On September 18, 2024 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported the approval of the Investigational New Drug (IND) to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor ICP-248 in combination with azacitidine for acute myeloid leukemia (AML) in China (Press release, InnoCare Pharma, SEP 18, 2024, View Source [SID1234646730]).

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Acute myeloid leukemia (AML) is a malignant hematological disease originating from hematopoietic stem/progenitor cells, accounting for about 80% of acute leukemia in adults. The risk of developing AML increases with age and is more common in middle-aged and elderly people. AML is also not uncommon in individuals under 18 years old, representing about 15-20% of pediatric leukemia and 80% of leukemia in neonates and infants1.

BCL2 is an important regulatory protein of apoptosis pathway, and its abnormal expression is related to the development of various hematologic malignancies. ICP-248 is a novel, orally bioavailable BCL2-selective inhibitor. It has anti-tumor effect by selectively inhibiting BCL2 and restoring the mechanism of programmed cell death.

Dr. Jasmine Cui, the co-founder, chairwoman and CEO of InnoCare, said, "With strong pipeline in hemato-oncology, InnoCare is dedicated to developing therapeutics with diverse mechanisms of action (MoA) to achieve comprehensive coverage of blood tumor indications. ICP-248 is an important global asset of our company in the field of hematology. We will accelerate clinical development and look forward to bringing greater benefits to patients with hematological malignancies early."

On September 18, 2024 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported the peer-reviewed journal Nature Medicine published results from the SCRUM-Japan GOZILA study confirming that selecting targeted therapy on the basis of Guardant360 CDx liquid biopsy results may significantly extend survival for patients with advanced cancer (Press release, Guardant Health, SEP 18, 2024, View Source [SID1234646731]).

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The study, led by a research group out of National Cancer Center Hospital East in Kashiwa, Japan, investigated the effects of personalized treatment based on results of the Guardant360 CDx test in 4,037 patients with advanced cancer. The results showed that 24% of participants were able to receive targeted treatment tailored to them based on comprehensive genomic profiling results from the test, which analyzes 74 cancer-related genes. The patients who received targeted treatment guided by liquid biopsy results lived approximately twice as long as those who did not.

"Compared to conventional tissue biopsies, liquid biopsies have several advantages: they are less invasive for patients, allow for repeated testing, and can simultaneously examine cancer characteristics from various parts of the body. However, until now, it was unclear whether treatment selection using liquid biopsies actually helped improve patient outcomes," said Yoshiaki Nakamura, M.D., Ph.D., chief, International Research Promotion Office, Department of Gastroenterology and Gastrointestinal Oncology at National Cancer Center Hospital East in Kashiwa, Japan, and a co-lead author of the study. "The GOZILA study is the first to demonstrate the survival-extending effect of liquid biopsy-based personalized cancer treatment on a large scale across various cancers. The results of this study have the potential to bring about a paradigm shift in cancer treatment."

Selecting therapies for patients based on the liquid biopsy results enabled study investigators to identify targeted treatment options they could not discern using traditional methods. The researchers then followed the progress of treated patients and analyzed their treatment response and survival time. Patients who received targeted therapy had a median survival of 18.6 months compared to 9.9 months for those who did not.

"The GOZILA study adds significantly to the body of evidence supporting the clinical utility of the Guardant360 CDx liquid biopsy to guide therapy selection in advanced cancer," said Craig Eagle, M.D., Guardant Health chief medical officer. "These study results confirm, across a large study population and multiple tumor types, that personalized therapy guided by liquid biopsy has the potential to significantly extend patient survival."

About Guardant360 CDx

The first FDA-approved comprehensive liquid biopsy for all advanced solid tumors, Guardant360 CDx provides oncologists with genomic profiling results from a simple blood draw in less than seven days to pair patients with targeted therapies. The test detects guideline-recommended actionable biomarkers across all four major alteration classes, with a panel that assesses 74 genes.1 Guardant360 CDx is FDA-approved as a companion diagnostic (CDx) for multiple targeted therapies in non-small cell lung cancer (NSCLC) and is the only FDA-approved CDx to identify patients eligible for breast cancer therapy targeting ESR1 mutations.

