On May 2, 2023 BioCentriq, Inc. and panCELLa, a Pluristyx Company, reported that they have signed a research agreement designed to evaluate the ability of panCELLa’s genetically-engineered, induced Pluripotent Stem Cell (iPSC)-derived, feeder cells to activate and positively impact the expansion rate, total yield and potency of manufactured NK cells (Press release, panCELLa, MAY 2, 2023, View Source [SID1234630853]).

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BioCentriq, a contract development and manufacturing organization (CDMO) focused exclusively on accelerating the development and manufacturing of cell therapies, will expand and cryopreserve the feeder cell bank for use as a reagent to activate adult and iPSC-derived NK cells and evaluate viable cell yield and potency in static and dynamic culture conditions.

panCELLa developed the feeder line from proprietary, genetically-modified, iPSCs to serve as a critical raw material for easy adoption by therapy developers and their partners to overcome the challenges associated with making NK cell therapies. The panCELLa feeder line provides a stable and easily scalable platform that can be fine-tuned to maximize NK cell activation, expansion, and manufacturability. Companies wishing to utilize panCELLa’s feeder line can access a research version for evaluation and a clinical-grade format for therapeutic use through panCELLa and its sub-licensees.

"We envision the panCELLa-provided feeder line as an effective tool in our quest to accelerate the manufacturing of NK cell therapies and can be added to BioCentriq’s proprietary LEAP platform," said Alex Klarer, Vice President of Business Strategy and Innovation at BioCentriq. "Our LEAP-NK platform is specifically designed to help biotech companies with promising NK cell therapies advance from lab to clinic in half the time it would normally take by leveraging proven technology, completed development work and established processes ready for GMP production."

Mahendra Rao, Chief Scientific Officer at Pluristyx, said, "We are pleased that BioCentriq selected our feeder line technology to better serve their cell therapy clients in solving the challenges of NK cell commercial manufacturing. We look forward to working with BioCentriq to bring the next generation of customized cell products and service solutions to speed the transition of these revolutionary therapies from the benchtop to the bedside."

On May 2, 2023 NKGen Biotech Inc. ("NKGen"), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic and CAR-NK Natural Killer ("NK") cell therapeutics, reported an upcoming presentation on its NK cell therapy (SNK01) at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, which will take place in Los Angeles, California from May 16 – 20, 2023 (Press release, NKGEN Biotech, MAY 2, 2023, View Source [SID1234630852]).

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Presentation Details

Presentation Title: SNK01 Autologous Enhanced Natural Killer Cells and an Immune Checkpoint Inhibitor Control Tumor Growth in Rare Chemotherapy-Resistant Advanced Soft Tissue Sarcomas

Presenting Author: Erlinda M. Gordon, M.D.

Session Type: Poster Presentation

Date & Time: Thursday, May 18, 2023; 12:00 p.m. – 2:00 p.m. PT

Abstract Number: 1136

Dr. Gordon will present data from three rare cases of chemotherapy-resistant advanced soft tissue sarcomas (STS) which achieved durable partial responses and disease control with SNK01 plus an immune checkpoint inhibitor. NKGen believes the data from these three cases support the potential combination of SNK01 and an immune checkpoint inhibitor in controlling tumor growth with manageable toxicity.

Full abstracts are available on the ASGCT (Free ASGCT Whitepaper) conference website View Source

On May 2, 2023 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Bank of America Securities 2023 Healthcare Conference at 10:00 a.m. Pacific Time (1:00 p.m. Eastern Time) on Tuesday, May 9, 2023. Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer, will present at the conference (Press release, Neurocrine Biosciences, MAY 2, 2023, View Source [SID1234630851]).

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The live presentation will be webcast and may be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

On May 2, 2023 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing Stealth Editors to perform in vivo gene editing without triggering the immune system, reported two abstracts have been accepted for two oral presentations at the American Society of Gene & Cell Therapy ("ASGCT") 26th Annual Meeting, which will take place in Los Angeles, CA and virtually on May 16-20, 2023 (Press release, NeuBase Therapeutics, MAY 2, 2023, View Source [SID1234630850]).

