Vesique (nitroxoline) / Asieris - LARVOL DELTA

Phase II study of oral APL-1202 plus tislelizumab or tislelizumab alone as neoadjuvant therapy in patients with muscle-invasive bladder cancer (MIBC). (ASCO-GU 2025) - P1/2 | "Clinical Trial Registration Number: NCT04813107 Background: Interim results of the randomized phase II trial of neoadjuvant tislelizumab +/- oral APL-1202 (nitroxoline) revealed promising pathological complete response (pCR) rates and met prespecified thresholds for study expansion (ASCO GU 2024). Neoadjuvant A+T in cisplatin-ineligible patients prior to RC was safe. Evaluation of the efficacy in MIBC is complicated by the large subset of patients enrolled retrospectively determined to have *pCR rate is calculated using UMVUE method when the final sample size of Simon's two-stage is changed."

Clinical • IO biomarker • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • PD-L1

Asieris Pharmaceuticals Phase II Clinical Data Selected for the American Society of Oncology Symposium [Google translation] (Eastmoney.com) - P2 | N=79 | ANTICIPATE (NCT04813107) | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris | "Yahong Pharmaceutical...issued an announcement, announcing that the data of its Phase II clinical trial of oral APL-1202 combined with tislelizumab for the neoadjuvant treatment of muscle-invasive bladder cancer has been selected for the 2025 American Society of Clinical Oncology Genitourinary Tumors Symposium and will be published in the form of a poster...In the analysis in accordance with the study protocol, the pathological complete remission rate (pCR) of the combination group and the monotherapy group was 41% and 20%, respectively. Especially in the PD-low expression subgroup, the combination group showed a stronger positive efficacy signal. In addition, the incidence of treatment-related adverse events (TRAEs) in the combination group and the monotherapy group was 59% and 44%, respectively, and 94% of TRAEs were ≤CTCAE grade 2."

P2 data • Bladder Cancer

YHGT-APL1501-NHS-103: A Study of APL-1501 Extended Release Capsules Compared to APL-1202 Immediate Release Tablets in Healthy Volunteers (clinicaltrials.gov) - P1 | N=12 | Terminated | Sponsor: Syneos Health | N=32 ➔ 12 | Trial completion date: Oct 2024 ➔ Jul 2024 | Not yet recruiting ➔ Terminated; Study terminated by sponsor due a business decision and development strategy.

Enrollment change • Trial completion date • Trial termination

Asieris and CDC Sign Investigational Product Supply Agreement for APL-1202 in Free-Living Amoeba Infections Treatment under Expanded Access Investigational New Drug Program (PRNewswire) - "Asieris Pharmaceuticals...announced the signing of an investigational drug supply agreement under the Expanded Access Investigational New Drug (IND) Program with the Centers for Disease Control and Prevention (CDC) in the United States for the independently developed APL-1202 (nitroxoline), subject to evaluation by CDC experts, to be used for treating Free-living Amoebae (FLA) infections....'We look forward to collaborating closely with the CDC in the future, sharing clinical experiences of treating FLA infections with APL-1202 to meet the unmet needs of patients. We will continue to uphold our company's mission of delivering innovative treatments to patients.'"

Commercial • Infectious Disease

Encouraging Efficacy Signals Achieved in Phase II Clinical Trial of Asieris APL-1202 in Combination with PD-1 Inhibitor Tislelizumab for Neoadjuvant Therapy in Muscle-Invasive Bladder Cancer (Asieris Pharma Press Release) - P1/2 | N=79 | ANTICIPATE (NCT04813107) | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris | "Asieris Pharmaceuticals... announced that the phase II clinical trial of oral APL-1202 in combination with the PD-1 inhibitor tislelizumab for neoadjuvant treatment of muscle-invasive bladder cancer (MIBC) has been completed and achieved encouraging efficacy signals....Results from the Phase II clinical trial indicate that APL-1202 in combination with tislelizumab shows encouraging efficacy signals with acceptable safety characteristics. In the population diagnosed with MIBC by central pathology at baseline (modified evaluable analysis set, mEAS), the pCR rate was 41% (9/22) for the APL-1202 combined with tislelizumab group and 20% (4/20) for the tislelizumab monotherapy group. Detailed data from this clinical trial will be disclosed at upcoming academic conferences."

