On May 7, 2024 Photocure ASA (OSE: PHO), the Bladder Cancer Company, reported its participation in the congress, and two abstract presentations at the AUA 2024: the American Urological Association Annual Congress 2024 was held May 3-6, 2024, in San Antonio, TX, USA (Press release, PhotoCure, MAY 7, 2024, View Source [SID1234642818]). The results of the BRAVO study performed within the VA healthcare system showed significant decreases in the risk of recurrence and progression, as well as the potential for improved overall survival in patients who received a blue light cystoscopy (BLC) compared to patients whose cystoscopy was only performed under white light. Another comparison of BLC with Hexvix/Cysview and white light cystoscopy (WLC), for the detection of bladder cancer using modern HD 4K equipment, was presented in an abstract from the multicenter phase III study of Hexvix in China, including new real world evidence data.

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On Sunday, May 5th, Dr. Sanjay Das presented the study, "Use of Blue Light Cystoscopy Among Non-Muscle Invasive Bladder Cancer Patients and Outcomes in an Equal Access setting: A Propensity Scored Matched Analysis."

The study, known as BRAVO (Bladder Cancer Recurrence Analysis in Veterans and Outcomes), is a retrospective, propensity score matched analysis that evaluated oncologic outcomes following BLC compared to WLC alone in patients from the Veterans Affairs Healthcare System. The study addresses a lack of practical real-world data comparing the impact of BLC versus WLC, specifically for recurrence, progression, and survival. The results of this study confirm that BLC use is associated with positive and statistically significant impacts on these outcomes. The Veterans’ Affairs (VA) Healthcare system accepts all U.S. Veterans, regardless of financial background, and retains its patients, allowing for high-quality data capture over a long-term follow-up period, therefore serving as a robust real-world model for equal access.

626 patients were included in this study, 313 in each study arm (WLC versus BLC). Recurrence and progression data for BRAVO was measured at a 3-year time point. Overall survival follow-up was for 10 years.

Study results include:

Risk of recurrence was significantly lower following BLC (HR 0.60, 95% CI 0.29-0.61) – 40% reduction in risk of recurrence. This confirms data from multiple RCT studies.
Patients who underwent BLC had significantly reduced risk of progression (HR 0.51, 95% CI 0.36-0.99) compared to patients who underwent WLC.
There was improved overall survival among BLC vs. WLC (HR 0.41, 95% CI 0.30-0.72)
Additionally, in the equal-access setting of the VA Healthcare System, benefits of BLC were equitably shared between race/gender.
The Principal Investigator of the BRAVO Study, Dr. Steven Williams, commented: "The results of the BRAVO study performed within the VA healthcare system showed significant decreases in the risk of recurrence and progression, as well as the potential for improved overall survival in patients who received a BLC compared to patients who received WLC only. These findings demonstrate the benefit of BL-enhanced cystoscopy as part of comprehensive care for NMIBC* patients, especially as improved tumor visualization helps to appropriately make determination of intravesical therapy use, such as BCG. The results are encouraging and consistent with prior clinical trial long-term oncological outcomes. It supports the generalizability of prior clinical trial results in the real-world clinical practice setting. The demonstrated impact on overall survival warrants future studies to better understand the oncologic benefit of BLC in NMIBC."

Read the abstract: View Source

On Monday, May 6th, a Poster presentation by Dr. Hailong Hu: Blue Light Cystoscopy versus White Light Cystoscopy for the Detection of Bladder Cancer using modern HD 4K equipment: An Analysis of Pivotal Trial and Real-World Data

This pooled meta-analysis presented data from a randomized clinical trial and a supporting real-world evidence study conducted in China. Both studies enrolled patients with known or suspected bladder cancer. A total of 177 patients were enrolled, 128 patients underwent blue light cystoscopy (BLC) with Cysview (HAL) and were included in the full analysis set. Among patients diagnosed with Ta, T1, or CIS, 46 out of 109 patients (42.2%) had at least one lesion detected by BLC but not by white light cystoscopy (WLC) (p<0.0001). Fifteen patients had CIS of which 12 (80%) showed at least one additional CIS lesions found by BLC but not by WLC. The BLC detection rates for CIS, Ta, T1, and T2-T4 tumors were 95.2%, 100%, 98.3%, and 100%, respectively, while the WLC detection rates were 42.9%, 76.5%, 91.7%, and 100%, respectively.

This study confirms the superiority of HAL BLC over WLC in the detection of bladder cancer even if improved WLC using HD 4K equipment is utilized. In particular, additional high-risk difficult to see CIS lesions have been identified in 80% of CIS patients only by HAL BLC. The quality of resection is still a key cornerstone in the treatment of NMIBC of which BLC remains a crucial part despite the further development of WLC imaging.

Read the abstract: View Source

Beyond this groundbreaking data on BLC/WLC comparison, Photocure provided attendees with hands-on experience in the blue light cystoscopy with Cysview procedure on its congress booth, that featured a Saphira HD equipment tower.

