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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Stephen Squinto, Gennao Bio chairman

Scoop: Stephen Squin­to’s Yale spin­out is rais­ing a Se­ries B for ge­net­ic med­i­cines

The nucleic acid therapeutics at Stephen Squinto’s Yale-originated biotech are en route to a $40 million R&D payday.

After a Series A of that size in the spring of 2021, Hopewell, NJ-based Gennao Bio is closing on a round of the same amount, Endpoints News has learned. A Series B is in the works, according to an investor’s post on LinkedIn and as confirmed by a spokesperson. An SEC filing on Thursday outlines $15 million in equity sold thus far in the round.

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Nashville-based CD­MO nets a $65M Se­ries B to ex­pand fa­cil­i­ty and ca­pa­bil­i­ties

Another $65 million is music to the ears of the team at August Bioservices, a contract manufacturer in Nashville.

The company announced the Series B round last week, which will fund equipment in a new building expected to open in 2023, according to CEO Jenn Adams. It was led by Oak HC/FT, the same firm that led August’s Series A round in July 2020.

August Bioservices, a producer of materials such as prefilled syringes, IV bags and vials, was formed back in 2020 after the acquisition of PMI BioPharma Solutions, also based in Nashville. Adams said the goal was to build a business that could “address the scarcity of supply relative to sterile injectable manufacturing based in the US” and provide a broad range of manufacturing services.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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