Claudia Ulbrich (Cardior Therapeutics)

A Bris­tol My­ers-backed biotech wants to treat heart dis­ease with non-cod­ing RNAs. It's one step clos­er to proof-of-con­cept

Is it pos­si­ble to use strands of non-cod­ing RNA, a bur­geon­ing ther­a­peu­tic area that re­searchers now be­lieve plays an im­por­tant part in how sim­i­lar genes are ex­pressed in dif­fer­ent tis­sues, to treat heart dis­ease? For Car­dior Ther­a­peu­tics, that’s the $76 mil­lion ques­tion.

The Hanover, Ger­many-based biotech com­plet­ed its Se­ries B round Wednes­day morn­ing with the promise of ad­vanc­ing its lead ncR­NA can­di­date in­to the next round of clin­i­cal tri­als. Hav­ing re­turned pos­i­tive Phase Ib re­sults last No­vem­ber, the pro­gram is now primed for a Phase II study in post-heart at­tack pa­tients suf­fer­ing from heart fail­ure, CEO Clau­dia Ul­brich told End­points News.

All Car­dior need­ed was some cash to get it there. The biotech plans to launch the tri­al in the first half of next year, with a da­ta read­out ex­pect­ed by the end of 2024. Am­s­ter­dam VC firm Inkef Cap­i­tal stepped in to lead Wednes­day’s round.

“The biggest achieve­ment from the Se­ries A was com­plet­ing the Phase Ib tri­al last year, the pro­gram is al­ready per­form­ing in sta­ble heart fail­ure pa­tients,” Ul­brich told End­points. “So fi­nal­ly this is now the next in­flec­tion step to get a ro­bust da­ta pack­age, proof-of-con­cept da­ta and to ad­vance the lead pro­gram fur­ther.”

That pro­gram, dubbed CDR132L, is at the cen­ter of the biotech’s ef­forts in heart dis­ease — though it has a few oth­er can­di­dates fur­ther down the pipeline. By uti­liz­ing non-cod­ing RNA, or RNA strands that are not trans­lat­ed in­to pro­teins, CDR132L can af­fect sev­er­al car­dio­vas­cu­lar path­ways at once.

Specif­i­cal­ly, CDR132L func­tions as an in­hibitor on a mi­cro-RNA known as mi­cro-RNA-132, giv­ing it the abil­i­ty to act as a “mas­ter switch” at the cel­lu­lar lev­el, Ul­brich said. The mi­cro-RNA it­self is im­pli­cat­ed dur­ing car­diac stress, in­creas­ing at high rates in such events and caus­ing a pletho­ra of down­stream com­pli­ca­tions.

On top of that, mi­croR­NA-132 plays a cru­cial part in how heart cells can change af­ter an in­jury, Ul­brich said. With the po­ten­tial abil­i­ty to in­hib­it these mech­a­nisms through block­ing mi­croR­NA-132, the ex­per­i­men­tal drug aims to slow down scar­ring and en­large­ment of the heart, as well as show pos­i­tive ef­fects on clin­i­cal­ly mean­ing­ful bio­mark­ers.

Ul­ti­mate­ly, that will lead to a re­duc­tion in heart vol­ume and car­diomy­ocytes, or the cells re­spon­si­ble for gen­er­at­ing con­trac­tile force in the heart’s mus­cles.

Car­dior is not the on­ly biotech to go af­ter non-cod­ing RNAs, as more than a few com­pa­nies have turned their at­ten­tion to the field. Just this year, Swiss biotech Haya Ther­a­peu­tics pulled in a $20 mil­lion seed round back in May, and UK-based Tran­sine Ther­a­peu­tics got $12.8 mil­lion in seed cash in June. While Tran­sine is shoot­ing for CNS and oph­thal­mo­log­ic dis­eases with a long non-cod­ing RNA ap­proach, Haya is gun­ning for heart dis­ease as well.

But Ul­brich ap­pears to en­joy the com­pe­ti­tion, point­ing to Bris­tol My­ers Squibb’s ac­qui­si­tion of MyoKar­dia last Oc­to­ber as a sign that there’s enough Big Phar­ma ap­petite for many car­dio­vas­cu­lar play­ers. And she feels the mech­a­nism di­rect­ed at mi­croR­NA-132 is unique enough for Car­dior to stand out as ncR­NA con­tin­ues to spur new biotech launch­es.

The $13 bil­lion MyoKar­dia buy­out “clear­ly shows new modal­i­ties are clear­ly a need for these pa­tients,” Ul­brich said, adding, “this one cru­cial mi­croR­NA dri­ves the pathol­o­gy in the heart.”

In ad­di­tion to Inkef, new in­vestors such as Fund+, Sun­stone, Hadean Ven­tures and Co­par­i­on joined the round. Car­dior al­so saw par­tic­i­pa­tion from ex­ist­ing in­vestors in­clud­ing LSP, Bio­Med­Part­ners, Bris­tol My­ers Squibb and High-Tech Grün­der­fonds.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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