Carcinoma of ampulla of Vater

LEXINGTON, Mass.--(BUSINESS WIRE)--Agenus Inc. (“Agenus”) (Nasdaq: AGEN), a leader in developing novel immunological agents to treat various cancers, today announced results for the second quarter 2023. Key accomplishments and highlights include: Botensilimab Update: Botensilimab/balstilimab combination data presented at ESMO-GI demonstrated median overall survival (mOS) of 20.9 months and a 23% overall response rate (ORR), both surpassing data reported for standard of care in 3L+ Non-MSI-H colorectal cancer (CRC) in patients without active liver metastases. Regulatory: The U.S. FDA granted Fast Track designation for the botensilimab/balstilimab combination in 3L+ Non-MSI-H CRC in patients without active liver metastases. Agenus is exploring global accelerated approval strategies for CRC. Clinical Trial Progress: Ongoing Phase 2 ACTIVATE trials in CRC and melanoma expected to fully enroll by end of year. AGEN2373 Update: AGEN2373 Phase 1 dose escalation data presented at ASCO demonstrated promising monotherapy clinical responses in patients with advanced solid tumors. “Botensilimab, alone or in combination with balstilimab, continues to display remarkable clinical activity in over 600 patients treated across nine late-stage, treatment resistant solid tumor cancers, demonstrating great potential to revolutionize the role of immunotherapy in cancer treatment,” said Chief Executive Officer, Garo Armen, Ph.D. “Agenus is committed to advancing our diverse clinical pipeline with a focus on expediting our first regulatory submission for the botensilimab/balstilimab combination in colorectal cancer. Our data has demonstrated an unprecedented survival benefit over what has been reported for standard of care, underscoring this combination as an important potential treatment option for patients with non-MSI-high colorectal cancer, which represents 85% of the population of patients with colorectal cancer1.” Botensilimab/Balstilimab (“Bot/Bal”) Combination in 3L+ Non-MSI-H Metastatic CRC Data Presented at the ESMO-GI World Congress on Gastrointestinal Cancer in June 2023: In 69 evaluable patients with non-MSI-High CRC without active liver metastases, results demonstrated a meaningful improvement over standard of care in both overall response rate (ORR%) and median overall survival (mOS): mOS of 20.9 months as compared to the recently reported 12.9-month benchmark with standard care.2 ORR of 23% as compared to the recently reported 2.8%1 benchmark with standard care. In April 2023, the U.S. FDA granted Fast Track designation to the bot/bal combination for patients with non-MSI-H/dMMR metastatic CRC without active liver metastases. The ACTIVATE Phase 2 trial in this patient population is on target to conclude enrollment by year-end. Planning is underway for a Phase 3 CRC study; with Fast Track designation from the FDA, study design and timing will be determined as we discuss our filing approach with the agency. A Biologics License Application to the FDA is planned in 2024. Botensilimab in Other Advanced Solid Tumor Cancers: Lung: Enrollment continues for a Phase 1b trial targeting PD-(L)1 +/- chemotherapy relapsed/refractory non-small-cell lung cancer (NSCLC); data update planned by year-end. Melanoma: Ongoing global enrollment in Phase 2 ACTIVATE trial with data update planned in 2024. Ovarian: Phase 1b trial in patients with platinum-resistant ovarian cancer (PROC) demonstrated a 33% ORR and 67% disease control rate among primarily high-grade, heavily pretreated patients (presented at SGO in March 2023). Sarcomas: Updated data for a Phase 1b cohort with advanced sarcomas to be presented at ESMO on October 21st 2023. AGEN2373 Data Presented at the ASCO 2023 Annual Meeting: Complete results from the first-in-human monotherapy dose escalation study