Pediatric medicines have long been neglected by the pharmaceutical industry, who mainly focused their efforts on adult medicines. There are reasons for this situation: the maturing organism of children poses specific and significant challenges with regards to clinical trials and pharmaceutical development and the market is often too small to attract major pharma consideration.
Consequently, there has been significant “off label” use of adult medicines in the pediatric population. Dosage forms designed for adults have often been modified for administration to children (e.g. segmenting tablets, cutting transdermal patches), thus creating a risk for misuse or overdosage.
At ORPHELIA Pharma, we believe that developing new pediatric drugs is not doomed to fail and could be successfully undertaken with the proper methodological approach and the ability to tap into the expertise of key partners. Commitment to children’s care is all we need to succeed.
We focus our efforts in bringing new medicinal products to the market in niche indications where the medical need is the most salient: in neuro-pediatry (epilepsy, neuro-degenerative diseases), in oncology (CNS tumors and hematology) and in metabolic diseases.
The current company set-up is focused on product development and commercialization.
We are actively seeking partners that share our vision and commitment to children care.
TargeOn SAS was incorporated in 2007 by Hugues Bienaymé. In the early years of the company, several potent and selective kinase inhibitors were discovered and optimized to target rare forms of childhood leukemia.
In 2009, the company decided to expand its operations to orphan indications in the Central Nervous System (CNS) arena. The company commenced several collaborations with academic teams to find new cures to rare forms of epilepsy and to Rett syndrome, a neurodegenerative condition for which no treatments are available.
In 2010, pursuant to the new European regulation on pediatric medicines, the company embarked on the development of a pediatric form of vigabatrin, a potent antiepileptic drug with a unique mechanism of action.
Since then, we have reinforced our project pipeline in CNS, oncology and metabolic diseases and continuously sought for new opportunities in these domains.
In 2015, TargeOn was renamed ORPHELIA Pharma to better reflect its commitment to children’s care. The company became a Société Anonyme, and a Board of Directors was set up.
ORPHELIA Pharma’s team is composed of seasoned experts in clinical research, drug discovery, development and regulatory activities.
We have developed a unique corporate model with experts in different fields that contribute to ORPHELIA Pharma and its product development as needed over time. Over the years, we have also developed close collaborations with several world-leading academic clinical and research teams. This allows ORPHELIA Pharma to benefit from very experienced specialists, and the best research experts in our field, while keeping the organisation lean and agile.
Hugues graduated from the Ecole Normale Supérieure de Saint-Cloud in 1986. After an Agrégation of physical sciences and chemistry, he gained a PhD in organic chemistry at the Paris XI University.
He joined the pharmaceutical industry in 1991 with Rhône-Poulenc (then Aventis, now Sanofi) where he became familiar with drug discovery. He created Chrysalon, a biotech company specialized in high-throughput drug discovery, in 2001. Hugues then joined Urogene, a biotech company specialized in urological diseases in 2002, as General Manager and CSO. There, he headed the preclinical development of several kinase inhibitors and GPCR modulators targeting prostate and bladder cancers. In 2005, he joined Pierre Fabre Medicament as GM/CSO of Pierre Fabre Urology where he pursued the discovery and development of several kinase inhibitors to target urological and pediatric cancers. In 2007, Hugues founded TargeOn SAS (now ORPHELIA Pharma SA).
Gilles has 30 years of experience in the European pharmaceutical industry. After its PhD at Institut Gustave-Roussy, Villejuif, France, Gilles joined the Mérieux Group in 1986, then founded and managed IMTIX, then IMTIX-SangStat from 1994 to 2000, and eventually founded OPi in 2000, a company dedicated to rare and severe diseases. OPi was acquired by EUSA Pharma in 2007, now part of Jazz Pharmaceuticals.
Gilles is the President and Founder of Octalfa SAS, a family-owned, independent investment company specializing in Life Sciences.
Gilles is the founder or co-founder of several biotech or pharma companies in France and has developed multiple products and devices, at least 5 of them are on the market, approved almost worldwide. Gilles was elected Executive Chairman of ORPHELIA Pharma in 2015.
Séverine Martin, MSc (Project Manager)
Séverine graduated from Montpellier University (MSc in organic chemistry) in 2004 and since then has gained a comprehensive professional experience in drug discovery for several companies, such as Servier, Proskelia (now Galapagos) and Cerep.
Séverine has been with ORPHELIA Pharma/TargeOn since its inception in 2007 and has been associated in all discovery and development programs of the company.
