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

Zo­genix teams with RNA up­start on rare epilep­sy

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

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Bomb squad called to As­traZeneca vac­cine plant; Lu­men nabs CARB-X award for low-cost an­tidiar­rheal

A plant located in Wrexham, Wales that is packing the Oxford/AstraZeneca Covid-19 vaccine into vials was surrounded by a bomb squad after officials called police to report a suspicious package.

The alert caused a partial evacuation of the plant, the BBC was among those to report Wednesday. The owner of the plant, British drugmaker Wockhardt UK, said it was cooperating with local authorities and that there were no reports of any injuries.

Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

As new Covid-19 task force gets un­der­way, threat looms of vac­cine, mon­o­clon­al an­ti­body-re­sis­tant vari­ants

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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