Anchiano Therapeutics Announces Initiation of Its Pivotal Codex Study of Inodiftagene Vixteplasmid in Patients with Non-Muscle-Invasive Bladder Cancer

By GlobeNewswire,  December 18, 2018, 08:30:00 AM EDT

CAMBRIDGE, Mass., Dec. 18, 2018 (GLOBE NEWSWIRE) — Anchiano Therapeutics (TASE: ANCN), a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat cancer, today announced the initiation of Codex, the Company’s pivotal Phase 2 study of inodiftagene vixteplasmid (BC-819), a first-in-class gene therapy for patients with non-muscle-invasive bladder cancer (NMIBC). The study will enroll 140 patients with NMIBC whose disease is unresponsive to adequate BCG therapy, as defined by U.S. Food and Drug Administration (FDA) guidance.

“The initiation of the Codex registration trial is a significant milestone for the company,” noted Dr. David Kerstein, Chief Medical Officer of Anchiano Therapeutics. “BCG-unresponsive NMIBC represents an area of high unmet medical need with limited standard treatment options, outside of surgical removal of the bladder. NMIBC is a cancer that has been lacking in new therapies for two decades, and we are excited to embark on this important next step in the development of inodiftagene vixteplasmid.”

About the Codex Study

Codex is a Phase 2 multicenter, open-label, single-arm clinical trial designed for registration. The study will evaluate the efficacy and safety of inodiftagene vixteplasmid in patients with NMIBC that have high-grade stage Ta or T1 papillary tumors or carcinoma in situ (CIS) and whose disease is considered unresponsive to adequate BCG therapy, as defined by FDA guidance. Under the trial protocol, inodiftagene vixteplasmid is administered as monotherapy via instillation into the urinary bladder at a dose of 20 mg for 10 weekly treatments, followed by treatment every three weeks for up to two years or until disease recurrence.  The primary endpoint of the trial is complete response rate in patients with CIS at or after 12 weeks, with responding patients followed for at least a year. An interim analysis will be performed on data from the first 35 CIS patients. Accrual of 140 patients is planned.

Further details about Codex can be found at clinicaltrials.gov (Identifier: NCT03719300).

About Inodiftagene Vixteplasmid

Inodiftagene vixteplasmid (BC-819) is a recombinant DNA plasmid that directs the expression of a potent toxin specifically in malignant cells but not in normal tissue. It has been designed to exploit the established biology of the H19 gene, which is upregulated and expressed at high levels only in malignant cells, to produce bacterial diphtheria toxin only in bladder cancer tissue. Inodiftagene vixteplasmid is administered directly into the bladder to enable maximal topical exposure to target bladder cancer cells. The plasmid inodiftagene vixteplasmid is a 4.5 kb recombinant DNA molecule containing 832 bp of H19 regulatory sequences driving the expression of diphtheria toxin A chain only in malignant cells.

Diphtheria toxin A is an extraordinarily potent payload: one molecule may kill a cell, binding NAD and EF2 and inhibiting protein translation. In inodiftagene vixteplasmid, the lack of the diphtheria toxin B chain prevents transfer of the toxin molecule between cells.  The mechanism of action of diphtheria toxin is very well understood. Unlike most cancer therapies, there is no need to validate a new target or pathway.

About Anchiano Therapeutics

Anchiano Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat cancer, with offices in Cambridge, MA, and Jerusalem, Israel. The Company’s most advanced product candidate, inodiftagene vixteplasmid (BC-819), is in development as a treatment for non-muscle invasive bladder cancer. For additional information on inodiftagene vixteplasmid and the Codex Trial please go to www.anchiano.com.

Forward Looking Statements

This press release contains “forward-looking statements” that are subject to risks and uncertainties. These forward-looking statements are based on information Anchiano Therapeutics has when those statements are made or its management’s good faith belief as of that time with respect to future events, and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. These risks and uncertainties include, but are not limited to: a lack of history of commercial sales; no anticipated earning operating income over the coming years; a dependence on the success of inodiftagene, the development of which will require significant additional clinical testing before regulatory approval can be sought and commercial sales launched; some scientific or technological difficulties that may be encountered and impede R&D activities; a need to raise substantial additional funds to complete R&D activities; the raising of additional capital may dilute holdings of our existing shareholders, restrict our operations or require us to relinquish rights to our technologies or products; and an ability to obtain and maintain intellectual property protection for product candidates, including pursuant to licensed patents.

Company Contact:

Frank Haluska, M.D., Ph.D.

President and Chief Executive Officer

info@anchiano.com

Investor Contact:

Ashley R. Robinson

Managing Director

LifeSci Advisors, LLC

617-535-7742

arr@lifesciadvisors.com

Source: Anchiano Therapeutics