Clinical Trials

Clinical trials—and their participants—are a key part of our programs’ success

Clinical trials are research studies that aim to evaluate the success of our methodology and to answer our scientific inquiries so that we can best accomplish our goal of treating rare cancers. These trials are one of the primary ways we are able to evaluate the potential success of our innovative treatments.

The science behind our trial

If you are a Plixorafenib clinical trial participant or candidate, you probably have questions about how the drug works. Here are the key facts.


Plixorafenib targets cancer cells with BRAF mutations

Plixorafenib is a small molecule that targets cancer cells with a mutated BRAF gene. Unlike conventional chemotherapy, this targeted therapy spares healthy cells, as their BRAF gene is not mutated.

Blocking mutant proteins can slow tumor growth

Our cells’ BRAF gene contains instructions for making BRAF proteins. BRAF proteins are large molecules that work with other proteins to relay a signal that tells the cell that it’s time to grow and divide. Changes, or mutations, to the BRAF gene can cause the BRAF protein to keep this signal permanently on, meaning that affected cells may multiply uncontrollably and possibly form a cancerous tumor. Plixorafenib is designed to slow tumor growth by locking into and silencing mutant BRAF proteins, thereby switching off the rogue cell division signal.

Targeted therapy differs from chemotherapy

Plixorafenib is designed to block only mutated BRAF proteins. Most BRAF mutations are not inherited; instead, they usually develop after birth and only in certain cells. Plixorafenib can slow tumor growth by blocking the multiplication of only these mutant cells, while sparing cells with normal BRAF genes. This selectivity means fewer side effects than traditional chemotherapies, which kill any rapidly reproducing cell, including healthy ones.

Plixorafenib differs from older BRAF inhibitors:

Studies show that Plixorafenib is potentially safer and more effective than first-generation BRAF-inhibitors. These first-generation inhibitors only target the most common BRAF mutations, and paradoxically, can even amplify cancerous activity in certain mutant cells. By contrast, Plixorafenib appears to inhibit a wider range of BRAF mutations, both common and rare, and does not trigger the drug resistance seen with first-generation inhibitors.

For more information about the trial:

Fore is running a clinical trial open for BRAF fusions patients and BRAF mutant glioma patients, as detailed in the NCT02428712 study protocol.


To be eligible for this study, participants must meet several criteria, including but not limited to the following:

  • Participants must have an advanced, inoperable solid or CNS tumor with a BRAF mutation. Examples include brain tumors, Langerhans cell histiocytosis, or any advanced inoperable solid tumor containing this mutation.
  • Participants’ cancers must persist despite prior therapy or be considered incurable using standard therapies.
  • Participants must be able to carry out work of a light or sedentary nature, e.g., light house work, office work.
  • This study includes adolescents and adults ages 10 years and older.

Clinical trial information


PLX 120-03: A Phase 1/2a Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of PLX8394 in Patients with Advanced, Unresectable Solid Tumors.


The purpose of this study is to assess the safety and effectiveness of the investigational drug Plixorafenib (previously PLX-8394) in patients with advanced, inoperable solid tumors that contain an altered form of a gene called BRAF. Certain mutations or “translocations” in the BRAF gene cause a change in the BRAF protein that can promote cancer growth and spread. Plixorafenib works by preventing these altered BRAF proteins from working, thereby blocking the growth and spread of cancer cells.

In this study, patients will receive Plixorafenib in combination with cobicistat, a drug used to increase the amount of Plixorafenib in the blood. Both drugs are tablets that are taken orally (by mouth).

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Fore Bio
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Suite 200
Philadelphia, PA 19104

Request more information

For information about the study or to check suitability for enrolling as a new patient, please contact us via the below form or at oib.e1709086894rof@s1709086894lairt1709086894.