Company (location)

Product

Description

Indication

Status

Date

Abbvie Inc. (North Chicago)

Imbruvica

Ibrutinib; oral, once-daily BTK inhibitor

Relapsed/refractory chronic lymphocytic leukemia/small lymphocytic leukemia

Long-term follow-up results from the pivotal phase III RESONATE trial (PCYC-1112) showed Imbruvica was associated with significantly longer progression-free survival (PFS; 59%) with median follow-up of 44 months, including in patient subgroups with genomic abnormalities that are traditionally considered high-risk for poor outcomes; three-year overall survival (OS; 74%) was longer in Imbruvica-treated patients

6/6/17

Adaptimmune Therapeutics plc (Philadelphia)

NY-ESO TCR

Targets the NY-ESO-1 target peptide on cancer cells

Synovial sarcoma

Updated data from its NY-ESO study; responses across all of its ongoing cohorts in the study, including in patients who express lower levels of NY-ESO, and in those receiving conditioning with modified doses of fludarabine/cyclophosphamide; a lower response rate was observed in the absence of fludarabine conditioning and that cohort is now closed

6/6/17

Affimed NV (Heidelberg, Germany)

AMF-26

Inhibitor of the Golgi system targeting ADP-ribosylation factor 1

Multiple myeloma

Data showed that AFM-26 induced lysis of BCMA-low cell lines that have been described as not sensitive to treatment with GSK-2857916

6/7/17

Agenus Inc. (Lexington, Mass.)

AGEN-1884

Anti-CTLA-4 antibody

Advanced solid malignancies

Interim analysis from an ongoing phase I dose-escalation trial showed an acceptable safety and tolerability profile at the 0.1-mg/kg, 0.3-mg/kg, 1-mg/kg and 3-mg/kg dose levels; a partial response was observed for a patient with angiosarcoma at week 33, with a 92% reduction in tumor volume upon treatment with 0.1 mg/kg

6/6/17

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-120

Ivosidenib

IDH1 mutant-positive cholangiocarcinoma

Updated data from the dose-escalation and expansion cohorts of the phase I study showed a durable disease control signal was observed; a safety analysis demonstrated that AG-120 was well-tolerated with a favorable safety profile

6/6/17

Aileron Therapeutics Inc. (Cambridge, Mass.)

ALRN-6924

p53-targeting drug; stapled peptide therapeutic

Advanced cancers

Antitumor activity was observed in a phase I trial, including complete responses, partial responses and evidence of stable disease

6/6/17

Amgen Inc. (Thousand Oaks, Calif.)

Xgeva

Denosumab

Multiple myeloma

The phase III '482 study met its primary endpoint, demonstrating it is noninferior to zoledronic acid in delaying the time to first on-study skeletal-related event; patients on Xgeva had a significantly lower rate of renal adverse events compared to zoledronic acid

6/6/17

Amgen Inc. (Thousand Oaks, Calif.)

Imlygic

Talimogene laherparepvec

Unresectable stage IIIB-IV melanoma

New data from the phase II '264 study demonstrated that Imlygic in combination with Yervoy (ipilimumab, Bristol-Myers Squibb Co.) more than doubled objective response rate, defined as the proportion of patients with tumor size reduction, compared to Yervoy alone, meeting the primary endpoint of the study; the analysis showed that 38.8% of patients treated with Imlygic plus Yervoy achieved an objective response vs. 18% of patients treated with Yervoy alone; patients in the combination arm also experienced nearly double the complete response rate compared to Yervoy alone

6/6/17

Angle plc (Guildford, Surrey, U.K.)

Parsortix system

Uses a micro-fluidic technology to capture and then harvest circulating tumor cells from blood

Colorectal cancer

Researchers were able to use the Parsortix system to harvest circulating tumor cells (CTCs) and were able to analyze those cells for the presence or absence of meEGFR (arginine methylation of the epidermal growth factor receptor); the total number of CTCs was not correlated to PFS, but the proportion of those CTCs that were positive for the meEGFR marker was found to be predictive: patients whose CTCs harvested by Parsortix had a higher proportion of meEGFR positive CTCs, had significantly shorter PFS compared to those patients whose CTCs had lower levels

6/6/17

Armo Biosciences Inc. (Redwood City, Calif.)

AM-0010

Pegilodecakin, pegylated interleukin-10

Advanced non-small-cell lung cancer (NSCLC) and metastatic renal cell carcinoma (RCC)

Data showed the combination of AM-0010 with either of the checkpoint inhibitors Opdivo (nivolumab, Bristol-Myers Squibb Co.) or Keytruda (pembrolizumab, Merck & Co. Inc.) induced high response rates and durable objective responses; the preliminary objective response rate (ORR) was 38.5% in 26 evaluable patients with advanced NSCLC treated with AM-0010 and an anti-PD-1 checkpoint inhibitor; the median progression-free survival (PFS) was 10.9 months

6/6/17

Arqule Inc. (Burlington, Mass.)

ARQ-087

Fibroblast growth factor receptor (FGFR) inhibitor

Intrahepatic cholangiocarcinoma

Data from a phase I/II trial demonstrated a meaningful clinical benefit to patients harboring FGFR2 fusions; the data show a robust response rate and prolonged duration of therapy for those patients well in excess of that reported for second-line chemotherapy

6/6/17

Aslan Pharmaceuticals Pte. Ltd. (Singapore)

Varlitinib

Reversible, small molecule pan-HER inhibitor

Metastatic solid tumors

Data from a phase Ib study in combination with doublet chemotherapy showed three of 15 evaluable patients showed partial response, 10 had stable disease and two had progressive disease; one cholangiocarcinoma patient had an 87% reduction in tumor size by cycle two of treatment and a complete resolution in one lesion by cycle four; another cholangiocarcinoma patient without measurable lesions in the liver or biliary tract achieved long-term disease control for 15.2 months with good tolerability on varlitinib as monotherapy

6/5/17

Astellas Pharma Inc. (Tokyo)

Gilteritinib

FLT3/AXL inhibitor

FLT3 mutation-positive relapsed/refractory acute myeloid leukemia

Data from an exploratory analysis of the phase I/II CHRYSALIS study demonstrated that patients with molecular response had improved overall survival compared with those without a molecular response when treated with either 120 mg/day or 200 mg/day doses

6/7/17

Astrazeneca plc (London)

Lynparza

Olaparib 300-mg twice-daily tablet

Germline BRCA-mutated (gBRCAm), platinum-sensitive, relapsed serous ovarian cancer

New data from a phase III SOLO-2 trial of Lynparza maintenance treatment demonstrating that quality of life (QOL) is sustained alongside improved progression-free survival; key data show that for the primary endpoint for patient-reported outcomes there was no appreciable detrimental effect on QOL for patients receiving maintenance treatment with olaparib versus patients on placebo -2.90 vs. -2.87, respectively

6/5/17

Astrazeneca plc (London)

Lynparza

Olaparib; poly ADP ribose polymerase inhibitor

BRCA-related metastatic breast cancer

Detailed results from the phase III OLYMPIAD trial showed those getting the drug were 42% less likely to see their cancer spread than those given conventional chemotherapy; women taking Lynparza had their disease progress after about seven months, compared to 4.2 months of median progression-free survival for those on chemotherapy

6/6/17

Astrazeneca plc (London)

Tagrisso

Osimertinib

EGFR T790M mutation-positive non-small-cell lung cancer

Data showed it has clinical activity in patients with disease progression to central nervous system metastases; Tagrisso extended the length of time patients with CNS metastases live without disease worsening or death to 11.7 months and chemotherapy to 5.6 months in the AURA3 trial

6/7/17

Aveo Oncology Inc. (Cambridge, Mass.) and Biodesix Inc. (Boulder, Colo.)

