ORPHELIA Pharma announces the inclusion of the first patient in the TEMOkids pediatric clinical trial

Paris and Lyon, on May 4^th, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, announces the inclusion of a first patient in the TEMOkids pediatric clinical study. The TEMOkids study aims to determine the pharmacokinetic parameters, tolerance, acceptability and response to treatment of Kimozo®, the first oral suspension of temozolomide, in the pediatric population (www.temokids.eu).

Temozolomide is an anti-cancer drug used in treatment regimens for refractory or relapsed neuroblastoma that mainly affect very young children. It is also used to treat rhabdomyosarcoma and certain brain tumors. The only oral forms of temozolomide currently available are capsules which are not suited to children. In fact, to be administered, the capsules must be opened and their content added to food. In young children, the formulation is essential to ensure correct medication intake, safety, accuracy of the delivered dose and adherence to treatment. To meet the requirements of onco-pediatricians and the needs of children, ORPHELIA Pharma, working in close collaboration with Gustave Roussy hospital teams, has developed Kimozo®, the first ready-to-use drinkable suspension of temozolomide.

“The TEMOkids trial, during which our oral suspension of temozolomide will be administered to children for the first time, is a key stage in the clinical development of Kimozo®,” says Caroline Lemarchand, Chief Pharmaceutical Development Officer for ORPHELIA Pharma, “This study will evaluate the pharmacokinetics of temozolomide, alone or in combination with other treatments, in children from 1 year of age. It will also define the tolerance and acceptability of Kimozo® within the study population, and collect data on tumor response to treatment. The first results of this clinical trial are expected mid-2022.“

“We are very pleased to be involved in the clinical development of this long-awaited new pediatric formulation of temozolomide for the treatment of cancer in children,” says Dr Samuel Abbou, pediatric oncologist at the Gustave Roussy hospital, and coordinating investigator of the TEMOkids study. “The study plans to recruit 40 children in 5 countries; that is France, Germany, Spain, the UK and the Netherlands, thanks to the support of a structured European-scale pediatric oncology network.”

“We have received an enthusiastic response from the members of the European pediatric oncology community who have chosen to participate in this clinical trial,” says Hugues Bienaymé, General Manager and Chief Scientific Officer for ORPHELIA Pharma, before adding by way of a conclusion that “TEMOkids complements our ongoing bioequivalence study in adult patients, and should support the filing of the Kimozo® registration dossier at European level in 2023.”

About the TEMOkids study (NCT04610736)

The clinical trial entitled “TEMOkids Study: A Phase I Pediatric Study for KIMOZO, Oral Suspension of temozolomide” is an international, open-label, non-randomized, phase 1 study involving 40 pediatric patients aged 1 year and over. The main objective of this study is to determine the pharmacokinetic parameters of Kimozo® in the population concerned by the study. The secondary objectives are to evaluate its tolerance and acceptability among children, and their response to treatment.

Ten clinical centers are involved in the TEMOkids study: Gustave Roussy in Villejuif (the coordinating center); Institut Curie in Paris, France; La Timone Children’s Hospital in Marseille, France; The Institute of Pediatric Hematology and Oncology in Lyon, France; The Oscar Lambret Center in Lille, France; The Charité University Medical Hospital in Berlin, Germany; The Hopp Children’s Cancer Center in Heidelberg, Germany; The Princess Máxima Center for Pediatric Oncology in Utrecht, the Netherlands; The Vall d’Hebron Research Institute in Barcelona, Spain; and Southampton General Hospital in the United Kingdom.

For further information, please go to www.temokids.eu.

About Kimozo®

Kimozo® (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address the needs of children. Kimozo® is an investigational medicinal product not yet approved for marketing.

ORPHELIA Pharma opens its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France

Paris and Lyon, February 2^nd, 2021. ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs, announces today that it has opened its operating pharmaceutical establishment (“laboratoire pharmaceutique exploitant”) in France. According to the French Public Health Code (“Code de la Santé Publique”), ORPHELIA Pharma will now be able to commercialize medicines under its own name. The authorized activities include importation, sales, advertising, medical information, pharmacovigilance and batch monitoring. The establishment will also be able to launch and monitor early access programs (“Autorisation Temporaire d’Utilisation”, or ATU) on its own behalf or on behalf of third parties.

“As of today, we will be able to commercialize our own medicines in France. We currently have two pediatric medicines which have been approved in Europe through a centralized procedure: Kigabeq® (vigabatrin) and Ivozall® (clofarabine)”, comments Laurent Martin, Chief Pharmaceutical Officer of ORPHELIA Pharma. “Becoming a pharmaceutical establishment will also allow us to initiate an early access program (ATU) for our experimental medicine Kimozo®, a liquid oral formulation of temozolomide developed for the treatment of relapsed or refractory neuroblastoma, following validation by health authorities”, he adds.

“After having obtained the authorization to open our pharmaceutical establishment from the French medicines agency (ANSM) on November 18^th, 2020, this effective opening is a major milestone for our company and is a testament to the efforts undertaken by the people at ORPHELIA Pharma”, concludes Hugues Bienaymé, General Manager.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorization in September 2018 and has been made available in several countries. Our second product, Ivozall®, obtained European marketing authorization in November 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.

ORPHELIA Pharma awarded a grant from the region Île-de-France to support business activity development in Europe and in the US

Paris and Lyon, January 26^th, 2021. ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric and orphan drugs announces today that it has received the support from the region Île-de-France as part of the PM’up program to accelerate the development of the company in Europe and the US. ORPHELIA Pharma will receive a grant of 250 000 € aimed at supporting the international development of the company.

“We are grateful to receive the support from region Île-de-France which will allow us to accelerate the development of our medicines portfolio worldwide. Our products address unmet medical needs of life-threatening paediatric diseases and our ambition is to make them available to all patients“, comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma.

“We will use the proceeds to implement the marketing operations of Kigabeq® in Europe and continue the efforts in finding partners for key territories elsewhere“, states Hugues Bienaymé, General Manager. “This program will also support the preparation of the US dossier and the evaluation of the market access strategy of Kimozo®, the first paediatric drinkable formulation of temozolomide currently under development for the treatment of relapsed or refractory neuroblastoma”, he adds.

About PM’Up program

The Région Ile-de-France supports the development of Small and Medium-sized Enterprises by providing financial assistance to support their growth strategy. PM’up is a grant of up to 250 000 € to finance a 3-year development plan. For more information, please visit https://www.iledefrance.fr/pmup.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorisation in September 2018 and is being launched in several countries. Our second product, Ivozall®, obtained European marketing authorisation in November 2019 for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.

ORPHELIA Pharma and Ethypharm sign a licensing agreement for the development and marketing of Kigabeq® in China

Saint-Cloud and Paris – January 19, 2021 – Ethypharm and ORPHELIA Pharma announce today the signature of an exclusive agreement for the development, registration and marketing in the People’s Republic of China of Kigabeq® (vigabatrin) indicated for the first-line treatment of infantile spasms (West syndrome).

 Kigabeq®, the first paediatric form of vigabatrin developed by ORPHELIA Pharma, is approved and marketed in Europe, where this medicine, intended exclusively for children, has been granted a Paediatric Use Marketing Authorisation (PUMA). Kigabeq® is indicated for the first-line treatment of infantile spasms (West’s syndrome), a severe epileptic encephalopathy in infants.

There is a significant unmet medical need in China for patients suffering from this rare and devastating form of epilepsy. By entering into this agreement, Ethypharm and ORPHELIA Pharma are committed to providing appropriate treatment for Chinese children suffering from this rare disease.

As part of this agreement, ORPHELIA Pharma grants Ethypharm exclusive rights to Kigabeq® in the People’s Republic of China. After registration, Ethypharm will be responsible for the distribution and sale of Kigabeq® in China.

 “Our objective is to make Kigabeq® available in many territories. After Europe and China, new agreements will be signed to enable the marketing of this formulation of vigabatrin, which is particularly suitable for children,” comments Hugues BIENAYME, founder and Managing Director of ORPHELIA Pharma.

 “We are very pleased to establish this partnership with ORPHELIA Pharma. This collaboration brings an effective solution to young Chinese patients. The upcoming commercialisation of Kigabeq® is fully in line with our strategy to strengthen our rare disease franchise in China” said Bertrand DELUARD, President and CEO of Ethypharm.

 Through this partnership, Ethypharm is strengthening its ambition to become a key player in the field of rare diseases in China. Operating successfully for more than 20 years in an ever-changing Chinese environment, Ethypharm has a fully integrated subsidiary located in Shanghai. “Our subsidiary is a partner of choice for companies wishing to make their existing internationally-marketed orphan drugs or drugs for rare diseases available to Chinese patients,” says François LIOT, General manager of Shanghai Ethypharm.

“I am delighted to sign this agreement with Ethypharm, which is based on the value of Kigabeq® in paediatrics. Thanks to the in-depth knowledge that the Ethypharm Group is developing in China, we are very confident that chinese children suffering from infantile spasms will have access to Kigabeq® as quickly as possible,” adds Gilles ALBERICI, Chairman of ORPHELIA Pharma.

About Kigabeq®

Kigabeq® is the first paediatric formulation of vigabatrin, an essential anti-epileptic drug. Kigabeq® is available in 100 mg and 500 mg soluble and scored tablets for oral or (naso)gastric administration and is indicated for the first-line treatment of childhood spasms (West’s syndrome), a severe epileptic encephalopathy in infants. Kigabeq® has been developed exclusively for children and has been granted Paediatric Use Marketing Authorisation (PUMA).

About infantile spasms

West’s syndrome, or infantile spasms, is a severe epileptic encephalopathy of the infant that combines epileptic spasms, a psychomotor deterioration and an hypsarrhythmic electroencephalogram tracing. It is a rare disease, with an estimated incidence of 5 per 10,000 births. It can occur in infants with previously normal development or with a pre-existing delay; in all cases, infantile spasms have a strong impact on the psychomotor development. Pharmacological treatment must be started quickly to allow the spasms to stop and improve the prognosis.

 About the Ethypharm Group

Ethypharm is a European pharmaceutical company focused on two therapeutic areas: the Central Nervous System and Critical Care. Ethypharm markets its drugs directly in Europe and China, and with partners in North America and the Middle East where its drugs are in high demand. The Group employs more than 1,500 people, mainly in Europe and China.

Ethypharm works closely with authorities and healthcare professionals to ensure the appropriate use of and access to its medicines, by as many people as possible.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of paediatric and rare diseases.

Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology with formulations adapted to the paediatric population. Our first product, Kigabeq®, received European marketing authorisation in September 2018 and is being launched in several countries. Our second product, Ivozall®, obtained European marketing authorisation in November 2019 for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma conducts research projects through academic and industrial collaborations.

Press contact

Avril PONNELLE / presse@ethypharm.com / + 33 (0)1 41 12 17 20

Séverine MARTIN / orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

Paris, October 12^th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs announces today that it will organize a symposium at the Société Internationale d’Oncologie Pédiatrique (SIOP) congress 2020. The session will focus on the pediatric use of temozolomide in the treatment of relapsed or refractory pediatric tumors and will showcase the development of Kimozo a ready-to-use and taste-masked oral suspension of temozolomide adapted to children.

“ORPHELIA Pharma is committed to improving the care of patients with pediatric and rare diseases, including developing innovative formulations to address children needs,” comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “Our commitment to develop child-friendly medicines is a historical pillar of our company and this session at the SIOP meeting highlights our long-term close collaboration with the pediatric oncology community”.

This industry symposium entitled “Improving children care with temozolomide oral suspension” will be held virtually on Friday, October 16^th from 9:15 AM to 9:35 AM local time (Ottawa), from 3:15 PM. to 3:35 PM. Central European Time (CET) and accessible by connecting on the SIOP meeting website (SIOP-congress.eu) in the “Product theatre” section.

Dr Lucas Moreno (Val d’Hebron Hospital, Spain) and Dr Dominique Valteau-Couanet (Gustave Roussy Hospital, France), two internationally renowned clinicians, will talk about the critical role of temozolomide in the management of relapsed or refractory pediatric tumors, including high-risk neuroblastomas. The third presenter, Dr Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma, will discuss the development of Kimozo, the first pediatric formulation of temozolomide under clinical development.

We look forward to welcoming you during this unique event.

About Kimozo

Kimozo (also known as Ped-TMZ or ORP-005) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under clinical development to address children needs. Kimozo is an investigational medicinal product not yet approved for use or marketing anywhere in the world.

About SIOP 2020 virtual congress

The SIOP Annual Meetings bring together more than 2,500 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. This congress represents an unique opportunity to stay up to date in this rapidly moving field and to bring the latest science to bedside. In light of the impact of the COVID-19 pandemic on travel restrictions and physical distancing requirements, the SIOP 2020 Annual Meeting will be held virtually on October 14-17, 2020.

ORPHELIA announces the inclusion of the first patient in a clinical study evaluating Kimozo, its pediatric formulation of temozolomide

Paris and Lyon, October 5^th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs is pleased to announce the inclusion of the first-in patient in a clinical study aimed at demonstrating the bioequivalence between Kimozo, the first pediatric formulation of temozolomide under clinical development, and Temodal® capsule.

Although not approved in this indication, temozolomide is an essential anticancer medicine recommended for use in treatment protocols of relapsed or refractory neuroblastoma, a condition that affects young children. However, the only oral formulations that are commercially available are in the form of capsules that are poorly adapted to children. Caretakers are thus compelled to open capsules and disperse the content into a soft food prior to administration to the child, which harbors several risks such as the uncertainty with regards to the delivered dose, the exposure of the caregiver to the cytotoxic drug and the instability of the drug in aqueous milieu. To overcome the risks this situation implies, Kimozo, a ready to use oral formulation has been specifically designed to address the needs of the pediatric population.

