On June 24, 2021 Aileron Therapeutics (NASDAQ:ALRN), a chemoprotection oncology company focused on fundamentally transforming the experience of chemotherapy for cancer patients, reported that it is set to join the Russell Microcap Index at the conclusion of the 2021 Russell indexes annual reconstitution, effective after the U.S. market opens on June 28, 2021 (Press release, Aileron Therapeutics, JUN 24, 2021, View Source [SID1234584309]).

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Membership in the Russell Microcap Index, which remains in place for one year, means automatic inclusion in the appropriate growth and value style indexes. FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings and style attributes.

"Protection against chemotherapy-induced toxicities represents a significant, largely unaddressed need in the oncology space, and one for which there is now increasing progress and momentum," said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer at Aileron. "Precision medicine has truly revolutionized cancer treatment. In line with that transformation, our use of the p53 biomarker gives us the potential to selectively chemoprotect healthy cells in a very large number of patients given that nearly one million cancer patients are annually diagnosed with a p53-mutated cancer in the United States alone. Our inclusion in the Russell Microcap Index will expand our investor base as we work to transform the experience of cancer patients undergoing chemotherapy."

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $10.6 trillion in assets are benchmarked against Russell’s US indexes. Russell indexes are part of FTSE Russell, a leading global index provider. For more information on the Russell Microcap Index and the Russell indexes reconstitution, go to the "Russell Reconstitution" section on the FTSE Russell website.

On June 24, 2021 Sirnaomics, Inc., a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, reported that dose administration for the first patient in a Phase 1 U.S. clinical study for liver cancer treatment, with the company’s siRNA (small interfering RNA) drug candidate, STP705 (Press release, Sirnaomics, JUN 24, 2021, View Source [SID1234584326]). STP705 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.

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This Phase 1 trial is a multicenter, open-Label, dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of STP705, with an intratumoral administration. In this "basket study" of up to 50 subjects suffering from cholangiocarcinoma, hepatocellular carcinoma, or liver metastases from colorectal cancer, the patients with advanced/metastatic or surgically unresectable solid tumors and are refractory to standard therapy will be treated with STP705. This therapeutic regimen is designed to take advantage of a dual-targeted inhibitory property of siRNAs and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expressions. Early pre-clinical and clinical studies using STP705 have shown an increase of active T cell infiltration into the tumor microenvironment. In addition, knocking down TGF-β1 and COX-2 gene expressions in animal fibrosis tissue can activate fibroblast apoptosis with significant antifibrotic efficacy.

"Advancing STP705 from skin cancer to liver cancer is a major milestone for Sirnaomics’ clinical programs, especially with a basket study design that consists of multiple tumor types. We are particularly interested in learning whether the mechanism of action validated in the skin cancer clinical study can be further verified in this liver cancer study," said Patrick Lu, Ph.D., founder, President and Chief Executive Officer of Sirnaomics. "We are expecting that the combination of anti-fibrotic effects and enhanced tumor immunity will provide a novel approach for the treatment of cholangiocarcinoma and hepatocellular carcinoma using our novel siRNA therapeutics. Sirnaomics is committed to advancing our polypeptide nanoparticle delivery system for innovative RNAi-based cancer therapy."

"Liver cancer is a devastating disease for patients with high mortality and high unmet medical need," stated Michael Molyneaux, MD, Chief Medical Officer of Sirnaomics. "The company is excited to announce first dosing, as we hope to gain important insight into the potential safety and efficacy of STP705 in this Phase 1 trial and build on the data from this study to expand into other oncology indications."

Sirnaomics expects to report initial clinical data from the Phase 1 trial in the second half of 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04676633

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About Liver Cancer

Liver cancer is a global health problem, with liver neoplasms representing the second-most frequent cause of cancer-related death. There are many different types of liver cancers including hepatocellular carcinoma (HCC), cholangiocarcinoma (CCA), liver angiosarcoma, hepatoblastoma and others. Additionally, liver is a highly metastasis-permissive organ. It is the most frequently afflicted organ by metastasis and liver metastases are much more common than primary hepatic tumors. The distinctive biology of the liver renders it intrinsically susceptible to metastases. The true prevalence of liver metastasis is unknown, but between 30% and 70% of patients dying of cancer have liver metastases and most patients with liver metastases will die of their disease.

