On May 11, 2020 Unum Therapeutics Inc. (NASDAQ: UMRX), a biopharmaceutical company focused on developing curative cell therapies for solid tumors, reported financial results for the first quarter ended March 31, 2020, and provided corporate updates (Press release, Unum Therapeutics, MAY 11, 2020, View Source [SID1234557514]).

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Recent Program and Corporate Highlights

Announced plans to prioritize resources towards advancing its preclinical program, BOXR1030, for the treatment of solid tumor cancers: On March 2nd, Unum announced a corporate restructuring plan to prioritize resources towards advancing its preclinical program, BOXR1030, for the treatment of solid tumor cancers. Unum’s BOXR1030 expresses a glypican-3 (GPC3) targeted CAR and incorporates the novel transgene glutamic-oxaloacetic transaminase 2 (GOT2) to improve T cell function in the solid tumor microenvironment by enhancing T cell metabolism. Unum has initiated formal preclinical development activities, including preclinical safety testing and cGMP manufacturing readiness activities, to support filing an investigational new drug (IND) application for BOXR1030 in late 2020.

Entered into a common stock purchase agreement for up to $25 million with Lincoln Park Capital Fund, LLC ("LPC"): Under the terms of the purchase agreement announced on March 20th, Unum Therapeutics will have the sole discretion to direct LPC to purchase up to $25 million in shares of its common stock over the 36-month term of the agreement based on the market prices prevailing at the time of each sale to LPC. Unum Therapeutics controls the timing and amount of any future sales of its stock, subject to various limitations including those under the NASDAQ listing rules, and there is no upper limit as to the price per share that LPC may pay for future stock issuances under the purchase agreement. LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Unum Therapeutics’ common stock. Unum Therapeutics maintains the right to terminate the common stock purchase agreement at any time, at its discretion, without any additional cost or penalty.

Exploring strategic options to maximize shareholder value. Following a review of its business, the Company recently initiated and continues a process to explore strategic alternatives focused on maximizing shareholder value, with Ladenburg Thalmann & Co. Inc. acting as Unum Therapeutics’ strategic financial advisor during this process. Potential strategic alternatives that may be evaluated include, but are not limited to, an acquisition, merger, business combination, in-licensing, or other strategic transaction. There can be no assurance that this process will result in any such transaction. Unum Therapeutics has not set a timetable for completion of this review process and does not intend to

comment further unless or until the Board of Directors has approved a definitive course of action, the review process is concluded, or it is determined that other disclosure is appropriate.

First Quarter 2020 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the first quarter ended March 31, 2020 of $7.0 million compared to $3.1 million in the same period of 2019. Collaboration revenue includes the recognition of a portion of the upfront payment received from Seattle Genetics, Inc. as reimbursements for research and development costs, and increased during the quarter ended March 31, 2020 as a result of the conclusion of the ATTCK-17-01 Phase 1 clinical trial and the termination of the collaboration agreement.

R&D Expenses: Research and development expenses of $9.5 million for the first quarter ended March 31, 2020 compared to $12.4 million for the same period of 2019. Research and development expenses relate to ongoing costs for Phase 1 trials and the BOXR1030 preclinical program, as well as personnel-related costs to support these programs.

G&A Expenses: General and administrative expenses for the first quarter ended March 31, 2020 were $3.7 million, compared to $2.5 million for the same period of 2019. The increase is primarily related to increased personnel-related costs, including severance costs, as well as expenses required to operate as a public company.

Net Loss: Net loss attributable to common stockholders was $6.1 million, or $0.20 per share, for the first quarter ended March 31, 2020 compared with a net loss attributable to common stockholders of $11.7 million, or $0.39 per share, for the same period of 2019.

Cash and Cash Equivalents: As of March 31, 2020, Unum had cash and cash equivalents of $29.6 million. Unum believes that its existing cash and cash equivalents will fund operating expenses and capital expenditure requirements into mid-2021.

About Unum’s BOXR1030 and BOXR Platform

Unum’s BOXR1030 was discovered from its Bolt-on Chimeric (BOXR) platform that is designed to discover novel "bolt-on" transgenes to be co-expressed with CARs, a T-cell receptor, or ACTR, to help T cells survive longer and perform better in the solid tumor microenvironment. BOXR candidates consist of two main components: 1) a targeting receptor that directs the T cell to attack tumor cells, which may be a traditional CAR receptor, a T-cell receptor, or Unum’s ACTR receptor, and 2) a novel "bolt-on" transgene that improves the intrinsic function of the T cell. Once discovered, BOXR transgenes are designed to be incorporated into

several different types of therapeutic T cells, including both ACTR T cells and CAR-T cells, to impart new functionality to T cells.

