On March 16, 2022 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the financial and operational results for its third quarter ended January 31, 2022, and provided an overview of recent operational highlights (PharmaCyte’s Fiscal Year begins May 1 and ends April 30) (Press release, PharmaCyte Biotech, MAR 16, 2022, View Source [SID1234610178]).

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Cash Position: PharmaCyte had approximately $87 million in cash on hand as of January 31, 2022.

Recent Q3 Highlights—Corporate:

PharmaCyte established FDIC insured accounts of approximately $50.3 million.
Recent Highlights—Pipeline Products:

In November 2021, PharmaCyte announced that the empty capsule material that makes up its CypCaps pancreatic cancer product does not cause skin irritation.
In November 2021, PharmaCyte launched its malignant ascites program with the commencement of a pivotal study to determine if its treatment for locally advanced, inoperable pancreatic cancer (LAPC)—Cell-in-a-Box (CypCaps) combined with the cancer killing prodrug ifosfamide—can also delay the production and accumulation of malignant ascites.
In December 2021, the Company successfully completed the Cytochrome P450 site of integration DNA sequencing assay and announced the results of an additional, more detailed, analysis of the integration site of the cytochrome P450 2B1 gene from the augmented HEK293 cell clone that PharmaCyte uses in its CypCaps product.
In December 2021, PharmaCyte successfully completed the 36-month time point in its ongoing Master Cell Bank stability study.
In January 2022, PharmaCyte announced that the empty capsule material that makes up PharmaCyte’s CypCaps pancreatic cancer product candidate is not toxic for the encapsulated cells inside the CypCaps.
In February 2022, PharmaCyte provided a comprehensive update of the status of its Investigational New Drug Application (IND) to the U.S. Food and Drug Administration (FDA). Among other things, PharmaCyte reported on: (i) the ongoing stability studies of its clinical trial product CypCaps; (ii) the additional studies it has commenced in response to the FDA’s requests related to the clinical hold; (iii) the exact sequence of the Cytochrome P450 2B1 gene in its clinical trial product; (iv) the biocompatibility studies it has completed and that are underway; (v) micro-compression and swelling assays being conducted on its clinical trial product; (vi) break force and glide testing on its clinical trial product; (vii) studies to show that its clinical trial product is not adversely affected by the catheters interventional radiologists use to deliver the CypCaps to a patient; and (vii) tests to show that its clinical trial product is not affected by the contrast medium used by interventional radiologists to help guide the implantation of the CypCaps into a patient.
Recent Highlights—Financial:

As of January 31, 2022, PharmaCyte’s cash balance and total assets were approximately $87 million.

On January 31, 2022, PharmaCyte’s total stockholder equity was approximately $91 million.

PharmaCyte’s "Other Expenses" decreased by approximately $45,000 and $68,000 for the three and nine months ended January 31, 2022.

Operating expenses increased for the three months ended January 31, 2022, by approximately $99,000 and $297,000 for the nine months ended January 31, 2022, as compared to the prior fiscal year, due to costs associated with: (i) research and development (R&D); (ii) an uplist to Nasdaq Capital Markets; (iii) the closing of two public offerings for approximately $90 million; and (iv) and conducting studies related to lifting the FDA’s clinical hold on PharmaCyte’s proposed treatment for LAPC.

PharmaCyte’s R&D expenses increased from the start of its fiscal year to about $526,000 to date. The two capital raises PharmaCyte conducted in August 2021 allowed for these necessary expenses to be possible.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On March 16, 2022 Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported a corporate and financial update for the fourth quarter and full year ended December 31, 2021 (Press release, Athenex, MAR 16, 2022, View Source [SID1234610163]).

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"We are pleased to announce our new vision for the future of Athenex, by deploying our resources and harnessing our expertise toward the advancement of our promising cell therapy programs. The encouraging data that we have generated so far, along with our belief that cell therapy is driving the next major innovation cycle in cancer treatment, give us confidence in our new strategy," said Johnson Lau, Chief Executive Officer of Athenex. "We are taking action to swiftly redirect our resources and have begun executing on cost savings measures to right-size the company and support our transformation to a lean, focused cell therapy company."

