On June 24, 2024 SOPHiA GENETICS (Nasdaq: SOPH), a cloud-native healthcare technology company and a global leader in data-driven medicine, reported its new Residual Acute Myeloid (RAM) Application (Press release, Sophia Genetics, JUN 24, 2024, View Source [SID1234644519]). The new offering expands the company’s comprehensive oncology portfolio to support measurable residual disease (MRD) capabilities and will be available to customers worldwide this summer.

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Acute Myeloid Leukemia (AML) represents about one percent of all cancers worldwide, yet is one of the most common forms of leukemia in adults1. Over 50 percent of AML patients relapse within 3 years after achieving complete remission2, therefore post-treatment monitoring is imperative for AML patients, particularly within the first two years, to help quickly detect any signs of relapse3. MRD solutions can help inform post-remission therapy and identify early relapse, and serve as a primary endpoint in clinical trials, helping researchers detect even the smallest trace of cancer and support better patient outcomes.

"AML unfortunately still remains an area of high unmet medical need today, with associated suboptimal patient outcomes. MRD measurement and monitoring has a critical role to play, for example by enabling research into the most optimal sequencing of therapies," said Philippe Menu, M.D., PhD., Chief Medical Officer and Chief Product Officer, SOPHiA GENETICS. "We are proud to contribute to the fight against AML through our SOPHiA DDM RAM Solution. In particular we feel that the capability to seamlessly track longitudinally the evolution of individual mutations over time through a dedicated add-on module of our SOPHiA DDM Platform has the potential to be a game-changer for clinical researchers."

Next-generation sequencing (NGS)-based MRD testing is among the most advanced in cancer screening and monitoring, and can be found only with highly sensitive methods. The SOPHiA DDM RAM Solution provides users with the confidence that MRD will detect even one cancer cell among 10,000 cells. This application will allow users to stay ahead of disease response with the analytical capabilities of the SOPHiA DDM Platform, enabling sensitive variant detection down to 0.01% VAF and covering guideline-recommended genes to deliver robust insights for residual acute myeloid.

Customers using the SOPHiA DDM RAM Solution will have access to longitudinal variant monitoring, allowing them to visualize the mutational landscape for each patient and its evolution over time. The solution also provides users with the most up-to-date databases and customizable reporting features to generate graphical representations and comprehensive MRD reports.

Additionally, the SOPHiA DDM RAM Solution will continually hone its machine learning algorithms to provide the most accurate MRD results in just four days.

Representatives from SOPHiA GENETICS are available at AMP (Association for Molecular Pathology) Europe June 24-27 to discuss AML monitoring with this new application.

For more information on SOPHiA GENETICS, visit SOPHiAGENETICS.com and connect on LinkedIn.

On June 24, 2024 IN8bio, Inc. (Nasdaq: INAB), a leading clinical-stage biopharmaceutical company focused on innovative gamma-delta T cell therapies, reported that William Ho, CEO and Co-founder, will participate in a fireside chat during the H.C. Wainwright 2nd Annual Immune Cell Engager Virtual Conference on Tuesday, June 25, 2024 at 2:30 p.m. ET (Press release, In8bio, JUN 24, 2024, View Source [SID1234644504]).

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A live webcast and replay will be available under "Events and Presentations" in the News & Presentations section of the IN8bio website at View Source

On June 24, 2024 Sustained therapeutics reported that the first patient has entered the Phase II/III trial of ST-02, innovative cancer medication targeting Upper Tract Urethral Carcinoma (UTUC) (Press release, Sustained Therapeutics, JUN 24, 2024, View Source [SID1234644520]).

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A rare form of cancer, UTUC affects over 7,000 individuals annually across North America. UTUC is a cancer of the inside lining of the kidney (the "collecting system" – where urine starts to drain after it is made by the kidney) and the ureter (the tube that carries urine from the kidney to the bladder). Its location in the upper collecting system makes it challenging to access and completely remove with traditional laser resection.

