On December 8, 2021 Shasqi, a clinical-stage biotechnology company developing precision activated oncology therapeutics with its proprietary Click Activated Protodrugs Against Cancer (CAPAC) Platform, reported that it has been awarded a second ‘Direct to Phase 2’ Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) (Press release, Shasqi, DEC 8, 2021, View Source [SID1234596618]). The $1.9 million grant will support a Phase 1b dose-expansion cohort in the company’s ongoing development program for its click-activated prodrug therapy, SQ3370. The safety and efficacy data from this cohort will support dose selection for the planned Phase 2 study of SQ3370 in patients with advanced soft tissue sarcoma.

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"The NCI has made two investments in our Phase 1 clinical studies of SQ3370, which will enable us to accelerate the development of SQ3370 and gather additional efficacy and safety data to inform our Phase 2 clinical trial design. We are grateful for the vision of the NCI and their support of SQ3370 and our click chemistry platform," said José M. Mejía Oneto, M.D., Ph.D., Founder and CEO of Shasqi. "The early data from our Phase 1 study of SQ3370 provides initial validation for our platform in humans, with an encouraging safety profile and initial hints of clinical activity. We believe we can improve the efficacy, safety, and biological effects of many existing cancer therapies and various modalities which are currently either too toxic to be used widely, have significant safety burden, or have limited efficacy due to a limited therapeutic window."

About CAPAC and SQ3370:

SQ3370 is the first click chemistry-based treatment to be tested in humans, and utilizes Shasqi’s proprietary CAPAC platform, an approach that activates cancer drugs at a tumor with decreased systemic toxicity. Shasqi is validating its platform with SQ3370, which is designed to activate a powerful chemotherapeutic, doxorubicin, at the site of the tumor. The investigational product is based on the chemical reaction between a drug protected through a trans-cyclooctene modification (a protodrug) and a tetrazine-modified biopolymer. The biopolymer is injected into the target tumor lesion, where it precisely activates an intravenously infused protodrug. Shasqi believes its click-chemistry approach can improve the efficacy and safety of many existing drugs and various modalities that have a limited therapeutic window.

On December 8, 2021 Cytovia Therapeutics, Inc., a biopharmaceutical company developing allogeneic "off-the-shelf" gene-edited iNK cells (NK cells derived from induced pluripotent stem cells, or iPSCs), CAR (Chimeric Antigen Receptor)-iNK cells, and Flex-NK cell engager multifunctional antibodies, reported that its scientific leadership will present at the following upcoming conferences (Press release, Cytovia Therapeutics, DEC 8, 2021, View Source [SID1234596617]):

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"We are proud of the scientific thought leadership of our colleagues Drs. Frankel, Li, and Kadouche, who will present Cytovia’s latest advances"

iPSC-Derived Cell Therapies Summit (Virtual Event)

Wednesday, December 8th, 2021, 9.30 AM
Demonstrating Safety & Efficacy of iPSC-derived Cells to Maximize Clinical Translation
(Industry Leaders’ Fireside Chat: Paving the Way For the Future of Cell Therapy with iPSCs)
Featuring Cytovia Therapeutics CMO Stanley Frankel

Wednesday, December 8th, 2021, 3PM
Advancing Clinical Trial Strategy for Successful Clinical Outcomes
(Developing iPSC-Derived NK Cell Therapies Against Solid Tumors)
Featuring Cytovia Therapeutics CSO Wei Li

Antibody Engineering & Therapeutics Conference (San Diego, California)

December 12th-15th, 2021
Dr. Jean Kadouche, Cytovia Therapeutics scientific co-founder and Global Head of Antibody R&D, will present the "Design, Manufacturing, and Activity of FLEX Immune Cell Engager Multi-functional Antibodies" poster on behalf of the Cytovia team.

"We are proud of the scientific thought leadership of our colleagues Drs. Frankel, Li, and Kadouche, who will present Cytovia’s latest advances," said Dr. Daniel Teper, CEO of Cytovia Therapeutics. "Cytovia is committed to translating disruptive innovation in cell therapy to create therapeutics that advance towards a cancer cure. Cytovia is the first biotech company to develop its own best-in-class gene-edited, iPSC-derived NK cells and NK Engager antibodies, with the potential to combine them for optimal clinical outcomes in both hematological and solid tumors."

On December 8, 2021 Amphera B.V., a late-stage biotechnology company developing MesoPher cell therapy to treat cancer, reported that significant progress in its two clinical programs in pancreatic cancer as well as the full recruitment of all patients in its pivotal Phase II/III study in mesothelioma (Press release, Amphera, DEC 8, 2021, View Source [SID1234596616]).

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Based on a preliminary efficacy analysis of the first cohort of the REACtiVe study in resected pancreatic cancer patients, an expansion cohort has been completed at a remarkable pace with results expected in H2 2022. In total 26 patients are now included in the study. Safety and efficacy data from the first cohort of the REACtiVe study in resected pancreatic cancer patients treated with MesoPher have been submitted for publication by Erasmus MC, Rotterdam, the Netherlands.

In addition a study in metastatic pancreatic cancer, REACtiVe-2, has started recruiting patients. This study will assess the safety and efficacy of a combination therapy of MesoPher and mitazalimab (a CD40 agonist from Alligator Bioscience). This follows the strong preclinical data published in the Journal for ImmunoTherapy of Cancer, which established the potency of the combination (available here).

Finally, the company announces that the Phase II/III pivotal study DENIM in mesothelioma patients is fully recruited and confirms the readout to be expected in Q4 2022.

Rob Meijer, CEO of Amphera said: "Over the last year Amphera has made exceptional progress with its clinical development programmes based on its MesoPher cell therapy platform. Despite the challenging pandemic environment, our trials saw strong enrolment, underlying the high medical need for patients. The pivotal Phase II/III DENIM study in pleural mesothelioma has now been fully recruited with the read-out expected in Q4 2022 and we are strongly encouraged by the rapid enrolment of the expansion cohort of the Phase II REACtiVe study in pancreatic cancer. In addition to the studies in pancreatic cancer and pleural mesothelioma, efficacy of MesoPher is investigated in abdominal mesothelioma and pleural mesothelioma in combination with surgical resection."

Prof. Joachim Aerts MD PhD, Medical Advisor to Amphera said: "Efficacy was first proven in mesothelioma and for me, as a clinician and a scientist, it is exciting to see that the previous efficacy found, translates into relevant clinical results for patients in other indications, especially one with a dire prognosis such as pancreatic cancer. We have also recently published highly compelling long-term survival data in Vaccines from three studies in mesothelioma patients (available here). In the three studies combined, the median overall survival was 27 months and the overall survival at 5 years was 21%. The expanding data set is showing the real potential of MesoPher in a wide range of oncology indications."

On December 8, 2021 Foundation Medicine, Inc. reported that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneCDx to be used as a companion diagnostic for two groups of current and future FDA-approved therapeutics in melanoma, which includes BRAF inhibitor monotherapies targeting BRAFV600E and BRAF/MEK inhibitor combination therapies targeting BRAFV600E or V600K mutations (Press release, Foundation Medicine, DEC 8, 2021, View Source [SID1234596615]). This approval makes FoundationOneCDx the only comprehensive genomic profiling (CGP) test approved as a companion diagnostic across two groups of targeted therapies, representing an important step toward simplifying decision making for oncologists.

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Melanoma is a serious form of skin cancer that accounts for an estimated 207,790 cases each year.1 BRAF mutations are the most common type of mutation in melanoma and are present in more than half of all melanoma cases.2 As a companion diagnostic for therapies targeting BRAFV600E and BRAFV600K mutations in melanoma, FoundationOneCDx offers oncologists flexibility when selecting the right therapy for their melanoma patients and ensures all treatment options are considered within these groups of therapies.

"As the first group therapy approval for any comprehensive genomic profiling test, this milestone reinforces our dedication to pioneering advances that expand the power of genomic testing in cancer care," said Mia Levy, MD, PhD, chief medical officer at Foundation Medicine. "This approval will allow oncologists to uncover all possible FDA-approved treatment options for these indications through just one test, providing more insights for physicians and patients, more efficiently than ever before."

This approval represents an innovative, more efficient regulatory approach that simplifies the companion diagnostic approval process for biopharma companies developing BRAF inhibitor therapeutics, while maintaining rigor and high-quality standards.

The first new therapeutics for which FoundationOneCDx is a companion diagnostic under the group approvals are Pfizer’s BRAFTOVI/MEKTOVI and Novartis Tafinlar (dabrafenib) + Mekinist (trametinib) combination therapeutics. Moving forward FoundationOneCDx will automatically become a companion diagnostic for future BRAF inhibitors that are approved by the FDA under these groups.

