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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Suresh Katta, Saama CEO (via YouTube)

As AI con­tin­ues to en­tice Big Phar­ma, a Car­lyle-led drug­mak­er syn­di­cate shells out $430M for cloud com­put­ing play­er

The AI revolution permeating Big Pharma took a big financial step forward Wednesday, with VCs and major drugmakers coming together to acquire a cloud-focused company.

Led by the Carlyle Group, the investors will put up $430 million for a majority stake in Saama, a company that collects patient data to help speed along the drug development process. The investment arms of Pfizer, Merck, Amgen and McKesson all participated in the financing, in addition to other prominent life sciences VCs like Northpond.

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Raju Mohan, Ventyx Biosciences CEO

Ven­tyx sprints to Wall Street less than a year af­ter emerg­ing from stealth

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

It took seven months from exiting “quiet mode” for Ventyx Biosciences to land its very own stock ticker, raising $165 million in venture funds along the way.

Now, after pricing a massive $151.5 million IPO, the Encinitas, CA-based biotech is gunning for Phase II.

Ventyx priced close to 9.5 million shares at $16 apiece on Wednesday, the midpoint of its $15 to $17 range. CEO Raju Mohan filed the S-1 papers at the end of September, just over a week after unveiling a $114 million Series B round. He penciled in the standard figure of $100 million at first, likely knowing that in the last year, it’s been common for biotechs to raise much more than those initial estimates.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Ep­i­darex, Sofinno­va dou­ble down on a par­al­lel take on 3rd-gen CAR-T — aim­ing straight at ovar­i­an can­cer

When John Maher treated the first head and neck cancer patient at Guy’s Hospital in London with his pan-ErbB CAR-T back in 2015, he was among a small club of researchers convinced they had an answer to the challenges that had kept those engineered T cells — wildly successful in hematological cancers — either too dangerous or out of reach for patients with solid tumors.

The field has blossomed since then, with a proliferation of technologies that promise to address any number of challenges identified as unique to solid tumors. And Maher himself has rethought his approach and come up with a new CAR-T platform to generate the next slate of candidates.