On May 2, 2023 Odimma Therapeutics, a biotechnology company specialized in personalized cancer immunotherapy reported a seed fundraising of €2 million in order to secure the preparation on its First-in-Man clinical trial with ODI-2001, an innovative first-in-class personalized immunotherapy against cancer (Press release, Odimma Therapeutics, MAY 2, 2023, View Source [SID1234630876]).

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This financing round is supported by Capital Grand Est, Alsace Business Angels, Angels Santé, historical founders and associates, several new Business Angels and BpiFrance.

In recent months, Odimma Therapeutics has completed the proof of activity experiments of its lead product ODI-2001 in several hard-to-treat preclinical cancer models. In parallel, the company has secured the pharmaceutical process for the production of ODI-2001. This relies on pharmaceutical partnerships with world-class players in the field as earlier communicated: myNEO (Ghent, Belgium) for neoepitope prediction by artificial intelligence, Touchlight Genetics (Hampton, UK) for the production of synthetic DNA and ABL Europe (Illkirch, France) for the production of the viral component of ODI-2001.

The current activities are focused on the preparation of the Phase I clinical trial in patients with advanced solid tumors. These activities include regulatory interactions, toxicology and clinical operations. R&D activities are strengthened to continue generating additional data and intellectual property.

Jean-Sébastien Schmitt, representative of Alsace Business Angels said: "Fighting cancer with a technological disruption which is personalized, more efficient and quickly available for the patient was very attractive for our members. We are delighted to support Odimma’s team in their ambition and, as a first step, provide them with the best conditions for their clinical trial."

"We are delighted about the success of this new refinancing round, and are very pleased to contribute to the development of this promising technology. We are confident the innovative personalized immunotherapy platform developed by Odimma Therapeutics will give the best chances of success to immunotherapy in oncology," said Patrick Squiban M.D. member and ambassador, Angels Santé Business Angels Network.

Jean-Marc Limacher, M.D., Chairman of the company said: "The renewed support of historical shareholders and the engagement of new investors is a mark of trust which acknowledges the advancements made by the company and its collaborators. This new investment will allow Odimma Therapeutics to bring ODI-2001, in the clinical stage of its development."

In relation with the preparation of its first clinical trial the company is actively working on the next financing round.

On May 2, 2023 Personalis, Inc. (Nasdaq: PSNL) reported it has partnered with the Academic Breast Cancer Consortium (ABRCC) and Criterium to carry out a prospective clinical trial, B-STRONGER-1 (Breast Cancer-Minimal Residual Disease Detection and Therapy Monitoring in Patients with Early Stage TNBC-Phase I), to evaluate the clinical performance of the NeXT Personal test for detecting minimal residual disease (MRD) during and after treatment and recurrent cancer in patients with early-stage resectable triple-negative breast cancer (TNBC) (Press release, Personalis, MAY 2, 2023, View Source [SID1234630875]).

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TNBC accounts for approximately 15%-20% of breast cancers diagnosed worldwide and is associated with worse outcomes compared to other breast cancer subtypes. Recurrence may be difficult to detect with other MRD assays, as TNBC in its earlier stages tends to ‘shed’ less circulating tumor DNA (ctDNA), a key marker for residual or recurrent cancer.

"The relatively high rate of recurrence in early stage TNBC patients makes detection of MRD after treatment important for assessing therapy response and providing opportunities for earlier intervention," said Richard Chen, MS, MD, Chief Medical Officer and Executive Vice President of R&D at Personalis. "However, in early-stage cancers like this, detection can be challenging because of lower ctDNA shedding rates. We specifically designed NeXT Personal to detect ctDNA with very high sensitivity. With the B-STRONGER-1 trial with ABRCC, Criterium and Dr. Chalasani, we hope to demonstrate that this high sensitivity enables earlier and more accurate detection of MRD in early stage TNBC patients."

Currently, a patient’s likelihood of developing a recurrent tumor is largely determined through pathological assessment of tissue samples collected from both the site of the primary tumor and regional lymph nodes following treatment—a process broadly referred to as pathological complete response (pCR) testing.

"While a valuable tool in estimating the risk of recurrence, pCR doesn’t help us to monitor for recurrence and response to treatment. More importantly, a large proportion of patients who do not have a pCR status have cancer recurrence. Currently we do not have good options in the clinic for detecting early recurrence. If we find it via imaging scans, it is too late, as this means the cancer has established a new ‘home’ already," explained the study’s principal investigator, Pavani Chalasani, MD, Division Director for Hematology/Oncology, George Washington Cancer Center, George Washington University. "MRD testing has shown significant promise in recent years as a viable alternative, and it could become the new standard for assessing not only a patient’s response to treatment, predicting their risk of recurrence, but also to detect early recurrence of disease. This early detection provides a window for the oncologist to potentially intervene and change treatment before the cancer can establish a new ‘home’."

