Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Blue­bird bio an­nounced it com­plet­ed the rolling sub­mis­sion of its BLA to the FDA for betibeglo­gene au­totem­cel gene ther­a­py.

The ther­a­py, de­signed for pa­tients with be­ta-tha­lassemia who re­quire reg­u­lar red blood cell trans­fu­sions, was pre­vi­ous­ly grant­ed break­through ther­a­py des­ig­na­tion for treat­ing trans­fu­sion-de­pen­dent be­ta-tha­lassemia (TDT). If ap­proved, beti-cel will be the first hematopoi­et­ic stem cell ex-vi­vo gene ther­a­py for pa­tients in the US.

“We look for­ward to work­ing with the FDA on its re­view of this BLA as we re­al­ize the promise that one-time gene ther­a­pies hold for pa­tients,” said new blue­bird pres­i­dent An­drew Oben­shain in a state­ment.

Blue­bird was in the news re­cent­ly as the pre­vi­ous CEO, Nick Leschly, branched off to lead a new com­pa­ny in 2sev­en­ty, split­ting the gene ther­a­py and rare dis­ease and gene ther­a­py pipeline from its ef­forts in on­col­o­gy.

The BLA sub­mis­sion for beti-cel is based on da­ta from two Phase III stud­ies and Phase I/II stud­ies with a com­bined 63 pa­tients treat­ed as of March 9 — the most re­cent up­date on the time­line. The da­ta in­clude two pa­tients with up to sev­en years of fol­low-up, eight with at least six years of fol­low-up and 19 with at least five years of fol­low-up. — Paul Schloess­er

CG On­col­o­gy en­ters a col­lab­o­ra­tion with Roche’s Tecen­triq

Cal­i­for­nia bio­phar­ma CG On­col­o­gy has en­tered in­to a clin­i­cal tri­al col­lab­o­ra­tion agree­ment with Roche to eval­u­ate com­bin­ing CG’s on­colyt­ic im­munother­a­py CG0070 with Roche’s PD-(L)1 drug Tecen­triq.

The Phase I/II tri­al will eval­u­ate safe­ty and ef­fi­ca­cy of this com­bi­na­tion in pa­tients with var­i­ous ad­vanced sol­id tu­mors yet to be dis­closed. CG On­col­o­gy will spon­sor the study and Roche will pro­vide Tecen­triq.

CG0070, CG On­col­o­gy’s lead I/O can­di­date, is in an on­go­ing Phase III monother­a­py study and has been ad­min­is­tered in over 100 pa­tients for non-mus­cle in­va­sive blad­der can­cer. CG0070 is based on a mod­i­fied ade­n­ovirus type 5 back­bone and is de­signed to de­stroy var­i­ous can­cer cells, in­clud­ing blad­der tu­mor cells, through a de­fec­tive retinoblas­toma path­way.

“We are pleased to en­ter in­to this col­lab­o­ra­tion agree­ment with Roche,” said CG CEO Arthur Kuan in a state­ment. “We are ex­cit­ed to ini­ti­ate this im­por­tant clin­i­cal tri­al with our lead on­colyt­ic im­munother­a­py, CG0070, in com­bi­na­tion with ate­zolizum­ab to eval­u­ate the po­ten­tial syn­er­gy of these two im­munother­a­pies in can­cer,” Kuan added.

Ad­di­tion­al de­tails of the col­lab­o­ra­tion were not dis­closed. — Paul Schloess­er

Months af­ter its pub­lic de­but, Celu­lar­i­ty strikes a de­vel­op­ment deal with Onc­ter­nal

Onc­ter­nal Ther­a­peu­tics is join­ing forces with Celu­lar­i­ty and its pla­cen­tal-de­rived cells to de­vel­op cell ther­a­pies tar­get­ing ROR1, the com­pa­nies said Mon­day.

Though they’re keep­ing the terms of the deal un­der wraps, the duo say they will al­so ex­plore the use of Onc­ter­nal’s Phase II mon­o­clon­al an­ti­body cirm­tuzum­ab in com­bi­na­tion with Celu­lar­i­ty’s NK cells. Specif­i­cal­ly, they’ll be look­ing at cirm­tuzum­ab and CYNK-101, a pla­cen­tal-de­rived NK cell ther­a­py that has been en­gi­neered to work to­geth­er with ther­a­peu­tic an­ti­bod­ies. Celu­lar­i­ty will al­so eval­u­ate ROR1-tar­get­ed CAR-NK and CAR-T cell ther­a­pies as ex­ten­sions of its CYNK and Cy­CART pro­grams, re­spec­tive­ly.

“Our re­search study­ing ROR1 sug­gests the po­ten­tial for a range of new tar­get­ed ther­a­peu­tics, ca­pa­ble of ad­dress­ing a wide va­ri­ety of both sol­id tu­mors and hema­to­log­i­cal ma­lig­nan­cies,” James Bre­it­mey­er, founder and CEO of Onc­ter­nal, said in a state­ment.

Celu­lar­i­ty CEO Bob Hariri steered the com­pa­ny to Wall Street a few months ago, merg­ing with a SPAC called GX Ac­qui­si­tion Corp. The deal gave the com­pa­ny $138 mil­lion to work with as it push­es its NK cell and T cell ther­a­pies deep­er in­to ear­ly-stage tri­als. Onc­ter­nal al­so jumped on­to Nas­daq by way of a re­verse merg­er, com­bin­ing with GTx back in 2019. — Nicole De­Feud­is

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 119,800+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'

 

Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

FDA ad­comm to de­cide on mol­nupi­ravir EUA; Can­cer at­las un­veils new po­ten­tial drug tar­get

The FDA has another adcomm coming down the pipeline — this time on Covid-19 oral antiviral molnupiravir.

The federal agency’s advisory committee will meet on November 30th to go over Merck and Ridgeback’s EUA request for their investigational antiviral drug, and discuss the available data supporting its use in Covid-19 patients.

This comes two weeks after Merck claimed that their antiviral pill reduced the chance that newly diagnosed Covid-19 patients would be hospitalized or die by 50%. The pharma made the announcement after interim data on 775 patients in their clinical trial showed the antiviral’s potential.

Tillman Gerngross, Adagio CEO

Q&A: Till­man Gern­gross ex­plains why his Covid mAb will have an edge over an al­ready crowd­ed field

If anyone knows about monoclonal antibodies, it’s serial entrepreneur, Adimab CEO, and Dartmouth professor of bioengineering Tillman Gerngross.

Even the name of Gerngross’ new antibody startup Adagio Therapeutics is meant to reflect his vision behind the development of his Covid-19 mAb: slowly, he said, explaining that “everyone else, whether it’s Regeneron, Lilly, or AstraZeneca, Vir, they all valued speed over everything.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.