On May 10, 2017 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported its financial results and recent corporate highlights for the quarter ended March 31, 2017 (Press release, Fortress Biotech, MAY 9, 2017, View Source [SID1234519030]).

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Dr. Lindsay A. Rosenwald, Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress had a strong start to 2017, with the launch of our subsidiaries Caelum Biosciences and Cyprium Therapeutics, which we believe strengthens our rare disease portfolio. Importantly, we announced a Cooperative Research and Development Agreement between Cyprium and the NICHD to advance the clinical development of Cyprium’s Phase 3 candidate CUTX ‐ 101 in Menkes disease, a rare and devastating pediatric illness. We also reported that Caelum announced the dosing of the final patient in its Phase 1b study of CAEL ‐ 101 for the treatment of AL amyloidosis, a fatal orphan disease that affects organ function. In addition, our established subsidiaries continued to deliver on key milestones, including Mustang Bio raising a total of approximately $95.0 million in private placement financings to support its CAR T pipeline. We look forward to the continued clinical advancement of our Fortress Companies, and are evaluating opportunities to expand our portfolio through compelling new indications and in ‐ licensing opportunities." Financial Results:  As of March 31, 2017, Fortress’ consolidated cash and cash equivalents totaled $134.0 million compared to $88.3 million at December 31, 2016, an increase of $45.7 million for the quarter. These totals exclude restricted cash of $15.9 million and cash deposits with clearing organizations of $1.0 million.  Net revenue totaled $44.7 million for the first quarter of 2017, compared to $0.7 million for the first quarter of 2016. Net total revenue as of March 31, 2017 includes $2.8 million of Fortress revenue and $41.9 million of revenue from National Holdings Corporation ("National"), which we acquired in September 2016, with no revenue attributable to National prior to the acquisition.  Research and development expenses were $7.1 million for the first quarter of 2017, of which $6.0 million was related to Fortress Companies. This compares to $7.7 million for the first quarter of 2016, of which $6.0 million was related to Fortress Companies. Non ‐ cash stock ‐ based compensation expenses included in research and development were $0.8 million for the first quarter of 2017, and $1.3 million for the first quarter of 2016.  Research and development expenses from license acquisitions totaled $1.3 million for the first quarter of 2017, compared to $0.1 million for the first quarter of 2016.  General and administrative expenses were $10.3 million for the first quarter of 2017, of which $7.1 million was related to Fortress Companies. This compares to $7.9 million for the first quarter of 2016, of which $4.0 million was related to Fortress Companies. Non ‐ cash stock ‐ based compensation expenses included in general and administrative expenses were $2.1 million for the first quarter of 2017, and $1.6 million for the first quarter of 2016.  National’s operating expenses totaled $43.1 million for the first quarter of 2017, with no expenses attributable to National prior to our acquisition of the company in September 2016.  Net loss attributable to common stockholders was $12.0 million, or $0.30 per share, for the first quarter of 2017, compared to a net loss attributable to common stockholders of $12.2 million, or $0.31 per share, for the first quarter of 2016. Recent Fortress Biotech and Fortress Company Highlights: Fortress Biotech, Inc.  Fortress recently launched two new Fortress Companies: Caelum Biosciences to develop therapies for amyloid light chain ("AL") amyloidosis, and Cyprium Therapeutics to develop novel therapies for the treatment of Menkes disease and related copper metabolism disorders.
Avenue Therapeutics, Inc.
In February 2017, two continuation patents covering methods of administration for intravenous tramadol for the treatment of acute pain were issued by the U.S. Patent and Trademark Office ("USPTO").
Caelum Biosciences, Inc.
In January 2017, Michael Spector was appointed Chief Executive Officer and a member of the Board of Directors of Caelum.
In January 2017, Caelum entered into an agreement with Columbia University ("Columbia") to secure exclusive worldwide license rights to CAEL ‐ 101, a chimeric fibril ‐ reactive monoclonal antibody.
In April 2017, the U.S. Department of Health & Human Services confirmed the transfer of two U.S. Food and Drug Administration ("FDA") Orphan Drug Designations for CAEL ‐ 101 from Columbia to Caelum. The designations cover use as a therapeutic agent for patients with AL amyloidosis and use as a radio ‐ imaging agent in amyloidosis.
In May 2017, Columbia dosed the final patient in the Phase 1b trial of CAEL ‐ 101. Preliminary Phase 1b data are expected mid ‐ 2017, with full data anticipated by the end of the year.
Checkpoint Therapeutics, Inc.
In February 2017, the USPTO issued a composition of matter patent for CK ‐ 101, an oral, third ‐ generation epidermal growth factor receptor ("EGFR") inhibitor in development for the treatment of EGFR mutation ‐ positive non ‐ small cell lung cancer.
In April 2017, preclinical data on CK ‐ 101 and anti ‐ programmed cell death ligand ‐ 1 ("PD ‐ L1") antibody, CK ‐ 301, were presented in poster sessions at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
Cyprium Therapeutics, Inc.
In March 2017, Lung Yam, M.D., Ph.D., was appointed Chief Executive Officer and a member of the Board of Directors of Cyprium.
In March 2017, Cyprium entered into a Cooperative Research and Development Agreement ("CRADA") with the Eunice Kennedy Shriver National Institute of Child Health and Human Development ("NICHD"), part of the National Institutes of Health ("NIH"), to advance the clinical development of Phase 3 candidate CUTX ‐ 101 (Copper Histidinate injection) for the treatment of Menkes disease.
Also effective in March 2017, Cyprium and the NICHD entered into a worldwide, exclusive license agreement to develop and commercialize the adeno ‐ associated virus ("AAV") ‐ based gene therapy AAV ‐ ATP7A to deliver working copies of the copper transporter that is defective in Menkes patients. AAV ‐ ATP7A will be used in combination with CUTX ‐ 101.
Mustang Bio, Inc.
From October 2016 to March 2017, Mustang closed on a total of approximately $95.0 million in private placement financings, prior to fees and expenses.
In April 2017, Mustang appointed Manuel Litchman, M.D., as President and Chief Executive Officer, as well as a member of the Board of Directors

