Exsilio Therapeutics Launches with $82 Million Series A Financing to Develop Redosable Genomic Medicines for A Broad Range of Diseases

On June 25, 2024 Exsilio Therapeutics ("Exsilio"), a biotechnology company developing genomic medicines for a broad range of diseases, reported its emergence from stealth with $82 million in Series A financing (Press release, Exsilio Therapeutics, JUN 25, 2024, View Source [SID1234644541]). The funding was co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. Exsilio was seed-funded by OrbiMed.

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Exsilio will use proceeds from the financing to advance its genomic medicines based on naturally occurring, programmable genetic elements that can precisely insert new genes into a cell through mRNA intermediates. Exsilio’s interdisciplinary team has built a platform that combines predictive in silico modeling and wet lab-based experimentation to discover and engineer such elements for integration of therapeutic genes into safe harbor sites. Because Exsilio’s medicines are encoded in mRNA, they can be delivered using existing lipid nanoparticle (LNP) platforms that are safe, efficient, scalable, and cost-effective, and can be redosed and titrated with curative intent.

"mRNA-based medicines allow for a software-like approach to creating new medicines," said Tal Zaks, M.D., Ph.D., who serves as Exsilio’s Chairman and Interim Chief Executive Officer. "Exsilio’s approach leverages the advantages of mRNA and goes a step further by encoding genes that integrate permanently, offering the possibility of curative rather than transient effects. The ability to insert whole genes with a repeatable and titratable approach should allow us to treat genetic diseases irrespective of the patient’s individual mutation. This financing will help us advance our genomic medicines and select promising lead candidates so that we can bring much-needed new options to patients."

"We were captivated by Exsilio’s genomic medicines approach that stands to enable large-gene integration in a safe and redosable manner," said Aaron Nelson, Managing Director at Novartis Venture Fund and Exsilio Board Member. "Through this significant investment, Exsilio will be able to select and advance promising candidates for difficult-to-treat diseases."

"We believe that the vision of safely and durably integrating therapeutic genes into a patient’s genome requires using RNA-based payloads that can leverage clinically validated non-viral gene delivery technologies," said Henry Chen, Managing Partner of Delos Capital and Exsilio Board Member. "Exsilio is bringing together a singular group of people to help establish this new pillar of genomic medicine."

Carisma Therapeutics to Participate in the Stifel 2024 Cell Therapy Forum

On June 25, 2024 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported that Steven Kelly, President and Chief Executive Officer, will participate in a fireside chat at the Stifel 2024 Cell Therapy Forum on Tuesday, July 9th at 9:10 am ET (Press release, Carisma Therapeutics, JUN 25, 2024, View Source [SID1234644540]).

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An audio webcast of the event will be available on the Company’s Investor Events section of the Investor Relations webpage and will be archived for a limited time following the event.

Carisma Therapeutics Granted FDA Fast Track Designation for CT-0525 for the Treatment of HER2-overexpressing Solid Tumors

On June 25, 2024 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for CT-0525, an ex vivo gene-modified autologous chimeric antigen receptor-monocyte (CAR-Monocyte) cellular therapy intended to treat solid tumors that overexpress human epidermal growth factor receptor 2 (HER2) (Press release, Carisma Therapeutics, JUN 25, 2024, View Source [SID1234644539]).

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The FDA’s Fast Track program is aimed to facilitate the development, and expedite the review, of novel potential therapies that are designed to treat serious conditions and have the potential to address significant unmet medical need.

"Receiving Fast Track designation for CT-0525 from the FDA marks a significant milestone for Carisma, highlighting the FDA’s recognition of the serious and life-threatening nature of these malignancies and the potential of CT-0525 to meet this critical medical need," said Eugene P. Kennedy, M.D., Chief Medical Officer of Carisma. "We are committed to working closely with the FDA to accelerate the development of CT-0525. Currently, we are enrolling patients in the Phase 1 clinical trial and remain on track to report initial clinical data by the end of 2024."

The Phase 1 clinical trial for CT-0525 is an open-label study designed to assess the safety, tolerability, and manufacturing feasibility of CT-0525. This trial will enroll participants with locally advanced (unresectable) or metastatic solid tumors overexpressing HER2 whose disease has progressed on standard approved therapies. The initial study design will consist of two dose escalation cohorts. Further details of the trial can be found at www.clinicaltrials.gov under NCT identifier: NCT06254807.

