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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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