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit View Source

On December 8, 2021 Alpha-1 Biologics, a biotherapeutics company developing innovative treatments for cancers and immune deficiencies, reported that positive data was published in frontiers in Oncology demonstrating Alphataxin, a small molecule that elevates circulating and tumor-infiltrating CD4+ T cells, suppressed kidney cancer and suppressed metastasis in mice (Press release, Alpha Biologics, DEC 8, 2021, View Source [SID1234596614]). Orally available Alphataxin, is the first and only drug in development to increase formation of CD4+ helper T cells. Immune checkpoint inhibitor therapy, the vanguard of cancer therapy, promotes the ability of CD8+ T cells to kill tumor cells. However, CD8+ T cells are unable to kill tumor cells in the absence of chemical mediators secreted by CD4+ helper T cells. The data showed that Alphataxin treatment is efficacious as a monotherapy in kidney cancer in mice and enhances anti-PD-1 immune checkpoint inhibitor therapy, with the potential to expand the number of human cancer patients who respond to checkpoint inhibitor therapy.

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"We are pleased with these pre-clinical results, which have further increased our confidence in the promising potential of Alphataxin to treat cancers and immune deficiencies. Based on this positive data, the company plans to raise additional capital to rapidly advance the Alphataxin development program."

Cynthia L. Bristow, PhD, CEO of Alpha-1 Biologics said, "We are excited to announce this positive pre-clinical data, which showed that Alphataxin, orally delivered in combination with an injected immune checkpoint inhibitor, may provide a powerful approach that can produce long-term remission in kidney cancer and other T cell-responsive tumors. Alphataxin as a monotherapy and in combination with anti-PD-1 immune checkpoint inhibitor therapy significantly suppressed tumor growth in a mouse model of kidney cancer and significantly elevated the number of circulating and tumor-infiltrating CD4+ T cells."

In the study, following implantation of mouse kidney tumor cells within the kidney of mice, combination treatment of Alphataxin and anti-PD-1 therapy resulted in 100% elimination of tumor growth. Moreover, in mice implanted with ten times more tumor cells into the kidney, doubling the Alphataxin dose in combination treatment with anti-PD-1 led to 100% elimination of tumors in one-third of mice and 81% suppression of tumor growth in the remaining two-thirds of mice. Both anti-PD-1 and Alphataxin monotherapy showed decreased tumor growth as compared with untreated mice. Lung metastasis was present in monotherapy but eliminated in combination-treated mice.

The study also investigated the effects of Alphataxin on the immune system in healthy mice. The data showed that Alphataxin increased the normally circulating numbers of CD4+ T cells, Pre-T cells, and CD4/CD8 ratio indicating that Alphataxin acts to increase the formation of CD4+ helper T cells.

"This combination treatment of Alphataxin with an anti-PD-1 therapy addresses a high unmet medical need in patients with kidney cancer who have very low survival rates. The 5-year survival rate for patients with renal adenocarcinoma undergoing anti-PD-1 treatment is estimated to be 27.7%. However, despite the efficacy of checkpoint inhibitors in promoting the cytotoxic activities of tumor infiltrating CD8+ T cells, approximately 87% of cancer patients do not respond to immune checkpoint therapy," said Dr. Bristow. "Kidney cancer is often not diagnosed until after metastasis, a disease stage for which there are few effective treatment options; however, recent promising advances demonstrate that easily accessible blood-based tests provide early detection, and this means that Alphataxin has the potential to be transformative in providing long lasting remission in kidney cancer."

Ron Winston, President, Institute for Human Genetics and Biochemistry said, "We are pleased with these pre-clinical results, which have further increased our confidence in the promising potential of Alphataxin to treat cancers and immune deficiencies. Based on this positive data, the company plans to raise additional capital to rapidly advance the Alphataxin development program."

The full article in frontiers in Oncology can be accessed here: Alphataxin, a Small-Molecule Drug That Elevates Tumor-Infiltrating CD4+ T Cells, in Combination With Anti-PD-1 Therapy, Suppresses Murine Renal Cancer and Metastasis.

About Alphataxin

Alpha-1 Biologics discovered that the protein alpha-1 proteinase inhibitor (α1PI, alpha-1 antitrypsin) regulates the number of circulating CD4+ T cells by stimulating the locomotion of Pre-T cells. The orally available small-molecule drug Alphataxin acts as a surrogate for α1PI in this pathway. The Company is focused on the development of Alphataxin, which suppressed tumor growth in a mouse model of kidney cancer. Alphataxin, in combination with anti-PD-1 antibody, significantly elevated circulating and tumor-infiltrating CD4+ T cells. Because orally available Alphataxin is the first and only drug developed to increase CD4+ T cells, Alphataxin is eligible for FDA Breakthrough Designation. In combination with anti-PD-1, Alphataxin is a powerful therapeutic method that provides long-term remission in kidney cancer in mice and is being tested in other T cell-responsive cancer models.