On June 5, 2018 Nimbus Therapeutics, a privately held biotechnology company applying deep computational expertise throughout drug discovery and development, reported that it has raised $65 million in new capital to accelerate the company’s pipeline progress and fuel its expansion into new high-value targets aimed at overlapping biological mechanisms in immunology, oncology and metabolic disease (Press release, Nimbus Therapeutics, JUN 5, 2018, View Source [SID1234527171]). Each of the company’s current investors participated in the financing, including Atlas Venture, SR One, Lilly Ventures, Bill Gates, Pfizer Venture Investments, Lightstone Ventures, and Schrödinger. Bruce Booth, Partner of Atlas Venture, Board Chair and co-founder of Nimbus noted, "this round of investment reflects the existing syndicate’s strong and enthusiastic support for Nimbus’ proven management team and exciting new programs."

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"The continued support from our world-class investor base is testament to our team’s repeated success in designing and developing promising candidates through our unique combination of massive computational-chemistry horsepower with founding partner, Schrödinger, coupled with additional cutting-edge technologies in structural biology, cryo-EM, and machine learning-augmented ADMET prediction to rapidly advance our pipeline to the clinic," said Don Nicholson, Ph.D., Chief Executive Officer. "We have made substantial progress across our entire portfolio, including inhibitors of Tyk2 (tyrosine kinase 2) and antagonists of STING (stimulator of interferon genes), both under our immunology alliance with Celgene. Additionally, our wholly owned STING agonist program has generated novel, highly potent, bona fide small molecules with compelling preclinical data."

"This most recent infusion of capital from our investors, together with proceeds from business development activity, enables Nimbus to remain a privately held LLC organization, which has allowed us to transact multiple deals with world leading partners such as Gilead, Celgene, and Genentech," said Jeb Keiper, Chief Financial Officer and Chief Business Officer. "Nimbus’ success has built a nine-figure balance sheet of resources for the rapid advancement and expansion of our pipeline and technology, which will allow us to develop several other undisclosed target programs forward to the clinic in the next few years."

About Tyk2 (tyrosine kinase 2)

Tyk2 is an important signal-transduction kinase for key pro-inflammatory cytokine receptors, including IL-23, IL-12 and interferons α and β. As a result, Tyk2 is a key target for the treatment of several challenging auto-immune disorders, including SLE (lupus), Crohn’s disease, psoriasis, multiple sclerosis, rheumatoid arthritis and others. In addition, some cancers, like T-ALL, appear to be driven by Tyk2 hyper-activation and are responsive to Tyk2 inhibition. Nimbus’ Tyk2 program is partnered under the immunology alliance with Celgene.

About STING (STimulator of INterferon Genes)

STING agonism (turning STING "on") plays a key role in anti-tumor immunity by activation of the innate immune system and induction of the Type-I interferon response, leading to recruitment and activation of cytotoxic T lymphocytes that attack tumor cells. STING agonism provokes anti-tumor responses alone, and in combination with checkpoint inhibitors and cell therapy. Further, STING agonists have been shown to provide benefit in areas of virology, such as efforts to achieve HIV cure. STING antagonists (turning STING "off") may have therapeutic potential in Type-I interferonopathies, such as SLE (lupus), where STING drives an exaggerated interferon response. Nimbus’ STING antagonist program is partnered under the immunology alliance with Celgene, while the STING agonist program remains wholly owned by Nimbus.

On June 5, 2018 BeiGene, Ltd. (NASDAQ:BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, reported that the company will present at the William Blair 2018 Growth Stock Conference in Chicago (Press release, BeiGene, JUN 5, 2018, View Source;p=RssLanding&cat=news&id=2353190 [SID1234527170]). The presentation is scheduled for 10:50 AM CT on Tuesday, June 12, 2018.

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A live webcast can be accessed from the investors section of BeiGene’s website at View Source An archived replay will be available for 90 days following the event.

On June 5, 2018 Verastem, Inc. (President and CEO: Robert Forrester)(NASDAQ:VSTM) and Yakult Honsha Co., Ltd. (President: Takashige Negishi)(Tokyo:2267), reported their entry into an exclusive licensing agreement for Yakult to develop and commercialize Verastem’s duvelisib, a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, for the treatment, prevention or diagnosis of all oncology indications in Japan (Press release, Verastem, JUN 5, 2018, View Source;p=RssLanding&cat=news&id=2353165 [SID1234527169]). Verastem’s New Drug Application (NDA) for duvelisib is currently under review with the U.S. Food and Drug Administration (FDA) and is seeking full approval for the treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and accelerated approval for the treatment of relapsed or refractory follicular lymphoma (FL). On April 9, 2018, Verastem announced that the FDA had accepted the NDA for filing with Priority Review.

