On August 7, 2018 Vical Incorporated (Nasdaq:VICL) reported financial results for the three months ended June 30, 2018 (Press release, Vical, AUG 7, 2018, View Source [SID1234528485]). Net loss for the second quarter of 2018 was $4.9 million, or $0.22 per share, compared with a net loss of $3.3 million, or $0.30 per share, for the second quarter of 2017. Revenues for the second quarter of 2018 were $0.7 million, compared with revenues of $3.4 million for the second quarter of 2017, reflecting a decline in revenues from Astellas Pharma Inc. for services performed under ASP0113 collaborative agreements.

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Vical recently announced plans to explore a range of strategic options to enhance shareholder value. The company retained MTS Health Partners, L.P. to assist in the strategic review process. There is no set timetable for the review process and there can be no assurance that the process will result in a transaction.

Vical had cash and investments of $54.3 million at June 30, 2018. The Company’s cash burn for the second quarter of 2018 was $4.0 million, which was consistent with the Company’s full year 2018 guidance of between $20 million and $24 million. The Company anticipates ending 2018 with a minimum of $40 million, which, in the absence of a strategic transaction, Vical believes to be sufficient to fund operations through the announcement of top-line data from its Phase 2 clinical trial of VL-2397, expected in 2020.

Program updates include:

VL-2397 Antifungal Candidate

The multinational Phase 2 registration trial comparing VL‑2397 to standard first-line treatment for invasive aspergillosis in immunocompromised adults with acute leukemia or recipients of an allogeneic hematopoietic cell transplant is ongoing (ClinicalTrials.gov Identifier: NCT03327727). Vical expects to conduct the trial in approximately 40 major cancer and transplantation centers in North America, Europe and Asia. The FDA has advised that VL‑2397 would be eligible for a Limited Use Indication (LUI) approval for the treatment of invasive aspergillosis for patients with limited treatment options. The FDA has also granted Vical Qualified Infectious Disease Product (QIDP), Orphan Drug and Fast Track designations for VL‑2397 for the treatment of invasive aspergillosis. VL-2397 has a novel mechanism of antifungal action and could be the first therapeutic in a new class of antifungals.
VR-CHB01 Hepatitis B Virus (HBV) Therapeutic Candidate

The Company is pursuing preclinical development of a novel treatment for chronic HBV infection based on its DNA and lipid-delivery technologies. The initial aim of this program will be to demonstrate proof of concept for inhibiting HBV infection in an in vivo model. The Company expects to complete the initial stage of preclinical development in the fourth quarter of 2018.

On August 7, 2018 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2018. In addition, the Company provided a clinical and business update (Press release, Syndax, AUG 7, 2018, View Source [SID1234528501]). As of June 30, 2018, Syndax had $98.4 million in cash, cash equivalents and short-term investments.

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"We made great progress across multiple programs during the first half of this year, including presentation of data from all three cohorts of ENCORE 601 and completion of target enrollment in ENCORE 602 and 603. We also initiated the first combination trial for the SNDX-6352 program, which will evaluate its safety in combination with durvalumab (IMFINZI)," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We continue to expect the progression free survival results from E2112, our ongoing Phase 3 trial of entinostat plus exemestane in HR+, HER2- breast cancer, later this quarter, and the third prespecified interim analysis of overall survival in November. We also anticipate sharing next steps for the entinostat-KEYTRUDA (pembrolizumab) combination program in both non-small cell lung cancer and melanoma by the end of the year."

The Company also announced today that it plans to initiate a Phase 1 trial of its monoclonal antibody inhibitor of Colony-Stimulating Factor 1 Receptor (CSF-1R), SNDX-6352, in patients with chronic graft versus host disease (cGVHD). Enrollment in this trial is anticipated to begin by the end of the year, with initial data expected in the second half of 2019.

"We are excited to begin the evaluation of SNDX-6352 as a treatment for cGVHD, a novel clinical path for a CSF1-R inhibitor," said Michael L. Meyers, M.D., Ph.D., Chief Medical Officer of Syndax. "Preclinical findings support that CSF-1R inhibition may serve as an effective approach for treating this debilitating, often deadly side effect of allogenic hematopoietic stem cell transplantation. We look forward to learning more about the potential of SNDX-6352 in this indication."

