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)

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.