tamuzimod (VTX002) / Ventyx Biosci - LARVOL DELTA

VTx-002-01-001: Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Vector Y Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • First-in-human • Amyotrophic Lateral Sclerosis • CNS Disorders • FUS • SOD1

Lilly to acquire Ventyx Biosciences to advance oral therapies targeting inflammatory-mediated diseases (Eli Lilly Press Release) - "Under the terms of the agreement, Lilly will acquire all of the outstanding shares of Ventyx for $14.00 per share of common stock in an all-cash transaction (equal to an aggregate equity value of approximately $1.2 billion)."

M&A • Inflammation • Inflammatory Bowel Disease • Lupus • Parkinson's Disease

Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Vector Y Therapeutics

New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Non-clinical development and phase 1/2 clinical trial design of VTx-002, an AAV5.2 vectorized intrabody targeting TDP-43 for amyotrophic lateral sclerosis (ALS-MND 2025) - "Collectively, our non-clinical findings suggest that AAV-mediated intrabody delivery targeting TDP-43 is a viable therapeutic strategy for ALS paving the way for clinical development in ALS and other TDP-43-related disorders. phase 1/2 clinical trial is planned to be an open-label, dose-escalating study in early-stage ALS patients. The primary objective of the study is safety and tolerability."

Clinical • P1/2 data • Amyotrophic Lateral Sclerosis • Anesthesia • CNS Disorders • FUS • SOD1 • TARDBP

Tamuzimod in patients with moderately-to-severely active ulcerative colitis: a multicentre, double-blind, randomised, placebo-controlled, phase 2 induction trial. (PubMed, Lancet Gastroenterol Hepatol) - P2 | "Induction therapy with tamuzimod was effective and well tolerated in patients with ulcerative colitis. These results and the favourable risk-benefit profile of tamuzimod collectively support the continued clinical development of tamuzimod for the treatment of moderately-to-severely active ulcerative colitis."

Journal • P2 data • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Macular Edema • Oncology • Ophthalmology • Pain • Respiratory Diseases • Ulcerative Colitis • S1PR1

No easy way out: blocking lymphocyte egress by the S1P1 receptor 1 modulator tamuzimod in ulcerative colitis. (PubMed, Lancet Gastroenterol Hepatol) - No abstract available

Journal • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Presents New 52-Week Results from the Phase 2 Trial of VTX002 (Tamuzimod) in Ulcerative Colitis at UEG Week 2024 (GlobeNewswire) - "Ventyx Biosciences, Inc...announced that a late-breaking abstract including new long-term extension (LTE) data from the Phase 2 trial of tamuzimod in ulcerative colitis was presented on October 15, 2024 during the United European Gastroenterology (UEG) Week meeting in Vienna, Austria."

P2 data • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Pharmacokinetics of S1P receptor modulators in the treatment of ulcerative colitis. (PubMed, Expert Opin Drug Metab Toxicol) - "S1P receptor modulators, including ozanimod and etrasimod (both currently on the market) as well as VTX002 (under development), are a new class of drugs for the treatment of moderate to severe ulcerative colitis, inducing and maintaining the remission. Due to its pharmacokinetic features, this class of drugs has certain advantages such as an oral administration, a short half-life, a high volume of distribution, and no immunogenicity. On the other hand, there are risks of cardiological and ophthalmological side-effects, as well as drug-drug interactions risk, that require special attention from the healthcare providers."

Journal • PK/PD data • Review • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease • Ophthalmology • Ulcerative Colitis

EFFICACY AND SAFETY OF THE ORAL SELECTIVE SPHINGOSINE-1-PHOSPHATE-1 RECEPTOR MODULATOR VTX002 IN MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS: RESULTS FROM A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 2 TRIAL (DDW 2024) - P2 | "The S1P1 receptor modulator VTX002 was well-tolerated and statistically superior to placebo for induction of clinical, endoscopic, histologic, HEMI, and symptomatic remission/response at week 13. These positive findings, including a dose-dependent pharmacodynamic reduction in ALC, support phase 3 development of VTX002 for treatment of UC."

Clinical • P2 data • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Immunology • Infectious Disease • Inflammatory Bowel Disease • Macular Edema • Ophthalmology • Ulcerative Colitis

5115: IBD Controlled Trials II (DDW 2024) - "Insights into novel agents including obefazimod, VTX002. MH002, risankizumab, and mirikizumab will be presented. Learning Objectives: Describe safety of oral sphongosine-1-phosphate receptor modulators, Determine safety and efficacy of MH002 and describe its mechanism of action, Describe safety and efficacy of interleukin-23 antagonosists"

Inflammatory Bowel Disease

Ventyx Biosciences Reports First Quarter 2024 Financial Results and Highlights Recent Corporate Progress (GlobeNewswire) - "We recently completed a productive End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) and we expect to conduct a Scientific Advice meeting with the European Medicines Agency (EMA) during the second quarter of this year. We intend to identify a partner or other source of non-dilutive financing to support the pivotal Phase 3 trial of VTX002 in UC....VTX958 (TYK2 Inhibitor): We are evaluating VTX958 in a Phase 2 trial in patients with moderately to severely active Crohn’s disease....The trial’s sole primary endpoint is now the change from baseline in the mean Crohn’s disease activity index (CDAI) score at Week 12. We have completed enrollment in the trial, and we expect to report topline results early in the second half of 2024."

