Since 2011, Kevelt has been developing the Virexxa project in cooperation with the Competence Centre for Cancer Research (Company Pipeline, Kevelt, AUG 29, 2016, View Source [SID:1234514786]). The objective of the project is to develop a new anti-cancer medicine.

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On August 29, 2016 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) reported top-line results from PERSIST-2, a randomized, controlled Phase 3 clinical trial comparing pacritinib, an investigational oral multikinase inhibitor, with physician-specified best available therapy (BAT), including ruxolitinib, for the treatment of patients with myelofibrosis whose platelet counts are less than 100,000 per microliter — a patient population with high-risk advanced disease (Press release, CTI BioPharma, AUG 29, 2016, View Source;p=RssLanding&cat=news&id=2197807 [SID:1234514783]).

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Three hundred eleven (311) patients were enrolled in the study, which formed the basis for the safety analysis. Two hundred twenty-one (221) patients who had a chance to reach Week 24 (the primary analysis time point) at the time the clinical hold was imposed and constituted the intent-to-treat (ITT) analysis population utilized for the evaluation of efficacy. Preliminary results demonstrated that the PERSIST-2 trial met one of the co-primary endpoints showing a statistically significant response rate in spleen volume reduction (SVR) in patients with myelofibrosis treated with pacritinib compared to BAT, including the approved JAK2 inhibitor ruxolitinib (p<0.01). Although the PERSIST-2 trial did not meet the other co-primary endpoint of greater than 50 percent reduction in Total Symptom Score (TSS), the preliminary analysis approached marginal significance compared to BAT (p=0.0791).

"Unlike patients with myelofibrosis who have normal baseline platelet counts where median survival is reported at 88 months, we recently reported from our institution’s experience that patients with severe thrombocytopenia (low platelets) had a median survival of about 14 months," said Srdan (Serge) Verstovsek, M.D., Ph.D., Director, Clinical Research Center for MPNs at the University of Texas MD Anderson Cancer Center and principal investigator for the PERSIST-2 Phase 3 clinical trial of pacritinib. "These patients represent up to 30 percent of all myelofibrosis patients and an unmet medical need. Data from the PERSIST-2 prospective randomized, controlled trial is encouraging because we need an effective therapy to treat the most challenging patients with low platelet counts we see in our practice."

The co-primary endpoints of the trial were the proportion of patients achieving a 35 percent or greater reduction in spleen volume from baseline to Week 24 as measured by magnetic resonance imaging (MRI) or computerized tomography (CT) and the proportion of patients achieving a Total Symptom Score (TSS) reduction of 50 percent or greater using MPN-SAF TSS 2.0 diary from baseline to Week 24.

The most common treatment emergent adverse events for pacritinib were generally manageable diarrhea, nausea and vomiting. The incidence of cardiac and bleeding adverse events (all grades and grade 3-4 including deaths) were similar across the arms. Overall mortality rates were comparable between arms. Additional data from ongoing analyses along with top-line results from PERSIST-2 will be submitted for presentation at an upcoming scientific meeting.

Myelofibrosis is associated with significantly reduced quality of life and shortened survival. Spleen enlargement (splenomegaly) is a common and debilitating symptom of myelofibrosis. As the disease progresses, the body slows production of important blood cells and within one year of diagnosis the incidence of disease-related thrombocytopenia (very low blood platelet counts), severe anemia and red blood cell transfusion requirements increase significantly.

"Having analyzed data from two Phase 3 trials with the only JAK inhibitor to be studied in severely thrombocytopenic patients, including patients on JAK2 therapy or those who failed prior JAK2, we are encouraged by pacritinib’s clinical profile in this difficult-to-treat group of patients with myelofibrosis," said James A. Bianco, M.D., President and Chief Executive Officer, CTI BioPharma. "We are grateful for the support and commitment of the investigators, our steering committee and, most importantly, all the patients who participated in PERSIST-2."

