Pharnext Amends the Protocol of the International Pivotal Phase 3 Trials of PXT3003 for Charcot-Marie-Tooth Disease Type 1A
Pharnext SA (Paris:ALPHA) (FR00111911287 - ALPHA), a biopharmaceutical company pioneering a new approach to the development of innovative drugs based on the combination and repositioning of known drugs, today announced an amendment in the protocol of the ongoing Phase 3 clinical program (PLEO-CMT and PLEO-CMT-FU studies) of PXT3003 for Charcot-Marie-Tooth disease type 1A (CMT1A) in adults to address a stability issue in the high dose formulation of PXT3003.
PLEO-CMT, a pivotal, multi-center, randomized, double blind, placebo-controlled, Phase 3 study, completed enrollment of 323 patients with mild to moderate CMT1A in 30 sites across Europe, the U.S. and Canada in December 2016. Patients have been randomized to receive during 15 months either the placebo or one of two doses of PXT3003: dose 1 (5 mL) or dose 2 (5 mL) with dose 2 equal to twice the dose 1. According to the protocol, all patients were supposed to continue treatment in a 9-month extension study (PLEO-CMT-FU), whilst placebo patients were randomized to dose 1 or dose 2 of PXT3003.
Overtime, after 12 months, a stability issue emerged in some batches of the high dose formulation (dose 2). This finding has raised no safety concern, but to ensure that the high dose patients get full exposure to the dose 2 level, Pharnext decided to switch these patients to receive double the amount of dose 1 (2 X 5 mL) in the 9-month open label extension study (PLEO-CMT-FU). Patients from the placebo and dose 1 arms in the 15-month double blind PLEO-CMT study will continue the Phase 3 clinical trial as planned: then, these patients will have the opportunity to continue treatment with PXT3003 in the PLEO-CMT-FU extension study for 9 months.
Main PXT3003 development milestones remain unchanged: adaptive design and futility analysis still planned by the end of 2017, results of the PLEO-CMT trial still expected in the second half of 2018, most likely in Q3. The statistical analysis plan will take the amendment into consideration. The data will form the basis of the submission package for market approval in the first quarter of 2019. Long-term safety data from PLEO-CMT-FU would then be submitted to regulatory authorities during their review of the marketing authorization application. Pharnext expects PXT3003 market approval during the second half of 2019, as scheduled.
As previously communicated, the independent Data Safety Monitoring Board (iDSMB) evaluated the safety data of all patients on September 5th, 2017, as it had no safety concern for both PXT3003 doses, it recommended study continuation. Of note, in agreement with regulatory agencies, the dose 2 was included in the PLEO-CMT study based on the dose response from the Phase 2 trial. Only the dose 1 was evaluated in the Phase 2 trial and demonstrated safety, tolerability and improvement beyond stabilization of CMT1A patient disability.
“We have found a satisfactory solution for this unexpected stability event of the PXT3003 highest dose that was not previously investigated in our Phase 2 trial.” said Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext . “All our objectives and development milestones remain unchanged and we look forward to bringing this innovative therapy to CMT1A patients.”
PXT3003, developed using Pharnext’s R&D platform PLEOTHERAPYTM, is a novel oral fixed-low dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol with Orphan Drug Designation in Europe and the United States.
Pharnext is an advanced clinical-stage biopharmaceutical company founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics. Pharnext has two lead products in clinical development. PXT3003 is currently in an international Phase 3 trial for the treatment of Charcot-Marie-Tooth disease type 1A and benefits from orphan drug status in Europe and the United States. PXT864 has generated positive Phase 2 results in Alzheimer’s disease. Pharnext is the pioneer of a new drug discovery paradigm: PLEOTHERAPY™. The Company identifies and develops synergic combinations of repositioned drugs at new optimal lower doses. These PLEODRUG™ offer several key advantages: efficacy, safety and intellectual property including several product or composition of matter patents already granted. The Company is supported by a world-class scientific team.
Pharnext is listed on Euronext Growth Stock Exchange in Paris (ISIN
For more information, visit www.pharnext.com
This press release contains certain forward-looking statements concerning Pharnext and its business. 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 the Company’s document de base registered by the French Financial Markets Authority (Autorité des marchés financiers) on June 2, 2016 under number I.16-0050 (a copy of which is available on www.pharnext.com ). The occurrence of all or part of such risks could cause actual results or achievements of Pharnext to be materially different from such forward-looking statements.
René Goedkoop, MD
Chief Medical Officer
+33 (0)1 41 09 22 30
Investor Relations (Europe)
MC Services AG
+49 211 529252 22
Investor Relations (U.S.)
Stern Investor Relations, Inc.
Financial Communication (France)
+33 (0)1 56 88 11 15
Media Relations (Europe)
+33 (0)1 44 54 36 64
Media Relations (U.S.)
