On February 28, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported financial results for the fourth quarter and full-year periods ended December 31, 2022 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, FEB 28, 2023, View Source [SID1234627877]).
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"2022 was a pivotal year for SpringWorks. We delivered highly positive data from our Phase 3 DeFi trial of nirogacestat in desmoid tumors, which served as the basis for our New Drug Application that was recently accepted by the FDA and granted Priority Review. We are excited to have the opportunity to change the treatment landscape for the desmoid tumor community following an anticipated approval later this year," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are equally encouraged by the potential for mirdametinib to be a best-in-class therapy for children and adults with NF1-PN. We look forward to reporting topline data from our Phase 2b ReNeu trial this year, which could support an NDA submission in the first half of 2024, and to advancing our emerging portfolio of BCMA combinations in multiple myeloma and biomarker-defined metastatic solid tumor programs. We believe that our continued strong execution, a financial position that we expect will fund us well into 2026, and durable intellectual property protections for our lead assets will support our long-term growth as we work towards our ambition of achieving two product launches by 2025."
Recent Business Highlights and Upcoming Milestones
Rare Oncology
In February 2023, the U.S. Food and Drug Administration (FDA) accepted SpringWorks’ New Drug Application (NDA) for nirogacestat, an investigational oral, small molecule gamma secretase inhibitor, in development as a monotherapy for the treatment of adults with desmoid tumors. The application has been granted Priority Review and given a Prescription Drug User Fee Act (PDUFA) action date of August 27, 2023. In addition, the FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application. SpringWorks also expects to file a Marketing Authorization Application for nirogacestat with the European Medicines Agency in 2024.
In the fourth quarter of 2022, the Phase 2 study sponsored by the Children’s Oncology Group evaluating nirogacestat in pediatric patients with desmoid tumors met its accrual goal.
In the fourth quarter of 2022, SpringWorks participated in a Type C meeting with the FDA to align on the statistical analysis plan for the Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in adult and pediatric patients with NF1-associated plexiform neurofibromas (NF1-PN) and expectations for an anticipated NDA submission in this indication. SpringWorks expects to present topline data from the pediatric and adult cohorts of the Phase 2b ReNeu trial of mirdametinib in NF1-PN in the second half of 2023. Pending these data, SpringWorks anticipates submitting an NDA for mirdametinib as a treatment for NF1-PN in the first half of 2024.
Patients continue to be enrolled in a Phase 2 trial evaluating nirogacestat in ovarian granulosa cell tumors.
B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma
SpringWorks continues to advance nirogacestat as a potential cornerstone of BCMA combination therapy across modalities in collaboration with industry leaders. In 2023, SpringWorks expects that additional clinical data of nirogacestat in combination with BCMA-directed therapies will be presented and that additional planned Phase 1 collaborator studies will be initiated.
Biomarker-Defined Metastatic Solid Tumors
The Phase 1 trial evaluating BGB-3245, a selective RAF dimer inhibitor, as a monotherapy in adult patients with RAF mutant solid tumors is advancing into cohort expansion studies. SpringWorks expects data from this trial to be presented at a medical conference in the first half of 2023. In February 2023, the first patient was dosed in a Phase 1/2a combination study of BGB-3245 and mirdametinib. BGB-3245 is being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene.
The BeiGene-sponsored Phase 1b/2 clinical trial evaluating mirdametinib with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with RAS/RAF mutant and other MAPK pathway aberrant solid tumors is ongoing. SpringWorks expects data from this trial to be presented at a medical conference in the first half of 2023.
In the fourth quarter of 2022, SpringWorks nominated TEAD inhibitor development candidate, SW-682. The Company expects to present additional preclinical data of SW-682 at a medical conference in the first half of 2023 and to file an Investigational New Drug Application for SW-682 in 2023.
General Corporate
SpringWorks has expanded and strengthened its intellectual property portfolio, including a new patent issued by the United States Patent and Trademark Office (USPTO) in November 2022 directed to pharmaceutical compositions of nirogacestat (U.S. Patent No. 11,504,354), which expires in 2042 and represents the seventh Orange Book-listable patent for nirogacestat. In addition, in February 2023, the USPTO granted a new patent directed to dispersible formulations of mirdametinib (U.S. Patent No. 11,571,402), which expires in 2041; this patent covers the formulation of mirdametinib that is being developed for pediatric patients with NF1-PN.
Fourth Quarter and Full Year 2022 Financial Results
Research and Development (R&D) Expenses: R&D expenses were $37.9 million and $146.1 million for the 2022 fourth quarter and full-year periods, respectively, compared to $29.3 million and $101.7 million for the comparable periods of 2021. The increase in R&D expense for the 2022 full-year period was primarily attributable to a $31.8 million increase in internal costs driven by the growth in employee costs associated with increases in the number of personnel, including an increase in stock-based compensation expense, and an increase of $22.6 million in external costs related to drug manufacturing, clinical trial and other research, with the full year increase partially offset by an $11.0 million decrease in licensing costs related to the nonrefundable upfront payment to Katholieke Universiteit Leuven and the Flanders Institute for Biotechnology for the in-licensing of the TEAD inhibitor program in May 2021.
General and Administrative (G&A) Expenses: G&A expenses were $40.5 million and $134.6 million for the 2022 fourth quarter and full-year periods, respectively, compared to $26.5 million and $71.8 million for the comparable periods of 2021. The increase in G&A expense was primarily attributable to an increase in internal costs driven by the growth in employee costs associated with increases in the number of personnel, including an increase in stock-based compensation expense as we continue to expand our operations to support the organization, and an increase in information technology costs and consulting and professional services, including legal, regulatory and compliance, as we continue to build new capabilities, including commercial.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $74.2 million, or $1.19 per share, for the fourth quarter of 2022 and a net loss of $277.4 million, or $5.21 loss per share, for the year ended December 31, 2022. This compares to a net loss of $56.1 million, or $1.15 per share, for the fourth quarter of 2021 and a net loss of $173.9 million, or $3.59 per share for the year ended December 31, 2021.
Cash Position: Cash, cash equivalents and marketable securities were $597.0 million as of December 31, 2022.