Win­ners and losers at #EHA18: Shares most­ly slide as da­ta from a range of promi­nent biotechs fail to wow in­vestors

News is pour­ing out of Stock­holm, Swe­den Fri­day morn­ing as the an­nu­al meet­ing for the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion gets ful­ly un­der­way. The con­fer­ence, which at­tracts some­thing like 10,000 hema­tol­o­gists, is on its sec­ond day — and many of this year’s news­mak­ers are work­ing in the com­pet­i­tive field of blood can­cers.

Blue­bird ar­rived with their lat­est in­cre­men­tal up­date on Lenti­Glo­bin, with da­ta from sev­er­al stud­ies, but it couldn’t hold on to ear­ly gains to­day as the crowd of on­look­ers played tough with the biotech shares on the line to­day. You can see the full sto­ry on blue­bird here; the sum­ma­ry of the rest lies be­low:

Glob­al Blood Ther­a­peu­tics — ↓ 19%

Glob­al Blood Ther­a­peu­tics $GBT came in­to the EHA meet­ing look­ing to de-risk its up­com­ing Phase III study re­sults, but stum­bled bad­ly in com­par­i­son with blue­bird bio. Its shares skid­ded 19% low­er as in­vestors com­pared the biotech’s less fa­vor­able clin­i­cal im­pact of 900 mg dose of vox­elo­tor on sick­le cell dis­ease.

Ted Love

The key points: 43% of pa­tients (9 of 21) achieved a he­mo­glo­bin re­sponse >1 g/dL at 24 weeks with a me­di­an he­mo­glo­bin change from base­line of 0.7 g/dL. Re­duced dai­ly symp­toms at 24 weeks as as­sessed by to­tal symp­tom scores (TSS), which im­proved in 13 of 21 pa­tients. 

Their Phase III HOPE Study is eval­u­at­ing vox­elo­tor at 900 mg and 1500 mg per day. And re­searchers “con­tin­ue to ex­pect to an­nounce top-line clin­i­cal da­ta from Part A of the HOPE study by the end of this quar­ter,” says CEO Ted Love. 

That’s right around the cor­ner.

→ Epizyme — ↓ 5%

De­spite be­ing slapped with a clin­i­cal hold ear­li­er this year on its lead ther­a­py tazeme­to­stat, Epizyme $EPZM is trot­ting out up­dat­ed da­ta on the drug that might give in­vestors hope. The da­ta are from a Phase II study test­ing tazeme­to­stat against fol­lic­u­lar lym­phoma with wild-type or mu­tant EZH2 genes. In­vestors were hop­ing to see the drug show con­tin­ued re­sponse rates, and Roth an­a­lyst Jotin Marango said to look for re­sponse rates above 70%.

And the Cam­bridge, Mass­a­chu­setts-based com­pa­ny came through — at least par­tial­ly. The EZH2 mu­tant group had a 71% over­all re­sponse rate, with 11% achiev­ing a com­plete re­sponse and 61% hav­ing a par­tial re­sponse. None of the pa­tients’ dis­ease pro­gressed and their me­di­an du­ra­tion of re­sponse was 32 weeks. But it’s im­por­tant to note that the clin­i­cal tri­al hold pre­vent­ed Epizyme from re­cruit­ing pa­tients to the mu­tant group, and that set of pa­tients start­ed treat­ment lat­er than the wild-type group.

The wild-type group had a low­er ORR of 33%, with 6% and 28% hav­ing com­plete and par­tial re­spons­es re­spec­tive­ly. The me­di­an du­ra­tion of re­sponse was 76 weeks.

Af­fimed — ↓ 14%

Af­fimed $AFMD had one of the worst morn­ings for EHA, with its shares tum­bling more than 15% on its re­lease of ear­ly-stage da­ta on AFM13, its lead NK cell en­gager can­di­date.

Re­searchers are pur­su­ing stud­ies us­ing a com­bi­na­tion of Af­fimed’s drug with Keytru­da in Hodgkin lym­phoma af­ter the fail­ure of Bren­tux­imab Ve­dotin. And they fo­cused in on the top dose re­sponse, where they had an ORR of 89%, hit­ting the mark with 16 of 18 pa­tients.

Stephen Ansell, prin­ci­pal in­ves­ti­ga­tor of the study, not­ed: 

“Im­por­tant­ly, these da­ta have shown that AFM13 can be safe­ly ad­min­is­tered in com­bi­na­tion with Keytru­da and has the po­ten­tial to im­prove pa­tient out­comes.”

It wasn’t enough to win over the skep­tics, though.

Karyopharm — 2%

Karyopharm $KP­TI shares edged up just 3.6% this morn­ing, but af­ter the big spike from six weeks ago on mid-stage da­ta for se­linex­or, not los­ing ground could be con­sid­ered a step for­ward. 

