8-K
false000185119400018511942025-06-172025-06-17
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
|
Date of Report (Date of earliest event reported): June 17, 2025 |
VENTYX BIOSCIENCES, INC.
(Exact name of Registrant as Specified in Its Charter)
|
|
|
|
|
Delaware |
001-40928 |
83-2996852 |
(State or Other Jurisdiction
of Incorporation) |
(Commission File Number) |
(IRS Employer
Identification No.) |
|
|
|
|
|
12790 El Camino Real Suite 200 |
|
San Diego, California |
|
92130 |
(Address of Principal Executive Offices) |
|
(Zip Code) |
|
Registrant’s Telephone Number, Including Area Code: 760 407-6511 |
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
|
|
|
|
|
Title of each class
|
|
Trading
Symbol(s) |
|
Name of each exchange on which registered
|
Common Stock, $0.0001 par value per share |
|
VTYX |
|
The Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 7.01 Regulation FD Disclosure.
On June 17, 2025, Ventyx Biosciences, Inc. (the “Company”), issued a press release announcing top-line data from its Phase 2a safety and biomarker trial evaluating its CNS-penetrant NLRP3 inhibitor, VTX3232, in patients with early-stage Parkinson’s disease. The press release is attached hereto as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated in this Item 7.01 by reference.
The information in this Item 7.01, including Exhibit 99.1 attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On June 17, 2025, the Company announced top-line data from its Phase 2a safety and biomarker trial evaluating its CNS-penetrant NLRP3 inhibitor, VTX3232, in patients with early-stage Parkinson’s disease. The single-center, open-label Phase 2a trial evaluated a 40mg oral daily dose of VTX3232 in ten patients with early-stage, idiopathic Parkinson’s disease over a 28-day treatment period.
The following was observed from the top-line data in the Phase 2a trial:
•All adverse events were mild or moderate in severity and assessed as unrelated to study treatment. No serious adverse events were reported.
•Steady state concentrations of VTX3232 in cerebral spinal fluid (“CSF”) and plasma exceeded the IC90 for NLRP3 inhibition by ≥3-fold for a full 24-hours post-dose, demonstrating a pharmacokinetic profile.
•A high correlation between plasma and CSF exposures across the Company’s Phase 1 trial and this Phase 2a trial of VTX3232 was observed.
•Reductions in levels of downstream biomarkers of NLRP3-inhibition in plasma and CSF were observed with VTX3232.
•Treatment was associated with improvement in both motor and non-motor symptoms of Parkinson’s disease, as measured by MDS-UPDRS.
•Other exploratory markers including sTREM2, GFAP, S100B, NfL, Aβ40 and Aβ42, were either unchanged and/or fluctuated within normal levels and no acute changes in exploratory PET imaging were observed, consistent with a 28-day study.
The observations disclosed above are historical, based on preliminary data and summarized by the Company, and thus reflective of the Company’s interpretation of the data from the Phase 2a study. The above should therefore be interpreted with appropriate caution and should not be interpreted as indicative of future results. The Company intends to present the complete dataset from the Phase 2a study at a future medical conference and to publish full results in a peer-reviewed medical journal.
The Company has begun planning for a double blind, placebo-controlled, dose ranging Phase 2 trial in Parkinson’s disease, and potentially in additional neurodegenerative disorders such as Alzheimer’s disease.
Forward Looking Statements
The Company cautions you that statements contained in this report regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding the Company’s plans to present the complete dataset from the Phase 2a study at a future medical conference and to publish the full results in a peer-reviewed medical journal and the Company’s plans with respect to a double blind, placebo-controlled, dose ranging Phase 2 trial in Parkinson’s disease, and potentially in additional neurodegenerative disorders such as Alzheimer’s disease.