"This is a new opportunity in the treatment of UTUC," says Dr. Peter Black, lead investigator of the clinical trial and a Senior Research Scientist with the Vancouver Prostate Centre. "Currently we have limited treatment options for this uncommon and challenging cancer, and this approach may allow better disease control without having to remove a kidney."

The first patient in the clinical trial has been enrolled at the Vancouver Prostate Centre, and it is anticipated that 5 other sites will take part in the study. The company plans on enrolling approximately 75 patients in the combined Phase II/III trial and believes that ST-02 may be designated an orphan drug. Orphan drug designation can qualify the company for incentives such as tax credits, exemption from user fees, and seven years of market exclusivity after approval.

Sustained Therapeutics’ ST-02 product for UTUC is a sustained release formulation that enables direct delivery of chemotherapy to the site of the carcinoma via a catheter. Using the company’s proprietary platform technology, the medication releases slowly over about 24 hours exactly where the cancer is located, coating the tissue and delivering chemotherapy at higher concentrations and for longer than the current standard of care. The company believes that this direct sustained delivery may be more effective and result in fewer side effects than other forms of chemotherapy that are a current standard of care.

"ST-02 is Sustained Therapeutic’s second product to reach Phase II trials, and demonstrates our potential to develop multiple products using our novel drug delivery platforms. As such, it is a very important development in our portfolio," says Dr. Martin Gleave, Founder and Chief Medical Office of Sustained Therapeutics and Distinguished Professor in the UBC Department of Urologic Sciences and Director of the Vancouver Prostate Centre (VPC). "This new therapeutic, designed specifically for UTUC, could address an important unmet need for patients. It’s an exciting time to be a part of this work."

On June 24, 2024 Nerviano Medical Sciences S.r.l. ("NMS" and "the Company"), a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc., a wholly owned subsidiary of NMS Group, focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy, reported that the United States Food and Drug Administration (FDA) has cleared the protocol for investigational new drug (IND) application for NMS-812, a first-in-class orally bioavailable and highly potent small molecule dual inhibitor of PERK/GCN2 (Press release, Nerviano Medical Sciences, JUN 24, 2024, https://www.nervianoms.com/nerviano-medical-sciences-announces-fda-protocol-clearance-of-new-ind-application-for-nms-812-a-first-in-class-dual-inhibitor-of-perk-gcn2-brian-sherer-phd-appointed-to-lead-and-accelerate-the-dev/ [SID1234644505]).

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NMS-812 has very strong scientific rational in the multiple myeloma indication by modulating the unfolded protein response via PERK in the integrated stress response (ISR) pathway since this is a high protein production setting. In addition, NMS-812 inhibits GCN2, another ISR component, affecting mainly amino acid deprivation stress, to augment cell death. Together the double inhibition likely overcomes resistance. The first in human (FIH) study showed excellent pharmacokinetic profile allowing daily oral dosing and likely permissive safety for further development. The multiple myeloma indication was deprioritized because it would require significant resources due to multiple treatment lines, but NMS is fully committed to continuing in partnership with other companies interested in the multiple myeloma space.

NMS-812’s ability to inhibit two key components, PERK and GCN2, of the ISR, may offer superiorapoptosis in other cancer settings such as AML. In addition, NMS-812 modulates the immune response via direct and indirect mechanisms which may contribute to anti-cancer activity.

Acute Myeloid Leukemia (AML) is an aggressive hematological malignancy. According to American Cancer Society, in 2024, an estimated 20,800 new cases of leukemia will be diagnosed in the US and 11,220 people will die from the disease.

NMS expects to initiate enrollment of patients with relapsed/refractory AML including patients with TP53 mutations in the Phase I PERKA-812-003 trial in the second half of 2024.

NMS today also announces the discontinuation of the FIH clinical trial with NMS-812 in the setting of relapsed/refractory multiple myeloma (NCT05027594) due to strategic reasons.

