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FDA Grants Orphan Drug Tag to AGIO's Tebapivat for Rare Cancer
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Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has granted orphan drug designation to its novel pyruvate kinase (PK) activator, tebapivat, for the treatment of myelodysplastic syndromes (MDS), a rare type of blood cancer.
The FDA generally grants orphan drug designation to investigational therapies that address rare medical diseases or conditions affecting fewer than 200,000 people in the United States. The perquisites that come from this designation include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity upon potential approval.
Agios is developing tebapivat (formerly, AG-946), its PK activator candidate, as a potential treatment for MDS and hemolytic anemias.
The company is currently starting a phase IIb study on tebapivat for treating lower-risk MDS. It has already achieved clinical proof-of-concept in a phase IIa study on tebapivat as a potential treatment for anemia in lower-risk MDS.
Shares of Agios have skyrocketed 98.9% so far this year compared with the industry’s rise of 14.2%.
Image Source: Zacks Investment Research
AGIO’s Lead PK Activator Mitapivat Holds Promise
Agios’ lead PK activator, Pyrukynd (mitapivat), was approved for treating hemolytic anemia in adults with PK deficiency in the United States and the EU in 2022.
Pyrukynd is the first disease-modifying therapy approved to treat adults with PK deficiency, a rare and debilitating blood disorder.
Agios is also conducting two phase III studies, namely ACTIVATE-kids and ACTIVATE-kidsT, on Pyrukynd for PK deficiency in pediatric patients who are not regularly transfused and are regularly transfused, respectively.
Apart from PK deficiency, Pyrukynd is also being developed for other hemolytic anemias, including sickle cell disease (SCD) and thalassemia, in several label expansion studies.
Based on the encouraging progress in clinical development across both indications, Agios expects to launch Pyrukynd in thalassemia commercially by 2025 and SCD by 2026.
The FDA has already granted orphan drug designations to mitapivat for both thalassemia and SCD.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.18 to $3.62. Earnings per share estimates for 2025 have improved from $2.93 to $4.43. Year to date, shares of ILMN have lost 10.3%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $1.98 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 60.3%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.53. Year to date, shares of FULC have jumped 31.1%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.
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FDA Grants Orphan Drug Tag to AGIO's Tebapivat for Rare Cancer
Agios Pharmaceuticals, Inc. (AGIO - Free Report) announced that the FDA has granted orphan drug designation to its novel pyruvate kinase (PK) activator, tebapivat, for the treatment of myelodysplastic syndromes (MDS), a rare type of blood cancer.
The FDA generally grants orphan drug designation to investigational therapies that address rare medical diseases or conditions affecting fewer than 200,000 people in the United States. The perquisites that come from this designation include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity upon potential approval.
Agios is developing tebapivat (formerly, AG-946), its PK activator candidate, as a potential treatment for MDS and hemolytic anemias.
The company is currently starting a phase IIb study on tebapivat for treating lower-risk MDS. It has already achieved clinical proof-of-concept in a phase IIa study on tebapivat as a potential treatment for anemia in lower-risk MDS.
Shares of Agios have skyrocketed 98.9% so far this year compared with the industry’s rise of 14.2%.
Image Source: Zacks Investment Research
AGIO’s Lead PK Activator Mitapivat Holds Promise
Agios’ lead PK activator, Pyrukynd (mitapivat), was approved for treating hemolytic anemia in adults with PK deficiency in the United States and the EU in 2022.
Pyrukynd is the first disease-modifying therapy approved to treat adults with PK deficiency, a rare and debilitating blood disorder.
Agios is also conducting two phase III studies, namely ACTIVATE-kids and ACTIVATE-kidsT, on Pyrukynd for PK deficiency in pediatric patients who are not regularly transfused and are regularly transfused, respectively.
Apart from PK deficiency, Pyrukynd is also being developed for other hemolytic anemias, including sickle cell disease (SCD) and thalassemia, in several label expansion studies.
Based on the encouraging progress in clinical development across both indications, Agios expects to launch Pyrukynd in thalassemia commercially by 2025 and SCD by 2026.
The FDA has already granted orphan drug designations to mitapivat for both thalassemia and SCD.
Zacks Rank & Stocks to Consider
Agios currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Illumina, Inc. (ILMN - Free Report) , Krystal Biotech, Inc. (KRYS - Free Report) and Fulcrum Therapeutics, Inc. (FULC - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.18 to $3.62. Earnings per share estimates for 2025 have improved from $2.93 to $4.43. Year to date, shares of ILMN have lost 10.3%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $1.98 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 60.3%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.53. Year to date, shares of FULC have jumped 31.1%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.