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Vaccinex Reports Completion of Last Patient Visit in Randomized, SIGNAL-AD Phase 1b/2 Study of Pepinemab Treatment for Alzheimer’s Disease

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Vaccinex announced the completion of the last patient visit in its Phase 1b/2 SIGNAL-AD study of pepinemab for Alzheimer's Disease (AD). The database lock is expected in June, with key outcomes to be reported in July.

Pepinemab targets astrocyte reactivity and neuroinflammation, believed to accelerate AD progression. Key endpoints include safety, tolerability, brain metabolic activity, and biomarkers like GFAP and p-tau 217. The study also uses cognitive scales to assess treatment effects on cognitive decline.

Funded partly by $4.75 million from the Alzheimer’s Drug Discovery Foundation and a $0.75 million grant from the Alzheimer’s Association, pepinemab has shown potential to offer alternative or complementary treatment to existing anti-Aβ therapies.

Positive
  • Completion of the last patient visit in the SIGNAL-AD study on schedule.
  • Database lock expected in June and outcomes to be reported in July.
  • Pepinemab targets important mechanisms in AD, including astrocyte reactivity and neuroinflammation.
  • The study aims to achieve primary endpoints such as safety, tolerability, and brain metabolic activity.
  • Biomarkers like GFAP and p-tau 217 help measure disease progression.
  • Exploratory endpoints include cognitive scales to assess cognitive decline.
  • Pepinemab has been well-tolerated in over 600 patients in prior trials.
  • Funded by significant grants: $4.75 million from ADDF and $0.75 million from the Alzheimer’s Association.
  • Potential to be an alternative or complementary treatment to current anti-Aβ amyloid therapies.
Negative
  • No efficacy results reported yet; success remains speculative until July data release.
  • High competition from established anti-Aβ amyloid treatments like Leqembi and donanemab.
  • Financial risks remain if study results are not favorable, impacting future funding and stock performance.
  • Potential delays in data analysis or reporting could impact investor confidence.

Pepinemab's Phase 1b/2 study completion represents a significant milestone in the field of Alzheimer’s Disease (AD) treatment. The drug targets astrocyte reactivity and neuroinflammation – important factors in the progression of neurodegenerative diseases. Research indicates that astrocytes, key support cells in the brain, become neurotoxic when activated. Blocking SEMA4D signaling with pepinemab may, therefore, protect neurons from this toxic environment.

However, the results from Huntington’s disease treatment with pepinemab, while promising, do not guarantee similar outcomes in AD due to differences in disease pathology. Additionally, the study’s endpoints, including brain metabolic activity, plasma levels of GFAP and phosphorylated tau peptide (p-tau 217), are well-chosen biomarkers reflecting disease progression but will need robust clinical improvement to convince stakeholders.

The broad implications for AD patients, if successful, are profound given the limited efficacy of current treatments. Retail investors should note the emphasis on safety and the potential for combination therapy with existing anti-Aβ treatments, which could enhance therapeutic outcomes.

The financial stakes for Vaccinex are high with the upcoming results. The significant investment from the Alzheimer’s Drug Discovery Foundation (ADDF) and the Alzheimer’s Association underscores the potential impact. If the study outcomes are positive, this could justify the $5.5 million in funding and attract further investments, boosting stock performance.

Conversely, should the results not meet expectations, the financial repercussions could be substantial, given the company’s current clinical stage and reliance on breakthrough drug developments. Investors should be prepared for volatility around the release of these results in July.

Importantly, the timing of the database lock and subsequent data release reflects an efficient trial process, which is important for investor confidence. The company’s previous clinical trials enrolling over 600 patients without major safety concerns is a positive indicator, but the efficacy in AD remains the pivotal factor.

The Alzheimer’s treatment market is poised for potential disruption with the introduction of innovative therapies like pepinemab. With an estimated 6 million people diagnosed with AD in the US alone, the demand for effective treatments is immense. Current treatments, such as Leqembi and donanemab, have shown limitations, which positions pepinemab as a potential alternative or adjunct therapy.

Market dynamics could shift significantly if pepinemab proves effective. The drug’s ability to potentially enhance the effects of existing treatments could carve out a substantial niche. Investors should consider the competitive landscape and the unmet needs in AD treatment, which pepinemab aims to address.

