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ASCO 2014 Small-Cap Cancer Biotech Preview

publication date: May 8, 2014
author/source: Mike Havrilla

Written by Mike Havrilla (updated on 5/8/14)--ASCO is the American Society of Clinical Oncology (


ASCO is the largest cancer-related conference of the year and it attracts the entire bio-pharmaceutical industry, including all types of public and private companies (along with a wide range of healthcare professionals in the cancer care space)--ranging from micro to mega-cap stocks since the development of new treatments and diagnostics for cancer is a highly profitable endeavor since there are typically few options for what are typically life-threatening conditions that represent unmet medical needs.


Every year small companies are invited to show what they are developing, and it gives them the opportunity to present on a ‘big stage’ and get noticed. A small company showing up on the agenda is usually enough to get the stock moving and start a share price run-up. The 2014 Conference is scheduled for May 30 to June 3 in Chicago.


Abstracts (a short summary of key data such as survival, response rate and side effects, etc.) will be released ( this year on 5/14/14 at 5pm (ET) and companies have began confirming their presentations this year at the conference.


Previous examples of big stock price movers in the small-cap cancer biotech space include Delcath Systems (DCTH) (2010) and Celldex Therapeutics (CLDX) (2008-2010). While any ASCO presentation can be a big catalyst; the most important presentations are those that include either first-time reporting of top-line results (a relatively rare occurrence since top-line results are typically announced via press release once known) or updated / new data from a previously reported trial (e.g. reporting final or updated survival results for a trial).


Below is a preview of some small-cap cancer biotech companies that have provided guidance or already confirmed presentations at ASCO this year. Please note that even small companies may have multiple presentations at ASCO; although many involve very early stage and preclinical research that is typically not as relevant as a trading catalyst and often gets lost in the noise of so much data being presented in a short period of time.


1.) ImmunoCellular Therapeutics (IMUC): ICT-107 (dendritic cell brain cancer vaccine)

- In DEC13 reported P2 brain cancer (GBM) trial failed to meet primary overall survival (OS) endpoint but improved progression-free survival (PFS) & will present updated OS results at ASCO on 6/1/14 w/ abstracts (#2005) to be published online 5/14/14 at 5pm ET

- Plans to conduct end of P2 mtg w/ FDA to discuss planned P3 trial for potential start by early 2015

- EU Orphan Drug status decision exp 2Q14 (already has FDA Orphan Drug status)


2.) TG Therapeutics (TGTX):


- Ublituximab (TG-1101) (CD20 targeted anti-cancer monoclonal antibody): Will present updated single-agent data at ASCO on 5/30/14 with abstracts (#8524) to be published online 5/14/14 at 5pm ET, will present data from P1/2 trial in combo w/ IMBRUVICA at EHA mtg (June 12-15) w/ abstracts to be published on 5/21/14 (, plans to begin one or more pivotal trial(s) evaluating TG-1101 and/or TGR-1202 in combination treatment regimens during 2H14


- TGR-1202 (oral PI3K delta inhibitor anti-cancer agent): Will present updated single-agent data at ASCO on 5/30/14 with abstracts (#2513) to be published online ( 5/14/14 at 5pm ET, being developed for relapsed or refractory blood-based cancers, will present clinical update for P1 trial in combo w/ Ublituximab at Pan Pacific Lymphoma conference (July 21-25) 


3.) OncoSec Medical (ONCS): ImmunoPulse Therapy (DNA-based IL-12 immune system cytokine for local and system anti-cancer effect)

- Will present updated P2 melanoma trial results at ASCO on 6/2/14 with abstracts (#9025) to be published on 5/14/14 at 5pm ET

- Llink to expected upcoming milestones at


4.) Threshold Pharma (THLD): TH-302 (a hypoxia-activated pro-drug or HAP anti-cancer agent). 1Q14 financial updates include $86.4 million (M) cash as of 3/31/14, 1Q14 operating loss of ($8.6M), 59.3M shares outstanding and a $150M mixed security SEC S-3 shelf filing declared effective as of mid-April.