Corporate Developments

Key Program and Business Updates

Focus R&D resources on developing cell therapy programs
Discontinue oral discovery program apart from oral paclitaxel
Reducing operating expenses: With the objective of extending its cash runway, the Company is implementing cost cutting initiatives and workforce reduction to lower operating expenses by over 50%
Plan to monetize non-core assets consistent with new focus and strategy of company
Key Anticipated Milestones

Update on ANCHOR data previously presented at ASH (Free ASH Whitepaper) in 2021 at ASTCT on April 23-26, 2022
Update from KUR-501, our autologous program in pediatric neuroblastoma at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting on May 16-19, 2022
Preclinical data presentation of allogeneic GPC3 CAR-NKT cell program in liver cancer at ASCO (Free ASCO Whitepaper) on June 3-7, 2022
Expand CD19 CAR-NKT ANCHOR study to a multi-center study under the newly allowed IND
Data update from KUR-502 at American Society of Hematology (ASH) (Free ASH Whitepaper) on December 10-13, 2022
Potential regulatory update from MHRA for Oral Paclitaxel in metastatic breast cancer (mBC) in UK
Data from I-SPY 2 trial of Oral Paclitaxel in combination with dostarlimab in neoadjuvant breast cancer expected in 2H22
IND filing for KUR-503, our allogeneic CAR-NKT program in liver cancer in 1H23
Fourth Quarter 2021 and Recent Business Highlights

Clinical Programs

Cell Therapy

Observed ORR of 80% with 3 CRs and 1 PR out of 5 evaluable patients in data presented from Phase 1 ANCHOR trial of KUR-502 in relapsed/refractory CD19-positive leukemia and lymphoma at the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting in December.
Announced agreement with the National Cancer Institute (NCI) to license TCRs targeting KRAS, TP53, and EGFR to combine with our allogeneic platform for application in solid tumors
Received IND clearance from the FDA to expand Phase 1 ANCHOR study of KUR-502 to up to 12 clinical sites
Orascovery

Initiated the expansion portion of Phase 1/2 trial of Oral Paclitaxel in combination with pembrolizumab in (Non-small Cell Lung Cancer) NSCLC
Commercial Update

Klisyri (tirbanibulin)

Commercial Partner Almirall reported 3% market share the U.S. and expects gains to continue
Over 2,000 HCPs have prescribed Klisyri since its launch, and it has access to over 70 million covered lives within the commercial space
Almirall expects Medicare Part D coverage in 2022
Specialty Pharmaceutical Business

Athenex Pharmaceutical Division (APD) currently markets a total of 29 products with 54 SKUs.
Athenex Pharma Solutions (APS) currently markets 5 products with 16 SKUs
Fourth Quarter and Full Year 2021 Financial Highlights

Revenues from product sales increased to $23.5 million for the three months ended December 31, 2021, from $21.8 million for the three months ended December 31, 2020, an increase of $1.7 million or 8%. Product sales for the full year 2021 were $92.3 million, down from $105.3 million in 2020, which represents a 12% decrease. This decrease was primarily attributable to non-recurring COVID-related sales in 2020 and international supply chain disruptions caused by COVID-19.

License fees and other revenue for three months and year ended December 31, 2021 were $1.5 million and $27.9 million, respectively, compared to $28.0 thousand and $39.1 million, respectively, for the same periods in 2020.

Cost of sales for the three months ended December 31, 2021 totaled $20.7 million, an increase of $2.4 million, or 13%, as compared to $18.3 million for the three months ended December 31, 2020. Cost of sales totaled $82.4 million for the full year in 2021, a decrease of 14% as compared to $95.4 million for the full year in 2020. The decrease in our cost of specialty product sales was in-line with the decrease in revenue partially offset by corresponding increases in 503B, Contract Manufacturing, and API product sales.

R&D expenses totaled $18.3 million for the three months ended December 31, 2021, in-line with expenses for the three months ended December 31, 2020. R&D expenses totaled $80.2 million for the full year in 2021, an increase of 6% as compared to $75.9 million for the full year in 2020. This increase is primarily due to an increase in costs related to Oral Paclitaxel, drug licensing costs, and cell therapy costs.

