Panitumumab

Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX-based therapies in pancreatic, colon and appendiceal cancersBASKING RIDGE, N.J., Dec. 10, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced the successful completion of patient enrollment in all three cohorts of the Phase 1b/2a CENDIFOX trial. This investigator-initiated trial, led by Dr. Anup Kasi at The University of Kansas (“KU”) Cancer Center, is evaluating the safety and efficacy of Lisata's iRGD cyclic peptide product candidate, certepetide, in combination with FOLFIRINOX-based therapies for pancreatic, colon, and appendiceal cancers. “The successful enrollment of all three cohorts in the CENDIFOX trial is another significant milestone, bringing us closer to validating certepetide’s potential as a transformative treatment for advanced solid tumors,” stated Kristen K. Buck, M.D., Executive Vice President of Research and Development and Chief Medical Officer of Lisata. “By addressing the unmet medical needs of patients with solid tumors, we aim to improve patient outcomes and augment the standard-of-care paradigm. We are excited about the progress of the CENDIFOX trial and eagerly anticipate reporting the results from this important study in 2025.” The open-label CENDIFOX trial is designed to assess the safety and therapeutic effects of combining certepetide with neoadjuvant FOLFIRINOX regimens, with or without panitumumab, across pancreatic, colon, and appendiceal cancers. The study, conducted solely at the KU Cancer Center, enrolled a total of 66 patients (35 resectable and borderline resectable pancreatic cancer patients, 18 high-grade colon and appendiceal cancer patients with peritoneal metastasis, and 13 colon cancer patients with oligo-metastatic disease). The trial will provide Lisata with valuable pre- and post-treatment tumor tissue data for immune profiling, along with long-term patient outcomes information. The trial is funded by the KU Cancer Center and Lisata is supplying certepetide. “We are delighted to complete enrollment in all three cohorts in the CENDIFOX study and are encouraged by certepetide’s potential to improve outcomes for patients with advanced solid tumors,” stated Dr. Anup Kasi, the study’s Principal Investigator at the KU Cancer Center. “We are eager to analyze the data from each cohort to determine the efficacy of this novel therapy.” For more information on the CENDIFOX trial, please visit www.clinicaltrials.gov/study/NCT05121038. About Certepetide Certepetide, an internalizing RGD, or arginylglycylaspartic acid, (iRGD) cyclic peptide product, is an investigational drug designed to selectively activate the C-end rule active transport mechanism in a tissue-specific manner, resulting in systemically co-administered agents more efficiently penetrating and accumulating in the tissue. To date, certepetide has demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing oncology clinical trials designed to demonstrate its ability to enhance the effectiveness of standard-of-care chemotherapy for pancreatic cancer, as well as the combination of chemotherapy and immunotherapy in a variety of solid tumors. Beyond its promising applications in oncology, certepetide's unique mechanism of action has the potential to be explored in various non-oncology settings. Its ability to selectively target specific tissues could offer new therapeutic possibilities for a range of diseases. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.), as well as Orphan Drug Designation for glioma, osteosarcoma, and cholangiocarcinoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). About Lisata Therapeutics Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s internalizing RGD, or arginylglycylaspartic acid, (iRGD) cyclic peptide product candidate, certepetide, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to selectively target and penetrate solid tumors more effectively. Lisata has already established noteworthy commercial and R&D partnerships based on its CendR Platform® technology. The Company expects to announce numerous milestones over the next 1.5 years and believes that its projected capital will fund operations into early 2026, encompassing anticipated data milestones from its ongoing and planned clinical trials. Learn more about certepetide’s mechanism of action in our short film. For more information on the Company, please visit www.lisata.com. About The University of Kansas Cancer Center The University of Kansas Cancer Center is transforming cancer research and clinical care by linking an innovative approach to drug discovery, delivery and development to a nationally accredited patient care program. Our consortium center includes cancer research and health care professionals associated with the University of Kansas Medical Center and The University of Kansas Health System; the University of Kansas, Lawrence; The Stowers