STP705 and Liver Cancer

Over expressions of TGF-β1 and COX-2 have been well-characterized as playing key regulatory roles in tumorigenesis. TGF-β is produced by different liver cells and is demonstrated to induce tumor cell migration and survival. TGF-β has been found to be overexpressed in metastatic HCC tissues. Overexpression of TGF-β is generally accepted to be associated with metastasis and poor prognosis. COX-2 is reported to be highly expressed in cancer stem cells and promotes cell migration in HCC cell lines. Additionally, inhibition of COX-2 suppresses cell migration and induces apoptosis. As such TGF-β1 and COX-2 are excellent therapeutic targets for treatment of liver cancer.

STP705 is composed of two siRNA oligonucleotides targeting TGF-β1 and COX-2 mRNA respectively and formulated in nanoparticles with a proprietary Histidine-Lysine Co-Polymer (HKP) peptide. Each individual siRNA has demonstrated the ability to inhibit the expression of their target mRNA and combining the two siRNAs produces a synergistic effect that diminishes pro-fibrogenic, pro-inflammatory, and pro-tumorigenic factors. Sirnaomics has completed several pre-clinical studies that demonstrate that inhibition of TGF-β1 and COX-2 is expected to result in the inhibition of tumor growth and provide an alternative approach for the treatment of various liver cancers. Molecular analyses of the effects of administering the combination demonstrated that the inhibition of these targets had effects on downstream gene products associated with numerous oncology targets.

Additional immunohistochemistry and image analyses of the liver and tumor tissues demonstrated that animals treated with STP705 resulted in increased CD4+ and CD8+ T cell infiltration within the tumor microenvironment. Using STP705 for treatments of hepatocellular carcinoma and cholangiocarcinoma have been designated as Orphan Drug indications by U.S. FDA. STP705 has also been evaluated in a Phase 2a clinical trial for treatment of Non-melanoma skin cancer.

On June 24, 2021 Sirnaomics, Inc., a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, reported dose administration for the first patient in a Phase 1 U.S. clinical study for liver cancer treatment, with the company’s siRNA (small interfering RNA) drug candidate, STP705 (Press release, Sirnaomics, JUN 24, 2021, View Source [SID1234584342]). STP705 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This Phase 1 trial is a multicenter, open-Label, dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of STP705, with an intratumoral administration. In this "basket study" of up to 50 subjects suffering from cholangiocarcinoma, hepatocellular carcinoma, or liver metastases from colorectal cancer, the patients with advanced/metastatic or surgically unresectable solid tumors and are refractory to standard therapy will be treated with STP705. This therapeutic regimen is designed to take advantage of a dual-targeted inhibitory property of siRNAs and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expressions. Early pre-clinical and clinical studies using STP705 have shown an increase of active T cell infiltration into the tumor microenvironment. In addition, knocking down TGF-β1 and COX-2 gene expressions in animal fibrosis tissue can activate fibroblast apoptosis with significant antifibrotic efficacy.

"Advancing STP705 from skin cancer to liver cancer is a major milestone for Sirnaomics’ clinical programs, especially with a basket study design that consists of multiple tumor types. We are particularly interested in learning whether the mechanism of action validated in the skin cancer clinical study can be further verified in this liver cancer study," said Patrick Lu, Ph.D., founder, President and Chief Executive Officer of Sirnaomics. "We are expecting that the combination of anti-fibrotic effects and enhanced tumor immunity will provide a novel approach for the treatment of cholangiocarcinoma and hepatocellular carcinoma using our novel siRNA therapeutics. Sirnaomics is committed to advancing our polypeptide nanoparticle delivery system for innovative RNAi-based cancer therapy."

"Liver cancer is a devastating disease for patients with high mortality and high unmet medical need," stated Michael Molyneaux, MD, Chief Medical Officer of Sirnaomics. "The company is excited to announce first dosing, as we hope to gain important insight into the potential safety and efficacy of STP705 in this Phase 1 trial and build on the data from this study to expand into other oncology indications."

Sirnaomics expects to report initial clinical data from the Phase 1 trial in the second half of 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04676633

About Liver Cancer
Liver cancer is a global health problem, with liver neoplasms representing the second-most frequent cause of cancer-related death. There are many different types of liver cancers including hepatocellular carcinoma (HCC), cholangiocarcinoma, liver angiosarcoma, hepatoblastoma, and others. Additionally, the liver is a highly metastasis-permissive organ. It is the most frequently afflicted organ by metastasis and liver metastases are much more common than primary hepatic tumors. The distinctive biology of the liver renders it intrinsically susceptible to metastases. The true prevalence of liver metastasis is unknown, but between 30% and 70% of patients dying of cancer have liver metastases and most patients with liver metastases will die of their disease.

