No­var­tis backs $30M round for can­cer biotech; Bay­er drug makes a ‘break­through’ at FDA

Ay­ala Ther­a­peu­tics — which fo­cus­es on rare can­cers that oc­cur due to dys­reg­u­la­tion of the Notch-ac­ti­vat­ing path­way — has raised $30 mil­lion in a round of fi­nanc­ing led by part­ner No­var­tis.

The funds, which al­so came from in­vestors in­clud­ing SBI JI In­no­va­tion Fund, Is­rael Biotech Fund, aMoon and Harel In­sur­ance & Fi­nance Group, will be used to fu­el the de­vel­op­ment of its lead drug, AL101. The pan-Notch in­hibitor is cur­rent­ly be­ing eval­u­at­ed for ade­noid cys­tic car­ci­no­ma (ACC) in a Phase II tri­al, and the com­pa­ny is al­so plan­ning to in­ves­ti­gate its use in a mid-stage study in triple neg­a­tive breast can­cer.

In 2017, Ay­ala inked a deal with Bris­tol-My­ers Squibb $BMY to de­vel­op treat­ments for can­cers with al­tered Notch genes. The fol­low­ing year, it joined forces with No­var­tis to de­vel­op and mar­ket an­oth­er drug, AL102, in com­bi­na­tion with BC­MA-tar­get­ing agents for mul­ti­ple myelo­ma pa­tients.

The Is­raeli biotech raised $17 mil­lion in its first round of fi­nanc­ing in April 2018.

→ The FDA grant­ed Bay­er’s Aliqopa (co­pan­lis­ib) — used for the treat­ment of adult pa­tients with re­lapsed mar­gin­al zone lym­phoma (MZL) who have re­ceived at least two oth­er ther­a­pies pri­or — break­through ther­a­py des­ig­na­tion based on over­all re­sponse rate (ORR) from its piv­otal Phase II CHRONOS-1 study. The com­pa­ny says that it “is con­duct­ing two ad­di­tion­al Phase III stud­ies to eval­u­ate the ef­fi­ca­cy and safe­ty of Aliqopa in com­bi­na­tion with oth­er ther­a­pies with iNHL (in­clud­ing MZL) who have re­lapsed fol­low­ing one or more pri­or ther­a­pies.”

→ Ger­man-based Bay­er $BAYRY and Foun­da­tion Med­i­cine $FMI are en­ter­ing a glob­al col­lab­o­ra­tion for the de­vel­op­ment and com­mer­cial­iza­tion of NGS-based com­pan­ion di­ag­nos­tic tests, which help find a pos­si­ble can­cer treat­ment ther­a­py via mol­e­c­u­lar in­for­ma­tion from a pa­tient’s tu­mor genome. Bay­er will al­so have ac­cess to Foun­da­tionOne CDx — Foun­da­tion Med­i­cine’s se­quenc­ing based in vit­ro di­ag­nos­tic de­vice.

The first project that will be un­der­tak­en is de­vel­op­ing a com­pan­ion di­ag­nos­tic for Vi­t­rakvi (larotrec­tinib), the first and on­ly TRK in­hibitor ap­proved in the US for adult and pe­di­atric pa­tients with “sol­id tu­mors that have a neu­rotroph­ic tropomyosin re­cep­tor ki­nase (NTRK) gene fu­sion with­out a known ac­quired re­sis­tance mu­ta­tion that are ei­ther metasta­t­ic or where sur­gi­cal re­sec­tion will like­ly re­sult in se­vere mor­bid­i­ty, and have no sat­is­fac­to­ry al­ter­na­tive treat­ments or that have pro­gressed fol­low­ing treat­ment.”

As­traZeneca spin­out Viela Bio has notched a com­mer­cial deal with sea­soned Chi­nese phar­ma Han­soh cen­tered around inebi­lizum­ab, the lead drug among six as­sets from the phar­ma gi­ant. The an­ti-CD19 drug treats neu­romyelitis op­ti­ca spec­trum dis­or­der, a rare dis­ease that af­fects the eye and spine. All told, the deal is worth up to $220 mil­lion in­clud­ing up­front and mile­stones.

The Park­er In­sti­tute is em­bark­ing on a new re­search jour­ney to find out why some pa­tients de­vel­op au­toim­mune dis­or­ders fol­low­ing can­cer im­munother­a­py. To­geth­er with two oth­er non­prof­its (ded­i­cat­ed to di­a­betes and health­care re­spec­tive­ly), it’s pledg­ing $10 mil­lion over three years to sup­port re­searchers at Yale, UCSF, Vir­ginia Ma­son and Dana-Far­ber. The hope is to both ad­vance can­cer treat­ment and bet­ter un­der­stand how type 1 di­a­betes de­vel­ops in gen­er­al.

→ In 2015, In­ovio joined forces with As­traZeneca’s $AZN Med­Im­mune unit for $27.5 mil­lion up­front, as well as an­oth­er po­ten­tial $700 mil­lion in mile­stone pay­ments — for the ex­clu­sive rights to In­ovio’s im­munother­a­py, de­signed to tar­get can­cers caused by hu­man pa­pil­lo­mavirus (HPV), in ad­di­tion to two ad­di­tion­al DNA-based can­cer vac­cine prod­ucts. On Wednes­day, In­ovio in a fil­ing dis­closed that Med­Im­mune was re­tain­ing its rights to the HPV im­munother­a­py, ME­DI0457 (pre­vi­ous­ly called INO-3112), but walk­ing away from the rest.

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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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 help@endpointsnews.com with any issues.

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As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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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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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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.