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Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), currently trading at $30.74 and showing a 6.5% gain year-to-date, issued a statement Monday addressing a securities analyst report published the same day regarding recent safety data for PYRUKYND, its treatment for hemolytic anemia in adults with pyruvate kinase (PK) deficiency. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet, though it’s currently experiencing rapid cash burn. The analyst report referenced data from the U.S. Food and Drug Administration’s Adverse Event Reporting System (FAERS), obtained through a Freedom of Information Act request.
According to Agios, the report highlighted four patient cases involving PYRUKYND. Three of these cases had previously been reported to both Agios and the FDA as part of routine pharmacovigilance. Two of these cases involved patients using commercially available PYRUKYND for PK deficiency. The third case concerned a patient with sickle cell disease who received PYRUKYND through expanded access, also known as compassionate use, at the treating physician’s request.
The company detailed the cases as follows:
- The first case involved a 61-year-old male with PK deficiency who died from metastatic liver cancer, as confirmed by a healthcare professional.
- The second case was a 93-year-old female with PK deficiency who entered hospice care due to kidney failure and cardiac issues and subsequently passed away.
- The third case involved a 26-year-old female with sickle cell disease, who had a history of heart arrhythmia, silent infarct, and aneurysm, and died from a pulmonary embolism while also being treated for a urinary tract infection. Elevated liver enzymes were noted but not attributed to PYRUKYND by the reporting healthcare provider.
The fourth case, which was reported directly to the FDA, involved a 28-year-old female with unspecified medical history and several adverse event terms, including hypertransaminasaemia. Bactrim was listed as another suspect drug in this case. Agios stated it is evaluating this case as part of its pharmacovigilance process.
Agios reported that, to date, the information available, including the cases referenced in the analyst report, has not altered the established benefit-risk profile of PYRUKYND as described in the U.S. Prescribing Information. The company’s financial health score from InvestingPro is rated as "GOOD," with particularly strong metrics in relative value and a current ratio of 14.48x, indicating robust short-term liquidity.
Separately, on Monday, the Saudi Food and Drug Authority approved PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
This information is based on a press release statement included in the company’s SEC filing.
In other recent news, Agios Pharmaceuticals reported its Q2 2025 earnings, which showed mixed results. The company experienced a larger-than-expected loss per share, posting an EPS of -$1.93, which did not meet the forecast of -$1.81. However, Agios Pharmaceuticals reported a notable revenue increase, reaching $12.5 million and surpassing expectations of $9.54 million. Despite the positive revenue figures, the earnings miss raised concerns among investors. There were no recent reports of mergers or acquisitions involving the company. Analysts have not provided any recent upgrades or downgrades for Agios Pharmaceuticals. These developments reflect recent activities surrounding the company’s financial performance.
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