of AGEN2373, a CD137 agonist, in patients with advanced solid tumors: Reported responses in metastatic prostate cancer, ampullary carcinoma, and vulvar squamous cell carcinoma highlight the potential impact of AGEN2373. AGEN2373 is the first CD137 agonist antibody to report single-agent responses with no major toxicities. Clinical data are consistent with mechanistic design intent of AGEN2373 to avoid hepatic toxicity, a typical constraint of CD137-targeted agonists. Leadership Appointments: In alignment with Agenus' next phase of growth and anticipated commercialization of the bot/bal combination, the company announced the following key executive appointments: Robin Taylor, Ph.D., Chief Commercial Officer. Homa Yeganegi, Chief Product Strategy and Global Medical Affairs Officer. Stephanie Fagan, Chief Communications Officer. Second Quarter 2023 Financial Results: We ended our second quarter 2023 with a cash, cash equivalent and short-term investment balance of $157.6 million, compared to $193.4 million at December 31, 2022. In addition, we have raised $20.3 million through sales of our common stock under our at market issuance sales agreement since the end of Q2 2023. For the three and six months ended June 30, 2023, we recognized revenue of $25.3 million and $48.2 million and incurred a net loss of $73.4 million and $144.3 million, respectively. Our net loss includes non-cash expenses of $28.9 million for the three months ended, and $53.8 million for the six months ended, June 30, 2023. Select Financial Information (in thousands, except per share data) (unaudited) June 30, 2023 December 31, 2022 Cash, cash equivalents and short-term investments $ 157,852 $ 193,358 Cash raised since quarter end $ 20,281 Three months ended June 30, Six months ended June 30, 2023 2022 2023 2022 Revenues, royalty sales milestone $ - $ 17,316 $ - $ 17,316 Revenues, non-cash royalty 22,068 144 41,174 17,778 Revenues, research and development 2,489 1,907 5,101 8,647 Revenues, other 739 1,559 1,923 3,126 Total Revenue 25,296 20,926 48,198 46,867 Research and development expenses 59,285 44,960 116,402 87,404 General and administrative expenses 20,415 18,914 38,653 37,866 Cost of service revenue 254 2,024 2,548 2,567 Other income (883 ) (8,966 ) (1,604 ) (8,776 ) Non-cash interest expense 19,647 13,636 36,920 28,588 Non-cash contingent consideration fair value adjustment 8 (407 ) (398 ) (943 ) Net loss* $ (73,430 ) $ (49,235 ) $ (144,323 ) $ (99,839 ) Net loss per share attributable to Agenus Inc. common stockholders: $ (0.20 ) $ (0.17 ) $ (0.41 ) $ (0.35 ) *Includes non-cash expenses Adjusted Net loss for non-cash expenses: Net loss as reported $ (73,430 ) $ (49,235 ) $ (144,323 ) $ (99,839 ) Non-cash expenses Depreciation and amortization 2,973 1,690 5,535 3,350 Share based compensation 6,042 4,634 11,527 9,625 Non-cash interest expense 19,647 13,636 36,920 28,588 Other 285 (409 ) (100 ) (943 ) Total non-cash expenses 28,947 19,551 53,882 40,620 Net loss excluding non-cash expenses $ (44,483 ) $ (29,684 ) $ (90,441 ) $ (59,219 ) References 1 2 Cohen et al. ASCO Annual Meeting 2023 About Botensilimab Botensilimab, an investigational multifunctional CTLA-4 antibody, is designed to extend immunotherapy benefits to “cold” tumors, which have not historically responded to standard of care or other investigational therapies. Besides binding to the CTLA-4 receptor, its Fc-enhanced structure induces a memory immune response, downregulates regulatory T cells, and activates T cells, thereby enhancing immune responses. Approximately 600 patients have been treated with botensilimab in phase 1 and phase 2 clinical trials. Botensilimab alone, or in combination with Agenus’ PD-1 antibody, balstilimab, has shown clinical responses across nine metastatic, late-line cancers. For more information about