"Acute Myeloid Leukemia (AML) remains an aggressive hematological malignancy with tremendous unmet medical need especially in the p53 mutant patient population. Based on preclinical data and unique dual Integrated Stress Response mechanism, NMS-812 may represent a novel strategy for AML through the unfolded protein response and amino acid deprivation stressors , with potential for synergies with other drugs and potential to overcome drug resistance." said Lisa Mahnke, MD, Ph.D., Chief Medical Officer at NMS. "We are thrilled to have Brian to join the NMS team to lead the development."

Brian has had leadership roles with Exelixis, EMD Serono, and AstraZeneca over a 25 year career. He has been instrumental in the discovery and early development of more than 10 small molecule clinical candidates.

In addition to PERK/GCN2, Brian will also lead NMS’ MPS1 asset, leveraging his extensive expertise.

"Both PERK/GCN2 and MPS1 assets hold potential as first-in-class drugs for subsets of cancers with high unmet medical needs. Our teams have made remarkable progress in developing these novel assets, and we are excited about the future under Brian’s leadership." said Lisa Mahnke.

On June 24, 2024 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that it has treated the sixth patient in the ongoing Phase 1 clinical trial of its novel chimeric antigen receptor T-cell (CAR-T) therapy for ovarian cancer (Press release, Anixa Biosciences, JUN 24, 2024, View Source [SID1234644521]). This patient is expected to be the last patient in the second dosage cohort. If there continue to be no adverse effects experienced by the second cohort participants, enrollment of the third dosage cohort may commence within the next month. The study is being conducted through a research partnership with Moffitt Cancer Center.

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Anixa’s FSHR-mediated CAR-T technology, also known as chimeric endocrine receptor T-cell (CER-T), differs from traditional CAR-T therapy by targeting the follicle-stimulating hormone receptor (FSHR), which research indicates is exclusively expressed on ovarian cells, including the vasculature of tumors. The first-in-human trial (NCT05316129) is enrolling female adult patients with recurrent/progressing ovarian cancer who have progressed on at least two prior therapies. The study is designed to evaluate safety and identify the maximum tolerated dose, while monitoring efficacy.

Safety was previously confirmed in the first three-patient cohort. The fourth through sixth patients, enrolled in the second cohort of the Phase 1 clinical trial, received triple the dose of CAR-T cells compared with the dose of the first cohort, with no dose-limiting toxicities observed.

Following the requisite wait time, of one month after dosing, enabling confirmation that it is safe to escalate, the trial will immediately begin enrolling patients in the third dose cohort, which will be at a ten times higher dose than the initial dosage.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, stated, "We are highly encouraged by the favorable safety profile observed thus far in both the first and second patient cohorts, and are eager to evaluate a higher dose in the next cohort. We are particularly encouraged by a notable response in one of the patients in the first cohort, even though the dosage was considered a subtherapeutic level. Our aim is to maintain a positive safety profile as we escalate dosing, with the goal of demonstrating additional objective evidence of efficacy. Unlike conventional CAR-T cell therapies, which have achieved amazing results in various hematological cancers, they have not been effective in solid tumors. In contrast, we believe Anixa’s novel technology has the potential to make CAR-T effective in ovarian cancer and perhaps across multiple solid tumor types. Our unique and highly targeted CER-T approach targets the FSHR, which is exclusively expressed on ovarian cells. A potential dual mechanism of action is operating with our therapy targeting tumor vasculature by starving or shrinking the tumor from the inside out, as well as direct targeting of ovarian cells."

Dr. Kumar continued, "We believe that intraperitoneal (IP) delivery may also be a significant advantage of our therapy, as it allows direct trafficking of the CAR-T cells to the tumor sites, which helps to minimize side effects such as cytokine release syndrome (CRS). We believe this method of delivery not only enhances the targeting of the tumor but also improves the overall safety profile of the treatment. We expect that IP delivery may enable us to use dosages that are much higher than possible with intravenous delivery."