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Agios Announces Data from Phase 2 Study of PK Deficiency


Dec. 4 2020, Updated 10:53 a.m. ET

Agios phase 2 PK data

Agios Pharmaceuticals (AGIO) was one of the top performers among the small-cap segment within the iShares Nasdaq Biotechnology ETF (IBB) on June 13, 2016. SciClone (SCLN) was the top outperformer in the small-cap segment. It rose by 5.4%. Read Part 1 in this series for more information on the stock. Agios Pharmaceuticals gained 3.2% as it released the data from the Phase 2 PK deficiency study. It outperformed the SPDR S&P 500 ETF (SPY). SPY fell by 0.8% for the day.

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In a press release, Agios Pharmaceuticals announced that “initial data demonstrating that AG-348 achieved proof-of-concept in an ongoing Phase 2 study (DRIVE-PK) of patients with pyruvate kinase (PK) deficiency, a rare, potentially debilitating, congenital anemia. AG-348 is a novel, first-in-class, an oral activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes. AG-348 is wholly owned by Agios. Data will be presented today at the 21st Congress of the European Hematology Association (EHA) taking place June 9-12, 2016 in Copenhagen.” The study indicated:

  • Nine of 18 total patients and nine of 13 patients with at least one missense mutation showed maximal hemoglobin increases between 2.3 to 4.9 g/dL.
  • AG-348 was tolerated well with up to six months of daily dosing.

Agios Pharmaceuticals closed at $51.97 and traded below 20-day moving average price of $53.80. The stock has fallen by 20% on a YTD basis. Agios has a book value of $8.77 per share. At its current price, the stock is trading at a price-to-book value of ~5.90x.

Analysts’ expectations and recommendations

Analysts expect Agios Pharmaceutical’s 12-month price target to be at $62.30. Five analysts recommended the stock as a “buy.” Three analysts recommended the stock as a “hold” and no analysts recommended the stock as a “sell.” Agios has a weight of ~0.4% in IBB.

On the other hand, Sucampo Pharmaceuticals (SCMP) was one of the worst performers within IBB’s small-cap segment. The stock fell due to profit-booking. It’s down for the third consecutive day. It closed at $10.83 and traded below the 20-day and 100-day moving averages. The stock has fallen by 37.4% on a YTD basis. Sucampo Pharmaceuticals carries a weight of 0.1% in IBB’s portfolio.


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