Ficlatuzumab

Humanized IgG1 antibody that binds to the HGF ligand

Cetuximab-resistant recurrent/metastatic head and neck squamous cell carcinoma

Results from two investigator-sponsored phase I studies showed the addition of ficlatuzumab to cetuximab resulted in a disease control rate of 67%, and prolonged progression-free and overall survival compared to historical controls, in addition to being well-tolerated

6/6/17

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL-101553

Tumor checkpoint controller

Various cancers

Data from the ongoing phase I/IIa trial showed that, in the cohorts completed so far, there was a dose-proportional up to fivefold higher weekly exposure at reduced maximum plasma concentrations compared with two-hour weekly infusion; of the 19 patients evaluated so far, eight had stable disease as best objective response

6/7/17

Beigene Ltd. (Cambridge, Mass.)

BGB-A317

Anti-PD-1 antibody

Multiple solid tumors

Initial data from the dose-escalation portion of the phase I trial of BGB-A317 in combination with BGB-290 suggest that the combination is generally well-tolerated and shows antitumor activity

6/6/17

Bergenbio ASA (Bergen, Norway)

BGB-324

Axl inhibitor

Myelodysplastic syndrome

Data from an ongoing phase I/II trial showed that one patient experienced a partial response and remained on treatment for more than 18 months; BGB-324 was well-tolerated and, at the time of data cut-off, the maximum tolerated dose had not yet been reached

6/7/17

Bluebird Bio Inc. (Cambridge, Mass.) and Celgene Corp. (Summit, N.J.)

BB-2121

Anti-BCMA CAR T-cell therapy

Relapsed/refractory multiple myeloma

Updated results indicated that 100% of the 15 evaluable patients in active dose cohorts achieved an objective response; the overall response rate across all cohorts is 89%, and 73% of evaluable patients in active dose cohorts achieved a very good partial response or better, and 27% had a complete response

6/6/17

Blueprint Medicines Corp. (Cambridge, Mass.)

BLU-285

Selective PDGFRalpha and KIT inhibitor

PDGFRalpha-driven gastrointestinal stromal tumors harboring a D842 mutation

New phase I data demonstrated an objective response rate of 60% and an estimated nine-month progression-free survival of 87%; tumor reduction was observed in eight of 14 evaluable patients treated at dose levels of at least 300 mg once daily

6/6/17

Boehringer Ingelheim GmbH (Ingelheim, Germany)

Nintedanib

Oral triple angiokinase inhibitor

Malignant pleural mesothelioma

Phase II results from LUME-Meso showed it demonstrated a 46% reduction in the risk of disease progression when added to standard first-line chemotherapy of pemetrexed/cisplatin in patients compared to chemotherapy alone; overall survival was also prolonged with the addition of nintedanib

6/6/17

Boston Biomedical Inc. (Cambridge, Mass.)

Napabucasin

Orally administered agent designed to inhibit cancer stemness pathways by targeting STAT3

Various cancers

Phase Ib/II data showed anticancer activity in advanced or metastatic disease in seven tumors types: colorectal, pancreatic, nonsquamous non-small-cell lung, ovarian, breast, hepatocellular and melanoma

6/6/17

Boston Biomedical Inc. (Cambridge, Mass.)

Amcasertib

Designed to inhibit cancer stemness pathways, including Nanog, by targeting stemness kinases

Advanced adenoid cystic carcinoma (ACC) and advanced head and neck cancers

Data from a phase Ib/II study showed amcasertib prolonged survival and was well-tolerated in patients with both types of cancer; of the 14 patients enrolled with ACC, disease control rate was observed in 86%, prolonged disease control of greater than six months was achieved in 57%, and median overall survival (mOS) was 27.8 months; among the 21 patients with head and neck cancers, 38% survived 12 months, and mOS was seven months

6/7/17

Bristol-Myers Squibb Co. (New York)

Opdivo

Nivolumab; checkpoint inhibitor

Advanced cervical, vaginal and vulvar cancers associated with infection by the human papillomavirus

The first data from a cohort of the phase I/II CHECKMATE -358 study showed that across the cohort, the objective response rate (ORR) – the primary endpoint – was 20.8% (95% CI: 7.1 to 42.2), with a 70.8% disease control rate of women experiencing complete or partial response or stable disease; median progression-free survival was 5.5 months (95% CI: 3.5 to not reached), and median overall survival was not reached; responses were seen only in cervical cancer patients; of these, five had complete and partial responses, with an ORR of 26.3% (95% CI: 9.1 to 51.2)

6/5/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

Sprycel

Dasatinib

Chronic phase chronic myeloid leukemia

Phase II data from CA180-226 evaluating Sprycel in imatinib-resistant or -intolerant and newly diagnosed pediatric patients showed at two-year follow-up, patients demonstrated a cumulative cytogenetic response rate of 55.2% three months into treatment, which increased over time to greater than 90% at 24 months

6/6/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

Yervoy

Ipilimumab

Resected high-risk melanoma

Relapse-free survival at three years was 56% for Yervoy 3 mg/kg and 54% for Yervoy 10 mg/kg; treatment-related grade 3 and 4 adverse events were experienced by 37% of patients in the 3-mg/kg arm and 57% in the 10-mg/kg arm

6/6/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

Opdivo

Nivolumab

DNA mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer

Interim data from the phase II CheckMate-142 trial of Opdivo monotherapy or in combination with Yervoy showed the objective response rate was 54.8%, median overall survival was not yet reached, and the overall survival rate at nine months was 87.6% for patients receiving Opdivo plus Yervoy

6/6/17

Bristol-Myers Squibb Co. (Princeton, N.J.)

BMS-986016

Anti-lymphocyte activation gene 3 (LAG-3) therapy

Advanced melanoma

Proof-of-concept data showed preliminary efficacy for BMS-986016 in combination with Opdivo in patients previously treated with anti-PD-1/PD-L1 therapy; phase I/IIa data demonstrated activity in heavily pretreated patients, and analyses suggest it may be promising in identifying patients most likely to benefit from the therapeutic combination

6/6/17

Bristol-Myers Squibb Co. (Princeton, N.J.) and the French Cooperative Thoracic Intergroup (Paris)

Opdivo

Nivolumab

Previously treated unresectable malignant pleural mesothelioma patients

Results from the IFCT-1501 MAPS-2 trial of nivolumab alone or in combination with ipilimumab showed the 12-week disease control rate, the primary endpoint, was 44.4% with nivolumab, and 50% with nivolumab plus ipilimumab; the objective response rate was 18.5% vs. 25.9%, respectively, and the median overall survival was 10.4 months for nivolumab and is not yet reached for the combination

6/6/17

Calithera Biosciences Inc. (South San Francisco)

CB-1158

Arginase inhibitor

Advanced solid tumors

It demonstrated safety, tolerability and target engagement in a phase I trial; it was generally well-tolerated with no drug-related serious adverse events

6/6/17

Carsgen Therapeutics Ltd. (Shanghai)

CAR-GPC3

T-cell therapy

Relapsed or refractory hepatocellular carcinoma

The treatment was well-tolerated among the 13 patients; among five evaluable patients, one achieved partial response, two maintained stable disease and two developed progressive disease

6/6/17

Celgene Corp. (Summit, N.J.) and Agios Pharmaceuticals Inc. (Cambridge, Mass.)

