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

On May 7, 2024 Hopewell Therapeutics, a biotechnology company focused on discovering, synthesizing, and developing the next generation of tissue-targeted lipid nanoparticles (ttLNPs) to deliver genomic medicines to patients, reported that the Company will present preclinical data demonstrating progression of its pulmonary, oncology and vaccine programs at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting being held this week in Baltimore, MD, as well as virtually (Press release, Hopewell Therapeutics, MAY 7, 2024, View Source [SID1234642813]). The findings will be highlighted in a poster presentation and support Hopewell’s commitment to redefine the non-viral delivery space for novel genomic medicines by designing systemically administered LNPs based on proprietary ionizable lipid chemistry that enables specific targeting of tissues and cells throughout the body.

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Hopewell has advanced product candidates that combine tissue-targeting LNPs with proprietary mRNA encoding for therapeutic proteins to treat rare and common diseases. In preclinical models across multiple species, systemic delivery to specific cell types in the lung and trachea has been demonstrated, and locally enriched and robust protein expression has been achieved in the lung and bronchoalveolar lavage fluid. This includes lung-specific expression of GM-CSF and concomitant reduction of disease specific biomarkers in a murine model of pulmonary alveolar proteinosis (PAP), a rare disease with no FDA-approved treatment.

In addition, Hopewell’s lead liver-targeting LNP has been used to deliver mRNA encoding novel bispecific T-cell engagers (BiTES), which have demonstrated deep and sustained B-cell depletion in a non-human primate model, as well as reduction in tumor volume in a hepatocellular carcinoma mouse model. For immune cell targeted delivery, Hopewell has designed and tested novel systemically or subcutaneously administered LNPs that provide enhanced delivery efficiency to T-cells, B-cells, dendritic cells, and macrophages in the spleen and lymph nodes. These immune targeting LNPs have been successfully deployed in preclinical cancer vaccine and infectious disease vaccine models.

"This is an exciting time for Hopewell," shared Louis Brenner, M.D., Hopewell’s President and CEO. "We have made remarkable progress with our ttLNP technology over the past year, highlighted by the preclinical experiments that demonstrate systemic delivery of genomic cargoes to specific cellular targets in the lung, spleen, and liver. As our field advances and seeks to realize the promise of genetic medicines, it continues to be clear that targeted delivery of increasingly complex therapeutic cargoes to tissues and cell types within those tissues is necessary. Our team is presenting data this week that showcase the progress of our ttLNPs and potential therapeutic applications that could be pursued for patient benefit."

Details for the poster presentation are as follows:

Session Date/Time: Thursday, May 9, 2024; 12:00 p.m. ET
Session Title: Thursday Posters: Cancer – Immunotherapy and Cancer Vaccines
Final Abstract Number: 1273
Presenter: Xin Kai, Ph.D., Director of Discovery Biology

On May 7, 2024 Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, reported that its self-developed novel Nectin-4-targeting ADC (R&D code: 9MW2821) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of esophageal cancer (Press release, Mabwell Biotech, MAY 7, 2024, View Source [SID1234642811]).

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The FDA grants Orphan Drug Designation to treatments for rare diseases in the United States that affect fewer than 200,000 patients. Orphan Drug Designation offers policy benefits to drug developers, including as aid with medication development, tax credits for a part of clinical trial expenditures, and seven years of market exclusivity upon approval.

9MW2821 was previously given Fast Track Designation (FTD) by the FDA for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma. It is the first therapeutic drug candidate targeting Nectin-4 in the world to reveal clinical effectiveness data for an esophageal cancer indication.

About 9MW2821

9MW2821 is the first site-specific conjugated novel Nectin-4-targeting ADC developed by Mabwell using ADC platform and automated high-throughput hybridoma antibody molecular discovery platform, and is the first drug candidate to enter clinical study among the Nectin-4-targeting ADCs developed by Chinese companies. Multiple clinical studies of 9MW2821 have been conducted in China to evaluate the safety, tolerability, pharmacokinetic characteristics, and efficacy of 9MW2821 in patients with various advanced solid tumors.

The phase III clinical study of 9MW2821 monotherapy has officially been initiated in patients with locally advanced or metastatic urothelial carcinoma who have previously received platinum-based chemotherapy and PD-(L)1 inhibitor therapy. The phase I/II clinical study of 9MW2821 in combination with PD-1 inhibitors is also ongoing, with the first patient already enrolled. 9MW2821 was granted Fast Track Designation (FTD) and Orphan Drug Designation (ODD) by the FDA. Currently, 9MW2821 is the first therapeutic drug candidate targeting Nectin-4 in the world to disclose clinical efficacy and safety data for indications of cervical cancer and esophageal carcinoma.