You are here

Research and Pipeline

Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.

These studies are investigating treatments or outcomes that have not received approval from a Health Authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a Health Authority.

Cystic Fibrosis

Ivacaftor

Cystic Fibrosis
Investigational studies in currently marketed product

Two Phase 3 clinical investigational studies are underway in children under 2 years of age, and children ages 3 through 5, with one of the following mutations in the cystic fibrosis conductance regulator (CFTR) gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.

Investigational studies in currently marketed product

How It Works

Ivacaftor is designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
 

Our Studies

Two Phase 3 clinical investigational studies are underway in children under 2 years of age, and children ages 3 through 5, who have one of the following mutations in the cystic fibrosis conductance regulator (CFTR) gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.

To learn more about the clinical investigational study in children under 2 years of age, visit clinicaltrials.gov.

To learn more about the clinical investigational study in children ages 3 through 5 measuring lung clearance index (LCI), a sensitive measure to assess lung function early on in the disease, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Lumacaftor/Ivacaftor

Cystic Fibrosis
Investigational studies in currently marketed product

A Phase 3 clinical investigational study is underway in children ages 2 through 5 who have two copies of the F508del mutation in the CFTR gene. 

Investigational studies in currently marketed product
Lumacaftor/Ivacaftor

How It Works

In people with certain types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no protein at the cell surface. Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. This helps improve the flow of salt and water in to and out of cells.
 

Our Studies

Vertex is conducting a Phase 3 clinical investigational study of lumacaftor/ivacaftor in children ages 2 through 5 who have two copies of the F508del mutation. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage

Tezacaftor (VX-661) + Ivacaftor

Cystic Fibrosis
Phase 3

A Phase 3 clinical investigational study is underway in people ages 12 and older who have one copy of the F508del mutation and a second mutation that has been shown to be clinically responsive to ivacaftor.

We also announced data for two other Phase 3 investigational clinical studies in people ages 12 and older.

Phase 3
Tezacaftor (VX-661) + Ivacaftor

How It Works

In people with two copies of the F508del mutation, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. Tezacaftor (VX-661) is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.

 

Our Studies

A Phase 3 clinical investigational study is underway in people ages 12 and older who have one copy of the F508del mutation and a second mutation that has been shown to be clinically responsive to ivacaftor. To learn more, visit clinicaltrials.gov.

For information about data announced in March 2017 for Phase 3 investigational clinical studies in people ages 12 and older who have two copies of the F508del mutation or one copy of the F508del mutation that results in residual CFTR function, Learn More.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-440 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 2

A Phase 2 clinical investigational study is underway in people ages 12 and older who have two copies of the F508del mutation or one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy.

Phase 2
VX-440 + Tezacaftor + Ivacaftor

How It Works

In people with certain types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-440 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex is conducting a Phase 2 clinical investigational study of VX-440 in triple combinations with tezacaftor and ivacaftor in people ages 12 and older who have two copies of the F508del mutation or who have one copy of the F508del mutation and a minimal function CFTR mutation that is not likely to respond to tezacaftor and/or ivacaftor therapy. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-152 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 2

A Phase 2 clinical investigational study is underway in people ages 18 and older who have one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy, or people who have two copies of the F508del mutation. 

Phase 2
VX-152 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-152 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex is conducting a Phase 2 clinical investigational study of VX-152 in triple combination with tezacaftor and ivacaftor in people ages 18 and older who have one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy, or people who have two copies of the F508del mutation. To learn more, please visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-371 + lumacaftor + ivacaftor

Cystic Fibrosis
Phase 2

A Phase 2a clinical investigational study is underway in people ages 12 and older who have two copies of the F508del mutation who are also being treated with lumacaftor/ivacaftor.

There is also a Phase 2 clinical investigational study of VX-371 in people ages 12 and older who have primary ciliary dyskinesia (PCD).

 

Phase 2
VX-371 + lumacaftor + ivacaftor

How It Works

In partnership with Parion Sciences, our VX-371 program is investigating the role of sodium epithelial channel (ENaC) inhibitors in people with CF. Learn more here.

 

Our Studies

Vertex is conducting a Phase 2a clinical investigational study of VX-371 in people ages 12 and older who have two copies of the F508del mutation who are also being treated with lumacaftor/ivacaftor. To learn more, visit clinicaltrials.gov.

There is also a Phase 2 clinical investigational study of VX-371 in people ages 12 and older who have primary ciliary dyskinesia (PCD), a rare genetic disease that results in a loss of function in key ciliary proteins. The defective proteins lead to dysfunctional beating of cilia on the surface of cells, especially in the lungs, where the accumulation of mucus can lead to chronic lung infections, bronchiectasis and progressive lung function decline. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-659 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 1

A Phase 1 clinical investigational study is underway in healthy volunteers and people with CF who have one copy of the F508del mutation and one minimal function mutation.

