Business Wire

Pharnext Amends the Protocol of the International Pivotal Phase 3 Trials 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 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

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

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

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

Kite Announces Data From ZUMA-3 Study of KTE-C19 in Adult Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia12.12.2017 00:30Pressemelding

Kite, a Gilead Company (Nasdaq: GILD), announced updated results from the ongoing Phase 1/2 ZUMA-3 study of KTE-C19, a CD19 chimeric antigen receptor T (CAR T) cell therapy, which is investigational for the treatment of adult patients with relapsed or refractory acute lymphoblastic leukemia (ALL). With a minimum of eight weeks of follow-up, 71 percent of ALL patients (n=17/24) who received a single infusion of KTE-C19 achieved complete tumor remission (complete remission (CR) or CR with incomplete hematological recovery). The ZUMA-3 study results were presented in an oral session at the Annual Meeting of the American Society of Hematology (ASH) in Atlanta. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171211005311/en/ ALL is an aggressive type of blood cancer which can also involve the lymph nodes, spleen, liver, central

NTT Communications Strengthens its DDoS Protection Services with New Detection Solution11.12.2017 23:00Pressemelding

NTT Communications Corporation (NTT Com), the global data and IP services arm of Fortune Global 500 telecom leader NTT (TYO: 9432), announced that it is expanding its Distributed Denial of Service Protection Services (DPS) with the addition of DPS Detect. This new solution can help customers detect potential attacks directed to their networks and initiate mitigations faster. DDoS attacks can strike at any time, potentially crippling network infrastructure and severely degrading the performance and reachability of a website or other IP-accessible assets. The expanded DPS suite of services will enable Global IP Network customers around the world to easily customize the level of service and assistance that best fits their needs. “Network security is one of the top priorities for our company,” said Michael Wheeler, executive vice president of the NTT Communications

Interactive Brokers Launches Bitcoin Futures Trading at the Start of Trading December 10th, 201711.12.2017 16:28Pressemelding

Interactive Brokers Group, Inc. (NASDAQ GS:IBKR), a global electronic brokerage firm, began offering clients the ability to trade bitcoin futures at the start of trading on the Cboe Futures Exchange (CFE) on Sunday night, December 10th, 2017. In addition to offering bitcoin futures from the CFE, under the ticker symbol GXBT, the company plans to offer bitcoin futures from the Chicago Mercantile Exchange (CME) at the scheduled start of trading December 18th using the symbol BRR. IBKR was there at the start of trading and its systems operated normally. “Interactive Brokers was on the buy side of the low print of 14,710,” said Thomas Peterffy, founder, Chairman and CEO of Interactive Brokers. “A Registered Investment Advisor on the Interactive Brokers platform purchased two March contracts in the first minute of trading.” As of 9:15am ET on Monday, the company said that

Boehringer Ingelheim Marks Great Progress in Immuno-oncology Research Partnerships in 201711.12.2017 13:00Pressemelding

2017 marks a year of significant advancement for Boehringer Ingelheim as it secured a range of external partnerships with the goal of transforming the lives of patients and winning the fight against cancer. External partnerships complement Boehringer Ingelheim’s two-pronged research strategy – investigating both cancer cell-directed therapies and immune cell-directed therapies. This multi-faceted approach offers the potential to bring innovative opportunities to the Boehringer Ingelheim oncology portfolio and novel therapies to patients. “Immuno-oncology is radically shifting the research and treatment landscape in oncology. We believe that the greatest success in difficult-to-treat cancers will be achieved with immuno-oncology and its combination with cancer cell directed therapy, which is why we are committing significant resources in both areas,” said Jonathon Sedgwick, Ph.D., Vice

Valence Advises SK Capital on Its Acquisition of ICL’s Fire Safety and Oil Additives Businesses11.12.2017 12:47Pressemelding

The Valence Group acted as lead financial advisor to SK Capital Partners on its announced acquisition of the Fire Safety and Oil Additives businesses of Israel Chemicals Ltd. (“ICL”) for approximately $1 billion. The transaction is expected to close during the first half of 2018. The Businesses, headquartered in St. Louis, Missouri, have global operations and are market leaders in each of their segments. The ICL Fire Safety business is a formulator and manufacturer of fire management chemicals. Its Phos-Chek fire retardants, Class A and B foams, and water enhancing gels are the world’s leading chemical solutions for managing wildland, military, industrial and municipal fires, and are used in partnership with fire management agencies across the globe. The ICL Oil Additives business provides high quality phosphorus pentasulfide used in the preparation of ZDDP-based lubricant additives, i

Smith & Nephew Announce European Launch of Handheld Imaging Technology Heralding a New Era of Evidence-Based Decision Making in Wound Care11.12.2017 10:18Pressemelding

Smith & Nephew (LSE: SN, NYSE: SNN), the global medical technology business, today announces the European launch of MolecuLight i:X TM, the easy to use, handheld imaging device that instantly measures wound surface area and visualises the presence and distribution of potentially harmful bacteria in wounds.2,3 This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171211005434/en/ Currently wound assessments are made with the naked eye which can lack the accuracy required to most effectively guide clinical decision making.4 Using fluorescence, MolecuLight i:X quickly, safely, and easily visualises potentially harmful bacteria1,2,3 in wounds which may otherwise lack signs or symptoms of infection. It enhances a clinician’s ability to choose the right therapy, at the right time for their patient1,

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom