FDA hits a green light on 2 more rare dis­ease drugs, giv­ing Alex­ion an ear­ly shot at pro­tect­ing PNH fran­chise

Faced with the prospect of a par­tial gov­ern­ment shut­down lat­er to­day, the FDA has pulled the trig­ger on a pair of new drug ap­provals.

Alex­ion got its nod 2 months ear­ly for ALXN1210 for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, or PNH, which will now be sold as Ul­tomiris. And Stem­line Ther­a­peu­tics won an FDA ap­proval to mar­ket El­zon­ris (tagrax­o­fusp-erzs) for rare cas­es of blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm, or BPD­CN.

The FDA has now ap­proved 59 new drugs through CDER in 2018, part of its record-set­ting year as the in­dus­try en­joys a surge of new mar­ket­ing OKs.


Ge­of­frey Porges

Alex­ion $ALXN is plan­ning to price Ul­tomiris at a dis­count to Soliris, long one of the most ex­pen­sive ther­a­pies in the world. ALXN1210 proved just as ef­fec­tive as their big drug Soliris in treat­ing PNH, with less fre­quent dos­ing that will now pave the way to a dis­count price. Leerink’s Ge­of­frey Porges counts him­self as a sup­port­er of this strat­e­gy ahead of prospec­tive com­peti­tors for their PNH fran­chise.

While in­vestors may re­act neg­a­tive­ly to the ini­tial im­pact of this pric­ing head­wind (rev­enue for the PNH in­di­ca­tion for es­tab­lished pa­tients down 10%), we be­lieve the stock al­ready dis­counts sub­stan­tial price ero­sion and mar­ket share loss to com­peti­tors. In­stead, Alex­ion’s de­ci­sion to move to­ward more sus­tain­able pric­ing with a su­pe­ri­or prod­uct should front-run and counter these po­ten­tial risks, and build to­ward a more sta­ble rev­enue tra­jec­to­ry and longevi­ty than cur­rent­ly ex­pect­ed. 

Stem­line $STML, mean­while, had to en­dure a ker­fuf­fle ear­li­er on re­gard­ing its tardy ac­knowl­edge­ment of a pa­tient death dur­ing a small study of their drug, but the agency nonethe­less pro­vid­ing a thumbs-up on a small set of da­ta.

The drug was waved through af­ter post­ing wide­ly vary­ing re­sults from two pa­tient co­horts which re­ceived the drug in a tiny sin­gle-arm study.

The ef­fi­ca­cy of El­zon­ris was stud­ied in two co­horts of pa­tients in a sin­gle-arm clin­i­cal tri­al. The first tri­al co­hort en­rolled 13 pa­tients with un­treat­ed BPD­CN, and sev­en pa­tients (54%) achieved com­plete re­mis­sion (CR) or CR with a skin ab­nor­mal­i­ty not in­dica­tive of ac­tive dis­ease (CRc). The sec­ond co­hort in­clud­ed 15 pa­tients with re­lapsed or re­frac­to­ry BPD­CN. One pa­tient achieved CR and one pa­tient achieved CRc.

The OK comes with a black box warn­ing alert­ing physi­cians to be on alert for a host of po­ten­tial side ef­fects, but this is one con­di­tion where there are no ap­proved drugs, putting it on a pri­or­i­ty list for the FDA, which of­fered its break­through drug des­ig­na­tion to help speed it along.

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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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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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.

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.