- ID NCT01440088 for pivotal fully enrolled P3 (Study 406) trial under SPA in combo w/ doxorubicin for soft tissue sarcoma (EU & US Orphan Drug status) w/ interim overall survival (OS) efficacy analysis (235 events) exp mid-2014 (2-3Q14 estimate) to allow for early termination of study for increased survival or continue as planned (there is no futility analysis to end trial early for failure) to topline OS data exp 1H15


- NCT01746979 for P3 pancreatic cancer (MAESTRO) trial under SPA in combo w/ gemcitabine w/ OS results exp 2-3Q15


- Global partner Merck KGaA funds 70% of expenses


- Multiple ongoing P1 & P2 trials with expected updates at medical conferences throughout 2014 including AACR & ASCO:


- Two posters will be presented at the American Association for Cancer Research's (AACR's) Special Conference on Pancreatic Cancer: Innovations in Research and Treatment, May 18-21, New Orleans, Louisiana:


Abstract #A102: Combination treatment with hypoxia-activated prodrug TH-302 and radiation reduce pancreatic tumor initiating cells and tumor growth in patient-derived xenografts; 4:30 PM - 7:00 PM Central Time on Monday, May 19, 2014 (Poster Session A).



Abstract #B88: Pharmacodynamic changes from the TH-302, gemcitabine, and nab-paclitaxel triplet combination in a xenograft model of pancreatic cancer; 12:30 PM - 3:00 PM Central Time on Tuesday, May 20, 2014 (Poster Session B). 


Abstract #2029 (Poster #20): Phase 1/2 study of investigational hypoxia-targeted drug, TH-302, and bevacizumab in recurrent glioblastoma (GBM) following bevacizumab failure; 1:00 PM - 4:00 PM Central Time on Friday, May 30, 2014, in E35b.


Abstract #8534 (Poster #14): Preliminary safety and efficacy of TH-302, an investigational hypoxia-targeted drug, and dexamethasone in patients with relapsed/refractory multiple myeloma (RR MM); 1:00 PM - 4:00 PM Central Time on Friday, May 30, 2014, in S405.


5.) Sunesis Pharma (SNSS): Vosaroxin (formerly voreloxin) (DNA targeted anti-cancer agent)

- ID NCT01191801 for fully enrolled P3 (VALOR) trial for blood-based cancer (AML) w/ top-line data expected during 3Q14 (delayed from previous 2Q14 guidance due to survival events occuring at slower pace than originally forecast)--while this is not an ASCO-related catalyst it is the major upcoming catalyst for the Company and similar to $MACK below I estimate slighly below average (40%) odds for success given the odds against success for small-cap bios in late-stage/pivotal trials and unproven nature of the drug which is not approved for any indication

- Updated P1B/2 AML/MDS data will be presented at ASCO on 6/2/14 with abstracts (#7098) to be published on 5/14/14 at 5pm ET


6.) DelMar Pharma (DMPI): VAL-083 (novel alkylating IV anti-cancer agent)

- ID NCT01478178 for open-label single-arm P1/2 dose-escalation trial for treatment of recurrent brain cancer (GBM) to evaluate safety and determine max tolerated dose (MTD) w/ exp completion mid-2014, currently enrolling pts in cohort 6 at 30mg/m2 dose w/ MTD not yet reached & expects to present updates at conferences througout 2014 including ASCO (most recently presented at AACR in April)


7.) CytRx (CYTR): Aldoxorubicin (INNO-206) (Doxorubicin EMCH conjugate anti-cancer agent)

- ID NCT01514188 for P2B sarcoma trial w/ stat sig progression-free survival (PFS) reported in DEC13 (will present detailed results at ASCO on 6/1/14 with abstract #10502 to be published at on 5/14/14 at 5pm ET) w/ overall survival (OS) results exp 2H14

- NCT02014844 for P2 brain cancer (GBM) trial w/ prelim results exp 2H14

- NCT02029430 for P2 Kaposi’s sarcoma trial w/ results exp 2Q15

- In MAR14 began pivotal P3 sarcoma trial under SPA (NCT02049905) during 1Q14 w/ PFS data exp 2Q16

- Plans to begin P2B trial for second-line small cell lung cancer (SCLC) 2H14

- Developed to bind circulating albumin for targeted delivery at tumor sites at a 3-4X higher equivalent dose to approved chemo drug doxorubicin


8.) Onconova Therapeutics (ONTX): Rigosertib (PI3K and PLK dual signaling pathway inhibitor IV and oral anti-cancer agent)

