Yingli Pharma and MD Anderson Initiate Strategic Collaboration to Advance Development of Multiple Oncology Programs

On January 20, 2022 Yingli Pharma US, Inc. (the "Company" or "Yingli"), a biotechnology company developing innovative small molecules, and The University of Texas MD Anderson Cancer Center reported a strategic alliance to advance several oncology programs from preclinical discovery through clinical development (Press release, Yingli Pharmaceutical, JAN 20, 2022, View Source [SID1234606682]).

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The five-year collaboration brings together Yingli’s experience in medicinal chemistry with MD Anderson’s comprehensive clinical and translational resources to drive existing Yingli therapeutic candidates into four U.S.-based clinical trials and to facilitate preclinical discovery and development in additional indications.

"This tremendous opportunity will accelerate our efforts as we look to bring new pharmacological agents to cancer patients in need," said Michael Hui, MBA, chief executive officer of Yingli. "We look forward to working with the experts at MD Anderson, complementing our own expertise as we continue to build a well-rounded oncology pipeline."

Two of the programs supported by the collaboration will leverage favorable clinical trial findings collected from China-based studies. The first program will focus on linperlisib, a PI3Kδ inhibitor with a novel structure, which has had positive results in eight active or completed Phase 1 and Phase 2 clinical trials for patients with lymphomas and solid tumors, including peripheral T cell lymphoma (PTCL).

"We are pleased to be working with Yingli to initiate a Phase 2 clinical trial evaluating linperlisib in patients with PTCL," said principal investigator Swaminathan Iyer, M.D., professor of Lymphoma & Myeloma at MD Anderson. "This collaboration brings a potentially effective treatment option with a favorable safety profile to the U.S. to address an unmet need for patients with this aggressive cancer. I look forward to leading this exciting study."

The second program will develop YL-13027, a novel, oral TGFβR1 inhibitor for use in advanced solid tumors with plans to initiate a U.S.-based Phase 1 clinical trial in 2022, led by Jordi Rodon, M.D., Ph.D., associate professor of Investigational Cancer Therapeutics at MD Anderson.

"The TGFβ pathway is an important immune regulator in the tumor microenvironment, and new treatments focused on enhancing anti-tumor immunity are desperately needed," Rodon said. "We look forward to working with Yingli to conduct the first U.S.-based clinical trial evaluating their TGFβR1 inhibitor."

In addition to advancing existing clinical programs, the collaboration also will focus on translational studies to further Yingli’s discovery programs. The collaborative approach will provide a steady stream of new therapeutic candidates for oncology clinical development, which the MD Anderson team, including David S. Hong, M.D., deputy chair of Investigational Cancer Therapeutics, will investigate.

About Linperlisib
Linperlisib (YY-20394) is a highly selective and potent PI3Kδ inhibitor that has shown a favorable safety profile, encouraging anti-tumor activities and promising PK and pharmaceutical properties as an oral once-a-day agent in late-stage clinical development. A Phase 1 clinical trial was completed in 2020 demonstrating linperlisib to be a safe and tolerable agent, and a recommended Phase 2 dose of 80 mg QD was established. Linperlisib was awarded NMPA Breakthrough Therapy status in China, leading to a Phase 2 study in follicular lymphoma (FL) that was completed and submitted in 2021 to China marketing authority for approval. In addition, linperlisib received FDA Orphan Drug Designations for FL, CLL/SLL and T cell lymphoma. Multiple linperlisib clinical trials being conducted in FL, PTCL, other lymphomas, solid tumors, combination with gemcitabine/oxaliplatin in r/r DLBC and combination with therapeutic PD1 antibody are underway in China and the U.S. Preliminary results of single agent linperlisib from a relapsed or recurrent PTCL Phase1b study were reported at ASCO (Free ASCO Whitepaper) 2021, indicating an overall response rate of 70% with 33% CRs for this difficult to treat and aggressive form of lymphoma.

