On February 10, 2020 Sirnaomics, Inc., a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported that the Office of Orphan Product Development division of the FDA has granted orphan drug designation to its leading therapeutic candidate, STP705, for the treatment of Hepatocellular Carcinoma (HCC) (Press release, Sirnaomics, FEB 10, 2020, View Source [SID1234554110]). This is the third such designation for this drug candidate, following the designation for Primary Sclerosing Cholangitis and Cholangiocarcinoma.

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Sirnaomics lead product candidate, STP705 is a siRNA (small interfering RNA) therapeutic that takes advantage of the Company’s proprietary dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. Sirnaomics has opened Investigational New Drug (IND) applications under the jurisdiction of the US FDA and Chinese FDA for clinical studies for Non-melanoma skin cancers (NMSC), Cholangiocarcinoma (CCA), and Hypertrophic Scar (HTS) Reduction.

"We are very pleased to reach another significant milestone and very excited to develop our lead product candidate for treatment of HCC with tremendous unmet needs" stated Patrick Lu, PhD, President and CEO. "This third grant of orphan drug designation for STP705 to treat HCC further consolidates our long-term strategy positioning us to take advantage of the regulatory arbitrage by achieving approval in US and then moving to the large market in China."

"Receipt of the orphan drug designation for Hepatocellular Carcinoma is a very important step for Sirnaomics. It provides the pathway allowing us to develop STP705 in a devastating oncology disease for which there is currently no effective therapy," stated Dr. Michael Molyneaux, MD, MBA, Chief Medical Officer. "This orphan drug designation aligns with our mission to alleviate human suffering and target diseases with high unmet clinical need and we anticipate the start of our clinical study for HCC in the second half of 2020."

About STP705 (Cotsiranib)
Sirnaomics leading product candidate, STP705, is an siRNA (small interfering RNA) therapeutic which takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product candidate has received multiple IND approvals from both the US FDA and Chinese NMPA, including treatments of Cholangiocarcinoma, Non-Melanoma skin cancer and Hypertrophic Scar. STP705 has also received Orphan Drug Designation for treatment of Cholangiocarcinoma and Primary Sclerosing Cholangitis. Preclinical animal models using STP705, have demonstrated a dramatic improvement in T-cell penetration into tumors in the liver with single agent action as well as improvement in the efficacy of an anti-PD-L1 antibody checkpoint inhibitor in an HCC model. This effect may improve other immune checkpoint inhibitor efficacies in addition to those targeting the PD-1/PD-L1 axis.

About Hepatocellular carcinoma (HCC)
Hepatocellular carcinoma (HCC) is the most common type of primary liver cancer in adults, and is the most common cause of death in people with liver cirrhosis. It occurs in the setting of chronic liver inflammation, and is most closely linked to chronic viral hepatitis infection (hepatitis B or C) or exposure to toxins such as alcohol or aflatoxin. Certain diseases, such as hemochromatosis and alpha 1-antitrypsin deficiency, markedly increase the risk of developing HCC. Metabolic syndrome and NASH are also increasingly recognized as risk factors for HCC. Worldwide, approximately 840,000 people are diagnosed with primary liver cancer each year and about 500,000 of the patients are in China, even though HCC is designated as an orphan drug indication by US FDA. Abundant literature has directly linked high level expression of TGF-β1 to liver epithelial to mesenchymal transition (EMT) and tumorigenesis. Inhibition of COX-2 is able to reverse the drug resistance to the chemo drug treatment of HCC and also increased CD4+ T cells within the tumor.

On February 10, 2020 Bold Therapeutics Inc., a clinical-stage biopharmaceutical company developing novel anti-cancer therapies, reported that it has received clearance from Health Canada to initiate a Phase 1b trial of its first-in-class anti-resistance therapeutic, BOLD-100, in combination with FOLFOX for the treatment of gastric, pancreatic, colorectal and bile duct cancers (Press release, Bold Therapeutics, FEB 10, 2020, View Source [SID1234554127]).

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"Based on compelling preclinical efficacy in combination with a wide range of anti-cancer agents, we are excited about the prospects of BOLD-100 in combination with FOLFOX," said Jim Pankovich, EVP of Clinical Development. "The combination of exciting preclinical data in a range of challenging solid tumor models, including gastric, pancreatic, triple-negative breast, and lung cancers, as well an encouraging safety profile seen in early clinical trials, supports the continued clinical development of BOLD-100. I am hopeful that these results translate into improved outcomes for patients, significantly advancing treatment of these devastating diseases."

Bold Therapeutics Inc., a Canadian federal corporation, was founded in 2018 by a team of biopharma industry veterans to develop and commercialize BOLD-100, a first-in-class anti-resistance therapeutic that appears to significantly enhance the activity of a wide range of other anti-cancer therapies by disabling a critical and previously untargeted resistance, survival and proliferation pathway common across cancers. Based on compelling preclinical and early clinical results, Bold Therapeutics is focusing its development efforts on some of the most challenging cancer indications where existing therapies are largely ineffective, resulting in a significant unmet medical need.

