On November 12, 2018 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology, reported financial results for third quarter ended September 30, 2018 and that it has completed enrollment of the UGN-101 OLYMPUS Phase 3 Trial in patients with low-grade upper tract urothelial cancer (LG UTUC) following a recent pre-New Drug Application (NDA) meeting held with the U.S. Food and Drug Administration (FDA) (Press release, UroGen Pharma, NOV 12, 2018, View Source [SID1234531211]).

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At the pre-NDA meeting, the company presented updated data from this open-label, single arm Phase 3 study. The meeting resulted in agreement on the required information for the NDA submission and suitability for NDA submission of primary endpoint (Complete Response) data for the approximately 65 patients enrolled to date.

UroGen plans to present the topline data in January 2019, with the full dataset expected at a medical meeting in the second quarter of 2019.

"We are pleased by the FDA’s support for UGN-101 and recognition of the potential clinical benefit that UGN-101 presents for LG UTUC as a potentially organ-sparing, non-invasive therapy for this disease," said Ron Bentsur, Chief Executive Officer of UroGen. "We are excited about the progress that we continue to make across our clinical pipeline and look forward to collaborating with the FDA to bring this potentially transformative therapy to patients with LG UTUC."

Breakthrough Therapy Designation (BTD) was granted by the FDA for UGN1-101 for the treatment of LG UTUC in October 2018. UroGen remains on track to initiate the UGN-101 Rolling NDA Submission to the FDA in Q4 2018 and complete the submission in Q2 2019, with potential approval expected in 2019.

Additional Highlights and Upcoming Milestones

UGN-102 Clinical Development:
UroGen is enrolling patients as part of its Phase 2b single-arm, open-label, multi-center trial designed to assess the efficacy and safety of UGN-102 (mitomycin gel) for intravesical instillation as a potential first-line chemoablation agent in the treatment of patients with LG Non-Muscle Invasive Bladder Cancer (NMIBC) at risk for recurrence.
Initial data from the trial is expected in 1H 2019.
Similar to LG UTUC, there are currently no drugs approved by the FDA as first-line treatment for NMIBC, and only three drugs have been approved by the FDA, all as adjuvant treatments, following TURBT (transurethral resection of bladder tumor).
UGN-102 represents a very substantial opportunity in UroGen’s pipeline with the potential to initially address up to approximately 85,000 patients for whom TURBT is no longer effective.1
Advancing the Potential of the RTGel Platform:
Allergan continues to enroll patients in its Phase 2 trial of BotuGel, UroGen’s RTGel in combination with BOTOX2, for the treatment of overactive bladder. This clinical trial, if successful, has the potential to demonstrate the broad applicability of the RTGel platform beyond uro-oncology. Phase 2 data is expected in 2019.
Corporate Developments:
UroGen strengthened its leadership team with the appointment of Jones "Woody" Bryan, Ph.D., as Senior Vice President, Business Development. Dr. Bryan is a seasoned industry veteran who brings over 25 years of industry experience. He is focused on the integration of corporate strategy and business development to assess potential partnerships, both inbound and outbound, and bolster UroGen’s product portfolio.
Third Quarter 2018 Financial Results

As of September 30, 2018, cash and cash equivalents totaled $109.5 million.
Research and development expenses for the nine months ended September 30, 2018 were $25.5 million, including non-cash share-based compensation expense of $9.1 million. Research and development expenses for the three months ended September 30, 2018 were $9.6 million, including non-cash share-based compensation expense of $3.8 million.
General and administrative expenses for the nine months ended September 30, 2018 were $27.0 million, including non-cash share-based compensation expense of $12.7 million. General and administrative expenses for the three months ended September 30, 2018 were $10.7 million, including non-cash share-based compensation expense of $5.7 million.
The Company reported a net loss of $51.9 million, or basic and diluted net loss per ordinary share of $3.30, for the nine months ended September 30, 2018. The Company reported a net loss of $20.5 million, or basic and diluted net loss per ordinary share of $1.28, for the three months ended September 30, 2018.
Conference Call & Webcast Information

Members of UroGen’s management team will host a live conference call and webcast today at 8:30 a.m. Eastern Time to review the Company’s financial results and provide a general business update. Due to observance of the Veteran’s Day holiday in the United States on November 12, 2018, UroGen’s corresponding 6-K will be filed with the Securities and Exchange Commission (SEC) before market on November 13, 2018.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (888) 771-4371 (U.S.) or (847) 585-4405 (International) to listen to the live conference call. The conference ID number for the live call will be 47697839. An archive of the webcast will be available for two weeks on the Company’s website.

