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Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

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Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale (‘ONLS’), after 4.5 years of total trial time.

A live conference call and webcast will be held tomorrow, Thursday April 29th at 2:00 p.m. CET
(8:00 a.m. ET)

PARIS, France, April 28, 2021, 6:00 p.m. CET – Pharnext SA (FR0011191287 – ALPHA) (the ‘Company’), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY™ platform, today announces new results from an interim analysis of an ongoing open-label follow-up extension study (‘PLEO-CMT-FU trial’) following the first double-blind, placebo-controlled Phase III study (‘PLEO-CMT trial’) of PXT3003 for the treatment of Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’). There is currently no approved drug treatment for CMT1A.

In January 2020, the company reported interim results suggesting sustained safety and efficacy of PXT3003 in patients with mild-to-moderate CMT1A after 24 months of total trial time (PLEO-CMT and PLEO-CMT-FU Period 1 trials). The new results announced today continue to show sustained treatment benefits for CMT1A patients treated with PXT3003 at High Dose (‘HD’) in the PLEO-CMT-FU Period 2 trial with a data readout at 54 months of total trial time (double-blind + open-label). Highlights from an analysis of available data include:

  • PXT3003 was safe and well tolerated. Data are consistent with observed safety profile in prior clinical trials.
  • Data on the Overall Neuropathy Limitations Scale (‘ONLS’), which measures patients’ functional motor disability, are as follows (please refer to the diagram below for graphical illustration):
    • During PLEO-CMT (double-blind Phase III study), patients treated with placebo on average declined on ONLS while patients treated with PXT3003 on average improved. The best efficacy signal was observed in the cohort of patients treated with PXT3003 HD.
    • During PLEO-CMT-FU (open-label Phase III Extension study), on average patients improved on ONLS across all patient cohorts.
      • Patients treated with placebo declined on ONLS during the double-blind phase, but then improved when switched to PXT3003 in the ongoing open-label phase.
      • Patients treated with PXT3003 during the double-blind phase continue to improve when pursuing their treatment with PXT3003 in the ongoing open-label phase.
  • New results with available ONLS data after 54 months of total trial time suggest a better efficacy signal with PXT3003 HD in this patient population.

Adrian Hepner, MD, PhD, Chief Medical Officer of Pharnext, said:Although these new data were generated from an open-label study, the findings are consistent with the safety and efficacy results of PXT3003 observed in prior clinical studies in CMT1A. In addition, the fact we have just initiated the PREMIER trial in a similar patient population, using the same High Dose of PXT3003 and measuring the same efficacy endpoint ONLS, reinforces our confidence in the potential positive outcome of our ongoing pivotal Phase III study.

Florian P. Thomas, MD, PhD, Founding Chair & Professor, Department of Neurology, Hackensack University Medical Center & Hackensack Meridian School of Medicine (NJ, USA) and U.S. lead investigator of the PLEO-CMT trial, said:These new results from the interim analysis of the ongoing open-label Phase III extension study show very promising safety and efficacy data of PXT3003 in CMT1A after more than four years of treatment. It reinforces our hope that PXT3003 could be the first treatment approved for patients suffering from this debilitating disease.

Results of First Double-Blind Phase III (PLEO-CMT) & Open-Label Extension (PLEO-CMT-FU)
Studies of PXT3003 with an ONLS Data Readout at 54 Months of Total Trial Time

aCohort of CMT1A patients treated with PXT3003 High Dose during PLEO-CMT and ongoing PLEO-CMT-FU trials
bCohort of CMT1A patients treated with PXT3003 Low Dose during PLEO-CMT + PLEO-CMT-FU Period 1, and then switched to PXT3003 High Dose during PLEO-CMT-FU period 2
cCohort of CMT1A patients treated with placebo during PLEO-CMT, PXT3003 Low Dose or High Dose during PLEO-CMT-FU Period 1 and PXT3003 High Dose during PLEO-CMT-FU Period 2
Please refer to a graphic illustration of first double-blind Phase III (PLEO-CMT) and open-label extension (PLEO-CMT-FU) studies design in the “About the PLEO-CMT-FU Trial” section below.

