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]).

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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.

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]).

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"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 United Therapeutics Corporation (Nasdaq: UTHR) reported that Dr. Martine Rothblatt, Chairman and Chief Executive Officer of United Therapeutics, will provide an overview and update on the company’s business during a fireside chat session at the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit (Press release, United Therapeutics, SEP 16, 2020, View Source;MedTech-Summit/default.aspx [SID1234565246]).

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The presentation will take place on Wednesday, September 23, 2020, from 11:40 a.m. to 12:20 p.m., Eastern Daylight Time, and can be accessed via a live webcast on the United Therapeutics website at View Source An archived, recorded version of the presentation will be available approximately 24 hours after the session ends and can be accessed at the same location for 90 days.

On September 16, 2020 Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, reported updated results from the ongoing investigator-initiated Phase 1/2 FRAME study evaluating VS-6766, its RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor, which demonstrated robust response rates, duration of response and a favorable safety profile in patients with low-grade serous ovarian cancer (LGSOC) (Press release, Verastem, SEP 16, 2020, View Source [SID1234565244]). These data will be presented in a virtual oral presentation today by Dr. Udai Banerji from The Institute of Cancer Research and The Royal Marsden at the 2nd Annual RAS-Targeted Drug Development Summit.

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"Existing treatments for patients with LGSOC are limited by either 10-25% response rates and/or increased toxicities, leading to high discontinuation rates. The FRAME data being presented today continue to demonstrate that RAF/MEK inhibition combined with FAK inhibition is well tolerated with a 56% overall response rate (ORR) in patients with KRAS-G12 mutant LGSOC and a 41% ORR in the overall LGSOC population. These data are still actively maturing with more than half of the patients still on treatment as of the data cutoff date, and responses in this patient population tend to deepen over time," said Dan Paterson, President and Chief Operating Officer of Verastem Oncology. "The response rates from this expanded data set are highly encouraging, consistent with the prior positive data from this study, and continue to speak to the significant potential of the VS-6766/defactinib combination for patients battling LGSOC."

Verastem recently met with the Food and Drug Administration (FDA), and the FDA is supportive of the Company’s adaptive study design for the planned Phase 2 registration-directed trials evaluating VS-6766 and defactinib in patients with recurrent LGSOC. Verastem expects to commence registration-directed clinical trials in both recurrent LGSOC and KRAS mutant non-small cell lung cancer by the end of 2020. Assuming a positive outcome from these registration-directed trials, Verastem expects to submit New Drug Applications to the FDA requesting accelerated approval for the VS-6766/defactinib combination in both LGSOC and KRAS mutant NSCLC.

Updated Phase 1/2 FRAME Study Results in Patients with LGSOC

Among the patients with LGSOC (n=17), the overall response rate (ORR) was 41% (7 of 17 patients), all partial responses (PRs). Among the patients with KRAS-G12 mutant LGSOC (n=9), the ORR was 56% (5 of 9 patients). Of the seven patients who responded, five had received one or more prior MEK inhibitors. In patients with KRAS mutant LGSOC receiving the recommended Phase 2 dosing (RP2D) regimen, the ORR was 50% (3 of 6 patients). The LGSOC cohort of the FRAME study remains ongoing, with 53% (9 of 17 patients) still on study as of the data cutoff date of August 17, 2020, with three patients on treatment for two years or more.

The most common Grade ≥3 treatment-related adverse events (TEAEs) observed for the recommended Phase 2 dosing regimen were rash (4%) and elevated creatine kinase (4%). No patients discontinued from the FRAME study due to TEAEs.

The novel, intermittent, combination dosing schedule used in the FRAME study continues to show encouraging clinical activity in patients with KRAS mutant LGSOC, including in patients who had previously progressed following treatment with a MEK inhibitor.

"These updated safety and efficacy results in both KRAS mutant LGSOC as well as the overall LGSOC population are highly encouraging. Of particular note in this early look at the data, is the strong, 50% response rate, durability, and tumor reduction seen in patients with KRAS mutant LGSOC receiving the recommended Phase 2 dosing (RP2D) regimen, which is the regimen we will be taking into our upcoming registration-directed study," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "With nine out of 11 patients at RP2D active in the study and responses still developing, we look forward to continued data outputs from this study and we remain on track to commence Phase 2 registration-directed trials in both LGSOC and KRAS mutant NSCLC by the end of this year."

