On May 11, 2021 Medigene AG (Medigene, FSE: MDG1, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies, reported an update on the first quarter of 2021 and confirms its financial guidance for the full year (Press release, MediGene, MAY 11, 2021, View Source [SID1234579693]). The full version of the Quarterly Statement Q1 2021 can be downloaded here: View Source

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Prof. Dolores Schendel, Chief Executive Officer and Chief Scientific Officer at Medigene: "Medigene’s development projects continued to progress well in the early part of 2021 as demonstrated by the increased tempo of presentations at international conferences and by the peer-reviewed publication of our work on the MAGE-A4-specific TCR in collaboration with bluebird bio.
While we anticipate reporting results later in 2021 for part 1 of our Phase I/II clinical trial of MDG1011 in blood cancer, we are pushing forward in our developments targeting solid tumors where we have achieved impressive preclinical results on multiple projects including the selection of optimal T cell receptors (TCRs), unique new tumor-specific antigens, and functional enhancements for T cell receptor-modified T cells (TCR-Ts) such as the PD1-41BB switch receptor to conquer the hostile tumor environment, among others.
To further enhance Medigene’s value, we continue to evaluate new partnering opportunities related to our suite of TCR technologies and portfolio of product candidates."

Business review since beginning of 2021 and outlook
In advancing its activities towards solid tumor indications, Medigene continues the research and development work on its suite of technologies and product candidates:

* TCR-4, Medigene’s lead TCR candidate against solid tumors, is a non-mutated TCR isolated using Medigene’s high-throughput screening platform that specifically recognizes a peptide stemming from the PRAME protein presented by human leukocyte antigen (HLA) A2. PRAME is expressed by a variety of solid cancer types. TCR-4 is highly sensitive for this PRAME epitope and its activity has been demonstrated both in vitro (against a variety of tumor cell types including lung cancer, uterine carcinoma, melanoma, and ovarian cancer, among others) and in an in vivo model against melanoma.

* The PD1-41BB switch receptor is the most advanced of the TCR-T enhancements currently being developed by Medigene. A key mechanism in the ability of solid tumors to evade attack by T cells includes the expression of the checkpoint molecule PD-L1 on the surface of tumor cells. Medigene’s PD1-41BB switch receptor turns the tumors’ off-signal sent by PD-L1 into an activation signal for the TCR-T cells. As shown at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) and the Association for Cancer Immunotherapy (CIMT) (Free CIMT Whitepaper) virtual meetings in March and May 2021, respectively, adding the PD1-41BB switch receptor improved the function of TCR-T cells in a challenging tumor environment over repeated cycles. The PD1-41BB switch receptor also enhanced the metabolic fitness of TCR-T cells and their antigen target-specific killing of tumor cells.

* Under the collaboration with the University of Montréal, Medigene gained access to 47 potential new tumor-specific antigens (TSAs) which were found to be shared among specimens of several patients with solid tumors of different origin, such as ovarian, breast, and lung cancer. As presented at the AACR (Free AACR Whitepaper) virtual meeting, Medigene’s high-throughput screening technology identified ten peptides as immunogenic and able to induce specific T cell responses. The sequences of more than 20 TCRs recognizing these novel TSAs were determined and additional characterization was presented recently at the CIMT (Free CIMT Whitepaper) virtual meeting. With ongoing work these TCRs have the potential to become next-generation TCR-T therapy candidates.

* Medigene continues to enroll and treat patients in the Phase I/II clinical trial of MDG1011 against blood cancers. Dosing of the first three dose cohorts of the Phase I safety and feasibility part of the trial is expected to be completed by the end of Q2 2021. In line with Medigene’s focus on solid cancers the Company has decided that, contingent on the results from the Phase I part, the Phase II part of the trial would only be conducted with or by a partner.

* Consistent with the company’s decision to focus its development efforts on solid cancers, the MDG1021 development program was discontinued in January 2021. All rights to the HA-1-specific TCR, as well as sponsorship of the Phase I clinical trial, are being transferred to a third party.

* Medigene has developed a new generation of dendritic cell (DC) vaccines. The positive results of the completed open-label Phase I/II clinical trial in patients suffering from acute myeloid leukemia were confirmed even after more than 3.5 years of median follow-up, as reported in February 2021. Consistent with Medigene’s development focus on TCR-T therapies, the DC vaccine project will only be continued with partners.

