- ADVANZ PHARMA secures exclusive rights to register and commercialise AT-007, a Phase 3 development compound for the treatment of several rare neurological diseases, including Galactosemia and SORD Deficiency, in the European Economic Area, the UK, and Switzerland
- ADVANZ PHARMA will leverage its existing specialty, hospital, and rare disease expertise and infrastructure to ensure successful commercialisation and patient access
London, UK – January 4, 2023: ADVANZ PHARMA Corp. Limited (“ADVANZ” or “the Company”), a UK headquartered global pharmaceutical company with a strategic focus on specialty, hospital, and rare disease medicines in Europe, today announced that it has entered into an exclusive agreement with Applied Therapeutics, a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.
Under the terms of the agreement, Applied Therapeutics will be responsible for the clinical development and supply of AT-007, while ADVANZ will manage the registration and commercialisation in the European Economic Area, the UK, and Switzerland.
Steffen Wagner, CEO of ADVANZ, commented: “We are very excited to announce this partnership with Applied Therapeutics, which is fully aligned with ADVANZ’s strategy to be a partner of choice for commercialisation of specialty, hospital, and rare disease medicines in Europe.”
“We believe ADVANZ PHARMA is an ideal partner for commercialisation of AT-007 in Europe,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “ADVANZ PHARMA’s commitment to improving the lives of patients alongside their critical infrastructure and expertise in rare diseases positions them well for commercial success. This partnership brings us one step closer to making AT-007 available to patients with Galactosemia and SORD Deficiency – both debilitating rare diseases with no other therapies currently available.”
Susanna El-Armale, Chief Corporate Development Officer of ADVANZ, stated: “This partnership with Applied Therapeutics delivers on our commitment to bring innovative products to market that serve an unmet medical need and it supports our ambition to build a strong product pipeline to drive ADVANZ’s mid- and long-term growth.”
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with Galactosemia. AT-007 is currently being studied in a Phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with Galactosemia, as well as a long-term open-label study in adults with Galactosemia. In a pilot study in adults with SORD Deficiency, AT-007 significantly reduced blood sorbitol levels. AT-007 is currently being studied in a Phase 3 trial (INSPIRE) investigating biomarker efficacy and clinical outcomes in adults and children 16 years and older with SORD Deficiency. The drug has been generally safe and well tolerated in all clinical studies to date.
About ADVANZ PHARMA
ADVANZ PHARMA is a global pharmaceutical company with a strategic focus on specialty, hospital, and rare disease medicines in Europe. The company has commercial affiliates in Europe, North America and Australia, and a Centre of Excellence in Mumbai, India, as well as an established global network of commercial partners throughout the rest of the world. With an agile and experienced team, including direct sales, marketing, and medical capabilities across Europe's major markets, Canada, and Australia, ADVANZ PHARMA is committed to improving lives of patients by providing and enhancing the specialty, hospital, and rare disease medicines they depend on. The company has a broad expertise in several therapeutic areas including hepatology, gastroenterology, critical care, anti-infectives, endocrinology, oncology, and more broadly rare disease medicines, making ADVANZ PHARMA a partner of choice for the commercialisation of specialty, hospital, and rare disease medicines.