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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Anthony Coyle (Pfizer via Youtube)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-Suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.