On March 13, 2019 CTI BioPharma Corp. (Nasdaq: CTIC) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Idera Pharmaceuticals, FEB 27, 2019, View Source [SID1234533745]).

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"The Company is focused on advancing its development program of pacritinib for the treatment for myelofibrosis patients with severe thrombocytopenia in the United States and Europe in 2019," commented Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "Following the previously-announced completion of full enrollment in the U.S. Phase 2 PAC203 study of pacritinib in myelofibrosis patients with severe thrombocytopenia, the Company expects to meet with the U.S. Food and Drug Administration and report the determination of an optimal dose of pacritinib in mid-2019, with a Phase 3 registration study targeted to commence enrollment shortly thereafter. The Company further expects to report topline efficacy and safety data from the Phase 2 study by the end of 2019."

Expected 2019 Milestones

Determination of the optimal dose of pacritinib – mid-2019
Commence enrollment in Phase 3 study of pacritinib in myelofibrosis patients with severe thrombocytopenia (platelet count of less than 50,000 per microliter) – 3Q 2019
Reporting of top-line efficacy and safety data from PAC203 at a major medical meeting by the end of 2019
Fourth Quarter Financial Results

Total revenues for the three months and year ended December 31, 2018 were $14.1 million and $26.3 million, respectively, compared to $0.5 million and $25.1 million for the respective periods in 2017. The increase in total revenues for the fourth quarter in 2018 compared to the same period in 2017 is primarily due to the recognition of license and contract revenue in 2018 related to the achievement of a regulatory milestone under the license and collaboration agreement for PIXUVRI with Servier as well as the attainment of a worldwide net sales milestone of TRISENOX under the agreement with Teva Pharmaceutical Industries Ltd.

GAAP operating income was $0.2 million for the fourth quarter of 2018 and GAAP operating loss was $32.9 million for the year ended December 31, 2018, compared to GAAP operating loss of $13.7 million and $39.5 million for the respective periods in 2017. Operating income in the fourth quarter of 2018 as compared to operating loss in the same period in 2017 resulted primarily from the increase in license and contract revenue as mentioned above. Operating loss for the year ended December 31, 2018 as compared to 2017 resulted primarily from the decrease in selling, general and administrative expenses.

Net income attributable to common stockholders for the fourth quarter of 2018 was $0.8 million, or $0.01 for basic and diluted earnings per share, compared to net loss attributable to common stockholders of $14.3 million, or $(0.33) per share, for the same period in 2017. Net loss attributable to common stockholders for the twelve months ended December 31, 2018, was $29.4 million, or $(0.52) per share, compared to a net loss of $45.0 million, or $(1.24) per share, for the same period in 2017.

As of December 31, 2018, cash, cash equivalents and short-term investments totaled $67.0 million, compared to $43.2 million as of December 31, 2017.

On March 13, 2019 LIDDS AB reported that first patient has now been enrolled in the Phase I NanoZolid-docetaxel study where NanoZolid combined with docetaxel will be injected into solid tumors (Press release, Lidds, MAR 13, 2019, View Source [SID1234555908]). The first injection was administered at Karolinska University Hospital.

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-The potential usefulness of this product is that it allows for higher drug concentrations to be achieved in the local treatment of cancer. These are probably drug concentrations that cannot be achieved with oral or intravenous treatments, says Dr Jeffrey Yachnin, Section Head Phase-I Unit, Center for Clinical Cancer Studies, Karolinska University Hospital.

-So far the treatment is without complications, says Dr Yachnin.

The first part of the Phase I study (NZ-DTX-001) will enroll patients for dose escalation after which patients will be treated intratumorally at a fixed dose to confirm tolerability. The primary objective is to study tolerability of NanoZolid-docetaxel and a secondary objective is to assess efficacy on tumor regression.

