Ono Enters into a Research Collaboration Agreement with Turbine to Identify and Validate Novel Therapeutic Targets in Oncology

On October 24, 2023 Ono reported that it has entered into a research collaboration agreement with Turbine (London, UK; CEO and Co-Founder: Szabolcs Nagy), a computational biology company that deploys empirically validated in silico cell simulations to guide biopharma R&D, to identify and validate novel therapeutic targets in the field of oncology (Press release, Ono, OCT 24, 2023, View Source [SID1234646640]).

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Under the terms of the agreement, Turbine will apply its end-to-end, interpretable cell simulation technology, the Simulated Cell platform, to the in silico discovery of novel targets for potential therapeutic development. Turbine will also conduct in vitro mechanism validation of identified targets in its state-of-the-art laboratory facility and manage in vivo validation studies. Ono will exclusively develop and commercialize drug candidates to the targets identified by Turbine worldwide. Ono will pay to Turbine an up-front payment, research funding, milestone payments based on the completion of target identification and validation by Turbine, as well as milestones on the progress of drug development and commercialization by Ono.

 "We appreciate Turbine’s AI-driven cell simulation platform for discovering novel therapeutic targets that cannot be identified using traditional approaches. Turbine will also provide information on the mechanisms of action and biomarkers of the target, which can accelerate drug development in Ono," said Toichi Takino, Senior Executive Officer / Executive Director, Discovery & Research of Ono. "Through this collaboration, we are committed to expanding our oncology development pipeline and bringing new therapeutic options to cancer patients with high unmet needs."

 "Ono has a history of developing transformative cancer therapies, including its important role in launching Opdivo (Generic name: nivolumab), the first ever approved PD-1 immune checkpoint inhibitor," said Szabolcs Nagy, Chief Executive Officer and Co-Founder of Turbine. "We believe that our cutting-edge cell simulation technology will enable the discovery of additional targets to fuel the expansion of Ono’s oncology pipeline. We are excited for the opportunity to demonstrate the combination of our Simulated Cells and proprietary validation assays that both enable validating computational predictions and drive the development of an ever more predictive simulation of human biology."

About Simulated Cell technology
 Turbine’s Simulated Cell technology leverages machine learning to build an end-to-end, interpretable cell simulation platform based on protein interactome and additional omics layers. The virtual cells capture patient biology better than currently available experimental models and are used for in silico experiments having never been run in the lab, testing more drug-like effects than current high throughput genetic screening approaches. Validating the uncovered hypotheses of causality in Turbine’s state-of-the-art laboratory facility and using the resulting data as feedback further improves the model’s predictive prowess. Informing biopharma R&D through in silico experiments improves the likelihood of success for truly novel therapies and allows existing assets to be optimally targeted at responder patients.

Lipella Pharmaceuticals Announces $2 Million Private Placement Priced At-The-Market Under Nasdaq Rules

On October 24, 2023 Lipella Pharmaceuticals Inc. (Nasdaq: "LIPO") ("Lipella," "our," "us" or the "Company"), a clinical-stage biotechnology company addressing serious diseases with significant unmet need, reported the entry, on October 23, 2023, into definitive agreements for the purchase and sale, in a private placement transaction priced at-the-market under Nasdaq rules, ofpre-funded warrants to purchase up to an aggregate of 1,315,790 shares and warrants to purchase up to an aggregate of 1,315,790 shares (Press release, Lipella Pharmaceuticals, OCT 24, 2023, View Source [SID1234636377]). The purchase price for each pre-funded warrant and accompanying warrant is $1.519.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants will have an exercise price of $1.40 per share, will be immediately exercisable upon issuance, and will expire three years from the date of issuance. The offering is expected to close on or about October 25, 2023, subject to the satisfaction of customary closing conditions.

The gross proceeds to Lipella from the private placement are expected to be approximately $2 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds for working capital and for general corporate purposes.

The offer and sale of the securities described above are being offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder, and such securities, along with the shares of common stock underlying the warrants and pre-funded warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the securities issued in the private placement and the shares of common stock underlying the warrants and pre-funded warrants, 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 Act and such applicable state securities laws.

The Company has agreed to file an initial registration statement with the Securities and Exchange Commission ("SEC") covering the resale of the shares of common stock underlying the warrants and pre-funded warrants, no later than seven business days following the date of the definitive agreements and to have the registration statement declared effective no later than 75 days following the date of the definitive agreements in the event of a "full review" by the SEC.

This press release does not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.

KaliVir Immunotherapeutics Announces FDA Clearance of Investigational New Drug (IND) for Systemic Oncolytic Virus ASP1012 for Phase I Clinical Trials for Locally Advanced or Metastatic Solid Tumors

On October 24, 2023 KaliVir Immunotherapeutics, Inc., a biotech company developing cutting-edge, multi-mechanistic oncolytic viral immunotherapy programs, reported clearance of an Investigational New Drug (IND) application by the United States Food and Drug Administration (FDA) to initiate a Phase 1 clinical study of ASP1012 in participants with locally advanced or metastatic solid tumors (Press release, KaliVir Immunotherapeutics, OCT 24, 2023, View Source [SID1234636320]).

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Discovered and developed by Kalivir and licensed to Astellas Pharma Inc. in December 2020, ASP1012 (formerly named VET2-L2) is a systemic oncolytic vaccinia virus therapy in which the virus is delivered intravenously and expresses Leptin-IL2 fusion protein as a therapeutic payload. The trial is expected to begin in Q1 2024.

