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Fibrocell Announces Positive Feedback from Type B End-of-Phase 2 Meeting with FDA on Phase 3 Clinical Trial Design for FCX-007
- Phase 3 clinical trial, named DEFI-RDEB, is expected to commence in the second quarter of 2019 -
- Additional data from Phase 1/2 clinical trial demonstrates FCX-007 continues to be well tolerated with continued positive trends in wound healing -
- Company reports full year 2018 financial results -
- Conference call & webcast scheduled at
FCX-007 Phase 3 Trial Design and FDA Type B Meeting Feedback
In the Type B face-to-face meeting, the
The proposed primary outcome measure for the DEFI-RDEB trial is a comparison of the proportion of FCX-007 treated wounds and untreated matched wounds with complete closure in a prospectively defined wound pair at 12 weeks post-administration of the first dose. Secondary endpoints include evaluation of the proportion of wounds achieving >50% wound closure, a patient reported outcome measure and an analysis of durability out to 24 weeks. The presence of type VII collagen (COL7) will be assessed from biopsy samples in a subpopulation of patients as an exploratory endpoint.
Fibrocell plans to submit a revised clinical trial protocol and statistical analysis plan based upon the FDA’s feedback and requested Chemistry, Manufacturing and Controls (CMC) information to the Investigational New Drug (IND) application.
“The FDA has provided us with invaluable advice on the design of the DEFI-RDEB trial, and we are grateful for their comprehensive feedback,” said
FCX-007 Phase 1/2 Trial Update
Fibrocell also reported today updated data from its ongoing Phase 1/2 clinical trial for FCX-007. To date, FCX-007 has been evaluated in eight wounds across five adult RDEB patients in the trial. Consistent with previously reported results, no product-related serious adverse events or circulating autoantibodies to COL7 have been reported.
The proportion of wounds healing at increasing closure percentages 12-week post-administration of a single injection session of FCX-007 are as follows:
|Wound Closure Percentage|
|FCX-007 Treated Wounds||88% (7/8)||75% (6/8)||63% (5/8)|
|Untreated Control Wounds||29% (2/7)||14% (1/7)||0% (0/7)|
The complete wound closure result (63% treated vs. 0% untreated) was the basis for the primary endpoint design in the DEFI-RDEB trial.
The proportion of wounds with >50% closure at various post-administration visits are as follows:
|4 Weeks||12 Weeks||25 Weeks||52 Weeks|
|FCX-007 Treated Wounds||100% (8/8)||88% (7/8)||67% (2/3)||66% (2/3)|
|Untreated Control Wounds||13% (1/8)||29% (2/7)||0% (0/2)||33% (1/3)|
“We continue to be encouraged by the positive safety and efficacy trends for FCX-007”, stated
Fibrocell recently completed dosing of a sixth patient—the first pediatric patient dosed with FCX-007—in the current Phase 1/2 clinical trial. Remaining Phase 2 patients who have not received dosing will be contacted to determine if they would agree to reconsent into the Phase 3 trial.
As a reminder, the
Fibrocell is also developing FCX-013 for the treatment of moderate to severe localized scleroderma. Fibrocell reported FCX-013 received Fast Track Designation from the
Financial Results for the Twelve Months Ended
For the year ended
Research and development expenses decreased 51% to approximately
Fibrocell used approximately
Conference Call and Webcast
To participate on the live call, please dial 888-394-8218 (domestic) or +1-323-794-2588 (international), and provide the conference code 8315981 five to ten minutes before the start of the call. The conference call will also be webcast live under the investor relations section of Fibrocell's website at www.fibrocell.com/investors/events and will be archived there for 30 days following the call.
FCX-007 is Fibrocell's clinical stage, gene therapy product candidate for the treatment of RDEB, a congenital and progressive orphan skin disease caused by the deficiency of the protein COL7. FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution.
Fibrocell is developing FCX-007 in collaboration with
FCX-013 is Fibrocell’s clinical stage, gene therapy candidate for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System®, a biologic switch activated by Veledimex—an orally administered compound—to control protein expression at the site of the localized scleroderma lesions. FCX‑013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation.
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell's most advanced product candidate, FCX-007, is the subject of a Phase 1/2 clinical trial for the treatment of RDEB. Fibrocell is also developing FCX-013 for the treatment of moderate to severe localized scleroderma, and is currently enrolling the Phase 1 portion of a Phase 1/2 clinical trial. For more information, visit www.fibrocell.com or follow Fibrocell on Twitter at @Fibrocell.
Fibrocell®, the Fibrocell logo, and
This press release contains, and our officers and representatives may from time to time make, statements that are “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to: Fibrocell's expectations regarding the timing and clinical development of FCX-007, including the Company’s plans to initiate a Phase 3 clinical trial for FCX-007 in the second quarter of 2019; the expected trial design of DEFI-RDEB, and expectation to enroll 15-20 patients therein; the timing of our Phase 1/2 clinical trial of FCX-013, including our expectation to complete enrollment of Phase 1 adult patients in the third quarter of 2019; the potential advantages of FCX-007 and Fibrocell’s other product candidates; the potential benefits of Fast Track Designation, Orphan Drug Designation and Rare Pediatric Disease Designation; the Company’s belief that its cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2019 and other statements regarding Fibrocell’s future operations, financial performance and financial position, prospects, strategies, objectives and other future events.
Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: Fibrocell has not yet received the FDA’s official meeting minutes, and they may differ materially from the Company’s understanding of the results of the Type B meeting with the
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