On January 20, 2022 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) ("Aptorum Group" or "Aptorum"), a clinical-stage biopharmaceutical company, reported that the United States Food and Drug Administration (FDA) Office has granted Orphan Drug Designation to SACT-1, a repurposed small molecule compound for the treatment of patients with Neuroblastoma (Press release, Aptorum, JAN 20, 2022, View Source [SID1234605672]). Aptorum Group plans to file an Investigational New Drug Application (IND) to commence a phase 1b/2a clinical trial for SACT-1 to test the drug in neuroblastoma patients in 2022.

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Mr. Darren Lui, President and Executive Director of Aptorum Group says, "The granting of orphan drug designation for SACT-1 for the treatment of neuroblastoma is another important step forward in the development of our drug candidate and reflects both the FDA’s and Aptorum’s commitment to addressing the unmet clinical needs of patients with neuroblastoma." Further to our recently announced completion of Phase 1 clinical trial and patent grant for SACT-1, we are currently focusing on our IND preparation for entering into the exciting Phase Ib/2a clinical trials for SACT-1 in the United States."

About SACT-1
SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma. SACT-1’s mechanism has been investigated in our preclinical studies to enhance tumor cell death and suppress MYCN expression (a common clinical diagnosis in high-risk or relapsed neuroblastoma patients where an amplification of MYCN is usually observed). SACT-1 is designed to be used especially in combination with standard-of-care chemotherapy.

About Neuroblastoma
Neuroblastoma is one of the most prevailing solid tumor cancers in children, representing 8% – 10% of all childhood tumors, accounting for c. 15% of all cancer related deaths in the pediatric population1. For the high-risk patient group, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society2 based on existing treatment.

1 View Source,deaths%20in%20the%20pediatric%20population.

2 View Source

On January 20, 2022 Bluestar Genomics, Inc., an early cancer detection company leading the development and commercialization of next-generation liquid biopsy approaches initially focused on non-invasive detection of high-mortality cancers, reported positive performance evaluation study results for its pancreatic cancer test (Press release, Bluestar Genomics, JAN 20, 2022, View Source [SID1234605671]). These data confirm that its pancreatic cancer test detects the disease with 55.2% sensitivity and 98.4% specificity in patients with and without diabetes. The study results will be presented on January 21 at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers (ASCO GI) Symposium in San Francisco.

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The new results underscore the importance of earlier pancreatic cancer detection when effective therapies are still possible. Research shows that one in four people diagnosed with pancreatic cancer were first diagnosed with Type 2 diabetes. For the hundreds of thousands of people with new-onset Type 2 diabetes who are potentially at risk for pancreatic cancer, early pancreatic cancer detection could help prevent a late-stage cancer diagnosis.

"Building on the previous data that led to FDA’s breakthrough device designation for our pancreatic cancer test, we continue to focus on addressing the deadliest of cancers with early detection," said Samuel Levy, Ph.D., chief scientific officer at Bluestar Genomics. "Now, with more than a dozen scientific publications and presentations, the precision of our proprietary 5-hydroxymethylcytosine-based platform is evident."

Bluestar Genomics’ pancreatic cancer test uses a standard blood draw to assess whether an individual has an abnormal epigenomic signal associated with pancreatic cancer. With no existing clinical standard of care for screening for pancreatic cancer, Bluestar Genomics’ groundbreaking epigenomics technology platform employs state-of-the-art machine learning coupled with the DNA-based 5-hydroxymethylcytosine (5hmC) biomarker, as a novel method to detect cancer earlier.

The study, which included 1,433 men and women between 45- and 75-years old from 146 sites in the U.S., evaluated the performance of the test in detecting pancreatic cancer signal in new-onset diabetes using Bluestar Genomics’ proprietary epigenomic platform technology based on plasma-derived cell-Free DNA 5-hmC signatures.

"Pancreatic cancer accounts for only three percent of new cancer cases, yet it is the third leading cause of cancer-related death in the United States," said Kelly Bethel, M.D., practicing pathologist at a major medical center in San Diego, and chief medical officer at Bluestar Genomics. "With pancreatic cancer having the poorest overall survival of all the major cancer types, it is imperative that we change the paradigm of late diagnosis depriving patients of potentially curative treatments."

