On October 10, 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 that China’s State Medical Insurance Administration (SMIA) has included VIDAZA (azacitidine for injection) on its national reimbursement drug list (NRDL) (Press release, BeiGene, OCT 10, 2018, View Source;p=RssLanding&cat=news&id=2370979 [SID1234530257]). VIDAZA is a nucleoside metabolic inhibitor and was approved in China for patients with intermediate-2 / high-risk myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) with 20-30% bone marrow blasts and chronic myelomonocyte leukemia (CMML). It is marketed in China by BeiGene under an exclusive license from Celgene Corporation.

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"VIDAZA became commercially available in China in February and is the only approved hypomethylating agent shown to prolong survival for patients with MDS. With this national reimbursement coverage, we expect more patients to have an opportunity to benefit from VIDAZA in China," commented Dr. Xiaobin Wu, General Manager of China and President of BeiGene, Ltd. "BeiGene is committed to improving patient access to high-quality, innovative cancer treatments."

VIDAZA is recommended by National Comprehensive Cancer Network (NCCN) Guidelines in the US as a front-line treatment. In a global Phase 3 trial (AZA-001) involving intermediate-2 and high-risk MDS patients, VIDAZA significantly prolonged the median overall survival to 24.5 months compared with 15 months for the conventional care regimens (CCR- best supportive care, low-dose cytarabine or intensive chemotherapy) group. In the VIDAZA group, 45% of patients who were dependent on red blood cell transfusions at baseline became transfusion independent compared with 11% in the CCR group.There was a higher objective response rate among patients treated with VIDAZA (49%) as compared to the CCR arm (29%). VIDAZA also delayed the onset of AML for these patients (17.8 months vs.11.5 months). The most common grade 3-4 events were peripheral blood cytopenias for all treatments.

"We are pleased that patients in China will have improved access to VIDAZA and other cancer treatments listed on the 2018 NRDL," said Professor Wang Jianxiang, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences. "VIDAZA is an important treatment for MDS with demonstrated survival benefit. The broadening access to new treatments like VIDAZA could significantly improve the care of our patients."

About Myelodysplastic Syndrome, Acute Myeloid Leukemia and Chronic Myelomonocyte Leukemia

MDS is a group of conditions that can occur when the bloodforming cells in the bone marrow become abnormal. This leads to low numbers of one or more types of blood cells. In about one-third of patients with MDS, the disease can progress to a rapidly growing cancer of bone marrow cells called AML.i CMML is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood; it affects mainly older adults. CMML has features of both MDS and myeloproliferative disorder and is considered the most common disease among myelodysplastic/myeloproliferative diseases.ii

About VIDAZA (Azacitidine for Injection)

VIDAZA is a nucleoside metabolic inhibitor indicated in China for the treatment of patients with intermediate-2 / high-risk MDS, AML with 20-30% bone marrow blasts and CMML. It is marketed in China by BeiGene under an exclusive license from Celgene Corporation.

In the US, VIDAZA is indicated for the treatment of patients with the following FAB MDS subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and CMML.

Important Safety Information

VIDAZA is contraindicated in patients with a known hypersensitivity to azacitidine or mannitol and in patients with advanced malignant hepatic tumors.

In Study 1 (a randomized, open-label, controlled trial carried out in 53 U.S. sites compared the safety and efficacy of subcutaneous VIDAZA plus supportive care with supportive care alone ("observation") in patients with any of the five FAB subtypes of myelodysplastic syndromes (MDS)) and Study 2 (a multi-center, open-label, single-arm study of 72 patients with RAEB, RAEB-T, CMMoL, or AML), most frequently observed adverse reactions occurring in at least 5% of patients by SC route were nausea (70.5%), anemia (69.5%), thrombocytopenia (65.5%), vomiting (54.1%), pyrexia (51.8%), leukopenia (48.2%), diarrhea (36.4%), injection site erythema (35.0%), constipation (33.6%), neutropenia (32.3%), and ecchymosis (30.5%). Other adverse reactions included dizziness (18.6%), chest pain (16.4%), febrile neutropenia (16.4%), myalgia (15.9%), injection site reaction (13.6%), and malaise (10.9%). In Study 3, the most common adverse reactions by IV route also included petechiae (45.8%), weakness (35.4%), rigors (35.4%), and hypokalemia (31.3%).