Enasidenib

Oral IDHIFA

Relapsed or refractory acute myeloid leukemia and an IDH2 mutation

Data from the ongoing phase I dose-escalation and expansion study showed an overall response rate of 40.3%, including a complete response rate of 19.3%

6/7/17

Celldex Therapeutics Inc. (Hampton, N.J.)

Glembatu-mumab vedotin

Fully human monoclonal antibody-drug conjugate that targets glycoprotein NMB

Stage III/IV checkpoint inhibitor-refractory and BRAF/MEK inhibitor-refractory metastatic melanoma

Phase II data showed median overall survival for all patients was nine months, and seven of 62 patients (11%) experienced a confirmed response, and an additional three patients also experienced single time point partial responses; patients who experienced rash in cycle 1 experienced a more prolonged overall survival with a median of 15.8 months as compared to those who did not experience rash

6/6/17

Celldex Therapeutics Inc. (Hampton, N.J.)

Varlilumab

CD27-targeting immune-activating antibody

Advanced cancer

Data from the phase I portion of a phase I/II study testing varlilumab and Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed the combo was well-tolerated, without any evidence of increased autoimmunity or inappropriate immune activation

6/7/17

Celsion Corp. (Lawrenceville, N.J.)

GEN-1

IL-12 DNA plasmid vector formulated as a nanoparticle in a nonviral delivery system to cause the sustained local production and secretion of the interleukin-12 protein locoregionally at the tumor site

Advanced ovarian cancer

Updated phase Ib OVATION data, combining GEN-1 with the standard of care for newly diagnosed patients who will undergo neoadjuvant chemotherapy, showed there was a 100% disease control rate and an 86% objective response rate reported; patients experienced either a partial response (80%) or a complete response (20%)

6/6/17

Compugen Ltd. (Holon, Israel)

COM-701

Immuno-oncology antibody candidate targeting poliovirus receptor-related immunoglobulin (PVRIG)

Solid tumors

Analysis of multiple human solid tumors, including those of lung, kidney, head and neck and endometrial cancers, identified expression of PVRIG and its ligand PVRL2, suggested that patients with such cancers could benefit from treatment with COM-701

6/6/17

Cortice Biosciences Inc. (New York)

TPI-287

Microtubule stabilizing agent that penetrates the blood-brain barrier

Recurrent glioblastoma (GBM)

Final results from the dose-escalation portion of a phase I/II trial evaluating TPI-287 plus Avastin (bevacizumab, Roche Holding AG/Genentech Inc.) in 24 patients with GBM that had progressed beyond first-line treatment and who had not received prior bevacizumab showed that 12 of 20 patients evaluable for overall response achieved an objective response, including three complete and nine partial responses; 10 of 23 patients evaluable for overall survival (OS) achieved stable disease and one patient had progressive disease at first assessment for response; median progression-free survival was 5.5 months [95% CI 4.1, 8.2], and median OS was 13.4 months [95% CI 10.9, 17.9] after the occurrence of 87% of possible events

6/6/17

Corvus Pharmaceuticals Inc. (Burlingame, Calif.)

CPI-444

Orally administered antagonist of the adenosine A2A receptor

Renal cell carcinoma (RCC) and non-small-cell lung cancer (NSCLC)

Interim safety and efficacy results from the RCC and NSCLC expansion cohorts in its ongoing phase I/Ib study of CPI-444 as a single agent and in combination with Tecentriq (atezolizumab, Genentech Inc./Roche Holding AG) showed antitumor activity in patients resistant or refractory to prior treatment with anti-PD-(L)1 antibodies and patients with PD-L1-negative tumors; the study included data on 75 patients with RCC (n=30) or NSCLC (n=45); of these, 73% of RCC patients and 82% of NSCLC patients were resistant or refractory to prior therapy with anti-PD-(L)1 antibodies; of the RCC (n=19) and NSCLC (n=28) patients with archived samples available, 95% and 54% of patients, respectively, had PD-L1 negative tumors

6/6/17

Daiichi Sankyo Co. Ltd. (Tokyo)

DS-8201

Chemotherapy candidate comprising a humanized HER2 antibody attached to a topoisomerase I inhibitor payload by a tetrapeptide linker

Metastatic breast and gastric cancer

Dose-expansion portion of the phase I study in a subgroup analysis of HER2-expressing metastatic breast cancer patients pre-treated with ado-trastuzumab emtansine (T-DM1/Kadcyla, Genentech Inc./Roche Holding AG) and Perjeta (pertuzumab, Genentech/Roche) showed an overall response rate (ORR) of 46.7% (14 of 30 patients) and a disease control rate (DCR) of 100% (30 of 30 patients) to date; an ORR of 45.7% (16 of 35 patients) and DCR of 100% (35 of 35 patients) was observed in patients pre-treated with only T-DM1

6/6/17

Debiopharm International SA (Lausanne, Switzerland)

Debio 1347/CH-5183284

FGFR 1,2,3 selective inhibitor

Advanced solid tumors

A phase I dose-escalation study showed a manageable safety profile, and encouraging antitumor activity was seen in several tumor types, mainly in patients with FGFR2 or 3 gene alterations, including fusion events

6/2/17

Deciphera Pharmaceuticals LLC (Waltham, Mass.)

DCC-2618

Pan-KIT and PDGFR-alpha inhibitor

Advanced malignancies and gastrointestinal stromal tumors (GISTs)

Updated results from the completed dose-escalation stage of its ongoing phase I trial in patients with advanced malignancies encouraging disease control with prolonged stable disease and objective responses in 38 heavily pretreated patients with GIST; at doses of 100 mg or greater daily, DCC-2618 produced a disease control rate (DCR) of 78% at 12 weeks (n=23) and a DCR of 60% at 24 weeks (n= 15) in GIST patients with KIT and PDGFRalpha-driven disease

6/6/17

Dendreon Corp. (Seal Beach, Calif.)

Provenge

Sipuleucel-T

Hormone-sensitive prostate cancer and metastatic castrate-resistant (hormone-refractory) prostate cancer

Findings from a study evaluating Provenge showed an immune response to PA2024 and prostatic acid phosphatase; responses were observed as early as six weeks following Provenge treatment, persisted for a minimum of six months and were associated with improved overall survival

6/6/17

Effector Therapeutics Inc. (San Diego)

eFT-508

Inhibitor of mitogen-activated protein kinase-interacting serine/threonine kinase-1 (MNK-1) and MNK-2 in

Advanced solid tumors

Data from a phase I dose-escalation study showed it was well-tolerated at doses that produced a level of target inhibition sufficient to maximize antitumor activity in preclinical models; disease stabilization through at least two cycles of therapy (42 days) was seen in four of 11 patients (36%) treated at the two highest tolerated doses in that refractory patient population

6/6/17

Eisai Inc. (Woodcliff Lake, N.J.)

