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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Rahul Singhvi, Resilience CEO

A Bob Nelsen start­up turns to Har­vard to help sharp­en its tech, in­spir­ing first spin­out

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.

John Oyler, BeiGene CEO (Paul Yeung/Bloomberg via Getty Images)

Bris­tol My­ers wants to pull out of its Abrax­ane deal in Chi­na. BeiGene says no way

A year and a half after Chinese officials ordered BeiGene to stop selling Bristol Myers Squibb’s Abraxane in the wake of an alarming inspection of a US facility, the manufacturing issues at the root of the import suspension still appear unresolved.

And Bristol Myers wants to axe the Abraxane supply deal altogether.

But BeiGene, which is currently in arbitration proceedings against its Big Pharma partner, won’t let it off the hook so easily.

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Madhu Natarajan, Takeda rare disease development head

Drawn to the idea of turn­ing B cells in­to 'pro­tein fac­to­ries,' Take­da jumps in­to a mile­stone-heavy, $900M pact

Madhu Natarajan can trace his fascination with the idea of taking B cells and turning them into protein factories back 20 years, when he had his own lab at UT Southwestern. So when Natarajan, now the rare disease development head for Takeda, sat down for a meet-up with execs from Seattle-based Immusoft at the last in-person JP Morgan conference, they went straight into a brainstorming session.

“That B cells can take up residence and do what they do for a long time,” says Natarajan, pumping out proteins and “leveraging it into a therapeutic context,” hits his sweet spot for discovery deals. And he was deeply impressed by what he heard.

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