Pipeline

We measure ourselves by the impact we have on people with cancer.
Let’s go big.

Preclinical Phase 1 Phase 2 Phase 3 Regulatory Submission Approved Collaborators

Nirogacestat (Gamma Secretase Inhibitor)

Monotherapy

Adult desmoid tumors

Approved in the U.S.

Approved in the U.S. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.

Monotherapy

Pediatric desmoid tumors

In collaboration with Children’s Oncology Group (COG), a Phase 2 clinical trial is evaluating nirogacestat in children and adolescents with progressive, surgically unresectable desmoid tumors.

Monotherapy

Ovarian Granulosa Cell Tumors

Ovarian granulosa cell tumors are a rare ovarian cancer subtype for which there are currently no FDA-approved therapies. SpringWorks is conducting a Phase 2 study of nirogacestat in recurrent ovarian granulosa cell tumors.

+ BCMA-Targeting Agents

Relapsed/refractory multiple myeloma

The word "Several" with footnote indicator reading "1"

SpringWorks 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 leaders.

Mirdametinib (MEK Inhibitor)

Monotherapy

Adult and pediatric NF1-associated plexiform neurofibromas

Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) is a rare tumor of the peripheral nerve sheath that causes significant pain and disfigurement. A New Drug Application for mirdametinib, an investigational MEK inhibitor, for the treatment of adult and pediatric patients with NF1-PN is under review by the FDA. Mirdametinib has also received Orphan Drug, Fast Track and Rare Pediatric Disease designations for this indication.

Monotherapy

Pediatric low-grade gliomas

In collaboration with St. Jude Children’s Research Hospital, a Phase 2 clinical trial is evaluating mirdametinib in children, adolescents, and young adults with low-grade glioma.

+ Lifirafenib (RAF dimer inhibitor)

RAS/RAF mutant and other MAPK pathway aberrant solid tumors

Under a collaboration agreement with BeiGene, a Phase 1b/2 clinical trial is evaluating mirdametinib in combination with lifirafenib, BeiGene’s RAF dimer inhibitor, in patients with advanced or refractory solid tumors harboring RAS mutations, RAF mutations, and other MAPK pathway aberrations.

Brimarafenib (BGB-3245) (RAF Fusion and Dimer Inhibitor)

Monotherapy

MAPK mutant solid tumors

Brimarafenib is a novel oral, selective small molecule inhibitor of monomer and dimer forms of activating B-RAF mutations including V600 B-RAF mutations, non-V600 B-RAF mutations and RAF fusions. A Phase 1 clinical trial is evaluating brimarafenib in patients with advanced or refractory solid tumors including those with B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor. Brimarafenib is being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene.

+ Mirdametinib

MAPK mutant solid tumors

A Phase 1/2a combination study is evaluating brimarafenib, a selective RAF dimer inhibitor, in combination with mirdametinib, a MEK inhibitor, in patients with MAPK mutant solid tumors. Brimarafenib is being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene.

+ Panitumumab

MAPK mutant solid tumors

MapKure will conduct a Phase 1b study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations.

SW-682 (TEAD Inhibitor)

Monotherapy

Hippo mutant tumors

SpringWorks is developing SW-682 , a novel small molecule inhibitor of the TEA Domain (TEAD) family of transcription factors, for the potential treatment of biomarker-defined solid tumors driven by Hippo pathway mutations.

Nirogacestat (Gamma Secretase Inhibitor)

Monotherapy

Adult desmoid tumors

Regulatory Approval

Approved in the U.S. for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.

Monotherapy

Pediatric desmoid tumors

Phase 2 In Progress:

Collaborators:

In collaboration with Children’s Oncology Group (COG), a Phase 2 clinical trial is evaluating nirogacestat in children and adolescents with progressive, surgically unresectable desmoid tumors.

Monotherapy

Ovarian Granulosa Cell Tumors

Phase 2 In Progress:

Ovarian granulosa cell tumors are a rare ovarian cancer subtype for which there are currently no FDA-approved therapies. SpringWorks is conducting a Phase 2 study of nirogacestat in recurrent ovarian granulosa cell tumors.