About YL-13027
YL-13027 is a potent small molecule antagonist of TGFβR1 kinase in early-stage clinical development. The compound was optimized with selectivity against TGFβR2 and other kinases, differentiating YL-13027 from other inhibitors blocking TGFβ signaling. In preclinical studies, YL-13027 demonstrated anti-tumor efficacy, tumor immune modulation and combination benefit with immunotherapy. In IND-enabling studies, YL-13027 showed reliable dose-proportionality and pharmacokinetic properties, and the agent proved to be safe and tolerable in toxicity studies. The preliminary results of a Phase 1 dose escalation clinical trial of single agent YL-13027 were reported at ASCO (Free ASCO Whitepaper) 2021, including the safety and efficacy findings that the open-label study is continuing in order to establish a recommended Phase 2 dose of YL-13027.

Tachyon Presents New Data Supporting the Development of TACH101, a Novel KDM4 Inhibitor, as a Potential Therapy for Gastrointestinal Cancers at the 2022 ASCO-GI Conference

On January 20, 2022 Tachyon Therapeutics, Inc. ("Tachyon" or "the Company"), a private biotechnology company creating novel therapeutics to unlock new pathways to treat advanced cancers, reported the presentation of data from its TACH101 program in a virtual poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancer Symposium being held from January 20-22, 2022 (Press release, Tachyon Therapeutics, 20 20, 2022, View Source [SID1234605673]).

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The data demonstrate the potent anti-cancer activity of TACH101, a first-in-class small molecule inhibitor of KDM4 histone demethylase, in preclinical models of gastrointestinal (GI) cancers. KDM4 is a novel target for cancer therapy, playing an important role in the self-renewal of cancer stem cells and regulating epigenetic processes. Overexpression of KDM4 can lead to inhibition of apoptosis, genetic instability, uncontrolled gene expression and cell proliferation, and metastasis. The Company plans to initiate a first-in-human Phase 1 clinical trial in the first half of 2022.

"These preclinical data for TACH101 highlight its potent anti-tumor activity in gastric, esophageal, and colorectal xenograft models and provide support for KDM4 as an important new target for cancer therapy," said Frank Perabo, MD, PhD, CEO of Tachyon Therapeutics. "There is a significant unmet need for new therapeutic options for patients with GI cancers, and we are excited about advancing TACH101 into a first-in-human clinical trial and develop it as a potential new treatment for GI cancers."

Highlights from the ASCO (Free ASCO Whitepaper)-GI poster presentation (Abstract #132) are summarized below:

TACH101 showed potent anti-proliferative activity in GI cancer cell lines and organoid models with IC50 as low as 0.001 µM.
Further evaluation in a panel of colorectal cancer (CRC) patient-derived xenograft (PDX) and organoid models showed a strong correlation of TACH101 sensitivity with MSI-H status (IC50 ranging from 0.001 – 0.270 µM).
TACH101 induced apoptosis in human CRC (HT-29) and esophageal (KYSE-150) cancer cell lines with EC50s of 0.033 μM and 0.092 μM, respectively.
In vivo, TACH101 triggered effective tumor control (≥70%) in xenograft models of CRC (SU60), esophageal (KYSE-150) and gastric (GXA-3036) cancers.
TACH101 treatment caused 86% repression of PNUTS mRNA (a direct target of KDM4) as well as a 51% increase in H3K9me3 (a mark of repressed transcription).
The virtual poster presentation titled, "TACH101, a First-in-Class KDM4 Inhibitor for Treatment of Gastrointestinal Cancers," is available for on-demand viewing beginning at 9 am EST on January 20 by conference attendees on the ASCO (Free ASCO Whitepaper)-GI conference website at View Source

Aptorum Group Receives FDA Orphan Drug Designation for its SACT-1 Repurposed Drug For The Treatment of Neuroblastoma

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

Bluestar Genomics Presents Positive Results of its Pancreatic Cancer Test Performance Evaluation at 2022 ASCO GI Cancers Symposium

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.

ADC Therapeutics to Host Webcast Highlighting its ADC Platform and Solid Tumor Pipeline on February 8, 2022

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.