Lipax (proliposomal intravesical paclitaxel) / TesoRx, Huons - LARVOL DELTA

PHASE 1/2A TWO YEAR RECURRENCE FREE SURVIVAL AND MARKER LESION RESPONSE RATE FOR PROLIPOSOMAL PACLITAXEL (TSD-001) IN INTERMEDIATE RISK NMIBC (SUO 2021) - P1/2 | "In patients with recurrent, Ta, LG NMIBC, intravesical administration of TSD-001 demonstrated no DLT, and it was found to be well tolerated with no significant reduction in urinary HR-QOL. Persistence of paclitaxel in the voided urine was observed up to 48 hours post instillation. The MLRR in pretreated and recurrent NMIBC patients was 63%."

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

AAV gene therapy for Tay-Sachs disease. (PubMed, Nat Med) - "Patient TSD-001 was treated at 30 months with an equimolar mix of AAVrh8-HEXA and AAVrh8-HEXB administered intrathecally (i.t.), with 75% of the total dose (1 × 10 vector genomes (vg)) in the cisterna magna and 25% at the thoracolumbar junction...TSD-002 developed anticonvulsant-responsive seizures at 2 years of age. This study provides early safety and proof-of-concept data in humans for treatment of patients with TSD by AAV gene therapy."

Journal • CNS Disorders • Epilepsy • Gene Therapies • Genetic Disorders • Metabolic Disorders

Proliposomal Intravesical Paclitaxel for Treatment of Low-Grade, Stage Ta, Non Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P1/2; N=15; Completed; Sponsor: Lipac Oncology LLC; Active, not recruiting ➔ Completed; Trial completion date: Aug 2022 ➔ Aug 2021

Clinical • Trial completion • Trial completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urethral Cancer • Urology • Urothelial Cancer

LIPAC Oncology to Present Two-year Data for Phase 1/2a Study of LiPax at the 22nd Annual Meeting of the Society of Urologic Oncology (PRNewswire) - "LIPAC Oncology...announced it will present two-year follow-up data for the Phase 1/2a study of LiPax in patients with non-muscle invasive bladder cancer (NMIBC) at the 22nd Annual Meeting of the Society of Urologic Oncology, which will be held in Orlando, Florida, and virtually on December 1-3, 2021."

P1/2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer

LIPAC Oncology Secures Broad Patent Protection for Proliposomal Paclitaxel Compositions Formulated for Delivery to the Bladder and Ureter for the Treatment of Solid Tumor Carcinomas (PRNewswire) - "LIPAC Oncology LLC...today announced the U.S. Patent and Trademark Office issued a Notice of Allowance of LIPAC's U.S. patent application for its next generation, proliposomal paclitaxel compositions formulated for delivery to the bladder and ureter to treat solid tumor carcinomas....This eventual U.S. patent, with an expected patent term to 2037, is a significant addition to LIPAC's worldwide patent portfolio, which includes formulation patents granted in the European Union, Japan, China and other countries...'We look forward to advancing our Phase 2b clinical trial of LiPax...for the treatment of low-intermediate risk non-muscle invasive bladder cancer.'"

New P2 trial • Patent • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

[VIRTUAL] PHASE 2 MARKER LESION RESULTS FOR PROLIPOSOMAL PACLITAXEL (LIPAX) IN INTERMEDIATE RISK NMIBC (SUO 2020) - P1/2 | "Introduction: TSD-001 (LiPax) is a pro-liposomal paclitaxel formulation specifically designed for intra-cavitary administration... In patients with highly recurrent, low grade, Ta NMIBC, intravesical administration of LiPax demonstrated no dose limiting toxcicity. Urinary HR-QOL remained unchanged after up to 8 intravesical instillations. Persistence of paclitaxel in the voided urine at therapeutic concentrations up to 72 hours after instillation were observed."

P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

LIPAC Oncology Announces Successful Type B Meeting with the U.S. FDA for the Study of LiPax for Non-Muscle Invasive Bladder Cancer (PRNewswire) - “LIPAC Oncology LLC today announced the conclusion of a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to discuss Phase 2b/3 clinical trials of LiPax in patients with low-grade highly recurrent non-muscle invasive bladder cancer (NMIBC). The feedback from the FDA provides a clear path for LIPAC Oncology to initiate the Phase 2b trial for LiPax in the second half of 2021.”

