Forward-Looking Statements
To the extent that statements contained in this presentation are not descriptions of historical facts regarding
GlycoMimetics, Inc. (“GlycoMimetics,” “we,” “us,” or “our”), they are forward-looking statements reflecting
management’s current beliefs and expectations. Forward-looking statements are subject to known and unknown
risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity,
performance, or achievements to be materially different from those anticipated by such statements. You can
identify forward-looking statements by terminology such as “may,” “will,” “should,” “expects,” “plans,”
“anticipates,” “believes,” “estimates,” “predicts,” “potential,” “intends,” or “continue,” or the negative of these
terms or other comparable terminology. Forward-looking statements contained in this presentation include, but are
not limited to, statements regarding: (i) the conduct of the ongoing Phase 3 clinical trial of GMI-1070 by Pfizer Inc.
(“Pfizer”); (ii) the timing of additional clinical trials for our other drug candidates; (iii) the timing of receipt of clinical
data for our drug candidates; (iv) our expectations regarding the potential safety, efficacy, or clinical utility of our
drug candidates; (v) the size of patient populations targeted by drug candidates we or our collaborators develop
and market adoption of our potential drugs by physicians and patients; (vi) the likelihood and timing of regulatory
filings and approvals; and (vii) and our cash needs and potential royalties and milestone payments under license
and collaboration agreements.
Various factors may cause differences between our expectations and actual results, including unexpected safety
or efficacy data, unexpected side effects observed during preclinical studies or in clinical trials, lower than
expected enrollment rates in clinical trials, changes in expected or existing competition, changes in the regulatory
environment for our drug candidates, failure of our collaborators to support or advance our collaborations or drug
candidates, our need for future capital, the inability to protect our intellectual property, and the risk that we
become a party to unexpected litigation or other disputes. For a further description of the risks associated with
forward-looking statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the
Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission on March1, 2017,
as well as other reports we file with the U.S. Securities and Exchange Commission from time to time, including those
factors discussed under the caption “Risk Factors” in such filings. Forward-looking statements speak only as of the
date of this presentation, and GlycoMimetics undertakes no obligation to update or revise these statements,
except as may be required by law.
2
GlycoMimetics - Investment Opportunity
Highly differentiated clinical programs for orphan diseases
– Portfolio targets significant unmet needs with large commercial potential
Phase 3 in vaso-occlusive crisis (VOC) of sickle cell disease
– Robust Phase 2 results across all endpoints
– Phase 3 top-line results expected 2H 2018
– Partnered with Pfizer
Phase 2 in acute myeloid leukemia
– Compelling clinical outcomes and biomarker proof-of-concept from ongoing Phase 1/2 trial
– Multiple data readouts next 6-12 months
Proven management team
– Pioneers in the field of glycobiology, with broad experience in drug development
Strong Balance Sheet provides funding through multiple milestones
3
Major Announcements & Upcoming Milestones
4
Rivipansel (Phase 3)
Phase 2 data selected as “Best of ASH” ✓
Orphan Drug Designation & Fast Track Status (US) ✓
Special Protocol Assessment with FDA ✓
Phase 3 enrollment initiated ✓
Anticipate announcing Phase 3 top-line results 2H-2018
GMI-1271 (Phase 1/2)
Orphan Drug Designation (US & EU) & Fast Track Status (US) ✓
Completion of Phase 1 and selection of phase 2 dose ✓
Completion of Phase 2 enrollment in newly diagnosed AML cohort ✓
Breakthrough Therapy Designation granted by the FDA ✓