Preclinical Results from Studies Investigating VS-6766 and Defactinib in Combination with G12C Inhibitors

KRAS-G12C inhibitors may benefit from novel combination approaches to enhance their inhibition of the ERK signaling pathway. In the preclinical results that will be presented today at the meeting, VS-6766 showed synergy with KRAS-G12C inhibitors in reducing cancer cell viability across a panel of KRAS-G12C mutant NSCLC and colorectal cancer (CRC) cell lines. This enhanced cellular anti-cancer activity of the combination correlated with deeper and more durable inhibition of ERK pathway signaling relative to G12C inhibition alone. In KRAS-G12C mutant NSCLC models in mice, the RAF/MEK dual inhibitor VS-6766 was more effective than trametinib when compared at equal dose level both alone and in combination with a G12C inhibitor. In the KRAS-G12C NSCLC models tested, the combination of G12C inhibitor with VS-6766 and FAK inhibitor induced tumor regressions of ≥30% in all mice.

"The anti-tumor effects of VS-6766 were generally comparable to those of KRAS-G12C inhibitors in KRAS-G12C NSCLC models in mice and were stronger than the effects of trametinib at a comparable dose," said Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem Oncology. "The tumor regressions observed with the triple combination of VS-6766, FAK inhibitor and G12C inhibitor were particularly striking. These data support clinical evaluation of VS-6766 and defactinib with G12C inhibitors in patients with KRAS-G12C mutant tumors."

About the Phase 1/2 FRAME Study

The FRAME study is an open-label, investigator-initiated study that is designed to assess safety, dose response and preliminary efficacy of the VS-6766/defactinib combination in patients with KRAS mutant solid tumors, including LGSOC, non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). The FRAME study is being led by Dr. Banerji and is being conducted in the United Kingdom. In this study, VS-6766 was administered using a twice-weekly dose escalation schedule and was administered three out of every four weeks. Defactinib was administered using a twice-daily dose escalation schedule, also three out of every four weeks. Dose levels were assessed in three cohorts: cohort 1 (VS-6766 3.2mg, defactinib 200mg); cohort 2a (VS-6766 4mg, defactinib 200mg); and cohort 2b (VS-6766 3.2mg, defactinib 400mg). The recommended Phase 2 dose was determined to be VS-6766 3.2mg, defactinib 200mg. The FRAME study is now expanding to include new cohorts in pancreatic cancer, KRAS mutant endometrial cancer and KRAS-G12V mutant NSCLC.

Details for the RAS-Targeted Drug Development Summit oral presentation are as follows:

Title: Clinical Combinations: Dual RAF-MEK Inhibitor & FAK for Treatment of KRAS Mutant Cancers With a Focus on Low Grade Ovarian Cancer

Lead author: Udai Banerji, The Institute of Cancer Research and The Royal Marsden

Date and Time: Wednesday, September 16, 2020; 3:35 p.m. ET (12:35 p.m. PT)

Title: Synergistic Combinations with the Dual RAF/MEK Inhibitor VS-6766 to Overcome Resistance Mechanisms

Lead author: Jonathan Pachter, Verastem Oncology

Date and Time: Wednesday, September 16, 2020; 12:10 p.m. ET (9:10 a.m. PT)

Conference Call and Webcast Information

The Verastem Oncology management team will host a conference call and webcast on Wednesday, September 16, 2020, at 8:00 AM ET to discuss the updated Phase 1/2 FRAME study data. The call can be accessed by dialing (877) 341-5660 (US and Canada) or (315) 625-3226 (international), five minutes prior to the start of the call and providing the passcode 5278200.

The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company’s website at www.verastem.com. A replay of the webcast will be archived on the Company’s website for 90 days following the call.

About VS-6766

VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

About Defactinib

Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

About the VS-6766/Defactinib Combination

RAS mutant tumors are present in ~30% of all human cancers, have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

The combination of VS-6766 and defactinib has been found to be clinically active in patients with KRAS mt tumors. In an ongoing investigator-initiated Phase 1/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Updated data from this study presented at the 2nd Annual RAS-Targeted Drug Development Summit in September 2020 demonstrated a 56% overall response rate and long duration of therapy among patients with KRAS-G12 mt LGSOC. Based on an observation of higher response rates seen in NSCLC patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study was expanded in August 2020 to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC.

On September 16, 2020 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that Jeff Goater, its chief executive officer, and Robert Ross, M.D., its chief medical officer, will participate in the upcoming Oppenheimer Fall Healthcare Life Sciences & MedTech Summit on September 21, where they will discuss Surface Oncology’s lead programs, SRF617 (targeting CD39) and SRF388 (targeting IL-27), as well as Surface’s emerging preclinical pipeline, highlighted by SRF813 (targeting CD112R, also known as PVRIG) (Press release, Surface Oncology, SEP 16, 2020, View Source [SID1234565243]).

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