Agios has cleared the first mid-stage hurdle for AG-348, its wholly owned drug for a rare inherited form of anemia called pyruvate kinase (PK) deficiency. Investigators managing the study said they captured a clear picture of an increase in hemoglobin, tracking a significant rise in half of the 18 patients currently evaluable.
The Mayo Clinic notes that hemoglobin levels--the protein that carries oxygen--that fall below 13.5 grams per deciliter among men are typically considered low, for example. In this study, 9 of the 18 patients taking the PK activator saw increases of 2.3 to 4.9 grams per deciliter, for a mean average of 3.4.
Historically, says Agios ($AGIO) CEO David Schenkein, patients being treated for low hemoglobin with a transfusion or some other therapy are looking for increases of 1 to 2 grams per deciliter.
“This is the first disease altering therapy (for this indication) ever,” says Schenkein from the European Hematology Association meeting in Copenhagen.
There are some 200 missense mutations linked to this condition, covering some 80% of all patients, and researchers noted that 9 of 13 of patients with mutations responded to the drug, further enhancing its clinical profile.
AG-348 is the first of two PK drugs that Agios is pursuing on its own. AG-519 hit its marks in an early study among healthy volunteers, but a single case of thrombocytopenia--flagged by analysts following an early release of its poster--spooked investors, sending the biotech’s shares down by 15% a couple of days ago.
Schenkein says he was surprised by that reaction, noting that the case was likely drug-induced but not mechanism-based and could be easily monitored and dealt with in the study. The next big step in this program will be picking one of the two drugs that looks best to put into a pivotal trial.
“We are designing the pivotal trial now,” adds the CEO, noting that there’s still no timeline on getting it to regulators.
Shares of Agios were up 9% in early trading on Monday, a lukewarm response to the bullish presentation over the weekend. Not all analysts were impressed by the results, with some questions circulating about the durability of the response and potential for safety issues to arise as investigators gather more data. Any preliminary results from a mid-stage study can only offer a snapshot of a company's progress, but it's one way to feed investors' appetite for catalysts.
Agios has a major collaboration underway with Celgene ($CELG), but the PK program is being kept in house as the biotech plots a course to the market with a product that will be entirely its own.
Schenkein likes to note that he founded the company with nothing but a blank sheet of paper. Now he’s well into the fourth chapter, with some major story lines coming to a head soon.
- here's the release