This is a multicenter, open-label, single-arm, phase II clinical trial to evaluate the efficacy and safety of margetuximab in combination with tucatinib and capecitabine in patients with human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer.

Start Date15 May 2022

Sponsor / Collaborator

Medica Scientia Innovation Research SL

[+2]

CTR20200660 / SuspendedPhase 2/3

一项评价Margetuximab联合INCMGA00012和化疗、或联合MGD013和化疗在初治HER2阳性、转移性或局部晚期胃癌或胃食管结合部癌患者疗效的II/III期临床试验

[Translation] A phase II/III clinical trial evaluating the efficacy of margetuximab in combination with INCMGA00012 and chemotherapy, or in combination with MGD013 and chemotherapy in previously untreated patients with HER2-positive, metastatic, or locally advanced gastric or gastroesophageal junction cancer

队列A：评估margetuximab＋INCMGA00012在人表皮生长因子受体2（HER2）免疫组织化学（IHC）3＋、并且IHC染色检验的PD-L1＋、排除微卫星不稳定（MSI）-高表达（MSI-H）的、未经治疗的局部晚期或转移性胃癌（GC）或胃食管结合部癌（GEJ）患者中的安全性和耐受性。评估margetuximab＋INCMGA00012在肿瘤缓解可评估人群（REP）中、通过中心影像以及研究者评估的客观缓解率（ORR）。队列B，第1部分：根据在主要肿瘤缓解可评估人群（PREP）中的安全性和ORR评估结果，不考虑PD-L1状态，选择在后续第2部分研究中将采用的margetuximab、化疗和包括CPI治疗的最佳联合治疗方案。队列B，第2部分：比较采用队列B，第1部分选择的margetuximab、化疗和包括CPI治疗的联合治疗方案与曲妥珠单抗联合化疗方案（对照组）在主要疗效人群（PEP）的总生存期（OS）

[Translation]

Queue A: To evaluate margetuximab + INCMGA00012 in human epidermal growth factor receptor 2 (HER2) immunohistochemistry (IHC) 3 +, and IHC staining for PD-L1 +, excluding microsatellite instability (MSI)-high expression (MSI-H), no Safety and tolerability in treated patients with locally advanced or metastatic gastric cancer (GC) or gastroesophageal junction cancer (GEJ). To evaluate the objective response rate (ORR) of margetuximab + INCMGA00012 in the tumor response evaluable population (REP), by central imaging, and by investigator assessment. Queue B, part 1: Margetuximab, chemotherapy, and optimal combination therapy including CPI therapy were selected for subsequent Part 2 studies based on safety and ORR assessments in the primary tumor response evaluable population (PREP) regardless of PD-L1 status Program. Queue B, part 2: To compare overall survival (OS) in the primary efficacy population (PEP) using cohort B, part 1 selected margetuximab, chemotherapy, and combination regimens including CPI therapy versus trastuzumab plus chemotherapy (control group)

Start Date15 Dec 2020

Sponsor / Collaborator

Zai Lab Ltd.

CTR20200242 / CompletedPhase 1

一项评价Margetuximab联合化疗在HER2表达阳性经标准治疗失败的转移性乳腺癌患者中的药代动力学、安全性及耐受性的I期研究

[Translation] A phase I study to evaluate the pharmacokinetics, safety, and tolerability of margetuximab in combination with chemotherapy in patients with HER2-positive metastatic breast cancer who failed standard therapy

主要研究目的：评估Margetuximab联合化疗治疗中国HER2表达阳性的既往接受过抗HER2靶向治疗失败的转移性乳腺癌患者的药代动力学（PK）特征。次要研究目的：评估margetuximab联合化疗治疗HER2表达阳性的既往接受过抗HER2靶向治疗失败的转移性乳腺癌的安全性及耐受性评价针对margetuximab的抗药物抗体（ADA）及其对PK、疗效和安全性的影响

[Translation]

MAIN STUDY OBJECTIVE: To evaluate the pharmacokinetic (PK) characteristics of margetuximab combined with chemotherapy in Chinese patients with HER2-positive metastatic breast cancer who had previously failed anti-HER2-targeted therapy. Secondary research objectives: To evaluate the safety and tolerability of margetuximab combined with chemotherapy in the treatment of HER2-positive metastatic breast cancer who had previously failed anti-HER2 targeted therapy. Efficacy and safety implications

Start Date28 Aug 2020

Sponsor / Collaborator

Zai Lab Ltd.

100 Clinical Results associated with Margetuximab

100 Translational Medicine associated with Margetuximab

100 Patents (Medical) associated with Margetuximab

Literatures (Medical) associated with Margetuximab

08 Feb 2024·AAPS PharmSciTech

Liquid Chromatography Tandem Mass Spectrometric Method for Quantification of Margetuximab in Rat Plasma and Application to a Pharmacokinetic Study.

