On March 16, 2022 Transgene (Euronext Paris: TNG) (Paris:TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for 2021 and provides an update on its product pipeline (Press release, Transgene, MAR 16, 2022, View Source [SID1234610180]).
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Hedi Ben Brahim, CEO of Transgene commented: "It has been more than a year since I joined Transgene as Chairman and Chief Executive Officer and I am pleased and proud to have experienced and delivered such a successful year for the Company.
Transgene achieved several important milestones in 2021. We announced the first positive data from two Phase I trials with TG4050, our individualized therapeutic vaccine based on our myvac platform. These results demonstrated the immunogenicity of the vaccine, which led to the first signs of clinical activity. We expect to confirm these results in the coming months and to provide more in-depth data at major scientific congresses in 2022.
The Phase II trial with TG4001, our therapeutic vaccine against HPV16-positive anogenital cancers, was launched in 2021, with the first patient enrolled in June; recruitment is continuing at a steady pace. First data from an interim analysis, including up to 50 patients, is anticipated in the fourth quarter of 2022.
We also made significant progress with our oncolytic virus pipeline. We recruited the first patient into the Phase I/IIa study evaluating BT-001, our first candidate from our Invir.IO platform. We also presented the first Phase I data with TG6002 at two major conferences in 2021: AACR (Free AACR Whitepaper) and ESMO (Free ESMO Whitepaper). These results confirm the feasibility of intravenous administration of this oncolytic virus. The ability to be given intravenously would considerably extend the use of our Invir.IO based virus therapies in oncology, significantly expanding the market opportunity they can address.
In addition, our expertise and the clear benefits that our Invir.IO platform can deliver was recognized with the first license option exercised by AstraZeneca for an oncolytic virus in late 2021. This exercise results in an upfront payment of $8 million. The collaboration with AstraZeneca continues under the agreement for the development of other oncolytic immunotherapies by Transgene.
Following the successful completion of a €34.1 million private placement in June 2021 and the additional sale of Tasly BioPharmaceuticals shares in September 2021 for €17.4 million, Transgene has financial visibility until the end of 2023. This puts us in strong position to pursue Transgene’s mission: create value by developing new innovative cancer therapies."
PRELIMINARY POSITIVE RESULTS WITH TG4050 CONFIRMING ITS POTENTIAL AND REINFORCING TRANSGENE’S POSITION AS A GLOBAL LEADER IN THE FIELD OF INDIVIDUALIZED THERAPEUTIC CANCER VACCINES
Transgene is developing TG4050, an individualized immunotherapy against cancer based on its highly innovative myvac platform. This platform delivers a customized approach, by combining Transgene’s expertise in viral engineering with NEC’s artificial intelligence capabilities. TG4050 is currently being assessed in two Phase I clinical trials in Europe and in the US (in ovarian and HPV-negative head and neck cancers); NEC is financing 50% of these studies. The product is manufactured by Transgene in an in-house GMP production unit in Strasbourg. The first results were announced in November 2021.
Positive data were generated in the first six patients treated and demonstrate the strong potential of TG4050. The specific immune responses observed show a robust T-cell response against multiple targeted mutations (neoantigens), with a median of 10 positive responses per patient. The development of adaptive responses also suggest that the vaccine is able to effectively prime the immune system. The studies also provide preliminary data on the clinical activity of this individualized immunotherapy. Four patients were treated in the ovarian cancer study, including one patient with a CA-125 elevation which was cleared by vaccination for nine months before death from an unrelated chronic disease, and one patient who remained stable for nine months after the appearance of radiological lesions and initiation of treatment. In the head and neck cancer study, patients treated for 10 and 5 months, respectively, were stable and disease-free as of November 22, 2021. More details are available here.
These data reinforce the rationale for TG4050’s prediction system and support the validation of the myvac platform as an efficient approach for anti-tumor vaccination. Additional data will be presented at the AACR (Free AACR Whitepaper) annual meeting on April 12, 2022, and at other scientific conferences in 2022.
ONGOING ENROLLMENT IN THE RANDOMIZED PHASE II STUDY WITH TG4001 IN HPV16-POSITIVE ANOGENITAL CANCERS
TG4001 is a therapeutic vaccine targeting HPV-positive tumors, and is engineered to express HPV16 E6 and E7 antigens and interleukin 2 (IL-2) which stimulates immune responses. TG4001 is being developed to target recurrent or metastatic HPV-16 positive cancers, without liver metastasis, based on a clinical benefit observed in the Phase Ib/II trial. TG4001 is currently being evaluated in a randomized Phase II trial of up to 150 patients comparing the efficacy of the combination of TG4001 with avelumab versus avelumab alone. The first patient was enrolled in June 2021. The trial is actively enrolling patients in Europe (France and Spain) and has recently been initiated in the US.
