Exelixis

Exelixis works on 10 rare diseases tracked on Trial Friend, including Cholangiocarcinoma, Essential Thrombocythemia, Familial Medullary Thyroid Carcinoma and 7 more, with 3 active clinical trials (2 recruiting) and 2 FDA-approved rare disease drugs.

Exelixis is an oncology-focused pharmaceutical company best known for cabozantinib (Cabometyx/Cometriq), a multikinase inhibitor FDA-approved for medullary thyroid cancer, renal cell carcinoma, and hepatocellular carcinoma. The company has a growing pipeline of next-generation tyrosine kinase inhibitors and antibody-drug conjugates targeting solid tumors.

Type
Diversified Pharma
Ticker
EXEL
Headquarters
Alameda, United States
Founded
1994
3
Active Rare Disease Trials
2
Approved Rare Disease Drugs
10
Rare Diseases in Portfolio
32
Years Active

Focus areas at Exelixis

Within its broader pharmaceutical portfolio, Exelixis has active clinical trial programs and drug development efforts across 10 rare diseases, including Cholangiocarcinoma, Essential Thrombocythemia, Familial Medullary Thyroid Carcinoma, Gastrointestinal Stromal Tumor, Medullary Thyroid Cancer, and 5 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows active and recruiting rare disease studies sponsored by Exelixis, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Exelixis is headquartered in Alameda, United States, founded in 1994, publicly traded under the ticker symbol EXEL. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Exelixis Drug Pipeline

Exelixis has 3 active clinical trials across 3 development stages, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.

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1
Phase 21 trial
Non-Small Cell Lung Cancer
Recruiting
1
Phase 31 trial
1
Phase 4 / Post-Market1 trial
Active Not Recruiting

Exelixis Clinical Trials (3)

Active and recruiting clinical trials sponsored by Exelixis, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

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RECRUITINGPHASE2, PHASE3Recently updatedNCT06943755

Zanzalintinib Versus Everolimus in Participants With Locally Advanced or Metastatic Neuroendocrine Tumors

Intervention: Zanzalintinib, Everolimus

The primary purpose of this study is to assess the effectiveness of zanzalintinib compared to everolimus in participants with previously treated, unresectable, locally advanced or metastatic neuroendocrine tumors.

Ages 18 Years+131 locations
View full study
RECRUITINGPHASE2Recently updatedNCT01639508

Cabozantinib in Patients With RET Fusion-Positive Advanced Non-Small Cell Lung Cancer and Those With Other Genotypes: ROS1 or NTRK Fusions or Increased MET or AXL Activity

Intervention: Cabozantinib

Non-Small Cell Lung Cancer

The purpose of this phase II study is to find out what effects cabozantinib (XL184) has, good and/or bad, in patients whose tumors one of the following gene changes RET, ROS1, or NTRK fusion, or increased MET or AXL activity. A phase II study looks at how effective a medication is at treating a specific type of cancer and collects information on the side effects of the study treatment. RET, ROS1, or NTRK fusion or increased MET or AXL activity gene leads to lung cancer cell growth. Cabozantinib is an oral medicine that inhibits of RET, ROS1, NTRK, MET, and AXL. In addition, this drug interferes with other cell pathways that also cause cancer cells to grow, form new blood vessels, and spread to other organs of the body. The goal of using cabozantinib is to shrink the cancer and to prevent it from growing Cabozantinib has been studied and shown to cause cancer shrinkage in other cancers such as medullary thyroid cancer and prostate cancer. We thus have a good idea of what side-effects it causes and can anticipate them.

Ages 18 Years+7 locations
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ACTIVE NOT RECRUITINGPHASE4Hasn't posted an update in over a yearNCT01896479

A Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer

Intervention: Cabozantinib (XL184) 140 mg, Cabozantinib (XL184) 60 mg, Placebo tablet, Placebo capsule

The objective of this study is to evaluate the efficacy and safety of oral cabozantinib at a 60 mg dose compared with a 140 mg dose in subjects with progressive, metastatic MTC. It will test if the lower dose results in similar progression free survival (PFS) and overall response rate (ORR) with fewer adverse events compared to the PFS, ORR and adverse events found in previous clinical trials of 140 mg.

Ages 18 Years+49 locations
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Exelixis FDA-Approved Drugs (2)

Medications developed or marketed by Exelixis that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Drug NameBrand NameRare DiseasesApproval Date
CABOZANTINIBCABOMETYX
oral
—Apr 25, 2016
CABOZANTINIBCOMETRIQ
oral
Nov 29, 2012

Exelixis Trial Locations

Exelixis clinical trials are running at 187 sites in 23 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
47▼
France
18▼
Italy
18▼
Spain
17▼
Australia
10▼
United Kingdom
10▼
South Korea
8▼
Germany
7▼
Netherlands
7▼
Poland
7▼
Canada
5▼
Belgium
4▼

Rare Disease Focus Areas (10)

Diseases targeted by Exelixis's clinical trial and drug development programs

CholangiocarcinomaRare Cancers

Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (...

Prevalence: About 8,000 new cases per year in the U.S.; rising incidence worldwide
Essential ThrombocythemiaBlood & Immune

Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts. Patients are at increased risk for blood...

Prevalence: Approximately 1-2 cases per 100,000 people; median age of diagnosis is 60 years
Familial Medullary Thyroid CarcinomaEndocrine & Hormonal

Familial medullary thyroid carcinoma is an autosomal dominant cancer predisposition syndrome caused by germline RET mutations. Nearly all mutation carriers develop medullary thyroid cancer by adulthoo...

Prevalence: Near-complete penetrance by adulthood in RET mutation carriers (varies by specific codon mutation); comprises ~25% of medullary thyroid cancers
Gastrointestinal Stromal TumorRare Cancers

Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract, arising from the interstitial cells of Cajal or their precursors. Most GISTs are driven by...

Prevalence: About 6,000 new cases per year in the U.S. (American Cancer Society); true incidence may be higher as small GISTs are often incidentally discovered
Medullary Thyroid CancerRare Cancers

Medullary thyroid cancer (MTC) is a rare neuroendocrine tumor arising from the parafollicular C cells of the thyroid gland, accounting for 2-4% of all thyroid cancers. About 75% of cases are sporadic ...

Prevalence: Approximately 1,000-1,200 new cases per year in the U.S.; 2-4% of all thyroid cancers
Merkel Cell CarcinomaRare Cancers

Merkel cell carcinoma is a rare, aggressive skin cancer that arises from Merkel cells, specialized touch receptors in the skin. It typically appears as a painless, firm nodule on sun-exposed areas, gr...

Prevalence: About 3,000 new cases per year in the U.S.; incidence has tripled over the past 2 decades

Patient Resources

Organizations and resources related to Exelixis's rare disease focus areas

Frequently Asked Questions About Exelixis

Common questions about Exelixis's rare disease programs, clinical trials, and treatments.