SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): September 5, 2018
FIBROCELL SCIENCE, INC.
(Exact Name of Registrant as Specified in its Charter)
(State or Other Jurisdiction of Incorporation or Organization)
(Commission File No.)
(I.R.S. Employer Identification No.)
405 EAGLEVIEW BLVD., EXTON, PA 19341
(Address of principal executive offices and zip code)
(Registrant’s telephone number, including area code)
(Former name or former address, if changed from last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-14(c))
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter). Emerging growth company o
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o
Item 8.01 Other Events.
On September 5, 2018, Fibrocell Science, Inc. (the "Company") issued a press release announcing that the U.S. Food and Drug Administration granted Fast Track Designation to FCX-013, the Company's clinical stage candidate for treatment of moderate to severe localized scleroderma. A copy of the press release is filed herewith as Exhibit 99.1 and is incorporated by reference herein.
On September 5, 2018, the Company also posted an updated corporate presentation on its website at www.fibrocell.com. A copy of the presentation is filed herewith as Exhibit 99.2 and is incorporated by reference herein.
Item 9.01 Financial Statements and Exhibits.
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, hereunto duly authorized.
Fibrocell Science, Inc.
/s/ John M. Maslowski
John M. Maslowski
President and Chief Executive Officer
Date: September 5, 2018
Fibrocell Announces FDA Fast Track Designation of FCX-013 for Treatment of Moderate to Severe Localized Scleroderma EXTON, PA – September 5, 2018 – Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to FCX-013, the Company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma—a chronic, debilitating genetic skin disorder with no FDA approved therapies. “Fast Track designation represents an important milestone in advancing clinical development of FCX-013,” said John Maslowski, President and Chief Executive Officer of Fibrocell. “We are pleased the FDA has awarded this designation to FCX-013 which, we believe, has the potential to be the first gene therapy to treat excessive collagen accumulation in the skin and soft tissue at the site of localized scleroderma lesions and to bring relief from the severe pain and functional disability associated with the disorder.” The Fast Track program is designed to facilitate development and expedite review of new therapies that address unmet medical needs of patients with serious conditions. The designation offers various benefits, including more frequent meetings with the FDA to discuss the drug's development plan, eligibility for Accelerated Approval or Priority Review if relevant criteria are met, and opportunity for Rolling Review, which allows the Company to submit completed sections of its Biologics License Application (BLA) to the FDA, rather than waiting until every section of the BLA is completed before the entire application can be reviewed. Previously, the FDA granted Orphan Drug Designation to FCX-013 for the treatment of localized scleroderma and Rare Pediatric Disease Designation for the treatment of moderate to severe localized scleroderma. In addition, Fibrocell is developing FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), which is currently under evaluation in the Phase 2 portion of a Phase 1/2 clinical trial. FDA previously granted Orphan Drug, Rare Pediatric Disease and Fast Track Designations to FCX-007. Fibrocell is developing FCX-007 and FCX-013 in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation (NYSE: XON), a leader in synthetic biology. Fibrocell plans to manufacture FCX-013 at its cGMP cell manufacturing facility located in Exton, Pennsylvania. About FCX-013
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 Precigen’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. About the Phase 1/2 Clinical Trial Fibrocell initiated the first investigator site for clinical enrollment for an open label, single arm Phase 1/2 clinical trial of FCX-013 in August 2018. The primary objective of the trial is to evaluate the safety of FCX-013. Secondary analyses consist of several fibrosis assessments including histology, skin scores, ultrasound and additional measurements of targeted sclerotic lesions and control sites at various time points up to 16 weeks post-administration of FCX-013. Fibrocell is targeting ten patients with any subtype of localized