On October 25, 2022 SK bioscience, a global innovative vaccine and biotech company committed to promoting human health from prevention to cure across the globe, reported that the Company made a new partnership agreement with the Coalition for Epidemic Preparedness Innovations (CEPI) for the development of mRNA vaccines to quickly respond to the spread of infectious diseases and to expand its vaccine portfolio (Press release, SK Bioscience, OCT 25, 2022, View Source [SID1234622376]).

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(from left) Richard Hatchett, CEO of CEPI, Park Min-soo, Second Vice Minister of the Ministry of Health and Welfare (MOHW), Chang Won (Andrew) Chey, Vice Chairman of SK discovery at the signing ceremony at Grand Walkerhill Seoul on October 25, 2022.
(from left) Richard Hatchett, CEO of CEPI, Park Min-soo, Second Vice Minister of the Ministry of Health and Welfare (MOHW), Chang Won (Andrew) Chey, Vice Chairman of SK discovery at the signing ceremony at Grand Walkerhill Seoul on October 25, 2022.
Richard Hatchett, CEO of CEPI, Park Min-soo, Second Vice Minister of the Ministry of Health and Welfare (MOHW), Chang Won (Andrew) Chey, Vice Chairman of SK discovery, and Jaeyong Ahn, CEO of SK bioscience attended the signing ceremony at Grand Walkerhill Seoul and discussed continuous cooperation between the two organizations for the development of mRNA vaccines.

The purpose of the agreement is that SK bioscience, which has secured the latest vaccine platform technologies such as cell culture, bacterial culture, and genetic recombination, expands its portfolio including the mRNA platform technology and establishes an R&D system that can prevent existing or unknown viruses in the future with global institutions. The Company will use the Japanese encephalitis virus (JEV) and Lassa virus to develop the mRNA vaccine platform.

SK bioscience is the CEPI’s first partner among global vaccine companies under the CEPI’s project of ‘RNA vaccine platform technologies and vaccine library development against emerging and select endemic infectious diseases’ to quickly respond to unknown infectious diseases (Disease-X) and solve the vaccine equity in low- and middle-income countries.

Under the agreement, SK bioscience will receive up to 140 million USD in R&D expenses from CEPI. Up to 40 million USD in initial funding will be made available to support phase 1/2 clinical trials of two mRNA vaccine platform projects. Pending results from phase 1/2 studies, a further $100 million in funding could be made available to support late-stage trials/licensure to further validate the mRNA platform and have it ready for use in outbreak situations.

SK bioscience and CEPI also agreed to expand cooperation on developing various vaccines based on the mRNA platform to respond to the spread of infectious diseases in low- and middle-income countries.

Earlier, SK bioscience had the partnership with the Bill & Melinda Gates Foundation in order to build the mRNA vaccine platform. The Company has been conducting the preclinical study on the mRNA vaccine platform using the COVID-19 virus with 2 million USD funded by the Foundation. The study is an important technology foundation for the collaboration project with CEPI.

SK bioscience will rapidly respond to the spread of infectious diseases based on the mRNA vaccine platform and global network. In particular, the Company plans to consolidate its global position by strengthening its portfolio with the mRNA vaccine platform and by developing new pipeline such as RSV vaccines, CMV vaccines, and anti-cancer vaccines using the mRNA platform.

The mRNA vaccine platform, which was first commercialized in the COVID-19 vaccine during the pandemic, is available for rapid mass production compared to existing platforms by utilizing genetic sequences. It is why the mRNA vaccine platform is considered to be proper in responding to the pandemic. In addition, the related market is expected to expand quickly due to the use possibility for developing treatments. According to Global Industrial Analyst (GIA), the global mRNA vaccine market will increase by 11.9% annually from 64.9 billion USD last year to 127.3 billion USD in 2027.

CEPI and SK bioscience are committed to enabling global equitable access to the vaccines they develop. Under the terms of the funding agreement, SK bioscience has committed to achieving equitable access to the outputs of this project including prioritization of supply for low-and middle-income countries, production of vaccine volumes required to meet public health needs, and affordable pricing, in line with CEPI’s Equitable Access Policy.

