On March 18, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on dermatological and immuno-inflammatory diseases, reported financial results for the fourth quarter and full year 2018, and provided an update on its clinical development and commercial programs (Press release, Aclaris Therapeutics, MAR 18, 2019, View Source [SID1234534422]).

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In November 2018, Aclaris acquired the worldwide rights to RHOFADE (oxymetazoline hydrochloride) cream, 1% from Allergan Sales, LLC.
In December 2018, Aclaris began promoting RHOFADE, which is approved in the United States for the topical treatment of persistent facial erythema (redness) associated with rosacea in adults.
In February 2019, at the annual National Sales Meeting, Aclaris appointed Jeff Wayne as interim Head of Commercial and officially relaunched RHOFADE.
Today Aclaris is providing an update, including new photos, from AUATB-201 Topical, an open-label clinical trial in patients with eyebrow loss due to alopecia areata (AA), including patients with alopecia totalis or alopecia universalis.
"On the commercial side of the company, we made a change in leadership as we focus on the relaunch of RHOFADE in 2019. Our development stage pipeline continues to advance with multiple data read outs expected during the course of the year across both our JAK inhibitor and A-101 clinical programs. Finally, we look to move our first Confluence originated asset into the clinic in the second half of the year; a significant milestone given we acquired and integrated the team just 18 months ago. Given the anticipated data readouts, we expect 2019 to be a watershed year as we become a fully integrated biopharmaceutical company," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris.

Clinical Pipeline Update:

A-101 45% Topical Solution:
Two pivotal Phase 3 trials, named THWART-1 and THWART-2, for the treatment of common warts are progressing as planned. THWART-2 has completed enrollment, and THWART-1 is expected to complete enrollment by the end of this month. These two trials will enroll a total of approximately 1,000 patients across both studies, and topline data are expected in the second half of 2019.
An open-label safety extension trial investigating A-101 45% Topical Solution for the treatment of common warts is also ongoing; if the results of these trials are positive, NDA submission is expected in the first half of 2020.
JAK Inhibitor Trials:
We have completed enrollment in all of the following JAK inhibitor trials:
AA-201 Topical – This ongoing Phase 2 randomized, double-blinded, parallel-group, vehicle-controlled trial is evaluating the safety, efficacy, and dose response of two concentrations of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 129 patients with AA. Data are expected in the second quarter of 2019.
AGA-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 31 patients with androgenetic alopecia (AGA), also known as male/female pattern hair loss. 6-month data are expected in the second quarter of 2019 and 12-month data are expected in the second half of 2019.
VITI-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the repigmentation of facial skin in 34 patients with vitiligo. 6-month interim data are expected in the second quarter of 2019 and 12-month data are expected in the second half of 2019.
AD-201 Topical – This ongoing Phase 2 open-label clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, in 22 adult patients with moderate-to-severe atopic dermatitis (AD). Data are expected in mid-2019.
AUAT-201 Oral – This ongoing randomized, double-blinded, parallel-group, placebo-controlled trial is evaluating the safety, efficacy, and dose response of three concentrations of ATI-501, an oral JAK 1/3 inhibitor, on the regrowth of hair in 87 patients with AA. Data are expected in the second half of 2019.
ATI-450 (MK-2 Inhibitor) – We expect to submit an Investigational New Drug (IND) application for rheumatoid arthritis to the FDA in mid-2019. If the IND is allowed by the FDA, we expect to initiate a Phase 1 and Phase 2 trial in the second half of 2019.
Recent Corporate Highlights:

