Io­n­is rolls out promis­ing PhI­II da­ta on volane­sors­en for rare en­zyme dis­or­der, but safe­ty ques­tions linger

Louis O’Dea, Akcea

Io­n­is has nailed down pos­i­tive Phase III da­ta on its lead ex­per­i­men­tal drug volane­sors­en, used to com­bat rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome. But the biotech $IONS is still bat­ting back ques­tions about safe­ty is­sues as it preps for fil­ings with the FDA and EMA.

In the Phase III in­ves­ti­ga­tors re­port­ed a huge drop in triglyc­erides among the 33 FCS pa­tients tak­ing the drug, hit­ting the pri­ma­ry end­point. The place­bo arm saw the lev­el of triglyc­erides go up, help­ing to il­lus­trate the treat­ment ef­fect.

The drug is de­signed to tack­le a dis­ease caused by hered­i­tary mu­ta­tions that in­hib­it the ac­tiv­i­ty of lipopro­tein li­pase, need­ed to break down triglyc­erides car­ried by chy­lomi­crons.

But re­searchers al­so not­ed that five pa­tients were forced out of the tri­al due to a threat­en­ing de­cline in platelet counts. Grade 4 throm­bo­cy­tope­nia oc­curred in three pa­tients, which end­ed af­ter they stopped dos­ing. There were no with­drawals due to platelet counts af­ter the com­pa­ny be­gan mon­i­tor­ing the side ef­fect, but the safe­ty is­sue did not pass un­no­ticed.

“(O)ve­r­all the dis­con­tin­u­a­tion rate in the study was high­er than we had ex­pect­ed, and high­er than pre­vi­ous­ly seen in the COM­PASS safe­ty tri­al in se­vere hy­per­triglyc­eridemia (20%),” not­ed Leerink’s Paul Mat­teis.

Io­n­is’ sub­sidiary Akcea al­so tracked some ad­di­tion­al ben­e­fits, in­clud­ing the end of pan­cre­ati­tis at­tacks dur­ing the 52-week study as well as a re­duc­tion in ab­dom­i­nal pain.

Some an­a­lysts, though, will well re­mem­ber that Io­n­is stunned in­vestors last May with the news that its drug IO­N­IS-TTR­Rx trig­gered throm­bo­cy­tope­nia in pa­tients with TTR amy­loid car­diomy­opa­thy. The FDA then put the pro­gram on a clin­i­cal hold, spurring its part­ner GSK to drop plans to pur­sue a late-stage study. CEO Stan­ley Crook al­so not­ed at the time that re­searchers had seen the same is­sue in their work on volane­sors­en, spurring fears that this could be a class ef­fect.

The biotech’s shares were down 6% by ear­ly Mon­day af­ter­noon.

Since then the com­pa­ny has gone to some lengths to down­play the im­pact of throm­bo­cy­tope­nia, not­ing that physi­cians can mon­i­tor pa­tients and con­trol its im­pact.

Io­n­is has had its ups and downs along the way, but 2016 end­ed on a high note as its part­ner Bio­gen scored an FDA ap­proval for Spin­raza, which costs $750,000 for its first year.

“The suc­cess of AP­PROACH rep­re­sents an im­por­tant mile­stone to­wards our first reg­u­la­to­ry sub­mis­sions for volane­sors­en in the U.S., Eu­rope and Cana­da in 2017,” said Dr. Louis O’Dea, chief med­ical of­fi­cer, Akcea Ther­a­peu­tics.  “We seek to bring this new treat­ment as ex­pe­di­tious­ly as pos­si­ble to FCS pa­tients who have a high un­met need with po­ten­tial­ly life-threat­en­ing con­se­quences.”

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.