Oxford Biodyn Regulatory News (OBD)

Oxford BioDynamics Plc

Massachusetts General Hospital reports results of Interim Analysis from REFINE-ALS biomarker trial by Mitsubishi Tanabe Pharma USA, utilizing Oxford Biodynamics' EpiSwitch® platform for prognostic stratification of fast progression ALS

· The multicenter REFINE-ALS prospective trial for Radicava® (Edaravone), sponsored by Mitsubishi Tanabe Pharma America (MTPA), has been conducted in collaboration with Massachusetts General Hospital and Harvard Medical School

· Dr. James Berry, Director of Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI), shared the interim analysis from the REFINE-ALS prospective trial, including the initial assessment of the EpiSwitch assay

· In the subset of data, baseline EpiSwitch predicted slow versus fast progressors. The proportion of fast progressors predicted by EpiSwitch was higher in the REFINE ALS participants compared with the control population from the ANSWER ALS participants

Oxford, UK - 16 December, 2022 - Oxford BioDynamics, Plc (AIM: OBD, the Company), a biotechnology company developing precision medicine tests for immune health based on the EpiSwitch® 3D genomics platform, announces the initial results from an interim analysis conducted for the REFINE-ALS prospective trial. The results from a subset of data, highlight the blood-based EpiSwitch prognostic stratification of patients with fast progressing Amyotrophic Lateral Sclerosis (ALS). The Phase 4 REFINE-ALS trial (NCT04259255)[1] is a prospective, multicenter study sponsored by Mitsubishi Tanabe Pharma America (MTPA) designed to identify biomarkers that could serve as quantifiable, biological and non-clinical measures of Edaravone's pharmacodynamic effect in ALS. To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 8,000 patients, with over 1.1 million days of therapy, and have been prescribed by nearly 2,000 HCPs].

ALS, also known as Lou Gehrig's disease in the US and motor neuron disease (MND) in the UK, is a terminal disorder that attacks nerve cells that control voluntary muscle movements, like chewing, walking, and breathing, usually leading to death from respiratory failure in 3-5 years[3]. Some patients exhibit fast disease progression, with shorter ( Currently, diagnosis of ALS averages 12 months from the onset of symptoms as there are no definitive, clinically validated measures available. This delay can have a significant impact on the timely treatment of patients, while also limiting the recruitment of early-stage patients to clinical trials.

The EpiSwitch platform was chosen for its ability to prognose at time zero, from a blood sample, which ALS patients are likely to have a fast or slow-progressing disease, before starting Radicava treatment[4]. 

Dr. Gustavo A. Suarez Zambrano, VP of Medical Affairs at MTPA, the US subsidiary of Mitsubishi Tanabe Pharma, said: "We are encouraged by these initial findings. The EpiSwitch platform, as part of other biomarker analyses being conducted in REFINE ALS, will allow us to examine clinically meaningful systemic biomarkers in the REFINE-ALS clinical trial, and enhance our understanding of the role that Radicava® (Edaravone) might play in treating ALS."

Dr. James Berry, Director, NCRI, Massachusetts General Hospital, shared interim analysis data from the study at the 2022 Annual Northeast Amyotrophic Lateral Sclerosis (NEALS)[5], summarized by news coverage here [6].

"We are very interested in investigating the biological impact of RADICAVA on this complex disease by using cutting-edge, meaningful biomarkers," said Dr. Berry. "Non-invasive EpiSwitch biomarkers could enable us to successfully predict slow vs fast progressors at baseline and compare the positive impact of treatment between REFINE-ALS and ANSWER-ALS cohorts, further confirmatory data will be analyzed in the coming future."

The non-invasive EpiSwitch 3D genomics platform continues to deliver clinically meaningful patient stratifications of high efficacy - including applications in neurology, oncology, and autoimmune indications.

OBD is the commercial pioneer and technology leader in the discovery, validation, and monitoring of systemic 3D genomic biomarkers within a liquid biopsy (blood). Today the Company's EpiSwitch CiRT Test (Checkpoint inhibitor Response Test), launched in February 2022, is in clinical use in the US for the accurate prognosis of response to Immune Checkpoint Inhibitor therapy (learn more at: myCiRT.com).

Dr Alexandre Akoulitchev, FRSM, OBD CSO said: "We are delighted with the initial interim results reported about the use of EpiSwitch in the REFINE-ALS trial. Robust clinical biomarkers based on liquid biopsy have been long predicted to change the practice of medicine, and if proven, could become part of medical standards in XXI century."

