On April 9, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that valemetostat (DS-3201), an investigational and potential first-in-class EZH1/2 dual inhibitor, has received SAKIGAKE Designation for the treatment of adult patients with relapsed/refractory peripheral T-cell lymphoma (PTCL) by the Japan Ministry of Health, Labour and Welfare (MHLW) (Press release, Daiichi Sankyo, APR 9, 2019, View Source [SID1234535072]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"There is a need for new and novel treatment approaches for patients with peripheral T-cell lymphoma, a group of heterogenous diseases for which relapse rates tend to be high and options beyond systemic chemotherapy are limited," said Kaszushi Araki, Valemetostat Global Team Leader, Oncology Clinical Development Department, Oncology Function, Daiichi Sankyo. "We look forward to working closely with the Japan Ministry of Health, Labour and Welfare to optimize development of valemetostat and to potentially offer a new, first-in-class targeted therapy option for patients with various subtypes of relapsed/refractory PTCL, including those that are more common in Japan."

The SAKIGAKE Designation System promotes R&D in Japan, driving early practical application for innovative pharmaceutical products, medical devices, and regenerative medicines. As a designated medicine under the SAKIGAKE Designation System, valemetostat will have prioritized consultation, a dedicated review system to support the development and review process, as well as reduced review time from the normal 12 months to 6 months.

Valemetostat targets epigenetic regulation by inhibiting both the EZH1 (enhancer of zeste homolog 1) and EZH2 (enhancer of zeste homolog 2) enzymes that act through histone methylation to regulate gene expression.[1] Reactivation of the silenced genes results in decreased proliferation of EZH2-expressing cancer cells.1 Preclinical research has shown that valemetostat suppressed trimethylation of H3K27 in cells more strongly than EZH2 selective inhibitors.[2] Valemetostat also displayed antitumor activity in various hematological malignancies in preclinical models.2,[3]

SAKIGAKE Designation was granted based on the preliminary results of an ongoing phase 1 study assessing the safety and efficacy of valemetostat in patients with non-Hodgkin lymphomas (NHL) including PTCLs, which were presented at the 2017 annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper).[4]

About Peripheral T-Cell Lymphoma (PTCL)

Peripheral T-cell lymphoma (PTCL) is a subtype of non-Hodgkin lymphoma (NHL), a form of cancer that originates in lymphocytes, a type of white blood cell.[5] The two main types of NHL are B-cell lymphomas and T-cell lymphomas, which are classified into subtypes based on the origin and stage of the cancer.[6]

There are at least eight different types of PTCL.[7] Each type is considered rare. Global incidence of PTCL subtypes varies by geographic region.[8] Most, but not all, types of PTCL are aggressive, and patients are usually treated with systemic chemotherapy; relapse is common after initial treatment.8

The incidence of lymphoma, including NHL, is increasing year by year around the world.[9] There were an estimated 509,000 new cases and about 248,000 deaths globally from NHL in 2018.[10] In Japan, there were nearly 21,000 new cases of NHL in 2012.[11] While recent treatment advances have led to improved outcomes for patients with certain types of NHL, patients with aggressive NHL subtypes or relapsed/refractory disease still face a poor prognosis.6,[12]

About Valemetostat

Part of the investigational AML Franchise of the Daiichi Sankyo Cancer Enterprise, valemetostat is an investigational and potential first-in-class EZH1/2 dual inhibitor in phase 1 clinical development for hematologic cancers including acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and Non-Hodgkin lymphoma (NHL), including adult T-cell leukemia/lymphoma (ATL/L), peripheral T-cell lymphoma (PTCL), and B-cell lymphomas. Valemetostat is an investigational agent and has not been approved by the FDA or any other regulatory agency worldwide as a treatment for any indication. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise

The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science Franchise, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/ immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in pivotal stage development include: DS-8201, an antibody drug conjugate (ADC) for HER2 expressing breast, gastric and other cancers; quizartinib, an oral selective FLT3 inhibitor, for newly-diagnosed and relapsed/refractory acute myeloid leukemia (AML) with FLT3-ITD mutations; and pexidartinib, an oral CSF-1R inhibitor, for tenosynovial giant cell tumor (TGCT). For more information, please visit: www.DSCancerEnterprise.com.

On April 9, 2019 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company focused on developing a pipeline of first-in-class drug candidates targeting AXL kinase to treat aggressive diseases including immune-evasive and therapy resistant cancers, reported that Richard Godfrey, BerGenBio’s Chief Executive Officer, will present an overview of the Company at the H.C. Wainwright Global Life Sciences Conference today, April 9, 2019 (Press release, BerGenBio, APR 9, 2019, View Source [SID1234535054]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Date:

Tuesday, April 9, 2019

Time:

09:10 AM BST

Location:

JW Marriott Grosvenor House, London, UK

The presentation slides will be available on www.bergenbio.com in the investor section at time of presentation.