Article

Author: Pridhvi Krishna Gaddey ; Raja Sundararajan

Margetuximab was approved for the treatment of advanced HER2+ breast cancer. A feasible analytical technique that can measure this drug was obligatory. In light of this, a novel and thoroughly validated liquid chromatographic (LC)-tandem mass spectrometric (MS/MS) approach was developed for the quantification of margetuximab in rat plasma. The liquid-liquid extraction method was used to extract the analyte from rat plasma. The analyte was separated using acetonitrile and formic acid buffer (30:70) as a mobile phase on Waters, alliance e-2695 model HPLC having Symmetry C₁₈ column, 150 mm × 4.6 mm, 3.5-µm column. The overall runtime was 6 min at a flow rate of 1.0 ml/min. The method showed significant sensitivity and acceptable linearity over the concentration range of 6-120 ng/ml. Accuracy was within 98.51-99.92%. The intraday precision ranged between 0.41 and 8.98% CV. Also, the findings of pharmacokinetic parameters such as C_max, t_max, AUC_0-∞, AUC_0-t, and half-life results of margetuximab showed that the technique was helpful for accurately measuring drug concentrations in rat plasma. The method that was developed was useful and effective for quantifying margetuximab.

29 Jan 2024·Anti-cancer agents in medicinal chemistry

An Expedition on Synthetic Methodology of FDA-approved Anticancer Drugs (2018-2021).

Article

Author: Vishakha S ; Navneesh N ; Balak Das Kurmi ; Ghanshyam Das Gupta ; Sant Kumar Verma ; Ankit Jain ; Preeti Patel

New drugs being established in the market every year produce specified structures for selective biological targeting. With medicinal insights into molecular recognition, these begot molecules open new rooms for designing potential new drug molecules. In this review, we report the compilation and analysis of a total of 56 drugs including 33 organic small molecules (Mobocertinib, Infigratinib, Sotorasib, Trilaciclib, Umbralisib, Tepotinib, Relugolix, Pralsetinib, Decitabine, Ripretinib, Selpercatinib, Capmatinib, Pemigatinib, Tucatinib, Selumetinib, Tazemetostat, Avapritinib, Zanubrutinib, Entrectinib, Pexidartinib, Darolutamide, Selinexor, Alpelisib, Erdafitinib, Gilteritinib, Larotrectinib, Glasdegib, Lorlatinib, Talazoparib, Dacomitinib, Duvelisib, Ivosidenib, Apalutamide), 6 metal complexes (Edotreotide Gallium Ga-68, fluoroestradiol F-18, Cu 64 dotatate, Gallium 68 PSMA-11, Piflufolastat F-18, 177Lu (lutetium)), 16 macromolecules as monoclonal antibody conjugates (Brentuximabvedotin, Amivantamab-vmjw, Loncastuximabtesirine, Dostarlimab, Margetuximab, Naxitamab, Belantamabmafodotin, Tafasitamab, Inebilizumab, SacituzumabGovitecan, Isatuximab, Trastuzumab, Enfortumabvedotin, Polatuzumab, Cemiplimab, Mogamulizumab) and 1 peptide enzyme (Erwiniachrysanthemi-derived asparaginase) approved by the U.S. FDA between 2018 to 2021. These drugs act as anticancer agents against various cancer types, especially non-small cell lung, lymphoma, breast, prostate, multiple myeloma, neuroendocrine tumor, cervical, bladder, cholangiocarcinoma, myeloid leukemia, gastrointestinal, neuroblastoma, thyroid, epithelioid and cutaneous squamous cell carcinoma. The review comprises the key structural features, approval times, target selectivity, mechanisms of action, therapeutic indication, formulations, and possible synthetic approaches of these approved drugs. These crucial details will benefit the scientific community for futuristic new developments in this arena.

11 Nov 2023·Clinics and research in hepatology and gastroenterology

New developments and standard of care in the management of advanced gastric cancer.

Review

Author: Léonard Depotte ; Juliette Palle ; Cosimo Rasola ; Chloé Broudin ; Vlad-Adrian Afrăsânie ; Antoine Mariani ; Aziz Zaanan

Advanced gastric adenocarcinoma is a common disease with a poor prognosis whose treatment has for decades been based on cytotoxic chemotherapy, including platinum salts in first-line, and taxane or irinotecan in second or later line. Recent years have seen the emergence of new drugs that have improved patient survival, such as trastuzumab in first-line for HER2-positive tumors, ramucirumab alone or in combination with paclitaxel in second-line, and trifluridine-tipiracil beyond the second-line treatment. More recently, two monoclonal antibodies have demonstrated their efficacy in combination with oxaliplatin-based first-line chemotherapy, nivolumab (anti-PD1) for PD-L1 CPS ≥5 tumors, and zolbetuximab for tumors overexpressing Claudin 18.2. In addition, regorafenib has been also showed effective in phase 3 trial for heavily pretreated patients. Based on phase 2 studies, trastuzumab-deruxtecan was approved in 2022 by the EMA for HER2-positive pretreated patients. This agent is currently evaluated in phase 3 study (DESTINY-Gastric04 trial), as are several other anti-HER2 (zanidatamab, margetuximab, tucatinib), immune checkpoint inhibitors, or targeted therapies (anti-FGFR2b).