Conference Call Details

Pharnext will host a live conference call and webcast at 2:00 p.m. CET / 8:00 a.m. ET tomorrow Thursday April 29th, 2021 to discuss the data. The conference call may be accessed by dialing +33 (0)1 70 70 07 81 (France), +1 646 7413 167 (USA), or +44 (0) 2071 928338 (International) and using conference ID 6197704. The live webcast and accompanying slides can be accessed via the Pharnext’s website at https://pharnext.com/en in the ‘Events’ section or by clicking here. An archived webcast will then be available on the Pharnext’s website approximately 4 hours after the conference call.

About the PLEOCMT Trial

The PLEO-CMT trial was an international, randomized, double-blind, placebo-controlled, Phase III study evaluating the efficacy and safety of PXT3003 in patients with CMT1A, over a 15-month period. Two dose levels, named low dose (‘LD’) and high dose (‘HD’), of PXT3003 in comparison to placebo were tested in patients diagnosed with mild-to-moderate CMT1A (HD equals double LD). A total of 323 patients were enrolled in 29 centers across Europe, the U.S. and Canada by December 2016 and last-patient-last-visit occurred in March 2018. Due to an unexpected issue in the HD formulation, the HD arm was prematurely stopped in September 2017. A revised statistical analysis plan was developed to take into account the premature HD arm discontinuation. Analysis of the primary endpoint, Overall Neuropathy Limitations Scale (‘ONLS’) from all investigated populations in the HD arm suggested preliminary efficacy in humans. The study further demonstrated the safety and tolerability of PXT3003. Further information on the PLEO-CMT trial, including study results, can be found on the ClinicalTrials.gov website (study identification number: NCT03023540) here.

About the PLEO-CMT-FU Trial

All randomized CMT1A patients who completed the PLEO-CMT trial (treated with PXT3003 or placebo) were eligible to pursue treatment with PXT3003 in the PLEO-CMT-FU trial. This trial enrolled a total of 187 patients and was designed to primarily assess the long-term safety and tolerability of PXT3003. It was initially planned to be a double-blind, nine-month, Phase III follow-up extension study where patients treated with PXT3003 in the PLEO-CMT trial were eligible to continue their treatment at the same dose (High dose ‘HD’ or Low Dose ‘LD’). Patients treated with placebo in the PLEO-CMT trial were randomized in PLEO-CMT-FU to receive LD or HD of PXT3003. Due to the PXT3003 HD formulation issue which occurred during the PLEO-CMT trial, the HD arm was discontinued in September 2017. Consequently, the PLEO-CMT-FU trial became an open-label study which is divided in 2 periods:

  • Period 1 (9-month treatment period) from March 2017 to April 2019. Patients randomized to PXT3003 LD in PLEO-CMT continued on the same dose. Patients randomized to PXT3003 HD in PLEO-CMT continued on the same dose, but it was given as twice the volume of PXT3003 LD formulation after the PXT3003 HD formulation issue. Patients randomized to placebo in PLEO-CMT continued only on PXT3003 LD after the HD formulation issue.
  • Period 2 from July 2018 (still on-going). The 153 patients who entered in PLEO-CMT-FU Period 2 were all switched to PXT3003 HD given as twice the volume of PXT3003 LD formulation.

In PLEO-CMT-FU, on top of safety and tolerability of PXT3003 which is evaluated every 3 months, long-term efficacy is evaluated with the Overall Neuropathy Limitations Scale (‘ONLS’) measured every 6 months. Results from the PLEO-CMT-FU trial will be reported on a yearly basis.
Further information on the PLEO-CMT-FU trial can be found on the ClinicalTrials.gov website (study identification number: NCT03023540) here.

Design of First Double-Blind Phase III (PLEO-CMT) and
Open-Label Extension (PLEO-CMT-FU) Studies of PXT3003

About the PREMIER Trial

The PREMIER trial is an international, randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, evaluating the efficacy and safety of PXT3003 versus placebo in mild-to-moderate CMT1A patients, over a 15-month period. The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose (‘HD’) tested in the prior Phase III trial (‘PLEO-CMT’). As agreed with regulatory agencies, the primary efficacy endpoint will be the Overall Neuropathy Limitations Scale (‘ONLS’) which measures functional motor disability. The secondary endpoints include the following outcome measures: 1) 10-Meter Walk Test (‘10mWT’), 2) Quantified Muscular Testing (bilateral foot dorsiflexion dynamometry), 3) Patient Global Impression of Severity (‘PGI-S’), 4) Patient Global Impression of Change (‘PGI-C’), 5) Charcot-Marie-Tooth Neuropathy Score, version 2 (‘CMTNS-v2’), and 6) Quantified Muscular Testing (hand grip). Safety and tolerability will be monitored throughout the study. Further information on the PREMIER trial can be found on the ClinicalTrials.gov website (study identification number: NCT04762758) here.

About Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

Charcot-Marie-Tooth (‘CMT’) disease encompasses a heterogeneous group of inherited, severe, debilitating, progressive and chronic peripheral neuropathies. CMT1A, the most common type of CMT, is an orphan disease with a prevalence of 1/5000 people affecting about 150,000 people in Europe and the U.S. and about 1,500,000 people worldwide. The genetic mutation responsible for CMT1A is a duplication of the PMP22 gene coding for a peripheral myelin protein. The duplication of this gene results in overexpression of the PMP22 protein and failure of Schwann cells to produce normal myelin (neuronal sheath). The lack of a normal myelin structure and function leads to abnormal peripheral nerve conduction and axonal loss. As a result of peripheral nerve degradation, patients suffer from progressive muscle atrophy in both the legs and arms causing problems with walking, running and balance as well as abnormal hand functioning. They might also suffer from mild to moderate sensory disorders. First symptoms usually appear during adolescence and will progressively evolve throughout life. Patients with the most severe form of CMT1A end up in wheelchairs, representing at least 5% of cases. To date, no curative or symptomatic medications have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery. More information can be found at https://pharnext.com/en/disease/charcot-marie-tooth.

About PXT3003

PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day. The three individual components of PXT3003 were selected to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves that are an essential part of the pathophysiology of this disease. PXT3003 could also have a positive effect on other cellular types of the motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells. PXT3003 has shown promising and consistent results across preclinical and clinical studies in Phase II and Phase III (PLEO-CMT and PLEO-CMT-FU). More information can be found at https://pharnext.com/en/pipeline/pxt3003.

About Pharnext

Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapeutics for orphan and common neurodegenerative diseases that currently lack curative and/or disease-modifying treatments. Pharnext has two lead products in clinical development. PXT3003 completed an international Phase III trial with positive topline results for the treatment of Charcot-Marie-Tooth disease type 1A (‘CMT1A’) and benefits from orphan drug status in Europe and the United States. An international pivotal Phase III study of PXT3003 in CMT1A, the PREMIER trial, is currently ongoing. PXT864 has generated encouraging Phase II results in Alzheimer’s disease and will be advanced through partnerships. Pharnext has developed a new drug discovery paradigm based on big genomics data and artificial intelligence: PLEOTHERAPY™. Pharnext identifies and develops synergic combinations of drugs called PLEODRUG™. More information can be found at www.pharnext.com.

Pharnext is listed on the Euronext Growth Stock Exchange in Paris (ISIN code: FR0011191287).

Disclaimer

This press release contains certain forward-looking statements concerning Pharnext and its business, including in respect of timing of and prospects for clinical trials and regulatory submissions of the Company’s product candidates as well as a potential financing transaction, the use of proceeds therefrom and cash runway. Such forward-looking statements are based on assumptions that Pharnext considers to be reasonable. However, there can be no assurance that the estimates contained in such forward-looking statements will be verified, which estimates are subject to numerous risks including the risks set forth in Pharnext’s URD approved by the AMF on November 9, 2020 under number N° R. 20-029 as well as in its annual periodic management reports and press releases (copies of which are available on www.pharnext.com) and to the development of economic conditions, financial markets and the markets in which Pharnext operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Pharnext or not currently considered material by Pharnext. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Pharnext to be materially different from such forward-looking statements. Pharnext disclaims any intention or obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Pharnext shares in any country, including the United States. The Company’s securities may not be offered or sold in the United States absent registration or an exemption from registration; any public offering of securities to be made in the United States will be made by means of a prospectus that may be obtained from the issuer that will contain detailed information about the Company and management, as well as financial statements.

Contacts

David Horn Solomon
Chief Executive Officer
[email protected]
+33 (0)1 41 09 22 30
   

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Automotive Digital Cockpit Domain Controller Power Expected to Double by 2030

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NEW YORK, March 28, 2024 /PRNewswire/ — The lengthening lifecycle of vehicles is driving automotive Original Equipment Manufacturers (OEMs) to plan several years of customer support through regular Over-the-Air (OTA) updates. Achieving this requires a digital Cockpit Domain Controller (CDC) with an architecture that supports long-term updating and maintenance. According to a new report from global technology intelligence firm ABI Research, the computing power of the CDC will increase significantly over the next few years, with graphical computing power and deep-learning processing power (for AI-powered functions) expected to double by 2030 for an average mid-market CDC.