On April 8, 2019 GT Biopharma, Inc. (OTCQB: GTBP) (GTBP.PA) an immuno-oncology biotechnology company focused on innovative treatments based on the Company’s proprietary NK-engager (TriKE) platform and Multi-Target Bispecific Drug Conjugate (MTBDC) platform, reported it has received authorization from the University of Minnesota’s Institutional Review Board (IRB) and Cancer Protocol Review Committee (CPRC) that it can proceed with its planned TriKE Phase 1 clinical trial (Press release, GT Biopharma , APR 8, 2019, View Source [SID1234539515]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Our Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) was earlier authorized allowing us to initiate a first-in-human Phase 1 study with GTB-3550, its first-in-class (TriKE), for the treatment of acute myelogenous leukemia (AML), myelodysplatic syndrome (MDS) and mastocytosis. The study will be led by Principal Investigator, Erica Warlick, MD, Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation at Masonic Cancer Center, University of Minnesota.

"GTB-3550 is a protein immune engager that binds to natural killer (NK) cells and targets them specifically to leukemia cells," said Jeffrey Miller, MD, Deputy Director, Masonic Cancer Center, University of Minnesota. "Our team has been working on the optimal construct for some time, and we are excited to see it is ready for clinical testing. In addition, the same TriKE protein will deliver an interleukin-15 stimulus, a growth factor that makes NK cells proliferate and be more active."

"The clinical trials team at the University of Minnesota are excited to commence the Phase 1 trial testing this novel immunotherapeutic agent, GTB-3550," said Anthony Cataldo (CEO GT Biopyarma). "The pre-clinical data are compelling, and success with GTB-3550 in this Phase 1 study will allow us to further investigate the TriKE in a larger number of cancer patients."

This single center, first-in-human Phase 1 clinical trial of GTB-3550 will enroll up to 60 subjects with CD33-expressing high risk for refractory/relapsed AML, MDS, or advanced systemic mastocytosis. Subjects will receive a single course of GTB-3550 TriKE given as 3 weekly treatment blocks. Each block consists of four consecutive 24-hour continuous infusions of GTB-3550 TriKE followed by a 72-hour break after Block #1 and #2. Disease response will be assessed by bone marrow biopsy performed between Day 21 and Day 42 after the start of the 1st infusion. Follow-up for response and survival continues through 6 months from treatment start. The primary objective from the Phase 1 dose finding portion of the study will be to identify the maximum tolerated dose (MTD) of GTB-3550 TriKE defined as the dose level that most closely corresponds to a dose limiting toxicity rate (DLT) of 20%. The primary objective from the Phase 2 extended portion of the study will be the potential efficacy of GTB-3550 TriKE, measured using rates of complete and partial remission. Subjects experiencing clinical benefit and no unacceptable side effects may be considered for a 2nd course of GTB-3550 TriKE on a compassionate basis.

About Acute Myelogenous Leukemia (AML)

AML is the most common form of adult leukemia with 21,000 new cases expected in 2018 alone, according to the American Cancer Society. AML patients typically receive frontline therapy, most commonly chemotherapy, which includes cytarabine and an anthracycline, a therapy that has not changed in over 40 years. However, there remains a significant unmet need in these therapies with about half of AML patients experiencing relapses or requiring alternative therapies. The Company is developing GTB-3550 to serve as a relatively safe, cost-effective, and easy-to-use therapy for resistant/relapsing AML and could also be combined with chemotherapy as frontline therapy thus targeting the larger patient population.

About Myelodysplastic Syndrome (MDS)

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become abnormal, leading to low numbers of one or more types of blood cells. There are several different types of MDS, based on how many types of blood cells are affected and other factors, although the most common finding in MDS is a shortage of red blood cells (anemia). The number of people with MDS diagnosed in the U.S. each year is estimated to be ~10,000. MDS is uncommon before age 50 and is most commonly diagnosed in people in their 70s. In about 1 in 3 patients, MDS can progress to AML, a rapidly growing cancer of bone marrow cells.

About Mastocytosis

Mastocytosis is a rare disorder characterized by abnormal accumulations of mast cells in the skin, bone marrow, and internal organs (liver, spleen, gastrointestinal tract and lymph nodes). Cases beginning during adulthood tend to be chronic and involve the bone marrow in addition to the skin, whereas, during childhood, the condition is often marked by skin manifestations with no internal organ involvement and can often resolve during puberty. In most adult patients, mastocytosis tends to be persistent, and may progress into a more advanced category in a minority of patients. Mastocytosis affects both males and females and can begin during childhood or adulthood. In children, 80% of cases appear during the first year of life, and the majority is limited to the skin. Adults who develop mastocytosis more often have systemic forms of the disease. Cutaneous forms of the disease account for less than 5% of adult cases. An estimate of prevalence from a recent population-based study is approximately 1 case per 10,000 people.

