On December 13, 2022 CoImmune, Inc., a clinical stage immuno-oncology company working to redefine cancer treatment using best-in-class cellular immunotherapies, reported the presentation of results from a Phase I/II clinical trial evaluating CARCIK-CD19, an investigational treatment based on the company’s chimeric antigen receptor (CAR) modified cytokine induced killer (CIK) cell platform, in pediatric and adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who had relapsed following hematopoietic stem cell transplantation (HSCT) (Press release, CoImmune, DEC 13, 2022, View Source [SID1234625229]). The results are being presented at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s (ASH) (Free ASH Whitepaper) 64th Annual Meeting and Exposition held December 10-13 in New Orleans.

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"For more than 15 years, we have been working to advance innovative pre-clinical and clinical research in the field of cellular and gene therapy for childhood and adult acute lymphoblastic leukemia, and these data presented at the ASH (Free ASH Whitepaper) annual meeting represent a promising breakthrough," said Andrea Biondi, M.D., Professor of Pediatrics at the University of Milano-Bicocca and Scientific Director of the M. Tettamanti Research Center. "In the Phase I/II dose-finding study, CARCIK-CD19 cells demonstrated an excellent safety profile with anti-leukemia activity in heavily pretreated patients with B-ALL relapsed after HSCT. Importantly, the persistence of these cells was associated with a longer duration of response and reduced risk of leukemia relapse."

The Phase I/II dose escalation clinical trial included 27 patients (4 children and 23 adults) with B-ALL relapsed post-HSCT, six of whom participated under a compassionate use protocol and received the highest dose level. The study was conducted as a collaboration between the Pediatric and Haematology Departments of Monza and Bergamo (Italy), respectively. Results show that 18 out of 27 patients (66.7%, 95%CI=46.0-83.5%) achieved complete response (CR), with 14 (77.8%) of the overall responders negative for minimal residual disease (MRD). In the two highest dose groups, 16 out of 21 patients (76.2%, 95%CI=52.8-91.8%) achieved CR, with 13 of the responders at the highest dose (81.3%) MRD-negative. The median follow-up was 2.8 years (range, 0.05-4.4 years, last update as of July 2022). Event-free survival (EFS) was significantly longer in the two highest dose groups (p=0.0265) and was associated with the percentage of bone marrow blasts after lymphodepletion (≥ 5% vs <5%) (p=0.0245). Graft versus host disease never occurred after treatment with CARCIK-CD19, and no dose limiting toxicity was observed.

In a prospective study also presented at the ASH (Free ASH Whitepaper) Annual Meeting, Giuseppe Gaipa, Ph.D., and Benedetta Rambaldi, M.D., Ph.D., evaluated the impact on clinical outcome of proliferation, differentiation, and expansion of CARCIK-CD19 cells in 20 of the patients treated in Phase I/II clinical trial. The cells were detectable up to 12 months in vivo, a majority of which presented with CD8+ memory phenotype. Their persistence after day 90 was correlated with duration of response (p=0.0045), and MRD-negativity was associated with longer survival (p=0.004).

CoImmune is also announcing the presentation two additional preclinical studies at the ASH (Free ASH Whitepaper) Annual Meeting that evaluated the CAR-CIK strategy in acute myeloid leukemia (AML). Both were performed at the M. Tettamanti Research Center, Monza, Italy.

In a poster titled, "Tuned IL3-Zetakine Coupled to a CD33 Costimulatory Receptor As a Dual CAR for Safer and Selective Targeting of Acute Myeloid Leukemia," Sarah Tettamanti, Ph.D., and colleagues describe a bispecific CAR-CIK strategy for AML that demonstrated the potential for improved efficacy and high specificity for CD123 and CD33 on leukemic cells while reducing the risk of life-threatening toxicities.

In a poster titled, "Overexpression of CXCR4 Enhances the Efficacy of CAR-T Therapy for Acute Myeloid Leukemia," Marta Serafini, Ph.D., and colleagues found that combining the expression of CD33-CAR and CXCR4 improves in vivo CAR-CIK cell migration and retention within the bone marrow and subsequent AML eradication. In an AML mouse model, median survival improved with CXCR4 overexpression (p<0.0001).
"Immunotherapies have the potential to allow the body to attack cancer cells with higher efficacy and improved safety when compared to traditional approaches to cancer treatment, but researchers have yet to advance the development of CAR-T cells to reach this full potential," said Charles Nicolette, Ph.D., Chief Executive Officer of CoImmune. "We are focused on advancing the science of immunotherapy with fundamentally new approaches as the only company developing gene-modified CAR-CIK cells. These new data demonstrate the potential for CAR-CIK cells to improve outcomes for B-ALL patients, while also opening the door to promising strategies that may increase the therapeutic potential in AML and solid tumor indications."

