On September 17, 2018 Cytori Therapeutics (NASDAQ: CYTX) ("Cytori" or the "Company") reported that it received FDA orphan drug designation for its ATI-1123 chemotherapy drug product candidate, an albumin-stabilized pegylated liposomal docetaxel, for the treatment of small cell lung cancer (Press release, Cytori Therapeutics, SEP 17, 2018, View Source [SID1234529464]).
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The FDA’s Orphan Drug Designation Program provides orphan status to drugs, such as ATI-1123, which are intended for the safe and effective treatment of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Sponsors of a drug receiving orphan designation qualify for various incentives including tax credits for clinical testing, 7 years of marketing exclusivity following FDA approval, and a waiver of prescription drug user fees.
Small cell lung cancer (SCLC), which accounts for approximately 15% of bronchogenic carcinomas with 33,375 new cases and 23,380 deaths estimated for 2017, has been identified by Cytori as a compelling target for ATI-1123. While SCLC is responsive to chemotherapy and radiation therapy, cure occurs only in ~20% of patients, generally restricted to those with limited stage disease. The remaining 80% of patients, including all patients with extensive disease, relapse within months of completing their initial therapy. Availability of 2nd line therapy options are limited, toxic, and provide little benefit in terms of extending survival. Topotecan is the only FDA-approved agent for 2nd line treatment of SCLC and is associated with an overall response rate of 24%, median response duration or time to progression of 14 weeks, and median overall survival of 25 weeks. Treatment usually involves a consecutive 5 day regimen of either IV or oral administration of drug, both which have black box warnings for severe myelosuppression as their use is associated with substantial morbidity including bone marrow suppression leading to neutropenia, thrombocytopenia and anemia requiring interventions of transfusion and growth factor support.
No major treatment advances for SCLC have occurred over the past 30 years. Hence, there remains a significant unmet need for novel agents with better safety profiles both for patients who cannot tolerate the adverse effects of 1st line chemo-radiotherapy and for relapsed/refractory patients who receive Topotecan. Cytori’s ATI-1123 has been designed to fill this need. ATI-1123’s combination of improved liposome stability, reduced toxicity, and superior delivery are expected to provide a therapeutic for SCLC that offers comparable or better efficacy to currently-available standards while having a less intensive administration routine and improved side effect profile.
Cytori is also exploring the development of ATI-1123 to address the shortcomings of docetaxel, a workhorse chemotherapy drug which generated $2.7B in worldwide sales at its peak. Compared to docetaxel, ATI-1123 may have potential to improve safety by removing the need for unwanted solvents, reduce morbidity by eliminating the requirement for standard pretreatment medications, provide better patient convenience and comfort via less time spent in the treatment center, decrease the cost of therapy, and enhance systemic docetaxel exposure.
A U.S. Phase 1 clinical study of ATI-1123 has been completed and published. Of the 29 patients in the study with cervical, gastric, melanoma, non-small cell lung, ovarian, pancreatic, prostate, thyroid, urachal and uterine cancers, 82% demonstrated a clinical benefit with ATI-1123. ATI-1123 exhibited an improved safety profile versus the Taxotere label with a 31% reduction in neutropenia and anemia. Further, ATI-1123 showed a 20% increase in maximum tolerated dose versus standard docetaxel and signs of efficacy with 1 partial responder.