Canaccord updateCompany Update CG conference takeaways:
Several shots on goal to address rare diseases; pivotal catalysts abound
We hosted Prometic CEO Pierre Laurin at our annual Canaccord Genuity Global Growth Conference this morning. Prometic is a unique rare disease story with several programs that could generate meaningful value as they win approval (Ryplazim) or gain clinical clarity (PBI-4050). In addition, the company is positioned to benefit from up to three priority review vouchers that could bring several hundred million USD in non-dilutive financing to support clinical advancement and product launches.
Clinical clarity on PBI-4050 could come soon after an FDA meeting next week. Prometic will meet with FDA next week to discuss the clinical path forward for PBI-4050. We expect that Prometic could pivot toward an Alstrom program given the recent RPDD receipt and what we think could be a manageable clinical program for the condition. Beyond Alstrom, Prometic will proceed with a larger IPF clinical program but we expect the company to partner on eventual IPF commercialisation.
Ryplazim refile could occur mid/19; significant opportunity in congenital plasminogen deficiency. Prometic plans to refile its BLA for Ryplazim in mid/19 after addressing manufacturing issues raised in a complete response letter. One hundred percent of congenital plasminogen deficient patients in the Ryplazim pivotal study showed a response to therapy, making the product a natural fit for the condition, in our view. In addition, Prometic sees an opportunity for acute use in thrombolysis that could meaningfully expand Ryplazim use.
Prometic now has three RPDDs that could lead to priority review vouchers and nondilutive financing. Yesterday Prometic announced the receipt of its third rare pediatric disease designation (RPDD) for PBI-4050 in Alstrom syndrome. The designations pave the way for potential priority review vouchers on the approvals for 4050, Ryplazim or Ialp and could be sources of non-dilutive financing to fund additional development work or commercialisation.