Far from the big hubs, Apel­lis is steer­ing its ri­val to Alex­ion's Soliris in­to a PhI­II pro­gram

A Ken­tucky biotech says they’re lay­ing the foun­da­tion for a piv­otal pro­gram for their C3 in­hi­bi­tion ther­a­py, which ex­ecs be­lieve can re­place Soliris in treat­ing PNH.

Louisville-based Apel­lis has been mak­ing progress on its lead drug far away from the spot­light that con­cen­trates at­ten­tion on the big biotech hubs. But it’s been well fund­ed, with a $47 mil­lion D round that dropped ear­ly last year af­ter they gave up on an IPO in chilly mar­ket wa­ters. And the com­pa­ny says they’ve been nail­ing down hard hu­man ev­i­dence that by mov­ing up­stream from C5 in­hi­bi­tion, where Soliris hits, they can do a bet­ter job in con­trol­ling ane­mia and trans­fu­sion de­pen­dence among pa­tients with this ex­treme­ly rare con­di­tion.

This week, Apel­lis is re­port­ing on two tiny stud­ies of 3 and 6 pa­tients. In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side.

As one of the world’s most ex­pen­sive ther­a­pies, Soliris has in­spired a range of ri­vals all look­ing to re­place it with their own drug. Com­pa­nies like Ra Phar­ma­ceu­ti­cals and Akari have been on the trail, while Soliris’ man­u­fac­tur­er, Alex­ion, has been mak­ing ad­vances with a sec­ond-gen prod­uct for their key mon­ey­mak­er. ALXN1210 — an an­ti-C5 an­ti­body that in­hibits ter­mi­nal com­ple­ment for pa­tients with parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH) — was about the on­ly ex­per­i­men­tal prod­uct that earned much re­spect from new CEO Lud­wig Hantson when he took over ear­li­er this year.

Cedric Fran­cois

Apel­lis has at­tract­ed con­sid­er­able fi­nan­cial sup­port for its work. At the time it filed its S-1, the biotech re­port­ed that Morn­ing­side Ven­ture In­vest­ments owned 32.6% of the com­pa­ny, mak­ing the VC their biggest in­vestor. And they say that they’re just get­ting start­ed with a lead fo­cus on PNH, with a range of oth­er dis­eases that they be­lieve can be treat­ed through the same path­way.

CEO Cedric Fran­cois said he found the da­ta en­cour­ag­ing as he steers the com­pa­ny to a Phase III study in a few months.

We be­lieve that C3-in­hibitor APL-2 can be the next gen­er­a­tion PNH treat­ment of­fer­ing pa­tients a pow­er­ful so­lu­tion to mean­ing­ful­ly im­prove their qual­i­ty of life.


Im­age: Down­town Louisville, Ken­tucky Shut­ter­stock

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.