P2 data • Bladder Cancer

Yahong Pharmaceuticals SH688176: Progress and prospects of the trial of oral APL-1202 combined with tislelizumab in the neoadjuvant treatment of MIBC [Google translation] (Sina Corp) - "The interim analysis results of the Phase II clinical trial of APL-1202 oral combined with tislelizumab for the neoadjuvant treatment of muscle-invasive bladder cancer (MIBC) have been disclosed, and all subjects of the Phase II clinical trial will be enrolled in January 2024. It is currently progressing as planned, and it is expected that the top-line data of the Phase II trial will be read out in the third quarter of 2024."

P2 data • Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A Study of APL-1501 Extended Release Capsules Compared to APL-1202 Immediate Release Tablets in Healthy Volunteers (clinicaltrials.gov) - P1 | N=32 | Not yet recruiting | Sponsor: Syneos Health

New P1 trial

APL-1202 enhances anti-tumor immune response through induction of cellular stress signaling and immunogenic cell death pathway in cancer cells (AACR 2024) - P1/2 | "APL-1202 may improve the efficacy of immune-checkpoint blockade therapies in bladder cancer patients by enhancing cancer cell immunogenicity and subsequent anti-tumor effector immune cell processes, particularly in tumors with deficits in antigen-specific immunity."

Immunogenic cell death • IO biomarker • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • CALR • CD8 • CXCL8 • GLI2 • HMGB1 • IL10 • IL6

#GU24 #bladdercancer highlights - AMBASSADOR @apolo_andrea (adjuvant pembrolizumab for high-risk muscle-invasive #urothelialcarcinoma following surgery), EV302 @MichvdHeijden (first-line EV+pembrolizumab for metastatic UC), Dato-DXd as salvage therapy, Pembro + cabozantinib @rohitjainMD as first-line therapy for cis/plat-ineligible/refusing metastatic UC, APL-1202 (oral MetAP2 inhibitor…

Oral APL-1202 in combination with tislelizumab as neoadjuvant therapy in patients with muscle-invasive bladder cancer (MIBC): Interim analysis of ANTICIPATE phase II trial. (ASCO-GU 2024) - P1/2 | "Clinical Trial Registration Number: NCT04813107 Background: APL-1202 (nitroxoline) is a reversible and orally available MetAP2 inhibitor with anti-angiogenic and anti-tumor activities. The pCR rates in both group 1 (APL-1202 plus tislelizumab) and group 2 (tislelizumab) exceeded thresholds to trigger expansion to stage 2 of the 2-stage design. The activity and safety of neoadjuvant APL-1202 plus tislelizumab support further evaluation of this novel regimen (NCT04813107). Clinical trial information: NCT04813107."

Clinical • Combination therapy • IO biomarker • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • METAP2

Asieris Presents for the First Time Interim Analysis Data of Oral APL-1202 in Combination with PD-1 Inhibitor Tislelizumab for Neoadjuvant Treatment of Muscle-Invasive Bladder Cancer at 2024 ASCO-GU (PRNewswire) - P2 | N=79 | ANTICIPATE (NCT04813107) | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris | "Asieris Pharmaceuticals...announced the first-time release of interim analysis data for the Phase II clinical trial of oral APL-1202 in combination with the PD-1 inhibitor tislelizumab for neoadjuvant treatment of muscle-invasive bladder cancer (MIBC). Release of the interim analysis data was made in the form of a rapid oral presentation abstract...at the 2024 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU). Patient enrollment of the trial was recently completed....The interim analysis results of the Phase II clinical trial showed that in the APL-1202 and tislelizumab combination treatment group, out of 18 subjects evaluated, 7 achieved pCR, accounting for 7/18 (39%). In the tislelizumab monotherapy group, out of 14 subjects evaluated, 3 achieved pCR, accounting for 3/14 (21%)."

P2 data • Bladder Cancer

Asieris will Present For the First Time Interim Analysis Data of Oral APL-1202 in Combination with PD-1 Inhibitor Tislelizumab for Neoadjuvant Treatment of Muscle-Invasive Bladder Cancer at 2024 ASCO-GU (PRNewswire) - "Asieris Pharmaceuticals...announced that the interim analysis data from the Phase II clinical trial of oral APL-1202 in combination with PD-1 inhibitor tislelizumab as neoadjuvant therapy for muscular invasive bladder cancer (MIBC) will be presented for the first time in the form of a rapid oral presentation abstract (Abstract No. 632) at the 2024 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU)."