REFINE-ALS is a NEALS-affiliated clinical trial sponsored by MTPA.

References

[1] ClinicalTrials.gov. Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS), updated 8 Aug 2022. https://clinicaltrials.gov/ct2/show/NCT04259255

[2] Mitsubishi Tanabe Pharma America, Inc. MTPA Presents Interim Data from REFINE-ALS Biomarker Study at 21st Annual NEALS Meeting, 2 Nov 2022. https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-presents-interim-data-from-refine-als-biomarker-study-at-21st-annual-neals-meeting-301666169.html

[3] Swinnen, B., Robberecht, W. The phenotypic variability of amyotrophic lateral sclerosis. Nat Rev Neurol 10, 661-670 (2014). https://doi.org/10.1038/nrneurol.2014.184

[4] Oxford BioDynamics PLC. Oxford BioDynamics notes first patient enrolled in ALS biomarker study sponsored by Mitsubishi Tanabe Pharma America, 25 Oct 2019. https://otp.tools.investis.com/clients/uk/oxford_biodynamics_plc/rns/regulatory-story.aspx?cid=2040&newsid=1339083

[5] Proceedings of the 21st Annual Meeting of NEALS Consortium. Interim Analysis of the Radicava/Edaravone Findings in Biomarkers from ALS (REFINE-ALS) Study, Nov 2022. https://onlinelibrary.wiley.com/doi/epdf/10.1002/mus.27729

[6] ALS News Today. Biomarkers May Determine ALS Progression, Edaravone Response, 7 Nov 2022. https://alsnewstoday.com/news/biomarkers-may-help-determine-als-progression-edaravone-response/

[7] RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.

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About Oxford BioDynamics Plc

Oxford BioDynamics Plc (AIM: OBD) is a global biotechnology company, advancing personalized healthcare by developing and commercializing precision medicine tests for life-changing diseases.

Its flagship product is EpiSwitch® CiRT (Checkpoint Inhibitor Response Test) for cancer, a predictive immune response profile for immuno-oncology (IO) checkpoint inhibitor treatments, launched in February 2022.

In March 2021, the Company launched its first commercial prognostic test, EpiSwitch® CST (Covid Severity Test) and the first commercially available microarray kit for high-resolution 3D genome profiling and biomarker discovery, EpiSwitch® Explorer Array Kit.

The Company has developed a proprietary 3D genomic biomarker platform, EpiSwitch®, which can build molecular diagnostic classifiers for prediction of response to therapy, patient prognosis, disease diagnosis and subtyping, and residual disease monitoring in a wide range of indications.

Oxford BioDynamics has participated in more than 40 partnerships with big pharma and leading institutions including Pfizer, EMD Serono, Genentech, Roche, Biogen, Mayo Clinic, Massachusetts General Hospital and Mitsubishi Tanabe Pharma.

The Company has created a valuable technology portfolio, including biomarker arrays, molecular diagnostic tests, bioinformatic tools for 3D genomics and an expertly curated 3D genome knowledgebase comprising hundreds of millions of data points from over 10,000 samples in more than 30 human diseases.

OBD is headquartered in Oxford, UK and is listed on AIM of the London Stock Exchange. It also has a commercial office in Gaithersburg, MD, USA and a reference laboratory in Penang, Malaysia.

For more information, please visit the Company's website, www.oxfordbiodynamics.com, or follow on Twitter or LinkedIn.

About EpiSwitch®

The 3D configuration of the genome plays a crucial role in gene regulation. By mapping this architecture and identifying abnormal configurations, EpiSwitch® can be used to diagnose patients or determine how individuals might respond to a disease or treatment.

Built on over 10 years of research, EpiSwitch® is Oxford Biodynamics' award-winning, proprietary platform that enables screening, evaluation, validation and monitoring of 3D genomic biomarkers. The technology is fully developed, based on testing of over 10,000 samples in 30 disease areas, and reduced to practice.

In addition to stratifying patients with respect to anticipated clinical outcome, EpiSwitch® data offer insights into systems biology and the physiological manifestation of disease that are beyond the scope of other molecular modalities. The technology has performed well in academic medical research settings and has been validated through its integration in biomarker discovery and clinical development with big pharma.

About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone)

The U.S. Food and Drug Administration (FDA) approved RADICAVA® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). RADICAVA is administered in 28-day cycles by IV infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.[7]

Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc. (MTDA), commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, RADICAVA was approved for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), China (July 2019), Indonesia (July 2020), Thailand (April 2021) and Malaysia (December 2021).