On January 9, 2023 Secura Bio, Inc. (Secura Bio) – (www.securabio.com), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, reported that the European Commission (EC) issued an Orphan Drug Designation for duvelisib for the treatment of patients with peripheral T-cell lymphoma (PTCL) (Press release, Secura Bio, JAN 9, 2023, View Source [SID1234626127]). Duvelisib was previously granted orphan drug designation by the United States Food and Drug Administration.

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Orphan Drug Designation in the EU is granted by the EC based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products. It is intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. If approved for marketing, this designation will provide ten years of marketing exclusivity and other special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.

Duvelisib is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first US-approved dual inhibitor of PI3K-delta and gamma pathways, which are involved in the proliferation and sustenance of malignant cells. Duvelisib was fully approved by the US Food and Drug Administration in September 2018 for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least two prior therapies.

"This Orphan Drug Designation recognizes the significant unmet need in patients with PTCL, especially those with relapsed/refractory disease. This patient group has very limited therapeutic options and duvelisib may offer a new choice of therapy." Said Dr. David Sidransky, Clinical Advisor to Secura Bio.

"Secura Bio is dedicated to developing duvelisib for the treatment of patients with difficult-to-treat cancers, which includes relapsed/refractory PTCL. We are investing significant corporate resources in this endeavor and hope to see new treatment options brought to the market which may benefit patients, such as those with relapse/refractory PTCL." Said Joseph M. Limber, President and CEO of Secura Bio.

On January 9, 2023 Bantam Pharmaceutical, LLC reported that it is presenting at the Biotech Showcase taking place in San Francisco, the 9th-11th of January and virtually, on demand the 18th and 19th of January (Press release, Bantam Pharmaceutical, JAN 9, 2023, View Source [SID1234626125]).

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Bantam Pharmaceutical is developing first-in-class therapeutics that leverage the "cellular power plants," known as mitochondria, to destroy cancer cells.

Bantam has a novel, oral therapeutic, BTM-3566, with an FDA-cleared IND featuring a phase 1 study in B cell lymphomas. BTM-3566 targets mitochondrial homeostasis, with exceptional single-agent activity in hematological malignancies, including complete responses in diffuse large B-Cell lymphoma (DLBCL) in vivo models. These models were derived from patient tumors with multiple genomic alterations that underlie the high death rates in these difficult-to-treat lymphomas.

The presentation by Bantam Pharmaceutical President and CEO Michael Stocum will cover BTM-3566 novel anti-cancer mechanism and the company’s potential to bring novel cancer agents closer to patients in need. Bantam features a leadership team of industry veterans, with most having over two decades of experience across several therapeutic areas in pharmaceuticals, biologics, and molecular diagnostics.

On January 9, 2023 Bio-Techne Corporation (NASDAQ: TECH) reported the expansion of its Advanced Cell Diagnostics (ACD)-branded RNAscope in situ hybridization (ISH) portfolio with the release of the RNAscope Plus smRNA-RNA detection assay (Press release, Bio-Techne, JAN 9, 2023, View Source [SID1234626124]). The RNAscope Plus smRNA-RNA assay enables the simultaneous fluorescent detection of a small regulatory RNA together with 3 target RNAs or RNA biomarkers in the same tissue section at single-cell and subcellular resolution. Designed to be easily scalable to support large studies, the assay will be offered for use on the Leica Bond Rx platform and as a manual assay.

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Small regulatory RNAs, including miRNAs, are instrumental in regulating human health and have been associated with a wide range of different diseases. Oligonucleotide therapies, including antisense oligonucleotides (ASOs) and RNA interference (RNAi), represent a unique class of gene therapies that have the potential to provide highly focused, long-lasting therapeutic effects by utilizing similar cellular processes. These therapeutic nucleic acids can be used to regulate the expression and function of specific target genes, enabling the development of tailored therapies for inherited or acquired diseases that have been previously resistant to standard therapies.

Advanced Cell Diagnostics miRNAscope ISH technology has become the gold standard for identifying the cellular localization of endogenous miRNAs and measuring delivery efficiency of therapeutic oligonucleotides in preclinical animal model systems. Combining proprietary RNAscope and miRNAscope ISH technologies, the RNAscope Plus smRNA-RNA detection assay provides researchers with a valuable new tool to quantify changes in gene expression and cellular function in response to the introduction of regulatory RNAs, essential for optimization of therapeutic efficacy and safety.

