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.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
Contact us about your next study.
Download our Frost & Sullivan APAC CRO Report here. 

The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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