On September 15, 2021 GT Biopharma, Inc. (the "Company") (NASDAQ: GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary natural killer (NK) cell engager, TriKE protein biologic technology platform, reported that Jeffrey Miller, MD, University of Minnesota Medical School, Professor of Medicine, Division of Hematology, Oncology and Transportation will present a mini-oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 to be held virtually September 16-21 (Press release, GT Biopharma, SEP 15, 2021, View Source [SID1234587783]).

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The mini-oral presentation will present updated positive Phase 1 safety data, progress, and preclinical data going beyond hematologic malignancies to solid tumors of a Phase 1 GTB-3550 TriKE trial. The Tri-Specific Killer Engager TriKE program is currently in pre-clinical and clinical development for the treatment of relapsed/refractory acute myelogenous leukemia (AML) and high-risk myelodysplastic syndrome (MDS) with solid tumor TriKE commercial manufacturing and IND enabling studies in progress.

Mini-oral Poster Presentation Details:

Title: GTB-3550 TriKE safely activates and delivers IL-15 to NK cells, but not T cells, in immune suppressed patients with advanced myeloid malignancies, a novel paradigm exportable to solid tumors expressing Her2 or B7H3 (Abstract #4068)

Speaker: Jeffrey Miller, MD

Mini-oral Session: Investigational Immunotherapy (Channel 2)

Presentation Time: September 17 at 6:10 PM EST

Mini-oral Presentation Number: 965MO

The abstract is currently available on the ESMO (Free ESMO Whitepaper) website at www.esmo.org. At the start of the mini-oral session the presentation will be available in the "Presentations" section of the Company’s website at View Source

Recent Announcement
The Company recently announced the advancement of GTB-3650 into IND-enabling studies, with which it plans to supplant the ongoing Phase 1 program with GTB-3550. GTB-3650 is a novel molecule based on camelid single-domain camelid antibody technology with advantages that build upon the strong proof-of-concept data from the Company’s first generation TriKE program, GTB-3550.

Therapeutic and commercial advantages of GTB-3650 compared to GTB-3550 include:

Based on second generation camelid single-domain antibody technology that holds several advantages over traditional IgG monoclonal antibodies
Improved potency and enhanced binding affinity
Similar preclinical safety profile
Commercial manufacturing capabilities through arrangement with Cytovance
Proprietary patented molecule, which unlike GTB-3550, is wholly owned by GT Biopharma
About Camelid Antibodies

Camelid antibodies are single domain antibodies (sdAbs) from the Camelidae family of mammals that include llamas, camels, and alpacas. These animals produce 2 main types of antibodies. One type of antibody camelids produce is the conventional antibody that is made up of 2 heavy chains and 2 light chains. They also produce another type of antibody that is made up of only 2 heavy chains and no light chain. This is known as heavy chain IgG (hcIgG). While these antibodies do not contain the CH1 region, they retain an antigen binding domain called the VHH region. VHH antibodies, also known as single domain antibodies, contain only the VHH region from the camelid antibody. Camelid antibodies have key characteristics, which include high affinity and specificity (equivalent to conventional antibodies), high thermostability, good solubility and strictly monomeric behavior, small size, relatively low production cost, ease of genetic engineering, format flexibility or modularity, low immunogenicity, and a higher penetration rate into tissues.

About GTB-3650

GTB-3650 is the Company’s lead second-generation Tri-Specific Killer Engager TriKE program currently in preclinical development for the treatment of relapsed/refractory acute myelogenous leukemia (AML) and high-risk myelodysplastic syndrome (MDS).

On September 15, 2021 PsiOxus Therapeutics, Ltd. (PsiOxus), a tumor re-engineering company, reported that they will present key safety and translational data from their first-in-human phase 1 FORTITUDE clinical study at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 this week. Data from the completed monotherapy dose-escalation part of the FORTITUDE study, initiated in 2019 to assess the safety and tolerability of the NG-350A T-SIGn vector, will be presented on Friday 16th September 2021, with the poster available in full at www.psioxus.com shortly afterwards.

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NG-350A is a T-SIGn vector designed to re-engineer cancers by selectively expressing a CD40 agonist monoclonal antibody, a potent activator of immuno-inflammatory responses, within the tumour microenvironment. PsiOxus is developing this agent as one of several products within its T-SIGn portfolio of vectors that combine systemic delivery with localized production of powerful transgene payloads to allow the selective re-engineering of both primary and metastatic tumors.

The data to be presented at the ESMO (Free ESMO Whitepaper) Congress show that IV delivery of NG-350A results in sustained elevations of inflammatory cytokines in the phase 1 FORTITUDE trial. In particular, marked and persistent dose-dependent increases in both IL-12 and IFNγ were observed after a single 1-week course of NG-350A, indicative of robust activation of antigen presenting cells via CD40 agonism generated within the tumor. Expansion of new T cell clones, a high proportion of which were new clones, was also observed following a single cycle of NG-350A. Safety data from the 25 patients treated with NG-350A as part of the now completed monotherapy dose-escalation part of FORTITUDE demonstrated that NG‑350A was well-tolerated, with few of the adverse events associated with systemic delivery of anti-CD40 agonists observed.