scleroderma for enrollment (approximately 5 patients per Phase). The Phase 1 portion will enroll adult patients, and dosing for the first three adult patients will be staggered prior to dosing the rest of the trial’s population. Fibrocell intends to include pediatric patients in the Phase 2 portion of the trial after submission and approval of safety and activity data from the adult Phase 1 patients from the FDA and the Data Safety Monitoring Board for the trial. About Localized Scleroderma Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix, specifically collagen, resulting in thickening of the skin and connective tissue. Localized scleroderma encompasses several subtypes which are classified based on the depth and pattern of the lesion(s). The moderate to severe forms of the disorder include any subtype that affects function or produces symptoms of discomfort, tightness and pain. Current treatments for localized scleroderma include systemic or topical corticosteroids which target inflammation, UVA light therapy and physical therapy. There are few treatment options to address excessive collagen accumulation in the skin and connective tissue. We estimate that there are approximately 90,000 patients in the U.S. considered to have moderate to severe localized scleroderma. About Fibrocell 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, the Company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon
Corporation, a leader in synthetic biology. For more information, visit www.fibrocell.com or follow Fibrocell on Twitter at @Fibrocell. Trademarks Fibrocell®, the Fibrocell logo, and Fibrocell Science® are trademarks of Fibrocell Science, Inc. and/or its affiliates. All other names may be trademarks of their respective owners. Forward-Looking Statements 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-013; the potential advantages of FCX-013 and Fibrocell’s other product candidates; the potential benefits of Fast Track Designation, Orphan Drug Designation and Rare Pediatric Disease Designation; 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: uncertainties and delays relating to the initiation, enrollment and completion of clinical trials; whether clinical trial results will validate and support the safety and efficacy of Fibrocell’s product candidates; unanticipated or excess costs relating to the development of Fibrocell’s gene therapy product candidates; Fibrocell’s ability to obtain additional capital to continue to fund operations; uncertainties associated with being able to identify, evaluate and complete any strategic transaction or alternative; the impact of the announcement of the Board of Directors’ review of strategic alternatives, as well as any strategic transaction or alternative that may be pursued, on the Company's business, including its financial and operating results and its employees; Fibrocell’s ability to maintain its collaboration with Precigen, Inc.; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing and Form 10-Q filings. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise. # # # Investor & Media Relations Contact: Karen Casey 484.713.6133 firstname.lastname@example.org
Corporate Presentation September 5, 2018
Forward-Looking Statements This presentation and our accompanying remarks contain “forward-looking statements” within the meaning 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: the potential advantages of our product candidates; the initiation, design and timing of pre-clinical studies and clinical trials and activities and the reporting of the results thereof; the timing of regulatory submissions and actions; expected milestones; and all other statements relating to our future operations, future financial performance, future financial condition, prospects or other future events. Forward-looking statements are based upon our current expectations and assumptions and are subject to a number of known and unknown risks, uncertainties and other factors that could cause actual results to differ materially and adversely from those expressed or implied by such statements. Factors that could cause or contribute to such differences include, among others: the impact of the announcement of the Board of Directors’ review of strategic alternatives, as well as any strategic transaction or alternative that may be pursued, on the Company’s business, including its financial and operating results and its employees; that interim clinical trial results are not necessarily indicative of final clinical results