On May 2, 2022 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 two abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy ("ASGCT") 25th Annual Meeting, taking place virtually and in person in Washington, D.C., May 16-19, 2022 (Press release, NeuBase Therapeutics, MAY 2, 2022, View Source [SID1234613291]).

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NeuBase will present new preclinical data on the biodistribution in key tissues of the company’s lead candidate, NT-0231.F, a precision genetic medicine in development to treat myotonic dystrophy, type 1 ("DM1"). NeuBase recently presented data at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of NT-0231.F in the HSALR model achieves clinically relevant molecular and functional rescue, including rescue of the spliceopathy, resolution of nuclear aggregates, and reversal of myotonia (delayed muscle relaxation after contraction). Pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice showed a single IV or SC dose achieved high volume of distribution.

The presentations are listed below, and the full abstracts are available on the ASGCT (Free ASGCT Whitepaper) meeting website. All times are listed in Eastern Time (ET).

Title: Pharmacokinetics, Biodistribution, and CNS Penetration of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy-Type 1 Following Systemic Administration Systemic Administration in Mice
Presenter: Noel Monks, Ph.D.
Session Date/Time: Tuesday, May 17, 5:30 PM – 6:30 PM
Poster Board Number: Tu-90
Session Title: Oligonucleotide Therapeutics II
Room: Hall D
Abstract Number: 585

Title: Pharmacology, Biodistribution and Tolerability of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Presenter: Sandra Rojas-Caro, M.D.
Session Date/Time: Wednesday, May 18, 5:30 PM – 6:30 PM
Poster Board Number: W-175
Session Title: Musculo-skeletal Diseases
Room: Hall D
Abstract Number: 1049

About NeuBase’s DM1 Program
Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splice proteins. NeuBase’s DM1 investigational genetic therapy, NT-0231.F, targets mutant DMPK pre-mRNA with a novel peptide-nucleic acid ("PNA") pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure, release the splicing proteins, and restore RNA splicing and downstream protein production. The PNA pharmacophore is conjugated to NeuBase’s novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1. For more information, visit www.neubasetherapeutics.com/pipeline/.

On May 2, 2022 Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo for patients with solid and hematologic malignancies, reported that it will have an oral presentation and two posters at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting, to be held May 16-19, 2022 (Press release, Umoja Biopharma, MAY 2, 2022, View Source [SID1234613290]).

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The oral presentation will include preliminary safety data of UB-VV100, a combination of VivoVec and RACR technology platforms. VivoVec is an in vivo lentiviral vector-based CAR T-cell platform developed for off-the-shelf use, while RACR is a rapamycin-activated cytokine receptor designed to drive in vivo CAR T-cell survival without the requirement for lymphodepletion through administration of rapamycin. The poster presentations will include descriptions of VivoVec platform advancements that expand particle mechanisms of action and enhance particle manufacturing processes.

Presentation details:

Oral Presentation Title: Preclinical Activity and Safety of UB-VV100, A Novel Lentiviral Vector Product Designed for Selective and Effective In Vivo Engineering of Therapeutic Anti-CD19 CAR T Cells for B cell Malignancies
Abstract Number: 1242
Session: Cell-Based Cancer Immunotherapies III
Presenting Author: Alissa Brandes, Ph.D., Principal Scientist, Umoja Biopharma
Presentation Date, Time: Thursday May 19, 2022; 10:15 AM – 12:00 PM ET

Poster Presentation Title: A Lentiviral-Based In Vivo CAR T Cell Generation Platform with Viral Particle Surface Engineering Incorporating Anti-CD3 Single Chain Variable Fragment and T Cell Costimulatory Molecules
Abstract Number: 879
Session: RNA Virus Vectors
Presenting Author: Christopher Nicolai, Ph.D., Senior Scientist, Umoja Biopharma
Presentation Date, Time: Wednesday May 18, 2022; 5:30 – 6:30 PM ET

