On January 8, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported that it will present at the 42nd Annual J.P. Morgan Healthcare Conference at 7:30 a.m. PT (10:30 a.m. ET), and a live webcast will be available at ir.springworkstx.com (Press release, SpringWorks Therapeutics, JAN 8, 2024, View Source [SID1234639111]). Ahead of the presentation, the Company highlighted its 2023 accomplishments and announced its anticipated key milestones for 2024.

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2023 Accomplishments

Nirogacestat

Received approval from the United States Food and Drug Administration (FDA) for OGSIVEO (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.
Published data from the Phase 3 DeFi trial of nirogacestat in adult patients with progressing desmoid tumors in the New England Journal of Medicine.
Presented additional data from the Phase 3 DeFi trial at several leading medical conferences. These presentations demonstrated rapid, sustained and consistent improvements in pain and functional status in patients receiving OGSIVEO using multiple assessment tools.
Completed enrollment in the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors (OvGCT).
Continued to evaluate nirogacestat in patients with multiple myeloma as part of several B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in collaboration with industry leaders. Clinical data from two BCMA combination studies were presented at the European Hematology Association (EHA) (Free EHA Whitepaper) congress and provided further validation of the mechanistic approach supporting nirogacestat’s ability to enhance the activity of BCMA-directed therapies across modalities. In addition, a Regeneron-sponsored study was initiated to evaluate nirogacestat in combination with linvoseltamab, Regeneron’s bispecific antibody targeting BCMA and CD3.
Mirdametinib (NF1-PN)

Presented positive topline data from the pivotal Phase 2b ReNeu trial evaluating mirdametinib, an investigational oral MEK inhibitor, in pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Data demonstrated a confirmed objective response rate of 52% in pediatric patients and 41% in adult patients, as assessed by Blinded Independent Central Review. Mirdametinib treatment resulted in deep and durable responses and led to significant improvements in key secondary patient-reported outcome measures. Mirdametinib was generally well tolerated in the ReNeu trial, with the majority of adverse events being Grade 1 or Grade 2.
Emerging Pipeline

Presented updated data from the Phase 1a/1b study of brimarafenib (BGB-3245), an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd., in adult patients with advanced or refractory solid tumors harboring MAPK pathway aberrations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. These data supported the advancement of brimarafenib into the Phase 1b cohort expansion portion of the study.
Shared updated clinical data from the Phase 1b trial evaluating mirdametinib in combination with BeiGene’s investigational RAF dimer inhibitor, lifirafenib, in patients with advanced or refractory solid tumors with RAS mutations, RAF mutations and other MAPK pathway aberrations at AACR (Free AACR Whitepaper). The combination showed antitumor activity in patients with various mutations across several solid tumor types and support the advancement of this combination into the dose-expansion portion of the study, which is evaluating the combination in patients with NRAS-mutated solid tumors.
Dosed the first patient in a Phase 1/2a combination study of brimarafenib and mirdametinib.
The FDA cleared the Investigational New Drug (IND) application submitted through MapKure for a combination study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations. Amgen Inc. is supplying panitumumab pursuant to a clinical trial collaboration agreement with MapKure.
Submitted an IND application for SW-682, a novel, potent, and selective TEAD inhibitor development candidate targeting tumors driven by Hippo pathway mutations.
General Corporate

Continued to expand and strengthen the intellectual property portfolios for nirogacestat and mirdametinib, with Orange Book listable patents providing protection past 2040 for both nirogacestat and mirdametinib.
Strengthened balance sheet with upsized public offering; gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $316.25 million. SpringWorks estimates that its cash, cash equivalents and marketable securities as of September 30, 2023 exceeded $700 million on a pro forma basis.
Anticipated 2024 Key Milestones

Nirogacestat

Continue to advance U.S. launch of OGSIVEO (nirogacestat) as the first and only approved therapy for adult patients with desmoid tumors and establish as standard of care.
Submit Marketing Authorisation Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors to the European Medicines Agency (EMA) in the first half of 2024.
Report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with OvGCT in the second half of 2024.
Expand data set with additional clinical data of nirogacestat in combination with BCMA-directed therapies.
Mirdametinib (NF1-PN)

Submit a New Drug Application (NDA) to the FDA for mirdametinib for the treatment of children and adults with NF1-PN in the first half of 2024.
Present data from the pediatric and adult cohorts of the Phase 2b ReNeu trial of mirdametinib in NF1-PN at a medical congress in the first half of 2024 and submit for publication in a peer-reviewed journal in 2024.
Emerging Pipeline

Present additional data for brimarafenib as a monotherapy in MAPK-mutant solid tumors in the second half of 2024.
Support initiation of Phase 1b trial of brimarafenib in combination with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations in the first quarter of 2024.
Initiate Phase 1 trial of SW-682, SpringWorks’ TEAD inhibitor, in Hippo mutant solid tumors in the first half of 2024.
"Our focus in 2024 is to deliver a successful U.S. launch of OGSIVEO as the first and only FDA-approved therapy for adults with desmoid tumors and to prepare for our second potential FDA approval by filing our NDA for mirdametinib as a treatment for patients with NF1-PN in the first half of the year," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are very pleased with our progress towards our goal of having two marketed products by 2025 given the highly positive topline data from our Phase 2b ReNeu trial and will simultaneously advance our broader pipeline of targeted oncology programs. Our achievements in 2023, our strong financial position and the durable IP protections for our lead assets position us for continued, long-term success as we execute on our mission to improve the lives of patients with devastating diseases."

Presentation at the 42nd Annual J.P. Morgan Healthcare Conference

SpringWorks will webcast its presentation from the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, 2024 at 7:30 a.m. PT (10:30 a.m. ET). To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.