On December 14, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported safety and promising activity of BPX-601 in Part 1 of a Phase 1/2 dose-escalation study in patients with advanced, metastatic pancreatic cancer expressing PSCA (prostate stem cell antigen) (Press release, Bellicum Pharmaceuticals, DEC 14, 2018, View Source [SID1234532061]). BPX-601 is a novel GoCAR-T cell candidate incorporating Bellicum’s co-activation domain, iMC, designed to boost T cell proliferation and persistence via administration of rimiducid. Data were reviewed during an oral presentation at the European Society for Medical Oncology Immuno-Oncology Congress (ESMO-IO) held in Geneva, Switzerland from December 13-16.

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"These initial results in advanced pancreatic cancer suggest that having greater control over the expansion and persistence of therapeutic cells may provide unique treatment benefits. We have observed promising initial clinical activity with BPX-601 in patients with one of the most challenging solid tumors, where there is a critical need for better treatments," commented Carlos R. Becerra, M.D., lead study investigator and oncologist and medical director of the Innovative Clinical Trials Center at Baylor University Medical Center at Dallas. "I am looking forward to the next phase of our study, which allows for a standard lymphodepletion regimen and administration of multiple doses of rimiducid to further increase the expansion and persistence of BPX-601 cells and the potential for greater impact against pancreatic and other tumor types."

Study Overview
A total of 12 patients with advanced metastatic pancreatic cancer expressing PSCA were treated with escalating doses of BPX-601 cells in a 3+3 design. Nine of 12 patients received a single dose of rimiducid following BPX-601 treatment to evaluate its effect on cell expansion and persistence. Patients in the study received a reduced conditioning regimen consisting of cyclophosphamide only.

Interim Results:

The administration of BPX-601 without rimiducid resulted in limited expansion of cells, but the cells did not persist; A single dose of rimiducid 7 days following BPX-601 administration resulted in significant expansion of cells (three-fold to 20-fold) in four patients in spite of a reduced conditioning regimen; BPX-601 cells persisted longer than three weeks in three patients after a single dose of rimiducid; Increases in key cytokine levels were observed in patients receiving higher doses of BPX-601 cells and rimiducid; 4 of 6 efficacy-evaluable patients treated with BPX-601 and a single dose of rimiducid had stable disease, with two patients demonstrating tumor shrinkage greater than 20 percent; No cytokine release syndrome or neurotoxicity of any grade was reported; The most frequently observed AEs were consistent with those experienced by advanced cancer patients undergoing cytotoxic chemotherapy or other cancer immunotherapies; Patients continue to be evaluated in the study.
"These results provide the first clinical evidence suggesting that our GoCAR-T technology drives expansion and persistence of therapeutic T cells in patients," said Rick Fair, President & CEO of Bellicum Pharmaceuticals. "We are excited about BPX-601 and the broader application of our technology intended to improve the safety and efficacy of cell therapies. With these promising results, we’re preparing to begin Part 2 of the BPX-601 study, adding patients with prostate and gastric cancers, allowing multiple doses of rimiducid and a more complete conditioning regimen. We’re looking forward to reporting updated results in 2019."

About BPX-601
BPX-601, the Company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence, and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, prostate and gastric cancers.

On December 14, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that Janssen Pharmaceutical K.K. has submitted a supplemental new drug application (sNDA) to the Ministry of Health, Labor and Welfare (MHLW) in Japan, for the use of daratumumab (DARZALEX) in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT) (Press release, Genmab, DEC 14, 2018, View Source [SID1234532058]). The application will receive a priority review. The submission of the application triggers a milestone payment of USD 2 million to Genmab from Janssen. In February 2018, the MHLW granted Orphan Drug Designation to DARZALEX for patients with newly diagnosed multiple myeloma. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"We are extremely pleased that daratumumab in front line multiple myeloma has now been submitted in Japan. Should this submission be approved, it would bring an exciting new therapeutic option to Japanese multiple myeloma patients in need," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The submission was based on data from the Phase III ALCYONE study that showed a reduction of the risk of disease progression or death by 50 percent in newly diagnosed ASCT ineligible multiple myeloma patients when daratumumab is combined with bortezomib, melphalan and prednisone. This data was presented as a Late-Breaking Abstract at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and published in The New England Journal of Medicine in December, 2017.