Richard Hatchett, CEO of CEPI said, "We are racing against the clock now, because we don’t know when the next pandemic virus will emerge. Key to making a future free of pandemics a reality is the ability to rapidly respond to the next Disease X with new vaccines and other countermeasures—in just 100 days. CEPI’s expanded partnership with SK bioscience will help kick start the world’s efforts to validate these mRNA platform technologies so that they can be used to create a library of vaccines ready for use against the next Disease X, bringing us another step closer achieving the 100 Days Mission, and preparing the world for the next pandemic."

Park Min-soo, Second Vice Minister of the Ministry of Health and Welfare (MOHW), said, "I would like to congratulate SK bioscience and CEPI’s partnership on development of mRNA vaccine. The Korean government will also make an effort to contribute to solving the vaccine issue as rapid vaccine development and vaccine equity are the most important to respond to the pandemic."

Chang Won Chey, Vice Chairman of SK discovery said, "We all agree that speed is the most important factor to protect humanity from the next life-threatening pandemic. Based on cooperation with global initiatives, including CEPI, we will achieve innovative vaccine development and ultimately contribute to promoting global public health."

On October 25, 2022 ImaginAb Inc., a global biotechnology company developing 89Zr crefmirlimab berdoxam (CD8 ImmunoPET) imaging agent and next generation radiopharmaceutical therapies (RPT) products, reported that the first patient has been dosed at the Macquarie University Hospital in Sydney, Australia as part of its Phase IIb iPREDICT clinical trial (Press release, ImaginAb, OCT 25, 2022, View Source [SID1234622375]).

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ImaginAb announced the launch of its Phase IIb iPREDICT trial in January this year, which is a part of its clinical development to support marketing approval for CD8 ImmunoPET. iPREDICT is being conducted globally in the US, Australia, and Europe.

The Phase IIb trial aims to assess predictive performance with its primary objective to evaluate the performance of CD8 ImmunoPET positron emission tomography/computed tomography (PET/CT) for predicting patient response to immuno-oncology therapy targeting Melanoma, Merkel Call, Renal Cell Carcinoma, Non-Small Cell Lung Cancer, and other selected solid tumors.

The Olivia Newton John Cancer Research Institute in Melbourne, which is also manufacturing doses for the iPREDICT trial in Australia, delivered the first clinical dose to the Macquarie University Hospital for use by Dr. Alison Zhang in the iPREDICT trial. Details of the study can be found on www.clinicaltrials.gov under the identifier NCT05013099.

Commenting on the announcement, Ian Wilson, Chief Executive Officer of ImaginAb, said:

"We are delighted to have reached this important clinical development milestone for ImaginAb as we move one step closer to our goal of helping to transform cancer care for patients. We are actively enrolling the iPREDICT trial over 22 clinical sites across the US, Australia and Europe and would like to thank our local sponsor/CRO, Accelagen, who played a key role in the achievement of this milestone by facilitating smooth communication with our investigators and the sites across the two time zones."

On October 25, 2022 Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, reported that received Phase 1/2a IND approval from the U.S FDA in October 10th for its hepatocellular carcinoma (HCC) treatment called RZ-001 and thus has achieved an important milestone for the company and the RNA editing field (Press release, Rznomics, OCT 25, 2022, View Source [SID1234622374]). Being the first U.S. FDA-approved ribozyme-based RNA reprogramming approach to be evaluated in patients, RZ-001, a gene therapy approach utilizing the company’s proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, is a replication-incompetent adenoviral vector that expresses an hTERT targeting ribozyme with multiple additional MoA to treat HCC patients. Rznomics also received IND approval of RZ-001 from the South Korean Ministry of Food and Drug Safety this June and already initiated a phase 1/2a clinical trial in Korea. Therefore, U.S. FDA approval allows Rznomics to start an international clinical study in HCC patients treating them with RZ-001 and therapeutic RNA editing.