and final clinical trial results may not be positive with regard to safety or efficacy of FCX-007 or FCX-013; uncertainties and delays relating to the initiation, enrollment and completion of pre-clinical studies and clinical trials, including with respect to FCX- 007, FCX-013 and our other product candidates; whether pre-clinical study and clinical trial results will validate and support the safety and efficacy of our product candidates; the risk that results seen in pre-clinical studies may not be replicated in humans; varying interpretation of pre-clinical and clinical data; unanticipated or excess costs relating to the development of our gene therapy product candidates; our ability to obtain additional capital to continue to fund operations; our ability to maintain our collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation; and the other factors discussed under the caption “Item 1A. Risk Factors” in our most recent Form 10-K and Form 10-Qs which are available through the “Investors—SEC Filings” page of our website at www.fibrocell.com. As a result, you should not place undue reliance on forward-looking statements. The forward-looking statements made in connection with this presentation represent our views only as of the date of this presentation (or any earlier date indicated in such statement). While we may update certain forward-looking statements from time to time, we specifically disclaim any obligation to do so, even if new information becomes available in the future. 2
Investment Highlights • Medical breakthroughs for rare diseases of the skin and connective tissue Focus on Rare Skin • Unmet needs with no approved therapies Diseases • Significant mortality and morbidity impact on pediatric populations Proprietary Ex-Vivo Gene • Autologous fibroblasts derived from skin are the vehicle to deliver target proteins Modified Fibroblast locally to the site of disease Platform • Extensive experience culturing dermal fibroblasts, including commercial scale • FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) . Four patients enrolled in Phase 2 (three pediatric and one adult), one completed first dose . Well tolerated safety and positive early trends noted in pharmacology and wound healing Two Clinical Trial Programs • FCX-013 for the treatment of moderate to severe Localized Scleroderma . IND allowed by FDA, and initiated first investigator site for clinical enrollment for Phase 1/2 clinical trial in August 2018 • Regulatory advantages including more frequent communications with FDA, eligibility Multiple FDA Designations for Accelerated Approval and Priority Review, and Rolling Review • Two Rare Pediatric Disease Designations to potentially receive Priority Review Vouchers (PRVs) upon market authorization • In-house 13,000 square foot cGMP cell therapy manufacturing facility supporting both Internal Manufacturing FCX-007 and FCX-013 Infrastructure • Existing capacity to serve the U.S. RDEB market 3
Development Pipeline Phase 1/2 Program Condition Target Research Pre-Clinical Clinical Trials FDA Designations Orphan Drug Type VII FCX-007 Recessive Dystrophic Rare Pediatric Disease Epidermolysis Bullosa Collagen (RDEB) Fast Track FCX-013 Moderate to Severe Orphan Drug MMP-1 Localized Scleroderma Rare Pediatric Disease Fast Track Research Arthritis and Related TBD Conditions Portfolio being developed in collaboration with Precigen, Inc., a wholly owned subsidiary of 4
Our Proprietary Fibroblast Platform Viral Vector Delivery Vehicle Vector Autologous fibroblasts Lentiviral vector gene transfer • Readily sourced, as fibroblasts • 3rd generation and self- are the most common cell type in skin and connective tissue inactivating • • Reduced rejection and Accommodates large gene immunogenicity concerns constructs • • Established manufacturing Transduces both dividing and non- process and regulatory dividing cells pathway Ex-vivo Gene • Target gene integration for long- term expression of the protein Ex-VivoModifiedGene Modified Cells Cells FCX-007 autologous dermal fibroblasts in culture 5
Personalized Biologics Approach 6
FCX-007 – Recessive Dystrophic Epidermolysis Bullosa FCX-007 is a dermal fibroblast transduced with a lentiviral (LV) vector encoded with the gene for type VII collagen (COL7) Orphan Drug Designation Rare Pediatric Disease Designation Fast Track Designation 7
