On September 16, 2020 Mateon Therapeutics "Mateon" (OTCQB: MATN), a leading developer of TGF-β therapeutics for oncology and COVID-19, reported that the US Food and Drug Administration (FDA) granted our request and designate OXi4503 (combretastatin A1-diphosphate; CA1P) for treatment of acute myeloid leukemia (AML) due to genetic mutations that disproportionately affect pediatric patients as a drug for a "rare pediatric disease," as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)) (Press release, Mateon Therapeutics, SEP 16, 2020, View Source [SID1234565247]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited about this Rare Pediatric Disease designation for AML.", said Dr. Vuong Trieu, Chief Executive Officer of Mateon. "This builds on our previous Rare Pediatric Disease designations for OT-101 and CA4P. It also completed our refocusing of the company to rare pediatric diseases. We are looking forward to initiate clinical programs among these indications post-COVID-19. In the meantime, we are focusing on deploying OT-101 against severe COVID-19 and Artemisinin against COVID-19 in general among various clinical trials globally.".

OXi4503 in combination with standard chemotherapy drug cytarabine was generally well tolerated by adult AML patients and a maximum tolerated dose level of OXi4503 was identified as the recommended dose for further clinical development of this novel two-drug combination. In 26 evaluable AML patients, there were 4 complete remissions (CR/CRi) and one partial remission (PR). The CR responses were associated with >1-year overall survival times. The combination therapy exhibited a manageable toxicity and a promising benefit to risk profile in older adults with relapsed AML. Four of the 5 objective responders were in the ≥65-years poor prognosis age category with adverse cytogenetic features.

Acute leukemia is the most common cancer in children accounting for one-third of all childhood cancers. Acute lymphoblastic leukemia (ALL) accounts for 80% and acute myeloid leukemia (AML) accounts for 15% of all acute leukemia cases in children. Children with AML have a worse prognosis with a 5-year survival rate of 64% than children with ALL who have a 5-year survival rate of ~90% on contemporary risk-adjusted treatment programs. Children with AML who have unfavorable risk factors, such as adverse cytogenetics, have a particularly poor survival outcome even after intensive multimodality therapy and hematopoietic stem cell transplantation. Approximately one-third of children with AML relapse after induction chemotherapy and only one-third of these patients become long-term survivors. Relapsed disease is the greatest challenge to a better survival outcome in AML. Although new drugs have recently been developed against several molecular targets in AML blast cells, the vast majority of relapsed pediatric AML patients still die of leukemia. Therefore, novel therapies are urgently needed for pediatric AML.

About Oxi4503

OXi4503 exhibited single-agent anti-leukemia activity in animal models of AML and in a Phase 1A clinical study for relapsed/refractory (R/R) AML. Notably, the combination of OXi4503 with cytarabine (ARA-C) in xenografted human AML models was more effective than either drug alone. The clinical safety profile of OXi4503 as a single agent has previously been evaluated in Phase 1A clinical trials. In the NCT00977210 Phase 1 dose-finding study in 43 advanced solid tumor patients, OXi4503 doses were escalated from 0.06 to 15.4 mg/m2, and 8.5 mg/m2 was defined as the maximum tolerated dose (MTD). In the NCT01085656 Phase 1A trial designed to evaluate the safety profile, MTD, and recommended Phase 2 dose of OXi4503 in patients with R/RAML and MDS, a total of 18 patients were treated with single-agent OXi4503 and showed a manageable safety profile at single-agent dose levels up to of 7.81 mg/m2 and there was early evidence of possible single-agent anti-AML activity. More recently, a Phase 1B study was performed to evaluate the safety, tolerability, and clinical activity of a combination of OXi4503 and the standard anti-AML drug ARA-C. The combination therapy exhibited a manageable toxicity and a promising benefit to risk profile in adults with relapsed AML. An MTD level of OXi4503 was identified as the recommended dose for further clinical development of this novel two-drug combination. In 26 evaluable AML patients, there were four complete remissions (CR/CRi) and one partial remission. The median overall survival time for the four patients who achieved a CR/CRi was 528 days (95% confidence interval [CI]: 434 – NA), which was significantly longer than the median overall survival time of 113 days (95% CI: 77–172) for the remaining 22 patients who did not achieve a CR (Log rank Chi-square = 11.8, P-value = 0.0006).