SG&A expenses totaled $13.4 million for the three months ended December 31, 2021, a decrease of 57% as compared to $31.4 million for the three months ended December 31, 2020. SG&A expenses totaled $72.6 million for the full year in 2021, a decrease of 22%, as compared to $92.9 million for the full year in 2020. This was primarily due to a $24.8 million decrease of costs for preparing to commercialize Oral Paclitaxel as significant pre-launch activities occurred in 2020 and slowed upon receipt of the Complete Response Letter in February 2021, offset by increased operating costs, professional fees and compensation expense.

The Company recorded impairment of $69.4 million, including goodwill impairment of $67.7 million, during the year ended December 31, 2021, based on the results of a quantitative goodwill impairment test for our reporting units.

Interest expense totaled $5.1 million and $4.4 million for the three months ended December 31, 2021 and 2020, respectively. Interest expense for the year ended December 31, 2021 totaled $20.7 million, an increase of $9.5 million, as compared to $11.2 million for the year ended December 31, 2020, primarily due to increased borrowings. Interest expense in the current period was incurred from the Senior Credit Agreement with Oaktree, while interest expense in the prior period was primarily incurred from debt under a former credit agreement with Perceptive Advisors LLC and its affiliates.

Income tax benefit (expense) for the three months ended December 31, 2021 amounted to ($15.0) thousand, compared to income tax expense of ($8.0) thousand for the same period in 2020. Income tax benefit totaled $10.6 million and income tax expense totaled ($4.1) million for the full year in 2021 and 2020, respectively. The income tax benefit in the current year is primarily the result of taxable temporary difference due to the deferred tax liability recognized for the indefinite lived intangible assets acquired in connection with the acquisition of Kuur’s in-process research and development. This taxable temporary difference is considered a source of taxable income to support the realization of deferred tax assets from the acquirer which resulted in a reversal of our valuation allowance. The income tax expense in the prior year was primarily attributable to foreign income tax withholdings on our revenue earned under our out-license arrangements.

Net loss attributable to Athenex for the three months and year ended December 31, 2021 were $104.4 million and $199.8 million, respectively, or ($0.95) and ($1.92) per diluted share, respectively, as compared to a net loss of $49.5 million and $146.2 million, or ($0.53) and ($1.72) per diluted share, for the same periods in 2020.

For further details on the Company’s financial results, including the results for the full year ended December 31, 2021, refer to the Form 10K filed with the SEC.

2022 Financial Guidance

Athenex’s product sales in 2021 amounted to $92.3 million, which represents 9% growth over the prior-year period, excluding one-time international sales of $21.0 million relating to the COVID pandemic. The Company expects 2022 year-over-year product sales growth to be in the range of 15-20%. While the Company does not give quarterly guidance, product sales are expected to increase throughout the year, as new products are launched.

Cash Conservation Update

As of December 31, 2021, the company had cash and cash equivalents of $35.2 million, restricted cash of $16.5 million, and short-term investments of $10.2 million, for a total of $61.9 million. The Company is implementing cost saving programs and monetizing non-core assets, resulting in plans to extend cash runway in 2022.

Conference Call and Webcast Information

Athenex will host a conference call and live audio webcast today, Wednesday, March 16, 2022, at 4:30 p.m. Eastern Time to discuss the financial results and provide a business update.

To participate in the call, dial either the domestic or international number fifteen minutes before the conference call begins:

The live conference call and replay can also be accessed via audio webcast here and on the Investor Relations section of the Company’s website under "Events and Presentations", located at View Source

On March 16, 2022 Provectus (OTCQB: PVCT) reported that data from an ongoing clinical trial of investigational cancer immunotherapy PV-10 (rose bengal sodium) for the treatment of neuroendocrine tumors (NET) metastatic to the liver (mNET) refractory to somatostatin analogs (SSAs) and peptide receptor radionuclide therapy (PRRT) (NCT02693067) was presented at the annual conference of the European Neuroendocrine Tumor Society (ENETS), held from March 10-11, 2022 in a hybrid setting in Barcelona, Spain and online (Press release, Provectus Biopharmaceuticals, MAR 16, 2022, View Source [SID1234610179]).

