Analyst Conference Summary


conference date: March 6, 2018 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2017 (fourth quarter 2017, Q4)

Forward-looking statements

Overview: Announced plan for Phase 3 trial of GMI-1271 for AML.

Basic data (GAAP):

Revenue was $0, up sequentially from $0, and down from $0 year-earlier.

Net income was negative $9.3 million, down sequentially from negative $8.0 million, and down from negative $8.3 million year-earlier.

EPS (diluted) was negative $0.27, down sequentially from negative $0.24, but up from negative $0.36 year-earlier.



Release & Conference Highlights:

Rachel King, Chief Executive Officer, said "Highlighting the fourth quarter of 2017, GlycoMimetics presented a robust data set for its Phase 1/2 study of GMI-1271 for the treatment of AML patients. This data provided the basis for discussions with the U.S. FDA focused on a Phase 3 trial design – the result of which we announced yesterday. The ongoing discussions were made possible via our Breakthrough Therapy designation for GMI-1271 for the treatment of relapsed/refractory AML patients, and we now plan to initiate our own Phase 3 trial in this patient population later this year."

GMI-1271 has Breakthrough Therapy designation from the FDA. Positive data was presented at ASH in December, both Phase 1/2 clinical and preclinical.

Registrational study for relapsed/refractory AML plans were announced. Will start the Phase 3 trial in late 2018. Data should be available in Q4 2020. Overall survival will be the primary endpoint for the trial, and will not be censored for transplants, allowing more patients to receive transplants. Believes gives best chance of statistically significant results with a single phase 3 trial. Will used a fixed dose of GMI-1271, and multiple cycles are allowed. Patients will also get one of two standard of care chemo regimens. Mucositis will be a secondary endpoint, as will CR.

Plans to expand 1271 into the unfit-for-chemo, newly diagnosed AML and high-risk MDS setting. After the quarter ended a trial was announced to be sponsored by the Hovon group. Could begin in second half of 2018.

Hopes to announce plans for 1271 for fit for chemo, newly diagnosed AML in the near future.

A European proof-of-concept trail of 1271 in multiple myeloma continued in Europe, with topline data expected in Q1 2019.

The Phase 3 rivipansel trial for VOC (vascular occlusive crisis) of sickle cell disease remains on track for completion in the second half of 2018. There is a special protocol agreement with the FDA. It is being conducted by licensed partner Pfizer. Possible peak sales are greater than $1 billion. Could get a milestone payment in 2019.

A third therapy, GMI-1359, Phase 1 trial continued, and may be an improvement on GMI-1271 in treating bone marrow cancers.

Preclinical studies to move other candidates to the clinic continue.

Cash balance ended at $123.9 million, up sequentially from $112.9 million. Shares outstanding ended at million.

Total cost of operations was $9.5 million, consisting of $6.7 million for R&D and $2.8 million for general and administrative expense.

Shares outstanding ended at 34,359,800.


Evolution of discussion with FDA, CR v. OS? Breakthrough therapy designation allowed for ongoing dialog. They said we could use response rate, we decided overall survival would best capture the constellation of benefits and give us the best positioning at launch.

Confidence in ability to expand label? We are gathering data with the consortium in parallel. OS endpoint gives us a strong case to expand the label later with other data.

Other AML trials with this methodology? Transplant is considered a success, because you achieved remission. Improving the rate of transplant improves OS. Other studies have been done that did not censor for transplant. The ASH Phase 2 data included transplants.

Historical reference for chemo in arms? One problem with salvage in AML is that it does not work well. Survival is not good for any of them; there is not a lot of range between them. Centers have preferences for MEC or FAI, so by allowing both in the trial we can include most centers in the trial. We will capture the standard of care.

Quality secondary endpoints? There are several, but they all would contribute to overall survival. Multiple consolidation cycles could drive a deeper response and longer duration. There will be a number of secondary endpoints, including short-term mortality rates.

Inclusion criteria will be very similar to the Phase 2 trial. We are loosening the medical factors that might limit enrollment. We are stratifying by age, disease factors, etc. between treatment and control arms.

Transplant rate expectations? Hard to capture in historical data, trending up over time. We had a 24% rate in the Phase 2 data, we think that is high, based on opinion leader feedback. Very dependent on nature of population. Should capture rates for different strata.

Mucositis rates of chemo backbones? Both are substantial, expect to see substantial mucositis in the control arm.

Any plans to use the at-the-market facility? We have about $80 million left, we have not used it since Q3 sales.

Cost estimates for Phase 3 trial? $50 million conservatively, costs starting this quarter and ramping in 2019.

There is no interim analysis built into the protocol.

Any concerns about safety given the extended cycles? We have had a very good safety profile. In newly diagnosed we have had patients with three rounds of therapy. We now have the ability to add up to three cycles of consolidation in the Phase 3 study.

The preclinical data supports trying 1271 in multiple myeloma, the mechanism of action is the same as in AML. We believe 1271 has potential to be used very broadly in blood cancers.

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Disclaimer: My analyst call summaries may include both condensations of statements made by company representatives and my own analysis. They are not covered by any warranty. I cannot guarantee anything said by company representatives is true. I try not to make errors, but it is possible. These are my personal notes which I share with other investors and which I use as the basis of my blog and Seeking Alpha articles.

Copyright 2018 William P. Meyers