Vaccinex (VCNX) FDA Approvals

Vaccinex's Drug in the FDA Approval Process

This section highlights FDA-related milestones and regulatory updates for drugs developed by Vaccinex (VCNX). Over the past two years, Vaccinex has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as Pepinemab. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.

Pepinemab FDA Regulatory Timeline and Events

Pepinemab is a drug developed by Vaccinex for the following indication: Alzheimer’s Disease (AD). This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Data Presentation - May 27,2025

• Drug: Pepinemab
• Announced Date: May 27, 2025
• Indication: Alzheimer’s Disease (AD)

Vaccinex, Inc. announced that it will present new data characterizing the unique mechanism of pepinemab to enhance immune responses to checkpoint therapies in the neoadjuvant setting that are associated with improved pathologic response in patients with head and neck cancer.

Vaccinex, Inc. will present new data at the 2025 Annual Meeting of Clinical Oncology showing how pepinemab enhances immune responses when combined with checkpoint therapies in the neoadjuvant setting for head and neck cancer. Researchers have found that pepinemab, a humanized antibody, works by blocking the SEMA4D inhibitory signal on dendritic cells. This blockage helps promote better interactions between immune cells, especially T cells and dendritic cells, which are crucial for forming tertiary lymphoid structures (TLS). These structures are key to driving a strong immune response and improving the pathologic outcome for patients. The data indicate that adding pepinemab to neoadjuvant treatments does not increase toxicity, yet it improves TLS maturity. This could be significant for patients with immunologically "cold" tumors that are typically resistant to standard immune checkpoint therapies.

Data Presentation - April 21,2025

• Drug: Pepinemab
• Announced Date: April 21, 2025
• Target Action Date: April 29, 2025
• Indication: Alzheimer’s Disease (AD)

Vaccinex, Inc. announced that it will present exciting new data characterizing the unique mechanism of pepinemab to enhance immune responses to checkpoint therapies, corresponding with improved survival benefit in patients with melanoma and head and neck cancer at the 2025 Annual Meeting of American Association for Cancer Research (AACR) in Chicago on April 29, 2025.

Vaccinex, Inc. announced it will present exciting new data at the 2025 AACR Annual Meeting in Chicago on April 29, 2025. The company will showcase how pepinemab, a Semaphorin 4D blocking antibody, enhances immune responses when used with checkpoint therapies in patients with melanoma and head and neck cancer. Data indicate that pepinemab’s unique mechanism helps to boost the formation of mature, tertiary lymphoid structures (TLS) within tumors. These TLS are key in turning immunologically “cold” tumors into “hot” immune centers by improving dendritic cell function and better coordinating T cell responses. Senior VP Elizabeth Evans, PhD, will present the findings in two sessions that detail the improved survival benefits observed in clinical studies, providing promising insights for the future of cancer immunotherapy.

Provided Update - August 14,2024

Phase 1b/2

• Drug: Pepinemab
• Announced Date: August 14, 2024
• Indication: Alzheimer’s Disease (AD)

Vaccinex provided an update on new clinical findings from its SIGNAL-AD Phase 1b/2 trial of pepinemab antibody in Alzheimer's disease.

Vaccinex recently provided an update on its SIGNAL-AD Phase 1b/2 trial that evaluated the effects of its pepinemab antibody in Alzheimer’s disease. The trial focused on whether pepinemab could impact key brain regions affected early in the disease. Results showed a statistically significant difference (p=0.0297) in the FDG-PET signal from the medial temporal cortex after 12 months of treatment compared to placebo, suggesting that pepinemab may help preserve brain metabolic activity.

The study also examined secondary markers such as blood levels of GFAP and p-tau 217, which relate to astrocyte activity and neuronal damage, respectively. These biomarkers, along with cognitive measures, indicated potential benefits for patients with mild cognitive impairment due to Alzheimer’s disease. Importantly, pepinemab was well-tolerated, supporting further research and future partnerships in targeting early-stage neurodegenerative diseases.

Top-line data - July 17,2024

Phase 1b/2

• Drug: Pepinemab
• Announced Date: July 17, 2024
• Indication: Alzheimer’s Disease (AD)

Vaccinex, Inc. announced that it will present topline data for its randomized, double-blind, phase 1b/2 SIGNAL-AD study of pepinemab treatment for Alzheimer's disease at the Alzheimer's Association International Conference in Philadelphia, July 28- Aug 1, 2024. Eric Siemers, MD, Principal Investigator, will present results of the study in a Featured Research Session.

Vaccinex, Inc. announced that it will present topline data from its randomized, double-blind Phase 1b/2 SIGNAL-AD study at the Alzheimer’s Association International Conference in Philadelphia, scheduled for July 28 to August 1, 2024. The study focuses on pepinemab, a SEMA4D blocking antibody, as a potential treatment for Alzheimer’s disease. Researchers are investigating how pepinemab may help slow the disease by interfering with the damaging activation of astrocytes, brain cells that become reactive when interacting with stressed neurons.

Eric Siemers, MD, the study’s Principal Investigator, will share the research findings during a Featured Research Session on July 31, 2024, at 9:00 AM EST in Ballroom A at the Pennsylvania Convention Center. His presentation will highlight key results from the SIGNAL-AD trial, including safety outcomes and the effect of pepinemab on brain metabolism, offering new insights into innovative approaches for Alzheimer’s treatment.