Chris Garabedian (Forge Biologics)

Chris Garabe­di­an's in­cu­ba­tor leads Se­ries A fund­ing for gene ther­a­py man­u­fac­tur­er/de­vel­op­er com­bo

Back in De­cem­ber, long­time in­dus­try in­vestor Chris Garabe­di­an helped launch a $210 mil­lion VC fund for ear­ly-stage biotechs. On Tues­day, Garabe­di­an and the Per­cep­tive Xon­toge­ny Ven­ture Fund in­tro­duced its lat­est start­up.

Tim Miller

The ex-Sarep­ta CEO has teamed with for­mer Abeona chief Tim Miller to cre­ate Forge Bi­o­log­ics, a gene ther­a­py com­pa­ny fo­cus­ing on both man­u­fac­tur­ing and de­vel­op­ment of GMP ade­no-as­so­ci­at­ed virus­es, with $40 mil­lion in Se­ries A fund­ing. Forge is the eighth biotech in which the PXV Fund has in­vest­ed — but on­ly the sixth an­nounced — and the first in the gene ther­a­py area, Garabe­di­an told End­points News.

Dri­ve Cap­i­tal al­so par­tic­i­pat­ed in the fund­ing. Be­cause Forge will serve as both a con­tract de­vel­op­ment and man­u­fac­tur­ing or­ga­ni­za­tion (CD­MO) and a pro­duc­er of its own pipeline, Garabe­di­an sees up­side here.

“That com­bi­na­tion CD­MO and pro­pri­etary pipeline is rare to find, that ca­pa­bil­i­ty in one shop,” Garabe­di­an, who will serve as Forge’s chair­man, said.

Own­ing and con­tract­ing out its man­u­fac­tur­ing fa­cil­i­ty, a 175,000-square-foot for­mer ware­house space dubbed “The Hearth” in Colum­bus, Ohio, will al­low Forge to cre­ate a net­work of clients en­com­pass­ing not on­ly biotechs but aca­d­e­m­ic in­sti­tu­tions and re­search foun­da­tions. There is huge de­mand for such spe­cial­ty sites in the gene ther­a­py field, and Miller said Forge has al­ready se­cured some po­ten­tial clients.

The high de­mand, com­bined with hav­ing the ca­pac­i­ty to de­vel­op AAVs on-site, makes for “a very com­pelling busi­ness mod­el,” Garabe­di­an said. Ear­li­er this year, Deer­field Man­age­ment in­vest­ed $1.1 bil­lion in a new CD­MO fa­cil­i­ty while two oth­er big play­ers, Catal­ent and Ther­mo Fish­er, had pre­vi­ous­ly spent over $1 bil­lion each on gene ther­a­py man­u­fac­tur­ers.

Forge’s chal­lenge in meet­ing that de­mand will be up­scal­ing its pro­duc­tion ca­pa­bil­i­ties, and the com­pa­ny plans to have ser­vices at the 500-liter scale by mid-2021, up from 50 liters cur­rent­ly.

“We’re us­ing what we con­sid­er to be a rel­a­tive­ly stan­dard way of ap­proach­ing us­ing a sus­pen­sion cul­ture, but with an ex­pe­ri­enced team who can take those cul­tures and scale those for the mass­es,” Miller said.

In terms of its own pipeline, Forge is ini­tial­ly re­search­ing an AAV in com­bi­na­tion with um­bil­i­cal cord bone mar­row (UCBM) trans­plants as a treat­ment for Krabbe dis­ease, a ge­net­ic dis­or­der that in many cas­es caus­es death by age 2. The pro­gram, la­beled FBX-101, is ex­pect­ed to be in ear­ly clin­i­cal tri­als by the end of 2020.

Miller specif­i­cal­ly men­tioned Philadel­phia-based Pas­sage Bio, whose Krabbe dis­ease treat­ment will hit Phase I/II clin­i­cal tri­als in the first half of 2021, as a po­ten­tial com­peti­tor. While Pas­sage Bio fo­cus­es sole­ly on AAV de­liv­ery, Forge aims to prove that its UCBM and AAV com­bo will ul­ti­mate­ly be safer and ef­fi­ca­cious.

“What this [com­bi­na­tion] does is it gives the pa­tients a re­set, or a new im­mune sys­tem,” Miller said, “so they don’t have an im­mune re­sponse against the pro­tein that would be ex­pressed dur­ing this gene ther­a­py, and it sup­press­es any im­mune re­sponse to the ac­tu­al AAV.”

Of course, Krabbe dis­ease treat­ments are just the first in what Forge hopes is a long fu­ture of ser­vic­ing and de­vel­op­ing gene ther­a­py. The biotech has two oth­er in-house projects it’s cur­rent­ly work­ing on, one of which is re­lat­ed to on­col­o­gy.

Look­ing for­ward, Forge in­tends to be a “de­vel­op­ment en­gine” that us­es Garabe­di­an’s seed fund back­ground with Miller’s vast net­work to ser­vice ear­ly-stage man­u­fac­tur­ing while al­so tak­ing on new projects it­self.

“Be­tween the two of us, we’re pret­ty plugged in to the emerg­ing gene ther­a­py re­search projects,” Garabe­di­an said.

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?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,800+ biopharma pros reading Endpoints daily — and it's free.

Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.