The company’s hypothesis and the promise of a differentiated approach targeting neuroinflammation could resonate well within the market, but only if backed by compelling clinical data. The strategic funding partnerships also highlight external confidence in the potential market impact.

Company expects to lock database in June and
remains on track to report key outcomes later in July.

Pepinemab targets astrocyte reactivity and neuroinflammation,
believed to be
key drivers of neurodegeneration.

ROCHESTER, N.Y., June 06, 2024 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating neurodegenerative disease and cancer through the inhibition of Semaphorin 4D (SEMA4D), today announced that the last patient completed their last visit in its randomized, placebo-controlled double-blind study of pepinemab treatment for Alzheimer’s disease.

What can we expect to learn from this study?

  • Vaccinex scientists discovered and published that SEMA4D, a molecule that binds to high affinity plexin-B1 receptors predominantly expressed on astrocytes in the brain, is highly upregulated on stressed or damaged neurons during progression of Alzheimer’s Disease (AD).
  • Astrocytes, which are key brain cells that support the health and function of neurons, undergo substantial changes in morphology and gene expression when SEMA4D binds to their plexin-B1 receptors. As a result, they switch from normal supportive functions to neurotoxic inflammatory activity that is believed to accelerate and aggravate progression of AD.
  • The Company’s hypothesis, which is being tested in the SIGNAL-AD study, is that treating with pepinemab antibody can block signaling by SEMA4D and prevent some or all the damaging consequences of astrocyte activation.
  • The Company has previously reported that antibody blockade of SEMA4D appears to protect and restore healthy astrocyte functions and to slow or prevent disease progression in patients with Huntington’s disease by several different measures.
  • Key outcomes of the SIGNAL-AD study will include safety and tolerability and the impact of pepinemab treatment on brain metabolic activity as detected by FDG-PET and astrocyte reactivity reflected in plasma levels of glial fibrillary acidic protein (GFAP), a molecule known to be released into blood by reactive astrocytes. Together, these are key biomarkers of disease progression.
  • Deposition of Aβ amyloid in the brain is considered to be the earliest recognized event in the pathologic cascade leading to AD. Aggregates of Aβ are believed to trigger a series of subsequent events, including astrocyte reactivity and formation of toxic tau tangles in neurons, which are believed to be key drivers of neurodegeneration. Accordingly, secondary endpoints will also include plasma levels of phosphorylated tau peptide (p-tau 217), a biomarker released into blood during formation of tau tangles in neurons. In addition, several validated cognitive scales will be employed as exploratory endpoints to evaluate potential treatment effects on cognitive decline, the main clinical symptom of AD.
  • The Company believes that the prevalence of AD (6 million people diagnosed with AD in the US alone) and current concerns about the limitations of treatment with anti-Aβ amyloid antibodies such as Leqembi (Eisai and Biogen) and donanemab (Eli Lilly) could make pepinemab, if approved, attractive as a potential alternative treatment or possibly for use in combination with anti-Aβ to enhance the benefit to patients. Pepinemab has, to date, been well-tolerated in clinical trials that enrolled a total of more than 600 patients.

The SIGNAL-AD study was funded in part by two investments from the Alzheimer’s Drug Discovery Foundation (ADDF) for a total of $4.75 million, and by an $0.75 million grant from the Alzheimer’s Association.

About Pepinemab
Pepinemab is a humanized IgG4 monoclonal antibody designed to block SEMA4D, which can bind to plexin-B1 receptors to trigger collapse of the actin cytoskeleton in cells and lead to loss of homeostatic functions of astrocytes and other glial cells in the brain and of dendritic cells in immune tissue. Pepinemab appears to have been well-tolerated with a favorable safety profile in multiple clinical trials in different neurological and cancer indications.

About Vaccinex Inc.
Vaccinex, Inc. is pioneering a differentiated approach to treating slowly progressive neurodegenerative diseases and cancer through the inhibition of semaphorin 4D (SEMA4D). The Company’s lead drug candidate, pepinemab, blocks SEMA4D, a potent biological effector that it believes triggers damaging inflammation in chronic diseases of the brain and prevents immune infiltration into tumors. Pepinemab is being studied as a monotherapy in the Phase 1b/2 SIGNAL-AD study in Alzheimer’s Disease, with ongoing exploration of potential Phase 3 development in Huntington’s disease. In oncology, pepinemab is being evaluated in combination with KEYTRUDA® in the Phase 1b/2 KEYNOTE-B84 study in recurrent or metastatic head and neck cancer (HNSCC) and in combination with BAVENCIO® in a Phase 1b/2 study in patients with metastatic pancreatic adenocarcinoma (PDAC). The oncology clinical program also includes several investigator-sponsored studies in solid tumors including breast cancer and melanoma.

