Liquid Biopsy · @Liquid_Biopsy
9th Nov 2015 from TwitLonger
Wedbush: #ASH15 Abstract Roundup:
$BLUE $CLDX $EPZM $FATE $GBT $INFI $KPTI $MEIP $STML $XNCR
• In this report, we provide a brief roundup of abstracts provided by covered companies for the upcoming American
Society of Hematology (ASH) Annual Meeting (Dec. 5th – Dec. 8th) in Orlando, FL.
• BLUE will present three LentiGlobin abstracts, providing clinical updates for the HGB-204 Northstar study, HGB-205
study and HGB-206 study. In addition, BLUE will present three abstracts highlighting its immuno-oncology program,
including its lead candidate bb2121, and manufacturing capabilities. We have published a more extensive BLUE ASH abstract
review here
• Data from a small Ph 1 IST of CLDX’s NY-ESO-1 vaccine (CDX-1401) in combination with decitabine for
myelodysplastic syndrome (MDS) patients will be presented. Of the first 6 patients analyzed, five patients have
demonstrated NY-ESO-1 specific CD4+ T-cell responses, and 4 patients had NY-ESO-1 CD8+ T-cell responses following
vaccination. In addition, no unexpected toxicities were observed, with the most frequent adverse events related to decitabine.
CDX-1401 is currently being evaluated in a Ph 1/2 study of CLDX’s varlilumab in combination with Yervoy, with patients that
express NY-ESO-1 set to receive CDX-1401 in the Ph 2 portion of the study.
• EPZM will present data from the ongoing Ph 1 study of tazemetostat (EPZ-6438) in r/r NHL patients (#473), as well as
providing updates on its Ph 1 studies of pinometostat (EPZ-5676) in adult and children with MLL-r acute leukemia
(#2547, #3792, respectively). Data in the abstract for the tazemetostat is similar to that presented in June at ICML, in which
9 of 15 evaluable patients had demonstrated an objective response. Five of these evaluable patients were still on study at the
time of that data cut-off, with an additional non-GCB DLBCL patient who had not yet been assessed. Results from this study
provided support for the ongoing five-arm Ph 2 in r/r NHL patients, for which initial data is expected mid:16.
• FATE will present preclinical data supporting its HSC modulator ProTmune, including results from a mouse model of
GvHD, in which its mobilized peripheral blood candidate demonstrated a stat sig reduction in GvHD risk score and
improved survival vs vehicle (#1884). In addition, FATE will present two posters (#2364, #3237) on its proprietary induced
pluripotent stem cell (iPSC) platform, including a method for the scalable generation of hiPSC-derived T and NK cells for
cancer immunotherapy applications, and engineering strategies to efficiently, precisely and safely integrate multiple genetic
modifications into pluripotent cells.
• As anticipated, the ASH abstract for GBT-440 is a placeholder for the conference. We look to ASH for confirmation of
promising early efficacy of GBT440 as a disease modifier in sickle cell disease. In a small cohort of eight patients (6 on
drug, 2 on placebo), treated with 700mg GBT440 for 28 days, blood markers such as hemoglobin and red blood cell count
increased and hemolysis markers decreased for patients compared to placebo. Importantly, most patients are seeing a near
complete decrease in sickled cells, suggesting the drug is having its intended effect. We expect to see data in 24 additional
patients at ASH; we believe these data should confirm early observations and act as a catalyst for shares.
• Two IST combination studies of INFI’s duvelisib will be presented, including a Ph 1b IST of duvelisib in combination
with FCR younger patients with CLL (Abstract #4158), and an IST combination study of duvelisib with RB or RB
(Abstract #3928). In addition, preclinical data will be presented on the effects of duvelisib on CLL tumor microenvironment
(#1753).
KPTI will present four clinical data abstracts on lead candidate selinexor, including an oral presentation of Ph 1 data that
compares efficacy and safety for a range of doses of selinexor in a number of different hematological malignancies
(#258). We believe recent concerns regarding selinexor tolerability in AML patients to be overdone and we expect this
presentation will help clarify its safety profile. In addition, 13 preclinical abstracts will be presented on selinexor and KPTI’s
additional pipeline candidates, including its second generation SINE compound KPT-8602, and PAK4 Allosteric Modulators
(PAMs).
• MEIP will present final response data and overall survival estimates for its Ph 2 study of pracinostat in combination with
azacitidine in elderly patients with AML (#453). Data presented at EHA showed 54% (27/50) of patients achieved the primary
endpoint of CR+CRi+morphologic leukemia-free state (MLFS), with 31 patients still being followed. MEIP will also present an
exploratory analysis from the failed Ph 2 study of pracinostat and azacitidine in MDS patients, indicating patients able to tolerate
the combination for at least four cycles appeared to derive benefit compared to azacitidine alone, with hazard ratios for
progression-free survival (0.37), event-free survival (0.33) and overall survival (0.59) all favoring the Pracinostat plus azacitidine
arm (#911). We also expect an update on for the development strategy going forward for pracinostat around the same time as
ASH.
• Data from the lead-in stage of STML’s pivotal trial for SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) or
AML patients will be presented in abstract #3795. Initial data for nine BPDCN patients presented in May, 2015 demonstrated
an ORR of 78% (5 CRs and 2 PRs). Abstract #3795 reiterates these results and also reports that out of eight evaluable AML
patients, three demonstrated stable disease with further dose escalation ongoing. STML will also present preclinical data on SL-
801, its novel XPO1 inhibitor (#4433).
• XNCR will present preclinical data for its bispecific CD38 x CD3 program (#1798), including demonstrating that
attenuation of CD3 affinity may improve the therapeutic window, potentially enabling improved tolerability and higher
dosing. The CD38 x CD3 program was licensed to Amgen (AMGN, not covered) in September, along with five other preclinical
bispecific programs from XNCR.