On February 26, 2021 Ipsen (Euronext: IPN; ADR: IPSEY) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for Cabometyx (cabozantinib) in combination with Bristol Myers Squibb’s Opdivo (nivolumab) for the first-line treatment of advanced renal cell carcinoma (aRCC) (Press release, Ipsen, FEB 26, 2021, View Source [SID1234575733]). The European Commission, which has the authority to approve medicines for the European Union (E.U.), will now review the CHMP recommendation and a final decision on the application in the E.U. is expected in the coming months.

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"Advanced renal cell carcinoma is a disease that significantly impacts the lives of people around the world. We’re proud to be able to share that the CHMP has confirmed a positive recommendation for Cabometyx in combination with Opdivo, bringing this impactful new treatment option one step closer for patients," said Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen. "At Ipsen, we are committed to progressing treatment for cancers which have an urgent need for additional therapeutic options and this recommendation marks an important milestone in achieving this."

The CHMP adopted the positive opinion based on results from the pivotal Phase III CheckMate -9ER trial, which demonstrated significant and clinically meaningful improvements in progression-free survival (PFS), overall survival (OS) and objective response rate (ORR) compared to sunitinib, with consistent efficacy benefits observed across key subgroups of patients.1 Cabometyx combined with Opdivo was well tolerated and reflected the known safety profiles of the immunotherapy and tyrosine kinase inhibitor (TKI) components in first-line advanced RCC.1 The full data from the CheckMate -9ER trial were presented during a Presidential Symposium at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020. At the recent American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Genitourinary Cancers Symposium (ASCO GU), additional data from the CheckMate -9ER trial were also presented, highlighting sustained superior efficacy with a longer duration of follow-up as well as significantly improved health-related quality of life outcomes for the combination versus sunitinib.2,3

Ipsen and its partners have shared the CheckMate -9ER data with regulatory authorities around the world. The combination of Cabometyx and Opdivo was approved by the U.S. Food and Drug Administration (FDA) as first-line treatment for patients living with advanced renal cell carcinoma in January 2021.

"Today’s news is welcomed by physicians treating people living with advanced renal cell carcinoma," said Dr. Cristina Suárez, Medical Oncologist at the Vall d´Hebron University Hospital, Barcelona, Spain, and a lead investigator on the Phase III CheckMate -9ER trial. "The positive CHMP opinion brings us one step closer to the promise of a new approach that combines improved treatment outcomes, a favorable tolerability profile and superior health-related quality of life for patients."

About renal cell carcinoma

There are over 400,000 new cases of kidney cancer diagnosed worldwide each year.4 Of these, renal cell carcinoma (RCC) is the most common type of kidney cancer, accounting for approximately 90% of cases.5,6 It is twice as common in men, and male patients account for over two thirds of deaths.4 If detected in the early stages, the five-year survival rate is high, but for patients with advanced or late-stage metastatic RCC the survival rate is much lower, around 12%, with no identified cure for this disease.7,8

About the CheckMate -9ER trial

CheckMate -9ER is an open-label, randomized, multi-national Phase III trial evaluating patients with previously untreated advanced or metastatic RCC. A total of 651 patients (23% favorable risk, 58% intermediate risk, 20% poor risk; 25% PD-L1 ≥1%) were randomized to Cabometyx plus Opdivo (n= 323) versus sunitinib (n= 328). The primary endpoint is progression-free survival (PFS). Secondary endpoints include overall survival (OS) and objective response rate (ORR). The primary efficacy analysis is comparing the doublet combination versus sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co and co-funded by Exelixis, Ipsen and Takeda Pharmaceutical Company Limited.

About Cabometyx (cabozantinib)