About rare pediatric disease voucher program

The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect people aged from birth to 18 years, and that affect fewer than 200,000 people in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application or biologics license application for a product for the prevention or treatment of a rare pediatric disease may be eligible for a voucher, which can be redeemed to obtain priority review for any subsequent marketing application, and may be sold or transferred. In August 2019, AstraZeneca has reportedly paid approximately $95 million to buy a priority review voucher from Swedish Orphan Biovitrum (Sobi) (View Source). Likewise, Biohaven Pharmaceutical Holding Company Ltd. reportedly paid approximately $105 million for a priority review voucher in March 2019 (View Source).

On September 16, 2020 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company") reported it has published a presentation that Joseph C. Papa, chairman and CEO, and Paul S. Herendeen, executive vice president and chief financial officer, are scheduled to present at the virtual 18th Annual Morgan Stanley Global Healthcare Conference today, Sept. 16, 2020, at 1:15 p.m. ET (Press release, Bausch Health, SEP 16, 2020, View Source [SID1234565263]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation provides an update on Bausch Health’s current business recovery in the wake of the COVID-19 pandemic, as well as an update and progress report regarding the Company’s previously disclosed intention to spin off its eye health business.

The presentation is available on the Investor Relations page of the Bausch Health Companies Inc. web site at: View Source A live webcast and audio archive of the event will be also be available for 90 days.

On September 16, 2020 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for the six-month period ended June 30, 2020, and provides an update on progress of its portfolio (Press release, Transgene, SEP 16, 2020, View Source [SID1234621821]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Philippe Archinard, Chairman and Chief Executive Officer of Transgene, commented:

"I would like to thank the teams at Transgene for their remarkable job in advancing all our clinical and preclinical projects in an environment that has been severely disrupted by the Covid-19 pandemic. During the period we delivered positive Phase 1b/2 results with TG4001, which have given us the confidence to progress the clinical development of this therapeutic vaccine against HPV-induced cancers. We intend to provide more detail on these positive Phase 1b/2 study results in the coming months. Our oncolytic virus TG6002 showed positive initial data in a Phase 1 trial, indicating that it induces the production of a chemotherapy agent in the tumor.

These promising results confirm the safety of TG6002 when given intravenously and are highly supportive of the new generations of oncolytic viruses that we are developing based on our exciting Invir.IO platform. Patient inclusion continues in line with expectations in the first trials of the individualized immunotherapy TG4050, which has been generated from our myvac platform. The myvac platform and the launch of these trials earlier this year exemplify our technological leadership in individualized immunotherapies. The data which were presented at the AACR (Free AACR Whitepaper) congress in June highlighted the power of NEC’s artificial intelligence and the integration of the first block chain solution into the myvac production process. In parallel, we successfully produced the first clinical batches of TG4050. The collaboration with AstraZeneca continues with the delivery of new oncolytic viruses. Finally, by selling part of approximately 40% of the stake in Tasly BioPharmaceuticals for $22.2 million, Transgene has the cash resources to fund its activities until 2022." Page 2/9 Promising initial data for TG4001 The analysis of the efficacy data from the Phase 1b/2 trial combining TG4001 with avelumab in HPV16-positive recurrent and/or metastatic malignancies showed a promising clinical activity in the overall study population (34 evaluable patients).

In addition, Transgene identified a selection criterion corresponding to patients showing particularly encouraging clinical activity. For more than 50% of these patients, the disease had not progressed at 12 weeks, compared to an expected median progression-free survival (PFS) of 8 weeks for this population with current treatment regimens or with immune checkpoint inhibitors in monotherapy. Responses are durable in most of the responder patients. Transgene is currently completing translational analyses. Patient follow-up is still ongoing. Complete data will be presented at an upcoming scientific conference. Transgene intends to pursue the development of TG4001 and is actively working on the preparation of a confirmatory clinical study. Advanced technological leadership with the myvac platform Transgene is developing the therapeutic vaccine TG4050, together with NEC. This individualized cancer vaccine is based on the myvac platform and integrates NEC’s artificial intelligence capabilities. The first Phase 1 clinical trials assessing TG4050 in patients with ovarian and head and neck cancers started in January 2020 in Europe and in the United States. NEC is financing 50% of these studies.