Om Business Wire
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
Sagemcom Unveils the Next-Generation Multi-Gigabit Solutions23.10.2017 16:05 | Pressemelding
AFTER THE SUCCESS OF ITS ONE GIGABIT GATEWAY RANGE, SAGEMCOM RELEASES TO THE OPERATORS THE NEXT GENERATION MULTI-GIGABIT GATEWAYS – VDSL 35B / G.FAST / PON, 2.5G/10G PON, DOCSIS 3.1 – INTEGRATING HIGH EFFICIENCY 802.11AX Wi-Fi, IN ADDITION TO NEW SMART HOME CONNECTIVITY SOLUTIONS INCLUDING DISTRIBUTED WI-FI, VOICE CONTROL AND HOME AUTOMATION. The VDSL 35B / G.fast / FTTH / PON universal gateway, F@st 5380, integrates G.fast 212MHz, GPON and 802.11ax Wi-Fi, enabling the operators to deliver Multi-Gigabit end-to-end services, and to meet the growing bandwidth required by the end-users. F@st 5380 benefits from the last innovations of the in-house field proven Sagemcom SWANTM software suite including big data, Wi-Fi distribution, smart diagnostics… The next generation Fiber gateways range includes two products: F@st 5688ax for 10G PON (XGS-PON
Moody’s Analytics Launches the CreditLens™ Platform23.10.2017 15:43 | Pressemelding
Moody’s Analytics has launched the CreditLens platform, our next-generation credit origination and risk monitoring solution. This new platform offers flexibility, configurability, and efficiency to lending institutions striving to establish smarter and leaner loan origination practices. The CreditLens platform reduces time-to-decision while tapping into powerful models and insightful analytics that help lenders manage risk and drive productivity. Lenders gain efficiency from the solution’s automated spreading capability and a flexible model authoring module. They also gain deep insights from built-in Moody’s Analytics Risk Grading features and Business Insights dashboards, which provide a complete view of the lender’s credit relationships. Automation in the platform also reduces errors and preserves data lineage throughout the credit assessment and origination process.
Knopp Biosciences Announces Renewal of NIH Blueprint Grant Award for Advancing KCNQ2 Modulators in Epilepsy23.10.2017 15:11 | Pressemelding
Knopp Biosciences LLC today announced the renewal and expansion of its grant award from the National Institutes of Health Blueprint Neurotherapeutics Network to advance novel treatments for epilepsy. The Phase 2 award under the NIH Small Business Innovation Research (SBIR) program anticipates NIH support of up to $2 million over the next three years of the project, subject to satisfactory completion of milestones. The renewal follows the successful completion of all milestones under a previously awarded Phase 1 grant of $400,000. Knopp is directing its potassium channel activator program to preclinical and clinical development of small-molecule drug candidates against a validated, anti-seizure pharmaceutical target encoded by the KCNQ2 gene. Knopp intends to advance novel, small-molecule KCNQ2 activators in neonatal epileptic encephalopathy, a rare disorder caused by inherit
HCL Technologies Powers Volvo Ocean Race 2017-1823.10.2017 13:02 | Pressemelding
The 2017-18 edition of the Volvo Ocean Race was flagged off at Alicante, Spain, with HCL Technologies as the strategic IT services provider. The leading global IT services company has provided end-to-end infrastructure support as well as connectivity between the Race Village and the Command Centre in Alicante further connecting them to 12 Host Cities on the race route globally. The world's longest and toughest professional sporting event began at Alicante and will finish eight months later at The Hague. At the flag-off, Jordi Neves, Chief Digital Officer, Volvo Ocean Race, said: “Over four decades, Volvo Ocean Race has drawn some of the greatest ever sailors. This edition of the race will be more digitally focused than before, with HCL as the strategic IT services provider. This edition is also special for us as we embark on a major Sustainability Program in partnership wi
CES Unveiled Paris Returns Tomorrow for Fifth Year with 70+ Exhibitors23.10.2017 13:00 | Pressemelding
Consumer Technology Association (CTA): WHAT: CES Unveiled Paris returns to Paris tomorrow, for its fifth consecutive year, hosting more than 70 exhibitors. More than 600 attendees are expected at the event which will unite French tech startups, top media, buyers and industry influencers around the latest tech innovation and provide a preview of CES® 2018. Schedule as follows:
Wipro Cited as a Leader in Everest Group PEAK Matrix™ for IT Security Services23.10.2017 12:59 | Pressemelding
Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today announced that it has been recognized as a ‘Leader’ by Everest Group, a global independent consulting and research firm, in its report titled “IT Security Services- Market Trends and PEAK MatrixTM Assessment 2017: Security- the Biggest Digital Insecurity”. The report analyzed the capabilities of 17 leading global IT service providers on Everest Group’s PEAK Matrix. Wipro has been named as a Leader for its strong global delivery network with a transformational mind-set, and its ability to deliver high quality of services across regions and service segments, in terms of both execution and responsiveness. The report highlights the trends influencing the IT security services market, focusing on the increasing complexities, size and
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom