Analyst Conference Summary

conference date: August 2, 2023 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2023 (second quarter 2023, Q2)

Forward-looking statements

Overview: Waiting for final uproleselan AML results in 2024.

Basic data (GAAP):

Revenue was $0 million, flat sequentially from $0.0 million, and flat from $0 year-earlier.

Net income was negative $8.2 million, up sequentially from negative $10.4 million, and up from negative $13.4 million year-earlier.

EPS (diluted) was negative $0.13, up sequentially from negative $0.17, and up from negative $0.25 year-earlier.

Guidance:

Cash runway to the end of 2024.

Conference Highlights:

Harout Semerjian, Chief Executive Officer, said "With the addition of a time-based analysis option to our pivotal Phase 3 study, we look forward to reporting topline uproleselan results in R/R AML by the end of Q2 2024. This readout will draw from a clinically mature dataset with more than three years of median follow-up and can potentially bring us closer to delivering this therapy to patients in need of new treatment options. We continue to execute our broad development strategy for uproleselan while we explore potential benefit in AML patients of all ages as evidenced by the FDA agreement on the pediatric study plan as well as the NCI sponsored, and investigator initiated pediatric studies. In addition to these advances, we are excited to expand our clinical pipeline and move GMI-1687, a novel, highly potent E-selectin antagonist into first-in-human studies. We look forward to initiating our Phase 1a study in the coming weeks."

The Uproleselan (GMI-1271) Phase 3 trial for relapsed/refractory AML completed enrollment in November 2021. Overall survival (OS) will be the primary endpoint for the trial, and will not be censored for transplants, allowing more patients to receive transplants. Mucositis will be a secondary endpoint, as will CR. The trial was event-driven, but the FDA allowed it to be amended to a time-based analsyis, so currently top line results should be in Q2 2024. In Q2 2023 the FDA agreed to an initial Pediatric Study Plan, and the NCI (National Cancer Institute)to sponsor the pediatric Phase 1/2 study in AML. [Taking a long time to reach the OS reporting point is a good thing - WM]

In June, 2023, the first pediatric patient was treated with uproleselan in an investigator-initiated single arm, multi-center Phase 1/2 study to assess safety and tolerability, as well as determine a recommended phase 2 dose (RP2D) of uproleselan plus myeloablative, busulfan-based, pre-transplant conditioning for treatment of AML. This study, led by John Horan, MD, MPH, of the Boston Children’s Hospital and Dana Farber Cancer Institute, will enroll up to 28 patients (Age =12 months and = 30 years) and will also assess preliminary uproleselan efficacy at the RP2D.

GlycoMimetics had asked, in 2022, to add an interim futility analysis, conducted by an Independent Data Monitoring Committee, to the AML trial. The FDA agreed. In February 2023 the results were that the trial was to continue as planned. That was at 80% of events (deaths). The analysis utilized a very high statistical threshold to preserve the statistical integrity of the originally planned final overall survival analysis. The overall survival events trigger is now expected to occur in the first half of 2024. A deeper explanation of the high statistical threshold was presented on the call. It did show better results than the Phase 1 trial. Patients continue to live longer than expected.

Uproleselan market opportunity is based on its novel mechanism of action. 20,000 AML diagnoses annually in the U.S., with incidence increasing. 29% current 5-year survival rate. Current OS for our target R/R population is just 6 months. Remarked on potential market size.

GlycoMimetics is gathering data that would help in preparation for commercialization of uproleselan. Uproleselan drug product registration batches were completed in Q3 2021.

At ASH (in December 2022) initial data was presented, in posters, from the investigator-sponsored trials. In addition to its own registrational trial, GlycoMimetics is collaborating with both the NCI and the Alliance for Clinical Trials in Oncology conducting a randomized, controlled clinical trial testing the addition of uproleselan (GMI-1271) to a standard cytarabine/daunorubicin regimen (7&3) in older adults with previously untreated AML who are eligible for intensive chemotherapy. Primary endpoint will be overall survival. Could be used for applciation to FDA for its patient population. The Phase 2 part of the trial completed enrollment in November 2021. EFS is the endpoint. An interim analysis is planned before proceeding to Phase 3 enrollment. In Q2 2021 three additional investigator-sponsored trials were initiated for AML, bone marrow transplantation, and multiple myeloma.

The Phase 3 uproleselan trial in China is planned to initiate soon. Other investigator-sponsored trials are underway or planned that could lead to label expansion.

GMI-1687 shows potential superiority for VOC in sickle cell disease to rivipansel. Preclinical data was presented at ASCAT in September 2020. A Phase 1 trial IND submission was cleared by the FDA in June 2022, with first patient start planned for Q3 2023. Currently there is no approved therapy for accute VOC. Looking for a partner.

GMI-1359 showed evidence of biologic activity in the initial patients treated in a Phase 1b proof-of-concept trial of various tumors (breast cancer) in December 2021. The analyses, based on pharmacodynamic biomarkers, were reported at AACR in April 2021, showing it hit the targets. GlycoMimetics is evaluating further clincial developement and strategic options.

A galactin 3 antagonist GMI-2093 is preparing to move towards development. Preclinical data was presented at AACR in 2021.

GlycoMimetics is looking at possible collaboration deals for 1687, 1359, and the galactin antagonist.

Cash balance ended at $58 million, down sequentially from $65 million.

Total cost of operations was $8.9 million, consisting of $4.1 million for R&D and $4.9 million for general and administrative expense. Loss from operations was $8.9 million. Other income was $0.7 million.

Q&A summary:

NCI trial, will it be a time-based analysis? Still on original trial plan, have not reached their number of events. Its Phase 2 analysis will be based on event-free survival. It finished enrolling in 2021, so it has taken a long time to mature. We do not anticipate any design changes.

Pediatric after NCI Phase 1/2? No decision yet.

Any additional analysis from the interim data? Patients continue to live longer, we only see pooled blinded data. Next year will be 3 year medium follow up, so data should be mature. We do not know which arm of patients are living longer.

1687 timeline, partner? We are excited to start the trial. It is a healthy volunteer study for safety and PK signals. It should not take too long, maybe results next year. We are open to patnering, but we are able to fund it ourselves.

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