About RDEB Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a monogenic disease resulting in chronic skin blistering and wounding Disease Current Treatments Epidemiology • Cause: A mutation in the • Current treatments only Dystrophic EB (DEB) COL7A1 gene that address symptoms ~5,500 – 12,500 US3 encodes for COL7 . Bandaging & • RDEB • Devastating, progressive, antibiotics – ~1,100 – 2,500 US4 painful blistering disease bandaging alone can that often leads to death exceed $10,000 per • Diagnosed at infancy month2 • High mortality rate – 76% . Feeding tubes of RDEB patients do not . Surgery, including 1 live beyond their 30’s hand and esophageal Photo credit: Science Photo Library 8
FCX-007 Phase 1/2 Clinical Trial Design A Phase 1/2 Trial of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) Title for Recessive Dystrophic Epidermolysis Bullosa (RDEB) NCT02810951 Primary Objective To evaluate the safety of a single intradermal injection session of FCX-007 • Pharmacology using immunofluorescence (IF), immunoelectron microcopy (IEM) and qPCR in wound and intact skin areas • Percent wound healing compared to baseline evaluation: Secondary Objectives . Wounds were evaluated during a monitoring period prior to dosing and were observed to be open up to 8 months . A matched size, untreated wound for each treated wound on the same patient was also monitored and evaluated • Stanford University School of Medicine Investigator Sites • Children’s Hospital Colorado Phase 1 Status • Four patients enrolled and dosed, continuing follow-up visits to 52 weeks • First patient dosed in June 2018 • Four patients enrolled: three pediatric and one adult patient Phase 2 Status • Six patients ages seven and older targeted for enrollment • Expect to complete enrollment by end of 3Q2018 9
Interim Phase 1 Data Update • Selected to present data at the International Investigative Dermatology (IID) meeting in Orlando on May 19, 2018 Dosing Parameters Number of adult patients dosed 4 Patient age range 20 - 37 Number of wounds treated 7 Wound size range (cm2) 4.4 - 23.2 Cell dose concentration range (cells/mL) 1.4 x 107 - 2.5 x 107 • Administered a single intradermal injection session at baseline to assess safety prior to increasing dose regimen . One patient received a second injection session in the remaining unhealed areas of the wound 25 weeks post-administration after the baseline dose • Performed on an outpatient basis using conscious sedation rather than in a surgical suite under general anesthesia • FCX-007 continues to be well tolerated up to 52 weeks post-administration . No serious adverse events or product related adverse events . No replication competent lentivirus (RCL) detected . No autoantibody response detected, including one null genotype patient (NC-1-) 10
Pharmacology: Immunofluorescence (IF) Patient A Patient B 332 - 1 1 Laminin - COL7 NC COL7 Merged with Merged stain nuclear 25 weeks 52 weeks 4 weeks 12 weeks 25 weeks 32 weeks* *following second injection at 25 weeks • COL7 expression continues to be detected in wounded and intact samples out to 52 weeks post-administration • Increased expression of COL7 over time • Laminin-332, a basement membrane zone (BMZ) marker and a binding partner of COL7, displays linear expression in the BMZ • Linear COL7 formation observed along the BMZ (white arrows) 11
Pharmacology: Immunoelectron Microscopy (IEM) All images 25 weeks Post-Administration Samples labeled with LH24 mAb, specific to the NC-2 domain of the COL7 molecule COL7 Structure • COL7 expression continues to be detected in wounded and intact samples out to 52 weeks post-administration • Anchoring fibrils detected (AF with arrow) • Black spots represent detectable COL7 in skin samples • Detection of the NC-2 domain indicates full length COL7 is expressed Reference: Bruckner-Tuderman, Leena. Can Type VII Collagen Injections Cure Dystrophic Epidermolysis Bullosa? Molecular Therapy (2008) 17 1, 6–7. 12
FCX-007 Interim Readout: Wound Healing Wounds Treated with FCX-007 Post-Baseline Visit Percent (Number) of Wounds > 50% Percent (Number) of Wounds > 75% Healed Compared to Baseline Healed Compared to Baseline 4 weeks 100% (7/7) 86% (6/7) 12 weeks 86% (6/7) 71% (5/7) 25/32 weeks 67% (2/3) 67% (2/3) 52 weeks 100% (1/1) 0% (0/1) Untreated Control Wounds Post-Baseline Visit Percent (Number) of Wounds > 50% Healed Compared to Baseline 4 weeks 14% (1/7) 12 weeks 17% (1/6) 25/32 weeks 0% (0/2) 52 weeks 0% (0/1) 13
