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.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

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Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.