A new CAR-T is cred­it­ed with a 'sur­pris­ing' im­pact in tiny study of ad­vanced liv­er can­cer — rais­ing hopes on sol­id tu­mors

A tiny study has pro­vid­ed the first glim­mer of hope that Eu­re­ka Ther­a­peu­tics’ CAR-T ther­a­py may work in sol­id tu­mors.

In a proof-of-con­cept study in­volv­ing six pa­tients with he­pa­to­cel­lu­lar car­ci­no­ma — the most preva­lent form of liv­er can­cer — re­searchers re­port­ed that one pa­tient had a com­plete re­sponse, which was main­tained at the sev­en-month check­up. Two oth­ers saw their tu­mors shrink.

These, the biotech em­pha­sizes, are pa­tients who have failed mul­ti­ple pri­or treat­ments.

“The re­sults were ex­treme­ly sur­pris­ing,” Chang Liu, a physi­cian at Xi’an Jiao­tong Uni­ver­si­ty in Chi­na and the pri­ma­ry in­ves­ti­ga­tor of the study, told Bloomberg. “This is a break­through in an area where there was pre­vi­ous­ly no known treat­ment. To be more dra­mat­ic, we can say it’s cre­at­ed a mir­a­cle.”

Cheng Liu

Emeryville, CA-based Eu­re­ka added that none of the pa­tients ex­pe­ri­enced cy­tokine re­lease syn­drome or drug-re­lat­ed neu­ro­tox­i­c­i­ty — well-known safe­ty con­cerns that have lim­it­ed the use of T cell ther­a­pies.

Of note, three pa­tients in the study died due to what the com­pa­ny de­scribes as “non-drug-re­lat­ed com­pli­ca­tions of liv­er dis­ease.”

The first CAR-Ts on the mar­ket from Gilead/Kite and No­var­tis have demon­strat­ed some amaz­ing re­sults for blood can­cers, but sol­id tu­mors rep­re­sent a high hur­dle for these new drugs. Clear­ing it — safe­ly — would open up a ma­jor mar­ket.

“We are en­cour­aged by the safe­ty pro­file and the po­ten­tial ef­fi­ca­cy of ET140202 for AFP-pos­i­tive liv­er can­cer,” said Cheng Liu, Eu­re­ka’s pres­i­dent and CEO. “Com­bin­ing T-cell ther­a­py with a TCR-mim­ic an­ti­body to tar­get in­tra­cel­lu­lar anti­gens is a nov­el ap­proach and can po­ten­tial­ly rep­re­sent a pow­er­ful way to treat sol­id tu­mors, and in par­tic­u­lar, liv­er can­cer, an area of sig­nif­i­cant un­met med­ical need.”

The treat­ment, dubbed ET140202, works by tar­get­ing a liv­er can­cer mark­er called al­pha-fe­to­pro­tein, or AFP.

In 2019, the com­pa­ny plans to launch a mul­ti­cen­ter Phase I tri­al for ET140202, in the US, where it’s al­so test­ing its lead pro­gram fo­cused on CD19+ non-Hodgkin lym­phoma.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Clay Siegall (Photo by Dimitrios Kambouris/Getty Images for Gabrielle's Angel Foundation)

UP­DAT­ED: Clay Sie­gall re­signs from Seagen amid in­ves­ti­ga­tion in­to do­mes­tic vi­o­lence claims

A week after Seagen revealed that longtime CEO Clay Siegall was on leave due to an allegation of domestic violence, he has resigned.

Since that shocking revelation, more details about the claims have emerged into the public eye. As Endpoints News reported, Siegall was arrested on April 23. A police report about that night and a subsequent temporary restraining order described a pattern of abusive behavior against his wife and a physical altercation that left her with multiple bruises. Siegall denied the claims.

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Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.