On November 12, 2018 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of novel cellular immunotherapies, reported financial results and provided a corporate update for the third quarter ended September 30, 2018 and recent activities (Press release, Unum Therapeutics, NOV 12, 2018, View Source [SID1234531239]).

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!

"In the third quarter of 2018, we continued to make significant progress advancing our clinical pipeline," said Chuck Wilson, CEO of Unum. "We are excited to announce today that we have selected ACTR707 in combination with rituximab for further development in patients with relapsed or refractory non-Hodgkin lymphoma. Early data from the ATTCK-20-03 trial continue to support our novel ACTR platform and potential best-in-class product profile. In addition, we remain on track with our two additional clinical studies, ATTCK-17-01 in multiple myeloma and ATTCK-34-01 in HER2+ solid tumors, and are looking forward to presenting further updates on all trials at upcoming scientific conferences in December. Finally, we are pleased with our significant progress in developing a second novel technology platform, BOXR, which is designed to improve T cell functionality in solid tumors, and we look forward to developing future products based on this platform."

Recent Highlights

Selected ACTR707 as Lead Candidate for Further Development in r/r NHL: Unum has elected to advance ACTR707 in combination with rituximab as its lead product candidate to treat patients with relapsed or refractory CD20-positive B cell non-Hodgkin lymphoma (r/r NHL). As a result of this decision, Unum intends to conclude enrollment in the ATTCK-20-2 study, in the first half of 2019. The selection of ACTR707 as the lead construct in r/r NHL was based on the emerging clinical data from the Phase I ATTCK-20-03 clinical trial, the continuing progress in the ATTCK-20-03 study, and the Company’s desire to efficiently manage resources. At the first dose level of that trial, three out of six treated patients achieved a complete response, two of which remained ongoing at the time of the September 4 data cut off. Additionally, no dose-limiting toxicities (DLTs), serious or severe adverse events of cytokine release syndrome or neurotoxicity were observed in any patients. The findings in ATTCK-20-03 build on the encouraging data observed in cohort 1 of the ATTCK-20-2 trial, which evaluated ACTR087 in combination with

rituximab in patients with r/r NHL. Updated data from the ATTCK-20-03 trial will be presented at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December.

Continued Progress with ATTCK-17-01 Phase I trial: Unum is continuing to enroll and dose patients in ATTCK-17-01, a Phase I, multi-center, open-label clinical trial designed to test the safety, tolerability, and anti-myeloma activity of ACTR087 used in combination with SEA-BCMA in patients with r/r multiple myeloma through the dose escalation phase of the trial. This is the first clinical trial conducted under the strategic collaboration with Seattle Genetics. Unum plans to report preliminary data from early dose cohorts at the upcoming ASH (Free ASH Whitepaper) meeting in December 2018.

On Track to Initiate ATTCK-34-01 Trial by Year End in Solid Tumors: Unum’s investigational new drug (IND) application for ACTR T cells in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers has been accepted, and Unum plans to initiate the multi-center Phase I ATTCK-34-01 trial by the end of 2018, and announce preliminary data in 2019. ATTCK-34-01, Unum’s first ACTR combination study in solid tumors, is a multicenter, single-arm, open-label dose escalation study evaluating ACTR T cells in combination with trastuzumab and includes a planned expansion at the recommended Phase 2 dose. The primary study objectives are to assess the safety and tolerability of the combination, and to define dose recommendations for further study. Additional objectives include assessment of anti-tumor activity, ACTR T cell persistence and trastuzumab pharmacokinetics. Unum expects to present updated preclinical data along with the clinical trial design at the San Antonio Breast Cancer Symposium in December 2018.

Announced New Technology Platform to Expand Clinical Development Efforts in Solid Tumors: At the Society for Immunotherapy (SITC) (Free SITC Whitepaper) meeting in November, Unum presented preclinical data on a new technology platform called "BOXR," or Bolt-On Chimeric Receptor, that improves the functionality of T cells, enabling them to be more effective in solid tumor cancers. BOXR works with ACTR T cells or CAR T cells to significantly improve T cell functionality. Unum will disclose more details on this new platform, as well as its broader efforts in solid tumors, at the upcoming investor event on November 19, 2018, in New York, featuring guest speaker Charles S. Fuchs, MD, MPH, of Yale Cancer Center. The event will be available also via live webcast.

Third Quarter 2018 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the third quarter ended September 30, 2018 and 2017, of $2.0 million and $2.3 million, respectively, reflects the recognition of a portion of the $25.0 million upfront payment received from Seattle Genetics under Unum’s collaboration agreement as well as reimbursements of research and development costs by Seattle Genetics. Effective January 1, 2018, Unum adopted the new revenue recognition standard, ASC 606, which changed the manner in which the Company recognizes revenue from this collaboration agreement compared to the prior year period.

R&D Expenses: Research and development expenses were $10.3 million for the third quarter ended September 30, 2018, compared to $8.2 million for the same period last year. The increase reflects higher clinical trial costs for the active Phase I clinical trials, as well as increased personnel-related costs, materials and facility-related costs related to scaling manufacturing processes, and increased consultant costs. This was partially offset primarily by a decrease in manufacturing costs incurred for the Phase I clinical trial of ACTR087 in combination with rituximab.

G&A Expenses: General and administrative expenses for the third quarter ended September 30, 2018, were $2.4 million, compared to $1.3 million for the same period last year. The increase is primarily due to expenses around operating as a public company and higher personnel related costs.

Net Loss: Net loss attributable to common stockholders was $10.2 million, or $0.34 per share, for the third quarter ended September 30, 2018, and $7.0 million, or $0.69 per share, for the same period last year.