The Company has set up an in-house production unit dedicated to the manufacturing of the individualized clinical batches of TG4050 needed for each patient. This unit is operational and complies with good manufacturing practice (GMP) norms. The manufacturing process and unit have been validated and the first clinical batches have been produced.

The myvac platform is being actively promoted as it exemplifies Transgene’s technological leadership in individualized immunotherapies.
✓ Data validating the vaccine design principle and underlining the accuracy of the artificial intelligence used to personalize TG4050 were presented at the AACR (Free AACR Whitepaper) congress (June 2020).
✓ Transgene has implemented the first block chain solution dedicated to the traceability of personalized treatment in clinical trials. This cloud-based solution monitors and orchestrates all of the processes related to the design and manufacturing of Transgene’s individualized therapeutic vaccine TG4050.
✓ Other innovative approaches were integrated into the myvac approach and will be detailed in the coming months. The initial translational data of TG6002 highlight the potential of the Invir.IO platform Initial data from the Phase 1 trial confirm the good tolerability of TG6002 in humans and demonstrate that this Vaccinia Virus, which is the viral backbone on which the Invir.IO platform is based, can reach the tumor and replicate within these cancer cells when administered intravenously. BT-001 is the first oncolytic virus from the Invir.IO platform. A first-in-human trial is being prepared; the trial protocol has been filed in France and in Belgium. Promising preclinical results for BT-001 were presented at the AACR (Free AACR Whitepaper) annual congress (June 2020). The collaboration with AstraZeneca continues with the development of new innovative oncolytic viruses.

AstraZeneca can exercise an option to further develop each of these novel drug candidates. Page 3/9 Summary of key ongoing clinical trials TG4001 + Bavencio (avelumab) Phase 1b/2 Targets: HPV16 E6 and E7 oncoproteins Advanced HPV-positive cancers including oropharyngeal head and neck cancer – 2 nd line
✓ Clinical collaboration with Merck KGaA and Pfizer, for the supply of avelumab
✓ Very promising results; patient follow-up is ongoing  Detailed results will be presented at an upcoming scientific conference  Transgene intends to launch a larger, controlled, confirmatory trial myvac TG4050 Phase 1 Targets: tumor neoantigens
✓ Data demonstrating the high accuracy of AI-based neoantigen prediction technology used to design TG4050 were presented at AACR (Free AACR Whitepaper) Ovarian cancer – after surgery and first-line chemotherapy ✓ Trial authorized in the United States and in France
✓ First patient enrolled in January 2020 – inclusions progressing in line with forecast  First scientific communication in 2021 TG4050 Phase 1 HPV-negative head and neck cancer – after surgery and adjuvant therapy ✓ Trial authorized in the United Kingdom and in France
✓ First patient enrolled in January 2020 – inclusions progressing in line with forecast  First scientific communication in 2021 TG6002 Phase 1/2a Payload: FCU1 for the local production of a 5-FU chemotherapy Gastro-intestinal cancer (colorectal cancer for Phase 2) – Intravenous (IV) administration
Multicenter trial ongoing in Belgium, France and Spain
✓ First findings confirm that 5-FU is produced in the tumor  Dose escalation is ongoing in the Phase 1 part, testing additional dose levels TG6002 Phase 1/2a Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration ✓ Multicenter trial authorized in the United Kingdom
✓ First patient treated in February 2020; enrollment resuming in September 2020 after pausing due to Covid-19  First observations in 2021 Invir.IO BT-001 Phase 1/2 Payload: anti-CTLA4 antibody and GM-CSF cytokine Solid tumors ✓ Collaboration with BioInvent
✓ First clinical trial applications submitted (France and Belgium) ✓ Presentation of very encouraging preclinical results at AACR (Free AACR Whitepaper) 2020  Approval from health authorities expected before the end of 2020 Key Financials The Board of Directors of Transgene met on September 16, 2020 and approved the financial statements for the six-month period ended June 30, 2020.