About PERSIST-2

PERSIST-2 was a randomized (1:1:1), controlled, open-label, multinational Phase 3 clinical trial evaluating pacritinib compared to best available therapy (BAT), including the approved JAK1/JAK2 inhibitor ruxolitinib, for patients with myelofibrosis whose platelet counts were less than or equal to 100,000 per microliter (≤100,000/μL). Three hundred eleven (311) patients were randomized to receive 200 mg pacritinib twice daily (BID), 400 mg pacritinib once daily (QD) or BAT. Clinical studies for pacritinib are currently subject to a full clinical hold issued by the U.S. Food and Drug Administration (FDA) in February 2016. The study was originally designed to enroll, and the Special Protocol Assessment (SPA) requires enrollment of 300 patients to evaluate the study objectives. Two hundred twenty-one (221) patients were enrolled at least 24 weeks prior to the full clinical hold and thus were potentially evaluable for efficacy. These patients were the population used to evaluate the study efficacy endpoints. The co-primary endpoints, originally agreed upon under the SPA, were the percentage of patients achieving a 35 percent or greater reduction in spleen volume measured by MRI or CT scan from baseline to Week 24 of treatment and the percentage of patients achieving a Total Symptom Score (TSS) reduction of 50 percent or greater using seven key symptoms as measured by the modified Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS 2.0) diary from baseline to Week 24. The primary objective of the study was to compare pooled pacritinib arms vs BAT. As a result of the full clinical hold on pacritinib, the SPA agreement is no longer in effect for PERSIST-2 and CTI BioPharma is no longer entitled to the benefit of the SPA.

About the Phase 3 Development Program of Pacritinib

Pacritinib was evaluated in two Phase 3 clinical trials, known as the PERSIST program, for patients with myelofibrosis, with one trial in a broad set of patients without limitations on platelet counts, the PERSIST-1 trial; and the other in patients with low platelet counts, the PERSIST-2 trial. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy; or patients who are intolerant of, or whose symptoms are not well controlled (sub-optimally managed) on other JAK2 therapy.

Clinical studies under the CTI BioPharma investigational new drug (IND) for pacritinib are currently subject to a full clinical hold issued by the U.S. Food and Drug Administration in February 2016.

PERSIST-1 was a randomized (2:1), controlled, open-label, multinational Phase 3 trial evaluating the efficacy and safety of pacritinib compared to BAT, excluding JAK2 inhibitors, which included a broad range of currently utilized treatments – in 327 patients with myelofibrosis (primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis), regardless of the patients’ platelet counts. The study included patients with severe or life-threatening thrombocytopenia. Patients were randomized to receive 400 mg pacritinib once daily or BAT, excluding JAK2 inhibitors. As previously reported, the trial met its primary endpoint of spleen volume reduction (35 percent or greater from baseline to Week 24 by MRI/CT scan) in the intent-to-treat population (ITT).

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia, or AML, myelodysplastic syndrome, or MDS, chronic myelomonocytic leukemia, or CMML, and chronic lymphocytic leukemia, or CLL, due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

CTI BioPharma and Shire are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Shire will jointly commercialize pacritinib in the U.S. while Shire has exclusive commercialization rights for all indications outside the U.S.

About Myelofibrosis and Myeloproliferative Neoplasms

Myelofibrosis is one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of progressive blood cancers. The three main types of MPNs are primary myelofibrosis (PMF), polycethemia vera (PV) and essential thrombocythemia (ET).1

Myelofibrosis is a serious and life-threatening bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue, and pain.

The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S., of which myelofibrosis accounts for approximately 18,000 patients.2 In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis3 and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia (AML).4 The median survival for high-risk myelofibrosis patients is less than 1.5 years, while the median survival for patients with myelofibrosis overall is approximately 6 years.4

On August 29, 2016 OncoCyte Corporation (NYSE MKT:OCX), a developer of novel, non-invasive blood based tests for the early detection of cancer, reported that it has closed two separate private placements with a select group of institutional and accredited investors, including both new and existing investors (Press release, BioTime, AUG 29, 2016, View Source;p=RssLanding&cat=news&id=2197996 [SID:1234514795]). The private placements in total will consist of 3,246,153 units for total gross proceeds of $10.55 million before deducting placement agent fees and offering expenses.