Licensing / partnership • P2 data • Crohn's disease • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Reports Clinical Data for its NLRP3 Inhibitor Portfolio and Provides Pipeline Updates at Virtual Investor Event (GlobeNewswire) - "At our virtual investor event, we will present data from the ongoing Phase 2 open-label extension. We believe these data continue to support the potentially differentiated profile of VTX002 in ulcerative colitis, with robust rates of clinical remission and endoscopic remission observed among participants completing 52 weeks of treatment with VTX002. At least half of the patients in the 60 mg treat-through group achieved clinical remission or endoscopic remission at Week 52. Activities are underway to prepare for a Phase 3 trial. We intend to identify a partner or other source of non-dilutive financing to support the pivotal Phase 3 trial of VTX002 in ulcerative colitis."

Clinical • P2 data • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Announces $100 Million Private Placement of Common Stock (GlobeNewswire) - "Ventyx Biosciences, Inc...announced today that it has entered into a stock purchase agreement for the sale of 11,174,000 shares of its common stock at an offering price of $8.95 per share in a private placement to certain qualified institutional buyers and institutional accredited investors. Gross proceeds of the private placement are expected to be approximately $100 million....Ventyx currently intends to use the net proceeds from the proposed private placement, together with existing cash and cash equivalents, to fund the clinical development of VTX3232, VTX2735, VTX002, and the preclinical development of other programs, research activities and working capital and other general corporate purposes."

Financing • CNS Disorders • Immunology • Inflammatory Bowel Disease

Ventyx Biosciences to Host Virtual Investor Event on March 11, 2024 (Yahoo Finance) - "Ventyx Biosciences, Inc...announced that it will host a virtual investor event on Monday, March 11, 2024 from 11:00 a.m. to 12:30 p.m. ET. The event will provide key clinical updates on our NLRP3 inhibitor portfolio...We will also provide a clinical update from the ongoing open-label extension of the VTX002 Phase 2 trial in ulcerative colitis."

Trial status • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Corporate Progress (Yahoo Finance) - "Activities are underway to support initiation of a Phase 3 trial of VTX002 in UC during the second half of 2024....Ventyx will host a virtual investor event on March 11, 2024, to provide key clinical updates on our NLRP3 portfolio, including...topline results from the Phase 1 trial of VTX3232 in healthy volunteers. We will also provide a clinical update from the ongoing open-label extension of the VTX002 Phase 2 trial in ulcerative colitis."

New P3 trial • P1 data • P2 data • CNS Disorders • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Efficacy and safety of the oral selective sphingosine-1-phosphate-1 receptor modulator VTX002 in moderately to severely active Ulcerative Colitis: results from a randomised, double-blind, placebo-controlled, phase 2 trial (ECCO-IBD 2024) - P2 | "Conclusion The S1P1 receptor modulator VTX002 was well-tolerated and statistically superior to placebo for induction of clinical, endoscopic, histologic, HEMI, and symptomatic remission/response at week 13. These positive findings, including a dose-dependent pharmacodynamic reduction in ALC, support phase 3 development of VTX002 for treatment of UC."

Clinical • P2 data • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences to Present Results of the Phase 2 Trial of VTX002 in Ulcerative Colitis at the 19th Congress of the European Crohn's and Colitis Organisation (ECCO) (GlobeNewswire) - "Ventyx Biosciences, Inc...today announced that data from the Phase 2 trial of VTX002 in ulcerative colitis will be presented during the 19th Congress of the European Crohn's and Colitis Organisation (ECCO) on February 22, 2024, in Stockholm, Sweden."

P2 data • Immunology • Inflammation • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Announces Positive Results from the Phase 2 Trial of VTX002 in Patients with Moderate-to-Severely Active Ulcerative Colitis (GlobeNewswire) - P2 | N=NA | "'The positive Phase 2 data for VTX002 establish a highly attractive profile for an oral agent in moderate-to-severely active UC, with the 60 mg dose achieving a compelling clinical remission rate, an unprecedented rate of complete endoscopic remission, and an excellent safety and tolerability profile,' said William Sandborn...'I would like to thank all of the patients and investigators for their participation in this trial.'"