On December 13, 2022 PreciseDx, a leading AI company leveraging the analysis of morphology features from histology slides to provide more precise patient-specific risk information, reported a sponsored research collaboration with the Breast Unit of the Champalimaud Clinical Center (CCC)/Champalimaud Foundation in Lisbon, Portugal (Press release, Champalimaud Foundation, DEC 13, 2022, View Source [SID1234625228]).

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As part of the research collaboration, PreciseDx and the CCC will evaluate the performance of the PreciseDx Breast Assay in predicting breast cancer recurrence and treatment choice. The objective is to obtain clinical data and Hematoxylin and Eosin (H&E) images from a cohort of patients treated at the CCC’s Breast Unit since its inception in 2011. Gaining access to these patient samples and outcomes data will allow for the development of models that are comparative to those previously produced by PreciseDx in the United States.

"The relationship with the Champalimaud Foundation Breast Unit under the leadership of Dr. Fatima Cardoso represents a unique opportunity for PreciseDx to introduce our AI breast cancer platform into the European oncology community," said Michael Donovan, MD, PhD, Co-Founder and Chief Medical Officer. "The ability to work alongside Champalimaud and Dr. Cardoso allows us to extend our understanding of recurrent and metastatic disease for breast cancer patients globally."

Breast cancer continues to remain the most lethal malignancy in women across the world. The European Cancer Information System (ECIS) estimated that there were more than 350,000 new breast cancer cases diagnosed in the European Union 27 countries (EU-27) in 2020 as well as over 90,000 deaths due to the disease. It was also reported that 1 in 7 women in the EU-27 have a lifetime risk of developing breast cancer. Consequently, breast cancer grading plays a critical role in patient management despite the considerable inter- and intra-observer variability, highlighting the vital need for decision support tools. These tools are key to improving reproducibility and prognostic accuracy in clinical practice.

"This research collaboration is exciting because it has the potential to advance our current practice of interrogating invasive breast cancer at the tissue level, providing insights into the underlying disease biology and hopefully improve our ability to determine accurate prognosis for the individual patient," said Dr. Fatima Cardoso, Director of the Breast Unit of the CCC.

PreciseDx and the CCC’s Breast Unit aim to facilitate the continued research, development and advancement of models and phenotyping related to early-stage invasive breast cancer (IBC). The organizations seek to improve recurrence score risk prediction in breast cancer through the incorporation of AI grading.

The Champalimaud Foundation conducts research in cutting-edge areas and has a priority to stimulate discoveries that promote the health and well-being of humanity. It also continually develops new knowledge standards by adopting translational methodology, establishing a direct link and interdependence between research and clinical activity.

On December 13, 2022 Cyclica Inc. ("Cyclica"), a neo-biotech that is unlocking the protein universe to discover the medicines of tomorrow, and SK Chemicals Co., Ltd. ("SK Chemicals"), a global leader in total healthcare providing diagnostic, therapeutic and prophylactic solutions, reported an AI-driven drug discovery and development partnership to develop therapies across a range of disease areas (Press release, Cyclica, DEC 13, 2022, View Source [SID1234625227]).

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Under the collaboration with SK Chemicals, Cyclica will deploy its robust, proprietary drug discovery platforms to identify novel drug candidates for challenging biological targets across therapeutic areas of mutual interest to Cyclica and SK Chemicals. SK Chemicals will be responsible for the preclinical and clinical development and worldwide commercialization of these pharmaceuticals.

"This partnership is a tremendous opportunity to create innovative drug therapies across a number of therapeutic areas. Having Cyclica and SK Chemicals join forces will provide both companies with a competitive advantage, allowing for major advances in understanding disease mechanisms and the potential development of new therapeutics for patients”, said Naheed Kurji, Co-Founder, President and CEO of Cyclica.

"We believe Cyclica’s technology will help SK Chemicals to develop novel therapeutic candidates against difficult biological targets with little information available. We seek to create synergy arising by combining SK Chemicals’ organizational excellence and development experience with Cyclica’s expertise and know-how," said Yun Ho Kim, Head of the Pharma Business Division at SK Chemicals.

"We are thrilled to collaborate with SK Chemicals. This partnership will leverage our respective strengths in creating new therapeutics for patients and while promoting human health," added Dr. Vern De Biasi, PhD, Chief Partnership Officer at Cyclica.

