Let the joust­ing be­gin: Which top bio­phar­ma com­pa­nies are look­ing to make a splash at ASH?

Any­one who fol­lows on­col­o­gy knows the field has at­tract­ed so much at­ten­tion and re­search mon­ey that it sparks a con­tin­u­al erup­tion of bio­phar­ma news as com­pa­nies joust over block­buster mar­kets. That’s kept me busy this year with a string of glob­al con­fabs — as AS­CO be­comes just one big event in a fren­zied com­pe­ti­tion.

John Car­roll

So now I’ll be at­tend­ing ASH in At­lanta in ear­ly De­cem­ber for the first time. What’s on my radar? I turned to Leerink to­day for some sug­ges­tions, and boiled them down here.

Ap­provals for No­var­tis $NVS and Gilead/Kite $GILD for their first CAR-T ap­pli­ca­tions are a his­toric first step, but this tech­nol­o­gy is just now be­gin­ning to write its first com­mer­cial chap­ter. Juno $JUNO is now back af­ter get­ting de­railed with the dis­as­ter that JCAR015 be­came. And in fig­ur­ing out what went wrong there, they are promis­ing to come up with the next big thing in CAR-T: JCAR017. The ther­a­py has pro­duced some jaw-drop­ping ef­fi­ca­cy re­sults and a much im­proved safe­ty pro­file, and now we all want to see every up­date to de­ter­mine if Juno can leapfrog the lead­ers.

No­var­tis, the big phar­ma com­pa­ny that fig­ured out how to work like a biotech on this one, will be of­fer­ing some im­por­tant de­tails on Kym­ri­ah for DL­B­CL, where it’s look­ing to go head-to-head with Gilead’s Yescar­ta. So the up­date on that front from the Gilead/Kite team will earn a close in­spec­tion.

Then there’s Pfiz­er $PFE, an­oth­er of the Big Phar­ma play­ers that has made on­col­o­gy a top pri­or­i­ty. The gi­ant bagged rights to Cel­lec­tis’ $CLLS UCART19 in ALL, and a pre­sen­ta­tion in At­lanta will be close­ly fol­lowed. Cel­lec­tis’ re­cent clin­i­cal hold for its own off-the-shelf ap­proach to CAR-T — an­oth­er po­ten­tial leapfrog ther­a­py — will keep the Paris-based biotech in the in­dus­try’s eye.

Blue­bird bio $BLUE is seem­ing­ly al­ways at ASH and al­ways has some­thing in­ter­est­ing to say. In this case it’s the “Lenti­Glo­bin gene ther­a­py pro­gram in trans­fu­sion-de­pen­dent be­ta-tha­lassemia and se­vere sick­le cell dis­ease, as well as up­dat­ed re­sults of the bb2121 an­ti- BC­MA CAR-T Phase I study in pa­tients with re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma,” to quote Leerink.

Blue­bird, by the way, just an­nounced that it has dosed the first pa­tient in its North­star-3 (HGB-212) tri­al, a Phase III study of Lenti­Glo­bin.

Cel­gene $CELG has had its ups and downs this year, with some stun­ning and un­char­ac­ter­is­tic set­backs that have trig­gered a small rout on their stock, ding­ing a large swath of big biotechs in the process. A good show­ing in At­lanta would go a long way to earn­ing back some of the re­spect it’s long en­joyed. Leerink’s as­sess­ment of what to watch for on their close­ly watched ap­pear­ance at ASH:

(O)ral pre­sen­ta­tions from the bb2121 Phase I study in mul­ti­ple myelo­ma, and from the phase I TRAN­SCEND study of JCAR017 in NHL. CELG pre­pares to ini­ti­ate a piv­otal Phase III tri­al with bb2121 in myelo­ma in the com­ing months. CELG al­so will re­port sev­er­al sets of Phase I da­ta from next-gen CelMod CC-122 in NHL in­di­ca­tions.

I’m al­so pluck­ing Alex­ion $ALXN out of the mix. Their next-gen PNH drug is about the on­ly im­por­tant pro­gram they have in the pipeline, for now, and the ear­ly-stage snap­shot on ALXN1210 will get some at­ten­tion as well.

This next part is web on­ly. I’m round­ing out my list now with sug­ges­tions com­ing in over e-mail along with some sug­ges­tions via an­a­lysts re­marks. 

Spark $ONCE has one of those land­mark drugs we all like to watch in Lux­tur­na, and an­a­lysts at Jef­feries say they’ll be on hand to make sure that the biotech demon­strates con­sis­tent­ly pos­i­tive da­ta on the lead­ing drug in the gene ther­a­py field. “At ASH, we ex­pect SPK-8011 to show gen­er­al con­sis­ten­cy of 11-14% and 20-25%, with the third co­hort ide­al­ly show­ing 50-100% high­er lev­els than the pri­or co­hort,” says an up­beat Michael Yee.

I al­so plan to talk to Ax­el Hoos at GSK about their ear­ly-stage BC­MA-tar­get­ing ther­a­py, an an­ti­body drug con­ju­gate dubbed GSK2857916. Just a few days ago GSK flagged the FDA’s break­through drug des­ig­na­tion, which will like­ly keep it on fast-track de­vel­op­ment. And the phar­ma gi­ant is like­ly to use this pro­gram to high­light a re­turn to late-stage de­vel­op­ment in on­col­o­gy.

Al­so adding Re­gen­eron, which has Phase I tri­als of their an­ti-PD-1 cemi­plimab and CD20xCD3 bis­pe­cif­ic REGN1979 in b-cell lym­phomas, specif­i­cal­ly B-cell non-Hodgkin lym­phoma.

There’s al­so Gami­da Cell, which will have the full Phase II da­ta avail­able on NiCord, now in late-stage de­vel­op­ment.

That’s it for now. These are on­ly the high­lights I’m ze­ro­ing in on. But if you have sug­ges­tions for any­thing im­por­tant I missed, let me know. You can email me at john@end­pointsnews.com.


Im­age: The Geor­gia World Con­gress Cen­ter in At­lanta Shut­ter­stock

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

Social image: UCB