Phase 2 Ready Oncology Drug Receives FDA Orphan Drug Designation
September 17 2018 - 9:28AM
Cytori Therapeutics (NASDAQ: CYTX) (“Cytori” or the “Company”)
today announced 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.
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.
About Cytori
Cytori is developing, manufacturing, and
commercializing nanoparticle-delivered oncology drugs and
autologous adipose-derived regenerative cell (ADRC) therapies
within its Nanomedicine™ and Cell Therapy™ franchises,
respectively. Cytori Nanomedicine™ is focused on the
liposomal encapsulation of anti-neoplastic chemotherapy agents,
which may enable the effective delivery of the agents to target
sites while reducing systemic toxicity. The Cytori
Nanomedicine™ product pipeline consists of ATI-0918 pegylated
liposomal doxorubicin hydrochloride for breast cancer, ovarian
cancer, multiple myeloma, and Kaposi’s sarcoma, a complex/hybrid
generic drug, and ATI-1123 patented albumin-stabilized pegylated
liposomal docetaxel for multiple solid tumors. Cytori Cell
Therapy™, prepared within several hours with the proprietary
Celution® System and administered to the patient the same day, has
been shown in preclinical and clinical studies to act principally
by improving blood flow, modulating the immune system, and
facilitating wound repair. As a result, Cytori Cell Therapy™ may
provide benefits across multiple disease states and can be made
available to the physician and patient at the point-of-care.
For more information, visit www.cytori.com.
Cautionary Statement Regarding Forward-Looking Statements
This communication includes forward-looking statements regarding
events, trends and business prospects, which may affect our future
operating results and financial position. Such statements are
subject to risks and uncertainties that could cause our actual
results and financial position to differ materially. Some of these
risks include clinical, regulatory and reimbursement uncertainties,
the challenges inherent in convincing physicians and patients to
adopt the new technology, dependence on third party performance,
performance and acceptance of our products, and other risks and
uncertainties described under the “Risk Factors” in
Cytori's Securities and Exchange Commission Filings,
including in its most recent annual and quarterly reports. Cytori
assumes no responsibility to update or revise any forward-looking
statements contained in this press release to reflect events,
trends or circumstances after the date of this communication.
Cytori Therapeutics, Inc.Tiago Girao,
1.858.458.0900ir@cytori.com
Cytori Therapeutics (NASDAQ:CYTX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Cytori Therapeutics (NASDAQ:CYTX)
Historical Stock Chart
From Apr 2023 to Apr 2024