On September 21, 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 preliminary results from the Phase 1 trial of its investigational BTK inhibitor zanubrutinib in Chinese patients with B-cell lymphoma in an oral presentation at the 21st Annual Meeting of the Chinese Society of Clinical Oncology (CSCO) in Xiamen, China (Press release, BeiGene, SEP 21, 2018, View Source;p=irol-newsArticle&ID=2368471 [SID1234529513]).

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"We continue to be encouraged by clinical data on zanubrutinib, including these results, which we believe support its broad global clinical development. Our recent new drug application filing for zanubrutinib in China for patients with relapsed/refractory mantle cell lymphoma (MCL), a type of B-cell lymphoma, is currently under review by the National Medical Products Administration of China, and we are hopeful that it will give patients in China, and across the world, a new treatment option where it is so greatly needed," said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene.

Summary of Preliminary Results from the Phase 1 Trial of Zanubrutinib in Chinese Patients with B-Cell Lymphoma

An ongoing Phase 1 trial of zanubrutinib as a monotherapy in patients with different subtypes of B-cell malignancies, including Waldenström macroglobulinemia (WM), chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and other non-Hodgkin’s lymphomas (NHL), is being conducted in China. The trial is fully enrolled and comprised of two parts – a dose escalation phase involving 21 patients and a dose-expansion phase of 23 patients treated with zanubrutinib at the recommended Phase 2 dose of 160 mg taken orally twice daily.

Preliminary findings suggested that there was no significant difference in the pharmacokinetic profile of zanubrutinib between Chinese and non-Chinese patients. Preliminary findings also showed complete or greater than 80 percent sustained BTK occupancy was achieved among these patients with both single- and multiple-dose administrations.

As of June 15, 2018, after a median follow-up of 9.5 months (2.3 months–23.4 months), 21 patients (47%) remained on treatment. With 44 patients enrolled in the trial, 34 were evaluable for response. Of the nine patients with CLL/SLL, the overall response rate (ORR) was 100 percent, with two complete responses (CRs), six partial responses (PRs), and a PR with lymphocytosis (PR-L). Of the two patients with mantle cell lymphoma (MCL), there was one CR and one stable disease (SD). Of the two patients with WM, there was one PR and one SD. Of the 26 patients with follicular lymphoma (FL), the ORR was 42 percent with two CRs and nine PRs. There were three patients with FL who were not evaluable at the time of the data cutoff. Of the five patients with marginal zone lymphoma (MZL), there were three SDs and two patients who were not evaluable.

At the time of data cutoff, no dose-limiting toxicities occurred during dose escalation portion of the trial and there were no unexpected safety signals identified in the trial. No deaths related to adverse events were observed in the trial. The most common adverse events (occurring in ≥ 20% of patients) of any attribution among all 44 patients were neutrophil count decreased (50%), anemia (32%), upper respiratory tract infection (25%), white blood cell count decreased (25%), platelet count decreased (23%), rash (23%), hematuria (20%), and hyperuricemia (20%).

"These preliminary safety, tolerability and pharmacokinetics data of zanubrutinib support its ongoing clinical study. In this study, the preliminary results suggest zanubrutinib has a high rate of activity and is generally well-tolerated, which we believe is based on its potency and high-degree of selectivity," said Jun Zhu, M.D., Medical Department Chief at the Beijing Cancer Hospital and study presenter.

About B-Cell Lymphomas
Lymphoma is a diverse group of malignancies that originates from B, T or NK cells. The most common type of B-cell lymphomas are non-Hodgkin’s lymphoma, of which diffuse large B-cell lymphoma (DLBCL) is the most common. Other types of B-cell non-Hodgkin’s lymphoma include FL, CLL/SLL, MCL, MZL, and WM.

About Zanubrutinib
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK) that is currently being evaluated in a broad pivotal clinical program globally and in China as a monotherapy and in combination with other therapies to treat various B-cell malignancies.

On September 20, 2018 Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, and Providence Cancer Institute, reported additional preliminary clinical data from cohort 3 of an investigator-initiated Phase 1b clinical trial of GR-MD-02 used in combination with KEYTRUDA (pembrolizumab) in patients with metastatic melanoma for which KEYTRUDA is indicated or those patients whose melanoma progressed during or recently after KEYTRUDA monotherapy (Press release, Galectin Therapeutics, SEP 20, 2018, View Source [SID1234530280]).

