Jason Coloma, Maze Therapeutics CEO

Maze Ther­a­peu­tics re­fu­els with an­oth­er $190M to prove its ge­net­ic mod­i­fiers ap­proach holds up in the clin­ic

Ja­son Colo­ma re­mem­bers when Maze Ther­a­peu­tics was noth­ing more than a bunch of Pow­er­Point slides. Sev­er­al months af­ter re­veal­ing ex­act­ly what his team of sci­en­tists have been up to, the chief ex­ec­u­tive is now gun­ning for the clin­ic with an­oth­er $190 mil­lion in tow — and he says an IPO isn’t en­tire­ly out of the ques­tion.

“I mean, the good thing is with the fi­nan­cial flex­i­bil­i­ty that we now have, we’ve done a nice job,” Colo­ma said when asked about a pub­lic de­but. “We’ll look at dif­fer­ent op­tions, but I think that would even in­clude even stay­ing pri­vate or look­ing at dif­fer­ent busi­ness de­vel­op­ment trans­ac­tions that might be help­ful for us to kind of think of dif­fer­ent ways to ad­vance our pipeline.”

Maze launched back in 2019 with $191 mil­lion, sup­port from some top-tier in­vestors, and a mis­sion to find so-called ge­net­ic mod­i­fiers, or genes that can change the sever­i­ty of a mono­genet­ic dis­ease like sick­le cell. The com­pa­ny’s lead can­di­date, MZE001, goes af­ter Pompe dis­ease, a rare mus­cle-wast­ing con­di­tion caused by the buildup of a sug­ar called glyco­gen in cells. Ac­cu­mu­la­tion in cer­tain or­gans and tis­sues (es­pe­cial­ly mus­cles) im­pairs their abil­i­ty to func­tion nor­mal­ly.

Pa­tients with Pompe dis­ease don’t have a func­tion­ing copy of the en­zyme for dis­pos­ing of glyco­gen. And while oth­er drug­mak­ers have large­ly set out to ei­ther in­fuse ar­ti­fi­cial copies of that en­zyme or de­liv­er a gene for a healthy one, Maze is tar­get­ing a gene called GYS1, which is re­spon­si­ble for mak­ing glyco­gen. The idea is that if you can in­hib­it GYS1, you can pre­vent glyco­gen from build­ing up in the first place.

“We’ve tar­get­ed GYS1 be­cause it has that abil­i­ty to de­plete glyco­gen, in par­tic­u­lar in the skele­tal mus­cles, which is im­por­tant be­cause that’s what’s re­al­ly im­por­tant for late-on­set Pompe dis­ease pa­tients,” Colo­ma said.

That can­di­date’s ex­pect­ed to en­ter the clin­ic in the first half of this year. But one bio­phar­ma com­pa­ny, in par­tic­u­lar, has its eyes on an­oth­er of Maze’s pre­clin­i­cal can­di­dates.

Maze’s sec­ond pro­gram is for APOL1 — a gene that has long been linked to a great­ly in­creased risk of kid­ney dis­ease — which puts the com­pa­ny in di­rect com­pe­ti­tion with Ver­tex. While the lat­ter drug­mak­er cur­rent­ly has a mol­e­cule in Phase II for APOL1-linked kid­ney con­di­tions, Colo­ma says Maze has a dif­fer­en­ti­at­ed way of tar­get­ing the gene.

“For many years, peo­ple un­der­stood the ge­net­ics, but they didn’t re­al­ly un­der­stand func­tion or mech­a­nism, right? There were about 10 dif­fer­ent hy­pothe­ses on what was ac­tu­al­ly, what APOL1 was do­ing in the kid­ney,” he said.

Maze’s orig­i­nal plan was to have one IND per year — and ac­cord­ing to Colo­ma, they’re right on track.

The com­pa­ny’s third pro­gram goes af­ter a gene called ATXN2, in the hopes that knock­ing it down can help pro­tect against the buildup of dan­ger­ous plaques. The ap­proach was pi­o­neered by co-founder Aaron Gitler, a pro­fes­sor of ge­net­ics at Stan­ford Uni­ver­si­ty.

There are nine pre­ci­sion med­i­cine pro­grams to­tal in Maze’s pipeline, span­ning meta­bol­ic, car­dio-re­nal, neu­ro­log­ic and oph­thalmic con­di­tions.

This lat­est fund­ing round was led by Ma­trix Cap­i­tal Man­age­ment, with par­tic­i­pa­tion from Gen­er­al Cat­a­lyst, a16z, Wood­line Part­ners, Cas­din Cap­i­tal, City Hill Ven­tures, Fore­site Cap­i­tal, Driehaus Cap­i­tal Man­age­ment, Moore Strate­gic Ven­tures, Ter­ra Mag­num Cap­i­tal Part­ners, NS In­vest­ments and sev­er­al oth­ers.

“Go­ing from ba­si­cal­ly an idea to the clin­ic in about three years is in­cred­i­bly re­ward­ing,” Colo­ma said. “Even though it’s been a crazy time, with Covid and every­thing around us, kind of sol­dier­ing for­ward and be­ing able to build a com­pa­ny has been a tremen­dous ex­pe­ri­ence.”

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms.

“While no clear trend in the adverse reactions or new safety signal has been identified by Gilead at this time, the partial clinical hold is being implemented by Gilead across all ongoing magrolimab and azacitidine (Vidaza) combination studies worldwide in the best interests of patients as additional data is gathered and analyzed to address the concerns raised by FDA,” the big biotech said in a statement.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.

Brian Thomas, Metagenomi CEO

Gen 2: Berke­ley spin­out lands $175M megaround to keep it on the cut­ting edge of the boom­ing gene-edit­ing field

The big bucks keep pumping into the gene-editing field.

This morning Metagenomi, allied with one of the biggest names in the mRNA field with a company DNA that includes the ubiquitous Jennifer Doudna, is showing off a $175 million B round that will pay for a rapid swelling of its staff in pursuit of some of the cutting-edge tech that keeps this field in the spotlight. And they’re aligning themselves with some major industry players with an eye on the clinic while getting behind some startups to help expand the work into new fields.

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Michael Egholm, Standard BioTools president and CEO (IsoPlexis)

Eli Cas­din co-leads $250M in­fu­sion in­to mi­croflu­idics play­er that land­ed NIH fund­ing for Covid-19 test­ing

In about 17 months, Fluidigm has gone from working with sharks to Vikings.

The South San Francisco-based company, which landed NIH money in a Shark Tank-style program for Covid-19 testing, announced that it will take on an investment worth $250 million from Casdin Capital and Viking Global Investors. It will also rebrand, and call itself Standard BioTools. The investment will help the company focus on the highest growth areas of discovery and development and expand its CRO and CMO service providers. Right now, the company’s customer reach is limited to basic research, it said.

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