*NMIBC: Non muscle-invasive bladder cancer

On May 7, 2024 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage immunology company, reported multiple poster presentations at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually (Press release, Myeloid Therapeutics, MAY 7, 2024, View Source;cell-therapy-asgct-2024-annual-meeting-302137520.html [SID1234642817]).

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"The data presented at ASGCT (Free ASGCT Whitepaper) showcase that Myeloid continues to lead the field of in vivo immune cell engineering and RNA-enabled gene editing and delivery. The data presented today underlies our in vivo mRNA CAR, MT-302, that continues to progress well in the ongoing clinical study. We are expanding plans for MT-302’s clinical use, given several inherent product advantages for patients," said Daniel Getts, Ph.D., Chief Executive Officer of Myeloid. "In addition, our groundbreaking CREATE platform demonstrates a full-scale revolution in gene editing. CREATE provides the ability to deliver large payloads with precision and solely using mRNA, which highlights the broad potential at Myeloid to transform cancer treatments in addition to rare disease indications."

Poster presentation details and abstract highlights include:

Title: "In vivo Programming of Immune Cells Using mRNA-LNP Chimeric Antigen Receptors"
Date & Time: Thursday, May 9, 2024, 12:00 PM-7:00 PM ET
Poster Session: Targeted Gene and Cell Therapy
Location: Poster Session F3
Session Title: Cancer – Targeted Gene and Cell Therapy
Published Abstract Number: 1284

Myeloid has designed novel CARs that achieve expression and function in targeted immune cell populations. These CARs, by activating innate and adaptive immune responses following the in vivo delivery of LNP-formulated mRNA encoded CARs, are capable of eliciting anti-tumor efficacy against a range of multiple target antigens evaluated.
Myeloid has demonstrated CAR activity in human cells, and following systemic mRNA/LNP delivery in mouse and non-human primates.
MT302, an in vivo CAR targeting TROP2+ epithelial malignancies (NCT05969041), is being evaluated in a Phase 1 clinical trial to assess the candidate’s safety and preliminary efficacy.
Title: "CRISPR-Enabled Autonomous Transposable Element (CREATE) for RNA-mediated gene editing and delivery"
Date & Time: Wednesday, May 8, 2024, 12:00 PM-7:00 PM ET
Poster Session: Gene Targeting and Gene Correction New Technologies
Location: Poster Session F3, Exhibit Hall
Session Title: Cancer – Targeted Gene and Cell Therapy
Published Abstract Number: 719

Myeloid developed a proprietary, RNA-based genome editing system called CREATE – CRISPR-Enabled Autonomous Transposable Element, that combines Prime Editing with a DNA transposase to overcome the current limitations of gene delivery technologies.
CREATE merges the capabilities of CRISPR/Cas9 with human L1 retrotransposon to insert gene-sized payloads without DNA donors or double strand breaks.
Mechanistic studies reveal that the CREATE system is highly specific with no observed off-target events.
These data establish CREATE system as a programmable gene delivery platform solely based on RNA components, enabling large-scale in vivo genome engineering with broad therapeutic potential.
More information on ASGCT (Free ASGCT Whitepaper) can be found here. More information on Myeloid’s CREATE platform can be found here.

On May 7, 2024 Biotheryx, Inc., a biopharmaceutical company discovering and developing a portfolio of first-in-class protein degraders with a focus on validated targets in cancer and inflammatory disease, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for BTX-9341, a novel cyclin-dependent kinase 4/6 (CDK4/6) bifunctional degrader (Press release, BioTheryX, MAY 7, 2024, View Source [SID1234642816]). The Company plans to initiate the Phase 1 clinical trial in the second half of 2024 and intends to enroll patients with HR+/HER2- breast cancer resistant to CDK4/6 inhibitor therapies.

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"Securing FDA clearance for our BTX-9341 IND application marks a significant milestone for Biotheryx, affirming our commitment to advancing innovative, orally bioavailable targeted protein degraders. As we transition from promising preclinical data to clinical trials, we are poised to explore the potential of BTX-9341 in offering tangible clinical benefits to patients battling breast cancer," said Leah Fung, Ph.D., CEO of Biotheryx.

The Phase 1 clinical trial includes dose escalation for the initial monotherapy administration of BTX-9341 and combination dose expansion combining BTX-9341 with fulvestrant. The initial clinical evaluation will be focused on safety, biological activity and preliminary efficacy.