Lenvima

Lenvatinib; multiple receptor tyrosine kinase inhibitor

Unresectable hepatocellular carcinoma (uHCC)

The phase III REFLECT study showed noninferiority in overall survival (OS), the primary endpoint, and demonstrated statistically significant improvements on the secondary endpoints of progression-free survival (PFS), time to progression (TTP) and objective response rate (ORR), compared to Nexavar (sorafenib, Bayer AG); the median OS for patients treated with lenvatinib was 13.6 months compared to 12.3 months for sorafenib (HR: 0.92; 95% CI: 0.79 – 1.06); median PFS was 7.4 months with lenvatinib, with a median TTP of 8.9 months compared to median PFS of 3.7 months (HR: 0.66; 95% CI: 0.57 – 0.77; p<0.00001) and median TTP of 3.7 months on sorafenib (HR 0.63; 95% CI; 0.53 – 0.73; p<0.00001); lenvatinib showed an ORR of 24% compared to sorafenib, at 9% (odds ratio: 3.13; 95% CI: 2.15-4.56; p<0.00001)

6/6/17

Eisai Inc. (Woodcliff Lake, N.J.)

Lenvima

Lenvatinib; multiple receptor tyrosine kinase inhibitor

Metastatic endometrial cancer

Interim results from the first evaluable cohort of the phase Ib/II Study 111, of lenvatinib in combination with Keytruda (pembrolizumab, Merck & Co. Inc.), in previously treated patients (n=23), showed a confirmed objective response rate at week 24 of 47.8% (95% CI: 26.8 – 69.4) based on investigator assessment and 52.2% based on independent radiologic review (95% CI: 30.6 – 73.2); interim data on secondary endpoints showed median progression-free survival was 9.7 months (95% CI: 4.2 – NE) based on investigator assessment and was not reached by IRR; DCR was 95.7% (95% CI: 78.1 – 99.9) based on investigator assessment and 91.3% (95% CI: 72.0 – 98.9) based on IRR

6/6/17

Eli Lilly and Co. (Indianapolis)

Abemaciclib

CDK 4/6 inhibitor

Hormone-receptor-positive (HR+) human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer

Results from the phase III MONARCH 2 study showed that abemaciclib, in combination with fulvestrant, improved progression-free survival (PFS) compared to fulvestrant alone in women who relapsed or progressed following endocrine therapy (median PFS, 16.4 vs. 9.3 months, respectively, HR: 0.553; 95% CI: 0.449, 0.681, p<0.0000001); patients with measureable disease treated with abemaciclib plus fulvestrant achieved an objective response rate of 48.1% (3.5% complete response [CR]), compared to 21.3% (0% CR) in patients treated with fulvestrant alone

6/6/17

Endocyte Inc. (West Lafayette, Ind.)

EC-1169

Prostate-specific membrane antigen-targeted tubulysin prostate cancer therapy

Taxane-exposed, metastatic castration-resistant prostate cancer (mCRPC)

Endocyte will narrow the EC1169 development program to focus only on the mCRPC cohort

6/5/17

Endocyte Inc. (West Lafayette, Ind.)

EC-1456

Folate receptor-targeted tubulysin cancer therapy

Folate receptor-positive non-small-cell lung cancer

Is halting development after a phase Ib study evaluating the candidate, dosed at 6 mg/m2 twice weekly, failed to yield a sufficient benefit to support continued development

6/5/17

Essa Pharma Inc. (Houston)

EPI-506

Ralaniten acetate; androgen receptor N-terminal domain inhibitor

Prostate cancer

Early data from the phase I portion of the ongoing phase I/II trial showed prostate-specific antigen declines ranging from 4% to 29% have been observed in four patients at higher doses (≥1,280 mg); an analysis of human drug exposures compared to exposure levels derived from a xenograft model of castration-resistant prostate cancer showed that EPI-506 doses of ≥2,400 mg were beginning to reach the anticipated target range for significant tumor growth inhibition as indicated by the model

6/6/17

Five Prime Therapeutics Inc. (South San Francisco)

FPA-008

Cabiralizumab

Pigmented villonodular synovitis

Initial pharmacokinetics (PK), pharmacodynamics (PD), safety and efficacy data from the ongoing phase I/II trial in 11 patients showed that the PK and PD of cabiralizumab supported dosing at 4 mg/kg every two weeks or less frequently; no dose-limiting toxicities were observed at doses up to 4 mg/kg; the most frequently reported adverse events (AEs) of periorbital and eyelid edema, rash and pruritus were similar to AEs reported in other agents in the drug class

6/6/17

Five Prime Therapeutics Inc. (South San Francisco)

FPA-144

ADCC-enhanced, FGFR2b isoform-specific monoclonal antibody

FGFR2b+ gastric cancer

Updated data from the ongoing phase I trial showed it was well-tolerated in doses up to 15 mg/kg, with no reports of hyperphosphatemia; no dose-limiting toxicities or grade 4 or higher treatment-related AEs were seen during dose escalation, and maximum tolerated dose was not reached; FPA-144 monotherapy showed early evidence of antitumor effect in heavily pretreated patients; confirmed radiographic responses included five partial responses (four confirmed, one unconfirmed) in 21 patients and an objective response rate of 19%

6/6/17

Genentech Inc. (unit of Roche Holding AG; Basel, Switzerland)

Alecensa

Alectinib; kinase inhibitor

Anaplastic lymphoma kinase-positive advanced non-small-cell lung cancer

The phase III ALEX study showed that Alecensa reduced the risk of disease worsening or death by 53% compared to Xalkori (crizotinib, Pfizer Inc.) as first-line treatment (hazard ratio=0.47, 95% CI: 0.34-0.65, p<0.0001); median progression-free survival reported by the investigators, the primary endpoint of the study, was not reached in people who received Alecensa (95% CI: 17.7-not reached) vs. 11.1 months (95% CI: 9.1-13.1 months) in those who received crizotinib

6/6/17

Genentech Inc. (unit of Roche Holding AG; Basel, Switzerland), the Breast International Group, Breast European Adjuvant Study Team and Frontier Science Foundation

Perjeta regimen

Pertuzumab

HER2-positive early breast cancer

The phase III APHINITY study showed adjuvant treatment with the combination of Perjeta, Herceptin (trastuzumab) and chemotherapy reduced the risk of breast cancer recurrence or death by 19% compared to Herceptin and chemotherapy alone (HR=0.81; 95% CI 0.66-1.00, p=0.045); at three years, 94.1% of people treated with the Perjeta-based regimen did not have their breast cancer return compared to 93.2% treated with Herceptin and chemotherapy

6/6/17

Glycomimetics Inc. (Rockville, Md.)

