On October 16, 2023 Theralase reported an update to its Phase II Bacillus Calmette Guérin ("BCG")-Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") (with or without resected Ta / T1 papillary disease) clinical study ("Study II") (Press release, Theralase, OCT 16, 2023, View Source [SID1234636147]).
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To date, Theralase has enrolled and provided the primary Study II Treatment for 62 patients.
The Study II Endpoints have been defined as:
Primary: Efficacy – Defined as Complete Response ("CR") rate at any point in time (CR is defined as at least one of the following:
Negative cystoscopy and negative (including atypical) urine cytology
Positive cystoscopy with biopsy-proven benign or low-grade NMIBC and negative cytology
Negative cystoscopy with malignant urine cytology, if urothelial cancer is suspected in the upper tract or prostatic urethra and random bladder biopsies are negative)
Secondary: Duration of CR – Defined as 12 months post initial CR assessment
Tertiary: Safety – Defined as the incidence and severity of Adverse Events ("AEs"), Grade 4 or higher, directly related to the Study Drug or Study Device, that do not resolve within 450 days post primary Study II Treatment; whereby: Grade 1 = Mild, Grade 2 = Moderate, Grade 3 = Severe, Grade 4 = Life-threatening or disabling, Grade 5 = Death.
In addition, survival surveillance for all patients, who achieve a CR or Indeterminate Response ("IR") (negative cystoscopy and positive urine cytology, without confirmatory negative bladder cancer biopsies) at 450 days and remain in Study II will be monitored past 450 days.
In performance to the primary, secondary and tertiary endpoints, refer to the clinical data below.
Achieved Primary Objective (n) Achieved Primary Objective (%) Achieved Secondary Objective (n) Achieved Secondary Objective (%) Achieved Tertiary Objective (n) Achieved Tertiary Objective (%)
Complete Response ("CR") 37 65% 14 33% 57 100%
Indeterminate Response ("IR") 5 9% 2 5% 0 0%
Total Response (CR and IR) 42 74% 16 38% 57 100%
Evaluable Patients 57 42 57
Note: Evaluable patients are defined as patients who have been assessed by a principal investigator during a particular assessment visit.
The CR, IR and Total Responders are detailed below by assessment visit.
TLD 1433-2 Clinical Study (Evaluable Patients)
Assessment 90 Day 90 Day 180 Day 180 Day 270 Day 270 Day 360 Day 360 Day 450 Day 450 Day
# % # % # % # % # %
Complete Response ("CR") 33 58% 31 56% 23 46% 16 36% 14 33%
Indeterminate Response ("IR") 4 7% 8 15% 6 12% 2 4% 2 5%
Total Responders (CR and IR) 37 65% 39 71% 29 58% 18 40% 16 38%
Total Evaluated 57 55 50 45 42
On August 1, 2020, the Company optimized the Study II Treatment. For patients that received the optimized Study II Treatment the CR, IR and Total Responders are detailed below by assessment visit.
TLD 1433-2 Clinical Study (Evaluable Patients) (Optimized: Post August 1, 2020)
Assessment 90 Day 90 Day 180 Day 180 Day 270 Day 270 Day 360 Day 360 Day 450 Day 450 Day
# % # % # % # % # %
Complete Response ("CR") 29 64% 28 62% 20 50% 13 37% 11 34%
Indeterminate Response ("IR") 3 7% 7 16% 5 13% 2 6% 2 6%
Total Responders (CR and IR) 32 71% 35 78% 25 63% 15 43% 13 41%
Total Evaluated 45 45 40 35 32
Theralase is currently working with its clinical study sites in Canada and the United States to compile information requested by the Food and Drug Administration ("FDA") for re-submission of a pre-Break Through Designation ("BTD").
If the pre-BTD submission is successful, this could lead to BTD approval.
Arkady Mandel, MD, PhD, DSc, Chief Scientific Officer of Theralase stated, "Theralase is delighted in its latest clinical data analysis. The Theralase RuvidarTM-based Anti-Cancer Therapy ("ACT") has shown remarkable single-agent activity by proving to be safe and effective on a very difficult to treat BCG-Unresponsive patient population that has been diagnosed with high-grade NMIBC CIS, with or without resected Ta / T1 papillary tumours. These patients have failed the standard of care, such as BCG therapy and a large majority of them have failed treatment with various modern immunotherapy drugs. Theralase has been able to demonstrate strong efficacy in the form of a CR or IR, with a well-tolerated safety profile, after predominately one treatment. This ACT technology, pending successful regulatory approval and commercialization, will be very attractive to patients, uro-oncologists and the insurance companies that insure these patients."
Mr. Roger DuMoulin-White, BSc, P.Eng, Pro.Dir, President and Chief Executive Officer of Theralase stated, "To date, the Theralase Study II clinical data has demonstrated best-in-class performance for a single agent, providing high efficacy, durable response and a high safety profile, with no serious adverse events directly related to RuvidarTM or the TLC-3200 Medical Laser System. Theralase hopes to complete patient enrollment with accompanying administration of the primary Study II Treatment by year end 2024. If successful, this will allow the Company the ability to complete assessment of the primary, secondary and tertiary endpoints of these patients by 2026. Based on the clinical data to date, Theralase is investigating potential partnerships for commercialization, financing and distribution of this ACT technology on an international basis. Theralase looks forward to commercializing this world-class technology for the benefit of all shareholders."