On September 27, 2018 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) reported it has entered into a strategic licensing and collaboration agreement with Novartis to manufacture and supply the CAR-T cell therapy Kymriah (tisagenlecleucel) in China (Press release, Cellular Biomedicine Group, SEP 27, 2018, View Source [SID1234529612]). Novartis will be the exclusive holder of the marketing license.

Upon the closing of the licensing and collaboration agreement, CBMG – a clinical-stage biopharmaceutical firm engaged in the development of immunotherapies for cancer and stem cell therapies for degenerative diseases – will receive $40 million in an equity purchase from Novartis at $27.43/share for approximately 9% equity. Novartis will receive certain royalty-free intellectual property worldwide rights to certain CBMG CAR-T related technology. CBMG will receive a single-digit escalating percentage collaboration payment based on net product sales. CBMG will receive a mark-up from Novartis on the manufacturing cost. CBMG will take the lead in the manufacturing process, and Novartis will lead distribution, regulatory and commercialization efforts in China.

"This collaboration with Novartis reflects our shared commitment to bringing the first marketed CAR-T cell therapy Kymriah, a transformative treatment option currently approved in the United States, European Union and Canada for two difficult-to-treat cancers, to China where the number of patients in need remains the highest in the world," said Tony Liu, Chief Executive Officer, CBMG. "Together with Novartis, we hope to bring the first CAR-T cell therapy to patients in China. In addition, we continue to focus on developing CBMG’s pipeline of immuno-oncology assets."

"Novartis is committed to bringing new hope to children and adults who are suffering from aggressive forms of blood cancer and currently have limited therapeutic options. We are proud to collaborate with CBMG in China to expand our Kymriah manufacturing capabilities and the potential to facilitate safe and seamless delivery of this innovative, one-time treatment to patients in need," said Pascal Touchon, Senior VP and Global Head, Cell & Gene, Novartis Oncology.

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On September 27, 2018 Targovax ASA (OSE: TRVX), a clinical stage biotechnology company developing immune activators to target hard to treat solid tumors, reported interim tumor response data from the first six patients treated with ONCOS-102 followed by the checkpoint inhibitor (CPI) pembrolizumab (KEYTRUDA) in patients with advanced melanoma whose disease has progressed after prior CPI treatment (Press release, Targovax, SEP 27, 2018, View Source [SID1234529606]). Results show that one of the six patients had a complete response to the treatment.

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One of the main aims of the trial has been achieved, which is to demonstrate that ONCOS-102 has the potential to immune activate CPI refractory patients to respond to PD-1 blockade. Complete responses are rarely seen in this patient population. The patients received three ONCOS-102 injections prior to KEYTRUDA treatment, which may be insufficient in highly advanced disease. As such, Targovax and the investigators now intend to optimize the dosing schedule by increasing the number of ONCOS-102 injections and expand the trial with additional patients.

Dr. Magnus Jäderberg, CMO of Targovax, said: "Given the limited number of patients who have completed the study to date, it is encouraging to already see a complete response to ONCOS-102 primed KEYTRUDA treatment in this CPI refractory patient population. This case is particularly interesting, as the patient became refractory to KEYTRUDA before entering our trial. At the same time, five patients progressed, which we believe may be partly due to an insufficient number of ONCOS-102 injections. Consequently, we have agreed with the investigators to expand the trial with additional patients, who will receive an increased number of ONCOS-102 injections. The complete response, combined with the optimized dosing regimen, makes us optimistic that we may demonstrate the full potential of ONCOS-102 in the checkpoint inhibitor refractory setting."

The results will be presented at a KOL event hosted by Targovax in New York City on 11 October 2018, which will also be available by webcast. Invitation and full event details has been issued separately and is posted on www.targovax.com.