About CT-0525

CT-0525 is a first-in-class, ex vivo gene-modified autologous chimeric antigen receptor-monocyte (CAR-Monocyte) cellular therapy intended to treat solid tumors that overexpress human epidermal growth factor receptor 2 (HER2). It is being studied in a multi-center, open label, Phase 1 clinical trial for patients with advanced/metastatic HER2-overexpressing solid tumors that have progressed on available therapies. The CAR-Monocyte approach has the potential to address some of the challenges of treating solid tumors with cell therapies, including tumor infiltration, immunosuppression within the tumor microenvironment, and antigen heterogeneity. CT-0525 has the potential to enable significant dose escalation, enhance tumor infiltration, increase persistence, and reduce manufacturing time compared to macrophage therapy.

IDEAYA Biosciences Announces First-Patient-In for Phase 1 Clinical Trial Evaluating IDE397 and Trodelvy® Combination in MTAP-Deletion Bladder Cancer

On June 25, 2024 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that it has dosed its first patient in the IDEAYA-sponsored Phase 1 trial evaluating the combination of IDE397, IDEAYA’s investigational MAT2A inhibitor, and Trodelvy (sacituzumab govitecan-hziy), Gilead’s Trop-2 directed ADC, in patients with MTAP-deletion bladder cancer (Press release, Ideaya Biosciences, JUN 25, 2024, View Source [SID1234644538]).

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"We are pleased to have dosed our first patient with MTAP-deletion bladder cancer in this Phase 1 trial evaluating combination treatment with IDE397 and Trodelvy. The MAT2A-Trop2 ADC combination targets two distinct, yet complementary nodes in patients with MTAP-deleted urothelial cancer and has first-in-class potential to improve clinical outcomes for bladder cancer patients with poor prognosis associated with MTAP-deletion," commented Dr. Darrin M. Beaupre, M.D., Ph.D., Chief Medical Officer, IDEAYA Biosciences.

The IDE397 and Trodelvy combination Phase 1 trial is included as an arm of an ongoing IDEAYA-sponsored clinical trial (NCT04794699), which includes a Phase 2 expansion arm of IDE397 monotherapy in MTAP-deletion solid tumors. The global Phase 1 clinical trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of IDE397 in combination with Trodelvy. IDE397 is a potent and selective small molecule inhibitor targeting methionine adenosyltransferase 2 alpha (MAT2A) in patients having solid tumors with methylthioadenosine phosphorylase (MTAP) deletion.

Gilead’s Trodelvy is currently approved in nearly 50 countries for 2L metastatic triple-negative breast cancer and in more than 30 countries for pre-treated HR+/HER2- metastatic breast cancer (mBC). In the U.S., Trodelvy has an accelerated approval for the treatment of patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and anti-PD-(L)1 therapy.

Pursuant to the clinical study collaboration and supply agreement, IDEAYA and Gilead retain the commercial rights to its respective compounds, including with respect to use as a monotherapy or combination agent. IDEAYA is the study sponsor and Gilead will provide the supply of Trodelvy to IDEAYA.

There is separately an Amgen-sponsored Phase 1/2 trial of IDE397 and AMG 193 combination in MTAP-Deletion NSCLC (NCT05975073) for which the companies intend to develop a joint publication strategy in 2024. The Company is targeting a clinical data update for the IDE397 Phase 2 monotherapy expansion dose in MTAP-deletion bladder and lung cancer in over ~15 evaluable patients in the second half of 2024. The Company is also advancing multiple preclinical stage MTAP-deletion programs to enable wholly-owned combinations with IDE397, including a program targeting a Development Candidate nomination in the second half of 2024.

IDE397 monotherapy or in combination with Trodelvy has not been approved by any regulatory agency and the efficacy and safety of this combination has not been established.

Allarity Therapeutics’ Dual PARP/Tankyrase Inhibitor, Stenoparib, Continues to Show Extended Clinical Benefit in Advanced Ovarian Cancer

On June 25, 2024 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing personalized cancer treatments, reported that multiple patients in its Phase 2 clinical trial of stenoparib for advanced recurrent ovarian cancer have been on treatment for more than 30 weeks (Press release, Allarity Therapeutics, JUN 25, 2024, View Source [SID1234644536]).

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The continued durability of clinical benefit further bolsters the Company’s announcement in early May 2024 that stenoparib had shown clear clinical benefit, including significant tumor shrinkage and long-term disease stability, in patients who had been heavily pre-treated for their ovarian cancer and otherwise have limited life expectancy. These results provided clinical proof of concept for stenoparib as a treatment in this patient population, prompting the company to halt patient enrollment to focus its resources on developing a follow-on trial designed to accelerate the path for stenoparib toward regulatory approval.

