BioCancell

Compassionate Use
BC-819 was introduced intravesically into the bladders of two patients suffering recurrent superficial TCC of the bladder, refractory to all the commonly used treatments. Nearly complete destruction of the tumor was determined by video imaging in the two patients after 6 weekly treatments. No trace of the plasmid could be detected in the bloodstream of the patients, and no adverse event or toxicity was detected, a strong indication of the safety of the treatment. As a result of the treatment, the two patients, who were candidates for bladder removal (radical cystectomy), have maintained their functioning bladders, without recurrence or with a considerable reduction of recurrence.

Phase I/IIa
A Phase I/IIa clinical trial was launched in patients suffering from refractory superficial bladder cancer on January 15, 2006. The trial took place at two medical centers in Israel under the supervision of the Israeli Ministry of Health and in accordance with principles agreed upon with the FDA. It was designed to determine the optimal dose and assess the safety and preliminary efficacy of BC-819 complemented with PEI (a transfection enhancer) given as intravesical infusions into the bladder of patients with superficial bladder cancer expressing the H19 gene, who had previously failed at least one standard treatment type. In the obligatory part of the trial each patient received six weekly treatments with BC-819 whereas in the elective part of the trial patients responding well to the treatment were offered up to nine additional monthly maintenance treatments.

Eighteen patients were enrolled in the trial and split into five groups, with each group receiving a higher dosage than the previous. Escalating doses consisted of 2, 4, 6, 12 and 20mg of BC-819. All patients had failed at least one standard treatment type, with a third failing at least two. The mean number of previous tumor recurrences after treatment in patients entering the Trial was 5.

On September 24, 2007, the successful results of the trial were announced.

- Safety: No Serious Adverse Events relating to the treatment were detected at any dose given.

- Optimal Dose: There was no dose-limiting toxicity, so the optimal dose to be used in Phase IIb is 20mg, which was the maximum dose administered to patients in Phase I/IIa.

- Primary Efficacy: 72% of patients responded to treatment. In total, 56% of patients remained free of tumors and 44% experienced tumor marker ablation or reduction. One patient showed disease progression, while 17 did not.

Phase IIb
Based on the positive results received in Phase I/IIa, BioCancell commenced a Phase IIb clinical trial in the first quarter of 2008. The aim of the phase is to test the efficacy and safety of BC-819 at a dose of 20mg. The treatment of 33 patients is taking place in 6 medical centers in Israel and one in the U.S., pursuant to an IND approval from the FDA.

Compassionate Use
A 69-year old patient has been the subject of two compassionate use tests of BC-819. In the first, she was given 10 courses of the drug intraperitoneally, in doses ranging from 80mg to 120mg. No severe adverse events were reported, tumor growth was arrested, the levels of cancer marker CA-125 dropped by approx 50%, the number of cancerous cells in the patient’s ascitic fluid dropped, and the ascitic fluid level itself was reduced to nothing. The patient and her doctors reported significant clinical improvement.

In a second test that commenced in Q1 2008, the patient has been given a combination of BC-819 and Taxtere (a derivative of chematherapeutic drug Taxol). Interim results show an additional drop in the levels of CA-125.

Phase I
BioCancell has received FDA approval for the protocol of a Phase I clinical trial in ovarian cancer, expected to commence during 2008.

Phase I
BioCancell has received FDA approval for the protocol of a Phase I clinical trial in pancreatic cancer, expected to commence during 2008, primarily at the NCI-designated Massey Medical Center in Virginia, in conjunction with the Virginia Biosciences Commercialization Center. A $950,000 grant by the BIRD Foundation has been approved for the performance of this trial.

Compassionate Use
One patient with colon cancer metastasising into the liver was treated with direct injection of plasmid into the tumor. After two weeks, promising results of tumor necrosis was observed through imaging techniques.

R&D Status