"As Bold Therapeutics’ lead investor in its Series A round, we are pleased to see BOLD-100 advance further into the clinic," said Glenn Walthall, Chairman of Bold Therapeutics’ Board and Chief Investment Officer of Gaston Capital Partners. "Bold’s team continues to execute, and we look forward to the achievement of other key development and commercialization milestones in 2020 and beyond."

"In 2019, the Bold Therapeutics team built a strong foundation for rapid growth and development: raising capital, engaging with potential development partners, completing cGMP manufacturing of BOLD-100, establishing collaborations with leading academic institutions to further elucidate the mechanism of action of BOLD-100, and finalizing a protocol that should efficiently determine safety and preliminary efficacy in the treatment of GI cancers," said E. Russell McAllister, CEO. "I look forward to an exciting, data-driven 2020, with preliminary results from our pioneering Phase 1b trial expected by year-end."

On February 9, 2020 KSQ Therapeutics, a biotechnology company using its proprietary CRISPRomics discovery platform to systematically screen the whole genome to identify optimal gene targets for oncology and autoimmune disease, reported the identification and validation of a novel target, CT-1, for the development of engineered tumor infiltrating lymphocyte (eTIL) therapies for refractory solid tumors. Data from two large-scale CRISPR-Cas9 functional screens using the company’s novel CRISPRomics platform and in vivo validation data will be presented at the Engineering the Genome conference, which takes place in Banff, Alberta, Canada (Press release, KSQ Therapeutics, FEB 9, 2020, View Source [SID1234554056]).

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"Adoptive cell therapies have long been hypothesized to be potentially curative treatments for refractory solid tumors, but their efficacy has been limited by the immunosuppressive tumor microenvironment," said Frank Stegmeier, Chief Scientific Officer of KSQ Therapeutics. "Our CRISPRomics platform enables us to identify gene targets that improve the ability of the T cell to function in this hostile tumor microenvironment. These insights allowed us to develop a pipeline of CRISPR/Cas9 eTIL programs that have the potential to unlock adoptive cell therapy in PD-1 refractory solid tumors."

KSQ’s proprietary CRISPRomics platform was used to identify the top targets across the T- cell genome that increase the efficacy of adoptive T cell transfer therapy (ACT) in PD-1 refractory mouse solid tumor models. CT-1, an undisclosed target identified by KSQ, emerged as a top target from these screens. CT-1-edited T cells produced a 10-fold increase in anti-tumor activity in vivo, and CT-1 edited human TILs exhibit an enhanced cytokine production profile. The data presented support the development of eTIL products with the potential to increase the efficacy of TIL adoptive cell therapy.

On February 9, 2020 Oncoheroes Biosciences Inc. ("Oncoheroes"), a biotech company exclusively focused on the development of innovative medicines to treat cancer in children and adolescents, reported that it has closed the first investment with Dreamers Startup Ventures (Press release, Oncoheroes Biosciences, FEB 9, 2020, View Source [SID1234568290]). Under the terms of the agreement, Dreamers Startup Ventures has committed to invest up to $1.1M, disbursed in installments subject to Oncoheroes milestone achievements in the coming months.

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The agreement goes beyond a traditional VC investment. Ricardo Garcia, Oncoheroes’ CEO, stated: "This is a great opportunity for Oncoheroes to take advantage of Dreamers Startup Ventures hands-on approach to support our efforts in all directions. We feel honored and acknowledge the responsibility of being the first investment of Dreamers Startup Ventures in the biotech sector."

Cesare Spadoni, Oncoheroes’ Chief Operations Officer added: "This funding is an important financial achievement for Oncoheroes and enables us to focus on continue working on the clinical development plan for volasertib, our first asset in-licensed from Boehringer Ingelheim, and identify two new assets and complete our pipeline".

Gorka Fius, cofounder of Dreamers Startup Ventures further notes: "From the beginning, we realized that Oncoheroes holds great potential as a biotech company, which made it a very strategic investment for us. The first reason that led us to invest in Oncoheroes is the uniqueness of being 100% focused on developing drugs for pediatric cancer, an estimated niche market of $5-10B/year. We also value the multi-asset portfolio strategy that contributes to diversifying our investment risk and the fact they already have volasertib, a very strong drug candidate in their pipeline. Moreover, Oncoheroes’ first-class team has wide expertise over all of the drug development spectrum, a clear strategy, and a very well-defined road map. Finally, we are confident that we are diminishing the risk of our first operation in the biotech sector by investing in the rare disease space, where normally the cost to develop a drug is lower and the time to reach the market is faster."

On February 7, 2020 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the Guggenheim Healthcare Talks Idea Forum on Thursday, February 13, 2020 (Press release, Eli Lilly, FEB 7, 2020, View Source [SID1234554032]). Jacob Van Naarden, chief operating officer for Loxo Oncology at Lilly; Eric Dozier, vice president, Lilly Oncology North America; and Maura Dickler, M.D., vice president, oncology late phase development, will participate in a fireside chat at 1:00 p.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.