+ BCMA-Targeting Agents

Relapsed/refractory multiple myeloma

Phase 2 In Progress:

Collaborators:

The word "Several" with footnote indicator reading "1"

SpringWorks 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 leaders.

Mirdametinib (MEK Inhibitor)

Monotherapy

Adult and pediatric NF1-associated plexiform neurofibromas

Phase 3 Complete:

Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) is a rare tumor of the peripheral nerve sheath that causes significant pain and disfigurement. A New Drug Application for mirdametinib, an investigational MEK inhibitor, for the treatment of adult and pediatric patients with NF1-PN is under review by the FDA. Mirdametinib has also received Orphan Drug, Fast Track and Rare Pediatric Disease designations for this indication.

Monotherapy

Pediatric low-grade gliomas

Phase 2 In Progress:

Collaborators:

In collaboration with St. Jude Children’s Research Hospital, a Phase 2 clinical trial is evaluating mirdametinib in children, adolescents, and young adults with low-grade glioma.

+ Lifirafenib (RAF dimer inhibitor)

RAS/RAF mutant and other MAPK pathway aberrant solid tumors

Phase 1 In Progress:

Collaborators:

Under a collaboration agreement with BeiGene, a Phase 1b/2 clinical trial is evaluating mirdametinib in combination with lifirafenib, BeiGene’s RAF dimer inhibitor, in patients with advanced or refractory solid tumors harboring RAS mutations, RAF mutations, and other MAPK pathway aberrations.

Brimarafenib (BGB-3245) (RAF Fusion and Dimer Inhibitor)

Monotherapy

MAPK mutant solid tumors

Phase 1 In Progress:

Collaborators:

Brimarafenib is a novel oral, selective small molecule inhibitor of monomer and dimer forms of activating B-RAF mutations including V600 B-RAF mutations, non-V600 B-RAF mutations and RAF fusions. A Phase 1 clinical trial is evaluating brimarafenib in patients with advanced or refractory solid tumors including those with B-RAF driver mutations and fusions that are likely to respond to a RAF dimer inhibitor. Brimarafenib is being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene.

+ Mirdametinib

MAPK mutant solid tumors

Phase 1 In Progress:

Collaborators:

A Phase 1/2a combination study is evaluating brimarafenib, a selective RAF dimer inhibitor, in combination with mirdametinib, a MEK inhibitor, in patients with MAPK mutant solid tumors. Brimarafenib is being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene.

+ Panitumumab

MAPK mutant solid tumors

Phase 1 In Progress:

Collaborators:

MapKure will conduct a Phase 1b study of brimarafenib with panitumumab, a monoclonal antibody targeting EGFR, in colorectal and pancreatic cancer patients with known MAPK pathway mutations.

SW-682 (TEAD Inhibitor)

Monotherapy

Hippo mutant tumors

Phase 1 In Progress:

SpringWorks is developing SW-682 , a novel small molecule inhibitor of the TEA Domain (TEAD) family of transcription factors, for the potential treatment of biomarker-defined solid tumors driven by Hippo pathway mutations.

1 Includes preclinical, Phase 1, and Phase 2 studies planned or ongoing with GSK, Janssen, Pfizer, Regeneron, and AbbVie. On June 6, 2024, GSK notified SpringWorks that it is terminating the collaboration agreement and winding down the study; no further enrollment is expected, though the study will continue for the patients enrolled at the time of termination notice.

Our pipeline

SpringWorks Therapeutics is advancing a diversified pipeline of targeted oncology programs as both standalone and combination therapies. Our development programs include late-stage rare oncology programs as well as multiple programs addressing highly prevalent, genetically defined cancers. Our team is working with urgency on behalf of the patients living with these devastating illnesses.

Let’s unlock the future of targeted oncology

We are meticulous about the science and passionate about its possibilities. This leads us to dig deeper – to explore the full potential of our molecules. You can see that approach in our pipeline. We have a diversified targeted oncology portfolio that includes standalone and combination therapies across various stages of development, including late-stage clinical trials in rare tumor types and multiple programs addressing highly prevalent, genetically defined cancers.