FDA event • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer

LIPAC Oncology Announces Two-Year Recurrence Free Survival Data for Phase 1/2a Study of LiPax in Patients With Non-Muscle Invasive Bladder Cancer (PRNewswire) - P1/2a, N=15; NCT03081858; Sponsor: Lipac Oncology LLC; “LIPAC Oncology LLC…announced the positive results from the two-year follow-up for TD-001, its Phase 1/2a study of LiPax in patients with non-muscle invasive bladder cancer (NMIBC) who have undergone transurethral resection of bladder tumor (TURBT). Results demonstrate a recurrence free survival (RFS) rate of 83% compared to 49% for current standard of care therapies. At both 12 and 24 months, LiPax was well tolerated with no change in patient reported urinary health related quality of life, no dose limiting toxicity (grade 3 or greater severity adverse events), and no evidence of systemic exposure to paclitaxel, the active product ingredient….The U.S. Food and Drug Administration (FDA) has agreed on Phase 2b and Phase 3 trial designs for LiPax, with the Phase 2b trial expected to initiate in Q4 2021.”

New P2b trial • P1/2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Urothelial Cancer

Proliposomal Intravesical Paclitaxel for Treatment of Low-Grade, Stage Ta, Non Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P1/2; N=15; Active, not recruiting; Sponsor: Lipac Oncology LLC; Recruiting ➔ Active, not recruiting; Trial completion date: May 2022 ➔ Aug 2022; Trial primary completion date: May 2020 ➔ Aug 2020

Clinical • Enrollment closed • Trial completion date • Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urethral Cancer • Urology • Urothelial Cancer

[VIRTUAL] PHASE 1 OUTCOMES OF A NOVEL THIRD GENERATION LIPOSOMAL PACLITAXEL FORMULATION (TSD-001) IN LOW-INTERMEDIATE RISK NON-MUSCLE INVASIVE BLADDER CANCER (NMIBC) PATIENTS (AUA 2020) - "NMIBC patients exposed to escalating TSD-001 dose (10-540 mg paclitaxel) until maximum deliverable dose (540 mg) demonstrated no DLT.  Voiding function and bother were unchanged from baseline to completion.  There was no evidence of systemic paclitaxel exposure based on a valid bioanalytical assess.  No evidence of TCCa recurrence  has been observed at a mean follow-up of 12 months.  TSD-001 delivers high urinary concentrations of paclitaxel with no measurable systemic exposure, and is very well tolerated in NMIBC patients. Source of Funding: Lipac Oncology, LLC"

Clinical • P1 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Proliposomal Intravesical Paclitaxel for Treatment of Low-Grade, Stage Ta, Non Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: Lipac Oncology LLC; Trial completion date: Jan 2022 ➔ May 2022; Trial primary completion date: Jan 2020 ➔ May 2020

Clinical • Trial completion date • Trial primary completion date

PHASE 1 OUTCOMES OF A NOVEL THIRD GENERATION LIPOSOMAL PACLITAXEL FORMULATION (TSD-001) IN PATIENTS WITH LOW-INTERMEDIATE RISK NON-MUSCLE INVASIVE BLADDER CANCER (NMIBC) (SUO 2019) - "NMIBC patients exposed to escalating TSD-001 dose (10-540 mg paclitaxel) until maximum deliverable dose (540 mg) demonstrated no DLT. Voiding function and bother were unchanged from baseline to completion. There was no evidence of systemic paclitaxel exposure based on a valid bioanalytical assess."

Clinical • P1 data

LIPAC Oncology announces successful completion of phase 1 bladder cancer clinical study and initiation of phase 2A study (PRNewswire) - "LIPAC Oncology LLC...announced the successful completion of a Phase 1 clinical study and progress in a Phase 2A study to further investigate TBC-1002 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC). Phase 1 study results showed escalating doses of TBC-1002 to be well tolerated with no dose limiting toxicity and no irritative voiding (cystitis) symptoms....LIPAC commenced a Phase 2A marker lesion clinical study to establish efficacy responder rates within 16 weeks, which predict long-term (two-year) recurrence free survival rates. Efficacy data from this open label study is expected as soon as November 2019 with the study results available in the first quarter of 2020."

P1/2 data • Trial completion

LIPAC Oncology and Huons Co. announce exclusive licensing agreement for TSD-001 in Korea (PRNewswire) - “LIPAC Oncology LLC and Huons Co., Ltd today announced that they have entered into an exclusive licensing agreement to develop, manufacture and commercialize TSD-001 for all indications in Korea. Huons is making an upfront payment and future milestone payments to LIPAC based on specific development, regulatory and commercial milestones. Huons will also make double-digit royalty payments based on sales. TSD-001 is a proliposomal intravesical paclitaxel formulation…in the treatment of non-muscle invasive bladder cancer (NMIBC).”

Licensing / partnership

"#LIPACOncology and Huons Co. Announce Exclusive #LicensingAgreement for #TSD001 in Korea https://t.co/goYjCRaqhp" (@1stOncology)

Proliposomal Intravesical Paclitaxel for Treatment of Low-Grade, Stage Ta, Non Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: TesoRx Pharma, LLC; Trial completion date: Jun 2019 ➔ Jan 2022; Trial primary completion date: Jun 2019 ➔ Jan 2020

Clinical • Trial completion date • Trial primary completion date