Significant presence at 2019 Annual Academy of Dermatology (AAD) Annual Meeting to support the relaunch of RHOFADE. Aclaris also presented 6 posters, including 2 oral presentations regarding the clinical results of ESKATA for the treatment of raised seborrheic keratosis and A-101 45% topical solution for the treatment of common warts.
Aclaris received approval from the Swedish Medical Products Agency to market ESKATA (hydrogen peroxide) cutaneous solution, 685 mg, for the treatment in adults of seborrheic keratoses that are not pedunculated and have up to a maximum diameter of 15 mm each. Aclaris also has received approval to market the medicine in the United Kingdom, Iceland, and Belgium. Aclaris is seeking a commercial partner or partners to market the medicine as an aesthetic skin treatment in various European countries with the brand name ESKATA in Finland, Iceland, Netherlands, Norway, Portugal, Spain, Sweden, Czech Republic, and Belgium, and the brand name ESKERIELE in Austria, France, Germany, Ireland, Italy, and the United Kingdom.
Canadian partner Cipher Pharmaceuticals Inc. submitted a New Drug Submission for A-101 40% Topical Solution for the treatment of raised SKs, which was accepted for review by Health Canada in December 2018.
Commercial Update:

Jeff Wayne, Aclaris’ Vice President of Business Development, was recently appointed as interim Head of Commercial. Mr. Wayne brings over 30 years of pharmaceutical experience with the majority spent in dermatology. During his career, he has held positions of increasing responsibility in sales, marketing, and general management with Galderma, Intendis, Promius Pharma (where he built, launched, and led the commercial organization), Onset Dermatologics, and LEO Pharma. He launched METROGEL in both the United States and Canada and was responsible for the marketing of FINACEA in the United States; two medications prescribed for the treatment of rosacea. In his role as Vice President, Business Development, he managed the RHOFADE transaction from the beginning, providing a seamless transition of leadership.
Following our National Sales Meeting held mid-February, RHOFADE was officially relaunched, and featured prominently at the Aclaris booth and throughout the convention venue at the 2019 AAD Annual Meeting.
The Aclaris field sales team was realigned to optimize reach and call frequency on current and potential RHOFADE prescribers, as well as the top 10 ESKATA accounts in each territory.
Comparing the 4-week period ended March 8, 2019 to the immediately prior 4-week period, the IQVIA data showed an 8.4% increase in total prescriptions for RHOFADE.
Based on these early prescription trends, we believe the response to the RHOFADE message, which emphasizes the need to treat all rosacea patients with persistent facial erythema, appears positive.
Financial Highlights

Liquidity and Capital Resources

As of December 31, 2018, Aclaris had aggregate cash, cash equivalents and marketable securities of $168.0 million compared to $208.9 million as of December 31, 2017. The $30.9 million decrease during the year ended December 31, 2018 included:

Aggregate net proceeds of $100.2 million from the sale of common stock in a follow-on public offering of common stock in October 2018;
$67.1 million of cash used to acquire the global rights to RHOFADE;
$29.9 million of cash, net of issuance costs, borrowed under the loan agreement with Oxford Finance LLC;
$1.4 million in property and equipment purchases; and
Net loss of $132.7 million, offset by $9.4 million of net cash provided by working capital and $21.9 million of non-cash stock-based compensation expense, depreciation, and amortization.
Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2018 will be sufficient to fund its operations into the fourth quarter of 2020, without giving effect to any potential new business development transactions or financing activities.

Fourth Quarter 2018 Financial Results

Net loss was $38.6 million for the fourth quarter of 2018, compared to $22.9 million for the fourth quarter of 2017.
Net revenues were $3.7 million for the quarter ended December 31, 2018, which consisted of $0.8 million of net ESKATA sales, $1.1 million of net RHOFADE sales (December only), $1.3 million of contract research revenues, and $0.5 million of other revenue. This compared to $1.0 million for the quarter ended December 31, 2017, all of which was contract research revenues. Cost of revenues was $3.5 million for the quarter ended December 31, 2018, compared to $0.8 million for the quarter ended December 31, 2017.
Total operating expenses for the fourth quarter of 2018 were $39.2 million, compared to $25.7 million for the fourth quarter of 2017.
Research and development expenses were $19.5 million for the fourth quarter of 2018, compared to $13.2 million for the fourth quarter of 2017. The increase of $6.3 million was mainly the result of the continued growth of Aclaris’ JAK inhibitor and common wart programs, as multiple Phase 2 trials of ATI-501 and ATI-502 and Phase 3 trials of A-101 45% were ongoing in the fourth quarter of 2018, as well as the increased headcount to support these programs.
Sales and marketing expenses were $13.0 million for the fourth quarter of 2018, compared to $6.6 million for the fourth quarter of 2017. The increase of $6.4 million was the result of increases in direct marketing and professional fees, as well as other commercial and personnel expenses incurred to support the continued commercialization of ESKATA following its launch in May 2018.
General and administrative expenses were $6.7 million for the fourth quarter of 2018, compared to $5.9 million for the fourth quarter of 2017. The increase was driven by headcount increases, and legal and business development costs related to the RHOFADE acquisition in the fourth quarter of 2018.
Full Year 2018 Financial Results