Unum’s first product candidate selected from the BOXR platform, BOXR1030, expresses GPC3+ targeted CAR and incorporates the bolt-on GOT2 transgene to improve T cell function in the solid tumor microenvironment (TME) by enhancing T cell metabolism. Preclinical data with BOXR1030 was presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2019. In preclinical studies, BOXR1030 T cells were resistant to suppressive TME-like conditions, showing improved T cell proliferation under both hypoxic and low glucose conditions compared with control GPC3+ CAR-T cells. In vivo, BOXR1030 demonstrated superior activity compared to the parental CAR-T with treated animals achieving complete tumor regressions. Tumor infiltrating lymphocytes isolated from the tumors of treated animals revealed that BOXR1030 cells were more resistant to dysfunction and had fewer markers of exhaustion as compared to the control CAR-T cells.

On May 11, 2020 Bionical Emas and Jazz Pharmaceuticals (NASDAQ: JAZZ) reported that they have entered into an agreement to provide appropriate patients in the United States (U.S.) who have relapsed Small Cell Lung Cancer (SCLC) lurbinectedin via an Expanded Access Program (EAP) (Press release, Bionical Emas, MAY 11, 2020, View Source [SID1234557531]). Lurbinectedin is an investigational drug under review by the U.S. Food and Drug Administration (FDA).

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The EAP is open to patients who are unable to enter clinical trials and for whom there are no appropriate alternative treatments while lurbinectedin is under regulatory review by the FDA. The EAP was originally launched in January 2020 between Bionical Emas and PharmaMar S.A. (PharmaMar), and now has successfully transitioned from PharmaMar to Jazz.

SCLC is an aggressive form of cancer that usually is diagnosed with advanced, often metastatic, disease, and often posing a worse prognosis when compared to other lung cancers.1 In the United States, approximately 10-15% of lung cancers are small cell.1 Approximately 30,000 new cases of SCLC are recorded in the U.S. every year.2

Lurbinectedin is a selective inhibitor of oncogenic transcription on which many cancers such as SCLC are particularly dependent.

"We are pleased to be working with Jazz on this important project allowing access to lurbinectedin to appropriate patients via an Expanded Access Program in the U.S.," said Tom Watson, Executive Vice President, Bionical Emas.

"Lurbinectedin provides further hope for patients suffering from relapsed SCLC who currently have limited treatment options. This EAP provides an important opportunity for those patients who are unable to enter clinical trials and for whom there are no appropriate alternative treatments, and we are pleased to support this program," said John Efthimiou, Chief Medical Officer, Bionical Emas.

A new drug application (NDA) for Lurbinectedin is under review by the FDA and has not yet been approved.

Healthcare professionals wishing to request access to lurbinectedin under the EAP or who would like to find out more should do so by emailing [email protected]. Further details concerning the EAP can be found on Clinicaltrials.gov.

On May 11, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2020 and provided an operational update (Press release, bluebird bio, MAY 11, 2020, View Source [SID1234557450]).

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"We remain grounded in our core values and priorities: our patients, our people, our community and our business," said Nick Leschly, chief bluebird. "This quarter, we successfully submitted the ide-cel BLA with our partners at BMS and I am pleased to announce today that we have alignment with FDA on our clinical data package and filing path for LentiGlobin for sickle cell disease, which accelerates our planned base case filing timeline into 2021. Additionally, we are amending our co-promotion/co-development agreement with BMS to enable both companies to focus their efforts on efficient commercialization of ide-cel and generate non-dilutive capital for our business. Lastly, after a rigorous review of all operational plans to reflect COVID-19 uncertainties and recent program shifts, we have prioritized our core four programs to drive ide-cel launch and three filings in 2021 for CALD, TDT and SCD and continue to drive forward high potential pipeline assets. This prioritization effort and operational review has led to significant efficiencies and spend reduction across our company and will extend our cash runway into 2022. The fundamentals of our business remain sound and our newly revised operating plan enables us to execute on the 2022 vision while putting us on a path towards financial sustainability. Our team remains excited and committed to our mission with the belief we will emerge from a very tough period in history stronger than ever to deliver for patients. Thank you birds!"

