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. 

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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