On September 17, 2024 Kairos Pharma, Ltd. ("Kairos Pharma" or the "Company") (NYSE American: KAPA), a clinical stage biopharmaceutical company developing cancer therapeutics designed to reverse cancer drug resistance and immune suppression, reported that it has closed its initial public offering of 1,550,000 shares of common stock at an offering price of $4.00 per share (Press release, Kairos Pharma, SEP 17, 2024, View Source [SID1234646701]). The gross proceeds to the Company from the offering were $6,200,000, before deducting underwriting discounts and commissions and other offering expenses. The Company’s common stock began trading on the NYSE American LLC on September 16, 2024 under the symbol "KAPA."

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The Company intends to use the net proceeds from the offering to fund its Phase 1 trial in lung cancer and Phase 2 trial in prostate cancer for its lead product candidate ENV 105, designed to reverse resistance to cancer drugs. The proceeds will also advance preclinical candidates, including KROS 101, a small molecule agonist for the GITR ligand, designed to promote T cell growth and cytotoxic function against cancer.

The Company has granted to the underwriters a 45-day option to purchase up to an additional 232,500 shares of common stock at the public offering price of $4.00 per share, less underwriting discounts and commissions.

Boustead Securities, LLC acted as lead managing underwriter for the offering, and EF Hutton LLC and Sutter Securities, Inc. acted as co-managing underwriters for the offering.

Dorsey & Whitney LLP served as counsel to the Company and Olshan Frome Wolosky LLP served as counsel to the underwriters.

A registration statement on Form S-1 (File No. 333-274805) relating to the offering was declared effective by the Securities and Exchange Commission (the "SEC") on September 16, 2024. The offering is being made only by means of a prospectus. A preliminary prospectus relating to the offering has been filed with the SEC. Electronic copies of the final prospectus, when available, may be obtained on the SEC’s website at View Source and may also be obtained, when available, by emailing [email protected] or by calling 1-949-502-4408 or by standard mail to Boustead Securities, LLC, Attention: Equity Capital Markets, 6 Venture, Suite 395, Irvine, California 92618.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On September 17, 2024 Novartis reported that the US Food and Drug Administration (FDA) has approved Kisqali (ribociclib) in combination with an aromatase inhibitor (AI) for the adjuvant treatment of people with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) stage II and III early breast cancer (EBC) at high risk of recurrence, including those with node-negative (N0) disease (Press release, Novartis, SEP 17, 2024, View Source [SID1234646702]).

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The approval is based on results from the pivotal Phase III NATALEE trial, which showed a significant and clinically meaningful 25.1% (HR=0.749; 95% CI: 0.628, 0.892; P=0.0006) reduction in risk of disease recurrence in a broad population of patients with HR+/HER2- stage II and III EBC treated with adjuvant Kisqali plus endocrine therapy (ET) compared to ET alone, including those with high-risk N0 disease3-6. The invasive disease-free survival (iDFS) benefit was consistently observed across all patient subgroups3-6.

"The FDA approval of Kisqali for this early breast cancer population, including those with N0 disease, is a pivotal moment in improving our approach to care," said Dennis J. Slamon, M.D., Director of Clinical/Translational Research, UCLA Jonsson Comprehensive Cancer Center and Chairman of the Board of Translational Research In Oncology (TRIO) and NATALEE trial lead investigator. "Today’s approval allows us to offer treatment with a CDK4/6 inhibitor to a significantly broader group of people as a powerful tool that, combined with endocrine therapy, can help further minimize their risk of cancer returning."