The Statutory Auditors have conducted a limited review of the interim consolidated financial statements. The half-year financial report is available on Transgene’s website, View Source Page 4/9 Key elements of the income statement (in thousands of euros) June 30, 2020 June 30, 2019 Operating revenues 5,731 4,909 Research and development expenses (13,831) (14,668) General and administrative expenses (3,297) (3,572) Other expenses-(141) Operating expenses (17,128) (18,381) Operating income/(loss) (11,397) (13,472) Financial income/(loss) 9,183 (1,870) Net income/(loss) (2,214) (15,342) Operating revenues amounted to €5.7 million for the first six months of 2020 compared to €4.9 million for the same period in 2019.

• In 2019, the Company entered into a collaboration agreement with AstraZeneca with exclusive licensing options to co-develop oncolytic immunotherapies derived from the Invir.IO platform. As a result, in the first half of 2019 Transgene received €8.9 million (US$10 million) in fees for access to its platform. This initial payment is recognized as revenue based on the stage of completion of the related activities. Over the period, the income recognized under this collaboration agreement was €2.2 million (€0.7 million in the first half of 2019). Of this amount €1.8 million reflects recognition of the initial payment for work done during the period and €0.4 million for the achievement of certain preclinical milestones.

• The research tax credit amounted to €2.9 million for the first half of 2020, compared to €3.1 million for the first half of 2019. Research and Development (R&D) expenses amounted to €13.8 million in the first half of 2020 compared to €14.7 million for the same period in 2019. External expenses for clinical projects decreased to €3.0 million from €4.7 million in the first half of 2019. This decrease is mainly due to a reduction in subcontracted clinical batch production expenses in the first half of 2020 compared to the same period in 2019. General and administrative expenses amounted to €3.3 million for the first half of 2020 compared to €3.6 million for the same period in 2019. Net interest income amounted to a gain of €9.2 million in the first half of 2020 compared to an expense of €1.9 million for the same period in 2019. This change is mainly due to the increase in the fair value of Tasly Biopharmaceuticals shares: in July 2020, the sale of the shares was carried out at a higher price than the acquisition price in July 2018. This sale price was applied to all the shares held. As a consequence, the net comprehensive loss amounted to €2.2 million for the first half of 2020 compared to a loss of €15.3 million for the same period in 2019. As of June 30, 2020, the Company’s cash, cash equivalents and other financial assets amounted to €33.2 million versus €43.3 million as of December 31, 2019. Transgene’s cash burn amounted to €10.1 million in the first half of 2020, compared with €4.1 million for the same period in 2019.

Transgene intends to reimburse the €10 million bank loan from the European Investment Bank in advance of its June 2021 maturity. The Company confirms its financial visibility until 2022. Page 5/9 Partial sale of the stake in Tasly BioPharmaceuticals On August 4, 2020, Transgene announced the receipt of $22.2 million (€19 million) following the sale to a Chinese investment fund of part of its minority stake in Tasly BioPharmaceuticals. This transaction involved the sale of 10.3 million shares of Tasly BioPharmaceuticals (38% of the shares held by Transgene). Following this share sale, Transgene holds 17.1 million shares in Tasly BioPharmaceuticals, equivalent to 1.58% of the Chinese company’s capital. Transgene’s remaining shareholding in Tasly BioPharmaceuticals is valued at approximately $36.9 million based on the price of the current share sale.

At the end of August 2020, Tasly BioPharmaceuticals filed its IPO documentation with the Science and Technology Innovation Board (STIB) of the Shanghai Stock Exchange. Succession of the Chairman and Chief Executive Officer planned at the end of 2020 Philippe Archinard, Chairman and Chief Executive Officer of Transgene, has informed the Board of Directors of his intention to leave his position at the end of 2020 to take up new responsibilities within Institut Mérieux. The Board has acknowledged his decision and proposes that his successor be Hedi Ben Brahim, who has been a Board member of Transgene since May 2019. This decision will be approved at the Board meeting scheduled on December 3, 2020. Philippe Archinard will remain a Board Member of Transgene thereafter.

