ORPHELIA Pharma and Gustave Roussy announce the publication in Pharmaceuticals of the scientific work related to the development of the first temozolomide oral suspension for children

Paris and Lyon, June 9th, 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.

ORPHELIA reports positive results of the bioequivalence study of KIMOZO® (oral suspension of temozolomide)

Paris and Lyon, May 31st, 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.

ORPHELIA Pharma has been awarded a € 1 million grant from Bpifrance to develop a therapy for a rare form of epilepsy

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.

International Childhood Cancer Day

Today, February 15th 2022, is the International Childhood Cancer Day. ICCD is a global collaborative campaign to raise awareness about pediatric cancer and to express support for children and adolescents with cancer. At ORPHELIA Pharma, we are committed to improving cancer treatments for children. We are proud to announce that, during the annual symposium of Imagine for Margo – Children without Cancer (e-colloque FAST) held few days ago, we signed the charter aimed at accelerating the development of new drugs, facilitating the sharing of data for research and improving the long-term follow-up of patients. More information: https://lnkd.in/d9yApGWs ORPHELIA Pharma: https://lnkd.in/efSUdMP

World Cancer Day

On the World Cancer Day, we stand in solidarity with patients and families affected by this disease. At ORPHELIA Pharma, we are committed to improving cancer treatments for children.  

ORPHELIA Pharma raises new money

ORPHELIA Pharma raises new money to accelerate its commercial and clinical operations

Paris, Lyon, December 14, 2021 – ORPHELIA Pharma, the French biopharmaceutical company dedicated to the development and commercialization of pediatric drugs in the fields of oncology and neurology, announces that it had made a capital increase to which all its shareholders subscribe: OCTALFA initiative and Gilles Alberici, Pierre Fabre Médicament/Pierre Fabre Invest, Ravenala holding (Alain Tornier), PAF Kapital (Jean-François Auffret) and Cemag (André Ulmann). The funds will be used to strengthen the company team and continue the clinical development of Kimozo®.

“I would like to thank all our shareholders for their renewed confidence”, said Mr. Hugues Bienaymé, CEO of ORPHELIA Pharma. “With their support, we will develop the company’s commercial infrastructure, set up new international partnerships and pursue our R&D efforts in the field of rare childhood diseases”.

About Kimozo®

Kimozo® is the first pediatric formulation of temozolomide developed with a patented formulation and adapted for the treatment of refractory or relapsed neuroblastoma in children. The product is the result of a partnership with Gustave-Roussy Hospital (Villejuif, France) and is currently in late clinical development.

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 products approved in the European Union and a project 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.

ORPHELIA Pharma and K.S. KIM sign an agreement to supply Kigabeq®

Paris and Tel Aviv – November 16th 2021 – ORPHELIA Pharma and K.S. KIM INTERNATIONAL (SK-PHARMA) LTD announce today the execution of an exclusive distribution and marketing agreement for Kigabeq® (vigabatrin) in the territory of the Russian Federation.

Kigabeq®, the first pediatric form of vigabatrin developed by ORPHELIA Pharma, is notably indicated in the treatment of infantile spasms (West syndrome). Kigabeq® is approved in the European Union, where this medicine, intended exclusively for children, benefits from a centralized marketing authorization (Pediatric Use Marketing authorization, PUMA).

We are delighted to sign this agreement with K.S. KIM, which is based on the value of Kigabeq® in paediatrics“, says Hugues BIENAYME, Founder and CEO of ORPHELIA Pharma, “With their strong footstep in the distribution of orphan medicines in the Russian territory, K.S. KIM is an excellent partner for Kigabeq®.

With Kigabeq®, the only pediatric presentation of vigabatrin, K.S. KIM is expanding its portfolio of drugs intended for rare and serious pathologies in children” adds Dr. Shlomo Sadoun, CEO of K.S. KIM, “Kigabeq® is a drug which address unmet medical needs for young patients affected by severe and resistant epilepsies. Our ultimate objective is to register Kigabeq® as an orphan drug in Russia, nevertheless we will start distributing it immediately as an unlicenced medicine augmenting unmet needs ”.

Our objective is to make Kigabeq® available as widely as possible, so that all children affected by infantile spasms can benefit.” concludes Gilles ALBERICI, President of ORPHELIA Pharma, “Thanks to this agreement with K.S. KIM, we are very confident that Russian children suffering from infantile spasms will have access to Kigabeq® as quickly as possible.

About K.S. KIM (SK-Pharma)

K.S. Kim International Ltd. operates as part of the SK-Pharma group of companies. The main areas of focus are the registration, commercialization and marketing of pharmaceutical specialty and orphan drugs, by utilizing data driven decision and distributive marketing strategies. The group is operating in 15 countries with a main focus on the Israeli and Russian markets.

For further information, please go to https://sk-pharma.com

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’s mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the pediatric population. With 2 products approved in the European Union and one project in late-stage clinical development, ORPHELIA Pharma recently sets up regional agreements in the EU, the USA and China and conducts research projects through academic and industrial partnerships.

ORPHELIA Pharma and Clinigen sign an agreement to supply Kigabeq®

Paris and Lyon, on July 21st, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs and Clinigen Group plc (AIM: CLIN, ‘Clinigen’), the global pharmaceutical Products and Services company, announce that they have signed an exclusive agreement to manage the supply and distribution of Kigabeq® into unlicensed markets.

