Getting medicines to those who need them most
We recognize that the patients and families affected by life-threatening diseases anxiously await more effective medicines with each passing day. Knowing this drives us to work harder to deliver transformative treatments more swiftly.
We also seek to ensure any medicine we develop is safe and effective and follows the rigorous guidelines established by regulatory agencies.
We strive to balance these two complementary and vital goals in all that we do.
Our clinical trials
Systemic mastocytosis (SM)
A clinical trial for people with advanced systemic mastocytosis
Trial Phase
Target Population
Aggressive SM, SM with associated hematologic neoplasm, and mast cell leukemia
Study Status
Active, fully enrolled
ClinicalTrials.gov
Study Number
NCT03580655
A clinical trial for people with indolent systemic mastocytosis
Trial Phase
Target Population
Indolent SM
Study Status
Active, fully enrolled
ClinicalTrials.gov
Study Number
NCT03731260
A clinical trial for people with indolent systemic mastocytosis
Trial Phase
Target Population
Indolent SM
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT04910685
Learn more: clinicaltrials.gov/ct2/show/NCT04910685
RET-altered cancers
A clinical trial for people with RET-altered cancers
Trial Phase
Target Population
RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) or other solid tumors with a RET fusion or mutation
Study Status
Active, fully enrolled
ClinicalTrials.gov
Study Number
NCT03037385
A clinical trial for people with RET fusion-positive, metastatic non-small cell lung cancer (NSCLC)
Trial Phase
Target Population
First-line RET fusion-positive, metastatic NSCLC
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT04222972
A clinical trial for people with RET-mutated medullary thyroid cancer (MTC)
Trial Phase
Target Population
Patients with locally advanced or metastatic RET-mutated MTC who have not previously received a standard of care multi-kinase inhibitor therapy
Study Status
Not yet recruiting
ClinicalTrials.gov
Study Number
NCT04760288
Learn more: https://clinicaltrials.gov/ct2/show/NCT04760288
EGFR-mutated non-small cell lung cancer (NSCLC)
A clinical trial for people with EGFR-mutated non-small cell lung cancer
Trial Phase
Target Population
Treatment-resistant EGFR-mutated NSCLC
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT04862780
Learn more: https://clinicaltrials.gov/ct2/show/NCT04862780
A clinical trial for people with non-small cell lung cancer harboring EGFR exon 20 insertion mutations
Trial Phase
Target Population
NSCLC and other cancers with EGFR exon 20 insertion mutations
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT05241873
Learn more: https://clinicaltrials.gov/ct2/show/NCT05241873
CDK2-vulnerable cancers and estrogen receptor-positive (ER+), HER2-negative (HER2-) breast cancer
A clinical trial for people with cyclin E aberrant cancers and estrogen receptor-positive, HER2-negative breast cancer
Trial Phase
Target Population
Cyclin E aberrant ovarian cancer, endometrial cancer, gastric cancer and other solid tumors, and ER+, HER2- breast cancer
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT05252416
Learn more: https://clinicaltrials.gov/ct2/show/NCT05252416
Pediatric cancers
A clinical trial for children and adolescents with solid tumors dependent on KIT or PDGFRA signaling
Trial Phase
Target Population
Solid tumors with a KIT or PDGFRA mutation and gliomas with an H3K27M mutation in pediatric patients
Study Status
Active, recruiting
ClinicalTrials.gov
Study Number
NCT04773782
Learn more: https://clinicaltrials.gov/ct2/show/NCT04773782