* Medigene continues its successful collaborations with bluebird bio, Inc. (bluebird bio) and Cytovant Sciences HK Limited, a biopharmaceutical company founded by Roivant Sciences (Roivant/Cytovant). To maximize the Company’s value, it continues to evaluate new partnering opportunities related to its suite of TCR technologies and portfolio of product candidates.

Financial development and financial forecast
* As of 31 March 2021, cash and cash equivalents and fixed-term deposits amounted to Eur26.482 m (31 December 2020: Eur30.033 m). The decrease is primarily due to research and development expenses to advance Medigene’s clinical and preclinical activities.

* Total revenue in the first quarter 2021 increased to Eur2.153 m from Eur1.403 m in the first quarter 2020 due to higher revenues resulting from research and development services from strategic partnerships. Revenue in the first quarter of 2021 also includes revenue from the derecognition of contract liabilities.

* Research and development expenses of Eur4.012 m were Eur2.126 m lower than in the prior-year quarter (Q1 2020: Eur6.138 m) which is mainly due to the increased focus on certain TCR-T therapies for the treatment of solid tumors.

* As a result, earnings before interest, taxes, depreciation and amortization (EBITDA) increased by Eur2.871 m on the prior-year quarter (Q1 2020: Eur-5.949 m), amounting to Eur-3.078 m in the first quarter 2021.
Medigene confirms its financial forecast for 2021 published in the Group Management’s Discussion and Analysis 2020. The Company continues to expect total revenue of Eur7 – 9 m in 2021, research and development expenses of Eur14 – 20 m, and an EBITDA loss of Eur10 – 17 m.
Currently, Medigene expects no material influence of the COVID-19 pandemic on total revenue, research and development expenses and loss at EBITDA level. Based on current planning, the Company has sufficient financial resources to fund operations into the third quarter of 2022.

Conference call
Medigene will not hold a telephone conference regarding the Quarterly Statement Q1 2021 but we remain available in the usual way for all enquiries.

On May 11, 2021 Australian clinical-stage drug development company Noxopharm Limited (ASX:NOX) reported its CEP-2 study will begin shortly. CEP-2 is related to the Investigational New Drug (IND) approval of Veyonda by the FDA based on evidence that Veyonda may increase generally poor response rates of sarcoma cancers to chemotherapy in combination with common chemotherapy drug, doxorubicin, for patients with soft tissue sarcomas (Press release, Noxopharm, MAY 11, 2021, View Source [SID1234579709]). The announcement marks the Company’s commitment to act on this valuable opportunity with the appointment of a contract research organization to oversee the study.

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Sarcoma was deliberately selected as a rare cancer carrying important funding and regulatory approval benefits including seven-year exclusive marketing opportunities. Sarcomas have very limited treatment options with only an estimated 14% of soft tissue sarcomas responding to chemotherapy. The Company is confident that Veyonda, with its unique mix of immunotherapy actions, has the means to increase both survival and response rates significantly when combined with doxorubicin.

"Soft tissue sarcomas are in that basket of cancers where there has been remarkably little advance in survival statistics over the past few decades, and the horizon has appeared to hold little reason to think that would change," said Graham Kelly, CEO of Noxopharm. "What we have seen to date with Veyonda both pre-clinically and clinically gives us confidence that a combination of Veyonda and doxorubicin has the capacity to deliver that change."

On May 11, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that it will give multiple oral and poster presentations at the American Society of Gene and Cell Therapy 2021 Virtual Annual Meeting being held May 11-14, 2021 (Press release, Poseida Therapeutics, MAY 11, 2021, View Source [SID1234579726]).

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The Company’s oral presentation will highlight new data demonstrating the potential of its proprietary piggyBac DNA Delivery System for the treatment of genetic liver disorders in children and infants. Two additional presentations will highlight preclinical data supporting Poseida’s first allogeneic CAR-T product candidate, P-BCMA-ALLO1 for R/R multiple myeloma, as well as preclinical data supporting the Company’s anti-c-kit CAR-T program as a potentially safer preconditioning regimen for hematopoietic stem cell transplantation in patients with AML.

