EXTON, PA — Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, reported financial results for the first quarter ended March 31, 2019 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 John Maslowski, President and Chief Executive Officer of Fibrocell. “Most recently, we announced our collaboration with Castle Creek Pharmaceuticals to develop and commercialize FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). We are excited about this partnership which, we believe, has the potential to deliver innovative treatments offering hope and relief to EB patients and their families.”
“Furthermore, we completed a Type B end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) to discuss the design of a Phase 3 clinical trial for FCX-007. Based on the FDA’s feedback from this meeting, we are incorporating their comments into our Investigational New Drug (IND) application for FCX-007, and expect to initiate the Phase 3 trial in the second quarter of 2019. In addition, we recently reported additional data from our ongoing FCX-007 Phase 1/2 clinical trial that continues to demonstrate positive safety and efficacy trends.”
“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 Pharmaceuticals to develop and commercialize FCX-007 for the treatment of RDEB. Under the terms of the collaboration agreement, Castle Creek Pharmaceuticals will receive an exclusive license to commercialize FCX-007 in the United States. Fibrocell received an upfront payment of $7.5 million at closing. In addition, Fibrocell will receive $2.5 million for the first patient enrolled in the Phase 3 clinical trial of FCX-007 and $30 million upon 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 million in sales milestones, consisting of $25 million when FCX-007 achieves $250 million in cumulative net sales and an additional $50 million upon attaining $750 million in cumulative net sales. In addition, Castle Creek Pharmaceuticals will pay Fibrocell a 30% share of the gross profits from FCX-007 sales. Castle Creek Pharmaceuticals will be responsible for all development and manufacturing expenses up to $20 million prior to the initial BLA filing with the FDA. If development spending exceeds $20 million, Castle Creek Pharmaceuticals will 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 FDA to 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 March 31, 2019
For the three months ended March 31, 2019, Fibrocell reported a diluted net loss of $0.40 per share, compared to a diluted net loss of $0.55 per share for the same period in 2018.
Research and development expenses increased approximately 48% to approximately $2.0 million for the three months ended March 31, 2019, as compared to approximately $1.3 million for the same period in 2018. Costs for their FCX-007 program increased approximately $0.8 million for the three months ended March 31, 2019 compared to the same period in 2018. The increase for the three-month period ended March 31, 2019 was related primarily to approximately $0.5 million in costs for vector development in the March 31, 2019 period, as well as approximately $0.3 million in increased costs from their clinical partner, Intrexon Corporation (Intrexon). The $0.8 million increase in FCX-007 program costs were partially offset by an approximately $0.2 million decrease in FCX-013 spending. This decrease was related primarily to decreased costs from Intrexon of approximately $0.1 million, as well as lower costs for materials and professional fees, as substantially all of the costs of the pre-clinical phase of the project were completed at the end of 2017, while 2018 and 2019 to date have been used primarily for clinical trial start-up activities.
Selling, general and administrative costs increased approximately $0.2 million to approximately $1.9 million, due primarily to increased legal fees related to the Castle Creek Pharmaceuticals agreement.
Fibrocell used approximately $3.1 million in cash for operations during the three months ended March 31, 2019, and used approximately $4.8 million in cash for operations during the three months ended March 31, 2018.
As of March 31, 2019, the Company had cash and cash equivalents of approximately $11.3 million and working capital of approximately $9.1 million. The Company believes that its cash and cash equivalents at March 31, 2019, along with the milestone payment received from Castle Creek Pharmaceuticals in April 2019, the potential milestone payment upon enrollment of the first patient in the Phase 3 clinical trial of FCX-007 and the reimbursement of development costs for FCX-007 under the Castle Creek Pharmaceuticals agreement, will be sufficient to fund operations into the third quarter of 2020.
Source: Fibrocell Science Inc., 405 Eagleview Blvd, Exton PA 19341
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