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)

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Stephen Squinto, Gennao Bio chairman

Scoop: Stephen Squin­to’s Yale spin­out is rais­ing a Se­ries B for ge­net­ic med­i­cines

The nucleic acid therapeutics at Stephen Squinto’s Yale-originated biotech are en route to a $40 million R&D payday.

After a Series A of that size in the spring of 2021, Hopewell, NJ-based Gennao Bio is closing on a round of the same amount, Endpoints News has learned. A Series B is in the works, according to an investor’s post on LinkedIn and as confirmed by a spokesperson. An SEC filing on Thursday outlines $15 million in equity sold thus far in the round.

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Nashville-based CD­MO nets a $65M Se­ries B to ex­pand fa­cil­i­ty and ca­pa­bil­i­ties

Another $65 million is music to the ears of the team at August Bioservices, a contract manufacturer in Nashville.

The company announced the Series B round last week, which will fund equipment in a new building expected to open in 2023, according to CEO Jenn Adams. It was led by Oak HC/FT, the same firm that led August’s Series A round in July 2020.

August Bioservices, a producer of materials such as prefilled syringes, IV bags and vials, was formed back in 2020 after the acquisition of PMI BioPharma Solutions, also based in Nashville. Adams said the goal was to build a business that could “address the scarcity of supply relative to sterile injectable manufacturing based in the US” and provide a broad range of manufacturing services.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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