We are a clinical-stage biotechnology company committed to engineering a targeted gene therapy to improve the standard treatment for early-stage bladder cancer, which is one of the most prevalent forms of cancer. We have discovered and are developing a biologic agent called inodiftagenevixteplasmid, or inodiftagene, that we believe can deliver a new treatment to patients who have options that are limited in efficacy and problematic in toxicity. Bladder cancer is a disease that typically causes symptoms early in its course and consequently presents the patient and the treating physician with an opportunity to gain control of the malignancy. However, the limitations of existing therapies, developed in the 1970s, often result in a prolonged series of unsuccessful treatments that can end in the radical removal of the bladder.
Our lead product candidate, inodiftagene, is a recombinant DNA construct that will be administered to patients whose therapy for early stage bladder cancer has failed: this is gene therapy for bladder cancer. Preclinical studies and clinical trials completed so far have demonstrated that our product candidate can deliver a lethal gene specifically to bladder cancer cells in a patient’s bladder. Based on our Phase 1 and Phase 2 clinical trial results, we believe our product candidate, inodiftagene, has the potential to improve patient outcomes substantially by delaying or in some cases eliminating disease progression, and consequently may significantly improve patients’ quality of life. Although there can be no assurance, based on our interactions with the U.S. Food and Drug Administration, or FDA, and other international regulators, we also believe the clinical trial program we have accomplished to date provides a foundation for the regulatory pathway to approval for commercialization and wide distribution of our product candidate. The patient population that is the subject of our development program exhibits a significant unmet need for more effective and safe treatments. Our proposed clinical development plan includes two pivotal clinical studies designed to address related but distinct indications that together may provide access to a large share of this affected population. This differentiates us from competitive approaches.
Bladder cancer affects a large population in the United States as well as worldwide. In 2018, it was expected that approximately 81,000 new cases of bladder cancer would be diagnosed in the United States. Nearly 141,000 new cases in the European Union are expected to be diagnosed in 2020. Of these cases, approximately 75% will be non-muscle invasive bladder cancer, or NMIBC. This is the earliest stage in bladder cancer classification. Bladder cancer usually causes symptoms as soon as it develops. Its diagnosis leads to a cycle of cystoscopic evaluation, surgical resection, and medical therapy, that is repeated as treatments fail successively and additional treatments are attempted. The standard medical treatment is Bacillus Calmette-Guerin, or BCG, which is live tuberculosis bacteria administered into the bladder. Most treatments with BCG ultimately fail. The effect of this is tumor recurrence. As a consequence of the prolonged natural history of early presentation and repeated treatment failures, it was estimated that in the United States in 2015, the latest year good estimates were available, about 710,000 patients were living with bladder cancer. Moreover, it is the most costly cancer to treat in the United States, with annual costs that have been estimated at over $4 billion in 2010, and costs of care for an individual nearing $200,000. Despite the need for new approaches to therapy for this disease, no drug has been approved by the FDA for the treatment of NMIBC since 1998. Patients with bladder cancer whose therapies have failed make up a large population that is in desperate need of new therapies.
Our lead product candidate, inodiftagene, is a biological agent designed and formulated to deliver a toxic gene to bladder cells in a manner that results in the gene’s being active only in the bladder tumor with consequent killing of only the malignant cells. The engineered gene has been compounded with an agent that enhances and optimizes the efficiency of its delivery to tissue. In experiments, we have demonstrated the uptake of inodiftagene by 85% of target cells after a single exposure.
We have tested inodiftagene in six clinical trials, three of which involved NMIBC patients, and we have observed substantial anti-tumor activity. The data from the three NMIBC Phase 1/2 and Phase 2 trials demonstrate that inodiftagene causes complete responses in 33% of bladder cancer patients with unresected measurable tumors; that one-year and two-year recurrence-free survivals are 46% and 33%, respectively; and that we can administer inodiftagene with BCG, the standard of care for NMIBC, with recurrence-free outcomes of 95% and 78% at three and six months, respectively.
We believe that these studies strongly support the potential for inodiftagene to provide new therapies for NMIBC patients. There are two distinct populations of unmet need in the field of NMIBC treatment: those patients for whom two treatments with standard BCG have failed (who are termed BCG-unresponsive), and those for whom one BCG treatment has failed. Accordingly, we are conducting two pivotal clinical trials in NMIBC patients: a single-arm study of inodiftagene in patients with BCG-unresponsive disease, and a randomized trial in patients whose disease has recurred after a single course of standard BCG therapy. Both of these studies have been reviewed by the FDA and other international regulatory bodies. The FDA has granted our program Fast Track designation for regulatory review. Additionally, the Phase 3 study has been granted a special protocol assessment, or SPA, by the FDA. This is a competitive area for drug development, and other companies are seeking to bring treatments for NMIBC to regulatory submission and to market. Although there are several planned and ongoing trials by our competitors that address the BCG-unresponsive patient population, in our view, our clinical development program in patients whose disease recurs after a first course of BCG treatment differentiates us from our competitors. Based on market data, including a commissioned independent research study, we believe the potential market for inodiftagene, should it achieve regulatory approval for both indications in the United States, the European Union and Japan, is over $1.5 billion.
We believe that the inodiftagene development program in early-stage bladder cancer may potentially allow us to meet the regulatory requirements for the development of a new therapy for this malignancy. ---
We were incorporated on September 22, 2011 under the laws of the State of Israel for the purpose of a reincorporation merger, or Reincorporation, which merged BioCancell Therapeutics Inc., or BTI, with and into a wholly-owned subsidiary of BioCancell Ltd., or BioCancell. BTI was incorporated in the United States under the laws of the State of Delaware on July 26, 2004, and commenced operations on October 1, 2004. The Reincorporation was consummated on August 14, 2012, and BTI survived as a wholly-owned subsidiary of BioCancell until it was formally dissolved in the State of Delaware on December 28, 2012. In August 2018, BioCancell changed its name to Anchiano Therapeutics Ltd. Our principal executive offices are located at 1/3 High-Tech Village, Givat Ram, P.O. Box 392649, Jerusalem, 9139102 Israel and our telephone number is +972 (2) 548-6555. Our website address is www.anchiano.com.展开