GMI-1271

E-selectin antagonist

Acute myeloid leukemia (AML)

Data from the phase II portion of its ongoing phase I/II trial showed high remission and low mortality rates; among the 54 of 79 relapsed/refractory AML patients enrolled in the trial for whom data are available, the overall response rate was 41%, higher than historical controls, and the 60-day induction-related mortality rate was 7%, lower than historical controls; oral mucositis was seen at low rates and severity, with only one grade 3/4 event observed; median overall survival time for phase I patients was 7.6 months

6/6/17

Halozyme Therapeutics Inc. (San Diego)

PEGPH20

Pegylated formulation of its recombinant human hyaluronidase

Pancreatic cancer

A randomized, multicenter phase II trial met its primary endpoints and the key secondary endpoint of progression-free survival (PFS) in patients with high levels hyaluronan (HA-High); PEGPH20 plus standard chemotherapy of Abraxane and gemcitabine improved median PFS by 77% over chemotherapy alone in HA-High patients; an exploratory endpoint of objective response rate by RECIST v1.1 in the HA-High population was 45% in the PEGPH20 plus chemotherapy arm, compared to 31% in patients who received chemotherapy alone

6/6/17

Helsinn Group (Lugano, Switzerland) and MEI Pharma Inc. (San Diego)

Pracinostat

Oral histone deacetylase (HDAC) inhibitor

Acute myeloid leukemia

Findings from a genetic mutation analysis of patients in a phase II study of pracinostat and azacitidine showed a correlation between genetic mutations in the DNA methylation pathway and clinical response; the most frequent mutations, occurring in 37% of samples studied (15/41), were found in the DNA methylation pathway, including DNMT3A, IDH1, IDH2 and TET2; patients with these mutations had a complete response (CR) rate of 60%, an improvement (p=0.027) over patients with the wild-type genes (22%); in the phase II analysis, median overall survival was roughly equivalent in patients with mutations typically associated with de novo AML (18.1 months) and secondary AML (17.7 months)

6/6/17

Hutchison China Meditech Ltd. (Hong Kong)

Fruquintinib

VEGFR kinase inhibitor

Locally advanced or metastatic colorectal cancer

Results from the pivotal phase III FRESCO trial showed that the study in patients in China met all primary and secondary endpoints, including significant improvements in overall and progression-free survival (PFS); the primary endpoint of median overall survival was 9.3 months in the fruquintinib group vs. 6.57 months in the placebo group, with a hazard ratio of 0.65 (two-sided p<0.001); PFS was 3.71 months in the fruquintinib group vs. 1.84 months in the placebo group, with a hazard ratio of 0.26 (two-sided p<0.001)

6/7/17

Immune Design Corp. (Seattle)

CMB-305 monotherapy

Prime-boost vaccine approach against NY-ESO-1-expressing tumors

Soft tissue sarcoma

Data showed median overall survival (OS) was not reached in the trial, with an OS rate at 12 and 18 months of 83% and 76%, respectively; a disease control rate of 64% (16/25) was observed, including durable tumor growth arrest in patients who had evidence of disease progression at study entry; CMB-305 generated a strong and broad anti-NY-ESO-1 immune response in more than 50% of the patients, with 32% experiencing an integrated response

6/6/17

Immunocellular Therapeutics Ltd. (Los Angeles)

ICT-121

Dendritic cell-based immunotherapy targeting CD133

Recurrent glioblastoma

Data from the phase I trial showed that six of 20 patients are alive, as of the April 2017 cut-off, with survival ranging from 10 months to 24 months

6/7/17

Immunocore Ltd. (Oxford, U.K.)

IMCgp100

Bispecific biologic designed to redirect T cells against the melanocyte-associated antigen gp100

Metastatic uveal melanoma

The intrapatient dose-escalation phase I trial recruited 19 patients and showed a median progression-free survival (PFS) of 5.6 months, compared with historical median PFS ranging from 2.6 to 2.8 months; PFS at 24 weeks was 57%, compared with 19% to 27% in similar studies in the indication

6/6/17

Incyte Corp. (Wilmington, Del.) and Bristol-Myers Squibb Co. (New York)

Epacadostat

Oral selective IDO1 enzyme inhibitor

Multiple advanced solid tumors

Updated data from the ongoing phase I/II ECHO-204 trial evaluating epacadostat in combination with the BMS' Opdivo (nivolumab) showed that patients with treatment-naïve advanced melanoma treated with epacadostat (100 mg or 300 mg) plus nivolumab (n=40) showed a combined objective response rate (ORR) of 63% (25/40), including two complete responses (CRs) and 23 partial responses (PRs); in previously treated patients with squamous cell carcinoma of the head and neck (SCCHN) who were treated with epacadostat (100 mg or 300 mg) plus nivolumab (n=31), the combined ORR was 23% (7/31), including one CR and six PRs, and the combined DCR was 61% (19/31)

6/6/17

Janssen Research & Development LLC (unit of Johnson and Johnson; New Brunswick, N.J.)

Darzalex

Daratumumab

Multiple myeloma (MM)

Pivotal phase III CASTOR and POLLUX studies showed that Darzalex in combination with bortezomib and dexamethasone (dex), or Revlimid (lenalidomide, Celgene Corp.) and dex, improved progression-free survival (PFS) and overall response rate (ORR) for previously treated patients with MM; daratumumab, in combination with bortezomib and dex, reduced the risk of disease progression or death by 55% (p=0.0053) in patients with high-risk cytogenetics (n=44) compared to dex alone; the combo resulted in an ORR of 82% vs. 62% (p=0.2028) for dex alone, with very good partial response or better achieved in 64% vs. 35% of patients and complete response or better achieved in 34% vs. 9% of patients

6/7/17

Janssen Research & Development LLC (unit of Johnson and Johnson; New Brunswick, N.J.)

Darzalex

Daratumumab

Multiple myeloma

Phase Ib data of daratumumab in combination with Kyprolis (carfilzomib, Amgen Inc.), lenalidomide and dex showed that the overall safety profile of the combo was consistent with the known safety profiles of the individual agents

6/7/17

Janssen Research & Development LLC (unit of Johnson and Johnson; New Brunswick, N.J.)

Imbruvica

Ibrutinib; Bruton's tyrosine kinase inhibitor

Relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma

Follow-up data for up to four years from the pivotal phase III RESONATE trial (PCYC-1112) of Imbruvica vs. ofatumumab (Arzerra, Genmab A/S) showed that, at a median follow-up of 44 months, results showed three-year progression-free survival of 59% vs. 3% with ibrutinib vs. ofatumumab, respectively; with longer follow-up, objective response rate has reached 91%, with a CR of 9%; the adverse events profile of ibrutinib included major hemorrhage, grade ≥3 atrial fibrillation and grade ≥3 hypertension occurring in 6%, 6% and 8% of patients, respectively, over a follow-up period of up to four years

6/7/17

Jounce Therapeutics Inc. (Cambridge, Mass.)

JTX-2011

Monoclonal antibody targeting ICOS

Advanced solid tumors

Preliminary phase I data from the ICONIC study showed it was well-tolerated in doses up to 0.3 mg/kg as a monotherapy and in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.); most adverse events were grade 1 or 2, with the most common being chills, decreased appetite, nausea and fever

6/6/17

Juno Therapeutics Inc. (Seattle)

JCAR-017

Chimeric antigen receptor T-cell candidate targeting CD19

Relapsed and refractory aggressive B-cell non-Hodgkin lymphoma

Updated data from the phase I TRANSCEND trial showed that, as of data cutoff of May 4, the overall response rate (ORR) was 86% (38/44) and the complete response (CR) was 59% (26/44); three-month ORR was 66% (21/32) and CR was 50% (16/32); for the full dataset, across dose levels, the best ORR was 76% (41/54) and CR was 52% (28/54); three-month ORR was 51% (21/41) and CR was 39% (16/41)

6/6/17

Kite Pharma Inc. (Santa Monica, Calif.)