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Details of the oral presentations are listed below, and the full abstracts are available on the ASGCT (Free ASGCT Whitepaper) meeting website.

Title: Toxicology, Pharmacokinetics and Biodistribution of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Presenter: Dr. William Riedl
Presentation Time: 1:45 PM – 2:00 PM PT
Session Date/Time: Thursday, May 18, 2023; 1:30 PM – 3:15 PM PT
Session Title: Gene Therapy Approaches for Muscle and Skeletal Diseases
Room: Room 408 AB
Abstract Number: 131

Title: Nuclease-Free Gene Editing with Peptide Nucleic Acids: A New Class of In Vivo Gene Editors
Presenter: Dr. Dani Stoltzfus
Presentation Time: 8:30 AM – 8:45 AM PT
Session Date/Time: Saturday, May 20, 2023; 8:00 AM – 9:45 AM PT
Session Title: Genome & Epigenome Editing Technologies II
Room: Concourse Hall 152 & 153
Abstract Number: 335

On May 2, 2023 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs, for hard-to-treat tumors and viruses, reported the successful completion of the first cohort in the Phase 1b portion of its Phase 1b/2 clinical trial using Annamycin in combination with Cytarabine for the treatment of AML (MB-106) (Press release, Moleculin, MAY 2, 2023, https://moleculin.com/moleculin-successfully-completes-first-cohort-in-phase-1b-2-trial-evaluating-annamycin-in-combination-with-cytarabine-ara-c-for-the-treatment-of-acute-myeloid-leukemia-aml/ [SID1234630849]).

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In this first cohort three subjects were treated, all of whom are believed to be relapsed from multiple prior therapies. Annamycin was dosed at 190 mg/m2, along with Cytarabine at 2.0 g/m2/day for 5 days (total dose of 10 g/m2), consistent with the familiar 7+3 regimen, combining daunorubicin and Cytarabine, typically used as a first-line induction therapy. In the Company’s study therapy Annamycin replaces daunorubicin. The Company, at the recommendation of the data safety committee, deemed the first cohort dose as safe and opened the second cohort with the Annamycin dose being increased to 230 mg/m2.

The median of prior therapies for these three subjects were 5 (2 to 7). One of the subjects, who was 78 years of age at the time of study initiation with 2 prior multi-year therapies, was preliminarily recorded as a complete response at three weeks, or "CR", per the protocol. This was subsequently confirmed to be a durable CR after four weeks. The other two subjects were shown to have disease progression. Subjects have already been identified as potential candidates for the second cohort.

"We are pleased with the rapid progress of this trial and to be advancing to the second cohort of escalated dosing. These preliminary results demonstrated by Annamycin in combination with Ara-C, add to our confidence in Annamycin’s potential to be an important treatment for refractory or relapsed AML. Since it wasn’t until we exceeded 210 mg/m2 in our single agent trials that we saw a consistent response rate (80%), we are glad to see a CR at just 190 mg/m2 and are eager to see how subjects respond at 230 mg/m2, especially since our preclinical animal studies showed a significant increase in activity when Annamycin is combined with Cytarabine, as it is in this trial. We are also encouraged to see a full slate of candidates already identified for the second cohort and our clinical team is working to get subject screening and dosing for this next cohort. We look forward to providing additional updates in the near future," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

The Phase 1b/2 MB-106 trial is an open label trial that builds on the safety and dosage data from the two successfully concluded single agent Annamycin AML Phase 1 trials, MB-104 and MB-105, conducted in the U.S. and Europe, respectively.

Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory AML and soft tissue sarcoma (STS) lung metastases and the Company believes it may have the potential to treat additional indications.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of STS lung metastases and the treatment of relapsed or refractory AML. For more information about the MB-106 Phase 1/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.