Net loss was $132.7 million for the year ended December 31, 2018, compared to $68.5 million for the year ended December 31, 2017.
Net revenues were $10.1 million for the year ended December 31, 2018, which consisted of $2.8 million of net ESKATA sales, $1.1 million of net RHOFADE sales (December only), $4.7 million of contract research revenues, and $1.5 million of other revenue. This compared to $1.7 million for the year ended December 31, 2017, all of which was contract research revenues. Cost of revenues was $6.9 million for the year ended December 31, 2018, compared to $1.2 million for the year ended December 31, 2017.
Total operating expenses were $138.7 million for the year ended December 31, 2018, compared to $72.9 million for the year ended December 31, 2017. Net cash used in operating activities was $100.8 million for the year ended December 31, 2018, compared to $54.7 million for the year ended December 31, 2017.
Research and development expenses were $63.0 million for the year ended December 31, 2018, compared to $39.8 million for the year ended December 31, 2017. The increase of $23.2 million was mainly the result of the continued growth of Aclaris’ JAK inhibitor and common wart programs, as multiple Phase 2 trials of ATI-501 and ATI-502 and Phase 3 trials of A-101 45% were conducted throughout 2018, as well as the increased headcount to support these programs. These increases were offset by a decrease in costs related to the development of ESKATA as Aclaris submitted the NDA for ESKATA in February 2017 following the completion of the clinical trials.
Sales and marketing expenses were $48.0 million for the year ended December 31, 2018, compared to $13.8 million for the year ended December 31, 2017. The increase of $34.2 million was the result of increases in direct marketing and professional fees, as well as other commercial expenses incurred to support the launch and commercialization of ESKATA in May 2018. Personnel expenses also increased as Aclaris completed the hiring of its field sales force early in 2018.
General and administrative expenses were $27.6 million for the year ended December 31, 2018, compared to $19.3 million for the year ended December 31, 2017. The increase of $8.3 million was the result of higher personnel expenses, due to increased headcount to support the commercial launch of ESKATA, as well as legal and business development costs related to the RHOFADE acquisition during 2018. General and administrative expenses for the year ended December 31, 2018 also included a $1.5 million ESKATA-related milestone payment, whereas the year ended December 31, 2017 included a $1.0 million ESKATA-related milestone payment.
As of December 31, 2018, Aclaris had 41.2 million shares of common stock outstanding.
2019 Financial Outlook

Aclaris expects 2019 GAAP research and development (R&D) expenses to be in the range of $61 to $64 million, including estimated stock-based compensation of $7 million. This expense guidance for R&D in 2019 contemplates the completion of Aclaris’ Phase 2 clinical trials in AA, open label trials in AGA, vitiligo and AD, and two pivotal Phase 3 trials in common warts, as well as the further advancement of Aclaris’ preclinical pipeline compounds, including ATI-450 and ATI-1777.
Aclaris expects 2019 GAAP sales and marketing (S&M) expenses to be in the range of $37 to $40 million, including estimated stock-based compensation of $4 million. This expense guidance for S&M in 2019 contemplates all salesforce costs and the selling and marketing initiatives to support our commercial brands.
Aclaris expects 2019 GAAP general and administrative (G&A) expenses to be in the range of $29 to $31 million, including estimated stock-based compensation of $10 million. This expense guidance for G&A in 2019 contemplates additional medical affairs, legal, and compliance activities to support our commercial brands.
Company to Host Conference Call

Management will conduct a conference call at 8:00 AM ET today to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 9765869prior to the start of the call.

On March 18, 2019 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, reported financial results and recent corporate highlights for the fourth quarter and full year ended December 31, 2018 (Press release, Fortress Biotech, MAR 18, 2019, View Source [SID1234534421]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress achieved significant milestones in 2018 and early 2019, closing numerous business development transactions and developing high-quality partnerships with Alexion Pharmaceuticals and InvaGen Therapeutics (a subsidiary of Cipla Limited), which could provide our shareholders with long-term revenue streams. These contingent sale agreements have the potential for combined aggregate proceeds to shareholders of up to $680 million (excluding potential contingent value right ("CVR") amounts based on drug sales). Also in 2018, our seven marketed specialty dermatology products generated net revenue of $23.4 million and $3.8 million in net income. Additionally, Fortress and our development partners, continued to efficiently advance more than 25 development-stage programs across seven therapeutic categories within a streamlined operating structure, allowing us to create value while mitigating risk for our shareholders."

Dr. Rosenwald continued, "2019 is positioned to be a successful year on the business development and partnership fronts. Our world-class business development team continues to identify and in-license high-potential marketed and development-stage assets to further expand our portfolio of product opportunities. We look forward to several important data readouts and the potential for our first New Drug Application ("NDA") filing later this year."

Financial Results1:

·As of December 31, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $99.2 million, compared to $114.4 million as of September 30, 2018, and $147.0 million as of December 31, 2017, a decrease of $15.2 million for the fourth quarter and a decrease of $47.8 million year-to-date.
·Fortress’ net revenue totaled $26.9 million as of December 31, 2018, compared to $17.2 million as of December 31, 2017, an increase of $9.7 million year-to-date.
·Research and development expenses were $83.3 million for the year ended December 31, 2018, of which $78.5 million was related to partner companies. This compares to $48.3 million for 2017, of which $40.6 million was related to partner companies. Non-cash, stock-based compensation expenses included in research and development were $5.3 million for the year ended December 31, 2018, compared to $4.0 million for 2017.

·Research and development expenses from license acquisitions totaled $4.1 million for the year ended December 31, 2018, compared to $4.2 million for 2017.
·General and administrative expenses were $53.4 million for the year ended December 31, 2018, of which $33.0 million was related to partner companies. This compares to $50.9 million for 2017, of which $31.4 million was related to partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $9.7 million for the year ended December 31, 2018, compared to $9.4 million for 2017.
·Net loss attributable to common stockholders was $84.1 million, or $1.94 per share, for the year ended December 31, 2018, compared to a net loss attributable to common stockholders of $66.9 million, or $1.61 per share, for 2017.

2018 and Recent Corporate Highlights2:

Marketed Dermatology Products

·In 2018, our seven marketed products generated net revenue of $23.4 million and $3.8 million in net income.
·In the fourth quarter of 2018, we launched Exelderm, a cream and solution for fungal infections.
·Also in 2018, we signed a co-promotion agreement with Crown Laboratories to promote Triderm, a topical corticosteroid indicated for the relief of the inflammatory and pruritic manifestations of corticosteroid-responsive dermatoses; Ala-quin, a cream for mixed infections; and Ala-scalp, a hydrocortisone lotion for corticosteroid-responsive dermatoses.
·Additionally, we anticipate launching at least one or two new prescription drugs in 2019.

IV Tramadol

·In May 2018, we announced that the first pivotal Phase 3 trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 48 hours compared to placebo in patients with moderate to moderately severe postoperative pain following bunionectomy surgery. In addition, the trial met its key secondary endpoints and demonstrated a clear dose response.
·In November 2018, we announced definitive agreements regarding an equity investment and contingent acquisition by InvaGen, a subsidiary of Cipla Limited, a leading pharmaceutical company. The first stage of the transaction closed in February 2019, and InvaGen acquired 5,833,333 shares of Avenue Therapeutics’ common stock at $6.00 per share for total gross consideration of $35.0 million, representing a 33.3% stake in Avenue’s capital stock on a fully diluted basis.
·In December 2018, we announced that the first patient has been dosed in a pivotal Phase 3 clinical trial of IV tramadol for the management of moderate to moderately severe pain in patients following abdominoplasty surgery. Topline data is expected to be available in mid-2019.
·IV Tramadol is currently in development at our partner, Avenue Therapeutics, Inc.