SCD REGULATORY PATH

Today, bluebird bio announced general agreement with FDA that the clinical data package required to support a BLA submission for LentiGlobin for sickle cell disease (SCD) will be based on data from a portion of patients in the HGB-206 study Group C that have already been treated. The planned submission will be based on an analysis using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint and at least 18 months of follow-up post drug product infusion. Globin response will be used as a key secondary endpoint. The company anticipates additional guidance from FDA regarding the commercial manufacturing process, including suspension lentiviral vector. The company is planning to seek an accelerated approval and expects to submit the U.S. Biologics Licensing Application (BLA) for sickle cell disease in the second half of 2021. bluebird bio plans to present updated data from the HGB-206 study at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting.

Additionally, to enhance its strategic, clinical and commercial manufacturing platform, bluebird bio has entered into expanded relationships with two subsidiaries of Hitachi Chemical Co., Ltd. (Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma GmbH). These agreements will support late-stage and commercial drug product manufacturing of LentiGlobin for SCD in both the United States and Europe, expand commercial drug product manufacturing capacity for ZYNTEGLO (betibeglogene autotemcel) in Europe, and expand clinical and commercial manufacturing capacity for Lenti-D for cerebral adrenoleukodystrophy (CALD) in Europe.

FINANCIAL AND BUSINESS UPDATES

IDE-CEL ROYALTY MONETIZATION AND BCMA RELATIONSHIP AMENDMENT

Today, bluebird bio announced in a separate press release that it has amended its existing co-promotion/co-development agreement with Bristol Myers Squibb (BMS) to enable the companies to focus their efforts on efficient commercialization of idecabtagene vicleucel (ide-cel; bb2121) in the U.S., the companies’ lead investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, currently in review with the FDA. The companies will continue to share equally profits and losses in the U.S. Under the terms of the amended agreement, BMS will buy out its obligations to pay bluebird bio future ex-U.S. milestone and royalty payments for ide-cel and bb21217, the companies’ second BCMA-directed CAR T immunotherapy, for a one-time upfront payment of $200 million. bluebird bio is currently in the process of building out and qualifying its wholly-owned manufacturing facility in Durham, North Carolina for the production of lentiviral vector (LVV) to support the U.S. commercial market for ide-cel and for bluebird bio’s pipeline. Over time, BMS will assume responsibility for manufacturing of LVV outside the U.S. In partnership with BMS, bluebird bio is planning to present updated ide-cel clinical data from the Phase 2 KarMMa study at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting.

REVISED BUSINESS PRIORITIES AND OPERATING PLAN

Given the ongoing impact of the COVID-19 global pandemic and recent shifts in regulatory timelines, bluebird bio has undertaken a comprehensive business review with the goal of ensuring the ability to achieve its 2022 vision with a path towards financial sustainability. Under the revised business priorities and operating plan, bluebird remains on track for potential regulatory approval and commercial launch for ZYNTEGLO, ide-cel, Lenti-D for CALD, and LentiGlobin for SCD by 2022.

Through this comprehensive business review, bluebird bio has prioritized key research and development programs and has made a number of changes to the future cost structure relative to the prior long-range plan, including:

Reduced investment in selling, general and administrative expenses, including a deferred investment in building a U.S. commercial organization, reduced facilities and IT infrastructure, and other cost-reduction measures.
Prioritized investment in R&D expenses, including an indefinite pause of the HGB-211 clinical study in SCD patients at high risk of stroke, adjustment to the timing of investment in ongoing clinical studies to reflect COVID-19 related delays in enrollment, reduction or elimination of investment in certain preclinical programs, and other cost-reduction measures.
Nick Leschly, chief bluebird, will decline nearly 100% of his salary for the next 12 months. Similarly, additional members of the bluebird bio senior leadership team and all members of the Company’s Board of Directors will forgo 20% of their salaries or Board cash retainers for the next 12 months. All will receive a grant of restricted stock units equal to 80% of the value of the released cash compensation, which will vest over one year.
In total, these changes are expected to result in over $500 million of net cash savings through 2022 compared to the prior long-range plan. As a result, bluebird bio expects cash, cash equivalents, and marketable securities of $1.02 billion as of March 31, 2020, together with projected revenue generated under collaborative arrangements, projected sales of products and cash inflows associated with the amended agreements with BMS, to fund the revised operating plan into 2022. bluebird bio expects to continue to drive additional savings through rigorous prioritization and focus on expenses, real estate optimization, and exploration of additional sources of funding to further strengthen its financial position.

