On March 31, 2020 AbbVie (NYSE: ABBV ), a global research-based biopharmaceutical company, reported that the clinical trial VIALE-A (M15-656) of VENCLEXTA (venetoclax) in combination with azacitidine versus the azacitidine combination with placebo achieved its two main primary objectives: statistically significant improvement in overall survival and compound complete remission rate for patients with prior treatment AML and ineligible for treatment with intensive chemotherapy (Press release, AbbVie, MAR 31, 2020, View Source;842586695.html [SID1234556055]). The complete results will be submitted to the United States regulatory agency, FDA ( Food and Drug Administration ), and to global health authorities, and will also be presented at medical congresses or published in specialized journals.

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!

"In the past three decades, few options have emerged for AML patients who cannot receive or tolerate intensive chemotherapy or bone marrow transplantation," said physician Neil Gallagher , Ph.D., AbbVie medical director and vice president of development . "The positive results of the VIALE-A study support the clinical benefit of the combination venetoclax plus azacytidine in AML patients who are not eligible for intensive chemotherapy and reflect our ongoing commitment to transforming the standards of care for patients with hematological neoplasms."

AML is one of the most aggressive and difficult to treat blood cancers, with a very low survival rate 1,2 . Despite advances in available therapies and care, the 5-year survival rate for patients diagnosed with AML is approximately 28% 3 . AML generally worsens rapidly and, due to age and existing comorbidities, not all patients are eligible for intensive chemotherapy 4 . AML is the most common acute leukemia in the world 5 .

It is estimated that 160,000 people are currently living with the disease globally, with an incidence rate of 103 new cases per 100,000 people 5

The Phase 3 VIALE-A study evaluated the efficacy and safety of venetoclax in combination with azacitidine compared to the placebo plus azacitidine combination. The study achieved its two primary outcomes of overall survival and complete composite remission (CR + CRi). The observed safety profile is generally consistent with the known safety profile of venetoclax with azacytidine, as seen in previous Phase 1/2 studies and the known safety profiles of the two drugs.

In November 2018, AbbVie received rapid US approval of VENCLEXTA in combination with azacytidine, decitabine or low-dose cytarabine for the treatment of newly diagnosed AML in adults aged 75 years or older, or with comorbidities that prevent the use of chemotherapy. intensive induction based on Phase 1/2 studies. Approval was also granted by regulatory authorities in Mexico, Israel , Puerto Rico, Peru , Brazil, Russia, Argentina , Guatemala , Uruguay, Lebanon, Bahrain , Kazakhstan, Panama, Saudi Arabia, Taiwan , Australia, Qatar and the United Arab Emirates.

VENCLEXTA is being developed by AbbVie and Roche. It is marketed jointly by AbbVie and Genentech, a member of the Roche Group , in the USA, and by AbbVie outside the USA.

About the VIALE-A Phase 3 Study (M15-656)

A total of 443 patients with AML without prior treatment were included in the study and 433 were randomized to the Phase 3 VIALE-A, double-blind, placebo-controlled study. The study was developed to assess the efficacy and safety of venetoclax in combination with azacitidine (n = 287) compared to placebo in combination with azacitidine (n = 146). 6

About VENCLEXTA (venetoclax)

VENCLEXTA (venetoclax) is the first drug in the class to selectively inhibit BCL-2. In some types of cancer, BCL-2 prevents cancer cells from going through the natural process of death or self-destruction, called apoptosis. VENCLEXTA targets the BCL-2 protein and, through its mechanism of action, helps to restore the apoptosis process.

VENCLEXTA is being developed by AbbVie and Roche. It is marketed jointly by AbbVie and Genentech, a member of the Roche Group , in the USA and by AbbVie outside the USA. Together, the companies are committed to BCL-2 research and clinical studies of venetoclax for the treatment of various types of blood and other cancers. VENCLEXTA is approved in more than 50 countries, including the United States and Brazil.

About AbbVie in Oncology

At AbbVie, we strive to discover and develop drugs that provide transformative improvement in cancer treatment, uniquely combining our deep knowledge in key areas of biology with advanced technologies, and by working together with our partners – scientists, clinical experts, peers from industry, patient support groups and patients. We remain focused on generating these transformative advances in the treatment of some of the most complex and aggressive types of cancer. AbbVie’s oncology portfolio consists of drugs already in use and new molecules being evaluated worldwide, through more than 300 clinical studies and more than 20 different types of tumors. For more information, visit www.abbvie.com/oncology.