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Under the terms of the agreement, Verastem will receive a one-time upfront payment of $10 million from Yakult. Verastem is eligible to receive up to an additional $90 million if certain future pre-specified development and commercialization milestones are successfully achieved by Yakult, plus double-digit royalties based on future net sales of duvelisib in Japan. In exchange, Yakult will receive exclusive rights to develop and commercialize duvelisib in Japan, at its own cost and expense. Yakult will also fund certain global development costs on a pro-rata basis. Verastem will retain all rights to duvelisib outside of Japan.

"In Japan, current therapies to treat CLL/SLL and FL are extremely limited and duvelisib has robust clinical data supporting its efficacy and safety in both indications, which we can build upon," said Masanori Ito, Head of Pharmaceutical Business Division/Managing Executive Officer, Member of the Board of Yakult. "We are eager to collaborate with Verastem to develop duvelisib in these initial hematologic malignancies, and then plan to later expand development to include the additional indications of PTCL and DLBCL. We believe this collaboration underscores our commitment to innovation, growing our oncology franchise, and commercializing medicines that positively impact the lives of patients in Japan."

"This agreement is an important, validating achievement for both duvelisib and Verastem Oncology and speaks to the significant global potential of this novel therapeutic for a broad range of hematologic malignancies," said Robert Forrester, President and Chief Executive Officer of Verastem. "Yakult is an established oncology leader in Japan that successfully markets several branded anti-cancer therapies, including Elplat and Campto. We look forward to working with the world-class development, regulatory and commercial teams at Yakult as they advance oral duvelisib toward commercialization in Japan."

About Duvelisib

Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On June 5, 2018 Verastem, Inc. (President and CEO: Robert Forrester)(NASDAQ:VSTM) and Yakult Honsha Co., Ltd. (President: Takashige Negishi)(Tokyo:2267), reported their entry into an exclusive licensing agreement for Yakult to develop and commercialize Verastem’s duvelisib, a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, for the treatment, prevention or diagnosis of all oncology indications in Japan (Press release, Verastem, JUN 5, 2018, View Source;p=RssLanding&cat=news&id=2353165 [SID1234527168]). Verastem’s New Drug Application (NDA) for duvelisib is currently under review with the U.S. Food and Drug Administration (FDA) and is seeking full approval for the treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and accelerated approval for the treatment of relapsed or refractory follicular lymphoma (FL). On April 9, 2018, Verastem announced that the FDA had accepted the NDA for filing with Priority Review.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Under the terms of the agreement, Verastem will receive a one-time upfront payment of $10 million from Yakult. Verastem is eligible to receive up to an additional $90 million if certain future pre-specified development and commercialization milestones are successfully achieved by Yakult, plus double-digit royalties based on future net sales of duvelisib in Japan. In exchange, Yakult will receive exclusive rights to develop and commercialize duvelisib in Japan, at its own cost and expense. Yakult will also fund certain global development costs on a pro-rata basis. Verastem will retain all rights to duvelisib outside of Japan.

"In Japan, current therapies to treat CLL/SLL and FL are extremely limited and duvelisib has robust clinical data supporting its efficacy and safety in both indications, which we can build upon," said Masanori Ito, Head of Pharmaceutical Business Division/Managing Executive Officer, Member of the Board of Yakult. "We are eager to collaborate with Verastem to develop duvelisib in these initial hematologic malignancies, and then plan to later expand development to include the additional indications of PTCL and DLBCL. We believe this collaboration underscores our commitment to innovation, growing our oncology franchise, and commercializing medicines that positively impact the lives of patients in Japan."

"This agreement is an important, validating achievement for both duvelisib and Verastem Oncology and speaks to the significant global potential of this novel therapeutic for a broad range of hematologic malignancies," said Robert Forrester, President and Chief Executive Officer of Verastem. "Yakult is an established oncology leader in Japan that successfully markets several branded anti-cancer therapies, including Elplat and Campto. We look forward to working with the world-class development, regulatory and commercial teams at Yakult as they advance oral duvelisib toward commercialization in Japan."

About Duvelisib

Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On June 4, 2018 Transgene (Paris:TNG), a biotech company that designs and develops virus-based immunotherapies against cancers and infectious diseases, and University of Leeds, reported that new clinical data1 obtained with Pexa-Vec further demonstrate anti-tumor activity after intravenous (i. v.) infusion (Press release, Transgene, JUN 4, 2018, View Source [SID1234621826]). These data were presented by Dr. Alan Anthoney (University of Leeds) in a poster presentation at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, June 4, in Chicago .

