On September 20, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported its clinical data on tislelizumab, an investigational anti-PD-1 antibody, in Chinese patients with lung cancers, in two oral presentations at the 21st Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) in Xiamen, China (Press release, BeiGene, SEP 20, 2018, View Source;p=RssLanding&cat=news&id=2368376 [SID1234529526]).

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"Advanced lung cancer is one of our focus areas for development of tislelizumab, where we hope to have an impact on the way patients are treated both in China and worldwide. This complex and difficult-to-treat disease has proven to be susceptible to treatment with immunotherapies," commented Amy Peterson, M.D., Chief Medical Officer, Immuno-Oncology, at BeiGene. "The preliminary data presented today demonstrate that tislelizumab is generally well tolerated and has antitumor activity both as monotherapy and in combination with several chemotherapy regimens used in small cell and non-small cell lung cancer patients. We are hopeful that further study of tislelizumab may lead to a new treatment option for a broad array of patients with lung cancers."

Summary of Preliminary Results of Phase 2 Trial in China of Tislelizumab Combined with Chemotherapy as First-Line Treatment in Advanced Lung Cancer Setting

The multi-center, open-label Phase 2 trial in China (CTR20170361) of tislelizumab in combination with chemotherapy enrolled 54 patients with previously untreated locally advanced or metastatic lung cancer. All patients received tislelizumab at 200 mg every three weeks, plus platinum doublet until disease progression. Patients with non-squamous non-small cell lung cancer (NSCLC) (n=16) received pemetrexed plus platinum; patients with squamous NSCLC received either paclitaxel plus platinum (cohort A, n=15) or gemcitabine plus platinum (cohort B, n=6); and patients with small cell lung cancer (SCLC) received etoposide plus platinum (n=17).

As of the June 5, 2018 data cutoff, 35 patients remain on treatment. Treatment discontinuation due to adverse events (AEs) occurred in three patients. Fifty-one patients had at least one post-baseline tumor assessment and were evaluable for efficacy. Objective responses (including confirmed and unconfirmed partial responses) were observed in 56 percent (31 percent confirmed; all patients with an unconfirmed partial response remained on treatment) of 16 evaluable patients with non-squamous NSCLC; 80 percent (all confirmed) in 15 evaluable patients with squamous NSCLC, cohort A; 67 percent (all confirmed) in six patients with squamous NSCLC, cohort B; and 82 percent (47 percent confirmed; all patients with an unconfirmed partial response remained on treatment) in 17 evaluable patients with SCLC. Data continue to mature with follow-up.

AEs were considered manageable and reversible, with chemotherapy dose modifications or tislelizumab dose holds, except for one fatal event of myocarditis/myositis. Five patients (9.3%) experienced at least one grade ≥3 AE (polymyositis, dyspnea, rhabdomyolysis, myocarditis/myositis, and myasthenia gravis) that were considered to be possibly related to tislelizumab. Immune-related AEs (irAEs) occurred in 13 patients (24%) and included hypothyroidism (n=3), decreased tri-iodothyronine (n=2), hyperthyroidism (n=2), pneumonitis (n=2), pyrexia (n=2), and rash (n=2).

"We are excited by the preliminary data of tislelizumab combined with chemotherapy in patients with advanced lung cancer. The safety and tolerability appear consistent with previous data, and high response rates of up to 80 percent in a squamous NSCLC cohort along with low discontinuation rates support continued investigation of tislelizumab in patients with advance lung cancer. We are hopeful that this combination therapy will offer improved outcomes in this advanced disease setting," said Professor Jie Wang, M.D., from the National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College in Beijing, China, and lead author of the presentation.

Summary of Preliminary Results of Tislelizumab in Chinese Patients with NSCLC from Phase 1/2 Trial

The multi-center, open-label Phase 1/2 trial in China (CTR20160872) of tislelizumab enrolled 46 patients with NSCLC in the Phase 2 indication expansion portion of the trial, with 21 patients having expression of PD-L1 in 10 percent or more of their tumor cells (PD-L1+); the remaining 25 patients were considered PD-L1 negative (PD-L1-).

