GlycoMimetics' Phase 3 Blood Cancer Trial Failure Dampens Investor Sentiment

GlycoMimetics, a biotechnology company, ended March with $31.3 million in cash and equivalents. CEO Harout Semerjian had previously informed investors that the results from a phase 3 blood cancer trial could significantly impact the company's direction. Unfortunately, the outcome was not what he had hoped for.

On Monday, GlycoMimetics announced the failure of their phase 3 trial, leading to a 72% drop in stock value. The company's share price plummeted to 51 cents in premarket trading, down from $1.83 at the previous Friday’s close. This significant drop pushed the biotech firm’s valuation into penny stock territory as investors reacted to the disappointing results of the phase 3 study. The trial was assessing the efficacy of uproleselan, an E-selectin antagonist, in patients with acute myeloid leukemia (AML).

The trial involved 388 participants with relapsed or refractory AML. These individuals were randomized to receive either uproleselan or a placebo, in addition to a chemotherapy regimen. The results showed an overall survival of 13 months for patients taking uproleselan, slightly higher than the 12.3 months for those on the placebo. This minor difference meant that the trial did not meet its primary endpoint, posing a significant challenge for GlycoMimetics.

The biotech company has faced several setbacks, including the failure of a sickle cell disease prospect in partnership with Pfizer and the subsequent loss of this Big Pharma collaboration. Despite these challenges, GlycoMimetics continued to focus its pipeline on uproleselan. Their next most advanced asset, a successor to the candidate dropped by Pfizer, successfully cleared phase 1a in January.

GlycoMimetics initially planned to complete the phase 3 AML trial in 2020 but faced delays, pushing the expected readout date. The company had hoped that the results would allow them to file for FDA approval, targeting a U.S. market opportunity worth between $650 million and $850 million. Instead, the trial's failure has left the company looking towards a phase 2/3 National Cancer Institute (NCI) trial for a potential breakthrough.

The NCI trial is evaluating uproleselan in a different group of patients: those newly diagnosed with AML. However, this study was suspended in November 2021 after completing the phase 2 portion. GlycoMimetics has stated that the results of a preplanned phase 2 event-free survival interim analysis will be reported once available.

Time is now a critical factor for GlycoMimetics. With $31.3 million in cash and equivalents at the end of March, the company had previously indicated that their funds would be sufficient to cover operations through the fourth quarter of 2024. The recent trial failure and subsequent financial impact have put additional pressure on the company to find a viable path forward.