About the ALCYONE study
This Phase III study (NCT02195479) is a randomized, open-label, multicenter study and includes 706 newly diagnosed patients with multiple myeloma who are ineligible for ASCT. Patients were randomized to receive 9 cycles of either VMP [bortezomib (a proteasome inhibitor), melphalan (an alkylating chemotherapeutic agent) and prednisone (a corticosteroid)] combined with daratumumab, or VMP alone. In the daratumumab treatment arm, patients received 16 mg/kg of daratumumab once weekly for six weeks (cycle 1; 1 cycle = 42 days), once every three weeks from cycles 2 to 9, once every 4 weeks from cycle 9 until disease progression. The primary endpoint of the study is progression free survival (PFS).

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in Japan, after leukemia and lymphoma.2 Approximately 8,200 new patients were projected to be diagnosed with multiple myeloma and approximately 4,200 people were projected to die from the disease in Japan in 2017.2 Globally, it was estimated that 124,225 people would be diagnosed and 87,084 would die from the disease in 2015.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4 Patients who relapse after treatment with standard therapies, including proteasome inhibitors or immunomodulatory agents, have poor prognoses and few treatment options.5

About DARZALEX(daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,7,8,9,10,

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, myelodysplastic syndromes, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On December 14, 2018 Leap Therapeutics, Inc. (NASDAQ:LPTX) reported that its clinical data from its ongoing Phase I/II study of TRX518 in combination with gemcitabine, KeytrudaÒ (pembrolizumab), or OpdivoÒ (nivolumab) in patients with advanced solid tumors at the European Society for Molecular Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress 2018 (Press release, Leap Therapeutics, DEC 14, 2018, View Source [SID1234532057]). In addition, Leap provided the top-line final data from the Phase I/II study of DKN-01 in combination with gemcitabine and cisplatin chemotherapy in patients with advanced biliary tract cancer.

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Leap will host a conference call and webcast on Monday, December 17, 2018 at 8:30 AM US Eastern Time with Jason J. Luke, MD, Assistant Professor of Medicine, Pritzker School of Medicine at the University of Chicago and Todd M. Bauer, MD, Sarah Cannon Research Institute/Tennessee Oncology PLLC, TN. Drs. Luke and Bauer will discuss patient outcomes and provide additional perspectives about the TRX518 program.

TRX518

TRX518 is unique among Glucocorticoid-Induced TNF Receptor (GITR) agonist antibodies for its aglycosyl design, permitting activation of GITR signaling without depleting CD8+ T-effector cells. In cancer patients, TRX518 has been shown to increase CD8+ T-effector cell infiltrate and the expression of granzyme B, as well as decrease CD4+ T-regulatory cell infiltrate. The dual function of TRX518 is designed to enhance the anti-tumor activity of chemotherapies and immune checkpoint inhibitors, such as anti-PD-1/PD-L1 antibodies.

TRX518 Clinical Data Presented at ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2018

The clinical trial is a continuation of the TRX518 multi-dose monotherapy study that has been expanded with three combination study arms to evaluate lower (2 mg/kg loading dose with 1 mg/kg maintenance doses) and higher (4 mg/kg loading dose with 1 mg/kg maintenance doses) dose levels of TRX518 in combination with gemcitabine chemotherapy or Keytruda or Opdivo anti-PD-1 immune checkpoint antibodies.

Key Findings:

· In monotherapy and combination studies, TRX518 demonstrated safety, tolerability, and clinical benefit in patients with heavily pretreated solid tumors.

· TRX518 in combination with Keytruda or Opdivo achieved durable complete and partial responses in patients not expected to respond to anti-PD-1 therapy alone, including a confirmed complete response in an esophageal squamous cell cancer patient and a confirmed partial response in an anti-PD-1 refractory urothelial cancer patient.

· TRX518 in combination with gemcitabine achieved meaningful clinical benefit and objective tumor reduction for heavily pretreated patients suffering from pancreatic, biliary tract, mesothelioma, appendiceal, and ovarian cancer.

· The biopsies of responding patients demonstrated an increase in CD8+ T-effector cells and granzyme B expression and a reduction in CD4+ T-regulatory cells and FoxP3 expression, hallmarks of immune activation and anti-tumor activity associated with GITR agonism.

TRX518/gemcitabine combination:

The combination arm evaluating TRX518 in combination with gemcitabine enrolled thirty patients who had received one to nine prior therapies. Four patients were treated at the lower dose of TRX518, and twenty-six patients were treated at the higher dose. The study enrolled fourteen patients with pancreatic cancer, five with biliary tract cancer, and eleven with other cancers including ovarian, appendiceal, and mesothelioma. Seventeen patients had previously progressed on gemcitabine therapy, and ten had previously progressed on anti-PD-1/PD-L1 therapy.

