Paris and Lyon, March 2nd, 2023 – ORPHELIA Pharma, the French biopharmaceutical company that develops and markets pediatric medicines in the fields of oncology and neurology, announces the recruitment of the last patient of its TEMOkids clinical trial evaluating its ready-to-use temozolomide oral suspension, named KIMOZO® or Ped-TMZ, in children with a solid tumor from the age 1 year.
The TEMOKids trial, during which KIMOZO® was administered to children for the first time, is a major milestone in its clinical development. In addition to generating pharmacokinetic data, the trial also evaluates the safety and acceptability of the suspension in the study population and collects data on treatment response.
TEMOkids complements the study previously carried out in adults, which demonstrated the bioequivalence between KIMOZO® oral suspension and the capsule form of temozolomide, which is not adapted to children and yet regularly used in the absence of a pediatric form. ORPHELIA Pharma will present the results of the TEMOkids study at an upcoming scientific congress and will submit the marketing authorization dossier to the European Medicines Agency in 2023.

“We have completed the enrollment of the TEMOkids clinical study thanks to the involvement and motivation of the investigating physicians, which underlines the medical need. With the support of a pediatric oncology network at the European level, we enrolled 43 children in 11 centers across 5 countries: France, Spain, the United Kingdom, the Netherlands and Germany“, says Caroline Lemarchand, Director of Pharmaceutical Development and coordinator of the TEMOkids study.

“The inclusion of the last patient in TEMOkids is a key milestone of the clinical development of KIMOZO®. This new drinkable formulation of temozolomide addresses major medical needs for the management of cancers affecting young children,” concludes Jeremy Bastid, Director of Development at ORPHELIA Pharma. “Our goal is to make the product available to all patients as soon as possible. The ORPHELIA Pharma team is mobilized to enable the filing of the European registration dossier in 2023 and to fill the needs of young patients through special access programs, in close liaison with and with the approval of the national regulatory authorities in Europe,” he adds.

About the TEMOkids study
The clinical trial “TEMOkids Study: A Phase I Pediatric Study for KIMOZO®, Oral Suspension of temozolomide®” (NCT04610736) is an international, open-label, non-randomized Phase I study in 40 pediatric patients aged one year or older. The primary objective of the study is to evaluate the pharmacokinetics of KIMOZO®/Ped-TMZ in the study population. Secondary objectives are to assess tolerability and response to treatment, as well as acceptability in children. Eleven clinical centers are actively involved in the TEMOkids study: Gustave Roussy, Villejuif, France (coordinating center); Institut Curie, Paris, France; CHU Timone Enfants, Marseille, France; Institute of Hematology and Pediatric Oncology, Lyon, France; Centre Oscar Lambret, Lille, France; Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands; Hospital Universitari Vall d’Hebron, Barcelona, Spain; Hospital La Fe, Valencia, Spain; University Pediatric Hospital Niño Jesús, Madrid, Spain; Southampton General Hospital, Southampton, United Kingdom; Great Ormond Street Hospital (GOSH) for Children NHS Trust, London, United Kingdom; Charité University Medicine Hospital, Berlin, Germany.
For more information, please visit www.temokids.eu.

About KIMOZO®/Ped-TMZ oral suspension, 40 mg/ml
KIMOZO® 40 mg/ml is a ready-to-use, taste-masked oral liquid formulation of temozolomide jointly developed by the ORPHELIA Pharma development team and the pharmacists and clinicians of Gustave Roussy for the treatment of relapsed or refractory neuroblastoma, a childhood cancer with a poor prognosis.
The product obtained an Early Access Authorization from the French authorities in March 2022.

About ORPHELIA Pharma
ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets medicines for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of rare diseases, with formulations adapted to the pediatric population. With two drugs approved in the European Union and one product in late-stage clinical development, ORPHELIA Pharma has recently established regional agreements in the European Union, the USA and China and conducts research projects through academic and industrial partnerships.
More information on www.orphelia-pharma.eu.

Contacts: Magali Rosbif / orphelia@orphelia-pharma.eu / +33 (0)1 42 77 08 18

ORPHELIA Pharma will participate to BIO-EUROPE 2022 to be held from October 24-26, 2022 in Leipzig, Germany. This annual meeting aims to gather and connect biotechnology, pharmaceutical industry, and financial sector on pharmaceutical innovative projects.

