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.

On January 6, 2020 Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform for patients with relapsed or refractory acute myeloid leukemia (AML) (clinical trial identifier: NCT03927261) (Press release, Precigen, JAN 6, 2020, View Source [SID1234552952]). Precigen announced in Q3 2019 that it had completed enrollment for the first cohort of this clinical trial and expects an initial data readout in the second half of 2020.

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The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization.

"This regulatory designation underscores the critical medical need for new therapies to treat AML patients. AML is a progressive, debilitating and often fatal disease with limited treatment options," said Helen Sabzevari, PhD, President and CEO of Precigen*. "As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease."

PRGN-3006 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS.

Preclinical data for PRGN-3006 UltraCAR-T evaluating non-viral, multigenic autologous CAR-T cells administered one day after gene transfer for the treatment of AML and MDS were presented at the 2019 American Society for Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition in Orlando, Florida.

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it was estimated that 21,450 new cases of AML would be diagnosed in the US2. AML is uncommon before the age of 45 and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall, and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age3.

About the FDA Orphan Drug Designation
FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization, such as grants, clinical design support, FDA fee waivers, tax incentives, and seven years of market exclusivity.

Precigen : Advancing Medicine with PrecisionTM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

*Precigen’s parent company, Intrexon Corporation (Nasdaq: XON), announced on January 2, 2020 that it will refocus the company on healthcare, will change its name to Precigen, Inc., and has appointed Helen Sabzevari, PhD, as President and CEO. The parent company will continue to hold, among its several health assets, all of Precigen’s discovery and clinical stage technology and programs.

Precigen’s UltraCAR-TTM Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.

On January 6, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as solutions for diseases with high unmet medical need, reported it will present at the 38th Annual J.P. Morgan Healthcare Conference to be held January 13-16 in San Francisco, CA (Press release, Aeglea BioTherapeutics, JAN 6, 2020, View Source [SID1234552791]).

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Presentation Details

Highlights: Aeglea will present on its strategic focus in developing innovative human enzyme therapeutics with defined potential to address Arginase 1 Deficiency, Homocystinuria and Cystinuria.
Date: Wednesday, January 15, 2020
Time: 2:30 p.m. PST; 5:30 p.m. EST
Presenter: Anthony G. Quinn, M.B. Ch.B., Ph.D., Aeglea’s president and chief executive officer
Location: Westin St. Francis Hotel, San Francisco, California
Webcast: View Source

To access the live and archived audio webcast, visit the Presentations & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary. Replays of the webcast will be available for 30 days thereafter.

On January 6, 2020 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, is scheduled to present at the Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top conference on Thursday, January 9, 2020, at 1:10 p.m. ET (Press release, CRISPR Therapeutics, JAN 6, 2020, View Source [SID1234552790]).

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A live webcast of the fireside chat will be available on the "Events & 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 14 days following the presentation.

On January 6, 2020 Yisheng Biopharma Co., Ltd. ("Yisheng Biopharma"), a biopharmaceutical company focusing on research, development, manufacturing, sales and marketing of immunological biologics and vaccines, and Tavotek Biotherapeutics, a biotech company focusing on novel multi-specific antibodies in immuno-oncology and autoimmune diseases, reported that the companies have entered into a strategic research alliance and collaborate in the development involving their lead assets in oncology (Press release, Yisheng US Biopharma, JAN 6, 2020, View Source [SID1234552768]). The objective of the alliance is to co-develop a combination therapy of YS-ON-001/002 and Tavo-301/303 for cancer treatment. The two companies are also in discussions regarding additional research and development collaborations beyond oncology.

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"We are excited to collaborate with Tavotek, which has a rich pipeline of multi-specific antibodies and advanced technology platforms in oncology and other therapeutic areas," commented Dr. David Shao, President and Chief Executive Officer of Yisheng Biopharma. "As potent agonists of TLR3, MDA5 and RIG-I pathways, YS-ON-001 and YS-ON-002 have demonstrated promising effects in activating the innate and adaptive immune systems and modulating the tumor microenvironment, and YS-ON-001 has shown an excellent safety profile with clinical data to date. By combining YS-ON-001/002 with multi-specific antibodies directed against tumors, we are well positioned in developing a first-in-class immunotherapy with potentially higher response rates. We look forward to working with the Tavotek team to explore the potential synergy of YS-ON-001/002 and Tavo-301/303."

"The collaboration with Yisheng aligns with Tavotek’s mission to develop life-changing therapies for patients with significant unmet medical need," said Dr. Mann Fung, Chief Executive Officer of Tavotek. "Current immuno-oncology approaches such as PD1/PDL1 antibody achieve only approximately 20 to 30 percent response rates in clinical settings. A majority of cancer patients still are not seeing a benefit from current therapies. We believe a combination regimen of YS-ON-001/002 with multi-specific IO antibodies, such as Tavo-301/303, will have beneficial impact on cancer care."

YS-ON-001 and YS-ON-002 are immunotherapeutic products based on the TLR3/RIG-I/MDA-5 signaling pathways of PIKA immunomodulating technology. They are capable of both reducing the immunosuppressive effect of tumor microenvironment and enhancing the anti-tumor function of the immune system to tumor cells. YS-ON-001 is currently in clinical development in China and Singapore, and has received orphan drug designations from the U.S. FDA for treatment of pancreatic and liver cancers. The product has been approved in Cambodia for the treatment of advanced solid tumors. YS-ON-002 is a clinical candidate ready for IND submission. YS-ON-001/002 can be an integral immunotherapy component with standard of oncology care, such as chemotherapies, targeted therapies and checkpoint inhibitors or with emerging immunotherapies for additive or synergistic treatment benefits.

Tavo-301/303 is a series of novel multi-specific antibody-based Immuno-Oncology assets that was developed using Tavotek’s proprietary TavoSelect Platform. Novel protein engineering employing TavoSelect technology generates multi-specific biologics with optimal molecular profiles for targeting multiple relevant epitopes simultaneously to manage difficult-to-treat solid tumors. These biologics modulate the immunosuppressive pathway by multiple mechanisms of action to promote anti-tumor response and efficacy. The TavoSelec technology also provides biologic molecules with favorable pharmacokinetic profiles and stable formats for ease of development and manufacturing.