NZ-DTX is a key project for LIDDS as most types of cancer tumors can benefit from the intratumoral delivery of cytotoxic drugs. The cancers of greatest interest to LIDDS are lung, head & neck, prostate and breast cancer, but other tumors may also benefit from treatment using NanoZolid loaded with cytotoxic drugs.

An intratumorally sustained release of cytotoxic drugs can result in substantial tumor regression and facilitate subsequent curative surgery or radiation therapy and may prevent the tumor from metastasizing during the diagnostic lag period. Depending on the cancer indication, intratumoral treatment can also be combined with systemic drugs.

Around four million people are diagnosed with solid-tumor cancers each year and a very large number undergo diagnostic biopsies. LIDDS aim is that a significant proportion of patients undergoing diagnostic biopsies will be offered an injection of NanoZolid combined with docetaxel.

-LIDDS has had very promising results with docetaxel in the preclinical trial which gives us confidence that tumor regression will also be observed during Phase I. If so, it is likely that a substantial number of patients could benefit from this new form of treatment, says Monica Wallter, CEO LIDDS.

-Our objective is that NanoZolid combined with docetaxel will be regularly used to treat solid tumors, including at the tumor biopsy phase, to decrease the tumor size and improve surgery and radiation therapy outcomes. This will also benefit cancer patients that often have to wait many weeks before their treatment plan is decided, says Monica Wallter.

LIDDS docetaxel strategy is to prove tolerability and tumor regression for a number of different types of cancer. LIDDS has combined NanoZolid with docetaxel as it is indicated for a large number of common types of cancer types. When proof of concept is reached, LIDDS objective is to divest or license the project to one or several pharmaceutical companies.

-The NanoZolid technology has patent coverage until 2037, which is an important asset when out-licensing NanoZolid with cytotoxic drugs, says Monica Wallter.

On March 13, 2019 Transgene (Euronext Paris:TNG), a biotech company that designs and develops virus-based immunotherapies against cancers and infectious diseases, reported that the NEOVIVA project was selected by the "Investments for the Future" (IFP) Programme (Programme d’Investissements d’Avenir) operated by Bpifrance for the development of an industrial sector focused on Transgene’s individualized immunotherapy platform myvac (Press release, Transgene, MAR 13, 2019, View Source [SID1234534268]). Under the agreement, the NEOVIVA project will receive €5.2 million over the five-year duration of the program from Bpifrance, of which Transgene will receive €2.6 million.

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Transgene holds the intellectual property of the myvac platform and is actively working to further develop this innovative technology in collaboration with its three French partners participating in the NEOVIVA project: HalioDx in Marseille, Traaser in Evry and the Curie Institute in Paris. The NEOVIVA project complements the already existing collaboration between Transgene and the Japanese company NEC focused on the clinical validation of an individualized immunotherapy based on artificial intelligence.

The goal of the NEOVIVA project is to develop and validate a manufacturing approach that would provide all solutions needed for the development of individualized immunotherapies. These are designed to stimulate the patient’s immune system to recognize and kill cancer cells by using their own genetic mutations. Two proof of concept clinical trials are being prepared in Europe and US for the treatment of HPV-negative head and neck cancers and ovarian cancer. These are expected to start in H2 2019.

"We would like to thank Bpifrance for its financial support which will accelerate the development of myvac. This funding is a further recognition of the myvac platform and reinforces our ambition to stand as a world leader in the development of individualized viral-vector (MVA) based immunotherapies. We believe the synergies among our partners and their respective teams are highly complementary and harness creativity which will lead to the success of our project. We are delighted to have access to our partners’ expertise through the NEOVIVA project," said Philippe Archinard, CEO and Chairman of Transgene.

Aïcha Douhou, Leader of the Health sector at the innovation department, Bpifrance commented: "We are delighted to support Transgene and its partners HalioDX, Traaser and the Curie Institute in their innovative approach for the design and development of individualized immunotherapy against solid tumors. We expect this project to further boost the development of Transgene bioproduction manufacturing unit for this individualized immunotherapy."

