Development Pipeline

Cytori is a specialty therapeutics company focused on bringing to market cell therapies and nanomedicines that address unmet medical needs. Our development pipeline is founded upon promising research data generated in a variety of preclinical studies, investigator driven studies, and Cytori-sponsored clinical trials.

Therapeutic Agent

Pre-clinical

Phase I/II

Phase III

Hand Scleroderma

HABEO™

48 Week Results

HABEO™

Fully Enrolled*

Knee Osteoarthritis

ECCO-50

48 Week Results

Male Stress Urinary Incontinence

ECCI-50

Fully Enrolled*

Thermal Burn Injury

DCCT-10

FDA IDE Approved#

Hand Scleroderma

HABEO™

Phase III - 48 Week Results

HABEO™

Phase III - Fully Enrolled*

Knee Osteoarthritis

ECCO-50

Phase I/II - 48 Week Results

Male Stress Urinary Incontinence

ECCI-50

Phase III - Fully Enrolled*

Thermal Burn Injury

DCCT-10

Phase I/II - FDA IDE Approved#
Cytori Nanomedicine

Therapeutic Agent

Pre-clinical

Phase I/II

Phase III

Breast and Ovarian Cancer; Multiple Myeloma and Kaposi’s Sarcoma

ATI-0918

Bioequivalence Study Complete

ATI-0918

Pre-clinical

Multiple Solid Tumors

ATI-1123

Results Published

Breast and Ovarian Cancer; Multiple Myeloma and Kaposi’s Sarcoma

ATI-0918

Phase III - Bioequivalence Study Complete

ATI-0918

Pre-clinical - Pre-clinical

Multiple Solid Tumors

ATI-1123

Phase I/II - Results Published

* Investigator-Initiated, Cytori-supported trial # BARDA funded program

Cytori Cell Therapy™ CLINICAL TRIALS

Cytori Therapeutics’ development pipeline consists of rigorous, well-designed, controlled studies and trials targeting clinical indications with significant medical needs inadequately addressed by currently available therapies.

Cytori Cell Therapy is comprised of ADRCs and specifically adapted to individual indications or clinical situations. For example, Habeo™ Cell Therapy has been developed for hand dysfunction associated with the rare autoimmune disease, scleroderma.

Completed and Ongoing Trials

HAND SCLERODERMA

STAR was an 88 patient, prospective, double-blind, randomized, U.S. multi-center, parallel-group clinical trial assessing the safety and efficacy of a single, subcutaneous administration of Habeo Cell Therapy (40 million cells per subject) into the fingers of patients with hand dysfunction due to scleroderma. The subjects were randomized 1:1 to receive either Habeo Cell Therapy or placebo. Investigators conducted final assessments at 48 weeks.  Details of the STAR trial can be found at clinicaltrials.gov.  Top-line results can be found here.  Additional results can be found here.

This STAR trial was based on the previously published trial, SCLERADEC I, conducted at Hȏpital de la Conception in France. SCLERADEC I reported significant improvement in hand function, pain, and Raynaud’s severity in patients with scleroderma affecting the hands.1,2,5 Cytori supported this trial in the form of devices, consumables, and training. Cytori is also providing support for SCLERADEC II, a multi-center, controlled study in France which recently completed enrollment of 40 patients.

KNEE OSTEOARTHRITIS

ACT-OA was a 94 patient, prospective, double-blind, randomized, U.S. multi-center, parallel-group, Phase IIA/B clinical trial assessing the safety and efficacy of a single, intra-articular administration of ECCO-50 Cell Therapy into patients’ knees with osteoarthritis. The patients were randomized 1:1:1 to receive ECCO-50 (20 million cells), ECCO-50 (40 million cells), or placebo.  Details of the ACT-OA trial can be found at clinicaltrials.gov.  Top-line results can be found here.

THERMAL BURN INJURY – BARDA CONTRACT

In December 2012 Cytori was awarded a research and development contract from the Biomedical Advanced Research and Development Authority (BARDA), a unit within the US Department of Health and Human Services. This contract funds the development of Cytori Cell Therapy™ as a medical countermeasure for thermal burn injury including burns that are complicated by radiation exposure. In 2014, upon achievement of specified development milestones by Cytori, BARDA executed contract Option 1 which funded the second phase of the project.  Cytori received U.S. FDA IDE approval for the RELIEF pilot clinical trial in April 2017 and executed a $13.4M contract option with BARDA in May 2017.  Details of the RELIEF trial can be found at reliefclinicaltrial.com and clinicaltrials.gov

MALE STRESS URINARY INCONTINENCE

Cytori has agreed to provide partial support to the ADRESU Japanese investigator/government sponsored trial of Cytori Cell Therapy for male urinary incontinence following radical prostatectomy. The ADRESU mutli-center trial is based on a previously published2 feasibility trial conducted at Nagoya University in Japan. This feasibility trial demonstrated improvements in leakage, urethral closure, and patient quality-of-life assessment in men with urinary incontinence following radical prostatectomy for prostate cancer. The primary funding and support of the ADRESU trial is from the Japanese Ministry of Health, Labor and Welfare and Nagoya University.

