Adap­ti­m­mune gets a par­tial hold on piv­otal T-cell study, but it seems OK to breathe now

The FDA is tap­ping the brakes on one of Adap­ti­m­mune T-cell stud­ies, look­ing for some clar­i­fi­ca­tions be­fore it gives the green light to start test­ing NY-ESO SPEAR T-cell ther­a­py in myx­oid round cell li­posar­co­ma.

No doubt there were a few skipped heart beats among in­vestors, but the UK biotech $ADAP hur­ried on to ex­plain that reg­u­la­tors are sim­ply look­ing to get a few an­swers for a reg­is­tra­tion tri­al that has yet to start re­cruit­ing pa­tients.

Im­muno-on­col­o­gy has ex­plod­ed in re­cent years, in a good way. New play­ers like Adap­ti­m­mune have at­tract­ed bil­lions in in­vest­ment cash. But as Juno demon­strat­ed re­cent­ly with its lead CAR-T study, a sud­den halt fol­low­ing the death of pa­tients can raise ques­tions about the en­tire field.

It’s im­por­tant to note, though, that the FDA lift­ed its hold on Juno just days af­ter im­pos­ing a clin­i­cal hold on the study, sat­is­fied that the biotech had an­swered its ques­tions about safe­guard­ing pa­tients from any fur­ther un­nec­es­sary risk. And Bank of Amer­i­ca an­a­lysts note that this pause is not tied to the use of flu­dara­bine, which Juno says was re­spon­si­ble for killing sev­er­al pa­tients.

“We spoke with man­age­ment who re­it­er­at­ed that the in­for­ma­tion re­quest­ed by FDA is clar­i­fi­ca­tion of de­f­i­n­i­tions rather than re­lat­ed to flu­dara­bine use or CMC man­u­fac­tur­ing is­sues that have plagued oth­er T cell ther­a­pies. ADAP plans to re­spond to the FDA short­ly and ex­pects the FDA to take 30 days to re­view their re­spons­es, re­sult­ing in a de­lay in the tri­al time­line.”

What­ev­er the case, Adap­ti­m­mune shares slipped 4% in af­ter mar­ket trad­ing as some ner­vous in­vestors fret­ted.

James No­ble, Adap­ti­m­mune CEO

“Adap­ti­m­mune is run­ning a num­ber of dif­fer­ent stud­ies with its NY-ESO pro­gram and con­tin­ues to en­roll pa­tients in syn­ovial sar­co­ma, ovar­i­an, and lung can­cer tri­als in the U.S.,” said James No­ble, Adap­ti­m­mune CEO, in a state­ment. “We have been in di­a­logue with the FDA since achiev­ing break­through sta­tus ear­li­er this year and this par­tial clin­i­cal hold re­quires a num­ber of ques­tions to be an­swered be­fore we can start a new MR­CLS tri­al in­tend­ed to be used for reg­is­tra­tion pur­pos­es. We will be pro­vid­ing a full re­sponse to the FDA short­ly and will up­date the mar­kets when we have fur­ther news to re­port.”

Adap­ti­m­mune’s lead pro­gram is a SPEAR T-cell ther­a­py tar­get­ing the NY-ESO can­cer anti­gen. The com­pa­ny is a spe­cial­ist in TCR tech.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

UP­DAT­ED: Bio­gen push­es in a fresh stack of chips and starts prep­ping a glob­al R&D game plan af­ter watch­ing the cards turn on ear­ly throm­bolyt­ic da­ta

After patiently steering through a decade-long journey for its early-stage clinical work, a small Tokyo biotech has clinched a deal to out-license its lead thrombolytic agent to US heavyweight Biogen — which sees a potentially game-changing impact on the clot-busting field after taking a careful look at some upbeat Phase IIa data.

Three years after Biogen anted up $4 million to gain an option on the drug from TMS, the big US biotech is making a small bet to beef up its stroke portfolio. The BD team inked a deal to go ahead and grab rights to the drug for $18 million, with another $335 million in milestone cash on the table for a successful outcome.

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Distribution of Moderna's Covid-19 Vaccine (Photo by Paul Sancya - Pool/Getty Images)

Opin­ion: Ado­les­cents can wait. The US needs to start do­nat­ing Covid-19 vac­cines to needy coun­tries now.

Now that the US is swimming in Covid-19 vaccines and the supply has officially eclipsed the demand, it’s time for America to lead the world and start shipping these excess doses to countries that desperately need them.

Unlike the IP waiver at the World Trade Organization, which Biden now supports and will likely take years to translate into actual shots in arms, the US could easily donate just a tiny fraction of the more than 60 million doses of Pfizer, Moderna and J&J vaccines sitting on American shelves right now.