Vaccinex has global commercial and development rights to pepinemab and is the sponsor of the KEYNOTE-B84 study which is being performed in collaboration with Merck Sharp & Dohme Corp, a subsidiary of Merck and Co, Inc. Kenilworth, NJ, USA. Additional information about the study is available at: clinicaltrials.gov.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co. Inc., Kenilworth, NJ, USA. BAVENCIO®/avelumab is provided by Merck KGaA, Darmstadt, Germany, previously as part of an alliance between the healthcare business of Merck KGaA, Darmstadt, Germany and Pfizer.

Forward Looking Statements
To the extent that statements contained in this press release are not descriptions of historical facts regarding Vaccinex, Inc. (“Vaccinex,” “we,” “us,” or “our”), they are forward-looking statements reflecting management’s current beliefs and expectations. Such statements include, but are not limited to, statements about expectations and objectives with respect to the results and timing of the SIGNAL-AD clinical trial; expectations with respect to compliance with Nasdaq listing standards; our plans, expectations and objectives with respect to the results and timing of the SIGNAL-AD and KEYNOTE-B84 clinical trials; the use and potential benefits of pepinemab in R/M HNSCC, lung cancer, metastatic pancreatic adenocarcinoma (PDAC) and other indications; the potential for benefits as compared to single agent KEYTRUDA® or BAVENCIO®; expectations with respect to the collaboration of Merck, the potential to initiate a Phase 3 trial in Huntington’s disease; and other statements identified by words such as “anticipate,” “believe,” “plans,” “schedule,” “being,” “will,” “appears,” “expect,” “ongoing,” “potential,” “suggest”, and similar expressions or their negatives (as well as other words and expressions referencing future events, conditions, or circumstances). Forward-looking statements involve substantial risks and uncertainties that could cause the outcome of our research and pre-clinical development programs, clinical development programs, future results, performance, or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, uncertainties inherent in the execution, cost and completion of preclinical studies and clinical trials, that interim and preliminary data may not be predictive of final results and does not ensure success in later clinical trials, uncertainties related to regulatory approval, risks related to our dependence on our lead product candidate pepinemab, the possible delisting of our common stock from Nasdaq if the Company is unable to regain compliance with the Nasdaq listing standards, and other matters that could affect our development plans or the commercial potential of our product candidates. Except as required by law, the Company assumes no obligation to update these forward-looking statements. For a further discussion of these and other factors that could cause future results to differ materially from any forward-looking statement, see the section titled “Risk Factors” in our periodic reports filed with the Securities and Exchange Commission and the other risks and uncertainties described in the Company’s annual year-end Form 10-K and subsequent filings with the SEC.

Investor Contact
Elizabeth Evans, PhD
Chief Operating Officer, Vaccinex, Inc.
(585) 271-2700
eevans@vaccinex.com


FAQ

What is the SIGNAL-AD study about?

The SIGNAL-AD Phase 1b/2 study investigates the safety and efficacy of pepinemab for Alzheimer's Disease.

When will Vaccinex report the results of the SIGNAL-AD study?

Key outcomes of the SIGNAL-AD study are expected to be reported in July 2024.

What are the primary endpoints in the SIGNAL-AD study?

Primary endpoints include safety, tolerability, and brain metabolic activity, measured by FDG-PET and biomarkers like GFAP.

What are the secondary endpoints in the SIGNAL-AD study?

Secondary endpoints include plasma levels of phosphorylated tau peptide (p-tau 217) and exploratory cognitive scales.

How was the SIGNAL-AD study funded?

The study received $4.75 million from the Alzheimer’s Drug Discovery Foundation and a $0.75 million grant from the Alzheimer’s Association.

What makes pepinemab a promising treatment for Alzheimer's?

Pepinemab targets astrocyte reactivity and neuroinflammation, important drivers of AD progression, and may serve as an alternative or complementary treatment to current therapies.

How many patients have been treated with pepinemab in clinical trials?

Pepinemab has been well-tolerated in clinical trials involving over 600 patients.

Vaccinex, Inc.

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