Kigabeq® was granted a European Paediatric Use Marketing Authorization (PUMA) in September 2018 and is currently approved for the treatment of infantile spasms (West Syndrome) and pharmaco-resistant focal-onset seizures (partial seizures).

Under the terms of the agreement, Clinigen will provide access to Kigabeq® 500 mg and 100 mg soluble tablets into markets where Kigabeq® is not commercially available. Clinigen will manage all elements of the supply including healthcare practitioner enquiry management, regulatory oversight, logistics and access management. The agreement currently covers all global territories except for USA, China, EU, UK and Bangladesh.

Sam Herbert, Chief Operating Officer and Head of Products Division, Clinigen, said: “We are pleased to be partnering with ORPHELIA Pharma and to be supplying Kigabeq® through our product lifecycle platform.  This agreement will offer healthcare professionals another option to treat difficult-to-manage types of epilepsy. This is a great example of how Clinigen’s lifecycle platform can help patients source medicines which are not otherwise available.”

Hugues Bienaymé, General Manager of ORPHELIA Pharma added: “We are delighted to have entered into this collaboration with Clinigen. With this agreement, our ambition to make Kigabeq® available worldwide, will be fulfilled.”

Healthcare professionals can obtain details about the products by calling the Clinigen customer service team at +44 (0) 1932 824100, emailing MedicineAccess@clinigengroup.com or going online at  www.clinigendirect.com.

Patients seeking medical information should contact their physician.

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. Its mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the paediatric population. ORPHELIA’s first product, Kigabeq®, which was approved during the latter part of 2018 for the treatment of West Syndrome in particular, has been launched in several European countries. Its second product, Ivozall®, obtained a European marketing authorization at the end of 2019 for the treatment of acute lymphoblastic leukaemia. ORPHELIA Pharma also conducts research projects through academic and industrial partnerships.

About Clinigen Group

Clinigen Group plc (AIM: CLIN) is a global, specialist pharmaceutical services and products platform focused on providing ethical access to medicines. Its mission is to deliver the right medicine to the right patient at the right time. The Group operates from sites in North America, Europe, Africa and the Asia Pacific. Clinigen has more than 1,000 employees across five continents in 16 countries, with supply and distribution hubs and operational centres of excellence in key long-term growth regions. The Group works with 34 of the top 50 pharmaceutical companies; interacting with over 5,000 hospitals across more than 115 countries.

For more information on Clinigen, please visit http://www.clinigen.com

European Patent covering Kimozo®

ORPHELIA Pharma and Gustave Roussy announce issuance of European Patent covering Kimozo®, the first oral liquid formulation of temozolomide

Paris and Lyon, on June 16, 2021 – ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs and Gustave Roussy, the leading cancer center in Europe, announce today that the European Patent Office has issued Patent EP3613436, which is directed to Kimozo®, the first pediatric, ready-to-use and taste-masked oral suspension of temozolomide. Kimozo® results from the collaboration between the pharmacists and clinicians from Gustave Roussy and ORPHELIA Pharma.

Temozolomide is an essential oncology drug used to treat relapsed or refractory neuroblastoma, a disease that unfortunately affects very young children. We are working in close collaboration with European clinicians and our goal is to make this pediatric form of temozolomide available as early as 2021 through early access programs”; commented Jeremy Bastid, Chief Development Officer of ORPHELIA Pharma.

“We have developed a hospital compounded liquid preparation of temozolomide in 2015 to address the needs of young patients treated at our center”, explain Maxime Annereau, pharmacist and Samuel Abbou, pediatric oncologist at Gustave Roussy, both at the origin of this project. “This hospital formulation was suitable for children but did not exhibit the physico-chemical and stability properties compatible with an industrialized product. This product was therefore not available to all patients. Three years of research and development between Gustave Roussy and ORPHELIA Pharma were necessary in order to achieve the targeted specifications: a highly concentrated, taste-masked and stable formulation”.

“Our research efforts led to the development of Kimozo®, the first drinkable formulation of temozolomide. The innovation is based on the discovery of a key excipient involved in the stability and the control of the rheological properties of the suspension, an invention that has been patented”, concludes Jeremy Bastid.

The grant of Patent EP3613436 entitled “oral suspension of temozolomide” and co-owned by ORPHELIA Pharma and Gustave Roussy has been published in the European Patent Bulletin. Based on the filing date of the application, the patent protection is expected to last until 2038.

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. Our mission is to provide patients with essential hospital products in the fields of epilepsy and oncology, with formulations that are suited to the pediatric population. Our first product, Kigabeq®, which was approved during the latter part of 2018 for the treatment of West Syndrome in particular, has been launched in several European countries. Our second product, Ivozall®, obtained a European marketing authorization at the end of 2019 for the treatment of acute lymphoblastic leukemia. ORPHELIA Pharma also conducts research projects through academic and industrial partnerships.

For further information, please go to www.orphelia-pharma.eu

Contact: Séverine Martin – +33 (0)1 42 77 08 18 – orphelia@orphelia-pharma.eu

About Gustave Roussy

Classed as the leading European Cancer Centre and the fifth on the world stage, Gustave Roussy is a center with comprehensive expertise and is devoted entirely to patients suffering with cancer. 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. 3,200 health 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

Contact : Claire Parisel – +33(0)1 42 11 50 59 – +33(0)6 17 66 00 26 – claire.parisel@gustaveroussy.fr

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.