We are glad to announce that Gilles Alberici, Executive Chairman and Hugues Bienaymé, General Manager and Chief Scientific Officer at ORPHELIA Pharma, will attend this event. If you would like to meet Gilles and Hugues, please contact them : contact@orphelia-pharma.eu.  

Sponsored by : La Région Auvergne – Rhône – Alpes

ORPHELIA Pharma, the French pharmaceutical company that develops and commercializes orphan and pediatric medicines in the fields of oncology and neurology, announces the publication of the results of its study performed in collaboration with the Pharmacy Department of Gustave Roussy and evaluating the temozolomide dose actually delivered to children when using, on the one hand, KIMOZO oral suspension and, on the other hand, Temodal capsule content mixed with soft food or drinks.

The poster #128  entitled “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures” (Lemarchand et al.), is displayed during the poster session of the SIOP 2022 Annual Congress held in Barcelona (Spain). The poster can also be accessed by using this link.

About SIOP

SIOP (International Society of Pediatric Oncology) is the only multidisciplinary and international learned society entirely dedicated to childhood and adolescent cancer. The society has more than 2,600 members worldwide, including doctors, nurses, other health professionals, scientists and researchers. Members are dedicated to increasing knowledge about all aspects of childhood cancer (https://siop-online.org/). The annual congress is held in Barcelona, Spain, from 28^th September to 1^st October, 2022 (https://siop-congress.org/).

ORPHELIA Pharma is proud to announce that it will attend 54th Annual SIOP Congress in Barcelona, Spain. M. Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma, will have the pleasure to present a poster entitled: “Dispensing oral temozolomide in children: precision and stability of a novel and ready-to-use liquid formulation in comparison with capsule-derived mixtures”.

The SIOP 2022 Annual Meetings will bring together more than 3,000 leading clinicians and scientists from over 100 countries to share recent momentous scientific advances in the field of paediatric oncology. The SIOP 2020 Annual Meeting will take place in Barcelona, Spain, from September 28-October 1, 2022 (https://siop-congress.org/). 

Hugues Bienaymé, General Manager, and Jeremy Bastid, Chief Development Officer at OPRHELIA Pharma, will attend this event. If you would like to meet with Hugues and Jeremy, please send an email to: contact@orphelia-pharma.eu.

ORPHELIA Pharma will participate to the 14th annual conference of The European Paediatric Formulation Initiative (EuPFI) to be held from September 20-22 in Rome, Italy. This annual meeting aims to gather industry, academia, clinical and regulatory professionals, to share expertise and interactive discussions on development of paediatric formulations.

We are glad to announce that Caroline Lemarchand, Chief Pharmaceutical Development Officer at ORPHELIA Pharma, will attend this event and present the following session:  “Formulation and assessment of Kimozo® an oral liquid formulation of temozolomide usable in children as young as 1 year old”. If you would like to meet Caroline, please contact her : caroline.lemarchand@orphelia-pharm.eu.

ORPHELIA Pharma will participate to the first meeting “Organisation Inter Régionale Hémato-Onco pédiatrique Hauts-de-France and Grand-Est” to be held on Monday September 19, 2022 at the Faculty of Medicine of Lille, France. This first annual meeting aims to gather doctors, researchers, clinicians, paramedics, from the EN-HOPE inter-region aims and to facilitate collaborations. Jeremy Bastid, Chief Operating Officer at ORPHELIA Pharma, will attend the event. If you would like to organize a meeting with Jeremy, please contact him : jeremy.bastid@orphelia-pharma.eu.

Lyon, June 22^nd, 2022 – OCTALFA announces that its subsidiary ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of orphan medicines for the treatment of rare and pediatric diseases, has included the first patient in the KIMOZO® French Early Access Program for the treatment of relapsed or refractory neuroblastoma.

KIMOZO® 40 mg/ml (oral suspension of temozolomide) was granted an early access pre-marketing authorization (Autorisation d’Accès Précoce, AAP) in France on March 31^st, 2022 by the Haute Autorité de Santé (HAS) for the treatment of relapsed or refractory neuroblastoma in patients aged 1 to 6 years as well as patients unable to swallow temozolomide capsules.

