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.

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"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.

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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]).

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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 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."

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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.

On November 12, 2018 Medigene AG (FSE: MDG1, Prime Standard, SDAX) reported that it has entered into an exclusive license agreement with Leiden University Medical Center (LUMC), the Netherlands, for worldwide rights to develop, manufacture, and commercialize an HA-1-specific T cell receptor (TCR) as a targeted immunotherapy for cancer (Press release, MediGene, NOV 12, 2018, View Source [SID1234531209]).

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The TCR specific for the minor histocompatibility antigen HA-1 was developed by LUMC and tested for preliminary safety and tolerability in a Phase I clinical trial involving five patients. HA-1 is a well-characterized antigen expressed in cells of the hematopoietic system, in leukemia and lymphoma cells, as well as expressed in various solid tumors.

Dr Kai Pinkernell, CMO and CDO of Medigene , said: "We are excited to have licensed this HA-1-targeting T cell receptor, which has already undergone early clinical testing, to complement Medigene’s preclinical and clinical development program. Scientists from LUMC and Medigene have had a longstanding shared scientific interest and exchange on HA-1 and we look forward to our future interactions. Our own extensive research and development for the HA-1 antigen confirms the potential of this approach in general and this TCR candidate in particular. Its potential applicability in both liquid and solid tumors makes strategic sense within our growing internal pipeline."

Mirjam H.M. Heemskerk, PhD, Associate Professor of the Department of Hematology at the Leiden University Medical Center , said: "Medigene is an ideal partner to further advance this promising TCR candidate given their deep scientific expertise, broad development work and strong focus on TCRs. We share a common vision of developing T cell receptor therapies to offer seriously ill cancer patients much-needed treatment alternatives."

Under the terms of the agreement, Medigene will receive the exclusive worldwide development and commercialization rights to the HA-1-specific TCR developed by LUMC. In return, LUMC will receive a one-time payment along with certain milestone payments. Upon commercialization, LUMC is eligible for royalties in the low-single digit percentage range. Confidentiality was agreed regarding further financial details.

Terms of the deal do not materially impact Medigene’s financial guidance for the balance of the year 2018.

About HA-1: The HA-1 antigen has been extensively studied by Medigene’s research team. It is a clinically validated T cell target used in stem cell transplantation (SCT). HA-1, as a single nucleotide polymorphism (SNP), is easily detected by a PCR method and shows a well-characterized tissue expression pattern.