CAEL-101

·In January 2019, Caelum Biosciences, Inc. ("Caelum") signed a collaboration agreement with Alexion Pharmaceuticals, Inc. ("Alexion") to advance the development of CAEL-101. Under the terms of the agreement, Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 2 data for pre-negotiated economics. Alexion will make payments to Caelum totaling $60 million, including the purchase price for the equity and milestone-dependent development funding payments. The collaboration also provides for potential additional payments of up to $500 million, including the upfront, regulatory and commercial milestone payments, in the event Alexion exercises the acquisition option.

·In March 2018, a new analysis of data from the Phase 1b trial of CAEL-101 (mAb 11-1F4), a light chain fibril-reactive monoclonal antibody ("mAb") 11-1F4, for the treatment of relapsed or refractory amyloid light chain ("AL") amyloidosis was presented at the 16th International Symposium on Amyloidosis. The data demonstrated a correlation between a sustained decrease in N-terminal pro-brain natriuretic peptide (NT-proBNP) levels and an improvement in global longitudinal strain ("GLS") following CAEL-101 treatment in patients with cardiac AL amyloidosis.
·In June 2018, Caelum announced a complete analysis of cardiac data from a Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment of relapsed or refractory AL amyloidosis demonstrating CAEL-101’s potential to improve myocardial function as assessed by GLS and generate a sustained decrease in N-terminal pro-brain natriuretic peptide levels in AL amyloidosis patients experiencing cardiac involvement. Columbia University presented the data at the American Society of Echocardiography’s 29th Annual Scientific Sessions.
·In December 2018, Caelum announced additional GLS data from the Phase 1b study of CAEL-101 in patients with cardiac AL amyloidosis, which further confirmed CAEL-101’s efficiency improving GLS and NT-proBNP. The Company also announced imaging data from a preclinical study that demonstrate the potential of using radiolabeled CAEL-101 for real-time imaging of human amyloidosis in vivo. The data were presented during two oral sessions at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting.

Triplex

·The multicenter Phase 2 study of Triplex for cytomegalovirus ("CMV") control in allogeneic stem cell transplant recipients has concluded, and its primary endpoint was met. We anticipate the full dataset will be presented at the 45th Annual Meeting of the European Society for Blood and Marrow Transplantation ("EBMT") being held in Frankfurt, Germany March 24-27, 2019.

MB-107 (XSCID Gene Therapy)

·In August 2018, we announced that we entered into an exclusive worldwide license agreement with St. Jude Children’s Research Hospital for the development of a potentially first-in-class ex vivo lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The therapy is currently being evaluated in a Phase 1/2 multicenter trial in infants under the age of two. This study is the world’s first lentiviral gene therapy trial for infants with XSCID. The therapy is also being investigated in patients over the age of two in a second Phase 1/2 trial at the National Institutes of Health ("NIH"). We believe these may be registration trials.
·MB-107 (XSCID Gene Therapy) is currently in development at our partner, Mustang Bio, Inc.

CK-301 (anti-PD-L1 antibody)

·In March 2018, we completed the dose escalation portion of the ongoing Phase 1 trial of CK-301, a fully human anti-PD-L1 antibody, in selected recurrent or metastatic cancers, and initiated the first dose expansion cohort, which is evaluating an 800 mg dose of CK-301 administered every two weeks.
·In January 2019, we announced that the ongoing multi-center clinical trial of anti-PD-L1 antibody CK-301 was expanded to enroll patients in three endometrial and colorectal cohorts intended to support requests for accelerated approval and Biologics License Application ("BLA") submissions to the FDA. The ongoing trial is also enrolling cohorts of patients with NSCLC and cutaneous squamous cell carcinoma.
·CK-301 (anti-PD-L1 antibody) is currently in development at our partner, Checkpoint Therapeutics, Inc.

