It's not de­vel­op­ing drugs that bind to RNA, or mor­ph­ing RNA in­to a ther­a­peu­tic — this Cam­bridge start­up has scored $63M to work on RNA-mod­i­fy­ing pro­teins

First came the con­cept of epi­ge­net­ics, the study of chem­i­cal mod­i­fi­ca­tions made to the blue­print of life — DNA — to switch genes on or off. Then, sci­en­tists re­al­ized DNA’s cousin RNA could al­so be sub­ject to such chem­i­cal ma­nip­u­la­tion, and the field of “epi­tran­scrip­tomics” was born in the last decade.

As re­searchers found that chem­i­cal mod­i­fi­ca­tions across a cell’s RNA were seem­ing­ly dis­tort­ed in some can­cers, a trio of biotech­nol­o­gy com­pa­nies spawned: Storm Ther­a­peu­tics, Ac­cent Ther­a­peu­tics and Gotham Ther­a­peu­tics. On Thurs­day, 2017-found­ed Ac­cent raised $63 mil­lion in a Se­ries B round, led by EcoR1 Cap­i­tal.

Chuan He, a pro­fes­sor of chem­istry at the Uni­ver­si­ty of Chica­go, first pro­posed the field of RNA epi­ge­net­ics, sug­gest­ing methyl mod­i­fi­ca­tions on RNA can be re­moved, in 2012. Five years lat­er, along with Stan­ford’s Howard Chang, and for­mer Epizyme and GSK ex­ec­u­tive Robert Copeland, Ac­cent was born.

Shak­ti Narayan

“What we’re do­ing is dif­fer­ent from a lot of oth­er com­pa­nies and oth­er re­searchers in the RNA field. There are many com­pa­nies that are us­ing RNA as a ther­a­peu­tic it­self, there are some com­pa­nies that are drug­ging the RNA di­rect­ly,” not­ed Ac­cent chief Shak­ti Narayan in an in­ter­view.

Akin to ac­cents in mu­sic that are used to mod­u­late tunes, the com­pa­ny was chris­tened Ac­cent Ther­a­peu­tics to re­flect mod­i­fi­ca­tions to chem­i­cal en­ti­ties that af­fect RNA mol­e­cules, which can ul­ti­mate­ly trans­form the func­tion of the mol­e­cule.

“What we’ve fo­cused on is tar­get­ing the en­zymes that work on RNA, and there­by, we’re ac­tu­al­ly able to take ad­van­tage of sev­er­al decades of med­i­c­i­nal chem­istry ex­pe­ri­ence and ex­per­tise that has de­vel­oped to specif­i­cal­ly tar­get en­zymes. So, we’re not hav­ing to take on a whole bunch of modal­i­ty risk in our ap­proach,” Narayan said.

The fresh in­jec­tion of fund­ing will be used to take Ac­cent’s lead ex­per­i­men­tal pro­grams — which are fo­cused on two pro­teins that are un­der­stood to mod­i­fy RNA, MET­TL3 and ADAR1 in AML and sol­id tu­mors, re­spec­tive­ly — in­to the clin­ic.

But, as with all best-laid plans, Covid-19 has put a span­ner in the works.

“We’ve been able to in­crease our work via CROs in cer­tain lo­ca­tions, ver­sus de­creas­ing it and oth­er lo­ca­tions de­pend­ing on the im­pact of the virus in par­tic­u­lar lo­ca­tions, which has been ex­treme­ly help­ful for us to be able to keep our pro­grams ad­vanc­ing to­wards the clin­ic,” Narayan said. “It’s cer­tain­ly not op­ti­mal, but it’s been a great way to mit­i­gate the im­pact of the dis­ease on our over­all op­er­a­tional mod­el.”

The new round of fi­nanc­ing in­clud­ed the par­tic­i­pa­tion by GV, Ab­b­Vie Ven­tures, The Mark Foun­da­tion for Can­cer Re­search, NS In­vest­ment and Droia Ven­tures as well as ex­ist­ing in­vestors, At­las Ven­ture and The Col­umn Group. The Lex­ing­ton, Mass­a­chu­setts-based com­pa­ny, which cur­rent­ly has 25 em­ploy­ees, raised $40 mil­lion at launch in 2018.

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Sta­da to place $50M+ in­vest­ment in a new fa­cil­i­ty in Ro­ma­nia

While Romania may conjure up images of vast mountain ranges and tales of medieval kings, one generic manufacturer has broken ground on a new facility there.

German pharma company Stada said Monday that it has placed a €50 million ($51.9 million) investment into a 100,000 square-meter (1.08 million square-foot) site in Turda, Romania, a city in the Southeast of the country. According to a Stada spokesperson in an email to Endpoints News, the company has developed only 281,500 square feet of the site so far.

Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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