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.

On May 7, 2024 Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company, reported that its product portfolio of FDA-approved brands delivered combined revenues of $8.5 million during the first quarter of 2024 (Press release, Cumberland Pharmaceuticals, MAY 7, 2024, View Source;company-update-302138742.html [SID1234642810]). The company ended the quarter with $82 million in total assets, $54 million in total liabilities and $27 million of shareholders’ equity.

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"It has been a steady start to 2024 – and despite some challenges the biopharmaceutical sector is facing in the financial markets amid stubborn inflation and stalled interest rate cuts, we remain optimistic about our industry’s fundamentals and future," said Cumberland’s CEO, A.J. Kazimi. "We continue to build our portfolio of innovative and differentiated products here at Cumberland, and our resulting, diversified product line has enabled us to weather external challenges while our team remains responsive to the evolving market. We look forward to sharing an update on our brands, pipeline and partnerships, including a number of growth opportunities."

Recent company developments include:

Caldolor Special Report

In March 2024, a Special Report was published in Anesthesiology News, General Surgery News and Pharmacy Practice News presenting the growing amount of clinical data supporting the use of Cumberland’s Caldolor (ibuprofen) injection as a standard of care for the treatment of pain and fever in adults, children and infants as young as 3 months of age.

Takeaways from the Special Report include:

Intravenous ibuprofen results in significant reduction in temperature compared with placebo in adults and with acetaminophen in pediatric patients.
Administration of the product prior to surgery leads to patients waking up in significantly less postsurgical pain, while also lessening or even eliminating the need for opioids.
The use of intravenous ibuprofen in the hospital emergency department for acute pain can minimize opioid requirements while achieving significant pain control.
Caldolor should be considered a foundation for any multimodal pain regimen.
Pain management has become one of the most common health care problems. As this Special Report states, comprehensive multimodal pain regimens have become key in preventing pain and optimizing pain control, while minimizing the need for opioids. A non-steroidal anti-inflammatory drug, such as Caldolor, can provide a cornerstone for many treatment paradigms.

Federal NOPAIN Act

Cumberland expects Caldolor will be eligible for special Medicare reimbursement under the Non-Opioids Prevent Addiction in the Nation Act (the "NOPAIN Act"), which was enacted as part of the Consolidated Appropriations Act of 2023.

The NOPAIN Act requires Medicare to provide separate and more favorable reimbursement for non-opioid products used to manage pain during surgeries conducted in hospital outpatient departments or in ambulatory surgical centers. The NOPAIN Act applies, in part, to products that are indicated to provide analgesia without acting upon the body’s opioid receptors.

The Centers for Medicare & Medicaid Services ("CMS") requested that manufacturers with potentially applicable non-opioid products submit comments and supporting clinical evidence regarding products that should be eligible for separate payment. Cumberland submitted a comment letter along with the requisite clinical information to the CMS in September 2023 explaining why Caldolor should be included and separately reimbursed. The company now awaits information from CMS regarding the reimbursement status and price for the product.

The Act is scheduled to go into effect in early 2025 and will initially apply to products that are furnished between January 1, 2025 and January 1, 2028.

New Manufacturing & Supplies of Sancuso

After acquiring U.S. rights to Sancuso, Cumberland successfully completed the transition from Kyowa Kirin in 2023, including the NDA transfer. A new facility was approved by the FDA for Sancuso and, during the first quarter of 2024, Cumberland completed the manufacturing of Cumberland-packaged product there. The company will launch these new supplies this year. Meanwhile, it continues to support the product through its expanded oncology sales division to help cancer patients by addressing certain side effects associated with their chemotherapy treatments.

Vaprisol Supply Update

Cumberland’s new manufacturing and distribution partner for Vaprisol is providing a special supply of compounded product in support of critically ill patients. The companies await FDA approval for the facility in order to relaunch the brand.

Vaprisol is the first and only intravenously administered vasopressin receptor antagonist. It is used to raise serum sodium levels in hospitalized patients with hyponatremia, the most common electrolyte disorder among such patients.

Clinical Development Pipeline

Cumberland has been evaluating its ifetroban product candidate, a selective thromboxane-prostanoid receptor antagonist, in a series of clinical studies. It has now been dosed in nearly 1,400 subjects and has been found to be safe and well tolerated in healthy volunteers and various patient populations.

Cumberland has initiated its newest clinical program in medical centers across the country and enrollment has now begun in patients with Idiopathic Pulmonary Fibrosis, the most common form of progressive fibrosing interstitial lung disease. This FIGHTING FIBROSIS trial is designed to enroll 128 patients in over 20 medical centers of excellence across the United States. Recent studies have shown ifetroban can both prevent and enhance resolution of lung fibrosis in multiple preclinical models.

Patient enrollment is already well underway in its two other company-sponsored Phase II clinical programs evaluating ifetroban in patients with: 1) Systemic Sclerosis, or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and internal organs and 2) cardiomyopathy associated with Duchenne Muscular Dystrophy, a rare, fatal, genetic neuromuscular disease results in deterioration of the skeletal, heart and lung muscles.

Cumberland’s plan going forward is to complete each of its company-sponsored studies, analyze their final data, announce top-line results and decide on the best development path for the registration of ifetroban, which the company continues to believe has the potential to benefit many patients with orphan diseases that represent unmet medical needs.

FINANCIAL RESULTS:

Net Revenue: For the three months ended March 31, 2024, net revenues were $8.5 million.

Net revenue by product for the first quarter of 2024 included $3.2 million for Kristalose, $1.8 million for Sancuso, $1.6 million for Vibativ and $1.5 million for Caldolor.

Operating Expenses: Total operating expenses for the first quarter were $10.4 million.

Net Income: The net loss for the first quarter of 2024 was $1.9 million, or $0.14 a share.

Adjusted Earnings: Adjusted earnings for the first quarter of 2024 were a loss of $0.6 million, or $0.05 per share. The adjusted earnings calculation does not include the benefit of the $0.5 million cost of goods for Vibativ and Sancuso during the quarter, which were received as part of each product’s acquisition.

Balance Sheet: At March 31, 2024, Cumberland had $82 million in total assets, including $19 million in cash and cash equivalents.

Total liabilities were $54 million, including $16 million outstanding on the company’s revolving line of credit. Total shareholders’ equity was $27 million at the end of the quarter.

EARNINGS REPORT CALL:

A conference call will be held on May 7, 2024, at 4:30 p.m. Eastern Time to provide a company update and discuss the financial results. To participate in the call, please register at: https://register.vevent.com/register/BIbb2d35bc9570474dabf8b2e3d2e4ca72.

Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.

A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting View Source