Media quote • P2 data

Ventyx Biosciences Announces Positive Results from the Phase 2 Trial of VTX002 in Patients with Moderate-to-Severely Active Ulcerative Colitis (GlobeNewswire) - P2 | 213 | NCT05156125 | Sponsor: Oppilan Pharma Ltd | "Topline results are summarized below: 28% of patients on the 60 mg dose and 24% of patients on the 30 mg dose achieved the primary endpoint of clinical remission at Week 13 compared to 11% of patients on placebo (p=0.018 for 60 mg; p=0.041 for 30 mg). 29% of patients on the 60 mg dose and 21% of patients on the 30 mg dose achieved the secondary endpoint of complete endoscopic remission (Mayo endoscopic subscore of 0) at Week 13 compared to 7% of patients on placebo (p=0.001 for 60 mg; p=0.014 for 30 mg)....VTX002 was well tolerated in both dose cohorts, with no treatment-related serious adverse events observed. There were no serious or opportunistic infections. There were no instances of atrioventricular block or bradycardia."

P2 data • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Efficacy and Safety of S1P1 Receptor Modulator Drugs for Patients with Moderate-to-Severe Ulcerative Colitis. (PubMed, J Clin Med) - "Oral administration route, rapidity and reliable safety profile are the main advantages of this class of drugs. The aim of this review is to summarize the available evidence for the efficacy, safety, and pharmacokinetics of ozanimod, etrasimod, and VTX002 in UC."

Journal • Review • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Announces Completion of Enrollment of the Phase 2 Trial of VTX002 in Ulcerative Colitis and the Phase 2 Trial (Bloomberg) - "'I am very proud of our team's execution as we mark another important milestone for Ventyx and for ulcerative colitis and plaque psoriasis patients,' said Dr. William Sandborn...followed by topline results from the Phase 2 SERENITY trial of VTX958 in plaque psoriasis, which are also expected in the fourth quarter of 2023."'"

Media quote

VTX002 Versus Placebo for the Treatment of Moderately to Severely Active Ulcerative Colitis (clinicaltrials.gov) - P2 | N=213 | Active, not recruiting | Sponsor: Oppilan Pharma Ltd | Recruiting ➔ Active, not recruiting

Enrollment closed • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis

Ventyx Biosciences Announces Completion of Enrollment of the Phase 2 Trial of VTX002 in Ulcerative Colitis and the Phase 2 Trial of VTX958 in Plaque Psoriasis (GlobeNewswire) - "Ventyx Biosciences, Inc...announced that the Company has completed patient enrollment in the Phase 2 trial of VTX002 in ulcerative colitis and the Phase 2 SERENITY trial of VTX958 in plaque psoriasis....'We look forward to reporting topline data from the Phase 2 trial of VTX002 in ulcerative colitis early in the fourth quarter of 2023, followed by topline results from the Phase 2 SERENITY trial of VTX958 in plaque psoriasis, which are also expected in the fourth quarter of 2023.'...In addition to the Phase 2 SERENITY trial, Phase 2 trials of VTX958 continue to enroll patients in moderately to severely active Crohn’s disease (HARMONY) and in active psoriatic arthritis (TRANQUILITY), with topline results from both trials expected in 2024."

Enrollment closed • Enrollment status • P2 data • Crohn's disease • Immunology • Inflammation • Inflammatory Bowel Disease • Psoriasis • Psoriatic Arthritis • Ulcerative Colitis

Ventyx Biosciences Reports Fourth Quarter and Full Year 2022 Financial Results and Highlights Recent Corporate Progress (Yahoo News) - "VTX958 (TYK2 Inhibitor): Enrollment is ongoing in the Phase 2 SERENITY trial of VTX958 in moderate to severe plaque psoriasis, the Phase 2 HARMONY trial in moderately to severely active Crohn’s disease and the Phase 2 TRANQUILITY trial in active psoriatic arthritis. Topline data from the Phase 2 SERENITY psoriasis trial are anticipated in Q4 2023. Topline readouts from the Phase 2 HARMONY and Phase 2 TRANQUILITY trials are expected in 2024....VTX002 (S1P1R Modulator): We continue to make significant progress enrolling the Phase 2 trial of VTX002 in moderately to severely active ulcerative colitis. We expect to complete enrollment by mid-2023 and topline results are anticipated in the second half of 2023."

Enrollment status • P2 data • Crohn's disease • Immunology • Inflammatory Bowel Disease • Psoriasis • Psoriatic Arthritis • Ulcerative Colitis

Pharmacokinetics, relative bioavailability, and dose proportionality of a tablet formulation of the sphingosine 1-phosphate-1 receptor modulator VTX002 (formerly OPL-002) (ECCO-IBD 2023) - P2 | "2020; 158(6):S1188. 1"

PK/PD data • Immunology • Inflammatory Bowel Disease • Ulcerative Colitis