On November 20, 2020 CARsgen Therapeutics, a clinical-stage biopharmaceutical company, reported that data from its global multiple myeloma program will be presented, including two oral presentations and one poster session, at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place virtually December 5-8, 2020 (Press release, Carsgen Therapeutics, NOV 20, 2020, View Source [SID1234571494]).

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Presentations will include updated safety and efficacy results from investigator-initiated clinical studies of CT053 and preliminary data from the ongoing Phase 1 and 1b/2 clinical studies of CT053 in China (LUMMICAR STUDY 1) and the United States (LUMMICAR STUDY 2). CT053 is an investigational anti-BCMA (B cell maturation antigen) autologous chimeric antigen receptor (CAR) T-cell product for the treatment of adult patients with relapsed and/or refractory multiple myeloma.

Results from LUMMICAR-1: A Phase 1 Study of Fully Human B-Cell Maturation Antigen-Specific CAR T Cells (CT053) in Chinese Subjects with Relapsed and/or Refractory Multiple Myeloma
Presenting Author: Wenming Chen, MD, PhD, Beijing Chao-Yang Hospital of Capital Medical University, Beijing, China
Date/Time: Poster #1396, Saturday, December 5, 2020: 7:00 AM-3:30 PM PST

Results from LUMMICAR-2: A Phase 1b/2 Study of Fully Human B-Cell Maturation Antigen-Specific CAR T Cells (CT053) in Patients with Relapsed and/or Refractory Multiple Myeloma
Presenting Author: Shaji K. Kumar, MD, Division of Hematology, Mayo Clinic, Rochester, MN
Date/Time: Oral #133, Saturday, December 5, 2020: 9:30 AM-11:00 AM PST

Two-Year Follow-up of Investigator-Initiated Phase 1 Trials of the Safety and Efficacy of Fully Human Anti-BCMA CAR T Cells (CT053) in Relapsed/Refractory Multiple Myeloma
Presenting Author: Siguo Hao, MD, PhD, Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine, Shanghai, China
Date/Time: Oral #132, Saturday, December 5, 2020: 9:30 AM-11:00 AM PST

About CT053 and LUMMICAR

CT053 is a CAR T-cell therapy that targets B-cell maturation antigen (BCMA), a protein expressed on the surface of malignant and normal plasma cells. The CT053 construct utilizes a fully human anti-BCMA scFv domain, hypothesized to reduce immunogenicity and improve safety. CT053 T cells are proposed to recognize, bind, and eradicate multiple myeloma cells that express BCMA.

CT053 has received regenerative medicine advanced therapy (RMAT) and orphan drug designations from the U.S. Food and Drug Administration and PRIority MEdicines (PRIME) and orphan drug designations from the European Medicines Agency.

CARsgen Therapeutics’ clinical development program for CT053 includes the clinical studies: LUMMICAR STUDY 1 (NCT03975907 Phase 1, China) and LUMMICAR STUDY 2 (NCT03915184 Phase 1b/2, United States and Canada). These are open-label, multicenter studies evaluating the safety and efficacy of CT053 in adult patients with relapsed and/or refractory multiple myeloma. CT053 studies also include three investigator-initiated trials (NCT03380039; NCT03716856; NCT03302403). For more information, visit clinicaltrials.gov.

On November 20, 2020 The Spanish government has announced the public grants corresponding to the State Program for research, development and innovation call — geared towards societal challenges and called "Collaboration Challenges of the year 2019" (Press release, Laminar Pharma, NOV 20, 2020, View Source [SID1234572575]). These are instruments to help meet the needs of the Spanish Science, Technology and Innovation System and its main agents.

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One of the approved proposals has been the project GLIOPROG561 (RTC2019-007399-1) led by our company in collaboration with the CIBIR- Biomedical Research Center of La Rioja (Fundación Rioja Salud) and the University of the Balearic Islands (UIB), whose objective is the "Identification and validation of prognosis response biomarkers in treatment with the LAM561 molecule in patients with glioma and evaluation of the pharmacological activity of its main metabolite".

This is an interdisciplinary project in which lipid chemistry, proteomics, bioinformatics, genomics and molecular biology are combined to maximize the chances of finding effective biomarkers. The participation of the CIBIR in this consortium guarantees — thanks to their extensive experience — high quality bioinformatics analyses and great reliability in the validation of the identified biomarkers.

This project answers one of the call’s objectives, which is the consolidation of R&D and innovation capabilities as well as the generation of knowledge and research teams that carry out their activities in universities, public research organizations and other research organizations. In total, the grant amounts to 1,058,759 euros from the budget of the Ministry of Science and that of the State Research Agency for 2020.

The financed projects all have a duration of several years: they will have begun their execution throughout the present year 2020 and will end on the date indicated in the grant request, between mid and late 2023. The public Challenges-Collaboration grants correspond to the State Program for Generation of Knowledge and Scientific and Technological Strengthening of the R&D and Innovation System, within the framework of the State Program for R&D and Innovation Geared towards the Challenges of Society, belonging to the State Plan for Scientific and Technical Research and Innovation 2017-2020.