NeXT Personal leverages next-generation sequencing technology to detect ctDNA in the bloodstream and may enable detection of micrometastases that currently evade pCR detection. The assay is designed to deliver industry-leading MRD sensitivity down to 1 part-per-million, an approximately 10- to 100-fold improvement over other available technologies. This may enable earlier detection across a broader variety of cancers and stages.

The B-STRONGER-1 study will enroll approximately 900 patients at up to 30 US sites and will be carried out in two stages. In the first stage, samples will be collected from each patient for both pCR and MRD analyses, to assess whether MRD using NeXT Personal correlates with standard of care pCR measurements. The second stage will involve a five-year follow-up to deepen the evidence on performance and establish clinical utility of NeXT Personal in early stage resectable TNBC.

About Triple Negative Breast Cancer

Triple negative breast cancer (TNBC) is aggressive and strongly correlated with hereditary BRCA1/2 mutations as well as a higher prevalence in young African American women. Despite advances in systemic therapies, currently 25–30% of early stage patients develop metastatic disease within 3–5 years of diagnosis and subsequent overall survival ranges from 8–13 months.

On May 2, 2023 Scorpion Therapeutics, Inc. ("Scorpion"), a pioneering clinical-stage oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, reported that the first patient has been dosed in a Phase 1/2 first-in-human dose escalation and expansion clinical trial evaluating STX-478, Scorpion’s highly differentiated, allosteric and central nervous-system ("CNS") penetrant inhibitor of mutant phosphoinositide-3-kinase alpha ("PI3Kα"), for the treatment of HR+/HER2- breast cancer and other solid tumors (Press release, Scorpion Therapeutics, MAY 2, 2023, View Source [SID1234630874]). The Phase 1/2 clinical trial will evaluate STX-478 as a monotherapy in a variety of solid tumors including breast and gynecological cancers, head and neck squamous cell carcinoma ("HNSCC") and others, as well as a monotherapy and in combination with approved agents in patients with HR+/HER2- breast cancer.

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STX-478 is designed to improve outcomes in patients harboring prevalent PI3Kα mutations in their tumors and has shown activity in both kinase and helical domain mutated tumors in preclinical models. PI3Kα mutations are among the most prevalent drivers of cancer, occurring in over 166,000 patients per year with breast, gynecological and head and neck cancers in the United States alone. The frequency of PI3Kα mutations and the scarcity of available treatment options has made the target a high priority for drug discovery.

"We are excited to begin clinical evaluation of STX-478, our mutant-selective PI3Kα inhibitor with a potentially best-in-class profile, for the treatment of HR+/HER2- breast cancer and a wide array of other solid tumors," said Axel Hoos, M.D., Ph.D, Chief Executive Officer of Scorpion. "The initiation of this clinical trial in just three years after our company was founded is an important validation of our Precision Oncology 2.0 strategy. The quality of the compound and the rapid progression of this program from target selection to clinical development demonstrate the expertise of our scientific team, the technical capabilities of our discovery platform, and their combined ability to create potentially transformational therapies."

Currently available treatments for PI3Kα-mutated cancers are limited by their inhibition of the normal, or wild-type, version of PI3Kα in healthy tissues, that can lead to significant side effects such as hyperglycemia and rash, which hinder the ability of patients to tolerate these therapies on a long-term basis. Further, these treatments also have little to no CNS penetrance, despite the fact that up to 50% of all solid tumor patients develop significant morbidity and mortality from brain metastases.

"STX-478 is a wild-type-sparing, CNS-penetrant, oral inhibitor of mutant PI3Kα with excellent pre-clinical pharmacokinetic properties," said Michael Streit, M.D., Chief Medical Officer of Scorpion. "In this Phase 1/2 trial, we will aim to demonstrate how these important traits translate into a potentially superior product profile that offers a wider therapeutic window and greater efficacy. We anticipate presenting initial safety, pharmacokinetic, and pharmacodynamic results in 2024, including data on STX-478’s impact on potential biomarkers suggestive of anti-tumor activity. We believe these initial data could differentiate STX-478 from existing agents currently on the market or in development, and support its profile as a safe and highly differentiated therapeutic for the treatment of solid tumors, especially HR+/HER2- breast cancer. We look forward to partnering with study investigators to evaluate STX-478 in patients with PI3Kα-mutated cancers."