In pa­tients whose mul­ti­ple myelo­ma was pro­tea­some in­hibitor (PI) naïve, there was a me­di­an pro­gres­sion-free sur­vival time of 17.8 months for the com­bo arm, which added dex and Vel­cade. The ORR was 84%. In PI re­frac­to­ry pa­tients, those num­bers dropped to 6.1 months and 43%.

Sharon Shacham

Karyopharm CSO Sharon Shacham not­ed: 

“Based on the pos­i­tive STOMP re­sults re­port­ed to date, we have ini­ti­at­ed a new all-oral STOMP arm to in­ves­ti­gate se­linex­or plus Revlim­id and dex in the front-line set­ting.  Giv­en the ob­served syn­er­gis­tic ac­tiv­i­ty of se­linex­or with stan­dard ap­proved myelo­ma ther­a­pies, we be­lieve oral se­linex­or has the po­ten­tial to be a fu­ture back­bone ther­a­py in myelo­ma, and we look for­ward to elu­ci­dat­ing its ac­tiv­i­ty as part of a front-line treat­ment reg­i­men.”

BeiGene — ↑ 0.2%

BeiGene $BGNE says it will be ready to file for an ap­proval of zanubru­ti­nib in Chi­na lat­er this year as they of­fer up an ear­ly snap­shot of da­ta for Walden­ström macroglob­u­line­mia. Re­searchers say that 43% of the evalu­able pa­tients achieved a very good par­tial re­sponse. And they added da­ta from the 86-pa­tient sin­gle-arm piv­otal Phase II study of zanubru­ti­nib in Chi­nese pa­tients with re­lapsed or re­frac­to­ry man­tle cell lym­phoma, with an over­all re­sponse rate (ORR) of 84 per­cent — and a 59% com­plete re­sponse rate. A US ap­pli­ca­tion is ex­pect­ed next year. Its stock was in the green this morn­ing, but just bare­ly.

→ Blue­print Med­i­cines — ↓ 5%

Six months af­ter pre­sent­ing some promis­ing Phase I da­ta at ASH, Blue­print Med­i­cines $BPMC rolled out up­dat­ed re­sults from its on­go­ing study of avapri­tinib. The drug, for­mer­ly coined as BLU-285, is be­ing test­ed as a treat­ment for sys­temic mas­to­cys­to­sis. Back in De­cem­ber, the com­pa­ny pre­sent­ed an over­all re­sponse rate of 72%, which rock­et­ed the com­pa­ny’s shares up 26% at the time.

In the up­dat­ed look pre­sent­ed to­day at EHA, Blue­print an­nounced an ORR of 83%. That be­ing said, 21% of pa­tients ap­pear to have dropped out of the tri­al through­out its 22-month course. The com­pa­ny plans to launch an open-la­bel, sin­gle-arm Phase II tri­al in the in­di­ca­tion in mid-2018.

→ Ar­Qule — ↓ 3%

Bri­an Schwartz

Back to back late-stage fail­ures had ham­mered Ar­Qule’s stock prices $AR­QL last year, and the very pre­lim­i­nary hu­man da­ta on its BTK in­hibitor com­ing out to­day are not help­ing.

The Phase I dose es­ca­la­tion study of ARQ 531 was con­duct­ed among a group of 11 pa­tients rep­re­sent­ing re­lapsed or re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia (CLL), small lym­pho­cyt­ic leukemia (SLL), Walden­strom’s macro­glo­bine­mia and B-cell Non-Hodgkin lym­phoma. Ar­Qule re­ports “an­ti-tu­mor ac­tiv­i­ty at all dose lev­els” — 5mg, 10mg, and 15mg — and plans to con­tin­ue push­ing the dose lim­it since no rel­e­vant tox­i­c­i­ties or drug-re­lat­ed se­vere ad­verse events were ob­served. On­ly four of the 11, how­ev­er, are still on the treat­ment, with oth­ers drop­ping out ei­ther be­cause of pro­gres­sion, physi­cian’s de­ci­sion or un­avail­able da­ta.

The ob­served tu­mor re­duc­tion rates came in at 35% for 5mg, 33% for 10mg, and 29% for 15mg dos­es, call­ing the dose-re­sponse re­la­tion­ship in­to ques­tion. The 29% re­duc­tion was ob­served in a 69 years old pa­tient with CLL/SLL with BTK C481S mu­ta­tion — who had re­ceived five pri­or sys­temic reg­i­mens — af­ter eight weeks.

Mean­while Bri­an Schwartz, CMO and head of R&D, main­tains that the re­sults show the drug’s po­ten­tial to be­come an op­tion for pa­tients with B-cell ma­lig­nan­cies, es­pe­cial­ly those with C481S-me­di­at­ed re­sis­tance to ir­re­versible BTK in­hibitors. The bio­mark­er ap­proach is a hall­mark of Ar­Qule de­vel­op­ment pro­grams, though it has not worked well so far, with dis­as­trous Phase III flops in 2012, 2013 and 2017. 

Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

Af­ter a run of CT­LA-4 com­bo fail­ures, sci­en­tists spot­light a way to make it work — in se­lect pa­tients

CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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