Bluebird, Agios: Preview of Next Week's Important Pipeline Updates

NEW YORK (TheStreet) -- Two members of the Biotech IPO Class of 2013 -- Bluebird Bio (BLUE) and Agios Pharmaceuticals (AGIO) -- will deliver important pipeline updates at the European Hematology Association (EHA) Congress on Sat. June 14. Let's preview both presentations, starting with Bluebird and its LentiGlobin gene therapy for beta-thalassemia.

Lenti what for beta who?

LentiGlobin is gene therapy, meaning it insertsa fully functional gene for human beta-globin into the patient's own hematopoietic stem cells. The theory behind gene therapy is relatively simple: For diseases caused by single, malfunctioning or missing gene, effective treatment or even a complete cure can be achieved (theoretically) by replacing the defective gene with one that is fully functional. Gene therapy sounds simple but the execution is obviously a lot more complicated.

I won't get into the weeds explaining the process by which BlueBird produces and delivers LentiGlobin except to say a working gene for human beta-globin would allows beta-thalassemia (B-Thal) patients to produce functional beta-globin -- the largest component of hemoglobin, which carries oxygen in red blood cells. B-Thal patients suffer from anemia and iron overload. There is no currently approved cure or effective treatment, so patients require regular blood transfusions to combat the anemia.

Bluebird went public last year partly on the back of some encouraging proof of concept data showing a first-generation gene therapy approach was feasible for B-thal patients. LentiGlobin is a second-generation gene therapy designed to be more potent -- and hopefully more effective.

Next Saturday, researchers will present data on two B-thal patients treated with LentiGlobin in an ongoing phase I/II study. Key data to look for include the engraftment success rate i.e. how many "gene-therapy treated" stem cells are populating the bone marrow; improvements in hemoglobin levels; achievement of transfusion independence; and if so, how quickly from the gene therapy.

Agios will be presenting a clinical update from an early-stage study of experimental cancer metabolism drugAG-221 last reported at the American Association of Cancer Research annual meeting in April.

Agios is developing AG-221 under a collaboration with Celgene (CELG). The companies are also working together on AG-120, a similar drug designed to block another mutated cancer-growing protein known a IDH1.

At the EHA meeting on Sat. June 14, researchers will be presented updated study results from additional patients treated with AG-221, some given higher doses of the drug.

In April at AACR, six of 10 patients with advanced, treatment-refractory acute myeloid leukemia carrying the IDH2 mutation had objective tumor responses, including three complete remissions and two complete remissions with incomplete platelet recovery. A single patient achieved a partial response.

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Bluebird, Agios: Preview of Next Week's Important Pipeline Updates