On September 16, 2020 Zai Lab Limited ("Zai Lab" or the "Company") (NASDAQ:ZLAB), an innovative commercial stage biopharmaceutical company, reported the launch of its Hong Kong public offering (the "Hong Kong Public Offering"), which forms part of the global offering (the "Offering") of 10,564,050 new ordinary shares (the "Offer Shares" or "Shares") and listing of its ordinary shares on the Main Board of The Stock Exchange of Hong Kong Limited (the "Hong Kong Stock Exchange") under the stock code "9688" (Press release, Zai Laboratory, SEP 16, 2020, View Source [SID1234565231]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company’s American depositary shares ("ADSs"), each representing one (1) ordinary share of the Company, will continue to be listed and traded on the Nasdaq Global Market ("Nasdaq"). Investors in the Offering will only be able to purchase ordinary shares and will not be able to take delivery of ADSs. Upon listing in Hong Kong, the ordinary shares listed on the Hong Kong Stock Exchange will be fully fungible with the ordinary shares represented by the ADSs listed on Nasdaq.

The Offering initially comprises 771,700 new Shares under the Hong Kong Public Offering and 9,792,350 new Shares for the international offering (the "International Offering"), representing approximately 7.3% and 92.7% of the total number of Shares in the Offering, respectively, subject to re-allocation and over-allotment. Subject to the level of any oversubscription in the Hong Kong Public Offering and pursuant to the clawback mechanism as described in the prospectus issued in Hong Kong, the total number of shares available under the Hong Kong Public Offering could be adjusted to up to a maximum of 3,591,800 new Shares, representing approximately 34% of the Offer Shares initially available under the Offering. In addition, the Company expects to grant the international underwriters an over-allotment option to purchase up to an additional 1,584,600 new Shares in the International Offering, representing not more than 15.0% of the Offer Shares initially available under the Offering.

The offer price for the Hong Kong Public Offering (the "Hong Kong Offer Price") will be not more than HK$648.00 per share (the "Maximum Offer Price"). The offer price for the International Offering tranche of the Offering (the "International Offer Price") may be set at a level higher than the Hong Kong Offer Price. The Company is expected to set the International Offer Price by September 22, 2020 Hong Kong time by making reference to, among other factors, the closing price of the ADSs on Nasdaq on the last trading day on or before September 21, 2020 and investor demand during the marketing process. The final Hong Kong Offer Price will be set at the lower of the final International Offer Price and the Maximum Offer Price of HK$648.00 per share. Shares will be traded in board lots of 50 shares.

The Company plans to use the net proceeds from the Offering for its core products, through strengthening R&D efforts and enhancing its commercialization capabilities, advancing its ongoing and planned clinical trials and preparation for registration filings of other drug candidates in its pipeline, exploring new global licensing and collaboration opportunities, funding working capital and other general corporate purposes.

Fully Electronic Application Process for the Hong Kong Public Offering

Zai Lab has decided to adopt a fully electronic application process for the Hong Kong Public Offering, with no printed copies of prospectuses or application forms to the public in relation to the Hong Kong Public Offering. A fully electronic application process is consistent with the way in which the Company’s users and stakeholders engage and interact with each other and the Company. As a company which has been highly committed to environmental, social and corporate responsibility matters since its founding, the Company believes such method will also help mitigate the environmental impact of printing and minimize the exploitation of natural resources, among others. The prospectus is available at the website of the Hong Kong Stock Exchange at www.hkexnews.hk and the Company’s website at www.zailaboratory.com.

The Company encourages applicants for the Hong Kong Public Offering to view its prospectus and apply online through the White Form eIPO service at www.eipo.com.hk, or apply through the CCASS EIPO service. The Hong Kong Public Offering will commence at 9:00 a.m. on Thursday, September 17, 2020 Hong Kong time and will close at 12:00 noon on Tuesday, September 22, 2020 Hong Kong time.

Potential applicants may call the enquiry hotline of Computershare Hong Kong Investor Services Limited if they have any question about making applications for the Hong Kong Offer Shares. The hotline number is +852 2862 8646, and will be open from 9:00 a.m. to 9:00 p.m. on Thursday, September 17, 2020, Friday, September 18, 2020 and Monday, September 21, 2020, from 9:00 a.m. to 6:00 p.m. on Saturday, September 19, 2020 and Sunday, September 20, 2020, and from 9:00 a.m. to 12:00 noon on Tuesday, September 22, 2020 Hong Kong time.

J.P. Morgan Securities (Far East) Limited (or its affiliates, as the case may be), Goldman Sachs (Asia) L.L.C. and Citigroup Global Markets Asia Limited (or its affiliate, as the case may be) are the joint sponsors, joint global coordinators, joint bookrunners and joint lead managers for the proposed Offering.

