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.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Look­ing to move past an R&D fi­as­co, Ipsen poach­es their new CEO from Sanofi

Ipsen has turned to another Paris-based biopharma company for its next CEO.

Sanofi Pasteur chief David Loew — who’s been leading one of the most advanced efforts to develop vaccines for Covid-19 — is making the journey to Ipsen, 5 months after David Meek jumped ship to run a startup in late-stage development.

Loew arrives as Ipsen works to get back on track with their rare bone disease drug palovarotene, picked up in the $1.3 billion Clementia buyout, which was slammed with a partial hold after researchers observed cases of “early growth plate closure” in patients under the age of 14. But they are pushing ahead with the over-14 crowd after writing down slightly more than half of its initial development.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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