On November 1, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported that new interim results from the Company’s ongoing Phase 2a study of cerdulatinib in patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL), will be presented during an oral session at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, December 1-4, 2018 in San Diego, California (Press release, Portola Pharmaceuticals, NOV 1, 2018, View Source;p=RssLanding&cat=news&id=2374918 [SID1234530526]).

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The Company also will present new outcomes-based research on the burden of hospital readmissions for venous thromboembolism among patients with cancer during an oral session on Sunday, December 2, and two investigator-initiated animal studies highlighting the anticoagulant reversal agent Andexxa [coagulation Factor Xa (recombinant), inactivated-zhzo] will be presented in poster sessions.

"As shown at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and European Hematology Association (EHA) (Free EHA Whitepaper) meetings earlier this year, cerdulatinib has shown encouraging results across a range of B- and T-cell malignancies and we look forward to presenting new data specifically focused on patients with PTCL and CTCL," said John Curnutte, M.D., Ph.D., head of research and development for Portola. "These data also will serve as a basis for further discussions with regulatory agencies regarding the potential regulatory path forward for cerdulatinib in certain tumor subtypes."

Cerdulatinib is an investigational oral, dual Syk/JAK kinase inhibitor that uniquely inhibits two key cell signaling pathways that promote cancer cell growth in certain hematologic malignancies. It is being developed for the treatment of resistant or relapsed hematologic cancer.

Oral Presentations

1001. The Novel Syk/JAK Inhibitor Cerdulatinib Demonstrates Good Tolerability and Clinical Response in a Phase 2a Study in Relapsed/Refractory Peripheral T-Cell Lymphoma and Cutaneous T-Cell Lymphoma
Abstract: View Source
Session: 624 (Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: T Cell Lymphoma: Chemotherapy and Targeted Approaches)
Presenter: Steven M. Horwitz, M.D., Memorial Sloan-Kettering Cancer Center
Date: Monday, December 3, 2018 at 7:15 p.m. PST; Room 6F

365. Burden of Hospital Readmissions for Venous Thromboembolism Among Patients with Cancer
Abstract: View Source
Session: 901 (Health Services Research—Non-Malignant Conditions: Thrombosis and Anticoagulation
Hematology Disease Topics & Pathways)
Presenter: Alpesh Amin, M.D., M.B.A., UC Irvine
Date: Sunday, December 2, 2018 at 10:30 a.m. PST; Room 8
Poster Presentations

2456. Reversal of Apixaban Anticoagulation with Reduced Doses of Andexanet Alfa in a Porcine Polytrauma Model
Abstract: View Source
Session: 321 (Blood Coagulation and Fibrinolytic Factors: Poster II Hematology Disease Topics & Pathways)
Presenter: Oliver Grottke, M.D., Ph.D., M.P.H., University Hospital RWTH Aachen, Germany
Date: Sunday, December 2, 2018 from 6:00 – 8:00 p.m. PST; Hall GH

3778. Comparison of Second and First Generation of Andexanet Alfa in a Porcine Polytrauma Model with Apixaban Anticoagulation
Abstract: View Source
Session: 321 (Blood Coagulation and Fibrinolytic Factors: Poster II Hematology Disease Topics & Pathways)
Presenter: Oliver Grottke, M.D., Ph.D., M.P.H., University Hospital RWTH Aachen, Germany
Date: Monday, December 3, 2018 from 6:00 – 8:00 p.m. PST; Hall GH

On November 1, 2018 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that the abstracts for the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) meeting (December 1st-4th, San Diego, California) have been released and include two melflufen presentations – one oral presentation (HORIZON) and one poster (ANCHOR) (Press release, Oncopeptides, NOV 1, 2018, View Source [SID1234530525]).

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The ANCHOR and HORIZON abstracts can be found on the company webpage. The abstract data cut for ANCHOR was July 18th and for HORIZON, May 10th. Further data supporting melflufen’s activity has been generated since the abstract data cut and will be presented at ASH (Free ASH Whitepaper).

Upcoming presentations at ASH (Free ASH Whitepaper)
The ANCHOR data will be presented as a poster on Saturday December 1st, at 6.15pm PST.

The HORIZON data will be presented as an oral presentation by Professor Paul G. Richardson, in the session "Antibodies and Targeted Therapies" on Monday December 3rd at 8.15am PST.

