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.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Rich Heyman (ARCH)

Rich Hey­man joins PMV Phar­ma, a p53 biotech, as it adds $70 mil­lion in Se­ries D

Less than a year after pulling in an impressive $62 million Series C round, PMV Pharma is back at it again.

The Cranbury, NJ-based biotech announced Monday an additional $70 million in Series D financing as it seeks to develop cancer therapies targeting p53 mutations. Additionally, PMV also introduced longtime biotech entrepreneur Rich Heyman as chairman of the board of directors.

“This financing provides PMV Pharma with the resources to expand our pipeline and to potentially advance multiple p53 therapies into the clinic,” said PMV president and CEO David Mack in a statement.

FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is facing a formal investigation on manslaughter charges, due to accusations that its epilepsy drug Depakine caused birth malfunctions and slow neurological development when taken during pregnancy.

The French pharma was formally charged in February, years after evidence surfaced that the drug, sodium valproate, posed neurodevelopmental risks. Sodium valproate first hit the market in 1967 for the treatment of epilepsy and bipolar disorder, and is currently prescribed in more than 100 countries.

Days af­ter seal­ing Sanofi pact, Kymera beats a path to the Nas­daq with $100M IPO pitch

Back in March, when Kymera Therapeutics closed $102 million in Series C funding led by Biotechnology Value Fund and Redmile Group, CEO Nello Mainolfi noted the protein degradation player was “at the cusp of transitioning” into a fully integrated R&D company. Five months and a major Sanofi pact later, he’s back asking for another little push to get there.

Kymera has penciled in $100 million in its first IPO pitch — although given the public market’s seemingly insatiable appetite for biotechs these days the final figure is anyone’s guess.

CymaBay flash­es pos­i­tive re­sults from the tri­al they have to re­launch

Two weeks after the FDA lifted its clinical hold on their lead drug, CymaBay said it showed positive results in an aborted Phase III trial.

The drug, a small molecule known as seladelpar, had been in development for three different liver conditions before an independent review of a NASH study last year showed that it might actually be damaging patient’s liver cells. The FDA slapped a clinical hold across all three trials, only lifting it last month when an FDA review determined that the drug hadn’t caused liver damage.

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH; Am­gen, Ab­b­Vie, Take­da team on a drug

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

Frank Zhang (AP Images)

CAR-T fil­ing in sight, Frank Zhang grabs full con­trol of J&J-part­nered Leg­end Biotech, steps down from Gen­Script

Two months after Yuan Xu steered Legend Biotech to a $424 million public debut on the Nasdaq, founder and chairman Frank Zhang is grabbing the reins as CEO.

In conjunction with the move, Zhang is also stepping down from the helm of GenScript — a position he’s held for 18 years. GenScript, a Hong Kong-listed CRO, hatched Legend as a subsidiary in 2015 before spinning it out, and remains a majority shareholder.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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