Simnova and Orna Therapeutics Collaborate to Advance Orna’s Next Generation Circular RNA Technology in China

On January 5, 2023 Shanghai Xianbo Biotech Co., Ltd., also known as Simnova outside of China, an affiliate of Simcere Investment Group and a clinical-stage biotechnology company developing proprietary off-the-shelf CAR-NK and BiTE-armed CAR-T cell therapies, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, reported a collaboration agreement to discover, develop, and commercialize multiple potential therapeutics in the area of oncology (Press release, Shanghai Simnova Biotechnology, JAN 5, 2023, View Source [SID1234625929]).

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Under the terms of the agreement, Simnova will gain access in greater China to certain programs built on Orna’s breakthrough isCAR technology, including Orna’s lead anti-CD19 in situ CAR (isCAR) program, ORN-101. In addition, Orna will gain access to investigator-initiated clinical trials in China to accelerate the validation of select programs in patients. Orna will receive an upfront payment from Simnova and be eligible for development, regulatory, and sales milestones as well as royalties on any approved products derived from the collaboration.

Orna’s proprietary isCAR technology comprises two components: elegantly engineered circular RNAs (oRNAs) expressing chimeric antigen receptors, and custom-built lipid nanoparticles (LNPs) designed to deliver the oRNAs to cells of the immune system. oRNAs have greater stability in immune cells and are engineered to produce more proteins inside the body, than linear mRNAs. More broadly, Orna’s oRNA-LNP platform has potential across a range of indications including oncology, genetic and infectious disease, and more.

"This strategic collaboration broadens Orna’s capability to develop circular RNA therapeutics in China through Simnova’s development capabilities," said Dr. Zhouxiao Cao, Simnova’s Chief Executive Officer. "We look forward to closely working with the innovative scientific and technical teams at Orna."

"This collaboration again recognizes the value of our oRNA-LNP platform and will allow us together to diversify the number of programs using isCAR technology while bringing the value of isCAR to patients more efficiently," said Tom Barnes Ph.D., Orna’s Chief Executive Officer. "This marks another important step in our overall business strategy where we look to thoughtfully engage partners to deliver on the promise of circular RNA therapies."

About ORN-101:

ORN-101, Orna’s lead program, is an in situ CAR therapy designed to modify a patient’s immune cells inside their body. Comprising an oRNA molecule packaged inside a proprietary LNP, this easily redosable format could avoid patient lymphodepletion and allow for reliable dose control, overcoming barriers of existing ex vivo CAR-T therapies without sacrificing efficacy. Preclinical data presented at a scientific conference demonstrated tumor suppression and eradication in an animal model, suggesting the possibility that oRNA-LNP based cancer therapies could eventually overtake cell therapies.

bluebird bio to Present at the 41st Annual J.P. Morgan Healthcare Conference

On January 5, 2023 bluebird bio, Inc. (Nasdaq: BLUE) reported that Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update at the 41st Annual J.P. Morgan Healthcare Conference on Thursday, January 12, 2023, at 7:30AM PT (Press release, bluebird bio, JAN 5, 2023, View Source [SID1234625892]).

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To access the live webcast of bluebird bio’s presentation, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 30 days following the event.

OncoResponse to Present at 41st Annual J.P. Morgan Healthcare Conference

On January 5, 2023 OncoResponse, a clinical-stage biotech company advancing immunotherapies derived from the immune systems of Elite Cancer Responders, reported that Clifford Stocks, Chief Executive Officer, will present a company overview at the 41st Annual J.P. Morgan Healthcare Conference on Thursday, January 12, 2023, at 11 a.m. PST (Press release, OncoResponse, JAN 5, 2023, View Source [SID1234625928]).

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Following the live event, the accompanying slide presentation will be accessible on the Events & Presentations page of the OncoResponse website at View Source

Jubilant Therapeutics Inc. receives Orphan Drug Designation for JBI-802 for Acute Myeloid Leukemia (AML) and Small Cell Lung Cancer (SCLC)

On January 5, 2023 Jubilant Therapeutics Inc., a clinical stage biopharmaceutical company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, reported that the United States Food and Drug Administration (US FDA) has granted Orphan Drug Designation for JBI-802 for the treatment of small cell lung cancer (SCLC) and acute myeloid leukemia (AML) (Press release, Jubilant Therapeutics, JAN 5, 2023, View Source [SID1234625927]).

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JBI-802 is a dual epigenetic modulator engineered in a single pharmacophore to achieve optimal inhibition of the transcriptional regulator CoREST, which regulates the development of cellular lineages responsible for neuroendocrine tumors like SCLC and hematopoietic tumors like AML.

This unique profile has shown synergistic anti-tumor activity and it is expected to overcome tolerability limitations of first-generation, single target epigenetic modulators.

"JBI-802 is the lead product candidate from our TIBEO (Therapeutic Index and Brain Exposure Optimization) Discovery Engine. It is our unique approach of structure-based drug design to generate novel pharmacophores with improved target product profile compared to existing agents. The Orphan Drug Designations (ODD) were supported by several relevant preclinical models. In SCLC, JBI-802 showed unique activity not just in normal neuroendocrine models but also in the ‘variant’ models driven by MYC amplification. This data also supports the ongoing Ph I/II clinical trial in neuroendocrine tumor patients. In AML the activity was uniquely seen in models of erythroleukemia, a subset of leukemia, with a unique erythroid phenotype and a very high unmet need based on its aggressive nature and limited therapy. This designation and emerging clinical data from the ongoing fist-in-human JBI-802 study will now underpin expansion of our clinical activities in thrombocythemia, leukemia and other erythroid tumors like MPN," said Syed Kazmi, Chief Executive Officer, Jubilant Therapeutics Inc.

The US FDA’s Office of Orphan Products Development (OOPD) grants orphan designation status to a drug that is intended to treat a rare disease or condition that affects fewer than 200,000 persons in the United States.

About JBI-802

JBI-802 is an oral, potent and selective dual inhibitor of two epigenetic targets of the CoREST complex: LSD1 and HDAC6. It targets stem cell modulation by inhibiting LSD1 and modulates immune suppression with isoform selective HDAC6 inhibition. Preclinical research has demonstrated its synergistic anti-tumor activity, which is superior vs. either target alone inhibitors and has a favorable safety profile with no significant safety concerns or accumulation. It is being clinically evaluated in multiple neuroendocrine tumors including SCLC, with a goal to expand in to hematological cancers such as acute myelogenous leukemia, essential thrombocythemia, and other myeloproliferative cancers. Positive clinical data was recently reported for bomedemstat, a LSD1 only inhibitor, in essential thrombocythemia, thereby establishing a pivotal role of epigenetic modulators in hematological malignancies.

CNS Pharmaceuticals to Present at the Virtual Investor 2023 Companies to Watch Event

On January 5, 2023 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported that John Climaco, Chief Executive Officer of CNS Pharmaceuticals will present at the Virtual Investor 2023 Companies to Watch Event on Wednesday, January 18, 2023 at 10:00 AM ET (Press release, CNS Pharmaceuticals, JAN 5, 2023, View Source [SID1234625926]).

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A live video webcast of the presentation will be available on the Events page of the Investors section of the Company’s website (cnspharma.com). A webcast replay will be available two hours following the live presentation and will be accessible for 90 days.