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

On May 2, 2023 Verismo Therapeutics, a clinical-stage CAR-T company and University of Pennsylvania spinout developing novel KIR-CAR platform technology, reported that it has activated its STAR-101 Phase 1 clinical trial at the University of Pennsylvania (Press release, Verismo Therapeutics, MAY 2, 2023, View Source [SID1234630870]).

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STAR-101 will evaluate Verismo’s lead candidate, SynKIR-110, an investigational new drug for the treatment of mesothelin-overexpressing malignant pleural mesothelioma, cholangiocarcinoma and ovarian cancer. Verismo Therapeutics is conducting a Phase 1 multicenter clinical trial in these tumor types to evaluate the feasibility and safety of SynKIR-110 (ClinicalTrials.gov Identifier: NCT05568680).

Verismo achieved clearance from the FDA to administer this multicenter clinical trial under the IND Verismo submitted for SynKIR-110, and Orphan Drug Designation for the treatment of mesothelin-expressing mesotheliomas with SynKIR-110 in September 2022. SynKIR -110 received Fast Track designation in April 2023.

"This trial marks a pivotal moment for our company," said Dr. Bryan Kim, Co-Founder and CEO of Verismo Therapeutics. We are grateful for the opportunity to partner with distinguished researchers at the University of Pennsylvania, including Dr. Janos Tanyi, MD, PhD, Dr. Andrew Haas, MD, PhD, and Dr. Mark O’Hara, MD, to bring this innovative therapy to those who require it the most."

Dr. Tanyi, an associate professor of Obstetrics and Gynecology in the Perelman School of Medicine at the University of Pennsylvania, and a principal investigator for the trial, said, "Our team is excited to be part of this clinical trial with Verismo. We are enthusiastic about the prospects of this innovative therapy and its capacity to provide new hope and improved outcomes for individuals who urgently need effective treatment options."

For more information about the STAR-101 clinical trial, please visit ClinicalTrials.gov NCT05568680.

About the KIR-CAR Platform
The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical animal models to be capable of maintaining antitumor T cell activity even in challenging solid tumor environments. DAP12 acts as a novel costimulatory molecule for T cells using additional T cell stimulating pathways, further sustaining chimeric receptor expression and improving KIR-CAR T cell functional persistence. This continued T cell function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those resistant to traditional CAR T cell therapies. The KIR-CAR platform is being investigated in combination with many additional emerging technologies, such as in vivo gene engineering, advanced cell manufacturing and reprogramming, combinational therapies, and even allogeneic cellular therapies to potentially provide the next-generation multimodal targeted immunotherapy for patients in need.

On May 2, 2023 EirGenix, Inc. (6589.TT) reported on 28th of April that it has completed the phase 1 clinical trial (EGC101) of its second breast cancer biosimilar EG1206A in Europe (Press release, EirGenix, MAY 2, 2023, View Source [SID1234630869]). Comparing EG1206A to Roche’s Perjeta US and EU, results of the clinical data analysis show that the main test indicators have met the pharmacokinetics bioequivalence standard. EG1206A is a biosimilar drug of the second-generation HER2-positive antibody drug Perjeta (pertuzumab). EG1206A has a unique binding mechanism for HER2 receptors which can produce a dual blockade effect. In combination with the first-generation HER2-positive antibody drug Trastuzumab, Pertuzumab has a synergistic effect in the treatment of early-stage HER2-positive breast cancer and metastatic breast cancer.

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The success of EG1206A’s phase 1 clinical trial is an important milestone for EirGenix and its development of biosimilar drug products for the treatment of HER2-positive breast cancer. Following the product launch of its first biosimilar drug EG12014 (trastuzumab biosimilar), EG1206A will soon begin its multi-country, multi-center global phase 3 clinical trials. After the initiation of the phase 3 clinical trials, it is expected that the evaluation of the primary end point indicators will be completed within two (2) years. Concurrently, EirGenix has been negotiating with global pharmaceutical companies for licensing marketing rights of EG1206A to further fast-track the launch of the biosimilar into the global market. Such achievements in EirGenix’s product development bring great excitement to the future of Taiwan’s biotechnology industry.

1. About EG1206A (pertuzumab biosimilar)

EG1206A is a biosimilar drug of the second-generation HER2-positive antibody drug Perjeta (pertuzumab). The clinical combination of trastuzumab and pertuzumab has a synergistic effect in the treatment of early-stage HER2-positive breast cancer and metastatic breast cancer. Patients with HER2-positive breast cancer account for approximately 20-30% of new breast cancer cases per year. The dual-drug therapy can help to prolong the survival period of breast cancer patients and is a standard treatment strategy recommended by many international treatment guidelines. According to 2022 sales data by Roche (developer of the originator drug Perjeta), global sales of Perjeta (pertuzumab) reached 4.087 billion Swiss Francs (4.56 billion U.S. Dollars). By the time the patent of Perjeta expires in 2026, it is expected that global sales will reach 6 billion U.S Dollars (approx. 180 billion New Taiwan Dollars). In 2021, Taiwan’s National Health Insurance paid 1.02 billion New Taiwan Dollars for Perjeta (pertuzumab) products. EirGenix’s EG1206A currently ranks amongst the top globally for most advanced developments of pertuzumab biosimilars. Once EirGenix’s products are launched onto the market, HER2-positive breast cancer patients will be provided with more accessible and affordable treatment options.