News (Medical) associated with Margetuximab

07 Mar 2024

·www.biospace.com

MacroGenics Provides Update on Corporate Progress and 2023 Financial Results

Presentation of preliminary data from TAMARACK Phase 2 study of vobra duo in mCRPC patients expected at ASCO 2024 Initiation of Phase 1 study of MGC026, MacroGenics' first topoisomerase I inhibitor-based ADC Preclinical data on two topoisomerase I inhibitor-based ADC product candidates, MGC026 and MGC028, to be presented in posters at AACR Conference call scheduled for today at 4:30 p.m. ET ROCKVILLE, Md., March 07, 2024 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative antibody-based therapeutics for the treatment of cancer, today provided an update on its recent corporate progress and reported financial results for the year ended December 31, 2023. “We expect that 2024 will be an important year for MacroGenics, with multiple pipeline advancements anticipated," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. “Late last year, we completed enrollment of 177 patients in the TAMARACK Phase 2 study of vobra duo, which was ahead of schedule. We plan to present the initial TAMARACK clinical data in the second quarter of this year. Later in the year, we expect to share updated clinical data from the trial. In addition, we continue to enroll the LORIKEET Phase 2 study of lorigerlimab in mCRPC and expect to start enrolling patients in the dose expansion portion of the combination study of vobra duo and lorigerlimab. Finally, we are excited about the potential of our topoisomerase I inhibitor-based ADCs, including MGC026, for which we recently began a Phase 1 study, and MGC028, for which we anticipate submitting an IND by year end.” Updates on Proprietary Investigational Programs Recent progress and anticipated events related to MacroGenics’ investigational product candidates are highlighted below. Vobramitamab duocarmazine (vobra duo) is an antibody-drug conjugate (ADC) that targets B7-H3, an antigen with broad expression across multiple solid tumors and a member of the B7 family of molecules involved in immune regulation. MacroGenics completed enrollment of the TAMARACK Phase 2 study of vobra duo in November 2023. A total of 177 patients have been dosed in the study, exceeding the study design goal of 100 participants. TAMARACK is being conducted in patients with metastatic castration-resistant prostate cancer (mCRPC) who were previously treated with one prior androgen receptor axis-targeted therapy (ARAT). Participants may have received up to one prior taxane-containing regimen, but no other chemotherapy agents. The TAMARACK study is designed to evaluate vobra duo at two different doses: 2.0 mg/kg or 2.7 mg/kg every four weeks (q4W). In late January 2024, the TAMARACK independent data safety monitoring committee (IDSMC) recommended continuing the study. Also, in early February, MacroGenics submitted an abstract to the American Society of Clinical Oncology Annual Meeting (ASCO) that included the safety data reviewed by the IDSMC, based on a January 2024 data cut-off. The Company anticipates presenting updated safety and preliminary efficacy data at ASCO. MacroGenics intends to expand the TAMARACK study of vobra duo by enrolling patients with non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), melanoma, squamous cell carcinoma of the head and neck (SCCHN) and anal cancer. The Company expects to initiate dosing in these additional cohorts in mid-2024. MacroGenics continues to enroll a Phase 1/2 dose escalation study of vobra duo in combination with lorigerlimab in patients with various advanced solid tumors. The Company anticipates commencing a dose expansion study of this combination in mCRPC and at least one additional indication in 2024. Lorigerlimab is a bispecific, tetravalent PD-1 × CTLA-4 DART® molecule. MacroGenics is enrolling LORIKEET, a randomized Phase 2 study of lorigerlimab in combination with docetaxel vs. docetaxel alone in second-line, chemotherapy-naïve mCRPC patients. A total of 150 patients are planned to be treated in the 2:1 randomized study. The current trial design includes a primary study endpoint of radiographic progression-free survival (rPFS). The Company anticipates providing a study update in the second half of 2024. MGD024 is a next-generation, humanized CD123 × CD3 DART molecule designed to minimize cytokine-release syndrome, while maintaining anti-tumor cytolytic activity, and permitting intermittent dosing through a longer half-life. MacroGenics continues to enroll patients in a Phase 1 dose-escalation study of MGD024 in patients with CD123-positive neoplasms, including acute myeloid leukemia and myelodysplastic syndromes. MGC026 is a clinical ADC directed against B7-H3, incorporating a novel site-specific linker and topoisomerase I inhibitor-based cytotoxic payload developed by Synaffix (a Lonza company). With distinct mechanisms of action, vobra duo and MGC026 may address different cancers, tumor stages, or be used in combination with alternate agents — or potentially with one another — to enhance their clinical utility. The Company plans to present MGC026 preclinical data at the upcoming American Association for Cancer Research (AACR) Annual Meeting in April 2024. MacroGenics recently initiated a Phase 1 dose escalation study of MGC026 in patients with advanced solid tumors. MGC028 is a preclinical ADC incorporating an ADAM9-targeting antibody and represents the second MacroGenics ADC molecule that incorporates Synaffix’s novel site-specific linker and topoisomerase I inhibitor-based cytotoxic payload. ADAM9 (a disintegrin and metalloprotease domain 9) is a member of the ADAM family of multifunctional type 1 transmembrane proteins that play a role in tumorigenesis and cancer progression and is overexpressed in multiple cancers, making it an attractive target for cancer treatment. MacroGenics plans to present preclinical data for MGC028 at the upcoming AACR Annual Meeting in April and currently anticipates submitting an investigational new drug (IND) application for MGC028 by the end of 2024. MGC028 is the second ADAM9-targeted ADC that MacroGenics has pursued. The first was IMGC936, a molecule with a maytansinoid payload that was advanced under a co-development arrangement with ImmunoGen, Inc. (ImmunoGen, now part of AbbVie). Under the 50/50 collaboration, ImmunoGen led clinical development and completed initial Phase 1 dose escalation and dose expansion studies. Neither MacroGenics nor AbbVie intends to further pursue development of IMGC936 as the molecule did not achieve pre-established clinical safety and efficacy benchmarks. The Company believes ADAM9 remains a promising target for delivery of an alternative cytotoxic payload. Enoblituzumab is an Fc-optimized monoclonal antibody that targets B7-H3. MacroGenics’ academic collaborators have initiated the HEAT study, an investigator-sponsored, randomized Phase 2 clinical trial. This study will evaluate the activity of neoadjuvant enoblituzumab given prior to radical prostatectomy in up to 219 men with high-risk localized prostate cancer. 