SpringWorks Therapeutics

What SpringWorks Therapeutics does

SpringWorks Therapeutics is a commercial-stage biopharmaceutical company founded in 2017 as a Pfizer spin-off, focused exclusively on developing targeted small-molecule therapies for underserved rare oncology indications. The company's technology centers on proprietary small-molecule platforms targeting specific oncogenic mechanisms, including gamma secretase inhibition (nirogacestat), MEK inhibition (mirdametinib), TEAD inhibition (SW-682), and PP2A activation (SW-3431 / RPT04402). Its two FDA-approved products are OGSIVEO (nirogacestat), approved in March 2023 as the first FDA-approved therapy for adult patients with progressing desmoid tumors, and GOMEKLI (mirdametinib), approved in February 2024 for adult and pediatric patients 2 years and older with neurofibromatosis type 1-associated symptomatic plexiform neurofibromas.

The company's business model is commercial pharmaceutical product sales through specialty pharmacy distribution and healthcare provider prescriptions, supported by a dedicated patient assistance program (CareConnections) and patient advocacy partnerships with organizations including the Desmoid Tumor Research Foundation, Children's Tumor Foundation, and NF Network. Customer segments are vertically defined by rare disease indication — primarily desmoid tumor patients (estimated 1,000–1,650 new U.S. cases annually) and NF1-PN patients (a subset of the NF1 population where 30–50% develop plexiform neurofibromas), with secondary exploration into multiple myeloma BCMA combinations, ovarian granulosa cell tumors, and pediatric low-grade glioma.

In April 2025, Merck KGaA acquired SpringWorks for approximately $3.4 billion, converting the previously NASDAQ-listed (SWTX) company into a wholly-owned healthcare subsidiary of Merck KGaA, Darmstadt, Germany. Post-acquisition, Merck KGaA discontinued several pipeline assets (SW-682, M9466, and the ovarian GCT exploration) while extending the approved medicines into the EU under the OGSIVEO and EZMEKLY brands during 2025.

Name

SpringWorks Therapeutics

Legal name

SpringWorks Therapeutics, Inc.

Website

https://www.springworkstx.com

Company type

Private

Founded year

2017

Operating status

Acquired

Headcount range

251–500 employees

Short description

SpringWorks Therapeutics is a commercial-stage biopharmaceutical company developing FDA-approved targeted small-molecule therapies OGSIVEO and GOMEKLI for rare oncology indications including desmoid tumors and NF1-associated plexiform neurofibromas, now operating as a wholly-owned subsidiary of Merck KGaA following its April 2025 $3.4 billion acquisition.

Ownership category

akta.pro rank

Product category

Rare Oncology Therapeutics

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Medicinal Chemicals & Botanical Products (2833)

akta.pro industry

PRIMARYHLAIAIAC

Rare Oncology & Hematologic Malignancy Therapies

Healthcare & Life Sciences/Pharmaceuticals/Orphan & Rare Disease Pharmaceuticals

SECONDARYHLAIAIAD

Rare Neurology & Neurodegenerative Disorder Therapies

Healthcare & Life Sciences/Pharmaceuticals/Orphan & Rare Disease Pharmaceuticals

SECONDARYHLAIAIAN

Rare Pediatric & Congenital Disorder Therapies

Healthcare & Life Sciences/Pharmaceuticals/Orphan & Rare Disease Pharmaceuticals

Keywords

• Rare oncology therapeutics
• Targeted cancer therapies
• Small molecule inhibitors
• Biopharmaceutical medicines
• Orphan oncology drugs

Headquarters

HQ city

Stamford

HQ country

United States

HQ region

North America

Offices4 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Marketing or Sales, Supply Chain, Operations, Infrastructure

Revenue model

1. Pharmaceutical Product Sales: Revenue generated from sales of FDA-approved drugs OGSIVEO (nirogacestat) and GOMEKLI (mirdametinib) for rare oncology indications. Commercial-stage biopharmaceutical company with approved products in the U.S. and E.U.

Pricing tiers

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

SpringWorks Therapeutics develops and commercializes FDA-approved targeted small molecule oncology medicines for patients with severe rare diseases and rare cancers. Its marketed products include OGSIVEO (nirogacestat), an oral gamma secretase inhibitor approved for adult patients with progressing desmoid tumors requiring systemic treatment, and GOMEKLI (mirdametinib), an oral MEK inhibitor approved for adult and pediatric patients aged 2 years and older with neurofibromatosis type 1-associated symptomatic plexiform neurofibromas not amenable to complete resection.

Differentiator

Problem solved

Functional benefit

Brands

• OGSIVEO: Brand name for nirogacestat, an oral gamma secretase inhibitor approved in the U.S. and E.U. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.
• GOMEKLI
• EZMEKLY

Products and services

• OGSIVEO (nirogacestat) Oral, selective, small molecule gamma secretase inhibitor (GSI) approved in the U.S. and E.U. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.
• OGSIVEO (nirogacestat) - European Union Approval European Union approval of oral, selective, small molecule gamma secretase inhibitor (GSI) for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.
• GOMEKLI (mirdametinib) Oral, small molecule MEK inhibitor approved in the U.S. for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
• EZMEKLY (mirdametinib) - European Union Conditional Approval European Union brand of oral, small molecule MEK inhibitor conditionally approved by the European Commission for the treatment of symptomatic, inoperable PN in adult and pediatric patients with NF1 aged 2 years and above.

Quantifiable outcome

• OGSIVEO received FDA approval for desmoid tumors based on Phase 3 DeFi trial data published in New England Journal of Medicine
• +3 more outcomes

Segments5 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Seven partnerships are on record, tiered core.

