A new RNA part­ner­ship hunts rare epilep­sies; Ab­b­Vie and I-Mab tack­le CD47

Zo­genix is get­ting a new part­ner as it looks to tack­le rare child­hood seizures.

The Cal­i­for­nia-based biotech, which de­vel­oped and pro­duces the seizure drug Fin­tepla, is team­ing with Tre­vard Bio­sciences to de­vel­op RNA-based gene ther­a­pies for Dravet syn­drome and oth­er ge­net­ic epilep­sies.

Spun out of MIT and ad­vised by David Liu and Bob Langer, Tre­vard is try­ing to use tran­scrip­tion­al RNA to in­crease the ex­pres­sion of healthy copies of the gene be­hind Dravet’s syn­drome in pa­tients and de­crease the ex­pres­sion of the faulty copy.

Zo­genix will pay Tre­vard $10 mil­lion up­front and $5 mil­lion in stock, with mile­stones be­tween $70 mil­lion and $100 mil­lion avail­able per pro­gram. — Ja­son Mast

Chi­na’s I-Mab push­es ahead with com­bo tri­als of Ab­b­Vie-part­nered CD47 drug

Ab­b­Vie’s CD47 part­ner I-Mab says the drug, lem­zopar­limab, is cruis­ing nice­ly along with clin­i­cal de­vel­op­ment, mov­ing in­to dose ex­pan­sion in a com­bo study.

Topline re­sults from the study, which tests the pair­ing of lem­zopar­limab with Rit­ux­an and Keytru­da, re­spec­tive­ly, are ex­pect­ed next year. Both will en­roll pa­tients with non-Hodgkin’s lym­phoma; the Rit­ux­an por­tion will take place in both the US and Chi­na.

I-Mab, which has of­fices in Shang­hai and Gaithers­burg, MD, said it’s al­so push­ing in­to late-stage tri­als in re­lapsed/re­frac­to­ry acute myeloid leukemia (AML) or myelodys­plas­tic syn­drome in Chi­na af­ter wrap­ping monother­a­py dose es­ca­la­tion.

It scored a hefty part­ner in Ab­b­Vie in Sep­tem­ber, scor­ing $180 mil­lion up­front for a deal that can add up to $3 bil­lion. — Am­ber Tong

Ma­gen­ta and blue­bird team up to look for sick­le cell clues

Blue­bird bio is en­list­ing Ma­gen­ta in its ef­forts to clear one of the hur­dles to sick­le cell gene ther­a­py.

The Cam­bridge biotech, which has long been one of the lead­ers in the sick­le cell gene ther­a­py field, is try­ing to find a bet­ter way of re­mov­ing pa­tients’ stem cells, so they can be edit­ed and re­turned.

In many bone mar­row trans­plants, you can get the cells in the right spot to be re­moved with a GCS-F an­ti­body. But in sick­le cell pa­tients, the GCS-F block­ers can lead to the painful va­so-oc­clu­sive crises that are a hall­mark of the dis­ease. An al­ter­na­tive, Pler­ix­afor, ex­ists but it’s not a per­fect re­place­ment, of­ten re­quir­ing pa­tients to come in for mul­ti­ple col­lec­tions.

Ma­gen­ta’s MG­TA-145 is de­signed to work bet­ter, when com­bined with Pler­ix­afor. — Ja­son Mast

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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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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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 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.

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.”

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