As of the May 11, 2018 data cutoff, 15 patients (33%) remained on treatment with the median treatment duration of 4.1 months (0.2–11.3 months) and a median follow-up of 8.4 months (0.2–11.8 months). Treatment discontinuation due to adverse events (AEs) occurred only in one patient. The median duration of treatment was 3.5 months (0.2-11.2 months) and 4.4 months (0.7–11.3 months) in PD-L1+ and PD-L1- cohorts, respectively. A total of 42 patients had at least 1 post-baseline tumor assessment and were evaluable for antitumor activity. Confirmed partial responses were observed in 17 percent of the evaluable patients, including 12 percent and 20 percent in PD-L1+ and PD-L1- patients, respectively.

Across the two arms, the most common treatment-related AEs (TRAEs) (occurring in ≥ 10% of patients) were increased transaminases (26%), rash (11%) and hypothyroidism (11%). A total of 14 patients had serious AEs and three of these patients experienced serious TRAEs, including nausea and vomiting (n=1), increased aspartate aminotransferase (AST) (n=1) and hyperglycemia (n=1). Three patients experienced a serious AE with a fatal outcome (multiple organ dysfunction syndrome [n=1], central nervous system metastases [n=1], hypotension [n=1]); none were determined to be related to treatment. Immune-related AEs occurred in 26 patients (57%) and many were overlapping with the TRAE cases.

"The prognosis for patients with late stage non-small cell lung cancer remains particularly poor. We are pleased that this trial demonstrated that treatment with tislelizumab was generally well tolerated. We are excited to see that Phase 3 trials evaluating tislelizumab, either as monotherapy or in combination with chemotherapy, in patients with advanced NSCLC are underway and look forward to the results," said Yi-Long Wu, M.D., President of Chinese Thoracic Oncology Group (CTONG) and lead author of the presentation.

Trial data with the same cut off time will be presented at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer (WCLC), which takes place September 23-26 in Toronto.

About Tislelizumab
Tislelizumab (BGB-A317) is an investigational humanized monoclonal antibody that belongs to a class of immuno-oncology agents known as immune checkpoint inhibitors. Discovered by BeiGene scientists in Beijing, tislelizumab is designed to bind to PD-1, a cell surface receptor that plays an important role in downregulating the immune system by preventing the activation of T-cells. Tislelizumab has demonstrated high affinity and specificity for PD-1. It is potentially differentiated from the currently approved PD-1 antibodies in an engineered Fc region, which is believed to minimize potentially negative interactions with other immune cells, based on preclinical data. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene and Celgene Corporation have a global strategic collaboration for the development of tislelizumab in solid tumor cancers outside of Asia (except Japan).

On September 20, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported the appointment of industry veteran Scott Garland as president and chief executive officer, effective October 8, 2018 (Press release, Portola Pharmaceuticals, SEP 20, 2018, View Source;p=irol-newsroomArticle&ID=2368241 [SID1234529523]). Mr. Garland brings to his new role more than two decades of broad executive leadership experience, including a strong track record driving multiple billion dollar product launches. He will also serve on the Company’s Board of Directors.

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Mr. Garland joins Portola from Relypsa, Inc., where he was president and previously chief commercial officer. During his tenure, Mr. Garland was responsible for the integration and growth of the U.S. operations after Relypsa was acquired by Vifor Pharma Group in 2016.

Prior to Relypsa, Mr. Garland was executive vice president and chief commercial officer of Exelixis, Inc., where he led global commercial operations and directed sales, marketing and market access in the United States. Previously, Mr. Garland spent close to a decade at Genentech leading full scale commercial franchises for two multi-billion dollar therapies – Avastin (bevacizumab) and Rituxan (rituximab). He started his career at Merck as a sales representative and then went on to serve at Amgen in various sales and marketing roles, including market development, to support the launch of Aranesp (darbepoetin alfa).

Hollings C. Renton, Chairman of the Board, said: "Scott is an exceptional choice to lead Portola through this exciting period of commercial evolution. His leadership and operational experience in hematology, oncology and hospital-based products aligns with our portfolio, and I am confident he will help realize the full potential of the Company’s life-changing medicines, drive long-term growth to maximize shareholder value and develop a best-in-class culture that will allow us to continue to attract and retain top industry talent. We look forward to introducing Scott during our third quarter conference call in early November."

