For­get RNA drugs. Ac­cent gets $40M to de­vel­op small mol­e­cules that mod­i­fy RNA in­stead

A Cam­bridge start­up is step­ping out to­day with some rev­e­la­tions on how pro­teins tweak RNA — and sub­se­quent­ly how a cell’s char­ac­ter and func­tion is de­ter­mined. The com­pa­ny, called Ac­cent Ther­a­peu­tics, scored $40 mil­lion in launch mon­ey to see if their new tech can de­liv­er pre­ci­sion meds in can­cer.

Howard Chang

The im­pe­tus for the com­pa­ny’s launch is a new un­der­stand­ing of RNA-mod­i­fy­ing pro­teins, and how they might link to dis­ease. Ac­cent’s new find­ings in the field just got pub­lished to­day in Na­ture Re­views Drug Dis­cov­ery.

The com­pa­ny’s pres­i­dent and CSO Bob Copeland tells me the lit­er­a­ture in the field is still rather sparse, as re­searchers have on­ly been study­ing these in­ter­ac­tions over the past five years or so.

Al­though it’s easy to con­fuse what Ac­cent is do­ing with RNA ther­a­peu­tics, Copeland says there’s ba­si­cal­ly noth­ing in com­mon.

“We’re not tar­get­ing the RNA, we’re not try­ing to make drugs that bind to RNA, and we’re not try­ing to use RNA as a ther­a­peu­tic it­self,” Copeland said. “In­stead, we’re us­ing small mol­e­cule drugs that in­hib­it the en­zymes that per­form mod­i­fi­ca­tions on RNA.”

Chuan He

If DNA is the code for life, and RNA is the blue­print for cells, then RNA-mod­i­fy­ing pro­teins are the lit­tle guys that tell cells what ex­act­ly to be and do in the body. Re­cent stud­ies have linked cer­tain can­cers to the ac­tiv­i­ty of some RNA-mod­i­fy­ing pro­teins, pro­vid­ing a rich new tar­get space for drug de­vel­op­ment.

Ac­cent hap­pens to have a cou­ple of ex­perts in the space as sci­en­tif­ic co-founders: Howard Chang of Stan­ford Uni­ver­si­ty and Chuan He of the Uni­ver­si­ty of Chica­go. The duo brings to­geth­er a broad and deep ex­per­tise in the emerg­ing bi­ol­o­gy of this field — called epi­tran­scrip­tomics — and its role in hu­man dis­eases.

This Se­ries A, backed by The Col­umn Group, At­las Ven­ture and EcoR1 Cap­i­tal, should get the com­pa­ny “quite far in pre­clin­i­cal dis­cov­ery.” For now, the com­pa­ny has se­lect­ed four tar­gets in both hema­to­log­i­cal can­cers and sol­id tu­mors, al­though Copeland is mum on the specifics.

Im­age: Bob Copeland. AC­CENT

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Af­ter sell­ing to Genen­tech, the old Je­cure team is back at an RNA-fo­cused start­up — and more en­thu­si­as­tic than ever

When Genentech swooped in to buy NASH-focused Jecure Therapeutics back in 2018, a handful of the startup’s executives weren’t quite ready to disperse.

It had been just three years since Jecure launched with a preclinical portfolio of NLRP3 inhibitors — and the takeover came sooner than anyone, including CEO Jeff Stafford, had expected. So he got talking with James Veal and Gretchen Bain, two serial entrepreneurs in charge of Jecure’s R&D.

Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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