Al­ler­gan, So­sei halt glob­al de­vel­op­ment for Alzheimer’s drug fol­low­ing tox­ic re­ac­tion in a non-hu­man pri­mate

Al­ler­gan and So­sei have hit the brakes on an in­ter­na­tion­al de­vel­op­ment pro­gram fea­tur­ing a key Alzheimer’s drug fol­low­ing an “un­ex­pect­ed tox­i­col­o­gy find­ing” for one of the non-hu­man pri­mates it test­ed the ther­a­py on.

The drug is HTL0018318, a mus­carinic M1 re­cep­tor ag­o­nist that Al­ler­gan part­nered on when it signed a de­vel­op­ment deal with So­sei’s UK sub­sidiary Hep­tares which was worth close to $840 mil­lion, plus bil­lions in po­ten­tial sales bonus­es. 

Tim Tasker

Ac­cord­ing to the com­pa­nies, they have sus­pend­ed clin­i­cal work, halt­ing a Phase I study Al­ler­gan is run­ning in the US and a Phase II tri­al in Japan cen­tered on de­men­tia with Lewy bod­ies. Hep­tares has al­ready com­plet­ed a Phase Ib Eu­ro­pean study and that da­ta are be­ing an­a­lyzed now.

Re­searchers did not spell out what kind of tox­ic re­ac­tion was tracked in the an­i­mal study.

The US biotech agreed to pay So­sei $125 mil­lion up­front — with hun­dreds of mil­lions in mile­stones and $50 mil­lion for re­search sup­port — to launch their al­liance in 2016. And this drug was spot­light­ed as one of the most im­por­tant ther­a­pies in the batch. Alzheimer’s in spe­cif­ic and cog­ni­tion in gen­er­al, though, re­mains one of the tough­est fields in R&D, fea­tur­ing fre­quent break­downs for in­ef­fec­tive­ness as well as the oc­ca­sion­al dan­ger­ous side ef­fect.

So­sei now ex­pects the de­lay will cost the com­pa­ny a ma­jor mile­stone next year as they re­set the clock on the de­vel­op­ment plan — pro­vid­ed they can un­der­stand what hap­pened in the pri­mate study and if the drug is still safe for pa­tients.

The ju­ry is still out, though, on this drug, which has now been test­ed in more than 300 peo­ple around the world.

“We were very sur­prised to see these re­sults giv­en the safe­ty pro­file HTL0018318 has ex­hib­it­ed across all pre­vi­ous an­i­mal and clin­i­cal stud­ies,” not­ed So­sei CMO Tim Tasker. “We are com­mit­ted to work­ing with clin­i­cal in­ves­ti­ga­tors, R&D teams and reg­u­la­to­ry au­thor­i­ties to un­der­stand bet­ter the rea­son for the find­ings from this an­i­mal tox­i­col­o­gy study and so en­able the hu­man clin­i­cal de­vel­op­ment pro­gram with HTL0018318 to con­tin­ue as soon as pos­si­ble.”

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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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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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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