On May 13, 2019 Transgene (Euronext Paris: TNG) (Paris:TNG), a biotech company that designs and develops virus-based immunotherapies against cancers and infectious diseases to transform the fight against solid tumors and infectious diseases, reported its business update for the quarter ending March 31, 2019 (Press release, Transgene, MAY 13, 2019, View Source [SID1234536214]).

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Operating revenue:

The following table summarizes the first quarter operating revenue for 2019 compared to the same period in 2018:

Q1
In millions of euros 2019 2018

Revenue from collaborative and licensing agreements 0.4 0.2
Government financing for research expenditures 1.5 1.6

Operating revenue 1.9 1.8
During the first quarter of 2019, revenue from collaborative and licensing agreements was mainly composed of research services and royalties.

As of March 31, 2019, government financing for research expenditures mainly consisted of 25% of the research tax credit expected for 2019 (€1.5 million in the first quarter of 2019, compared to €1.6 million for the same period in 2018).

Cash, cash equivalents, available-for-sale financial assets and other financial assets:

In the first quarter of 2019, Transgene’s cash burn was €7.8 million, compared to €5.8 million for the same period in 2018. Cash, cash equivalents, available-for-sale financial assets and other financial assets stood at €9.1 million as of March 31, 2019, compared to €16.9 million as of December 31, 2018. This cash position does not include the €20 million credit facility available for the Company or the $10 million receivable from AstraZeneca.

Key achievements:

Finance:
Transgene secured a €20 million revolving credit facility with a 30-month term with Natixis. Transgene will be able to draw on and repay the facility at its discretion. Transgene has used its shares in the Chinese biotech company Tasly Biopharmaceuticals as collateral for this loan (press release distributed on March 18, 2019). As of May 13, 2019, the Company has not drawn down on this facility.
Invir.IO:
Transgene and AstraZeneca signed a collaborative research, option and exclusive license agreement to co-develop five armed oncolytic vaccinia virus candidates deriving from the Invir.IO platform.
Transgene is to receive $10 million in Q2 2019 and additional pre-clinical success milestones of up to $3 million. Transgene is eligible to receive an option exercise payment on each candidate in the event AstraZeneca exercises one or more of its license option, as well as development and commercial milestones and royalties (press release distributed on May 2, 2019).
Transgene and BioInvent extend their collaboration to develop additional multifunctional oncolytic viruses encoding for antibodies capable of treating a broad range of solid tumors (press release distributed on March 26, 2019).
myvac:
Transgene announced that the NEOVIVA project, that is focused on Transgene’s individualized immunotherapy platform myvac, was selected by the "Investments for the Future" Program (Programme d’Investissements d’Avenir) operated by Bpifrance. The NEOVIVA project will receive €5.2 million over the five-year duration of the program from Bpifrance, of which Transgene will receive €2.6 million (press release distributed on March 13, 2019).
TG4050:
Transgene announced its decision to initiate clinical developments of its lead myvac candidate, TG4050, and the finalization of its collaboration agreement with NEC (press release distributed on March 5, 2019).
Transgene received FDA IND clearance for TG4050 to commence clinical development in ovarian cancer (press release distributed on May 13, 2019).
Outlook:

Transgene expects its cash burn for 2019 to be between €15 million and €20 million, based on its current development plan and cash-in from the collaboration with AstraZeneca.

As a result of the financing agreement with Natixis and the signing of the collaboration with AstraZeneca, Transgene has extended its financial visibility to Q4 2020.

Transgene confirms that it expects readouts from its key clinical trials in the second half of 2019.

On May 13, 2019 Ayala Pharmaceuticals, a clinical-stage company developing medicines for cancers that are genetically defined, reported that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) for AL101, a potent and selective inhibitor of gamma secretase-mediated Notch signalling, for the treatment of ACC AL101 is currently in Phase 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY) (Press release, Ayala Pharmaceuticals, MAY 13, 2019, View Source [SID1234536212]). For additional information about AL101 ACCURACY clinical trial, please go to www.clinicaltrials.gov. Interested patients and physicians can contact Medical Affairs at Ayala for more information at: [email protected] or call refer +1-857-444-0553..