botensilimab trials, visit with the identifiers NCT03860272, NCT05608044, NCT05630183, and NCT05529316. About AGEN2373 AGEN2373 is a novel anti-CD137 agonist that has been designed to activate T and NK cells while mitigating liver toxicities common to the CD137 target class. CD137 (4-1BB) is an activating receptor expressed on T and NK cells. Upon binding to CD137, AGEN2373 is designed to stimulate the growth and activation of cytotoxic T and NK cells, triggering a lasting memory response to cancer. AGEN2373 binds to a unique epitope designed to achieve this response specifically within the tumor microenvironment. This selective binding is designed to avoid serious side effects associated with CD137 activation in the liver that have been reported by competitor molecules. AGEN2373 has demonstrated preliminary clinical activity and has been well tolerated by patients without signs of liver toxicity (Barve et al. ASCO 2023). About Agenus Agenus is a leading immuno-oncology company targeting cancer and infectious diseases with a comprehensive pipeline of immunological agents. The company’s mission is to expand patient populations benefiting from cancer immunotherapy through combination approaches, using a broad repertoire of antibody therapeutics, adoptive cell therapies (through MiNK Therapeutics) and adjuvants (through SaponiQx). Agenus is headquartered in Lexington, MA. For more information, visit or follow us on LinkedIn and Twitter @agenus_bio. Non-GAAP Financial Measures To supplement our financial disclosures that are prepared and presented in accordance with generally accepted accounting principles in the United States (“GAAP”), this press release includes Adjusted Net Loss, which is a non-GAAP financial measure that we believe is helpful to our investors. We use these non-GAAP financial measures for financial and operational decision-making purposes. We believe these measures provide investors with meaningful period-to-period comparisons of our underlying performance. Adjusted Net Loss is a supplemental measure of our operating performance and excludes the impacts of certain costs required to be recorded under GAAP. We calculate Adjusted Net Loss as net loss adjusted to exclude non-cash expenses of depreciation and amortization, share based compensation, non-cash interest expense and certain other non-cash expenses. Accordingly, Adjusted Net Loss should not be considered in isolation or as a substitute for net loss, which is the most directly comparable financial measure presented in accordance with GAAP. Forward-Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements relating to our technologies, therapeutic candidates, and capabilities, for instance, statements regarding therapeutic benefit and efficacy, mechanism of action, potency, durability, and safety and tolerability pro our therapeutic candidates, both alone and in combination with each other and/or other agents; statements regarding future plans, including research, clinical, regulatory, and commercialization plans; and any other statements containing the words "may," "believes," "expects," "anticipates," "hopes," "intends," "plans," "will" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially. These risks and uncertainties include, among others, the factors described under the Risk Factors section of our most recent Quarterly Report on Form 10-Q or Annual Report on Form 10-K filed with the Securities and Exchange Commission and available on our website: . Agenus cautions investors not to place considerable reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this press release, and Agenus undertakes no obligation to update or revise the statements, other than to the extent required by law. All forward-looking statements are expressly qualified in their entirety by this cautionary statement.