About BTX-9341

BTX-9341 is a first-in-class, oral degrader of cyclin-dependent kinase 4 (CDK4) and cyclin-dependent kinase 6 (CDK6), important targets for a range of cancers and clinically validated in the context of certain breast cancers. In preclinical breast cancer models, BTX-9341 demonstrated superiority to CDK4/6 inhibitors through potent and highly selective degradation of CDK4 and CDK6, robust inhibition of Cyclin E and CDK2 transcription, cell cycle arrest and ultimately superior in vivo efficacy in breast cancer xenografts. Beyond this increased efficacy potential, BTX-9341 is differentiated from CDK4/6 inhibitor approaches through the potential ability to overcome key resistance mechanisms that limit the impact of inhibitors (~20% of patients have intrinsic resistance and up to 70% have acquired resistance to CDK4/6 inhibitors) and significantly enhanced penetration of the blood-brain-barrier.

On May 7, 2024 IceCure Medical Ltd. (Nasdaq: ICCM) ("IceCure" or the "Company"), developer of the ProSense System, a minimally-invasive cryoablation technology that destroys tumors by freezing as an alternative to surgical tumor removal, reported that positive data from an independent study (the "Study") performed in Japan was published in an article titled "Percutaneous ultrasound–guided cryoablation for early–stage primary breast cancer: a follow–up study in Japan," in the journal Breast Cancer on April 27, 2024 (Press release, IceCure Medical, MAY 7, 2024, View Source [SID1234642815]). The Study, which focused on the local control of cancer, safety, patient quality of life, patient satisfaction, and cosmetic outcomes of cryoablation for patients with early-stage breast cancer, is the continuation of a prior pilot study that demonstrated percutaneous cryoablation treated with ProSense is a potential standard treatment for early-stage breast cancer patients, given compliance to pre-defined patient selection criteria.

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The Study was led by Dr. Hisanori Kawamoto, M.D., Ph.D. from the Department of Breast Surgery, Breast and Imaging Center at St. Marianna University School of Medicine in Japan. Eighteen early-stage breast cancer patients, with a mean age of 59.0 [±9.0 years], with a mean tumor size of 9.8 ±2.3 mm, who underwent treatment with ProSense were followed for a mean of 44.3 months. No patients had local recurrence or distant metastasis in the 5-year follow-up. No serious adverse events were reported. Cosmetic outcomes were excellent and the overall patient satisfaction level and patient quality of life improved post-cryoablation.

The article states that minimally invasive non-surgical techniques, including cryoablation, aim for curative outcomes while also acknowledging the importance of preserving or enhancing the quality of life and cosmetic appearance following the procedure. The authors of the Study refer to recent trials, including IceCure’s ICE3 trial, for evidence that cryoablation results in local cancer control rates comparable to lumpectomies in early-stage breast cancer patients.

Dr. Kawamoto commented, "This is a very important study in Japan, where we are experiencing an upward trend in breast cancer cases, especially among women in their late 40s to early 60s. These are women who often are at very active stages of their life and therefore there is a growing demand for treatment options that minimize the chance of or avoid hospitalization, in addition to resulting in favorable clinical and cosmetic outcomes. We are very pleased with these results and are hopeful that ProSense may become a favored option in Japan upon regulatory approval for early-stage breast cancer."

"We thank Dr. Kawamoto and his colleagues for conducting this independent study, which adds to the growing body of efficacy and safety data for ProSense as a minimally-invasive option for early-stage breast cancer," stated IceCure CEO, Eyal Shamir. "We anticipate that our in-country distribution partner, Terumo Corporation, will file for regulatory clearance of ProSense for breast cancer in Japan later this year, which may soon lead to the approval and commercialization for it in a market where there is high demand for a minimally-invasive option."

Terumo Corporation owns the exclusive distribution rights for ProSense in Japan for a 5-year term following regulatory approval. In exchange for exclusive distribution rights, IceCure is expected to receive a total of $13.2 million in proceeds from Terumo during this initial term, of which it has received $4 million to date, with an additional $8.2 million expected upon the completion of orders, as well as $1 million expected for milestone-based payments.

About ProSense

The ProSense Cryoablation System provides a minimally invasive treatment option to destroy tumors by freezing them. The system uniquely harnesses the power of liquid nitrogen to create large lethal zones for maximum efficacy in tumor destruction in benign and cancerous lesions, including breast, kidney, lung, and liver.

ProSense enhances patient and provider value by accelerating recovery, reducing pain, surgical risks, and complications. With its easy, transportable design and liquid nitrogen utilization, ProSense opens that door to fast and convenient office-based procedure for breast tumors.

On May 7, 2024 GC Genome Corporation, a leading diagnostics company, reported that it will present the new clinical data of its AI-based liquid biopsy platform on colorectal cancer detection at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, May 31–June 4 (Press release, GC Genome, MAY 7, 2024, View Source [SID1234642814]). This research was conducted through a collaborative effort with Genece Health Inc., a strategic partner based in San Diego.

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Poster Presentation Details:

Title: Non-invasive colorectal cancer detection using multimodal deep learning ensemble classifier.

Date and Time: Saturday, June 1, 2024 9:00 AM – 12:00 PM CDT
Format: Poster Presentation
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Poster Board Number: 211
Abstract Number: 3066