"We are excited to further expand our unique offering for the gene therapy community with the launch of the RNAscope Plus smRNA-RNA detection assay. We received an overwhelmingly positive customer response to this novel assay following introduction by our Professional Assay Services and are thrilled to provide broader access to accelerate the groundbreaking work of our customers," said Kim Kelderman, President of Bio-Techne’s Diagnostics and Genomics Segment.

On January 9, 2023 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company reported its expected milestones and strategic priorities for 2023 (Press release, Alaunos Therapeutics, JAN 9, 2023, View Source [SID1234626122]).

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"Achieving the first-in-human objective clinical response in a patient with a solid tumor using a non-viral TCR-T cell therapy made for an exciting 2022. We believe we are well positioned to increase the pace of enrollment in 2023 and with the addition of two new TCRs to our library we have doubled the potential addressable market of our therapy," commented Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. "In December we treated our third patient, a pancreatic patient, with a KRAS-G12V mutation. The year ahead will focus on increasing patient enrollment with an aim towards advancing the program to Phase 2 readiness. We are excited about our progress to date and as leaders in the TCR-T cell therapy space we look forward to bringing the promise of our therapies to even more patients in need."

Anticipated 2023 Milestones and Strategic Priorities

Expand TCR library using hunTR TCR discovery platform to increases addressable market: In the fourth quarter of 2022, the Company submitted an IND amendment to the U.S. Food and Drug Administration (FDA) adding two new TCRs to its clinical trial targeting frequent mutations and HLAs, with the potential to double the addressable market of its TCR-T program. The addition of these new TCRs highlights our strategy to add both more HLAs to existing mutations (KRAS-G12V and HLA-DRB1*07:01) and new mutations within our targeted gene families (TP53-R273C and HLA-DPB1*04:02). In 2023, the Company expects to further expand its library with exclusively owned TCRs targeting recurrent hotspot mutations in KRAS, TP53 and EGFR.

Advance TCR-T library program to Phase 2 readiness: The Company continues to actively enroll patients in its TCR-T Library Phase 1/2 trial targeting KRAS, TP53, and EGFR hotspot mutations across six solid tumor indications. In September 2022, the Company announced the first objective clinical response from a TCR-T cell therapy using non-viral Sleeping Beauty targeting solid tumors. The Company successfully dosed its third patient in the trial in December 2022 and expects to enroll multiple patients in the first half of 2023. Alaunos anticipates providing an interim data update by mid-2023 as it works towards advancing the program into Phase 2.

Optimize manufacturing process towards commercial scalability: The Company continues to execute on its multi-pronged strategy to expand manufacturing capacity and efficiency. The Company doubled its manufacturing capacity in 2022 allowing for production of two products simultaneously. The Company also filed an IND amendment to move from fresh to cryopreserved product and expects to begin implementing this change in the first half of 2023. The use of cryopreserved cell products will reduce manufacturing process time from 30 days to 26 days, a 13% decrease, while increasing flexibility for patient scheduling and treatment. The Company has ongoing initiatives to optimize the process and further reduce the manufacturing time.

Explore next generation TCR-T cell therapy approaches to deepen clinical responses: The Company is advancing its mbIL-15 TCR-T cell therapy program towards an IND filing anticipated in the second half of 2023. The Company believes mbIL-15 has the potential to increase the survival of TCR-T cells in the harsh tumor microenvironment and deepen clinical responses. In addition, Alaunos continues to conduct translational assessments of treated patients to guide next generation TCR-T therapy approaches including potential combination and multiplexed TCR-T cell therapies.

Cash Position and Financial Guidance

Alaunos ended the fourth quarter of 2022 with unaudited cash and cash equivalents of approximately $39.1 million and restricted cash of approximately $13.9 million. Based on current operating plans, the Company expects its operating cash flow for 2023 to be between approximately $35 million and $40 million. The Company expects to have sufficient cash resources to fund research and development programs and operations into Q4 2023.

On January 9, 2023 Geron corporation presented its corporate presentation (Presentation, Geron, JAN 9, 2023, View Source(Jan-2023).pdf [SID1234626123]).

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