The inclusion of forward-looking statements should not be regarded as a representation by the Company that any of its plans or anticipated outcomes will be achieved. Actual results may differ from those set forth in this Current Report on Form 8-K due to the risks and uncertainties associated with clinical development, including that the Phase 2a study is a short-duration open label study in a small group of patients (and that results observed in the study may not be indicative of future results in a larger study with longer duration), and that the top-line data above has been reported in a summary format and is reflective of the Company’s interpretation of such data, which is preliminary, and has not been presented with the complete dataset, as well as the risks and uncertainties inherent in Company’s business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; the
Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; disruptions in the supply chain, including raw materials needed for manufacturing and animals used in research, delays in site activations and enrollment of clinical trials; the results of preclinical studies and clinical trials; early clinical trials not necessarily being predictive of future results; interim results not necessarily being predictive of final results; the potential of one or more outcomes to materially change as a trial continues and more patient data become available and following more comprehensive audit and verification procedures; regulatory developments in the United States and foreign countries; economic uncertainty in global markets caused by, among other things, geopolitical conditions, tariffs, military conflicts, and inflation volatility; unexpected adverse side effects or inadequate efficacy of the Company’s product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; the Company’s ability to obtain and maintain intellectual property protection for its product candidates; the use of capital resources by Ventyx sooner than expected; and other risks described in the Company’s prior press releases and the Company’s filings with the Securities and Exchange Commission (“SEC”), including in Part II, Item 1A (Risk Factors) of the Company’s Quarterly Report on Form 10-Q for the period ended March 31, 2025, filed on May 8, 2025, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
|
|
|
|
|
|
|
VENTYX BIOSCIENCES, INC. |
|
|
|
|
Date: |
June 17, 2025 |
By: |
/s/ Raju Mohan |
|
|
|
Raju Mohan, Ph.D.
Chief Executive Officer and Director
(Principal Executive Officer) |
EX-99.1
Exhibit 99.1
Ventyx Biosciences Announces Positive Top-Line Data from its Phase 2a Safety and Biomarker Trial Evaluating VTX3232 in Patients with Early-Stage Parkinson’s Disease
The study met its primary goal of establishing safety and tolerability of VTX3232 in patients with early-stage Parkinson’s disease
CSF and plasma exposures reinforce VTX3232’s potential as a
once-daily oral therapy for neurodegenerative diseases
VTX3232 treatment resulted in significant reductions in NLRP3-related biomarkers in
CSF and plasma, demonstrating sustained target engagement
VTX3232 is also being studied in a 12-week Phase 2 trial in participants with obesity and cardiometabolic risk factors; topline results expected in H2 2025
SAN DIEGO, CA, June 17, 2025 (GLOBE NEWSWIRE) -- Ventyx Biosciences, Inc. (Nasdaq: VTYX) (“Ventyx”, the “Company”), a clinical-stage biotech company developing oral therapies for autoimmune, inflammatory and neurodegenerative diseases, today announced positive top-line results from its Phase 2a study of VTX3232, a novel, CNS-penetrant NLRP3 inhibitor, in patients with early-stage Parkinson’s disease (NCT06556173).
The study accomplished the primary objective of demonstrating safety and tolerability, with no drug-related treatment-emergent adverse events (TEAEs) observed throughout the dosing period. The trial also met pharmacokinetic and pharmacodynamic endpoints, demonstrating high drug exposures in plasma and cerebral spinal fluid (CSF) as well as clear evidence of target engagement in plasma and CSF, with potent suppression of NLRP3-related biomarkers.
“We are thrilled that our Phase 2a data show that a once-daily dose of VTX3232 can safely maintain plasma and CSF levels above the IC90 for IL-1b for 24 hours in patients with early Parkinson’s disease,” said Mark Forman, MD, PhD, Chief Medical Officer. “In addition, we observed biomarker changes in CSF and plasma that reflect potent NLRP3 inhibition by VTX3232.”
