Teplizumab

More than 1.4 million Americans live with type 1 diabetes, an autoimmune disease. Screening for type 1 diabetes can reduce the risk of life-threatening complications at diagnosis. NEW YORK, March 26, 2024 /PRNewswire/ -- In recognition of Autoimmune Awareness Month, JDRF, the leading global type 1 diabetes (T1D) research and advocacy organization, recognizes all those who live with T1D, an autoimmune condition that causes the pancreas to make very little insulin or none at all. T1D is one of nearly 100 autoimmune diseases that occur when the body's immune system mistakenly attacks another part of the body. While there are currently no cures for autoimmune diseases, screening options may be available, which can reduce the risk of life-threatening complications at diagnosis and provide people time to develop a plan in partnership with their healthcare team. JDRF continues advancing breakthroughs to find cures for T1D, including research that has the potential to impact other autoimmune diseases. Research shows about 25% of people with one autoimmune disease may develop a second, and around 50 million Americans have one or more autoimmune diseases. This includes one-fifth of the T1D community. Chelsea Morra was diagnosed with T1D at the age of seven and lived with it for 14 years before she was diagnosed with a second autoimmune condition, celiac disease, at age 21. She was then diagnosed with vitiligo in 2021. Chelsea has a family history of T1D and celiac disease, and she knew her T1D put her at a higher risk of developing a second autoimmune disease. Unfortunately, this didn't diminish the complexity that an additional diagnosis added to her day-to-day T1D management. "When I learned I had celiac disease, the stress of a new diagnosis impacted my type 1 diabetes and all aspects of my life," said Chelsea Morra. "I had to relearn my T1D in a sense. Identifying what new foods I could eat and understanding how they impacted my blood sugars was challenging," she said. "Fortunately, I've been able to manage my autoimmune diseases and have remained healthy, but knowing your risk for developing an autoimmune disease and accessing any available screening can be helpful for your overall health." In recent years, JDRF-funded research has discovered that it's possible to detect T1D before a clinical diagnosis, even before symptoms start, by identifying the presence of specific autoantibodies through a simple blood test. Screening for T1D and follow-up monitoring can have significant benefits, particularly if results indicate that a person is likely to develop T1D in their lifetime. "Screening for T1D before symptoms occur presents the opportunity to change the course of the disease and improve the health of those who may develop it," said Anastasia Albanese-O'Neill, JDRF Associate Vice President of Community Screening and Clinical Trial Education. "The ability to identify T1D early, before insulin is required, is a huge breakthrough that can help people avoid serious complications at diagnosis, give them time to prepare, consider research, and potentially access therapies that could delay the clinical diagnosis. This is why it's so important that people understand the importance of screening and monitoring for T1D and other autoimmune conditions." In the fall of 2022, the FDA approved the first disease-modifying therapy that can delay clinical T1D in individuals in early stages, teplizumab. JDRF supported the development of teplizumab for over 30 years. JDRF encourages those with T1D and other autoimmune diseases to share their stories and continue to build awareness of their conditions and opportunities for screening. For more information about T1D screening and monitoring, visit jdrf.org and jdrf.org/T1Detect. About JDRF JDRF's mission is to accelerate life-changing breakthroughs to cure, prevent, and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally and globally for efficiency and broader fundraising impact, and uniting on a global stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement, and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter (@JDRF), Facebook (@myjdrf), and Instagram (@jdrfhq). About Type 1 Diabetes (T1D) T1D is an autoimmune condition that causes the pancreas to make very little insulin or none at all. This leads to dependence on insulin therapy and the risk of short or long-term complications, which can include highs and lows in blood sugar; damage to the kidneys, eyes, nerves, and heart; and even death if left untreated. Globally, it impacts nearly 9 million people. Many believe T1D is only diagnosed in childhood and adolescence, but diagnosis in adulthood is common and accounts for nearly 50% of all T1D diagnoses. The onset of T1D has nothing to do with diet or lifestyle. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. There is currently no cure for T1D. Contact: Casey Fielder [email protected] 509-651-0087 SOURCE JDRF International

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