2015 Will Be A Defining Year for Gene Therapy, Says Analyst

December 1, 2014

By Riley McDermid, BioSpace.com Breaking News Sr. Editor

The year 2015 will be a defining period for biopharma as emerging gene therapy programs and companies have increasingly come to the forefront, said the biotech team at Piper Jaffray Monday.

In a note to investors titled “Biopharm: A Defining Year Ahead for Gene Therapy,” the biotech team led by Joshua Schimmer at Piper Jaffray said the “stage is now set” for a series of key events which “should further highlight this evolving field and showcase” its potential to transform multiple therapeutic categories.

“We continue to see gene therapy as an exceptionally powerful platform that has the potential to transform the biotech industry over the coming years,” wrote Schimmer.

Right now, Piper Jaffray said it is closely watching the pricing used during the launch of lipoprotein lipase deficiency gene therapy Glybera, noting that company's European Union commercial partner, Chiesi Farmaceutici SpA , has priced the product at €1.1M in Germany for treatment of LPL deficiency.

“Germany will assess the product and price point next year as this sets the stage for reimbursement in additional countries,” said the note. “Some investors have expressed skepticism in this price point, so success in procuring reimbursement would be a positive. As well, some payors may consider alternative or creative pricing models in 2015 or beyond.”

Gene therapy for hemophilia is also in the spotlight, with programs entering or approaching clinical trials in 2015 including gene therapy for Hemophilia B and for Hemophilia A.

“We believe clinical data will start to emerge next year,” wrote Schimmer. “Success has been seen with academic gene therapy programs for hemophilia B, and the corporate programs are borrowing from that experience.“

For the rest of 2015, ophthalmology gene therapies are advancing, with Avalanche Biotech reporting new Phase 2 data for its wet AMD gene therapy program in 2015, after having established preliminary proof of concept in a small early study.

“Repeated success should solidify prospects of gene therapy for this sizable indication. Applied Genetic Technologies Corporation will report initial Phase 1/2 data for its XLRS and ACHM gene therapy Orphan disease programs next year,” said Schimmer. “Additionally, Spark Therapeutics expects to report Phase 3 results from its gene therapy program for Leber's Congenital Amaurosis next year.