On May 9, 2017 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2017, and provided an operating forecast and program updates (Filing, Q1, Ligand, 2017, MAY 9, 2017, View Source [SID1234519019]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time.

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"We are pleased to be reporting a substantial increase in first quarter royalty revenue led by Promacta, Kyprolis and EVOMELA, as well as strong cash flow from operations. In addition to achieving solid sales growth, our partners made important clinical, regulatory and commercial progress on a global basis," said John Higgins, Chief Executive Officer. "During the first quarter we completed enrollment in a Phase 2 clinical trial with our novel, small-molecule GRA program for the treatment of type 2 diabetes mellitus, and we look forward to reporting topline results this September. We also added to our Shots-on-Goal business model with new licensing agreements including those for OmniAb and Captisol."

First Quarter 2017 Financial Results

Total revenues for the first quarter of 2017 were $29.3 million, compared with $29.6 million for the same period in 2016. Royalties were $24.2 million, compared with $14.4 million for the same period in 2016, an increase of 68%, primarily due to higher royalties from Promacta and Kyprolis and new royalties from EVOMELA this period, compared to a year ago. Material sales were $1.1 million, compared with $5.3 million for the same period in 2016 due to timing of Captisol purchases for use in clinical trials and commercial products. License fees, milestones and other revenues were $3.9 million, compared with $9.9 million for the same period in 2016, which included receipt of a $6.0 million approval milestone for EVOMELA.

Cost of goods sold was $0.3 million for the first quarter of 2017, compared with $1.0 million for the same period in 2016 due to the timing and mix of Captisol sales. Amortization of intangibles was $2.7 million, compared with $2.5 million for the same period in 2016. Research and development expense was $8.7 million, compared with $4.0 million for the same period of 2016 due to enrollment costs of our Phase 2 GRA trial and non-cash stock-based compensation expense. General and administrative expense was $7.3 million, compared with $7.1 million for the same period in 2016.

Net income for the first quarter of 2017 was $5.1 million, or $0.22 per diluted share, compared with $6.6 million, or $0.30 per diluted share for the same period in 2016. Adjusted net income for the first quarter of 2017 was $12.6 million, or $0.57 per diluted share, compared with $13.6 million, or $0.63 per diluted share for the same period in 2016.


As of March 31, 2017, Ligand had cash, cash equivalents and short-term investments of $159.4 million. Cash generated from operations was $24.2 million for the 2017 first quarter.

2017 Financial Forecast

The Company expects 2017 revenues to consist of three components: royalties, material sales and contract (license and milestone) revenue. Ligand affirms previous guidance of 2017 core revenue to include royalties of approximately $87 million, material sales of approximately $23 million and contract payments of at least $20 million. During 2017, Ligand estimates it could potentially receive up to an additional $24 million of contract payments; however, external events are out of Ligand’s control so the Company will provide more information about the timing and probability for additional contract revenue, if any, expected to be booked in 2017 as the year progresses. Ligand notes that with core revenue of $130 million, adjusted earnings per diluted share would be approximately $2.70. This amount is expected to be higher in the event additional contract revenue is received in 2017.

