#AS­CO21: Watch out Bris­tol My­ers, J&J is back at AS­CO with some com­pet­i­tive BC­MA CAR-T da­ta

Bris­tol My­ers Squibb and blue­bird beat John­son & John­son to mar­ket back in March with their BC­MA CAR-T drug, Abec­ma, for heav­i­ly pre-treat­ed mul­ti­ple myelo­ma pa­tients. But watch out: J&J and their part­ners at Leg­end Biotech are now head­ing to AS­CO with up­dat­ed da­ta that could give Abec­ma a run for its mon­ey.

A longer-term fol­low-up from the Phase Ib por­tion of a Phase Ib/II tri­al shows that all pa­tients re­spond­ed to J&J’s JNJ-4528, 86% of whom achieved a strin­gent com­plete re­sponse at a me­di­an of 11.5 months. In Phase II, there was a 95% over­all re­sponse rate as of the Feb­ru­ary da­ta cut­off, with a 75% strin­gent com­plete re­sponse, the com­pa­ny said Fri­day.  The da­ta are part of the pack­age J&J is sub­mit­ting in rolling ap­pli­ca­tions for ap­provals in Eu­rope and the US.

Je­sus Berde­ja, prin­ci­pal in­ves­ti­ga­tor of the study, said in a state­ment that it shows a “con­tin­ued treat­ment ef­fect for heav­i­ly pre­treat­ed pa­tients who faced a dis­mal prog­no­sis.” Mul­ti­ple myelo­ma is a blood can­cer that starts in the bone mar­row and is char­ac­ter­ized by an ex­ces­sive pro­lif­er­a­tion of plas­ma cells. While some pa­tients achieve re­mis­sion, most re­lapse, and 29% die with­in one year of di­ag­no­sis.

Pa­tients in the Phase Ib por­tion of the study had re­ceived a me­di­an of five pri­or treat­ments (but ranged from three to 18 pri­or ther­a­pies). Most of them (86%) were triple-re­frac­to­ry, and 28% were pen­ta-re­frac­to­ry. In Phase II, pa­tients had a me­di­an of three lines of treat­ment.

“We’re en­cour­aged by not on­ly the rel­a­tive­ly high rate of strin­gent com­plete re­spons­es, but al­so the pro­gres­sion-free sur­vival seen in these pa­tients,” Berde­ja said.

A to­tal of 85% of pa­tients in the Phase II achieved what’s known as a “very good par­tial re­sponse” or bet­ter.

Back in De­cem­ber, up­on scor­ing break­through ther­a­py des­ig­na­tion, J&J said 69% of pa­tients had achieved a com­plete re­sponse. The new da­ta mea­sure strin­gent com­plete re­sponse, which is de­fined by the ab­sence of two key mark­ers of myelo­ma in the blood and bone mar­row.

As with pre­vi­ous CAR-Ts, there is some risk. All pa­tients in the Phase Ib ex­pe­ri­enced neu­trope­nia, an ab­nor­mal­ly low count of a type of white blood cell. And 93% ex­pe­ri­enced cy­tokine re­lease syn­drome (CRS). In the Phase II, CRS oc­curred in 85% of pa­tients, with 10% ex­pe­ri­enc­ing a Grade 3 or 4 case. The me­di­an time of on­set of CRS was 7 days, with a ma­jor­i­ty of pa­tients on­ly ex­pe­ri­enc­ing a Grade 1 or 2 event. At ASH in De­cem­ber, the com­pa­nies said there were 14 deaths in the study, six of which were due to treat­ment-re­lat­ed events in­clud­ing CRS, sep­sis, lung ab­scess, res­pi­ra­to­ry fail­ure and neu­ro­tox­i­c­i­ty.

Ac­cord­ing to Mark Wild­gust, Janssen’s VP of on­col­o­gy glob­al med­ical af­fairs, 99% of CRS was re­solved with­in 14 days. “We see that pre­dictable on­set at about Day 7,” he told End­points News. “I don’t be­lieve cy­tokine re­lease is go­ing to be an in­hi­bi­tion to use cil­ta-cel.”

J&J will con­tin­ue to fol­low these pa­tients for the long term, Wild­gust said. But so far, these re­sults best the da­ta that led BMS and blue­bird to an OK for Abec­ma, the first ap­proved cell-based gene ther­a­py for mul­ti­ple myelo­ma.

Abec­ma put up a 72% over­all re­sponse rate, with a 28% com­plete re­sponse rate. An es­ti­mat­ed 65% of pa­tients who achieved com­plete re­sponse re­mained there for at least 12 months, ac­cord­ing to the FDA.

This sto­ry has been up­dat­ed with Phase II re­sults.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

On the hunt for the next Mod­er­na, in­vestors have pumped 'plat­form plays' with cash. Can any­thing slow the run­away train?

It didn’t take an expert to see that mRNA platforms could be huge.