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The Providence Cancer Institute (Portland, OR) translational medicine team is conducting this phase 1b clinical trial, initiated under direction of principal investigator Brendan D. Curti, M.D., Director, Providence Melanoma Program. The objectives of this study were to determine a safe dose of GR-MD-02 used in combination with KEYTRUDA and to measure the response rate to combined therapy. "We are very encouraged by the objective response rate and the disease control rate observed in patients with advanced melanoma. These response rates were higher than expected with KEYTRUDA alone," said Dr. Curti. "An objective response rate of seven out of fourteen patients (50%) and a disease control rate of nine out of fourteen patients (64%) with advanced melanoma is very encouraging. The published objective response rates in randomized studies using KEYTRUDA in patients with advanced melanoma range from 21% in patients who have had prior therapy to 39% in patients who had not received prior systemic therapy. Importantly, the combination was also very well tolerated, and treatment appears to be associated with fewer adverse events than expected with KEYTRUDA alone."

When aggregated with the cohorts previously reported, the data shows a 50% objective response rate in advanced melanoma with GR-MD-02 in combination with KEYTRUDA and a significant decrease in the frequency of suppressive myeloid-derived suppressor cells (MDSC) following treatment in the responding patients (on day 85 post-treatment) was observed. The published data on KEYTRUDA alone have shown an objective response rate of 33% in this patient population.

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Fourteen advanced melanoma patients across three dose cohorts now have Objective Response Rate (ORR) and Disease Control Rate (DCR) data. Six patients in cohort 3 (8 mg/kg GR-MD-02) have now been added to the three patients in cohort 2 (4 mg/kg GR-MD-02) and the five patients in cohort 1 (2 mg/kg GR-MD-02). Cohorts 1 and 3 each had two patients with an objective response. All three patients in cohort 2 had an objective response.
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N=13; 1 patient in cohort 3 not depicted due to clinical progression prior to scans. Dotted line at -30% change from baseline indicates the RECIST 1.1 threshold for definition of partial response.

Generally, the U.S. Food and Drug Administration has defined objective response rate as the sum of partial responses plus complete responses. Disease control rate is the objective responses plus those with stable disease.

In addition to the fourteen advanced melanoma patients, six patients with head and neck cancer were enrolled in this phase 1b trial with a 33% objective response rate and a 67% disease control rate. Dr. Curti states "the response rates observed overall in advanced melanoma and head and neck cancer patients were better than expected with KEYTRUDA alone and are the basis for moving forward with both tumor types, particularly given the low response rates of anti-PD-1 monotherapy in head and neck cancer. There is a significant clinical need for better options for these patients and our initial objective response rates were encouraging enough to warrant inclusion of additional patients to help determine whether we should also pursue these challenging patient populations in a phase 2 trial. Taken together with the observed favorable safety and tolerability of the combination, these results provide a compelling rationale to move forward with this approach." Given that all three melanoma patients (100%) were responders at 4 mg/kg dose, the investigators plan to continue the trial with expansion of the 4 mg/kg GR-MD-02 and KEYTRUDA cohort to include additional advanced melanoma patients and additional head and neck cancer patients.

"In addition to the encouraging clinical responses seen thus far, we continue to make progress on identifying immunological biomarkers that correlate with favorable responses," said William L. Redmond, Ph.D., Associate Member, Laboratory of Cancer Immunotherapy, and Director, Immune Monitoring Laboratory at the Earle A. Chiles Research Institute, a division of Providence Cancer Institute. "We have observed a significant decrease in the frequency of suppressive myeloid-derived suppressor cells (MDSC) following treatment in the responding patients (on day 85 post-treatment). Comprehensive laboratory studies are being performed to further identify the biological mechanisms associated with this response."

"Galectin Therapeutics is very pleased with our continuing collaboration with Providence Cancer Institute, and we are encouraged that Dr. Curti and his team are expanding the trial to include additional patients," said Harold Shlevin, Ph.D., CEO and President of Galectin Therapeutics. "The planned expansion of the size of the 4 mg/kg dose cohort, and inclusion of both advanced melanoma patients and patients with head and neck cancer, will permit further evaluation that the use of GR-MD-02 in combination with KEYTRUDA has a better objective response rate and fewer adverse events than KEYTRUDA alone. We believe this collaboration with Providence to be a fruitful approach to helping to determine the potential of GR-MD-02 in combination immuno-therapy, and it also leverages our ability to collect additional data related to the immunological monitoring of these patients before potentially proceeding to the next phase of development."