OEMs are beginning to plan for several years of support, both in software patches and bug fixes, and for delivering new added value features to the driving experience. “This support and feature roadmap requires a hardware and software architecture that supports the continuous updating of vehicles over time. OEMs need a system that accommodates quick, targeted updates by shipping vehicles with planned overhead in computing power, software containerization, and robust hypervisors. These can be accommodated by silicon vendors such as NVIDIA or Qualcomm with their suite of high-powered System-on-Chips (SoCs), along with hypervisor and software specialists such as Blackberry QNX. The ecosystem hasn’t fully adjusted to OTA updates in mixed-criticality systems like the digital cockpit domain controller yet, with most OEMs speculating about the level of computing power that is needed for several years of support and few going to their silicon and tier-one partners with roadmaps of planned features,” explains Abu Miah, Smart Mobility and Automotive Analyst at ABI Research.
The computing power of the CDC will increase significantly over time. The Tera Floating-Point Operations per Second (TFLOPS) of an average mid-market CDC is expected to rise from 1 TFLOPS in 2023 to 2.5 TFLOPS by 2030. Miah adds, “One of the primary drivers of this increase is the implementation of a larger number of higher resolution screens in the vehicle to accommodate new high-end gaming and video-on-demand features.”
Building a ‘future-proofed’ CDC is not as simple as throwing compute power at the vehicle. “OEMs, tier ones, and silicon vendors must all work toward an ecosystem of hardware and software agnosticism, modular architecture, and collaborative software development if they are to match customers’ expectations of updates, patches, and bug fixes from the consumer electronics space,” Miah concludes.
These findings are from ABI Research’s Future-Proofing Digital Cockpit Domain Controllers application analysis report. This report is part of the company’s Smart Mobility and Automotive research service, which includes research, data, and ABI Insights. Based on extensive primary interviews, Application Analysis reports present an in-depth analysis of key market trends and factors for a specific technology.
About ABI Research
ABI Research is a global technology intelligence firm uniquely positioned at the intersection of technology solution providers and end-market companies. We serve as the bridge that seamlessly connects these two segments by providing exclusive research and expert guidance to drive successful technology implementations and deliver strategies proven to attract and retain customers.
ABI Research是一家全球性的技术情报公司,拥有得天独厚的优势,充当终端市场公司和技术解决方案提供商之间的桥梁,通过提供独家研究和专业性指导,推动成功的技术实施和提供经证明可吸引和留住客户的战略,无缝连接这两大主体。
For more information about ABI Research’s services, contact us at +1.516.624.2500 in the Americas, +44.203.326.0140 in Europe, +65.6592.0290 in Asia-Pacific, or visit www.abiresearch.com.
Contact Info: 
GlobalDeborah Petrara Tel: +1.516.624.2558 [email protected] 
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ACL Digital in Collaboration with AWS and Infineon to Participate at Embedded World 2024

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SAN JOSE, Calif., March 28, 2024  /PRNewswire/ — ACL Digital, an ALTEN group company, is a pioneer in design-led digital experience, innovation, enterprise IT modernization, and product engineering services, announced that ACL Digital, in collaboration with AWS and Infineon, is going to showcase its AWS IoT & Cloud capabilities at Embedded World 2024, Hall 4 (Booth #4-552) from April 9 to April 11 in Nuremberg, Germany.

