On November 2, 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 third quarter financial results for the period ended September 30, 2023 and provided an update on recent business highlights (Press release, SpringWorks Therapeutics, NOV 2, 2023, View Source [SID1234636795]).

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"We continue to work closely with the FDA as they complete their review of our nirogacestat NDA and are ready to serve the desmoid tumor community following an approval," said Saqib Islam, Chief Executive Officer of SpringWorks. "We are also on track to report topline data from our Phase 2b ReNeu trial of mirdametinib in patients with NF1-PN, which, if positive, would position us to file an NDA in the first half of 2024. Together, these anticipated milestones position us to have two medicines approved to treat patients with two distinct devastating diseases. In addition to these near-term events, we continue to advance our broader targeted oncology pipeline, which includes rare oncology, BCMA combinations in multiple myeloma, and biomarker-defined metastatic solid tumor programs, and look forward to providing further updates on our progress over the coming months."

Recent Business Highlights and Upcoming Milestones

Rare Oncology

The Prescription Drug User Fee Act (PDUFA) target action date for the New Drug Application (NDA) for nirogacestat for the treatment of adults with desmoid tumors is November 27, 2023. SpringWorks expects to file a Marketing Authorisation Application for nirogacestat with the European Medicines Agency (EMA) in the first half of 2024.
In November 2023, additional data from the Phase 3 DeFi trial evaluating the impact of nirogacestat on functional status in patients with desmoid tumors were presented at the 2023 Connective Tissue Oncology Society (CTOS) Annual Meeting. Statistically significant and clinically meaningful improvements in physical and role functioning were observed in patients receiving nirogacestat compared with those who received placebo by Cycle 10 across the three prespecified assessment tools evaluated. Patients receiving nirogacestat were five times more likely to have a clinically meaningful improvement in physical functioning and two times more likely to have a clinically meaningful improvement in role functioning compared to placebo at Cycle 10. These improvements began as early as Cycle 2 (the first post-treatment timepoint) and were sustained through Cycle 24 (the final assessment). Improvements in functioning were consistent with the improvements in pain measures, disease-related symptoms, and overall health-related quality of life that were previously reported with nirogacestat.
The ongoing Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors is fully enrolled. SpringWorks expects to report initial data from the trial in 2024.
SpringWorks expects to present topline data from the pediatric and adult cohorts of the Phase 2b ReNeu trial evaluating mirdametinib, an investigational MEK inhibitor, in NF1-associated plexiform neurofibromas (NF1-PN) in the fourth quarter of 2023. If these data are positive, SpringWorks plans to submit an NDA to the FDA for mirdametinib for the treatment of NF1-PN in the first half of 2024.

B-cell Maturation Antigen (BCMA) Combinations in Multiple Myeloma

SpringWorks continues to evaluate nirogacestat as part of several BCMA combination therapy regimens across treatment lines in collaboration with industry leaders.
In September 2023, the first patient was dosed in the Regeneron-sponsored Phase 1b study arm evaluating nirogacestat in combination with Regeneron’s linvoseltamab, a bispecific antibody targeting BCMA and CD3.

Biomarker-Defined Metastatic Solid Tumors

The dose expansion portion of the Phase 1b trial evaluating brimarafenib (BGB-3245) is ongoing in adult patients with RAF mutant solid tumors. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.
Patients continue to be enrolled in the dose escalation phase of the SpringWorks-sponsored Phase 1/2a combination study of brimarafenib and mirdametinib.
In August 2023, MapKure filed an Investigational New Drug Application (IND) for a combination study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations and expects to initiate a Phase 1/2a study in the first quarter of 2024. Amgen Inc. is supplying panitumumab pursuant to a clinical trial collaboration agreement with MapKure.
Dose expansion cohort is ongoing in the BeiGene-sponsored Phase 1b/2 trial evaluating mirdametinib in combination with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with NRAS mutant solid tumors.
In September 2023, preclinical data were published in Molecular Cancer Therapeutics characterizing SWTX-143, a TEAD inhibitor tool compound, and describing the potential of TEAD inhibitors to treat multiple Hippo-mutant solid tumor types. SpringWorks plans to file an IND for SW-682, the Company’s TEAD inhibitor development candidate, in the fourth quarter of 2023.

General Corporate

In September 2023, SpringWorks appointed Tai-An Lin, Ph.D., as Chief Scientific Officer. Dr. Lin brings more than 25 years of biotechnology and global pharmaceutical experience in advancing drug discovery programs from target identification through early clinical trials across the therapeutic areas of oncology, immuno-oncology, and immunology.

Third Quarter 2023 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $37.5 million for the third quarter, compared to $36.1 million for the comparable period of 2022. The increase in R&D expenses 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, partially offset by a decrease in external costs related to drug manufacturing, clinical trials and other research.
General and Administrative (G&A) Expenses: G&A expenses were $46.5 million for the third quarter, compared to $35.7 million for the comparable period of 2022. The increase in G&A expenses was largely attributable to commercial readiness activities to support the U.S. launch of nirogacestat, if approved, for the treatment of adults with desmoid tumors.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $79.4 million, or $1.27 per share, for the third quarter of 2023. This compares to a net loss of $72.4 million, or $1.37 per share, for the comparable period of 2022.
Cash Position: Cash, cash equivalents and marketable securities were $422.4 million as of September 30, 2023.