• Rappta TherapeuticscoreStrategic or Co-development Partner · 13 January 2025Finland's Rappta Therapeutics entered into a global license agreement with SpringWorks for RPT04402, a first-in-class molecular glue targeting the PP2A enzyme for cancer treatment. SpringWorks paid $13 million upfront and will oversee global development and commercialization, including filing an Investigational New Drug (IND) application by end of 2025. Rappta remains eligible for clinical, regulatory, and commercial milestone payments plus tiered royalties.
• Children's Tumor Foundation (CTF)coreStrategic or Co-development PartnerCTF, led by CEO Annette Bakker, worked to revive shelved rare disease drug candidates by partnering with pharmaceutical companies. CTF successfully brokered a deal with Pfizer to license a shelved drug to spin-off SpringWorks Therapeutics, which received FDA approval and was subsequently acquired by Merck KGaA for $3.4 billion.
• Children's Oncology Group (COG)coreStrategic or Co-development PartnerPhase 2 clinical trial collaboration evaluating nirogacestat as monotherapy in children and adolescents with progressive, surgically unresectable desmoid tumors. The clinical trial is fully enrolled.
• St. Jude Children's Research HospitalcoreStrategic or Co-development PartnerPhase 2 clinical trial collaboration evaluating mirdametinib in children, adolescents, and young adults with low-grade glioma.
• BCMA Therapy Developers (GSK, Janssen, Pfizer, Regeneron)coreStrategic or Co-development PartnerSpringWorks is evaluating nirogacestat as a potential treatment for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with industry-leading BCMA therapy developers. Note: GSK notified SpringWorks in June 2024 that it is terminating its collaboration agreement and winding down the study.
• PfizercoreStrategic or Co-development PartnerPfizer originally licensed a shelved experimental drug for rare genetic disorder to SpringWorks Therapeutics. SpringWorks developed it into Gomekli (mirdametinib), receiving FDA and EMA approval. This drug became the foundation for Merck KGaA's $3.4 billion acquisition of SpringWorks.
• Merck KGaAcoreOthersMerck KGaA acquired SpringWorks Therapeutics for $3.4 billion in April 2025, representing its largest acquisition in a decade. SpringWorks now operates as a healthcare company of Merck KGaA, Darmstadt, Germany.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Blueprint Medicines: Blueprint Medicines develops targeted small molecule therapies for genomically defined rare cancers and hematologic disorders, including FDA-approved Ayvakit and Gavreto. Its focus on biomarker-defined oncology populations closely parallels SpringWorks' strategy of addressing small, genetically defined rare tumor populations with proprietary small molecules.
• Deciphera Pharmaceuticals: Deciphera developed Qinlock, an FDA-approved kinase inhibitor for rare gastrointestinal stromal tumors, before being acquired by Otsuka. It is one of the closest direct comparables to SpringWorks in terms of business model: a focused rare-oncology company commercializing a targeted small molecule for an ultra-rare indication.
• Rigel Pharmaceuticals: Rigel commercializes small molecule drugs for rare hematology and oncology indications, including Tavalisse and Rezlidhia. Its model of selling rare-disease specialty drugs through targeted field sales and specialty pharmacy channels is structurally similar to SpringWorks' commercial approach with OGSIVEO and GOMEKLI.
• Karyopharm Therapeutics: Karyopharm developed Xpovio, a first-in-class selective inhibitor of nuclear export (SINE) compound approved for multiple myeloma and diffuse large B-cell lymphoma. As a small-molecule rare oncology company with an approved drug and ongoing label-expansion work, it offers a comparable commercial and pipeline profile to SpringWorks.
• Forma Therapeutics (now Novo Nordisk): Forma Therapeutics focused on rare hematology and hematologic malignancies with small molecule therapies (e.g., Rezlidhia for AML) before being acquired by Novo Nordisk. Its trajectory of FDA approvals in rare hematologic indications and subsequent strategic exit mirrors the SpringWorks playbook closely.
• Epizyme (an Ipsen company): Epizyme developed Tazverik, an EZH2 inhibitor approved for epithelioid sarcoma and follicular lymphoma, before being acquired by Ipsen. Its profile as a small-molecule rare oncology company with an approved drug for ultra-rare cancers is highly analogous to SpringWorks.

Emerging players

• Cogent Biosciences: Cogent Biosciences is developing targeted kinase inhibitors for rare diseases, including bezuclastinib for systemic mastocytosis and GIST. It is an emerging peer pursuing a similar rare-oncology small-molecule strategy, though at an earlier commercial stage than SpringWorks.
• Revolution Medicines: Revolution Medicines develops targeted inhibitors of RAS oncogene mutations and other oncology drivers with a focus on biomarker-defined solid tumors. Its emphasis on novel mechanism targeted oncology for defined populations overlaps with SpringWorks' approach to molecularly defined rare tumors.

Broad incumbents

• Pfizer: Pfizer is the originator of the mirdametinib asset licensed to SpringWorks and a prior equity holder. As a large oncology incumbent with rare disease programs of its own, Pfizer provides context on the broader competitive and licensing dynamics around the shelved-drug revival model SpringWorks epitomizes.
• Merck KGaA: Merck KGaA is the parent company of SpringWorks following the April 2025 acquisition. Its Healthcare segment, including the established oncology and neurology franchises, defines the strategic context and resource pool within which SpringWorks now operates.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/springworkstx
• YouTubeyoutube.com/@SpringWorksTx
• Facebookfacebook.com/SpringWorksTherapeutics
• LinkedInlinkedin.com/company/springworks-therapeutics
• Instagraminstagram.com/springworkstx

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds5 records

Investors18 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about SpringWorks Therapeutics