P2 data • Bladder Cancer

Two Asieris Studies in the Field of Bladder Cancer Selected for Oral and Poster Presentations at the 2024 American Society of Clinical Oncology Genitourinary Cancer Symposium, Potentially Offering New Disease Management Options for Patients (Asieris Pharma Press Release) - "Asieris Pharmaceuticals...announced that data from two studies in the field of bladder cancer will be presented at the 2024 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO-GU)....The interim analysis of ANTICIPATE data, to be presented at the 2024 ASCO-GU, indicates that the combination of APL-1202 with the PD-1 inhibitor, tislelizumab, shows promising therapeutic potential as neoadjuvant treatment for MIBC patients."

P1/2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses of APL-1501 Extended Release (ER) Capsules Compared to APL-1202 Immediate Release (IR) Tablets in Healthy Volunteers (clinicaltrials.gov) - P1 | N=48 | Not yet recruiting | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris

New P1 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Asieris Announces Positive Interim Analysis Results in Phase II Clinical Trial of Oral APL-1202 in Combination with Tislelizumab, a PD-1 inhibitor, as Neoadjuvant Therapy for Muscle Invasive Bladder Cancer (PRNewswire) - "Asieris Pharmaceuticals...announced that the clinical trial of oral APL-1202 in combination with PD-1 inhibitor Tislelizumab as neoadjuvant therapy for muscular invasive bladder cancer (MIBC) completed the phase II interim analysis with positive results. Detailed results will be released on academical meeting."

P1/2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ANTICIPATE phase I: Oral APL-1202 in combination with tislelizumab as neoadjuvant therapy in patients with muscle-invasive bladder cancer (MIBC). (ASCO 2023) - P1/2 | "Background: APL-1202 (nitroxoline) is a reversible and orally available MetAP2 inhibitor with anti-angiogenic and anti-tumor activities. APL-1202 plus tislelizumab was well tolerated. The RP2D of APL-1202 was identified as 1125 mg daily dose. Early efficacy signals were observed including pathologic downstaging to < pT2 (5/8, 62.5%) and pT0 (1/8, 12.5%)."

Clinical • Combination therapy • IO biomarker • Bladder Cancer • Genito-urinary Cancer • Hepatology • Liver Failure • Oncology • Solid Tumor • Urothelial Cancer • PD-L1

Asieris’ progress in MIBC receives renewed attention with its inclusion in ASCO 2023 (PRNewswire) - P1/2 | N=79 | ANTICIPATE (NCT04813107) | Sponsor: Jiangsu Yahong Meditech Co | "A total of 9 subjects were enrolled In the Phase I stage of the trial, and the clinical data showed oral APL-1202 in combination with tislelizumab was well tolerated. No dose-limiting toxicities were observed at either 375 mg, 750 mg, or 1125 mg daily dose of APL-1202 and the recommended phase II dose (RP2D) was identified as 1125 mg daily dose. Treatment-related adverse events (TRAE) were observed in 6 patients. All AEs were grade 1, except 1 case of CTCAE grade 2 T wave abnormality on ECG and 1 case of CTCAE grade 3 liver dysfunctions....The trial is actively recruiting for Phase II with the first patient enrolled in December 2022."

P1 data • Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Non-chemo neoadjuvant combo explored in MIBC (Urology Times) - "Matthew Galsky, MD, is a principal investigator on the trial, recruiting patients at Mount Sinai Medical Center in New York City."

Asieris Enrolled First Patient for its Phase II Clinical Study of Oral APL-1202 in Combination with Tislelizumab, a PD-1 inhibitor, as Neoadjuvant Therapy for Muscle Invasive Bladder Cancer (PRNewswire) - "Asieris Pharmaceuticals...announced that the first patient enrolled in phase II clinical study of oral APL-1202 in combination with PD-1 inhibitor Tislelizumab as neoadjuvant therapy for muscular invasive bladder cancer (MIBC)....This is an open-label, multi-center Phase I/II clinical study with the following objectives: to evaluate the safety in MIBC patients, to determine the recommended Phase 2 dose (RP2D), and to assess efficacy as neoadjuvant therapy for MIBC."

Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology

ANTICIPATE: A phase I/II, open-label, multicenter study to evaluate the safety and efficacy of oral APL-1202 in combination with tislelizumab compared to tislelizumab alone as neoadjuvant therapy (NAC) in patients with muscle invasive bladder cancer (MIBC). (ASCO 2022) - P1/2 | "Background: Standard treatment for muscle invasive bladder cancer (MIBC) is radical cystectomy (RC) and administration of NAC in patients who are eligible to receive cisplatin...APL-1202 (nitroxoline) is a reversible and orally available MetAP2 inhibitor with anti-angiogenic and anti-tumor activities...In phase II patients will be randomly assigned to group 1 (APL-1202 + tislelizumab) or group 2 (tislelizumab only), stratified by PD-L1 expression. The IND has been approved by FDA and NMPA, and the study execution is under preparation both in US and China."

Clinical • Combination therapy • IO biomarker • New P1/2 trial • P1/2 data • Bladder Cancer • Genito-urinary Cancer • Immune Modulation • Inflammation • Oncology • Solid Tumor • Urothelial Cancer • PD-L1

Asieris to present a study protocol of APL-1202 in combination with BeiGene's tislelizumab as neoadjuvant therapy（NAC）for muscle invasive bladder cancer (MIBC) patients at 2022 ASCO annual meeting (PRNewswire) - "Asieris Pharmaceuticals (688176.SH)...announced that ANTICIPATE Study will be presented at the 2022 American Society of Clinical Oncology (ASCO) annual meeting (3-7 June) in Chicago. This is a study of oral APL-1202 in combination with BeiGene's tislelizumab compared to tislelizumab alone as NAC in patients with MIBC."

Clinical protocol • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer

Ascertain: A Phase 3 Study to Evaluate the Safety and Efficacy of APL-1202 as a Single-agent Oral Treatment Versus Intravesical Instillation of Epirubicin Hydrochloride in naïve Intermediate-risk NMIBC Patients (clinicaltrials.gov) - P3 | N=800 | Recruiting | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris | Not yet recruiting ➔ Recruiting

Enrollment open • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

A Study to Evaluate the Safety and Efficacy of Oral APL-1202 in Combination With Tislelizumab Compared to Tislelizumab Alone as Neoadjuvant Therapy in Patients With Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P1/2 | N=79 | Recruiting | Sponsor: Jiangsu Yahong Meditech Co., Ltd aka Asieris | Not yet recruiting ➔ Recruiting | Initiation date: Sep 2021 ➔ Dec 2021

Combination therapy • Enrollment open • IO biomarker • Trial initiation date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • PD-L1 • TMB

Asieris Announces the World's First Patient Dose Administered in Combination of Asieris'APL-1202 and BeiGene's Tislelizumab as Neoadjuvant Therapy for MIBC Patients (PRNewswire) - "Asieris Pharmaceuticals...announced that the world's first patient dose has been administered in U.S. for its oral APL-1202 in combination with BeiGene's tislelizumab as neoadjuvant therapy in patients with muscle invasive bladder cancer (MIBC)....This is an open-label, multi-center Phase I/II clinical study with the following objectives: to evaluate the safety in MIBC patients; to determine the recommended Phase 2 dose (RP2D), and to assess efficacy as neoadjuvant therapy for MIBC."

Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

The CDE Approved IND Application to Investigate Combination of Asieris’ APL-1202 and BeiGene’s Tislelizumab as Neoadjuvant Therapy for MIBC Patients (PRNewswire) - “Asieris Pharmaceuticals (Asieris) today announced that the Center for Drug Evaluation (CDE) NMPA has approved the Investigational New Drug (IND) application of oral APL-1202 in combination with BeiGene's tislelizumab as neoadjuvant therapy in patients with muscle invasive bladder cancer (MIBC). This is an open-label, multi-center Phase I/II clinical study with the following objectives: to evaluate the safety in MIBC patients; to determine the recommended Phase 2 dose (RP2D), and to assess efficacy as neoadjuvant therapy for MIBC.”

New P1/2 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer