On November 8, 2021 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Vaccinex, NOV 8, 2021, View Source [SID1234594781]).

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"We made steady progress on our pepinemab clinical programs since our last quarterly update. Enrollment is now underway in the Phase 1b/2 head and neck cancer trial and the Phase 1/2a Alzheimer’s disease trial, two of the serious indications in which SEMA4D is overexpressed and is believed to contribute to disease pathology," stated Maurice Zauderer, Ph.D., President and Chief Executive Officer.

"As presented at the September European Huntington’s Disease Network meeting, we believe post-hoc analysis of the Phase 2, SIGNAL trial of pepinemab in patients with early manifest Huntington’s disease (HD) supports the potential cognitive benefit of treatment with pepinemab in HD patients, particularly those with mild advanced disease. We are engaged in discussions with potential partners for the advancement of this important program."

Pepinemab Clinical Updates:

Head and Neck Cancer. Enrollment is underway in the Phase 1b/2 clinical trial evaluating pepinemab in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in advanced recurrent or metastatic head and neck cancer.

The study will enroll up to 65 subjects across 18 U.S. trial sites and will assess whether combination therapy can improve responses in this population. Key endpoints of the study will include objective response, progression free survival and overall survival. Vaccinex anticipates data from this study in the second half of 2022.

Multiple prior studies suggest that inhibition of SEMA4D increases immune infiltration and alters the balance of cytotoxic and immunosuppressive cells in the tumor microenvironment. As SEMA4D is highly expressed in head and neck cancer, there is strong rationale for development in this indication.

Alzheimer’s Disease. Patient screening and enrollment have been initiated in the Phase 1/2a clinical trial of pepinemab in Alzheimer’s disease. The Alzheimer’s trial is being funded in part by the Alzheimer’s Drug Discovery Foundation by the Alzheimer’s Association under the 2020 Part the Cloud Program.

The randomized, double-blind, placebo-controlled, multi-center safety and biomarker study of pepinemab in early AD is planned to enroll 40 subjects across 14 U.S. trial sites. Vaccinex anticipates topline data from this study in late 2022 or early 2023.
Huntington’s disease. We believe that post-hoc analysis of the Phase 2, double-blind, placebo-controlled SIGNAL trial of pepinemab in patients with early manifest Huntington’s disease (HD) supports the potential cognitive benefit of treatment with pepinemab in HD patients, particularly those with mild cognitive deficits.
Highly significant improvement (p=0.007) in the (Huntington’s Disease Cognitive Assessment Battery (HD-CAB) Composite score, a widely employed measure comprised of 6 different cognitive assessments.
Significant benefit in reducing apathy severity (p=0.017, 1-sided), a problem behavior that has previously been correlated with cognition in both HD and AD.
Striking increase in brain metabolic activity as measured by FDG-PET in most brain regions. Decline in FDG-PET signal has been reported to correlate with cognitive decline in several studies of AD.
The company continues to actively explore advancing pepinemab into a Phase 3 HD trial in collaboration with a biopharmaceutical partner; discussions are ongoing.

Other Trials. Pepinemab is also being evaluated in multiple investigator-sponsored trials (ISTs) being conducted by the Winship Cancer Institute of Emory University to evaluate pepinemab in combination with checkpoint inhibitors in "Window of Opportunity" studies in head and neck cancer and melanoma.
Upcoming Anticipated Milestones:

H2:2022– Meaningful data from open label head and neck cancer trial
Late 2022/Early 2023 – Topline data from randomized Alzheimer’s trial
ActivMAb Updates:

As previously announced, we have entered into several collaborations with pharmaceutical and biotechnology companies employing the unique capabilities of our ActivMAb antibody discovery platform to address difficult to drug G-protein Coupled Receptors (GPCRs) known to be strongly associated with diseases. "We believe this enabling technology will allow us and our collaborators to address significant market opportunities," said Ernest S. Smith, Ph.D., Chief Scientific Officer.

Financial Results for the Three Months Ended September 30, 2021:

Research and Development Expenses. Research and development expenses for the three months ended September 30, 2021 were $3.6 million as compared to $7.3 million for the comparable period in 2020.

General and Administrative Expenses. General and administrative expenses for the three months ended September 30, 2021 were $1.5 million as compared to $1.9 million for the comparable period in 2020.

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on September 30, 2021 were $13.8 million, as compared to $10.6 million as of December 31, 2020.