David Russell, MD, PhD, Principal Investigator on the trial and member of the Yale University Parkinson’s Disease Research Group, remarked, “This was a thorough and well-conducted trial demonstrating clear evidence of target engagement in the CSF and plasma, with significant reduction to near-normal levels or the limit of quantitation (LOQ) in downstream biomarkers of NLRP3 inhibition, including IL-1b, IL-6 and high-sensitivity C-reactive protein (hsCRP). Our investigators also noted clinically significant reductions in MDS-UPDRS Parts II and III. With the caveat that this was a small, open-label study, all patients reported a subjective sense of improvement. Exploratory microglial PET imaging revealed no acute changes - not unexpected given the short duration of the trial.”
“Neuroinflammation is recognized as a potential trigger for neurodegenerative diseases. By inhibiting NLRP3-mediated cytokine production and inflammatory markers in the CNS, VTX3232 provides a unique opportunity for a disease-modifying therapy for Parkinson’s disease,” said Raju Mohan, PhD, Chief Executive Officer. “We are delighted that this trial met its goals of establishing that treatment with VTX3232 was safe and well tolerated, with high exposure levels in CSF and clear reductions in NLRP3-related biomarkers in a Parkinson’s disease patient population. We have initiated internal and external planning discussions for a placebo-controlled Phase 2 trial in Parkinson’s disease and potentially in additional neurodegenerative disorders such as Alzheimer’s disease.”
The single-center, open-label Phase 2a study evaluated a 40mg oral daily dose of VTX3232 in ten patients with early-stage, idiopathic Parkinson’s disease over a 28-day treatment period. The primary objective of this study was to evaluate safety and tolerability. Key secondary objectives included characterization of VTX3232’s pharmacokinetic profile in plasma and CSF, and its pharmacodynamic activity, by measuring effects on plasma, and CSF biomarkers of NLRP3-inhibition at Day 28, including IL-1, unbound, biologically active IL-18, IL-6, and hsCRP (Tier 1 biomarkers). The study also incorporated exploratory markers of neurodegeneration (Tier 2 biomarkers), motor and non-motor aspects of Parkinson’s disease as measured by MDS-UPDRS, and a microglial PET imaging assessment using [18F]-DPA 714 binding to translocator protein (TSPO).
Key takeaways from the Phase 2a study in Parkinson’s patients:
•VTX3232 was safe and well tolerated through the 28-day dosing period
oAll adverse events (AEs) were mild or moderate in severity and assessed as unrelated to study treatment; no serious adverse events were reported
oThe majority of AEs were attributed to PET-tracer administration or lumbar puncture for CSF sampling, and self-resolved
•VTX3232 demonstrated an excellent pharmacokinetic profile
oSteady state concentrations in CSF and plasma exceed the IC90 for NLRP3 inhibition by ≥3-fold for a full 24-hours post-dose
oHigh correlation between plasma and CSF exposures between this Phase 2a study and the Phase 1 study in healthy volunteers, reinforcing the profile of VTX3232 as a potential once-daily, oral therapy for neurodegenerative diseases
•VTX3232 demonstrated target engagement by decreasing biomarkers of NLRP3 inhibition, IL-1b in plasma* and IL-18, in both the plasma and CSF (Tier 1 biomarkers)
*Insufficient samples above the limit of quantification (LOQ) to quantify IL-1b in the CSF
oDownstream biomarkers, specifically IL-6, hsCRP and SAA, were also reduced, and in some cases, approached the LOQ
oEffects on biomarkers in early-stage Parkinson’s patients were consistent with those observed in healthy volunteers in the Phase 1 study
oTier 2 exploratory markers including sTREM2, GFAP, S100B, NfL, Ab40 and Ab42, were either unchanged and/or fluctuated within normal levels
•VTX3232 treatment was associated with improvement in both motor and non-motor symptoms of Parkinson’s disease, as measured by MDS-UPDRS
oStatistically significant reductions in Part I (p<0.05), Part II (p<0.01) and Part III (p<0.01) were observed
oPer the Principal Investigator, Dr. Russell, “Encouraging results, with the usual caveats associated with an open-label study.”
•No acute changes in exploratory PET imaging were observed, consistent with a relatively short, 28-day study
Ventyx intends to present the complete dataset at a future medical meeting and to publish full results in a peer-reviewed medical journal.