LEXINGTON, Mass.--( BUSINESS WIRE )--Agenus (Nasdaq: AGEN), a leading immuno-oncology company with a pipeline of immunological agents targeting cancer and infectious disease, presented complete results from the monotherapy arm of the first-in-human dose escalation study of AGEN2373 (CD137 agonist) at the American Society of Clinical Oncology (ASCO) Annual Meeting. AGEN2373 demonstrated objective responses, clinical benefit, and was well tolerated in heavily pre-treated patients with solid tumors. “AGEN2373 has shown meaningful single agent activity and a favorable safety profile without evidence of liver toxicity in patients with heavily pretreated cold and immunotherapy resistant tumors,” said Dr. Steven O’Day, MD, Chief Medical Officer at Agenus. “AGEN2373 is designed to selectively boost tumor immunity while limiting hepatotoxicity and off-target effects associated with systemic CD137 activation. These encouraging monotherapy results support further clinical trials for AGEN2373 alone and in combination with our novel immunotherapy programs.” Study Design: AGEN2373 was administered intravenously at doses ranging from 0.03 mg/kg to 10 mg/kg in a cohort of 46 patients with advanced solid tumors and a median of 4 prior lines of therapy. Objective responses: Notable responses in the dose escalation study include: Confirmed partial response in a patient with vulvar squamous cell carcinoma who progressed on prior pembrolizumab Confirmed partial response with complete resolution of the pancreatic lesion in a patient with ampullary carcinoma Confirmed 38% reduction in target liver lesions in a castrate-resistant prostate cancer that was non-evaluable by RECIST due to palliative radiation for bone metastases Tolerability: No hepatic toxicities, grade ≥3 treatment-related adverse events, or dose-limiting toxicities were observed, consistent with the molecule’s design. Presently, AGEN2373 is being evaluated in combination with botensilimab (multi-functional anti-CTLA-4) at a dose of 10 mg/kg in patients with PD-(L)1 refractory melanoma. Presentation Details: Abstract Title: A Phase 1 Study of AGEN2373, a Novel CD137 Agonist Antibody Designed to Avoid Hepatoxicity, in Patients with Advanced Solid Tumors (NCT04121676) Abstract Number: 2524 Poster Number : 366 Presenting Author: Dr. Minal Barve, MD, Executive Director and Chief Medical Officer, Mary Crowley Research The poster presentation can be accessed in the publications section of our website at https://agenusbio.com/publications/ . About AGEN2373 AGEN2373 is a novel anti-CD137 agonist designed to stimulate T and NK cells for a durable memory response to cancer. AGEN2373’s selective binding to a unique epitope is designed to prevent serious side effects associated with CD137 activation in the liver, as reported by competitor molecules. About Agenus Agenus is a clinical-stage immuno-oncology company focused on developing therapies that engage the body's immune system in fighting cancer and infections. The Company's mission is to broaden the patient populations benefiting from cancer immunotherapy through combination approaches, leveraging a broad repertoire of antibody therapeutics, adoptive cell therapies (through its subsidiary MiNK Therapeutics), and adjuvants (through its subsidiary SaponiQx). Agenus is headquartered in Lexington, MA. For more information, please visit www.agenusbio.com and our Twitter handle @agenus_bio. Forward-Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements relating to the use of AGEN2373, for instance, statements regarding therapeutic benefit and efficacy, mechanism of action (including validation of mechanism of action), potency, durability, and safety profile (including the absence of specific toxicities) of the Company’s therapeutic candidates; and any other statements containing the words "may," "believes," "expects," "anticipates," "hopes," "intends," "plans," "forecasts," "estimates," "will," “establish,” “potential,” “superiority,” “best in class,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially. These risks and uncertainties include, among others, the factors described under the Risk Factors section of our most recent Quarterly Report on Form 10-Q or Annual Report on Form 10-K filed with the Securities and Exchange Commission. Agenus cautions investors not to place considerable reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this press release, and Agenus undertakes no obligation to update or revise the statements, other than to the extent required by law. All forward-looking statements are expressly qualified in their entirety by this cautionary statement.

Compass to Host Investor Event on Monday, January 23rd at 8:30am ET CTX-009 in combination with paclitaxel demonstrated a 37.5% overall response rate (ORR) in 24 patients with BTC treated in the second- and third- line settings; at least one response was observed in each of the four anatomical subtypes of BTC Sub-group analysis showed that an ORR of 63.6% was observed in the 11 patients treated in the second-line setting compared with an ORR of 14.9% in the 13 patients treated in the third-line setting Median progression free survival (PFS) was 9.4 months and median overall survival (OS) was 12.5 months Safety and tolerability of CTX-009 were consistent with prior studies Compass has initiated a randomized Phase 2/3 trial of CTX-009 in combination with paclitaxel in the U.S. in patients with BTC. Depending on study results, this study may support a biologics license application (BLA) submission in 2024 BOSTON, Jan. 19, 2023 (GLOBE NEWSWIRE) -- Compass Therapeutics Inc. (Nasdaq: CMPX), a clinical-stage, oncology-focused biopharmaceutical company developing proprietary antibody-based therapeutics to treat multiple human diseases, today announced the presentation of data from a Phase 2 study of CTX-009 in combination with paclitaxel in patients with biliary tract cancers (BTC). These data will be presented at the 2023 American Society of Clinical Oncology Gastrointestinal Cancers (ASCO GI) Symposium taking place in San Francisco, CA on January 20, 2023. Data highlights from the poster presentation include: In the primary analysis of all 24 patients participating in the study, CTX-009 with paclitaxel demonstrated a 37.5% ORR based on 9 patients with Partial Responses (PRs) that were confirmed by RECIST 1.1. Importantly, of the 11 patients who had received one prior systemic therapy, 7 patients achieved a PR, leading to an ORR of 63.6% in this sub-group. After a median follow up of approximately 12 months, the median PFS was 9.4 months, and the median OS was 12.5 months. For reference, one regimen that has been studied in patients with advanced BTC is FOLFOX, the regimen recommended in the guidelines of the National Comprehensive Cancer Network (NCCN) for patients with BTC treated in the second-line setting. FOLFOX demonstrated a median PFS of 4.0 months and a median OS of 6.2 months in a randomized study against best supportive care. As the data continue to mature, safety and tolerability of CTX-009 in combination with paclitaxel remain consistent with previous studies. Treatment emergent adverse events (TEAEs) were observed in all patients, including those TEAEs related to the patients’ disease and the two study drugs in the treatment regimen, paclitaxel and/or CTX-009. Grade 3 or greater TEAEs were reported in 95.8% of patients, including decreased neutrophil count (83.3%), hypertension (16.7%), anemia (20.8%) and decreased platelet count (12.5%). The poster presentation will be available in the Pipeline section of the Compass Therapeutics website at . “We are very pleased to see the maturing data from this study, particularly the median progression free survival of greater than 9 months and median overall survival of 12.5 months supporting the durable effect of CTX-009 in this patient population,” said Thomas Schuetz, M.D., Ph.D., Chief Executive Officer and Scientific Founder of Compass. “Given that patients with advanced BTC have a poor prognosis, have been heavily pre-treated, and have few treatment options, we are particularly enthused with these results.” “The results from the Phase 2 trial in BTC continue to show the potential of CTX-009, which has demonstrated activity in a broad range of solid tumors. We are presently enrolling patients in two clinical studies of CTX-009 in the United States and expect to report initial data from the colorectal study in the second half of this year,” added Vered Bisker-Leib Ph.D., President and Chief Operating Officer of Compass. Investor Event Details Compass will host a webcast on Monday, January 23rd at 8:30am ET. Registration for the webcast or access to a replay of the call is available on the Company’s website or by clicking here. CTX-009 Phase 2 BTC Study Design The Phase 2 study is being conducted at four leading medical centers in Korea and has a Simon Two-Stage adaptive design. As of November 9, 2022, the data cut-off date, 24 patients were enrolled and dosed with at least one cycle of CTX-009 and paclitaxel. Of these patients, 22 were evaluable for response. Nearly all patients (23/24) received gemcitabine plus cisplatin as a first-line treatment. Among patients enrolled in the study, 45.8% of patients received one prior therapy and 54.2% received at least two prior therapies. Patients with all four BTC subtypes were enrolled in the study: intrahepatic cholangiocarcinoma (37.5%), extrahepatic cholangiocarcinoma (12.5%), gallbladder cancer (29.2%) and ampullary cancer (20.8%). Patients had a median age of 62.1 years and an ECOG performance status of 0 (54.2%) or 1 (45.8%). CTX-009 Phase 2/3 BTC Study Design Compass has initiated a randomized Phase 2/3 study of CTX-009 in combination with paclitaxel in the United States in patients with advanced BTC. The study will enroll up to 150 patients with BTC who have received no more than one prior systemic therapy and those patients will be randomized to CTX-009 in combination with paclitaxel or paclitaxel alone in a 2:1 ratio. Based upon regulatory feedback and depending upon the study results, this study potentially could support a biologics license application (BLA) submission as soon as H2 2024. The primary endpoint of the study is ORR as measured by RECIST 1.1. Secondary endpoints of the study include PFS, OS, Quality of Life and other measures. Study details are on Identifier: NCT05506943 About CTX-009 CTX-009 is a bispecific antibody that simultaneously blocks Delta-like ligand 4/Notch (DLL4) and vascular endothelial growth factor A (VEGF-A) signaling pathways, which are critical to angiogenesis and tumor vascularization. Preclinical and early clinical data of CTX-009 suggest that blockade of both pathways provides robust anti-tumor activity across several solid tumors, including colorectal, gastric, cholangiocarcinoma, pancreatic and non-small cell lung cancer. Partial responses to CTX-009 as a monotherapy have been observed in heavily pre-treated patients with cancer who were resistant to currently approved anti-VEGF therapies. Compass holds the global rights to CTX-009 (also known as ABL001) with the exception of rights in Korea, held by Handok, Inc. ( ) and rights in China, which were out-licensed to Elpiscience Biopharma, Ltd. ( ). About Biliary Tract Cancers Biliary tract cancers (BTC) are a group of aggressive gastrointestinal (GI) cancers that form in the cells of the bile ducts (cholangiocarcinoma), gallbladder, or ampulla of Vater (where the bile duct and pancreatic duct connect to the small intestine). In the United States approximately 18,400 cases of BTC are diagnosed annually,¹ including cholangiocarcinoma, gallbladder, and ampullary subtypes. Only 10% of these patients present at an early stage when they would be candidates for surgical resection. The vast majority present with locally advanced or metastatic BTC, for which there are very few therapeutic options.² ¹ seer.cancer.gov/statfacts/html/livibd.html ² cancer.gov/types/liver/patient/bile-duct-treatment-pdq#_66 About Compass Therapeutics Compass Therapeutics, Inc. is a clinical-stage, oncology-focused biopharmaceutical company developing proprietary antibody-based therapeutics to treat multiple human diseases. Compass’s scientific focus is on the relationship between angiogenesis, the immune system, and tumor growth. Compass’s pipeline of novel product candidates is designed to target multiple critical biological pathways required for an effective anti-tumor response. These include modulation of the microvasculature via angiogenesis-targeted agents, induction of a potent immune response via activators on effector cells in the tumor microenvironment, and alleviation of immunosuppressive mechanisms used by tumors to evade immune surveillance. Compass plans to advance its product candidates through clinical development as both standalone therapies and in combination with proprietary pipeline antibodies based on supportive clinical and nonclinical data. Compass was founded in 2014 and is headquartered in Boston, Massachusetts. For more information, visit the Compass Therapeutics, Inc. website at . Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, references to Compass’s product candidate, CTX-009, its development, regulatory plans with respect thereto and therapeutic potential thereof. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, Compass’s ability to raise the additional funding it will need to continue to pursue its business and product development plans, the inherent uncertainties associated with developing product candidates and operating as a development stage company, Compass’s ability to identify additional product candidates for development, Compass’s ability to develop, complete clinical trials for, obtain approvals for and commercialize any of its product candidates, competition in the industry in which Compass operates and market conditions. These forward-looking statements are made as of the date of this press release, and Compass assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents Compass files with the SEC available at including without limitation Compass’s latest Form 10-Q and subsequent filings with the SEC. Investor Contact Mario Corso, Investor Relations ir@compasstherapeutics.com Media Contact Anna Gifford, Communications Manager media@compasstherapeutics.com 617-500-8099