Development Pipeline

Target

Zelboraf™

(vemurafenib)
BRAF V600E
    

Zelboraf™ (vemurafenib) is the first small molecule drug approved for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved companion diagnostic test. Zelboraf™ is also indicated for the treatment of patients with Erdheim-Chester Disease with BRAF V600 mutation. In addition, Zelboraf™ forms the basis for several combination therapies, including with MEK inhibitors and checkpoint blockade. Zelboraf™ is the first drug designed using fragment/scaffold-based lead discovery to gain regulatory approval.  

Partnered with Roche. Further information is available at www.zelboraf.com.

TURALIO™

(pexidartinib)
CSF1R Inhibitor
    

TURALIO™ (pexidartinib) is an oral small molecule inhibitor of the colony stimulating factor-1 (CSF1) receptor (CSF1R), and first therapy approved in the United States for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. TGCT, a rare tumor of the joint or tendon sheath, overexpresses CSF1; inhibition of signaling between CSF1 and CSF1R targets the underlying cause of the disease. Progress in treating rare cancers such as TGCT was named by the American Society of Clinical Oncology (ASCO) as the 2019 Advance of the Year.

Partnered with Daiichi Sankyo. Further information is available at https://www.turalio.com.

PLX8394

BRAF
    

PLX8394 is a second generation BRAF inhibitor which inhibits ERK signaling by specifically disrupting BRAF-containing dimers. PLX8394 selectively inhibits ERK signaling in tumors driven by dimeric BRAF mutants, including BRAF fusions and splice variants, as well as BRAF V600E monomers, but spares RAF function in normal cells.

Partnered with Novellus.

PLX9486

KIT
    

PLX9486 is a highly selective Type I inhibitor of KIT receptor tyrosine kinase, targeting specifically oncogenic KIT with mutations in the activation loop encoded by exons 17 and 18, including KIT D816V, the driver mutation in nearly all patients with systemic mastocytosis. Combination with sunitinib, a Type-II inhibitor potent against KIT ATP-binding pocket mutations, was associated with prolonged progression-free survival in heavily pretreated patients with gastrointestinal stromal tumor.

Partnered with Cogent Biosciences.

PLX2853 is a small molecule inhibitor of the BET (bromodomain and extra terminal domain) proteins. PLX2853 shows a slight preference for bromodomain-2 (BD2) versus bromodomain-1 (BD1) and has a broad therapeutic window due to its differentiated PK profile.

https://clinicaltrials.gov/ct2/show/NCT03297424

PLX2853 is a small molecule inhibitor of the BET (bromodomain and extra terminal domain) proteins. PLX2853 shows a slight preference for bromodomain-2 (BD2) versus bromodomain-1 (BD1) and has a broad therapeutic window due to its differentiated PK profile.

https://clinicaltrials.gov/ct2/show/NCT03787498

PLX2853 is a small molecule inhibitor of the BET (bromodomain and extra terminal domain) proteins. PLX2853 shows a slight preference for bromodomain-2 (BD2) versus bromodomain-1 (BD1) and has a broad therapeutic window due to its differentiated PK profile.

https://clinicaltrials.gov/ct2/show/NCT04493619

PLX2853 is a small molecule inhibitor of the BET (bromodomain and extra terminal domain) proteins. PLX2853 shows a slight preference for bromodomain-2 (BD2) versus bromodomain-1 (BD1) and has a broad therapeutic window due to its differentiated PK profile.

https://clinicaltrials.gov/ct2/show/NCT04556617

Preclinical Pipeline

Target

Expanded Access to Investigational Products

Plexxikon strives to achieve our goal of developing personalized medicine through the process of sponsoring clinical trials and discovering new drug candidates.

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