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.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Andrew Allen, Gritstone CEO (Gritstone via website)

Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Bris­tol My­ers Squibb gets re­view date for Op­di­vo com­bo in gas­tric can­cer, look­ing to over­turn Keytru­da's 3-year lead

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.