Together, these data suggest NG-350A contributes to the re-programming of the tumour microenvironment while avoiding the toxicity associated with systemic non-localized dosing of anti-CD40 antibodies.

"The headline data shared at the ESMO (Free ESMO Whitepaper) Congress confirms previous findings that our T-SIGn vector replicates selectively in primary tumor cells and metastases and persists for several months after intravenous delivery. Even more importantly, the biomarker data indicates that ongoing vector replication in tumors effectively translates into sustained production of the transgene payload, in this case a CD40 agonistic antibody. This translational data is a first in class demonstration of a downstream effect of tumor re-engineering, using T-SIGn vectors to turn the patient’s tumor cells into small drug factories," said Tom Lille, M.D., Ph.D., Chief Medical Officer, PsiOxus.

Based on these highly promising data, NG-350A will be assessed in combination with an anti–PD-1 checkpoint inhibitor in Part B of FORTITUDE.

On September 15, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will present a corporate overview at the virtual Oppenheimer Fall Healthcare Life Sciences & MedTech Summit being held September 20 – 23 (Press release, NeuBase Therapeutics, SEP 15, 2021, View Source [SID1234587743]).

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Oppenheimer Fall Healthcare Life Sciences & MedTech Summit

Date: Tuesday, September 21st
Time: 3:45 p.m. ET
Location: Webcast Link – or at the company’s website (click here)

On September 15, 2021 Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported participation in the following scientific conferences during the month of September (Press release, Exicure, SEP 15, 2021, View Source [SID1234587761]):

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TIDES USA 2021
Poster presentation: "Biodistribution of Spherical Nucleic Acids in the Rat CNS Following ICM and IT Delivery"
September 20 – 30, 2021

17th Annual Meeting of the Oligonucleotide Therapeutics Society
Poster presentation: "Development of Spherical Nucleic Acids Targeting FXN for the Treatment of Friedreich’s Ataxia"
September 26 – 29, 2021

Posters will go live at the start of the conference and will be available to conference attendees.

Exicure will add the posters to its website after each conference.

On September 15, 2021 Starpharma reported that it’s DEP cabazitaxel is a proprietary nanoparticle version of leading prostate cancer drug cabazitaxel (Jevtana), which had global sales of US$536 million in 2020 (Press release, Starpharma, SEP 15, 2021, View Source [SID1234587784]). Starpharma’s DEP cabazitaxel is a water soluble, polysorbate-80 free formulation, without requirement for pre-treatment with steroids nor G-CSF to reduce the risk of severe bone marrow toxicity .

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In preclinical and clinical studies, DEP cabazitaxel has shown an improved side effect profile, notably markedly reduced bone marrow toxicity demonstrated by lower rates of severe neutropenia, thrombocytopenia, and severe anaemia, which are experienced by a significant proportion of Jevtana treated patients.

The composition of matter patent builds on Starpharma’s suite of existing international patents for DEP cabazitaxel. It specifically covers a DEP dendrimer conjugated to multiple cabazitaxel drug molecules via a particular releasable linker, with a patent term to 2039 and potential for a 5-year extension.

Starpharma CEO, Dr Jackie Fairley, commented: "The grant of this new US patent illustrates the unique and compelling benefits of Starpharma’s DEP drug delivery technology and DEP cabazitaxel. We look forward to completing the phase 2 clinical program for DEP cabazitaxel, in parallel with commercial licensing discussions."

DEP cabazitaxel is in late phase 2 clinical development, recruiting patients with solid tissue tumours, including prostate, ovarian and gastro-oesophageal cancers.

Encouraging efficacy signals have been observed in multiple tumour types, including in prostate cancer where radiological responses, significant reductions in prostate-specific antigen (PSA) and no new bone metastases were observed. These efficacy signals were observed despite patients having been heavily pre-treated with an average of 30 prior cycles of treatment, and in some cases with more than 100 cycles and up to 10 different treatment regimens. Patients treated with DEP cabazitaxel have also exhibited encouraging efficacy signals in gastro-oesophageal, ovarian, cholangiocarcinoma, lung and head and neck cancers.

DEP cabazitaxel was developed using Starpharma’s proprietary DEP drug delivery platform, used by the company and partners to create novel nanoparticle formulations of existing and new drugs to enhance their therapeutic and commercial value. DEP drug delivery is applicable to a wide range of drugs in oncology and other therapeutic areas. Starpharma has three phase 2 clinical-stage DEP assets, multiple preclinical DEP programs, and several DEP commercial partnerships with companies, including AstraZeneca, Chase Sun and Merck & Co., Inc., to develop DEP versions of their products or ADCs.