Cabometyx is currently approved in 57 countries, including in the European Union, the U.K., Norway, Iceland, Australia, New Zealand, Switzerland, South Korea, Canada, Brazil, Taiwan, Hong-Kong, Singapore, Macau, Jordan, Lebanon, Russian Federation, Ukraine, Turkey, United Arab Emirates, Saudi Arabia, Serbia, Israel, Mexico, Chile, Peru, Panama, Guatemala, Dominican Republic, Ecuador and Thailand for the treatment of advanced RCC in adults who have received prior VEGF-targeted therapy; in the European Union, the U.K., Norway, Iceland, Canada, Australia, Brazil, Taiwan, Hong Kong, Singapore, Lebanon, Jordan, Russian Federation, Ukraine, Turkey, United Arab Emirates, Saudi Arabia, Israel, Mexico, Chile, Peru, Panama, Guatemala, Dominican Republic, Ecuador and Thailand for previously untreated intermediate- or poor-risk advanced RCC; and in the European Union, the U.K., Norway, Iceland, Canada, Australia, Switzerland, Saudi Arabia, Serbia, Israel, Taiwan, Hong Kong, South Korea, Singapore, Jordan, Russian Federation, Ukraine, Turkey, Lebanon, United Arab Emirates, Peru, Panama, Guatemala, Chile, Dominican Republic, Ecuador and Thailand for HCC in adults who have previously been treated with sorafenib.

The detailed recommendations for the use of Cabometyx are described in the Summary of Product Characteristics (SmPC) and in the U.S. Prescribing Information (PI).

Cabometyx is marketed by Exelixis, Inc. in the United States and by Takeda Pharmaceutical Company Limited in Japan. Ipsen has exclusive rights for the commercialization and further clinical development of Cabometyx outside of the U.S. and Japan. Cabometyx is a registered trademark of Exelixis, Inc.

On February 26, 2021 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates" or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer and other serious diseases, reported that it will present and participate in 1-on-1 investor meetings at the Cowen 41st Annual Health Care Conference, the Barclays Global Healthcare Conference and the Oppenheimer 31st Annual Healthcare Conference (Press release, Molecular Templates, FEB 26, 2021, View Source [SID1234575759]).

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Cowen 41st Annual Health Care Conference
Conference Dates: March 1-4
Presentation Date and Time: Thursday, March 4th at 11:10 AM ET

Barclays Global Healthcare Conference
Conference Dates: March 9-11
Presentation Date and Time: Wednesday, March 10th at 3:00 PM ET

Oppenheimer 31st Annual Healthcare Conference
Conference Dates: March 16-17
Presentation Date and Time: Wednesday, March 17th at 11:20 AM ET

Live webcasts of these presentations will be available in the "News and Events" section of the MTEM website at www.mtem.com. Additionally, replays of the webcasts will be available on the corporate website following the conferences.

On February 26, 2021 GlaxoSmithKline (GSK) plc reported the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending dostarlimab, an anti-programmed death-1 (PD-1) monoclonal antibody, for use as monotherapy in women with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer who have progressed on or following prior treatment with a platinum containing regimen (Press release, GlaxoSmithKline, FEB 26, 2021, View Source [SID1234575775]). The CHMP opinion is one of the final steps in the marketing authorisation procedure prior to approval by the European Commission.

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Dr Axel Hoos, Senior Vice President and Head of Oncology R&D, GSK, said: "Treatment options are limited for women with recurrent or advanced endometrial cancer and prognosis is typically poor. This positive CHMP opinion brings us one step closer to providing dostarlimab as a new treatment option to women with endometrial cancer in Europe with the hope of improving outcomes. If approved by the European Commission, dostarlimab would be the first anti-PD-1 therapy approved for endometrial cancer in Europe."

The application is based on data from the GARNET study, which represents the largest dataset of an anti-PD-1 monotherapy treatment in endometrial cancer. Data from the GARNET study were initially presented at the 2019 Society for the Gynecologic Oncology (SGO) Annual Meeting, and updated data were presented at the 2020 SGO Annual Meeting and the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020.

Endometrial cancer is the sixth most common cancer in women worldwide.[1] While endometrial cancer can be treated successfully when diagnosed early, there is a significant unmet need for treatment options for women with recurrent or advanced endometrial cancer whose disease progresses on or after first-line therapy.[2]

Dostarlimab is under review with the US Food and Drug Administration (FDA) for the treatment of women with recurrent or advanced endometrial cancer who have progressed on or following platinum-based chemotherapy and whose tumours are dMMR. It is also under FDA review for the treatment of adult patients with dMMR recurrent or advanced solid tumours. Dostarlimab is not currently approved for use anywhere in the world.