On December 13, 2022 Tactical Therapeutics, Inc. (TTI), a clinical stage biopharma, reported that it has been selected to present at the MedInvest Oncology Investor Conference taking place December 14-15, 2022, in New York, NY (Press release, Tactical Therapeutics, DEC 13, 2022, View Source [SID1234625226]).

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"We are honored to be selected to present at this unique event," said Rashida Karmali, Ph.D., CEO of Tactical Therapeutics. "We welcome the chance to interact with many of the world’s most accomplished leaders and investors who are all dedicated to improving the health and lives of patients with glioblastoma (GBM) and difficult to treat solid cancers."

About Glioblastoma, recurrent (rGBM) and newly diagnosed (nGBM)
Glioblastoma, the brain tumor that killed Senator Ted Kennedy, Beau Biden and Senator John McCain, is still mostly untreatable. Recent reports suggest veterans of Vietnam, Iraq and Afghanistan wars who were exposed to burn pits later developed GBM. GBM exhibits debilitating neurological symptoms and suicide rates for GBM patients are higher compared to those with other cancers.

Since 2005, GBM, an orphan disease (approximately 18,000/yr), has a median overall survival (OS) of between 12 to 20 months in newly diagnosed GBM with surgery, radiotherapy (with temozolomide) and adjuvant temozolomide, plus tumor treating fields. rGBM recurs frequently within a year with a median OS of 10 months.

TTI’s oral, safe and brain-penetrating drug, Carboxyamidotriazole Orotate (CTO), showed efficacy with significantly improved OS from 10 months to 28 months+ in nGBM in Phase IB trial, with published results (J. Clin Oncology, (2018)36:1702-1709).

Dr. Antonio Omuro, who oversaw the CTO Phase I clinical trial, had this to say about the promising treatment:

"In 27 Recurrent GBM patients, 1 complete and 6 partial responses were observed in patients who had been in several previous trials, including patients previously exposed to bevacizumab, who are the most challenging patients to treat. Radiographic responses are rarely seen in glioblastoma trials, therefore these are really promising results. Excellent survival was observed in 15 newly diagnosed GBM (13/15), with median overall survival not reached after 28 months; 2-year survival 62%; 1-year survival 93%. Two patients remain recurrence free at 5 years.

"The safety profile was excellent, and we were also able to demonstrate that CTO crosses the blood brain barrier and can achieve high concentrations in brain tumors. Overall, the Phase IB trials of CTO in GBM patients showed highly promising efficacy and safety in patients with very advanced disease."

About Carboxyamidotriazole Orotate (CTO) & Intellectual Property (IP)
CTO is a patented first-in-class inhibitor of genes of non-voltage dependent calcium signaling associated with multiple oncolytic pathways and calcium channels. At clinically relevant CTO levels, differential expression of some mRNA showed inhibition of transcriptional signatures of several oncogenes, and in contrast activation of tumor suppressors.

The IP portfolio includes 8 U.S. and 75 International patents lasting to 2033.

On December 13, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that the U.S. Food and Drug Administration (FDA) has provided a safe to proceed letter and cleared the IND application for VIP236, the Company’s front-runner SMDC for the treatment of advanced solid tumors (Press release, Vincerx Pharma, DEC 13, 2022, View Source [SID1234625225]).

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VIP236 is a first-in-class SMDC with a tailored design to efficiently treat patients with cancer with aggressive and metastatic disease. VIP236 binds to activated αVβ3 integrin allowing specific homing to the tumor and is efficiently cleaved by neutrophil elastase (NE). Both proteins are present in the tumor microenvironment (TME), are highly expressed in advanced metastatic tumors, and are associated with poor prognosis in patients with cancer. Anticancer activity occurs after a specific and targeted release of an optimized camptothecin (CPT) payload by NE in the TME. The CPT payload of VIP236 is optimized for high permeability with low active efflux potential to overcome transporter-mediated resistance observed with SN38, the active metabolite of irinotecan.

"We are excited to advance our lead SMDC, VIP236, to the clinic," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "Preclinical results provide validation of our targeting mechanism and demonstrate how VIP236 can deliver up to 40 times more drug to the cancer than the surrounding tissues or normal organs. This is evident in the durable tumor regressions and significant reduction of metastases in patient-derived xenograft (PDX) cancer models, including PDX models of triple negative breast cancer, renal cell carcinoma and colorectal cancer."

Dr. Hamdy continued, "We look forward to starting our first-in-human dose- escalation study early next year to evaluate the maximum tolerated dose, safety and tolerability in patients with advanced or metastatic solid tumors. Bringing VIP236 to the clinic while continuing to be strategic about our resources remains one of our top priorities. We will be pushing the VIP236 program forward into Phase 1 with our existing capital and continue to expect our cash runway to lead us into late 2024."