Additional information about this clinical trial may be found at www.clinicaltrials.gov/ct2/show/NCT02575404

About GR-MD-02

GR-MD-02 is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of fatty liver disease and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. The drug binds to galectin-3 proteins and disrupts its function. Preclinical data in animals have shown that GR-MD-02 has robust treatment effects in reversing liver fibrosis and cirrhosis. GR-MD-02 also has robust efficacy in pre-clinical cancer models in combination with immunotherapy agents.

On September 20, 2018 Cell Medica reported the treatment of the first patient world-wide to receive CMD-501, an autologous CAR-NKT therapy targeting pediatric neuroblastoma (Press release, Cell Medica, SEP 20, 2018, View Source [SID1234530199]). This is the first time an engineered NKT cell therapy has been used in humans. Cell Medica is a clinical-stage biopharmaceutical company that is transforming the treatment of solid and hematological cancer by developing the next generation of CAR therapies.

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This open-label Phase 1 study, GINAKIT2, is being carried out in collaboration with both Baylor College of Medicine (BCM) and Texas Children’s Hospital.

Dr. Andras Heczey, Principal Investigator, Assistant Professor, Pediatrics-Oncology at Baylor College of Medicine and Physician-Scientist, Texas Children’s Cancer Center commented: "Dosing the first patient with this novel CAR-NKT therapy is an important milestone for all pediatric patients with neuroblastoma. CAR-NKTs may offer an exciting new therapeutic option for these patients and potentially for others with solid and hematological cancers. I am extremely grateful to the patients and families participating in this ground-breaking study."

Chris Nowers, Cell Medica’s CEO, said: "We believe that our CAR-NKT platform has a unique profile, with a potential to target solid and hematological tumors, as well as the possibility of a subsequent allogeneic "off the shelf" CAR-NKT therapy that could address some challenges of current autologous CAR-T therapies. This study marks an important step forward for Cell Medica and we are proud to be leading the development of this innovative class of next generation CAR therapies with our colleagues at BCM and Texas Children’s."

Innovative CAR-NKT Platform
CMD-501 is based on Cell Medica’s novel CAR-NKT platform, a next-generation technology of engineered immune cells with enhanced functions for the treatment of hematological and solid tumors, utilizing the unique properties of NKT cells, a specialized type of innate lymphocytes, sharing properties of T and NK cells. CMD-501 is the initial study from Cell Medica’s CAR-NKT pipeline and utilizes an autologous approach. The patient’s own NKT cells are genetically engineered with a CAR targeting GD2, a molecule expressed on the surface of nearly all neuroblastoma cells. In collaboration with its partners at BCM and Texas Children’s, Cell Medica designed this CAR-NKT cell therapy to also secrete the cytokine IL-15, which has been shown in pre-clinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the immunosuppressive tumor microenvironment.

Dr. Leonid Metelitsa, Professor of Pediatrics, Hematology-Oncology, Baylor College of Medicine and Co-Director, Neuroblastoma Program, Texas Children’s Cancer Center added: "It has been a great pleasure leading the multi-disciplinary team in the development of this versatile CAR-NKT platform. NKT cells effectively traffic to the tumor site, so expressing tumor-specific CARs in these cells ensures delivery to the site of disease for maximum efficacy. We’re now exploiting another natural feature of NKT cells, their lack of allo-reactivity and we are developing allogeneic, "off the shelf", therapies that will further harness the unique advantages of NKT cells."

First-in-Human Study
GINAKIT2 is a first-in-human, dose escalation evaluation of CMD-501 in children with relapsed or refractory (R/R) high-risk neuroblastoma, (NCT03294954). Neuroblastomas occur primarily in children and account for 7-10 percent of all pediatric cancers. Ninety percent of patients are younger than 5 years at diagnosis. R/R high risk neuroblastoma is one of the deadliest types of childhood cancer and the current median survival is around 1-3 years. Almost all neuroblastomas express GD2, which is targeted by CMD-501. This study is supported by a grant from Alex’s Lemonade Stand Foundation (ALSF), awarded to BCM investigators, Drs. Heczey and Metelitsa.

