Bob Langer-found­ed cell ther­a­py play­er loads up $65M to fund Roche-part­nered can­cer work while ex­pand­ing in­to vac­cines for in­fec­tious dis­eases

SQZ Biotech isn’t plan­ning to test any­thing against Covid-19 for now. But when the next pan­dem­ic strikes, CEO Ar­mon Sharei wants its cell ther­a­py plat­form — which is es­sen­tial­ly al­so a vac­cine plat­form — to be ready for plug-and-play.

The ex­pan­sion in­to in­fec­tious dis­eases is one of many things the Wa­ter­town, MA-based com­pa­ny plans to ac­com­plish with its lat­est $65 mil­lion Se­ries D.

Bob Langer

Vi­ral and bac­te­r­i­al in­fec­tions, both the chron­ic kind and the ones that can pose a sud­den, glob­al threat, had been on his radar since his PhD days at Bob Langer’s MIT lab, Sharei told End­points News. Now that they have pushed the first im­ple­men­ta­tion of their cell en­gi­neer­ing tech­nique in­to the clin­ic and fig­ured out the man­u­fac­tur­ing set­up — in the form of an on­col­o­gy pro­gram part­nered with Roche — with an­oth­er lined up for lat­er this year, the time is ripe for some prepa­ra­tion for an IND fil­ing in 2021.

As with the ap­pli­ca­tions in can­cer, two of SQZ’s three tech plat­forms, name­ly the anti­gen pre­sent­ing cells (APC) and ac­ti­vat­ing anti­gen car­ri­ers (AAC), can come in­to play here. Un­der both ap­proach­es, cells get squeezed and, as their mem­branes get tem­porar­i­ly dis­rupt­ed, have ma­te­r­i­al slipped in­side. That car­go of in­ter­est can be a vi­ral pro­tein for well-char­ac­ter­ized virus­es, such as the spike pro­tein in SARS-CoV-2, or even a whole chunk of the virus in sit­u­a­tions where sci­en­tists have yet to de­ci­pher a nov­el pathogen. The for­mer in­volves white blood cells while the lat­ter us­es red blood cells as a kind of Tro­jan horse at var­i­ous lym­phoid or­gans to kick off an im­mune re­sponse.

Ei­ther way, it di­verges from the tra­di­tion­al goal of in­duc­ing an an­ti­body re­sponse.

“Ul­ti­mate­ly the core prob­lem we ad­dress with the APC pro­gram and the AAC pro­gram re­lates to gen­er­at­ing CD8 T cells re­spons­es which all these pri­or at­tempts at can­cer vac­cines had failed,” Sharei said. “A lot of times vac­cines can on­ly ever work pro­phy­lac­ti­cal­ly. But this mech­a­nism of vac­ci­na­tion, be­cause it lever­ages killer T cells, it could be used for both con­texts.”

On top of that dual func­tion, this plat­form promis­es to soothe the man­u­fac­tur­ing headache that’s be­set even de­vel­op­ers of the most ad­vanced vac­cine can­di­dates in the Covid-19 race.

Be­gin­ning with the very first pa­tient they dosed this Jan­u­ary, SQZ has been able to com­plete pro­cess­ing the cells with­in 24 hours and re­turn them to pa­tients for in­fu­sion — ship­ping and re­lease test­ing in­clud­ed — in a week. The rel­a­tive­ly sim­ple pro­ce­dure and ab­sence of pre­con­di­tion­ing re­quire­ments have al­so al­lowed their tri­al, in­volv­ing pa­tients with HPV+ tu­mors, to con­tin­ue.

To go even faster, Sharei said his team will be look­ing to con­sol­i­date that whole process — from the ac­tu­al squeez­ing to cell wash­ing and oth­ers — in­to a sin­gle box that can be de­ployed at a point-of-care fa­cil­i­ty, shav­ing even more time off.

“There’s ob­vi­ous­ly some en­gi­neer­ing work to do, I don’t want to triv­i­al­ize it, but there is no big leap nec­es­sary to in­te­grate it,” Sharei said.

Dis­cus­sions with reg­u­la­tors will al­so need to es­tab­lish the kinds of qual­i­ty con­trol and test­ing that would be need­ed if the ma­te­r­i­al no longer needs to be shipped across the coun­try.

With 100 full timers on staff, SQZ is al­so con­tin­u­ing to plough on the au­toim­mune field with its third and ear­li­est-stage plat­form, the to­ler­iz­ing anti­gen car­ri­ers (TACs), which al­so lever­ages red blood cells.

The Ju­ve­nile Di­a­betes Re­search Foun­da­tion is a col­lab­o­ra­tor here, and their T1D Fund has chipped in on the lat­est round af­ter back­ing SQZ’s Se­ries C.

Oth­er ex­ist­ing in­vestors such as GV, Il­lu­mi­na Ven­tures, In­vus, Nan­oDi­men­sion and Po­laris Part­ners al­so par­tic­i­pat­ed in the round, which was led by Temasek.

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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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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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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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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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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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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Alex Leech, Alchemab CEO (SV Health Investors)

Alchemab bags fresh round of in­vestor for tar­get-ag­nos­tic an­ti­body de­vel­op­ment for Hunt­ing­ton's, Covid-19

With a “target-agnostic” approach to antibody development, the UK’s Alchemab has used lessons learned from patients with resistance to certain diseases to chase after conditions as far apart as Huntington’s and Covid-19. Now, investors are jumping on board the concept with an $86 million Series A.

The proceeds will go toward advancing the company’s target-agnostic drug discovery program, a release said. That approach looks at the antibody repertoires of patients who show resistance to typically destructive diseases regardless of genetic disposition.

Craig Parker, Surrozen CEO

The world of Wnt heads to Nas­daq as Sur­rozen an­nounces a $212M SPAC deal

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Another day, another SPAC merger in the world of biotech.

Less than 24 hours after Tango Therapeutics announced its own leap to Nasdaq through the blank check route, Surrozen has decided to take a similar step. The Wnt pathway-focused biotech is reverse-merging with Consonance Capital Management’s SPAC in a $212 million deal, which includes $92 million from the shell company and $120 million in PIPE financing.