On November 12, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of novel cellular immunotherapies, reported that the Company will host an investor event to discuss its efforts in solid tumors on Monday November 19, 2018, from 8:30 a.m. to 10:00 a.m. ET in New York City (Press release, Unum Therapeutics, NOV 12, 2018, View Sourcenews-releases/news-release-details/unum-therapeutics-host-investor-event-its-efforts-realize" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-host-investor-event-its-efforts-realize" rel="nofollow">View Source [SID1234531331]).

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The event will feature guest speaker Charles S. Fuchs, MD, MPH, Director of the Yale Cancer Center, who will discuss the challenges and opportunities in HER2 malignancies. In addition, the Company will discuss the unmet need, clinical validation of ACTR707 and design of the ATTCK-34-01 Phase I trial using ACTR707 in combination with trastuzumab to target HER2-positive advanced cancers. The Company also will provide more details on the new BOXR platform to support its growing efforts in solid tumors.

To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at View Source at least 10 minutes before the event begins.

On November 11, 2018 Harpoon Therapeutics, Inc. ("Harpoon"), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported the closing of a $70 million Series C equity financing (Press release, Harpoon Therapeutics, NOV 11, 2018, View Source [SID1234531110]). OrbiMed served as the lead new investor, along with new investors Cormorant, Ridgeback Capital Investments, Lilly Asia Ventures (LAV) and NS Investment. Harpoon’s existing investors MPM Capital, Oncology Impact Fund, Arix Bioscience, New Leaf Venture Partners and Taiho Ventures, LLC also participated in the Series C financing round. Harpoon intends to use the proceeds from the financing to support further advancement of its immunotherapy programs based on its TriTAC (Tri-specific T cell Activating Construct) and ProTriTAC (Protease-activated Tri-specific T Cell Activating Construct) platforms, which are designed to harness the natural power of the body’s immune system to fight cancer and other diseases.

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Harpoon’s TriTAC platform was designed to advance the therapeutic potential of T cell engagers, with a long half-life extended format. Harpoon is developing a pipeline of four wholly owned TriTAC product candidates. Harpoon’s lead product candidate, HPN424, is currently in a Phase 1 clinical trial for the treatment of metastatic castration-resistant prostate cancer, or mCRPC.

"This financing is a significant milestone for us as we continue to advance our pipeline, including HPN424, our lead product candidate, with which we initiated a Phase 1 trial this summer as a potential treatment for prostate cancer," said Jerry McMahon, Ph.D., President and Chief Executive Officer. "With these funds, we intend to pursue preclinical and clinical development of additional product candidates based on our TriTAC platform as well as our second technology platform, ProTriTAC, which we believe can expand access to a broader landscape of tumor targets and indications."

"Harpoon has assembled a world-class team to drive forward an ambitious pipeline across a range of indications and patient populations," said Luke Evnin, Ph.D., Co-Founder and Chairman of Harpoon and Founder and Managing Director at MPM Capital. "With this financing, we believe we have the capacity not only to advance those pipeline programs but also to continue to innovate in the immuno-oncology arena."

In addition, in 2019, Harpoon plans to initiate Phase 1 clinical trials for HPN536 (a mesothelin-targeting TriTAC) for the treatment of mesothelin-expressing tumors, and HPN217 (a BCMA-targeting TriTAC) for the treatment of multiple myeloma. The ProTriTAC platform effort is yielding T-cell engagers against additional targets based on tumor protease-dependent activation in the tumor microenvironment, and Harpoon expects to advance its first ProTriTAC product candidate to IND-enabling studies in 2019.

