Paxalisib to Dominate Forum

Dana Farber hosting this conference for PI3K/AKT/mTOR in cancer and even other disease. ( PI3K alpha drug Alpelisib, recently has been approved for an overgrowth disease - bottom)

Fantastic to see this conference with FDA support and Europe Agency involvement. Extremely encouraging really.... given the regard shown at Dana Farber - for our safe, brain penetrant multi-isoform PI3K drug, Paxalisib  (any involvement of the FDA with PI3K is significant and high value)  
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Abstract

Introduction: Paxalisib is a potent, oral, selective, brain-penetrant, small molecule inhibitor of class I phosphoinositide 3-kinase and mammalian target of rapamycin
July 1 2021  Patrick Y. Wen; John de Groot; Alan Olivero;Timothy Cloughesy
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I would suggest this conference would not even be taking place without Paxalisib - a clearly dominant, potent and effective drug in this class.


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11^th Paediatric Strategy Forum for Medicinal Product Development of PI3K/AKT/mTOR Pathway Inhibitors in children and adolescents



3 & 4 April 2023



Dana-Farber Cancer Institute

Boston, Massachusetts


Paediatric Strategy Forums have been created to evaluate the current state of the science, facilitate dialogue, and provide an opportunity for constructive discussions between relevant stakeholders (patient advocates, clinicians, academics, biotechnology/pharmaceutical companies, and regulators) on specific topics in an open forum to assure development of medicines in the best interests of children and adolescents with cancer. The goal of these meetings is to share information, to facilitate the development of innovative medicines and ultimately their introduction into the standard-of-care of children with malignancies.

The Eleventh Multi-Stakeholder Paediatric Strategy Forum organised by ACCELERATE in collaboration with the European Medicines Agency (EMA) and with the participation of the U.S. Food and Drug Administration (FDA) will focus on PI3K/AKT/mTOR Pathway Inhibitors.

The PI3K-mTOR-AKT pathway is upregulated in many paediatric cancers, particularly neuroblastoma, central nervous system tumours and leukaemia. Therefore this pathway is a potential important target in which to develop new drugs for childhood cancer. A large array of inhibitors of this pathway have been developed for adult cancers, however the best use of these inhibitors and how best to combine them with other agents commonly used in children is unknown. Despite the large number inhibitors and the relevance to children’s cancer, inhibition of the pathway has not to date had a major impact for children and adolescents with cancer and there is only one front-line trial incorporating this class of drugs.

The Forum will focus on PI3K, mTOR, AKT, TORC1/2 kinase and GSK3β inhibitors.

The Paediatric Strategy Forum aims to:

• Clearly  ascertain the role of this  pathway in paediatric cancer
• Identify which of the very many  types of inhibitors are of highest value for children
• Suggest optimal combinations.

Specifically the Forum will overview the relevant biology; highlight genetic disorders for which mutations in this pathway are drivers of the diseases (SEGA and overgrowth conditions) in which PI3K/ATK/mTOR inhibitors are active; discuss specific indications in adults where these inhibitors are active and the reasons for this; present the genomic landscape in children and potential genomic predictors for activity; describe lessons learnt from mTOR inhibitors and finally discuss combination strategies.

The immune and metabolic effects of PI3K inhibitors, the potential for toxicity from combinations and optimal scheduling will be considered

The goal is to formulate a consensus between all stakeholders on how best to take forward the development of this class of products.

Format: This forum will be held in person and all participants are strongly encouraged to attend in person. For attendees unable to travel or if travel restrictions prevent an in person event, a virtual option will be available.   

Forum Output: Publication of a summary and manuscript from all participants addressing the challenges and documenting the conclusions of the meeting. Potentially further specific meetings relating to areas which require more in-depth discussion will be arranged.

Participation: possible only by invitation or by submitting an Expression of Interest (see below) before the 6 January 2023.

Submit your Expression of Interest: The call for expressions of interest is open now – please use the box available below. Invitations will be sent out by the end of January 2023.

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FDA approves Novartis Vijoice® (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS)
Apr 06, 2022

• Vijoice is first approved treatment to specifically address the root cause of PROS conditions in select patients 2 years of age and older¹
  
  
• PROS is a spectrum of rare conditions and is characterized by atypical overgrowths and anomalies in blood vessels, the lymphatic system and other tissues^2,3

Basel, April 6, 2022 — Novartis today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval to Vijoice^® (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy.¹ Vijoice is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million.