Y-mAbs Submits Omburtamab Marketing Authorization Application to the European Medicines Agency

On April 27, 2021 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that the Company has submitted its Marketing Authorization Application ("MAA") to the European Medicines Agency for omburtamab for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma (Press release, Y-mAbs Therapeutics, APR 27, 2021, View Source [SID1234578560]). Omburtamab is an investigational, monoclonal antibody that targets B7-H3 and is radiolabeled before intraventricular central nervous system ("CNS") administration. B7-H3 is an immune checkpoint molecule that is widely expressed in tumor cells of several cancer types.

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"We believe omburtamab is on track to potentially become the first EMA approved targeted therapy for pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma," said Thomas Gad, founder, Chairman and President at Y-mAbs. "With this submission to EMA, Y-mAbs is continuing to advance the omburtamab program to potentially provide access to this innovative therapy to pediatric patients globally as quickly as possible."

In addition, Y-mAbs recently concluded a Type B meeting with the U.S. Food and Drug Administration ("FDA") regarding omburtamab and the Company continues to be in close dialog with the FDA and maintains its aim of resubmitting the Biologics License Application ("BLA") to the FDA late in the second quarter or in the third quarter of 2021.

Dr. Claus Moller, the Company’s Chief Executive Officer, continued, "We are excited to submit the MAA for omburtamab, and very pleased about the progress we are making. We believe omburtamab can potentially address a significant unmet medical need for children with CNS/leptomeningeal metastasis from neuroblastoma. We also are continuing to work closely with the FDA to resubmit the omburtamab BLA."

Researchers at Memorial Sloan Kettering Cancer Center ("MSK") developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interest related to the compound and Y-mAbs.

Transgene reports business update and end Q1 2021 financial position

On April 27, 2021 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its business update for the quarter ending March 31, 2021, and provides an update on the progress of its portfolio of clinical trials (Press release, Transgene, APR 27, 2021, View Source [SID1234578576]).

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Since January 2021, Transgene has achieved significant milestones on all candidates of its clinical portfolio, with:

ü the first patients dosed with TG4050, the individualized therapeutic vaccine against cancer based on Transgene’s myvac technology – the first data from the two ongoing Phase I clinical trials are expected in 4Q 2021;

ü regulatory clearance received in the United States, in France and in Spain, which are expected to allow enrolment of patients with HPV-positive anogenital cancers in the randomized Phase II trial of TG4001 + avelumab versus avelumab alone in 2Q 2021;

ü first patients dosed in the Phase I trial evaluating BT-001, an oncolytic virus based on the Invir.IO platform – first data are expected in 1H 2022;

ü TG6002 initial data, presented at AACR (Free AACR Whitepaper), providing the clinical proof of concept of the intravenous administration of an oncolytic virus. After intravenous administration, TG6002 reached the tumor, multiplied within tumor cells, and induced the local expression of its payload (the FCU1 gene). These data also suggest that candidates derived from Transgene’s unique Invir.IO platform could also be given intravenously, extending the use of these therapies to a broad range of solid tumors – Next data with TG6002 are expected in 2H 2021 (intra-hepatic artery route).

SUMMARY OF KEY ONGOING CLINICAL TRIALS
AND EXPECTED MILESTONES

myvac

TG4050

Phase I

NCT03839524

Targets: tumor neoantigens

Ovarian cancer – after surgery and first-line chemotherapy

ü Trial ongoing in the United States and in France

ü Inclusions and patient dosing progressing in line with forecast

Ü First data expected in 4Q 2021

myvac

TG4050

Phase I

NCT04183166

HPV-negative head and neck cancer – after surgery and adjuvant therapy

ü Trial ongoing in the United Kingdom and in France

ü First patient treated in Jan. 2021 – Inclusions and patient dosing progressing in line with forecast

Ü First data expected in 4Q 2021

TG4001

+ avelumab
Phase II

NCT03260023

Targets: HPV16 E6 and E7 oncoproteins

Recurrent/metastatic anogenital HPV-positive – 1st and 2nd line

ü A Phase II randomized trial comparing the efficacy of TG4001 + avelumab versus avelumab single-agent benefits from the extended clinical collaboration with Merck KGaA and Pfizer, for the supply of avelumab

ü Regulatory authorizations received in the Unites States, Spain, and France

Ü Patient enrollment in the randomized trial expected to start in 2Q 2021

Ü First data from the randomized trial are expected around the end of 2022. This timeline is based on patient enrollment starting in 2Q 2021 and there being no major impact on recruitment from the Covid-19 pandemic.