Clinical outcomes data as of December 5, 2018 includes:

TRX518 + gemcitabine

N

Response
Evaluable

Stable
Disease

Progressive
Disease

Disease
Control Rate
(Response
Evaluable)

Overall

30

25

13

12

52

%

Lower dose TRX518

4

2

0

2

0

%

Higher dose TRX518

26

23

13

10

56.5

%

Pancreatic Cancer

14

10

5

5

50

%

Biliary Tract Cancer (BTC)

5

5

4

1

80

%

Other Cancers

11

10

4

6

40

%

Nineteen pancreatic or biliary tract cancer (PBC) patients were enrolled, and nine remain on study. Nearly all of these patients had received prior gemcitabine therapy, and nine (47%) remained on study for more than four cycles. Eight (67%) of twelve evaluable PBC patients previously treated with gemcitabine who received the higher dose of TRX518 have had stable disease, including a fourth-line pancreatic cancer patient who remains on study in Cycle 9 with a 21% reduction in tumor burden. Pancreatic cancer patients who have progressed on gemcitabine have extremely poor outcomes with studies indicating a range of 1.6 to 2.9 months between median progression and median overall survival.

Additional reductions in tumor burden and durable clinical benefit have been noted in appendiceal cancer (-4% in Cycle 7), mesothelioma (-5% in Cycle 6), and two in ovarian cancer (-15% in Cycle 5, -9% off after 4 cycles).

TRX518/Keytruda or Opdivo combination

The combination arms evaluating TRX518 in combination with Keytruda or Opdivo enrolled fourteen patients in the dose escalation cohorts as of December 5, 2018. Both patients treated with the higher dose of TRX518 plus Keytruda have had clinical benefit. An esophageal squamous cell carcinoma patient has had a confirmed complete response, which remains ongoing for seven months, and an ocular melanoma patient has had a 23% reduction in tumor volume and six months of ongoing stable disease. In the low dose TRX518 plus Opdivo combination arm, a patient with urothelial carcinoma who had failed prior Keytruda had a confirmed partial response and remained on therapy for six months.

Enrollment is now complete in the dose escalation cohort for TRX518 and Keytruda and continues in the high dose escalation cohort of TRX518 and Opdivo. Leap anticipates that dose expansion cohorts for both combinations will initiate in the first quarter 2019.

DKN-01

DKN-01 is a humanized monoclonal antibody targeting the Dickkopf-1 (DKK1) protein, a Wnt pathway modulator. DKN-01 is in clinical trials in patients with esophagogastric cancer, hepatobiliary cancer, and gynecologic cancers.

At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, Leap announced that the combination of DKN-01 and paclitaxel generated a 46.7% overall response rate, 19.6 weeks median progression free survival, and 61.1 weeks median overall survival in fifteen evaluable esophagogastric cancer patients as a second-line therapy. In the subgroup of twelve evaluable patients with heavily pre-treated esophageal squamous cell carcinoma, DKN-01 and paclitaxel produced a 33.3% overall response rate, 13.7 weeks median progression free survival, and 31.0 weeks median overall survival.

At the ESMO (Free ESMO Whitepaper) 2018 Annual Meeting, Leap announced that the combination of DKN-01 and Keytruda demonstrated promising clinical activity with a 23.5% overall response rate and 58.8% disease control rate in evaluable gastric or gastroesophageal junction cancer patients who have been heavily pretreated and have not had prior anti-PD-1/PD-L1 therapy. The combination has generated durable responses in subgroups less likely to respond to pembrolizumab monotherapy, for example, patients whose tumors are microsatellite stable and/or PD-L1 negative. Additional data from the DKN-01/Keytruda combination is expected in the second quarter of 2019.

DKN-01 in combination with gemcitabine and cisplatin in Advanced Biliary Tract Cancer

The open-label, Phase I/II study enrolled fifty-one patients with advanced biliary tract cancer (BTC). Seven patients received one of two dose levels (150 mg or 300 mg) of DKN-01 in combination with gemcitabine and cisplatin during Part A, with forty-four additional patients treated at the 300 mg dose level of DKN-01 in the Part B expansion cohort. Forty-two patients were chemotherapy treatment-naïve, and nine patients had received 1-2 prior therapies. The primary objective of this study was to evaluate the safety, pharmacokinetics, and efficacy of DKN-01 in combination with gemcitabine and cisplatin.

