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PTC Therapeutics to Host Conference Call to Discuss Third Quarter 2019 Financial Results

On October 15, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the Company will host a webcast conference call to report its third quarter 2019 financial results and provide an update on the company’s business and outlook on Tuesday, October 29, 2019 at 4:30 p.m. (ET) after closing of the market (Press release, PTC Therapeutics, OCT 15, 2019, https://www.prnewswire.com/news-releases/ptc-therapeutics-to-host-conference-call-to-discuss-third-quarter-2019-financial-results-300938644.html [SID1234542280]).

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The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 9224968. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

Denovo Biopharma Receives FDA’s Permission to Proceed with a Biomarker-Guided Phase 2b Clinical Trial with DB102 (Enzastaurin) In First-Line Treatment of Glioblastoma (GBM)

On October 15, 2019 Denovo Biopharma LLC, a pioneer in applying precision medicine to develop innovative therapies, reported FDA’s approval to initiate Denovo’s Phase 2b clinical study of DB102 in patients with newly-diagnosed glioblastoma (GBM) in combination with radiation and temozolomide (Press release, Denovo Biopharma, OCT 15, 2019, View Source [SID1234542279]).

Denovo’s GBM clinical study is an extension of its work to identify a genetically-enriched patient population with DLBCL who may benefit from DB102 treatment. After Denovo acquired DB102 from Eli Lilly & Co., it discovered a novel genetic biomarker, DGM1, that is a potentially predictive biomarker for DB102 response in patients with DLBCL. Since DGM1 is a germline biomarker, Denovo found that DGM1 also predicts a survival benefit in patients with GBM treated with DB102 plus temozolomide. Denovo’s clinical trial is expected to enroll approximately 200 patients with newly-diagnosed GBM.

"GBM remains one of the toughest cancers to treat and numerous attempts have failed including anti-PD-1 antibodies. DB102 treatment guided by the novel DGM1 biomarker could potentially provide a breakthrough for this severely unmet medical need," said Lei Zhang, M.D., Denovo Biopharma’s Chief Medical Officer. "We are very excited to receive FDA’s approval of our IND and begin to initiate this potentially pivotal GBM study."

About Glioblastoma

Glioblastoma Multiforme (GBM) is the most common type of adult primary malignant brain cancer, with 18,000 newly-diagnosed patients in the US and 13,000 deaths annually. Standard treatment for patients with newly diagnosed GBM can include surgery followed by radiation and chemotherapy, but treatment options are limited. The five-year survival rate of patients with GBM is less than five percent.

McKesson and Aetion Collaborate to Advance Cancer Research With Real-World Data

On October 15, 2019 Aetion and McKesson reported a strategic collaboration focused on advancing the use of real-world evidence (RWE) in cancer research to benefit patients, regulators, the biopharma industry, and payers (Press release, McKesson, OCT 15, 2019, View Source [SID1234542278]). The partnership will provide best-in-class solutions in multiple tumor types, including breast, lung, and melanoma cancers.

The joint solutions combine the Aetion Evidence Platform with data from McKesson’s iKnowMedSM oncology electronic health record (EHR) system to power regulatory-grade outcomes research. They will first be made available to the team of researchers at Brigham and Women’s Hospital who are leading the Food and Drug Administration (FDA) demonstration project, RCT DUPLICATE, in order to replicate oncology randomized controlled trials (RCTs) with real-world data. Aetion and McKesson are also both part of the Friends of Cancer Research Real-World Evidence pilot project to assess several frontline treatment regimens in real-world patients with advanced non-small cell lung cancer (aNSCLC), which will inform the FDA’s acceptance of real-world evidence for drug approvals.

"This collaboration is an important step in informing rigorous standards for regulatory-grade data and analysis. Our goal is to get therapies to market faster to help life sciences companies and drive better health for patients," said Derek Rago, vice president and general manager of Oncology Data, Evidence, and Insights at McKesson Life Sciences. "Based on the ability of observational studies to gain insight into treatment patterns and outcomes in clinical practice, the FDA is supportive of the expanded use of RWE to inform a variety of regulatory decisions."

The solutions allow users to conduct regulatory-grade studies in major cancers and accelerate time to insight on oncology data. It also enables researchers to develop evidence for synthetic control arm and label expansion studies, to fulfill post-marketing safety requirements, and to assess the economic impact of treatments.

"Combining our real-world evidence platform with McKesson’s cancer data accelerates time to insight when conducting oncology analyses," said Carolyn Magill, CEO of Aetion. "This collaboration enables us to provide researchers with the tools to advance cancer research with greater efficiency and precision."

McKesson’s iKnowMed was named the top-ranked EHR platform for oncologists and hematologists for the eighth year in a row by Black Book Research and is ONC Health IT certified. By joining Aetion’s scientifically-validated RWE platform with McKesson’s best-in-class data, the offering enables faster and more cost-effective regulatory-grade cancer research.

Expanding the possibilities for cost-effective cancer research through RWE meets a pressing need. In 2018, biopharma invested $50 billion to support oncology research and development, with an approximate three percent probability of success for any individual product. Real-world evidence can provide critical information about treatments while reducing the costs and time of developing a drug.

Abalos Therapeutics Launches with €12M Series A Round to Develop Novel Immuno-Virotherapies Against Cancer

On October 15, 2019 Abalos Therapeutics reported a EUR 12 million Series A financing round establishing its operations and leadership (Press release, Abalos Therapeutics, OCT 15, 2019, View Source [SID1234542275]). The Company’s objective is to develop new immuno-oncology therapeutics based on a specific arenavirus strain that preferentially infects and proliferates in cancer cells generating a strong anti-tumor immune response. The Series A round was co-led by Boehringer Ingelheim Venture Fund (BIVF) and Gruenderfonds Ruhr, with participation from NRW.BANK and High-Tech Gruenderfonds (HTGF). Representatives from all investors will join the company’s newly formed Supervisory Board.

