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.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Eu­ro­pean Par­lia­ment calls mem­ber states to ac­tion on an­timi­cro­bial re­sis­tance

Members of the European Parliament have called on EU countries to develop national action plans against antimicrobial resistance (AMR), calling it a top-three priority health threat.

Parliament on Thursday announced recommendations for the fight against AMR, including national action plans that must be updated at least every two years, an EU-level database tracking AMR and antimicrobial use and increased partnership between the pharma industry, patient groups and academia.

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Andrey Zarur, GreenLight Biosciences CEO

Green­Light Bio­sciences se­cures merg­er as it looks to go pri­vate

GreenLight Bioscience, the developer and manufacturer of RNA vaccines and therapeutics, is set to be acquired.

The company announced earlier this week that it would be acquired by a group of buyers led by Fall Line Capital in a cash deal valuing GreenLight at around $45.5 million. According to a release, Fall Line and the group agreed to acquire all of the shares of the company for $0.30 per share. The deal is expected to close sometime in the third quarter of this year.

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Roche plans to di­vest from lega­cy Genen­tech man­u­fac­tur­ing fa­cil­i­ty in Cal­i­for­nia

Roche is planning to make some changes to its subsidiary’s manufacturing network in California.

The Swiss pharma announced Wednesday that it plans to divest from Genentech’s manufacturing facility in Vacaville, CA, around 58 miles northeast of San Francisco. According to a statement from Roche, the move is part of a “broader strategy” to bring its manufacturing capabilities in line with its future pipeline. Roche is starting the process of finding a buyer for the site but has not named any candidates yet.

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FDA lifts hold on Mol­e­c­u­lar Tem­plates’ mul­ti­ple myelo­ma tri­al af­ter less than two months

The FDA has lifted a partial clinical hold on Molecular Templates’ early-stage trial for a multiple myeloma drug, the biotech company announced Thursday morning.

Regulators had put the trial on partial hold in early April, pausing patient enrollment, following two adverse heart-related events in patients who received the highest dose of Molecular Templates’ treatment MT-0169 last year. One patient had asymptomatic grade 2 myocarditis, or heart muscle inflammation, while the other had a grade 3 cardiomyopathy. Both recovered within two months.

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Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Who's con­fi­dent­ly in­vest­ing in biotech star­tups dur­ing these tense days? We've got some an­swers

We’ve got a changeup to our event schedule in Boston next week, where we’ll be doing a mix of live/streaming events at our base at The Seaport Hotel as part of a two-day lineup of webinars, virtual firesides and a cocktail hour Q&A with a veteran of the biotech financing scene.

The 9:30-10:30 am ET live slot on Tuesday, June 6, will now feature a panel conversation on the current state of affairs for VC investing in biotech, focusing on what startups are getting cash — and how. Alaa Halawa, head of US ventures at Mubadala, is confirmed, along with Brian Goodman at MPM and Geoff von Maltzahn, a general partner at Moderna-buoyed Flagship. I have a couple of other invites out and will let you know how that plays out.

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As­traZeneca gives up on late-stage IL-23 drug due to tri­al de­lays, com­pet­i­tive land­scape

AstraZeneca is shelving an IL-23 antibody that’s been through a winding journey around pharma — including stops at Amgen and Allergan — and putting an end to Phase II and III trials testing the drug for inflammatory bowel disease.

“The decision to discontinue brazikumab’s IBD development follows a recent review of brazikumab’s development timeline and the context of a competitive landscape that has continued to evolve,” a press release reads.

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