The goal of Early Access Programs in France is to accelerate access to innovative drugs before or after Market Authorization is granted once all conditions specified in Article L.5121-12 of the French Public Health Code (Code de la Santé Publique) are met:

• No appropriate treatment exists;
• The treatment’s implementation cannot be postponed;
• The efficacy and safety of these drugs are strongly presumed based on therapeutic trials;
• These drugs are considered to be innovative, particularly when compared to a possible clinically-relevant comparator.

Full details of the early access program can be found at the Haute Autorité de Santé and the ANSM websites.

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed to treat rare pediatric cancers.

In France, KIMOZO® has been granted an Early Access Program, to be used as monotherapy or in combination with irinotecan or topotecan in the treatment of pediatric patients aged 1 to 6 years and in patients with incapacity to swallow capsules of temozolomide and who suffer from:

• refractory high-risk neuroblastoma or presenting an insufficient response to induction chemotherapy;
• relapsed high-risk neuroblastoma after at least a partial response to induction chemotherapy followed by myeloablative therapy and stem cell transplantation.

KIMOZO® was developed by ORPHELIA Pharma in collaboration with Gustave Roussy.

About ORPHELIA Pharma

Founded by Hugues Bienaymé, ORPHELIA Pharma is a biopharmaceutical company based out of Paris and Lyon which develops and markets drugs for the treatment of pediatric and rare diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the rare diseases therapeutic area, in the form of formulations adapted to a pediatric population. With two drugs approved in the European Union, and another investigational medicine in a late stage of clinical development, ORPHELIA Pharma has recently established regional agreements in Europe, the United States and China, and is conducting research projects through academic and industrial partnerships.

For more information, please visit www.orphelia-pharma.eu

About OCTALFA

Set up by Gilles Alberici, OCTALFA is an independent family-owned company that has been developing and innovating for 15 years in the fields of life sciences, biotechnology and healthcare. Thanks to our subsidiaries and participations, we assert our presence in branches of medicines, particularly in pediatrics, cancer immunology, diagnostics and nanomaterials. We also provide support to patients living with cancer and people with disabilities, particularly children, in France and Madagascar, thanks to our endowment fund and the Dominique & Tom Alberici – OCTALFA Corporate Foundation, whose actions have been fully integrated into OCTALFA’s governance strategy since 2008.

OCTALFA is a shareholder of ORPHELIA Pharma since 2015.

For more information, please visit initiative-octalfa.eu

Contacts

Charlotte Maddalena / e-mail: charlotte.maddalena@octalfa.eu / phone: +33 (0)4 37 49 87 20

Paris and Lyon, June 9^th, 2022 – ORPHELIA Pharma and Gustave Roussy jointly announce today the publication in the scientific journal Pharmaceuticals of the work related to the development of the first temozolomide oral suspension for the treatment of young children and adults with cancer and presenting difficulties to swallow temozolomide capsules (Annereau M. et al., Pharmaceuticals, 2022).

“Since 2015, we have been manufacturing a liquid temozolomide hospital compounded formulation to address the needs of young patients treated in our hospital and a few partner centers,” explains Maxime Annereau, pharmacist at Gustave Roussy and the originator of this project. “In collaboration with ORPHELIA Pharma, we carried out the necessary research work to optimize the physico-chemical properties and the stability of the suspension.  Then we conducted a palatability study in order to select the aroma capable of covering the bitterness of temozolomide in the most efficient way. The development of this concentrated suspension has made it possible to obtain a form adapted to the needs of patients and caregivers”, he adds.

“The work done in collaboration with our partner Gustave Roussy led to a prototype of KIMOZO®, an industrialized and patented version of this drinkable and highly concentrated suspension. KIMOZO® is currently in an advanced phase of clinical development,” says Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “Temozolomide is an essential drug in the management of many pediatric cancers, including relapsed or refractory neuroblastoma which unfortunately affects very young children. Thanks to the results of this collaboration, we intend to make this pediatric formulation available to patients soon,” he concludes.

About KIMOZO® 40 mg/ml

KIMOZO® 40 mg/ml is a liquid, taste-masked and ready-to-use oral formulation of temozolomide developed in collaboration between the pharmacists and clinicians of Gustave Roussy and the development team of ORPHELIA Pharma. The formulation of KIMOZO® has been covered by a patent application in Europe and in the US.

