Look­ing to cap­i­tal­ize on ge­net­ic mod­i­fiers, Maze Ther­a­peu­tics launch­es with $191M in fi­nanc­ing led by Arch, Third Rock

Some peo­ple with dis­ease-caus­ing ge­net­ic mu­ta­tions seem to es­cape ill­ness, while mem­bers of a fam­i­ly with the same ge­net­ic aber­ra­tion present with dif­fer­ent forms and/or sever­i­ty of a dis­ease — for ex­am­ple cys­tic fi­bro­sis. These in­con­sis­ten­cies sug­gest that the ef­fects of a sin­gle ge­net­ic vari­ant can be mod­i­fied by oth­er genes in our DNA, and mono­genic dis­eases give sci­en­tists the op­por­tu­ni­ty to iden­ti­fy these ge­net­ic mod­i­fiers and har­ness them for ther­a­peu­tic in­ter­ven­tion. A com­pa­ny work­ing on trans­lat­ing these in­sights in­to drugs — Maze Ther­a­peu­tics — burst on to the scene on Thurs­day, with a $191 mil­lion in­fu­sion led by sto­ried in­vestors Third Rock Ven­tures and ARCH Ven­ture Part­ners.

The San Fran­cis­co-based com­pa­ny plans to com­bine the strength of large-scale hu­man ge­net­ics with func­tion­al ge­nomics to un­der­stand the ways mod­i­fi­er genes can con­fer pro­tec­tion.

Mark Daly

“Al­most two and a half, three years ago, we had a meet­ing with the sci­en­tif­ic ad­vi­so­ry board about whether the time was ripe to in­te­grate these two ca­pa­bil­i­ties, build a plat­form-based com­pa­ny, with the un­der­stand­ing that re­al­ly the great­est risk in drug dis­cov­ery is pick­ing the right tar­get — a tar­get that will have quan­ti­ta­tive­ly im­por­tant, clin­i­cal­ly mean­ing­ful out­come,” founder and in­ter­im CEO Charles Hom­cy told End­points News.

Stephen Elledge

“The con­cept of find­ing ge­net­ic val­i­da­tion of a tar­get, where the genome ac­tu­al­ly tells you what the tar­get is — that it is an­oth­er gene that mod­i­fies the dys­func­tion­al ac­tiv­i­ty of a mendelian dis­ease (dis­eases caused by mu­ta­tions in one gene), gives one great con­fi­dence that tar­get as a drug dis­cov­ery tar­get will pro­vide clin­i­cal ben­e­fit…that re­al­ly is the rai­son d’être for this plat­form build at the com­pa­ny,” said Hom­cy, who is al­so a part­ner at Third Rock.

The com­pa­ny has about 3 to 4 drug dis­cov­ery pro­grams on the go, Hom­cy said, de­clin­ing to pro­vide de­tail on the dis­eases they are tar­get­ing. “We be­lieve there was low-hang­ing fruit out there in terms of val­i­dat­ed ge­net­ic mod­i­fiers…two of those pro­grams will hope­ful­ly be in the clin­ic in two and a half years.”

The most ad­vanced pro­gram is a small mol­e­cule tar­get­ing a clas­sic mendelian dis­ease, he added.

Sekar Kathire­san

The com­pa­ny is called Maze for two rea­sons: the idea be­hind nav­i­gat­ing the maze of hu­man ge­net­ics as well as a way to pay homage to No­bel prize win­ner Bar­bara Mc­Clin­tock and her work with mo­bile ge­net­ic el­e­ments in maize corn. It sports a star stud­ded group of sci­en­tif­ic founders, in­clud­ing not­ed ge­neti­cists Mark Daly (Broad In­sti­tute) and Stephen Elledge (Har­vard), Sekar Kathire­san (Broad In­sti­tute, Har­vard) as well as neu­ro­sci­en­tist Aaron Gitler (Stan­ford) and sci­en­tist Jonathan Weiss­man (Uni­ver­si­ty of Cal­i­for­nia).

The cap­i­tal raise al­so in­clud­ed the par­tic­i­pa­tion of GV (the ven­ture cap­i­tal in­vest­ment arm of Al­pha­bet), Fore­site Cap­i­tal, Cas­din Cap­i­tal, Alexan­dria Ven­ture In­vest­ments and oth­er undis­closed in­vestors.


Im­age: Charles Hom­cy. (Third Rock)

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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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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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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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.

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)

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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Daphne Koller, insitro CEO (insitro)

Daphne Koller’s AI start­up gets $143M in new cash from a16z, oth­ers

Biotech is becoming saturated with machine learning companies promising to reinvent and hasten drug development, but few, if any, have amassed the war chest Daphne Koller has.

Entering Tuesday, the former Stanford professor, MacArthur Fellowship recipient, Coursera founder and chief computing officer of Google’s secretive anti-aging biotech Calico had raised $100 million for her AI startup insitro. Now she’s raised $143 million more.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.