In Study 4 (the AZA-001 survival trial, an international, multicenter, open-label, randomized trial in MDS patients with RAEB, RAEB-T or modified CMMoL according to FAB classification and Intermediate-2 and High risk according to IPSS classification), most frequently observed adverse reactions occurring in at least 5% of patients were thrombocytopenia (69.7%), neutropenia (65.7%), anemia (51.4%), constipation (50.3%), nausea (48.0%), injection site erythema (42.9%), and pyrexia (30.3%). The most frequently observed adverse reactions in the percentage with NCI CTC Grade 3/4 reactions were neutropenia (61.1%), thrombocytopenia (58.3%), leukopenia (14.9%), anemia (13.7%), and febrile neutropenia (12.6%).

Because treatment with VIDAZA is associated with anemia, neutropenia and thrombocytopenia, complete blood counts should be performed as needed to monitor response and/or toxicity, but at a minimum, prior to each dosing cycle.

Because azacitidine is potentially hepatotoxic in patients with severe preexisting hepatic impairment, caution is needed in patients with liver disease. In addition, azacitidine and its metabolites are substantially excreted by the kidneys and the risk of toxic reactions to this drug may be greater in patients with impaired renal function. Because elderly patients are more likely to have decreased renal function, it may be useful to monitor renal function.

VIDAZA may cause fetal harm when administered to a pregnant woman. Females of reproductive potential should be advised to avoid pregnancy during treatment with VIDAZA. Males with female sexual partners of reproductive potential should not father a child and should use effective contraception during treatment with VIDAZA.

The importance of the drug to the mother should be taken into consideration before the breastfeeding mothers decide to discontinue breastfeeding or the drug.

On October 10, 2018 Gotham Therapeutics, a biotechnology company developing a novel drug class targeting RNA-modifying proteins, reported with a $54 million Series A financing co-led by founding investor Versant Ventures, Forbion and S.R. One (Press release, Gotham Therapeutics, OCT 10, 2018, View Source [SID1234550889]). The syndicate also included Celgene Corporation and Alexandria Venture Investments. Gotham is part of New York’s rapidly growing biopharma community with a subsidiary at one of Europe’s leading life science clusters near Munich, Germany.

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Versant created and seeded Gotham based upon the seminal discoveries of co-founder Samie Jaffrey, M.D. Ph.D., a pioneer in an exciting new field within RNA metabolism called epitranscriptomics. Dr. Jaffrey is a professor of pharmacology at Weill Cornell Medicine and a member of the Scientific Advisory Board for Gotham Therapeutics. His work has shed light on the role of post-transcriptional mRNA modifications in health and disease. These modifications and their biological effects are driven by protein complexes commonly described and categorized as writers, erasers and readers of the epitranscriptomic code.

With its seed funding, Gotham built a platform to assess the impact of RNA-modifying proteins on disease biology and developed small molecules against priority targets. The Series A proceeds will allow Gotham to establish clinical proof of concept and to invest broadly in a pipeline of preclinical candidates that have potential to treat diseases intractable to classical approaches.

"As we pursue several important targets, the information we glean will help us further validate and build our platform for increasingly broad applications. Our goal is to become the leader in drugging key proteins that modulate mRNA functionality, thereby impacting disease onset and progression," said Lee Babiss, Ph.D., CEO of Gotham.

"While academic research and the pharmaceutical industry focused initially on modifications of DNA, a growing body of evidence indicates that mRNA modifications help determine to which degree genes are translated into proteins. RNA modifications and their associated protein complexes therefore represent an untapped frontier that could yield new therapeutic approaches," added Dr. Jaffrey.

Gotham has assembled an experienced founding team led by Dr. Babiss, former President of Pharma Research at Roche. Dr. Babiss is an early adopter of RNA drug discovery approaches who has a track record of translating discoveries into therapeutic candidates. He also has served as CSO of PPD, and as Head of Human Genetics and Personalized Healthcare at Glaxo Wellcome.