Invir.IO

BT-001

Phase I/IIa

NCT04725331

Payload: anti-CTLA4 antibody and GM-CSF cytokine

Solid tumors

ü Co-development with BioInvent

ü Trial ongoing in France and Belgium

ü First patient enrolled in February 2021 – Inclusions and patient dosing progressing in line with forecast

Ü US IND expected in 2021

Ü First Phase I data expected in 1H 2022

TG6002

Phase I/IIa

NCT03724071

Payload: FCU1 for the local production of a 5-FU chemotherapy

Gastro-intestinal cancer (colorectal cancer for Phase II) – Intravenous (IV) administration

ü Multicenter trial ongoing in Belgium, France and Spain

ü Poster presentation at AACR (Free AACR Whitepaper) 2021 on initial data of the trial, demonstrating the clinical proof of concept of the intravenous route of administration

Ü Phase I part ongoing

TG6002

Phase I/IIa

NCT04194034

Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration

ü Multicenter trial ongoing in the United Kingdom

Ü First observations expected in 2H 2021

During the first quarter of 2021, revenue from collaborative and licensing agreements was mainly composed of the revenue from the collaboration with AstraZeneca.

As of March 31, 2021, government financing for research expenditures mainly consisted of 25% of the research tax credit expected for 2021 (€1.5 million in the first quarter of 2021, in line with the same period in 2020).

CASH, CASH EQUIVALENTS

AND OTHER FINANCIAL ASSETS

Cash, cash equivalents and other financial assets stood at €19.1 million as of March 31, 2021, compared to €26.3 million as of December 31, 2020. In the first quarter of 2021, Transgene’s cash burn was €7.2 million, compared to €8.0 million for the same period in 2020.

In addition, Transgene has access to a €15 million credit line available from Natixis, the maturity date of which has been extended to April 15, 2023. The Company holds shares of Tasly BioPharmaceuticals valued at €32.3 million at the end of December 2020.

As a result, the Company has a financial visibility until the second half of 2022.

Alligator Bioscience AB: Interim report January-March 2021

On April 27, 2021 Alligator Bioscience reported that Interim report January-March 2021 (Press release, Alligator Bioscience, APR 27, 2021, View Source [SID1234578593])

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High activity during the first quarter

"So far, 2021 has been characterized by high activity, with a research collaboration that validates our Discovery concept, and good progress of our clinical programs, with ATOR-1017 and mitazalimab entering clinical efficacy studies this year."

Malin Carlsson, Interim CEO Alligator Bioscience AB

SIGNIFICANT EVENTS JANUARY-MARCH

Pipeline:

New preclinical data were released in an abstract for a poster presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2021, demonstrating that mitazalimab synergizes with chemotherapy.
Company:
Søren Bregenholt was appointed as new CEO to strengthen Alligator’s business development activities and clinical progress on an international level.
Oversubscribed rights issue generated proceeds of SEK 86 million before transaction costs.
SIGNIFICANT EVENTS AFTER THE END OF THE PERIOD

Alligator and the US biopharmaceutical company MacroGenics entered into a joint research collaboration to develop Neo-X-PrimeTM, next generation immune oncology therapy building on Alligator’s CD40 expertise.
The Annual General meeting was postponed to June 1, 2021.
The Nomination Committee proposed that the Annual General Meeting 2021 re-elects Anders Ekblom and Graham Dixon as board members, and that Hans-Peter Ostler, Eva Sjökvist Saers and Veronica Wallin are elected as new board members.
FINANCIAL SUMMARY