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Each unit, consisting of one share of common stock and one warrant to purchase one share of common stock, was sold at a price of $3.25 per unit, for an aggregate issuance of 3,246,153 shares of common stock and warrants to purchase 3,246,153 shares of common stock. The warrants have an exercise price of $3.25 per share of common stock, become exercisable for the underlying shares of common stock upon shareholder approval of the issuance of such shares, and may be exercised for five years from the date they become exercisable. During the previous 30 trading days, OncoCyte’s common stock has closed at a price per share ranging from $3.60 to $4.40.

The first private placement for $3.25 million was with George Karfunkel, who with his son Bernard is a founder of OncoCyte and together held 6.2 million shares of OncoCyte common stock prior to the transaction. The second private placement for $7.3 million was with a group of institutional and accredited investors, including new and existing holders of OncoCyte. OncoCyte expects to use the proceeds from the private placements for funding operations or for working capital or other general corporate purposes, including to continue the research and development of its non-invasive cancer diagnostics tests, build out its CLIA lab and commercial infrastructure as it anticipates launching its lung cancer diagnostic test in the first half of 2017. With the transaction closings, OncoCyte now has approximately 28.7 million common shares outstanding.

Cowen and Company acted as sole agent for the placement with the group of institutional and accredited investors.

The securities offered in these private placement transactions have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws, and accordingly may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. OncoCyte has agreed to file a registration statement with the Securities and Exchange Commission registering the resale of the shares of common stock issued in this private placement and the shares of common stock issuable upon the exercise of the warrants issued in this private placement.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus

On August 26, 2016 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs being developed to treat inflammatory diseases, cancer and sexual dysfunction, reported financial results for the six months ended June 30, 2016 and updates on its drug development programs (Filing, Q2, Can-Fite BioPharma, 2016, AUG 26, 2016, View Source [SID:1234514753]).

Clinical Development Program and Corporate Highlights Include:

● Piclidenoson (CF101) – Upcoming Phase III Trials in Rheumatoid Arthritis & Psoriasis

Rheumatoid Arthritis: Can-Fite reached an agreement with the European Medicine Agency (EMA) on the protocol design of its upcoming Phase III trial of Piclidenoson in the treatment of rheumatoid arthrosis. Based on the suggestion of the EMA, Piclidenoson will be developed as a first line therapy and replacement for the current gold standard, Methotrexate (MTX), the most widely used drug for rheumatoid arthritis, a treatment market forecast to reach $38.5 billion by 2017. The planned Phase III trial will aim to show Piclidenoson is not inferior to MTX. Based on this clinical study design, Can-Fite is now conducting preparatory work for the trial including drug tableting, packaging and labeling work. The Company plans to submit its study protocol to the Institutional Review Boards (IRBs) of clinical sites in the first quarter of 2017.

Psoriasis: Can-Fite submitted a Phase III clinical trial protocol for Piclidenoson in the treatment of moderate-to-severe psoriasis with the EMA in the first half of 2016. Based on a pre-submission meeting the Company had with the EMA, the planned trial will be a head-to-head study comparing Piclidenoson to apremilast (Otezla), a recently approved oral drug from Celgene. Can-Fite expects a meeting with the EMA to discuss the trial’s design in the third quarter of 2016.

New mechanism of action data showing Piclidenoson may offer efficacy similar to industry-leading biologics, without the associated harmful side effects, were presented by Can-Fite at Psoriasis 2016, the 5th Congress of the Psoriasis International Network, in Paris, France. The oral presentation titled, "CF101 via A3AR Activation inhibits IL-17 and IL-23," was delivered on July 7, 2016.

The peer reviewed scientific journal, Journal of Drugs in Dermatology, published data from a Phase II/III trial of Piclidenoson in the treatment of moderate to severe psoriasis. The study titled, "Treatment of Plaque-Type Psoriasis With Oral CF101: Data from a Phase II/III Multicenter, Randomized, Controlled Trial," was published in August 2016.

