Monday morning Global Blood Therapeutics (GBT) announced the FDA has agreed to GBT’s plan to file for accelerated approval of voxelotor, a unique therapy created to treat sickle cell disease (SCD). Additionally, the FDA agreed to the use of transcranial Doppler (sonography) for a post-approval study that reflects stroke risk reduction for SCD patients. Share prices for the stock have risen nearly 48% since the markets closed on Monday, following the news. Cantor’s Elemer Piros says the FDA response confirms his belief in GBT, as voxelotor could be a meaningful benefit for patients in the fundamental progression of SCD. His positivity for the company’s future success leads him to reiterate an Overweight rating and price target of $96 for GBT stock, showing an upside of 106%. (To watch Piros’ track record, click here)
In addition to FDA approval, Piros cites GBT presentations at the American Society of Hematology, an event held the first four days of December and ends today. The event boasted a positive 24-week update from the GBT’s HOPE Part A study in 154 patients. After 24 weeks of treatment with 1,500 mg of voxelotor per day, 65% of patients showed a 1g/dL improvement, compared with 10% of placebo-treated patients (p<0.0001). The improvements were observed with and without hydroxyurea usage and coincided with a decrease in markers of hemolysis.
“[…] we believe this study is well positioned for the assessment on the full cohort of patients,” Piros said. “Several key measures support our view that the observed hemoglobin increase is indicative of substantial clinical benefit and safety. While the baseline characteristics of enrolled patients highlighted a low number of historical vaso-occlusive crises (VOCs, 40-44% experienced only 1 VOC in the past year), voxelotor treatment led to 109 VOCs and 113 VOCs for 1,500mg and 900mg dose cohorts, compared to placebo’s 131 events. We believe maintaining (or potentially improving) this profile will be important for investigators as we think about a commercialization of voxelotor following subpart H approval. We are encouraged by the results of erythropoietin which remained stable or decreased, suggestive of maintained or improved tissue oxygenation, a theoretical concern of hemoglobin modifier therapies.”
GBT is planning a pre-NDA meeting with the FDA in the first quarter of the year. After this, GBT plans to file its package in 2H19. The data is set to include a six-month follow-up for the last enrolled patient of the study. Some early talks of commercialization revealed the sales force for GBT will likely include 75 or less representatives.
With talks of rapid FDA approval and the imagery of voxelotor sales reps pushing the product, it makes sense that Wall Street would consider GBT to be a Strong Buy. TipRanks finds out of 10 analysts who have reviewed GBT stock in the last three months, 9 are bullish and only one is sidelined. With a consensus price target of $90.88, there’s a 95% upside from where the stock is currently trading. (See GBT’s price targets and analyst ratings on TipRanks)