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. 

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.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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+ 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.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,000+ biopharma pros reading Endpoints daily — and it's free.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.