Pipeline Updates

The Phase 3 registration trial of entinostat plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer, E2112, is 98% enrolled as of the end of July. ECOG-ACRIN Cancer Research Group, the trial sponsor, had notified the Company that the Data Safety Monitoring Committee (DSMC) completed the final progression free survival (PFS) analysis in November 2017. The trial is proceeding as planned, and Syndax continues to anticipate that enrollment will be complete in the third quarter of 2018, at which time the result of the PFS analysis will be released to the Company. In addition, interim overall survival (OS) analyses are scheduled to occur every May and November. Two interim OS analyses have already occurred, with the next analysis expected this November.

The Company presented data from a subset of PD-(L)1 refractory non-small cell lung cancer (NSCLC) patients enrolled in the expanded Phase 2 ENCORE 601 cohort (n = 57) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. Updated data from all patients (n = 76) enrolled in this cohort will be presented at the World Conference on Lung Cancer Meeting in Toronto next month, including updated results from the Company’s biomarker analyses. The Company expects to communicate its development plans for entinostat in this indication in the fourth quarter.

The Company presented data from a subset of PD-1 refractory melanoma patients enrolled in the expanded Phase 2 ENCORE 601 cohort (n = 34) at the ASCO (Free ASCO Whitepaper) Annual Meeting in June. Updated results from the full cohort (n = 55) are expected by the end of this year, at which time the Company will make a decision on registration plans for entinostat in this indication.

Enrollment in the expanded stage 1 ENCORE 601 cohort of patients with microsatellite stable colorectal cancer (MSS-CRC, n = 37) is expected to complete in the third quarter. A decision on whether to continue to the second stage of this cohort is expected in the first half of 2019.

Target enrollment in both the Phase 2 portion of ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-L1 inhibitor, atezolizumab (TECENTRIQ), in patients with triple negative breast cancer, and the Phase 2 portion of ENCORE 603, evaluating entinostat in combination with Pfizer/Merck KGaA’s PD-L1 inhibitor, avelumab (BAVENCIO), in patients with ovarian cancer, is complete. Topline results from each study are anticipated in the first half of 2019.

ENCORE 606, the Phase 1b/2 trial evaluating entinostat in combination with NKTR-214, Nektar’s CD122-biased agonist, is expected to begin enrolling patients in the first half of 2019.

Dosing of patients with solid tumors in the Phase 1/1b trial evaluating the safety of SNDX-6352 continues as planned. Testing of SNDX-6352 in combination with durvalumab (IMFINZI), AstraZeneca’s human monoclonal antibody directed against PD-L1, was recently initiated, and dosing of patients with SNDX-6352 as a monotherapy is ongoing. The Company anticipates identifying the recommended Phase 2 dose and schedule for SNDX-6352 monotherapy and in combination with durvalumab in the first half of 2019.

The Company expects to commence enrollment in a Phase 1 dose escalation trial of SNDX-6352 in patients with cGVHD by the end of the year. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule. Initial results are anticipated in the second half of 2019.

Development of the Company’s portfolio of Menin-Mixed Lineage Leukemia (MLLr) inhibitors is ongoing. The Company continues to expect clinical trials to initiate in the first half of 2019.
Second Quarter 2018 Financial Results

As of June 30, 2018, Syndax had cash, cash equivalents and short-term investments of $98.4 million and 22,705,794 shares issued and outstanding.

On June 18, 2018, the Company signed an exchange agreement with Biotechnology Value Fund and certain affiliated funds ("BVF") under which BVF exchanged 2,000,000 shares of common stock for 2,000,000 Warrant Shares. BVF can exercise the Warrant Shares at an exercise price per share equal to $0.0001 per share. The warrant is issued for a period of 20 years.

In the third quarter of 2018, through August 6th, the Company sold 633,231 shares of its common stock with net proceeds of approximately $4.4 million pursuant to its at-the-market arrangement.

Second quarter 2018 research and development expenses increased to $14.9 million from $9.9 million for the comparable period in the prior year. The increases were primarily due to increased activities in manufacturing for SNDX-6352, increased development activities for the Menin-MLLr and ENCORE 602 programs partially offset by completion of pharmacology trials and lower program cost for E2112. Employee compensation increased due to increased headcount.

General and administrative expenses totaled $4.5 million during the second quarter of 2018, compared to $4.3 million for the comparable period in the prior year. The increase in general and administrative expenses was primarily due to increased pre-commercialization activities and increased patent related legal expenses.

