Op-ed: What we’re watch­ing close­ly as an un­usu­al #ASH21 kicks off in At­lanta

Re­mem­ber when med­ical meet­ings were live and in per­son?

Kyle Blanken­ship

Step­ping off the plane from Austin in­to this year’s Amer­i­can So­ci­ety of Hema­tol­ogy an­nu­al meet­ing in At­lanta, I can’t help but feel some of the ex­cite­ment and sense of in­tel­lec­tu­al fer­ment we used to feel around the biggest con­fer­ences of the year — but it’s un­de­ni­able this year is dif­fer­ent.

No longer re­strict­ed to a wonky In­ter­net plat­form, #ASH21 has adopt­ed a live-vir­tu­al hy­brid mod­el, and I em­braced the chance to re­turn to my first live meet­ing in rough­ly two years — vaxxed, boost­ed and masked, of course. It’s good to be back, but I, like the rest of the world, am still ad­just­ing to the new nor­mal.

Where be­fore my week­end might be packed with side­bars with bio­phar­ma ex­ec­u­tives shared over luke­warm cups of cof­fee from the me­dia room, quick meet-and-greets with fel­low journos and PR flacks, and wolf­ing down a sand­wich from the buf­fet bar, this year’s event al­ready feels more mut­ed and a lit­tle more somber.

The cof­fee and sand­wich­es still flow freely, and a cou­ple of folks are still brav­ing a short sit­down and in­tro­duc­tion, but most of my calls have al­ready hap­pened this week on the now-fa­mil­iar con­fines of Zoom and Mi­crosoft Teams. So where some of the ex­cite­ment is gone, this meet­ing has al­so giv­en me some time ahead to re­flect on what is still a fas­ci­nat­ing time in hema­tol­ogy.

In con­ver­sa­tion with more than a dozen bio­phar­ma ex­ecs and re­searchers, I’ve com­piled a bit of a self-guide to some of the biggest trends and ab­stracts ex­pect­ed at this year’s #ASH21. Many of the biggest names in this space are cer­tain­ly throw­ing their weight around, but there are still a few un­der­told sto­ries float­ing around out there that are worth watch­ing.

If you’re com­fort­able at home in your PJs or cram­ming your­self in­to sud­den­ly too-small work clothes in At­lanta (pag­ing: me), I hope you en­joy my list of what I’m watch­ing this week­end.

As cur­rent-gen CAR-T scours for more pa­tients, a spate of next-gen drugs vie for a break­through

Ear­li­er to­day, Bris­tol My­ers Squibb and Gilead’s Kite read out com­pet­ing da­ta for their cur­rent-gen CAR-Ts in sec­ond-line B cell lym­phoma as head-to-head chal­lenges to an ug­ly stan­dard of care.

Those read­outs are big news in that they rep­re­sent a big ad­vance for the cur­rent gen­er­a­tion of CAR-Ts — with oner­ous safe­ty pro­files and the weight of the un­known on their shoul­ders — look­ing to break new ground in ear­li­er lines of ther­a­py. And this isn’t the end for those am­bi­tions, ei­ther. Kite is al­so pre­sent­ing more da­ta for Yescar­ta in first-line, high-risk LB­CL pa­tients on Mon­day, which will be some­thing to watch not on­ly for in­ter­me­di­ate ef­fi­ca­cy mark­ers but al­so whether physi­cians will be able to stom­ach a cell ther­a­py so ear­ly in the treat­ment par­a­digm.

That bat­tle may come down to safe­ty, where Bris­tol My­ers’ Breyanzi ap­pears to show a safer pro­file over Kite’s ri­val. Time will tell on whether that gap holds up.

But as the es­tab­lished CAR-Ts duke it out over what could be a siz­able slice of pa­tients and sales, a grow­ing menagerie of biotechs and phar­mas look­ing for break­throughs in next-gen cell ther­a­py for can­cer are al­so loud­ly shop­ping their wares this week­end.

This list runs long and in­cludes some drug­mak­ers that have re­cent­ly made waves. The field has shown some ear­ly ef­fi­ca­cy promise, but con­cerns over dura­bil­i­ty and a cou­ple sur­pris­es in the clin­ic have churned out mixed re­sults, so far. Take Al­lo­gene, for in­stance.

One of the pre­mier names in craft­ing al­lo­gene­ic — or “off-the-shelf” — cell ther­a­pies de­rived from donor cells rather than a pa­tient’s own, Al­lo­gene was re­cent­ly hit with a clin­i­cal hold for its AL­LO-501 pro­gram af­ter some pa­tients re­port­ed chro­mo­so­mal ab­nor­mal­i­ties. The com­pa­ny has an up­date on those Phase I tri­als Mon­day so at least we’ll know whether they were on the right track.