The aforementioned State Plan is the main instrument of the General State Administration for the development and achievement of the objectives of the Spanish Strategy for Science and Technology and Innovation 2013-2020 and the European 2020 Strategy. It includes state grants for R&D and innovation, which are preferably awarded through competitive calls. These projects are evaluated by experts from the fields of basic science, clinical science, economics and finance.

On November 20, 2020 Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage company focused on pioneering the development of exosome-based therapeutics, reported third quarter 2020 financial results and operational progress (Press release, Codiak Biosciences, NOV 20, 2020, View Source [SID1234571473]).

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"Codiak was founded to realize the potential of engineered exosomes as a new and important class of biologic medicines for the treatment of a broad range of diseases," said Douglas E. Williams, Ph.D., President and Chief Executive Officer of Codiak. "The creativity, expertise, and execution of our team has enabled us to bring the field’s first two engineered exosome candidates into the clinic and we are on track to initiate a third clinical program next year. The recent successful completion of our initial public offering provides additional funding to progress our development programs, invest in our platform and sustain the positive momentum we have built."

Third Quarter 2020 and Recent Highlights

Closed initial public offering (IPO) in October 2020, raising $74.4 million in net proceeds
Initiated healthy volunteer dosing of exoIL-12 in a Phase 1 clinical trial designed to transition into patients with cutaneous T cell lymphoma (CTCL)
Initiated subject dosing in a Phase 1/2 clinical trial of exoSTING for the treatment of advanced/metastatic, recurrent and injectable solid tumors
Continued to advance exoASO-STAT6 for the intravenous treatment of myeloid-rich cancers through IND-enabling studies
Presented preclinical data on exoIL-12 and the engEx Platform at the 35th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
Expanded executive team with addition of Yalonda Howze as Executive Vice President, Chief Legal Officer
Welcomed Jason Haddock to the Board of Directors and as Audit Committee Chairman
Anticipated Milestones and Events

Initial safety, tolerability and systemic exposure/pharmacokinetics data from the healthy volunteer portion of the exoIL-12 Phase 1 clinical trial are expected by year-end, with biomarker, safety and preliminary pharmacodynamics and efficacy data in CTCL patients expected by mid-2021
Safety and preliminary pharmacodynamics and efficacy data from exoSTING Phase 1/2 clinical trial in patients with solid tumors expected by mid-2021
Evercore ISI HEALTHCONx Virtual Conference, December 2, 2020
Third Quarter 2020 Financial Results
Total revenues for the quarter ended September 30, 2020 were $1.0 million, compared to $0.2 million for the same period in 2019. This increase was primarily due to an increase in collaboration revenue driven primarily by activities in connection with the collaboration with Sarepta.

Net loss for the quarter ended September 30, 2020 was $35.3 million, compared to a net loss of $20.7 million for the same period in 2019. Net loss for the quarter ended September 30, 2020 was driven primarily by clinical development, general and administrative, and personnel expenses, and ongoing development of the engEx Platform. The current quarter net loss was inclusive of a $15.0 million milestone payment to Kayla Therapeutics in connection with dosing of the first patient in the Phase 1/2 clinical trial of exoSTING.

Research and development expenses were $30.7 million for the quarter ended September 30, 2020 compared to $16.5 million for the same period in 2019. The increase was primarily driven by an increase in license milestones, personnel costs, and clinical development expenses related to the initiation of the exo-IL12 and exoSTING clinical trials in September 2020.

General and administrative expenses were $5.3 million for the quarter ended September 30, 2020 compared to $4.8 million for the same period in 2019. The increase was primarily driven by an increase in consulting and legal fees, personnel costs, and other administrative expenses in anticipation of operating as a public company.

As of September 30, 2020, Codiak had cash, cash equivalents, and marketable securities of $48.3 million. In October 2020, the completion of the IPO resulted in net proceeds of $74.4 million.

On November 20, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Evercore ISI 3rd Annual HealthCONx on Wednesday, December 2, 2020 at 12:35 p.m. ET and at the Piper Sandler 32nd Annual Healthcare Conference (Press release, BioCryst Pharmaceuticals, NOV 20, 2020, https://biocryst.gcs-web.com/news-releases/news-release-details/biocryst-present-upcoming-virtual-investment-conferences [SID1234571475]). Both conferences are being conducted as virtual events.

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Piper Sandler is pre-recording company presentations at the conference and the pre-recorded presentation may be accessed on Monday, November 23, 2020 in the Investors section of BioCryst’s website at http://www.biocryst.com.

Links to a live audio webcast and replay of the presentation at the Evercore ISI conference may be accessed on December 2, 2020 in the Investors section of BioCryst’s website at http://www.biocryst.com.

On November 20, 2020 Bicycle Therapeutics plc (NASDAQ:BCYC), a clinical-stage biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that it has entered into a settlement and license agreement with Pepscan Systems B.V. regarding Bicycle’s use of Pepscan’s CLIPS peptide technology (Press release, Bicycle Therapeutics, NOV 20, 2020, View Source [SID1234571517]).

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The companies have agreed to settle all intellectual property disputes worldwide. Under the terms of the settlement, Bicycle has been granted a license to use CLIPS peptide technology in the development of its product candidates BT1718 and THR-149. Bicycle will pay €3 million upfront, will pay €1 million on the first anniversary of the date of settlement, and will make potential additional payments to Pepscan based on achievement of specified clinical, regulatory and commercial milestones.