Paul G. Richardson, MD, is Professor of Medicine at Harvard Medical School and Clinical Program Leader, Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center Dana-Farber Cancer Institute in Boston, Massachusetts, USA.

The data to be presented at ASH (Free ASH Whitepaper) will be based on data cuts during November (ANCHOR) and October (HORIZON) when more patients have been treated for evaluation than what is available in the abstracts.

CEO

"We are happy to announce that HORIZON has been selected for an oral presentation at ASH (Free ASH Whitepaper) and very much look forward to present new data from our ongoing clinical trials in patients with relapsed refractory multiple myeloma. Over the last 2 years, we have consistently generated good efficacy and tolerability data in RRMM patients on our path to establish melflufen as a new potential back-bone therapy after IMiDs and PIs in RRMM patients. From the trial ANCHOR, we will for the first time show efficacy and tolerability data when treating RRMM patients with melflufen in combination with other myeloma drugs. The combinations we will present at ASH (Free ASH Whitepaper) are melflufen with either daratumumab or bortezomib. Furthermore, we will present updated data from HORIZON where we treat very ill patients that have few or no remaining treatment options with melflufen. HORIZON is a study where patients first have failed on lenalidomide and proteasome inhibitor (PI) based therapy and then also failed on treatment with pomalidomide and/or daratumumab. The new HORIZON data will be presented in an oral session by Prof. Paul G. Richardson. This is a significant recognition of the progress we are making in the development of melflufen", said Jakob Lindberg CEO of Oncopeptides.

For more information about the abstracts go to:
www.oncopeptides.com / Investors & Media / Presentations / ASH (Free ASH Whitepaper) Abstracts 2018ANCHOR abstract number #1967
HORIZON abstract number #600
ANCHOR

ANCHOR is an ongoing Phase I / II study that will include up to 64 patients. It is an open, single-arm study, in which melflufen (Ygalo) and dexamethasone (steroid) is administered in combination with bortezomib (cohort A) or daratumumab (cohort B) in relapsed refractory multiple myeloma (RRMM) patients. Melflufen is administred as either 20mg, 30mg or 40mg every 28 days.

Summary of the interim results in the abstract

As of July 18th 2018, 8 RRMM patients had been enrolled in the study. 2 patients in cohort A and 6 patients in cohort B. None of the patients in either cohort had ever achieved Complete Response to any therapy prior to inclusion.

In cohort A, treatment was done in combination with bortezomib. As of the data cut-off, a total of four cycles in 2 patients were available for safety evaluation with 30mg of melflufen. The combination was found to be well tolerated. With regard to early signs of efficacy after one cycle of treatment, 1 patient achieved an MR (Minimal Response) and 1 patient achieved SD (Stable Disease).

In cohort B, treatment was done in combination with daratumumab. As of the data cut-off, a total of nine cycles in 3 patients were available for safety evaluation with 30 mg of melflufen. The combination was found to be well tolerated and an additional three patients were dosed with 40mg melflufen together with daratumumab (no data in abstract). With regard to early signs of efficacy after one cycle of treatment, 1 patient achieved PR (Partial Response) and 2 patients achieved MR.

Since the data cut for the abstract, further data has been gathered and will be presented at the ASH (Free ASH Whitepaper) meeting.

HORIZON

HORIZON is an ongoing open single-armed phase II trial in which melflufen (Ygalo) and dexamethasone (steroid) is used in relapsed refractory multiple myeloma patients with few or no remaining treatment options.

Summary of the interim results in the abstract

The results presented in the abstract show promising activity in heavily pre-treated and multi-refractory RRMM patients where a majority of patients in addition also have high-risk cytogenetics and/or poor prognosis through the ISS disease staging system. The data in the abstract are in line with the data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) meeting in June 2018. Preliminary efficacy results showed an encouraging 32% Overall Response Rate, and a 39% Clinical Benefit Rate.

Since the data cut for the abstract, further data has been gathered and will be presented (including Progression Free Survival (PFS)) at the ASH (Free ASH Whitepaper) meeting.

For further information, please contact:

Jakob Lindberg, CEO of Oncopeptides
E-mail: [email protected]
Telephone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 70 853 72 92

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons above, on November 1, 2018 at 14.00 (CET).

