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MEDIGENE REPORTS FINANCIAL & BUSINESS RESULTS FOR THE FIRST NINE MONTHS OF 2019

On November 13, 2019 Medigene AG (Medigene, FSE: MDG1), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies, reported on its business performance and financial results for the first nine months of 2019 and confirms the financial guidance (Press release, MediGene, NOV 13, 2019, View Source [SID1234551163]).

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The full version of the quarterly statement 9M-2019 can be downloaded here: View Source

Prof. Dolores Schendel, CEO/CSO of Medigene AG commented: "In the past nine months, we have continued to work intensively on the progress of our immunotherapies. Preparations are ongoing for our next clinical trial with the T cell receptor (TCR) immunotherapy MDG1021 in cancer patients suffering from a disease relapse after hematopoietic stem cell transplantation. This trial will start next year and will be conducted in cooperation with the Leiden University Medical Center.
Our first-in-human clinical phase I/II trial with MDG1011 in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and multiple myeloma (MM) continues to recruit patients. While recruitment has been below desired levels we have adapted the study protocol and successively expanded the study centers. We expect data from the first three dose cohorts to be available in Q4 2020. We plan to further expand our pipeline of potential TCR candidates and are continuously researching the innovation potential of our technologies. We aim to increase the number of possible targets for our TCRs and are developing innovative next-generation TCR therapies, especially for difficult conditions such as the immunosuppressive environments in solid tumors. After promising interim data for our dendritic cell (DC) vaccine, we expect the topline results to be reported at the beginning of next year. Our partnerships as well as our finances are on track and we look forward to the coming months."

Major events since the beginning of 2019
– Medigene enters a clinical trial agreement for its second TCR immunotherapy MDG1021 with Leiden University Medical Center. Phase I clinical trial with the HA-1-specific TCR therapy scheduled to start in 2020
– Medigene publishes interim analysis from ongoing Phase I/II clinical trial with DC vaccines in acute myeloid leukemia (AML) patients at the Congress of the European Hematology Association (EHA) (Free EHA Whitepaper)
– bluebird bio presents preclinical data of the first TCR candidate from the Medigene collaboration and announces that clinical development of the TCR candidate targeting the MAGE-A4 tumor antigen, which is expressed on a variety of solid tumor types, will start in 2020
– Medigene doses the first patient in Phase I/II clinical trial of Medigene’s first T cell immunotherapy MDG1011 to treat the blood cancers: AML, MDS, and MM, and opens additional clinical trial centers
– Medigene and Roivant/Cytovant enter into a strategic partnership for the research and discovery of cell therapies in Asia. Medigene receives upfront payment of USUSD10 m and reimbursement of future R&D expenditure, potential milestone payments and royalties
– Medigene licenses a co-stimulatory receptor to enhance TCR therapies for solid tumors
– Medigene presents the results from in vitro tests to assess the potential TCR-mediated off-target toxicity for neuronal cells at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper)
– Medigene presents preclinical data on the selective killing of tumor cells by PRAME TCR-transduced T cells at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) conference
– Medigene presents its new ‘inducible Medigene (iM)-TCR’ which enables controllable cytotoxicity of tumor-specific TCR-T cells to potentially tune safety and efficacy according to clinical needs at the CAR-TCR Summit 2019
– Medigene obtains two European patents for its DC vaccine platform and for a TCR building block library to develop neoantigen-specific TCRs
– Medigene sells the remaining rights and stocks of Veregen to Aresus Pharma and thereby completes its transformation into a pure-play immunotherapy company

Key figures in the first three quarters of 2019
– Revenue from the core business of immunotherapies increased by 32% to Eur 6.2 m (9M 2018: Eur 4.7 m)
– Research and development expenses increased as expected by 24% to Eur 16.5 m (9M 2018: Eur 13.3 m) due to more intense activities in the development and production activities for Medigene’s immunotherapy programs
– EBITDA loss increased as anticipated by 48% to Eur 18.6 m (9M 2018: Eur 12.6 m*) (without taking the non-core business Veregen effect into account: increase of EBITDA loss by 11% to Eur 13.9 m)
– Net loss for the period increased as expected by 52% to Eur 20.0 m (9M 2018: Eur 13.2 m*)
– Cash and cash equivalents and time deposits of Eur 60.5 m as at September 30, 2019 (December 31, 2018: Eur 71.4 m)
– Confirmation of financial guidance 2019

* IAS 8 correction – see note (3) to the consolidated financial statements

Outlook
Financial guidance 2019

Medigene confirms the financial guidance issued in the 6M-2019 financial statement.

