On December 19, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the 38TH Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco (Press release, Ultragenyx Pharmaceutical, DEC 19, 2019, View Source [SID1234552511]).

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 live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On December 19, 2019 MacroGenics, Inc. (Nasdaq: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, reported that the Company has submitted a Biologics License Application (BLA) for margetuximab, an investigational, Fc-engineered, monoclonal antibody that targets HER2 (Press release, MacroGenics, DEC 19, 2019, View Source [SID1234553174]). The margetuximab BLA is for the treatment of patients with metastatic HER2-positive breast cancer in combination with chemotherapy. The submission is based on the safety and efficacy results of the pivotal phase 3 SOPHIA study, which were first presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, with updated data recently presented at the 2019 San Antonio Breast Cancer Symposium.

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!

"As the Company’s first BLA submission, this is a key milestone for MacroGenics. We are grateful to the patients who participated in this study, as well as their families, and all involved in developing margetuximab," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "With this submission, we look forward to working with the Agency to bring margetuximab to appropriate patients. We believe the positive clinical trial results in SOPHIA demonstrate margetuximab’s potential as a treatment option for patients living with this devastating disease."

About the SOPHIA Study

The SOPHIA study (NCT02492711) is a randomized, open-label Phase 3 clinical trial evaluating margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in patients with HER2-positive metastatic breast cancer, who have previously been treated with anti-HER2-targeted therapies. All study patients had previously received trastuzumab and pertuzumab, and approximately 90% had previously received ado-trastuzumab emtansine, or T-DM1.

The study enrolled 536 patients who were randomized 1:1 to receive either margetuximab (n=266) given intravenously at 15 mg/kg every three weeks or trastuzumab (n=270) given intravenously at 6 mg/kg (or 8 mg/kg for loading dose) every three weeks in combination with one of four chemotherapy agents (capecitabine, eribulin, gemcitabine or vinorelbine) given at the standard dose. Intent-to-treat PFS analysis occurred after 265 PFS events, and the first and second interim OS analyses occurred after 158 and 270 OS events, respectively. Final OS analysis is planned after 385 events and is expected to occur in the second half of 2020.

Primary endpoints are sequentially-assessed PFS, determined by centrally-blinded radiological review, and OS. Key secondary endpoints are PFS by investigator assessment and ORR. Tertiary endpoints include ORR by investigator assessment and safety. PFS and ORR were assessed according to Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1). Effect of CD16A (FcγRIIIa) 158 genotype on margetuximab activity is a pre-specified exploratory endpoint.

About Margetuximab

Margetuximab is an investigational monoclonal antibody that targets the HER2 oncoprotein. Margetuximab was designed to provide HER2 blockade and has similar HER2 binding and antiproliferative effects as trastuzumab. In addition, the Fc region of margetuximab has been engineered to enhance the engagement of the immune system. Margetuximab is also being evaluated in combination with anti-PD-1 therapy for the potential treatment of patients with HER2-positive gastric or gastroesophageal junction cancer. MacroGenics has initiated the Phase 2/3 MAHOGANY study (NCT04082364). For more information, please visit www.clinicaltrials.gov.

On December 19, 2019 Oxford Vacmedix UK Limited (OVM), the UK-based biopharma company focusing on the development of cancer vaccines, reported the award of an Innovate UK grant to fund research that capitalises on OVM’s proprietary recombinant overlapping peptide (ROP) technology (Press release, Oxford Vacmedix, DEC 19, 2019, View Source [SID1234553210]). The research will be carried out in collaboration with Imperial College in London and the Ditan hospital in Beijing, the leading infectious disease hospital in China. The project aims to develop a low-cost diagnostic test to detect bacteria resistant to carbapenem antibiotics. This will have advantages in terms of speed, simplicity and cost over DNA sequencing approaches, and will be accessible for low and middle income countries. These prototype assays will be produced at OVM and Imperial College and will be trialled at the Ditan Hospital. Patients in intensive care are at high risk of bacterial infections, a significant proportion of which are resistant to front-line antibiotics.

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 need to monitor and control resistance to antimicrobial therapy has been identified as an urgent global priority by both the WHO and UN. The rise in carbapenem resistance is of particular concern given the importance of this class of antibiotics in the intensive care setting, and its role as a marker for multiple antibiotic drug resistance. The availability of an inexpensive and simple test for carbapenem resistance will enable early identification of affected patients in the community. Once identified, patients can be isolated, appropriate treatment started earlier and clinical control measures put in place.

Spun out from the University of Oxford, OVM is commercialising the research on ROPs developed in the Department of Oncology at the University of Oxford. The principal application of the technology is in the development of a novel type of therapeutic cancer vaccine with potential for increased efficacy, simpler regulatory pathways and lower costs.

This new diagnostic project will run alongside OVM’s main development programmes and will be largely outsourced, allowing the company to focus on the development of its two lead cancer vaccines; OVM-100, an HPV vaccine targeted at cervical cancer; and OVM-200 a new type of vaccine based on survivin that targets solid tumours. Both vaccines address areas of significant unmet medical need and will be tested as single agents and also in combination with other immune-oncology (IO) agents.

