Drugmaker with rare disease work · based in South San Francisco, United States · founded 1996 · ticker RIGL · rigel.com
Rigel Pharmaceuticals, Inc. works on 6 rare diseases we track, including Acute Myeloid Leukemia, Diffuse Intrinsic Pontine Glioma and 4 more. It has 7 active clinical trials, and all of them are looking for volunteers now. It also has 3 FDA-approved rare disease drugs.
Latest news. On August 4, 2026, Rigel said it expects to choose a Phase 2 dose of R289 for lower-risk MDS in the second half of 2026 and to share early dose-expansion data by the end of the year. Rigel second quarter 2026 results, August 4, 2026
Rigel Pharmaceuticals is a drug company founded in 1996 and based in South San Francisco, California, that works on blood disorders and cancer. It sells Tavalisse (fostamatinib) for chronic immune thrombocytopenia, Rezlidhia (olutasidenib) for relapsed or refractory AML with an IDH1mutation, Gavreto (pralsetinib) for RET fusion-positive lung and thyroid cancers, and, since August 2026, Veppanu (vepdegestrant) for a form of advanced breast cancer.
Rigel Pharmaceuticals, Inc. pipeline
Each row lists the diseases Rigel Pharmaceuticals, Inc. is studying at that stage, from the ones closest to approval down to the earliest work.
These are Rigel Pharmaceuticals, Inc. trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
7 trials looking for volunteers now
Looking for volunteersPhase 2Recently updatedNCT06566742
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
TestsOlutasidenib
For Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Clonal Cytopenia of Undetermined Significance
To learn if olutasidenib can help to control CCUS, MDS, and/or CMML. The safety of the drug will also be studied.
This is a prospective, single-arm phase 2 pilot study to assess the response rate of IDH1 mutated relapsed/refractoryacute myeloid leukemia (AML) patients who receive olutasidenib after progressing on venetoclax based regimens. Each cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission. Subjects with at least a PR after 6 cycles of treatment will continue treatment as previously described. Subjects without at least a partial response (PR) after 6 cycles of treatment will move to long term follow up.
The goal of this study is to determine the efficacy of the study drug olutasidenib to treat newly diagnosed pediatric and young adult patients with a high-grade glioma (HGG) harboring an IDH1mutation.
The main question the study aims to answer is whether the combination of olutasidenib and temozolomide (TMZ) can prolong the life of patients diagnosed with an IDH-mutant HGG.
A open-labeldrug-drug interaction (DDI) study to evaluate the effects of olutasidenib on the pharmacokinetics (PK) of a CYP450 and OATP1B1 probe substrate cocktail in participants with IDH1mutation-positive malignancies.
The purpose of this study is as follows:
1. Determine whether people receiving the combination treatment of olutasidenib, venetoclax, and azacitidine have the same, more, or fewer side effects compared to the usual chemotherapy treatment that people with this condition receive.
2. Determine how well the combination treatment of olutasidenib, venetoclax, and azacitidine works compared to the usual chemotherapy treatment that people with this condition receive.
This is an open-label phase I study of fostamatinib in combination with ruxolitinib for the treatment of chronic GvHD with a suboptimal response to corticosteroids. The primary objective is to identify a minimum safe and biologically effective dose of fostamatinib when combined with standard of careruxolitinib for the treatment of steroid refractory and steroid dependent cGVHD. The secondary objective is to estimate the efficacy of the combination of ruxolitinib and fostamatinib for the treatment of steroid refractory and steroid dependent cGVHD.
The target enrollment is 24-30 subjects. The study will begin with an initial dose escalationcohort employing a modified 3+3 design to investigate up to three doses of fostamatinib. Using safety, efficacy, pharmacodynamic (PD), and pharmacokinetic data (PK), an interim assessment will be performed to determine two candidate doses of the biologically optimal dose to investigate further. A safety expansion cohort will be opened to backfill these two candidate doses up to a total 12 patients per dose, including those in the dose escalationcohort who received the candidate doses. Patients will then be randomized to one of these two candidate doses in the expansion. If there is an imbalance in the two expansion cohorts, the remaining patient slots after 1:1 randomization will be sequentially backfilled to a total of 12 patients per cohort. A final analysis of safety, efficacy, and PK/PD data in patients who received the two candidate doses will be conducted to determine a minimum safety and biologically effective dose, which will be the recommended phase II dose (RP2D).
