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.

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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The Strand team (L-R): COO Samta Kundu, SVP of R&D Prashant Nambiar, CEO Jacob Becraft, and president/head of R&D Tasuku Kitada

Lil­ly joins in ex­pand­ed Se­ries A round for BeiGene-part­nered mR­NA biotech

With the buzz around the Covid-19 mRNA vaccines wearing off, biotechs attempting to create the next generation of messenger RNA therapeutics are in full swing, and MIT-founded Strand Therapeutics has picked up $45 million more to do so.

The Boston-based drug developer added that amount to its original $52 million Series A, disclosed in June 2021, to get its first solid tumor mRNA candidate into human trials next year. The BeiGene-partnered startup gathered the Series A1 funds from Eli Lilly, FPV, Potentum Partners, existing backer Playground Global and an “unannounced syndicate.”

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In­dus­try groups, CVS pick apart FDA's pro­posed path­way for gener­ics to carve out OTC in­di­ca­tions

Pharma industry groups like the Association for Accessible Medicines (AAM) and PhRMA are raising pointed questions about an FDA plan to create a new pathway for marketing prescription drugs with an additional condition for nonprescription use (ACNU), which would require more safeguards than the current OTC pathway but essentially carve out new OTC uses for some generic drugs.

Chief among the concerns were: Insurance companies dropping coverage for the Rx version, new ACNU patents to block competition, and industry essentially governing the pathway.

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