- In FEB14 announced P3 (IV) trial for higher risk blood cancer (MDS) failed to meet primary endpoint of overall survival (OS) but showed stat sig improved in subgroup that progressed on HMA therapy w/ detailed results to be presented at ASCO (May 30-June 3) & regulatory discussions 2Q14

- Plans to seek SPA agmt w/ FDA for a pivotal P3 (oral) trial for transfusion dependent lower-risk MDS w/ results from 20-patient validation cohort evaluating prognostic genomic biomarker and start of P3 exp 2H14

- Updated results expected during  2H14 from P1/2 trial in combo w/ VIDAZA for first-line treatment of MDS

- Fully enrolled P2 (oral) trial for head and neck cancer w/ exp update 2Q14

- In DEC13 discontinued P3 (IV) pancreatic cancer trial after failed interim analysis

- Licensed to $BAX in Europe & SymBio in Japan

- US & EU Orphan Drug status for MDS


9.) Ambit Biosciences (AMBI): Quizartinib (AC220) (oral once-daily FLT3 inhibitor anti-cancer agent)

- ID NCT02039726 for pivotal P3 (QUANTUM-R) adaptive trial as mono-therapy treatment for relapsed or refractory blood cancer (FLT3-ITD mutant AML) w/ interim analysis planned for potential increase in enrlmt, exp to complete enrlmt during 2H15 if there is no increase and report top-line data expected during 1Q16

- In APR14 began P2 cohort in P1/2 combo trial for same indication w/ full results to be presented in 2014

- Will present P2 results at ASCO with abstracts (#7100 & 7093) to be published on 5/14/14 at 5pm ET



10.) ImmunoGen (IMGN)

- IMGN529 (CD37-targeting ADC anti-cancer agent) ID NCT01534715 for P1 trial in pts w/ B-cell blood based cancer (NHL) w/ initial clinical results scheduled for presentation at ASCO with abstracts ( to be published on 5/14/14 at 5pm ET 

- IMGN853 (folate receptor α-positive ADC anti-cancer agent) ID NCT01609556 w/ final results exp late 2014-early 2015, being developed for a variety of FOLR1-positive solid tumors, will present updated results at ASCO with abstracts to be published on 5/14/14 at 5pm ET


11.) Array BioPharma (ARRY)--on 4/29/14 reported quarterly results and announced that ASCO accepted 16 abstracts for presentation with late-stage compounds highlighted below (the Company has a diverse pipeline with numerous early-mid stage trials ongoing so the main focus will be pivotal P3 results expected next year). 


- Binimetinib (MEK162) (ARRY-162) (MEK inhibitor anti-cancer agent):  Three ongoing P3 trial in collaboration w/ $NVS including ovarian cancer ( ID NCT01849874 w/ results expected mid-2016), BRAF-mutant melanoma (NCT01909453 w/ results expected mid-2017), and lead indication NRAS-mutant melanoma (NCT01763164 w/ results expected during 1H15 to support potential regulatory approval filings in 2015) 


- Selumetinib (AZD6244) (MEK inhibitor anti-cancer agent) (oral twice-daily capsule):  Three ongoing pivotal P3 trials being conducted by partner $AZN w/ primary completion date of mid-2015 for first indication (uveal melanoma), other trials include KRAS-mutant NSCLC (non-small cell lung cancer) and thyroid cancer



Binimetinib will be featured in seven presentations, including one oral presentation, at the 2014 American Society for Clinical Oncologists (ASCO) annual meeting including the following:


"A phase 1b/2 study of LEE011 in combination with binimetinib (MEK162) in patients with NRAS-mutant melanoma: Early encouraging clinical activity" Abstract #9009 – oral presentation


"A phase 1b dose escalation study of BYL719 plus binimetinib (MEK162) in patients with selected advanced solid tumors"  Abstract #9051


"The BRAF inhibitor LGX818 induces endoplasmic reticulum stress and sensitizes NRAS-mutant melanoma cells to the MEK inhibitor MEK162" Abstract #9062


"The Role of MEK inhibition in Neuroblastoma Tumor Cells" Abstract #10068        


Trials in Progress posters will also showcase the trial designs for MILO and NEMO.



