Peter Ghoroghchian, Ceptur CEO

A new start­up wants to take an­ti­sense and siR­NA one step fur­ther, and build more po­tent drugs than Io­n­is and Al­ny­lam

Drug­ging RNA has be­come a hot bio­phar­ma top­ic in re­cent years, par­tic­u­lar­ly af­ter the mas­sive suc­cess­es of the Covid-19 vac­cines. But one biotech is aim­ing to take things a step fur­ther, uti­liz­ing the sin­gle-strand­ed mol­e­cules be­fore they even be­come RNA.

Cep­tur Ther­a­peu­tics launched Wednes­day morn­ing with a $75 mil­lion Se­ries A, the com­pa­ny an­nounced. The goal is to push for­ward a slate of oligonu­cleotide-based ther­a­pies that can con­trol gene ex­pres­sion, and even si­lence some tar­get­ed genes, at the “pre-mR­NA lev­el,” co-founder and CEO Pe­ter Ghoroghchi­an told End­points News.

The biotech has re­cruit­ed a blue-chip syn­di­cate as well, with ven­Bio and Qim­ing Ven­tures co-lead­ing the round. In ad­di­tion, Cep­tur re­ceived new sup­port from Per­cep­tive Xon­toge­ny Ven­ture Fund, Bris­tol My­ers Squibb and Janus Hen­der­son In­vestors.

Cep­tur’s main idea cen­ters around a pro­tein com­plex known as the U1 snRNP (pro­nounced SNURP), which Ghoroghchi­an said serves as a bi­o­log­i­cal gate­keep­er when DNA is tran­scribed in­to RNA and RNA in­to pro­teins. When cells go through this process, they’re con­stant­ly splic­ing ge­net­ic se­quences in and out, with some left out of the fi­nal mR­NA.

“The splic­ing ma­chin­ery that helps mR­NA to ma­ture is re­cruit­ed by the U1 snRNP, and the U1 snRNP al­so helps to turn off and con­trol the lev­el of tran­scripts, so how much of a giv­en mR­NA is made,” Ghoroghchi­an said. “It does this for every sin­gle gene in the body, so it has this very cen­tral role in bi­ol­o­gy.”

To take ad­van­tage of these gate­ways and cre­ate drugs, re­searchers led by Rut­gers pro­fes­sor Sam Gun­der­son, an­oth­er Cep­tur co-founder, de­vel­oped bi­va­lent oligonu­cleotides called U1 Adap­tors. The adap­tors tar­get spe­cif­ic ge­net­ic se­quences and re­cruit the U1 snRNP to de­stroy the de­sired pre-mR­NA, es­sen­tial­ly con­trol­ling which genes make it in­to the fi­nal pro­teins.

It’s an ap­proach sim­i­lar to an­ti­sense oligonu­cleotides and siR­NA ther­a­pies, Ghoroghchi­an said, on­ly Cep­tur aims to drug the RNA at a much more gran­u­lar lev­el. And be­cause the U1 snRNP is so ubiq­ui­tous through­out the body — Ghoroghchi­an says there are more than one mil­lion copies in every cell — Cep­tur be­lieves dif­fer­ent cell types and genes won’t im­pede the com­pa­ny’s progress.

Col­in Walsh

Col­in Walsh, a part­ner at Qim­ing Ven­tures, not­ed the RNA drug­ging field has boomed in re­cent years, ever since the idea took off in the ear­ly days of Io­n­is and Al­ny­lam. But he be­lieves Cep­tur rep­re­sents a big step for­ward in how such ap­proach­es will shape the space in the fu­ture, par­tic­u­lar­ly once it moves past the gene si­lenc­ing ap­pli­ca­tion.

“There is a large ap­petite for ways to con­trol pro­tein func­tion at the RNA lev­el,” Walsh said. “The U1 Adap­tor pro­tein com­plex is re­al­ly a mas­ter reg­u­la­tor of the tran­scrip­tome, it’s splic­ing and oth­er things. Though knock­down is the first place where you can take this, we see this as a re­al­ly broad po­ten­tial plat­form for mod­u­lat­ing RNA us­ing oli­go-based ther­a­peu­tics.”

For its in-house pipeline, Cep­tur isn’t re­veal­ing much just yet. Ghoroghchi­an said the team has a few can­di­dates that could hit IND-en­abling stud­ies some­time in 2023, in­clud­ing on­col­o­gy pro­grams tar­get­ing KRAS and MYC in can­cer, as well as one for Hunt­ing­ton’s dis­ease. Past that, though, every­thing re­mains in the dis­cov­ery phase.

And though the biotech is try­ing to con­trol ge­net­ic se­quenc­ing, Ghoroghchi­an be­lieves Cep­tur’s drugs will prove far safer than gene ther­a­pies that some­times con­tin­ue edit­ing DNA be­yond the ther­a­peu­tic use. Be­cause Cep­tur isn’t en­gag­ing in DNA or base edit­ing, its drugs will on­ly work as long as the adap­tor is in the pa­tient’s sys­tem.

Cep­tur’s oligonu­cleotides are ex­treme­ly small, Ghoroghchi­an said, and “every nu­cleotide is chem­i­cal­ly mod­i­fied. And we can use a lot of ex­ist­ing and val­i­dat­ed chemistries to di­al in how long it lasts, ei­ther to how long they can knock the gene down, or how quick­ly it can be de­grad­ed, in or­der to di­al in the safe­ty as­pect.”

On top of the new in­vestors, Cep­tur al­so re­ceived Se­ries A sup­port from ex­ist­ing seed in­vestors Affin­i­ty As­set Ad­vi­sors, Box­er Cap­i­tal and LifeSci Ven­ture Part­ners.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Lanny Sun, co-founder, CEO and chairman of Full-Life Technologies

Se­quoia Chi­na leads $37M in­fu­sion in­to ra­dio­phar­ma­ceu­ti­cals play­er set­ting up shop in Chi­na and Bel­gium

It’s not just American startups that are tuning into the rising interest in radiopharmaceuticals.

Sequoia China is leading a $37 million Series A into Full-Life Technologies, a biotech headquartered in Shanghai with offices in Brussels, Belgium, to develop a pipeline of radioactive cancer therapies.

The idea isn’t new: As clinicians started routinely deploying radiation to kill cancer cells, scientists and drugmakers have long been exploring ways to limit that powerful effect only to cancer cells while sparing healthy cells. But recent progress in the production of radioisotopes — coupled with big investments from Big Pharma, most notably Novartis and Bayer — has inspired a new wave of startups.

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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