On May 16, 2018 Tiziana Life Sciences plc (AIM: TILS), a clinical stage biotechnology company developing targeted drugs for cancer and inflammatory diseases, reported that the independent data monitor committee (IDMC) completed a second, interim analysis of tolerability data from the first eleven treated patients and recommended expansion of the initial cohort to continue enrolment of an additional 20 patients to complete the trial (Press release, Tiziana Life Sciences, MAY 16, 2018, View Source;intolerable-Unresectable-or-Metastatic-Hepatocellular-Carcinoma-HCC-Patients [SID1234526674]).

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This phase 2a multi-centre and multi-country clinical trial (CDKO-125A-010) in sorafenib-refractory or -intolerable patients with unresectable or metastatic HCC is being conducted in Greece, Italy and Israel. Since, this was the first trial with milciclib in HCC patients, a second, interim analysis was scheduled following completion of treatment for the first 11 patients before initiating enrolment of the next 20 patients. Thus, demonstration of good tolerability with acceptable incidence of serious adverse events is an important milestone to initiate a phase 2b trial evaluating combination of milciclib with sorafenib (Nexavar; Bayer Germany (BAYN.GR)) in HCC patient.

Major findings were as follows:

Milciclib treatment was well-tolerated with manageable drug-related toxicities. The IDMC concluded that there were no major signals of tolerability concerns and therefore favours proceeding to expand enrolment.

Four patients have completed the study per protocol (6 cycles of treatment in 6 months). Two of these patients and their care provider opted to continue receiving milciclib at full dose as part of compassionate use. A third patient is awaiting ethical committee (EC) approval.

Gabriele Cerrone, Chairman of Tiziana Life Sciences, commented: ”Establishment of tolerability of milciclib as a single agent in HCC patients is a key pre-requisite to initiate the phase 2b trial to evaluate dosing, tolerability and clinical activity of milciclib in combination with sorafenib (Nexavar; Bayer Germany) in HCC patients”.

Kunwar Shailubhai, CEO & CSO of Tiziana Life Sciences, commented: "We are pleased with the conclusion of IDMC that milciclib treatment showed no major signals of tolerability concerns in sorafenib-refractory or -intolerable HCC patients. These findings are consistent with the findings reported earlier on the long-term tolerability and clinical activity of milciclib in thymic carcinoma, thymoma1 and other solid cancers2. Results from these clinical studies strongly warrant further clinical development of milciclib for treatments of HCC and other cancers".

Cited References

1. Press Release on announcement of clinical data in thymoma and thymic carcinoma.
www.tizianalifesciences.com

2 . Aspeslagh, S., Shailubhai, K., Bahleda, R. et al. (2017). Phase I dose-escalation study of Milciclib in combination with gemcitabine in patients with refractory solid tumors. Cancer Chemother Pharmacol. 79:1257-1265

About Hepatocellular Carcinoma

Hepatocellular cancer 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 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. Therefore, more effective systemic therapy is required 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 (CDKs) 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 signalling pathways that regulate cell cycles have been frequently found to be associated with development of resistance towards chemotherapies. In a phase I study, oral treatment with Milciclib was found to be well-tolerated and the drug showed promising clinical responses in patients with advanced solid malignancies such as in thymic carcinoma, pancreatic carcinoma and colon cancer.

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), advanced primary liver cancer (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 May 16, 2018 BerGenBio ASA (OSE: BGBIO) reported that the company and its collaborators will present new interim clinical and biomarker data from its extensive Phase II clinical development programme with bemcentinib, a selective, oral AXL inhibitor, at the 2018 annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) at McCormick Place in Chicago, IL (1-5 June 2018) (Press release, BerGenBio, MAY 16, 2018, View Source [SID1234526697]). Abstracts are now available online at View Source and details of the presentations are below. The posters presented at ASCO (Free ASCO Whitepaper) will be made available on www.bergenbio.com in the Investors / Presentations section following the sessions.

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Presentations at ASCO (Free ASCO Whitepaper)
Monday 4 June, 8:00 AM – 11:30 AM Central Daylight Time (Hall A)

Phase II open-label, multi-centre study of bemcentinib (BGB324), a first-in-class selective AXL inhibitor, in combination with pembrolizumab in patients with advanced NSCLC.
James Lorens, PhD et al
Session: Developmental Therapeutics – Immunotherapy
Poster Board: #292, Abstract 3078
Identification of predictive and pharmacodynamic biomarkers associated with the first-in-class selective AXL inhibitor bemcentinib across multiple Phase II clinical trials.

