Look­ing to cure Type 1 di­a­betes, in­vestors front $114M to launch a pi­o­neer­ing hu­man study at Sem­ma

Three years ago, Har­vard’s Doug Melton pub­lished a land­mark study out­lin­ing how he had suc­cess­ful­ly used stem cells to cre­ate in­sulin-pro­duc­ing pan­cre­at­ic be­ta cells that were in­sert­ed in bulk in­to mice and suc­cess­ful­ly pro­tect­ed from an im­mune re­sponse — a break­through in re­gen­er­a­tive med­i­cine that bore re­al promise to pro­vide a cu­ra­tive ap­proach for Type 1 di­a­betes that could con­ceiv­ably end a life­time of in­sulin shots.

It was the cul­mi­na­tion of 23 years of lab work, launched when his son was di­ag­nosed with Type 1 di­a­betes. And that achieve­ment marked the be­gin­ning of some­thing new in biotech.

That same year Sem­ma Ther­a­peu­tics would be launched — with a $44 mil­lion A round land­ing in 2015 — in pur­suit of a mis­sion to com­plete one of the most am­bi­tious pre­clin­i­cal pro­grams in the re­gen­er­a­tive med field. And af­ter work­ing on all the nit­ty grit­ty re­search need­ed to see if this tech could be scaled up to hu­man size, an ex­pand­ed syn­di­cate of ven­ture in­vestors have put to­geth­er a whop­ping $114 mil­lion round with plans to take this in­to hu­mans for a first-of-its-kind proof-of-con­cept study.

One of the big chal­lenges Sem­ma faced in scal­ing up, Melton tells me, was to cre­ate a mem­brane specif­i­cal­ly de­signed with pores that were large enough for mol­e­cules to pass through but too small for im­mune cells to pen­e­trate. Us­ing some cal­cu­la­tions from the lab, Melton and his col­leagues es­ti­mat­ed that they would need some 150 mil­lion cells — pos­si­bly rang­ing up to three times that amount — in or­der to pro­vide the nat­ur­al in­sulin need­ed to elim­i­nate the shots.

Melton com­pares the mem­brane to a tea bag, but one that couldn’t be over­loaded. The re­place­ment cells, he said, “will on­ly se­crete the right amount de­pend­ing on the lev­el of sug­ar in the blood.”

Mark Fish­man

The big round marks an in­flec­tion point for the 35 staffers at Sem­ma, says chair­man Mark Fish­man, who joined Har­vard af­ter a 13-year stint run­ning the No­var­tis In­sti­tutes for Bio­Med­ical Re­search.

“Un­til you get this kind of fund­ing,” says Fish­man, “you don’t know how broad your strat­e­gy can be. With this fund­ing, we can get through a proof-of-con­cept tri­al, with enough in­for­ma­tion to know whether this works.” They can fol­low par­al­lel tracks and al­so start think­ing through some new di­rec­tions to pur­sue as their di­a­betes treat­ment pro­ceeds.

Typ­i­cal­ly, you nev­er see VCs back­ing a di­a­betes play. A few ma­jor multi­na­tion­als con­trol the bulk of the de­vel­op­ment work be­cause the reg­u­la­to­ry re­quire­ments for ap­proval are daunt­ing. But that’s in Type 2, which is spread­ing at epi­dem­ic pro­por­tions. In­her­it­ed Type 1 di­a­betes has a much small­er pop­u­la­tion, adds Fish­man, which makes it pos­si­ble to con­sid­er push­ing ahead in­to late-stage de­vel­op­ment alone.

As for time­lines, Fish­man is play­ing his cards close to the vest. Ear­ly-stage re­search, as he knows all too well, has a lot of vari­ables that can af­fect time­lines. An IND is com­ing, he says, and the com­pa­ny will see how it plays out, with a spe­cial fo­cus in start­ing to look at how durable a sin­gle treat­ment can be — one of the the big is­sues that Melton is most in­trigued by.

The mega-round in play al­so un­der­scores the will­ing­ness of ven­ture back­ers to go big these days when they’re fo­cused on mak­ing a pi­o­neer­ing ad­vance.

Eight Roads Ven­tures and Cowen Health­care In­vest­ments co-led the fi­nanc­ing with help from MPM Cap­i­tal, F-Prime Cap­i­tal Part­ners and Arch Ven­ture Part­ners. Ex­ist­ing strate­gic part­ners in­clude No­var­tis, Medtron­ic and the JDRF T1D Fund, and new in­vestors jump­ing in in­clude ORI Health­care Fund, Wu Cap­i­tal, 6 Di­men­sions Cap­i­tal and SinoPharm Cap­i­tal. Sem­ma named Daniel Auer­bach from Eight Roads and Kevin Raidy from Cowen to its board of di­rec­tors.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.