Hard, not soft, plaque buildup may predict risk of heart attack
Nicox Announces Recruitment Of Last Patient In US In Denali Phase 3 Trial Of NCX 470
According to the company, the Denali trial is evaluating NCX 470 safety and efficacy in patients with open-angle glaucoma or ocular hypertension and topline results continue to be expected in H2 2025
(Image credit: Adobe Stock)
Nicox SA announced the recruitment and randomization of the last patient in the U.S. In its Denali Phase 3 trial of NCX 470.
The company noted that NCX 470 is a novel NO-donating bimatoprost eye drop currently in Phase 3 clinical development.
According to a news release, the Denali trial is evaluating NCX 470 safety and efficacy in patients with open-angle glaucoma or ocular hypertension and topline results continue to be expected in H2 2025, based on this completion of recruitment of patients in the U.S. And continuing good progress of the trial in China, where recruitment of patients is still ongoing. More than 95% of the total target number of patients have been randomized in this trial so far.1
Doug Hubatsch, Chief Scientific Officer of Nicox, offered his thanks to patients, the clinical sites, the company's internal team and investors for their efforts and support in enabling Nicox to reach this important milestone, the completion of US recruitment in the Denali trial.
"We will remain fully focused on NCX 470 and on continuing recruitment of patients in China to complete Denali as we move towards expected topline results in H2 2025," he said in the news release.
The company noted its Denali trial is evaluating the intraocular pressure (IOP) lowering efficacy of once-daily dosed NCX 470 ophthalmic solution 0.1% compared to latanoprost ophthalmic solution 0.005% in patients with open-angle glaucoma or ocular hypertension.
The trial is a multi-country (U.S. And China) clinical trial financed equally by Nicox and Ocumension, Nicox's exclusive licensee for China, Korea and Southeast Asia. The Denali trial, together with the Mont Blanc trial, was designed to fulfill the regulatory requirements to support New Drug Application (NDA) submissions of NCX 470 in the U.S. And China. The U.S. NDA for NCX 470 is expected to be submitted in H1 2026.1
Topline results from the first Phase 3 trial, Mont Blanc, showed the IOP-lowering effect from baseline was 8.0 to 9.7 mmHg for NCX 470 vs. 7.1 to 9.4 mmHg for latanoprost. Statistical non-inferiority was met vs. Latanoprost in the primary efficacy analysis and 4 out of 6 timepoints additionally demonstrated superiority; the trial therefore met the efficacy requirements for approval in the U.S. NCX 470 was well tolerated and discontinuation rates were low.1
Reference:Clinical Trial Recruitment And AI: Easing The Burden Of GA Studies
Researchers train deep learning model on OCT scans to ID patients eligible for GA trials.
(Image Credit: AdobeStock/ALL YOU NEED studio)
Reviewed by Dominic J. Williamson
Artificial intelligence (AI) is finding its niche in medical research, with more applications being explored. A team of researchers in the United Kingdom (UK) are exploring AI to simplify appropriate patient recruitment in trials focused on geographic atrophy (GA) associated with age-related macular degeneration (AMD).
First authors and PhD candidates Dominic J. Williamson and Robbert R. Struyven point out that developing new treatments is expensive, can take decades, and relies heavily on data from clinical trials to demonstrate the safety and efficacy of the new treatments.1
"Today, challenges in recruiting participants stand as a significant obstacle in clinical trials.2,3 Time taken to fully recruit a trial adds significantly to the cost and can lead to delays, with up to 86% of trials not finishing on schedule,"4,5 the researchers write.
Williamson and Struyven are from the National Institute for Health and Care Research Biomedical Research Centre for Ophthalmologyat Moorfields Eye Hospital National Health Service Foundation Trust in London, UK, and the Centre for Medical Imaging Computing at University College London.
In light of this, Williamson, Struyven, and their colleagues conducted a cross-sectional study to explore the role of AI in clinical trial recruitment of individuals with GA. The investigators used a retrospective data set from theINSIGHT Health Data Research Hub at Moorfields Eye Hospital that included 306,651 patients (602,826 eyes) with suspected retinal disease who underwent optical coherence tomography (OCT) imaging between January 1, 2008, and April 10, 2023.
The investigators recount that they trained a deep learning model on OCT scans to identify patients potentially eligible for GA trials using AI-generated segmentations of retinal tissue. This method was compared with a keyword-based electronic health record (EHR) search for accuracy. They also carried out clinical validation with fundus autofluorescence (FAF) images to calculate the positive predictive value (PPV) of this approach by comparing AI predictions with expert assessments.
The main outcome measure was the PPV of AI for identifying trial-eligible patients. The secondary outcome was the intraclass correlation between the GA areas segmented on FAF by experts and AI-segmented OCT scans.
AI performanceThe authors report, "The AI system shortlisted a larger number of eligible patients with greater precision (1139[;] PPV, 63%; 95% CI, 54%-71%) compared [with] the electronic health records search (693[;] PPV, 40%; 95% CI, 39%-42%)." When AI and the EHR approach were combined, a total of 604 eligible patients with a PPV of 86% (95% CI, 79%-92%) were identified.
