GlycoMimetics Announces Positive Initial Safety and Pharmacokinetic Results from Phase 1a Healthy Volunteer Study of GMI-1687

Rhea-AI Summary

GlycoMimetics, Inc. (GLYC) announces positive initial safety, tolerability, and pharmacokinetic results from a Phase 1a study of GMI-1687, a potential patient-controlled point-of-care treatment for inflammatory diseases, with a focus on sickle cell disease (SCD). The study met its primary and secondary endpoints, with no dose-limiting toxicities or safety signals observed. Subcutaneous dosing achieved target plasma concentrations and linear pharmacokinetics across all dosing levels. Full study results are expected to be presented at an upcoming medical meeting.

Insights

Medical Research Analyst

The completion of a Phase 1a study for GMI-1687 marks a significant step forward in the development of treatments for inflammatory diseases, particularly sickle cell disease (SCD). The absence of dose-limiting toxicities and safety signals indicates a favorable safety profile for the drug, which is crucial at this early stage of clinical development. The confirmation of linear pharmacokinetics suggests that the drug's behavior in the body is predictable, which is advantageous for dosing considerations in later-stage trials.

From a medical research perspective, the drug's rapid renal clearance is noteworthy as it implies that the drug may not accumulate in the body, potentially reducing the risk of long-term toxicity. This characteristic could differentiate GMI-1687 from other treatments on the market, making it a compelling candidate for further investigation. The focus on patient-controlled point-of-care treatment reflects a trend in personalized medicine, aiming to improve patient autonomy and outcomes.

Healthcare Industry Analyst

For investors and stakeholders in the biotechnology sector, GlycoMimetics' announcement is an indicator of potential future growth, especially given the unmet medical need in SCD treatment. The emphasis on GMI-1687 as a patient-controlled treatment may resonate with healthcare providers and patients seeking more agency in managing chronic conditions. The advancement of this drug could position GlycoMimetics favorably in the niche market of SCD therapies.

It is also important to note that the successful completion of the Phase 1a trial may facilitate partnership and financing opportunities, which are crucial for the continuation of the drug's development. These partnerships could provide the necessary capital and strategic support to navigate through subsequent trial phases, regulatory hurdles and eventual commercialization. The biotech industry is highly competitive and GlycoMimetics' progress with GMI-1687 could attract attention from larger pharmaceutical companies interested in expanding their portfolios in the inflammatory disease space.

Financial Analyst

The positive outcome of the Phase 1a trial could have immediate implications for GlycoMimetics' stock valuation. Typically, announcements of successful trial results lead to increased investor confidence and can result in a short-term uptick in stock prices. However, long-term value will depend on the drug's continued success in clinical development, regulatory approval and market acceptance.

Given the high costs associated with drug development, the company's ability to secure financing and partnerships will be critical to sustaining its operations and funding future trials. Investors should monitor GlycoMimetics' subsequent announcements for indications of such partnerships, as well as for the detailed results of the Phase 1a study to be presented at the upcoming medical meeting. These results will provide deeper insights into the drug's efficacy and safety, which are key determinants of its commercial viability and, by extension, the company's financial health.

• First in human trial evaluating highly potent E-selectin antagonist, GMI-1687, met its primary and secondary endpoints with no dose-limiting toxicities or safety signals
• Single ascending dose study confirmed that subcutaneous dosing generated linear pharmacokinetics and achieved target plasma concentrations across all dosing levels
• GMI-1687 is being developed as a potential patient-controlled point-of-care treatment for inflammatory diseases, with initial focus on sickle cell disease (SCD)
• Data analysis is ongoing, with full study results to be presented at an upcoming medical meeting

ROCKVILLE, Md.--(BUSINESS WIRE)-- GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, today announced positive initial safety, tolerability, and pharmacokinetic results from a Phase 1a study of GMI-1687 in healthy volunteers.

“These positive results represent an important milestone in the development of GMI-1687 as a potential point-of-care treatment option intended to help people living with sickle cell disease when they need it most, at the onset of pain crises,” said Harout Semerjian, Chief Executive Officer of GlycoMimetics. “Our Phase 1a data confirm this highly potent, second-generation E-selectin antagonist has an excellent profile for further development, with no dose-limiting toxicities or safety signals observed along with linear pharmacokinetics. We look forward to completing analysis of the study and advancing partner and financing discussions that support further development of this potentially important new therapeutic option for SCD.”

This double-blind, single-center, randomized, placebo-controlled, sequential, single ascending dose Phase 1a trial in healthy adult volunteers enrolled 40 subjects. Eligible subjects received a single subcutaneous injection of GMI-1687 or placebo (6:2 ratio). Five dose levels were evaluated, including 3.3, 10, 20, 40, and 80 mg. The study met its primary and secondary endpoints of safety/tolerability and pharmacokinetics. There were no observed dose limiting toxicities or safety signals. Subcutaneous dosing achieved target therapeutic plasma concentration and linear pharmacokinetics with rapid renal clearance across all dosing levels. Analysis of data is ongoing with full results expected to be presented at an upcoming medical conference.

About Sickle Cell Disease

SCD is the most common inherited blood disorder in the United States, afflicting approximately 100,000 people. Worldwide, about 100 million people carry the SCD trait, and an estimated five million live with the disease. While a majority are of African descent, SCD can affect all ethnic groups, in particular those from areas of endemic malaria, including the Southern Mediterranean, Middle East, and India. Acute pain crises, or vaso-occlusive crises (VOCs), are the most common clinical manifestation of SCD. VOCs occur when sickled red blood cells irritate the lining of blood vessels and cause an inflammatory response that leads to vascular occlusion, tissue ischemia and pain.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a highly potent E-selectin antagonist that is bioavailable after subcutaneous administration. This second-generation compound has potential application in inflammatory diseases, and the company’s initial clinical development will focus on SCD. E-selectin is believed to play a major role in VOCs, vascular clots and blockages that cause pain crises in people living with the disease. Administration of GMI-1687 by subcutaneous injection, if successfully developed in the clinic, may enable this study drug to be approved as a patient controlled, point-of-care treatment option at time of VOC onset. There currently exists no FDA approved treatment for acute onset of painful and debilitating VOCs.

About GlycoMimetics, Inc.

GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including Acute Myeloid Leukemia, and for inflammatory diseases. GlycoMimetics’ science is based on an understanding of the role that carbohydrates play in cell recognition. The company’s specialized chemistry platform is being deployed to discover small molecule drugs--known as glycomimetics--that alter carbohydrate-mediated recognition in diverse disease states. As a leader in this science, GlycoMimetics leverages this unique approach to advance its pipeline of wholly-owned drug candidates with the goal of developing transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at www.glycomimetics.com.

Forward-Looking Statements

This press release contains forward-looking statements. These forward-looking statements may include, but are not limited to, statements regarding the conduct of and results from clinical trials, including presentation of data from such studies; planned or potential clinical development, partnering or financing opportunities; and the potential benefits and impact of the company’s drug candidate, GMI-1687. Actual results may differ materially from those described in these forward-looking statements. For a further description of the risks associated with these 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 (SEC) on March 29, 2023, and other filings GlycoMimetics makes with the SEC from time to time. Forward-looking statements speak only as of the date of this release, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240104347772/en/

Investor:

Argot Partners

Leo Vartorella

212-600-1902 / Glycomimetics@argotpartners.com

Public Relations:

Geoff Cook/973-652-7927

Source: GlycoMimetics, Inc.

FAQ

What are the primary and secondary endpoints of the Phase 1a study of GMI-1687 conducted by GlycoMimetics, Inc. (GLYC)?

The Phase 1a study met its primary and secondary endpoints of safety/tolerability and pharmacokinetics, with no observed dose-limiting toxicities or safety signals.

What is the initial focus of GMI-1687 being developed by GlycoMimetics, Inc. (GLYC)?

The initial focus of GMI-1687 is on sickle cell disease (SCD) as a potential patient-controlled point-of-care treatment for inflammatory diseases.

What were the dose levels evaluated in the Phase 1a trial of GMI-1687 by GlycoMimetics, Inc. (GLYC)?

Five dose levels were evaluated, including 3.3, 10, 20, 40, and 80 mg.

What type of dosing achieved target plasma concentrations and linear pharmacokinetics across all dosing levels in the Phase 1a trial of GMI-1687 by GlycoMimetics, Inc. (GLYC)?

Subcutaneous dosing achieved target therapeutic plasma concentration and linear pharmacokinetics with rapid renal clearance across all dosing levels.

When are the full study results of GMI-1687 expected to be presented?

Full results of the Phase 1a study are expected to be presented at an upcoming medical conference.