2023 Financial Results Cash Position: Cash, cash equivalents and marketable securities balance as of December 31, 2023, was $229.8 million, compared to $154.3 million as of December 31, 2022. Revenue: Total revenue was $58.7 million for the year ended December 31, 2023, compared to total revenue of $151.9 million for the year ended December 31, 2022. The decrease was primarily due to a decrease in revenue from collaborative and other agreements. R&D Expenses: Research and development expenses were $166.6 million for the year ended December 31, 2023, compared to $207.0 million for the year ended December 31, 2022. The decrease was primarily due to decreased manufacturing-related costs for vobra duo, decreased development and clinical trial costs related to margetuximab, and decreased costs related to discontinued studies, partially offset by increased expenses related to MGC026 and MGC028 development. SG&A Expenses: Selling, general and administrative expenses were $52.2 million for the year ended December 31, 2023, compared to $58.9 million for the year ended December 31, 2022. The decrease was primarily related to decreased selling costs for MARGENZA®. Other Income: During the year ended December 31, 2023, MacroGenics received $100.0 million proceeds from the sale of its single-digit royalty interest on global net sales of TZIELD® to DRI Healthcare Acquisitions LP. In addition, the Company received a $50.0 million milestone payment from Sanofi S.A. related to the achievement of a primary endpoint in a TZIELD clinical study. Under GAAP guidelines and pursuant to Financial Accounting Standards Board’s Accounting Standards Codification 470, this combined $150.0 million was included in Other Income as a “Gain on royalty monetization arrangement” in 2023. Net Loss: Net loss was $9.1 million for the year ended December 31, 2023, compared to net loss of $119.8 million for the year ended December 31, 2022. Shares Outstanding: Shares of common stock outstanding as of December 31, 2023 were 62,070,627. Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $229.8 million as of December 31, 2023, in addition to projected and anticipated future payments from partners and product revenues should extend its cash runway into 2026. The Company’s expected funding requirements reflect anticipated expenditures related to the Phase 2 TAMARACK clinical trial, the Phase 2 LORIKEET study as well as MacroGenics’ other ongoing clinical and preclinical studies. Conference Call Information To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at A recorded replay of the webcast will be available shortly after the conclusion of the call and archived on MacroGenics’ website for 30 days following the call. MACROGENICS, INC. SELECTED CONSOLIDATED BALANCE SHEET DATA (Amounts in thousands) December 31, 2023 December 31, 2022 Cash, cash equivalents and marketable securities $ 229,805 $ 154,346 Total assets 298,418 280,468 Deferred revenue 80,893 69,468 Total stockholders' equity 152,613 142,013 MACROGENICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Amounts in thousands, except share and per share data) 2023 2022 2021 Revenues: Collaborative and other agreements $ 28,990 $ 119,303 $ 63,294 Product sales, net 17,939 16,727 12,349 Contract manufacturing 9,833 13,988 — Royalty revenue 431 — — Government agreements 1,556 1,923 1,804 Total revenues 58,749 151,941 77,447 Costs and expenses: Cost of product sales 619 3,351 2,651 Cost of manufacturing services 7,603 4,033 — Research and development 166,583 207,026 214,577 Selling, general and administrative 52,188 58,949 63,014 Total costs and expenses 226,993 273,359 280,242 Loss from operations (168,244 ) (121,418 ) (202,795 ) Gain on royalty monetization arrangement 150,930 — — Interest and other income 9,686 1,660 680 Interest expense (1,430 ) — — Net loss (9,058 ) (119,758 ) (202,115 ) Other comprehensive income (loss): Unrealized gain (loss) on investments (1 ) 56 (54 ) Comprehensive income (loss) $ (9,059 ) $ (119,702 ) $ (202,169 ) Basic and diluted net loss per common share $ (0.15 ) $ (1.95 ) $ (3.37 ) Basic and diluted weighted average common shares outstanding 61,929,198 61,433,124 59,944,717 About MacroGenics, Inc. MacroGenics (the Company) is a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at MacroGenics, the MacroGenics logo, MARGENZA and DART are trademarks or registered trademarks of MacroGenics, Inc. Cautionary Note on Forward-Looking Statements Any statements in this press release about future expectations, plans and prospects for MacroGenics (“Company”), including statements about the Company’s strategy, future operations, clinical development of the Company’s therapeutic candidates, including initiation and enrollment in clinical trials, expected timing of results from clinical trials, discussions with regulatory agencies, commercial prospects of or product revenues from MARGENZA and the Company’s product candidates, if approved, manufacturing services revenue, milestone or opt-in payments from the Company’s collaborators, the Company’s anticipated milestones and future expectations and plans and prospects for the Company, as well as future global net sales of TZIELD and the Company’s ability to achieve the milestone payments set forth under the terms of the agreement with DRI (or its successors or assigns with respect to such agreement), and other statements containing the words “subject to”, "believe", “anticipate”, “plan”, “expect”, “intend”, “estimate”, “potential,” “project”, “may”, “will”, “should”, “would”, “could”, “can”, the negatives thereof, variations thereon and similar expressions, or by discussions of strategy constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks that TZIELD, vobramitamab duocarmazine, lorigerlimab, ZYNYZ, MARGENZA or any other product candidate’s revenue, expenses and costs may not be as expected, risks relating to TZIELD, vobramitamab duocarmazine, lorigerlimab, ZYNYZ, MARGENZA or any other product candidate’s market acceptance, competition, reimbursement and regulatory actions; our ability to provide manufacturing services to our customers; the uncertainties inherent in the initiation and enrollment of future clinical trials; the availability of financing to fund the internal development of our product candidates; expectations of expanding ongoing clinical trials; availability and timing of data from ongoing clinical trials; expectations for the timing and steps required in the regulatory review process; expectations for regulatory approvals; expectations of future milestone payments; the impact of competitive products; our ability to enter into agreements with strategic partners and other matters that could affect the availability or commercial potential of the Company's product candidates; business, economic or political disruptions due to catastrophes or other events, including natural disasters, terrorist attacks, civil unrest and actual or threatened armed conflict, or public health crises such as the novel coronavirus (referred to as COVID-19 pandemic); and other risks described in the Company's filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views only as of the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so, except as may be required by law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. CONTACTS: Jim Karrels, Senior Vice President, CFO 1-301-251-5172 info@macrogenics.com