The intraclass correlation of GA area segmented on FAF vs AI-segmented area on OCT was 0.77 (95% CI, 0.68-0.84) for eligible cases. The AI also adjusts to the distinct imaging criteria from several clinical trials, generating tailored shortlists ranging from 438 to 1817 patients, the investigators report.
The investigators conclude that AI has the potential to streamline the clinical trial process, first by identifying patients with greater ease and later in other stages of the clinical trial process.
The researchers noted that they demonstrated that an AI tool can facilitate clinical trial recruitment in AMD.
"Given the intense clinical trial activity currently underway, we believe that implementing this AI-driven strategy will offer a scalable solution to the recruitment challenges in GA trials," they concluded. "Our future efforts will concentrate on assessing the real-world robustness of this AI solution, ensuring it performs well with routinely collected images of varying quality, protocols, [and] equipment and across diverse populations."
They also believe that, in addition to prescreening patients, AI may enable site feasibility assessments, data-driven protocol design, and cost reduction. Once treatments are available, similar AI systems could also be used to identify individuals who may benefit from treatment, they hypothesize.
Dominic J. Williamson
E: dominic.Williamson.21@ucl.Ac.Uk
Williamson is a PhD candidate and has no financial interest in this subject matter.
Robbert R. Struyven
E: robbert.Struyven.20@ucl.Ac.Uk
Struyven is a PhD candidate and has no financial interest in this subject matter. References:
NurExone's ExoPTEN Being Studied As Glaucoma Treatment For US$3.4 Billion Market
TORONTO and HAIFA, Israel, June 28, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX), (OTCQB: NRXBF), (Germany: J90) (the "Company" or "NurExone"), a pioneering biopharmaceutical company, is pleased to announce a pre-clinical study to explore the potential of NurExone's exosome-based therapies in regenerating damaged optic nerves. The study, initiated by renowned ophthalmologist and serial entrepreneur Prof. Michael Belkin from Tel Aviv University's Goldschleger Eye Research Institute, and led by the principal investigators Prof. Ygal Rotenstreich and Dr. Ifat Sher from the Sheba Medical Center Eye Institute1, is the latest step in expanding potential clinical indications for Nurexone Biologic's exosome-loaded drugs.
The optic nerve, a critical component of the visual system, transmits visual information from the retina to the brain. Since the optic nerve, part of the central nervous system, does not regenerate spontaneously, and damage thereto, whether due to injury, glaucoma, or other conditions, can result in significant vision loss and blindness. According to experts, current treatments are limited and focus on preventing additional damage rather than regenerating or repairing damaged nerves. Based on NurExone's trials on the spinal cord, which is also part of the central nervous system, exosome-loaded drugs may be able to change this paradigm with their potentially regenerative properties with respect to damaged nerves.
The global optic nerve disorders treatment market size was valued at US$3.4 billion in 2021, and is projected to reach US$5.3 billion by 2031, growing at a Compound Annual Growth Rate of 4.5% from 2022 to 2031. Key players in the optic nerve disorder treatment market, include AbbVie Inc., Novartis AG, Santen Pharmaceutical Co., Ltd., and Teva Pharmaceutical Industries Ltd. ⁽¹⁾ ⁽²⁾
Prof. Michael Belkin commented: "We are excited to perform preclinical studies on optical nerve regeneration at the Sheba Medical Center Eye Institute. If this experimental direction is successful, I believe we may be able to translate the success quickly to clinical practice. Our ultimate goal is to restore and improve the quality of life for individuals affected by optic nerve diseases and injuries."
Dr. Lior Shaltiel, CEO of Nurexone Biologic, added: "This investigation is part of our ongoing commitment to using our ExoTherapy platform to advance the field of regenerative medicine. Through pre-clinical investigations, we aim to address this critical and unmet medical need and bring hope to individuals suffering from vision loss. This also represents the next phase in our strategy to expand the clinical indications for our exosome-loaded drugs, paving the way for future breakthroughs."
Learn more about Prof. Belkin's vision for treating glaucoma with ExoPTEN in NurExone's latest podcast available on NurExone's website.
⁽¹⁾ Optic Nerve Disorders Treatment Market Research, 2031. Available from: https://www.Alliedmarketresearch.Com/optic-nerve-disorders-treatment-market-A14042
⁽²⁾ Optic Nerve Disorders Treatment Market 2024 Business Insights, Development Plans, And Growth Analysis Report To 2033. Available from: https://medium.Com/@bharadwajvanteru/optic-nerve-disorders-treatment-market-2024-business-insights-development-plans-and-growth-d3384e03ea94
About The Tel Aviv University's Goldschleger Eye Research Institute and Sheba Medical Center Eye Institute
The Goldschleger Eye Research Institute focuses on studying the visual system in health and disease and transform laboratory research into clinical practice. The Institute is part of the Tel Aviv University School of Medicine, located at Sheba Medical Center in Tel HaShomer. This location enables close collaboration between basic and clinical research. Sheba was recently ranked as one of the ten best hospitals in the world. Prof. Belkin, the founder and first director of the Institute, is a member of NurExone's Scientific Advisory Board.