For the three months ended June 30, 2018, Syndax reported a net loss attributable to common stockholders of $18.4 million or $0.74 per share compared to $13.6 million or $0.70 per share for the comparable prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the third quarter and full year 2018. For the third quarter and full year 2018, research and development expenses are expected to be $14 to $16 million and $59 to $62 million, respectively, and total operating expenses are expected to be $18 to $20 million and $77 to $81 million, respectively. Total operating expenses for 2018 are expected to include approximately $6 million of non-cash stock compensation expense.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, August 7, 2018.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 4980058
Domestic Dial-in Number: 1- 855-251-6663
International Dial-in Number: 281-542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On August 7, 2018 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported its financial results for the second quarter ended June 30, 2018 (Press release, Protagonist, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362550 [SID1234528527]).

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"Protagonist continues to advance well-differentiated therapeutic candidates discovered through its novel peptide technology platform," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "We very recently reported results from a comprehensive review of the data from the Phase 2 PROPEL study of PTG-100 for the treatment of moderate to severe active ulcerative colitis. We are pleased to conclude that this drug candidate showed signals of clinical efficacy, has no safety concerns, and warrants further clinical development for potential treatment of ulcerative colitis. We are planning to discuss next steps in the clinical development of PTG-100 with the U.S. Food and Drug Administration (FDA) and other global regulatory authorities in the second half of this year. We are glad to report on the continued progress of the clinical development of PTG-200 in collaboration with our partner Janssen for potential treatment of Crohn’s disease. In addition, we look forward to initiating an open label Phase 2 study in beta-thalassemia patients in the fourth quarter of 2018 with PTG-300, our product candidate for rare blood disorders."

Product Development Update:

PTG-100

In March 2018, Protagonist had announced discontinuation of the Phase 2 PROPEL study following a planned interim analysis conducted by an independent Data Monitoring Committee. The interim data had revealed an unusually high placebo rate of clinical remission (24 percent, approximately four times higher than historical norms for similar UC studies) that led to a futility decision and discontinuation of the trial. A re-read of the endoscopies by the subcontractor of the contract research organization (CRO) and a subsequent fully blinded re-read of the endoscopies by an independent third party, Robarts Clinical Trials, confirmed that a subset of the initial endoscopy reads provided by the CRO were in error. If the re-read of endoscopy results had been utilized for the interim futility analysis, the trial would have continued. Based on this entire analysis, the Company plans to discuss the next steps in advancing the clinical development of PTG-100 with the FDA and other global regulatory authorities in the second half of 2018.
PTG-200

In the fourth quarter of 2018, Protagonist expects a U.S. IND filing by its partner Janssen Biotech that will support the initiation of a global Phase 2 study of PTG-200, an oral, gut-restricted interleukin-23 receptor antagonist peptide in Crohn’s disease patients. This IND filing would trigger a milestone payment from Janssen of $25 million under the existing exclusive license and collaboration agreement between Janssen and Protagonist.
PTG-300

The results of a Phase 1 study in healthy volunteers and supportive pre-clinical data for PTG-300, an injectable hepcidin mimetic peptide, were the subject of oral presentations in June 2018 at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Protagonist completed discussions with U.S. and global regulatory agencies and filed a U.S. IND in the second quarter of 2018. The Company plans other global regulatory submissions to support the initiation of a global Phase 2 trial in patients with beta-thalassemia in the fourth quarter of 2018.
Preclinical Programs

The Company presented data related to preclinical product candidates, oral peptide agonists targeting the delta/mu opioid receptors, in a podium presentation at the Digestive Diseases Week conference in June 2018.
Corporate Update – Financing:

Protagonist recently announced the completion of an equity financing with investors including BVF Partners L.P. and their affiliates for gross proceeds of $22 million. Proceeds from the financing will be used to advance development of drug candidate PTG-100.
Financial Results

Protagonist reported a net loss of $8.7 million and $16.3 million, respectively, for the second quarter and first six months of 2018, as compared to a net loss of $15.0 million and $29.1 million, respectively, for the same periods of 2017. The decrease in net loss for both the second quarter and year-to-date periods was driven primarily by license and collaboration revenue recognized during the first and second quarters of 2018, partially offset by increased research and development (R&D) and general and administrative (G&A) expenses. The net loss for the second quarter and first six months of 2018 includes non-cash stock-based compensation of $1.6 million and $2.8 million, respectively, as compared to $1.0 million and $1.8 million, respectively, for the same periods of 2017.