"At Poseida, we are applying our proprietary technology platforms to develop the next wave of cell and gene therapies to not only treat severe cancers but to also unlock the potential of single treatment cures," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer. "The preclinical data being presented today further validate our CAR-T approach in multiple myeloma and in preconditioning for patients with AML, as well as demonstrate the exciting potential of our liver directed gene therapies, particularly in juvenile patients."

Presentation Highlights:

Oral Presentation: "Preclinical Evaluation of Combined Adeno-Associated Virus and Nanoparticle Delivery of piggyBac Transposon System for Durable Transgene Expression in the Growing Neonatal Murine Liver"
Session Date/Time: Tuesday, May 11, 2021, 5:45pm – 6:00pm ET
Abstract Number: 30

In a preclinical study, Poseida evaluated concomitant delivery of recombinant adeno-associated virus (rAAV) vectors and novel nanoparticle (NP) vectors using its piggyBac and "Super" piggyBac (SPB) technologies in order to deliver transposon and transposase in a growing neonatal mouse model. Data demonstrated that the piggyBac DNA Delivery System was effective in using both rAAV and NP vectors to introduce edited genes into targeted hepatocyte genomes. Poseida also found that SPB, a hyperactive form of the transposase, produced stable vector integration into the hepatocyte genome for more than three months, compared to transpose alone. Similarly, delivery of a novel NP formulation using SPB produced efficient delivery of mRNA to the liver hepatocytes, with similarly high levels of durability in the transgene expression. Taken together, these preclinical findings suggest the potential of piggyBac and SPB technology for gene therapies that treat congenital liver disease in infants and young children.

Poster Presentation: "P-BCMA-ALL01: A Fully Allogeneic Stem Cell Memory T Cell (TSCM) CAR-T Therapy Targeting BCMA for the Treatment of Multiple Myeloma Shows Potent Anti-Tumor Activity"
Session Date/Time: Tuesday, May 11, 2021, 8:00am – 10:00am ET
Abstract Number: 789

P-BCMA-ALLO1 is Poseida’s first fully allogenic product candidate targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. In in vitro and in vivo preclinical studies, P-BCMA-ALL01 showed effective, targeted cancer cell killing and cytokine secretion, with similar or superior performance in anti-tumor efficacy compared to an autologous CAR-T therapy. Inclusion of a proprietary "booster molecule" in the allogeneic manufacturing process further improved expansion of gene-edited cells and enabled production of hundreds of patient doses from a single manufacturing run, thereby reducing the manufacturing cost per dose into the same range as that of a monoclonal antibody.

Poster Presentation: "Anti-c-kit CAR-T Cells Afford Effective Eradication of Human AML and Normal Hematopoietic Cells in a Preclinical Model of Safer Non-Genotoxic Stem Cell Transplant Conditioning"
Session Date/Time: Tuesday, May 11, 2021, 8:00am – 10:00am ET
Abstract Number: 715

Poseida is investigating its anti-c-kit CAR-T program, which leverages its proprietary piggyBac DNA Delivery System in preclinical studies as a potentially safer precursor conditioning therapy to the transplantation of hematopoietic stem cells (HSC) for patients suffering from AML. The piggyBac delivery vectors under investigation include a transposon that generates pure CAR+ product as well as a safety switch that allows rapid clearance of the reactive CAR-T cells prior to donor transplant of hematopoietic stem cells. Preclinical data to be presented in the poster showed that the lead CAR-T cells that express the anti-c-kit binder (CAR 1) deplete up to 92% of human CD34+ stem and progenitor cells in bone marrow within 48 hours. Additionally, an enhanced anti-c-kit CAR-T product, CAR 2, killed an estimated >99% of leukemia cells, exceeding the killing ability of a single dose of 30 mg/kg dose of busulfan. These encouraging data suggest that stem cell-directed CAR-T cells may be a safer preconditioning regimen compared to the current standard of care and may expand access to treatment for acute myeloid leukemia patients needing HSC transplant.

On May 11, 2021 Genmab A/S (Nasdaq: GMAB) reported that it will increase its share capital by 33,418 shares as a consequence of the exercise of employee warrants (Press release, Genmab, MAY 11, 2021, View Source [SID1234579663]).

