'Miss­ing link': Bay­er, Morn­ing­side help cat­a­pult a new kind of de­liv­ery tech to cell and gene ther­a­py

Robert Mill­man

Robert Mill­man co-found­ed and led Sem­ma Ther­a­peu­tics as CEO while al­so a man­ag­ing di­rec­tor of MPM Cap­i­tal. By the time he left the stem cell ther­a­py pi­o­neer — two years be­fore it would be sold to Ver­tex — he had left VC life be­hind.

In­stead, he went around scout­ing new tech­nolo­gies, vis­it­ing with tech trans­fer of­fices and aca­d­e­mics in the Boston/Cam­bridge area to find wor­thy ideas that could ben­e­fit from his IP ex­per­tise.

And he found quite a few.

One of them is Vesi­gen Ther­a­peu­tics, which is launch­ing with $28.5 mil­lion to turn a new class of ex­tra­cel­lu­lar vesi­cles in­to pack­ag­ing and de­liv­ery tools for a va­ri­ety of car­gos — mR­NA, Cas9, base ed­i­tors, PRO­TACs, you name it.

“The bar­ri­er to be­ing a drug com­pa­ny right now is not tech­nol­o­gy or tar­gets,” he told End­points News. “There’s plen­ty of tar­gets, there’s plen­ty of tech­nol­o­gy, but there’s no way of get­ting the two of those in­to a pa­tient.”

Quan Lu

Vesi­gen’s plat­form stems from sci­en­tif­ic co-founder and Har­vard pro­fes­sor Quan Lu’s work on AR­MMs, or ar­restin do­main-con­tain­ing pro­tein 1 [AR­RDC1]-me­di­at­ed mi­crovesi­cles. As he wrote in Na­ture Com­mu­ni­ca­tions in March 2018, AR­MMs are “dis­tinct from ex­o­somes,” in that they lend them­selves to more con­trolled pro­duc­tion and car­gos that can be re­cruit­ed in­to the vesi­cle dur­ing the process of man­u­fac­tur­ing rather than put to­geth­er af­ter the fact.

“I don’t un­der­stand re­al­ly why a whole lot of ven­ture firms didn’t jump on it at the time,” Mill­man said. “I think it’s be­cause most were fo­cused on ex­o­somes and just thought that this was an­oth­er ex­o­some com­pa­ny, and there was al­ready Co­di­ak and Evox.”

But Lu showed in his pa­per de­liv­ery of func­tion p53 pro­tein to an p53 de­fi­cient an­i­mal and restor­ing p53 ac­tiv­i­ty: “To me it was rev­o­lu­tion­ary.”

And com­pared to the cur­rent work­hors­es of ge­net­ic med­i­cines such as vi­ral vec­tors and lipid nanopar­ti­cles, AR­MMs promise to pack­age big­ger and more var­ied pay­loads, get in­to more or­gans, and stay in cir­cu­la­tion longer with­out trig­ger­ing tox­i­c­i­ty.

Faced with the “Willy Won­ka-like” range of pos­si­bil­i­ties, Mill­man said the top or­der right now for his team of sev­en is to fig­ure out a man­u­fac­tur­ing set­up that would load the car­gos ef­fi­cient­ly and be cleared by reg­u­la­tors.

In the three years that it will like­ly take to get to the cusp of the clin­ic, Vesi­gen is al­so col­lab­o­rat­ing with re­searchers to show how AR­MMs can get in­to “tis­sues that no one can go to” — with im­pli­ca­tions in oc­u­lar and neu­ro­log­i­cal dis­eases as well as can­cer.

The Se­ries A, co-led by Leaps by Bay­er and Morn­ing­side Ven­tures and joined by Lin­den Lake Ven­tures and Alexan­dria Ven­ture In­vest­ments, should fund the com­pa­ny un­til 2025, when Mill­man ex­pects to have built out the team to 30.

Jür­gen Eck­hardt

It’s ex­act­ly the kind of projects that his group likes to in­vest in ear­ly, Leaps by Bay­er head Jür­gen Eck­hardt said, es­pe­cial­ly as big be­liev­ers in the cell and gene ther­a­py space who see de­liv­ery as the “miss­ing link.” For Vesi­gen, it al­so opens up op­por­tu­ni­ties to find part­ners with­in the phar­ma gi­ant’s net­work down the road.

While Mill­man is keen not to lose fo­cus, he al­so isn’t hid­ing am­bi­tions to make this “the next big one.”

“I saw all of the prob­lems — Al­ny­lam I was there for three years, we couldn’t get de­liv­ery. I worked very ear­ly at Cel­era and es­tab­lished col­lab­o­ra­tions with Io­n­is and again de­liv­ery was lim­it­ed. I was help­ing set up Verve Ther­a­peu­tics and again de­liv­ery of Cas9 was a prob­lem,” he said. “And here was an elo­quent so­lu­tion. I was just pleased to be at the right place at the right time.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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