On May 7, 2024 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519), National Cancer Center Hospital (hereafter, NCCH), Osaka Medical and Pharmaceutical University (hereafter, OMPU) and MICIN reported introduction of a new decentralized clinical trial (DCT)* structure and the start of DCT in a Chugai-sponsored phase I clinical trial for patients with advanced solid tumors (Press release, Chugai, MAY 8, 2024, View Source;category= [SID1234642809]). NCCH and OMPU will collaborate using telemedicine as the primary institution and the satellite institution,** respectively, and some tests and assessments that were conventionally performed at institutions will be performed at the satellite institution.

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This is the first study in Japan to utilize a satellite institution for a company-sponsored phase I clinical trial in oncology. Since the number of institutions is very limited in phase I clinical trials, it is expected to improve access to clinical trials of new drug candidates for patients living far from institutions. We will evaluate this new DCT structure, with the aim of building an implementation structure that provides access to clinical trials for many patients regardless of where they live.

[Background and details]
Since clinical trials of new drug candidates are conducted at a limited number of medical institutions, some patients living far from the institution give up their participation in the clinical trial due to the time and financial burden associated with in-person visits. Establishing an environment that improves the patients’ access to clinical trials is a common challenge in clinical development, including pharmaceutical companies and academias.1 A retrospective study conducted at NCCH also showed that the participation rate in clinical trials tended to decrease for patients with travel time of 120 minutes or more.2 In addition, in phase I clinical trials evaluating drug safety for cancer patients, it is necessary to closely monitor patients’ condition while ensuring the patients’ safety. Therefore, there are few medical institutions where phase I clinical trials can be conducted.

DCTs, which are not dependent on in-person visits, have been attracted attention as a new approach in recent years. In Japan, some guidelines have been issued, and DCT is gradually being introduced into clinical trials, but the use in the oncology area is still limited.

This is a Chugai-sponsored phase I clinical trial for patients with advanced solid tumors, conducted with NCCH as a primary institution and OMPU as a satellite institution. Since the travel time from OMPU to NCCH exceeds 120 minutes, DCT is expected to reduce patients’ burdens. Patients will be able to visit the satellite institution instead of the primary institution to receive some tests and assessments for this clinical trial via telemedicine. The DCT platform MiROHA, provided by MICIN, will also be utilized for telemedicine visits and for obtaining remote re-consent using eConsent.

Improving access for patients is a common challenge in the pharmaceutical industry because clinical trials of new drug candidates are conducted at a limited number of medical institutions. The utilization of satellite institutions is expected to reduce the burden on patients and improve access to clinical trials. Chugai’s most prioritized value is patient-centricity. We will develop new drugs together with patients as partners, with the aim of realizing advanced and sustainable patientcentric healthcare.
Dr. Osamu Okuda, President and CEO, Chugai Pharmaceutical Co., Ltd.

To patients living in remote areas, access to clinical trials is drastically reduced, especially true for rare cancers and those of rare fractions, which trials are concentrated in hospitals in urban centers. By removing the need for patients to commute long hours, DCTs can address this issue. With new means, our team will reinvigorate drug development in Japan, delivering treatments expeditiously to patients nationwide, which is our mission.
Dr. Yasuyuki Seto, Director of National Cancer Center Hospital

In the phase I clinical trial for solid tumors, our institution will play a role as a satellite institution, contributing to the implementation of "no one left behind in cancer care" practices. In the face of disparities in access to clinical trials and information, particularly in rural areas, we have established a decentralized clinical trial system aimed at reducing the burden on patients while addressing these disparities. We have experience in many phase I trials of new anti-cancer drugs, and we are committed to the success of this challenging trial and will endeavor to ensure the realization of cancer patients’ "Well Being" even in rural regions.
Dr. Takahiro Katsumata, Osaka Medical and Pharmaceutical University Hospital

It is hoped that this trial initiative, which utilizes satellite medical institutions, will reduce the burden on patients and expand their options for participating in clinical trials.
As a leading DCT company, we are committed to contributing to a new way of conducting clinical trials through this new attempt.
Dr. Seigo Hara, MICIN, Inc., Representative CEO

On May 8, 2024 Corvus Pharmaceuticals, Inc. reported its first quarter 2024 results (Press release, Corvus Pharmaceuticals, MAY 8, 2024, View Source [SID1234642791]).

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On May 7, 2024 Alltrna, a Flagship Pioneering company unlocking transfer RNA (tRNA) biology and pioneering tRNA therapeutics to regulate the protein universe and resolve disease, reported the presentation of new data at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting demonstrating that Alltrna’s platform, enabled by machine learning (ML), can optimize sequences and modifications from natural tRNAs to significantly increase in vivo activity to readthrough premature termination codons (PTCs) caused by nonsense mutations (Press release, Alltrna, MAY 7, 2024, https://www.alltrna.com/press/alltrna-applies-machine-learning-to-engineer-trna-oligonucleotides-with-significantly-improved-activity-and-demonstrates-in-vivo-readthrough-of-the-two-most-common-premature-termination-codons-in-genetic-disease [SID1234646024]).