In EBC, Kisqali is taken with or without food as a once-daily oral dose of 400 mg (two 200 mg tablets) for three weeks, followed by one week off treatment, in combination with four weeks of any AI1. Patients should take Kisqali for three years. The NATALEE trial showed the safety profile of Kisqali at the 400 mg dose was well tolerated, with discontinuations mainly driven by asymptomatic laboratory findings3. Adverse events (AEs) of special interest in the Kisqali + ET arm of the NATALEE trial include (all Grades, and Grades 3/4, respectively): neutropenia (62.5%, 44.3%), liver-related AEs (26.4%, 8.6%), QT interval prolongation (5.3%, 1.0%), and interstitial lung disease/pneumonitis (1.5%, 0.0%)4.

An updated analysis from the NATALEE trial recently presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 reinforces the data analyzed by the FDA. Results showed a deepening benefit beyond the three-year treatment period and reduced the risk of recurrence by 28.5% (HR=0.715; CI 95% 0.609–0.840; P<0.0001), compared to ET alone, in patients with stage II and III HR+/HER2- EBC7. Novartis will continue evaluating NATALEE patients for longer-term outcomes, including overall survival.

Raising the bar for EBC survivors
Approximately 90% of breast cancer cases in the US are diagnosed early (stages I-III) and treated promptly with curative intent – sometimes with adjuvant ET21,22. In spite of this, people with stage II and III HR+/HER2- EBC remain at risk of cancer coming back – in most cases, as incurable metastatic disease8,9. Recurrence remains a lifelong concern, though most tumors return within the first years, even in cases with no lymph node involvement8,23. Despite ET, 10% of people with high-risk N0 disease may face recurrence within the first three years after diagnosis24.

"With this approval, we are redefining treatment options for a broader population of people impacted by breast cancer and facing the persistent risk of recurrence," said Victor Bultó, President, US, Novartis. "We continue to transform cancer care with Kisqali, building on its established profile in the metastatic setting and now helping a wide range of people as they strive to stay cancer-free following an early-stage diagnosis."

"Breast cancer treatment can take a toll on your physical and mental health, and you may worry about the risk of your cancer coming back. This risk is different for everyone, depending on many factors, but should not be underestimated," said Valarie Worthy, Co-Founder & Vice President of Community Outreach and Engagement, Touch, The Black Breast Cancer Alliance. "The FDA approval of Kisqali for more people with breast cancer is welcome news and empowers people diagnosed with early breast cancer with a new option to help manage and control their risk of cancer coming back."

Novartis prioritizes patient access by offering the Novartis Patient Support program. This resource assists eligible patients in navigating treatment initiation, providing educational materials, clarifying insurance coverage, and identifying potential financial assistance options. For additional information, patients and healthcare professionals can call 1-800-282-7630.

About NATALEE
NATALEE is a global Phase III multi-center, randomized, open-label trial to evaluate the efficacy and safety of Kisqali (ribociclib) with ET as an investigational adjuvant treatment versus ET alone in patients with stage II and III HR+/HER2- EBC, being conducted in collaboration with TRIO25. The adjuvant ET in both treatment arms was a non-steroidal AI (NSAI; anastrozole or letrozole) and goserelin if applicable25. The primary endpoint of NATALEE is iDFS as defined by the Standardized Definitions for Efficacy End Points (STEEP) criteria25. A total of 5,101 adult patients with HR+/HER2- EBC across 20 countries were randomized in the trial25.

About Kisqali (ribociclib)
Kisqali (ribociclib) is a selective cyclin-dependent kinase inhibitor, a class of drugs that help slow the progression of cancer by inhibiting two proteins called cyclin-dependent kinase 4 and 6 (CDK4/6). These proteins, when over-activated, can enable cancer cells to grow and divide too quickly. Targeting CDK4/6 with enhanced precision may play a role in ensuring that cancer cells do not continue to replicate uncontrollably.

Beyond today’s FDA approval of Kisqali for EBC patients in the US, regulatory reviews for Kisqali as an EBC treatment are ongoing worldwide, including in the EU and China.