Completion of Phase 2 enrollment in R/R AML cohort ✓
Presentation of interim Phase 2 results at ASCO and EHA ✓
Define registration program in AML with regulatory agencies Q4’17 - ‘18
Anticipate announcing Phase 2 durability of response and OS Q4’17 - ‘18
GMI-1359 (Phase 1)
Initiation of Phase 1 study ✓
Anticipate announcing optimal dose and initial cancer indication Q4’17 - ‘18
A Balanced Portfolio of Product Candidates
5
Compound Therapeutic Area Discovery Pre-Clinical
Ph 1 Ph 2 Ph 3 Registration Partner
Selectins
Rivipansel(Pan-selectin Inhibitor)
Sickle Cell Anemia Vaso-occlusive Crisis Pfizer
GMI-1271 and Follow-ons(E-selectin Inhibitor)
Acute MyelogenousLeukemia
--
Multiple Myeloma --
Various Tumor Types & Inflammatory Diseases
--
GMI-1359(E-selectin & CXCR4 Inhibitor)
Various Tumor Types --
Galectins
Galectin-3Inhibitor
Fibrosis & Oncology --
Undisclosed GalectinInhibitor
Various Tumor Types --
GMI-1271: A First-in-Class Therapeutic Disrupts
Tumor Microenvironment
Small-molecule, potent, selective, E-selectin antagonist
Compelling efficacy, safety and biomarker data from Phase 1/2 AML
study - clinical proof-of-concept established
– High remission rates, low induction mortality and promising initial
survival in highly pre-treated R/R AML population
• Biomarker correlated with achievement of remission
– High remission rates and low induction mortality in newly
diagnosed, elderly, AML patients
– Benign safety profile with observed reductions in chemotherapy-associated toxicities (e.g. mucositis)
Preclinical rationale for use in multiple indications and combination
approaches - “proteasome-rescue” study underway in MM
IP through 2032
7
Study Design
– Dose Escalation: 3 dose levels tested (5, 10 & 20 mg/kg) in
Relapsed/refractory AML (MEC) population
• Phase 2 dose selected - 10mg/kg
– Dose Expansion:
• 25 patients with newly diagnosed AML > 60 (7&3)
• 47 patients with R/R AML (MEC)
Endpoints: Safety, PK, Biomarkers & Efficacy (ORR)
Lead Investigator: Dan DeAngelo, MD, PhD – Dana Farber Cancer Institute
– Additional centers in US, Ireland and Australia
GMI-1271 Phase I / II Study in AML
Treatment Follow-Up
BM Biopsy
Screening
-22 to 0 15 28Visit DayGMI-1271 + Chemo
GMI-1271 GMI-1271
Long-Term Follow-Up
6 Mon 12 Mon
8
Re-TreatmentPhase 2 Expansion
✓
✓
✓
High-risk, heavily pre-treated patient
population
– All patients had intermediate or
unfavorable cytogenetics
– >60% were either refractory or early
relapsed (<6 months) AML
Higher than expected CR/CRi rate and
promising initial median overall survival
– mOS 7.6 mons vs. ~5.4 mons (MEC-alone)
– Baseline expression of E-selectin ligand is
predictive of remission
– For responders in Phase 1, median
survival not reached at 12+ months
GMI-1271 + MEC well tolerated
– No DLTs observed - AE profile consistent
with MEC alone
Signs of protective effects - oral mucositis
/stomatitis occurred at lower rates and
intensity than expected for MEC (2% incidence
Grade 3/4 vs. 25% for historical MEC-alone)
9
Key Phase I / II Interim Results
in R/R AML Population
Clinical proof-of-concept established
Complete Remission Rate
‡ Response criteria per Cheson, 2003; Dohner 2010.Feldman J Clin Oncol 2005 Jun 20:23 (18): 4110-6.
Percent of Patients
CR + CRi
28
41
0 20 40 60
MEC(Feldman)
GMI-1271
Elderly, high-risk patient population
– 50% had sAML; >92% had
intermediate - unfavorable
cytogenetics
GMI-1271 + 7&3 well tolerated
– AE profile consistent with 7&3 alone -
no obvious incremental toxicity
– 0% grade 3-4 mucositis
E-selectin ligand broadly expressed at
baseline in majority of patients - target
clinically relevant
Efficacy data compares favorably to well-
established historical controls (7&3 or
Vyxeos)
– Responses appear durable with100%
of responding patients remaining in
remission at 6 months
10
Key Phase II Interim Results
in Frontline AML Population
Promising Complete Remission Rates in High-Risk, Elderly AML Patients
Percent of Patients
Complete Remission Rate
‡ Response criteria per Cheson, 2003; Dohner 2010.Foran et al. Blood 2015 126:217.Lancet et al. ASCO Annual Meeting 2016.