Selumetinib will be featured in five presentations at the 2014 ASCO annual meeting:


"NCI #8412: A randomized phase II trial of AZD6244 alone and AZD6244 plus temsirolimus for soft-tissue sarcomas"  Abstract #10526


"Phase I Study of the MEK1/2 inhibitor selumetinib (AZD6244 hydrogen sulfate) in children and young adults with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs)"  Abstract #10018


"A phase 1 study of AZD6244 in children with recurrent or refractory low-grade gliomas: A Pediatric Brain Tumor Consortium report" Abstract #10065


"ABC-04: A phase 1b trial of cisplatin, gemcitabine and selumetinib in patients with advanced biliary tract cancers"  Abstract #4090

"Phenformin combines with selumetinib in targeting KRAS mutant non-small cell lung cancer cells with alternative LKB1 status"  Abstract #2589


12.) Celldex Therapeutics (CLDX)--Varlilumab ("varli"; CDX-1127), an immune modulating mAb targeting CD27 in solid tumors and hematologic malignancies:

- Celldex continues to advance plans to initiate multiple Phase 1/2 studies of varlilumab in combination with various agents in the second half of 2014, including a Phase 1/2 study of varlilumab and Yervoy(R) (and potentially other checkpoint inhibitors) plus CDX-1401 in NY-ESO+ patients with metastatic melanoma and a Phase 1/2 study of varlilumab plus B-raf targeted pathway agents (followed sequentially by a checkpoint inhibitor) for patients with B-raf mutated metastatic melanoma.


- Celldex will present data from the Phase 1 varlilumab program in two separate Poster Highlight Sessions at the 2014 American Society of Clinical Oncology (ASCO) Annual Meeting. The first presentation will include dose-escalation data from the solid tumor arm of the study and data from the expansion cohorts in metastatic melanoma and renal cell carcinoma; the second will include dose-escalation data from the hematologic malignancies arm of the study.


13.) Merrimack Pharma (MACK): 1Q14 financial updates & summary includes approx. $124 million (M) cash as of 3/31/14, used ($31.4M) cash to fund operations during 1Q14, 103M shares of common stock outsanding & $111M in total debt


a.) MM-398 (novel encapsulated liposomal formulation of approved anti-cancer drug irinotecan):  On 5/1/14 reported pivotal P3 trial for pts w/ metastatic pancreatic cancer that progressed on gemcitabine treatment met primary endpt w/ stat sig improved overall survival (OS) (6.1 vs. 4.2 months for control arm) to support planned NDA filing in 2014 as combination regimen (monotherapy arm failed to improve OS), US and EU Orphan Drug status, developed to extend activity and reduce side effects vs. standard irinotecan, will present P3 results at ESMO (June 25-28)

Link to study protocol ( which I believe is flawed since the active comparator arm did not include the standard formulation of irinotecan so there is no way to know whether MM-398 provides any survival advantage over an already approved chemotherapy drug that is widely available as a generic drug. 

- Active Comparator: 5-FU & Leucovorin (control arm which does not include standard formulation of irinotecan)

- Experimental: MM-398, 5-FU & Leucovorin (1.9 month OS advantage which was stat sig & serves as basis for NDA filing)

- Experimental: MM-398 (this is the monotherapy arm that failed to improve OS)


b.) MM-121 (ErbB3 inhibitor anti-cancer agent):  In OCT13 reported P2 ovarian cancer trial failed to meet primary endpoint of improved progression-free survival (PFS), P2 trial for wild-type EGFR non-small cell lung cancer also failed to meet primary endpoint, in NOV13 reported P2 results from ER/PR+ breast cancer trials which continued trend of potential benefit in subgroup of pts w/ two biomarkers previously identified in ovarian cancer trials, P2 triple-negative breast cancer top-line data expected during 2Q14, will present P2 data for MM-121 at ASCO (May 30-June 3) 


c.) MM-111 (HER2/3 signaling inhibitor anti-cancer agent): ID NCT01774851 for P2 trial in combo w/ HERCEPTIN and paclitaxel for HER2-expressing cancer of the esophagus, GE junction and stomach w/ results expected during 2H15, FDA Orphan Drug status for advanced gastric and esophageal / GE junction cancers


d.) The Company also plans to begin P2 trials in 2014 for MM-302, MM-151 & MM-141. As illustrated on the image below from the Company’s annual report SEC 10-K filing there are multiple ongoing clinical trials and pipeline compounds in development with MM-398 & MM-121 being the primary value drivers at this stage. 