About GTB-3550

GTB-3550 (OXS-3550) is the Company’s first Tri-specific Killer Engager (TriKE) product candidate being initially developed for the treatment AML. GTB-3550 is a single-chain, tri-specific scFv recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. When the NK stimulating cytokine human IL-15 is used as a crosslinker between the two scFvs, it provides a self-sustaining signal that activates NK cells and enhances their ability to kill. We intend to study this anti-CD16-IL-15-anti-CD33 tri-specific killer engager, or TriKE, in CD33 positive leukemias, a marker expressed on tumor cells in AML, myelodysplastic syndrome, or MDS, and other hematopoietic malignancies. CD33 is primarily a myeloid differentiation antigen with endocytic properties broadly expressed on AML blasts and, possibly, some leukemic stem cells. CD33 or Siglec-3 (sialic acid binding Ig-like lectin 3, SIGLEC3, SIGLEC3, gp67, p67) is a transmembrane receptor expressed on cells of myeloid lineage. It is usually considered myeloid-specific, but it can also be found on some lymphoid cells. The anti-CD33 antibody fragment that will be used for these studies was derived from the M195 humanized anti-CD33 scFV and has been used in multiple human clinical studies. It has been exploited as target for therapeutic antibodies for many years. Improved survival seen in many patients when the antibody-drug conjugate gemtuzumab was added to conventional chemotherapy validates this approach. GT Biopharma believes that GTB-3550 could serve as a relatively safe, cost-effective, and easy-to-use therapy for resistant/relapsing AML and could also be combined with chemotherapy as frontline therapy thus targeting the larger patient population.

On April 8, 2019 Kymab, a clinical-stage biopharmaceutical company developing antibody-based therapeutics, reported the appointment of Simon Sturge as its new CEO, effective May 1, 2019 (Press release, Kymab, APR 8, 2019, View Source [SID1234537017]). Simon succeeds David Chiswell, who remains a scientific adviser to the Company.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are delighted that such an experienced leader as Simon will lead Kymab to its next stage of growth," said Martin Nicklasson, Non-Executive Chair. "Led by Simon, Kymab will continue to progress its innovative portfolio of products through the clinic and to market, driving our strategy to transform patients’ lives. This appointment is part of Kymab’s strategy to position itself for long-term success, building on the incredibly strong R&D foundation of the Company. We’d also like to thank David for many years of leadership at Kymab as CEO and previously as Chair – his leadership has been instrumental to the Company’s evolution."

Mr. Sturge brings to Kymab over 30 years of international leadership experience in both the biotechnology and pharmaceutical industry. He joins Kymab from Merck KGaA, where he was Executive Vice President Global Strategy, Business Development & Global Operations. Previously, he was Chief Operating Officer of Merck Healthcare, responsible for company’s global commercial and manufacturing operations. Over the past five years, he has been responsible for the continued growth in global sales at Merck KGaA, as well as the commercial launches of BAVENCIO (anti-PD-L1 avelumab) in solid tumours and MAVENCLAD (cladribine) for relapsing multiple sclerosis. Prior to joining Merck KGaA, Mr. Sturge served as Corporate Senior Vice President, Biopharmaceuticals at Boehringer Ingelheim, where he had responsibility for the research, development and commercialisation of the company’s global biosimilar portfolio. Before that, he held CEO roles at a number of healthcare companies including Celltech Biologics, and he served as founder and CEO of Ribotargets, which was acquired by British Biotech and subsequently renamed Vernalis.

"I am excited to have this opportunity to head Kymab at such a transformative time in its development," said Simon Sturge. "With its competitive antibody research platforms and evolving clinical pipeline, Kymab is well positioned as it progresses into an integrated discovery and development company."

"It has been rewarding to guide Kymab through a period of significant growth. Kymab is now a recognized leader in the antibody space, with an extensive pipeline and two lead candidates in the clinic," said Dave Chiswell, former CEO of Kymab. "I have known Simon for some years and I am pleased to hand over the reins to him. Simon is highly experienced and well positioned to lead Kymab and its exceptional staff, pipeline and partnerships, as it continues to discover and develop transformative antibody therapies for patients in need."

On April 8, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it intends to offer and sell 4,000,000 American Depositary Shares ("ADSs") representing 4,000,000 ordinary shares in an underwritten public offering in the United States (Press release, Autolus, APR 8, 2019, View Source [SID1234535077]). All ADSs to be sold in the proposed offering will be offered by Autolus. Autolus also intends to grant the underwriters a 30-day option to purchase up to an additional 600,000 ADSs at the public offering price, less underwriting discounts and commissions. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed or the actual size or terms of the offering.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Goldman Sachs & Co. LLC and Jefferies LLC are acting as joint book-running managers for the offering. Wells Fargo Securities, LLC and William Blair & Company, L.L.C. are acting as lead managers.

The proposed offering will be made only by means of a prospectus. A copy of the preliminary prospectus related to the offering can be obtained from either of the joint book-running managers for the offering, Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, or by telephone at +1 866 471 2526 or by email at [email protected]; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at +1 877 821 7388 or by email at [email protected]. For the avoidance of doubt, such prospectus will not constitute a "prospectus" for the purposes of Directive 2003/71/EC (and amendments thereto, including Directive 2010/73/EU, to the extent implemented in each relevant EU member state) and will not have been reviewed by any competent authority in any EU member state.

A registration statement on Form F-1 relating to the public offering of the ADSs described above has been filed with the U.S. Securities and Exchange Commission (the "SEC") but has not yet become effective. These securities may not be sold nor may offers to buy be accepted prior to the time the registration statement becomes effective. This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.