48
63
44
68
0 20 40 60 80
Vyxeos (Phase 3)
GMI-1271 sAML
(10mg/kg)
7 & 3 (Foran)
GMI-1271 Newly
Diagnosed (10mg/kg)
CR + CRi
GMI-1271-201: Overall Response by Cytogenetic
& Other Risk Factors
Remission Rates (CR/CRi) in Defined Subgroups, n (%)
AML SubgroupPhase 1 R/R
(MEC)
Phase 2 R/R
(MEC)
RP2D R/R
(MEC)
Total R/R
(MEC)
Elderly
Newly
Diagnosed
(7+3)
N Completing Induction
Period19 35 42 54 25
Favorable risk (SWOG) 0 0 0 0 1/1 (100%)
Intermediate risk (SWOG) 5/9 (56%) 6/15 (40%) 9/17 (53%) 11/24 (46%) 10/15 (67%)
Unfavorable risk (SWOG) 4/10 (40%) 7/22 (32%) 9/23 (39%) 11/32 (34%) 5/8 (63%)
FLT3-ITD mutated 1/2 (50%) 2/5 (40%) 3/7 (43%) 3/7 (43%) 1/1 (100%)
Extramedullary disease 1/1 (100%) 1/1 (100%) 2/2 (100%) 2/2 (100%) 0
11
Higher than expected remission rate maintained across all subgroups,
including those with unfavorable risk
% E
-Se
lec
tin
(H
EC
A-4
52)
Po
sitiv
e A
ML
Bla
sts
PD+PR+
MLFS+CRi
CR
PD+PR+MLFS+
CRiCR
N 17 8
Mean 22.13 48.89
Median 24.56 50.39
Min 0.86 14.87
Max 60.65 76.21
Lower 95% CI
12.57 31.11
Upper 95% CI
31.70 66.66
P=0.0039
0
20
40
60
80
100
Relapse/Refractory Cohort: Correlation of Baseline
E-Selectin Expression And Clinical Remission
12
GMI-1271: US Regulatory Update & Future Plans
Breakthrough Therapy Designation (BTD) granted “for the treatment of
adult patients with relapsed/refractory acute myeloid leukemia” on
May 17, 2017
– BTD expedites the development and review of new medicines that
• treat Serious or life-threatening disease
• demonstrate substantial improvement over existing therapy
US Fast Track status granted in June 2016
Orphan Drug Designation (USA May 2015 / Europe May 2016)
13
Potential for expedited development program and
FDA regulatory review
High ST3GAL6/low FUCA1
FISH/ISS others
E-Selectin Expression is Associated with Poorer
Clinical Outcomes in Multiple Myeloma
15
NDMM: Newly
Diagnosed
(n=33)
rrMM:
Relapsed/Refractory
(n=50)
E-Selectin Expression Overall Survival
E-selectin ligand is over-expressed in high risk MM patients and confers poor
prognosis (e.g. mOS of 10 months vs. 33 months)
A. Natoni et al. Leukemia.2017.123
16
Addition of GMI-1271 Restores Sensitivity of Bortezomib
in an E-Selectin Overexpressing Model of MM
0 20 40 60 800
50
100
Days post tumor injection
Pe
rce
nt
su
rviv
al
0 50 100 1500
50
100
Days post tumor injection
Pe
rce
nt
su
rviv
al
TreatmentMST
(days)%
survival P vs
saline
saline 33 0
GMI-1271(40 mg/kg IP qd x 21 days
31 0
Bortezomib(0.75 mg/kg IP qw x 3 wks
42 0 0.0622
GMI-1271 + Bortezomib 60b 0 0.0101
TreatmentMST
(days)%
survival P vs
saline
saline 25.5 0
GMI-1271(40 mg/kg IP qd x 21 days
30 0
Bortezomib(0.75 mg/kg IP qw x 3 wks
24 0
GMI-1271 + Bortezomib 56.5c 37.5 0.0028
aP=0.0363 vs. bortezomib
aP=0.0123 vs. bortezomib
Parent
E-Selectin
Enriched
aadditional study at lower tumor inoculum gives similar outcome
A. Natoni et al. Leukemia.2017.123
Study Design: Phase I Dose Escalation
– Patient Population
• Patients with relapsed/refractory MM continuing on
bortezomib/carfilzomib-based regimen
– 4 Dose Levels (5, 10 & 20 mg/kg) - 6 patients/Cohort
• Weekly or Bi-weekly bortezomib/carfilzomib backbone
Endpoints: Safety, PK, Biomarkers & Efficacy
Lead Investigator: Michael O’Dwyer, MD: NUI – Galway
Proof-of-Concept Study in MM Ongoing
“Proteasome Inhibitor Rescue”
Bort/Carf
Baseline
17
Screening Treatment (Up to 6 Cycles)
Weekly or Bi-Weekly Standard Regimen Bortezomib/Carfilzomib
QD dosing same time and 24 hrs following each dose of Bort/CarfGMI-1271