PREVIOUSLY DISCLOSED RECENT HIGHLIGHTS

IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On March 31, 2020, bluebird bio and BMS announced the submission of their BLA to the U.S. FDA for ide-cel, the companies’ lead investigational BCMA-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The BLA submission includes results from pivotal KarMMa study evaluating ide-cel in a heavily pre-treated patient population with relapsed and refractory multiple myeloma.
COVID-19 IMPACT – On March 26, 2020, bluebird bio provided an assessment of the impact of the COVID-19 pandemic and outlined steps the company has taken to ensure the safety of its patients and employees, while working to ensure the sustainability of its business operations as this unprecedented situation continues to evolve. Generally, the company expects the COVID-19 pandemic to shift the timing of enrollment and completion of clinical studies by at least three months and expects timing shifts to vary by clinical trial and by program.
UPCOMING ANTICIPATED MILESTONES

Regulatory
Submission of a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical
Presentation of ide-cel clinical data from the KarMMa study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting later this month, in partnership with Bristol-Myers Squibb.
Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes at the 2020 Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).
Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Updated data presentation from HGB-206 clinical study in patients with SCD at the 2020 Annual Congress of EHA (Free EHA Whitepaper).
Presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.
Updated data presentation from ALD-102 in patients with CALD by the end of 2020.
Commercial and Foundation Building
ZYNTEGLO first commercial patients treated in Europe in the second half of 2020.
ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
FIRST QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2020 and December 31, 2019 were $1.02 billion and $1.24 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $21.9 million for the three months ended March 31, 2020 compared to $12.5 million for the three months ended March 31, 2019. The increase was primarily attributable to an increase in ide-cel license and manufacturing service revenue under our agreement with BMS, as well as an increase in royalty revenue.
R&D Expenses: Research and development expenses were $154.1 million for the three months ended March 31, 2020 compared to $122.6 million for the three months ended March 31, 2019. The increase was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $73.2 million for the three months ended March 31, 2020 compared to $60.3 million for the three months ended March 31, 2019. The increase was largely attributable to costs incurred to support the company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $202.6 million for the three months ended March 31, 2020 compared to $164.4 million for the three months ended March 31, 2019.
CONFERENCE CALL DETAILS

bluebird bio will hold a conference call to discuss business updates and first quarter 2020 financial results on Monday, May 11 at 8:00AM ET.

Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 335-5158.

On May 11, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported business highlights and financial results for the first quarter ended March 31, 2020 (Press release, Fate Therapeutics, MAY 11, 2020, View Source [SID1234557488]).

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"We are encouraged by the resilience of our employees, our clinical trial investigators and participating patients, and our collaboration partners in the face of the challenge posed by the global pandemic. Like others, we have been affected by COVID-19, which has impacted clinical site initiation, slowed the cadence of new patient enrollment, and changed how we conduct our day-to-day business," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Nevertheless, we have continued to enroll patients across our three Phase 1 clinical programs, expanded the clinical footprint of our FT596 program into relapse prevention following autologous HSCT, and submitted our IND application to the FDA for FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy, in multiple myeloma. Additionally, we entered into a transformative collaboration with Janssen that leverages our iPSC product platform and Janssen’s proprietary tumor-targeting antigen binders to develop novel CAR NK and CAR T-Cell product candidates for hematologic malignancies and solid tumors, supporting our fundamental goal of bringing off-the-shelf, iPSC-derived cell-based cancer immunotherapies to patients."

