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CAMBRIDGE, Mass. - Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a biotechnology company with a market capitalization of $2 billion and strong financial health according to InvestingPro analysis, has appointed Jay Backstrom, M.D., M.P.H., to its Board of Directors, effective Tuesday, according to a press release from the company.
Dr. Backstrom, who most recently served as President and Chief Executive Officer of Scholar Rock, brings decades of leadership experience in pharmaceutical development. At Scholar Rock, he led the company’s spinal muscular atrophy program through Phase 3 development and regulatory filings in both the U.S. and European Union.
Prior to Scholar Rock, Backstrom held executive positions at Acceleron Pharma before its acquisition by Merck in 2021, and served as Chief Medical Officer and Head of Global Regulatory Affairs at Celgene Corporation, where he contributed to regulatory approvals in beta thalassemia and myelodysplastic syndromes.
"It is an honor to join Agios’ Board of Directors and work alongside such a talented group as the company enters its next phase of commercial growth and innovation," said Dr. Backstrom in the statement.
Brian Goff, Chief Executive Officer of Agios, noted that Backstrom’s appointment comes at "a pivotal time for Agios" as the company works to maximize the potential of PYRUKYND (mitapivat), its first-in-class PK activator, and advance its pipeline of rare disease medicines.
Backstrom will serve on Agios’ Science and Technology Committee.
Agios Pharmaceuticals focuses on developing treatments for rare diseases and currently markets a pyruvate kinase activator for adults with PK deficiency in the U.S. The company’s clinical pipeline includes programs for thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes-associated anemia and phenylketonuria. While revenue grew 26% in the last twelve months to $37 million, InvestingPro analysis suggests the stock is currently undervalued, with 8 additional exclusive ProTips and a comprehensive research report available for subscribers.
In other recent news, Agios Pharmaceuticals has been at the center of several notable developments. The company is anticipating key clinical trial data for its mitapivat drug, with TD Cowen reiterating a buy rating due to high confidence in its potential for reducing vaso-occlusive crises in sickle cell disease. This optimism is reflected in the projected $4 billion sales potential for the drug. Agios also faces a September 7 PDUFA date for mitapivat in thalassemia, which could approach $1 billion in global sales, according to TD Cowen. Meanwhile, H.C. Wainwright has adjusted its price target for Agios to $56 from $61, maintaining a Buy rating despite the reduction. This adjustment follows Agios’s commercial and distribution agreement with Avanzanite for the drug Pyrukynd in Europe, Switzerland, and the UK.
The partnership with Avanzanite is structured as a revenue share agreement, expanding the reach of Pyrukynd, a treatment for pyruvate kinase deficiency. Agios’s collaboration with Avanzanite is expected to facilitate market penetration across 32 European countries within the next year. Cantor Fitzgerald continues to maintain an Overweight rating on Agios, expressing confidence in the company’s potential to achieve sales targets for its thalassemia treatment. The firm’s analyst anticipates sales between $300 million and $500 million, with the pricing strategy aligned with that of polycystic kidney disease treatments. These recent developments highlight Agios’s strategic efforts to expand its market presence and address unmet needs in rare disease treatments.
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