– The company is expecting to generate total revenue of Eur 10 – 11 m in 2019.
– Due to progress in the development projects, Medigene is projecting research and development expenses of Eur 24 – 29 m and forecasts an EBITDA loss of Eur 23 – 28 m.
– This assessment does not include potential future milestone payments or cash flows from existing or future partnerships or transactions, as the timing and extent of such events depends to a large extent on external parties and therefore cannot be reliably predicted by Medigene.
– Based on its current planning, the Company has sufficient financial resources to fund business operations beyond the planning horizon of two years.

Immunotherapy Outlook

Current Phase I/II clinical trial with Medigene’s first TCR therapy MDG1011
Medigene commenced the Phase I/II clinical trial of its TCR-based T cell therapy MDG1011 and began treating patients in the first quarter of 2019. In 2019, the focus of the trial is the recruitment for the first dose cohorts to assess the safety and tolerability of the treatment with MDG1011.

Planned Phase I/II clinical trial with Medigene’s second TCR therapy MDG1021
Based on the Clinical Trial Agreement entered into with the Leiden University Medical Center in May 2019, Medigene plans to conduct a Phase I clinical trial with its TCR therapy MDG1021, targeting the HA-1 antigen. Medigene will assess the safety, feasibility and preliminary efficacy of MDG1021 in patients with relapsed or persistent hematologic malignancies after allogeneic hematological stem cell transplantation. The study is scheduled to start in 2020 as Medigene’s second company-sponsored clinical TCR-T trial. In preparation for the trial, Medigene first needs to establish the manufacturing process, develop the design of the trial and then obtain the official trial approval.

Development of additional TCR candidates
Now that a robust platform for the discovery and characterization of new TCR candidates has been fully established, building a solid pipeline of potential TCR development candidates is an important goal to secure future clinical programs for both internal and existing or future partners.
In 2019, in addition to the MDG1011 clinical trial, Medigene will therefore continue to work on characterizing new TCR candidates for future clinical trials under the responsibility and funding of Medigene and collecting preclinical data to prepare further clinical TCR trials.

Optimization of future TCR therapies for solid tumors
The new inducible Medigene (iM)-TCR approach as well as the chimeric co-stimulator receptor (the PD-1/4-1BB molecule) licensed from HMGU will be assessed in combination with Medigene’s tumor-specific TCR-Ts in preclinical models in order to optimize future TCR therapies for solid tumors.

TCR partnerships
Medigene continues its successful collaboration with bluebird bio and expects to make further progress on TCR candidate discovery. Within the framework of the partnership entered into with Roivant/Cytovant, Medigene will now, together with the collaboration partner, undertake the preparations to generate TCR constructs tailored specifically to Asian patient populations using its proprietary TCR discovery and isolation platform.

Dendritic cell vaccines (DCs)
Conclusion of the Phase I/II clinical trial at the end of 2019
Medigene will continue the current Phase I/II clinical trial for DC vaccines for the treatment of AML as planned and bring it to a conclusion at the end of 2019. Topline data will be reported in the beginning of 2020.

Conference call and webcast
A telephone conference in English will be held today at 2:00 pm CET (Munich/Frankfurt) / 8:00 am EST (New York) and transmitted live via webcast. Access and transmission of the synchronized presentation slides and a recording of the presentation will be available on the Medigene website at View Sourcewebcasts/

F1 Oncology’s International Affiliate, EXUMA Biotechnology, Announces Acquisition of Strategic CAR-T Assets

On November 13 2019 F1 Oncology, Inc., a biotechnology company discovering and developing adoptive cellular therapies (ACTs) for solid tumors, reported that its international affiliate, EXUMA Biotechnology Hong Kong, has formed Shanghai EXUMA Biotechnology Co., Ltd., a wholly foreign-owned enterprise (WFOE), to acquire all rights to novel chimeric antigen receptor T (CAR-T) cell therapy and manufacturing assets in Shanghai and Shenzhen for the development and commercialization of cellular therapies in Greater China to be used in the treatment of solid tumor malignancies (Press release, EXUMA Biotechnology, NOV 13, 2019, View Source [SID1234551150]).

