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Fibrocell Reports First Quarter 2019 Financial Results and Recent Operational Highlights
“We achieved important milestones during the first several months of 2019 for our gene therapy clinical programs focused on devastating, rare genetic conditions of the skin and connective tissue with high unmet medical needs,” said
“Furthermore, we completed a Type B end-of-Phase 2 meeting with the
“Our FCX-013 gene therapy program for the treatment of moderate to severe localized scleroderma continues to progress. We are currently enrolling the Phase 1 portion of the FCX-013 Phase 1/2 clinical trial, and expect to complete enrollment of the Phase 1 adult patients in the third quarter of 2019,” said Mr. Maslowski.
Recent program highlights are as follows:
- Fibrocell announced completion of a collaboration agreement with
Castle Creek Pharmaceuticalsto develop and commercialize FCX-007 for the treatment of RDEB. Under the terms of the collaboration agreement, Castle Creek Pharmaceuticalswill receive an exclusive license to commercialize FCX-007 in the United States. Fibrocell received an upfront payment of $7.5 millionat closing. In addition, Fibrocell will receive $2.5 millionfor the first patient enrolled in the Phase 3 clinical trial of FCX-007 and $30 millionupon approval of a Biologics License Application (BLA) for FCX-007 and commercial readiness of FCX-007. Fibrocell is also eligible to receive up to $75 millionin sales milestones, consisting of $25 millionwhen FCX-007 achieves $250 millionin cumulative net sales and an additional $50 millionupon attaining $750 millionin cumulative net sales. In addition, Castle Creek Pharmaceuticalswill pay Fibrocell a 30% share of the gross profits from FCX-007 sales. Castle Creek Pharmaceuticalswill be responsible for all development and manufacturing expenses up to $20 millionprior to the initial BLA filing with the FDA. If development spending exceeds $20 million, Castle Creek Pharmaceuticalswill be responsible for 70% of the excess costs and Fibrocell will cover 30% of these additional expenses. Fibrocell will retain sole ownership of the Rare Pediatric Disease Priority Review Voucher (PRV), which may be granted upon market approval of FCX-007. The PRV can be used to obtain priority review for a subsequent New Drug Application or BLA, and can be sold to another entity.
- In connection with the successful completion of the agreement with
Castle Creek Pharmaceuticals, Fibrocell also concluded the strategic alternative review process announced last year.
- Fibrocell completed a Type B face-to-face meeting with the
FDAto discuss various design aspects of the Company’s proposed Phase 3 clinical trial, named DEFI-RDEB (dermal fibroblasts-RDEB), to support a BLA filing. The Phase 3 trial is designed as an open label, multi-centered, intra-patient controlled trial expected to enroll 15-20 patients. 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 information to the IND application. The Company expects to initiate the Phase 3 clinical trial in the second quarter of 2019. Fibrocell projects enrollment and dosing of Phase 3 patients will be completed in the third quarter of 2020 and data collection for the primary endpoint will be completed in the fourth quarter of 2020. If the Phase 3 clinical trial is successful and completed within the projected timeframe, Fibrocell expects to file a BLA for FCX-007 in 2021.
- Fibrocell reported additional data from its ongoing Phase 1/2 clinical trial that demonstrates FCX-007 continues to be well tolerated with continued positive trends in wound healing. 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.
- Fibrocell completed dosing of a sixth patient—the first pediatric patient dosed with FCX-007—in the current Phase 1/2 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.
- Fibrocell is currently enrolling the Phase 1 portion of a Phase 1/2 clinical trial for FCX-013, and expects to complete enrollment of Phase 1 adult patients in the third quarter of 2019. The Company projects that safety and efficacy data for the adult patients will be available in mid-2020.
Financial Results for the Three Months Ended
For the three months ended
Research and development expenses increased approximately 48% to approximately
Selling, general and administrative costs increased approximately
Fibrocell used approximately
Conference Call and Webcast
To participate on the live call, please dial 888-220-8451 (domestic) or +1-786-789-4776 (international), and provide the conference code 7919925 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.
FCX-007 has been granted Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation by the
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 a cell and gene therapy company focused on improving the lives of people with rare diseases of the skin and connective tissue. The Company is utilizing its proprietary autologous fibroblast technology to develop personalized biologics that target the underlying cause of disease. Fibrocell’s pipeline of localized gene therapy candidates include FCX-007 for the treatment of RDEB, a life-threatening genetic disorder diagnosed in infancy with no cure or treatment approved by the
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 potential benefits of the collaboration between
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: the ability of
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Condensed Consolidated Statements of Operations (unaudited)
($ in thousands, except share and per share data)
|Three Months Ended
|Total cost of revenue||—||—|
|Gross profit (loss)||—||—|
|Research and development expense||1,943||1,645|
|Research and development expense - related party (see Note 9)||44||(303||)|
|Selling, general and administrative expense||1,870||1,639|
|Other income (expense):|
|Warrant revaluation income (expense)||(29||)||235|
|Derivative revaluation income (expense)||55||(63||)|
|Other income, net||362||98|
|Loss before income taxes||(3,666||)||(2,901||)|
|Dividend paid in-kind to preferred stockholders||(85||)||(82||)|
|Deemed dividend on preferred stock (see Note 11)||(140||)||(121||)|
|Net loss attributable to common stockholders||$||(3,891||)||$||(3,104||)|
|Per Share Information:|
|Weighted average number of common shares outstanding:|
|Condensed Consolidated Balance Sheets Data:||March 31,||December 31,|
|Cash and cash equivalents||$||11,322||$||14,430|
|Warrant liability, long term||181||152|
|Total stockholders’ equity||6,003||9,557|