[February 27, 2015]

Edimer Pharmaceuticals Celebrates Rare Disease Day 2015 by Initiating Yearlong Series of Regional Conferences for Healthcare Providers and Patients

Edimer Pharmaceuticals, a biotechnology company focused on developing an innovative therapy for the rare genetic disorder, X-linked Hypohidrotic Ectodermal Dysplasia (XLHED), today announced a new initiative in collaboration with the National Foundation of Ectodermal Dysplasias (NFED) -- the Ectodermal Dysplasias Regional Conferences. This series of regional conferences is being planned in collaboration with hospitals and academic centers around the United States and will provide healthcare providers and families an opportunity to come together to discuss ectodermal dysplasias. Conferences are being planned throughout 2015 in the Northeast, Midwest, South and West Coast. Additional information can be found at: www.ectodermaldysplasiasconference.com.

"Individuals with rare diseases, such as XLHED, often receive care from a variety of healthcare providers," Neil Kirby, President and CEO of Edimer Pharmaceuticals . "Inter-disciplinary meetings, such as these, represent an important opportunity to share information that will hopefully lead to better overall quality care for these individuals."

The two-part initial event will be held on Rare Disease Day, Saturday, February 28, 2015. The healthcare provider (HCP) portion of the conference, occurring in New York City, will bring together more than 180 pediatric dentists, geneticist, genetic counselors, students and other sub-specialists. Speakers will discuss genetics, dentistry, dermatology, and treatment approaches to the ectodermal dysplasias.

Concurrently, the Regional Family Conference will be held in White Plains, N.Y. The conference will bring together more than fifty individuals living with ectodermal dysplasias. Families and healthcare providers will be able to share experiences, learn about available resources, hear the latest research, and receive updates about current clinical trials of EDI200 for XLHED.

This year's Rare Disease Day theme, Living with a Rare Disease, pays tribute to the millions of parents, siblings, grandparents, spouses, aunts, uncles, cousins, and friends who are living day-by-day, hand-in-hand with patients affected by rare disease. In keeping with that theme, a three-generation family living with XLHED will share their life story at the ectodermal dysplasias healthcare provider conference in New York City.

"We are pleased to be patnering with Edimer Pharmaceuticals on this new initiative," said Mary Fete, NFED Executive Director. "The Regional Conferences provide a local opportunity for families to gather who otherwise may not be able to attend our Annual National Family Conference in July. During these Regional Conferences parents and children will have an opportunity to hear from experts on ectodermal dysplasia, get to know each other, make connections, and have a network of support closer to home. As February is also Ectodermal Dysplasias Awareness Month, we couldn't think of a better way to wrap up the month."

Rare Disease Day occurs the last day of February each year (www.rarediseaseday.org) with a goal of raising awareness about rare diseases and their impact on families among the general public, policy makers, public authorities, industry representatives, researchers, and health professionals. Since it's inception in 2008, Rare Disease Day has become a global event, with participation from over eighty countries in 2014. You can find information about events in your area and around the world at http://www.rarediseaseday.org/events/world.

About EDI200

EDI200 is an ectodysplasin-A1 (EDA-A1) replacement protein, representing the first of a new class of molecules rationally designed to correct a specific developmental disorder. EDI200 has been shown to bind specifically to the EDA-A1 receptor (EDAR), activating the signaling pathways that lead to normal ectoderm development. EDI200 has demonstrated substantial and durable efficacy in animal models of XLHED with notable reduction in mortality and morbidity. The U.S. Food and Drug Administration (FDA) granted Orphan Drug designation and Fast Track status to EDI200. EDI200 also has Orphan Drug designation in Europe.

EDI200 is currently being tested in a phase 2 clinical trial designed to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of EDI200 in XLHED-affected male newborns in the first two weeks of life. For additional information on this clinical trial, please visit clinicaltrials.gov, identifier NCT01775462.

About XLHED

XLHED (also known as Christ-Siemens-Touraine Syndrome) is a rare disorder of development resulting from genetic mutations in the ectodysplasin gene (EDA). Patients affected by XLHED are at risk for life-threatening hyperthermia based on their inability to regulate body temperature, and for clinically-significant pneumonias resulting from their abnormality in respiratory secretions. Cardinal signs and symptoms in XLHED include diminished/absent sweat, reduced and abnormal airway secretions, few and often misshapen teeth, and absent or early hair loss from face and scalp.

XLHED patients surviving infancy are predisposed to atopy presenting with eczema and asthma, chronic sinusitis, recurrent nose bleeds, and dry eye complications. Almost uniformly they require dental interventions including early prostheses and later implants. Their susceptibility to hyperthermia, may impact normal participation in outdoor activities, sports and school attendance. Both medical and self-esteem issues are life-long in this disorder. As is generally true with X-linked inheritance, males are fully affected while females are variably affected.

About Edimer Pharmaceuticals

Edimer is a privately held biotechnology company based in Cambridge, Massachusetts dedicated to delivering a significant and durable improvement in the health and quality of life for future generations affected by XLHED. Edimer was established in 2009 with investment from Third Rock Ventures and VI Partners. NEA and Sanofi-Genzyme BioVentures joined the initial investors in a Series B round of equity financing that closed in July of 2013.

For further information on Edimer Pharmaceuticals, please visit www.edimerpharma.com. To receive regular updates about Edimer Pharmaceuticals' progress please join the XLHED network at www.xlhednetwork.com. For further information on Edimer Pharmaceuticals, please visit www.edimerpharma.com.

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