On December 3, 2018 Tiziana Life Sciences plc (Nasdaq: TLSA / AIM: TILS), a biotechnology company focusing on the discovery and development of innovative therapeutics for inflammation and oncology indications, reported that patient enrollment in the ongoing Phase 2a clinical trial (CDKO-125A-010) is completed (Press release, Tiziana Life Sciences, DEC 3, 2018, View Source [SID1234531836]). This is a single-arm, repeated-dose, 6-month duration study to evaluate safety, tolerability and anti-tumor activity of Milciclib in Sorafenib-refractory or -intolerant patients with unresectable or metastatic HCC. Topline data from this multi-center trial, being conducted in Italy, Greece and Israel, will be available in the second quarter of 2019.

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Previously we reported interim analysis data from the first 10 patients, following 6 months of treatment, showing that Milciclib (100 mg once daily; 4 days on/3 days off every 4 weeks defining each cycle) was well-tolerated in this HCC patient population. It was concluded by an Independent Data Monitoring Committee (IDMC) that there were no major signals of tolerability concerns, and the IDMC allowed continuation of patient enrollment in the trial. Following completion of 6 months of treatment, three patients opted to continue treatment under the compassionate use program. Notably, one patient is still continuing treatment in the 14th month and the other two patients received treatment until 9th month and 13th month, respectively.

About HCC

HCC is the 5th most common cancer and the 3rd cause of cancer mortality worldwide. In 2007 the approval by the European Medical Agency (EMA) and Food and Drug Administration (FDA) of Sorafenib (Nexavar), an inhibitor of several receptor tyrosine kinases, in HCC represented the first systemic therapy for improving outcome in patients unsuitable for loco-regional and surgical therapies and created a new standard of treatment for the disease. However, although significant in respect to placebo, the benefits of Sorafenib are modest; the response rate is less than 3%, the improvement in median survival is 2-3 months and the drug-related symptoms are not ordinary. The complex multi-factorial etiology of HCC warrants a need for systemic therapies that target different signaling cascades to provide improved efficacy and safety for both naive patients presenting with unresectable, advanced stage and those who suffer recurrence after curative treatments (resection, ablation and transplantation).

About Milciclib

Milciclib (PHA-848125AC) is a small molecule inhibitor of several cyclin dependent kinases such as CDK1, CDK4, CDK5 and CDK7. CDKs are serine threonine kinases that play crucial roles in progression of the cell cycle from G1 to S phase. Overexpression of CDKs and other downstream signaling pathways that regulate cell cycles have been frequently associated with development of resistance towards chemotherapies. In a Phase 1 study, oral treatment with Milciclib was well-tolerated and the drug showed promising clinical responses in patients with advanced solid malignancies such as in NSCLC, pancreatic and colon cancer, thymic carcinoma and thymoma. Additionally, milciclib met its primary endpoint in two separate Phase 2 multi-center clinical trials (CDKO-125A-006: 72 patients and CDKO-125A-007: 30 patients) in thymic carcinoma and thymoma patients.

About Sorafenib

Sorafenib (co-developed and co-marketed by Bayer and Onyx Pharmaceuticals as Nexavar) is a small molecular multi-tyrosine kinase inhibitor drug approved for the treatment of primary kidney cancer (advanced renal cell carcinoma), HCC, and radioactive iodine resistant advanced thyroid carcinoma. Treatment with Sorafenib induces autophagy, which may suppress tumor growth. However, autophagy can also cause drug resistance.

On December 3, 2018 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on using the body’s immune system to fight cancer, presented the latest data from its ongoing study focusing on early detection of breast cancer, utilizing Cchek, its artificial intelligence (AI) driven cancer detection technology, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy (Press release, Anixa Biosciences, DEC 3, 2018, View Source [SID1234531852]). The conference is designed to integrate multidisciplinary facets of basic cancer immunology and immunotherapy to broaden the understanding of ways to harness the immune system to address cancer. The conference was held November 27–30, 2018 in Miami Beach, Florida.

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Anixa presented data demonstrating the ability of Cchek to identify the presence of early stage breast cancer in a subject by using its AI technology to analyze a simple blood draw. The Cchek technology demonstrated a sensitivity of 89.3% when detecting early stage breast cancer (Stage I or II) and a specificity (the ability to correctly identify healthy subjects) of 94.7% when used to test blinded samples. Furthermore, Cchek was also able to detect the early stages of breast cancer (Stage 0) in subjects with biopsy-confirmed ductal carcinoma in situ (DCIS), a type of pre-cancerous/non-invasive breast lesion that often leads to invasive breast cancer, with 72% sensitivity. The presentation is available on Anixa’s website, or it may be requested by sending an email to AACR (Free AACR Whitepaper)[email protected] and including your name, title, and contact information.

