Business Wire

Pharnext Amends the Protocol of the International Pivotal Phase 3 Trials of PXT3003 for Charcot-Marie-Tooth Disease Type 1A

Jaa

Regulatory News:

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.

Quote:

“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.”

About PXT3003
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.

About Pharnext
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 code: FR00111911287).
For more information, visit www.pharnext.com

DISCLAIMER
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.

Contact information

Pharnext
René Goedkoop, MD
Chief Medical Officer
medical@pharnext.com
+33 (0)1 41 09 22 30
or
Investor Relations (Europe)
MC Services AG
Anne Hennecke
anne.hennecke@mc-services.eu
+49 211 529252 22
or
Investor Relations (U.S.)
Stern Investor Relations, Inc.
Matthew Shinseki
matthew@sternir.com
+1 212-362-1200
or
Financial Communication (France)
Actifin
Stéphane Ruiz
sruiz@actifin.fr
+33 (0)1 56 88 11 15
or
Media Relations (Europe)
ALIZE RP
Caroline Carmagnol
Margaux Pronost
pharnext@alizerp.com
+33 (0)1 44 54 36 64
or
Media Relations (U.S.)
RooneyPartners
Marion Janic
mjanic@rooneyco.com
+1 212.223.4017

Tietoja julkaisijasta

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Tilaa tiedotteet sähköpostiisi

Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

Warm welcome for ABB Formula E in Davos19.1.2019 12:00Tiedote

Just a few days before the start of the World Economic Forum 2019, Davos is celebrating a world premiere: The latest ABB FIA Formula E racing car - dubbed Gen2 – was transported on a train from the iconic Swiss Rhaetian Railway to Europe’s highest city, where it will be an exciting eyecatcher during the coming week at the Davos Platz railway station. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190119005003/en/ ABB FIA Formula E racing car in Davos (Photo: Business Wire) From this Saturday until next Sunday, January 27, the Gen2 will be on display in a special glass case. Interested passersby can learn about the ABB FIA Formula E racing series as well as about the activities of ABB in e-mobility via an information system. With over 8,500 fast charging stations installed in 70 countries around the world, ABB is the global market leader for charging infrastructures. To watch the video, click here ABB (ABBN: SIX Swiss Ex) is

PMI’s Mission Winnow Goes Full Throttle with Ducati Corse for 2019 MotoGP™18.1.2019 20:00Tiedote

Philip Morris International Inc. (PMI) (NYSE: PM) is pleased to announce that its Mission Winnow initiative is expanding in 2019 to include the Ducati Corse racing team. Mission Winnow is a PMI-led campaign to raise global awareness of our passion and determination to constantly improve and evolve, as well as highlight the power of science, technology and innovation to build a better future. PMI has partnered with Ducati Corse since 2002 and has extended the relationship for another three years until the end of 2021. As of the start of this year’s MotoGP season, the team will be officially known as Mission Winnow Ducati. Mission Winnow was first launched in October 2018 with Scuderia Ferrari Mission Winnow, which – like Ducati Corse – has a passion for innovation and a relentless drive to improve in the team’s pursuit of victory. Ducati Corse is one of the most inspiring and resilient teams in MotoGP, with a 70-year history in racing. Ducati fans form a passionate community who appreci

IFF’s Frutarom Division Completes Acquisition of 60% of Thailand-based Mighty18.1.2019 15:50Tiedote

Regulatory News: International Flavors & Fragrances Inc. (NYSE:IFF) (Euronext Paris:IFF) (TASE:IFF), announced that its Frutarom Division has completed the acquisition of 60% of the share capital of The Mighty CO. LTD. (“Mighty”), a leading savory solutions provider in Thailand, thus continuing its growth strategy in Southeast Asia. Amos Anatot, President of IFF's Frutarom Division, said, “The completion of this deal with Mighty underscores that the Frutarom division will continue on its growth strategy and pursue attractive companies that create new opportunities or build on current capabilities.” Mr. Anatot continued, “And in this case, we are helping to grow our capabilities in savory solutions – already an area of strength for legacy Frutarom, now IFF." Mighty, founded in 1989, develops, produces and markets reaction flavors, with particular expertise in savory solutions. The company’s portfolio includes flavors, seasoning blends, marinades, and specialty functional raw materials f

