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)

In­vestors pony up $476M for the lat­est round of biotech IPOs to hit the Street

Three biotechs — and a genome se­quenc­ing play­er — have caught the lat­est tide to the Gold Coast of IPOs, round­ing out the first half of 2019 with 23 new drug de­vel­op­ers mak­ing it on Nas­daq.

Most of these com­pa­nies filed their IPOs al­most si­mul­ta­ne­ous­ly, though we’re still wait­ing on word of fel­low class­mate Bridge­Bio’s pric­ing af­ter CEO Neil Ku­mar set the terms at $14 to $16 a share on Mon­day in search of a $240 mil­lion (or so) wind­fall. If he’s suc­cess­ful, that would take the one-week haul past the $700 mil­lion mark, a fresh sign that in­vestors’ en­thu­si­asm for new­ly coined pub­lic biotechs hasn’t cooled.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

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In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.