Follow-Up
Bone Marrow
Biopsy
Cycle 3 Cycle 6 Folllow-Up
Rivipansel Value Proposition
Significant unmet medical need
Quantifiable clinical and health-
economic endpoints
Defined regulatory path to approval
– US FDA Special Protocol
Assessment
Excellent economics to GLYC from
Pfizer
19(1) Hassell KL. (2010) Am J Prev Med.;38(4 Suppl):S512-21
(2) Brousseau D et. al. (2010) Am J Hematol. 85(1) 77-8
(3) Yusuf HR et. al. (2010) Am J Prev Med. 38(4 0)
Initial US Approval Goal
Inpatient Hospitalizations
(80-100,000 Events/Yr1,2)
Outpatient Treatment
in ER & Clinic Settings (~140,000 Events/Yr3)
Attractive commercial opportunity; substantial market expansion potential
– On-demand approach
• Avoids historical noncompliance challenges with chronic, prophylactic
therapy
– Longer term potential: Treat VOC earlier in ER/clinic
Sickle Cell Anemia Healthcare
Utilization for VOC
Rivipansel Clinical Development Highlights
Improvements observed in
every efficacy endpoint
evaluated across all
subgroups studied in Phase
2 study
– Reduction in time in to
hospital discharge by
> 3.5 days
Efficacy data supported by
improvements on
biomarkers of inflammation
and coagulation in
patients in biomarker study
Benign safety profile
Phase 2 data recognized
as “Best of ASH”
Rivipansel
Placebo
Pro
ba
bili
tyTime to Discharge (Hours)
0 50 100 150 200
1.0
0.8
0.6
0.4
0.2
0.0
Median time to discharge
reduced by 84 hours
(p=0.092; Kaplan-Meier)
Time to Discharge From Hospital
20Telen, Blood. 2015 Apr 23;125(17):2656-64
Phase 2 Results – Substantial Reduction in
Time to Resolution of VOC and Opioid Use
Ho
urly IV
Op
ioid
Use
Mean Hourly Opioid Use
0.0
0.2
0.4
0.6
0.8
1.0
Day 1 Day 2 Day 3 Day 4 Day 5 Day 6 Day 7
Rivipansel
Placebo
24 hour reduction p<0.001***
Cumulative opioid analgesic
administered reduced by 83%
(p=0.010; ANCOVA)
21
Opioid Use Over Time
Rivipansel
Placebo
Pro
ba
bili
ty
Time to Resolution (Hours)
0 50 100 150 200
1.0
0.8
0.6
0.4
0.2
0.0
Median time to resolution of VOC
reduced by 63 hours
(p=0.187; Kaplan-Meier)
Time to Resolution of VOC
Telen, Blood. 2015 Apr 23;125(17):2656-64
Tiered, ranging from low double digits to low teens
Total Milestones: up to $320.0 million
Development: up to $115.0 million
$35.0 million upon start of Phase 3
$15.0 million advance payment received in May 2014,
$20.0 million received August 2015 with dosing of the first
patient.
Regulatory: up to $70.0 million
Commercial: up to $135.0 million
$22.5 million
Pfizer responsible for all further clinical development, regulatory approval and potential commercialization for all indications worldwide
Rights
Upfront
Milestones
Royalties
Pfizer Deal Economics
22
GlycoMimetics is Positioned for Success
23
Promising Pipeline
Rivipansel: Sole “on-demand” treatment in late-stage,
registration trial for acute VOC; SPA in place with FDA
GMI-1271: Clinical outcomes and biomarker demonstrate
clinical proof-of-concept in AML; Breakthrough Therapy
Designation granted by the FDA
GMI-1359: Simultaneous blockade of CXCR4 & E-Selectin
inhibits established pathways of cancer cell trafficking
Significant Revenue
Opportunities
Rivipansel: ~100,000 patients in USA
GMI-1271: > 40,000 AML patients in 7MM
Strong Investment BaseCash balance provides runway through key milestones
Top-tier biotech investors
Experienced Team
Pioneers in the field of glycobiology and small-molecule,
therapeutic “mimetics”Relationships with leading KOLs and national / international
oncology networks