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The oral presentation was made by the principal investigator of the clinical trial’s single center at The Queen Elizabeth Hospital (TQEH) in Adelaide, Australia: Tim Price, MBBS, DHlthSc (Medicine), FRACP, Head of Clinical Oncology Research and Chair of the combined Hematology and Medical Oncology Unit at TQEH, and Clinical Professor in the Faculty of Medicine at the University of Adelaide.

Highlights from the 2022 ENETS Presentation:

Patient characteristics
N = 12 patients: 50% male; median age of 66 years (range 47-79)
Primary tumor sites: 7 small bowel (58%), 3 pancreas (25%), 1 caecal (8%), and 1 unknown (8%; likely pancreas)
NET grades: 5 Grade 1 (42%) and 7 Grade 2 (58%)
All patients were refractory to SSA (100%), and 11 received PRRT (92%) as part of their prior treatment
All patients had symptomatic, progressive disease
Baseline chromogranin A (CgA): median 1,585 µg/L (range 35-10,370)
PV-10 treatment
4 patients (33%) received more than 1 dose (range 2-4)
8 patients (67%) received 1 dose
Safety
All treatment-emergent adverse events (TEAEs) were Grade 1 or 2, primarily injection site pain (75%)
Single subjects experienced Grade 3 TEAEs of photosensitivity reaction or transaminases increased
Efficacy
Median progression-free survival (mPFS): 9.4 months (range 1.0-41.8)
Median overall survival (mOS): 22.5 months (range 5.5-42.3); 4 patients alive
Subgroup analysis of primary NET histology, pancreas vs intestine: mPFS 2.7 months vs 19.7 months; mOS 11.8 months vs 25.5 months
Quality of life (QOL)
Stable or improved health status and symptoms after 3 months in most patients
A recording of Dr. Price’s presentation and a copy of his slides are available on Provectus’ website at View Source

Dominic Rodrigues, Vice Chair of the Company’s Board of Directors, said, "Study data continue to show encouraging local and systemic disease control, as well as symptom control, in a heavily pre-treated population and to support a role for monotherapy PV-10 as a treatment for neuroendocrine cancer patients who fail standard therapy."

Mr. Rodrigues added, "PV-10-led combination therapies for neuroendocrine cancer patients may represent opportunities to improve quality of life and clinical outcomes. Front-line approaches may include combining liver-directed PV-10 therapy with systemically-delivered immune checkpoint inhibitors to enhance PV-10’s immune mechanisms, or using PV-10 to enhance the activity of cytotoxic treatments such as peptide receptor radionuclide therapy."

About PV-10

Intralesional (IL) administration of PV-10 for the treatment of solid tumor cancers can yield immunogenic cell death within hours of tumor injection, and induce tumor-specific reactivity in circulating T cells within days. This PV-10-induced functional T cell response may be enhanced and boosted in combination with immune checkpoint blockade (CB). In CB-refractory disease, PV-10 may restore disease-specific T cell function. IL PV-10 has been administered to over 450 patients with melanoma and cancers of the liver in both monotherapy and combination therapy settings. IL PV-10 is administered under visual, tactile, or ultrasound guidance to superficial malignancies, and under CT or ultrasound guidance to visceral hepatic tumors.

Systemic administration of PV-10 is undergoing preclinical study as prophylactic and therapeutic treatments for high-risk and refractory adult solid tumor cancers, and as a therapeutic treatment for relapsed and refractory blood cancers.

On March 16, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality services to biotechnology and pharmaceutical companies, reported that the company will participate in the KeyBanc Capital Markets Life Sciences & MedTech Forum. Nick Green, president and chief executive officer of Avid Bioservices, will be the featured speaker in a fireside chat at the conference, which will take place March 22-23, 2022 in a virtual format (Press release, Avid Bioservices, MAR 16, 2022, View Source [SID1234610164]).

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Details of the company’s participation are as follows:

KeyBanc Capital Markets Life Sciences & MedTech Forum
Conference Date: March 22-23, 2022
Fireside Chat Time/Date: 1:30 – 2:05 p.m. Eastern on Wednesday, March 23, 2022
Format: Virtual Conference; webcast available
To listen to the live webcast of the KeyBanc fireside chat, or access the archived webcast, please visit: View Source

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.