Phase 1Phase 2Financial StatementASCOAACR

04 May 2023

·www.biospace.com

Compass Therapeutics Reports First Quarter Financial Results and Provides Corporate Update

Initiated patient enrollment in a U.S. Phase 2/3 study of CTX-009 (DLL4 /VEGF-A bispecific antibody) in patients with advanced biliary tract cancers (BTC). Top line data is expected in the first half of 2024 Continue to enroll in a U.S. Phase 2 study of CTX-009 in patients with advanced colorectal cancer (CRC). Initial data expected in the third quarter of 2023 Presented results of a Phase 2 study of CTX-009 in combination with paclitaxel in patients with BTC at the 2023 ASCO GI Cancers Symposium Expanded the management team with the appointment of Minori Rosales, M.D. PhD, as Senior Vice President & Head of Clinical Development Appointed Richard Lindahl, M.B.A., EVP & CFO of Emergent BioSolutions, to the Compass Board Ended the first quarter with $175 million in cash and marketable securities, providing cash runway for the company into 2026 BOSTON, May 04, 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 reported first quarter 2023 financial results. “We are very pleased with the progress we are making toward our enrollment goals in both of our CTX-009 clinical trials and look forward to reaching our goal of making CTX-009 available to patients with various cancers,” said Thomas J. Schuetz, MD, PhD, Co-Founder and Chief Executive Officer. “We anticipate initial results from the CTX-009 colorectal study in Q3 of this year and top-line data from the CTX-009 study in BTC in the first half of 2024. Additionally, we expect initial data from our CTX-471 combination study later this year.” “In addition to our progress in the clinic, Compass is excited to welcome two experienced leaders to the Company. Dr. Minori Rosales has joined our leadership team as SVP & Head of Clinical Development. She brings with her years of successful oncology drug development from her tenure at Eli Lilly, Merck, MacroGenics and other BioPharma companies. Richard Lindahl, currently the EVP & CFO at Emergent BioSolutions and prior to this the SVP and Treasurer of Sprint-Nextel, was appointed to our board as a new independent director and as the Chair of our Audit Committee,” said Vered Bisker-Leib, PhD, MBA, President and Chief Operating Officer. “We look forward to leveraging Rich’s broad financial expertise on our board and Minori’s unique clinical and medical expertise in advancing our clinical programs.” Development Pipeline Update and Highlights: CTX-009 (DLL4 and VEGF-A bispecific antibody) Initiated enrollment and dosing of patients in the U.S. Phase 2/3 study of CTX-009 in combination with Paclitaxel in BTC This randomized Phase 2/3 study is designed to enroll 150 patients with BTC who have received one prior systemic therapy The primary endpoint of the study is overall response rate (ORR), and secondary endpoints include progression free survival (PFS), overall survival (OS), clinical benefit rate (CBR) and duration of response (DOR) Top line data from this study is expected in the first half of 2024 Enrolling patients in the U.S. Phase 2 study of CTX-009 as a monotherapy in patients with advanced, metastatic colorectal cancer The study design is an Adaptive Simon Two-Stage, with Stage 1 of the study enrolling 37 patients. If there are 3 or more responses confirmed in Stage 1, the study will advance to Stage 2 and an additional 47 patients will be enrolled Patients are being evaluated for safety and tolerability, as well as clinical response First patient dosed in January 2023. Initial results from this study are expected in the third quarter of 2023 Presented Phase 2 results of CTX-009 in combination with paclitaxel in patients with BTC at the 2023 ASCO GI Cancers Symposium Data showed 9 confirmed partial responses (PRs) among 24 evaluable patients for an ORR of 37.5% in the second- and third-line settings In the second-line setting, an ORR of 63.6% was observed (7 out of 11 patients responded) Median PFS was 9.4 months and median OS was 12.5 months Safety and tolerability were consistent with prior studies CTX-471 (CD137 + PD-1) Advancing enrollment of the Phase 1 combination arm of CTX-471 (CD137 agonistic antibody) and KEYTRUDA® (pembrolizumab) in patients with select solid tumors The first two dose cohorts in the study (n=6) have been fully enrolled Initial results from the combination arm are expected in the second half of 2023 CTX-8371 (PD-1 x PD-L1) Targeting IND submission in the third quarter of 2023 and initiating a clinical trial in the second half of 2023 Corporate Update In April 2023, the company appointed Minori Rosales, MD PhD, as Senior Vice President & Head of Clinical Development. Dr. Minori Rosales brings significant clinical experience as a proven leader in both biotech and pharmaceutical industries. Most recently, Dr. Rosales was Chief Development Officer for Sesen Bio. Previously, she served as Vice President, R&D at MacroGenics where she advanced margetuximab in clinical development. Earlier