Three Proof-of-Concept Studies Support GBT440 Mechanism of Action and Safety Profile
Clinically Meaningful Efficacy Results Not Achieved in Patients with IPF
Data Supports Ongoing GBT440 Program in Sickle Cell Disease
SOUTH SAN FRANCISCO, CA, USA I October 23, 2017 I Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced the discontinuation of its GBT440 program for the treatment of idiopathic pulmonary fibrosis (IPF). This decision is based on results from three proof-of-concept studies including a Phase 1 study in healthy volunteers called Basecamp and two Phase 2a studies in patients with IPF called GBT440-006 and ZEPHYR.
“From the outset, we set a high bar for success in our IPF program. The results re-affirm our confidence in the mechanism of action of GBT440. However, the data from these proof-of-concept studies did not demonstrate sufficient overall clinical benefit to justify continuing the program. While we are disappointed that we didn’t meet our high bar for success, and we are disappointed that we will not be able to help the IPF community, we are grateful to the patients, healthy volunteers and healthcare professionals who participated in the trials and supported us in these efforts,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We are encouraged that the safety and mechanistic data reinforce the potential of GBT440 as a disease-modifying treatment for individuals living with sickle cell disease. We remain on track to announce top-line clinical trial results from our Phase 3 HOPE Study in the first half of 2019.”
GBT440 was generally well tolerated across all three studies, and no new safety signals emerged. In Basecamp, healthy volunteers who received GBT440 900 mg under hypoxic conditions demonstrated a statistically significant increase in oxygen saturation, confirming the mechanism of action of the drug in hypoxemia and demonstrating that GBT440 is a potent and effective hemoglobin modifier. In GBT440-006, adults with IPF and low oxygen levels during exercise who received GBT440 1,500 mg demonstrated a statistically significant but more modest dose-dependent improvement in oxygen-saturation. However, this increase in oxygen-saturation at the highest dose tested did not appear to be large enough to translate into clinically meaningful benefit based on multiple endpoints including oxygen dependency with exercise. In ZEPHYR, adults with more severe IPF (on oxygen at rest) who received GBT440 900 mg did not demonstrate improvement in oxygen saturation.
Overall, these studies demonstrate proof-of-concept regarding improvement in oxygen saturation in healthy volunteers and patients with IPF. However, based on the totality of the data, GBT believes this improvement is unlikely to translate into a transformative clinical benefit for IPF patients. The company plans to present the full data for each of the three studies at a future medical meeting. GBT intends to make no further investments in additional clinical studies in hypoxemia with GBT440 and intends to stop further enrollment in any ongoing studies.
Conference Call and Webcast Details
Management will host a conference call today at 8:30 a.m. Eastern Time/5:30 a.m. Pacific Time. To participate in the conference call, please dial (844) 471-0808 (domestic) or (480) 696-7309 (international) and refer to conference ID 3198319. Live audio of the conference call will be simultaneously webcast and will be available under the Investors section of the company’s website at www.globalbloodtx.com. The webcast will be archived under the Investors section of the company’s website and will be available for replay for at least one month.
About Global Blood Therapeutics
Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. GBT is developing its late-stage product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit www.globalbloodtx.com and follow the company on Twitter @GBT_News.
SOURCE: Global Blood Therapeutics
Post Views: 116
Three Proof-of-Concept Studies Support GBT440 Mechanism of Action and Safety Profile
Clinically Meaningful Efficacy Results Not Achieved in Patients with IPF
Data Supports Ongoing GBT440 Program in Sickle Cell Disease
SOUTH SAN FRANCISCO, CA, USA I October 23, 2017 I Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced the discontinuation of its GBT440 program for the treatment of idiopathic pulmonary fibrosis (IPF). This decision is based on results from three proof-of-concept studies including a Phase 1 study in healthy volunteers called Basecamp and two Phase 2a studies in patients with IPF called GBT440-006 and ZEPHYR.
“From the outset, we set a high bar for success in our IPF program. The results re-affirm our confidence in the mechanism of action of GBT440. However, the data from these proof-of-concept studies did not demonstrate sufficient overall clinical benefit to justify continuing the program. While we are disappointed that we didn’t meet our high bar for success, and we are disappointed that we will not be able to help the IPF community, we are grateful to the patients, healthy volunteers and healthcare professionals who participated in the trials and supported us in these efforts,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We are encouraged that the safety and mechanistic data reinforce the potential of GBT440 as a disease-modifying treatment for individuals living with sickle cell disease. We remain on track to announce top-line clinical trial results from our Phase 3 HOPE Study in the first half of 2019.”
GBT440 was generally well tolerated across all three studies, and no new safety signals emerged. In Basecamp, healthy volunteers who received GBT440 900 mg under hypoxic conditions demonstrated a statistically significant increase in oxygen saturation, confirming the mechanism of action of the drug in hypoxemia and demonstrating that GBT440 is a potent and effective hemoglobin modifier. In GBT440-006, adults with IPF and low oxygen levels during exercise who received GBT440 1,500 mg demonstrated a statistically significant but more modest dose-dependent improvement in oxygen-saturation. However, this increase in oxygen-saturation at the highest dose tested did not appear to be large enough to translate into clinically meaningful benefit based on multiple endpoints including oxygen dependency with exercise. In ZEPHYR, adults with more severe IPF (on oxygen at rest) who received GBT440 900 mg did not demonstrate improvement in oxygen saturation.
Overall, these studies demonstrate proof-of-concept regarding improvement in oxygen saturation in healthy volunteers and patients with IPF. However, based on the totality of the data, GBT believes this improvement is unlikely to translate into a transformative clinical benefit for IPF patients. The company plans to present the full data for each of the three studies at a future medical meeting. GBT intends to make no further investments in additional clinical studies in hypoxemia with GBT440 and intends to stop further enrollment in any ongoing studies.
Conference Call and Webcast Details
Management will host a conference call today at 8:30 a.m. Eastern Time/5:30 a.m. Pacific Time. To participate in the conference call, please dial (844) 471-0808 (domestic) or (480) 696-7309 (international) and refer to conference ID 3198319. Live audio of the conference call will be simultaneously webcast and will be available under the Investors section of the company’s website at www.globalbloodtx.com. The webcast will be archived under the Investors section of the company’s website and will be available for replay for at least one month.
About Global Blood Therapeutics
Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. GBT is developing its late-stage product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit www.globalbloodtx.com and follow the company on Twitter @GBT_News.
SOURCE: Global Blood Therapeutics
Post Views: 116
