Rare Diseases: Inserm Transfert Novartis Announces Signing A License Agreement With MyPharma Edition

Posted on Tuesday, May 17th, 2022

Inserm Transfert has just announced the signing of a licensing agreement with Novartis for the work, based on research by Professor Guillaume Canaud at the Institut Necker – Enfants Malade (INEM), of which Inserm is a supervisor. The results of this study aim to restore alpelisib in the indications of Clovis syndrome so that patients with the disease can be treated and relieved, primarily in young children of severe size. The announcement comes at a time when American marketing approvals for the molecule have been granted by the FDA following clinical studies, particularly at Necker Hospital.

A rare disease

Dysharmoniasis hypergrowth syndrome associated with PIK3CA mutations, including Clovis syndrome (congenital lipomatous overgrowth, vascular malformation, epidermal navy) is a group of rare diseases whose symptoms cause mutilation of certain parts of the body. Numerous symptoms such as severe pain, bleeding, thrombosis, fatigue, and sometimes life-threatening for patients. These pathologies are responsible for severe obstruction and have so far only been the subject of symptomatic treatment, interventional radiology or often distorted surgery.

In 2015, thanks to the first patient suffering from a very serious form of this pathology who came to consult with him, Professor Guillaume Cannaud became interested in the disease. Dr. Canwood is familiar with this signaling path and is working on it in other contexts.

Repositioned a molecule for the treatment of breast cancer

At the same time, Novartis is clinically developing a specific PIK3-alpha inhibitor and conducting a clinical trial in breast cancer. Professor Canwood received the necessary approval for the sympathetic use of this molecule with this first patient. The first results are very encouraging with a significant improvement in the patient. Together with his team, he created the first preclinical experimental model of pathology, showed interest in pharmacological inhibitors in this model, and then treated 18 additional patients, including children, thanks to a sympathetic use protocol. The startling results were published in the journal Nature in 2018. Based on these results, many more patients around the world were treated following this protocol and then EPIK P1 clinical trials (real life data) were born.

A clinical study at Necker Hospital

“In fact, the clinical study was conducted on 57 patients by a team from PR Guillaume Canada, of whom 44 were hospitalized at Necker Hospital – Children’s Sick AP-HP, and demonstrated the efficacy of Alpelisib (a drug used in breast cancer) in inconsistent growth. Syndrome associated with activation of mutations in the PIK3CA pathway.

This study found that patients with Cloves syndrome or related disorders who were treated with this molecule had significantly reduced the amount of target lesions and significantly improved symptoms and manifestations related to PROS (PIK3CA-related overgrowth spectrum), looking at their health and quality of life. Significant improvement “

Insaram is a project supported by multiple skills of transfer

Inserm Transfert filed a patent for this new indication and protected the first results by helping teams strengthen scientific rationality throughout the project. Supported Canaud and his parties Novartis Pr. For clinical research. Has begun collaborating with Canaud and AP-HP and has begun negotiating a license agreement regarding the intellectual property generated by Inserm Transfert.

The support of the RHU COZY collaborative project deepened the relationship between Professor Canada and Insom Transfer in 2019, with the goal of improving the medical pathway as well as the future of asymptomatic hypergrowth syndrome patients.

A new study was published by Pr Canned’s team in the journal Science Translational Medicine in October 2021, followed by a recent study on children in the Journal of Experimental Medicine (2022).

A license negotiated by Inserm Transfert with Novartis

Novartis is granted exclusive rights in a patent held by Inserm Transfert. As a result, in February 2022, an exclusive licensing agreement was signed. This was followed, on April 5, by regulatory approvals from the FDA in the United States. In addition, a clinical trial is still ongoing at the European level.

“We are delighted with the doubling of the success of Insarum Transfer, on the one hand, and the support of a medical researcher, Prof. Connaught and his team, on the other. With the promotion of one of the patents issued by Inserm and its partners.

“I am proud of this exceptional achievement that will provide the potential for drug treatment for patients with overgrowth syndrome or vascular abnormalities associated with PI3KCA mutations. This is the result of a team that worked hand-in-hand with several teams at Necker Hospital – Children’s Sick – AP-HP and also in the laboratory (Institut Necker – Children’s Sick – AP-HP – Inserm – University of Paris Cité). The laboratory that owns the molecule (Novartis), the Patient Association and the FDA. The results of the EPIK-P1 study are derived from our previous pre-clinical discoveries, ”points out Professor Guillaume Cannaud (Necker Hospital – AP-HP Sick Children / INEM – Molecular Medicine Center – Insarm / Imagine Institute / Paris City University). “We have been supported at all stages of this development by Inserm Transfert teams who have always stood by us to help our research work the most. We welcome this result, which represents a major step in improving patient care. A

Source: Inserm Transfer

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