"We are pleased to have made our presentation titled, Combining the immunophenotyping of MDSCs and lymphocytes with artificial intelligence (AI) to predict early stage breast cancer, at the AACR (Free AACR Whitepaper) Tumor Immunology and Immunotherapy conference. This data was focused on using our Cchek technology to detect breast cancer in its early stages, primarily stage I and II when the cancer is more easily treated. The majority of screening technologies currently used for breast cancer detection, such as mammography, have the ability to detect later stage breast malignancies rather successfully but have shown difficulty with earlier stages. A major challenge with mammography is the large number of false positives resulting in overtreatment and many unnecessary biopsies," stated Dr. Amit Kumar, President and CEO of Anixa Biosciences. "As our study continues, we hope to enable a better approach for identifying breast cancer as early as possible including even when a non-invasive breast cancer lesion is present, such as in the case of DCIS. We have previously announced that our initial commercial focus is on a prostate cancer test for which we will be meeting with the USFDA on December 17, 2018. We recently presented our latest prostate cancer data at the 33rd Annual Meeting of The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)," added Dr. Kumar.

American Association of Cancer Research (AACR) (Free AACR Whitepaper)
The American Association of Cancer Research (AACR) (Free AACR Whitepaper) (www.aacr.org) is a 501(c)(3) public charity headquartered in Philadelphia, PA. The mission of the AACR (Free AACR Whitepaper) is to prevent and cure cancer through research, education, communication and collaboration. Through its programs and services, the AACR (Free AACR Whitepaper) fosters cancer research and related biomedical science; accelerates the dissemination of new research findings among scientists and others dedicated to the conquest of cancer; promotes science education and training; and advances the understanding of cancer causes, prevention, diagnosis and treatment throughout the world.

On December 3, 2018 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that previously announced interim data from the Company’s Phase 2 study evaluating prexigebersen as a treatment for acute myeloid leukemia (AML) were presented in a poster at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place from December 1-4, 2018 in San Diego, CA (Press release, Bio-Path Holdings, DEC 3, 2018, View Source [SID1234531869]).

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Maro Ohanian, M.D., Assistant Professor of the Department of Leukemia at The University of Texas MD Anderson Cancer Center, presented the poster titled, "Interim Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with Untreated Acute Myeloid Leukemia (AML)." The poster reviewed interim data from the Company’s open-label Phase 2 study evaluating the efficacy and safety of prexigebersen in conjunction with low-dose cytarabine (LDAC), a therapeutic regimen well established in treatment of AML patients who cannot or elect not to be treated with more intensive chemotherapy. The primary objective of the study is to determine whether the combination of prexigebersen and LDAC provides greater efficacy than what would be expected with LDAC alone in this de novo patient population.

Prexigebersen was safely administered to patients with untreated AML, who were considered unsuitable for standard chemotherapy. Of the 17 evaluable patients, there were four patients (24%) who achieved complete responses and four patients with stable disease including one patient who achieved a morphologic leukemia free state and two patients who had significantly reduced bone marrow blasts. In total, 47% of the evaluable patients showed some form of response, including stable disease, to the combination treatment. Efficacy data are encouraging in this challenging population in which the majority of patients had secondary AML or adverse-risk AML, and compares favorably to the reported CR (complete remission), CRp (complete remission with incomplete platelet recovery), and CRi (complete remission with incomplete hematologic recovery) rate with LDAC alone of 7-13%1.

"We are excited to be presenting these important data at ASH (Free ASH Whitepaper) before an audience of world-leading scientists and oncologists, as they demonstrate the potential for the combination of prexigerbesen and LDAC to safely and effectively treat these de novo AML patients, including doubling the complete response to treatment compared to LDAC treatment alone," noted Peter H. Nielsen, chief executive officer of Bio-Path. "We look forward to presenting final data from this study as we expect they will provide even better results for these patients suffering with AML."

1 Heiblig, Mediterr J Hematol 2016; Kantarjian, J Clin Oncol 2012; Dohner, Blood 2014

On December 3, 2018 Shenzhen Salubris Pharmaceuticals Co. Ltd. (Salubris, SZSE: 002294) and Viracta Therapeutics, Inc. reported the initial closing of a financing, with Salubris committing $10 million as the lead investor (Press release, Viracta Therapeutics, DEC 3, 2018, View Source [SID1234534657]). New investor Virtus Inspire Ventures, as well as Viracta’s existing investors, NantKwest, Inc., Latterell Venture Partners and Forward Ventures, all participated in the financing. In addition to the financing, Salubris and Viracta have entered into an exclusive Collaboration and License Agreement to bring Viracta’s novel treatment approach for virus-associated malignancies to China.