First Quarter 2017 and Recent Business Highlights

Portfolio Program Progress

Promacta/Revolade

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Novartis reported first quarter 2017 net sales of Promacta/Revolade (eltrombopag) of $175 million, a $44 million or 34% increase over the same period in 2016.
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Novartis reported Revolade (eltrombopag) was approved in Canada for the treatment of pediatric (≥1 years to <18 years) chronic immune thrombocytopenia purpura to increase platelet counts in patients who have had an insufficient response to corticosteroids or immunoglobulins.
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Novartis announced the publication of a study conducted by the National Institutes of Health demonstrating that 58% of patients with treatment-naïve severe aplastic anemia achieved complete response at six months when treated with eltrombopag at the initiation of and concurrent with standard immunosuppressive treatment. The data are published in the latest issue of The New England Journal of Medicine.

Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

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On April 26, 2017, Amgen reported first quarter 2017 net sales of Kyprolis (carfilzomib) of $190 million, a $36 million or 23% increase over the same period in 2016.
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On February 28, 2017, Amgen announced positive results from a planned overall survival (OS) interim analysis of the Phase 3 head-to-head ENDEAVOR trial. The study met the key secondary endpoint of OS, demonstrating that patients with relapsed or refractory multiple myeloma treated with Kyprolis (carfilzomib) and dexamethasone (Kd) lived 7.6 months longer than those treated with Velcade (bortezomib) and dexamethasone (Vd) (median OS 47.6 months for Kd versus 40.0 for Vd, HR = 0.79, 95 percent CI, 0.65 – 0.96).
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On March 1, 2017, Amgen announced that new data from the Kyprolis (carfilzomib) clinical development program would be presented at the 16th International Myeloma Workshop, March 1-4, 2017, in New Delhi.

Additional Pipeline and Partner Developments

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Melinta Therapeutics announced that the new drug applications (NDAs) for IV and oral Baxdela (delafloxacin) for the treatment of patients with acute bacterial skin and skin structure infections (ABSSSI) were accepted for filing by the Food and Drug Administration (FDA) and were granted a Prescription Drug User Fee Act (PDUFA) date of June 19, 2017. Additionally, Melinta announced that the FDA does not plan to hold an Advisory Committee meeting for the NDAs. If approved, Ligand is entitled to receive a 2.5% royalty on net sales of the IV formulation of Baxdela and a $1.5 million approval milestone payment.
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Melinta Therapeutics announced signing a development and commercialization agreement with Menarini Group, granting Menarini exclusive rights to commercialize delafloxacin under its own brands in 68 countries in Europe, Asia-Pacific including China, South Korea and Australia (excluding Japan), and the Commonwealth of Independent States including Russia.




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Retrophin announced plans to initiate a single Phase 3 clinical trial to enable an NDA filing for sparsentan for the treatment of focal segmental glomerulosclerosis. The trial will include an interim analysis of proteinuria as a surrogate endpoint to serve as the basis for an NDA filing for Subpart H accelerated approval of sparsentan. Retrophin expects to initiate the trial in the second half of 2017.
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Sage Therapeutics presented brexanolone data at the American Academy of Neurology 2017 annual meeting.
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Aldeyra provided an update on its Phase 3 clinical program of ADX-102 in noninfectious anterior uveitis and anticipates beginning the Phase 3 trial in the second quarter of 2017.
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Aldeyra announced the last patient had completed dosing in Aldeyra’s multicenter, double-blind, randomized Phase 2b clinical trial of ADX-102 in allergic conjunctivitis.
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Biocad announced receiving marketing authorization from the Ministry of Health of the Russian Federation for its interferon beta-1a biosimilar of Merck’s Rebif.
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Merck announced it stopped the Phase 2/3 EPOCH study evaluating verubecestat in people with mild-to-moderate Alzheimer’s disease due to the conclusion that the efficacy endpoint could not be achieved. No safety concerns were noted. Results from EPOCH will be analyzed and presented at an upcoming scientific meeting. The external Data Monitoring Committee recommended that the ongoing Phase 3 APECS study, which is evaluating verubecestat in people with prodromal Alzheimer’s disease, continue unchanged. Results from the APECS study are expected in February 2019.
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Novartis announced that it had exercised an option to in-license ECF843 (Lubricin) for ophthalmic indications from Lubris Biopharma. Ligand acquired economic rights to the Lubricin program from Selexis, SA in 2015.
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Opthea Limited announced positive results from its Phase 1/2a clinical trial of OPT-302 for wet age-related macular degeneration (wet AMD). Opthea is planning to initiate a Phase 2b trial in wet AMD and a Phase 2a trial in diabetic macular edema in the second half of 2017.
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Viking Therapeutics announced positive initial results from a proof-of-concept study of VK2809 in an in vivo model of glycogen storage disease 1a (GSD 1a) and announced funding of initial clinical development of VK2809 for treatment of GSD 1a with plans to file an investigational new drug (IND) application in the second half of 2017.
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Janssen filed an IND application for an antibody discovered using Ligand’s OmniAb technology. The IND filing resulted in a $1 million milestone payment to Ligand. Janssen has a royalty-free license to the OmniAb technology (entered into with OMT in October of 2013), but will potentially pay Ligand further development and commercial milestones upon clinical success and regulatory approval of any therapeutic developed using the OmniAb technology.
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Marinus Pharmaceuticals presented Phase 1 clinical data showing the safety and tolerability of ganaxolone IV at the 6th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures.
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Merck KGaA announced it licensed rights to develop Captisol-enabled VX-970 from Vertex Pharmaceuticals. Economic terms of the original agreement between Ligand and Vertex remained unchanged.
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XTL Biopharmaceuticals announced the receipt of additional preclinical data regarding the role of hCDR1 as a potential treatment for Sjögren’s syndrome from Prof. Edna Mozes of The Weizmann Institute of Science and the developer of hCDR1.