Robert J Holt, PhD et al
Session: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics
Poster Board: #385, Abstract 2559
Analysis of anti-leukemic activity, predictive biomarker candidates, immune activation and pharmakodynamics in R/R AML and MDS in response to treatment with bemcentinib (BGB324), a first-in class selective AXL inhibitor, in a Phase II open-label, multi-centre study.

Bjørn T. Gjertsen, MD, PhD et al
Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Poster Board: #80, Abstract 7020
To be discussed at the Poster Discussion Session on Monday 4 June, 11:30 AM – 12:45 PM, at E450
Monday 4 June, 1:15 PM – 4:45 PM CDT (Hall A)

A randomized Phase Ib/II study of the selective small molecule AXL inhibitor bemcentinib (BGB324) in combination with either dabrafenib/trametinib or pembrolizumab in patients with metastatic melanoma.
Oddbjørn Straume, MD, PhD et al
Session: Melanoma/Skin Cancers
Poster Board: #375, Abstract 9548
BerGenBio reception
Coinciding with ASCO (Free ASCO Whitepaper), BerGenBio will host a reception for collaborators, investors, analysts, media and other interested parties on 2 June at The School of the Art Institute Ballroom in Chicago. At this event, short presentations will be given by clinical investigators participating in the bemcentinib clinical trials and by KOL experts in AXL kinase function. For further details and to receive an invitation, please click here.

The presentations will be made available on BerGenBio’s website in the Investors / Presentations section following the event.

On May 16, 2018 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO FR0010095596), a biotechnology company specializing in the development of innovative drugs in oncology, notably against rare or resistant forms of cancer, reported its consolidated revenues and cash position at March 31, 2018 (Press release, Onxeo, MAY 16, 2018, View Source [SID1234526713]).

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Judith Greciet, Chief Executive Officer of Onxeo, said: "The first quarter of 2018 was highlighted by strong momentum in the development of AsiDNA, our lead product candidate. Following the significant efforts of our R&D team over the previous months, we recently initiated the DRIIV phase I clinical trial in order to evaluate the potential of AsiDNA, our first-in-class DNA repair inhibitor, administered intravenously, in patients with advanced solid tumors. We expect interim data from this study to be available in the second half of 2018. If these results confirm both the safety profile of AsiDNA and its activity, we will have achieved a key milestone in our AsiDNA development program. Importantly, we continue to advance our core R&D programs according to plan while maintaining strict cost control. As such, we expect that our current cash position of €9.2 million will support our currently planned activities until mid-2019, including through multiple potentially value-creating inflection points for our company.

On May 16, 2018 FogPharma reported the closing of a $66 million Series B financing (Press release, FogPharma, MAY 16, 2018, View Source [SID1234552458]). The round was led by 6 Dimensions Capital, with participation by additional new investors, including GV (formerly Google Ventures), Blue Pool Capital, Horizons Ventures, Nan Fung Group, and Leerink Partners. All existing investors participated in the round including Deerfield Management, Boyu Capital, WuXi AppTec Corporate Ventures, and a prominent international group of non-institutional investors.

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FogPharma was founded by renowned scientist-entrepreneur Dr. Gregory Verdine, whose Harvard lab invented cell-penetrating miniproteins and who coined the term "drugging the undruggable" to describe his life’s mission. FogPharma’s research and development strategy is unique in that it pairs a broadly-enabling new drug class, designed to access the cell interior, with a massively parallelized engine to discover – rapidly and on-demand – drugs that engage the most intractable of disease targets. FogPharma’s drug discovery engine has been configured to deliver multiple new medicines in rapid succession, with clinical entry for the first product, a first-in-class beta-catenin antagonist, by the end of 2019, followed by a steady stream of first-in-class clinical product candidates addressing other intractable targets.

While FogPharma’s approach has many therapeutic applications, the company’s early focus is on drugging the major, intractable drivers of cancer and on pharmacological management of the immune response.

"One of the most important challenges of our time is making actionable the enormous, inactionable trove of biological data on human disease targets. FogPharma is addressing this challenge by bringing forward a new class of medicines that combine the cell-penetrating ability of small molecules with the broad, target power of biologics, and by learning how to discover these medicines better, faster and smarter," said Verdine, chief executive officer and chief scientific officer, FogPharma.

Added Verdine, "We are thrilled to have such an incredible group of investors who share our vision of fundamentally advancing the treatment of cancer and other serious diseases with few if any current treatment options. We are excited at the opportunity provided by this financing to propel our programs and drug discovery engine forward."