Poster Presentation Title: Development of a Scalable, Suspension Cell Culture-Based Manufacturing Process for VivoVec, a Lentiviral Vector Platform for In Vivo CAR-T Cell Generation
Abstract Number: 1166
Session: Vector Product Engineering, Development or Manufacturing III
Presenting Author: Jeff Plomer, Senior Director Process Development, Umoja Biopharma
Presentation Date, Time: Wednesday May 18, 2022; 5:30 PM – 6:30 PM ET

Presentations can be accessed from the ASGCT (Free ASGCT Whitepaper) website at View Source

On May 2, 2022 Geron Corporation (Nasdaq: GERN) reported that it will release its first quarter 2022 financial results after the market closes on Monday, May 9, 2022 via press release, which will be available on the Company’s website at www.geron.com/investors (Press release, Geron, MAY 2, 2022, View Source [SID1234613289]). Geron will host a conference call to discuss the financial results as well as recent highlights at 4:30 p.m. ET the same day.

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A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events. An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link, View Source Participants that are unable to register online can access the conference call via telephone by dialing domestically +1 (888) 330-2434 or internationally +1 (240) 789-2725. The conference ID is 67335.

On May 2, 2022 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing next-generation multifunctional biotherapeutics, reported that management will participate in an upcoming investor conference (Press release, Zymeworks, MAY 2, 2022, View Source [SID1234613288]):

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H.C. Wainwright Global Investment Conference. Zymeworks will participate virtually in one-on-one meetings on May 24th – 26th and a corporate presentation will be available virtually on May 24th at 7 a.m. ET.

The presentation will be available on Zymeworks’ website at View Source

On May 2, 2022 NANOBIOTIX (Euronext: NANO – NASDAQ: NBTX – the "Company"), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported the presentation of three abstracts featuring potential first-in-class radioenhancer NBTXR3 at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 3-7 (Press release, Nanobiotix, MAY 2, 2022, View Source [SID1234613287]).

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"We are aiming to significantly improve outcomes for patients with cancer through NBTXR3, and to do so we must validate the safety and efficacy of our innovation across solid tumor indications and in combination with existing treatment modalities," said Laurent Levy, co-founder and chief executive officer of Nanobiotix. "Taken together with data we have previously reported on NBTXR3 as a single agent and in combination with checkpoint inhibitors, we expect the findings that will be presented at ASCO (Free ASCO Whitepaper) this year to add a new dimension to the story of NBTXR3 as we observe the radioenhancer’s performance in combination with chemotherapy. We will also highlight the design of our ongoing pivotal phase III study evaluating NBTXR3 as a single agent for frail, elderly patients with head and neck cancer."

The accepted abstracts include:

Abstract #18041: PEP503 (NBTXR3), a Radioenhancer, in Combination with Concurrent Chemoradiation (CCRT) in Locally Advanced or Recurrent Head and Neck Squamous Cell Carcinoma (HNSCC): Dose-Finding of a Phase 1b/2 Trial
Abstract #3603: A Phase 1b/2 Study of Radioenhancer, PEP503 (NBTXR3), in Combination with Concurrent Chemoradiation in Locally Advanced or Unresectable Rectal Cancer
Abstract #TPS6110: A Phase III Pivotal Study of NBTXR3 Activated by Investigator’s Choice of Radiotherapy Alone or Radiotherapy in Combination with Cetuximab for Platinum-based Chemotherapy-ineligible Elderly Patients with Locally Advanced Head & Neck Squamous Cell Carcinoma
About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product, composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physics-based mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering an adaptive immune response and long-term anti-cancer memory. Given the MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly, with immune checkpoint inhibitors.

NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) as the primary development pathway. The company-sponsored phase I dose escalation and dose expansion study has produced favorable safety data and early signs of efficacy. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy.

Nanobiotix has also prioritized an Immuno-Oncology development program—beginning with a Company sponsored phase I clinical study, evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/metastatic HNSCC and for patients with lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy, either naïve or resistant to prior PD-1 (either primary or secondary as per SITC (Free SITC Whitepaper) criteria).

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in strategic collaborations to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations. In 2021, the Company entered into an additional strategic collaboration agreement with LianBio to support its global phase III study in Asia along with four future registrational studies.