LTI Q3 FY19: Constant currency revenue growth up 6.1% QoQ and 20.6% YoY; Net Profit jumps 32.8% YoY18.1.2019 15:02Tiedote

Larsen & Toubro Infotech (BSE code: 540005, NSE: LTI), a global technology consulting and digital solutions company, announced its Q3 FY19 results today. In US Dollars: Revenue at USD 346.9 million; growth of 5.6% QoQ and 18.2% YoY Constant Currency Revenue growth of 6.1% QoQ and 20.6% YoY In Indian Rupees: Revenue at Rs 24,729 million; growth of 6.1% QoQ and 31.3% YoY Net Income at Rs 3,755 million; growth of (6.2%) QoQ and 32.8% YoY “We are pleased to deliver another strong quarter with 5.6% QoQ growth in USD revenues. Our broad-based revenue growth, superior margin delivery and steady cash generation in Q3 is a testimony of our focused execution and client centricity. We are also thrilled to welcome Ruletronics to LTI family. Ruletronics enables businesses to transform and evolve digitally by providing innovative BPM and CRM solutions leveraging Pega Platform.” - Sanjay Jalona, Chief Executive Officer & Managing Director, LTI Recent Deal Wins Nets, the leading payments company in th

Schlumberger Announces Full-Year and Fourth-Quarter 2018 Results18.1.2019 15:00Tiedote

Schlumberger Limited (NYSE: SLB) today reported results for full-year 2018 and the fourth quarter of 2018. (Stated in millions, except per share amounts) Full-Year Results Twelve Months Ended Change Dec. 31, 2018 Dec. 31, 2017 Year-on-year Revenue $32,815 $30,440 8% Pretax operating income $4,187 $3,921 7% Pretax operating margin 12.8% 12.9% -12 bps Net income (loss) - GAAP basis $2,138 $(1,505) n/m Net income, excluding charges & credits* $2,261 $2,085 8% Diluted EPS (loss per share) - GAAP basis $1.53 $(1.08) n/m Diluted EPS, excluding charges and credits* $1.62 $1.50 8% Full-Year Consolidated Revenue by Area North America $11,984 $9,487 26% Latin America 3,745 3,976 -6% Europe/CIS/Africa 7,158 7,072 1% Middle East & Asia 9,543 9,394 2% Other 385 511 n/m $32,815 $30,440 8% North America revenue $11,984 $9,487 26% International revenue $20,446 $20,442 - North America revenue, excluding Cameron $9,668 $7,518 29% International revenue, excluding Cameron $17,675 $17,423 1% *These are non

FLIR Systems Awarded $89 Million Contract from French Armed Forces to Deliver Black Hornet Personal Reconnaissance System18.1.2019 14:00Tiedote

FLIR Systems, Inc. (NASDAQ: FLIR) announced today it has been awarded a contract from the French Defense Procurement Agency (DGA) in support of the French Operational Pocket Drone (DrOP) program. The contract has a ceiling value of $89 million to provide the FLIR Black Hornet® 3 nano-unmanned aerial vehicle (UAV) and Personal Reconnaissance System (PRS) to support French Armed Forces operations. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190118005085/en/ The French Armed Forces awarded FLIR Systems a contract to deliver the Black Hornet Personal Reconnaissance System for military operations. (Photo: Business Wire) The Black Hornet PRS is the world’s smallest combat-proven nano-Unmanned Aerial System (UAS) and is currently deployed in more than 30 countries. The Black Hornet enables the warfighter to maintain situational awareness, threat detection, and surveillance no matter where the mission takes them. Equipped with el

Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.

Tutustu uutishuoneeseemme