The primary hypothesis is that Fostamatinib combined with ruxolitinib is a safe therapy for and has synergistic activity in cGvHD. The recommended phase II dose will be determined by the study investigators in collaboration with the sponsors. The decision to select the recommended phase II dose will occur only after all patients in the part 1 have completed at least 28 days of therapy. The decision will be based on the valuation of all relevant, available data, and not solely on dose-limiting toxicities.
Follow a condition Rigel Pharmaceuticals, Inc. is studying
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Its own pipeline drug R289, which blocks the IRAK1 and IRAK4 enzymes, is in a Phase 1b trial in lower-risk myelodysplastic syndromes, and Rigel expects early results by the end of 2026.
Where Rigel Pharmaceuticals, Inc. trials take place
The trials above run at 42 study sites in 7 countries. Tap a country to see its states, cities and hospitals.
United States
33▼
Australia
3▼
Canada
2▼
Germany
1▼
Netherlands
1▼
New Zealand
1▼
United Kingdom
1▼
Rare diseases Rigel Pharmaceuticals, Inc. works on
Each disease page lists every open trial, not only Rigel Pharmaceuticals, Inc.'s, plus approved treatments.
Acute myeloid leukemia (AML) is a fast-growing cancer of the bone marrow in which immature white blood cells called blasts crowd out normal blood cells, causing fatigue, infections and bleeding...
How common: The National Cancer Institute's SEER program estimates 22,720 new US cases and 11,500 deaths in 2026, about 1.1% of new cancers, and 83,311 people were living with AML in 2023
Diffuse intrinsic pontine glioma (DIPG) is an aggressive childhood brain tumor that grows in the pons, the part of the brainstem controlling breathing, swallowing, and movement.
How common: About 300 children diagnosed in the U.S. each year, most often between ages 5 and 10
Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.
How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months
RET fusion cancers are a group of rare tumors driven by chromosomal rearrangements that fuse the RET gene with a partner gene, creating an abnormal protein that drives uncontrolled cell growth.
How common: RET fusions occur in 1-2% of non-small cell lung cancers (roughly 2,300-4,600 new cases per year in the U.S.), 7-12% of papillary thyroid cancers (higher in pediatric cases), and rarely in other tumor types
Warm autoimmunehemolytic anemia (wAIHA) is a rare autoimmune disease in which the immune system makes antibodies, usually of the IgG type, that stick to red blood cells at normal body temperature...
How common: About 1 to 3 new cases per 100,000 people each year, according to the FDA and NORD
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Rigel Pharmaceuticals, Inc.'s own site is rigel.com, and our help paying for treatment page lists drugmaker programs and charity funds.
3 shared diseases, including Acute Myeloid Leukemia and Myelodysplastic Syndromes
Questions about Rigel Pharmaceuticals, Inc.
Rigel sells 4 FDA-approved medicines, 3 of them for rare blood disorders or rare cancers.
Tavalisse (fostamatinib): a twice-daily pill for adults with chronicimmune thrombocytopenia whose platelets did not respond well enough to an earlier treatment, approved April 17, 2018. See the Tavalisse page.
Rezlidhia (olutasidenib): a twice-daily capsule for adults with relapsed or refractoryAML that carries an IDH1mutation, approved December 1, 2022. See the Rezlidhia page.
Gavreto (pralsetinib): a pill for adults with metastaticRET fusion-positive non-small cell lung cancer, and, under accelerated approval, advanced RET fusion-positive thyroid cancer.
Veppanu (vepdegestrant): for ER-positive, HER2-negative, ESR1-mutated advanced breast cancer, available since August 2026.
R289 is an investigational Rigel pill that blocks IRAK1 and IRAK4, 2 enzymes in an inflammation signaling pathway. It is not FDA approved.
Trial: a Phase 1b study in adults with lower-risk myelodysplastic syndromes, mainly people whose MDS came back or did not respond to earlier treatment (NCT05308264), now enrolling its dose-expansion part.
Timing: Rigel said on August 4, 2026 that it expects to finish expansion enrollment and choose a Phase 2 dose in the second half of 2026, and to share early expansion data by the end of the year.
Rigel Pharmaceuticals, Inc. has active programs across 6 rare diseases: Acute Myeloid Leukemia, Diffuse Intrinsic Pontine Glioma, Immune Thrombocytopenia, Myelodysplastic Syndromes, RET Fusion Cancer, Warm AutoimmuneHemolytic Anemia. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Rigel Pharmaceuticals, Inc., pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Rigel Pharmaceuticals, Inc. has 3 FDA-approved drugs with a rare disease use that we track, as of November 2026.
Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.
Eligibility is decided by each program, so call the number listed before a prescription is filled.