On February 14, 2022 Oxilio, a pioneering drug development company repurposing existing drugs to address unmet needs in cancer treatment, reported that following successful formulation development it has commenced work on the clinical evaluation of its first novel formulation of OXL001 (Press release, Oxilio, FEB 14, 2022, View Source [SID1234621598]).

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Oxilio signed a research agreement with TRx Biosciences in January 2021, to evaluate the performance of TRx Biosciences’ technology platform within Oxilio’s OXL001 programme. The TRx Biosciences technology offers the potential to address bioavailability and pharmacokinetic challenges in achieving and maintaining efficacious exposure whilst enhancing tumour uptake to maximise the utility of OXL001’s pharmacology.

As a result of successful formulation optimisation and encouraging preclinical evaluation, Oxilio and TRx Biosciences have begun work on the first human study for the clinical evaluation of the novel formulation of OXL001. It is anticipated that the first subject will be dosed later in 2022, with the study concluding by the end of the year.

Oxilio Limited Co-founder and Director, Dr Simon Yaxley said "Together with our colleagues at TRx Biosciences, we are excited and encouraged by what we have been able to achieve in formulation optimisation which has led to significant pharmacokinetic improvements in preclinical models. We look forward to taking this science forward into the clinic later this year which, if successful, offers tremendous near-term potential benefit to cancer patients.

TRx Biosciences Co-founder and CEO, Dr Robin Bannister said "Our work with Oxilio has enabled us to rapidly establish our underlying technology, both in terms of pharmacokinetics and pharmacodynamics and we are delighted to see the progression of OXL001 towards clinic, given its immense potential as a pan-cancer therapy. We will continue to closely support Oxilio throughout its clinical development activities, which will be equally transformative for TRx as the first clinical validation of our targeted oral delivery platform."

On February 14, 2022 BioDuro-Sundia, a leading drug discovery, development and commercial services organization backed by Advent International, reported the appointment of Kent M. Payne, PhD, as Chief Executive Officer (Press release, Bioduro-Sundia, FEB 14, 2022, View Source [SID1234611016]). He originally joined BioDuro-Sundia in 2019 as President, Global CMC Solutions.

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"For the past 2+ years Kent has distinguished himself, setting a vision for growth, building strong teams and processes to execute that growth most recently as Corporate Head of Transition," said David Preston, Chairman of the Board, BioDuro-Sundia. "Under his leadership, we have generated record growth across all business units. The Board has full confidence and is excited to have him formally lead our next phase of growth with planned expansions in nearly every aspect of our China and US operation."

The executive team, led by Payne, will continue to be co-located in China and the US, providing balance for the global contract research, development and manufacturing organization (CRDMO) for both drug substance and drug product.

"I am honored to serve as CEO at this time of remarkable growth; we will be adding 1,000 people to our company in 2022 alone and bringing on board new capacities at every facility, including discovery, development and manufacturing," said Kent M. Payne, Ph.D., CEO.

"With support from Advent International, we are making the needed investment into our teams, systems and processes to make sure we deliver ‘right first time’, stay fast and flexible, and maintain our customer focus culture. This is our promise to each and every client, and to the patients awaiting new life-saving medicines."

On February 14, 2022 Roivant Sciences reported that has spawned another Vant (Press release, Roivant Sciences, FEB 14, 2022, View Source [SID1234608336]). The latest addition to the clan is Hemavant, a biotech that starts life with a former Eisai drug and plans to start a study in patients with myelodysplastic syndromes (MDS) this year.

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Hemavant is developing RVT-2001, the small-molecule modulator of splicing factor 3B subunit 1 that Roivant licensed from Eisai late last year. Eisai, working with its H3 Biomedicine subsidiary, took the oral candidate into a phase 1 clinical trial in 2016 but saw no complete or partial responses in the first 84 patients enrolled in the study of patients with MDS and other blood cancers.

Work stalled on the candidate after the 2019 data drop, with Roivant saying in January that the number of subjects treated with RVT-2001 still stands at "over 80." Yet, Roivant sees promise in some of the data generated by Eisai, leading it to license the asset and narrow the focus of the program.

Eisai enrolled patients with acute myeloid leukemia, chronic myelomonocytic leukemia and lower- and higher-risk MDS in its phase 1 study. In that population, the lack of responses was a blow, but Roivant has zeroed in on another endpoint that suggests RVT-2001 may have a future in lower-risk, transfusion-dependent MDS patients.

RELATED: Roivant ditches plan to reacquire Immunovant, invests $200M

Thirty percent of the 19 lower-risk MDS patients treated with RVT-2001, formerly known as H3B 8800, gained red blood cell transfusion independence. Most of the patients had previously received Bristol Myers Squibb’s Revlimid, hypomethylating agents or both therapies.

Based on the signal, Hemavant is developing RVT-2001 as an oral therapy for transfusion-dependent anemia in patients with lower-risk MDS. A phase 1/2 clinical trial is set to start in the first half of the year.

Roivant has paid Eisai $8 million in cash and $7 million in stock for the global rights to the candidate. As RVT-2001 advances, Roivant will pay up to $65 million in development and regulatory milestones in the first indication plus up to $18 million in additional indications. The deal also features up to $295 million in commercial milestones and a tiered high single-digit to sub-teens royalty.

On February 14, 2022 Datar Cancer Genetics Inc reported that the US Food and Drug Administration (FDA) has granted ‘Breakthrough Device Designation’ for its ‘TriNetra-Prostate’ blood test to detect early-stage prostate cancer (Press release, Datar Cancer Genetics, FEB 14, 2022, View Source [SID1234608106]). This is the second test from the Company that has received the Breakthrough Device Designation from the US FDA. Last year, the Company’s early-stage breast cancer detection test became the first such test to receive the Breakthrough Device Designation.