Institute for Medical Research; Children’s Mercy; and in partnership with members of the Masonic Cancer Alliance. Forward-Looking Statements This communication contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding the Company’s clinical development programs are forward-looking statements. In addition, when or if used in this communication, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Lisata or its management, may identify forward-looking statements. Examples of forward-looking statements include, but are not limited to, the potential efficacy of certepetide as a treatment for patients with cholangiocarcinoma and other solid tumors; statements relating to Lisata’s continued listing on the Nasdaq Capital Market; expectations regarding the capitalization, resources and ownership structure of Lisata; the approach Lisata is taking to discover and develop novel therapeutics; the adequacy of Lisata’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; and the difficulty in predicting the time and cost of development of Lisata’s product candidates. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: results observed from a single patient case study are not necessarily indicative of final results and one or more of the clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available, including the risk that unconfirmed responses may not ultimately result in confirmed responses to treatment after follow-up evaluations; the risk that product candidates that appeared promising in early research and clinical trials do not demonstrate safety and/or efficacy in larger-scale or later clinical trials; the safety and efficacy of Lisata’s product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in Lisata’s clinical programs, Lisata’s ability to finance its operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of Lisata’s scientific studies, Lisata’s ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in Lisata’s markets, the ability of Lisata to protect its intellectual property rights; and legislative, regulatory, political and economic developments. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Lisata’s Annual Report on Form 10-K filed with the SEC on February 29, 2024, and in other documents filed by Lisata with the Securities and Exchange Commission. Except as required by applicable law, Lisata undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. Lisata Therapeutics Contact: Investors:Lisata TherapeuticsJohn MendittoVice President, Investor Relations and Corporate CommunicationsPhone: 908-842-0084Email: jmenditto@lisata.com Media:ICR HealthcareElizabeth ColemanSenior AssociatePhone: 203-682-4783Email: elizabeth.coleman@icrhealthcare.com University of Kansas Cancer Center Contact: Media:Kay HawesAssociate Director of News and Media Relationskhawes@kumc.edu

– Achieved $49.3 million in OGSIVEO® (nirogacestat) net product revenue in the third quarter – – Long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors highlight further reductions in tumor size, increase in ORR, sustained improvement in desmoid tumor symptoms, and consistent safety profile – – Granted FDA Priority Review on NDA and received validation of EU Marketing Authorization Application for mirdametinib for the treatment of adults and children with NF1-PN – – $498 million in cash, cash equivalents and marketable securities as of September 30, 2024; expected to fund the Company through profitability, which is anticipated in the first half of 2026 – – Conference call and webcast scheduled for 8:30 a.m. ET today – STAMFORD, Conn., Nov. 12, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, today reported financial results for the third quarter of 2024 and provided an update on recent company developments. "In the third quarter, we continued to see robust growth in the U.S. launch of OGSIVEO for adults with desmoid tumors. As we approach nearly one full year on market, we are very encouraged by the metrics we are seeing regarding the breadth of physician prescribing, the number of patients on OGSIVEO, and the number of patients identified who may stand to benefit in the future from our medicine. We were also very pleased to receive Priority Review on our NDA for mirdametinib in NF1-PN this quarter, which positions us to potentially have a second approved medicine in early 2025," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for the remainder of the year is to continue building on OGSIVEO’s momentum in the U.S. while working to also bring it to patients in Europe, to advance our commercial preparations for mirdametinib in anticipation of serving patients with NF1-PN in the U.S. and Europe, and to progress our emerging portfolio." Recent Business Highlights and Upcoming Milestones OGSIVEO® (Nirogacestat) Continued strong commercial execution of the OGSIVEO launch, with net product revenue of $49.3 million in the third quarter