Business Overview and History

SpringWorks Therapeutics, Inc. is a commercial-stage biopharmaceutical company that is pioneering the development and commercialization of life-changing medicines for patients suffering from devastating rare diseases and cancer. The company's strategic approach and operational excellence have enabled it to successfully launch two products, OGSIVEO and GOMEKLI, while also advancing a pipeline of promising clinical and preclinical candidates.

SpringWorks was formed in Delaware in August 2017 with the goal of applying a precision medicine approach to develop and commercialize innovative therapies for underserved patient populations. The company's origins can be traced back to the strong ties its founders had with Pfizer, from whom SpringWorks licensed the rights to develop and commercialize its lead product candidates, nirogacestat and mirdametinib.

In September 2019, SpringWorks completed its initial public offering, issuing and selling 10.35 million shares of its common stock at a public offering price of $18.00 per share, resulting in net proceeds of $169.7 million. This successful IPO provided the company with additional capital to fund its operations and advance its product candidates through clinical development.

In November 2023, SpringWorks received FDA approval for OGSIVEO (nirogacestat) for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. This milestone marked the company's successful transition into a commercial-stage entity and established OGSIVEO as the first and only FDA-approved therapy for this indication. To support the commercialization of OGSIVEO, SpringWorks built out its U.S. commercial sales team.

Just over a year later, in February 2025, SpringWorks again made history when the FDA approved GOMEKLI (mirdametinib) for the treatment of adult and pediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

The approval of OGSIVEO and GOMEKLI underscores SpringWorks' ability to effectively navigate the complex drug development and commercialization landscape. The company has demonstrated its prowess in executing successful clinical trials, securing regulatory approvals, and building the necessary commercial infrastructure to drive the uptake of its innovative therapies.

As of December 31, 2024, SpringWorks reported $461.9 million in cash, cash equivalents, and marketable securities, providing it with a strong financial foundation to fund its operations and continue expanding its pipeline. The company's robust balance sheet, coupled with its strategic partnerships and in-licensing agreements, positions SpringWorks well to capitalize on future growth opportunities.

Throughout its history, SpringWorks has faced some challenges. In 2022, the company terminated its expanded global, non-exclusive license and collaboration agreement with GlaxoSmithKline for the development of nirogacestat in combination with GSK compounds. This termination came after interim analysis results showed the objective response rate did not meet the pre-specified threshold for continued development. Despite this setback, SpringWorks remained focused on advancing its pipeline of product candidates and ultimately achieved success with the approval and commercialization of OGSIVEO and GOMEKLI.

Financial Performance and Liquidity

SpringWorks' financial performance has been marked by significant investments in research and development, as well as the successful commercialization of its approved products. For the full year 2024, the company reported $191.59 million in total revenue, with $172.0 million attributed to OGSIVEO net product revenue, demonstrating the strong commercial traction of this therapy.

The company's total operating expenses for 2024 were $469.72 million, with $200.52 million allocated to research and development and $256.65 million directed towards selling, general, and administrative activities. This spending reflects SpringWorks' commitment to advancing its pipeline and establishing a strong commercial presence for its approved products.

Despite the substantial investments, SpringWorks reported a net loss of $258.13 million for the full year 2024. The company's operating cash flow was negative $175.60 million, while free cash flow stood at negative $196.30 million. However, the company's strong cash position, with $461.9 million in cash, cash equivalents, and marketable securities as of the end of 2024, provides it with the financial flexibility to continue funding its operations and growth initiatives.

SpringWorks' liquidity position is further strengthened by its ability to generate positive cash flow from operations. For the fourth quarter of 2024, the company reported $61.55 million in revenue, primarily driven by OGSIVEO net product revenue, indicating the continued strong demand for this therapy. The company's debt-to-equity ratio is 0.016, while its current ratio and quick ratio stand at 3.81 and 3.70, respectively, demonstrating a solid financial position.