Mr. Garland said: "This is a rare opportunity to join a company with two FDA-approved medicines, a promising compound in development and a financial position to support long-term growth. I am excited to partner with such a committed Board and leadership team to build on the Company’s current pace of innovation and focus on execution to deliver potentially life-saving therapies to patients."

Mr. Garland holds an M.B.A. from the Fuqua School of Business at Duke University and a Bachelor of Science degree in biological sciences from California Polytechnic State University, San Luis Obispo. He has been a member of the Board of Directors of Karyopharm Therapeutics since 2014.

On September 20, 2018 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq:YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported the pricing of its initial public offering of 6,000,000 shares of common stock at a public offering price of $16.00 per share, for gross proceeds of $96 million, before underwriting discounts and commissions and estimated offering expenses payable by the Company (Press release, Y-mAbs Therapeutics, SEP 20, 2018, View Source [SID1234529522]). All shares are being offered by the Company.

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In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 900,000 shares of common stock from the Company at the initial public offering price, less underwriting discounts and commissions.

The Company’s common stock is expected to begin trading on the Nasdaq Global Select Market under the ticker symbol "YMAB" on Friday, September 21, 2018. The offering is expected to close on Tuesday, September 25, 2018, subject to customary closing conditions.

BofA Merrill Lynch and Cowen are acting as joint book-running managers for the offering. Canaccord Genuity is acting as lead manager for the offering and BTIG is acting as co-manager for the offering.

A registration statement relating to the securities being sold in the offering was declared effective by the Securities and Exchange Commission (the "SEC") on September 20, 2018. The offering is being made only by means of a prospectus. Copies of the final prospectus relating to this offering may be obtained, when available, by contacting BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, NC 28255-0001, Attn: Prospectus Department or by email at [email protected]; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, or by phone at (631) 274-2806.

Additionally, when available, copies of these documents may be obtained for free by accessing the EDGAR database on the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or the 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 the registration or qualification under the securities laws of any such state or jurisdiction.

On September 20, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that it has entered into a clinical trial collaboration agreement with a subsidiary of Merck & Co., Inc., Kenilworth, New Jersey, U.S.A., known as MSD outside the United States and Canada, to evaluate the combination of Daiichi Sankyo’s investigational HER2 targeting antibody drug conjugate DS-8201 and KEYTRUDA (pembrolizumab) in HER2 expressing advanced/metastatic breast and HER2 expressing or HER2 mutant non-small cell lung cancers (NSCLC) (Press release, Daiichi Sankyo, SEP 20, 2018, View Source [SID1234529511]).

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"We are excited to pursue this opportunity to evaluate the safety, tolerability and activity of DS-8201 in combination with KEYTRUDA and whether this combination may provide a potential new treatment approach for patients with HER2 expressing advanced breast and non-small cell lung cancer," said Tom Held, Vice President, Head, Antibody Drug Conjugate Task Force, Oncology Research and Development, Daiichi Sankyo. "Strategic collaborations like this support our goal to pursue, investigate and maximize the application of DS-8201 in combination with other compounds that target different pathways to address unmet needs of patients with cancer."

About the Study

Under the terms of the agreement, Daiichi Sankyo will conduct a two-part phase 1b multicenter, open-label study to:

・ Determine the safety, tolerability and dose of DS-8201 in combination with KEYTRUDA and evaluate efficacy of the combination in patients with HER2 expressing advanced/metastatic breast cancer and patients with HER2 expressing or HER2 mutant advanced/metastatic NSCLC.

・ Enroll patients into one of four cohorts: patients with HER2 positive advanced breast cancer who have been previously treated with ado-trastuzumab emtansine (T-DM1) (cohort 1); patients with HER2 low expressing advanced breast cancer (IHC 1+ or IHC 2+/ISH-) who have received available standard of care (cohort 2); patients with HER2 expressing advanced NSCLC (IHC 1+, 2+, or 3+) who have not received prior treatment with anti-PD-1 or anti-PD-L1 agents (cohort 3); and patients with HER2 mutant advanced NSCLC who have not received prior treatment with anti-PD-1 or anti-PD-L1 agents (cohort 4).