of 2024, representing a 23% increase over the second quarter of 2024.In September, more than 800 unique desmoid tumor patients filled an OGSIVEO script. As of the end of the third quarter of 2024, approximately 420 treatment centers have ordered OGSIVEO since approval and approximately 65 percent of OGSIVEO patients on OGSIVEO are receiving the product in the 150 mg or 100 mg tablets blister packages. SpringWorks expects to complete the full transition to blister packages by the end of the year. In addition, through August 2024, approximately 10,000 unique desmoid tumor patients have been identified through the new desmoid tumor ICD-10 diagnostic code since the introduction of the code in October 2023. A Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors is under review with the European Medicines Agency (EMA).Long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors will be presented as a late-breaking oral presentation at the upcoming Connective Tissue Oncology Society (CTOS) 2024 Annual Meeting. These results, utilizing an August 2024 data cutoff date, showed that longer-term treatment with nirogacestat (median duration of treatment: 34 months) was associated with further reductions in tumor size, increase in objective response rate with additional partial responses and complete responses, sustained improvements in desmoid tumor symptoms including pain, and a consistent safety profile compared to the April 2022 data cutoff date for the primary results of the trial.SpringWorks expects to report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors in the first half of 2025.SpringWorks is continuing to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma. Mirdametinib SpringWorks' New Drug Application (NDA) for mirdametinib for the treatment of children and adults with NF1-PN was granted Priority Review by the U.S. Food and Drug Administration (FDA) and assigned a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2025.SpringWorks’ MAA for mirdametinib for the treatment of children and adults with NF1-PN was validated by the EMA and is currently under review.Results from the Phase 2b ReNeu trial were published in the Journal of Clinical Oncology.Additional data from the pediatric and adult cohorts of the Phase 2b ReNeu trial will be presented in oral and poster presentations at the 2024 Society for Neuro-Oncology (SNO) meeting. Data demonstrate that the deep responses in tumor volume reduction seen in ReNeu were achieved regardless of age, sex, target PN volume at baseline, tumor location, or progression status at baseline and suggest a trend between deep response and earlier achievement of a first confirmed response. Patients with deep responses also had a trend of longer treatment duration. In addition, adults and children with NF1-PN had clinically meaningful, early, and sustained improvement in health-related quality of life over the course of mirdametinib treatment.Data from the Phase 1/2 trial evaluating mirdametinib in pediatric and young adult patients with low-grade gliomas (LGG) are being presented in an oral presentation at the 2024 SNO meeting. Results from 23 patients enrolled in the Phase 1 portion of the study suggested that mirdametinib, which has shown high blood brain barrier penetration, has encouraging clinical activity in patients with recurrent/progressive LGG across a variety of MAPK pathway aberrations. Twelve (63%) of the 19 patients with measurable tumors achieved an objective response (one major, six partial, and five minor responses). The Phase 2 portion of the study is ongoing and recruiting patients. Emerging Pipeline A Phase 1b trial evaluating brimarafenib (BGB-3245) in adult patients with RAF mutant solid tumors is ongoing; additional data from the dose expansion portion of the study is expected to be presented in the second half of 2025. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.Several combination therapy oncology programs are ongoing: a Phase 1/2a study of brimarafenib and mirdametinib in MAPK mutant solid tumors, a Phase 1b trial of brimarafenib and Amgen’s EGFR inhibitor, panitumumab, in colorectal and pancreatic cancer patients with known MAPK pathway mutations, and a Phase 1b trial of mirdametinib with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with NRAS mutant solid tumors.In October, SpringWorks presented preclinical data at the EORTC-NCI-AACR symposium on Molecular Targets and Cancer therapeutics demonstrating the utility of SW-682 as a monotherapy and in combination with mirdametinib in subsets of head and neck cancer as well as in combination with KRAS G12C inhibitors in KRAS G12C mutant NSCLC. SW-682 is an investigational novel, oral, potent, and selective pan-TEAD inhibitor and patients are being enrolled in a Phase 1a trial in Hippo-mutant solid tumors. General Corporate In July, SpringWorks appointed Martin Mackay, Ph.D. to the Company’s Board of Directors. Dr. Mackay is a highly accomplished R&D executive with more than 30 years of pharmaceutical and biotech R&D experience, including leadership roles at Pfizer, AstraZeneca and Alexion. Third Quarter 2024 Financial Results Product Revenue: OGSIVEO net product revenue was $49.3 million in the third quarter of 2024.Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $61.6 million for the third quarter of 2024, compared to $46.5 million for the comparable period of 2023. The increase in SG&A expense was primarily attributable to commercial readiness activities to support the U.S. launch of mirdametinib, if approved.Research and Development (R&D) Expenses: R&D expenses were $42.3 million for the third quarter of 2024, compared to $37.5 million for the comparable period of 2023. The increase in R&D expenses was primarily attributable to an increase in costs related to drug manufacturing, clinical trials, other research, consulting and professional services.Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $53.5 million, or $0.72 per share, for the third quarter of 2024. This compares to a net loss of $79.4 million, or $1.27 per share, for the comparable period of 2023.Cash, Cash Equivalents, and Marketable Securities: Cash, cash equivalents and marketable securities were $498.1 million as of September 30, 2024. SpringWorks expects this cash position to fund the Company through profitability, which is expected in the first half of 2026. Conference Call Information SpringWorks will host a conference call and webcast today, Tuesday, November 12, at 8:30 a.m. ET to review its third quarter 2024 financial results and discuss recent business updates. To join the live webcast and view the corresponding slides, please click here. To access the live call by phone, please pre-register for the call by clicking here. Once registration is complete, participants will be provided with a dial-in number and conference code to access the call. A replay of the webcast will be available for a limited time following the event on the Investors and Media section of the Company’s website at https://ir.springworkstx.com. About SpringWorks Therapeutics SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. OGSIVEO® (nirogacestat), approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is the Company’s first FDA-approved therapy. SpringWorks also has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with programs ranging from preclinical development through advanced clinical trials. In addition to its wholly owned programs, SpringWorks has also entered into multiple collaborations with innovators in industry and academia to unlock the full potential for its portfolio and create more solutions for patients in need. For more information, visit www.springworkstx.com and follow @SpringWorksTx on X (formerly Twitter), LinkedIn, and YouTube. SpringWorks uses its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on SpringWorks' website in the Investors & Media section. Accordingly, investors should monitor such portions of the SpringWorks website, in addition to following press releases, SEC filings and public conference calls and webcasts. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, relating to our business, operations, and financial conditions, including but not limited to current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development and commercialization plans, our preclinical and clinical results, the market potential of OGSIVEO for adult patients with desmoid tumors, expectations to transition to blister packaging for OGSIVEO by the end of the year, expectations regarding the adequacy of the data contained in the nirogacestat MAA to serve as the basis for marketing approval of nirogacestat for the treatment of desmoid tumors in the European Union, the potential for mirdametinib to become an important new treatment for adult and pediatric NF1-PN patients, expectations regarding the timing and results of the reviews by the FDA and the EMA, as applicable, of each of the NDA and the MAA for mirdametinib for the treatment of adult and pediatric NF1-PN patients, including the FDA's PDUFA target action date for the NDA, our plans to report additional data from the Phase 2b ReNeu clinical trial at an upcoming medical conference in the fourth quarter of 2024, our plans to present additional data from the Phase 3 DeFi trial of nirogacestat at upcoming conferences, our plans for seeking regulatory approval for and making mirdametinib available for NF1-PN patients, if approved, expectations regarding the timing and initial data from the Phase 2 trial evaluating nirogacestat in patients with recurrent ovarian granulosa cell tumors, our expectations and the timing of the Phase 1a trial of SW-682, our plans to report additional clinical data of nirogacestat in combination with BCMA-directed therapies and initiate additional planned Phase 1 collaborator studies, our expectations and the timing of the Phase 1b dose expansion phase of brimarafenib, our expectations regarding the timing of enrollment in our combination therapy oncology programs, expectations about whether our patents for our lead assets will adequately protect SpringWorks against competition, as well as relating to other future conditions. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “would,” “should” and “could,” and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success of our commercialization efforts with respect to OGSIVEO, (ii) our limited experience as a commercial company, (iii) our ability to obtain or maintain adequate coverage and reimbursement for OGSIVEO, (iv) the success and timing of our product development activities, including the initiation and completion of our clinical trials, (v) our expectations regarding the potential clinical benefit of OGSIVEO for adult patients with desmoid tumors who require systemic treatment, (vi) the potential for OGSIVEO to become the new standard of care for adult patients with desmoid tumors, (vii) estimates regarding the number of adult patients who are diagnosed with desmoid tumors annually per year in the U.S. and the potential market for OGSIVEO, (viii) the fact that topline or interim data from clinical studies may not be predictive of the final or more detailed results of such study or the results of other ongoing or future studies, (ix) the success and timing of our collaboration partners’ ongoing and planned clinical trials, (x) the timing of our planned regulatory submissions and interactions, including the timing and outcome of decisions made by the FDA, EMA, and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, (xi) whether FDA, EMA, or other regulatory authorities will require additional information or further studies, or may fail or refuse to approve or may delay approval of our product candidates, including nirogacestat and mirdametinib, (xii) our ability to obtain regulatory approval of any of our product candidates or maintain regulatory approvals granted for our products, (xiii) our plans to research, discover and develop additional product candidates, (xiv) our ability to enter into collaborations for the development of new product candidates and our ability to realize the benefits expected from such collaborations, (xv) our ability to maintain adequate patent protection and successfully enforce patent claims against third parties, (xvi) the adequacy of our cash position to fund our operations through any time period indicated herein, (xvii) our ability to establish manufacturing capabilities, and our and our collaboration partners’ abilities to manufacture our product candidates and scale production, and (xviii) our ability to meet any specific milestones set forth herein. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks’ expectations and actual results, you should review the “Risk Factors” in Item 1A of Part II of SpringWorks’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks’ subsequent filings. SpringWorks Therapeutics, Inc.Condensed Consolidated Statements of Operations(Unaudited) ​Three Months Ended September 30, Nine Months Ended September 30,(in thousands, except share and per-share data) 2024 2023 2024 2023 Revenue: Product revenue, net$49,301 $— $110,493 $— Other revenue (1) — — 19,547 — Total revenue 49,301 — 130,040 — Operating costs and expenses: Cost of product revenue 3,341 — 6,996 — Selling, general and administrative 61,601 46,546 179,553 137,715 Research and development 42,296 37,453 140,280 106,835 Total operating costs and expenses 107,238 83,999 326,829 244,550 ​ Loss from operations (57,937) (83,999) (196,789) (244,550)Interest and other income: Interest and other income, net 6,216 5,586 20,565 16,971 Total interest and other income 6,216 5,586 20,565 16,971 Equity method investment loss (1,809) (1,024) (4,610) (3,203)Net loss$(53,530) $(79,437) $(180,834) $(230,782)​ Net loss per share, basic and diluted$(0.72) $(1.27) $(2.44) $(3.70)Weighted average common shares outstanding, basic and diluted 74,264,501 62,521,772 74,052,151 62,386,496 (1) Related to recognition of all previously deferred revenue associated with the GSK License Agreement following notice of termination of the agreement received from GSK, as announced in June 2024. SpringWorks Therapeutics, Inc.Selected Balance Sheet Data(Unaudited) September 30, 2024 December 31, 2023(in thousands) Cash, cash equivalents and marketable securities$498,123 $662,588 Working Capital (1) 364,498 422,742 Total assets 608,878 725,788 Total liabilities 76,297 99,569 Accumulated deficit (1,075,868) (895,034)Total stockholders’ equity 532,581 626,219 (1) We define Working Capital as current assets less current liabilities.Contacts: Investorsinvestors@springworkstx.com Mediamedia@springworkstx.com