ACL Digital, a top-tier AWS Services partner, propels organizations of all sizes to navigate digital transformation to accelerate time-to-market. The company provides comprehensive support, from adopting to modernizing IT infrastructure on AWS. By leveraging expertise in architecture, security, migration, and operations, ACL Digital unlocks the full potential of AWS, streamlining IoT and cloud journeys and fast-tracking business growth and innovation.
The AWS Advanced Tier partnership enables ACL Digital to leverage AWS expertise, its robust support ecosystem and best practices to deliver customer delight.
ACL Digital offers visitors at Embedded World 2024 a chance to experience the exclusive demo of a Smart Stove Solution, built by leveraging the AWS Cloud and Infineon platform and how it has added value to our customers.
With over 100 AWS-certified experts, ACL Digital empowers clients to achieve breakthrough results in their digital transformation. Also, the leading digital transformation company supports global clients in navigating the complexities of cloud implementation, migration and digital transformation with ease and helping them unlock new growth opportunities.
About AWS
Since 2006, Amazon Web Services has been the world’s most comprehensive and broadly adopted cloud. AWS has been continually expanding its services to support virtually any workload, and it now has more than 240 fully featured services for compute, storage, databases, networking, analytics, machine learning and artificial intelligence (AI), Internet of Things (IoT), mobile, security, hybrid, media, and application development, deployment, and management from 105 Availability Zones within 33 geographic regions, with announced plans for 12 more Availability Zones and four more AWS Regions in Malaysia, New Zealand, Thailand, and the AWS European Sovereign Cloud. Millions of customers—including the fastest-growing startups, largest enterprises, and leading government agencies—trust AWS to power their infrastructure, become more agile, and lower costs. To learn more about AWS, visit https://aws.amazon.com .
About Infineon
Infineon Technologies AG is a global semiconductor leader in power systems and IoT. Infineon drives decarbonization and digitalization with its products and solutions. The company has around 58,600 employees worldwide and generated revenue of about €16.3 billion in the 2023 fiscal year (ending 30 September). Infineon is listed on the Frankfurt Stock Exchange (ticker symbol: IFX) and in the USA on the OTCQX International over-the-counter market (ticker symbol: IFNNY). To learn more about Infineon, visit https://www.infineon.com/
About ACL Digital
ACL Digital an ALTEN Group Company, is a digital product innovation and engineering leader. We help our clients design and build innovative products (AI, Cloud, and Mobile ready), content and commerce-driven platforms, and connected, converged digital experiences for the modern world through a design-led Digital Transformation framework.
Headquartered in Silicon Valley, ACL Digital is a leader in design-led digital experience, innovation, enterprise modernization, and product engineering services converging to Technology, Media & Telecom. The company has a workforce of 57,000+ spread across more than 30+ countries.

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Gilbarco Veeder-Root champions fuel efficiency, clean fuels and diesel rebate solutions in mining

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JOHANNESBURG, March 28, 2024 /PRNewswire/ — Global leader in technology solutions OEM Gilbarco Veeder-Root (GVR) is dedicated to enhancing operational performance by delivering comprehensive end-to-end wetstock, industrial, mining, and business-to-business solutions, all tailored to meet the specific needs of the customer.

 
 
By encompassing every aspect of mining operations, GVR commercial and industrial Middle East and Africa director Westtar Kapito says, “the company is setting new benchmarks for fuel efficiency, safety and sustainability within the mining industry”.
As part of a holistic approach to mining excellence, Kapito explains that Gilbarco’s integrated fuel and fleet management technology solutions are designed to address the multifaceted challenges of the mining industry.
Some of these facets include wetstock control, equipment maintenance and management, fleet management and automation, compliance monitoring and environmental sustainability, as well as driving productivity and profitability through innovation.
In addition, the introduction of Gilbarco’s clean fuel solution exemplifies the company’s commitment to maintaining equipment integrity and performance.
This technology, GVR says, monitors in real-time the status of up to 16 “clean fuels” key performance matrices, thereby ensuring that dirty fuel is flagged and not transferred into mining equipment which would affect engines and injectors and thus lower productivity.
Gilbarco’s comprehensive site automation solutions empower mining companies with critical data analytic insights, facilitating efficient monitoring and management of fleet and fuel inventory. Gilbarco’s dataFLEX360 platform plays a pivotal role, offering near real-time reporting and analytics to drive informed decision-making and operational agility.
dataFLEX360 is a Web-based, cloud-hosted strategic operational insights platform. The system ensures accurate, reliable and relevant reporting of all fuel, fleet and asset transactions, and provides for proactive corrective measures to reduce complex reporting and gives a consolidated and comprehensive view across all sites and assets.
With reconciliations at its core, dataFLEX360 provides solution accuracy on operational data.
Integral to the company’s solutions is compliance with Global Industry Standards and environmental stewardship, from leak detection to vapour recovery and clean fuel technologies. Gilbarco’s products are designed to ensure compliance and minimise the carbon footprint of mining operations.
Additionally, through the company’s innovative telematics technology and the data generated, it can systematically and seamlessly generate South African Revenue Services- (SARS-) compliant fuel rebate reports for any selected tax period.  
GVR’s technology provides a full audit trail required for eligibility for SARS rebates, and its reporting platform simplifies logbook and data gathering required, enabling successful rebate claims and return on investment.
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