Kimozo has been developed in collaboration with Gustave Roussy, one of the leading cancer centers in Europe. The current clinical trial conducted by ORPHELIA Pharma aims at demonstrating, first for regulatory purposes, the bioequivalence between Kimozo and the Temodal® capsules in adult patients having brain cancers, with additional clinical investigation to come in the pediatric population of interest.

“We have reached an important milestone for Kimozo with the clinical trial approval from competent authorities and the recruitment of a first patient“, said Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma “We plan to enroll 30 patients by mid-2021 thanks to the support of the three neuro-oncology teams involved in the study: Professor Ducray of the Hospices Civils de Lyon (coordinating investigator), Professor Chinot of the Timone Hospital in Marseille and Dr Bronnimann of Saint-André Hospital in Bordeaux. ”

“We are pleased to contribute to the development of this new pediatric formulation of temozolomide. A liquid form is unambiguously of interest for treating children.“, underlines Pr. François Ducray. Hugues Bienaymé, General Manager of ORPHELIA Pharma further comments: “This first administration is a major milestone in the development of Kimozo. We are now preparing the opening of our second clinical trial, which will evaluate Kimozo in pediatric patients, by the end of the year ”, he concludes.

About the Bioequivalence Study (NCT04467346)

The clinical trial entitled “Bioequivalence Study between temozolomide oral suspension (Ped-TMZ) and Temodal® capsules” (NCT04467346) is an open label phase I study, randomized, crossover, 2-period study in 30 male/female patients with primary CNS malignancies. Patients will receive, under fasting conditions, 200 mg/m² of Temozolomide Oral Suspension (Ped-TMZ, the code name of Kimozo) or Temodal®, as single oral administration in 2 different study periods depending on the randomization, with no wash out period between administrations. The primary objective is to evaluate the bioequivalence between Kimozo and Temodal® capsules for oral administration. The secondary objectives are to define the pharmacokinetic parameters of Kimozo administration and to assess its buccal safety.

The clinical centers are Hospices Civils de Lyon, Bron, France ; CHU de Bordeaux, Bordeaux, France and Hôpital de la Timone (AP-HM), Marseille, France. The Study sponsor is ORPHELIA Pharma.

About Kimozo

Kimozo (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address children needs. Kimozo is an investigational medicinal product not yet approved for marketing anywhere in the world.

Meet us at BIO-Europe 2020, October 26-29

Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma, will participate in the upcoming BIO-Europe conference to be held virtuallly on october 26-29, 2020. BIO-Europe is Europe’s largest partnering conference serving the global biotech industry.

Jeremy will actively take part in this business event, registered under the name of the holding company, initiative OCTALFA.

ORPHELIA Pharma will support and attend the upcoming SIOP Congress from October 14^th to 17^th (52^nd Congress of the International Society of Paediatric Oncology), which will be virtual this year.

This event aims to bring together the oncology community and share the latest advances in pediatric oncology.

Join the ORPHELIA Pharma virtual symposium to share on innovation in the use of temozolomide in children, which will take place on Friday October 16^th between 9:15 AM and 9:35 AM (Ottawa time) by logging on to the SIOP website (SIOP-Congress.org).

Please contact us if you have any questions (pierre-julien.cotte@orphelia-pharma.eu).

Paris and Lyon, March 11th, 2020 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of paediatric drugs in the fields of oncology and neurology, announces the appointment of Laurent Martin as Chief Pharmaceutical Affairs Officer, effective January 6^th, 2020.

Pharmacist and scientist by background, Laurent has got more than 25 years of experience in drug development, especially in rare and serious pathologies. Laurent contributed to the development, the European approval and the marketing of several orphan drugs at Orphan Europe (now Recordati Rare Diseases). More recently, he was Chief Development Officer and Qualified Person at DBV Technologies, where he led global developments (USA, Europe) of innovative biological drugs mainly for children.

« Laurent’s large experience in the development of drugs for rare and paediatric diseases is a significant asset for ORPHELIA Pharma » comments Hugues Bienaymé, Founder and General Manager. « Laurent will be instrumental in strengthening ORPHELIA Pharma’s pharmaceutical structure. We plan to file an application to operate as a pharmaceutical licensed company (the French “Exploitant” status) in order to market our products in France. »

« I was convinced by ORPHELIA Pharma’s model, which I am very happy to join at this period of strong development of the company » says Laurent Martin. « The essential paediatric products developed by ORPHELIA Pharma perfectly match with my regulatory expertise and my wish to work on the marketing of medicines with high medical benefit.»