● CF102 – Ongoing Phase II in Liver Cancer & Plans to Commence Phase II in NASH

Liver Cancer: Can-Fite continues to enroll and dose patients in its global Phase II study of CF102 in the treatment of hepatocellular carcinoma, the most common form of liver cancer. Enrollment of approximately 78 patients in the U.S., Europe, and Israel is expected to conclude in the second half of 2016.

NASH: Can-Fite worked with world renowned Key Opinion Leaders in the field of liver diseases to complete the protocol design for its upcoming Phase II trial of CF102 in the treatment of non-alcoholic fatty liver disease (NAFLD), the precursor to non-alcoholic steatohepatitis (NASH). By 2025, the addressable pharmaceutical market for NASH is estimated to reach $35-40 billion. The Company plans to file its protocol with IRBs in the second half of 2016.

● CF602 – Preparing for Phase I in the Treatment of Sexual Dysfunction

Can-Fite is currently conducting Investigational New Drug (IND) enabling studies of CF602 in the treatment of sexual dysfunction to support commencing a Phase I study in the first quarter of 2017. The Company presented data at the American Urology Association’s Annual 2016 Meeting in San Diego, California. The presentation titled, "CF602 Improves Erectile Dysfunction in Diabetic Rats," was delivered on May 10, 2016. CF602’s mechanism of action, its efficacy in increasing penile intracavernous pressure (ICP), and single dose efficacy were included in the presentation.

"In the first half of 2016, we were particularly encouraged by feedback received from the EMA, indicating we conduct head-to-head studies of Piclidenoson in psoriasis and rheumatoid arthritis. These studies will compare Piclidenoson to drugs that are used as the standard of care today. Because of Piclidenoson’s well established safety profile, proving efficacy that is equivalent to the comparative drugs would highlight the benefits of Piclidenoson in delivering a safe, effective and oral treatment," stated Can-Fite CEO Dr. Pnina Fishman. "In addition to heading into Phase III studies of Piclidenoson, we are pleased to continue the development programs of CF102 and CF602 to address unmet clinical needs."

Revenues for the six months ended June 30, 2016 were NIS 0.43 million (U.S. $0.11 million) compared to NIS 0.27 million (U.S. $0.07 million) in the first six months of 2015. The increase in revenue was due to the recognition of a portion of the NIS 5.14 million (U.S. $1.36 million) upfront payment received in March 2015 under the distribution agreement with Cipher Pharmaceuticals.

Research and development expenses for the six months ended June 30, 2016 were NIS 9.97 million (U.S. $2.59 million) compared with NIS 5.75 million (U.S. $1.5 million) for the same period in 2015. Research and development expenses for the first half of 2016 comprised primarily of expenses associated with the Phase II study for CF102, preclinical study for CF602, as well as expenses for ongoing studies of CF101. The increase is primarily due to costs associated with preparations of the CF101 Phase III studies in the treatment of rheumatoid arthritis and psoriasis.

General and administrative expenses were NIS 4.99 million (U.S. $1.3 million) for the six months ended June 30, 2016 compared to NIS 4.67 million (U.S. $1.21 million) for the same period in 2015. The increase is primarily due to an increase in share based compensation expense.

Financial income, net for the six months ended June 30, 2016 aggregated NIS 3.19 million (U.S. $0.83 million) compared to financial income, net of NIS 1.88 million (U.S. $0.49 million) for the same period in 2015. The increase in financial income, net in the first half of 2016 was mainly due to a larger decrease in the fair value of warrants that are accounted for as financial liability as compared to the same period in 2015. In addition, the increase in financial income, net in the first half of 2016 was attributable to a decrease in financial expenses due to exchange rate differences as compared to the same period in 2015.

Can-Fite’s net loss for the six months ended June 30, 2016 was NIS 11.35 million (U.S. $2.95 million) compared with a net loss of NIS 8.27 million (U.S. $2.15 million) for the same period in 2015. The increase in net loss for the first half of 2016 was primarily attributable to an increase in research and development expenses offset by an increase in financial income, net.