Prof. Ygal Rotenstreich and Dr. Ifat Sher are affiliated with Sheba Medical Center Eye Institute. The Eye Institute at Sheba Medical Center is a diverse team that utilizes cutting-edge technological advancements in ophthalmology to deliver patient care of the utmost safety and quality standards. Comprising an inpatient department, day care unit, surgery rooms, and various outpatient clinics, the Eye Institute boasts a staff of over 50 eye specialists, 30 skilled nurses, and medical technicians with specialized training.
About NurExone Biologic Inc.
NurExone Biologic Inc. Is a TSXV listed pharmaceutical Company that is developing a platform for biologically-guided exosome-based therapies to be delivered, non-invasively, to patients who have suffered Central Nervous System injuries. The Company's first product, ExoPTEN for acute spinal cord injury, was proven to recover motor function in 75% of laboratory rats when administered intranasally. ExoPTEN has been granted Orphan Drug Designation by the FDA. The NurExone platform technology is expected to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications.
For additional information, please visit www.Nurexone.Com or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube.
For more information, please contact:
Dr. Lior ShaltielChief Executive Officer and DirectorPhone: +972-52-4803034Email: info@nurexone.Com
Thesis Capital Inc.Investment Relation - CanadaPhone: +1 905-347-5569Email: IR@nurexone.Com
Dr. Eva ReuterInvestment Relation - GermanyPhone: +49-69-1532-5857Email: e.Reuter@dr-reuter.Eu
FORWARD-LOOKING STATEMENTS
Expectations and projections about its future results. Wherever possible, words such as "may", "will", "should", "could", "expect", "plan", "intend", "anticipate", "believe", "estimate", "predict" or "potential" or the negative or other variations of these words, or similar words or phrases, have been used to identify these forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements relating to the Company's pre-clinical trials, including the pre-clinical trials being the latest step in the Company's plans in expanding potential clinical indications for the Company's Biologic's exosome-loaded drugs, the potential benefits to individuals affected by optic nerve diseases and injuries; the potential for exosome-loaded drugs in regenerating or repairing damaged nerve; the Company's ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine and bring hope to individuals suffering from vision loss; and the Company's expectations to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications.
These statements reflect management's current beliefs and are based on information currently available to management as at the date hereof. In developing the forward-looking statements in this press release, we have applied several material assumptions, including the Company's ability to carry out its pre-clinical trials and realize upon the stated benefits of the pre-clinical trials; the Company's ability to realize upon the stated potential for exosome-loaded drugs in regenerating or repairing damaged nerves; the Company's ability to maintain its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine; and the Company's ability to deliver upon its expectations to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications.
Forward-looking statements involve significant risk, uncertainties and assumptions. Many factors could cause actual results, performance or achievements to differ materially from the results discussed or implied in the forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to the Company's early stage of development; lack of revenues to date; government regulation; market acceptance for its products; rapid technological change; dependence on key personnel; protection of the Company's intellectual property; dependence on the Company's strategic partners; the fact that preclinical drug development is uncertain, and the drug product candidates of the Company may never advance to clinical trials; the fact that results of preclinical studies and early-stage clinical trials may not be predictive of the results of later stage clinical trials; the uncertain outcome, cost, and timing of product development activities, preclinical studies and clinical trials of the Company; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; the potential inability to obtain or maintain regulatory approval of the drug product candidates of the Company; the introduction of competing drugs that are safer, more effective or less expensive than, or otherwise superior to, the drug product candidates of the Company; the initiation, conduct, and completion of preclinical studies and clinical trials may be delayed, adversely affected or impacted by unforeseen issues; the potential inability to obtain adequate financing; the potential inability to obtain or maintain intellectual property protection for the drug product candidates of the Company; risks that the Company's intellectual property and technology won't have the intended impact on the Company and/or its business; the Company's inability to carry out its pre-clinical trials and realize upon the stated benefits of the pre-clinical trials; the Company's inability to realize upon the stated potential for exosome-loaded drugs in regenerating or repairing damaged nerves; the Company's inability to maintain its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine; and the Company's inability to deliver upon its expectations to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications; and the risks discussed under the heading "Risk Factors" on pages 29 to 36 of the Company's Annual Information Form dated March 30, 2023, a copy of which is available under the Company's SEDAR+ profile at www.Sedarplus.Ca. These factors should be considered carefully and readers should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in this press release are based upon what management believes to be reasonable assumptions, the Company cannot assure readers that actual results will be consistent with these forward-looking statements. These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update or revise them to reflect new events or circumstances, except as required by law.
Neither TSXV nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release.
Dr. Lior Shaltiel, Professor Michael Belkin, Professor Ygal Rotenstreich and Dr. Ifat Sher. (Credit: Edenvideo)
_________________1 Photo: Dr. Lior Shaltiel, Prof. Michael Belkin, Prof. Ygal Rotenstreich and Dr. Ifat Sher. (Credit: Edenvideo)
A photo accompanying this announcement is available at https://www.Globenewswire.Com/NewsRoom/AttachmentNg/8d4b0f07-b60a-4068-a562-18f53cef6575
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