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Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation by the FDA entitles Ayala to seven years of market exclusivity for the use of AL101 for the treatment of ACC, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

"Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop AL101 as a potential treatment for people with ACC," said Roni Mamluk, Ph.D., Chief Executive Officer at Ayala Pharmaceuticals. "Given the proof of concept demonstrating that AL101 has a significant inhibitory effect on tumor growth in a mouse model using implanted ACC patient-derived xenograft tumors coupled with the significant unmet need in ACC, we look forward to accelerating AL101 development for patients in the U.S."

ACC is a rare form of cancer. In the U.S., there are approximately 566,000 people diagnosed with cancer each year, and only about 1,224 of them are diagnosed with ACC. According to the Adenoid Cystic Carcinoma Organization International, there are approximately 14,873 Americans alive today living with this disease. Current treatment options include surgery, chemotherapy and/or radiation therapy; however, there is no approved drug for the treatment of ACC.

About AL101

AL101 is a gamma secretase inhibitor developed as a Notch inhibitor for oncology indications. Notch signaling pathway plays an important role in tumorigenesis in several solid and hematological malignancies. Upon ligand binding of the Notch receptor, an important step in the activation of Notch receptors is cleavage by gamma secretase, which frees the Notch intracellular signaling domain.

AL101 is currently in Phase 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY). For additional information about AL101 ACCURACY clinical trial, please go to www.clinicaltrials.gov. Interested patients and physicians can contact Medical Affairs at Ayala for more information at: [email protected] or call refer +1-857-444-0553.

On May 13, 2019 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that its research partner, the University of Northern Colorado, has bioengineered a human cell line designed to activate a cannabinoid prodrug in its quest to develop a Cell-in-a-Box/cannabinoid therapy for serious forms of cancer, particularly brain cancer (Press release, PharmaCyte Biotech, MAY 13, 2019, View Source [SID1234536211]).

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Mark L. Rabe, MD, PharmaCyte’s Director of Cannabis Program Development, commenting on the bioengineered cell line said, "We now have a cell line into which the gene for a putative cannabinoid prodrug-activating enzyme has been ‘transfected’ or inserted into the cell’s DNA. The gene was synthesized de novo using knowledge of the underlying genetic code of the cell. The cell line is the same human cell line that will be used in PharmaCyte’s planned clinical trial in locally advanced, inoperable pancreatic cancer (LAPC).

"The next step is to test the efficiency of the transfected cells in converting cannabinoid prodrugs into their active cancer-fighting forms. If the cells are suitably active, they would then be propagated to the point that they can then be encapsulated using the Cell-in-a-Box technology. Also, we will continue our analysis of other genes of interest that could be used in a similar way."

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, stated, "As we advance our Cell-in-a-Box + ifosfamide therapy for LAPC with the U.S. FDA through preparation and submission of an Investigational New Drug application, we are pleased to report on the work done at the University of Northern Colorado. Such news as this serves to contribute to PharmaCyte’s efforts as we work diligently to develop treatments for deadly diseases and build shareholder value."

The current objective of PharmaCyte’s Cannabis Program is to develop targeted cannabinoid-based chemotherapy by bioengineering a cell line that produces a cannabinoid prodrug-activating enzyme, encapsulating this cell line utilizing the Cell-in-a-Box platform and implanting the encapsulated cells near the site of a tumor. A cannabinoid prodrug would then be administered and activated at the site of the tumor where its anti-cancer effects are needed. PharmaCyte’s research conducted by the University of Northern Colorado has confirmed that a cannabis-based approach may prove to be efficacious in the treatment of several different types of cancers.

On May 13, 2019 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies to treat cancer based on its novel T cell technology platforms, reported financial results and provided a corporate update for the first quarter ended March 31, 2019, and recent activities (Press release, Unum Therapeutics, MAY 13, 2019, View Source [SID1234536207]).

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"We remain on track to deliver on key milestones across our pipeline of hematologic and solid tumor cancer programs," said Chuck Wilson, President and CEO of Unum. "The dose escalation phases of our non-Hodgkin lymphoma, multiple myeloma, and HER2+ advanced cancer trials based on our Antibody-Coupled T cell Receptor (ACTR) platform are proceeding as planned, positioning us to report data and drive decisions on next steps. Simultaneously, we continue to advance and expand our preclinical pipeline of Bolt-On Chimeric Receptor (BOXR) programs, aiming to address the unmet need in solid tumor cancers by engineering improved T cell functionality."