Proceeds from the Series B raise will enable the company to advance its first-in-class beta-catenin inhibitor (iCat) program into Phase 2 development for cancer indications involving Wnt pathway activation. The financing will also be used to advance clinical development of its first-in-class Cbl-b inhibitor program and a third as-yet-undisclosed program through IND-enabling studies, and FogPharma’s drug discovery platform for three additional, distinct and differentiated forms of cell-penetrating miniproteins.

In association with the Series B financing, FogPharma has appointed to its board of directors: Dr. Leon Chen, chief executive officer, 6 Dimensions Capital; Dr. Krishna Yeshwant, general partner, GV; and Dr. Rick Klausner, founder and director, Juno Therapeutics, GRAIL and Mindstrong.

"There is substantial and persistent interest in tackling targets like beta-catenin and Cbl-b, which are clearly important biologically and medically, but untouchable by conventional therapeutics. I was captivated by the FogPharma team’s unprecedented ability to go after these and other intractable targets," said Klausner, formerly director of the National Cancer Institute and executive director of the Bill and Melinda Gates Foundation.

"The opportunity in the near term to bring cell-penetrating miniproteins to the 20-25 percent of cancer patients whose disease is driven by the Wnt pathway is tremendous, and the opportunity beyond that to be the first to drug Cbl-b for immuno-oncology indications, is extraordinary. On behalf of 6 Dimensions Capital, I am thrilled to have led this exceptional investor syndicate and foster FogPharma’s mission," said Chen, chief executive officer, 6 Dimensons Capital, and member of the FogPharma board of directors.

Through seed and Series A financing, FogPharma previously secured $11 million bringing the company’s total funding to-date to $77 million.

In addition to FogPharma, Verdine founded and leads LifeMine Therapeutics, which has buit a first-of-its-kind, genomically-enabled drug discovery platform that can rapidly develop new drugs from fungi. Both LifeMine Therapeutics and FogPharma are headquartered in Cambridge, Mass. and were established operationally in 2016. Verdine is highly regarded for having moved seamlessly between successful roles as life scientist, entrepreneur, investor, and chief executive throughout his career. He is Erving Professor at Harvard University and Harvard Medical School and has founded multiple NASDAQ-listed biotech companies including Wave Life Sciences, Enanta, Eleven Bio, Variagenics, Tokai, Aileron, and a private company, Gloucester Pharmaceuticals, acquired by Celgene in 2010.

On May 16, 2018 bluebird bio, Inc. (Nasdaq: BLUE) reported that updated interim data from CRB-401, its Phase 1 study of bb2121, an anti-BCMA CAR T cell therapy being developed by the company and Celgene, will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois (Press release, bluebird bio, MAY 16, 2018, View Source [SID1234526698]).

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"Since we first presented data for bb2121 in heavily pretreated patients with multiple myeloma, we have been hopeful about its potential to markedly improve the outcomes and expectations for this incurable disease," said David Davidson, M.D., chief medical officer, bluebird bio. "One of the most striking changes has been to see patients testing negative for minimal residual disease (MRD), which was previously unexpected in this population with advanced disease. As the data from the Phase 1 study continue to advance our understanding of the bb2121 dose response and safety profiles, we and Celgene are applying these lessons to optimize outcomes in KarMMA, the ongoing registration-enabling study of bb2121 in patients with relapsed/refractory multiple myeloma."

Oral Presentation
bb2121 anti-BCMA CAR T-cell therapy in patients with relapsed/refractory multiple myeloma: Updated results from a multicenter phase I study. (Abstract 8007)

Presenter: Noopur S. Raje, M.D., Massachusetts General Hospital, Boston, Massachusetts
Date & Time:Friday, June 1, 2018, 2:45-5:45 p.m. CT (3:45-6:45 p.m. ET)
Location: E450
Session Title: Hematologic Malignancies—Plasma Cell Dyscrasia

Poster Presentation
Early MRD negativity to predict deepening myeloma response in relapsed/refractory multiple myeloma (RRMM) patients treated with bb2121 anti-BCMA CAR T cells. (Abstract 8024)

Presenter: Nikhil C. Munshi, M.D., Dana-Farber Cancer Institute, Boston, Massachusetts
Poster Session Date & Time:Monday, June 4, 2018, 8:00 – 11:30 a.m. CT (9:00 a.m. – 12:30 p.m. ET)
Location: Poster Area Hall A
Session Title: Hematologic Malignancies—Plasma Cell Dyscrasia

Conference Call & Webcast Information

bluebird bio will host a conference call and live webcast at 6:30 p.m. CT (7:30 p.m. ET) on Friday, June 1, 2018. To access the live webcast, please visit the "Events & Presentations" page within the Investors and Media section of the bluebird bio website at View Source Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 1489304. A replay of the webcast will be available on the bluebird bio website for 90 days following the call.