STP705 and Liver Cancer
Over expressions of TGF-β1 and COX-2 have been well-characterized as playing key regulatory roles in tumorigenesis. TGF-β is produced by different liver cells and is demonstrated to induce tumor cell migration and survival. TGF-β has been found to be overexpressed in metastatic HCC tissues. Overexpression of TGF-β is generally accepted to be associated with metastasis and poor prognosis. COX-2 is reported to be highly expressed in cancer stem cells and promotes cell migration in HCC cell lines. Additionally, inhibition of COX-2 suppresses cell migration and induces apoptosis. As such TGF-β1 and COX-2 are excellent therapeutic targets for treatment of liver cancer.

STP705 is composed of two siRNA oligonucleotides targeting TGF-β1 and COX-2 mRNA respectively and formulated in nanoparticles with a proprietary Histidine-Lysine Co-Polymer (HKP) peptide. Each individual siRNA has demonstrated the ability to inhibit the expression of their target mRNA and combining the two siRNAs produces a synergistic effect that diminishes pro-fibrogenic, pro-inflammatory, and pro-tumorigenic factors. Sirnaomics has completed several pre-clinical studies that demonstrate that inhibition of TGF-β1 and COX-2 and is expected to result in the inhibition of tumor growth and provide an alternative approach for the treatment of various liver cancers. Molecular analyses of the effects of administering the combination demonstrated that the inhibition of these targets had effects on downstream gene products associated with numerous oncology targets.

Additional immunohistochemistry and image analyses of the liver and tumor tissues demonstrated that animals treated with STP705 resulted in increased CD4+ and CD8+ T cell infiltration within the tumor microenvironment. Using STP705 for treatments of hepatocellular carcinoma and cholangiocarcinoma have been designated as Orphan Drug indications by U.S. FDA. STP705 has also been evaluated in a Phase 2a clinical trial for treatment of non-melanoma skin cancer.

On June 23, 2021 Servier, a global independent pharmaceutical Group, and Biolabs Global, ("BioLabs") a developer and operator of premium co-working laboratories in the US and Europe, reported that they have entered into a services agreement to manage Servier’s start-up incubator located in the future Servier R&D Institute in Paris-Saclay due to open in 2023 (Press release, Servier, JUN 23, 2021, View Source [SID1234584278]).

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The start-up co-working laboratory created by Servier within its Research and Development Institute in Paris-Saclay aims to be a major player in therapeutic innovation. The unprecedented synergy between the dynamism of young, promising companies in the healthcare sector and Servier’s pharmaceutical expertise offers unique conditions for developing and accelerating drug discovery, both in France and worldwide.

"The start-up incubator encourages a new level of knowledge sharing. Networked innovation without barriers is crucial for serving patients’ unmet needs even more effectively and rapidly. Agile methods to accelerate R&D programs are based on synergies and teamwork," comments Christophe Thurieau, Executive Director of Servier Research.

This incubator inside the Servier Research and Development Institute will offer a unique combination of space and services in France, and represents Servier’s ambition to renew itself and its new approach to R&D. It will allow start-ups to have their independence regarding Servier, while being close-by and benefiting from Servier’s scientific and technological expertise.

A place for innovation
Spanning 1,850 m2, the co-working laboratory has been designed with a capacity for, approximately 20 companies with space for over 100 scientists. The beautifully designed spaces will offer fully equipped and permitted premium shared and private laboratory spaces, conference facilities, central café and adjacent office and hot desks for the start-up companies. Residents will have access to the central Servier support spaces and core facilities and benefit from an easy to use e-procurement solution and integrated biohazard and chemical waste management. Additionally, residents are connected to fellow entrepreneurs from around the globe and to sponsor companies from many industries through the BioLabs network. Participants join this fertile ecosystem on their first day at the lab.

Admission to the Center will be through an application and selection process managed by BioLabs. The application process will be opened in Summer 2022. To promote and facilitate interactions prior to opening, Servier and BioLabs are offering regular webinars, Masterclasses and mentoring programs. The first Masterclass mentoring workshop for start-ups will take place on November 2021 in Boston.

A recognized player with international experience to offer a unique range of services
Servier will partner with BioLabs to operate and manage this tailor-made space. BioLabs is experienced and recognized in health/life sciences at a global level. Both companies are interested and highly motivated to support the acceleration of R&D projects.

"Launching this site in collaboration with Servier is an extraordinary opportunity for us. Servier has been a valuable partner in the US and has shown that they have the vision and enthusiasm to support entrepreneurs at this very early stage. This commitment to health science innovation matches our own mission and values. We look forward to supporting the community alongside Servier with webinars, masterclasses and mentoring prior to the launch and welcoming quality start-ups in 2023," said Johannes Fruehauf, Founder, President & CEO of Biolabs.

BioLabs will oversee the day-to-day management of the site, the provision of supplies, services and equipment, the practical support and events and programming specific to the interests of life science start-ups. They will also establish contacts with industry leaders and the entrepreneurial ecosystem at large, in Saclay and throughout France and beyond.

Paris-Saclay, an unprecedented location to bio-innovate in France
With 15% of national research, 40% of public and private research jobs in Île-de-France with the jewels of French academic and private research, the scientific and technological cluster of Paris-Saclay is among the eight most powerful innovation hubs in the world. This density and variety of resources makes Paris-Saclay unique. It gives Paris-Saclay all the advantages of one of the most attractive innovation clusters for investors, innovators and entrepreneurs from all over the world.

The Paris-Saclay cluster offers a promising array of potential future partners.
"Combining this unique place for health start-ups to grow and learn from experienced professionals with the vibrant ecosystem of Paris Saclay makes a unique opportunity to support therapeutic and technology innovation and improve the life of patients," concludes Olivier Nosjean, Head of Global Open Innovation and Scientific Affairs at Servier.

On June 23, 2021 Bionaut Labs, a company focused on revolutionizing the treatment of central nervous system disorders (CNS) with its Bionaut precision medicine treatment modality, reported that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for BNL-101 for the local treatment of all malignant gliomas including diffuse intrinsic pontine glioma in pediatric and adult patients (Press release, Bionaut Labs, JUN 23, 2021, View Source [SID1234584294]). BNL-101 is a drug-device combination comprised of doxorubicin as the active drug component together with the company’s Bionaut microscale robots.

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"The granting of orphan drug designation for BNL-101 represents a significant milestone for Bionaut Labs as it recognizes the potential of our approach to transform the standard of care for devastating CNS diseases like malignant gliomas," said Michael Shpigelmacher, co-founder and CEO, Bionaut Labs. "The award of orphan drug designation is the first step in a program of regulatory optimization that Bionaut Labs has initiated to allow us to move BNL-101 into the clinic in the swiftest and most effective way possible. We believe BNL-101 has the potential to play a significant role in shifting the treatment paradigm for malignant gliomas, and we look forward to continuing to work with the FDA as we fulfill our mission of helping patients suffering from debilitating brain diseases who lack treatment options."

"Gliomas remain some of the most devastating tumors for which there are few, if any, effective treatment options and for which there remains significant unmet medical need," said Alex Kiselyov, chief science officer, Bionaut Labs. "Receiving orphan drug designation from the FDA is an important regulatory milestone as we believe it validates our Bionaut-based approach. We look forward to advancing our BNL-101 therapeutic program into the clinic."

The FDA’s Office of Orphan Drug Products grants orphan drug status to support development of drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, and seven years of marketing exclusivity.

About Bionaut Treatment
A Bionaut is a novel treatment modality that uses remote-controlled microscale robots to deliver biologics, nucleic acids, small molecule, gene or cellular therapies locally to targeted CNS disease areas. Through precise targeting, Bionaut therapeutics could offer better efficacy and safety that cannot be achieved by other traditional treatment or delivery modalities.

Bionauts can be constructed in different designs with custom geometries and surface characteristics. Smaller than a millimeter, they contain moving parts controlled remotely by a magnetic controller, allowing them to safely reach the target and release a therapeutic payload from the cargo compartment. Engineering flexibility provides a broad foundation for designing Bionaut therapies for nearly any disease of interest.

Bionaut Labs has demonstrated safe and controlled navigation of its therapeutic Bionaut to and from the treatment locus in the brain, in a large animal in vivo model. Furthermore, the Company has successfully treated human glioma tumors established in mice, utilizing guided delivery of therapeutic cargos directly into these tumors to eliminate systemic toxicity. These results pave the way to the clinical trials of the Bionaut platform.