Dr Shisong Jiang, CSO and Founder of OVM, said:

"We are very pleased to have this opportunity to work with Imperial College and with the Ditan hospital in Beijing on this important project. This novel application of our recombinant overlapping peptide technology could show real utility in the diagnosis and management of carbapenem resistance and could make a significant difference to the effectiveness of treatment for infectious disease. We very much appreciate the support and funding from Innovate UK for this project"

Professor Xu Xiao-Ning, Chair of Human Immunology at the Imperial College London added:

"This project is an excellent opportunity to widen the applications of the Oxford Vacmedix’s ROP technology and to build on the expertise in immunology and infectious disease at Imperial College. We look forward to a successful collaboration and to developing early diagnosis of carbapenem resistance, to address this real need and benefit a wide population with sometimes limited access to healthcare".

On December 19, 2019 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR, reported that it has entered into definitive agreements to sell securities in a private placement with a group of institutional investors and certain members of the Company’s Board of Directors (Press release, Selecta Biosciences, DEC 19, 2019, View Source [SID1234552494]). The lead institutional investors in the private placement were Vivo Capital LLC, Mangrove Partners, EcoR1 Capital LLC, BVF Partners L.P., and Boxer Capital LLC. The transaction is expected to result in gross proceeds to the company of approximately $70 million, before deducting placement agent fees and other offering expenses.

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 Company plans to use the net proceeds from the financing primarily to fund the ongoing head-to-head Phase 2 COMPARE clinical trial of its lead product candidate, SEL-212, for the treatment of chronic refractory gout, its gene therapy programs, and for general corporate purposes.

Pursuant to the terms of the securities purchase agreement, the Company will issue 45,977,011 units at a price of approximately $1.5225 per unit. Each unit consists of approximately one share of common stock and a warrant to purchase 0.5 of a share of common stock at an exercise price of $1.46. Certain institutional investors have elected to receive pre-funded warrants to purchase common stock in lieu of a portion of their common stock. The closing of the offering is subject to certain conditions and is expected to occur on Monday, December 23, 2019.

Cantor Fitzgerald & Co. served as placement agent for the offering.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws, and will be sold in a private placement pursuant to Regulation D of the Securities Act. The securities being issued in the private placement may not be offered or sold in the United States absent registration or pursuant to an exemption from the registration requirements of the Securities Act and applicable state securities laws. The Company has agreed to file a registration statement covering the resale of the securities acquired by the investors in the private placement.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On December 19, 2019 Bridge Biotherapeutics Inc., a clinical stage biotech company headquartered in Seongnam, South Korea, reported that the company filed an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) and the Ministry of Food and Drug Safety (MFDS) in Korea to initiate phase I/II study of BBT-176, a clinical candidate of targeted lung cancer therapy (Press release, BridgeBio, DEC 19, 2019, View Source [SID1234552532]).

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!

BBT-176, a novel epidermal growth factor receptor – tyrosine kinase inhibitor (EGFR-TKI) is designed to inhibit EGFR with C797S mutations, which arise as Tagrisso (osimertinib) resistant mutations following Tagrisso treatment in non-small cell lung cancer (NSCLC). BBT-176 exhibited strong antitumor efficacy in xenograft models harboring C797S triple mutations including Del19/T790M/C797S and L858R/T790M/C797S. Furthermore, BBT-176 displayed markedly enhanced efficacy when combined with anti-EGFR antibodies.

Since the EGFR C797S mutation was reported 3 years ago, as the first evidence of Tagrisso resistance, no major breakthroughs have been achieved to target the clinically relevant mutant variant that impedes covalent bond formation with irreversible EGFR inhibitors.[i]

The company plans to initiate dose escalation studies in advanced NSCLC patients in Korea next year and to develop further clinical studies in both Korea and the US afterwards. In the Phase I/II study with NSCLC patients, the safety, tolerability and efficacy of the candidate will be observed.

"We are proud of the IND submission for BBT-176, which has shown a potential to be developed as a highly mutant-selective, fourth-generation EGFR-TKI for NSCLC treatment," stated James Lee, CEO of Bridge Biotherapeutics. "Our team will make our best effort to develop novel targeted lung cancer therapy inhibiting C797S EGFR mutation."

BBT-176 was discovered by Korea Research Institute of Chemical Technology (KRICT), a Korean government research institute, and was licensed to Bridge Biotherapeutics in December 2018 for the worldwide exclusive right for further development.

Lung cancer is the leading cause of cancer death, accounting for about one-fifth of all cancer deaths. It is split into NSCLC and small cell lung cancer (SCLC) and NSCLC accounts for approximately 85% of all lung cancers. Overall, across 8 major countries including the U.S., 5 EU countries, China and Japan, the total NSCLC population as of 2015 is assumed 2 million and the incidence of NSCLC is expected to increase at an annual growth rate of 3.1% from 2015 to 2025.[ii]