Updated results from a trial evaluating GMI-1271 in relapsed or refractory acute myeloid leukemia (AML) sent GlycoMimetics (NASDAQ:GLYC) shares soaring earlier this week, but more good news next year could send shares even higher next year.

The company plans to begin a phase 3 study of GMI-1271 by the middle of 2018, and in the second half of 2018, data is expected from a phase 3 study evaluating GlycoMimetics and Pfizer's (NYSE:PFE) sickle-cell disease drug, rivipansel.

The potential to improve AML treatment and improve the lives of sickle-cell disease patients could make this company an intriguing takeover target, so let's learn more about GlycoMimetics' story.

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Subtraction by addition

Usually, when adding a new drug to existing standard of care increases efficacy, it also increases safety risks. That doesn't appear to be the case with GMI-1271. In phase 2 studies, tacking GMI-1271 on to the standard of care chemotherapy not only improved remission rates, but it also lowered the risk of severe mucositis.

Specifically, the clinical remission rate among 54 relapsing or refractory patients was 43%. That beat the 28% for the historically matched control group receiving 7+3 chemotherapy. GMI-1271 also improved median overall survival to 9.4 months from the historical median overall survival of 5.4 months.

GMI-1271 also worked in older, newly diagnosed patients with AML. In those patients, adding GMI-1271 to the commonly used MEC chemotherapy cocktail resulted in a clinical remission rate of 68%. The rate historically associated with MEC in this patient population is 44%. 

GMI-1271 may also improve patients' tolerability of chemotherapy. For instance, about one-quarter of MEC chemotherapy patients experience grade 3 or 4 mucositis. However, the rate fell to below 4% when GMI-1271 was included in the cocktail.

Improving outcomes in sickle-cell disease

In 2011, Pfizer licensed the rights to GlycoMimetics' vaso-occlusive crisis (VOC) sickle-cell disease drug, rivipansel. Soon, it will find out if that was a smart decision. 

VOC causes about 100,000 hospitalizations per year, and currently there aren't any FDA-approved treatments for it. Typically, patients are provided supportive care during their hospital stay, including narcotics that reduce the significant pain associated with VOC.

Rivipansel is particularly interesting because it reduced the time to discharge from a hospital by 84 hours in phase 2 studies. In addition, rivipansel patients used 83% fewer narcotics than non-rivipansel patients. Given that reducing hospitalization costs is important to payers and patients and reducing narcotics use helps combat opioid addiction, rivipansel could prove to be an important option for these patients. As a result, Pfizer lists rivipansel as one of 15 billion-dollar blockbuster drugs that it has in its pipeline that could win FDA approval by 2020. 

Phase 3 studies are expected in the second half of 2018, and if that data leads to an FDA OK, GlycoMimetics can receive milestone payments and royalties on rivipansel sales. Management's mum on how much it will get in milestone payments, but it says it can get payouts if the FDA and EU accept Pfizer's new drug application and that it will get another payment upon the first commercial sale in both markets. As for royalties, Pfizer will pay GlycoMimetics a low-double-digit percentage to low teens percentage of sales. Based on Pfizer's blockbuster projections, rivipansel could be a nine-figure cash cow for GlycoMimetics.

Why it should be on your radar

Because the majority of clinical trials fail, there's a lot that can still go wrong for GlycoMimetics. If things go right, however, an argument could be made that an acquirer will come knocking.

Last year, Jazz Pharmaceuticals paid $30.25 per share in cash, or about $1.5 billion, to acquire Celator Pharmaceticals after Celator's AML drug Vyxeos improved overall survival by 3.6 months in pivotal trials. According to GlycoMimetics' corporate slide deck, Vyxeos delivered a complete remission rate in newly diagnosed AML of 48%. It's bad science to compare results from separate trials, but an argument could be made that GMI-1271's performance in that patient group in phase 2 matches up favorably to Vyxeos.

GlycoMimetics plans to update investors on its phase 3 trial design in relapsing or refractory AML early next year, then it plans to begin that trial by mid-year. The company's fielding offers from outsiders that are interested in conducting combination studies of GMI-1271 in newly diagnosed patients and it hopes to update investors on those discussions early in 2018, too. Based on the flurry of news that's expected, 2018 could be a very interesting year for this company and its investors.

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.