Conference call

At 14:00 CET (08:00 EST) today, Targovax will host a conference call to answer questions about the results. Call-in details can be found below.Call-in numbers:

Norway Toll-Free Number: 80062196
Norway Toll Number: +47-23500243
UK Toll-Free Number: 08003589473
UK Toll Number: +44-3333000804
US Toll-Free Number: +1-855-85-70686
US Toll Number: +1-6319131422

Access code: 60258218#

Please make sure to dial in at least 5-10 minutes ahead to complete your registration.

See attached list for more dial-in numbers:

View Source

About the trial

In this open label trial running in three centers in the USA, patients that have progressed on CPI treatment receive three intra-tumoral ONCOS-102 injections during the first week, followed by eight infusions of the programmed cell death protein 1 (PD-1) blocking CPI pembrolizumab (KEYTRUDA). As reported in December 2017 and January 2018, the first planned safety review passed without any issues. Both local and systemic immune activation, which includes T-cell tumor infiltration and increased PD-1 expression on CD8+ T-cells, has been confirmed in all evaluated patients.

On September 26, 2018 Arvinas, Inc. (Nasdaq: ARVN), a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development and commercialization of therapies to degrade disease-causing proteins, reported the pricing of its initial public offering of 7,500,000 shares of its common stock at a public offering price of $16.00 per share, for aggregate gross proceeds of approximately $120 million, before deducting underwriting discounts and commissions and estimated offering expenses (Press release, Arvinas, SEP 26, 2018, View Source [SID1234531302]). In addition, Arvinas has granted the underwriters an option for a period of 30 days to purchase up to 1,125,000 additional shares of common stock at the public offering price, less underwriting discounts and commissions. All of the shares are being offered by Arvinas.

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The common stock is expected to begin trading on the Nasdaq Global Select Market on September 27, 2018 under the symbol "ARVN." The offering is expected to close on October 1, 2018, subject to customary closing conditions.

Goldman Sachs & Co. LLC, Citigroup and Piper Jaffray & Co., are acting as joint book-running managers for the offering.

A registration statement relating to the shares being sold in this offering was declared effective by the Securities and Exchange Commission on September 26, 2018. The offering is being made only by means of a prospectus forming part of the registration statement relating to these shares. Copies of the prospectus, when available, may be obtained by contacting: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by telephone at 800-831-9146; or Piper Jaffray & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at 800-747-3924 or by email at [email protected].

This press release shall not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On September 26, 2018 Zai Lab Limited (NASDAQ: ZLAB), a Shanghai-based innovative biopharmaceutical company, reported its interim results of its Phase 2 study of ZL-2301 (brivanib) in Chinese patients with previously-treated advanced hepatocellular carcinoma (HCC) (Press release, Zai Laboratory, SEP 26, 2018, View Source;p=RssLanding&cat=news&id=2368997 [SID1234530331]). Results from the study show ZL-2301 continues to demonstrate evidence of anti-tumor activity with a manageable safety profile in Chinese patients. The interim data were presented at the 21st Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) on Sept. 22, 2018, at the Xiamen International Conference and Exhibition Center (XICEC) in Xiamen, China.

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In the multi-center, open-label Phase 2 trial, 90 patients were enrolled to receive either 800 mg of ZL-2301 once-daily (QD) or 400 mg of ZL-2301 twice-daily (BID), administered orally. The primary endpoints in the trial are: (1) disease control rate (DCR) at 12 weeks from randomization, defined as the percentage of patients with a complete response, partial response or stable disease, and (2) time to disease progression (TTP).

The interim analysis was based on 75 patients (36 on 800mg QD; 39 on 400mg BID). The 12 weeks DCR was 41.7% in the 800mg QD arm and 35.9% in the 400mg BID arm. TTP was 4.2 months (95% CI, 2.8-4.2) in 800mg QD arm and 2.8 months (95% CI, 1.4-4.2) in the 400mg BID arm. ZL-2301 was relatively well-tolerated in the study; its toxicity profile appears in-line with that of the VEGFR inhibitor drug class.

