ViaCell Announces Completion Of CB001 Phase I Clinical Trial
August 28, 2018
(Nasdaq: VIAC) today announced that it has enrolled and treated the last
patient in the CB001 Phase I clinical trial and has begun the 100-day
post-transplant follow-up. CB001 is an investigational therapy being
studied as a potential therapeutic option for people who need stem cell
transplants, such as bone marrow, for hematopoietic recovery after
chemo-radiation therapy or chemotherapy in the treatment of a variety of
blood cancers. The Phase I clinical trial is a safety study of CB001 using
stem cells that have been isolated from umbilical cord blood and expanded
using ViaCell's proprietary expansion technology. Ten patients received
treatment in the study. A preliminary data review indicates that there were
no infusion toxicity events related to CB001. ViaCell expects to announce
top-line results from the Phase I clinical trial in the fourth quarter of
2006 and, if positive, the Company intends to advance the product into a
Phase II clinical trial.
Umbilical cord blood stem cells are an important source of hematopoietic cells and often the preferred source of stem cells for transplants, especially in children. A single umbilical cord blood unit is generally too small to be suitable to treat an adult patient. CB001 consists of donor cord blood stem cells expanded using ViaCell's Selective Amplification technology, a proprietary approach to control and optimize growth of the stem cell population.
"There is an important medical need for patients who are unable to find a suitable bone marrow donor and we believe an expanded umbilical cord blood derived stem cell product has the potential to be a therapeutic alternative," said Marc D. Beer, President and Chief Executive Officer of ViaCell. "We are pleased to have completed the treatment phase of this study and look forward to reporting results later this year."
The Phase I clinical trial is primarily designed to evaluate safety in patients with advanced hematologic cancers in need of a hematopoietic stem cell transplant and who are unable to find a suitable bone marrow donor. Patients requiring this type of therapy are typically very sick with few therapeutic options. Patients participating in the study receive CB001 plus a standard cord following full myeloablative therapy. Patients are followed for 100 days post-transplant. ViaCell also expects to generate preliminary data on the clinical activity of CB001 including rate and durability of blood and immune system reconstitution. Additional background information on the clinical trial protocol is available online at clinicaltrials.
ViaCell is a biotechnology company focused on enabling the widespread use of human cells as medicine. The Company is developing a pipeline of proprietary stem cell product candidates intended to address cancer, cardiac disease, and diabetes. CB001, its lead cord blood derived stem cell therapy product candidate, is being developed for hematopoietic stem cell transplantation in patients affected by a variety of cancers. In addition to its therapeutic development programs, ViaCell's reproductive health business commercializes ViaCord(R), a product that offers expecting families the option of preserving their baby's umbilical cord blood. The Company is working to leverage its commercial infrastructure and product development capabilities by developing ViaCyte(SM), its investigational product intended to broaden reproductive choices for women through the cryopreservation of human unfertilized eggs. ViaCell is headquartered in Cambridge, Massachusetts with a processing and storage facility in Kentucky and additional research and development operations in Singapore. Additional information about ViaCell is available online at viacellinc.
This press release contains forward-looking statements regarding the potential for CB001 and the possibility of advancing to Phase II trials if the Phase I data is positive. Such statements are based on management's current expectations, and are subject to a number of risks and uncertainties which could cause actual results to differ materially from the Company's current expectations. There is no assurance that the Company will advance CB001 into Phase II clinical trials or be successful with development efforts. The Company may decide that the data from the Phase I clinical trial does not warrant further clinical development or requires changes in the program that cause there to be a need for additional preclinical testing or another Phase I clinical trial. Later stage clinical trials of CB001 may fail to show the desired safety and efficacy. The Company may also encounter other unexpected technical, regulatory or manufacturing hurdles and issues. For example, the FDA may view CB001 made with an improved Selective Amplification process as sufficiently different from the product candidate being used in our current Phase 1 clinical trial so as to warrant a new Phase 1 clinical trial. The Company may need to repeat trials for CB001 to incorporate new procedures such as mini-transplants. Repeating clinical trials for any reason would significantly delay the Company's development efforts related to CB001. Drug development involves a high degree of risk. For more information on the risks and uncertainties associated with the Company and its products and programs, see the factors set forth under the heading "Risk Factors" in the Company's report on Form 10-Q for the quarter ending March 31, 2006, which is on file with the Securities and Exchange Commission and which factors are incorporated herein by reference. ViaCell does not undertake any obligation to update forward-looking statements.
ViaCell(R) and ViaCord(R) are federally registered trademarks and ViaCyte(SM) is a service mark of ViaCell, Inc.