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)

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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Spanish Prime Minister Pédro Sanchez and European Commission President Ursula von der Leyen (AP Photo/Geert Vanden Wijngaert)

EU to launch vac­cine de­vel­op­ment and man­u­fac­tur­ing part­ner­ship with Latin Amer­i­can and Caribbean coun­tries

While European companies, including BioNTech, are focused on increasing vaccine access to African countries by setting up vaccine manufacturing facilities, the European Union is looking westward to Latin America and the Caribbean.

Speaking at a press conference with Spanish Prime Minister Pédro Sanchez, EU Commission president Ursula von der Leyen said that the EU is launching a new initiative for vaccines and medicines manufacturing in Latin America, to get drugs to Latin America and the Caribbean faster.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Kelly Martin, Radius Health CEO

VC firms take os­teo­poro­sis drug­mak­er Ra­dius Health pri­vate for al­most $900M

After attacks from activist investors and disappointing returns on share prices, Radius Health has now agreed to new ownership, a direction resulting in leaving the Nasdaq.

Radius Health, a biotech out of Massachusetts with one approved product in its arsenal, announced Thursday morning that it agreed to be acquired by two VC firms: Gurnet Point Capital and Patient Square Capital. The deal, worth around $890 million, will include debt assumption and the payout of $1 CVR per share for investors. And on top of that, OrbiMed is providing debt financing.