Look­ing to cure Type 1 di­a­betes, in­vestors front $114M to launch a pi­o­neer­ing hu­man study at Sem­ma

Three years ago, Har­vard’s Doug Melton pub­lished a land­mark study out­lin­ing how he had suc­cess­ful­ly used stem cells to cre­ate in­sulin-pro­duc­ing pan­cre­at­ic be­ta cells that were in­sert­ed in bulk in­to mice and suc­cess­ful­ly pro­tect­ed from an im­mune re­sponse — a break­through in re­gen­er­a­tive med­i­cine that bore re­al promise to pro­vide a cu­ra­tive ap­proach for Type 1 di­a­betes that could con­ceiv­ably end a life­time of in­sulin shots.

It was the cul­mi­na­tion of 23 years of lab work, launched when his son was di­ag­nosed with Type 1 di­a­betes. And that achieve­ment marked the be­gin­ning of some­thing new in biotech.

That same year Sem­ma Ther­a­peu­tics would be launched — with a $44 mil­lion A round land­ing in 2015 — in pur­suit of a mis­sion to com­plete one of the most am­bi­tious pre­clin­i­cal pro­grams in the re­gen­er­a­tive med field. And af­ter work­ing on all the nit­ty grit­ty re­search need­ed to see if this tech could be scaled up to hu­man size, an ex­pand­ed syn­di­cate of ven­ture in­vestors have put to­geth­er a whop­ping $114 mil­lion round with plans to take this in­to hu­mans for a first-of-its-kind proof-of-con­cept study.

One of the big chal­lenges Sem­ma faced in scal­ing up, Melton tells me, was to cre­ate a mem­brane specif­i­cal­ly de­signed with pores that were large enough for mol­e­cules to pass through but too small for im­mune cells to pen­e­trate. Us­ing some cal­cu­la­tions from the lab, Melton and his col­leagues es­ti­mat­ed that they would need some 150 mil­lion cells — pos­si­bly rang­ing up to three times that amount — in or­der to pro­vide the nat­ur­al in­sulin need­ed to elim­i­nate the shots.

Melton com­pares the mem­brane to a tea bag, but one that couldn’t be over­loaded. The re­place­ment cells, he said, “will on­ly se­crete the right amount de­pend­ing on the lev­el of sug­ar in the blood.”

Mark Fish­man

The big round marks an in­flec­tion point for the 35 staffers at Sem­ma, says chair­man Mark Fish­man, who joined Har­vard af­ter a 13-year stint run­ning the No­var­tis In­sti­tutes for Bio­Med­ical Re­search.

“Un­til you get this kind of fund­ing,” says Fish­man, “you don’t know how broad your strat­e­gy can be. With this fund­ing, we can get through a proof-of-con­cept tri­al, with enough in­for­ma­tion to know whether this works.” They can fol­low par­al­lel tracks and al­so start think­ing through some new di­rec­tions to pur­sue as their di­a­betes treat­ment pro­ceeds.

Typ­i­cal­ly, you nev­er see VCs back­ing a di­a­betes play. A few ma­jor multi­na­tion­als con­trol the bulk of the de­vel­op­ment work be­cause the reg­u­la­to­ry re­quire­ments for ap­proval are daunt­ing. But that’s in Type 2, which is spread­ing at epi­dem­ic pro­por­tions. In­her­it­ed Type 1 di­a­betes has a much small­er pop­u­la­tion, adds Fish­man, which makes it pos­si­ble to con­sid­er push­ing ahead in­to late-stage de­vel­op­ment alone.

As for time­lines, Fish­man is play­ing his cards close to the vest. Ear­ly-stage re­search, as he knows all too well, has a lot of vari­ables that can af­fect time­lines. An IND is com­ing, he says, and the com­pa­ny will see how it plays out, with a spe­cial fo­cus in start­ing to look at how durable a sin­gle treat­ment can be — one of the the big is­sues that Melton is most in­trigued by.

The mega-round in play al­so un­der­scores the will­ing­ness of ven­ture back­ers to go big these days when they’re fo­cused on mak­ing a pi­o­neer­ing ad­vance.

Eight Roads Ven­tures and Cowen Health­care In­vest­ments co-led the fi­nanc­ing with help from MPM Cap­i­tal, F-Prime Cap­i­tal Part­ners and Arch Ven­ture Part­ners. Ex­ist­ing strate­gic part­ners in­clude No­var­tis, Medtron­ic and the JDRF T1D Fund, and new in­vestors jump­ing in in­clude ORI Health­care Fund, Wu Cap­i­tal, 6 Di­men­sions Cap­i­tal and SinoPharm Cap­i­tal. Sem­ma named Daniel Auer­bach from Eight Roads and Kevin Raidy from Cowen to its board of di­rec­tors.

As­traZeneca trum­pets the good 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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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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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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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

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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No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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Stymied by the pan­dem­ic, Im­munomedic­s' new CEO bows out, tak­ing a mil­lion bucks plus perks as he heads out the vir­tu­al ex­it

Just a little more than a month since taking over as the latest CEO to helm Immunomedics, $IMMU Harout Semerjian is exiting the company after being confronted by “logistical” obstacles thrown up by the pandemic that made it impossible for him to move from London to carry out the job. And he’s getting a little over a million dollars in cash plus perks to grease the skids on the way out.

Word of the changeup arrived right after the market closed Wednesday.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.