IDO-de­rived can­cer vac­cine scores in com­bo tri­al with Keytru­da; Vir teams up with WuXi on coro­n­avirus an­ti­bod­ies

→ Copen­hagen-based IO Biotech said that its can­cer vac­cine has passed the high bar set for its in­ter­im fu­til­i­ty analy­sis in Phase II, show­ing “a sub­stan­tial­ly high­er” over­all re­sponse rate among non-small cell lung can­cer pa­tients when com­bined to Keytru­da than the PD-1 alone —which ac­cord­ing to the com­pa­ny in­duced his­tor­i­cal ORR of 39%.

In­ves­ti­ga­tors will car­ry on eval­u­at­ing IO-102 plus Keytru­da, with fi­nal da­ta ex­pect­ed by the end of the year. The can­di­date is an IDO-de­rived pep­tide se­quence de­signed to stir up T cell ac­tion against IDO — the en­zyme tar­get for an ill-fat­ed class of small mol­e­cule drugs. “In that sense you have flags on the sur­face of im­mune sup­pres­sive cells and [tu­mor cells] that our T cells can rec­og­nize and there­by ac­tu­al­ly get rid of those IDO pos­i­tive cells, hence a com­plete­ly dif­fer­ent MOA than IDO SMI,” CEO Mai-Britt Zoc­ca told End­points News.

IO Biotech is fund­ing the study while part­ner Mer­ck gets to share the re­sults. A cock­tail of IO102 and IO103 — which di­rects T cells against PD-L1 — is be­ing test­ed in com­bi­na­tion with Op­di­vo for melanoma pa­tients.

→ A month af­ter join­ing the hunt for an­ti­bod­ies that could treat Covid-19, Vir Biotech­nol­o­gy is en­list­ing WuXi Bi­o­log­ics to do the cell-line de­vel­op­ment, process and for­mu­la­tion de­vel­op­ment as well as ini­tial man­u­fac­tur­ing. The San Fran­cis­co-based start­up said it’s iden­ti­fied a num­ber of mon­o­clon­al an­ti­bod­ies that bind to SARS-CoV-2, iso­lat­ed from in­di­vid­u­als who had sur­vived SARS. A deal with WuXi — which gives the pow­er­ful CRO rights to com­mer­cial­ize in Chi­na — po­si­tions them for quick ad­vance­ment, CEO George Scan­gos said.

As­traZeneca has di­vest­ed an­oth­er gas­troin­testi­nal drug, col­lect­ing $52.5 mil­lion up­front from Red­Hill Bio­phar­ma for par­tial rights to Movan­tik. The drug, which was orig­i­nal­ly de­vel­oped by Nek­tar Ther­a­peu­tics to treat opi­oid-in­duced con­sti­pa­tion, gen­er­at­ed US sales of $96 mil­lion last year (Dai­ichi Sankyo shares some of the prof­its). As­traZeneca will con­tin­ue to sup­ply the drug for a while.

→ Parisian biotech Sci­pio bio­science, which helps re­search and clin­i­cal labs with sin­gle-cell se­quenc­ing, has se­cured €6 mil­lion in Se­ries A fund­ing. The mon­ey will fu­el the fi­nal de­vel­op­ment of its sin­gle-cell RNA-se­quenc­ing kit, as well as mar­ket­ing and busi­ness de­vel­op­ment ef­forts to pre­pare for com­mer­cial launch in 2022.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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TG Ther­a­peu­tics pre­pares to ap­proach reg­u­la­tors with MS drug; In­vestors bet $93M on a life sci­ences tools com­pa­ny

Gearing up for a battle with pharma giants, TG Therapeutics says it’s nearly ready to approach regulators with its CD20 drug for multiple sclerosis.

The biotech read out results from two Phase III trials at AAN, which show its candidate ublituximab reduced patients’ annualized relapse rate by 60% and 50% compared to Sanofi’s Aubagio, or teriflunomide — an 8-year-old drug that’s often used as a benchmark.

J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Roche touts 2-year ef­fi­ca­cy da­ta for SMA ther­a­py Evrys­di; NIH funds new net­work to study flu and virus­es with ‘pan­dem­ic po­ten­tial’

Two years after therapy, 61% of babies with spinal muscular atrophy Type 1 treated with Roche’s Evrysdi (risdiplam) were able to sit up without support for at least five seconds compared with just 29% of patients after a year, the drugmaker said Thursday.

As part of the FIREFISH-2 Phase III study, researchers tested Evrysdi’s ability to help 41 infant patients sit up without support at 5 and 30 seconds. Data showed that 44% of patients were able to sit without support for 30 seconds at the two-year check-in compared with 17% after one year.