Business Wire

Pharnext Announces That the DSMB Recommends Continuing the Ongoing Phase 3 Trial 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 that the independent Data Safety Monitoring Board (DSMB) has completed its second pre-specified safety evaluation of PXT3003 in the ongoing PLEO-CMT Phase 3 clinical trial. Based on a review of safety data from all randomized patients, the DSMB recommended to continue the PLEO-CMT study as planned.

PLEO-CMT is a pivotal, multi-center, randomized, double blind, placebo-controlled, three-arm Phase 3 study that was initiated in December 2015 and has enrolled 323 patients with mild-to-moderate CMT1A in 30 sites across Europe, the U.S. and Canada. Diagnosis of CMT1A has been confirmed genetically through detection of PMP22 gene duplication. Over 15 months, Pharnext will compare in parallel groups the efficacy and safety of two orally administered doses of PXT3003 to placebo. Efficacy will be assessed through one primary endpoint: change in the ONLS score at 12 and 15 months of treatment to measure improvement of patients’ disability with PXT3003. Additional secondary outcome measures will be assessed including functional and electrophysiological endpoints.

The DSMB is an independent body of experts drawn from the fields of clinical medicine, biostatistics and study methodology, chartered to provide recommendations to Pharnext upon regular pre-specified review of the accumulated data during the conduct of the clinical trial.

“This second positive recommendation from an independent board of experts reinforces PXT3003’s safety profile, even in patients who received up to 15 months of treatment. This Phase 3 clinical trial is highly significant for patients suffering from CMT1A where only supportive care is available today,” said Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext. “We believe that our PLEODRUG™ PXT3003, if successful, has the potential to transform the treatment of CMT1A in adults. We look forward to completing this clinical trial and sharing top-line results in mid-2018.”

About PXT3003

PXT3003, Pharnext’s lead PLEODRUG™ in development for the treatment of Charcot-Marie-Tooth type 1A disease (CMT1A), is a novel, synergistic, low-dose combination of baclofen, naltrexone, and D-sorbitol formulated as an oral solution given twice-daily. PXT3003 has multiple main mechanisms of action: a synergistic inhibition of PMP22 gene overexpression associated with myelination improvement, direct nerve protection and additional positive effects on other cellular types: muscle cells, neuromuscular junctions and immune cells. PXT3003 obtained positive results in a Phase II clinical trial in 80 adult patients with CMT1A. In 2014, the EMA and FDA granted orphan drug designation to PXT3003 for the treatment of CMT1A in adults. An international pivotal Phase III trial (PLEO-CMT) in over 300 adult patients with CMT1A is now underway at 30 sites across Europe, the U.S. and Canada.

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.

The company Pharnext is listed on Euronext Growth Stock Exchange in Paris (ISIN code: FR00111911287).
For more information, visit www.pharnext.com

Contact information

Pharnext
Xavier Paoli, +33 (0)1 41 09 22 30
Chief Commercial Officer
contact@pharnext.com
or
Investor Relations (Europe)
MC Services AG
Anne Hennecke, +49 211 529252 22
anne.hennecke@mc-services.eu
or
Media Relations (Europe)
ALIZE RP
Caroline Carmagnol
Margaux Pronost
+33 (0)1 44 54 36 64
pharnext@alizerp.com
or
Pharnext
René Goedkoop, +33 (0)1 41 09 22 30
Chief Medical Officer
contact@pharnext.com
or
Investor Relations (U.S.)
Stern Investor Relations, Inc.
Matthew Shinseki, +1 212-362-1200
matthew@sternir.com
or
Media Relations (U.S.)
Russo Partners
Tony Russo
Scott Santiamo
tony.russo@russopartnersllc.com
scott.santiamo@russopartnersllc.com
+1 212-845-4251
+1 718-344-5843
or
Financial Communication (France)
New CAP
Emmanuel Huynh, +33 (0)1 44 71 20 40
pharnext@newcap.eu

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 jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme tältä julkaisijalta, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.

Lue lisää julkaisijalta Business Wire

California Sports Surfaces Announces New Senior Leadership22.9.2017 23:21Tiedote

The California Sports Surfaces (CSS) brand, as part of ICP Construction (ICP), today announces the appointments of Steve Lock as global vice president; John Graham as leader, strategic accounts and global relationships; and Malcolm Parkes as managing director, Australia California Sports Surfaces. CSS is the leading supplier of such prestigious sports surfacing solutions as DecoTurf, Plexicushion Prestige, Rebound Ace, Premier Sports Coatings, Premier Court and Plexitrac. These world-renowned product lines supply the US Open, the Australian Open and other distinguished sporting events. Steve Lock has assumed the role of global vice president, responsible for managing the US and global sales and marketing teams for California Sport Surfaces. He was previously the leader for California Sports Surfaces in Australia and the Asia/Pacific region, and will be relocating to ICP global headquar

