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. 

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.