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Phio Pharmaceuticals to Present at Biotech Showcase™ 2019 in San Francisco

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.

Delcath Announces 4th Independent Safety Review of Registration Trial Data for Metastatic Ocular Melanoma: Recommended Continuation with no Trial Modification

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.

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."

AOP ORPHAN ANNOUNCES POSITIVE CHMP OPINION FOR ROPEGINTERFERON ALFA-2B/BESREMI®

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]).

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.

C4XD and Horizon Discovery enter exclusive partnership

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.

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."

TG Therapeutics, Inc. and Dana-Farber Cancer Institute Announce Publication of Clinical Data from the Phase 1/1b Trial of Umbralisib in Combination with Ibrutinib in Lancet Haematology

On December 17, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX) and Dana-Farber Cancer Institute reported the publication of results from the multicenter Phase 1/1b trial of umbralisib (TGR-1202), TG Therapeutics’ novel once-daily PI3K delta inhibitor, in combination with ibrutinib, the oral Bruton’s tyrosine kinase (BTK) inhibitor, in Lancet Haematology (Press release, TG Therapeutics, DEC 17, 2018, View Source [SID1234532247]). This investigator-initiated trial was conducted at Dana-Farber Cancer Institute and four additional academic and community sites across the USA in collaboration with the Leukemia and Lymphoma Society Blood Cancer Research Partnership with funding by TG Therapeutics. The publication includes safety and efficacy information from a total of 42 relapsed or refractory patients, 21 with chronic lymphocytic leukemia (CLL) and 21 with mantle cell lymphoma (MCL).

In this study, the combination of umbralisib and ibrutinib was well tolerated and consistent with the additive toxicity profile of the two drugs individually. No dose-limiting toxicities were observed, and the maximum-tolerated dose of umbralisib when combined with ibrutinib was not reached. The recommended phase 2 dose of umbralisib when given in combination with ibrutinib was 800 mg once daily. Importantly, serious immune-mediated toxicities were not observed with this combination, as had previously been reported with combinations of different agents targeting this pathway, with only one case of transient Grade 3 transaminitis and no Grade 3/4 colitis or pneumonitis. The combination of umbralisib and ibrutinib was also clinically active, with 90% of relapsed/refractory CLL patients achieving an overall response (n=19), of which 62% (n=13) achieved a partial response or partial response with lymphocytosis, and 29% (n=6) achieved a complete response. Of the 21 patients treated with MCL, 67% (n=14) achieved an overall response, of which 48% (n=10) achieved a partial response and 19% (n=4) achieved a complete response.

These data are described further in the manuscript entitled, "Umbralisib in combination with ibrutinib in patients with relapsed or refractory chronic lymphocytic leukaemia or mantle cell lymphoma: a multicenter phase 1–1b study," which was published today in Lancet Haematology. The online version of the article can be accessed at http://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(18)30196-0/fulltext.

"Our study demonstrates for the first time that it is feasible to combine two agents targeting B cell receptor pathway kinases in patients with B cell malignancies," said Matthew Davids, MD, MMSc, Associate Director of the Center for Chronic Lymphocytic Leukemia at Dana-Farber. Dr. Davids continued, "We are particularly encouraged by the depth of response in the CLL patients, which compares favorably to historical data for ibrutinib monotherapy in this relapsed population. Our data support further exploration of dual BCR pathway blockade in CLL and other B cell malignancies."

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated "We want to thank Dr. Davids and the team at Dana-Farber, and most importantly the patients who participated in this trial. We are excited to publish the first ever data evaluating the all oral combination of a PI3K delta inhibitor with a BTK inhibitor and believe this paper further highlights the favorable safety profile and combinability of umbralisib as compared to prior generation PI3k deltas. We believe these data support our plans to develop combinations utilizing umbralisib plus our BTK inhibitor, TG-1701, which has already demonstrated activity in patients in early clinical studies."