On November 1, 2018 OncoMed Pharmaceuticals, Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics, reported third quarter 2018 financial results and provided a corporate update (Press release, OncoMed, NOV 1, 2018, View Source [SID1234530524]). As of September 30, 2018, cash, cash equivalents, and short-term investments totaled $70.9 million.

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"As anticipated, our development efforts have culminated in a stream of data this year and set the stage for additional data flow in 2019," said John Lewicki, Ph.D., President and Chief Executive Officer of OncoMed. "The efficacy of navicixizumab, both as a single agent and in combination with chemotherapy, has been impressive in patients with heavily pretreated, late stage recurrent ovarian cancer. We continue to enroll additional patients in our ongoing Phase 1b clinical trial as we consider the possible next steps for this program. Concurrently, clinical investigation and proof of concept continues for our other clinical candidates: etigilimab (anti-TIGIT) and GITRL-Fc in metastatic solid tumor settings."

Pipeline Highlights

Navicixizumab (anti-DLL4/VEGF bispecific; OMP-305B83)

In the third quarter, OncoMed reported publication of results from its Phase 1a study of single-agent navicixizumab in patients with refractory solid tumors in Investigational New Drugs. The results showed that 19 of the 66 patients with various types of refractory solid tumors had tumor shrinkage following treatment with navicixizumab. Notably, 3 of the 12 (25%) heavily pretreated ovarian cancer patients treated in the trial achieved a partial response with single-agent navicixizumab therapy.

In addition, the company announced interim results from its Phase 1b clinical trial of navicixizumab in combination with weekly paclitaxel in ovarian cancer patients who had received a median of four prior therapies. In addition, all patients had previously received paclitaxel and 69% had received bevacizumab. The results, which were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting, showed that 22 of the 26 patients (85%) treated with the novel regimen experienced clinical benefit. Notably 11 of the 26 patients (42%) achieved a partial response, the GCIG CA-125 response rate was 61% and the median progression-free survival was 5.4 months (95% CI: 3.5-8.0 months). Historical response rates for patients with heavily pretreated platinum-resistant ovarian cancer treated with chemotherapy are typically 15% or less.
Etigilimab (Anti-TIGIT monoclonal antibody; OMP-313M32)

Enrollment continues in the company’s Phase 1a/1b clinical trial of etigilimab. Specifically, the company is continuing to enroll patients with select tumor types in the single-agent expansion phase of the study and is also enrolling patients who have progressed on prior immunotherapy in the Phase 1b portion of the trial with these patients being treated with etigilimab plus anti-PD1 (nivolumab). Phase 1a data from the dose-escalation portion of the trial, designed to assess safety and tolerability of escalating doses of etigilimab monotherapy, will be reported at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in a poster presentation on Friday and Saturday, November 9 and 10, 2018 and in a rapid oral presentation on Saturday, November 10, 2018 from 12:35-1:35 pm Eastern Time.
GITRL-Fc (OMP-336B11)

Enrollment continues in the Phase 1a single-agent study of its wholly-owned GITRL-Fc in patients with advanced or metastatic solid tumors. The company is pleased that enrollment in this trial has been robust to date. GITRL-Fc is a fusion protein with an Fc-linked fully human trimer ligand and is designed to activate the co-stimulatory receptor GITR (glucocorticoid-induced tumor necrosis factor receptor-related protein) to enhance T-cell modulated immune responses. Data from the Phase 1a trial are expected to be presented in 2019.
Third Quarter 2018 Financial Results

Cash, cash equivalents and short-term investments totaled $70.9 million as of September 30, 2018, compared to $103.1 million as of December 31, 2017.

Revenues were $19.5 million for the third quarter of 2018, an increase of $14.4 million, compared to $5.1 million for the same period in 2017. The increase in revenue was due to the Company’s adoption of Accounting Standards Codification (ASC) Topic 606, Revenue from Contracts with Customers effective January 1, 2018.

Research and development (R&D) expenses were $10.0 million for the third quarter of 2018, a decrease of $2.2 million, compared to $12.2 million for the same period in 2017. The decrease in R&D expenses was due to decreases in clinical development costs and a decrease in personnel cost, including stock-based compensation.

General and administrative (G&A) expenses were $3.7 million for the third quarter of 2018, a decrease of $0.2 million, compared to $3.9 million for the same period in 2017. The decrease in G&A expenses was primarily due to a decrease in personnel cost, including stock-based compensation.