About Gustave Roussy

Classed as the leading European Cancer Centre and the sixth on the world stage, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering with cancer. The Institute is a founding member of the Paris Saclay Cancer Cluster. It is a source of diagnostic and therapeutic advances. It caters for almost 50,000 patients per year and its approach is one that integrates research, patient care and teaching. It is specialized in the treatment of rare cancers and complex tumors and it treats all cancers in patients of any age. Its care is personalized and combines the most advanced medical methods with an appreciation of the patient’s human requirements. In addition to the quality of treatment offered, the physical, psychological and social aspects of the patient’s life are respected. 4,100 professionals work on its two campuses: Villejuif and Chevilly-Larue. Gustave Roussy brings together the skills which are essential for the highest quality research in oncology: a quarter of patients treated are included in clinical trials.

For further information: www.gustaveroussy.fr/en, Twitter, Facebook, LinkedIn, Instagram

Contact : Claire Parisel / presse@gustaveroussy.fr / +33 (0)1 42 11 50 59

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products with formulations adapted to the pediatric population. With two medicines approved in the European Union and one investigational medicine in an advanced clinical development phase, ORPHELIA Pharma has recently signed regional distribution agreements in the European Union, the USA and China with several partners and is carrying out research projects through academic and industrial partnerships.

Paris and Lyon, May 31^st, 2022 – ORPHELIA Pharma, the French biopharmaceutical company focused on developing and commercializing orphan medicines in oncology and neurology, announces today the successful completion of the phase 1 study of KIMOZO®, novel ready-to-use oral suspension of temozolomide, aimed at determining bioequivalence between KIMOZO® suspension and temozolomide capsules.

The abstract #368704 entitled “bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary central nervous system malignancies“ by Ducray et al., was selected by the Scientific Program Committee of the 2022 ASCO annual meeting. The data released online demonstrate the equivalence between KIMOZO® oral suspension and temozolomide capsules.

“The positive results of the bioequivalence study are an important milestone in the development of KIMOZO®”, comments Caroline Lemarchand, Chief Pharmaceutical Development Officer at ORPHELIA Pharma. “KIMOZO® is the first drinkable form of temozolomide that has been specifically developed to address the needs of young children with cancer”, she adds.

“Temozolomide is part of the standard of care for the treatment of relapsed or refractory neuroblastoma, a devastating cancer that affects young children. KIMOZO® is currently undergoing further clinical evaluation (TEMOkids trial, NCT04610736) and our goal is to make this pediatric formulation of temozolomide available to children in the shortest timeframe possible”; concludes Jeremy Bastid, Chief Development Officer at ORPHELIA Pharma.

About the bioequivalence study (NCT04467346)

The NCT04467346 bioequivalence study was an open label, randomized, crossover, 2-period study in 30 adult patients with primary CNS malignancies. The study objective was to evaluate primarily the bioequivalence between KIMOZO® oral suspension and TEMODAL® capsules, to define KIMOZO® pharmacokinetic parameters and to assess the safety and tolerability including buccal tolerance of KIMOZO®.

About ORPHELIA Pharma

ORPHELIA Pharma is a biopharmaceutical company based in Paris and Lyon that develops and markets drugs for the treatment of pediatric and orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential products in the fields of neurology and oncology, with formulations adapted to the pediatric population. With two medicines approved in the European Union and one investigational medicine in an advanced clinical development phase, ORPHELIA Pharma has recently signed regional agreements in the European Union, the USA and China with several partners and is carrying out research projects through academic and industrial partnerships.

Paris and Lyon, May 11, 2022 – ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of pediatric drugs in the fields of oncology and neurology, announces today that it has obtained a one million Euro grant from Bpifrance to develop a therapy to treat a rare epileptic encephalopathy in children.

“We collaborate actively with academic teams on this program, the clinical development of which could begin within 2 years,” comments Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma. “This is an ambitious project for which we have a strong scientific rationale and that could improve the care of these patients“, he adds.

“We would like to thank the Bpifrance teams for their renewed support, which has enabled us to initiate the development of a drug which could become a new therapeutic option in this orphan indication,” 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 orphan diseases. ORPHELIA Pharma’s mission is to provide patients with essential hospital products in the fields of neurology and oncology, with formulations adapted to the paediatric population. With two medicines approved in the European Union and one product in an advanced phase of clinical development, ORPHELIA Pharma has recently set up regional agreements in the European Union, the USA and China and is carrying out research projects through academic and industrial partnerships.