Phase 1 Observations • Product is well-tolerated up to 52 weeks post-administration, with no evidence of circulating autoantibodies to COL7 • Pharmacology: . Various pharmacology signals continue to be detected throughout the data set out to 52 weeks post- administration, including COL7 expression using IF and IEM, and anchoring fibrils using IEM . Increased expression of COL7 over time noted using IF . Patients dosed with higher cell concentrations resulted in more robust COL7 expression signals . LV-COL7 vector was detected in skin 25 weeks post-administration, indicating FCX-007 cells are still present in the skin • Wound healing: . Positive healing trends maintained at 4 and 12 weeks post-administration, while the majority of untreated control wounds are unhealed . A higher healing percentage of wound healing is noted 25 weeks and beyond post-administration of FCX-007 compared to untreated control . A patient’s wound receiving second injection at 25 weeks exhibited continued healing effect at 32 weeks . Based on observations, the addition of a second dose at 12 weeks post-administration may contribute to effect durability 14
Moving Forward with Phase 2 • Based on safety, pharmacology and wound healing data, we have incorporated learnings on dose and administration in our clinical trial protocol, including an increase in the overall cells administered and a reduction of the interval between injections • First Phase 2 patient dosed in June 2018 • Total of four patients enrolled (three pediatric/one adult) in Phase 2 . Expect to complete enrollment by end of 3Q2018 • Next anticipated interim data readout and trial update in 1Q2019 15
FCX-013 – Moderate to Severe Localized Scleroderma FCX-007 is a dermal fibroblast transduced with a lentiviral (LV) vector encoded with the gene for MMP-1, under control of the RheoSwitch Therapeutic System® (RTS®) Orphan Drug Designation Rare Pediatric Disease Designation Fast Track Designation 16
About Localized Scleroderma Disease Epidemiology • Excess production of collagen • Localized Scleroderma characterized by skin fibrosis ~160,000 sufferers US5 comprised of different sub- • Focus on moderate to severe types subtypes . ~90,000 patients are • Thickening may extend to considered moderate to 6 underlying tissue and muscle severe in children which may impair growth in affected limbs or forehead Current Treatments Current treatments: • Lesions appearing across joints impair motion and may • Systemic or topical be permanent corticosteroids target inflammation • UVA light therapy • Physical therapy Photo: Reprinted from the Journal of the American Academy of Dermatology, Volume 59, Issue 3, Stéphanie Christen-Zaech, Miriam D. Hakim, F. Sule Afsar, Amy S. Paller. Pediatric morphea (localized scleroderma): Review of 136 patients, Figure 1, pp. 385-396. Copyright Sept 2008. Used with permission from Elsevier Ltd. 17
FCX-013 Moving into Clinical Trials • Product profile . Dermal fibroblasts genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen . Incorporates Precigen’s RheoSwitch Therapeutic System® (RTS®) to control protein expression • Activated by an oral ligand (Veledimex) • FDA allowed IND in March 2018 • Completed GLP toxicology/biodistribution study . Bleomycin fibrosis model using immunocompromised mice (NOD/SCID) . No test article-related clinical observations, body weight changes, changes in clinical pathology parameters, gross observations or organ weight change . No significant vector biodistribution to target organs • Initiated the first investigator site for clinical enrollment in the Phase 1/2 clinical trial in August 2018 • FDA granted Fast Track Designation in late August 2018 18
FCX-013 Phase 1/2 Clinical Trial Design A Phase 1/2 Trial of a Combination of FCX-013 (Genetically-Modified Human Title Dermal Fibroblasts) plus Veledimex for the Treatment of Moderate to Severe Scleroderma (Morphea) Primary Objective To evaluate the safety of a single intradermal injection session of FCX-013 To evaluate fibrosis resolution through the following measurements: Secondary Objectives • Histology • MRI, ultrasound and durometry • Various scale assessments Investigator Site Parish Dermatology, Philadelphia, PA - Jennifer Parish, MD, Principal Investigator • 10 (approximately 5 per Phase) Number of Patients • Staggered enrollment for first 3 adults • Starting with adult patients; addition of pediatric patients after submission and approval of safety and activity data from adult patients to FDA and the DSMB Initiated the first investigator site for clinical enrollment for Phase 1/2 clinical Status trial in August 2018 19