Net income was $6.1 million ($0.16 net income per share, basic and diluted) for the third quarter of 2018, compared to a net loss of $10.7 million ($0.28 net loss per share, basic and diluted) for the same period of 2017. The net income in the third quarter of 2018 was primarily due to higher collaboration revenue as a result of the new revenue recognition accounting standard adopted on January 1, 2018 and lower operating expenses.

2018 Financial Guidance

With resource reprioritization and additional cash management measures, OncoMed’s current cash runway has been extended by one quarter and is now estimated to fund operations through at least the fourth quarter of 2019, without taking into account future potential milestone or opt-in payments from its partners. OncoMed estimates 2018 operating cash burn to be less than $55 million, before considering potential milestone or opt-in payments.

On November 1, 2018 NuCana plc (NASDAQ: NCNA) announced the first patients have been enrolled in the NuTide:302 Study (Press release, Nucana BioPharmaceuticals, NOV 1, 2018, View Source [SID1234530523]). This study is evaluating NUC-3373 in combination with other agents typically administered with 5-fluorouracil (5-FU) in patients with advanced colorectal cancer. NUC-3373 is NuCana’s ProTide transformation of the active anti-cancer metabolite of 5-FU.

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The NuTide:302 study was preceded by the first-in-human Phase I study (NuTide:301) of single-agent NUC-3373 in patients with advanced solid tumors. Initial results from NuTide:301 were presented in October 2018 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in which NUC-3373 was observed to have had durable single-agent anti-cancer activity in patients who had exhausted all current standards of care. Furthermore, NuCana believes NUC-3373 demonstrated potential safety and dosing administration advantages as compared with 5-FU. In that study, three patients achieved durable Stable Disease with responses lasting more than nine months at the time of data cutoff. Both dosing regimens were observed to be well tolerated with no unexpected adverse events (AEs). Of particular note, no patients developed hand-foot syndrome, a common and debilitating side effect of fluoropyrimidine treatment.

Hugh Griffith, NuCana’s Chief Executive Officer, stated: "NUC-3373 is our second product candidate developed from our proprietary ProTide technology. The goal, as with all our ProTides, is to significantly improve the efficacy and safety of commonly used anti-cancer agents. We believe NUC-3373 has the potential to replace 5-FU as the standard of care in the treatment of a wide range of cancers and we are pleased to have enrolled the first patients in this combination study."

NuTide:302 is a two-part study of NUC-3373 administered every two weeks as an intravenous (IV) infusion. In part 1, NUC-3373 will be combined with leucovorin, with a plan to enroll 12 patients. In part 2, NUC-3373, with or without leucovorin, will be studied in separate combinations with oxaliplatin, oxaliplatin plus bevacizumab, oxaliplatin plus panitumumab and irinotecan, and irinotecan plus cetuximab, with six patients planned per cohort. The primary objective of the study is to identify a recommended dose of NUC-3373 in combination with standard agents used in treating advanced colorectal cancer. The study will also make a preliminary assessment of the anti-tumor activity of the combinations.

More information about this study may be found at: View Source

On November 1, 2018 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported positive progress in the Phase 1 clinical trial of its immuno-stimulating STAT3 inhibitor, WP1066, with initial results showing bioavailability of the drug in patients (Press release, Moleculin, NOV 1, 2018, View Source [SID1234530522]).

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"Although this data is preliminary, it represents a significant milestone for the development of WP1066," commented Dr. Donald Picker, Moleculin’s Chief Science Officer. "In the first two cohorts of the Phase 1 study, we are already seeing measurable levels of the drug in the patient’s plasma resulting from oral administration. Knowing we can deliver drug this way opens the door for further development and expanded clinical activity."

Walter Klemp, Moleculin’s Chairman and CEO added, "We believe WP1066 is a first-in-class compound capable of stimulating a natural immune response in animal models while directly attacking tumors by modulating transcriptional activity and repressing what we call ‘oncogenic transcription factors.’ Chief among these is STAT3, considered a master regulator of tumor progression. While activity in animal models has been very promising, one of the goals of this trial was to determine the potential for bioavailability in humans. The initial positive indications of this clinical trial increase our confidence that WP1066 has the potential to become an important drug in the treatment of certain cancers. The initial demonstration of human bioavilability is an important milestone."