The International Offering is being made only by means of a preliminary prospectus supplement dated September 16, 2020 and the accompanying prospectus included in an automatic shelf registration statement on Form F-3ASR filed with the U.S. Securities and Exchange Commission (the "SEC") on March 29, 2019 and was subsequently amended and became automatically became effective upon filing with the SEC on January 21, 2020. The registration statement on Form F-3ASR and the preliminary prospectus supplement are available at the SEC website at: View Source Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from: (i) J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 1-866-803-9204 or by email at [email protected], (ii) Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282 or by telephone at 1-866-471-2526, or (iii) Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 1-800-831-9146 or by email at [email protected].

The proposed Offering is subject to market and other conditions, and there can be no assurance as to whether or when the Offering may be completed, or as to the actual size or terms of the Offering. This press release shall not constitute an offer to sell or the solicitation of an offer or an invitation to buy any securities of the Company, nor shall there be any offer or sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. This press release does not constitute a prospectus (including as defined under the laws of Hong Kong) and potential investors should read the prospectus of the Company for detailed information about the Company and the proposed offering, before deciding whether or not to invest in the Company. This press release has not been reviewed or approved by the Hong Kong Stock Exchange or the Securities and Futures Commission of Hong Kong.

The price of the Shares of the Company may be stabilized in accordance with the Securities and Futures (Price Stabilization) Rules. The details of the intended stabilization and how it will be regulated under the Securities and Futures Ordinance (Chapter 571 of the laws of Hong Kong) will be contained in the prospectus of the Company dated September 17, 2020

On September 16, 2020 Transgene (Paris:TNG) (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported its financial results for the six-month period ended June 30, 2020, and provides an update on progress of its portfolio (Press release, Transgene, SEP 16, 2020, View Source [SID1234565248]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Philippe Archinard, Chairman and Chief Executive Officer of Transgene, commented:

"I would like to thank the teams at Transgene for their remarkable job in advancing all our clinical and preclinical projects in an environment that has been severely disrupted by the Covid-19 pandemic.

During the period we delivered positive Phase 1b/2 results with TG4001, which have given us the confidence to progress the clinical development of this therapeutic vaccine against HPV-induced cancers. We intend to provide more detail on these positive Phase 1b/2 study results in the coming months.

Our oncolytic virus TG6002 showed positive initial data in a Phase 1 trial, indicating that it induces the production of a chemotherapy agent in the tumor. These promising results confirm the safety of TG6002 when given intravenously and are highly supportive of the new generations of oncolytic viruses that we are developing based on our exciting Invir.IO platform.

Patient inclusion continues in line with expectations in the first trials of the individualized immunotherapy TG4050, which has been generated from our myvac platform. The myvac platform and the launch of these trials earlier this year exemplify our technological leadership in individualized immunotherapies. The data which were presented at the AACR (Free AACR Whitepaper) congress in June highlighted the power of NEC’s artificial intelligence and the integration of the first block chain solution into the myvac production process. In parallel, we successfully produced the first clinical batches of TG4050.

The collaboration with AstraZeneca continues with the delivery of new oncolytic viruses.

Finally, by selling part of approximately 40% of the stake in Tasly BioPharmaceuticals for $22.2 million, Transgene has the cash resources to fund its activities until 2022."

Promising initial data for TG4001

The analysis of the efficacy data from the Phase 1b/2 trial combining TG4001 with avelumab in HPV16-positive recurrent and/or metastatic malignancies showed a promising clinical activity in the overall study population (34 evaluable patients).

In addition, Transgene identified a selection criterion corresponding to patients showing particularly encouraging clinical activity. For more than 50% of these patients, the disease had not progressed at 12 weeks, compared to an expected median progression-free survival (PFS) of 8 weeks for this population with current treatment regimens or with immune checkpoint inhibitors in monotherapy. Responses are durable in most of the responder patients. Transgene is currently completing translational analyses. Patient follow-up is still ongoing. Complete data will be presented at an upcoming scientific conference.

Transgene intends to pursue the development of TG4001 and is actively working on the preparation of a confirmatory clinical study.

Advanced technological leadership with the myvac platform

Transgene is developing the therapeutic vaccine TG4050, together with NEC. This individualized cancer vaccine is based on the myvac platform and integrates NEC’s artificial intelligence capabilities.