This consortium which combined bio-engineering, bioIT and a recognized know-how in viral vectorisation will be led by Transgene and each partner will contribute to the project as follows:

Transgene will be responsible for the project’s strategy and the clinical development plan. The product design for each patient will be generated in collaboration with NEC which will provide bioIT predictions needed to finalise the immunotherapy. Transgene will also manufacture the myvacTM clinical batches in its pilot manufacturing unit on the short timelines required for the treatment;
Traaser will be in charge of the interpretation of the genomic profiles determined by DNA sequencing. This will be used to facilitate, guide and secure the study of the individualized virotherapy artificial intelligence algorithms results;
HalioDx will study biomarkers to monitor and maximize the clinical efficacy of myvacTM with Immunogram, a high-tech clinical research platform that includes a suite of proprietary tests including Immunosign and the Immunoscore assay suite;
The Curie Institute (Immunotherapy Cancer Centre, led by Dr Amigorena) will contribute to the project in generating translational data and characterizing the therapy’s mechanism of action.

On March 12, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that data from the Phase 2 multicenter trial evaluating ublituximab (TG-1101), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, in relapsing forms of Multiple Sclerosis (RMS) has been selected for presentation at the upcoming American Academy of Neurology (AAN) annual meeting, to be held May 4 – 10, 2019 in Philadelphia, Pennsylvania (Press release, TG Therapeutics, MAR 12, 2019, View Source [SID1234534250]). Final data from the core Phase 2 trial has been previously presented, most recently at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) annual meeting in Dallas, TX. In addition to highlighting the final Phase 2 data, the AAN presentation plans to include data from the open label extension (OLE), a trial made available to any patient who completed the core Phase 2 trial allowing them to continue treatment with ublituximab.

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The abstract is available online and can be accessed via the below link or on the AAN meeting website at www.aan.com.

Presentation Details:

Title:Open Label Extension (OLE) of Phase 2 Multicenter Study of Ublituximab (UTX), a Novel Glycoengineered Anti-CD20 Monoclonal Antibody (mAb) in Patients with Relapsing Forms of Multiple Sclerosis (RMS)
— Presentation Date & Time:Tuesday May 7, 2019, 5:30 PM – 6:30 PM ET
— Session Title: Poster Session P3: MS Clinical Trials and Therapeutic Research
— Presenter:Edward Fox, MD, PhD, Central Texas Neurology Consultants, Round Rock, TX
— Location:Pennsylvania Convention Center
— Abstract Number: 2055
These data support the ongoing, fully enrolled, international Phase 3 program evaluating ublituximab for the treatment of RMS. The Phase 3 trials, entitled ULTIMATE I and ULTIMATE II, are being conducted under Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) and are being led by Lawrence Steinman, MD, of Stanford University.

On March 12, 2019 Bio-Path Holdings, Inc., (Nasdaq: BPTH) (Bio-Path), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that it has entered into definitive agreements with several healthcare focused institutional investors for the issuance and sale in a registered direct offering of 712,910 shares of its common stock, at a purchase price of $25.95 per share, for aggregate gross proceeds of approximately $18.5 million (Press release, Bio-Path Holdings, MAR 12, 2019, View Source [SID1234534269]). The offering is expected to close on or about March 14, 2019, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

Bio-Path currently intends to use the net proceeds from the offering for working capital and general corporate purposes.

The shares of common stock described above are being offered and sold by Bio-Path pursuant to a "shelf" registration statement on Form S-3 (Registration No. 333-215205), including a base prospectus, previously filed with and declared effective by the Securities and Exchange Commission (SEC) on January 9, 2017. The offering of the shares of common stock will be made only by means of a prospectus supplement that forms a part of the registration statement. A final prospectus supplement and an accompanying base prospectus relating to the registered direct offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the prospectus supplement and the accompanying base prospectus may also be obtained from H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at 646-975-6996 or e-mail at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.