Recent Highlights

Dose Escalation in ATTCK-20-03 Phase I Trial in Non-Hodgkin Lymphoma Continuing: Building upon results from the first two dose cohorts presented at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, Unum has continued dose escalation in Cohorts 3 and 4 in its ongoing Phase I study of ACTR707 in combination with rituximab in patients with relapsed/refractory CD20+ B cell non-Hodgkin lymphoma (r/r NHL). Cohort 3 has completed enrollment and dosed patients with 55M ACTR+ T cells, whereas Cohort 4 enrollment and dosing of patients with 80M ACTR+ T cells is ongoing. As of May 7, 2019, no dose-limiting toxicities (DLTs) and no severe adverse events of cytokine release syndrome (CRS) or neurologic events have been reported. Unum plans to complete dose escalation in the second half of 2019 and subsequently to initiate safety expansion at the preliminary recommended Phase II dose of ACTR707. Unum plans to report results from the dose escalation phase in late 2019.

Expansion Cohort in ATTCK-20-2 Phase I Trial in Non-Hodgkin Lymphoma Ongoing: Enrollment has completed in the ATTCK-20-2 study, a Phase I clinical trial evaluating safety and anti-lymphoma activity of ACTR087 in combination with rituximab in patients with r/r NHL. Unum is continuing ACTR087 treatment, safety, and response assessments in the expansion cohort at the preliminary recommended Phase II dose (35M ACTR+ T cells). No severe adverse events of CRS or neurologic events have been observed as of May 7, 2019. Unum plans to report data on all enrolled patients from ATTCK-20-2 at the end of 2019. These findings will be used to advise other ACTR programs, in particular ATTCK-17-01, an ongoing Phase I trial of ACTR087 in combination with SEA-BCMA.

Dose Escalation in ATTCK-17-01 Phase I Trial in Multiple Myeloma Continuing: After reporting data at the 2018 ASH (Free ASH Whitepaper) Annual Meeting from the first three dose cohorts in the ATTCK-17-01 study, combining ACTR087 with very low doses of SEA-BCMA antibody, dose escalation is continuing at doses of SEA-BCMA that may be expected to have pharmacological activity based upon preclinical studies. Enrollment and dosing of patients at Dose Level 4 (30 MM ACTR+ T cells and 2.0 mg/kg SEA-BCMA) has completed, and enrollment and dosing of patients at Dose Level 5 (50M ACTR+ T cells and 2.0 mg/kg SEA-BCMA) is ongoing. As of May 7, 2019, no DLTs and no severe adverse events of CRS or neurologic events have been reported in this trial. Unum expects to continue to enroll and dose patients through the dose escalation phase of the trial and to report data from multiple dose cohorts in the second half of 2019.

Dose Escalation with ATTCK-34-01 Phase I Trial in HER2+ Advanced Cancers Ongoing: In December, 2018, Unum initiated the first clinical site in the ATTCK-34-01 study, a Phase 1, multicenter, single-arm, open-label dose escalation study evaluating ACTR T cells in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers. Enrollment, dosing, and assessment of patients in the first dose cohort are ongoing. As of May 7, 2019, no DLTs or severe adverse events of CRS or neurologic events have been reported in this trial. Unum plans to report initial clinical data from ATTCK-34-01 at the end of 2019.

Preclinical Development of BOXR1030 Targeting GPC3+ Advanced Cancers Ongoing: Earlier this year, Unum nominated BOXR1030 as the first product candidate from their BOXR platform, which seeks to counter immunosuppression, improving T cell functionality for solid tumors. IND-enabling preclinical studies of BOXR1030 are underway, as well as research to characterize its mechanism of action. Unum plans to present additional data regarding BOXR1030 in the second half of 2019.

First Quarter 2019 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the first quarter ended March 31, 2019 was $3.1 million, compared to $2.2 million in the same period of 2018. The increase reflects the recognition of a portion of the $25.0 million upfront payment received from Seattle Genetics under Unum’s collaboration agreement as well as reimbursements of research and development costs attributed to the collaboration agreement.

R&D Expenses: Research and development expenses were $12.4 million for the first quarter ended March 31, 2019, compared to $8.1 million for the same period of 2018. The increase reflects higher clinical trial costs for the active Phase I clinical trials, as well as increased personnel-related costs, materials and facility-related costs related to scaling manufacturing processes, and increased consultant costs to support these activities.