KTE-C19

Axicabtagene ciloleucel

High burden relapsed/refractory acute lymphoblastic leukemia

Reported that 73% of patients in the phase I ZUMA-3 trial achieved complete remission, including those with incomplete or partial recovery of bone marrow; all responders tested negative for minimal residual disease (MRD). No dose-limiting toxicities occurred

6/6/17

Lion Biotechnologies Inc. (San Carlos, Calif.)

LN-144

Tumor-infiltrating lymphocyte (TIL) technology

Metastatic melanoma

Data from 16 patients enrolled in the first cohort of its ongoing phase II study showed a 29% objective response rate among evaluable patients, including one complete response (CR) continuing beyond 15 months post-administration of a single TIL treatment; 77% of patients had reduction in target tumor size, and the mean time to first response was 1.6 months, with the CR developing at six months

6/6/17

Loxo Oncology Inc. (Stamford, Conn.)

LOXO-101

Larotrectinib

TRK fusion cancers

Interim clinical data from all three ongoing trials in patients whose tumors harbor tropomyosin receptor kinase (TRK) fusions demonstrated a 76% confirmed objective response rate across tumor types

6/7/17

Mabvax Therapeutics Holdings Inc. (San Diego)

MVT-5873

Antibody

Advanced pancreatic cancer and other CA19-9 positive cancers

Results from its phase I trial showed that, as a single agent, it appeared safe and well-tolerated in patients at biologically active doses; in the dose-escalation safety trial of 32 patients, one patient achieved a complete response and 11 achieved stable disease; after completing the first treatment cycle of 28 days, 40% of patients had a sustained decrease of 50% or more in serum tumor marker CA19-9 levels; patients with these higher reductions in CA19-9 levels continued treatment for a median of four cycles (range two to 9.75+), compared to one cycle (range 0.25 to three) for patients with less than 50% decrease; 12 of the 32 patients achieved stable disease, and one patient achieved a complete response

6/7/17

Merck & Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab

Unresectable or metastatic melanoma

Updated longer-term overall survival data from the phase III trial, KEYNOTE-006, showed sustained superior survival outcomes for Keytruda monotherapy compared to ipilimumab in treatment-naïve patients or in those who received one prior line of therapy; Keytruda was associated with a 30% improvement in survival: 50% in the Keytruda group were alive nearly three years after starting treatment, compared to 39% in the ipilimumab group; Keytruda doubled the rate of progression-free survival at 33.9 months: 31% vs. 14% for ipilimumab

6/5/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Locally advanced triple-negative breast cancer (TNBC) or hormone receptor-positive/HER2-negative (HR+/HER2-) breast cancer

Merck, along with Quantumleap Healthcare Collaborative, reported results from the phase II I-SPY 2 TRIAL investigating Keytruda combined with standard therapy as a neoadjuvant treatment; the addition of Keytruda increased the estimated pathologic complete response (pCR) rate nearly threefold in patients with TNBC (60% vs. 20%) and in patients with HR+/HER2- breast cancer (34% vs. 13%) compared to standard therapy

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Advanced microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors

Findings from KEYNOTE-164 and KEYNOTE-158, two phase II studies, showed objective response rates (ORR), regardless of histology, between 28% and 38% across patients with MSI-H/dMMR colorectal cancer and other advanced MSI-H/dMMR solid tumors, respectively

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Metastatic non-small-cell lung cancer (NSCLC) with high levels of PD-L1

Updated data from KEYNOTE-024 showed continued overall survival (OS) benefit of Keytruda compared to chemotherapy in the first-line treatment; the study demonstrated a reduction in the risk of death by 37% for Keytruda compared to chemotherapy, based on 19 months of median follow-up; specifically, data showed an 18-month OS rate of 61.2% in the Keytruda group compared to 43% in the chemotherapy group; the 12-month OS rate was 70.3% in the Keytruda group compared to 54.8% in the chemotherapy group.

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Advanced nonsquamous non-small-cell lung cancer

Updated results from KEYNOTE-021, cohort G1, which studied Keytruda combined with pemetrexed and carboplatin (pem/carbo), irrespective of PD-L1 expression, demonstrated continued benefit, including an overall response rate (ORR) of 56.7% compared to 30.2% with pem/carbo alone; progression-free survival was longer in the Keytruda combination group, with the median not reached

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Advanced gastric or gastroesophageal junction adenocarcinoma

Findings from cohort 1 of the phase II registrational KEYNOTE-059 trial in previously treated patients showed an overall response rate of 11.6% in patients treated with Keytruda who had received two or more prior lines of treatment, with higher response rates among patients with PD-L1-positive tumors

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Advanced nonsquamous non-small-cell lung cancer

In an ongoing phase I/II ECHO-202 trial, Keytruda combined with epacadostat (Incyte Corp.) showed an ORR of 35% among all patients, irrespective of PD-L1 status

6/6/17

Merck and Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; anti-PD-1 therapy

Advanced urothelial carcinoma

In the phase III KEYNOTE-045 trial, Keytruda showed continued overall survival (OS) over chemotherapy as a second-line treatment for patients post-platinum failure; specifically, Keytruda improved OS compared to chemotherapy – reducing the risk of death by 30% – and, in the first-line setting, it demonstrated an ORR of 29%

6/6/17

Miragen Therapeutics Inc. (Boulder, Colo.)

MRG-106

Inhibitor of microRNA-155

Mycosis fungoides form of cutaneous T-cell lymphoma

Phase I data showed that 18 participants (95%) showed improvement in either individual lesion or total skin disease, as measured by maximal change in Composite Assessment of Index Lesion Severity (CAILS) or modified Severity Weighted Assessment Tool (mSWAT)

6/7/17

Nanobiotix SA (Paris)

NBTXR-3

Injectable nanoparticles

Head and neck cancer

Results from the phase I/II trial showed it has a good safety profile, with no adverse events related to the product; it was shown to be feasible, with the product remaining in the tumor from the first day until the last day of radiotherapy; initial efficacy data showed an overall response rate of 91% (10 of 11 evaluable patients), with complete response in seven of nine patients at a dose level of 10% or more

6/7/17

Nantkwest Inc. (Culver City, Calif.) and Nantworks

Nant

Vaccine; personalized, molecularly informed therapy guided by Nanthealth's GPS Cancer test

Pancreatic cancer

The Nant cancer vaccine program was expanded to target a number of additional tumor types

6/7/17

Nektar Therapeutics Inc. (San Francisco)

NKTR-214

CD122-biased agonist

Stage IV renal cell carcinoma

New phase I data showed confirmed partial responses observed in three of four patients who were immuno-oncology treatment-naïve and who experienced stable disease with tumor shrinkage while on NKTR-214 monotherapy and then received sequential treatment with Opdivo (nivolumab, Bristol-Myers Squibb Co.); data from blood and tumor samples show that NKTR-214 increases immune cells in the blood and tumor microenvironment even in subjects who have failed multiple prior immunotherapeutic agents