License and collaboration revenue was $11.7 million and $22.5 million for the second quarter and first six months of 2018, respectively, and consisted of revenue from activities performed under the Janssen Collaboration Agreement. Protagonist did not recognize any license and collaboration revenue for the second quarter or first six months of 2017.

R&D expenses for the second quarter and first six months of 2018 were $17.7 million and $33.1 million, respectively, as compared to $12.0 million and $23.3 million, respectively, for the same periods of 2017. The increases in R&D expenses were primarily due to costs related to contract manufacturing and the preparation for and conduct of clinical trials for our product candidates. R&D expenses for the quarter also included an increase in salaries and employee-related expenses due to an increase in R&D personnel.

G&A expenses for the second quarter and first six months of 2018 were $3.2 million and $6.8 million, respectively, as compared to $3.1 million and $6.1 million, respectively, for the same periods of 2017. The increases in G&A expenses were primarily due to increases in salaries and employee-related expenses primarily due to an increase in headcount to support the growth of our operations, partially offset by a decrease in legal expenses.

Protagonist ended the second quarter with $125.2 million in cash, cash equivalents and investments. With the financing announced yesterday, the company expects to have sufficient financial resources to fund operations to mid-2020.

On August 7, 2018 Stony Brook University, iCell Gene Therapeutics, and the University of Louisville, have received Food and Drug Administration (FDA) clearance for an Investigational New Drug (IND) for the treatment of relapsed and refractory T-cell leukemia and lymphoma (Press release, Stony Brook University, AUG 7, 2018, View Source [SID1234528670]). The approach is the first to use chimeric antigen receptor engineered T-cells directed against the target protein CD4 (CD4CAR). Together, Stony Brook University, the University of Louisville, and iCell Gene Therapeutics expect the first in-human Phase I clinical trial to begin accruing patients before the end of 2018.

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"We are excited to partner with the University of Louisville and iCell Gene Therapeutics to offer this innovative first-in-human CAR T cell immunotherapy clinical trial for patients who are suffering from these extremely difficult to treat T cell lymphomas and leukemias," said Huda Salman, MD, Principal Investigator for the IND and an oncologist at Stony Brook University Cancer Center. "CD4CAR T cells may prove to be a promising and novel therapy in this setting."

"The development of this trial using CD4 as a target is the first of what we expect to be many CAR T-based clinical trials available to our patients over time," said Yusuf Hannun, MD, Director of the Stony Brook University Cancer Center. "The pending trial is an example of the type of bench-to-bedside research that is building up at Stony Brook due to the growing expertise and collaborative research environment we are creating and new opportunities that will emerge upon the opening of our Medical and Research Translation (MART) Building."

William Tse, MD, FACP, Chief of the Blood and Marrow Transplantation at the University of Louisville School of Medicine, is the Co-PI of the CD4CAR clinical trial at University of Louisville site.

About CD4CAR

CD4CAR is a novel development for the treatment of CD4+ T-cell malignancies. The CD4-redirected chimeric antigen receptor (CAR) T-cells are engineered to express an anti-CD4scFV antibody domain. CD4CAR has received Orphan Drug Designation by the FDA for Peripheral T cell Leukemia (PTCL) in 2016. Over the past few years, CAR T-cell therapy has proven its efficacy in clinical trials for various types of leukemia, lymphoma, and myeloma. CAR T-cell therapy is a type of adoptive immunotherapy. A CAR-engineered T-cell is genetically modified to express a protein on its surface with the capability to bind to a target protein on another cell. The CD4CAR is manufactured from the patient’s own cells to target CD4 expressed on tumor cells. Once these cells are infused back into a patient’s body through an IV, they multiply and attack tumor cells efficiently throughout the body.

About T-cell leukemias and lymphomas

Although there are clinical development programs ongoing with CAR T-cells for B-cell hematological malignancies, CD4 positive T-cell malignancies (T-cell lymphomas (TCLs) and T–cell acute lymphoblastic leukemia (T-ALLs)), have not been targeted by a CAR therapy in a human trial. TCLs account for 15–20 % of all non-Hodgkin’s lymphomas (NHLs), while T-ALLs affect about 25% of ALLs in adults. These malignant entities are significantly more difficult to treat in comparison to B-cell malignancies. Furthermore, T-cell malignancies almost exclusively have poorer outcomes with few exceptions, lower response rates, shorter times of disease control and survival. As a result, the standard of care for T-cell malignancies is not well-established and the only potential curative approach is allogeneic blood and marrow transplant (BMT) for which patients need to achieve complete disease control and to have suitable marrow donors. This leaves many patients with no curative options.