"After following the developments in the RNA drug discovery field for a number of years, we felt this was the right time to build a company that could capitalize on translating the scientific discoveries into a whole new class of drug candidates," said Carlo Rizzuto, Ph.D., Partner at Versant. "With our initial investment, the Gotham team constructed a platform able to validate critical links between specific types of RNA modifications and disease biology. We look forward to advancing a number of drug candidates with this new round of financing."

"We are excited to invest in Gotham, one of the pioneers in the fast-emerging field of RNA metabolism, which could create a paradigm shift in both cancer therapy and other major diseases," commented Holger Reithinger, Ph.D., General Partner at Forbion. "Gotham represents the first investment by our recently announced Forbion IV fund. Our new fund aims to help build leading companies around exciting new science, proven teams or in-licensed assets."

Dr. Reithinger will join Dr. Babiss, Dr. Rizzuto and Jill Carroll, Principal, SR One, on Gotham’s Board of Directors. Jorge DiMartino M.D., Ph.D., Vice President, Translational Development Oncology at Celgene and Head of Celgene’s Epigenetics Thematic Center of Excellence, has joined the board in an observer role.

On October 10, 2018 Allogene Therapeutics, Inc., a clinical-stage biotechnology company pioneering the development of allogeneic CAR T(AlloCAR T) therapies for cancer, reported the pricing of its initial public offering of 18,000,000 shares of its common stock at a price to the public of $18.00 per share (Press release, Allogene, OCT 10, 2018, View Source [SID1234529851]). The gross proceeds to Allogene Therapeutics from the offering, before deducting the underwriting discounts and commissions and offering expenses, are expected to be $324.0 million. The shares are expected to begin trading on the Nasdaq Global Select Market on October 11, 2018 under the symbol "ALLO." The offering is expected to close on October 15, 2018, subject to customary closing conditions. In addition, Allogene Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 2,700,000 shares of common stock.

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Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC, Cowen and Company, LLC and Jefferies LLC are acting as the joint book-running managers for the offering.

The offering will be made only by means of a prospectus. Copies of the final prospectus related to the offering, when available, may be obtained from:

Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, or via telephone: 1-866-471-2526, or via email: [email protected]; or
J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or via telephone: 1-866-803-9204; or
Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, or via email: [email protected]; or
Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or via telephone: 1-877-547-6340, or via email: [email protected].
Registration statements relating to these securities have been filed with the Securities and Exchange Commission and became effective on October 10, 2018. This press release shall not constitute an offer to sell or a 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.

On October 9, 2018 Incyte (Nasdaq:INCY) reported that interim Phase 2 data on its investigational, selective FGFR1/2/3 inhibitor, pemigatinib (INCB54828), will be presented at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress taking place in Munich, Germany from October 19-23, 2018 (Press release, Incyte, OCT 9, 2018, View Source [SID1234529818]).

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Data at ESMO (Free ESMO Whitepaper) 2018 will include poster presentations on the FIGHT-202 study of pemigatinib in patients with previously treated advanced/metastatic or surgically unresectable cholangiocarcinoma (bile duct cancer) with fibroblast growth factor (FGF)/FGFR genetic alterations, as well as the FIGHT-201 study of pemigatinib in patients with metastatic or surgically unresectable urothelial carcinoma (bladder cancer) harboring FGF/FGFR genetic alterations.

"We are pleased that data on pemigatinib – part of our targeted therapy portfolio – have been selected for presentation at this year’s ESMO (Free ESMO Whitepaper) Congress," stated Steven Stein, M.D., Chief Medical Officer, Incyte. "We look forward to sharing updated interim data from the ongoing FIGHT-202 trial of pemigatinib in patients with cholangiocarcinoma, which continue to support our plan for a 2019 NDA submission in this indication, as well as updated data from the FIGHT-201 study of pemigatinib in patients with urothelial carcinoma, which support recruitment into the continuous dosing cohort of this study."