Jeremy Bastid, PharmD, PhD, advisor
Jeremy holds a Doctor of Pharmacy degree (PharmD) from University Claude Bernard Lyon 1 and obtained his PhD in oncology at the Centre Léon Bérard (Lyon). His expertise combines drug discovery and development as well as business development and management of biotechs. He held several positions at the Hospices Civils de Lyon and the University Claude Bernard Lyon 1. As shareholder and Chief Operating Officer, Jeremy has been involved in the inception and manages OREGA Biotech, a company focusing on the discovery and development of antibodies targeting novel immune checkpoints. Since 2010, he is also a member and now R&D Director of initiative OCTALFA, an organization dedicated to the inception and management of drug development companies in the fields of pediatric oncology, neurology and metabolic disorders.
Virginie Beaurent, PharmD (Regulatory Affairs Director)
Virginie graduated in Pharmacy from Faculté de Châtenay-Malabry, Université Paris XI in 2002. Prior to joining ORPHELIA Pharma as head of Regulatory Affairs in 2016, Virginie was appointed at Stallergenes as CMC and regulatory pharmacist, both for France and International activities. Prior, she was senior regulatory manager at IDD (International Drug Development), a development and regulatory consulting company. Virginie has also experience with the production of pharmaceuticals at Chiesi and Servier. Virginie's experience in regulatory affairs was implemented in the successful filing of ORP-001 marketing autorisation dossier.
Mathieu Schmitt, PharmD, MSc (CMC Director)
Mathieu graduated in Pharmacy from the University of Strasbourg in 2009. After an appointment with Catalent - Beinheim, where he was responsible for the development of lipidic and semi-solid formulations, he joined Santen SAS (formerly Novagali), as head of the formulation laboratory. He was subsequently appointed as senior CMC development manager at Aicuris GmBH (Wuppertal), where he was in charge of all CMC activities, from preclinical to clinical. Mathieu joined ORPHELIA Pharma in 2017.
Pierre Attali, MD, MSc (Chief Medical Officer, acting)
Pierre Attali is a hepatologist with a long-lasting commitment to clinical development. He was involved in the development of multiple products during his career at Synthelabo (now Sanofi) as well as at several biotech companies (Urogene, Bioalliance Pharma/Onxeo, Sensorion, Selexel). Pierre is advising ORPHELIA Pharma on its overall clinical strategy and is involved in the preparation of the clinical protocols.
Pierre has contributed to the approval of more than 10 new drugs and has authored or co-authored more than 100 articles in peer-reviewed journals and patents.
Emmanuèle Gerdil, PharmD (Head of Regulatory Affairs, acting)
Emmanuèle graduated in Pharmacy from the University of Lyon, and after 15 years as Clinical Biologist she joined Sanofi Pasteur in 1990 as vaccines Quality Control Manager. In 1994 she was appointed Qualified Person and Regulatory Affairs Manager for Sanofi Pasteur MSD. She was then promoted Executive Director, Regulatory Affairs Europe at Sanofi Pasteur MSD. Emmanuèle was an active member of the Regulatory Affairs Working Group of the European Vaccine Manufacturers (EVM), a specialized group of the European Federation of Pharmaceutical Industry Associations (EFPIA).
Shareholders of ORPHELIA Pharma
Pierre Fabre is the 3rd largest French pharmaceutical group and the 2nd largest dermo-cosmetics laboratory in the world. In 2014, its sales reached €2.1 Billion, with revenues in dermo-cosmetics accounting for 55% and international sales for 55%. Founded and its headquarters still based in the South-west of France, Pierre Fabre currently has branches in 44 countries and distribution agreements in over 130 countries. Covering a continuum of healthcare products, from prescription drugs and consumer health care products (family care, oral care, natural health) to dermo-cosmetics, Pierre Fabre Laboratories employ over 10,000 people worldwide. In 2014, Pierre Fabre dedicated more than 17% of its drug revenues to R&D. www.pierre-fabre.com
Founded by Gilles Alberici, initiative OCTALFA is a family-owned organization based in France comprising investment companies and private foundations. Its two investment companies OCTALFA and OTONNALE are based on entrepreneurship spirit applied to life sciences and sustained development, respectively. Since 2007, initiative OCTALFA has supported 14 innovative companies in France, with up to 2.5 m€ funding per company. It also seeded OCTALFA 360 enjeux d’avenir (a mutual fund specialized in life sciences, energy and cleantech) and is a shareholder of R2V, a French venture fund investing in the South-East of France. In addition, and through the Dominique & Tom Alberici Foundation, more than 60 charity projects have been financed by initiative OCTALFA since 2008 to improve well-being of disabled people and cancer patients in France and in Madagascar. www.initiative-octalfa.eu
Board of Directors
Gilles Alberici, Chairman of the Board
Hugues Bienaymé, Member of the Board
André Tartar, Member of the Board
Frédéric Desdouits, Observer (on behalf of Laboratoires Pierre-Fabre)
The Board of Directors will be strengthened in the coming months in order to support development activities aiming at building international partnerships.
At ORPHELIA Pharma, we highly welcome external innovation. Over the years, we have built strong collaborative relationships with several leading academic institutions.