Shanghai EXUMA will develop these clinical programs into multicenter trials under a centralized regulatory pathway based on the results of investigator-initiated trials (IITs) of CAR-T therapies for the treatment of metastatic renal cell carcinoma (mRCC) and other solid tumor malignancies. Shanghai EXUMA will be expanding its CAR-T cell processing capabilities at its new facility in the Shanghai International Medical Zone within Zhangjiang Innopark in Shanghai’s Pudong district and its viral manufacturing capabilities at its newly constructed Shenzhen facility.

"The treatment of cancer is advancing at unprecedented speed, and we are pleased to be playing a role in this rapid and positive evolution, of which the formation of Shanghai EXUMA Biotechnology is an important component," said Gregory Frost, Ph.D., Chairman and CEO of F1 Oncology. "The decision to integrate these programs and processes into EXUMA was driven by the favorable early clinical profile, and we are excited to support their advancement toward multicenter trials next year. By turning a hostile tumor microenvironment (TME) into an activating signal for CAR-T cells, this platform could open the therapeutic window of CAR-T to many solid tumors."

EXUMA Biotech integrates BioAtla’s conditionally active biologic (CAB) protein therapeutic technology with F1 Oncology’s proprietary CAR-T technologies to develop and commercialize CAB-CAR-T therapies and other ACTs for the treatment of solid tumor malignancies. CAB-CAR-T cellular therapies are designed to be conditionally active only in the TME and may therefore help reduce potential adverse events associated with on-target, off-tumor effects of CAR-T therapies in solid tumors. While BioAtla’s option to purchase F1 Oncology expired in May 2018, F1 Oncology retains an exclusive global license to discover and develop CAB-based ACTs.

ProMIS Neurosciences Undertaking $6.5M Private Placement

On November 13, 2019 ProMIS Neurosciences, Inc. ("ProMIS" or the "Company") (TSX: PMN; OTCQB:ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported that it is undertaking a private placement of up to 32.5M Units at a price of CDN$0.20 (or US$0.15) per Unit for gross proceeds of up to CDN$6,500,000 (the "Offering") (Press release, ProMIS Neurosciences, NOV 13, 2019, View Source [SID1234551157]). ProMIS expects to complete a first closing in November 2019 in the amount of approximately CDN$2,055,000.

"We are pleased to offer this private placement designed to afford a cash runway well into 2020. Proceeds of the offering will support further development of our programs targeting Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, each of which is the subject of ongoing partnering discussions with large biopharmaceutical companies," stated Dr. Elliot Goldstein, ProMIS President and CEO.

Each Unit will consist of one common share of the Company and one share purchase warrant of the Company (each a "Warrant"). Each Warrant will entitle the holder thereof to purchase one share at an exercise price of $0.35 per share at any time through the fifth anniversary of the offering.

All securities issued in connection with the Offering will be subject to a four-month statutory hold period in accordance with applicable provincial securities laws in Canada. Net proceeds from the Offering are intended to be used to advance the Company’s antibody therapeutic candidates selectively targeting toxic oligomers implicated in neurodegenerative diseases, for working capital and general corporate purposes. Closing of the Offering is subject to TSX approval.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful. The securities issued, or to be issued, under the Offering have not been, and will not be, registered under the United States Securities Act of 1933, as amended, and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.

Cellectis Publishes Creation of “Smart CAR T-Cells” for Potentially Safer, More Effective Treatments for Cancer in Nature Communications

On November 13, 2019 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported a paper published in Nature Communications that describes a proof-of-concept for rewiring the cell pathway to create highly intelligent T-cells that can recognize cancerous tumors and cause a micro secretion of therapeutic proteins onto these tumors, which ultimately reshapes the tumor microenvironment and improves the T-cells ability to fight cancer (Press release, Cellectis, NOV 13, 2019, View Source [SID1234551149]). By utilizing gene editing techniques to rewire the TCRα, CD25 and PD1 genes, the study enabled CAR T-cells to micro secrete the pro-inflammatory cytokine, IL-12, in a tumor and time-dependent manner, paving the way for a next generation of tightly controlled, highly active and potentially safer CAR T-cell treatments.

