GlycoMimetics Inc. (Rockville MD) soared Thursday (May 18, 2017), up 99% to $10.67.GlycoMimetics Inc. exploded on news its drug GMI-1271 received Breakthrough Therapy by @US_FDA Click To Tweet
The unforeseen blastoff followed Tuesday (May 16, 2017) night’s announcement that the company’s clinical-stage acute myeloid leukemia (AML) drug, GMI-1271, has been designated as a Breakthrough Therapy by the US Food and Drug Administration. The agency had already given the product Orphan Drug Designation, as well as Fast Track Status for its use as an AML therapy.
Giving a product Breakthrough status helps the drugmaker speed up the development process–as well as the FDA review period, notes Economic Calendar. The designation is bestowed when preliminary clinical data show that the treatment may be a considerable improvement over others that are already on the market. The goal is to get the critically-needed product to patients as soon as possible.
The drug, which is an E-selectin antagonist (E-selectin is a cell adhesion molecule that plays a crucial role in inflammatory processes and cancer), is currently being assessed in the Phase 2 portion of a Phase 1/2 clinical study in AML patients. The program is evaluating the effects of GMI-1271 in patients who have relapsed or refractory AML and patients over the age of 60 who have been newly diagnosed.
GlycoMimetics also furnished additional updates on how the Phase 2 trial is proceeding. So far, patients who were given GMI-1271 in combination with chemotherapy have reached higher remission rates than previously anticipated, as well as lower-than-expected 30-day and 60-day mortality rates–which were 0% and 7%, respectively. Overall, the median Overall Survival (OS) was 7.6 months.
“It is particularly noteworthy to see in the relapsed/refractory cohort that patients who have higher levels of the E-selectin ligand biomarker on their leukemic blasts appear to be more likely to achieve remission of their disease,” said the company’s Chief Medical Officer, Helen Thackray, MD. “This observation builds directly on what we and others have reported in the preclinical and clinical settings about the key role E-selectin plays in many forms of cancer, including AML.”
In March, GlycoMimetics announced that it had finished enrolling one of two patient cohorts in the Phase 2 trial. Last month, the company told shareholders that it would provide additional data updates on both patient populations when it presents at this year’s American Society of Clinical Oncology (ASCO) annual meeting next month.
According to Dr. Thackray, the results from the Phase 1/2 program have consistently demonstrated good tolerability and increased remission rates. The data announced Wednesday have already been submitted to the FDA.
Break out the sunglasses. Another rocket just blasted off and is heading skyward. Is it too late to tag along for the ride?
Late last year GlycoMimetics showed up at annual meeting of the American Hematology Society to report out an early picture of some positive outcomes from the first stage of a Phase 1/2 study of GMI-1271, its E-selectin antagonist, for a small group of patients with acute myeloid leukemia.
And with another batch of data being readied for ASCO in a couple of weeks, the biotech says that the FDA’s breakthrough designation will help it accelerate the development work into a formal marketing application.
Currently in the Phase 2 portion of the study, investigators for the company have been recruiting two groups of AML patients: Treatment resistant subjects and newly diagnosed patients over the age of 60. The drug is designed to target pathways tumors use to defend against therapies, heightening the effect of chemo, says EndPoints.
Last December investigators noted that for 33 study participants with relapsed or refractory disease in one arm of the trial, the complete response rate was 45%. For 11 newly diagnosed study participants 60 or more years of age in the second arm of the trial, the CR rate was 73%.
Dan Cohen and Scott Matusow at Seeking Alpha write that identifying clinical-stage biotech with a legitimate chance at platform success is extremely hard. In the past, the majority of them ultimately failed. However, these days the conditions are changing for the right type of players.
They see immune-therapy as the primary area in which to prospect for clinical-stage biotech, “notwithstanding the importance of bio-markering in this segment, and in relation to new FDA trends.”
Their latest choice is GlycoMimetics. Why? Because its GMI-1271 is a novel E-selectin (CD62E) antagonist built on the company’s proprietary glycomimetic platform and is designed to block interactions between tumor cells and endothelial cells.
That means only GlycoMimetics produces the med.
Curious about the company’s name? The term “glycomimetics” is a reference to a class of synthetic molecules which mimic specific carbohydrate structures. The company has designed these molecules to have an enhanced pharmacokinetic/pharmacodynamic (PK/PD) profile enabling them to behave more as traditional therapeutics–a potentially compelling alternative to antibody therapy.
And again, GMI-1271 belongs (for the moment) to GlycoMimetics’ portfolio of proprietary medicines. There are no co-owners.
What do the analysts think?
As of May 12, 2017, the consensus forecast among 5 polled investment analysts covering GlycoMimetics advises investors to Buy equity in the company.
The five firms are Cowen & Co., Canaccord Genuity, SunTrust Banks, Jefferies Group and Stiefel Nicolaus.
1 YEAR CHANGE +64.15%
Share price forecast
The 4 analysts offering 12 month price targets for GlycoMimetics have a median target of $12.50, with a high estimate of $16.00 and a low estimate of $9.00. The median estimate represents a 17% increase from the last price of $10.67.Steve's Take: GlycoMimetics Inc. smacks of more upside in the short- to mid-term Click To Tweet
Although a strong yet speculative long-term play, I agree that this tongue-twister name smacks of more upside than down in the short- to mid-term.