CORRECTING and REPLACING Smiths Detection Wins Biggest Ever Single Order for Next-Generation Hold Baggage Scanner for Frankfurt Airport22.9.2017 19:02Tiedote

Please replace the release with the following corrected version due to multiple revisions to the headline, subhead and body of the release. The corrected release reads: SMITHS DETECTION WINS BIGGEST EVER SINGLE ORDER FOR NEXT-GENERATION HOLD BAGGAGE SCANNER FOR FRANKFURT AIRPORT Advanced HI-SCAN 10080 XCT hold baggage scanners offer flexible, sophisticated detection capabilities Smiths Detection has won its largest single order ever for its advanced HI-SCAN 10080 XCT hold baggage scanners, with an associated eight-year service agreement. The contract covers 35 of our HI-SCAN 10080 XCT next-generation scanners to support the upgrade to the ECAC Standard 3 explosives detection systems. The solution will deliver increased security and operational efficiency, and can easily be upgraded to mee

California Sports Surfaces’ Rebound Ace Portable Court to be Used at Inaugural Laver Cup22.9.2017 17:37Tiedote

California Sports Surfaces (CSS), a brand under ICP Construction (ICP), is pleased to announce its innovative Rebound Ace Portable sport surfacing system is being used in the inaugural Laver Cup in Prague, Czech Republic, Sept. 22-24, 2017. CSS’s Rebound Ace system was selected to provide comfort, consistency and playability to the Laver Cup participants, who represent the best tennis players in the world. For this event, organizers worked with California Sports Surfaces to create a stunning black surface that would keep spectators focused on the game play. Rebound Ace Portable is an interlocking, temporary court system that provides premium cushion and comfort to maximize players’ performance. It has been chosen for use for several high-profile events, including the Fed Cup, Davis Cup, and the Singapore-based WTA Finals, for which it was specifically created. Laver Cup is modeled afte

Mobile RPG ‘King’s Raid’ Set to Launch in European Markets22.9.2017 14:00Tiedote

Mobile RPG “King’s Raid” which was launched in February in Korea, North America and SEA, is officially launching in European Markets (Google Play and Apple App Store) this month. This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170922005006/en/ Mobile RPG King’s Raid is officially launching in European Markets (Google Play and Apple App Store). It is a Character Collection mobile RPG game full of charming characters and high-end graphics. It includes real-time PvP and large scale Raid content. King’s Raid was launched in February in Korea, North America, and SEA. King’s Raid developed by Vespa Interactive went viral and became a hot Mobile RPG even with limited marketing and lack of brand recognition. As it slowly made the charts in Popularity and Grossing Sales in both markets, it succeeded in reaching the Top 5 in both major App markets in Korea within a month

Smiths Detection is Supplying the Latest Technology for ECAC Standard 3 EDS Upgrade at Frankfurt Airport22.9.2017 14:00Tiedote

Smiths Detection is providing the latest hold baggage scanning technology to support the upgrade to ECAC Standard 3 explosives detection systems at Frankfurt Airport. Awarded by the Procurement Office of the Federal Ministry of the Interior in Germany, the contract includes 35 x HI-SCAN 10080 XCT next generation scanners which will not only detect the explosive threats specified by the new standard, but can also easily be upgraded to meet future requirements to identify evolving threats, such as homemade explosives. Frankfurt is Europe’s second largest airport in terms of cargo and fourth in passenger numbers (over 60 million in 2016) and these figures are expected to continue growing. The new technology will deliver the security and operational efficiencies required by such a busy airport both now and into the future. For deployment ahead of the upcoming ECAC deadline of 20

IBC2017 Reports Record Visitor Numbers in Its 50th Year22.9.2017 12:32Tiedote

In its 50th anniversary, IBC2017 has confirmed a year-on-year increase in visitor numbers, marking a record attendance of 57,669 across the six day event. Visitors from across the expanding media ecosystem had the opportunity to learn about the opportunities offered by technologies - including social media TV, OTT, VR, AR, Cloud, IP, Bots and 5G - and to meet the most intelligent robots. Visitors to the 15 exhibition halls witnessed many exciting new launches; Launch Pad grew year-on-year with a record number of exhibitors; the Future Reality Theatre and Content Everywhere Hub free programmes were packed with diverse and new exciting content, embracing the transformation. Conference delegates packed the Forum to hear from leading experts from Facebook, Google, CNN, HTC Viveport, Dolby, C4 and ITV to discuss and debate the hot topics challenging the industry with a focus on m

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