As of June 30, 2016, Can-Fite had cash and cash equivalents of NIS 46.42 million (U.S. $12.07 million) as compared to NIS 66.03 million (U.S. $17.17 million) at December 31, 2015. The decrease in cash during the six months ended June 30, 2016 is due to operating expenses.

For the convenience of the reader, the reported NIS amounts have been translated into U.S. dollars, at the representative rate of exchange on June 30, 2016 (U.S. $1 = NIS 3.846).

The Company’s consolidated financial results for the six months ended June 30, 2016 are presented in accordance with International Financial Reporting Standards.

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On August 25, 2016 argenx (Euronext Brussels: ARGX), a clinical-stage biopharmaceutical company focused on creating and developing differentiated therapeutic antibodies for the treatment of cancer and severe autoimmune diseases, reported its second quarter business update and half year financial results for 2016, in accordance with IFRS as adopted by the European Union (Press release, arGEN-X, AUG 25, 2016, View Source [SID:1234514755]).

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The half year results will be discussed during a conference call and webcast presentation today at 3 pm CET / 9 am EDT. To participate in the conference call, please select your phone number below, and use the confirmation code 49998398. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"This quarter was a seminal one for us as we made substantial progress against several very important corporate goals: advancing our clinical and other pipeline programs and in closing a significant financing with key U.S. institutional investors as well as entering into a strategic transaction with AbbVie for our oncology candidate AGRX-115. We believe these accomplishments have driven argenx forward to become a new and more substantial company with a full and mature clinical pipeline, an advanced platform and the financial and strategic support to derive value from them," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "During the quarter we announced data from our Phase 1 MAD and SAD studies of ARGX-113 which led to the selection of our Phase 2 dose and demonstrated the drug’s strong safety profile and its ability to rapidly reduce IgG levels in healthy volunteers. Our lead oncology candidate ARGX-110 showed further evidence of anti-tumor activity in T-cell lymphoma patients and is on track to announce top-line data in this expansion cohort by end of year. We are looking forward to executing on our strategic plan for the remainder of 2016 to bring ARGX-113 into two Phase 2 indications and to examine the breadth of potential for ARGX-110 as a monotherapy and a combination agent in TCL and AML."

SECOND QUARTER 2016

Announced initial results from its Phase 1 multiple ascending dose (MAD) study of ARGX-113 in healthy volunteers. The compound continues to show favorable safety and tolerability across multiple doses and dosing regimens with promising pharmacodynamics effects relating to speed, depth and duration of IgG reduction.
Published efficacy and safety data from its ARGX-111 Phase 1 expansion study in patients with MET amplified tumors in conjunction with the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting (Chicago, USA). The data confirm ARGX-111 to have a favorable safety profile and to continue to show signs of anti-tumor activity.
Presented efficacy and safety data from its Phase 1 expansion study of ARGX-110 in patients with T-cell lymphoma (TCL) during an e-poster session at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress (Copenhagen, Denmark). The data from the Phase 1 expansion study show evidence of clinical and/or biological anti-tumor activity with ARGX-110 in highly refractory cutaneous TCL & peripheral TCL patients with confirmed overexpression of CD70.
Entered into placement agreements with several predominant U.S. institutional investors relating to the issue of a total of 2,703,000 new shares for an aggregate amount of €30,003,300. The transaction was led by MPM Oncology Impact Fund with participation from Aquilo Capital, Burrage Capital, DAFNA Capital, Perceptive Advisors and certain other existing and new institutional investors.
Announced collaboration with AbbVie to develop and commercialize ARGX-115. ARGX-115 is argenx’ preclinical-stage human antibody asset targeting the novel immuno-oncology target GARP, a protein believed to contribute to immunosuppressive effects of T-cells. argenx received an upfront payment of $40M.
FINANCIAL HIGHLIGHTS (as of 30 June, 2016) (compared to financial highlights as of 30 June 2015)

Operating income of EUR 7.0 million (30 June 2015: EUR 4.3 million).
Net loss of EUR 7.4 million (30 June 2015: EUR 7 million).
Cash position of EUR 108.7 million (cash, cash-equivalents and financial assets) allowing Company to pursue development of its product portfolio as planned.