Carina Biotech Receives FDA “Safe to Proceed” Letter for IND Application for Phase 1/2a Clinical Trial of LGR5-Targeted CAR-T Cell Therapy Candidate for Treatment of Advanced Colorectal Cancer

• Receives greenlight from the FDA for Phase 1/2a clinical trial of CNA3103
• On track to commence patient enrollment during the first half 2023

ADELAIDE, Australia , Jan. 23, 2023 (GLOBE NEWSWIRE) — Carina Biotech Pty Ltd (“Carina,” “the Company”), a cell therapy immuno-oncology company developing CAR-T and other adoptive cell therapies for the treatment of solid cancers, announced today that it has received the “safe to proceed” letter from the U.S. FDA for its Investigational New Drug (NDA) application to conduct a first-in-human Phase 1/2a clinical trial of CNA3103, its LGR5-targeted chimeric antigen receptor T cell (CAR-T) therapy candidate, in patients with advanced colorectal cancer (CRC).

The Phase 1/2a clinical trial will enroll a total of 44 patients with metastatic disease failing prior lines of chemotherapy and who express LGR5 on their cancer cells. Twenty-four patients in the Phase 1 segment will be enrolled in select Australian centers. Following a BOIN (Bayesian Optimal Interval) design, ascending CAR-T cell doses will be administered to cohorts of three patients each, to assess the safety and tolerability of CNA3103, and to determine its optimal dose. The subsequent Phase 2 segment will enroll 20 patients at the optimal dose, in both Australia and the U.S., to characterize the activity of CNA3103, in terms of antitumor response, duration of response and time to disease progression.

“Our positive engagement with the FDA marks a major step forward for Carina and our continued efforts to develop CNA3103 as a potential treatment for the third most common cancer in the U.S. and worldwide, colorectal cancer,” said Deborah Rathjen, Carina’s Chief Executive Officer. “The FDA’s letter provides the pathway for this clinical trial and further validates that a significant unmet need exists for more effective treatment options for colorectal cancer. Preparations, including site selection, are underway as we aim to commence enrolling patients during the first half of 2023.”

“This interaction with the FDA corroborates our ability to gain the agency’s support for our proposed trial of CNA3103 in metastatic colorectal cancer patients,” said José Iglesias, MD, Carina’s Chief Medical Officer. “Our approach is doubly innovative, in being one of the of emergent CAR-T treatment protocols in solid tumors and in utilizing a novel, and to our knowledge, unique CAR-T construct against LGR5 – an important cancer stem cell-associated antigen linked to the pathogenesis, dissemination, and treatment resistance of colorectal cancer.”

About CNA3103

Carina’s proprietary CNA3103 CAR-T cell targets LGR5, a cancer stem cell marker that is highly expressed on advanced colorectal cancer and some other cancers. In colorectal cancer patients, LGR5 expression has been correlated with poor prognosis. Cancer stem cells are a small sub-population of cells within a tumor with the ability to self-renew, differentiate into the many cell types of a tumor, initiate new tumors, and resist chemotherapy and radiotherapy (leading to relapses). By targeting cancer stem cells, it is hoped that this therapy will reduce the tumor’s ability to generate new cancer cells, resulting in durable tumor suppression and preventing the relapses that are very common in patients with colorectal cancer. Carina’s pre-clinical studies of CNA3103 have shown promising results with complete tumor regression and no tumor recurrence following a single administration. CNA3103 has also demonstrated impressive tumor access and prolonged survival, enabling rejection of new tumors.

About Carina Biotech

Immuno-oncology company Carina Biotech is developing CAR-T and other adoptive cell therapies for the treatment of solid cancers. In addition to its LGR5-targeted CAR-T cell therapy CNA3103 for advanced colorectal cancer, Carina has a deep pipeline of CAR-T programs. Using its proprietary chemokine receptor platform, Carina aims to improve access to and infiltration of solid cancers by CAR-containing cells, resulting in more potent and specific cancer killing and reduced off-target effects. Carina also has a fully integrated, proprietary CAR-T cell manufacturing process that has both reduced manufacturing time and improved CAR-T cell quality, capable of delivering robust “serial-killing” CAR-T cells to patients.

Forward Looking Statements

Investment in Carina Biotech is subject to investment risk, including possible loss of income and capital invested. Carina does not guarantee any particular rate of return or performance, nor does it guarantee the repayment of capital. This press release does not take into account the investment objectives, financial situation and particular needs of the investor. Before making any investment in Carina, the investor or prospective investor should consider whether such an investment is appropriate to their particular investment needs, objectives and financial circumstances, and consult an investment advisor as necessary.

This press release contains forward-looking statements regarding the potential of Carina’s projects and interests, and the development and therapeutic potential of the company’s investigational CAR-T therapies. Any statement describing a goal, expectation, intention or belief of the company is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing therapies that are safe and effective for use as human therapeutics and the financing of such activities.

There is no guarantee that Carina’s research and development projects and interests (where applicable) will receive regulatory approvals or prove to be technically and/or commercially successful in the future. Actual results of further research could differ from those projected or detailed in this press release. As a result, potential investors are cautioned not to rely on forward-looking statements. Consideration should be given to these and other risks concerning research and development programs, including but not limited to those specifically referred to in this press release.

Contact                                                                  Investor Inquiries

Deborah Rathjen, PhD                                        ICR Westwicke                
CEO & Managing Director                                 Stephanie Carrington                        
Carina Biotech                                                      646-277-1282        
+61 418 160 425                                                      [email protected]         
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