The primary endpoints of the study are maximum tolerated dose/recommended expansion dose and overall response rate. Secondary endpoints include duration of response, disease control rate, progression-free survival, overall survival, time to response and safety. The study is expected to enroll approximately 125 patients in the U.S. and Europe. Additional details of the agreement were not disclosed.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About DS-8201

DS-8201 is the lead product in the investigational ADC Franchise of the Daiichi Sankyo Cancer Enterprise. ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Designed using Daiichi Sankyo’s proprietary ADC technology, DS-8201 is comprised of a humanized HER2 antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells and reduce systemic exposure to the cytotoxic payload (or chemotherapy) compared to the way chemotherapy is commonly delivered.

DS-8201 is currently in pivotal phase 2 clinical development for HER2 positive unresectable and/or metastatic breast cancer resistant or refractory to T-DM1 (DESTINY-Breast01) in North America, Europe and Asia; pivotal phase 2 development for HER2 positive advanced gastric cancer resistant or refractory to trastuzumab (DESTINY-Gastric01) in Japan and South Korea; phase 2 development for HER2 expressing advanced colorectal cancer in North America, Europe and Japan; phase 2 development for unresectable and/or metastatic non-squamous HER2 overexpressing or HER2 mutated non-small cell lung cancer (NSCLC) in North America, Europe and Japan; and phase 1 development for other HER2 expressing advanced/unresectable or metastatic solid tumors in the U.S. and Japan.

DS-8201 has been granted Breakthrough Therapy designation for the treatment of patients with HER2 positive, locally advanced or metastatic breast cancer who have been treated with trastuzumab and pertuzumab and have disease progression after ado-trastuzumab emtansine (T-DM1), and Fast Track designation for the treatment of HER2 positive unresectable and/or metastatic breast cancer in patients who have progressed after prior treatment with HER2 targeted therapies including T-DM1 by the U.S. Food and Drug Administration. DS-8201 has also been granted SAKIGAKE Designation by the Japan Ministry of Health, Labour and Welfare for the treatment of HER2 positive advanced gastric or gastroesophageal junction cancer.

DS-8201 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

On September 20, 2018 ERYTECH Pharma (Euronext Paris: ERYP – Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating drug substances inside red blood cells, reported that the first three patients have been enrolled in its pivotal Phase 3 clinical trial, named ‘TRYbeCA1’, evaluating its lead product candidate eryaspase for the treatment of second line metastatic pancreatic cancer (Press release, ERYtech Pharma, SEP 20, 2018, View Source;p=RssLanding&cat=news&id=2368155 [SID1234529509]).

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The TRYbeCa1 (NCT03665441) trial will enroll approximately 500 patients with second line metastatic pancreatic cancer in 120-130 clinical sites in Europe and the US. Patients who meet the eligibility criteria are randomized 1-to-1 to receive eryaspase in combination with standard chemotherapy (gemcitabine/abraxane or irinotecan -based regimen) or chemotherapy alone until disease progression. The primary endpoint is overall survival. An interim analysis is foreseen when approximately two-thirds of events have occurred.

The launch of the TRYbeCA1 Phase 3 trial follows the positive Phase 2b results in the same patient population, that were reported in September 2017. This open-label, multi-center, 2-to-1 randomized study in 141 patients demonstrated significant improvement in both overall survival and progression-free survival. Overall, eryaspase was well tolerated and showed a safety profile comparable to that of standard chemotherapy.

"The results from our landmark Phase 2b study are highly promising and underscore the importance of targeting tumor metabolism pathways in pancreatic cancer. We are hopeful to provide a novel treatment modality for this highly unmet medical need. We are very pleased that eryaspase has now moved into Phase 3 and patient enrollment has started as planned. Our first three enrolled patients mark the initiation of the trial in Europe. Early next year, we expect sites in the United States will begin enrolling as well," commented Iman El-Hariry, Chief Medical Officer.

About pancreatic cancer

Pancreatic cancer is a disease in which malignant (cancer) cells are found in the tissues of the pancreas. Every year, there are approximately 150,000 new cases of pancreatic cancer diagnosed in Europe and the United States. Advanced pancreatic cancer is a particularly aggressive cancer, with a five-year survival rate of less than 10%. It is currently the fourth leading cause of cancer death in Europe and the United States and is projected to rise to the second leading cause by 2030. Limited therapeutic options are currently available for this indication, thereby reinforcing the need to develop new therapeutic strategies and rational drug combinations with the aim of improving overall patient outcomes and quality of life.