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Promising responses in Phase 1b segment of the open-label KEYNOTE B84 study of pepinemab with KEYTRUDA® in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC);
Enrollment in Phase 2 expansion underway
Patient enrollment also underway in Phase 1/2a study of pepinemab in Alzheimer’s disease
“I am pleased to report that the last 15 months have been extremely productive for
“In addition, phase 1 has been completed and enrollment is underway in Phase 2a expansion of the SIGNAL-AD trial in early Alzheimer’s Disease. Looking ahead to 2022 and early 2023, we expect to complete enrollment in the KEYNOTE-B84 and SIGNAL-AD trials. Data from these studies will help to guide the regulatory and product development path for the pepinemab programs. We look forward to continue to update the clinical community and investors on our progress at other medical conferences in 2022.”
Pepinemab Clinical Updates:
Oncology: 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) for first-line treatment in recurrent or metastatic head and neck cancer.
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 and has been shown to promotes immunosuppression in head and neck cancer, there is strong rationale for development in this indication.
The KEYNOTE-B84 study is expected to enroll up to 65 subjects across 18 U.S. trial sites and will assess whether immunotherapy with pepinemab in combination with pembrolizumab can improve responses in the front-line setting. The primary outcome of the study is objective response, and additional outcomes include progression free survival and overall survival.
Other Oncology Trials. Pepinemab is also being evaluated in multiple investigator-sponsored trials (ISTs) in pancreatic and breast cancer and in “window of opportunity” studies, including head and neck cancer and melanoma to evaluate pepinemab in several combination treatments.
Alzheimer’s Disease. Enrollment continues in the Phase 1/2a SIGNAL-AD trial of pepinemab as a single agent in early Alzheimer’s disease. This trial is being funded in part by the Alzheimer’s
The randomized, double-blind, placebo-controlled, multi-center safety and biomarker study of pepinemab in early AD is planned to enroll 40 subjects across 15 U.S. trial sites.
Huntington’s disease. The Phase 2 double-blind, placebo-controlled SIGNAL trial of pepinemab in patients with early Huntington’s disease (HD) has been completed, and
While the Phase 2 study did not meet the prespecified primary endpoints, we believe that multiple exploratory and post-hoc analyses support the potential cognitive benefit of treatment with pepinemab in early manifest HD patients, particularly those with evidence of mild cognitive or functional deficits at baseline including:
- Highly significant improvement (p=0.007) in the Huntington’s Disease Cognitive Assessment Battery (HD-CAB) Composite score, a measure comprised of 6 different cognitive assessments that has also been employed in other HD trials.
- 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.
- Reduced atrophy (p=0.017) in caudate region of striatum, a brain region known to degenerate early in HD progression, along with a 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 and clinical progression in several studies of AD.
The Company continues to actively explore advancing pepinemab into a Phase 3 HD trial in collaboration with biopharmaceutical partners.
Upcoming Anticipated Milestones:
- Phase 1b/2 Keynote B84 Trial: Open label head and neck cancer trial of pepinemab in combination with KEYTRUDA/pembrolizumab. Multiple interim data read-outs expected in 2022. Enrollment is expected to be completed and primary outcome data presented in 2023.
- AACR Presentation:
Monday, April 11in the Phase II Clinical Trials 1 between 9:00 a.m. and 12:30 p.m. CST.
- Phase 1/2a Alzheimer’s Disease Trial: Topline data are expected in 2023.
As previously announced, the Company has entered into several collaborations with pharmaceutical and biotechnology companies employing the unique capabilities of our ActivMAb® antibody discovery platform to address difficult to drug multi-pass membrane receptors including G-protein Coupled Receptors (GPCRs) and ion channels known to be strongly associated with diseases.
Financial Results for the Twelve Months Ended
Research and Development Expenses. Research and development expenses for the year ended
Research and Development expenses are lower in 2021 compared to 2020 primarily attributed to a smaller number of patients enrolled in clinical trials, especially the CLASSICAL-Lung and SIGNAL studies.
General and Administrative Expenses. General and administrative expenses for the year ended
The difference in general and administrative expenses is primarily attributable to planned cost reductions, as part of cost control measures.
Comprehensive loss/Net loss per share. The Comprehensive Loss and Net loss per share for the year ended
Financial results are included below. For further details on Vaccinex’s financials, refer to its Form 10-K filed
Pepinemab is a humanized IgG4 monoclonal antibody that inhibits SEMA4D, which regulates chronic inflammation in the tumor microenvironment. Preclinical and clinical data show that pepinemab promotes infiltration of activated immune cells while reducing immune suppression in tumors and repair or prevention of neurological damage in neuroinflammatory and neurodegenerative diseases.
Results of a Phase 1b/2 study were presented at ASCO 2020 and were highlighted in the
KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co. Inc.,
Pepinemab is being evaluated in studies in oncology and neurodegenerative disease. In oncology, pepinemab is being evaluated in the Phase 1b/2 open label KEYNOTE-B84 study, in combination with KEYTRUDA/pembrolizumab, in patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HSNCC). In neurodegenerative disease, pepinemab is being evaluated in a Phase 1/2a randomized, double-blinded, placebo-controlled trial in people with early Alzheimer’s Disease, with ongoing exploration of potential Phase 3 development in Huntington’s disease.
The Company has also developed a proprietary drug discovery platform, ActivMAb®, that it intends to use to create opportunities for future pipeline expansion and strategic collaborations, particularly by exploiting its unique capability to select high value antibodies against important multi-pass membrane receptors including GPCR and ion channels.
Forward Looking Statements
To the extent that statements contained in this presentation are not descriptions of historical facts regarding
Consolidated Balance Sheets
(in thousands, except share and per share data)
|Cash and cash equivalents||$||8,589||$||10,596|
|Prepaid expenses and other current assets||816||533|
|Total current assets||9,405||11,286|
|Property and equipment, net||297||416|
|Operating lease right-of-use asset||141||-|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Senior secured convertible debt, net||-||8,074|
|Current portion of long-term debt||74||-|
|Operating lease right-of-use liability||141||-|
|Total current liabilities||2,256||13,180|
|Commitments and contingencies (Note 7)|
|Stockholders’ equity (deficit):|
|Common stock, par value of
|Additional paid-in capital||307,281||250,914|
|TOTAL STOCKHOLDERS’ EQUITY (DEFICIT)||7,412||(2,612||)|
|TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY||$||9,843||$||11,702|
Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share data)
|Costs and expenses:|
|Cost of revenue||-||2|
|Research and development||17,160||21,549|
|General and administrative||6,230||7,405|
|Total costs and expenses||23,390||28,956|
|Loss from operations||(22,490||)||(28,331||)|
|Gain on forgiveness of PPP loan||876||-|
|Other income (expense), net||43||(31||)|
|Loss before provision for income taxes||(22,380||)||(28,851||)|
|Provision for income taxes||-||-|
|Net loss attributable to noncontrolling interests||-||-|
|Net loss attributable to
|Net loss per share attributable to
|Weighted-average shares used in computing net loss per share attributable to Vaccinex, Inc. common stockholders, basic and diluted||28,849,197||18,786,768|
Source: Vaccinex, Inc.