On November 11, 2018 Generon Corporation, a leading biopharmaceutical company in China, reported it received approval for the Company’s Investigational New Drug (IND) application from the State Food and Drug Administration (SFDA) of the People’s Republic of China to initiate a Phase I clinical trial for A-319 in patients with B cell malignancies (Press release, Generon (Shanghai), NOV 11, 2018, View Source [SID1234531111]). A-319 is Generon’s second bispecific antibody in clinical development.

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A-319 is the first CD19 engaging bispecific antibody approved by the SFDA for clinical trials in China. The CD3/CD19 bispecific antibody was developed using Generon’s Immuno-Therapy Antibody (ITab)TM technology platform. The IND approval enables Generon to commence Phase I clinical trials in China enrolling patients with B cell malignancies including acute lymphoblastic leukemia (ALL) and B cell lymphoma.

Dr. Mi Jian Qing, Professor at Ruijin Hospital, Shanghai Jiaotong University expressed his enthusiasm about the biology of A-319 and the potential benefits for ALL patients. He commented: "Obtaining the SFDA’s approval for the A-319 Phase I trial is a significant accomplishment for Generon’s ITabTM platform. The IND approval is another step in demonstrating Generon’s innovative capabilities".

Yifan Pharmaceuticals, Generon’s parent company, congratulated Generon’s team on the continued effort to develop innovative therapies. Dr. Xiao Qiang Yan, CEO and CSO of Generon, said, "Initiation of a Phase I study for A-319 in China is one of Generon’s goals this year. A-319 has a similar mechanism of action to eliminate malignant B cells to those of CAR-T and other CD19/CD3 bispecific antibodies, but it is more convenient for patient dosing and potentially with better safety. A-319 is our second T-cell activating bispecific antibody to enter clinical development. Generon is expanding its ITabTM pipeline for both liquid and solid tumors and committed to bringing innovative immune-oncology antibodies to cancer patients in China and the world".

B cell malignancies

B cell malignancies refer to the different types of cancers that form in B cells in the immune system, including B-cell lymphomas and B-cell leukemia. B-cell lymphoma may be either indolent (slow-growing) or aggressive (fast-growing). Most B-cell lymphomas are non-Hodgkin lymphomas (NHL). There are many different types of B-cell non-Hodgkin lymphomas including chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), Burkitt lymphoma (BL), diffuse large B-cell lymphoma (DLBL), follicular lymphoma (FL), and mantle cell lymphoma (MCL). B-cell leukemia includes B-cell chronic lymphocytic leukemia (CLL), Acute lymphoblastic leukemia (ALL), B-cell prolymphocytic leukemia (PLL), and hairy cell leukemia (HCL). The prognosis and treatment of B cell malignancies depend on the specific type of the B cell lymphoma/leukemia, as well as the stage and grade. Recent immunotherapy (T-cell activating) approaches have demonstrated significant clinical benefits for patients.

About A-319

A-319 is a T-cell activating bispecific antibody (BsAb) designed to target CD19 and CD3 (anti-CD19, anti-CD3) and is under development for the treatment of patients with B cell malignancies including B-cell leukemia and B-cell lymphoma. A-319 activates T lymphocytes in a patient to kill CD19 expressing malignant B-cells.

On November 11, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported new clinical data for FATE-NK100, an investigational, first-in-class, allogeneic donor-derived natural killer (NK) cell cancer immunotherapy, and provided a regulatory update on the development of FT500, a universal, off-the-shelf NK cell product candidate derived from a master induced pluripotent stem cell (iPSC) line, on November 10, 2018.