Mean­while, you have a com­pa­ny like Fate, which is read­ing out da­ta on its CAR-NK pro­gram FT596 — ef­fec­tive­ly strap­ping a CAR on­to nat­ur­al-killer cells de­rived from donor stem cells. Ear­li­er this year, Fate turned out da­ta ques­tion­ing the dura­bil­i­ty of that drug de­spite some promis­ing ef­fi­ca­cy num­bers.

All of these sto­ries are part of a greater push in­to the fu­ture of cell ther­a­py — par­tic­u­lar­ly drugs that lever­age the in­nate im­mune sys­tem. It will be in­ter­est­ing to watch how that de­vel­ops.

New an­swers in he­mo­phil­ia

An ab­stract to watch close­ly on Tues­day is Sanofi’s up­date on fi­tusir­an, an siR­NA ther­a­py that si­lences the SER­PINC1 gene lead­ing to he­mo­phil­ia A/B, could prove a game chang­er in treat­ing that hema­to­log­i­cal dis­ease.

Ear­ly da­ta in the Phase III set­ting from this pro­gram showed a whop­ping 90% re­duc­tion in an­nu­al­ize bleed­ing rate, which would like­ly prove to be best-in-class ef­fi­ca­cy if it holds up. Mean­while, the safe­ty pro­file for the drug has shown some hur­dles, par­tic­u­lar­ly on the clot­ting front.

Mean­while, there are a range of po­ten­tial gene ther­a­pies in the works for this pro­gram. Just this week, uniQure read out piv­otal da­ta for its gene ther­a­py en­tranaco­gene deza­parvovec hit­ting its pri­ma­ry non-in­fe­ri­or­i­ty end­point in an­nu­al­ized bleed­ing rate (ABR) af­ter 18 months com­pared to base­line Fac­tor IX pro­phy­lac­tic ther­a­py. Fac­tor IX is a pro­tein that’s nat­u­ral­ly pro­duced in the body to help form blood clots and stop bleed­ing, and com­mon treat­ments to­day are de­signed to re­place the pro­tein to achieve ad­e­quate clot­ting.

It’s worth not­ing that ASH isn’t just a blood can­cer con­fer­ence but a hema­tol­ogy con­fer­ence as well, and this race will prove one to watch.

CD19/20 still dom­i­nates blood can­cer drug de­vel­op­ment, but new tar­gets are catch­ing eye­balls

An­oth­er big late-break­ing ab­stract on Tues­day is an up­date on Roche’s Po­livy, an an­ti­body-drug con­ju­gate tar­get­ing the CD79b pro­tein.

Po­livy is one of a rare group of blood can­cer meds ap­proved to tar­get any­thing but CD19 or CD20, two of the most high­ly val­i­dat­ed tar­gets in can­cer care that have been the back­bone for a range of ap­provals for decades. Roche, with a his­to­ry dri­ving three of the in­dus­try’s most suc­cess­ful can­cer drugs in Avastin, Her­ceptin and Rit­ux­an, knows that CD19/20 will play a big role in the fu­ture but is al­so will­ing to dab­ble in some in­ter­est­ing ex­per­i­ments.

One to watch is an up­date from Roche on com­bi­na­tions of Po­livy with its in­ves­ti­ga­tion­al CD20xCD3 bis­pe­cif­ic an­ti­bod­ies mo­sune­tuzum­ab and glofitimab. There are lots of com­pet­ing bis­pecifics work­ing off those tar­gets, and Roche is get­ting ag­gres­sive in pur­su­ing com­bi­na­tions to get ahead.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

AS­CO ab­stracts mint some ear­ly win­ners, but Io­vance, Spring­Works get hit by mas­sive loss­es

Before cancer-focused biotechs start their trek to the first in-person American Society of Clinical Oncology (ASCO) annual conference since the pandemic began, investors have taken a good look at the teasers for the data scheduled to be presented — and started placing bets.

With its power to confer overnight fame, ASCO is a stage where impressive or surprise debut performances can go a long way. On the other hand, disappointing details could be punishing.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Levi Garraway, Roche CMO (Genentech)

Roche's CD20xCD3 does­n't beat Gen­mab at ORR, but sets bar for CR da­ta on lym­phoma drug

On its way to potentially becoming the first to market with a CD20xCD3 bispecific for aggressive lymphoma, Big Pharma’s largest R&D spender has some more data to pad its case with an FDA filing slated for later this year.

Roche dropped some more details from a Phase II expansion study of its fixed-duration glofitamab, to be presented at next week’s ASCO annual meeting, in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). The patients had received a median of three prior therapies.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.