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Enrollment underway in combination Phase 1b/2 study of pepinemab with KEYTRUDA® in Metastatic Head and Neck Squamous Cell Carcinoma (HNSCC)
Patient screening and enrollment underway in Phase 1/2a study of pepinemab in Alzheimer’s disease
Engaged in partnering discussions for a randomized Phase 3 trial in Huntington’s disease
“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
“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.
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 Foundationby 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.
Vaccinexanticipates 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 Universityto 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
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
Financial Results for the Three Months Ended
Research and Development Expenses. Research and development expenses for the three months ended
General and Administrative Expenses. General and administrative expenses for the three months ended
Forward Looking Statements
To the extent that statements contained in this presentation are not descriptions of historical facts regarding
Condensed Consolidated Balance Sheets (Unaudited)
(in thousands, except share and per share data)
|Cash and cash equivalents||$||13,741||$||10,596|
|Prepaid expenses and other current assets||1,071||533|
|Total current assets||14,862||11,286|
|Property and equipment, net||327||416|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Senior secured convertible debt, net||-||8,074|
|Total current liabilities||2,165||13,180|
|Commitments and contingencies (Note 7)|
|Stockholders’ equity (deficit):|
|Common stock, par value of
shares issued as of
30,801,110 and 22,387,175 shares outstanding as of
|Additional paid-in capital||307,128||250,914|
|TOTAL STOCKHOLDERS’ EQUITY (DEFICIT)||11,890||(2,612||)|
|TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY||$||15,189||$||11,702|
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited)
(in thousands, except share and per share data)
|Three Months Ended
||Nine Months Ended
|Costs and expenses:|
|Cost of revenue||-||2||-||2|
|Research and development||3,629||7,334||13,206||17,300|
|General and administrative||1,484||1,872||4,666||5,565|
|Total costs and expenses||5,113||9,208||17,872||22,867|
|Loss from operations||(5,063||)||(8,583||)||(16,972||)||(22,242||)|
|Other income (expense), net||(1||)||(23||)||48||(14||)|
|Loss before provision for income taxes||(5,206||)||(8,754||)||(17,749||)||(22,406||)|
|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
Source: Vaccinex, Inc.