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Twenty heavily pre-treated subjects, each presenting with progressive disease at the time of enrollment, have been treated with FATE-NK100 in the dose-escalation phases of three Phase 1 clinical trials. As of a October 22, 2018 data cutoff, one-month follow-up data were available on fourteen subjects1, with clinical benefit indicated in seven of these fourteen subjects:

In the DIMENSION study for the treatment of advanced solid tumors (n=5 in the monotherapy regimen; n=1 in the monoclonal antibody combination regimen1), one subject at the second dose level (1-3×107 cells per kg) and two subjects at the third dose level (3-10×107 cells per kg) treated with a single intravenous infusion of FATE-NK100 in the monotherapy regimen had stable disease at one month. These two subjects at the third dose level were each subsequently treated with a second dose of FATE-NK100 and remain on study (3.1 and 5.0 months, respectively) with ongoing disease control. The study is currently enrolling at the third dose level in the monotherapy regimen, at the second dose level in the cetuximab combination regimen (1-3×107 cells per kg) and at the run-in dose level in the trastuzumab combination regimen (1×106 cells per kg).
In the APOLLO study for the treatment of recurrent ovarian cancer (n=4), one subject treated with a single intraperitoneal infusion of FATE-NK100 at the second dose level (1-3×107 cells per kg) had stable disease at one month. The subject was subsequently treated with a second dose of FATE-NK100 and maintained disease control for 6.2 months. The study is currently enrolling at the third dose level (3-10×107 cells per kg).
In the VOYAGE study for the treatment of refractory or relapsed acute myelogenous leukemia (n=4), all three subjects treated with a single intravenous infusion of FATE-NK100 at the second dose level (1-3×107 cells/kg) showed complete clearance of leukemic blasts in the bone marrow and achieved a morphologic leukemia-free state at Day 14 following treatment. Each of these three subjects received a single dose of FATE-NK100 only, and the anti-leukemic response in each of these subjects was transient. The study is currently enrolling at the second dose level.
No dose limiting toxicities related to FATE-NK100 were reported. One serious adverse event related to FATE-NK100 was reported (Grade 3: abdominal pain) in the APOLLO study.

"The safety and clinical benefit observed with a single infusion of FATE-NK100 as a monotherapy in heavily pre-treated cancer patients, including in refractory AML patients that have high leukemic blast burden in the marrow and in advanced solid tumor patients with progressive disease, are encouraging," said Sarah Cooley, M.D., Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation at the University of Minnesota and the lead investigator of the VOYAGE study. "We are particularly excited that a repeat dose of FATE-NK100 was well-tolerated and showed persistence. Importantly, all three subjects re-treated with a second dose have demonstrated disease control. These data provide compelling proof-of-concept for FATE-NK100 and support earlier intervention with NK cell therapy using a multi-dose treatment cycle."

In addition, the Company announced that adventitious agents testing of the master iPSC bank for the clinical production of FT500, a universal, off-the-shelf NK cell product candidate, has been completed. The FT500 master iPSC bank was found to be free of adventitious agents as determined by in vivo and in vitro testing. The Company has submitted these results to the U.S. Food and Drug Administration (FDA) in furtherance of the agency’s review of the Company’s FT500 Investigational New Drug (IND) application. Upon allowance by the FDA of the FT500 IND, the Company plans to initiate Phase 1 clinical testing of FT500 as a monotherapy and in combination with checkpoint inhibitor therapy for the treatment of advanced solid tumors. This first-in-human study is expected to evaluate the safety and tolerability of multiple doses of FT500 in multiple dosing cycles.

An updated presentation on the Company’s NK cell cancer immunotherapy franchise can be found under "Events & Presentations" in the Investors and Media section of the Company’s website at www.fatetherapeutics.com.

1 Excludes one subject treated at the run-in dose level in the cetuximab combination regimen (1×106 cells per kg)

About FATE-NK100
FATE-NK100 is an investigational, first-in-class, allogeneic donor-derived natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells, a highly specialized and functionally distinct subset of activated NK cells expressing the maturation marker CD57. Higher frequencies of CD57+ NK cells in the peripheral blood or tumor microenvironment in cancer patients have been linked to better clinical outcomes. In August 2017, non-clinical data describing the unique properties and anti-tumor activity of FATE-NK100 were published by Cancer Research (doi:10.1158/0008-5472.CAN-17-0799), a peer-reviewed journal of the American Association of Cancer Research. Three clinical trials of FATE-NK100 are currently being conducted: VOYAGE for the treatment of refractory or relapsed acute myelogenous leukemia; APOLLO for the treatment of recurrent ovarian cancer; and DIMENSION for the treatment of advanced solid tumors, including in combination with monoclonal antibody therapy.