In­ves­ti­ga­tors de­tail some of the hopes – and fears – be­hind Cel­lec­tis' off-the-shelf CAR-T

Cel­lec­tis is right at the cut­ting edge of de­vel­op­ing a new, off-the-shelf CAR-T that hopes to dis­rupt a mar­ket that has yet to be cre­at­ed. So far, about the on­ly tan­gi­ble hu­man ev­i­dence of its po­ten­tial dura­bil­i­ty has been seen in a pair of in­fants who were giv­en Cel­lec­tis’ $CLLS lead ther­a­py to ward off lethal cas­es of leukemia. And while an up­date on their con­di­tion shows that the two young pa­tients are still alive and do­ing well months af­ter treat­ment, one had to fight off graft-vs-host dis­ease trig­gered by the ther­a­peu­tic, a key sign of tox­i­c­i­ty that in­ves­ti­ga­tors are watch­ing close­ly.

Writ­ing in Sci­ence Trans­la­tion­al Med­i­cine, the in­ves­ti­ga­tors han­dling these two cas­es of­fered de­tails on their pi­o­neer­ing cas­es, us­ing TAL­EN en­gi­neered cells to di­rect­ly tar­get B cell leukemia. Both chil­dren had re­lapsed, drug-re­sis­tant cas­es of B cell acute lym­phoblas­tic leukemia.

In the first case, the in­ves­ti­ga­tors were al­lowed to use Cel­lec­tis’ off-the-shelf treat­ment on an 11-month-old child. Grade 2 skin GVHD de­vel­oped but was suc­cess­ful­ly treat­ed with steroids, and 18 months lat­er the in­fant re­mains in re­mis­sion.

An­oth­er ba­by girl was treat­ed in much the same way af­ter re­laps­ing in late 2015 and re­spond­ed well, with­out GVHD. An ag­gres­sive ther­a­peu­tic in­ter­ven­tion leaves her “clin­i­cal­ly well at home.”

The sur­vival times for these girls is not new. The da­ta were first dis­cussed at a sci­en­tif­ic meet­ing in De­cem­ber, along with oth­er cas­es in­volv­ing short­er pe­ri­ods of post-treat­ment sur­vival times. (Dura­bil­i­ty will be a key fo­cus.) But this new re­port does pro­vide more in­sights in­to the ther­a­py, un­der­scor­ing Cel­lec­tis’ ra­tio­nale that the lead CAR-Ts now in late-stage de­vel­op­ment – which ex­tract T cells from pa­tients, en­gi­neer them and then in­fuse them back in­to pa­tients – leave a lot to be de­sired. In some cas­es, the in­ves­ti­ga­tors note, pa­tients aren’t go­ing to have enough T cells left to reengi­neer in­to ther­a­pies, mak­ing an off-the-shelf al­ter­na­tive us­ing cells from healthy donors es­sen­tial.

The threat of GVHD, though, once again spot­lights the se­vere ad­verse events that have been tied to these ther­a­pies. Juno’s $JUNO CAR-Ts have been tied to sev­en deaths from cere­bral ede­ma, in­clud­ing a tal­ly of five for their lead pro­gram, which is now in lim­bo. Kite $KITE and No­var­tis $NVS, mean­while, are rac­ing to win the first ap­provals in the field.

Cel­lec­tis isn’t in the frank ranks of that race. But it’s an­gling to come in not far be­hind, if it can es­cape a cat­a­stro­phe.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.