Clinical Programs

First Patient Treated with FT596 Monotherapy for Advanced Diffuse Large B-cell Lymphoma. FT596 is the industry’s first investigational cellular immunotherapy engineered with three active anti-tumor modalities to be evaluated in patients. The Company is currently conducting an open-label Phase 1 clinical trial of FT596 in patients with relapsed / refractory B-cell malignancies and chronic lymphocytic leukemia. FT596 is an off-the-shelf, multi-antigen targeted, chimeric antigen receptor (CAR) natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a proprietary CD19-targeting CAR, a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, and a unique interleukin-15 receptor fusion (IL-15RF). The hnCD16 Fc receptor enables concurrent targeting of additional tumor-associated antigens bound by therapeutic antibodies to overcome antigen escape and promote multifaceted tumor cell killing, and IL-15RF is a potent cytokine complex that promotes survival, proliferation and trans-activation of NK cells and CD8 T cells without the need for exogenous cytokine support.
Second FT596 IND Application Allowed by FDA for Relapse Prevention after Autologous HSCT. The open-label Phase 1 study, which is sponsored by investigators from the Masonic Cancer Center, University of Minnesota, is intended to assess the potential of FT596 to prevent relapse in patients undergoing autologous hematopoietic stem cell transplant (HSCT) for the treatment of non-Hodgkin lymphoma. The clinical trial is expected to enroll up to 18 patients considered high risk for early relapse based on failure to achieve complete or partial remission on, or relapse within twelve months of, prior therapy. Up to three dose levels of FT596 will be administered in combination with rituximab approximately 30 days following HSCT.
FT500 Phase 1 Dose-Escalation Stage Successfully Completed for Advanced Solid Tumors. Three patients have been treated in the dose-escalation stage of the FT500 Phase 1 study at 300M cells per dose in combination with checkpoint inhibitor therapy. There were no dose-limiting toxicities, no FT500-related Grade ≥3 adverse events (AEs) or serious adverse events (SAEs), and no incidents of cytokine release syndrome, neurotoxicity, or graft-versus-host disease reported by investigators. Enrollment into the dose-expansion stage of the FT500 Phase 1 study is proceeding in patients with non-small cell lung cancer (NSCLC) who are refractory to, or have relapsed following, checkpoint inhibitor therapy. The Company intends to treat up to 15 patients in the outpatient setting, administering three once-weekly doses of FT500 at 300M cells per dose over up to two 30-day cycles with IL-2 cytokine support and the same checkpoint inhibitor on which the patient failed or progressed.
FT516 Clinical Investigation Expanded to Solid Tumors. In January, the Company announced that the U.S. Food & Drug Administration (FDA) allowed its second IND application for FT516, the Company’s off-the-shelf NK cell cancer immunotherapy derived from a clonal master iPSC line engineered to express hnCD16 Fc receptor, enabling clinical investigation in combination with PDL1-, PD1-, EGFR- and HER2-targeting monoclonal antibody (mAb) therapies across a broad range of solid tumors. The Company intends to initially evaluate FT516 in combination with avelumab in patients with advanced solid tumors who are refractory to, or have relapsed following, at least one line of anti-PDL1 mAb therapy. The multi-dose treatment course consists of three once-weekly doses of FT516 over up to two 30-day cycles. The Company is currently conducting an open-label, multi-dose Phase 1 clinical trial of FT516 as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma.
IND Application Submitted for FT538, the First CRISPR-edited, iPSC-derived Cellular Immunotherapy. FT538 is derived from a clonal master iPSC line engineered with hnCD16 and IL-15RF and edited for elimination of CD38 expression (CD38KO) to mitigate anti-CD38 antibody-mediated fratricide. The Company has recently submitted an IND application to the FDA for the clinical investigation of FT538 in combination with anti-CD38 monoclonal antibody therapy for the treatment of multiple myeloma.
FT516 Investigator-initiated Clinical Trial for COVID-19 Opened to Enrollment. Sponsored by investigators from the Department of Medicine, Division of Infectious Diseases and International Medicine, University of Minnesota, the open-label Phase 1 study is designed to assess the clinical safety and tolerability of FT516 for the treatment of Coronavirus Disease 2019 (COVID-19) (NCT04363346). The study is evaluating three FT516 dose-escalating strategies (90M cells on Day 1; 90M cells on Day 1 and 300M cells on Day 4; and 90M cells on Day 1, 300M cells on Day 4, and 900M cells on Day 7) in up to 20 patients at a high risk of developing critical life-threatening illness. Secondary objectives of the study include time to elimination of viral shedding, to discontinuation of supplemental oxygen support, and to hospital discharge.
Corporate Highlights

Strategic Collaboration Formed with Janssen for Novel iPSC-derived Cell-based Cancer Immunotherapies. In April, the Company entered into a global collaboration and option agreement with Janssen Biotech, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop iPSC-derived chimeric antigen receptor (CAR) NK and CAR T-cell product candidates targeting up to four tumor-associated antigens for which Janssen is contributing proprietary antigen binding domains. The Company is eligible to receive payments of up to $3.0 billion upon the achievement of certain development, regulatory and commercial milestones, plus tiered double-digit royalties on worldwide net sales of products targeting the antigens. In addition, the Company has the right to elect to co-commercialize each product candidate in the U.S. and share equally in profits and losses in the U.S., subject to its payment of certain clinical development costs and adjustments in milestone and royalty payments. The Company received $100 million upon entering into the collaboration, including $50 million in an upfront cash payment and $50 million from the purchase by Johnson & Johnson Innovation – JJDC, Inc. of newly issued shares of the Company’s common stock at a price per share of $31.00.
Lease Executed for New Corporate Headquarters with cGMP Cell Manufacturing Facility. In January, the Company entered into a lease agreement for the entirety of the Scripps Northridge Corporate Center, a 200,000-square-foot life sciences complex in San Diego, CA that is designed to include a 40,000 square foot cGMP cell manufacturing facility. The Company intends to move its corporate headquarters to the campus in the middle of 2021.
Upcoming Scientific Presentations