An interim analysis will be performed after the inclusion of approximately 50 patients. Transgene expects to report the results of this analysis in the fourth quarter of 2022.
BT-001, FIRST ONCOLYTIC VIRUS BASED ON INVIR.IO PLATFORM, CONTINUES ITS CLINICAL DEVELOPMENT IN EUROPE AND IN THE US
BT-001 is a patented VVcopTK-RR- oncolytic virus, with high antitumor potential, based on the Invir.IO platform. It is being co-developed with BioInvent. It has been engineered to express both a human recombinant anti-CTLA-4 antibody and the human GM-CSF cytokine locally in the tumor, aiming at inducing a local Treg depletion, and providing significant therapeutic activity by limiting systemic exposure.
Promising preclinical results with BT-001 were presented at the SITC (Free SITC Whitepaper) 2021 annual meetings. They demonstrated high and robust intratumoral antitumor activity, resulting in tumor disappearance in in vivo models. In January 2022, preclinical proof-of-concept data were published in the Journal for ImmunoTherapy of Cancer (JITC). The published results demonstrated the potential of the virus to provide therapeutic benefit beyond current anti-PD1/anti-CTLA-4 immune checkpoint inhibitors. The article can be downloaded here.
Further preclinical data will be presented at the AACR (Free AACR Whitepaper) on April 12, 2022.
A multicenter, open-label Phase I/IIa study is evaluating ascending doses of BT-001 alone and in combination with pembrolizumab. The first patient in this trial, approved in Europe (France and Belgium) and in the US, was enrolled in February 2021. Patient recruitment is progressing as expected.
The next clinical update on the ongoing Phase I trial is expected in the second quarter of 2022.
TG6002 PROVIDES THE CLINICAL PROOF-OF-CONCEPT OF THE INTRAVENOUS ADMINISTRATION OF TRANSGENE’S ONCOLYTIC VIRUSES
TG6002 is based on Transgene’s patented VVcopTK-RR- strain, it has been engineered to express a chemotherapeutic agent (5-FU) directly in the tumor. TG6002 is being assessed in two Phase I/II clinical trials in gastrointestinal cancers, for which 5-FU is a common treatment. Its administration is evaluated by the intravenous and intrahepatic artery routes.
Initial Phase I data were presented at AACR (Free AACR Whitepaper) 2021 and ESMO (Free ESMO Whitepaper) 2021. The Phase I data provide the clinical proof-of-concept of the feasibility of the intravenous (IV) administration of Transgene’s patented oncolytic virus backbone. The data showed that, after IV administration, TG6002 is able to selectively replicate and persist in tumor cells leading to the local expression of its functional payload (the FCU1 gene).
This finding supports the potential of IV administration of oncolytic virus based on the VVcopTK-RR- strain behind the Invir.IO platform, extending the use of Transgene’s oncolytic therapies to a broad range of solid tumors.
The Phase I trial evaluating TG6002 administered intravenously is expected to be completed by mid-2022. Comprehensive translational data will be presented in the fourth quarter of 2022.
SUMMARY OF ONGOING CLINICAL TRIALS
myvac
TG4050
Phase I
NCT03839524
Targets: tumor neoantigens
Codeveloped with NEC
First positive data in first 6 patients demonstrating the immunogenicity of the vaccine as well as first signs of clinical activity
Ovarian cancer – after surgery and first-line chemotherapy
Trial ongoing in the US and in France
First patient treated in 2020 – patient enrollment progressing in line with forecast
> Additional data expected in 2022, including at the AACR (Free AACR Whitepaper)
TG4050
Phase I
NCT04183166
HPV-negative head and neck cancer – after surgery and adjuvant therapy
Trial ongoing in the UK and in France
First patient treated in Jan. 2021 – patient enrollment progressing in line with forecast
> Additional data expected in 2022, including at the AACR (Free AACR Whitepaper)
TG4001
+ avelumab
Phase II
NCT03260023
Targets: HPV16 E6 and E7 oncoproteins
Recurrent/metastatic anogenital HPV-positive – 1st (patients ineligible for chemotherapy) and 2nd line
Randomized Phase II trial comparing the combination of TG4001 with avelumab versus avelumab alone
First patient treated in June 2021. Active patient enrollment in Europe (France and Spain), trial initiation in the US
> Results of the interim analysis expected in Q4 2022 (N≈50)
Invir.IO
BT-001
Phase I/IIa
NCT04725331
Payload: anti-CTLA4 antibody and GM-CSF cytokine
Solid tumors
Co-development with BioInvent
Very encouraging preclinical results presented at SITC (Free SITC Whitepaper) 2021 and soon at AACR (Free AACR Whitepaper) 2022
Trial ongoing in France, Belgium and recently approved in the US. First patient included in February 2021