Phase 1
VX-659 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-659 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
 

Our Studies

Vertex is conducting a Phase 1 clinical Investigational study of VX-659 alone and in triple combiniation in healthy volunteers and people with CF who have one copy of the F508del mutation and one minimal function mutation. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-445 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 1

A Phase 1 clinical investigational study of VX-445 alone, and in triple combination with tezacaftor and ivacaftor, is underway in healthy volunteers.

Phase 1
VX-445 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-445 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex is conducting a Phase 1 study of VX-445 alone and in triple combination with tezacaftor and ivacaftor in healthy volunteers.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Gene Editing

Cystic Fibrosis
Research & Investigation
Research & Investigation
Gene Editing

Gene Editing

Vertex is collaborating with CRISPR Therapeutics to investigate the use of CRISPR's gene editing technology, known as CRISPR-Cas9, to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. As part of the collaboration, Vertex and CRISPR will evaluate the use of CRISPR-Cas9 to potentially correct the mutations in the CFTR gene known to result in the defective protein that causes CF and to edit other genes that contribute to the disease. Check back for future updates.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Messenger Ribonucleic Acid (mRNA) Therapies

Cystic Fibrosis
Research & Investigation
Research & Investigation
Messenger Ribonucleic Acid (mRNA) Therapies

Messenger Ribonucleic Acid (mRNA) Therapies

Vertex is collaborating with Moderna Therapeutics in an exclusive research collaboration and licensing agreement aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics™ for the treatment of cystic fibrosis (CF). The research collaboration focuses on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery. Check back for future updates.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Acute Spinal Cord Injury

VX-210

Acute Spinal Cord Injury
Phase 2

A Phase 2b/3 clinical investigational study is underway in people with certain types of acute cervical spinal cord injuries.

Phase 2
VX-210

How it Works

 

Vertex is investigating VX-210 as a potential medicine for acute spinal cord injury. VX-210 is designed to promote neurologic recovery by inhibiting a protein known as Rho, which blocks neural regeneration after injury.  

 

Our Studies

In 2016, Vertex initiated the SPRING Trial: a randomized, double blind, placebo controlled Phase 2b/3 study to evaluate the efficacy and safety of VX-210 in patients with certain types of acute cervical spinal cord injuries. Learn more about the VX-210 SPRING trial at www.vertexscitrial.com or at clinicaltrials.gov.

About Acute Spinal Cord Injury

Spinal Cord Injury, Severity, and Treatment

The spinal cord is a bundle of nerves that connects the body to the brain. These nerves carry signals to every part of the body. A spinal cord injury disrupts the signals.

Spine injuries are fractures or compressions of the vertebrae, the bone disks that make up the spine. Most injuries don't cut through the spinal cord. Spinal cord injury can occur when pieces of vertebrae tear spinal cord tissue or create pressure on the nerves.

Cervical spinal cord injury refers to a type of injury in the neck area of the spine.

An acute spinal cord injury is a medical emergency. Treatments may include surgery to stabilize the spine and relieve pressure on the spinal cord.

The SPRING Trial is evaluating an investigational therapy, VX-210, which is applied to the spinal cord during initial decompression/stabilization surgery. Learn more at www.vertexscitrial.com.

Pain

VX-150

Pain
Phase 2

Vertex completed a Phase 2 proof-of-concept study in osteoarthritis and plans to initiate two additional proof-of concept studies in 2017, in acute pain and neuropathic pain.

Phase 2
VX-150

How it Works

Vertex is investigating VX-150 as a potential medicine for the treatment of acute and neuropathic pain. VX-150 is designed to block pain sensation through inhibition of NaV1.8, a voltage-gated sodium channel expressed in peripheral neurons that transmit pain.

Our Studies

In 2016, Vertex completed a Phase 2 proof-of-concept study in osteoarthritis and plans to initiate two additional proof-of concept studies in 2017, in acute pain and neuropathic pain. To learn more, visit clinicaltrials.gov.

   

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)

VX-970

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 2
Phase 2
VX-970

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

VX-803

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1
Phase 1
VX-803

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

VX-984

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1
Phase 1
VX-984

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

Influenza (Outlicensed to Janssen Pharmaceuticals, Inc.)

VX-787

Influenza (Outlicensed to Janssen Pharmaceuticals, Inc.)
Phase 2
Phase 2
VX-787

In June 2014, Vertex entered into a licensing agreement with Janssen Pharmaceuticals, Inc. for the worldwide development and commercialization of VX-787, a novel medicine discovered by Vertex for the treatment of influenza. Learn more.