14.) Galena (GALE): 

GALE-301 (Folate Binding Protein (FBP) vaccine) advances into Phase 2 to prevent recurrences in high-risk ovarian cancer patients after completing standard of care therapy. Full Phase 1 results to be presented at the American Society of Clinical Oncology (ASCO) 50th Annual Meeting, May 30 - June 3, 2014.


15.) Ariad Pharma (ARIA):


- Iclusig: PRAC mtg being held by European regulators (EMA) to assess risks & benefits of drug w/ final list of follow-up questions exp by 5/9/14 & PRAC recommendation (July 7-10 mtg) along with CHMP opinion (July 21-24 mtg) exp in JUL14, in DEC13 revised FDA prescribing info & REMS to reflect potential for serious blood clots (drug was temporarily off the market in US from OCT13-DEC13) which resulted in stopping P3 (EPIC) trial, currently approved for specific types of blood-based cancers (T315I-positive Ph+ ALL & certain types of CML)


Through March 31, 2014, approximately 340 patients in the U.S. received Iclusig obtained commercially based on physicians’ prescriptions. By the end of the first quarter, there were nearly 300 unique prescribers of Iclusig.


Approximately 60 percent of prescribers are community-based physicians, with the remainder being physicians practicing in academic hospitals. We expect that adoption of Iclusig among community oncologists and hematologists will continue to increase as commercialization progresses further.


In Europe, we are selling Iclusig in Germany, the United Kingdom, France, Austria, the Netherlands, Norway, and Sweden. This year, we expect to expand commercialization of Iclusig to all of the major markets in Europe – 16 countries – based on staged achievement of pricing and reimbursement approvals in each country. We anticipate that most of the EU pricing approvals will occur in the second half of 2014.


Five investigator-sponsored trials have been cleared to resume enrollment: a U.S. trial in patients with non-small cell lung cancer (NSCLC) with RET translocation, a U.S. trial in patients with medullary thyroid cancer with or without RET mutations, a U.S. trial in endometrial cancer patients with FGFR2 mutations, a French trial in patients with FLT3-positive acute myeloid leukemia (AML), and a UK trial in patients with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).


Eight additional investigator-sponsored trials are pending regulatory and/or institutional review board (IRB) approval; these include investigations in blast-phase chronic myeloid leukemia (CML), Ph+ALL, AML, and NSCLC with RET translocation and/or FGFR1 amplification.


We will present multiple clinical updates on Iclusig at the 2014 annual meeting of the American Society of Clinical Oncology (ASCO) in June. This will include updates from the ongoing Iclusig Phase 1 and PACE trials, data from the discontinued EPIC trial in patients with newly diagnosed CML, and the Phase 2 trial in patients with refractory gastrointestinal stromal tumors (GIST).


- AP26113:

A pivotal global Phase 2 trial of AP26113 in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who were previously treated with crizotinib is now open and enrolling patients. The ALTA trial is designed to determine the safety and efficacy of AP26113 in refractory NSCLC patients who test positive for the anaplastic lymphoma kinase (ALK+) oncogene. The trial will enroll approximately 220 patients including those with brain metastasis.


We will present an update from the ongoing Phase 1/2 clinical trial of AP26113 at the 2014 ASCO annual meeting.


16.) Nektar Therapeutics (NKTR):


Etirinotecan Pegol (NKTR-102) (long-acting, tumor-targeted formulation of approved cancer drug class, topo I inhibitor): ID NCT01492101 w/ top-line results expected during 1Q15 for fully enrolled pivotal P3 (BEACON) trial in pts w/ metastatic breast cancer (FDA Fast Track status)


Abstract Title: "Etirinotecan pegol (EP, NKTR-102) in the treatment of high grade glioma (HGG): a phase 2 trial," Nagpal et al.

Abstract Number: 2096

Session Title/Track: Central Nervous System Tumors

Date: May 31, 2014, 1:15 p.m. – 5:00 p.m. Central Time

Location: S Hall A2

Abstract Title: "Combination Immunotherapy: Synergy of a Long-Acting Engineered Cytokine (NKTR-214) and Checkpoint Inhibitors Anti-CTLA-2 or Anti-PD1 in Murine Tumor Models," Kantak et al.

