Global Blood Therapeutics Inc (NASDAQ:GBT) was another big winner at ASH. The drug in the spotlight is Voxelotor, an oral, once-daily therapy for patients with Sickle Cell Disease that works by increasing hemoglobin’s affinity for oxygen. Our sense right now is that the “powers that be” are looking for strong candidates to beat SCD, and Vox is a striking candidate to take the lead.

At ASH, the company presented surprisingly compelling data. And, in a rare simultaneous announcement, the FDA informed GBT the same day through discussions and written correspondence that the agency is in favor of an accelerated approval pathway.

Global Blood Therapeutics Inc (NASDAQ:GBT) shares ramped nearly 50% in a day on the double dose of good news. This was perhaps the most dramatic reaction we saw to any stock presenting at ASH in 2018.

We noted above that the company presented “surprisingly compelling data”. The surprising part is that the company is zeroing in on meeting an endpoint for SCD, which has become a sort of bogeyman in the biotech space. It’s a nut no one seems to be able to crack with the FDA.

Basically, the drug works by inflating the sickling cells to prevent them from taking the familiar sickle shape.

Data on this drug was first reported in June, and it showed that 58% of Part A Vox patients who received a 1,500 mg daily dose saw an increase of 1g/dl in their hemoglobin by the 12th week. That compared with only 9% for the placebo arm.

Now, if we fast forward to ASH, the same data came in at 65% versus 10%. So, it’s clearly a stable phenomenon versus placebo, and it is a significant margin of efficacy.

The question is whether or not this will translate into an actual benefit for patients who are likely more concerned with shortened lifespans and regular discomfort. Hemoglobin count is likely tied in, but it isn’t a guarantee.

In any case, the FDA is looking for a promising new direction on SCD and GBT might have it.

At this point, the stock is hovering around the 200-day simple moving average in the $43/share zone. This is up from the $31 area where the stock was into the conference.

One can assume there was some “dead money” hanging out in the name that saw this as a chance to get out. But plenty of new money has been flowing in, and we would assume analysts will start to leak out positive sell-side views in coming weeks as Vox gets put in the fast lane toward some sort of approval.

 

Upper Deck Binoculars

Global Blood Therapeutics Inc (NASDAQ:GBT) bills itself as a clinical-stage biopharmaceutical company that engages in the discovery, development, and delivery of treatments for underserved patient communities.

It is developing its lead product candidate, voxelotor, an oral, once-daily therapy for sickle cell disease (SCD). The company is evaluating voxelotor in SCD in a Phase III clinical trial in adult and adolescent patients with SCD. It is also evaluating the safety and pharmacokinetics of single and multiple doses of voxelotor in a Phase IIa clinical trial of adolescent and pediatric patients with SCD.

Global Blood Therapeutics, Inc. was founded in 2011 and is headquartered in South San Francisco, California.

According to company materials, “GBT is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. GBT is developing two therapies for the potential treatment of sickle cell disease, including its late-stage product candidate, voxelotor, as an oral, once-daily therapy.”

Global Blood Therapeutics Inc (NASDAQ:GBT) had no reported sales in its last quarterly financial data. In addition, the company has a strong balance sheet, with cash levels far exceeding current liabilities ($383.2M against $26.9M).

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