Agios Pharmaceuticals ($AGIO) had hoped to showcase some early data on one of its rare disease drugs over the weekend, looking to spotlight some helpful signs of biomarker activity at a meeting of the European Hematology Association (EHA). Instead, the poster was released early, at least one analyst quickly zeroed in on an adverse event experienced by one of the patients in the Phase I trial, and the stumble wound up costing the company a few hundred million in market cap as its stock tumbled 15%.
The message that Cambridge, MA-based Agios was focused on at the end of this week centered on AG-519, a second pyruvate kinase-R (PKR) activator which is designed to correct pyruvate kinase (PK) deficiency, which causes a rare, congenital anemia. It hit its marks among healthy volunteers, an early proof-of-mechanism snapshot which the company thought might offer some encouragement to investors.
Instead, a note on a single case of grade 2 thrombocytopenia--a low blood platelet count--was flagged.
Alarm bells rang, the stock dropped, even though the company noted that the patient’s platelet levels recovered 7 days after the last dose.
“(O)ur conversation with management suggests that this subject likely had a rare case of drug-induced immune thrombocytopenia, which would be unrelated to AG-519's mechanism of action, and the company does not expect to see additional cases,” noted a helpful analyst at Sun Trust. But in this market, bad news travels faster than an explanation.
Agios also has another PKR activator in Phase II, the more advanced AG-348, to protect against the destruction of red blood cells.
Agios can’t seem to catch a break on the PR front. The biotech announced that it grabbed $200 million upfront when it restructured its pact with Celgene ($CELG), but investors zeroed in on the bigger company’s decision to abandon one of the drugs in its collaborations in the process. Since then, though, its stock shot up ahead of the EHA meeting--until this latest snafu.
- here's the release