Kathleen N. Moore, MD, MS, Deputy Director of the Stephenson Cancer Center, Professor of the Section of Gynecologic Oncology and the Virginia Kerley Cade Chair in Developmental Therapeutics, serves as the Principal Investigator for the current Allarity trial: "Inhibition of poly-ADP-ribose polymerase or PARP has been transformational for the treatment of ovarian cancer with first-generation agents significantly improving progression-free and overall survival especially among patients with biomarkers for their use. However, there are a significant number of patients who either don’t benefit at all from inhibition of PARP or only modestly and for them, continual development of next-generation agents remains a high priority. The promising results observed with stenoparib – particularly with the tolerability demonstrated so far, warrant further development of this drug. I look forward to continuing the discussions with the team at Allarity to refine the future trial design and subsequently continue to investigate the potential of this novel PARP/Tankyrase inhibitor."

"The continued durability of clinical benefit from stenoparib furthers our enthusiasm for stenoparib for patients who traditionally have limited treatment options and often only a few months of continued life expectancy. We continue to see patients maintain their quality of life with minimal side effects for extended periods," stated Allarity Therapeutics CEO Thomas Jensen. He elaborated, "Not only does the safety profile stand out when compared to chemotherapies, which is often the alternative for this patient group, but the safety metrics are indeed also favorable when compared to first-generation PARP inhibitors. As we see it, stenoparib may represent the next-generation alternative for advanced ovarian cancer patients. Therefore, we are aggressively working with Dr. Moore and other leading experts to design a trial that will help advance and quicken stenoparib’s clinical progress toward registration with the FDA."

After conducting a detailed review of the stenoparib monotherapy trial data, including the data announced today, the Company has concluded that these data may be of significant interest among key oncologists, potential commercial partners, and other relevant stakeholders and therefore warrant presentation within a high-level scientific conference. Further release of these clinical data is intended to be staged to comply with the common rules of scientific conferences, which do not allow data to be published before the event.

The PARP inhibitor market saw a major shift in 2022 as rucaparib, olaparib, and niraparib were withdrawn for heavily pretreated ovarian cancer patients, underscoring the need for new, effective PARP inhibitors with a more favorable safety profile. The PARP inhibitor market is expected to reach $22 billion in revenue by 2028, and has historically seen significant partnerships and acquisitions. Recent interest in the market has focused on the development of PARP inhibitors with improved tolerability and safety profiles, leading to notable activity in the sector. For example, one significant acquisition included a multi-asset deal potentially totaling $1.5 billion, which featured an advanced PARP inhibitor. Stenoparib is orally available and also shares an advantaged safety profile relative to 1st generation PARP inhibitors. It also inhibits the Tankyrase 1 and 2 enzymes, which helps block the activity of the WNT/Beta-catenin pathway, a pathway often overactive in ovarian and many other solid cancers. The safety and unique, dual inhibitory activities of stenoparib make it a differentiated and compelling potential therapeutic product.

Background Information about the Trial
The above-mentioned trial is a Phase 2, prospective open-label, single-arm study with multiple sites in both the US and the UK. Investigators prescreened women with advanced, recurrent ovarian cancer using Allarity’s DRP companion diagnostic (CDx), which comprises a complex transcriptomic signature of 414 mRNA biomarkers indicative of drug response or resistance. Each participant was assigned a DRP score, and those with scores above 50 -suggesting a higher likelihood of benefiting from treatment – were selected to receive stenoparib. The selected patients were administered stenoparib under a revised protocol implemented in Q1 2023, which involved a twice-daily dosing regimen (200 mg in the morning and 400 mg in the evening) instead of the previous once-daily 600 mg dose. This change was made to optimize daily drug exposure and target inhibition.

The patients enrolled have advanced through multiple lines of therapy, including platinum, taxanes, anti-angiogenesis inhibitors, and even the recently approved Antibody Drug Conjugate, Elahere. Importantly, all but two enrolled patients to date have been previously treated with a PARP inhibitor. These patients have few, if any, effective treatment options and typically advance through available therapies after only a few months.

About stenoparib
Stenoparib is an orally available, small-molecule dual-targeted inhibitor of PARP1/2 and Tankyrase 1 and 2. At present, tankyrases are attracting significant attention as emerging therapeutic targets for cancer, principally due to their role in regulating the Wnt signaling pathway. Aberrant Wnt/β-catenin signaling has been implicated in the development and progression of numerous cancers. By inhibiting PARP and blocking Wnt pathway activation, stenoparib’s unique therapeutic action shows potential as a promising therapeutic. Allarity has exclusive global rights for the development and commercialization of stenoparib, which was originally developed by Eisai Co. Ltd. and was formerly known under the names E7449 and 2X-121.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has proven its ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients dozens of clinical studies (both retrospective and prospective). The DRP platform, which can be used in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature.