A phase 2 clinical trial for people
with Pyruvate Kinase (PK) deficiency.

The DRIVE PK trial for people with Pyruvate Kinase deficiency

DRIVE PK is a phase 2 clinical trial for an investigational therapy, AG-348, developed to treat the underlying cause of Pyruvate Kinase (PK) deficiency.1,2 AG-348 is an oral medication that is being studied to determine whether it may increase the levels of healthy red blood cells in the body that are critical for people with PK deficiency.1,2 It is not known if AG-348 will help with increasing the levels of healthy red blood cells in the body—that is why this study is being conducted.

Sign up to learn more about the DRIVE PK trial.

A closer look at PK deficiency

PK deficiency is a rare inherited disease that can result in lifelong problems.1,2 It occurs when the protein (or enzyme) that is needed to make healthy (or mature) red blood cells is deficient—this enzyme is called Pyruvate Kinase.1 Because of this enzyme deficiency, people with PK deficiency have fewer healthy red blood cells (a condition known as anemia),1 and may experience signs and symptoms impacting quality of life, including:

  • Fatigue (extreme tiredness)3
  • Jaundice (a yellowing of the skin and/or whites of the eyes)4
  • Shortness of breath3,5

People with PK deficiency may also require frequent blood transfusions to help manage their PK deficiency.4 Though doctors can help manage signs and symptoms, there is no approved drug that treats the underlying cause of PK deficiency.2

The AG-348 DRIVE PK trial

The DRIVE PK trial will study an investigational therapy, AG-348, which is being developed to treat the underlying cause of this disease—the deficiency of the Pyruvate Kinase enzyme.1,2 AG-348 attaches to the defective enzyme and may help to activate it, which, in turn, may help increase the amount of healthy red blood cells in the body.2 This could help people with PK deficiency avoid the damaging effects of the disease, but this has not been proven.

In the DRIVE PK trial, doctors will study different doses of AG-348 to see if the treatment is safe and well tolerated in adults. Doctors will also look for early signs that AG-348 helps increase the amount of healthy red blood cells. This trial will last up to 24 weeks, and all participants who qualify to participate in the study will receive AG-348. There is no cost to be a part of this trial.

Sign up for more information on the DRIVE PK trial and PK deficiency.

SIGN UP FOR MORE INFORMATION ABOUT THE DRIVE PK TRIAL

Thank you for your interest in DRIVE PK, a clinical trial for people with PK deficiency.

We will be in touch by phone or email soon.

Learn more about PK deficiency at agios.com

Thank you for your interest in more information about PK Deficiency.

From time to time, Agios will share news, updates, and the latest information about PK deficiency and upcoming clinical trials.

Learn more about PK Deficiency at agios.com

DRIVE PK trial locations

The DRIVE PK trial will take place at multiple clinical centers across North America and Europe. Since the trial is just beginning, additional locations will be added over time.

See the current
DRIVE PK trial locations

A closer look at PK deficiency

PK deficiency is a rare inherited disease that can result in lifelong problems.1,2 It occurs when the protein (or enzyme) that is needed to make healthy (or mature) red blood cells is deficient—this enzyme is called Pyruvate Kinase.1 Because of this enzyme deficiency, people with PK deficiency have fewer healthy red blood cells (a condition known as anemia),1 and may experience signs and symptoms impacting quality of life, including:

  • Fatigue (extreme tiredness)3
  • Jaundice (a yellowing of the skin and/or whites of the eyes)4
  • Shortness of breath3,5

People with PK deficiency may also require frequent blood transfusions to help manage their PK deficiency.4 Though doctors can help manage signs and symptoms, there is no approved drug that treats the underlying cause of PK deficiency.2

DRIVE PK trial locations

The DRIVE PK trial will take place at multiple clinical centers across North America and Europe. Since the trial is just beginning, additional locations will be added over time.

See the current
DRIVE PK trial locations

The AG-348 DRIVE PK trial

The DRIVE PK trial will study an investigational therapy, AG-348, which is being developed to treat the underlying cause of this disease—the deficiency of the Pyruvate Kinase enzyme.1,2 AG-348 attaches to the defective enzyme and may help to activate it, which, in turn, may help increase the amount of healthy red blood cells in the body.2 This could help people with PK deficiency avoid the damaging effects of the disease, but this has not been proven.

In the DRIVE PK trial, doctors will study different doses of AG-348 to see if the treatment is safe and well tolerated in adults. Doctors will also look for early signs that AG-348 helps increase the amount of healthy red blood cells. This trial will last up to 24 weeks, and all participants who qualify to participate in the study will receive AG-348. There is no cost to be a part of this trial.

Sign up for more information on the DRIVE PK trial and PK deficiency.

About Agios

Agios is a biopharmaceutical company focused on discovering and developing novel medicines to treat cancer and rare genetic disorders through its scientific leadership in the field of cellular metabolism. Agios is developing multiple first-in-class investigational medicines, including a potential treatment for PK deficiency, a rare, genetic, red blood cell disorder.

Learn more at agios.com

Questions? Comments? Want more information?

Whether you have PK deficiency or are caring for someone with the disease—you are the reason we do what we do. If you have questions or comments, or just want to know more, email us at PKdeficiencytrials@agios.com or contact us at 617-649-8600. Please remember not to send any specific information relating to your medical condition or other personal health information via email.