On January 6, 2020 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported that it exercised its option to sell $50 million of its common stock to Royalty Pharma, pursuant to the terms of the companies’ recent funding agreements announced on November 4, 2019 (Press release, Epizyme, JAN 6, 2020, View Source [SID1234552704]). The company notified Royalty Pharma that it was exercising the prespecified option when the trailing average share price reached the $20 per share cap. The closing of the sale and issuance of the shares is subject to the satisfaction or waiver of customary conditions.

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Epizyme expects that this additional capital, combined with its existing cash, cash equivalents and marketable securities on December 31, 2019 of approximately $381 million (preliminary and unaudited), will further support the company’s current operating runway into 2022, which includes the planned epithelioid sarcoma and follicular lymphoma launch activities for tazemetostat, as well as other potential value-creating initiatives.

"2020 is set to be a transformational year as we complete our evolution to a commercial enterprise," said Robert Bazemore, president and chief executive officer of Epizyme. "Our top priorities this year include successfully launching tazemetostat for the first two indications upon approvals, advancing life-cycle development for tazemetostat to support its potential utility in additional treatment settings and combinations, and progressing our research efforts to expand our pipeline. With a strong balance sheet and an exceptional team in place, I am confident in our ability to execute these activities and to realize our mission of rewriting treatment for people with cancer."

Epizyme has outlined the following key milestones for 2020:

Gain U.S. Food and Drug Administration (FDA) approval for and launch tazemetostat for patients with metastatic or locally advanced epithelioid sarcoma in the U.S.;

Gain FDA approval for and launch tazemetostat for patients in the U.S. with relapsed or refractory follicular lymphoma (FL), both with and without EZH2 activating mutations, who have received at least two prior lines of systemic therapy;

Complete the safety evaluation of tazemetostat in combination with doxorubicin and advance the confirmatory trial in the front-line epithelioid sarcoma treatment setting;

Complete the safety evaluation of tazemetostat in combination with "R2" (Revlimid plus Rituximab) and advance the confirmatory trial in the second-line FL treatment setting;

Expand clinical investigation of tazemetostat in combination with R-CHOP in the front-line treatment setting for patients with FL;

Initiate the Phase 2 efficacy portion of the ongoing Phase 1b/2 clinical trial exploring tazemetostat in combination with standard-of-care treatments for chemo-naïve patients with metastatic castration-resistant prostate cancer;

Initiate clinical investigation of tazemetostat in combination with a PARP inhibitor for certain platinum-resistant solid tumors;

Support investigator-sponsored studies designed to evaluate clinical activity of tazemetostat in various combinations for multiple tumor types; and

Pursue additional development candidates for its preclinical programs.

Epizyme’s figure for cash, cash equivalents and marketable securities as of December 31, 2019 is based on preliminary unaudited information and is subject to change as the company has not yet completed its financial closing procedures and its auditors have not reviewed this information.

On January 6, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company addressing key mechanisms of tumor drug resistance, reported that Steve Hoerter, President and Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 11:00 AM PT in San Francisco (Press release, Deciphera Pharmaceuticals, JAN 6, 2020, View Source [SID1234552703]).

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On January 6, 2020 Sysmex Corporation (HQ: Kobe, Japan; Chairman & CEO: Hisashi Ietsugu) reported its launch of the ipsogen JAK2 DX reagent (Press release, Sysmex, JAN 6, 2020, View Source [SID1234552701]). The company received marketing approval on the reagent on December 19, 2018. This product is a gene testing kit that measures the JAK2V617F mutation1 quantitatively, used in the diagnosis of certain hematopoietic tumors generally referred to as blood cancers, specifically polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF).

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This product obtained health insurance coverage effective January 1, 2020, becoming Japan’s first diagnostic aid for PV, ET, and PMF to be covered. Going forward, tests using the reagent will be covered under health insurance. This is expected to increase the opportunities for more patients to receive testing.

Representative disorders of Philadelphia chromosome-negative myeloproliferative neoplasms—PV, ET, and PMF—are caused by the oncogenesis of hematopoietic stem cells.2 According to the Japanese Society of Hematology, each year some 5,000–6,000 people in Japan suffer from these disorders.

In the tumor classification guidelines (WHO 2017 classifications) formulated by the International Agency for Research on Cancer, the agency of the World Health Organization (WHO) that specializes in cancer, identification of the causative gene mutation is indicated as a key aspect (major criteria) of the diagnostic standard for PV, ET, and PMF. The JAK2V617F mutation is the major gene mutation seen in most PV, ET, and PMF patients. Hence, the mutation is considered to be of major diagnostic importance to patients with these three disorders.

Doctors diagnose JAK2V617F mutations based on a quantitative value of the JAK2V617F mutation ratio (allele burden).3 An in vitro diagnostic (IVD) medical device for diagnosing the JAK2V617F mutation based on international standards that had received Japanese marketing approval, as well as insurance coverage, was long awaited, as none had previously existed.

In response to these medical needs, on January 1, 2020 Sysmex received health insurance coverage for, and commenced sales of, the ipsogen JAK2 DX reagent, an IVD medical device for measuring the allele burden of the JAK2V617F mutation. Now that this IVD medical device has received marketing approval, as well as insurance coverage, Sysmex believes that more people who are suspected of having PV, ET, or PMF will have opportunities to receive appropriate diagnoses based on international standards.