in her career, Minori served in executive and senior clinical leadership roles at Merck and Eli Lilly, where she led label-expansion and registrational studies for pembrolizumab and ramicirumab respectively in various oncology indications, including biliary tract cancer, gastric, esophageal, and HCC, among others. Dr. Rosales obtained her medical degree from Yamaguchi University and her PhD in tumor immunology from Kansai Medical University in Japan. Also in April 2023, the Compass’s board of directors unanimously appointed Richard Lindahl, M.B.A, Executive Vice President, Chief Financial Officer and Treasurer at Emergent BioSolutions, as a director and as Chair of the Audit Committee. Mr. Lindahl has over 20 years of experience in financial leadership roles. Prior to Emergent BioSolutions, from 2009-2017, Mr. Lindahl was Chief Financial Officer at CEB, a NYSE-listed technology company. Earlier in his career, Mr. Lindahl was at Sprint Nextel Corporation in roles of increasing responsibility, culminating in the position of Senior Vice President & Corporate Treasurer. Mr. Lindahl has a BA from Dartmouth College and an MBA from the University of Virginia. Financial Results Net loss for the quarter ended March 31, 2023, was $7.8 million or $0.06 per share, compared to $7.2 million or $0.07 per share for the same period in 2022. Cash Position As of March 31, 2023, cash and marketable securities were $175 million as compared to $187 million as of December 31, 2022, providing the Company with an anticipated cash runway into 2026. During the first quarter of 2023, the Company used $12 million of cash to fund operations. Research and development (R&D) Expenses R&D expenses were $6.6 million for the first quarter ended March 31, 2023, as compared to $4.4 million for the same period in 2022, an increase of $2.2 million or 50%. The change for the year was primarily attributable to a net increase of $2.5 million in program costs, resulting primarily from $3.3 million additional spending related to CTX-009 partially offset by $0.8 million decrease in spending on other programs. General and Administrative (G&A) Expenses G&A expenses were $3.1 million for the quarter ended March 31, 2023, as compared to $2.8 million for the same period in 2022, an increase of $0.3 million or 11%. Upcoming Investor Conferences Compass management will participate in three upcoming investor conferences: Inaugural EF Hutton Global Conference Date: May 10-11, 2023 Location: New York, NY Jefferies Global Healthcare Conference Date: June 7-9, 2023 Location: New York NY 2023 World Medical Innovation Forum (Bank of America and Mass General) Date: June 12-14, 2023 Location: Boston, MA Live webcasts presentations, when available, will be under “News & Events” in the Investors section of the Company’s website located at . KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. 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. The company 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. The company was founded in 2014 and is headquartered in Boston, Massachusetts. For more information, visit the Compass Therapeutics 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 financial position to continue advancing its product candidates, expectations about cash runway, business and development plans, and statements regarding Compass’s product candidates, their 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 ir@compasstherapeutics.com Media Contact Anna Gifford, Communications Manager media@compasstherapeutics.com 617-500-8099 Compass Therapeutics, Inc. and Subsidiaries Consolidated Statements of Operations (In thousands, except per share data) Three Months Ended March 31, 2023 2022 (unaudited) Operating expenses: Research and development $ 6,638 $ 4,415 General and administrative 3,073 2,767 Total operating expenses 9,711 7,182 Loss from operations (9,711 ) (7,182 ) Other income 1,874 20 Loss before income tax expense (7,837 ) (7,162 ) Income tax expense — — Net loss $ (7,837 ) $ (7,162 ) Net loss per share - basic and diluted $ (0.06 ) $ (0.07 ) Basic and diluted weighted average shares outstanding 126,375 100,858 Compass Therapeutics, Inc. and Subsidiaries Condensed Consolidated Balance Sheets (In thousands) March 31, 2023 December 31, 2022 (unaudited) Assets Current assets: Cash and cash equivalents $ 27,027 $ 34,946 Marketable securities 148,143 151,663 Prepaid expenses and other current assets 8,432 8,182 Total current assets 183,602 194,791 Property and equipment, net 1,373 1,567 Operating lease, right-of-use ("ROU") asset 2,680 2,967 Other assets 320 320 Total assets $ 187,975 $ 199,645 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 1,125 $ 3,382 Accrued expenses 8,943 11,690 Operating lease obligations, current portion 1,122 1,097 Total current liabilities 11,190 16,169 Operating lease obligations, long-term portion 1,520 1,838 Total liabilities 12,710 18,007 Total stockholders' equity 175,265 181,638 Total liabilities and stockholders' equity $ 187,975 $ 199,645