IND-enabling Data for FT819 iPSC-derived CAR T-cell to be Presented at AACR (Free AACR Whitepaper) II. IND-enabling data for FT819, which is derived from a clonal master iPSC line engineered with a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus and edited for elimination of T-cell receptor (TCR) expression, is scheduled to be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II (June 22-24). In the second quarter of 2020, the Company intends to submit an IND application to the FDA for clinical investigation of FT819, the Company’s first off-the-shelf, iPSC-derived CAR T-cell product candidate.
FT576 Master iPSC Line Engineered with Four Anti-Tumor Modalities to be Highlighted at ASGCT (Free ASGCT Whitepaper). FT576 is the Company’s off-the-shelf, iPSC-derived, multi-antigen targeted CAR-BCMA NK cell product candidate for multiple myeloma that is currently undergoing preclinical development. At the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Virtual Annual Meeting (May 12-15), the Company will highlight an innovative paradigm to generate new clonal master iPSC lines for next-generation product development. Starting with an existing clonal master iPSC line engineered with three anti-tumor modalities (hnCD16; IL15-RF; and CD38KO) as a cell backbone, a CAR-BCMA transgene was engineered into the backbone, followed by single-cell selection and generation of a new clonal master iPSC line incorporating CAR-BCMA as a fourth functional element.
New iPSC-derived CAR MICA/B Solid Tumor Program to be Presented at ASGCT (Free ASGCT Whitepaper). The Company plans to present initial preclinical data for its new off-the-shelf, iPSC-derived CAR MICA/B cancer immunotherapy program at ASGCT (Free ASGCT Whitepaper). MICA and MICB are pan-tumor associated stress proteins induced by malignant transformation, and proteolytic shedding of MICA/B by cancer cells is a common mechanism of immune cell evasion broadly observed across solid tumors. The Company’s proprietary CAR MICA/B program targets a specific protein region which prevents shedding to overcome tumor escape.
First Quarter 2020 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of March 31, 2020 were $219.4 million. The Company’s cash, cash equivalents and investments exclude the receipt of $100.0 million in April 2020 in connection with entering into the Janssen collaboration.
Total Revenue: Revenue was $2.5 million for the first quarter of 2020, which was derived from the Company’s collaboration with Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $29.3 million for the first quarter of 2020, which includes $4.3 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $7.7 million for the first quarter of 2020, which includes $2.7 million of non-cash stock-based compensation expense.
Shares Outstanding: Common shares outstanding were 76.0 million, and preferred shares outstanding were 2.8 million, as of March 31, 2020. Each preferred share is convertible into five common shares. Common shares outstanding excludes the issuance of 1.6 million shares in April 2020 in connection with entering into the Janssen collaboration.
Today’s Conference Call and Webcast

The Company will conduct a conference call today, Monday, May 11, 2020 at 5:00 p.m. ET to review financial and operating results for the quarter ended March 31, 2020. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 8623509. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

On May 11, 2020 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company, reported financial results and recent corporate highlights for the first quarter ended March 31, 2020 (Press release, Fortress Biotech, MAY 11, 2020, View Source [SID1234557515]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "The first quarter of 2020 was another record revenue quarter for Fortress Biotech. Revenue from our dermatology products marketed by our partner company, Journey Medical Corporation, increased 95% compared to the first quarter of 2019, demonstrating the strength of our commercial operations. We intend to acquire one to two new prescription products this year."

Dr. Rosenwald continued, "We also made significant progress across our robust pipeline of development-stage product candidates in the first quarter. Notably, IV tramadol was assigned a PDUFA date of October 10, 2020. If IV tramadol is approved by the FDA and Avenue Therapeutics is acquired, we would expect to realize approximately $48 million, plus future contingent value rights. After a strong start to the year, we look forward to delivering key value-creating catalysts throughout the rest of 2020."