In the study, DKN-01 in combination with gemcitabine and cisplatin was well tolerated with no new emerging safety trends. Forty-seven patients overall were treated at the 300 mg DKN-01 dose level, and their median overall survival was 53.7 weeks (12.4 months). Median progression free survival was 37.7 weeks (8.7 months). Ten patients (21.3%) had a partial response and thirty-one patients (66.0%) experienced a best response of stable disease, representing a disease control rate of 87.2%. Two patients (4.3%) had progressive disease, and four patients (8.5%) were non-evaluable for response. The one-year probability of overall survival was 0.51, and the six-month probability of progression free survival was 0.58. The median number of cycles of DKN-01 was seven (range of 1 to 23), and the median duration on study was 331 days.

"Patients with metastatic biliary tract cancer have a poor prognosis with an unmet medical need, with median overall survival of less than one year. We are very pleased by the progression-free survival, overall survival, and favorable safety profile demonstrated by DKN-01 in combination with gemcitabine and cisplatin in this single arm study," commented by Andrew X. Zhu, M.D., Director of Liver Cancer Research at Massachusetts General Hospital Cancer Center and Professor of Medicine at Harvard Medical School. "The activity of DKN-01 in biliary tract cancer warrants further development in the front-line setting as well as in second-line in combination with anti-PD-1/PD-L1 antibodies."

TRX518 Clinical Perspectives Conference Call and Webcast

On Monday, December 17, 2018 at 8:30AM ET, Leap will be hosting a conference call and webcast for the investment community. To access the conference call, please dial (866) 589-0108 (US/Canada Toll-Free) or (409) 231-2048 (international) and refer to conference ID 9187987. The presentation will also be webcast live and will be available on Leap’s website, View Source A replay of the webcast will be available on Leap’s website after the event and will be available for a limited time.

On December 13, 2019 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage immuno-oncology corporation, reported that investigators shared new positive data from the company’s ongoing DeCidE1 (DPX-Survivac with low dose CyclophosphamIDe and Epacadostat) clinical trial at the 2018 ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress (Press release, IMV, DEC 13, 2018, View Source [SID1234534100]). The phase 1b/2 study is evaluating the safety and efficacy of the combination of IMV’s lead candidate DPX-Survivac, low dose cyclophosphamide, and 100 mg or 300mg of Incyte’s IDO1 enzyme inhibitor epacadostat in patients with advanced recurrent ovarian cancer.

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In a poster presentation, Oliver Dorigo, M.D., Ph.D., Associate Professor of Obstetrics and Gynecology (Oncology), Stanford University Medical Center, who served as the trial’s lead investigator and author on the poster, shared topline safety results from 53 enrolled patients and efficacy data from the 32 participants evaluable for immune-related and clinical responses, as well as blood sample and tumor biopsy analyses.

Key findings include:

Evidence of a clinical marker based on Baseline Tumor Burden (BTB), a measure of tumor size predictive of patient response to DPX-Survivac

37.5% (12/32) of evaluable study subjects began treatment with a non-bulky disease defined as BTB < 5 cm.

73% (8/11) of tumor regressions and 80% of clinical responses (4/5) observed in subset of patients with BTB < 5 cm.

Responders showing prolonged duration of clinical benefits reaching up to more than two years, surpassing the progression-free interval from their previous chemotherapy treatment

Robust systemic survivin-specific T cell responses and evidence of survivin-specific T cells tumor infiltration correlated with clinical benefits

100% of durable clinical responses correlated with T cell infiltration

Epacadostat triggered inhibition of the conversion of tryptophan into kynurenine that was dose dependent

Cohort demographics were balanced and the combination yielded a tolerable safety profile

"This data set provided meaningful information on how the potential benefits of DPX-Survivac may best be translated to patients, including the connection between tumor regressions and T cell infiltration in the tumor microenvironment," said Frederic Ors, Chief Executive Officer at IMV. "We believe that DPX-Survivac is the first targeted T cell therapy to induce significant tumor regressions in challenging tumors such as those seen in ovarian cancer. We remain committed to developing DPX-Survivac for patients with significant unmet medical needs, and look forward to our upcoming discussions with regulatory authorities in the USA, Canada and Europe."