"ASP1012 was the first candidate built from our unique Vaccinia Enhanced Template (VET) platform and we are thrilled to reach this important milestone," said Steve Thorne, Ph.D, Chief Scientific Officer and founder of Kalivir. "Our partnership with Astellas underscores our mission to bring critical cancer therapies to patients and we look forward to continued collaborations."

"Immuno-oncology is a core focus of the Astellas R&D strategy and enriching our pipeline with the oncolytic virus ASP1012 is a testament to our commitment to provide new options to treat cancers where there are no effective treatment options," said Peter Sandor, M.D., MBA, Senior Vice President and Primary Focus Lead, Immuno-Oncology, Astellas. "The combination of KaliVir’s expertise in oncolytic viruses and Astellas’ drug development capabilities has been a great partnership and it’s exciting to see the collaboration move forward in this way, bringing us one step closer to developing an immuno-oncology therapy for patients."

Nutcracker Therapeutics to Present Data for Two mRNA Drug Candidates at SITC 2023

On October 24, 2023 Nutcracker Therapeutics, Inc., a biotechnology company dedicated to developing transformative RNA therapies through its proprietary technology platform, reported two poster presentations: one on the company’s lead mRNA therapeutic candidate, NTX-250, which targets human papillomavirus (HPV)-driven cancers; the other on its latest therapeutic candidate, NTX-471, which targets CD47 and CCR4 (Press release, Nutcracker Therapeutics, OCT 24, 2023, View Source [SID1234636319]). These data will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC’s) 38th Annual Meeting & Pre-Conference Programs (SITC 2023) in San Diego, CA, from November 1 through 5, 2023.

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Nutcracker’s presentation on NTX-250 will describe how its three RNA-encoded protein components (HPV E6/E7, IL-12, and LIGHT) work together to drive enhanced immune responses and tumor clearance in murine models of HPV-driven tumors, while also providing immunogenic memory. The presentation on NTX-471 will describe unique mRNA compositions that can selectively target CD47 on cancerous cells over those on red blood cells, potentially alleviating the anemic side effects seen with anti-CD47 drugs currently under development.

Poster Presentations at SITC (Free SITC Whitepaper) 2023

Title: mNTX-250, a novel multimodal HPV-16 mRNA-based therapeutic, induces potent anti-tumor responses and establishes HPV-16 specific immune memory
Abstract Number: 134
Session Date and Time: Saturday, Nov 4th, 2023 9:00 AM – 7:00 PM
Lead Author: Ou Li

Title: NTX-471, engineered multivalent SIRPa and bispecific SIRPa-antiCCR4 molecules demonstrate superior activity providing path for mRNA-expressed in vivo biologics
Abstract Number: 1359
Session Date and Time: Friday, November 3, 2023: 9:00 AM – 7:00 PM
Lead Author: Gunasekaran Kannan

Poster presentations will be accessible in person and virtually. All accepted abstracts will be available in a Journal for ImmunoTherapy of Cancer (JITC) supplement, which will be published on October 31 at 9:00 a.m. EST. For more information about SITC (Free SITC Whitepaper) 2023, please visit View Source

Ankyra Therapeutics Receives FDA IND and Health Canada CTA Approval to Advance First-In-Class Selective Anchored Immunotherapy Agent, ANK-101, into Human Trials in Solid Tumors

On October 24, 2023 Ankyra Therapeutics, a clinical stage biotechnology company pioneering anchored immunotherapies to treat cancer, reported approval of its investigational new drug (IND) application by the U.S. Food & Drug Administration (FDA) and clinical trial application (CTA) by Health Canada for its lead agent, ANK-101, a novel tumor-directed anchored immune medicine (Press release, Ankyra Therapeutics, OCT 24, 2023, View Source [SID1234636318]). The Company plans to initiate a first-in-human Phase I clinical trial of ANK-101 as a single agent in early 2024 at a limited number of clinical sites in the U.S. and Canada in patients with advanced solid tumors who have failed standard of care treatments.

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ANK-101, an interleukin-12 (IL-12) cytokine anchored to aluminum hydroxide, is locally delivered and retained in the tumor microenvironment for several weeks where it mediates recruitment and activation of effector immune cells. Ankyra has demonstrated single agent activity of ANK-101 in preclinical studies of various solid tumors in mice as well as in canine melanoma (cANK-101) with a tolerable safety profile.

"By anchoring to and being retained at the tumor site, ANK-101 has the ability to avoid hallmark challenges of both systemic and intratumoral therapies, namely by preventing cytokine diffusion, effectively training the body to potently destroy cancer cells," said Howard L. Kaufman, MD, CEO and President of Ankyra Therapeutics. "We are very excited about the clearance of our CTA and IND, which will allow us to bring ANK-101 to patients who may benefit from this new approach to cancer treatment."

Joe Elassal, MD, MBA, Ankyra’s Chief Medical Officer, added "ANK-101 represents potential treatment benefit without unwanted side effects. Our hope is that by anchoring the IL-12 at the site of tumor growth, we will see therapeutic activity without systemic toxicity. We look forward to advancing our first-in-class asset to realize this vision, and to paving the way for anchored immunotherapy to safely deliver other biologically active agents in the future."

About ANK-101
ANK-101 is an anchored drug complex composed of interleukin-12 (IL-12) linked to aluminum hydroxide. ANK-101 enables local delivery of functional IL-12 to the tumor microenvironment where it remains biologically active for several weeks but does not diffuse into the systemic circulation, thereby avoiding systemic toxicity. Treatment with ANK-101 in animal models has been associated with recruitment and retention of tumor-specific CD8+ T cells, NK cells and M1 macrophages activating innate and adaptive anti-tumor immunity. ANK-101 is being evaluated for the treatment of advanced solid tumors alone and in combination with anti-PD-1 agents.