About dostarlimab

Dostarlimab is a humanised PD-1 monoclonal antibody that binds with high affinity to the PD-1 receptor and blocks its interaction with the ligands PD-L1 and PD-L2.[3] In addition to GARNET, dostarlimab is being investigated in other registrational enabling studies, including the phase 3 RUBY study for patients with recurrent or primary advanced endometrial cancer in combination with standard of care (SOC) chemotherapy[4] and the phase 3 FIRST study of platinum-based therapy with dostarlimab and niraparib versus SOC platinum-based therapy as first-line treatment of stage III or IV non-mucinous epithelial ovarian cancer. It is also being evaluated in combination with other therapeutic agents for patients with advanced solid tumours or metastatic cancer.

Dostarlimab was discovered by AnaptysBio and licensed to TESARO, Inc., under a Collaboration and Exclusive License Agreement signed in March 2014. The collaboration has resulted in three monospecific antibody therapies that have progressed into the clinic. These are: dostarlimab (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialization, and manufacture of each of these products under the Agreement.

About GARNET

The ongoing phase 1 GARNET trial is evaluating dostarlimab as monotherapy in patients with advanced solid tumours. Part 2B of the study includes five expansion cohorts: dMMR/MSI-H endometrial cancer (cohort A1), mismatch repair proficient/microsatellite stable (MMRp/MSS) endometrial cancer (cohort A2), non-small cell lung cancer (cohort E), dMMR/MSI-H non-endometrial or POLE-mut solid tumour basket cohort (cohort F), and platinum-resistant ovarian cancer without BRCA mutations (cohort G). GARNET is ongoing and enrolling patients.

About endometrial cancer

Endometrial cancer is a main type of uterine cancer that forms in the inner lining of the uterus, known as the endometrium.[5] Endometrial cancer can be classified as dMMR/MSI-H or MMRp/MSS. There are limited treatment options for patients whose disease progresses on or after first-line therapy.

GSK in Oncology

GSK is focused on maximising patient survival through transformational medicines. GSK’s pipeline is focused on immuno-oncology, cell therapy, cancer epigenetics and synthetic lethality. Our goal is to achieve a sustainable flow of new treatments based on a diversified portfolio of investigational medicines utilising modalities such as small molecules, antibodies, antibody drug conjugates and cells, either alone or in combination.

On February 26, 2021 Horizon Therapeutics plc (Nasdaq: HZNP) reported that the Company will participate in the following conference in March (Press release, Horizon Pharma, FEB 26, 2021, https://ir.horizontherapeutics.com/news-releases/news-release-details/horizon-therapeutics-plc-present-cowen-and-company-41st-annual [SID1234575793]):

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Cowen and Company 41st Annual Health Care Conference (Virtual)

Date: Tuesday, Mar. 2, 2021
Presentation Time: 1:30 p.m. ET
The conference presentation will be webcast live and may be accessed by visiting Horizon’s website at View Source A replay of the webcast will be available following the event.

On February 26, 2021 Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2020 (Press release, Mersana Therapeutics, FEB 26, 2021, View Source [SID1234575734]).

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"In 2020, we demonstrated compelling proof of concept for UpRi in heavily pretreated ovarian cancer and advanced our pipeline of highly differentiated DolaLock and Immunosynthen ADCs. We are well positioned for an equally productive 2021 as we focus on building UpRi as a foundational therapy for the treatment of ovarian cancer and building out our robust and maturing pipeline of ADC candidates. For UpRi, we are on track to initiate UPLIFT, our single-arm registration strategy in platinum-resistant ovarian cancer in the first quarter and plan to initiate the UPGRADE combination umbrella study to explore the role of UpRi in earlier stages of the disease in the third quarter of 2021. This is an important first step in a lifecycle management plan in earlier lines of therapy," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "With respect to building out the pipeline, we are planning to report data from the ongoing Phase I expansion cohort of UpRi in lung adenocarcinoma as well as data from the Phase I dose escalation study of XMT-1592 in the second half of 2021. We also intend to progress IND-enabling activities for XMT-1660 and XMT-2056 with the intention of advancing into clinical development in 2022."