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The trans-splicing ribozyme is derived from the self-splicing Tetrahymena group I intron, which both recognizes and reprograms the target RNA into the therapeutic transcript of interest. Ribozyme-based RNA editing technology developed by Rznomics has unique features, differentiating it from other nucleic acid-based editing approaches, as follows: (1) A single RNA molecule is capable of both suppressing target RNA expression and simultaneously expressing a therapeutic RNA. Thus, no potentially antigenic proteins or cofactors are required. (2) Safety can be improved by selectively inducing therapeutic RNA expression only in cells/tissues where the target gene is expressed. (3) Therapeutic gene expression can be regulated proportionally to endogenous cellular target RNA levels. (4) Editing occurs at the RNA level, not the genomic level, thus eliminating concerns about genomic toxicity and eternal genome changes. (5) Indications with multiple mutation sites scattered throughout a target RNA can be edited with a single RNA designed to react upstream of all mutations and by replacing and editing large stretches of RNA. (6) Additional safety can be conferred by building control mechanisms into the ribozyme itself, without the need to modulate intrinsic cellular mechanisms or external proteins.

More specifically, RZ-001 engenders effective anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which over-express hTERT, and simultaneously inducing a cytotoxic effect by trans ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. Moreover, the result of such editing efficiently induces immune cell infiltrations into HCC tumors in preclinical animal models. (View Source). The Phase 1/2a clinical trial will be a dose escalation/expansion study to assess the safety and tolerability of RZ-001 and to determine the most effective dose with the least toxicities of RZ-001 in HCC patients with no extrahepatic metastasis.

"The translation of the first trans-splicing-based RNA editing approach into an FDA-approved phase 1/2a clinical trial is an exciting achievement and a critical milestone for the RNA editing field. I am very excited about Rznomics’ preclinical progress on ribozyme design and gene delivery optimization. The advances have allowed them to create and now translate a promising therapy, RZ-001, into the clinic. I and the entire editing field are eager to learn if RZ-001 and mRNA reprogramming is safe and able to combat hepatocellular carcinoma in patients. Rznomics is clearly a leader at bringing novel editing strategies to cancer patients that desperately need innovative, breakthrough therapies," said Dr. Bruce Sullenger, Joseph, and Dorothy Beard Professor of Surgery at Duke University. Dr. Sullenger is a scientific advisory board member of Rznomics and the initial pioneer developing approaches to therapeutically edit RNA and DNA using RNA guided endonucleases (RGENs) such as the group trans-splicing I ribozyme..

"It’s a monumental achievement of Rznomics that RZ-001, the first trans-splicing ribozyme therapy at the front of our therapeutic pipeline, has successfully received the IND approval in both Korea and the United States. I am really grateful that RZ-001 earned the opportunity to potentially fulfill the unmet needs of HCC patients. Through the advanced development phase, I hope Rznomics can provide more new therapeutic options to patients suffering from intractable diseases. Rznomics will further expand our pipeline by targeting indications with highly unmet medical needs for which the unique characteristics of our platform technology may be the most competitively applied." said Dr. Seong-Wook Lee, CEO and founder of Rznomics.

In addition to the HCC, Rznomics is expanding the indication of RZ-001 to glioblastoma multiforme and planning to submit the IND this year. Also under development are ribozyme-based RNA editing treatments for Alzheimer’s disease (RZ-003) and inherited retinal dystrophies, called Retinitis pigmentosa (RZ-004).

On October 25, 2022 Prelude Corporation (PreludeDx), a leader in molecular diagnostics and precision medicine for early-stage breast cancer, reported study results demonstrating that DCISionRT provides patients with ductal carcinoma in situ (DCIS) superior risk and RT benefit prediction compared to common clinicopathologic features. The data was presented in an oral presentation at the 64th Annual American Society for Radiation Oncology (ASTRO) Meeting, held on October 23 – 26, 2022 at the Henry B. Gonzalez Convention Center in San Antonio, TX (Press release, Prelude Therapeutics, OCT 25, 2022, View Source [SID1234622373]).

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This study, titled ‘Re-thinking clinicopathologic risk assessment in DCIS: Pooled data from validation studies comparing a 7-gene DCIS assay to clinicopathologic features alone’, highlighted that clinicopathologic criteria were poor predictors of 10-year ipsilateral breast recurrence (IBR) rates and radiation therapy (RT) benefit in 926 women from four international cohorts.

