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Pharnext Regulatory Filings 2017

Sep 7, 2017

1594_iss_2017-09-07_0a6b4be8-bd3a-4d1d-af85-db65c74de788.pdf

Regulatory Filings

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Pharnext's PXT3003 Featured at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2017 Annual Meeting

Paris, France, 5:45pm, September 7, 2017 (CEST) – Pharnext SA (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 data related to PXT3003, the Company's lead PLEODRUG™, in development for the treatment of Charcot-Matrie-tooth type 1A (CMT1A) disease will be presented in a poster viewing session at the AANEM 2017 Annual Meeting, September 13-16, in Phoenix, Arizona, U.S.

Details are as follow:

Date & Time Title
September
14 -15, 2017
All day
Poster #90: "A
multicenter, double-blind, placebo-controlled,
pivotal Phase
III
study
(PLEOCMT) of a fixed combination of baclofen, naltrexone and sorbitol (PXT3003), for the
treatment of CMT1A" S. Attarian et al.

The abstract is available online at :http://www.aanem.org/Meetings/Annual-Meeting/Abstracts

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

CONTACTS

Pharnext Xavier Paoli Chief Commercial Officer [email protected] +33 (0)1 41 09 22 30

Investor Relations (Europe) MC Services AG Anne Hennecke [email protected] +49 211 529252 22

Media Relations (Europe) ALIZE RP Caroline Carmagnol Margaux Pronost [email protected] +33 (0)1 44 54 36 64

Pharnext René Goedkoop Chief Medical Officer [email protected] +33 (0)1 41 09 22 30

Investor Relations (U.S.) Stern Investor Relations, Inc. Matthew Shinseki [email protected] +1 212-361-1200

Media Relations (U.S.) Russo Partners Tony Russo Scott Santiamo [email protected] [email protected] +1 212-845-4251 +1 718-344-5843

Financial Communication (France) New CAP Emmanuel Huynh [email protected] +33 (0)1 44 71 20 40