On December 18, 2018 CTI BioPharma Corp. (NASDAQ:CTIC) reported that it has received input from the U.S. Food and Drug Administration (FDA) at a recent Type C meeting on key elements of the design of a new randomized Phase 3 study of pacritinib in adult patients with myelofibrosis (primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis) and who have severe thrombocytopenia (as defined by patients with platelet counts of less than 50,000 per microliter), an indication that has been recognized by the medical community as an important unmet medical need (Press release, CTI BioPharma, DEC 18, 2018, View Source [SID1234532119]).

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The planned Phase 3 study is designed to evaluate the effects of pacritinib as compared to physician’s choice of treatment. The primary efficacy endpoint will be the proportion of patients achieving a greater than or equal to 35% spleen volume reduction (SVR) between baseline and Week 24. Secondary efficacy endpoints of the study include total symptom score reduction and overall survival.

Before commencing the Phase 3 study, CTI plans to meet with the FDA to discuss the final optimal dose analysis from the PAC203 Phase 2 study. To expedite the transition to Phase 3, CTI intends to amend the PAC203 protocol to include a Phase 3 component. The PAC203 Phase 3 component is designed to enroll approximately 200 patients with enrollment expected to commence in the third quarter of 2019. The anticipated cost of the Phase 3 study is approximately $25 million. Taking into account the impact of recently-announced cost saving efforts and the anticipated cost of the new Phase 3 trial, the current CTI financial analysis projects a cash runway that extends into 2020.

On December 18, 2018 Phio Pharmaceuticals Corp. (NASDAQ: PHIO) (formerly RXi Pharmaceuticals Corporation), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported that Dr. Gerrit Dispersyn, President and Chief Operating Officer, will present at Biotech Showcase 2019, to be held January 7 – 9, 2019 at the Hilton San Francisco Union Square (Press release, Phio Pharmaceuticals, DEC 18, 2018, View Source [SID1234532115]).

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Dr. Gerrit Dispersyn will present an overview of the Company’s novel self-delivering RNAi (sd-rxRNA) technology and the multiple business development and commercial opportunities available with this proprietary platform on:

Date: Monday, January 7, 2019
Time: 9:00 a.m. PST
Room: Yosemite C (Ballroom Level)

He will also be available for investor and executive meetings throughout the conference. The presentation will be webcast and available on the "Investors – Events and Presentations" section of the Company’s website, www.phiopharma.com.

About Biotech Showcase
Now in its eleventh year, Biotech Showcase, produced by Demy-Colton and EBD Group, is an investor and networking conference devoted to providing private and public biotechnology and life sciences companies with an opportunity to present to, and meet with, investors and executives in one place during the course of one of the industry’s largest annual healthcare investor conferences, J.P. Morgan Annual Healthcare Conference.

On December 18, 2018 Delcath Systems, Inc. (OTCQB: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported that the independent Data Safety Monitoring Board (DSMB) of the Registration trial for Patients with Hepatic Dominant Ocular Melanoma (The FOCUS Trial) completed another pre-specified review of safety data for treated patients in the trial (Press release, Delcath Systems, DEC 18, 2018, View Source;p=RssLanding&cat=news&id=2381000 [SID1234532112]). This review was conducted on data collected from both the prior randomized protocol and the amended single-arm protocol for the FOCUS Trial. The DSMB again recommended continuation of the study without modification.

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In July, the Company announced that it has amended the protocol for the FOCUS trial, which is now enrolling as a single-arm, multi-center open label study. Safety data collected have not been modified as a result of the amendment, and safety data from both the randomized and single-arm protocols will be pooled in any analyses submitted to the Food & Drug Administration as part of a New Drug Application.

The FOCUS trial, now entitled A Single-arm, Multi-Center, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Melphalan/HDS Treatment in Patients with Hepatic-Dominant Ocular Melanoma (The FOCUS Trial), is enrolling a minimum of 80 patients with ocular melanoma metastatic to the liver. Patients previously enrolled under the prior randomized protocol continue to be treated and evaluated as part of the amended trial, and periodic DSMB reviews will continue to be conducted.

Commenting on the announcement, Jennifer K. Simpson, Ph.D., MSN, CRNP President and CEO of Delcath, said, "We are pleased with the safety profile observed by our therapy in the trial thus far, and the trial remains on track to complete enrollment by June 2019."

On December 17, 2018 AOP Orphan Pharmaceuticals AG (AOP Orphan) reported that EMA´s CHMP adopted a positive opinion for approval of Ropeginterferon alfa-2b/BESREMi indicated as monotherapy in adults for the treatment of Polycythaemia vera without symptomatic splenomegaly (Press release, AOP Orphan Pharmaceuticals, DEC 17, 2018, View Source [SID1234533574]).

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Ropeginterferon alfa-2b/BESREMi is a novel, long-acting interferon, which is administered once every two weeks, or monthly after stabilization of hematological parameters. This treatment schedule is expected to lead to overall better safety, tolerability and adherence compared to conventional pegylated interferons.

AOP Orphan has been running the BESREMi clinical development in PV since 2010. The latest phase III data, three years treatment, and phase II data, up to seven years treatment, were presented at ASH (Free ASH Whitepaper) 2018. In summary, BESREMi showed high hematologic and clinical response rates with good tolerability.


