­so scored CHMP’s pos­i­tive opin­ion, rec­om­mend­ing the com­pa­ny’s drug Mep­se­vii to treat non-neu­ro­log­i­cal man­i­fes­ta­tions of Mu­copolysac­cari­do­sis VII in Eu­rope. Mep­se­vii is an en­zyme re­place­ment ther­a­py de­signed to re­place the de­fi­cient lyso­so­mal en­zyme be­ta-glu­curonidase in pa­tients with MPS VII, a pro­gres­sive and de­bil­i­tat­ing rare ge­net­ic dis­ease.

→ Pre­ci­sion Ther­a­peu­tics, an Ea­gan, Min­neso­ta-based com­pa­ny ap­ply­ing AI to per­son­al­ized med­i­cine and drug dis­cov­ery, is merg­ing with Pitts­burgh di­ag­nos­tics com­pa­ny Helomics. The merg­er ups Pre­ci­sion’s stake in Helomics from 25% to 100%, and gets Pre­ci­sion ac­cess to Helomics’ suite of AI, pre­ci­sion di­ag­nos­tic, and in­te­grat­ed CRO ca­pa­bil­i­ties, the com­pa­nies said in a state­ment. Carl Schwartz, Pre­ci­sion’s CEO, com­ment­ed, “Up­on com­ple­tion of the merg­er, we will have com­plete own­er­ship of Helomics’ one of a kind tu­mor data­base, which has been de­vel­oped over 15 years of clin­i­cal test­ing and con­tains drug re­sponse pro­files of over 149,000 pa­tient can­cer tu­mors, and its D-CHIP bioin­for­mat­ics en­gine that pro­vides ac­tion­able in­sights in­to this da­ta.”

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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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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UP­DAT­ED: Pay­back? An­a­lysts say Sarep­ta was blind­sided by an FDA re­jec­tion dri­ven by reg­u­la­to­ry re­venge

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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FDA de­ci­sion on Ver­tex's CF triple will come just ahead of planned CEO shake­up

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.

An MIT spin­out kills one of its ‘liv­ing ther­a­peu­tics’ af­ter flunk­ing an ear­ly-stage study — shares rout­ed

Just a few weeks after bagging $80 million in a deal to collaborate with Gingko Bioworks on its special blend of engineered bacteria used for “living therapeutics,” little Synlogic in Boston $SYBX is tossing one of its two clinical programs after watching an early-stage study go down in defeat.

Their Phase Ib/IIa study for SYNB1020 to counter the accumulation of ammonia in the body, a condition called hyperammonemia or urea cycle disorder, floundered at the interim readout, forcing the biotech to kill it and reserve its cash for pipeline therapies with greater potential.

Elan­co to buy Bay­er's an­i­mal health busi­ness for $7.6B, as deal­mak­ing gath­ers steam in the sec­tor

Last week, Elanco explicitly dodged answering questions about its rumored interest in Bayer’s animal health business in its post-earnings call. On Tuesday, the Eli Lilly spinoff disclosed it was purchasing the German drug maker’s veterinary unit in a cash-and-stock deal worth $7.6 billion. 

Elanco $ELAN has been busy on the deal-making front. In April, it laid out plans to swallow its partner, Kansas-based pet therapeutics company Aratana $PETX. A July report by Reuters suggested a potential Bayer deal was being explored, and Bloomberg last week said the deal was imminent, citing sources. 

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.

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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