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These first clinical results confirm Pexa-Vec’s activation of anti-tumor immunity and targeted oncolytic activity. The key findings of the trial show:

selective expression and replication of Pexa-Vec in the tumor tissues;
induction of a robust anti-tumor immune response:
with the stimulation of an adaptive response (T cells) targeted to tumor specific antigens,
the activation of innate immune response (NK cells), and
an elevation of cytokines associated with immune stimulation;
one partial and one complete necrosis of tumors among the patients with colorectal cancer and liver metastases (pathological responses);
upregulation of PD-L1 and PD-1 signaling molecules, a finding that strongly supports the rationale for combining Pexa-Vec with anti-PD-1 inhibitors.
Dr Alan Anthoney, Consultant in Medical Oncology at Leeds Teaching Hospitals, Senior Lecturer in the Institute of Cancer & Pathology at the University of Leeds and principal investigator of the trial, said: "We are very encouraged to report that a single IV administration of Pexa-Vec displayed cytolytic activity at tumor sites, where it elicited a robust activation of tumor-antigen specific immune cells. In one patient with colorectal cancer liver metastasis a complete pathological response has been observed. These data clearly support the anti-tumor activity of Pexa-Vec. The final data from this trial will be published in an upcoming paper. Our decision to lead this clinical trial, investigating the potential of new weapons against cancers, testifies to our commitment at Leeds University and Leeds Teaching Hospitals NHS Trust to evaluate new innovative options that might improve the lives of our patients with cancer."

Maud Brandely, Chief Medical Officer of Transgene, added: "These very positive translational data confirm the targeted oncolytic activity and the potential of Pexa-Vec in advanced stages cancers. The observed upregulation of PD-1 and PD-L1 positive pathways strongly supports the rationale for combining Pexa-Vec with anti-PD-1 immunotherapies, which is the focus of an ongoing Phase 1/2 trial in the first-line treatment of liver cancer (HCC). These data are also crucial for our next generation of multifunctional oncolytic viruses based on our Invir.IOTM platform: this trial clearly shows that Vaccinia virus based immunotherapeutics can reach the tumor sites after i. v. administration, and selectively replicate within cancer cells. This neoadjuvant trial is the first clinical trial led by Transgene to readout this year. We look forward to announcing additional clinical results this year, not only with Pexa-Vec, but also on our four other clinical-stage immunotherapeutics."

About the Pexa-Vec "neo-adjuvant" trial:
This clinical study is aimed at evaluating the biological effects of pre-operative intravenous administration of Pexa-Vec prior to planned surgical resection of locally advanced/poor prognosis or metastatic cancers. This single center, open label, non-randomized trial recruited 9 patients including 8 evaluable patients (3 with metastatic melanoma and 5 with colorectal cancer metastases to the liver). They received a single intravenous dose of 1×109 pfu of Pexa-Vec, 14 days prior to planned surgery. Up to 6 blood samples were collected pre- and post- injection for each patient. Imaging was performed prior at baseline and within 7 days prior to surgery. Tumor tissue was collected at surgery for histologic and translational assessments.
University of Leeds is the sponsor of the trial that was supported by Transgene and run through the NIHR Clinical Research Facility at St James’ Hospital, Leeds.

The poster is available on Transgene’s website.

About Pexa-Vec
Pexa-Vec (JX594) is an oncolytic immunotherapeutic based on an oncolytic vaccinia virus armed with a GM-CSF gene that promotes an anti-tumor immune response. Pexa-Vec is designed to selectively target and destroy cancer cells through three different mechanisms of action: selectively destroy cancer cells through the direct lysis (breakdown) of cancer cells through viral replication, reduce the blood supply to tumors through vascular disruption, and stimulate the body’s immune response against cancer cells.
Pexa-Vec is currently being evaluated in a Phase 3 trial in hepatocellular carcinoma (HCC, liver cancer) in combination with sorafenib (current standard of care). Other trials evaluating the oncolytic virus in solid tumors are underway and expected to readout in 2018, including a Phase 2 trial in combination with nivolumab (HCC).
Transgene has exclusive rights to develop and commercialize Pexa-Vec for the treatment of solid tumors in Europe. Its partner SillaJen, Inc. is focused on developing Pexa-Vec for the North American market and has also granted exclusive development and commercial rights to Pexa-Vec in Hong Kong and The People’s Republic of China to Lee’s Pharmaceutical.