Peter Smith (file photo)

Is there room for a new way to drug RNA? ARCH, Fore­site join At­las, The Col­umn Group for $81M bet

By now, it ap­pears that drug hunters have ex­ploit­ed just about every ther­a­peu­tic an­gle hav­ing to do with RNA. Lever­ag­ing RNA as a ther­a­py? Check. Mak­ing an­ti­sense oligonu­cleotides that in­duce a par­tic­u­lar change? Check. Find­ing small mol­e­cules that bind to RNA, tar­get­ing the en­zymes that mod­i­fy it, or even cre­at­ing a way to ed­it it let­ter by let­ter? Check, check, check. But At­las and The Col­umn Group — both ac­tive in­vestors of those ef­forts — saw yet an­oth­er ap­proach to be tried.

About a year ago they seed­ed Remix Ther­a­peu­tics with $16 mil­lion to look at RNA pro­cess­ing, where the nu­cle­ic acid and its ex­pres­sion gets fine tuned. Tak­ing place post-DNA tran­scrip­tion but be­fore pro­teins are made, said pres­i­dent and CSO Pe­ter Smith, it’s “re­al­ly core to de­ter­min­ing the fate of the mes­sage.”

Now Fore­site, ARCH and Alexan­dria Ven­ture In­vest­ments are com­ing on board with a Se­ries A that’s brought their to­tal haul to $81 mil­lion.

Heather Wasser­man

A com­bi­na­tion of da­ta an­a­lyt­ics, high-through­put screen­ing and new chem­istry means Remix has “a num­ber of dif­fer­ent av­enues” to mea­sure how RNA is processed, with the as­say sys­tems and bio­physics ex­per­tise to tease out how the com­pounds in their li­braries in­ter­act with tar­gets.

“So we’re in­ter­est­ed in splic­ing, we’re in­ter­est­ed polyadeny­la­tion, we’re in­ter­est­ed in [5’ end cap­ping],” Smith said. “We’ve got some great in­sights in­to the way we can use the tech to elim­i­nate RNA, to in­crease RNA ex­pres­sion, to cor­rect RNA dys­reg­u­la­tion.”

Smith co-found­ed the start­up as en­tre­pre­neur-in-res­i­dence in At­las, a role he took up af­ter some years at Mil­le­ni­um and H3, the Ei­sai sub­sidiary fo­cused on pre­ci­sion on­col­o­gy. He is keep­ing those ex­act in­sights to him­self right now, but said they promise to hit both known un­drug­gable tar­gets and new ones in can­cer, cen­tral ner­vous sys­tem dis­or­ders, au­toim­mune dis­eases and rare ge­net­ic con­di­tions.

It’s still ear­ly days, with just in vit­ro proof of con­cept da­ta for the most ad­vanced pro­grams. But the com­pa­ny has al­ready re­cruit­ed Heather Wasser­man — who un­til Au­gust was still a VP of cor­po­rate busi­ness de­vel­op­ment at Eli Lil­ly — to scout part­ner­ships as CBO and COO.

Big Phar­ma has warmed up to the idea of drug­ging RNA over the past few years, tee­ing up plat­form deals with Ar­rakis and Sky­hawk. Oth­ers like Ac­cent and Ex­pan­sion are qui­et­ly work­ing on ear­ly-stage pipelines with sig­nif­i­cant ven­ture back­ing.

“RNA has long been con­sid­ered a prime ther­a­peu­tic tar­get, but its in­nate com­plex­i­ties have made it no­to­ri­ous­ly dif­fi­cult to drug,” At­las part­ner Kevin Bit­ter­man, who’s chair­ing the board, said in a state­ment. “By fo­cus­ing on RNA pro­cess­ing, the Remix team has iden­ti­fied a com­pelling way to pur­sue pre­vi­ous­ly un­drug­gable dis­ease tar­gets.”

For Smith, the past 12 months hasn’t just been about gen­er­at­ing al­ready ex­cit­ing da­ta and chem­istry. It’s al­so test­ed the re­silience nim­ble­ness of the team he’s as­sem­bled — 18 and grow­ing — at the Alexan­dria Launch­Labs in Kendall Square, which has had to adapt to the new pan­dem­ic nor­mal and move the out­sourced work around the globe.

Aside from Bit­ter­man, Fore­site man­ag­ing di­rec­tor Michael Rome, The Col­umn Group’s Leon Chen, Kristi­na Bur­row of ARCH and Scott Biller, ex-CSO of Agios, will be plot­ting the next steps with him on the board.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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Spanish Prime Minister Pédro Sanchez and European Commission President Ursula von der Leyen (AP Photo/Geert Vanden Wijngaert)

EU to launch vac­cine de­vel­op­ment and man­u­fac­tur­ing part­ner­ship with Latin Amer­i­can and Caribbean coun­tries

While European companies, including BioNTech, are focused on increasing vaccine access to African countries by setting up vaccine manufacturing facilities, the European Union is looking westward to Latin America and the Caribbean.

Speaking at a press conference with Spanish Prime Minister Pédro Sanchez, EU Commission president Ursula von der Leyen said that the EU is launching a new initiative for vaccines and medicines manufacturing in Latin America, to get drugs to Latin America and the Caribbean faster.

GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.