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Viracta is advancing its innovative approach for viral-associated cancers in a Phase 1b/2 clinical trial for Epstein Barr Virus (EBV)-associated lymphomas in the US and Brazil. The approach is the first targeted, orally administered therapy for EBV-associated malignancies. It incorporates Viracta’s proprietary drug candidate, nanatinostat (VRx-3996) in combination with an antiviral as a targeted treatment to eradicate a range of viral-associated cancers. Viracta plans to initiate a clinical trial for the treatment of EBV-associated solid tumors in the coming year.

EBV-associated nasopharyngeal carcinoma is endemic in Southern China. EBV is also highly associated with the incidence and progression of other solid tumors, including gastric carcinoma, as well as NK/T cell lymphomas found throughout China. Salubris CEO, Kevin Ye highlighted, "EBV-driven cancers disproportionately impact patients in China. We look forward to partnering with Viracta to develop this new treatment option in China. The approach holds the potential to provide a valuable new treatment option for these patients, who currently still face considerable morbidity and mortality."

"We are committed to bringing our treatment approach to cancer patients around the world," commented Viracta CEO, Ivor Royston, MD. "We look forward to developing nanatinostat in collaboration with Salubris to address these major healthcare needs in China."

Under the terms of the license agreement, in addition to the equity investment made by Salubris, Viracta is eligible to receive up to $58 million in pre-commercial milestones as well as significant sales level-triggered commercial milestones and tiered royalties on sales. The Companies will collaborate for clinical development of the treatment approach for viral-associated cancers with Salubris taking on responsibility for development within the Peoples Republic of China (excluding Hong Kong, Macau and Taiwan). Viracta retains development responsibility as well as commercial rights outside of China.

On December 3, 2018 ArQule, Inc. (Nasdaq: ARQL) reported its presented preliminary results from the Company’s Phase 1 dose escalation study for ARQ 531, an orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK) in patients with relapsed or refractory hematologic malignancies at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego (Press release, ArQule, DEC 3, 2018, View Source [SID1234531820]).

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Dr. Brian Schwartz, Chief Medical Officer of ArQule commented, "We are encouraged by the pace at which this trial is progressing and by the level of target engagement we are observing as we continue to dose escalate. Four out of the five heavily pretreated CLL patients enrolled in the last two cohorts with the BTK-C481S mutation have experienced tumor shrinkage."

"These data provide further evidence of ARQ 531’s potential to become a new therapeutic option for patients with B cell malignancies," commented Paolo Pucci, Chief Executive Officer of ArQule. "There is significant unmet need for patients with relapsed or refractory B-cell malignancies in particular those with the C481S-mediated resistance to irreversible BTK inhibitors such as ibrutinib."

The reported data are from the ongoing Phase 1, open label, single arm dose escalation 3+3 study and include the first six cohorts (n=20) at dose levels of 5, 10, 15, 20, 30 and 45 mg once a day in patients with relapsed or refractory chronic lymphocytic leukemia (CLL), small lymphocytic leukemia (SLL), Waldenstrom’s macroglobinemia and B-cell Non-Hodgkin lymphomas.

Key findings presented include the following:

ARQ 531 has demonstrated a manageable safety profile with no dose-limiting toxicities observed to date
Pharmacokinetic data are nearly dose proportional with a mean plasma half-life that supports QD dosing
Pharmacodynamic biomarkers for cohorts 4 through 6 showed profound pBTK inhibition
Anti-tumor activity, with reduction of tumor burden, was observed in 9 out of 20 patients
At the first assessment of tumor response, 80% of the ibrutinib refractory, heavily pretreated CLL patients (4 out of 5) in the highest dose cohorts (30 and 45 mg) experienced tumor shrinkages
Four out of 5 lymphoma patients derived benefit with shrinkages between 27 and 58%, including 1 PR in a Follicular Lymphoma patient who began at 5 mg, was dose escalated to 15 and then 45 mg, and remains on therapy after 70 weeks
The study is on-going and dose escalation continues
The presented poster, A Phase 1 Dose Escalation Study of ARQ 531 in Selected Patients with Relapsed or Refractory Hematologic Malignancies, is available on the company’s website at View Source

The Company will hold a conference call and webcast today at 9:00 a.m. ET to discuss these results. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

About BTK and ARQ 531

Bruton’s tyrosine kinase, BTK, is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in blood cancers. ARQ 531 is an orally bioavailable, potent and reversible BTK inhibitor. Biochemical and cellular studies have shown that ARQ 531 inhibits both the wild type and C481S-mutant forms of BTK. The C481S-mutation is a known resistance mechanism for first generation irreversible BTK inhibitors. In preclinical studies, ARQ 531 has demonstrated good oral bioavailability as well as favorable pharmacokinetic, pharmacodynamic and metabolic properties.