Recent Corporate Highlights1:

Marketed Dermatology Products

·Our dermatology products are marketed by our partner company, Journey Medical Corporation.
·Our five marketed specialty dermatology products generated first quarter 2020 net revenues of $11.9 million, compared to first quarter 2019 net revenues of $6.1 million, representing growth of 95% year-over-year.
·We intend to acquire one to two new prescription products in 2020.

IV Tramadol

·In February 2020, the U.S. Food and Drug Administration ("FDA") accepted the submission of Avenue Therapeutics’ New Drug Application ("NDA") for IV tramadol for review and assigned a Prescription Drug User Fee Act ("PDUFA") date of October 10, 2020. Pending a positive outcome resulting in FDA approval and other certain conditions being satisfied, a merger between Avenue and InvaGen will be completed shortly thereafter, which would result in a potential net distribution to Fortress of approximately $48 million and future contingent value rights.

1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words "we", "us" and "our" may refer to Fortress individually or together with our affiliates and partners, as dictated by context.

·In April 2020, Avenue announced that two e-posters highlighting efficacy and safety results from its Phase 3 program are available for online viewing from the cancelled Annual Regional Anesthesiology and Acute Pain Medicine Meeting hosted by the American Society of Regional Anesthesia and Pain Medicine ("ASRA").
oThe e-poster (816) titled "Intravenous Tramadol is Effective in Management of Postoperative Pain Following Abdominoplasty: A 3-arm Randomized Controlled Trial" presents data from the Phase 3 abdominoplasty study and can be found here.
oThe e-poster (1001) titled "IV tramadol – A New Treatment Option for Management of Post-Operative Pain: A Safety Trial Including Various Types of Surgery" presents data from the Phase 3 safety study and can be found here.
·IV Tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

CUTX-101

·In January 2020, the FDA granted Rare Pediatric Disease Designation to CUTX-101 for the treatment of Menkes disease.
·We intend to begin the rolling submission of the NDA for CUTX-101 to the FDA in the fourth quarter of 2020.
·CUTX-101 is currently in development at our partner company, Cyprium Therapeutics, Inc.

CAEL-101

·In March 2020, Caelum Biosciences, Inc. ("Caelum") began dosing patients in its Phase 2 dose selection clinical trial of CAEL-101, a light chain fibril-reactive monoclonal antibody for the treatment of AL amyloidosis.
·Caelum expects to begin its pivotal Phase 3 program in the second half of 2020.

Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

·In January 2020, we announced confirmation of the registration path for cosibelimab in metastatic cutaneous squamous cell carcinoma ("CSCC"). FDA feedback supports the plan to submit a BLA based on data from the ongoing Phase 1 clinical trial. Over one-third of enrollment is complete in the cohort of patients with metastatic CSCC. There is potential for cosibelimab to be differentiated both clinically and as a lower-cost alternative to available anti-PD-1/L1 mAbs.
·In April 2020, we announced that the U.S. Patent and Trademark Office has issued a composition of matter patent for cosibelimab. U.S. Patent No. 10,590,199 specifically covers the antibody, cosibelimab, or a fragment thereof, providing protection through at least May 2038, exclusive of any additional patent-term extensions that might become available.
·We anticipate presenting additional cosibelimab data in the second half of 2020 and expect to complete enrollment of our registration-enabling Phase 1 clinical trial in CSCC in 2021.
·Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc.

MB-107 (Lentiviral Gene Therapy for XSCID)

·In April 2020, we announced that the European Medicines Agency ("EMA") granted Advanced Therapy Medicinal Product ("ATMP") classification to MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease.
·In May 2020, Mustang Bio submitted an Investigational New Drug ("IND") application with the FDA to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multicenter trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplant ("HSCT"). The primary efficacy endpoint will be event-free survival. The initiation of this trial is currently on hold pending CMC clearance by the FDA. Mustang is targeting topline data from the trial in the second half of 2022.

·Mustang further expects to file an IND in the third quarter of 2020 for a registrational multi-center Phase 2 clinical trial of its lentiviral gene therapy in previously transplanted XSCID patients. This product will be designated MB-207. Mustang anticipates enrolling 20 patients and comparing them to matched historical control patients who have undergone a second HSCT. Mustang is targeting topline data for this trial in the second half of 2022.
·MB-107 is currently in development at our partner company, Mustang Bio, Inc.