These data meet Ventyx’s internal criteria for continued clinical development in Parkinson’s disease. Planning for a double blind, placebo-controlled, dose ranging Phase 2 trial in Parkinson’s disease, and potentially in additional neurodegenerative disorders such as Alzheimer’s disease, is underway.
About VTX3232
VTX3232 is a CNS-penetrant, potent and selective inhibitor of the NLRP3 inflammasome. VTX3232 has an excellent non-clinical in vitro and in vivo safety profile including in 6- and 9-month chronic toxicology studies.
VTX3232 has completed a Phase 1 trial in healthy volunteers and has shown good safety and tolerability with once-daily doses achieving therapeutic CSF exposures and significant reductions in NLRP3-mediated biomarkers in plasma and in the CSF.
VTX3232 is also being studied in a 12-week Phase 2 trial in participants with obesity and cardiometabolic risk factors with topline results expected in H2 2025.
About Ventyx Biosciences
Ventyx Biosciences is a clinical-stage biopharmaceutical company developing innovative oral therapies for patients with autoimmune, inflammatory, and neurodegenerative diseases. Our expertise in medicinal chemistry, structural biology, and immunology enables the discovery of differentiated oral small molecule therapeutics for conditions with high unmet medical need, and our extensive experience in clinical development allows the rapid progression of these drug candidates through clinical trials.
Our portfolio of NLRP3 inhibitors includes VTX2735, a peripherally restricted NLRP3 inhibitor in Phase 2 development for recurrent pericarditis, and VTX3232, a CNS-penetrant NLRP3 inhibitor in Phase 2 development for neurodegenerative and cardiometabolic diseases. Our inflammatory bowel disease portfolio includes two Phase 2 compounds: tamuzimod (VTX002), an S1P1R modulator, and VTX958, a TYK2 inhibitor.
For more information on Ventyx, please visit our website at https://ventyxbio.com.
Forward-Looking Statements
Ventyx cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Ventyx’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the potential of VTX3232 as a disease-modifying therapy for Parkinson’s disease and additional neurodegenerative disorders such as Alzheimer’s disease, the anticipated timing and results of future clinical trials, including the topline results of the ongoing Phase 2 trial of VTX3232 in participants with obesity and cardiometabolic risk factors, management’s plans with respect to a double blind, placebo-controlled, dose ranging Phase 2 trial in Parkinson’s disease, and potentially in additional neurodegenerative disorders such as Alzheimer’s disease, and management’s plans with respect to future presentations and publications of VTX3232 data.
The inclusion of forward-looking statements should not be regarded as a representation by Ventyx that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties associated with a short-duration open label study in a small group of patients (including that results observed in this study may not be indicative of future results in a larger study with longer duration of dosing), and risks and uncertainties inherent in Ventyx’s business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; Ventyx’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; disruptions in the supply chain, including raw materials needed for manufacturing and animals used in research, delays in site activations and enrollment of clinical trials; the results of preclinical studies and clinical trials; early clinical trials not necessarily being predictive of future results; interim results not necessarily being predictive of final results; the potential of one or more outcomes to materially change as a trial continues and more patient data become available, and following more comprehensive audit and verification procedures; regulatory developments in the United States and foreign countries; economic uncertainty in global markets caused by, among other things, geopolitical conditions, tariffs, military conflicts, and inflation volatility; unexpected adverse side effects or inadequate efficacy of Ventyx’s product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Ventyx’s ability to obtain and maintain intellectual property protection for its product candidates; the use of capital resources by Ventyx sooner than expected; and other risks described in Ventyx’s prior press releases and Ventyx’s filings with the Securities and Exchange Commission (SEC), including in Part II, Item 1A (Risk Factors) of Ventyx’s Quarterly Report on Form 10-Q for the quarter year ended March 31, 2025, filed on May 8, 2025, and Ventyx’s subsequent filings with the SEC.
You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Ventyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
Investor Relations Contact:
Joyce Allaire
Managing Director
LifeSci Advisors
IR@ventyxbio.com
