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.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”