Gly­coMimet­ics stock rock­ets up on the lat­est ‘break­through’ ti­tle in can­cer R&D

Late last year Rockville, MD-based Gly­coMimet­ics turned up at ASH to re­port out an ear­ly snap­shot of some pos­i­tive out­comes from the first stage of a Phase I/II study of GMI-1271, its E-se­lectin an­tag­o­nist, for a small group of pa­tients with acute myeloid leukemia. And with an­oth­er batch of da­ta be­ing read­ied for AS­CO in a cou­ple of weeks, the biotech says that the FDA has help­ful­ly pro­vid­ed a break­through drug des­ig­na­tion to help them ac­cel­er­ate the de­vel­op­ment work through to a po­ten­tial mar­ket­ing ap­pli­ca­tion.

The news was cheered on by in­vestors Wednes­day af­ter­noon with a 35% spike for the biotech’s stock $GLYC.

Now in the Phase II por­tion of the study, in­ves­ti­ga­tors for the com­pa­ny have been re­cruit­ing two groups of AML pa­tients: Treat­ment re­sis­tant sub­jects and new­ly di­ag­nosed pa­tients over the age of 60. The drug is de­signed to tar­get path­ways tu­mors use to de­fend against ther­a­pies, height­en­ing the ef­fect of chemo.

Last De­cem­ber in­ves­ti­ga­tors not­ed that for 33 study par­tic­i­pants with re­lapsed or re­frac­to­ry dis­ease in one arm of the tri­al, the com­plete re­sponse rate was 45%. For 11 new­ly di­ag­nosed study par­tic­i­pants 60 or more years of age in the sec­ond arm of the tri­al, the CR rate was 73%.

He­len Thack­ray

BTD sta­tus for this ther­a­py fol­lows or­phan and fast-track sta­tus from the FDA, which has been wide open to push­ing through ac­cel­er­at­ed ap­provals in the on­col­o­gy field, trans­form­ing de­vel­op­ment work for new can­cer drugs.

“The FDA’s grant­i­ng to GMI-1271 of Break­through Ther­a­py des­ig­na­tion will fur­ther help Gly­coMimet­ics to ac­cel­er­ate the de­vel­op­ment of GMI-1271 as a treat­ment for this very dif­fi­cult-to-treat pa­tient pop­u­la­tion,” said He­len Thack­ray, the CMO of Gly­coMimet­ics, in a pre­pared state­ment. “We be­lieve GMI-1271 when com­bined with chemother­a­py has the po­ten­tial to ad­dress an un­met ther­a­peu­tic need for in­di­vid­u­als liv­ing with AML.”

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.

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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?

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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.

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

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Jacob Van Naarden, Senior VP, CEO of Loxo Oncology at Lilly; President, Lilly Oncology

Eli Lil­ly bags FDA nod for Verzenio in ear­ly breast can­cer, but a con­tro­ver­sial di­ag­nos­tic could dog its roll­out

As Eli Lilly works to consolidate its internal and Loxo teams into an oncology powerhouse, the drug giant is putting high hopes on CDK 4/6 inhibitor Verzenio to help drive the portfolio into the future. Now, the drug has scored a paradigm-altering win in early breast cancer — but will a controversial companion diagnostic hamstring Lilly’s market plans?

The FDA on Wednesday approved CDK 4/6 inhibitor Verzenio in combination with physician’s-choice endocrine therapy to cut the risk of relapse in patients with high-risk HR-positive, HER2-negative breast cancer, Lilly said in a release.

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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.

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.