New Licensing Deals

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Ligand announced a worldwide platform license agreement with bluebird bio, Inc. Under the license, bluebird will be able to use the OmniRat, OmniMouse and OmniFlic platforms to discover fully human mono- and bispecific antibodies and antibody fragments. Ligand is eligible to receive annual platform access payments, development milestone payments and royalties for each product incorporating an OmniAb antibody. Bluebird will be responsible for all costs related to the programs. Ligand previously disclosed rights to a single-antibody partnership had been licensed to bluebird, but this new agreement gives bluebird full access to the OmniAb platform.
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Ligand announced an expansion of its license with Sermonix Pharmaceuticals to include worldwide rights to develop and commercialize oral lasofoxifene. Ligand originally licensed U.S. rights to oral lasofoxifene to Sermonix in February of 2015, and has now expanded the agreement to include the rest of the world. Ligand is entitled to commercial milestones and royalties on net sales ranging from 6-10% upon commercialization of oral lasofoxifene.

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Ligand announced a commercial license and supply agreement with Marinus Pharmaceuticals granting rights to use Captisol in the formulation of IV ganaxolone. Ligand is entitled to milestone payments, royalties and revenue from Captisol material sales related to IV ganaxolone.
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Ligand entered into a Captisol Clinical Use/Supply Agreement with Eisai.

Internal Glucagon Receptor Antagonist (GRA) Program

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Ligand announced the completion of enrollment in the Company’s Phase 2 clinical trial with its novel, small-molecule GRA program (LGD-6972) for the treatment of type 2 diabetes mellitus. The Company expects to report topline results in September 2017.

Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share, in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include stock-based compensation expense, amortization of debt-related costs, amortization related to acquisitions, changes in contingent liabilities, net losses of Viking Therapeutics, mark-to-market adjustment for amounts owed to licensors, fair value adjustments to Viking Therapeutics convertible note receivable and warrants, unissued shares relating to the Senior Convertible Note, and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included in this press release. However, other than with respect to total revenue, the Company only provides guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, net losses of Viking Therapeutics, mark-to-market adjustments for amounts owed to licensors, effects of any discrete income tax items and fair value adjustments to Viking Therapeutics convertible note receivable. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

On May 9, 2017 Atossa Genetics, Inc. (NASDAQ: ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that the Institutional Review Board associated with Montefiore Medical Center (Biomedical Research Alliance of New York IRB) has approved the Fulvestrant Microcatheter Phase 2 study that was recently transferred to Montefiore (Press release, Atossa Genetics, MAY 9, 2017, View Source [SID1234518985]).

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The Fulvestrant Microcatheter Phase 2 study includes women with ductal carcinoma in-situ (DCIS) or invasive breast cancer slated for mastectomy or lumpectomy. This study will assess the safety, tolerability and distribution of fulvestrant when delivered directly into breast milk ducts of these patients compared to those who receive the same product intramuscularly. The secondary objective of the study is to determine if there are changes in the expression of Ki67 as well as estrogen and progesterone receptors between a pre-fulvestrant biopsy and post-fulvestrant surgical specimen. Digital breast imaging before and after drug administration in both groups will also be performed to determine the effect of fulvestrant on any lesions as well as breast density of the participant. Six study participants will receive the standard intramuscular fulvestrant dose of 500 mg to establish the reference drug distribution, and 24 participants will receive fulvestrant by intraductal instillation utilizing Atossa’s microcatheter device.