CK-101 (third-generation EGFR inhibitor)

·In September 2018, we announced interim safety and efficacy data from our Phase 1/2 clinical trial of CK-101, a third-generation EGFR tyrosine kinase inhibitor ("TKI") being evaluated in advanced non-small cell lung cancer ("NSCLC"). The data were disclosed in an oral presentation at the International Association for the Study of Lung Cancer ("IASLC") 19th World Conference on Lung Cancer in Toronto. CK-101 was well tolerated across multiple dose groups. Durable anti-tumor activity was observed, particularly in treatment-naïve EGFR mutation-positive NSCLC patients.
·In March 2019, we announced two new patent issuances by the U.S. Patent and Trademark Office and the European Patent Office for CK-101. The patents cover CK-101 in the U.S. and Europe through at least August 2034, not including any potential patent term extensions.

·CK-101 (third-generation EGFR inhibitor) is currently in development at our partner, Checkpoint Therapeutics, Inc.

CUTX-101 and AAV-based gene therapy

·In July 2018, we announced that the FDA granted Fast Track Designation to CUTX-101 ("Copper Histidinate"), a product candidate for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression. CUTX-101 is currently in a Phase 3 clinical trial.
·In September 2018, we announced the publication of preclinical data on adeno-associated virus (AAV)-based gene therapy combined with subcutaneous CUTX-101 for Menkes disease in Molecular Therapy: Methods & Clinical Development.

MB-102 (CD123 CAR T)

·In July 2018, we completed a pre-Investigational New Drug ("pre-IND") meeting with the U.S. Food and Drug Administration ("FDA") for MB-102 ("CD123 CAR T"). Based on the meeting, we expect to initiate a multicenter Phase 1/2 trial of MB-102 in acute myeloid leukemia ("AML"), blastic plasmacytoid dendritic cell neoplasm ("BPDCN") and high-risk myelodysplastic syndrome in the second half of 2019.
·In November 2018, we announced that additional safety and efficacy Phase 1 data evaluating MB-102 (CD123 CAR) in relapsed or refractory AML and BPDCN were presented in an oral session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Special Conference on Tumor Immunology and Immunotherapy.
·In December 2018, the FDA granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of BPDCN, a rare and incurable blood cancer. Historically the median survival for BPDCN has been less than 18 months.
·MB-102 (CD123 CAR T) is currently in development at our partner, Mustang Bio, Inc.

MB-101 (IL13Rα2-specific CAR T)

·In May 2018, we announced the publication of preclinical data in JCI Insight demonstrating that glioblastoma-targeted CD4+ CAR T cells mediate superior antitumor activity over CD8+ CAR T cells. The data, published by research partner City of Hope, will be applied in the ongoing Phase 1 trial of IL13Rα2-specific CAR T MB-101 in glioblastoma.
·MB-101 (IL13Rα2-specific CAR T) is currently in development at our partner, Mustang Bio, Inc.

Oncolytic Virus (C134)

·In February 2019, we announced that we partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop an oncolytic virus (C134) for the treatment of glioblastoma multiforme. We intend to combine the oncolytic virus with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme.

·C134 (oncolytic virus) is currently in development at our partner, Mustang, Bio, Inc.

MB-103 (HER2-specific CAR T)

·In October 2018, we announced that a first-of-its-kind Phase 1 clinical trial evaluating the safety and effectiveness of intraventricular delivery of CAR T cells to the brains of patients with HER2-positive breast cancer with brain metastases was initiated at City of Hope; the first patient was dosed in December 2018. In addition, we announced that City of Hope dosed the first patient in a Phase 1 clinical trial of HER2-specific CAR T cells in treating recurrent or refractory grade III-IV glioma. The trial is evaluating the side effects and best dose of HER2-specific CAR T cells in treating patients with grade III-IV glioma that has come back or does not respond to treatment.
·MB-103 (HER2-specific CAR T) is currently in development at our partner, Mustang, Bio, Inc.

