On November 13, 2018 RadioMedix Inc. (a Texas-based clinical stage biotechnology company) and Orano Med (a nuclear biotechnology company) reported that the United States Food & Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AlphaMedixTM for the treatment of neuroendocrine tumors (NETs) (Press release, RadioMedix, NOV 13, 2018, View Source [SID1234531347]). AlphaMedixTM is a 212Pb-labeled somatostatin analogue used for the targeted alpha therapy (TAT) of neuroendocrine tumors (NETs).

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"TAT has brought new hope to our patients with cancer, and the range of available therapies in this area will only increase in the near future. We are pleased to conduct the first TAT clinical trial in the U.S. for neuroendocrine cancers, with the aim of registration of the drug," said Dr. Ebrahim S. Delpassand, CEO of RadioMedix and Medical Director of the clinical studies. "We are hoping that AlphaMedixTM will provide a breakthrough therapy that will benefit our patients soon," added Dr. Delpassand. "We are excited about the progress of our dose escalation studies of AlphaMedixTM", echoed Dr. Izabela Tworowska, Ph.D., CSO of RadioMedix.

The mechanism of action of AlphaMedixTM relies on the delivery of a cytotoxic load of alpha-emitters to SSTR-overexpressing neuroendocrine tumors. Alpha- emitters are highly energetic radioactive particles that can cause irreversible damage to the cancer cells. RadioMedix and Orano Med initiated the Phase 1 clinical trial of the safety and preliminary efficacy of AlphaMedixTM in February of 2018 (NCT03466216); the study is currently recruiting patients at the Excel Diagnostics and Nuclear Oncology Center in Houston, TX U.S.

"FDA’s decision to grant Orphan drug Designation is an important regulatory milestone for AlphaMedixTM, a key program in our pipeline of 212Pb-labeled compounds," said Julien Dodet, CEO of Orano Med. "We believe TAT with 212Pb represents a promising new approach and that AlphaMedixTM has the potential to have a meaningful impact on patients".

Orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. The designation provides certain benefits to drug developers such as seven-year marketing exclusivity upon FDA approval, tax credits for qualified clinical testing or prescription drug user fee exemption.

About Neuroendocrine Tumors

Neuroendocrine tumors (NETs) are a heterogeneous group of rare neoplasms that originate from neuroendocrine cells. These neoplasms occur mostly in the gastrointestinal tract and pancreas, but can also occur in other tissues including thymus, lung, and other uncommon sites such as ovaries, heart, and prostate. Most NETs strongly express somatostatin receptors (SSTRs).

About AlphaMedixTM

AlphaMedixTM is a radiolabeled SSTR-targeting therapeutic investigational drug for the treatment of NETs patients. The product consists of SSTR-targeting peptide complex radiolabeled with 212Pb and serve as an in vivo generator of alpha-emitting particles. 212Pb isotope is particularly suitable for SSTR therapy applications based upon its half-life, energy and decay properties.

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.

On November 12, 2018 Complix, a biopharmaceutical company developing a pipeline of transformative Cell Penetrating Alphabodies (CPABs) reported that its Chief Scientific Officer, Dr Yvonne McGrath, will be presenting recent developments on CPABs against intracellular targets at the 10th Annual PEGS Europe Meeting (Protein and Antibody Engineering Summit) in Lisbon Portugal, November 12-16 (Press release, Complix, NOV 12, 2018, View Source [SID1234531276]).

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In her presentation Dr McGrath will provide an update on Complix’ recent progress with respect to CAPBs acting on important intracellular cancer targets. In particular Dr McGrath will be showing results obtained with CPABs targeting the enhanceosome of the Wnt pathway, a key pathway involved in development of cancer and other major diseases.

Details of the talk are as follows:

Title: "Cell Penetrating Alphabodies to Access Undruggable Intracellular Targets"
Session: "Engineering Antibodies"
Time: November 14 at 09:05 CET

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 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.