On August 7, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported the positive results from the lead-in period of the GENESIS trial, a double-blind, placebo-controlled Phase 3 trial comparing BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF), to G-CSF alone, for the mobilization of hematopoietic stem cells (HSCs) used for autologous transplantation in multiple myeloma patients (Press release, BioLineRx, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362414 [SID1234528751]).

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The open-label, single-arm, lead-in period of the study was designed to include up to 30 patients, with Data Monitoring Committee (DMC) review after completion of approximately 10, 20 and 30 patients, in order to assess safety and efficacy following treatment with BL-8040 plus G-CSF. Results of the first 11 patients show that BL-8040 in combination with standard G-CSF treatment is safe and tolerable. In addition, the data show that 9/11 patients (82%) reached the primary endpoint threshold of ≥ 6×106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions. Furthermore, 7/11 patients (64%) reached the threshold of ≥ 6×106 CD34 cells/kg in a single apheresis session only. These data demonstrate the potential of BL-8040 treatment to reduce the number of administrations and apheresis sessions, as well as hospitalization costs, related to the preparation of multiple myeloma patients for autologous HSC transplantation.

"Autologous HSC transplantation following high-dose chemotherapy has significantly improved outcomes for multiple myeloma patients," said Dr. John F. DiPersio, Chief, Division of Oncology at the Washington University School of Medicine, and lead investigator of the study. "Current practice involves mobilizing HSCs from the bone marrow to the peripheral blood, after which the cells are collected by apheresis. Results so far show that mobilizing HSCs with a single BL-8040 dose combined with G-CSF is not only safe and tolerable, but also demonstrates robust efficacy regarding the number of collected cells, and may reduce the number of required apheresis sessions to a single session in the majority of patients. This is a very encouraging result that, if corroborated in the placebo-controlled part of the trial, will be of great value to patients as well as to the medical community. I am looking forward to participating in the trial and to potentially improving the quality of treatment available to multiple myeloma patients."

"We are extremely encouraged by these results. Based on the robust data received from the first 11 patients, the DMC issued a positive recommendation to stop the lead-in part of the study and move immediately to the randomized placebo-controlled part of the study," stated Philip Serlin, Chief Executive Officer of BioLineRx. "This is the first Phase 3 trial for our lead BL-8040 program, and as such, it is an important milestone in BL-8040’s comprehensive development plan. We look forward to the top-line results from the randomized, double-blind, placebo-controlled part of the study, which are expected in 2020."

About the GENESIS Study

The GENESIS study is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of BL-8040 in combination with G-CSF, compared to placebo and G-CSF, for the mobilization of CD34 HSCs for autologous transplantation in multiple myeloma patients. The placebo-controlled part is designed to include 177 patients in more than 25 centers. Treatment will include 5 days of G-CSF, with a single dose of BL-8040 or placebo on day 4 with the option to expand treatment to up to 8 days of G-CSF and up to 2 days of BL-8040. Apheresis for collection of CD34 cells will be performed on day 5. An additional 3 apheresis sessions may be conducted if needed in order to reach the goal of ≥ 6×106 mobilized CD34 cells/kg.

The primary objective of the study is to demonstrate the superiority of a single dose of BL-8040 in combination with G-CSF, over placebo and G-CSF, in the mobilization of ≥ 6×106 CD34 cells/kg in up to 2 apheresis sessions, in preparation for autologous stem cell transplantation in multiple myeloma patients. Secondary objectives include time to engraftment of neutrophils and platelets, durability of the engraftment, as well as safety and other efficacy parameters.

About BL-8040

BL-8040 is a short synthetic peptide for stem cell mobilization and for treatment of hematological malignancies and solid tumors. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in the retention of stems cells in the bone marrow, as well as tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity.

HSCs express CXCR4 and are retained in the protective bone marrow niche via binding to CXCL12 (also known as SDF-1). Blocking of the CXCR4-SDF1 interaction by BL-8040 leads to the mobilization of HSCs into the peripheral blood. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of HSCs.

Furthermore, BL-8040 induce mobilization of leukemic cells and immune-cells from the bone marrow, thereby sensitizing leukemic cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.