> Next clinical update expected in Q2 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
Multicenter trial ongoing in Belgium, France and Spain
Proof-of-concept data of the intravenous administration presented at the AACR (Free AACR Whitepaper) 2021 and ESMO (Free ESMO Whitepaper) 2021
Dose escalation completed to the maximum projected dose (3*109 pfu), confirming the good safety profile. Ongoing dose escalation (109 and 3*109 pfu)
> End of Phase I expected mid-2022
TG6002
Phase I/IIa
NCT04194034
Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration
Multicenter trial ongoing in the UK and in France
Ongoing enrollment of patients from the latest dose escalation cohort (109 pfu)
> First data expected mid-2022
ASTRAZENECA COLLABORATION : NEW MILESTONE
WITH THE FIRST LICENSE OPTION EXERCISE
AstraZeneca has exercised a first license option in December 2021 for an oncolytic virus from Transgene’s Invir.IO platform. Transgene received an $8 million payment for the exercise of this option and is also eligible to receive development, regulatory and sales-based milestones payments as well as a royalty based on future commercial sales.
The collaboration with AstraZeneca, which includes co-development of other potential oncolytic immunotherapies, is ongoing. AstraZeneca has an option to acquire the rights to each of these innovative drug candidates for further clinical development.
A NEW COLLABORATION WITH INVIR.IO PLATFORM
In January 2022, Transgene announced the launch of a preclinical collaboration with PersonGen BioTherapeutics. This collaboration aims to evaluate the feasibility and efficacy of a combination therapy against solid tumors, combining PersonGen’s CAR T cell injection with an oncolytic virus from the Invir.IO platform.
APPOINTMENT OF STEVEN BLOOM
AS CHIEF BUSINESS OFFICER
Steven Bloom joined Transgene as Vice President, Chief Business Officer (CBO) in February 2022. He joined Transgene’s executive committee to lead global business development strategy, alliance management and program management of the Company, with a focus on building a strong visibility in the US as part of establishing Transgene as a world leader in virus-based immunotherapies.
KEY FINANCIALS FOR 2021
– Operating income of €17.4 million in 2021, compared to €9.9 million in 2020.
R&D services for third parties amounted to €10.0 million in 2021 (€3.0 million in 2020), mainly due to the collaboration with AstraZeneca which generated €9.9 million in revenues in 2021 (€2.9 million in 2020). This increase is related to the first license option exercised by AstraZeneca in 2021 for €7.1 million, for an oncolytic virus developed by Transgene.
Research tax credit amounted to €7.0 million in 2021 (€6.3 million in 2020).
– Net operating expenses of €40.9 million in 2021, compared to €33.9 million in 2020.
R&D expenses were €32.9 million in 2021 (€27.3 million in 2020). This increase is mainly due to the acceleration of clinical trials in 2021 and the start of a new process development project.
General and administrative expenses amounted to €7.4 million in 2021 (€6.5 million in 2020).
– Financial income of €4.0 million in 2021, compared to €6.8 million in 2020.
The partial sale of the Tasly BioPharmaceuticals shares in September 2021 generated a net gain on asset disposal of €1.3 million. Transgene’s remaining shareholding was revaluated and resulted in financial income of €2.4 million in 2021. This figure corresponds to the difference between the last market price compared with last year.
– Net loss of €19.5 million in 2021, compared to a net loss of €17.2 million in 2020.
– Net cash burn of €10.0 million in 2021 (excluding capital increase), compared to €17.0 million in 2020.
– Cash available at year-end 2021: €49.6 million, compared to €26.3 million at the end of 2020, following the completion of a €34.1 million private placement in June 2021. In addition, Transgene still holds Tasly BioPharmaceuticals shares valued at €18.9 million at the end of December 2021.
– Transgene has a financial visibility until the end of 2023.
The financial statements for 2021 as well as management’s discussion and analysis are attached to this press release (Appendices A and B).
The Board of Directors of Transgene met on March 16, 2022, under the chairmanship of Hedi Ben Brahim and closed the 2021 financial statements. Audit procedures have been performed by the statutory auditors and the delivery of the auditors’ report is ongoing.
The Company’s universal registration document, which includes the annual financial report, will be available early April 2022 on Transgene’s website, www.transgene.fr.