FCX-013 Proof-of-Concept Study • Study Design D0 D28 D29 D39 . Bleomycin treated SCID mouse model . N=30 mice over test and control groups Blecomycin treatments Ligand Treatment . Assessed histologically for reduction of dermal thickness and sub-dermal muscle in the presence Cell Harvest skin of FCX-013 and oral ligand injection samples • Result . Bleomycin treatment resulted in skin fibrosis, measured by a significant increase in dermal thickness . Demonstrated that FCX-013 with ligand reduced the dermal thickness of fibrotic tissue to levels similar to non-bleomycin (saline) with ligand treated skin . Further reduced the thickness of the sub-dermal muscle layer CONTROL: Saline (no Bleo) No Cells CONTROL: Bleomycin Non-Modified Cells TEST: Bleomycin FCX-013 20
Manufacturing Experience and Solutions • Fibrocell’s existing cGMP cell therapy manufacturing facility in Exton, PA has been designated as the production site for FCX-007 and FCX-013 . ~13,000 square foot facility includes cleanroom manufacturing, cryogenic storage, QC laboratories and shipping/receiving areas . Multiple site inspections by FDA . Site for remaining clinical and future commercial manufacture of FCX-007 and FCX-013 . Existing capacity to serve the U.S. RDEB market commercially • Implemented a more efficient manufacturing process . Targets the large scale culture portion of the process with new technology . Reduces time in culture by ~50% . Aids scalability and site capacity due to smaller culture footprint without sacrificing yield . The new technology was already in use as part of our FCX-013 IND • Sufficient cGMP vector available to supply remaining clinical trials, at a minimum 21
Anticipated Milestones 2018-2019 January 2018 2Q2018 3Q2018 1Q2019 FDA allowance of Interim Phase 1 data Complete enrollment Interim data readout pediatric patient readout and trial of Phase 2 patients and trial update FCX-007 enrollment in Phase 2 update Dose first Phase 2 patient FCX-013 March 2018 3Q2018 FDA allowance of IND Initiate enrollment of Phase 1 adult patients Category Amount Cash at June 30, 2018 $15.4M, plus ~$3.5M net from July offering Share Count (Outstanding/Fully Diluted) 7.8M/16.4M as of June 30, 2018 Cash Runway Cash into 4Q2019 22
Investment Highlights • Medical breakthroughs for rare diseases of the skin and connective tissue Focus on Rare Skin • Unmet needs with no approved therapies Diseases • Significant mortality and morbidity impact on pediatric populations Proprietary Ex-Vivo Gene • Autologous fibroblasts derived from skin are the vehicle to deliver target proteins Modified Fibroblast locally to the site of disease Platform • Extensive experience culturing dermal fibroblasts, including commercial scale • FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) . Four patients enrolled in Phase 2 (three pediatric and one adult), one completed first dose . Well tolerated safety and positive early trends noted in pharmacology and wound healing Two Clinical Trial Programs • FCX-013 for the treatment of moderate to severe Localized Scleroderma . IND allowed by FDA, and initiated first investigator site for clinical enrollment for Phase 1/2 clinical trial in August 2018 • Regulatory advantages including more frequent communications with FDA, eligibility Multiple FDA Designations for Accelerated Approval and Priority Review, and Rolling Review • Two Rare Pediatric Disease Designations to potentially receive Priority Review Vouchers (PRVs) upon market authorization • In-house 13,000 square foot cGMP cell therapy manufacturing facility supporting both Internal Manufacturing FCX-007 and FCX-013 Infrastructure • Existing capacity to serve the U.S. RDEB market 23
References 1 Fine, J. et. al. (ed.). Epidermolysis Bullosa: Clinical, Epidemiologic, and Laboratory Advances and the Findings of the National Epidermolysis Bullosa Registry. The John Hopkins University Press, Baltimore, MD, 1999. 2 The Dystrophic Epidermolysis Research Association of America (DebRA). EB brochure, page 6: http://www.debra.org/DebraBrochure; accessed 11/10/17. 3 DEBRA International. What is EB Infographic: http://www.debra-international.org/what-is-eb.html; accessed 11/10/17. 4 Murauer, E, Koller, U, Pellegrini, G, De Luca, M, Bauer, J. Advances in Gene/Cell Therapy in Epidermolysis Bullosa. The Keio Journal of Medicine. 2015; 64. 5 Peterson LS et al. The epidemiology of morphea (localized scleroderma) in Olmsted County 1960-1993. J Rheumatol. 1997; 24:73-80. 6Leitenberger, et. al. Distinct autoimmune syndromes in morphea: a case study of 245 adult and pediatric cases. Arch Dermatol. 2009 May; 145(5):545-550. 24