Abstract Number: 3082

Session Title/Track: Developmental Therapies - Immunotherapy

Date: June 1, 2014, 8:00 a.m. – 11:45 a.m. Central Time


Location: S Hall A2


17.) Immunomedics (IMMU):


Clivatuzumab tetraxetan: ID NCT01956812 w/ results expected 2H15 for pivotal P3 trial in combo w/ low-dose gemcitabine and best supportive care for treatment of metastatic pancreatic cancer in pts who have received at least two prior therapies w/ a primary endpoint of overall survival (OS) 

Outside clinical investigator will present results from the randomized Phase Ib study of fractionated yttrium-90-labeled clivatuzumab tetraxetan in patients with pancreatic cancer having 2 or more prior therapies at the American Association for Cancer Research (AACR) Special Conference on Pancreatic Cancer: Innovations in Research and Treatment on Tuesday, May 20, 2014, and at the 2014 Annual Meeting of the American Society of Clinical Oncology (ASCO) on Sunday, June 1, 2014, in a Poster Highlights Session.


IMMU-130 & IMMU-132 (antibody-drug conjugate anti-cancer agents): ID NCT01915472 w/ results expected 2H15 for IMMU-130 in P2 metastatic colon cancer trial, NCT01631552 for P2 portion of dose-escalation trial for IMMU-132 for a variety of solid tumor indications w/ results expected 1H15, in DEC13 received FDA Orphan Drug status for small cell lung cancer (SCLC)

Multiple partial responses, including 2 patients with small-cell lung cancer, 2 with triple-negative breast cancer and 1 with colorectal cancer, from the ongoing Phase I/II study of IMMU-132 in patients with solid cancers were reported at the 2014 Annual Meeting of AACR. More information on the results can be obtained from the Company's press release at 

Results of 13 pancreatic cancer patients with CT-assessments from the Phase I/II trial of IMMU-132 will be presented at the AACR Special Conference on Pancreatic Cancer: Innovations in Research and Treatment on Tuesday, May 20, 2014.

Additional Phase II data from the IMMU-132 trial will be provided at the 2014 Annual Meeting of ASCO on Monday, June 2, 2014, in a Poster Highlights Session.

IMMU-130 (anti-CEACAM5-SN-38)

Two partial responders with durable responses were reported at the 2014 AACR Annual Meeting from the 3 Phase I studies using 3 different IMMU-130 dosing schedules in patients with metastatic colorectal cancer. For more information, please refer to the Company's press release at 


Results from the more frequent dosing schedules of IMMU-130 once or twice-weekly for 2 weeks followed by one week of rest in a 3-week cycle will be updated at the 2014 ASCO Annual Meeting on Sunday, June 1, 2014.


18.) Ignyta (RXDX)

RXDX-101 (oral pan-Trk, ROS1 & ALK inhibitor anti-cancer agent): ID NCT02097810 for P1/2 trial in adults w/ locally advanced or metastatic solid tumors w/ a targeted molecular alteration w/ final results expected mid-2016

Date/time:   Saturday, May 31, 2014, 1:39 PM - 1:51 PM, Central time


Phase 1 open label, dose escalation study of RXDX-101, an oral pan-Trk, ROS1, and ALK inhibitor, in patients with advanced solid tumors with relevant molecular alterations. (Abstract number 2502)

Presenter: Filippo G. De Braud, M.D., Fondazione IRCCS Istituto Nazionale dei Tumori

Session: Developmental Therapeutics - Clinical Pharmacology and Experimental Therapeutics.


OUTLOOK & TRADE SUMMARY: There are no major (i.e. first-time announcement of top-line survival results for a mid or late-stage trial) presentations scheduled for this year's ASCO conference and many of the companies outlined above are presenting updated/detailed or preliminary results; but these presentations could still have an impact on trading and as with any year there are bound to be some surprises & late-breaking data presented. For more detailed analysis of the companies mentioned above search the site for recent articles or if there are no recent articles published then please reference the catalyst database since many of the companies have other catalysts aside from the data being presented at ASCO.


Disclosure: No positions