Eric Ostertag, Poseida CEO

Take­da en­lists Po­sei­da in $3.6B+ non­vi­ral gene ther­a­py deal, though da­ta re­main years away from hu­man stud­ies

Take­da has been be­hind oth­er ma­jor play­ers in terms of cell and gene ther­a­py de­vel­op­ment, but af­ter an­nounc­ing a col­lab­o­ra­tion with Se­lec­ta Bio­sciences last week, it’s now on a bit of a spend­ing spree.

The Big Phar­ma has re­cruit­ed Po­sei­da Ther­a­peu­tics to de­vel­op in vi­vo gene ther­a­pies in six dif­fer­ent in­di­ca­tions, the com­pa­nies an­nounced Tues­day, with Take­da re­tain­ing an op­tion to ask for two more. Po­sei­da will get a $45 mil­lion up­front pay­ment in the deal, $125 mil­lion in short-term pre­clin­i­cal mile­stones and up to $435 mil­lion in clin­i­cal mile­stones per pro­gram.

If all eight can­di­dates hit all their check­points, Take­da will be on the hook for more than $3.6 bil­lion. Each of the pro­grams will be di­rect­ed at the liv­er and hematopoi­et­ic stem cells, with he­mo­phil­ia A be­ing the first tar­get.

Though last week’s Se­lec­ta deal was much nar­row­er in scope, fo­cus­ing on tamp­ing down the im­mune re­sponse seen in AAV-based gene ther­a­pies, Take­da’s Tues­day agree­ment is a bet on the fu­ture of gene ther­a­py, Po­sei­da CEO Er­ic Os­tertag told End­points News. The biotech’s gene ther­a­py plat­form is cen­tered around a non­vi­ral ap­proach, which Os­tertag says can pro­vide a true cure for ge­net­ic dis­eases.

Po­sei­da ar­gues it is dif­fer­ent from ear­li­er gene ther­a­py at­tempts be­cause that non­vi­ral ap­proach is bet­ter suit­ed at in­te­grat­ing in­to a pa­tient’s genome. Os­tertag said the pro­grams in Tues­day’s deal will use its pro­pri­etary nanopar­ti­cle tech­nol­o­gy to ed­it tar­get­ed genes in vi­vo, re­sult­ing in high­er ex­pres­sion lev­els, and give Po­sei­da the op­por­tu­ni­ty to go af­ter pe­di­atric pa­tients.

It’s an ap­proach un­like those be­ing in­ves­ti­gat­ed by oth­er com­pa­nies that he says are pri­mar­i­ly treat­ing adults, though Os­tertag did not name names.

“I think there are some ap­proach­es that as­pire to be sin­gle treat­ment cures. We’ve ac­tu­al­ly proven it with nu­mer­ous an­i­mal mod­els,” Os­tertag told End­points. “That is unique.”

As Os­tertag points out, how­ev­er, Po­sei­da has yet to put any of its gene ther­a­pies in hu­mans and like­ly won’t do so for sev­er­al years. The ear­li­est gene ther­a­py slat­ed for IND-en­abling stud­ies is an OTC de­fi­cien­cy pro­gram not in­volved in Tues­day’s deal shoot­ing for 2023, and even that isn’t en­tire­ly cer­tain — Po­sei­da is still choos­ing be­tween two dif­fer­ent tech­nolo­gies.

The lack of in­te­gra­tion seen in AAV-based gene ther­a­py and some nanopar­ti­cles-based ap­proach­es are “par­tic­u­lar­ly a prob­lem when you have a rapid­ly di­vid­ing tis­sue,” Os­tertag said. “Tran­sient ap­proach­es are not go­ing to work at all in that [pe­di­atric] pa­tient pop­u­la­tion.”

He lat­er added, “To date, the oth­er gene ther­a­py com­pa­nies are re­al­ly on­ly ad­dress­ing those few pa­tients that had mild dis­ease that made it in­to adult­hood. We can ad­dress the pe­di­atric pop­u­la­tion.”

Po­sei­da has large­ly made noise for its CAR-T pro­grams, pro­vid­ing proof of ef­fi­ca­cy for a next-gen sol­id tu­mor CAR-T in a da­ta read­out at the end of Au­gust. The re­sults came about 10 months af­ter the FDA re­moved a clin­i­cal hold, spurred by a pa­tient death on its lead can­cer drug.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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Benjamin Oakes, Scribe Therapeutics CEO

CEO of Doud­na spin­out: With­in five years, genome ed­i­tors will have a 're­al­ly big im­pact' on pa­tients' lives

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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