On March 31, 2020 IONpath, Inc. reported that it has jointly produced with Bristol Myers Squibb an article entitled Multiplexed Ion Beam Imaging (MIBI) for Characterization of the Tumor Microenvironment Across Tumor Types (Multiplexed ionic ray images to characterize the tumor microenvironment in different types of tumors) for the journal Laboratory Investigation of the scientific journal Nature (Press release, IONpath, MAR 31, 2020, View Source [SID1234556054]).

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!

The ability to understand all cells present within a tumor by differentiating different cell types at the same time in a single tissue section has been limited by the technology available. In the article, the authors demonstrate how Multiplexed Ion Beam Imaging (MIBI ) technology (multiplexed ion beam images) is able to detail the characteristics of a wide variety of tumor types by providing identification of the cell phenotype along with the analysis of their spatial relationships. For this collaboration, Bristol Myers Squibb provided samples of 50 tumor biopsies to IONpath, which were simultaneously marked with a panel of 15 antibodies, each labeled with a specific metal isotope. The highly detailed mapping of the tumor microenvironment generated an immunological profile(fig. 1) and a spatial organization (fig. 2) through a multi-step process that segmented the sample to the individual cell.

Such information was collected as an exercise to observe the characteristics of the samples in question. However, the implications of this new level of tumor mapping at the cellular level can be far-reaching. Scientists and drug developers can now measure the proximity between immune cells and cancer cells. Within the same sample, they can also measure protein expression levels of potential drug targets and other variables associated with the response to certain therapies, such as those that target immunological checkpoints.

"IONpath’s mission is to enable medical discovery, specifically in the field of immuno-oncology, by characterizing the tumor microenvironment down to the cellular level," said Dr. Jessica Finn , director of pathology and one of the authors of the article. She added that "examining these detailed and interrelated structures is a difficult but important job in the fight to cure certain types of cancer."

Exemplifying how this mission for IONpath can impact on real-world decisions, the study demonstrated the possibilities for calculating distances between different cell subsets, including tumor and immune cells, as well as subsets of immune cells that express PD-1 and PD -L1. Further studies in this area can be designed to support better results in the field of immuno-oncology.

On March 31, 2020 GenScript Biotech Corp. (Stock Code: 1548.HK), a leading global biotechnology company, reported full year 2019 financial results and provided a corporate update (Press release, GenScript, MAR 31, 2020, View Source [SID1234556053]). In 2019, overall revenue of the Group was approximately US$273.4 million, representing an increase of 18.4% as compared with that for the year ended December 31, 2018. Gross profit was approximately US$180.3 million, representing an increase of 13.8% YoY.

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!

GenScript’s four major business segments continued to grow steadily in 2019. Revenue growth was primarily attributable to the continued stable growth of life science products and services, an increase in contract revenue from Legend’s collaboration with Janssen on CAR-T cell therapy (JNJ-4528), the rapid conversion of biologics CDMO service backlog, and an increase in customers and their purchase volumes of industrial synthetic biology products.

GenScript has made significant investments in R&D in each of its business segments. During the reporting period, the Company’s R&D expense increased by 151.0% to US$186 million compared with 2018.

"GenScript’s current investments and our bold initiatives across all four business units are significantly advancing our long-term goals and illustrate our ongoing commitment to building a strong foundation for high-quality growth," said Dr. Zhang Fangliang, Founder, Chairman & CEO of GenScript, "We intend to continue to invest heavily in R&D, talent and facilities to drive the growth of each business segment."

Business Highlights – Legend Biotech and Cell Therapy

GenScript’s cell therapy program received multiple international designations for expediting clinical development, as the company prepared to spin off Legend Biotech as a publicly listed company.

The ongoing clinical trials for the B-cell maturation antigen (BCMA)-directed Chimeric Antigen Receptor T-cell (CAR-T) (BCMA CAR-T) program continued to proceed smoothly in the U.S. and China in 2019. The program has received Breakthrough Therapy Designation and Orphan Drug Designation from the US FDA, as well as PRIME Designation from EMA. Legend Biotech’s BCMA CAR-T product has also recently obtained Orphan Drug approval from European Commission, which will be announced by EMA soon.