January–March 2021

Net sales, SEK 0.6 million (0)
Operating result, SEK -32.5 million (-44.9)
Result for the period, SEK -32.7 million (-42.9)
Earnings per share before and after dilution, SEK -0.38 (-0.60)
Cash flow for the period, SEK 40.4 million (108.4)
Cash and cash equivalents, incl. interest-bearing securities, SEK 143.7 million (103.3)

IGM Biosciences to Present at the Truist Securities Life Sciences Summit

On April 27, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that Fred Schwarzer, Chief Executive Officer, will participate in a fireside chat at the Truist Securities Life Sciences Summit on May 4, 2021 at 4:20 p.m. EDT (Press release, IGM Biosciences, APR 27, 2021, View Source [SID1234578545]). The conference will be held in a virtual meeting format.

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A live webcast of the event will be available on the "Events and 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 90 days following the presentation.

Fate Therapeutics Announces Four Presentations at the 2021 ASGCT Annual Meeting

On April 27, 2021 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported that two oral and two digital presentations of the Company’s induced pluripotent stem cell (iPSC) product platform were accepted for presentation at the 24th American Society of Gene & Cell Therapy Annual Meeting (ASGCT) (Free ASGCT Whitepaper) being held virtually from May 11-14, 2021 (Press release, Fate Therapeutics, APR 27, 2021, View Source [SID1234578561]).

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In addition to the Company’s presentations at ASGCT (Free ASGCT Whitepaper), its iPSC-derived natural killer (NK) cell product pipeline is expected to be featured in a meeting symposium on May 11 by Jeffrey S. Miller, M.D., Professor of Medicine, University of Minnesota and Deputy Director of the Masonic Cancer Center and scientific advisor and collaborator of the Company, and its iPSC-derived CAR T-cell product platform is expected to be highlighted during the meeting’s plenary session on May 12 by Michel Sadelain, M.D., Ph.D., Stephen and Barbara Friedman Chair and Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center and collaborator of the Company.

The Company also plans to host a virtual investor event on May 13 to highlight interim Phase 1 clinical data from its FT516 and FT538 programs for the treatment of relapsed / refractory acute myeloid leukemia (AML). The Phase 1 clinical trial of FT516 has enrolled the first and second dose cohorts (90 million and 300 million cells per dose, respectively), and dose escalation is ongoing in the third dose cohort (900 million cells per dose). The Phase 1 clinical trial of FT538 is ongoing in the first dose cohort (100 million cells per dose).

ASGCT Oral Presentations

Sequential CRISPR-mediated Engineering and Clonal Banking for the Generation of Multiplexed Engineered Master Pluripotent Cell Lines for the Mass Manufacture of Off-the-Shelf Immune Cells Targeting Solid Cancers
Room 9 – Advances in Ex Vivo Modified Cell Therapies; Abstract 5; May 11, 2021; 6:30pm – 6:45pm EDT
Enhanced Generation of T-cell Derived Naïve Pluripotent Cells as a Renewable Cell Source for the Mass Manufacture of Off-the-shelf CAR T-cell Therapies
Room 5 – CAR Modified Cellular Therapies; Abstract 76; May 12, 2021; 6:45pm – 7:00pm EDT
ASGCT Digital Presentations

Development and Application of a Pluripotent Stem Cell Platform to Generate Precision-Engineered Single Cell-derived Renewable Clonal Master Cell Lines for Therapeutic Use
Abstract # 793
Temporal Gene Regulation of T-cell Enhancers by Locus Targeted Engineering Enables Cytokine Autonomy and Augments Anti-tumor Efficacy of iPSC-derived Off-the-shelf CAR-T Therapy
Abstract #784
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT516
FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in a multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in a multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About FT538
FT538 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components: a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; an IL-15 receptor fusion (IL-15RF) that augments NK cell activity; and the deletion of the CD38 gene (CD38KO), which promotes persistence and function in high oxidative stress environments. FT538 is designed to enhance innate immunity in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms. In preclinical studies, FT538 has shown superior NK cell effector function, as compared to peripheral blood NK cells, with the potential to confer significant anti-tumor activity to patients through multiple mechanisms of action. FT538 is being investigated in a multi-dose Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-targeted monoclonal antibody therapy, for the treatment of multiple myeloma (NCT04614636).