6/6/17

Newlink Genetics Corp. (Ames, Iowa)

NLG-802

Indoximod

Metastatic castration-resistant prostate cancer

Results from a phase II investigator-initiated study with indoximod in combination with Provenge (sipuleucel-T) showed the indoximod arm showed a statistically significant improvement in radiographic progression-free survival when compared to placebo (10.3 months vs. 4.1 months) and was well-tolerated

6/6/17

Newvac LLC (San Diego)

Quisinostat

Selective oral HDAC1 inhibitor

Ovarian cancer

Phase II trial demonstrated its safety profile and high efficacy against platinum-resistant ovarian cancer in combination with paclitaxel and carboplatin; the primary efficacy endpoint of the study, objective response rate, was 51.6%, which Newvac said greatly exceeded the value of 30% expected by study protocol

6/27/17

Novartis AG (Basel, Switzerland)

Tafinlar + Mekinist

Dabrafenib + trametinib

BRAF V600 mutation-positive metastatic melanoma

Phase II results showed a durable survival benefit for some patients when treated with the combination; findings from the five-year analysis of the trial, BRF113220, in which 20 patients remained on study, including seven in the Tafinlar monotherapy arm and 13 in the combo arm, showed that overall survival with combination therapy is superior to monotherapy

6/6/17

Novartis AG (East Hanover, N.J.)

Kisqali

Ribociclib

Hormone receptor positive, human epidermal growth factor receptor-2 negative advanced or metastatic breast cancer

Phase III MONALEESA-2 data of Kisqali plus letrozole in postmenopausal women showed that, after an additional 11 months of follow-up, a median progression-free survival of 25.3 months for Kisqali plus letrozole and 16 months for letrozole alone was observed (p<0.0001); after two years of treatment, the progression-free survival rate was 54.7% in the Kisqali plus letrozole arm compared to 35.9% in patients treated with letrozole alone

6/5/17

Onconova Therapeutics Inc. (Newtown, Pa.)

Rigosertib

A dual P13 kinase and pololike kinase inhibitor

Higher-risk myelodysplastic syndromes

Results of a phase II study with rigosertib, after failure of hypomethylating agents (HMAs), showed treatment with rigosertib resulted in a reduction in bone marrow blast count, including complete bone marrow responses, confirming findings in earlier studies

6/6/17

Orbus Therapeutics Inc. (Palo Alto, Calif.)

Eflornithine

Cytostatic agent

Anaplastic astrocytoma

Detailed the design of the pivotal phase III STELLAR study testing it in patients whose cancer has recurred following radiation and adjuvant chemotherapy

6/6/17

Peregrine Pharmaceuticals Inc. (Tustin, Calif.)

Bavituximab

Phosphatidylserine-targeting monoclonal antibody

Previously treated locally advanced or metastatic nonsquamous non-small-cell lung cancer

New data from its phase III SUNRISE trial demonstrated that patients in the study's docetaxel plus bavituximab treatment arm with a pre-treatment PD-L1 expression level on tumor cells of <1% (TC0) had a median overall survival (mOS) of 12.1 months compared to a mOS of 6.1 months for patients with PD-L1 expression ≥1% (TC1/2/3) (HR = 0.42 p=0.007)

6/6/17

Prima Biomed Ltd. (Sydney)

IMP-321

LAG-3Ig

Hormone receptor-positive metastatic breast cancer

Data from the safety run-in stage of its phase IIb AIPAC (Active Immunotherapy PAClitaxel) study showed it was safe and well-tolerated at both the 6-mg and 30-mg dose levels; the 30-mg dose showed a stronger immune response and was determined to be the recommended phase II dose for the ongoing randomized phase of 226 patients

6/6/17

Puma Biotechnology Inc. (Los Angeles)

PB-272

Neratinib

HER2-positive metastatic breast cancer that metastasized to the brain

Data from a subgroup of patients (n=37) without prior lapatinib treatment in the third cohort from the ongoing phase II trial showed that 49% of the patients experienced a central nervous system (CNS) objective response, using composite criteria, with a CNS response rate of 24% using Response Assessment in Neuro-Oncology-Brain Metastases criteria; median time to CNS progression was 5.5 months and median overall survival was 13.5 months, although 49% of patients remain alive and survival data are immature

6/6/17

Radius Health Inc. (Waltham, Mass.)

Elacestrant (RAD-1901)

Oral selective estrogen receptor degrader

Estrogen receptor positive (ER+) breast cancer

Data from an ongoing phase I study showed that 22 patients met the RECIST measurable disease criteria at baseline, and there were five confirmed partial responses; elacestrant was well-tolerated, with the most common adverse events being low grade nausea and dyspepsia

6/6/17

Rexahn Pharmaceuticals Inc. (Rockville, Md.)

RX-3117

Oral small-molecule nucleoside compound

Metastatic bladder cancer

Interim data from an ongoing phase IIa trial showed two of the 10 treated with RX-3117 showed progression-free survival of more than six months, and one remains in the study with stable disease at 175 days; two patients had reductions of 19% and 15% in tumor size

6/6/17

Roche Holding AG (Basel, Switzerland)

Perjeta

Pertuzumab

HER2-positive breast cancer

Data from the phase III Aphinity trial showed that to add Perjeta on top of Herceptin (trastuzumab) after surgery may help, but the benefit was not especially powerful; at an early follow-up of three years, 93.2% of women given Herceptin alone had not developed invasive disease compared with 94.1% of those who received Perjeta plus Herceptin, a difference of just 1%; adding Perjeta to Herceptin lowered the chance of developing invasive breast cancer by 19% compared to trastuzumab alone and, at a median follow-up of almost four years, 171 (7.1%) of patients in the Perjeta group had developed invasive breast cancer, compared to 210 (8.7%) patients in the placebo group

6/7/17

Sanofi SA (Paris) and Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.)

REGN-2810

Checkpoint inhibitor targeting PD-1

Advanced cutaneous squamous cell carcinoma

Pooled data from two expansion cohorts of the phase I trial showed the investigator-assessed overall response rate was 46.2% (12 of 26 patients) and the disease control rate was 69.2% (18 of 26 patients); median progression-free survival and overall survival were not reached at the data cutoff date, with a median follow-up of 6.9 months (range: 1.1 to 13.8 months; ongoing)

6/6/17

Seattle Genetics Inc. (Bothell, Wash.) and Astellas Pharma Inc. (Tokyo)

Enfortumab vedotin (ASG-22ME)

Antibody-drug conjugate (ADC) targets Nectin-4

Metastatic urothelial cancer

Updated phase I data showed, of the 71 patients evaluated for response, 29 (41%) had an objective response, including three (4%) complete responses and 26 (37%) partial responses; disease control was achieved in 51 patients (72%); the preliminary estimate of median duration of response for all patients was 24 weeks; in 30 patients treated at the RP2D level, 16 (53%) had an objective response, including one (3%) complete response and 15 (50%) partial responses; disease control was achieved for 22 patients (73%)

6/6/17

Sierra Oncology Inc. (Vancouver, British Columbia)

SRA-737

Checkpoint kinase 1 inhibitor

Advanced cancer

Dose escalation will continue in the trial until a maximum tolerated dose is reached, in parallel with ongoing cohort expansion enrollment; SRA-737 has been well-tolerated to date, with no report of grade 2 or higher adverse events related to the study drug; no dose-limiting toxicities were observed, and a maximum tolerated dose was not reached

6/7/17

Sirtex Medical Inc. (Woburn, Mass.)