Phase 1Phase 2Executive ChangeFinancial StatementClinical Result

17 Oct 2022

·endpts.com

Gilead calls dibs on a PhI bispecific, hands over $60M to cash-strapped MacroGenics

On a continual search for more baskets to put its oncology eggs in, Gilead has landed on a hot target: CD123. Specifically, Gilead is grabbing a bispecific antibody from MacroGenics, a seasoned biotech player that’s seen its share of setbacks. For $60 million upfront, Gilead buys an exclusive option to license MGD024 after MacroGenics wraps up the ongoing Phase I study and calls dibs on two other early-stage bispecific research programs. The whole deal, inclusive of target nomination, option fees and milestones, can add up to $1.7 billion. For MacroGenics, that represents significant cash infusion “as cash runway has been weighing on the stock,” SVB Securities analyst Jonathan Chang wrote in a note. CD123 has been pursued by multiple drugmakers as a promising target for hematologic cancers such as acute myeloid leukemia and myelodysplastic syndromes — and tripped some of them up. Because CD123 also sits on healthy cells, the therapeutic window is relatively narrow, which can present challenges when it comes to dosing frequency. It was the difficulty, alongside a competitive landscape for AML, that pushed Novartis to bow out of a major deal with Xencor, according to the biotech. MacroGenics’ MGD024 is made on its DART platform, which it says produces molecules with more stable structure. Like most cancer-killing bispecifics, it binds to a tumor antigen on one side and CD3 on T cells on the other; but according to the company, its CD3 component is designed to minimize cytokine release syndrome, thus lowering toxicity. Bill Grossman “We believe MGD024, with its potential to reduce CRS and permit intermittent dosing through a longer half-life, could translate to more patient-friendly dosing and enhanced clinical outcomes for people living with AML and MDS,” said Bill Grossman, Gilead’s senior vice president, oncology clinical development. Gilead, which boasted about a network of 30 oncology partners in April, has continued to grow the cancer pipeline in pursuit of a goal to accrue more than 20 indication approvals by 2030. In a recent pact, it wagered $300 million cash on Dragonfly’s 5T4-targeting therapy designed to drag natural killer — NK cells — along with other cancer killing T cells into the tumor microenvironment. Gilead joins a lineup of Big Pharma partners betting big on TriNKETs, with a $300M cash ante to get started on a 5T4 As Chang of SVB Securities noted, MGD024 is actually MacroGenics’ second CD123xCD3 bispecific after the company discontinued a first-gen compound dubbed flotetuzumab earlier this year. While MacroGenics once soared on high expectations for its HER2 drug Margenza, which won a head-to-head against Roche’s top-selling Herceptin in progression-free survival, the commercialization effort proved barren. Final results from the trial suggesting Margenza performed worse than Herceptin on overall survival didn’t help — and total 2021 sales were only $12.3 million. It was so strapped for cash that execs noted in a recent earnings call they didn’t have enough money to fund a Phase II/III study for the lead experimental drug, the antibody-drug conjugate MGC018, in prostate cancer. The latest cash infusion may boost investors’ confidence that they will find the money, as promised, to begin the trial later this year.