Dr. Shukui Qin, Executive Member of the Asian Clinical Oncology Society, Senior Vice President of Chinese Society of Clinical Oncology and Lead Investigator for ZL-2301 study, commented, "This is a well conducted clinical trial that produced additional data to better characterize the clinical profile of ZL-2301 in Chinese patients. The data helped to set the foundation for further development of ZL-2301 as a potential therapy for HCC which are still in dire need of additional treatment options."

The efficacy and tolerability results of the study are in-line with our expectations in this heavily pre-treated HCC population. Given the large, unmet needs in HCC, a common, difficult to treat cancer, primarily driven by hepatitis B infection in China, and the rapidly changing landscape in HCC, Zai Lab plans to conduct a combination study to evaluate ZL-2301 with anti-PD1 therapy for HCC patients in China.

About ZL-2301 (brivanib)

ZL-2301 (brivanib) is an oral, small molecule dual target tyrosine kinase inhibitor, or TKI. Zai Lab licensed exclusive rights for China, Hong Kong, Macau and Taiwan from Bristol Myers Squibb. ZL-2301 has been tested in four Phase III studies in hepatocellular cancer (HCC) and showed anti-tumor activity and a manageable safety profile. Based on Zai Lab’s review of the results from Bristol-Myers Squibb’s development program for ZL-2301, its understanding of the etiology and current standard of care of HCC in Chinese patients and its ongoing research, Zai Lab believes that ZL-2301 has the potential to be an effective treatment option for Chinese HCC patients and merits further clinical development. In the second quarter of 2017 Zai Lab initiated a Phase II trial of ZL-2301 as a second-line treatment for advanced HCC patients in China.

On September 26, 2018 ai Lab Limited (NASDAQ:ZLAB), a Shanghai-based innovative biopharmaceutical company, presented results of its open-label study to evaluate the pharmacokinetic (PK) profile of ZL-2306 (niraparib) made in China in Chinese ovarian cancer patients (Press release, Zai Laboratory, SEP 26, 2018, View Source;p=RssLanding&cat=news&id=2368996 [SID1234530330]). Results from the study show comparable PK profile of the Chinese patients administered ZL-2306 to that of patients evaluated in Tesaro’s global PK study. These data were presented at the 21st Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) on Sept. 21, 2018, at the Xiamen International Conference and Exhibition Center (XICEC) in Xiamen, China.

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The open-label PK Study enrolled 36 Chinese patients with stage III or IV epithelial ovarian, fallopian tube or primary peritoneal cancer. Patients had received no more than two lines of platinum-based therapy and were responsive to the most recent platinum-based treatment. Subjects were randomly assigned to dose levels of 100mg, 200mg and 300mg. The primary objective of the study was to assess the PK profile of ZL-2306 in Chinese patients following both single and multiple doses. The secondary objective was a safety assessment.

The study demonstrated that the drug exposure increased proportionally from 100mg to 300mg, with a Tmax of approximately three hours. Systemic exposure of ZL-2306, as measured by Cmax and AUC, increased approximately proportionally with increased dose. The half-life is 31-37 hrs. There were no unexpected safety issues noted during the trial. All key PK and safety parameters were comparable to those in global studies. The study results and population PK data did not identify ethnicity differences between Chinese and non-Chinese patients.

The positive outcome of this PK study will further enhance ZL-2306 application package in China.

About ZL-2306

ZL-2306 (niraparib) is a highly potent and selective oral, once-daily small molecule poly (ADP-ribose) PARP 1/2 inhibitor. Niraparib was approved in March 2017 by the FDA in the U.S. and by the EMA in the EU under the trade name ZEJULA in November 2017 as a maintenance treatment for women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Based on the approval status in the U.S. and EU, Zai Lab submitted a market registration application for niraparib in Hong Kong and plans to launch and commercialize niraparib in Hong Kong in the second half of 2018. Zai Lab believes ZL-2306 has the potential to be a first-in-class Category 1 drug for treatment across multiple solid tumor types in China.