The first Phase 1 clinical trials assessing TG4050 in patients with ovarian and head and neck cancers started in January 2020 in Europe and in the United States. NEC is financing 50% of these studies.

The Company has set up an in-house production unit dedicated to the manufacturing of the individualized clinical batches of TG4050 needed for each patient. This unit is operational and complies with good manufacturing practice (GMP) norms. The manufacturing process and unit have been validated and the first clinical batches have been produced.

The myvac platform is being actively promoted as it exemplifies Transgene’s technological leadership in individualized immunotherapies.

✓ Data validating the vaccine design principle and underlining the accuracy of the artificial intelligence used to personalize TG4050 were presented at the AACR (Free AACR Whitepaper) congress (June 2020).
✓ Transgene has implemented the first block chain solution dedicated to the traceability of personalized treatment in clinical trials. This cloud-based solution monitors and orchestrates all of the processes related to the design and manufacturing of Transgene’s individualized therapeutic vaccine TG4050.
✓ Other innovative approaches were integrated into the myvac approach and will be detailed in the coming months.

The initial translational data of TG6002 highlight the potential of the Invir.IO platform

Initial data from the Phase 1 trial confirm the good tolerability of TG6002 in humans and demonstrate that this Vaccinia Virus, which is the viral backbone on which the Invir.IO platform is based, can reach the tumor and replicate within these cancer cells when administered intravenously.

BT-001 is the first oncolytic virus from the Invir.IO platform. A first-in-human trial is being prepared; the trial protocol has been filed in France and in Belgium. Promising preclinical results for BT-001 were presented at the AACR (Free AACR Whitepaper) annual congress (June 2020).

The collaboration with AstraZeneca continues with the development of new innovative oncolytic viruses. AstraZeneca can exercise an option to further develop each of these novel drug candidates.

Summary of key ongoing clinical trials

TG4001
+ Bavencio

(avelumab)
Phase 1b/2

Targets: HPV16 E6 and E7 oncoproteins

Advanced HPV-positive cancers including oropharyngeal head and neck cancer – 2nd line

✓ Clinical collaboration with Merck KGaA and Pfizer, for the supply of avelumab

✓ Very promising results; patient follow-up is ongoing

→ Detailed results will be presented at an upcoming scientific conference

→ Transgene intends to launch a larger, controlled, confirmatory trial

myvac

TG4050

Phase 1

Targets: tumor neoantigens
✓ Data demonstrating the high accuracy of AI-based neoantigen prediction technology used to design TG4050 were presented at AACR (Free AACR Whitepaper)
Ovarian cancer – after surgery and first-line chemotherapy
✓ Trial authorized in the United States and in France
✓ First patient enrolled in January 2020 – inclusions progressing in line with forecast
→ First scientific communication in 2021

TG4050

Phase 1

HPV-negative head and neck cancer – after surgery and adjuvant therapy
✓ Trial authorized in the United Kingdom and in France
✓ First patient enrolled in January 2020 – inclusions progressing in line with forecast
→ First scientific communication in 2021

TG6002
Phase 1/2a

Payload: FCU1 for the local production of a 5-FU chemotherapy

Gastro-intestinal cancer (colorectal cancer for Phase 2) – Intravenous (IV) administration

✓ Multicenter trial ongoing in Belgium, France and Spain

✓ First findings confirm that 5-FU is produced in the tumor

→ Dose escalation is ongoing in the Phase 1 part, testing additional dose levels

TG6002
Phase 1/2a

Colorectal cancer with liver metastasis – Intrahepatic artery (IHA) administration

✓ Multicenter trial authorized in the United Kingdom

✓ First patient treated in February 2020; enrollment resuming in September 2020 after pausing due to Covid-19

→ First observations in 2021

Invir.IO
BT-001
Phase 1/2

Payload: anti-CTLA4 antibody and GM-CSF cytokine

Solid tumors

✓ Collaboration with BioInvent

✓ First clinical trial applications submitted (France and Belgium)

✓ Presentation of very encouraging preclinical results at AACR (Free AACR Whitepaper) 2020

→ Approval from health authorities expected before the end of 2020

Key Financials

The Board of Directors of Transgene met on September 16, 2020 and approved the financial statements for the six-month period ended June 30, 2020. The Statutory Auditors have conducted a limited review of the interim consolidated financial statements.