CollaborateAntibodyADC

100 Deals associated with Margetuximab

External Link

KEGG	Wiki	ATC	Drug Bank
D10446	Margetuximab	L01XC	DB14967

R&D Status

Indication	Highest Phase	Country/Location	Organization
HER2 Positive Breast Cancer	Approved	CN More	Zai Lab Ltd. More
Breast Cancer	Phase 2	US More	MacroGenics, Inc. More
Stomach Cancer	Phase 2	KR More	MacroGenics, Inc. More
Non-Small Cell Lung Cancer	Discontinued	US More	MacroGenics, Inc. More
Esophageal Carcinoma	Pending	US More	MacroGenics, Inc. More

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04082364 (MAHOGANY, Pubmed) Manual	Phase 2/3	HER2 Positive Gastroesophageal Adenocarcinoma First line HER2 Positive \| PD-L1 Positive	43	Margetuximab+Retifanlimab	whzicrknlg(nidgpiflti) = sahxndohmq xpyragxgvi (hnqzfyrxmh ) View more	Positive	24 Aug 2022
NCT04082364 (NCT04082364, ESMO_GI2021)	Phase 2/3	Metastatic HER2 positive gastroesophageal junction cancer First line HER2+ \| PD-L1+ \| microsatellite instability	-	Margetuximab+Retifanlimab	(varotqfrcb) = toplnltnxw omqqvdusmq (sjohtnmala )	Positive	03 Jul 2021
ASCO2022	Phase 3	Advanced breast cancer	-	Margetuximab	(ecnvhfxjer) = these therapies are generally well tolerated with manageable side effects as listed in the table. tbymyjfhit (mldgtsdfnk ) View more	Positive	02 Jun 2022
				Trastuzumab
NCT02492711 (SOPHIA, Pubmed) Manual	Phase 3	Advanced HER2-Positive Breast Carcinoma HER2 Positive	536	Chemotherapy+Margetuximab	(dekgtoxvwv) = iwmwvgvjor ljsekfenpy (uphamqgrkx, 18.89 ~ 25.07)	Non-superior	04 Nov 2022
				Chemotherapy+Trastuzumab	(dekgtoxvwv) = aysugbrzdl ljsekfenpy (uphamqgrkx, 18.69 ~ 24.18)
NCT03219268 (ASCO2020) Manual	Phase 1	Advanced Malignant Solid Neoplasm \| Hematologic Neoplasms	207	MGD013 (Dose Escalation)	ikvmiajaxb(pcqaocccxa) = consistent with events observed with anti-PD-1 antibodies. behnszxxxg (zkhzsnawpq ) View more	Positive	29 May 2020
				MGD013 (epithelial ovarian cancer)
NCT02689284 (CP-MGAH22-05, Pubmed)	Phase 1/2	HER2 Positive Gastroesophageal Adenocarcinoma	86	Margetuximab and Pembrolizumab combination therapy	(pweflwhtzd) = rxtfontatp rubxojvkzk (gaptjilosv )	-	25 Apr 2023
NCT01148849 (Pubmed \| Ann Oncol) Manual	Phase 1	HER2 Positive Solid Tumors HER2-positive	66	margetuximab (HER2 positive)	(pknahvptjr) = Treatment was well-tolerated, with mostly Grade 1 and 2 toxicities consisting of constitutional symptoms such as pyrexia, nausea, anemia, diarrhea, and fatigue. pfecahbsjr (doiluckvhq )	Positive	01 Apr 2017
NCT02689284 (CP-MGAH22-05, Pubmed \| Lancet Oncol) Manual	Phase 1/2	HER2 Positive Gastroesophageal Adenocarcinoma Second line HER2 Positive	95	pembrolizumab+Margetuximab	(uyijoozwau) = floqjrjnbp yxcatlbwxa (rjlfbkujzw ) View more	Positive	01 Aug 2020
NCT02492711 (SOPHIA, Pubmed \| JAMA Oncol)	Phase 3	HER2 Positive Breast Cancer	536	Margetuximab	(izjitnften) = ccfjjvbwav sexzlvlubf (smhsqmsola ) View more	Positive	22 Jan 2021
				Trastuzumab	(izjitnften) = lnqqqzerxd sexzlvlubf (smhsqmsola ) View more
SOPHIA (NEWS) Manual	Not Applicable	HER2 Positive Breast Cancer HER2 positive	-	Chemotherapy+Margetuximab	(ehnrmnkreu) = ynsrhvoyva yatawykjcx (rjrzsxiuif ) View more	Superior	09 Nov 2022
				Chemotherapy+Trastuzumab	(ehnrmnkreu) = kjpyxdfaip yatawykjcx (rjrzsxiuif ) View more

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