Concurrent with the financing, Abalos announced the appointment of experienced entrepreneurs Dr. Marcus Kostka as Chief Executive Officer and Dr. Jörg Vollmer as Chief Scientific Officer.

"Abalos’ goal is to capture the potent immune activation and highly specific tumor tropism of the arenavirus to propel a differentiated immuno-oncology approach towards clinical evaluation," said Dr. Marcus Kostka, CEO of Abalos Therapeutics. "After funding and supporting a range of biotechnology companies over the years, this promising technology and the chance to build a company with Jörg as a highly-experienced immunology expert made this opportunity extremely attractive."

The proceeds from the financing will be used to advance Abalos’ arenavirus-based lead candidates towards clinical testing. The Company will develop the initial product candidates using its proprietary Fast Evolution platform, which will generate virus strains with optimized anti-tumoral properties. These candidates will be selected based on their ability to re-program the immune system to specifically and efficiently eliminate the malignant tumor tissue. The approach is based on the innovative research of immunologists Prof. Dr. Karl Lang, Chair of Immunology at the Medical Faculty, University Duisburg-Essen and Prof. Dr. Philipp Lang, Director of the Department of Molecular Medicine II, University Düsseldorf. Abalos builds on a close collaboration with the Universities Duisburg-Essen and Düsseldorf.

Dr. Aristotelis Nastos from Gruenderfonds Ruhr commented: "Immuno-oncology continues to make very exciting progress, however, developing drug candidates that can more fully harness the power of the immune system remains a challenge, in particular, addressing not only the main tumor but also distant metastases. We look forward to supporting Abalos’ experienced management team and its dedicated researchers to work on an arenavirus-based solution."

Dr. Marcus Kostka is a highly seasoned industry expert bringing over 20 years and a range of experiences from Boehringer Ingelheim (BI), where he held positions defined by the ability to locate and advance innovation. Most recently, he was Venture Fund Partner at BI and held board positions at several companies and participated in the successful exits of Rigontec and ICD Therapeutics.

In his role as CSO, Dr. Jörg Vollmer brings to Abalos Therapeutics significant knowledge and experience in the areas of immunology, oncology and infectious diseases, which he obtained in various R&D and executive positions over the course of his career. Most recently, Jörg was CSO at Rigontec where he helped advance a novel immuno-oncology treatment approach into the clinic and led the company’s R&D efforts until the acquisition by MSD in 2017.

Aro Biotherapeutics Debuts Lead Centyrin Candidate at 2019 OTS Meeting

On October 15, 2019 Aro Biotherapeutics Company reported that co-founder and Chief Scientific Officer Karyn O’Neil, PhD, debuted ABX300, the company’s lead Centyrin-pan-KRAS siRNA program, during a presentation today at the 2019 Oligonucleotide Therapeutic Society (OTS) meeting in Munich, Germany (Press release, Aro Biotherapeutics, OCT 15, 2019, View Source [SID1234542274]).

In her presentation entitled, "Cell Specific Centyrin Targeting of Oligonucleotides for Cancer," Dr. O’Neil highlighted preclinical data demonstrating ABX300’s efficacy in treating KRAS-driven epithelial tumors. ABX300 comprises the Company’s proprietary Centyrin protein scaffold technology, conjugated to a pan-KRAS small-interfering RNA (siRNA).

KRAS has been one of the most elusive and difficult to drug targets in cancer. Various point mutations can occur in the KRAS oncogene, resulting in persistent KRAS activation, which drives tumor growth. Recent reports on preclinical and clinical results with small molecule KRAS G12C mutant inhibitors have shown promise; however, this mutant represents only about 12 percent of all KRAS mutations. In contrast, ABX300 is designed to address all known KRAS mutations identified in human tumors.

"Tumor cells treated with ABX300 internalized the Centyrin-siRNA conjugate and exhibited potent, sustained knockdown of mutant KRAS RNA and protein that persisted for days, resulting in complete inhibition of proliferation," said Dr. O’Neil. "This long-term intracellular activity reflects the unique stability and biochemical properties of Centyrins inside the cellular microenvironment."

ABX300 contains two Centyrin-based binding domains that allow for selective targeting of the KRAS siRNA to epithelial-derived tumor cells. ABX300 was shown in preclinical studies to selectively reduce KRAS mRNA levels in cell lines with KRAS G12C, G12D, G12S and G12V mutations, providing proof of concept for broad KRAS inhibition with a single therapeutic.

Sue Dillon, PhD, co-founder and Chief Executive Officer of Aro, commented, "Dr. O’Neil’s presentation at the OTS Meeting is an important milestone for Aro Biotherapeutics. As demonstrated by ABX300 in our research to date, Centyrin-siRNA targeting is a powerful approach to addressing disease target genes in specific cell types, broadly expanding opportunities for RNA medicines."

Building a Pipeline of Life Changing Therapies

Centyrins are small, structurally simple, ultra-stable, highly soluble proteins. These characteristics enable the discovery of medicines with new mechanisms of action for cancer and other devastating diseases. This first-of-its-kind combination of properties is designed to address unmet medical needs by targeting drug payloads in high concentration to the site of disease, while lowering the toxicity to non-target organs. The company holds an exclusive worldwide license for research, development, manufacturing and commercialization of Centyrin protein therapeutics.