Julie Sunderland partnered with both Moderna and BioNTech about a decade ago while she was running program-related investments for the Bill & Melinda Gates Foundation — and even then the potential for their platforms was obvious despite some well-founded concerns about whether the next-gen tech would ever cross the finish line.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

Mer­ck­'s Keytru­da blazes a path in first-line cer­vi­cal can­cer, mak­ing good on drug­mak­er's push for ear­li­er pa­tients

In the years since I/O wonder drug Keytruda’s initial approval, Merck has struck an aggressive clinical trial program, which is now firmly focused on earlier lines of therapy. The drugmaker has scored some success there so far, and now it’s earned one of its biggest wins yet.

Keytruda plus chemotherapy with or without background Avastin significantly extended patients’ lives over those dosed with a placebo control in first-line patients with persistent, recurrent or metastatic cervical cancer, according to top-line data from the Phase III KEYNOTE-826 study revealed Tuesday.

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Neu­rona Ther­a­peu­tics is dash­ing to the clin­ic with its cell ther­a­py for epilep­sy — but first, an­oth­er ven­ture round

Six years ago, a band of neuroscientists from the University of California, San Francisco combined decades of research and jumped into the hunt for an off-the-shelf cell therapy. Now, that team is sprinting toward the clinic with a treatment for epilepsy — but first, it’s making a pit stop at the venture well.

Neurona Therapeutics unveiled a $41.5 million round on Tuesday morning, bringing the San Francisco-based biotech’s total raise to $135 million. The cash will be used to advance the company’s pipeline, including an upcoming Phase I/IIa for its lead candidate, NRTX-1001, in chronic focal epilepsy.

Fred Upton and Diana DeGette

New DARPA-like NIH agency preps for re­al­i­ty as E&C un­veils bi­par­ti­san Cures 2.0 draft bill

House Energy & Commerce leaders Fred Upton (R-MI) and Diana DeGette (D-CO) on Tuesday released new draft legislation with wide-ranging implications for public health, the FDA, NIH, and that would create a new, $6.5 billion federal advanced research agency under NIH, with an aim to cure cancer, Alzheimer’s and other difficult diseases.

Similar to DARPA, the new NIH division to be known as ARPA-H, would be run by a small group of program managers with more latitude to pursue high-risk, high-reward projects that other government agencies would likely shy away from.

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Sen. Ron Wyden (D-OR) with reporters in the Senate Subway (Graeme Sloan/Sipa via AP Images)

Top Wyden pri­or­i­ty for drug price re­forms: Medicare ne­go­ti­a­tions

As the Biden administration tries to wrangle the details of its infrastructure bill, Senate Finance Committee Chair Ron Wyden (D-OR) took a concrete step forward on drug pricing reforms on Tuesday and unveiled five principles for such reforms, including providing Medicare with the ability to negotiate prices.

“Allowing the Secretary of HHS to negotiate the price Medicare will pay creates a much needed mechanism to achieve fairer prices when the market has failed to do so,” Wyden wrote.

End­points News is now 5 years old. Here's how you can sup­port us for the next phase of growth

Endpoints News turned five years old over the weekend. I wanted to mark the happy occasion by extending our deepest gratitude to Endpoints’ premium subscribers while outlining several other ways to support us as we go broader and get bigger this year and beyond.

Same as any business, we’ve got to create value and get paid for delivering it. So if you depend on Endpoints to stay abreast on biopharma developments, we depend on you too.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

Ac­tivist in­vestor El­liott in talks with oth­er Glax­o­SmithK­line in­vestors about re­plac­ing Em­ma Walm­s­ley, spin­ning off vac­cine busi­ness — re­port

As Emma Walmsley reveals details this Wednesday about the upcoming split of GlaxoSmithKline’s pharma and consumer units, some tough questions may be coming her way.

Elliott Management, the activist investor that’s previously threatened an attack on GSK (but eventually backed off), is floating more radical changes like replacing the CEO, further breaking up the company and spinning out the vaccine unit, or reviewing the focus on cancer drugs, the Financial Times reported.

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Roche's Alzheimer's drug spurred bio­mark­er changes but no cog­ni­tive im­pact — pa­per; vTv out-li­cens­es for­mer lead pro­gram

More than a year ago, Roche and Eli Lilly were forced to contend with a Phase II/III failure of their respective Alzheimer’s drugs. But while Eli Lilly essentially threw in the towels, Roche wasn’t ready to give up yet.

The Swiss drugmaker now has some biomarker data to spotlight as investigators continue monitoring patients in an open-label extension study.

Dubbed DIAN-TU, the study had been designed to see whether Roche’s gantenerumab and Lilly’s solanezumab could spur a cognitive benefit for a group of patients who had a rare, inherited form of Alzheimer’s that’d tied to early-onset. In short, they didn’t: Both failed the primary endpoint.