About STX-478 Phase 1/2 Trial
Scorpion’s Phase 1/2 clinical trial is a multi-center, open-label study designed to evaluate the safety and tolerability of STX-478 in multiple ascending doses for patients with locally advanced or metastatic HR+/HER2- breast cancer driven by PI3Kα-mutations. The goal of the trial is to characterize the safety profile of STX-478 and establish a maximum tolerated dose ("MTD") or a lower optimal-biologically active dose, if appropriate, as the recommended Phase 2 dose ("RP2D") as a monotherapy for breast cancer and other solid tumor types, and as a combination agent in PI3Kα-mutant HR+/HER-2- breast cancer. Once the MTD is reached, or a RP2D is established, Scorpion intends to open expansion cohorts evaluating STX-478 as monotherapy for several indications such as breast cancer, gynecological cancers, HNSCC, gastrointestinal cancers and other solid tumors, all harboring PI3Kα-mutations. In addition, the combination of STX-478 and fulvestrant, as well as other standard of care agents, will be investigated in breast cancer patients harboring PI3Kα mutations. Secondary objectives for this Phase 1/2 trial include assessing the pharmacokinetic profile, pharmacodynamic effects and clinical response as measured by Response Evaluation Criteria In Solid Tumors ("RECIST") version 1.1. To learn more about the first-in-human trial of STX-478, please visit this page.

On May 2, 2023 EdiGene, Inc., a clinical-stage biotechnology company focused on developing transformative gene-editing therapies, reported an oral presentation on the preclinical proof-of-concept (POC) data in non-human primate (NHP) model, including NHP disease model, for LEAPERTM 2.0-based in vivo RNA editing therapies at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) to be held on May 16-20, 2023 in Los Angeles, California (Press release, EdiGene, MAY 2, 2023, View Source [SID1234630873]). LEAPERTM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.

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Data from specific NHP disease model have demonstrated the preclinical safety and efficacy of LEAPERTM 2.0-based in vivo therapeutic approach with outstanding editing efficiency. With endogenous ADAR, LEAPERTM has achieved precise and highly efficient RNA base editing using only one guide RNA (arRNA). Additionally, the exogenous protein-free feature significantly reduces delivery difficulty and minimizes the potential for immune response induced by foreign proteins. Algorithms are also developed to limit bystander effects, ensuring the high specificity of LEAPERTM 2.0. These results represent a significant step forward in the development of novel therapies.

"We are thrilled about the tremendous potential of our in vivo RNA editing therapeutic approach, which may have the capability to address significant unmet medical needs by enabling A-to-G editing, and these needs are not limited to correcting G-to-A mutations." said Pengfei Yuan, Ph.D., Chief Technology Officer of EdiGene. "Our team is relentlessly working to advance our leading programs into life-changing therapies for patients, with an initial focus on ophthalmic, neuromuscular, and CNS diseases, through both our internal efforts and external collaborations."

Details of the presentation:
Title: Efficient and Safe RNA Editing in Non-Human Primates Using AAV Delivered LEAPER Agents
Session Date/Time: Thursday, May 18, 2023, 3:45 PM – 5:30 PM
Session title: Gene Targeting and Gene Correction: New Technologies
Room: Room 515 AB
Presentation Time: 5:00 pm – 5:15 pm
Final abstract number: 233

On May 2, 2023 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, reported that it will share initial preclinical data on its in vivo Gene Placement System (iGPS) technology during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, occurring May 16-20, 2023, in Los Angeles (Press release, Kelonia Therapeutics, MAY 2, 2023, View Source [SID1234630872]).

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The preclinical data presented will demonstrate that Kelonia’s iGPS technology uniquely enables safe and efficient in vivo generation of highly efficacious CAR T cells. In a humanized mouse model of multiple myeloma treatment, when an anti-BCMA CAR transgene was delivered directly in vivo using iGPS particles, low intravenous dose levels resulted in complete initial tumor regression comparable to ex vivo-manufactured CAR T cells. However, functional CAR T cell persistence after iGPS particle treatment resulted in significantly longer tumor control compared to ex vivo-generated CAR T cells. Despite efficient in vivo CAR gene transfer to T cells, no concerning or unexpected "off-target" transduction was observed including in vital, progenitor or reproductive organs.

"CAR T cells have been transformative for patients suffering from lethal cancers. But manufacturing unique CAR T cells for every patient has limited access to this incredible medicine. The preclinical data we’ll present at ASGCT (Free ASGCT Whitepaper) demonstrates the potential for potent, durable and safe CAR T cells generated by our iGPS in vivo delivery technology, without the requirement for preparative chemotherapy," said Kevin Friedman, Ph.D., Founder, President, and Chief Scientific Officer, Kelonia Therapeutics. "This is the first step toward our vision of solving the central challenge of genetic medicines: democratizing and enabling access when and where patients need them. We look forward to sharing detailed data at the conference."

Details for the oral presentation are as follows:

Abstract Title: Potent in vivo transduction by iGPS particles generates CAR T cells with durable anti-tumor activity

Presenter: Shannon Grande Contrastano, Ph.D., Senior Director, Preclinical Sciences, Kelonia Therapeutics

Session Date/Time: Wednesday, May 17, 2023: 3:45-5:30 PM PDT

Abstract number: 90