Speakers: Audra Boscoe, VP HEOR; Erin Zagadailov, Dir. HEOR; Lenny Dang, VP Translational Sciences; Varsha Iyer, Sr. Dir. Clinical Pharmacology; Ophelia Yin, Sr. Dir. Pharmacometrics; Chandra Prakash, Sr. Dir. DMPK; Vanessa Beynon, Sr. Clinical Director (Agios)
Organizers: Lenny Dang, VP Translational Sciences (Agios); Hanlan Liu, Senior Vice President, Rare Disease and Early Pipeline Research (Be Biopharma)
Date: 2022-06-02
Time: 13:00-17:00 Eastern Time
Registration fee: Free
Location: Webcast from PBSS Boston.
Major Sponsor:
Vendor show vendors registered to date:
Registration: http://www.PBSS.org
Registration deadline:2022-06-01  (it will close sooner if the seating cap is reached)

About the Topic

Note: This is an online meeting, The Zoom link will be sent to the registrants 1+ days before the event.

Pyruvate kinase deficiency (PKD) is a rare disease caused by mutations in the PKLR gene that results in lifelong, chronic hemolytic anemia.  It is an autosomal recessive disease with a variable clinical presentation, ranging from mild to life threatening, but often associated with severe and debilitating comorbidities.  PK-deficient red blood cells (RBCs) are characterized by dysregulation in cellular metabolism associated with defective glycolysis, including a build-up of the upstream metabolite 2,3-disphosphoglycerate (2,3-DPG) and deficiency in critical energetic metabolites such as ATP.  Since mature RBCs are entirely dependent on glycolysis for ATP production, it is hypothesized that insufficient energy production affects red cell homeostasis and survival, promoting premature removal of PK-deficient RBC from the circulation by the spleen.

Pyrukynd™, a first-in-class oral allosteric activator of mutant and wild-type red cell pyruvate kinase (PK), has been recently approved by the FDA for the treatment of adults with PK Deficiency.  The team at Agios Pharmaceuticals will share with the audience their findings and experiences learned from the journey of Pyrukynd discovery and development from bench to bedside.

Outline:

1. PKD beyond anemia and the challenges of defining a rare, neglected disease (Audra Boscoe, VP HEOR; Erin Zagadailov, Dir. HEOR, Agios, ~ 45 min)
2. Discovery of Pyrukynd as first-in-class pyruvate kinase activator (Lenny Dang, VP Translational Sciences, Agios, ~25 min)
3. Understanding the drug properties & human-dosage development of Pyrukynd (Varsha Iyer, Sr. Dir. Clinical Pharmacology; Ophelia Yin, Sr. Dir. Pharmacometrics; Chandra Prakash, Sr. Dir. DMPK, Agios, ~ 1hr)
4. Clinical Development & FDA Approval of Pyrukynd (Vanessa Beynon, Sr. Clinical Director, Agios, 45 min)