Gen­Sight vet Com­bal grabs $40M from No­var­tis, Roche group for sec­ond-wave gene ther­a­py biotech

Jean-Philippe Com­bal

Up un­til some months ago, Jean-Philippe Com­bal had been the COO at Paris-based Gen­Sight Bi­o­log­ics, fo­cused on a close­ly-watched late-stage study of an ex­per­i­men­tal gene ther­a­py for Leber’s Hered­i­tary Op­tic Neu­ropa­thy (or LHON), a rare mi­to­chon­dr­i­al dis­ease that de­stroys vi­sion. But in a quick leap, he’s now jumped ship, leav­ing CEO Bernard Gilly’s crew and launch­ing a new wave ther­a­py start­up of his own that has at­tract­ed about $40 mil­lion in start­up cash.

Vivet Ther­a­peu­tics is not a com­pet­ing ven­ture. Com­bal is jump­ing in­to liv­er and meta­bol­ic dis­eases, build­ing a plat­form tech to go af­ter a new set of rare dis­eases. First up for a clin­i­cal pro­gram is Wil­son Dis­ease, an or­phan dis­ease trig­gered by an er­rant gene that scram­bles the body’s abil­i­ty to reg­u­late lev­els of cop­per in the liv­er.

Work­ing with Jens Kurth, ex-Anokion and No­var­tis along with Glo­ria Gon­za­lez Aseguino­laza from CIMA at the Uni­ver­si­ty of Navar­ra, they have a lead gene ther­a­py dubbed VTX801 ready to go in­to the clin­ic next year. The mon­ey should get them through first steps in ear­ly-stage R&D work with enough cash to pay for a cou­ple of more INDs as they start pipeline con­struc­tion.

No­var­tis Ven­ture Fund and Colum­bus Ven­ture Part­ners led the round, with a syn­di­cate that in­cludes Roche Ven­ture Fund, Health­Cap, Kur­ma Part­ners and Ys­ios Cap­i­tal.

The nascent gene ther­a­py field has learned a lot from the first two gene ther­a­pies to ar­rive in Eu­rope. Gly­bera from uniQure is now be­ing yanked af­ter one use, and Strimvelis from GSK is find­ing con­sid­er­able re­sis­tance from pay­ers as well.

But Com­bal is look­ing on the bright side, gain­ing lessons from the re­al world ex­pe­ri­ence of the pi­o­neers to see how to build a gene ther­a­py com­pa­ny that can thrive, avoid­ing the pit­falls that await­ed the lead­ers.

“I think the top com­pa­nies to­day (which in­cludes US leader Spark Ther­a­peu­tics) of­fer a good ex­am­ple of what is pos­si­ble and not pos­si­ble,” Com­bal, al­so a Fovea and Sanofi vet­er­an, tells me. To be a suc­cess in this field, com­pa­nies are go­ing to need strong da­ta from at least two years of ex­pe­ri­ence in tri­al pa­tients.

“We need to get strong out­comes,” he adds, in or­der to get an ap­proval with the kind of demon­stra­tion of ef­fi­ca­cy that can win over pay­ers and agen­cies. “Strimvelis was very help­ful, show­ing that big com­pa­nies can go in­to gene ther­a­py.” And he be­lieves the he­mo­phil­ia stud­ies un­der­way now can de­liv­er the kind of strong da­ta need­ed to start build­ing mar­kets.

Com­bal is hap­py to let the pi­o­neers break these paths. He plans to be close be­hind, learn­ing at every step and in­cor­po­rat­ing those lessons in­to the busi­ness plan.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

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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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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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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