G&A Expenses: General and administrative expenses for the first quarter ended March 31, 2019, were $2.5 million, compared to $1.1 million for the same period of 2018. The increase is primarily related to higher personnel related costs due to increased headcount and increased expenses around operating as a public company.

Net Loss: Net loss attributable to common stockholders was $11.7 million, or $0.39 per share, for the first quarter ended March 31, 2019, and $6.8 million, or $0.66 per share, for the same period of 2018.

Cash, Cash Equivalents and Marketable Securities: As of March 31, 2019, Unum had cash, cash equivalents, and marketable securities of $67.1 million. Unum believes that its existing cash, cash equivalents, and marketable securities, will fund operating expenses and capital expenditure requirements into early 2021.

Investor Call and Webcast Information

Unum will host a live conference call and webcast today, May 13, 2019, at 8:00 a.m. ET, to discuss these financial results and company updates. To access the call, please dial 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 1443149. A webcast will be available at View Source at least 10 minutes before the event begins. The archived webcast will be available at the same location approximately two hours after the event and will be archived for 90 days.

On May 13, 2019 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapiesfor the treatment of solid tumors, reported that it has received Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA) to proceed with a Phase 1 clinical trial of its lead myvac candidate TG4050 as a potential treatment for ovarian cancer patients after first-line surgery and chemotherapy (Press release, Transgene, MAY 13, 2019, View Source [SID1234536206]).

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TG4050 is an individualized MVA-based immunotherapy derived from the myvac platform. It has been designed to stimulate and educate the immune system of patients to recognize and destroy tumor cells. Tumor cells accumulate mutations and each patient has a set of mutations that are unique to his tumor. TG4050 is designed to target a panel of patient specific mutations selected using a NEC’s Neoantigen Prediction System

"We are very pleased to have been granted an IND for TG4050 by the FDA allowing us to commence the first trial with our lead myvac candidate in ovarian cancer patients who have already received first-line treatment" said Maud Brandely, Chief Medical Officer of Transgene. "We believe individualized vaccination is a promising solution with significant potential to transform treatment outcomes for a wide range of solid tumors. With TG4050, we are confident that we can show that this therapeutic modality will improve patient outcome. We look forward to updating you on the progress of this clinical trial, which is expected to start later this year."

The Phase 1 clinical trial will evaluate the safety and the tolerability of TG4050 in patients with ovarian, fallopian or peritoneal serous cell carcinoma. Antitumor activity will also be measured. This multicenter, one-arm trial will recruit patients in the United States and Europe.

The study, sponsored by Transgene, will be co-financed by Transgene and its partner NEC, which will also support the trial by contributing to the therapeutic vaccine design and the selection of target neoantigens (see press release dated March 5, 2019.

Contacts Transgene:

Lucie Larguier Director Corporate Communications & IR +33 (0)3 88 27 91 04 [email protected] Media: Citigate Dewe Rogerson EU: David Dible/Sylvie Berrebi US: Marine Perrier-Barthez + 44 (0)20 7638 9571/+1 424 341 9140 [email protected]

About TG4050
TG4050 is an immunotherapy designed to stimulate the immune system of patients in order to induce a response that is able to recognize and destroy tumor cells in a specific manner. This personalized immunotherapy is developed for each patient, on the basis of mutations identified through sequencing of tumor tissue, prioritized using NEC’s Neoantigen Prediction System and delivered using the myvacTM technological platform which allows development and manufacture of a product that is specific to the patient within time frames compatible with clinical management.

About myvacTM myvacTM is a viral vector (MVA) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. The myvacTM-derived products are designed to stimulate the patient’s immune system, recognize and destroy tumors using the patient’s own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization know-how and unique manufacturing capabilities. Transgene has been awarded an "Investments for the Future" funding from Bpifrance for the development of its platform myvacTM.

About NEC’s Neoantigen Prediction System NEC’s neoantigen prediction utilizes its proprietary AI, such as graph-based relational learning, which is combined with other sources of data to discover candidate neoantigen targets. NEC comprehensively evaluates the candidate neoantigens with a primary focus placed on its in-house MHC-binding affinity prediction. These allow NEC to effectively prioritize the numerous candidate neoantigens identified in a single patient.