About Neuroblastoma and GD2
Neuroblastoma is a cancer of the sympathetic nervous system which can occur in the chest, neck, abdomen and adrenal glands, and can metastasize to the bone marrow and other organs. Children with low or intermediate risk neuroblastoma can be cured through surgical intervention and/or chemotherapy, however, at least half of all children with neuroblastoma have high risk disease, which often requires combined surgical, radio-, immuno-, and chemotherapy, in addition to autologous stem cell transplantation. Patients with relapsed/refractory high-risk neuroblastoma have one of the deadliest types of childhood cancer and a poor prognosis, with median survival of 1-3 years.

GD2 is a molecule expressed on tumors of neuroectodermal origin, including almost all neuroblastomas, and a substantial fraction of small cell lung cancer and melanoma, with restricted expression on normal tissues, making it a good target for CAR-NKT cell therapy.

About CMD-501
CMD-501 is an innovative autologous product in which NKT cells are genetically engineered with a CAR targeting GD2. NKT cells are a subset of T lymphocyte with the cytotoxic and anti-tumor properties of conventional T cells, but with other biological attributes that are expected to improve their ability to attack tumors. GD2 is a molecule expressed on the surface of most neuroblastoma cells.

In collaboration with its partners at BCM, Cell Medica has engineered a GD2-specific CAR construct that is additionally designed to secrete the cytokine IL-15, which has been shown in pre-clinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the immunosuppressive tumor microenvironment. CMD-501 is an autologous product, meaning that each patient’s own cells are collected, modified and activated outside the body, and then infused back into the same patient. However, NKT cells also have significant potential for so-called off-the-shelf use, where cells from a healthy donor could be prepared in large quantities in advance and used to treat many different patients. Cell Medica is collaborating with BCM to bring an off-the-shelf CAR-NKT cell product into the clinic in the near future.

On September 20, 2018 Supernus Pharmaceuticals, Inc. (NASDAQ: SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases, reported that the Company’s management will present an overview and Company update as well as host investor meetings, at the 2018 Cantor Fitzgerald Global Healthcare Conference (Press release, Supernus, SEP 20, 2018, View Source [SID1234529925]).

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Date: Wednesday, October 3, 2018

Time: 8:35 a.m. ET
Place: InterContinental New York Barclays Hotel, New York City

Investors interested in arranging a meeting with the Company’s management during this conference should contact the conference coordinator.

A live webcast of the presentation can be accessed by visiting ‘Events & Presentations’ in the Investors Section on the Company’s website at www.supernus.com. An archived replay of the webcast will be available for 60 days on the Company’s website after the conference.

On September 20, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported the pricing of an underwritten public offering of 9,259,260 shares of its common stock at a public offering price of $13.50 per share, before underwriting discounts, for an aggregate offering of approximately $125.0 million (Press release, Fate Therapeutics, SEP 20, 2018, View Source [SID1234529896]). Fate Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 1,388,889 shares of its common stock. The proceeds to Fate Therapeutics from this offering are expected to be approximately $117.2 million after deducting underwriting discounts and commissions and other estimated offering expenses but excluding any exercise of the underwriters’ option. Fate Therapeutics intends to use the net proceeds from the offering to fund clinical trials and nonclinical studies, the manufacture of clinical product candidates and the conduct of preclinical research and development, and for general corporate purposes. All shares of common stock to be sold in the offering are being offered by Fate Therapeutics. The offering is expected to close on or about September 25, 2018, subject to customary closing conditions.

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Jefferies, Piper Jaffray, and Wells Fargo Securities are acting as joint book-running managers for the offering. Wedbush PacGrow is acting as a co-manager for the offering.

The securities described above are being offered by Fate Therapeutics pursuant to a shelf registration statement on Form S-3 (File No. 333-224680) previously filed with and declared effective by the Securities and Exchange Commission (the "SEC"). The securities may be offered only by means of a prospectus. A preliminary prospectus supplement related to the offering was filed with the SEC on September 20, 2018 and is available on the SEC’s website at View Source and a final prospectus supplement related to the offering will be filed with the SEC and will be available on the SEC’s website at View Source. Copies of the final prospectus supplement and the accompanying prospectus for the securities being offered may also be obtained, when available, by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 821-7388; Piper Jaffray & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by e-mail at [email protected] or by telephone at (800) 747-3924; or Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 375 Park Avenue, New York, New York 10152, by email at [email protected] or by telephone at (800) 326-5897.

This press release does not constitute an offer to sell or the solicitation of an offer to buy, 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.