Updated Clinical Response and Safety Data for DeCidE1

At the time of data cut-off, 53 patients were enrolled in the phase 1b clinical trial, including 14 from the 100mg epacadostat dosing cohort and 39 from 300mg epacadostat cohort. Based on 300 mg cohort results, IMV and Incyte agreed to stop dosing patients with epacadostat before completion of the study. Patients who completed at least one CT scan, as required per the trial protocol, were evaluable for response analysis.

71% of patients were evaluable for responses in the 100mg cohort and 56% in the 300mg dose cohort. At time of data cut-off, 8 participants remained on treatment and were being evaluated for clinical responses.

(1) Partial Response (PR) is defined as ≥30% decrease in sum of target lesions
(2) Stable Disease (SD) is defined as  30% decrease and ≤ 20% increase in sum of target tumor lesions
(3) Disease Control Rate (DCR) refers to the total number of patients achieving complete response, partial response, and stable disease.

"Recurrent ovarian cancer treatment remains a significant unmet need and represents a challenge for immunotherapy," said Gabriela Nicola Rosu, M.D., Chief Medical Officer at IMV Inc. "What we have showed here is that the dynamic interaction between the survivin specific T cells induced by DPX-Survivac and the tumor size and its growth kinetics can be a determinant of clinical responses. We

believe that this information is significant for the future development of DPX-Survivac and may indicate a pathway to more efficacious immunotherapeutic treatments for patients."

Poster Session Details

Session Title: Poster Display Session
Location: Foyer, Geneva Palexpo
Poster ID: 87P; Abstract ID 262
Abstract Title: "New clinical data from the DeCidE1 trial: Results on DPX-Survivac, low dose cyclophosphamide (CPA), and epacadostat (INCB024360) in subjects with advanced recurrent epithelial ovarian cancer"
Date: December 14 – 15, 2018
Time: 12:30 p.m. – 13:00 p.m. (local time)
Presenter: Dr. Oliver Dorigo, DeCidE1 Clinical Investigator and Lead Author
Investor Call Information

IMV will host a webcast and conference call on Thursday, December 13 at 8:30 a.m. ET to provide an overview of its ESMO (Free ESMO Whitepaper)-IO presentation.

Dial-in: (844) 461-9932 (U.S. and Canada) or (636) 812-6632 (International)

Conference ID#: 6192578

A live audio webcast and presentation will be available via this link, or by pasting this URL in an internet browser: View Source

About the DeCidE1 Phase 1b/2 Trial

The phase 1b/2 trial is an open label, uncontrolled, safety and efficacy study for individuals with advanced, platinum-sensitive and resistant ovarian cancer. The phase 1b portion has two dosing cohorts:

100mg of epacadostat twice daily (BID), with DPX-Survivac and low dose cyclophosphamide, and

300mg of epacadostat BID in combination with DPX-Survivac and low dose cyclophosphamide.

The primary endpoints are to determine:

The safety profile of the combination regimen,

Induction of systemic survivin specific T cells in the blood, and

Induction of T cell infiltration into tumors.

Secondary endpoints include objective response rate (ORR) using modified RECIST v1.1 criteria; duration of response based on modified RECIST criteria; time to progression (TTP); and overall survival (OS.)

IMV conducted the phase 1b/2 study in collaboration with Incyte Corporation. IMV recently announced that, based on the 300 mg cohort results, IMV and Incyte have agreed to stop dosing patients in this trial with epacadostat. IMV is continuing the phase 2 portion of the trial as a monotherapy study evaluating DPX-Survivac in the advanced and recurrent ovarian cancer subpopulation with BTB < 5 cm.

IMV intends to report updated results from the phase 1b when data from at least 16 evaluable participants in the second dosing cohort are available. Investigators plan to submit final results for publication in a peer-reviewed journal.

About Ovarian Cancer

According to the American Cancer Society (ACS), ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. Often diagnosed in its advanced stages, about 21,290 women received a new diagnosis of ovarian cancer in 2015; approximately 14,180 women would die from the disease, according to ACS estimates.

Ovarian cancer has a significant impact globally as well. The World Cancer Research Fund reports that ovarian cancer is the seventh most common cancer in women worldwide (18 most common cancer overall), with 239,000 new cases diagnosed in 2012.