"This study was initiated at The Columbia University Medical Center Breast Cancer Program and then transferred to Montefiore Medical Center at the beginning of 2017 when the principal investigator moved his practice to Montefiore," commented Steven Quay, CEO and President. "We are pleased that the study continues to move forward at Montefiore, which is a leading center for breast cancer treatment."

On May 9, 2017 Aeglea BioTherapeutics, Inc., (NASDAQ:AGLE) a biotechnology company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat rare genetic diseases and cancer, reported a corporate update and reported financial results for the first quarter ended March 31, 2017 (Press release, Aeglea BioTherapeutics, MAY 9, 2017, View Source [SID1234518984]).

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"AEB1102 remains the primary focus of our clinical and preclinical work across rare genetic diseases and cancer," said David G. Lowe, Ph.D., chief executive officer of Aeglea. "We are especially pleased with recent preclinical data which demonstrated that AEB1102 was not immunosuppressive in combination with anti-PD-1 immune checkpoint inhibitors. We believe this opens a unique opportunity at the intersection of tumor metabolism and existing or emerging therapies in immuno-oncology."
Recent Highlights

Presented preclinical data at the 2017 American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating that reducing systemic arginine with AEB1102 did not suppress the immune response induced by anti-PD-1 and anti-PD-L1, and exerted an additive anti-tumor and synergistic survival benefit.

Expanded research and development capabilities with the opening of an internal laboratory at Aeglea in Austin, TX.

Upcoming Events
David G. Lowe, Ph.D., chief executive officer of Aeglea, will present a corporate update at the UBS 2017 Global Healthcare Conference being held May 22 – 24, 2017 in New York City.

First Quarter 2017 Financial Results At March 31, 2017, Aeglea had available cash, cash equivalents and marketable securities of $57.9 million. Based on Aeglea’s current operating plan, management believes that it has sufficient capital resources to fund anticipated operations through March 31, 2019. Aeglea recognized grant revenues of $1.0 million in the first quarter of 2017, compared with $0.9 million in the first quarter of 2016. The grant revenues are the result of a $19.8 million research grant received from the Cancer Prevention and Research Institute of Texas (CPRIT). The increase was primarily due to higher qualifying expenditures associated with AEB1102 for grant-related clinical trials. Research and development expenses totaled $4.9 million for the first quarter of 2017, compared with $3.6 million for the first quarter of 2016. The increase was primarily associated with hiring additional personnel to expand Aeglea’s internal regulatory, laboratory, and clinical development capabilities. General and administrative expenses totaled $2.3 million for the first quarter of 2017, compared to $1.8 million in the first quarter of 2016. This increase was primarily due to additional compensation and personnel costs, and increased costs associated with operating as a public company. Net loss totaled $6.2 million and $4.5 million for the first quarter of 2017 and 2016, respectively.

On May 9, 2017 Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, and Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported that two abstracts will be presented in oral sessions at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) from May 10-13, 2017 in Washington, D.C (Press release, Intrexon, MAY 9, 2017, View Source [SID1234518983]).

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The schedule for each presentation is as follows:

Session: Pharmacology, Toxicology and Assay Development
Presentation: Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Presenter: Anna Malyala, PhD, Director, New Product Development, Fibrocell Science
Abstract ID: 301
Presentation on Thursday, May 11, 2017, 4:45 – 5:00 p.m. ET
Room: Maryland ABC Room

Session: Somatic Stem Cell Therapies
Presentation: Development of an Autologous Gene-Modified Cell Therapy for the Treatment of Linear Scleroderma
Presenter: Darby Thomas, PhD, Director, Rare Diseases, Human Therapeutics Division, Intrexon Corporation
Abstract ID: 740
Presentation on Saturday, May 13, 2017, 11:00 – 11:15 a.m. ET
Location: Delaware AB Room

About FCX-007

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 type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7 and is being developed in collaboration with Intrexon Corporation. 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. The FDA has granted Orphan Designation to FCX-007 for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, FCX-007 has been granted Rare Pediatric Disease Designation and Fast Track Designation by the FDA for treatment of RDEB.

About FCX-013

Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment of linear scleroderma, a form of localized scleroderma. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound to control future protein expression once the initial fibrosis has been resolved. FCX-013 is designed to be injected under the skin at the location of the fibrosis where the genetically-modified fibroblast cells will produce a protein to break down excess collagen accumulation. The patient takes an oral compound to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further production of the subject protein by FCX-013. Fibrocell has received Orphan Drug Designation from the FDA for FCX-013 for the treatment of localized scleroderma.