Recent Highlights and Anticipated Milestones

Upifitamab Rilsodotin (UpRi, XMT-1536), first-in-class Dolaflexin ADC targeting NaPi2b:

UPLIFT single-arm registration strategy in platinum-resistant ovarian cancer on track to initiate in first quarter of 2021. Informed by feedback from a meeting with the U.S Food and Drug Administration (FDA), the Company plans to initiate UPLIFT, a single-arm registration strategy to evaluate the safety and efficacy of UpRi in platinum-resistant ovarian cancer patients who have received up to four lines of therapy. Consistent with the bevacizumab label, platinum-resistant ovarian cancer patients previously treated with three or four lines of therapy may enroll without regard to prior bevacizumab treatment. Platinum-resistant ovarian cancer patients who received one or two lines of therapy will be required to have had prior bevacizumab treatment. Patients may enroll without regard to NaPi2b expression; however, the role of the biomarker will be evaluated. The primary endpoint will be the objective response rate (ORR) in the higher NaPi2b population and the secondary endpoints will be the ORR regardless of NaPi2b expression, as well as duration of response and safety. The single-arm registration strategy will be initiated as an amendment to the ongoing multinational, multi-center, open label study protocol, and the Company expects to enroll approximately 100 patients with higher NaPi2b expression and up to 180 patients overall. The Company is on track to finalize the biomarker strategy and the cutoff for the proposed commercial diagnostic in UPLIFT.

Reported updated Phase 1 ovarian cancer expansion cohort study data in January 2021. In January 2021, the Company presented updated data with a cutoff of December 3, 2020, which included 72 patients evaluable for safety and 47 patients evaluable for RECIST response in the ongoing expansion portion of the Phase 1 study of UpRi in ovarian cancer. These data continued to demonstrate encouraging antitumor activity in heavily pretreated patients with ovarian cancer, with an ORR of 32% including complete responses and a disease control rate (DCR) of 74% in the higher NaPi2b population. Activity was also observed in the overall population, regardless of NaPi2b expression, with an ORR of 28% and a DCR of 68%. The majority of responses occurred by the first scan, and the median duration of response was approximately five months in the higher NaPi2b population. UpRi’s tolerability profile remained consistent with previous data disclosures and continues to demonstrate a differentiated profile without the severe neutropenia, peripheral neuropathy, or ocular toxicity that can be observed for other ADCs. These data suggest the potential to achieve a clinically meaningful benefit in platinum-resistant ovarian cancer, where the single-agent chemotherapy standard of care has an ORR of 4% to 12% and expected overall survival of less than a year.

UPGRADE umbrella combination study in ovarian cancer expected to initiate in the third quarter of 2021. The Company plans to initiate the UPGRADE study in the third quarter of 2021 to evaluate the combination of UpRi with other agents, starting with a platinum chemotherapy combination dose escalation cohort. This study is designed to inform the lifecycle management strategy for UpRi in earlier lines of ovarian cancer, including platinum-sensitive disease.

NSCLC adenocarcinoma cohort of the expansion portion of Phase 1 study continues to enroll patients. The Company is on track to recruit approximately 40 patients in the expansion phase of the study. The Company plans to report interim data in the second half of 2021.
XMT-1592, first Dolasynthen ADC targeting NaPi2b:

Phase 1 dose escalation study of XMT-1592 enrolling patients with interim data anticipated in the second half of 2021. XMT-1592 is the Company’s first clinical candidate created using its new Dolasynthen ADC platform. In preclinical studies, XMT-1592 showed four times greater efficacy in a patient-derived lung tumor model in comparison to UpRi. The Company continues dose escalation and plans to disclose interim data in the second half of 2021 and outline the XMT-1592 development plan in NSCLC in the fourth quarter of 2021.
XMT-1660, first-in-class Dolasynthen ADC targeting B7-H4:

Completion of XMT-1660 IND-enabling studies expected in the fourth quarter of 2021. B7-H4 is expressed in high unmet need tumors such as triple-negative breast cancer, ER-positive breast cancer, and NSCLC. B7-H4 is expressed on both tumor cells and immunosuppressive tumor-associated macrophages (TAMs). This provides the potential for both a direct, cytotoxic antitumor effect as well as for additional payload delivery to the tumor microenvironment that can further contribute to immunogenic cell death, dendritic cell activation, and stimulation of an immune response consistent with the features of the Company’s unique DolaLock payload. The Company plans to initiate a Phase 1 dose escalation study of XMT-1660 in early 2022.
XMT-2056, first Immunosynthen STING-agonist ADC:

