News brief­ing: New senes­cent cell play launch­es with $12M; Jun­shi leads Se­ries C+ in­fu­sion for Chi­nese syn­thet­ic lethal­i­ty play­er

The idea of tar­get­ing senes­cent cells to treat age-re­lat­ed dis­eases is con­tin­u­ing to gath­er steam, and Khosla Ven­tures is help­ing get the lat­est play go­ing with a $12 mil­lion seed round.

Rube­do Life Sci­ences is fo­cus­ing its ini­tial ef­forts on res­pi­ra­to­ry dis­eases such as id­io­path­ic pul­monary fi­bro­sis and ob­struc­tive pul­monary dis­ease, with pre­clin­i­cal pro­grams in on­col­o­gy, neu­rode­gen­er­a­tive con­di­tions, skele­tal mus­cle dis­or­ders and oth­ers to fol­low.

With the mon­ey, the goal is to move sev­er­al com­pounds in­to IND-en­abling stud­ies in pul­monary dis­eases start­ing next year.

“We were at­tract­ed to Rube­do by the speed of which the com­pa­ny is ad­vanc­ing mul­ti­ple lead com­pounds in­to IND-en­abling stud­ies across dif­fer­ent ar­eas of un­met med­ical need,” Zal Bil­imo­ria, man­ag­ing di­rec­tor of Refac­tor Cap­i­tal, said in a state­ment. The VC firm joined Longevi­ty Fund, Shan­da and oth­ers in the fi­nanc­ing.

Af­ter spend­ing some years at Stan­ford re­search­ing ag­ing and re­gen­er­a­tive med­i­cine, CEO Mar­co Quan­ta co-found­ed the com­pa­ny with 5AM Ven­ture Part­ners en­tre­pre­neur-in-res­i­dence Mark Gal­lop and Ju­lian Klein, who al­so did his grad­u­ate work at Stan­ford. The big idea at Rube­do has to do with a com­pu­ta­tion­al drug dis­cov­ery plat­form that comes up with small mol­e­cule can­di­dates to drug senes­cent cells.

Jun­shi leads Se­ries C+ in­fu­sion for Chi­nese syn­thet­ic lethal­i­ty play­er

Chi­nese biotech Im­pact Ther­a­peu­tics has grabbed $50 mil­lion to fu­el more work on a slate of drugs that tar­get syn­thet­ic lethal­i­ty, in­clud­ing PARP and oth­er DNA dam­age re­sponse path­ways.

Jun­shi Bio­sciences — known it­self for a slate of an­ti­bod­ies aimed at can­cer, au­toim­mune dis­eases and more re­cent­ly, Covid-19 — led the Se­ries C+. Lil­ly Asia Ven­tures and Chi­na Sum­mit Cap­i­tal re­turned for more, flanked by new in­vestors AJ As­set Mgmt, Zhen­ji Cap­i­tal, West Foun­tain Glob­al Fund and Ausun Phar­ma.

“2021 is ex­pect­ed to be a sig­nif­i­cant turn point for us, as we are start­ing a se­ries of clin­i­cal tri­als in the US,” CEO Jun Bao said in a state­ment. — Am­ber Tong

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.