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In the United States, prostate cancer is the third most common type of cancer; each year, more than 200,000 men are detected with prostate cancer and is associated with more than 32,000 deaths. The test can identify individuals who are more likely to harbour cancer in the prostate and aids clinical decision making such as the need to undergo a biopsy for confirmatory diagnosis.

Studies have shown that TriNetra-Prostate can detect early-stage cancer with high accuracy (>99%) without any false positives. TriNetra-Prostate requires 5 ml blood and is indicated for males of age 55-69 years with serum PSA of 3 ng/mL or higher. TriNetra-Prostate is based upon the detection of prostate adenocarcinoma specific Circulating Tumor Cells (CTCs) in the blood.

"The breakthrough device designation is a recognition of the potential benefits of TriNetra-Prostate in the clinical setting. The test can help reduce the number of biopsies among individuals with benign conditions of the prostate and it can also improve detection rates among those who do have prostate cancer. With our proprietary CTC-enrichment and detection technology, there is virtually no risk of false positives among individuals who do not have prostate cancer," said Dr Vineet Datta, Executive Director of the Company. The test has previously received CE certification and is already available in Europe as ‘Trublood-Prostate’.

The Breakthrough Device Designation is granted by the FDA for devices that demonstrate a potential for more effective diagnosis of life-threatening diseases such as cancer. The Breakthrough Devices Program intends to provide patients and healthcare providers with timely access to medical devices granted such designation by prioritized review to expedite development and assessment.

On February 14, 2022 Children’s Hospital Los Angeles reported A new study independently verified the value of a system that assesses hepatoblastoma risk in children (Press release, Children’s Hospital Los Angeles, FEB 14, 2022, View Source [SID1234608105]). Hepatoblastoma is a rare childhood liver cancer, usually seen within the first three years of a child’s life with 50 to 70 cases occurring in the U.S. each year. The researchers also discovered the potential for tumor histology—the examination of a tumor’s tissue and its structure—to predict a patient’s hepatoblastoma prognosis.

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Leo Mascarenhas, MD, MS, Deputy Director of the Cancer and Blood Disease Institute, co-led the study with Shengmei Zhou, MD, a pediatric pathologist and diagnostician at CHLA. The findings are published in JAMA Network Open.

Risk stratification for hepatoblastoma

The risk assessment process, called risk stratification, has the potential to improve success rates for children undergoing hepatoblastoma treatment. Risk stratification can also reduce unnecessary exposure to chemotherapy. Often, treatment for low-risk cases is too aggressive, or liver transplants are not prioritized for high-risk cases.

In 2016, leading hepatoblastoma researchers from around the world created the Children’s Hepatic tumors International Collaboration Hepatoblastoma Stratification (CHIC-HS). Information such as age at diagnosis, whether the tumor has spread within the body and alpha fetoprotein level (which is usually increased in liver cancer) contributes to the CHIC-HS risk categorization. This database of clinical trial data aims to establish a common approach to staging and risk stratification. The CHIC-HS system has yet to be globally adopted, however, due to a lack of validation.

"Independent validation is valuable for others to use this risk stratification," emphasizes first author Dr. Zhou. "It gives them confidence."

Validating the CHIC-HS system

The study retrospectively tested the CHIC-HS system on an independent cohort of patients diagnosed and treated at Children’s Hospital Los Angeles from 2000 to 2016. The team examined the electronic medical records, imaging and pathology of 96 patients.

The investigators confirmed that the CHIC-HS system successfully predicts how much risk a tumor poses. Children in the lower-risk categories improved with minimal treatment. Those in higher-risk groups required a more intense treatment approach.

The CHIC-HS system also corresponded with long-term patient outcomes after treatment. These outcomes include overall survival, and how long a patient lives without relapse or cancer progression.

For 84 of the patients included in the study, tumor histology collected before treatment was available for analysis. Pretreatment biopsies, which provide tissue samples of the tumor, are not always possible or commonly collected. Having a cohort of this size offered a unique opportunity to assess the relationship between histological characteristics in the tumor, risk category and patient survival.

The investigators discovered that certain histological features predicted patient risk and long-term outcomes, including relapse and survival rates. "Histology may help further enhance the risk stratification scale," concludes Dr. Mascarenhas. "Our hope is that our work will be proven in the ongoing Pediatric Hepatic International Tumor Trial." (NCT03533582, North America; NCT03017326, Europe).

Ongoing research at CHLA focuses on the identification of genetic markers that may further contribute to risk stratification and inform hepatoblastoma treatment.

"This cancer, from being a pretty deadly cancer, is now highly curable in a lot of patients," says Dr. Mascarenhas. "A lot of our goals should really be aimed at limiting the toxicity of treatment in these patients and, for those with high-risk disease, figuring out how to improve their outcomes."

Additional authors of the paper include: Jemily Malvar, MS, of Children’s Hospital Los Angeles and Yueh-Yun Chi, PhD, James Stein, MD, Larry Wang, MD, PhD, Yuri Genyk, MD, and Richard Sposto, PhD, of Children’s Hospital Los Angeles and the Keck School of Medicine at USC.

This research was funded in part by grants UL1TR001855 from the National Center for Advancing Translational Science and 5P30CA014089-44 from the National Cancer Institute of the U.S. National Institutes of Health. The research also received support from the Society for Pediatric Pathology Young Investigator Research Grant and the Names Family Foundation.