In addition, BESREMi showed high molecular response rates, associated with the ability to reduce allelic burden of both mutant JAK2 and importantly also non-JAK2 mutations, which are believed to have a role in disease progression.

Andreas Steiner, Chief Executive Officer of AOP Orphan commented: "Although interferons are a treatment modality widely used throughout the myeloproliferative neoplasms including CML, BESREMi will be the first licensed interferon in any of these indications. Physicians experienced in the management of the disease and administration of BESREMi during the clinical studies expect many advantages for the patients with PV."

About Ropeginterferon alfa-2b/BESREMi
Ropeginterferon alfa-2b/BESREMi is a novel, long-acting, mono-pegylated proline interferon (ATC L03AB15) with improved pharmacokinetic properties offering improved tolerability and adherence to treatment. It is administered once every two weeks, or monthly during long-term maintenance, and is expected to be the first interferon approved for PV worldwide.

Ropeginterferon alfa-2b was discovered by PharmaEssentia, a long-term partner of AOP Orphan. In 2009, AOP Orphan has in-licensed from PharmaEssentia Corporation the exclusive rights for clinical development and commercialization of Ropeginterferon alfa-2b in PV, other MPNs and CML for European, Commonwealth of Independent States (CIS), and Middle Eastern markets.

About Polycythemia Vera
Polycythemia Vera (PV) is a cancer of the blood-building cells in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. This condition may result in circulatory disorders such as thrombosis and embolism, as well as malignant transformation to myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2 that make the malignant clone.

On December 17, 2018 C4X Discovery Holdings plc (AIM: C4XD), a pioneering drug discovery company, reported that it has entered into an exclusive target discovery partnership with Horizon Discovery Group plc ("Horizon", AIM: HZD), a global leader in the application of gene editing and gene modulation technologies (Press release, C4X Discovery, DEC 17, 2018, View Source [SID1234533248]). The partnership aims to validate novel synthetic lethal oncology targets that have been identified by Horizon’s cutting-edge CRISPR-Cas9 technology leading to the generation of potential new drugs for patients with limited effective treatments such as colorectal and lung cancer.

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C4XD aims to apply its proprietary 4D shape-based chemistry technology (Conformetrix) to discover drug candidates directed against these high value, novel, synthetic lethality targets and out-license them to clinical development partners. This discovery partnership bolsters C4XD’s oncology pipeline, a high priority therapeutic area for the company.

The therapeutic concept of harnessing synthetic lethality in cancer with tumour-specific mutations has been demonstrated with the recent approval of the poly(ADP-ribose) polymerase (PARP) inhibitors Lynparza Zejula and Rubraca. These drugs are effective in indications that are poorly served by immunotherapy drugs, such as checkpoint inhibitors, and have become an established treatment in ovarian cancer. They work by targeting a specific DNA repair pathway that cancer cells, with mutations such as BRCA, become over-reliant on. Inhibiting this critical DNA repair pathway causes the tumour cells to self-destruct, resulting in the terminology ‘synthetic lethality’. The pharmaceutical industry is now rushing to fill its clinical pipelines with investigational drugs, identified by empirical research, that might produce similar efficacy in cancers where PARP inhibitors are not effective.

However, recent advancements in the gene-editing tool, CRISPR-Cas9, has enabled systematic ‘functional genomic’ screening to identify novel synthetic lethal genes in cancer cells with specific mutations. Horizon has utilised its target discovery platform to conduct high quality CRISPR gene knock-out studies across multiple cancer cell lines, screening around ~3000 genes suitable as the basis for small molecule drug targets. This cutting-edge approach has identified a shortlist of ~20 novel, high value synthetic lethal genes following a secondary screen to further validate them as targets. The partnership has been established to complete the target validation package for these novel targets, leading to the initiation of drug discovery programmes by C4XD should it exercise its options on a target-by-target basis. C4XD will provide the funding for the work plan and Horizon will receive a share of all future revenues C4XD receives should any drug discovery programmes emerging from the partnership be out-licensed for clinical development.

Dr Craig Fox, Chief Scientific Officer of C4XD, said: "PARP inhibitors are transforming the treatment of ovarian cancer, particularly in patients with BRCA mutations and so identifying the next generation of synthetic lethality drug targets is a key priority to develop new therapies for cancers patients who are not responsive to current treatments. This partnership with Horizon gives C4XD access to a comprehensive proprietary CRISPR screening dataset that has selected the most promising novel drug targets in colon and lung cancer. We look forward to working with the highly experienced team at Horizon to complete the target validation package for these novel genes and initiate drug discovery programmes to generate high value pre-clinical licensable assets for partnering."

Dr Jon Moore, Horizon’s Chief Scientific Officer added: "Drugging the cancer genome now requires new synthetic lethal therapies that can exploit the vulnerabilities in patient’s tumours that are created by mutations in undruggable cancer driver genes. Horizon’s internal research program has used powerful CRISPR technology to open a new window into how cancer cells are wired and has identified a cohort of novel targets. We are delighted to have secured this collaboration with C4X Discovery, which applies a powerful drug discovery engine to the first-in-class opportunities for transformational medicines represented by the targets Horizon has found and allows Horizon to share in the upside."