SIR-Spheres

Y-90 resin microspheres

Advanced hepatocellular carcinoma (HCC)

Results of the 360-patient SIRveNIB study found that the Asian patients with locally advanced HCC who were treated with Y-90 resin microspheres had a significantly better tumor response rate of 16.5% compared to 1.7% for Nexavar (sorafenib, Bayer AG) (p<0.001) in the intent-to-treat analysis, and 23.1% for selective internal radiation therapy compared to 1.9% (p<0.001) in the treated population, which represents the patients who actually received their allocated treatment

6/6/17

Syndax Pharmaceuticals Inc. (Waltham, Mass.)

Entinostat

Oral histone deacetylase (HDAC) inhibitor

Non-small-cell lung cancer

Results from the melanoma cohort of the ongoing phase II ENCORE 601 trial of entinostat in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed the first cohort of 13 melanoma patients who had progressed on or after prior immune checkpoint inhibitor therapy met the pre-specified objective response criteria; data from the first cohort of patients indicate that four patients achieved an objective response by irRECIST criteria and of those, two patients had stable disease and two had progressive disease as best response to their prior anti-PD-1 therapy prior to progressing, with a median duration on prior anti-PD-1 therapy of 4.9 months (range 2.7-12.5)

5/18/17

Targovax ASA (Oslo, Norway)

TG-01

Immunotherapy

Resected pancreatic cancer

Phase I/II data showed that TG-01 generated early immune response in 89% of patients (17/19) in combination with GM-CSF, and 95% (18/19) showed immune activation in either DTH or PMBC tests; median overall survival was 33.1 months

6/7/17

Tesaro Inc. (Waltham, Mass.)

Zejula

Niraparib

Recurrent, platinum-resistant ovarian cancer or triple negative breast cancer

Initial data from the phase I/II Topacio trial of Zejula plus Keytruda (pembrolizumab, Merck & Co. Inc.) further characterized the positive, durable treatment effects of Zejula and the clinical benefit in women who have residual disease following treatment with platinum-based chemotherapy; in the dose escalation phase, a disease control rate of 69% was observed in patients with platinum-resistant ovarian cancer

6/6/17

Tesaro Inc. (Waltham, Mass.)

TSR-042

Monoclonal antibody targeting PD-1

Various tumors

The phase I study is now complete, and no dose limiting toxicities were observed; among the 21 heavily pretreated patients in Part 1 of the study, two had a partial response and five had stable disease

6/6/17

TG Therapeutics Inc. (New York)

TG-1101

Ublituximab; CD20 antigen inhibitor

Previously treated high-risk chronic lymphocytic leukemia

The phase III Genuine trial of TG-1101 plus Imbruvica (ibrutinib, Abbvie Inc./Johnson & Johnson) met its primary endpoint, demonstrating a statistically significant improvement in overall response rate compared to ibrutinib alone in both the intent-to-treat and the treated populations, (p=0.001) and (p < 0.001) respectively

6/6/17

Tracon Pharmaceuticals Inc. (San Diego)

TRC-105

Antibody to endoglin

Soft tissue sarcoma, angiosarcoma, renal cell carcinoma

Phase Ib data showed that patients who had greater than a 10% reduction in tumor volume following treatment with TRC-105 and Votrient (pazopanib, Glaxosmithkline plc) were significantly more likely to have lower baseline levels of soluble intracellular adhesion molecule-1 (p=0.018) and thrombospondin-2 (p=0.041); in another phase Ib study, data showed patients with a partial response by RECIST 1.1 following treatment with TRC-105 and Inlyta (axitinib, Pfizer Inc.) were more likely to have lower levels of soluble osteopontin (p=0.026) and higher levels of soluble transforming growth factor-beta receptor III (p=0.0028)

6/7/17

Tyme Technologies Inc. (New York)

TYME-88

Breaks down the cellular defenses of tumors, leading to tumor cell death

Recurrent, non-metastatic prostate cancer

Interim data from an ongoing phase Ib/II trial showed improvement in the study's primary endpoints, including prevention of radiographically detectable lesions (100%), reduction in circulating tumor cells (87.5%), stabilization or improvement in PSA doubling time (87.5%), delay of subsequent toxic therapy, including ADT or chemotherapy (100%), and avoidance of worsening patient-reported outcomes (all subjects)

6/7/17

Tyme Technologies Inc. (New York)

TYME-88

Breaks down the cellular defenses of tumors, leading to tumor cell death

Refractory pancreatic cancer

Results from a phase I trial in 11 patients indicated a reduction in tumor size in three subjects, including one complete response with progression-free survival (PFS) of at least six months and two partial responses with one known PFS of 15 weeks; eight of the 11 had stable disease for six to 61 weeks

6/7/17

VBL Therapeutics Ltd. (Tel Aviv, Israel)

VB-111

Ofranergene obadenovec

Recurrent glioblastoma (rGBM)

New data demonstrated that treatment with VB-111 induced durable tumor regression and attenuation of tumor growth; 12-month overall survival was 57% in the VB-111 continuous exposure cohort, compared with only 24% in historical pooled Avastin trials (p=0.03); VBL analyzed the tumor growth kinetics in all rGBM patients who participated in the VB-111 phase II trial, with those new data demonstrating that tumor growth kinetics were significantly attenuated upon longer treatment with VB-111

6/6/17

Xbiotech Inc. (Austin, Texas)

Hutruo

MABp1 antibody

Advanced colorectal cancer

Phase III data showed that, of the patients receiving at least one dose of study drug who completed the eight-week treatment regimen, 40% achieved the primary endpoint, which was nearly double that of placebo patients (40% vs. 23%, p = 0.0003); patients in that population that achieved the primary endpoint had a median survival of 11.7 months vs. 5.7 months for those that did not (p<0.0001)

6/2/17

Ymabs Therapeutics Inc. (New York)

131I-burtomab

CD276 antigen inhibitor

Refractory leptomeningeal metastasis from neuroblastoma

Top-line results from a pivotal study showed a 58-month average survival for the patients treated with 131I-burtomab in the study, compared to an average of 4.7 months and no long-term survival or cure, for a contemporary cohort in the Central German Childhood Cancer Registry; after more than a decade of follow-up, data show more than 40% overall long-term survival, indicating that the treated children have been cured

6/6/17

Ziopharm Oncology Inc. (Boston)

Ad-RTS-hIL-12

Gene therapy

Recurrent or progressive glioblastoma

Updated results from its phase I study of Ad-RTS-hIL-12 + veledimex showed that, at the cutoff date of May 24, that median overall survival of all patients was maintained at 12.5 months, with a mean follow-up time of 9.2 months

6/6/17

Zymeworks Inc. (Vancouver, British Columbia)

ZW-25

Azymetric bispecific antibody targeting two domains of the HER2 receptor

Advanced HER2-expressing cancers

Results from the dose-escalation portion of the first-in-human study showed it was well-tolerated at all dose levels evaluated with single-agent antitumor activity

6/6/17

Notes

The date indicated refers to the BioWorld issue in which the news item can be found.

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