Merck Establishes Strategic Oncology Collaboration with Taiho and Astex

On January 6, 2020 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported an exclusive worldwide research collaboration and license agreement with Taiho Pharmaceutical Co, Ltd., ("Taiho") and Astex Pharmaceuticals (UK), a wholly owned subsidiary of Otsuka Pharmaceutical Co., Ltd. ("Astex"), 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, Merck & Co, JAN 6, 2020, View Source [SID1234553268]).

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"At Merck 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, Merck 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."

Under the terms of the agreement, Merck, Taiho and Astex will combine preclinical candidates and their data with knowledge and expertise from their respective research programs. In exchange for providing Merck 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. Merck 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.

"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 Merck, allows us to combine our expertise to significantly accelerate the global research, development, and commercialization of a number of our mutant KRAS programs by accessing external talent and resources."

"Together with our Taiho colleagues we are delighted to be working with Merck, 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," said Harren Jhoti, Ph.D., president and CEO of Astex.

KRAS is among the most frequently mutated oncogenes in cancer. It is estimated to occur in more than 90% of pancreatic cancers and approximately 20% of non-small cell lung cancers (NSCLC) and is associated with poorer outcomes.

Merck’s Focus on Cancer

Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.

Sysmex Launches Gene Testing Kit for Blood Cancer (ipsogen JAK2 DX Reagent)

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.

Kaleido Biosciences to Present at the 38th Annual J.P. Morgan Healthcare Conference

On January 6, 2020 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to leveraging the microbiome organ to treat disease and improve human health, reported that Alison Lawton, President and Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2020 at 4:00 p.m. PT (7:00 p.m. ET) in San Francisco, CA (Press release, Kaleido Biosciences, JAN 6, 2020, View Source [SID1234552722]).

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A live audio webcast of the presentation and question and answer session can be accessed on the Investors & Media section of Kaleido’s website at View Source An archived replay will be available for 30 days following the event.

Rocket Pharmaceuticals Announces Participation at the 38th Annual J.P. Morgan Healthcare Conference

On January 6, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that Gaurav Shah, M.D., Chief Executive Officer and President of Rocket is scheduled to present on Monday, January 13, 2020, at 4:00 p.m. Pacific Time at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco, California (Press release, Rocket Pharmaceuticals, JAN 6, 2020, View Source [SID1234552739]).

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A live audio webcast of the presentation and Q&A will be available on the Investors section of the company’s website, www.rocketpharma.com. A replay of the presentations will be archived on the Rocket website following the conferences.

Neurocrine Biosciences to Present at the 38th Annual J.P. Morgan Healthcare Conference Live Audio Webcast will be on January 13, 2020

On January 6, 2020 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported that it will present at the 38th Annual J.P. Morgan Healthcare Conference at 11:30 a.m. Pacific Time (2:30 p.m. Eastern Time) on Monday, Jan. 13, 2020, in San Francisco (Press release, Neurocrine Biosciences, JAN 6, 2020, View Source;live-audio-webcast-will-be-on-january-13-2020-300981951.html [SID1234552756]). Kevin Gorman, Chief Executive Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.