In December 2019, at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) annual conference, Johnson & Johnson and Legend presented for the first time the latest Phase Ib/II pivotal trial data of CARTITUTE-1 on JNJ-4528’s efficacy and safety. JNJ-4528 showed excellent efficacy in the treatment of 29 patients with relapsed/refractory multiple myeloma (R/R MM). With a median follow-up of 6 months, a 100% overall response rate (ORR) was achieved. Legend Biotech anticipates filing a Biologics License Application (BLA) with the FDA by the end of 2020. Legend Biotech has ongoing IIT trials treating DLBCL, AML, gastric cancer and pancreatic cancer, and is also conducting IIT on allogeneic anti-CD20 CAR-T therapy. Furthermore, Legend Biotech expects to file IND for CD4 CAR-T treating TCL in the second half of 2020 and another IND for Ovarian cancer in the first half of 2021.

To date, the cell therapy business has achieved four milestones related to the BCMA CAR-T LCAR-B38M program and received milestone payments of 110 million USD from Jassen. Going forward, Legend Biotech is eligible for up to 125 million USD payment if certain manufacturing milestone is reached and additional up to 1.115 billion USD payments if other milestones are reached.

Recently, GenScript has obtained conditional approval from the Hong Kong Stock Exchange to spin off its cell therapy business and seek a public listing in the US. This is intended to enable operational focus and strategy development at both GenScript and Legend Biotech, better align management responsibilities and increase the financial and operational transparency for both GenScript and Legend Biotech. It is anticipated that after a potential Legend IPO, Legend will continue to be consolidated under GenScript Group. Existing GenScript shareholders, through their interest in GenScript, will continue to own a majority interest in Legend Biotech.

In addition, GenScript announced this morning that Legend has just closed a round of private financing, a syndicate of highly reputable institutional investors have agreed to invest approximately 150.5 million USD into Legend, this deal values Legend at approximately 1.95 billion USD on a fully diluted basis. This round of financing is co-led by Hudson Bay Capital, JNJ Innovation, and Lily Asia Ventures, other participating investors included Capital Group, Vivo Capital, and RA Capital. These pre-IPO investors can also choose to invest more into Legend at the IPO stage. This is endorsement for Legend’s technology and commercial potential in the future.

Business Highlights – Life Sciences, Biologics CDMO, Industrial Synthetic Biology

In addition to Legend Biotech’s progress in the cell therapy segment, GenScript’s other segments continued to grow steadily.

GenScript’s life science services and products segment, as the traditional business of the Company, remains a strong and stable revenue source for the overall business. GenScript has maintained its position as one of the world’s largest molecular biology CRO companies. In 2019, the Company continued to focus on successful commercial operations in synthetic biology. The Company has grown its commercial operations by establishing sites in Europe and Asia Pacific, organizing a new management team and a local team, building its brand and launching an online service platform. The Company expanded its gene synthesis throughput by 50% through investments in automation and maintained its number one market share position globally. Commissioning the production facility in Zhenjiang, along with the automated peptide production line, boosted production capacity.

By the end of 2019, revenue from life science services and products segment was approximately US$170.4 million, representing an increase of 20.9% as compared with approximately US$141.0 million for the year ended December 31, 2018. During the same period, the gross profit was approximately US$110.6 million, representing an increase of 15.7% as compared with approximately US$95.6 million for the year ended December 31, 2018.

In the biologics CDMO segment, the Company entered into significant collaborations at home and abroad and leveraged its R&D strength to efficiently expand capacity. Bringing in Berkeley Lights optofludic single cell platform, the company became the first CDMO in Asia Pacific to apply this global leading digital cell biology technology in the antibody development technology service. In late 2019, China’s largest plasmid and virus facility for clinical sample production began operations. During the reporting period, revenue from the biologics CDMO business was approximately US$22.5 million, representing an increase of 8.7% as compared with approximately US$20.7 million for the year ended December 31, 2018. During the same period, the gross profit was approximately US$7.0 million, representing a decrease of 20.5% as compared with approximately US$8.8 million for the year ended December 31, 2018. Total backlog for biologics development services increased by 172.9% from US$18.1 million from the year ended December 31, 2018 to US$49.4 million for the year ended December 31, 2019.

The Industrial synthetic biology products business continued its rapid growth. In 2019, the Company launched several innovative products and entered the bio-synthesis market, shifted its marketing strategy from a product seller to a solution provider in order to expand market share, and optimized the production process for cost reduction and quality improvement. During the reporting period, revenue from industrial synthetic biology products was approximately US$23.1 million, representing an increase of 30.5% as compared with approximately US$17.7 million for the year ended December 31, 2018. Excluding impact from foreign currency conversion, constant currency revenue increased by 34.6%. During the same period, the gross profit was approximately US$5.3 million, representing an increase of 112.0% as compared with US$2.5 million for the year ended December 31, 2018.

