Business Wire

Pharnext Announces That the DSMB Recommends Continuing the Ongoing Phase 3 Trial of PXT3003 for Charcot-Marie-Tooth Disease Type 1A

Del

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

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

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

OTEZLA® (Apremilast) Phase III Data Showed Significant Improvements in Patients with Active Behçet’s Disease with Oral Ulcers17.2.2018 21:00Pressemelding

Celgene Corporation (NASDAQ:CELG) today announced that data from the phase III RELIEF™ clinical trial of OTEZLA® (apremilast) in patients with active Behçet’s Disease with oral ulcers were presented in a late-breaking oral presentation at the 2018 American Academy of Dermatology (AAD) Annual Meeting. The results showed statistically significant reductions in oral ulcers with apremilast 30 mg twice daily (BID) versus placebo through week 12. OTEZLA (apremilast) is Celgene’s oral selective inhibitor of phosphodiesterase 4 (PDE4). Behçet’s Disease is a rare, chronic, multi-system inflammatory syndrome. Oral ulcers, the most common manifestation of Behçet’s Disease, can be disabling and have a substantial effect on quality of life. This study primarily evaluated the effect of apremilast on recurring oral ulcers in patients with active Behçet’s Disease who were previously treated with at least one topical or systemic medication. “Reducing oral ulcers, which are painful and can negatively im

CT-P13 (biosimilar infliximab) is comparable to reference infliximab and adalimumab in highly anticipated real-world study17.2.2018 08:00Pressemelding

Twelve-month data from the Personalised Anti-TNF therapy in Crohn’s disease Study (PANTS) was presented at the 13th Congress of the European Crohn’s and Colitis Organisation (ECCO). The results indicate that the clinical effectiveness, safety and immunogenicity of Celltrion Healthcare’s CT-P13 (biosimilar infliximab) in patients with Crohn’s disease (CD) is comparable to those treated with reference infliximab as well as those treated with adalimumab.1 The UK-wide, three-year prospective observational study investigated primary non-response (PNR), loss of response (LOR) and adverse drug reactions (ADR) to infliximab (reference infliximab and CT-P13) and adalimumab in 1610 CD patients. The data show comparable efficacy between CT-P13, reference infliximab and adalimumab in relation to PNR, LOR and ADR rates. In addition, at week 54, the remission rate was 39.7%, 39.0% and 32.7% for reference infliximab, CT-P13 and adalimumab treated patients, respectively.1 The PANTS study investigates

Avanti Communications HYLAS 4 Satellite Arrives in French Guiana16.2.2018 18:26Pressemelding

Avanti Communications Group plc (“Avanti”), a leading provider of satellite data communications services in Europe, the Middle East and Africa (“EMEA”), announces its HYLAS 4 satellite has arrived safely in French Guiana ahead of its March 2018 launch. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180216005586/en/ Avanti Communications HYLAS 4 Satellite Arrives in French Guiana (Photo: Business Wire) The spacecraft, built by Orbital ATK was packed and shipped at their Satellite Manufacturing Facility in Dulles, Virginia, United States and flown to French Guiana, where it was unloaded and will be taken to the Arianespace launch facilities in Kourou. HYLAS 4 uses the latest High Throughput Satellite technology, doubling Avanti's Ka-band capacity across EMEA. The latest addition to Avanti's HYLAS fleet will provide: Backhaul services for mobile network operators Wholesale broadband for ISPs Connectivity for governments (civil a

General Cable Corporation Stockholders Approve Acquisition By Prysmian Group16.2.2018 17:05Pressemelding

General Cable Corporation (NYSE: BGC) today announced the voting results from the Company’s special meeting of stockholders held this morning. Stockholders of General Cable approved the Company’s previously announced acquisition by Prysmian Group (BIT: PRY) for $30.00 per share in cash. A total of 38,140,754 shares, representing approximately 75.34% of the total number of shares of common stock outstanding and approximately 99% of the total votes cast, were voted in favor of the merger. Subject to regulatory approvals and other customary closing conditions, the transaction is expected to close by the third quarter of 2018. About General Cable General Cable (NYSE:BGC), with headquarters in Highland Heights, Kentucky, is a global leader in the development, design, manufacture, marketing and distribution of aluminum, copper and fiber optic wire and cable products for the energy, communications, automotive, industrial, construction and specialty segments. General Cable is one of the larges

Systemic Sclerosis World Congress:Inspirational Patient Stories Reveal the Challenging Realities of Living with Devastating Rare Disease16.2.2018 09:03Pressemelding

Boehringer Ingelheim today unveils a new phase of 'More Than Scleroderma: The Inside Story', and launches the new patient website www.morethanscleroderma.com. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180216005188/en/ Anna, 36, living with scleroderma (Photo: Business Wire) The global initiative highlights the importance of understanding the ‘inside story’ of each individual living with scleroderma, also known as systemic sclerosis. To coincide with the Systemic Sclerosis World Congress in Bordeaux, 15-17 February 2018, new and truly inspiring patient stories are revealed, reflecting the real-life, diverse and very moving journeys of people living with scleroderma across the world. The campaign will also be launched in the U.S. The new website www.morethanscleroderma.com features a powerful collection of photographs and video stories of people living with scleroderma across the world, revealing the life-changing impact o

Philip Morris International Recognized as a Global Top Employer for the Second Year in a Row16.2.2018 08:00Pressemelding

Philip Morris International Inc. (PMI) (NYSE/Euronext Paris: PM) today is recognized for the second year in a row as a Global Top Employer. This year’s certification from the Top Employer Institute is awarded to PMI teams in 44 countries, a testament to the company’s consistency and excellence in offering an enriching and dynamic work environment, and exceptional development opportunities for employees across the globe. PMI has been recognized by the Top Employer Institute for six consecutive years. This year, PMI was awarded with five regional certifications in Europe, the Middle East, Africa and, for the first time, in North America and Latin America. Additionally, Indonesia was the first PMI affiliate to be certified in Asia. “It is a proud moment for all of us at PMI,” said Charles Bendotti, PMI Senior Vice President, People and Culture. “Our success as a company relies on the men and women who come to work every day with a passion to achieve, and a willingness to learn, grow, and