"With DCISionRT, clinicians can now identify which DCIS patients, despite having low-risk clinicopathologic features, who can decrease their risk of recurrence by 70% or more with RT after breast conserving surgery. Utilizing this genomic tool, we can also confidently identify a truly Low Risk group of patients who can safely forego RT, even if they have high risk clinicopathologic factors," said Chirag Shah, MD, Co-Director of Comprehensive Breast Program and Director of Clinical Research in the Department of Radiation Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH. "It is important to follow the clinical evidence when making the best treatment decisions with our patients. As clinicians, we should not rely on clinicopathologic factors alone, which are inadequate to personalize radiation treatment decisions, especially when compared to the DCISionRT predictive test."

"DCISionRT provides unique game-changing information to enable physicians and DCIS patients to confidently make a personalized treatment decision," said Dan Forche, President and CEO of PreludeDx. "We are honored by the overwhelming recognition of this latest data with oral presentations at three national medical conferences and the recent publication of validation data in ASTRO’s prestigious Red Journal."

About DCISionRT for Breast DCIS
DCISionRT is the only risk assessment test for patients with ductal carcinoma in situ (DCIS) that predicts radiation therapy benefit. Patients with DCIS have cancerous cells lining the milk ducts of the breast, but they have not spread into surrounding breast tissue. In the US, over 60,000 women are newly diagnosed with DCIS each year. DCISionRT, developed by PreludeDx on technology licensed from the University of California San Francisco, and built on research that began with funding from the National Cancer Institute, enables physicians to better understand the biology of DCIS. DCISionRT combines the latest innovations in molecular biology with risk-based assessment scores to assess a woman’s individual tumor biology along with other pathologic risk factors and provide a personalized recurrence risk. The test provides a Decision Score that identifies a woman’s risk as low or elevated. Unlike other risk assessment tools, the DCISionRT test combines protein expression from seven biomarkers and four clinicopathologic factors, using a non-linear algorithm to account for multiple interactions between individual factors in order to better interpret complex biological information. DCISionRT’s intelligent reporting provides a woman’s recurrence risk after breast conserving surgery alone and with the addition of radiation therapy. In turn, this new information may help patients and their physicians to make more informed treatment decisions.

On October 25, 2022 Quadriga BioSciences, a clinical-stage oncology company developing QBS10072S (QBS72S) for the targeted treatment of cancer, reported the completion of enrollment for a Phase 1 dose escalation study investigating QBS72S in patients with advanced or metastatic solid cancers (Press release, Quadriga BioSciences, OCT 25, 2022, View Source [SID1234622372]).

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"Completing the enrollment of our dose-ranging Phase 1 study is an important milestone in the development of QBS72S," said Gordon Ringold, Ph.D., Chief Executive Officer of Quadriga BioSciences. "With the study complete, Quadriga is able to launch two Phase 2 studies investigating QBS72S for brain malignancies, each of which is supported by funding from an SBIR grant. We are fortunate for the support of all involved as we continue to advance QBS72S development."

The Phase 1, multi-center, open-label, dose-escalation study [NCT04430842] was designed to evaluate the safety and tolerability of QBS72S in patients with advanced or metastatic solid tumors. Results of the Phase 1 study have informed the recommended Phase 2 dose (RP2D) for two imminent Phase 2 studies: one investigating QBS72S for the potential treatment of brain metastases of triple negative breast cancer at Stanford University [NCT05305365] and one investigating QBS72S for the potential treatment of glioblastoma at the Dana-Farber Cancer Institute [NCT02977780]. Each of the studies is funded by a Small Business Innovation Research (SBIR) grant; the two grants were awarded to Quadriga by the U.S. National Institutes of Health (NIH) to support the development of QBS72S. The studies are expected to launch in Q4’2022.

About QBS72S

QBS72S is a novel, first-in-class chemotherapeutic agent that mimics an aromatic amino acid for cellular uptake by the amino acid transporter LAT1 (L-type amino acid transporter 1) thereby enabling the drug to cross the blood brain barrier (BBB) as well as to selectively target numerous types of rapidly growing cancer cells. Once inside the cell QBS72S causes double-stranded DNA breaks resulting in cell death. Most aggressive cancers express high LAT1, which is commonly associated with poor prognoses.1