With the prevalence of diabetes increasing worldwide, it is estimated that by 2025, more than 37 million patients will have been diagnosed with diabetes in the U.S. This growing prevalence highlights the need for a non-invasive early pancreatic cancer detection approach.

On January 20, 2022 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company improving the lives of those affected by cancer with its next-generation, targeted antibody drug conjugates (ADCs) for patients with hematologic malignancies and solid tumors, reported that it will host a webcast highlighting its ADC platform and solid tumor pipeline on Tuesday, February 8, from 9:00 to 11:00 a.m. ET (Press release, ADC Therapeutics, 20 20, 2022, View Source [SID1234605670]).

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Chris Martin, PhD, Chief Executive Officer, Patrick van Berkel, PhD, Senior Vice President of Research & Development, and Joseph Camardo, MD, Chief Medical Officer, will provide insight into the company’s proprietary ADC platform technology and a deep dive into the company’s promising pipeline of programs for the treatment of various solid tumors.

A live webcast will be available via the Events & Presentations page in the Investors section of ADC Therapeutics’ website, ir.adctherapeutics.com. A replay of the webcast will be available for approximately 30 days.

On January 20, 2022 -Pluristyx, Inc., an advanced therapy tools and services biotechnology company, panCELLa, a privately held biotechnology company, and Implant Therapeutics, a developer of genetically engineered stem cells, reported that they have entered into a manufacturing and distribution strategic alliance that offers cell therapy companies streamlined access to the next generation of safe, universal, cost-effective, "off-the-shelf" induced pluripotent stem cells (iPSCs) (Press release, Pluristyx, 20 20, 2022, View Source [SID1234605668]). The agreement will enable commercial access to iPSCs containing panCELLa’s FailSafe and Implant’s hypoimmunogenic technology derived from fully consented and regulatory appropriate donors. Both research and clinical grade panCELLa-owned iPSC lines will be manufactured and distributed by Pluristyx, who will also make custom iPSC lines incorporating panCELLa and Implant technology to meet unique customer needs.

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"We evaluated many manufacturing partners and concluded that Pluristyx’s proprietary platform and mRNA reprogramming technologies provided the ideal complement to our platform technologies. We think this combination of complementary skills will enable wide dissemination of our technology."

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Dr. Mahendra Rao, Chief Executive Officer of Implant, commented, "We evaluated many manufacturing partners and concluded that Pluristyx’s proprietary platform and mRNA reprogramming technologies provided the ideal complement to our platform technologies. We think this combination of complementary skills will enable wide dissemination of our technology."

"Pluristyx is excited to be working with panCELLa and Implant to commercialize their unique gene-edited platforms and iPSC lines. The combination of Pluristyx’s iPSC manufacturing and reprogramming technologies with panCELLa and Implant’s Stealth and FailSafe platforms will provide immediate access to a unique and ideal raw material for making the next generation of cell therapies," said Dr. Benjamin Fryer, Chief Executive Officer of Pluristyx.

On January 20, 2022 Palleon Pharmaceuticals, a company pioneering the field of glyco-immunology to treat cancer and inflammatory diseases, reported that the U.S. Food and Drug Administration has cleared the company’s investigational new drug (IND) application for its lead candidate E-602 (Press release, Palleon Pharmaceuticals, JAN 20, 2022, View Source [SID1234605669]). This clearance paves the way for Palleon’s first-in-class cancer immunotherapy which targets immunosuppressive sialoglycans to enter clinical testing.

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"We are proud to have reached this milestone in advancing glyco-immunology from a promising, nascent scientific field to an entirely new class of medicines," said Jim Broderick, M.D., Chief Executive Officer and Founder of Palleon. "We continue to leverage our unrivaled expertise in glycobiology drug discovery and development to expand our product pipeline in the pursuit of better therapies for patients with cancer and inflammatory diseases."

E-602 is a first-in-class engineered human sialidase enzyme genetic fusion from Palleon’s EAGLE platform which is designed to desialylate both immune cells and tumor cells, potentiating an anti-tumor immune response. Palleon’s Phase 1/2 trial of E-602 is expected to begin enrolling patients with solid tumors refractory to standard of care in Q1 2022.