"Discussion around the tumor microenvironment has become a popular topic in the CAR T-cell space, and with recent advancements in gene editing technologies, especially TALEN, it is now possible to manipulate the way a T-cell regulates itself to adapt to its environment," said Dr. Philippe Duchateau, Ph.D., Chief Scientific Officer, Cellectis. "With seamless modification of multiple genes, and subsequently rewiring their natural regulatory processes, this approach causes the T-cells to secrete therapeutic proteins of interest in a tightly controlled and localized manner. We have essentially transformed the current T-cells used today into precise and powerful micro-robots that can spray IL-12 specifically onto cancer cells – potentially avoiding the toxicity of a systemic injection of IL-12, while enhancing CAR-T activity."

"Our extensive knowledge in TALEN-based gene editing and DNA donor template design enabled us to develop this groundbreaking proof-of-concept, a milestone that paves the way to the next generation of CAR T-cells," added Dr. Julien Valton, Ph.D., Innovation Team Leader, Cellectis. "These highly intelligent CAR T-cells can sense and remodel their microenvironment in a tailored, highly regulated, and antigen-specific manner, allowing us to have more control over increasingly potent treatments and less risk of general secretion into healthy tissues. This engineering strategy could bring smarter, safer and more effective treatments to the forefront for patients in need."

Julien Valton, Ph.D., Innovation Team Leader, Cellular Engineering & Adoptive CAR T-Cell Immunotherapy

Dr. Julien Valton obtained his Ph.D. at the University Joseph Fourier in Grenoble, France, where he was trained as an enzymologist. He then joined the Yale School of Medicine to apply his knowledge to therapeutic research by investigating the mechanism of inhibition of receptor tyrosine kinases that are involved in the development of gastrointestinal cancer. In 2009, he moved a step further into the field of applied science by joining the Innovation Department of Cellectis, where he actively participated in using and improving TALEN gene editing technology for targeted gene therapy and genome engineering. He is now using TALEN along with protein engineering techniques to develop the next-generation CAR T-cells to treat different malignancies.

Moleculin Announces Beginning of Preclinical Development of New Approach to Pancreatic Cancer

On November 13, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported it has begun preclinical testing of its drug candidate, WP1122, which it believes may present a new approach to treating highly glycolytic tumors like pancreatic cancer and glioblastoma (Press release, Moleculin, NOV 13, 2019, View Source [SID1234551156]).

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

"WP1122 represents an opportunity to attack the metabolism of cancer by exploiting the Warburg principle, which explains that some tumors are highly dependent on glycolysis, a specific metabolism of glucose, for growth and survival," commented Walter Klemp, Moleculin’s Chairman and CEO. "What this means in practice is that tumors are vulnerable by being highly dependent on glucose availability. Cancer cells often consume up to 18 times as much glucose as their healthy normal cells’ neighbors, suggesting that we may be able to starve tumors by supplying them with glucose decoys that would inhibit glucose-based energy production. However, until the creation of WP1122, glucose decoys like 2-deoxy-D-glucose (‘2-DG’) lacked the drug-like properties to be effective, primarily because of rapid metabolism and a very short circulation time in the body, which then limits desired organ and tumor uptake."

Dr. Don Picker, Chief Science Officer for Moleculin, added: "WP1122 is a prodrug of 2-DG that has been shown in animal models to significantly increase the half-life of 2-DG and allows for increased uptake to targeted organs and tumors like the brain tumors and pancreatic cancer. Recent discoveries now also suggest that such a glucose decoy could critically impact a process known as glycosylation and glycan formation and that this type of activity can directly impact the function of PD-L1, enabling increased immune system response to cancer cells. We have been working on the clinical formulation of WP1122 for some time, and we are eager to now be taking the next key steps to getting WP1122 into the clinic."