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Global Blood's (GBT) Candidates for SCD Get Two FDA Tags
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Global Blood Therapeutics, Inc. announced that the FDA has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT021601 (GBT601) for the treatment of sickle cell disease (“SCD”), a chronic, inherited blood disorder that affects hemoglobin levels.
The orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes one that affects fewer than 200,000 individuals in the United States. The designation also includes incentives, including financial aid for clinical testing, and seven-year marketing exclusivity in the event of regulatory approval.
The rare pediatric disease designation is granted by the FDA in case of serious or life-threatening diseases, which usually affect individuals aged 18 years and below. The designation also qualifies for a priority review voucher if the drug is approved upon meeting certain conditions.
Shares of Global Blood have lost 17% so far this year compared with the industry’s decline of 24%.
Image Source: Zacks Investment Research
Global Blood is currently enrolling patients in two pivotal phase III studies evaluating the safety and efficacy of inclacumab (dosed quarterly) for the potential treatment of vaso-occlusive crisis (“VOC”) associated with SCD.
The first registrational study — GBT2104-131 — is investigating the effect of inclacumab on the frequency of VOCs while the second registrational study — GBT2104-132 — is evaluating the effect of a single dose of inclacumab on hospital readmission rates.
Global Blood has an exclusive license agreement with drug giant Roche (RHHBY - Free Report) for the development and commercialization of inclacumab, GBT’s P-selectin inhibitor.
Under the license agreement entered into in August 2018, Global Blood licensed inclacumab from RHHBY. The partnership with Roche for inclacumab looks like a strategic fit for Global Blood.
GBT has recently restarted the phase I study of GBT601, the company’s investigational next-generation sickle hemoglobin (HbS) polymerization inhibitor, to study a 150-mg daily dose, which is an increase over the 100-mg daily maintenance dose previously studied. GBT is also planning to advance GBT601 into a phase II/III study and anticipates initiating the phase II portion by mid-2022.
Per the company, GBT601 has the same mechanism of action as GBT’s sole marketed drug, Oxbryta (voxelotor), which is approved by the FDA as an oral, once-daily treatment for SCD in patients aged 12 years or above.
The Zacks Consensus Estimate for Leap Therapeutics’ loss per share has narrowed 11.1% for 2022 and 5.9% for 2023 in the past 60 days.
Earnings of Leap Therapeutics have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion. LPTX delivered an earnings surprise of 1.92%, on average.
Precision BioSciences’ loss per share estimates narrowed 21.7% for 2022 and 31.4% for 2023 in the past 60 days.
Earnings of Precision BioSciences have surpassed estimates in each of the trailing four quarters. DTIL delivered an earnings surprise of 76.15%, on average.
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Global Blood's (GBT) Candidates for SCD Get Two FDA Tags
Global Blood Therapeutics, Inc. announced that the FDA has granted both orphan drug and rare pediatric disease designations to inclacumab and GBT021601 (GBT601) for the treatment of sickle cell disease (“SCD”), a chronic, inherited blood disorder that affects hemoglobin levels.
The orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes one that affects fewer than 200,000 individuals in the United States. The designation also includes incentives, including financial aid for clinical testing, and seven-year marketing exclusivity in the event of regulatory approval.
The rare pediatric disease designation is granted by the FDA in case of serious or life-threatening diseases, which usually affect individuals aged 18 years and below. The designation also qualifies for a priority review voucher if the drug is approved upon meeting certain conditions.
Shares of Global Blood have lost 17% so far this year compared with the industry’s decline of 24%.
Image Source: Zacks Investment Research
Global Blood is currently enrolling patients in two pivotal phase III studies evaluating the safety and efficacy of inclacumab (dosed quarterly) for the potential treatment of vaso-occlusive crisis (“VOC”) associated with SCD.
The first registrational study — GBT2104-131 — is investigating the effect of inclacumab on the frequency of VOCs while the second registrational study — GBT2104-132 — is evaluating the effect of a single dose of inclacumab on hospital readmission rates.
Global Blood has an exclusive license agreement with drug giant Roche (RHHBY - Free Report) for the development and commercialization of inclacumab, GBT’s P-selectin inhibitor.
Under the license agreement entered into in August 2018, Global Blood licensed inclacumab from RHHBY. The partnership with Roche for inclacumab looks like a strategic fit for Global Blood.
GBT has recently restarted the phase I study of GBT601, the company’s investigational next-generation sickle hemoglobin (HbS) polymerization inhibitor, to study a 150-mg daily dose, which is an increase over the 100-mg daily maintenance dose previously studied. GBT is also planning to advance GBT601 into a phase II/III study and anticipates initiating the phase II portion by mid-2022.
Per the company, GBT601 has the same mechanism of action as GBT’s sole marketed drug, Oxbryta (voxelotor), which is approved by the FDA as an oral, once-daily treatment for SCD in patients aged 12 years or above.
Zacks Rank & Stocks to Consider
Global Blood currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector are Leap Therapeutics, Inc. (LPTX - Free Report) and Precision BioSciences, Inc. (DTIL - Free Report) , both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
The Zacks Consensus Estimate for Leap Therapeutics’ loss per share has narrowed 11.1% for 2022 and 5.9% for 2023 in the past 60 days.
Earnings of Leap Therapeutics have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion. LPTX delivered an earnings surprise of 1.92%, on average.
Precision BioSciences’ loss per share estimates narrowed 21.7% for 2022 and 31.4% for 2023 in the past 60 days.
Earnings of Precision BioSciences have surpassed estimates in each of the trailing four quarters. DTIL delivered an earnings surprise of 76.15%, on average.