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"Alltrna’s ML-driven platform explores at an unprecedented scale the expansive combinatorial space of tRNA sequences and modifications, which has the potential to generate more engineered tRNA oligonucleotides than atoms in the universe," said Michelle C. Werner, CEO of Alltrna. "The data demonstrate the power of Alltrna’s platform to identify key combinations of tRNA sequences and modifications and precisely design tRNA oligonucleotides with significantly improved in vivo activity. With optimized engineered tRNAs for the two most prevalent premature termination codons, we are advancing preclinical studies for our first Stop Codon Disease indications."

tRNAs are programmable molecules with a diverse biology of sequences and modifications that are key to their structure, function, and stability. There’s the potential for approximately 10^34 tRNA sequences and more than 120 natural and synthetic modifications for each nucleotide. Using ML-powered screens, Alltrna scientists applied high-throughput sequence optimization to increase engineered tRNA activity by ~100-fold. ML-driven modification optimization further increased engineered tRNA activity.

Data were presented for the optimization of engineered tRNA oligonucleotides to address two different PTCs: Arg-TGA and Gln-TAG. Delivered using a liver-directed lipid nanoparticle (LNP), both optimized tRNAs showed robust in vivo activity in two transgenic mouse models. The first carries a human rare disease gene with a PTC mutation, the second carries a reporter gene with a different PTC as a general in vivo model for Stop Codon Disease. Alltrna previously presented in vitro data on the engineered tRNA for Arg-TGA showing that it can readthrough nonsense mutations regardless of gene or location in 25 disease reporter models, 14 different genes, and seven different mutation locations on a single gene.

"These data confirm that the application of high-throughput sequence and ML-driven modification optimization through Alltrna’s unique platform can significantly increase the in vivo activity of engineered tRNAs," said Stephen W. Eichhorn, Ph.D., Head of Computational and Molecular Biology at Alltrna. "We’ve also demonstrated that we can engineer robust tRNA activity for two different premature termination codons, each of which are highly prevalent in Stop Codon Disease."

About Stop Codon Disease

Stop Codon Disease encompasses thousands of rare and common diseases that stem from premature termination codons (PTC) also called nonsense mutations, where the code for an amino acid has been mutated into a premature "stop" codon. This results in a truncated or shortened protein product with no or altered biological activity that causes disease. Approximately 10% of all people with a genetic disease have Stop Codon Disease, representing approximately 30 million people worldwide. Alltrna is engineering tRNA medicines that can read these PTC mutations and deliver the desired amino acid, thereby restoring the production of the full-length protein.

On May 7, 2024 Kyowa Hakko Kirin reported its Consolidated Financial Summary (IFRS) Fiscal 2024 First Quarter (Press release, Kyowa Hakko Kirin, MAY 7, 2024, View Source [SID1234645288]).

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On May 7, 2024 Flagship Pioneering, the bioplatform innovation company, reported Prologue Medicines, a company unlocking the therapeutic potential of the viral proteome for the creation of powerful new medicines (Press release, Prologue Medicines, MAY 7, 2024, View Source [SID1234643565]). Flagship has initially committed $50 million to advance the company’s Decoding Evolutionary Logic of Variant Ensembles (DELVE) Platform and harness its power to develop a pipeline of medicines fora wide range of diseases, with an initial focus in immunological, oncology and metabolic indications.

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"We asked, what if we could expand beyond the functional boundaries of the human proteome by harnessing viral evolution," said Noubar Afeyan, Ph.D., Founder and CEO of Flagship Pioneering and Co-Founder and Chairman of the Strategic Oversight Board of Prologue. "By exploring the viral proteome, we can discover novel proteins with enhanced or completely unique therapeutic properties to develop medicines for optimal patient outcomes."

Prologue is unlocking viral proteome-assisted drug discovery with its novel DELVE Platform. DELVE combines computation and high-throughput biology to continually mine the expanding viral proteome to uncover viral proteins and their unique features and evaluate their potential to modulate human physiology for the treatment of disease. The platform contains the largest known database of viral protein structures layered both with detailed data from known human proteins and annotations of previously uncharacterized viral proteins predicted through Prologue’s proprietary computational frameworks. By applying the learned rules of natural viral evolution, Prologue creates enhanced therapeutic proteins programmed to have properties, some previously unique to viruses, that expand the functional boundaries of human proteins.

"The viral proteome dwarfs the human proteome by orders of magnitude, enabling us to reveal a trove of powerful new proteins that can be used as medicines across nearly any therapeutic area," said Lovisa Afzelius, Ph.D., MBA, Co-Founder and CEO of Prologue and Origination Partner at Flagship Pioneering. "Nature has evolved proteins with unique characteristics across the viral proteome. Advances in machine learning are enabling us to quickly characterize and select those that can precisely regulate human physiology to create powerful new medicines with impeccable precision."

In addition to Afeyan and Afzelius, Prologue is led by Theonie Anastassiadis, Ph.D., Founding President of Prologue and Senior Principal at Flagship Pioneering, and Hozefa Bandukwala, Ph.D., Founding Chief Scientific Officer of Prologue and Science Partner at Flagship Pioneering.