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. 

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.