On December 13, 2018 ESSA Pharma Inc. ("ESSA" or the "Company") (TSX-V: EPI,NASDAQ: EPIX), a pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported financial results for the fiscal fourth quarter and year ended September 30, 2018 (Press release, ESSA, DEC 13, 2018, View Source [SID1234532082]). All references to "$" in this release refer to United States dollars, unless otherwise indicated.

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"We have narrowed our selection of an IND candidate to a small number of compounds with high potency, metabolic stability and, therefore, predicted long half-lives, as well as superior pharmaceutical properties," stated David Parkinson, MD, President and CEO of ESSA. "We will make a final IND candidate selection following full compound selectivity characterization and in vivo animal model results, which are expected in the first calendar quarter of 2019. We look forward to preparing our lead candidate efficiently in order to enter the clinic as expeditiously as possible after our IND submission."

2018 Year Highlights

Achieved significant progress in advancing the Company’s next-generation aniten program toward identifying a lead clinical product candidate and submitting an Investigational New Drug Application to the U.S. Food and Drug Administration
Lead compounds are at least fifteen times more potent and five times more stable in vitro compared to the first-generation aniten N-terminal domain inhibitor compound, EPI-506.
Two poster abstracts accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Symposium (ASCO-GU) in February 2019, which will be the first public presentation of preclinical data from ESSA’s new aniten compounds.
Closed equity financings totaling $26 million in January 2018, issuing a total of 4,321,000 common shares and 2,189,000 prepaid warrants exercisable at a nominal price of $0.002.
Strengthened the Company’s preclinical development capabilities.
Summary Financial Results
Effective April 25, 2018, the Company consolidated its issued and outstanding common shares on the basis of one post-consolidation share for every 20 pre-consolidation shares. The consolidation applied uniformly to all ESSA common shares, incentive stock options, prepaid warrants, and other securities convertible into or exercisable for common shares. Unless otherwise stated, all ESSA common share and per share amounts have been restated retrospectively to reflect this share consolidation.

Net Income (Loss). ESSA recorded a net loss of $11.6 million ($2.55 loss per common share based on 4,566,519 weighted average common shares outstanding) for the year ended September 30, 2018, compared to a net loss of $4.5 million ($3.09 loss per common share based on 1,454,936 weighted average common shares outstanding) for the year ended September 30, 2017, which included a gain on derivative liability of $7.3 million. The net loss for the fourth quarter ended September 30, 2018 was $2.3 million compared to a net loss of $1.9 million for the fourth quarter ended September 30, 2017.
Research and Development ("R&D") expenditures. R&D expenditures for the year ended September 30, 2018 were $4.9 million net of grants ($5.1 million gross) compared to $5.7 million net of grants ($10.9 million gross) for the year ended September 30, 2017. For the fourth quarter ended September 30, 2018, R&D expenditures were $0.9 million net of grants ($1.2 million gross), as compared to $1.2 million (net and gross) for the fourth quarter ended September 30, 2017. The decreases in R&D expenditures for the full year and fourth quarter were primarily related to decreases in manufacturing and clinical trial costs as ESSA focused its R&D resources on preclinical research related to the Company’s next-generation aniten compounds in the current year. ESSA concluded its Phase I clinical study of EPI-506 in September 2017.
General and administration ("G&A") expenditures. G&A expenditures for the year ended September 30, 2018 were $5.9 million compared to $5.1 million for the year ended September 30, 2017. For the fourth quarter ended September 30, 2018, G&A expenditures were $1.2 million, compared to $1.1 million for the fourth quarter ended September 30, 2017. The increases in the full year and fourth quarter primarily reflected increased corporate activity, such as the 1:20 share consolidation, filing of the base shelf prospectus, as well as compensation expenses and increased share-based payments reflecting the vesting of stock options.
Liquidity and Outstanding Share Capital
Cash on hand at September 30, 2018, was $14.8 million, with working capital of $12.3 million, reflecting the aggregate gross proceeds of the completed January 2018 financing, which totaled $26 million.

As of September 30, 2018, the Company had 5,776,098 common shares issued and outstanding, and 2,189,000 common shares issuable on the exercise of prepaid warrants at a nominal exercise price of $0.002 per common share. If all prepaid warrants are exercised, there would be approximately 7,965,098 ESSA common shares outstanding.

In addition, there were 474,937 common shares issuable upon the exercise of warrants and broker warrants at a weighted-average exercise price of $34.35 per ESSA common share and 888,709 ESSA common shares issuable upon the exercise of outstanding stock options at a weighted-average exercise price of $4.81 per common share.