The half-year financial report is available on Transgene’s website, View Source

Key elements of the income statement

(in thousands of euros)

June 30, 2020

June 30, 2019

Operating revenues

5,731

4,909

Research and development expenses

(13,831)

(14,668)

General and administrative expenses

(3,297)

(3,572)

Other expenses

–

(141)

Operating expenses

(17,128)

(18,381)

Operating income/(loss)

(11,397)

(13,472)

Financial income/(loss)

9,183

(1,870)

Net income/(loss)

(2,214)

(15,342)

Operating revenues amounted to €5.7 million for the first six months of 2020 compared to €4.9 million for the same period in 2019.

In 2019, the Company entered into a collaboration agreement with AstraZeneca with exclusive licensing options to co-develop oncolytic immunotherapies derived from the Invir.IO platform. As a result, in the first half of 2019 Transgene received €8.9 million (US$10 million) in fees for access to its platform. This initial payment is recognized as revenue based on the stage of completion of the related activities. Over the period, the income recognized under this collaboration agreement was €2.2 million (€0.7 million in the first half of 2019). Of this amount €1.8 million reflects recognition of the initial payment for work done during the period and €0.4 million for the achievement of certain preclinical milestones.
The research tax credit amounted to €2.9 million for the first half of 2020, compared to €3.1 million for the first half of 2019.
Research and Development (R&D) expenses amounted to €13.8 million in the first half of 2020 compared to €14.7 million for the same period in 2019. External expenses for clinical projects decreased to €3.0 million from €4.7 million in the first half of 2019. This decrease is mainly due to a reduction in subcontracted clinical batch production expenses in the first half of 2020 compared to the same period in 2019.

General and administrative expenses amounted to €3.3 million for the first half of 2020 compared to €3.6 million for the same period in 2019.

Net interest income amounted to a gain of €9.2 million in the first half of 2020 compared to an expense of €1.9 million for the same period in 2019. This change is mainly due to the increase in the fair value of Tasly Biopharmaceuticals shares: in July 2020, the sale of the shares was carried out at a higher price than the acquisition price in July 2018. This sale price was applied to all the shares held.

As a consequence, the net comprehensive loss amounted to €2.2 million for the first half of 2020 compared to a loss of €15.3 million for the same period in 2019.

As of June 30, 2020, the Company’s cash, cash equivalents and other financial assets amounted to €33.2 million versus €43.3 million as of December 31, 2019.

Transgene’s cash burn amounted to €10.1 million in the first half of 2020, compared with €4.1 million for the same period in 2019.

Transgene intends to reimburse the €10 million bank loan from the European Investment Bank in advance of its June 2021 maturity.

The Company confirms its financial visibility until 2022.

Partial sale of the stake in Tasly BioPharmaceuticals

On August 4, 2020, Transgene announced the receipt of $22.2 million (€19 million) following the sale to a Chinese investment fund of part of its minority stake in Tasly BioPharmaceuticals. This transaction involved the sale of 10.3 million shares of Tasly BioPharmaceuticals (38% of the shares held by Transgene).

Following this share sale, Transgene holds 17.1 million shares in Tasly BioPharmaceuticals, equivalent to 1.58% of the Chinese company’s capital. Transgene’s remaining shareholding in Tasly BioPharmaceuticals is valued at approximately $36.9 million based on the price of the current share sale.

At the end of August 2020, Tasly BioPharmaceuticals filed its IPO documentation with the Science and Technology Innovation Board (STIB) of the Shanghai Stock Exchange.

Succession of the Chairman and Chief Executive Officer planned at the end of 2020

Philippe Archinard, Chairman and Chief Executive Officer of Transgene, has informed the Board of Directors of his intention to leave his position at the end of 2020 to take up new responsibilities within Institut Mérieux. The Board has acknowledged his decision and proposes that his successor be Hedi Ben Brahim, who has been a Board member of Transgene since May 2019. This decision will be approved at the Board meeting scheduled on December 3, 2020. Philippe Archinard will remain a Board Member of Transgene thereafter.

A conference call in English is scheduled today, on September 16th, 2020, at 6:00 p.m. CET.