Al­ny­lam makes RNAi his­to­ry again as patisir­an gets ap­proved in Eu­rope; Ver­tex buys in­to UK-based ma­chine learn­ing group

→ Just days af­ter Al­ny­lam land­ed a his­toric FDA OK on patisir­an — and quick­ly launched it as On­pat­tro — the Eu­ro­pean Com­mis­sion has come through with its own mar­ket­ing au­tho­riza­tion. The drug, which treats hered­i­tary AT­TR amy­loi­do­sis by si­lenc­ing mes­sen­ger RNA and block­ing the pro­duc­tion of TTR pro­tein, is the first RNAi ther­a­py to be ap­proved in Eu­rope. It’s al­so a like­ly block­buster-to-be, with Clar­i­vate peg­ging 2022 pro­ject­ed sales at $1.21 bil­lion even though the dis­ease af­flicts on­ly about 30,000 peo­ple world­wide. In­ot­ersen, a ri­val prod­uct from Akcea and Io­n­is, was ap­proved in Eu­rope in Ju­ly.

Ver­tex is part­ner­ing with UK-based Ge­nomics plc on their plat­form for ge­net­ics and ma­chine learn­ing. Ver­tex $VRTX made a £10.5 mil­lion eq­ui­ty in­vest­ment in Ge­nomics plc as part of its £25 mil­lion Se­ries B fund­ing round — al­so an­nounced to­day. “Hu­man bi­ol­o­gy and ge­net­ics are a foun­da­tion of drug dis­cov­ery at Ver­tex and the team at Ge­nomics brings to­geth­er a unique com­bi­na­tion of so­phis­ti­cat­ed da­ta sci­ence and hu­man ge­net­ics,” said Ver­tex CSO David Alt­shuler. “This part­ner­ship will pi­o­neer new us­es of ge­nom­ic tools and tech­nolo­gies to ad­vance Ver­tex’s on­go­ing work and in­vest­ment at our Ox­ford Re­search Site and glob­al­ly to bring new med­i­cines to the pa­tients that need them.”

→ We fi­nal­ly know what to call Chi­na’s re­vamped FDA. The drug reg­u­la­tor will now go by the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion, or NM­PA, to re­flect its new man­date af­ter a gov­ern­ment re­or­ga­ni­za­tion sep­a­rat­ed med­i­cine reg­u­la­tion from oth­er con­sumer goods — in­clud­ing every­thing food-re­lat­ed. NM­PA re­ports to the new­ly cre­at­ed State Ad­min­is­tra­tion for Mar­ket Reg­u­la­tion.

Evotec and the CH­DI Foun­da­tion have ex­tend­ed their neu­ro­science col­lab­o­ra­tion for a few more years. In the pact, CH­DI will help fund the work of up to 75 sci­en­tists fo­cused on Hunt­ing­ton’s dis­ease.

Tem­pus, a pre­ci­sion med­i­cine com­pa­ny pro­vid­ing da­ta an­a­lyt­ic ser­vices to physi­cians and re­searchers, has raised $110 mil­lion in Se­ries E fi­nanc­ing. Er­ic Lefkof­sky — best known for co-found­ing Groupon — start­ed the com­pa­ny to as­sist re­al-time de­ci­sion-mak­ing for pa­tients, first in can­cer care. Har­ness­ing their pool of pa­tient da­ta, the com­pa­ny al­so op­er­ates an organoid de­vel­op­ment and test­ing fa­cil­i­ty to build bi­o­log­i­cal mod­els that they be­lieve can in­form clin­i­cal tri­als.

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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