In terms of market performance, SpringWorks reported significant quarter-over-quarter growth for OGSIVEO, with Q3 2024 net product revenue of $49.3 million representing a 23% increase from the previous quarter. The company expressed confidence in the continued strong momentum of the OGSIVEO U.S. launch, driven by robust demand from both new patient starts and existing patients.

Looking ahead, SpringWorks expects to fund operations through profitability, which they are on track to achieve in the first half of 2026. This milestone will further enhance the company's long-term financial stability and growth prospects.

Product Portfolio and Pipeline

OGSIVEO (nirogacestat)

OGSIVEO is SpringWorks' first approved product, indicated for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. Desmoid tumors are rare, locally aggressive, and invasive soft tissue tumors that can cause severe morbidities, such as pain, disfigurement, and functional impairment.

The approval of OGSIVEO was based on the positive results from the pivotal Phase 3 DeFi trial, which demonstrated a statistically significant improvement in progression-free survival compared to placebo. The therapy has also shown consistent benefits in terms of objective response rate, symptom improvement, and quality of life for patients.

Since its launch in late 2023, OGSIVEO has rapidly become the standard of care systemic therapy for adults with desmoid tumors in the United States. In the fourth quarter of 2024, the company reported $61.5 million in OGSIVEO net product revenue, reflecting the strong and steady demand for this therapy.

SpringWorks has successfully introduced 150mg and 100mg strength tablets and blister packaging to increase patient convenience, adherence, and renewal times. Real-world data suggests that the addressable patient population for desmoid tumors is larger than initially estimated, further supporting the commercial potential of OGSIVEO. Additionally, the company has observed very high satisfaction from physicians, with expectations that the majority will increase OGSIVEO usage in the coming year.

Looking ahead, SpringWorks is focused on expanding the global reach of OGSIVEO. The company's marketing authorization application for OGSIVEO is currently under review by the European Medicines Agency, with potential approval and launch in Europe expected in 2025. Additionally, SpringWorks is working to initiate a bridging study for OGSIVEO in Japan in the coming year.

GOMEKLI (mirdametinib)

GOMEKLI is SpringWorks' second approved product, indicated for the treatment of adult and pediatric patients two years of age and older with NF1 who have symptomatic PN not amenable to complete resection. NF1 is a rare, autosomal dominant tumor predisposition disorder that can lead to the development of debilitating PN tumors.

The FDA approval of GOMEKLI was based on the positive data from the pivotal Phase 2b ReNeu trial, which demonstrated robust objective response rates, deep and durable reductions in tumor volume, and a manageable safety profile in both adult and pediatric patients. Importantly, GOMEKLI is the first and only therapy approved for the treatment of NF1-PN in both adults and children.

SpringWorks has assembled a 35-person U.S. commercial field organization to support the launch of GOMEKLI, which received FDA approval in February 2025. The company is also pursuing regulatory approvals for GOMEKLI in Europe, with the marketing authorization application currently under review by the European Medicines Agency.

Pipeline and Future Growth Opportunities

Beyond its two approved products, SpringWorks maintains a diverse pipeline of clinical and preclinical candidates targeting rare diseases and cancer. Some key pipeline programs include:

• Nirogacestat in ovarian granulosa cell tumors: SpringWorks is evaluating nirogacestat as a monotherapy in a Phase 2 trial for the treatment of this rare ovarian cancer subtype.

• Nirogacestat in combination with BCMA-directed therapies for multiple myeloma: SpringWorks has entered into several collaborations to evaluate nirogacestat in combination with various BCMA-directed therapies, leveraging its ability to potentiate the activity of these therapies.

• Mirdametinib in pediatric and young adult low-grade glioma: The company is supporting an ongoing Phase 2 study of mirdametinib in this patient population, with encouraging data presented at recent scientific conferences.

• SW-682, a TEAD inhibitor, in Hippo-mutant solid tumors: SpringWorks is advancing this first-in-class program, with a Phase 1a trial currently underway.