By working to increase testing opportunities for patients and creating high-value testing and diagnosis technologies, going forward Sysmex aims to continue contributing to the development and advancement of personalized medicine.
Product Overview
Generic name: JAK2 gene mutation kit
Name: ipsogen JAK2 DX reagent
(in vitro diagnostic medical device registration number: 23000EZX00061000)
Target market: Japan
Manufacture and sale: Sysmex Corporation
Details of Insurance Coverage
Item of measurement: JAK2 gene test
Measurement method: Allele-specific quantitative PCR (AS-qPCR) method
Objective of use: To measure the percentage of JAK2V617F mutations in genome DNA extracted from blood components (diagnostic aid for polycythemia vera, essential thrombocythemia and primary myelofibrosis)
NHI points: 2,504
Terminology
1 JAK2V617F mutation:
JAK2 refers to the tyrosine kinase JAK2 protein, which transduces the signals for regulating the growth and differentiation of blood cells. JAK2V617F indicates a mutation in which an amino acid (valine) at position 617 of JAK2 protein is replaced by phenylalanine.

2 Oncogenesis of hematopoietic stem cells:
Blood cells (red blood cells, white blood cells, and platelets) are differentiated from hematopoietic stem cells in the bone marrow. Gene mutations in these hematopoietic stem cells leads to oncogenesis, causing a rapid increase of the blood cells.

3 Allele burden:
A quantitative expression of the degree of the JAK2V617F mutation.

On January 6, 2020 Taiho Pharmaceutical Co, Ltd., ("Taiho") reported an exclusive worldwide research collaboration and license agreement with Astex Pharmaceuticals (UK), a wholly owned subsidiary of Otsuka Pharmaceutical Co., Ltd. ("Astex"), and a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA, known as MSD outside the United States and Canada ("MSD") focused on the development of small molecule inhibitors against several drug targets, including the KRAS oncogene, which are currently being investigated for the treatment of cancer (Press release, Taiho, JAN 6, 2020, View Source [SID1234552700]).

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"Taiho has used its unique and proprietary drug discovery platform to generate a number of small molecule inhibitors," said Teruhiro Utsugi, Ph.D., managing director at Taiho. "This alliance builds on our KRAS research up to now, and together with MSD it allows us to combine expertise to significantly accelerate the global research, development and commercialization of a number of our mutant KRAS programs by accessing external talent and resources."

Under the terms of the agreement, Taiho, Astex and MSD will combine preclinical candidates and their data with knowledge and expertise from their respective research programs. In exchange for providing MSD an exclusive global license to their small molecule inhibitor candidates, Taiho and Astex will receive an aggregate upfront payment of $50 million and will be eligible to receive approximately $2.5 billion contingent upon the achievement of preclinical, clinical, regulatory and sales milestones for multiple products arising from the agreement, as well as tiered royalties on sales. MSD will fund research and development and will be responsible for commercialization of products globally. Taiho has retained co-commercialization rights in Japan and an option to promote in specific areas of South East Asia.

"At MSD we continue to pursue new regimens designed to extend the benefits of highly selective therapies to more patients with cancer," said Dr. Roger M. Perlmutter, president, MSD Research Laboratories. "This agreement with Taiho and Astex combines our respective small molecule assets and industry-leading expertise in cancer cell signaling to enable development of the most promising drug candidates."

Harren Jhoti, Ph.D., president and CEO of Astex Pharmaceuticals (UK), commented,

"Together with our Taiho colleagues we are delighted to be working with MSD, one of the global leaders in oncology drug development, on this strategic alliance. This collaboration is another testament to Astex’s position as the leader in fragment-based drug discovery."

KRAS is among the most frequently mutated oncogenes in human cancers. It is estimated to occur in approximately 90% of pancreatic cancers1 and 20% of non-small cell lung cancers (NSCLC)2, where they are associated with poorer outcomes.

1: Journal of Cell Science (2016) 129, 1287-1292
2: Journal of the National Comprehensive Cancer Network; Cancer Genome Atlas Research N. Nature 2014;511:543-50

On January 6, 2020 Novartis AG (NYSE: NVS) ("Novartis") reported the successful completion of the previously announced tender offer by its indirect wholly-owned subsidiary, Medusa Merger Corporation ("Purchaser"), for all of the outstanding shares of common stock, par value USD 0.001 per share, of The Medicines Company (NASDAQ: MDCO) (the "Company") at a price of USD 85.00 per share, net to the seller in cash, without interest and subject to any tax withholding (Press release, Novartis, JAN 6, 2020, View Source [SID1234552699]).

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American Stock Transfer & Trust Company, LLC, the depositary for the tender offer, has advised Purchaser that, as of 12:00 midnight, New York City time, at the end of the day on Friday, January 3, 2020 (the "Expiration Time"), the expiration of the tender offer, (i) 60,669,325 shares were validly tendered and not withdrawn in the tender offer, representing approximately 75.0% of the outstanding shares of the Company’s common stock, and (ii) Notices of Guaranteed Delivery had been delivered with respect to 13,655,837 additional shares, representing approximately 16.9% of the outstanding shares of the Company’s common stock. On January 4, 2020, Purchaser accepted for payment all shares validly tendered and not withdrawn and will promptly pay for such shares.

Novartis will promptly complete its acquisition of the Company through consummation of a merger of Purchaser with and into the Company, with the Company surviving the merger, without a vote of the Company’s stockholders in accordance with Section 251(h) of the Delaware General Corporation Law. Following the merger, the Company will be an indirect wholly-owned subsidiary of Novartis, and each share of the Company’s common stock outstanding immediately prior to the effective time of the merger (other than shares owned by Novartis, Purchaser, the Company, any other subsidiary of Novartis or any subsidiary of the Company, or shares that are held in the Company’s treasury, or shares held by any Company stockholder who has properly demanded and perfected appraisal rights under Delaware law) will be converted into the right to receive USD 85.00 per share, net to the seller in cash, without interest and subject to any tax withholding, the same consideration received by stockholders who tendered their shares in the tender offer. As a result of the merger, as of January 6, 2020, the Company common stock will cease to be traded on the NASDAQ Global Select Market.