Abstracts were made available today on the ESMO (Free ESMO Whitepaper) Congress website at View Source

Poster details:

Interim Results of FIGHT-202, a Phase 2, Open-Label, Multicenter Study of INCB054828 in Patients (pts) with Previously Treated Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma (CCA) with/without Fibroblast Growth Factor (FGF)/FGF Receptor (FGFR) Genetic Alterations (Abstract #756P, poster display session)

Sunday, 21 October 2018 from 12:45 p.m. CEST to 1:45 p.m. CEST (6:45 a.m. ET to 7:45 a.m. ET) in Hall A3 – Poster Area Networking Hub
Interim Results of FIGHT-201, a Phase 2, Open-Label, Multicenter Study of INCB054828 in Patients (pts) with Metastatic or Surgically Unresectable Urothelial Carcinoma (UC) Harboring Fibroblast Growth Factor (FGF)/FGF receptor (FGFR) Genetic Alterations (GA) (Abstract #900P, poster display session)

Monday, 22 October 2018 from 12:45 p.m. CEST to 1:45 p.m. CEST (6:45 a.m. ET to 7:45 a.m. ET) in Hall A3 – Poster Area Networking Hub
Full session details and data presentation listings for ESMO (Free ESMO Whitepaper) 2018 can be found at:
View Source

About FGFR and Pemigatinib (INCB54828)

Fibroblast growth factor receptors (FGFRs) play an important role in tumor cell proliferation and survival, migration and angiogenesis (the formation of new blood vessels). Activating mutations, translocations and gene amplifications in FGFRs are closely correlated with the development of various cancers.

Pemigatinib is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations. Phase 2 studies investigating the safety and efficacy of pemigatinib monotherapy across several FGFR-driven malignancies are ongoing—the FIGHT (FIbroblast Growth factor receptor in oncology and Hematology Trials) clinical trial program currently comprises FIGHT-201 in patients with metastatic or surgically unresectable bladder cancer, including with activating FGFR3 alterations; FIGHT-202 in patients with metastatic or surgically unresectable cholangiocarcinoma who have failed previous therapy, including with activating FGFR2 translocations; and FIGHT-203 in patients with myeloproliferative neoplasms with activating FGFR1 translocations.

On October 8, 2018 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported it has entered into a purchase agreement with several institutional investors for the purchase of common units and pre-funded units for a combined total of 4,629,630 units in a registered direct offering, for expected gross proceeds of approximately $25 million before placement agent fees and other offering expenses payable by Altimmune (Press release, Altimmune, OCT 9, 2018, View Source [SID1234529900]).

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Each common unit is being sold at a public offering price of $5.40 and consists of one share of common stock and a warrant to purchase one share of common stock at an exercise price of $5.40. Each pre-funded unit is being sold at a public offering price of $5.39 and consists of a pre-funded warrant to purchase one share of common stock at an exercise price of $0.01 per share and a warrant to purchase one share of common stock at an exercise price of $5.40. The public offering price of each pre-funded unit is equal to the public offering price of each common unit being sold to the public in this offering, minus $0.01. The pre-funded warrants will be immediately exercisable and may be exercised at any time until all of the pre-funded warrants are exercised in full. The other warrants will be exercisable immediately and will expire five years from the date of issuance. The terms of the warrants and the pre-funded warrants will be substantially the same as the terms of the warrants and the pre-funded warrants issued in connection with the Company’s public offering completed October 2, 2018 For a summary of the material terms of the warrants and pre-funded warrants, please refer to Exhibit A attached to this press release.

The offering is expected to close on or about October 10, 2018, subject to customary closing conditions. Altimmune intends to use the net proceeds from this offering for the continued advancement of development activities for our clinical-stage product pipeline, general corporate purposes and strategic growth opportunities.

Roth Capital Partners is acting as sole placement agent for the offering.

The securities described above are being offered by Altimmune pursuant to a registration statement on Form S-3 (File No. 333-217034) that was declared effective by the Securities and Exchange Commission (SEC) on April 6, 2017. A final prospectus supplement and an accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s web site at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, by contacting Roth Capital Partners, LLC, Attention: Equity Capital Markets, 888 San Clemente Drive, Suite 400, Newport Beach, California 92660, by telephone at (800) 678-9147 or e-mail at [email protected].

This press release shall not constitute an offer to sell or a 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.