CK-103 (BET Inhibitor)

·In April 2018, preclinical data was presented on our BET inhibitor, CK-103, at the AACR (Free AACR Whitepaper) Annual Meeting. CK-103 demonstrated combinatorial effects in an in vivo model with anti-PD-1 antibodies, which may support the development of CK-103 as an anti-cancer agent alone and in combination with CK-301.
·CK-103 (BET Inhibitor) is currently in development at our partner, Checkpoint Therapeutics, Inc.

CNDO-109

·In June 2018, data from a Phase 1 trial evaluating CNDO-109-activated allogeneic natural killer ("NK") cells in AML patients were published in the journal Biology of Blood and Marrow Transplantation. The data demonstrated that CNDO-109-activated NK cells are safe, well tolerated and may be capable of extending complete remissions in high-risk AML patients.

AVTS-001 (AAV Gene Therapy)

In January 2018, we entered into a sponsored research agreement with the laboratory of Guangping Gao, Ph.D., at the University of Massachusetts Medical School to evaluate construct optimization for our AAV gene therapy treatment for complement-mediated diseases, including dry age-related macular degeneration, paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
·In August 2018, we announced that we entered into a sponsored research agreement with the laboratory of Wenchao Song, Ph.D., at the University of Pennsylvania to evaluate our AAV gene therapy technology in the university’s proprietary animal models of complement-mediated diseases.

On March 18, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, was selected to participate as a panelist on "The Brave New World of Myeloid Tumors (Press release, Stemline Therapeutics, MAR 18, 2019, View Source [SID1234534420])." The panel will take place on Monday, March 18 at 3 PM PT during the 31st Annual ROTH conference at the Ritz-Carlton Laguna Niguel, CA.

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About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to, and validated by, the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, and other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

On March 18, 2019 Veracyte, Inc. (Nasdaq: VCYT) reported that new data derived from the Afirma Xpression Atlas, which help characterize the genomic underpinning of medullary thyroid cancer (MTC), will be presented at ENDO 2019, the Endocrine Society’s annual meeting (Press release, Veracyte, MAR 18, 2019, View Source [SID1234534419]). The findings on this rare–but aggressive–form of thyroid cancer will be presented in a guided poster presentation during the conference, which is taking place March 23-26 in New Orleans, La.

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"The Afirma Xpression Atlas is enabling us to shed new light on the scope and scale of gene alterations found in medullary thyroid cancer," said Giulia C. Kennedy, Ph.D., chief medical and scientific officer of Veracyte. "We believe that, in the emerging era of precision medicine, such information will be vital to advancing the diagnosis and treatment of this aggressive cancer and we look forward to sharing our findings at ENDO 2019."

The following guided poster will be presented during the meeting:

Title: The Genomic Landscape of Preoperative FNAs Positive for the Afirma GSC Medullary Thyroid Cancer Classifier

Presenter: S.G. Waguespack, M.D., University of Texas MD Anderson Cancer Center, Houston, Texas

Date: Monday, March 25, 1:00 – 3:00 PM CT

Location: ENDOExpo, Ernest N. Morial Convention Center

About Afirma

The Afirma Genomic Sequencing Classifier (GSC) and Xpression Atlas provide physicians with a comprehensive solution for a complex landscape in thyroid nodule diagnosis. The Afirma GSC was developed with RNA whole-transcriptome sequencing and machine learning and helps identify patients with benign thyroid nodules among those with indeterminate cytopathology results in order to help patients avoid unnecessary diagnostic thyroid surgery. The Afirma Xpression Atlas provides physicians with genomic alteration content from the same fine needle aspiration samples that are used in Afirma GSC testing and may help physicians decide with greater confidence on the surgical or therapeutic pathway for their patients. The Afirma Xpression Atlas includes 761 DNA variants and 130 RNA fusion partners in over 500 genes that are associated with thyroid cancer.

On March 18, 2019, Vaccinex, Inc. presented the corporate presentation (Presentation, Vaccinex, MAR 18, 2019, View Source [SID1234534418]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!