• SW-3431, a potential first-in-class protein phosphatase 2A (PP2A) activator, for aggressive uterine cancers: SpringWorks recently in-licensed this program and plans to file an investigational new drug application with the FDA by the end of 2025.

These pipeline programs, coupled with SpringWorks' strong commercial execution and financial position, position the company well for continued growth and the delivery of transformative therapies to patients in need.

Market Opportunity and Industry Trends

SpringWorks operates in a rapidly growing market for innovative therapies targeting rare diseases and oncology. The market for immunotherapy medications, which includes some of SpringWorks' product candidates, is expected to experience significant growth. Industry projections estimate that this market will expand from $240 billion in 2023 to $1.3 trillion by 2033, representing a substantial compound annual growth rate.

This robust market growth is driven by factors such as increasing prevalence of cancer and rare diseases, advancements in precision medicine, and growing demand for more effective and targeted therapies. SpringWorks' focus on developing innovative treatments for underserved patient populations positions the company well to capitalize on these industry trends.

Currently, SpringWorks sells its products exclusively in the United States. However, the company is actively pursuing regulatory approvals in other key markets, including Europe and Japan. These geographic expansions represent significant growth opportunities for SpringWorks and will allow the company to address the unmet medical needs of patients on a global scale.

Risks and Challenges

While SpringWorks has demonstrated impressive achievements, the company faces several risks and challenges that investors should consider:

• Reliance on Approved Products: The success of SpringWorks is currently heavily dependent on the commercial performance of OGSIVEO and GOMEKLI. Any setbacks or unexpected challenges in the commercialization of these products could significantly impact the company's financial results and growth trajectory.

• Competition and Pricing Pressures: SpringWorks operates in highly competitive therapeutic areas, and it may face challenges in maintaining market share and pricing power for its approved products over the long term.

• Regulatory Hurdles: The drug development and approval process is inherently risky, and SpringWorks may encounter delays or setbacks in advancing its pipeline candidates through clinical trials and regulatory reviews.

• Manufacturing and Supply Chain Disruptions: Disruptions in the company's manufacturing operations or supply chain could adversely affect its ability to produce and distribute its approved products and pipeline candidates.

• Dependence on Key Personnel: SpringWorks' success is heavily dependent on the continued contributions of its experienced management team and key scientific personnel. The loss of any of these individuals could have a significant impact on the company's operations and future prospects.

• Financial Risks: While SpringWorks has a strong cash position, the company continues to incur significant expenses as it advances its pipeline and commercializes its approved products. The company may require additional capital raises or partnerships to fully execute on its growth strategy.

Despite these risks, SpringWorks has demonstrated its ability to navigate the complexities of the biopharmaceutical industry and deliver innovative therapies to patients in need. The company's strong financial position, diversified pipeline, and proven track record of execution position it well for continued growth and success in the years to come.

Conclusion

SpringWorks Therapeutics has established itself as a leader in the rare disease and oncology treatment landscape, driven by the successful commercialization of OGSIVEO and GOMEKLI, as well as the advancement of a robust pipeline of promising product candidates. The company's strategic focus, operational excellence, and strong financial foundation provide a solid foundation for continued growth and the delivery of transformative therapies to patients suffering from devastating diseases.

With total revenue of $191.59 million in 2024 and a strong cash position of $461.9 million, SpringWorks is well-positioned to fund its operations and growth initiatives. The company's expectation to achieve profitability in the first half of 2026 further underscores its potential for long-term success.

As SpringWorks continues to execute on its ambitious goals, including expanding the global reach of its approved products and advancing its diverse pipeline, the company is well-positioned to create significant value for shareholders and make a profound impact on the lives of the patients it serves. The projected growth in the immunotherapy market, coupled with SpringWorks' innovative approach to drug development and commercialization, presents a compelling opportunity for investors seeking exposure to the rapidly evolving biopharmaceutical sector.