Kisqali has been approved as a treatment for metastatic breast cancer (MBC) patients in 99 countries worldwide, including by the US FDA and the European Commission1,26. In the US, Kisqali is indicated for the treatment of adults with HR+/HER2- advanced or MBC in combination with an AI as initial ET or fulvestrant as initial ET or following disease progression on ET in post-menopausal women or in men1. In the EU, Kisqali is approved for the treatment of women with HR+/HER2- advanced or MBC in combination with either an AI or fulvestrant as initial ET or following disease progression. In pre- or peri-menopausal women, the ET should be combined with a luteinizing hormone-releasing hormone agonist26.

In MBC, Kisqali has consistently demonstrated statistically significant overall survival benefit across three Phase III trials10-20. The NCCN Guidelines for breast cancer recommend ribociclib (Kisqali) as the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of people living with HR+/HER2- when combined with an AI, making Kisqali the preferred first-line treatment of choice for US prescribers in HR+/HER2- MBC27. Additionally, Kisqali has the highest rating of any CDK4/6 inhibitor on the ESMO (Free ESMO Whitepaper) Magnitude of Clinical Benefit Scale, achieving a score of five out of five for first-line pre-menopausal patients with HR+/HER2- advanced breast cancer28. Further, Kisqali in combination with either letrozole or fulvestrant has uniquely, among other CDK4/6 inhibitors, received a score of four out of five for post-menopausal patients with HR+/HER2- advanced breast cancer treated in the first line29.

Kisqali was developed by Novartis under a research collaboration with Astex Pharmaceuticals.

Please see full Prescribing Information for Kisqali, available at www.Kisqali.com

On September 17, 2024 Rakovina Therapeutics Inc. (TSX-V: RKV), a biopharmaceutical company committed to advancing new cancer therapies based on novel DNA-damage response technologies, and Variational AI, developer of the Enki generative artificial intelligence (AI) platform for drug discovery, reported a research collaboration aimed at leveraging each company’s expertise to identify and develop novel small-molecule therapies against DNA-damage response (DDR) targets for the treatment of cancer (Press release, Rakovina Therapeutics, SEP 17, 2024, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-and-variational-ai-announce-drug-discovery-collaboration [SID1234646703]).

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Under the terms of the agreement, Rakovina Therapeutics has been granted an exclusive right to compounds generated by the Enki platform against the selected target product profiles and an option to license validated drug candidates for further development. Variational AI will employ the Enki platform to identify novel inhibitors of specific DDR kinase targets selected by Rakovina Therapeutics. Rakovina Therapeutics will synthesize and evaluate the viability of these drug candidates as potential cancer therapies in its laboratories at the University of British Columbia.

According to Rakovina Therapeutics Executive Chairman Jeffrey Bacha, "This collaboration is an ideal addition to our already established Deep Docking AI partnership as it expands Rakovina Therapeutics’ pipeline beyond our current focus of developing next-generation PARP inhibitors."

"Leveraging Variational AI’s expertise in kinases where it overlaps with our DDR interest will significantly increase partnering opportunities as ‘big pharma’ maintains a close interest in novel therapies against these targets," added Bacha.

Rakovina Therapeutics will pay a low upfront fee to initiate work against each selected target and a "TPP Fee" upon exercise of its option to acquire rights to drug candidates meeting a pre-specified target-product profile (TPP) and additional payments upon achievement of specified development milestones. Additional financial terms were not disclosed.

According to Variational AI CEO Handol Kim, "We are thrilled to be partnering with Rakovina Therapeutics in their quest for lifesaving cancer drugs. Variational AI is at the forefront of Artificial Intelligence drug discovery, and we look towards a successful and lasting partnership. Our unique Enki platform is the first commercially accessible foundation model for small molecules and represents an ideal drug discovery solution for Rakovina Therapeutics for these initial targets and beyond."

Bacha said, "Variational AI will deliver a short list of potential drug candidates within four to six weeks of target selection. We anticipate determining the validity of these novel drug candidates against our target-product profile within 12 to 18 months upon receipt of each Variational AI deliverable."

"We are excited about the potential of this collaboration. The Enki AI platform, already used by leading pharmaceutical companies, brings a powerful addition to our AI-driven drug discovery toolkit as we expand our research focus to additional DDR targets," Bacha added.