Sickle cell disease impacts about 100,000 Americans, according to RethinkSCD.com. The incidence is about one in every 365 African American births and about one in every 16,300 Hispanic American births. The incidence of a sickle cell trait is also said to be as frequent in many as one in 13 African American births. With major advances in sickle cell disease screening and interventions over the past four decades, life expectancy has increased, but that life expectancy is still over 30 years shorter than that of the general population.
One relatively small clinical-stage biopharmaceutical player called Global Blood Therapeutics Inc. (NASDAQ: GBT) may be about to change how sickle cell disease treatment goes. The company’s lead product candidate is voxelotor (GBT440), which is an oral once-daily therapy for sickle cell disease that is currently in a Phase 3 clinical trial in adult and adolescent patients with sickle cell.
According to Wedbush Securities, the key near-term catalyst for GBT440 is 24-week safety and efficacy results from the Phase 3 HOPE study at the 24th European Hematology Association Congress taking place from June 13 to June 16 in Amsterdam.
The abstract is embargoed until the presentation date of June 14 and time. Wedbush’s Liana Moussatos is looking for a substantial improvement in hemolytic anemia efficacy, fewer “VOC” events, as well as a favorable safety and tolerability profile. Her take is that a new drug application filing will be coming in the second half of 2019, and she thinks there is the potential for close to $1 billion in annual sales — hence the blockbuster drug status.
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Moussatos said in her report:
In our view, preliminary HOPE Phase 3 results (n=154) presented at ASH 2018 substantially de-risked voxelotor’s clinical risk and we anticipate EHA results to be supportive. We understand that the FDA and the American Society of Hematology (ASH) cohosted a workshop in October 2018 supporting hemoglobin as an appropriate endpoint to show potential for clinical benefit. Global Blood guided to NDA submission under accelerated approval in H2:19. We project potential achievement of almost $1 billion in gross worldwide sales in 2023 after our estimated U.S. launch in August 2020. We recommend buying shares of GBT in front of key catalysts in 2019-2020.
While new sickle cell treatments are needed to treat the population, Global Blood Therapeutics is a potential double-your-money stock pick if Moussatos is correct. Her Outperform rating comes with a $107 price target, and that compares with a $55.81 closing price on June 11.
Refinitiv consensus analyst data shows no projected revenues in 2019 and only $87 million (from a rather wide range) in revenues for 2020. Through the end of 2018, the company had accumulated a deficit of −$472 million in accumulated losses over its existence.
Global Blood Therapeutics shares closed down 2.5% at $55.81 on Tuesday, in a 52-week range of $30.15 to $63.00. Its market cap is about $3.1 billion, and the dozen or so analysts who cover the stock have target prices as low as $59 and as high as $150.
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