MB-105 (PSCA-targeted CAR T cell therapy)

·In the ongoing Phase 1 trial at City of Hope with MB-105, a PSCA-directed CAR T administered systemically to patients with PSCA-positive castration resistant prostate cancer, the first patient to receive the therapy following a standard CAR T conditioning regimen experienced a significant reduction in his prostate-specific antigen (PSA) at day 28. This PSA response was associated with radiographic improvement of the patient’s metastatic disease.
·MB-105 is currently in development at our partner company, Mustang Bio, Inc.

MB-106 (CD20-targeted CAR T cell therapy)

·In February 2020, we announced that the first subject treated with the optimized MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang Bio and the Fred Hutchinson Cancer Research Center, achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas.
·MB-106 is currently in development at our partner company, Mustang Bio, Inc.

ONCOlogues (proprietary platform technology using PNA oligonucleotides)

·In May 2020, we entered into an exclusive worldwide licensing agreement with Columbia University to develop novel oligonucleotides for the treatment of genetically driven cancers. The proprietary platform produces oligomers, known as "ONCOlogues," which are capable of binding gene sequences 1,000 times more effectively than complementary native DNA.
oONCOlogues invade a DNA double helix and displace native mutated strands. This prevents the mRNA that antisense binds to from ever being created. It is higher upstream than an antisense approach as well as potentially more potent and broader in its utility.
·In addition, we are exploring the potential of the platform to treat novel coronaviruses, such as COVID-19.
·The "Suppression of KRAS-G12D and BRAF-V600E Oncogene Transcription with PNA Conjugates" data presentation from the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in 2019 can be found here.
·The ONCOlogues platform is currently in development at our partner company, Oncogenuity, Inc.

General Corporate

·In February 2020, we closed on a gross total of approximately $14.4 million in an underwritten public offering of our 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock.
·In March 2020, our Board of Directors authorized the repurchase of up to $5 million of Fortress’ 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock (Nasdaq: FBIOP).

Financial Results:

To assist our stockholders in understanding our company, we have prepared non-GAAP financial results for the three months ended March 31, 2020 and 2019. These results exclude the operations of our three public partner companies: Avenue Therapeutics, Inc., Checkpoint Therapeutics, Inc. and Mustang Bio, Inc., as well as any one-time, non-recurring, non-cash transactions, such as the gain of $18.4 million we recorded in the first quarter of 2019 resulting from the de-consolidation of Caelum. The goal in providing these non-GAAP financial metrics is to highlight the financial results of Fortress’ core operations, which are comprised of our commercial-stage business, our privately held development stage entities, as well as our business development and finance functions.

·As of March 31, 2020, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $152.5 million, compared to $153.4 million as of December 31, 2019, a decrease of $0.9 million during the quarter.
·Fortress’ net revenue totaled $12.9 million for the first quarter of 2020, which included $11.9 million in net revenue generated from our marketed dermatology products. This compares to net revenue totaling $6.5 million for the first quarter of 2019, which included $6.1 million in net revenue generated from our marketed dermatology products.
·On a GAAP basis, consolidated research and development expenses were $14.9 million for the first quarter of 2020, compared to $23.3 million for the first quarter of 2019. On a non-GAAP basis, research and development expenses were $2.3 million for the first quarter of 2020, compared to $1.6 million for first quarter of 2019.
·On a GAAP basis, consolidated research and development expenses from license acquisitions totaled $0.3 million for the first quarter of 2020, compared to $0.5 million for the first quarter of 2019.
·On a GAAP basis, consolidated general and administrative expenses were $15.5 million for the first quarter of 2020, compared to $13.5 million for the first quarter of 2019. On a non-GAAP basis, general and administrative expenses were $11.6 million, of which $5.6 million is attributed to Journey, for the first quarter of 2020, compared to $8.5 million, of which $3.9 million is attributed to Journey, for the first quarter of 2019.
·On a GAAP basis, consolidated net loss attributable to common stockholders was $12.4 million, or $0.19 per share, for the first quarter of 2020, compared to net income attributable to common stockholders of $1.4 million, or $0.03 per share for the first quarter of 2019, which includes the $18.4 million gain due to the de-consolidation of Caelum.
·Fortress’ non-GAAP loss attributable to common stockholders was $4.2 million, or $0.07 per share, for the first quarter of 2020, compared to Fortress’ non-GAAP loss attributable to common stockholders of $4.7 million, or $0.07 per share, for the first quarter of 2019.