Cash, Cash Equivalents and Marketable Securities: As of September 30, 2018, Unum had cash, cash equivalents, and marketable securities of $87.1 million. Today, the Company updated its cash runway and now believes that its existing cash, cash equivalents, and marketable securities will fund operating expenses and capital expenditure requirements through at least June 2020, without considering $15.0 million in available borrowings under its loan and security agreement, as a result of its decision to conclude enrollment in the ATTCK-20-2 study in the first half of 2019.

On November 12, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted an exemption to the Import Alert placed on the Centre for Probe Development and Commercialization (CPDC), the sole supplier of the CLR 131 (Press release, Cellectar Biosciences, NOV 12, 2018, View Source [SID1234531227]). The exemption for CLR 131 is effective immediately for all hematology studies and, in response, Cellectar is preparing to dose patients in the second fractionated dose cohort of the Phase 1 relapsed refractory (R/R) multiple myeloma study and the Phase 2 study for R/R hematologic malignancies. The company awaits authorization from the FDA for any future shipments in connection with its Phase 1 study of pediatric patients with neuroblastoma, sarcomas, lymphomas (including Hodgkin’s lymphoma) and malignant brain tumors.

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!

"We thank the FDA for their diligence and for providing this exemption for CLR 131 hematology studies. Our ability to advance our clinical trials and achieve stated business objectives remains our top priority," said James Caruso, president and CEO of Cellectar Biosciences. "I also want to recognize our team for their outstanding execution in support of a rapid resolution."

In its efforts to obtain an exemption for CLR 131 from the Import Alert in hematology and pediatrics, Cellectar has collaborated with the various divisions within the FDA that oversee the company’s investigational new drug applications evaluating CLR 131 in multiple indications. Cellectar executed a series of actions requested by the FDA to obtain an exemption to the Import Alert for its hematology programs. Similarly, the company continues to work with the appropriate division of the FDA to secure an exemption for the pediatric program.

As background, on August 10, 2018, Cellectar announced that CPDC was informed of an FDA Import Alert that prohibited CPDC from supplying CLR 131. While the Import Alert disrupted CLR 131 supply, the basis of the Import Alert was not related to CLR 131 specifically, or to CPDC’s production facility associated with CLR 131. The company actively supported CPDC’s efforts to have the Import Alert lifted as quickly as possible. The FDA subsequently initiated direct talks with Cellectar concerning a possible exemption for CLR 131 from the Import Alert. Those discussions and subsequent actions resulted in the exemption Cellectar is announcing today.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131 is in a Phase 2 clinical study in relapsed/refractory multiple myeloma (R/R MM) and a range of B-cell malignancies, and a Phase 1b clinical study in patients with R/R MM exploring fractionated dosing. The objective of the multicenter, open-label, Phase 1b dose-escalation study is the characterization of safety and tolerability of CLR 131 in patients with R/R MM. Patients in Cohorts 1-4 received single doses of CLR 131 ranging from 12.5 mCi/m2 to 31.25 mCi/m2 as well as a fractionated dose of 15.625 mCi/m2 given twice over seven days in Cohort 5. All study doses and regimens have been deemed safe and well tolerated by an independent Data Monitoring Committee. The company plans to initiate a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma as well as a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On November 12, 2018 Ziopharm Oncology, Inc. (Nasdaq: ZIOP), reported that it has entered into definitive securities purchase agreements for the sale of its common stock and warrants to purchase common stock in a private placement that is expected to result in gross proceeds to the Company of approximately $50 million, before deducting placement agent and other offering expenses (Press release, Ziopharm, NOV 12, 2018, View Source [SID1234531224]). The private placement is being led by existing stockholder, MSD Partners, L.P. Other participants include Miller Value Partners, White Rock Capital Management and Level One Partners, LLC.

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!

Pursuant to the terms of the securities purchase agreement, at the closing of the private placement, Ziopharm will issue and sell 18,939,394 shares of common stock and warrants to purchase up to 18,939,394 additional shares of common stock at a per unit purchase price of $2.64. The warrants will become exercisable on the date that is six months following the date of issuance, have a per share exercise price of $3.01 and will expire five years from the date of issuance. The private placement is expected to close on or about November 13, 2018, subject to the satisfaction of customary closing conditions. Additional details regarding the private placement will be included in a Form 8-K filed by Ziopharm with the Securities and Exchange Commission (the "SEC").

Neither the shares of Ziopharm common stock nor the warrants to be issued in the private placement have been registered under the Securities Act of 1933, as amended (the "Securities Act"). Accordingly, these securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act. Ziopharm has agreed to file a registration statement with the SEC to register the resale of Ziopharm common stock to be issued in the private placement as well as the Ziopharm common stock issuable upon exercise of the warrants.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

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]).

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!

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 Bicycle Therapeutics, a biotechnology company pioneering a new class of therapeutics based on its proprietary bicyclic peptide (Bicycles) product platform, reported that its scientific co-founder, Sir Gregory Winter, the 2018 winner of the Nobel Prize in chemistry, will deliver the plenary keynote at the 2018 Protein and Antibody Engineering (PEGS) Summit Europe, held in Lisbon, Portugal (Press release, Bicycle Therapeutics, NOV 12, 2018, View Source [SID1234531210]). The title of his address, which will be delivered today at 4:20 p.m. GMT, is "Bicycles and Bicycle Drug Conjugates."

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!

In addition, Bicycle will present a poster highlighting preclinical research that supports the development of CD137 Bicycle agonists as novel cancer immunotherapies. The poster, which has been selected as a highlighted oral presentation, is titled "Novel Multimers of Bicyclic Peptides Cluster and Activate CD137 (4-1BB): A Costimulatory T-Cell Checkpoint Receptor" and will be presented on Thursday, Nov. 15 at 3:20 p.m. GMT.