Completion of XMT-2056 IND-enabling studies expected in the fourth quarter of 2021. In November 2020, the Company introduced XMT-2056 and presented preclinical data that supported the potential differentiation of the Immunosynthen platform from other innate immune stimulatory approaches and its potential applicability across multiple targets and indications. The Company plans to disclose the target for this program in the fourth quarter of 2021 and to initiate a Phase 1 dose escalation study in early 2022.
Corporate

Appointed Chief Human Resources Officer. In January 2021, Mersana announced that Carla Poulson has joined the company as Chief Human Resources Officer. Ms. Poulson was most recently Chief Human Resources Officer at Akcea Therapeutics where she played an integral role in building the organization including recruiting several members of the senior management team. Before that, she served in multiple roles at Vertex Pharmaceuticals for over 10 years including as Head of International Human Resources where she was instrumental in helping build the organization to over two hundred fifty employees in just two years.
Upcoming Events

Mersana will participate in a virtual panel presentation at the Cowen 41st Annual Health Care Conference on Tuesday, March 2, 2021 at 12:50 p.m. ET.
Mersana will present preclinical data for XMT-1660, XMT-2056 and its novel Immunosynthen STING-agonist ADC platform in the e-poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting scheduled for April 10-15, 2021.
2020 Financial Results

Cash and cash equivalents as of December 31, 2020, were $255.1 million, compared to $99.8 million in cash, cash equivalents and marketable securities as of December 31, 2019. In addition, the Company has the option to draw additional funds through its debt financing agreement with Silicon Valley Bank.

Net cash used in operating activities in the fourth quarter of 2020 was $17.3 million. The Company expects that its available funds will be sufficient to support its operating plan commitments for approximately the next two years.

Fourth Quarter 2020

Research and development expenses for the fourth quarter of 2020 were approximately $22.9 million, compared to $12.4 million for the same period in 2019. The difference was primarily due to an increase in UpRi and XMT-1592 clinical expenses, an increase in manufacturing activities for UpRi and discovery stage programs, an increase in headcount, and a non-cash increase in valuation of stock-based awards as a result of stock appreciation. The increase was partially offset by a decrease in preclinical development and manufacturing expenses for XMT-1592.
General and administrative expenses for the fourth quarter of 2020 were approximately $5.9 million, compared to $4.2 million during the same period in 2019 primarily due to an increase in consulting and professional fees, an increase in facility-related costs as a result of the extension of the Company’s lease, and a non-cash increase in valuation of stock-based awards as a result of stock appreciation.
Net loss for the fourth quarter of 2020 was $28.8 million, or $0.42 per share, compared to net loss of $16.2 million, or $0.34 per share, for the same period in 2019. Weighted average common shares outstanding for the quarters ended December 31, 2020 and December 31, 2019, were 68,630,078 and 47,886,144, respectively.
Full Year 2020

Collaboration revenue for the full year 2020 was approximately $0.8 million, compared to $42.1 million for the full year 2019. The decrease in collaboration revenue was primarily a result of the recognition of the remaining deferred revenue under the Takeda agreements of $40.0 million in 2019.
Research and development expenses for the full year 2020 were approximately $67.0 million, compared to $55.0 million for the full year 2019. The difference was primarily due to an increase in clinical and regulatory activities for UpRi and XMT-1592, and increase in manufacturing activities for UpRi, an increase in headcount and a non-cash increase in valuation of stock-based awards as a result of stock appreciation and an increase in manufacturing activities for preclinical programs. The increase was partially offset by a decrease in XMT-1592 preclinical development and manufacturing activities and discontinuation of XMT-1522.
General and administrative expenses for the full year 2020 were approximately $21.9 million, compared to $17.3 million for the full year 2019, primarily due to an increase in consulting and professional fees, an increase in facility-related costs as a result of the extension of the Company’s lease, and a non-cash increase in valuation of stock-based awards as a result of stock appreciation.
Net loss for the full year 2020 was $88.0 million, or $1.43 per share, compared to net loss of $28.2 million, or $0.65 per share, for the full year 2019. Weighted average common shares outstanding for the periods ended December 31, 2020 and December 31, 2019, were 61,485,205 and 43,492,113, respectively.
Conference Call Details
Mersana Therapeutics will host a conference call and webcast today at 8:00 a.m. ET to report financial results for the fourth quarter and full year 2020 and provide certain business updates. To access the call, please dial 877-303-9226 (domestic) or 409-981-0870 (international) and provide the Conference ID 2354447. A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com.