INVESTIGATOR-INITIATED STUDIES

By providing access to Cytori Cell Therapy processing technology in markets where it has class I or CE Mark approval, the Celution® System has become an important research and development tool for physicians. This enables them to study the potential of our technology in Institutional Review Board (IRB) and Medical Ethics Committee (MEC)-approved studies. A number of investigator-led trials have been completed, are in process, for a range of conditions and applications, including the following:

  • Anterior cruciate ligament repair
  • Bone repair
  • Burn scar
  • Chronic wounds
  • Critical limb ischemia
  • Cryptoglandular and Crohn’s Fistulae
  • Erectile Dysfunction
  • Meniscal Repair
  • Stress urinary incontinence
  • Scleroderma hand disease
  • Vocal cord repair

Other Clinical Trials

HEART DISEASE

PRECISE was a 27 patient safety and feasibility study in Europe designed to evaluate the use of Cytori Cell Therapy in patients with chronic myocardial ischemia. Data from the PRECISE trial indicated the feasibility of obtaining and delivering Cytori Cell Therapy to patients with significant heart disease. The data provided early indications of efficacy that provided the rationale for the ATHENA trials in the US.

The ATHENA I and II trials are multi-center, randomized, double-blind safety and feasibility trials to investigate the use of Cytori Cell Therapy in patients with heart failure due to ischemic heart disease. The trials were originally planned to enroll 45 patients each and examine two dosages of Cytori Cell Therapy.

The trials incorporated several endpoints, including peak oxygen consumption, perfusion defects, heart failure symptoms, health-related quality of life, left ventricle end-systolic and diastolic volume, and left ventricular ejection fraction at six and 12 months. Enrollment was truncated at 31 patients in 2014 due to a prolonged trial delay in 2014; however, data will be available for review in mid-2016.

BREAST RECONSTRUCTION

The RESTORE-2 study was a Phase IV (post-market) European study that evaluated Cytori Cell Therapy for breast deformities post segmental breast resection (lumpectomy) with or without radiation therapy. This prospective, single-arm, open-label, multi-center study enrolled 71 patients with defects ranging from 25-150 mL. The procedure was shown to be safe and well tolerated. The data reported patient and investigator satisfaction (75% and 85% respectively) following cell-enriched fat grafting to treat breast defects post-breast conservation therapy.4

  • 1 Gabizon A, Shmeeda H, and Barenholz Y (2003) “Pharmacokinetics of pegylated liposomal Doxorubicin: review of animal and human studies” Clin Pharmacokinet. 42(5):419-36
  • 2 Rafiyath et al. 2012 “Comparison of safety and toxicity of liposomal doxorubicin vs. conventional anthracyclines: a meta-analysis” Exp Hematol Oncol 1:10-19

CYTORI NANOMEDICINE CLINICAL TRIALS

Completed Trials and Future Plans

ATI-0918 Bioequivalence Trial

ATI-0918 is Cytori’s lead nanomedicine product, a generic pegylated liposomal doxorubicin hydrochloride.  Pegylated liposomal doxorubicin was approved many years ago by the U.S. FDA and the European Medicines Agency (EMA), but is now off-patent. This creates an expedited path for regulatory approval of generic formulations in which clinical trials are targeted at showing “bioequivalence” which means that the drug levels in the blood and tissue following injection of ATI-0918 must be equivalent (using FDA/EMA-specified bioequivalence criteria) to the levels seen when the original patented approved product is injected. Bioequivalence studies typically require only 1 clinical trial as opposed to the conventional Phase I, Phase II, and Phase III pathway required for new drugs. Bioequivalence of ATI-0918 and the reference listed drug in Europe, Caelyx®, has already been demonstrated in a clinical trial. Cytori is now preparing to submit an application for approval of ATI-0918 by the EMA. If granted, this approval will allow marketing of ATI-0918 for use in breast cancer and ovarian cancer with the possibility of extending this to both multiple myeloma and Kaposi’s sarcoma. Bioequivalence with the FDA-approved reference listed drug,Lipodox®, in the U.S. is in the planning stages.

ATI-1123

ATI-1123, a patented protein-bound liposomal formulation of docetaxel, is being developed as a next-generation version of the currently approved product, Taxotere. Docetaxel is approved in the U.S. for the treatment of a number of cancers including breast cancer, head and neck cancer, stomach cancer, prostate cancer and non small-cell lung cancer. Use of docetaxel is associated with several side effects including injury to the bone marrow. Cytori believes that by encapsulating docetaxel in a liposome,  ATI-1123 may reduce these side effects. A 29 patient Phase I trial has been completed and published6. This published data suggests that the toxicity may be less than that reported for non-encapsulated docetaxel. This will need to be confirmed in larger Phase II and Phase III clinical trials.

  • 6. Mahalingam D, Nemunaitis JJ, Malik L, et al. Phase I study of intravenously administered ATI-1123, a liposomal docetaxel formulation in patients with advanced solid tumors. Cancer Chemother Pharmacol. 2014 Dec;74(6):1241-50. doi: 10.1007/s00280-014-2602-x. Epub 2014 Oct 11.
Caelyx® and Doxil® are registered trademarks of Janssen Products, LP.