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The increase is effected without any preemption rights for the existing shareholders of the company or others. The shares are subscribed in cash at the following price per share of nominally DKK 1:

2,600 shares at DKK 40.41,
1,440 shares at DKK 225.30,
8,750 shares at DKK 337.40,
1,375 shares at DKK 636.50,
400 shares at DKK 815.50,
6,050 shares at DKK 939.50,
4,594 shares at DKK 1,032.00,
1,317 shares at DKK 1,145.00,
2,865 shares at DKK 1,210.00,
1,000 shares at DKK 1,233.00, and
3,027 shares at DKK 1,432.00.

Proceeds to the company are approximately DKK 25.6 million. The increase corresponds to approximately 0.05% of the company’s share capital.

The new shares are ordinary shares without any special rights and are freely transferable negotiable instruments. The new shares give rights to dividends and other rights in relation to the company as of subscription, i.e. inter alia full rights to dividends for the financial year 2021. The new shares will be listed on Nasdaq Copenhagen after registration with the Danish Business Authority. The capital increase is expected to be finalized shortly.

Pursuant to section 32 of the Danish Capital Markets Act No. 1767 of November 27, 2020, it is hereby announced, that the total nominal value of Genmab A/S’ share capital after the capital increase is DKK 65,620,740 which is made up of 65,620,740 shares of a nominal value of DKK 1 each, corresponding to 65,620,740 votes.

On May 11, 2021 VBL Therapeutics (Nasdaq: VBLT) reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, VBL Therapeutics, MAY 11, 2021, View Source [SID1234579678]).

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"Our OVAL Phase 3 clinical trial remains on track and passed its most recent safety review, with the next one coming in the third quarter," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "During the first quarter and in early April, we strengthened our balance sheet, raising a total of approximately $40 million in additional capital, $12.3 million primarily through the exercise of outstanding warrants and gross proceeds of $28.3 million through a public financing. The additional capital extends our cash runway until year-end 2023. We have a number of important milestones anticipated through the rest of 2021 and look forward to keeping investors apprised of our progress."

First Quarter and Recent Corporate Highlights

In February, a successful pre-planned DSMC review of the OVAL study found no safety issues with the trial and recommended its continuation as planned.
In March, the Company announced the initiation of randomized, controlled and blinded trial of VB-111 in patients with recurrent Glioblastoma Multiforme (rGBM).
In March, the Company announced the publication of positive results in peer-reviewed journal Gynecologic Oncology of the pre-specified interim analysis of the OVAL Phase 3 Registration Enabling Study of VB-111 in Ovarian Cancer. The analysis showed a CA-125 response of at least 58% in the VB-111 treatment arm.
Development of VB-111 in rGBM and colorectal cancer, as well as of pipeline assets, continues as planned.
Corporate

In January, the Company entered into an ordinary share purchase agreement of up to $20 million with Aspire Capital Fund LLC
During the first quarter, the Company raised an additional $12.3 million through a combination of sales under the Aspire share purchase agreement, sales through its At-the-Market (ATM) facility and the exercise of outstanding warrants
In April, the Company closed a public offering raising gross proceeds of $28.3 million
Financial Results for the First Quarter 2021

As of March 31, 2021, VBL had cash, cash equivalents, short-term bank deposits and restricted bank deposits totaling $36.6 million. After the end of the first quarter, VBL raised gross proceeds of $28.3 million in a public offering of shares and pre-funded warrants. VBL expects that its cash and cash equivalents and short-term bank deposits will be sufficient to fund operating expenses and capital expenditure requirements until year-end 2023.
Revenues for the first quarter 2021 were $185 thousand, as compared to $366 thousand in the comparable period in 2020.
R&D expenses, net were $4.8 million for the first quarter compared to $4.5 million in the comparable period in 2020.
G&A expenses were $1.7 million for the fiscal year, compared to $1.3 million in the comparable period in 2020.
VBL reported a net loss for the quarter ended March 31, 2021, of $6.3 million, or ($0.12) per basic share, compared to a net loss of $5.4 million, or ($0.15) per basic share, in the comparable period in 2020.
Conference Call:

The live webcast will be available online and may be accessed from the "Events and Presentation" page of the company website. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.