GenScript 2020

In January 2020, GenScript hosted the GenScript Biotech Global Forum during the J.P. Morgan Healthcare Conference in San Francisco, drawing thousands of attendees. According to media coverage, "At the top global industry event, GenScript’s role has changed from an attendee to a speaker to an independent sponsor… As an emerging innovative pharmaceutical company in China, GenScript is narrowing the gap in terms of global biological innovation… As China’s innovation ecosystem continues to improve, ‘China’s rising power’ will stand the test of time."

The life science services and products segment, GenScript’s traditional business, has significant growth opportunities around the world. GenScript will focus on expanding its industrial customer base, relying on the Europe and Asia Pacific Divisions to support global expansion, investing in automation to improve CRO business efficiency, and leveraging its leading R&D strength to develop higher industry standards.

The company’s cell therapy segment will focus on moving the BCMA program towards commercialization in last line R/R MM patients and clinical trials in earlier line MM treatment. The next-generation cell therapy pipeline will target malignant hematological tumors, solid tumors and infectious diseases, using autologous and allogeneic CAR-T technology. This will create new growth potential for Legend Biotech and help grow the business to become the world’s leading biopharmaceutical company.

GenScript’s Biologics CDMO segment has become a new growth driver of the Company. With well-defined strategic positioning, GenScript’s CDMO segment focuses on biologics CDMO and gene and cell therapy CDMO. The Company’s planned and implemented construction projects are intended to increase the GMP capacity to meet the manufacturing requirements of Phase I/II/III clinical trials and commercialization, to offer end-to-end biologics CDMO services to customers, and to make the Company a leading biologics and gene and cell therapy CDMO provider.

For the industrial synthetic biology products segment, the Company will continue to focus on industry key accounts, optimizing capacity, and combining R&D and applications to maintain rapid growth.

"GenScript aims to be one of the leaders in global gene and cell therapy in five years, while maintaining our leadership and commitment to our fundamental business of gene synthesis technology. In the next decade, our main course business will focus on biologics CDMO, cell and gene therapy, life science services and products, and bio-enzyme and synthetic biology products. To this end, we must invest resolutely. To make breakthroughs in the most prospective cell therapy for cancer treatment and even get far ahead, a Chinese biotech company must be committed to bold continued investment," said Dr. Zhang Fangliang.

On March 31, 2020 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that company management will participate in the 1-on-1 meetings at the upcoming 19th Annual Needham Virtual Healthcare Conference from Tuesday, April 14, 2020 through Wednesday, April 15, 2020 (Press release, Personalis, MAR 31, 2020, View Source [SID1234556052]).

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!

On March 31, 2020 AVEO Oncology (NASDAQ: AVEO) reported that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tivozanib, the Company’s vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI), as a treatment for relapsed or refractory renal cell carcinoma (RCC) (Press release, AVEO, MAR 31, 2020, View Source [SID1234556051]).

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!

"NDA submission is a distinguishing milestone for any development stage biotechnology company, and our tivozanib NDA is an important step in our goal of providing an effective and more tolerable therapeutic option to patients with relapsed or refractory RCC," said Michael Bailey, president and chief executive officer. "The TIVO-3 study provides valuable insight into the potential sequencing of therapy following earlier TKI and immunotherapy treatment, an area of significant need for kidney cancer patients whose disease has relapsed or become refractory to multiple lines of therapy. All of us at AVEO offer our continued gratitude to the patients, caregivers, and investigators who participated in our clinical trials. We look forward to working closely with the FDA during their review process and remain hopeful that the study’s overall survival (OS) hazard ratio (HR) will continue to favor tivozanib at the time of the final readout, expected by June 2020."

The NDA submission is based on the pivotal active comparator-controlled Phase 3 study, TIVO-3, comparing tivozanib to sorafenib in 3rd and 4th line RCC patients. The application is supported by three additional trials, including an active comparator-controlled supportive Phase 3 study, TIVO-1, comparing tivozanib to sorafenib, and two Phase 2 studies, Study 902, the open-label, crossover clinical study of tivozanib for patients who progressed on sorafenib in TIVO-1, as well as placebo-controlled Study 201 in first line RCC patients.

A final OS analysis of the TIVO-3 study will be conducted in the second quarter based on a May 1, 2020 data cutoff date. AVEO expects to report results from the final OS analysis by June 2020. The FDA and the Company agreed that if, during the review, the final analysis yields an OS HR above 1.00, the Company will withdraw its NDA.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union, the United Kingdom, Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for relapsed/refractory RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers.