Agios Pharmaceuticals, a Cambridge-based biotech company says that its experimental drug can help treat a rare and inherited form of anemia. The results of the mid-stage trial showed that the drug can boost hemoglobin significantly in half of the patients. Agios is one of the most valuable biotech companies with approximately $1.0 billion market cap.
The company announced in a conference at Copenhagen, Denmark, the results of the interim data for its experimental trial. There are 18 patients who participated in the Phase 2 Trial of AG-348. The results came in two days after the early stage data from AG-519, which is another potential drug for PK disorder or Pyruvate kinase deficiency. AG-519 had safety issues among some investors that led to a 24 percent stock sellof last Thursday and Friday. This has resulted to a loss of half a billion dollar in the market value of the company.
The concern was raised when 16 healthy volunteers took AG-519 for two weeks. After the clinical trial, all patients ended up with low platelet count which is called thrombocytopenia. They all recover after few days of stopping the intake of the drug. David Schenkein, Agios CEO, said in an interview that the reduction in platelet count may be related to the patient’s immune response to the drug.
Seamus Fernandez, Leerink Partners noted that the AG-348 data help boost the company’s stock performance. The participants of AG-348 are those who are taking the drug anywhere from week three to twenty-four weeks. Nine out of eighteen patients observed an increase in hemoglobin count of at least one gram per deciliter.
Schenkein noted that the normal range of hemoglobin for healthy people is around 13 to 17 grams. Patients having the disease have had seven to ten grams. The average increase of hemoglobin count in nine patients is around 3.4 grams.
Agios reported that the nine patients who had an increase in hemoglobin count belong to the group of 13 patients in the trial who had a missense gene mutation that were identified during the study. The company said that there was a 69 percent success rate of the experimental trial. They also pointed out that there was no any safety issues reported in the study.
Schenkein noted that the drug work by binding to the enzyme that helps produce hemoglobin and make it work efficiently. Agios is known to produce drugs that focused on correcting cell metabolism among sick patients. The lead drugs of Algios are in the late stage trials and it is aimed for treating blood cancer. Another drug is being developed in collaboration with Celgene.
As of now, there are no drugs that are used to treat PK disorder. This disease is known to have severe impact among infants. These patients may need a lifelong blood transfusion.
Creating New Hemoglobin
Algios candidate drug aims to help patients suffering from rare forms of anemia. The good thing about this drug is that there are no known serious side effects which have become an issue with the previous experimental trial drugs. Hopefully, if this drug will be approved it will improve the lives of patients with PK disorders.