France's Erytech Closer To Realizing Market Ambitions With Eryaspase US Filing
Positive FDA Feedback For Leukemia Submission
Executive Summary
It has taken almost two decades but it looks as though Erytech could get approval for eryaspase in the US in two indications next year – acute lymphoblastic leukemia and pancreatic cancer.
Founded in 2004, ERYTECH Pharma S.A. is finally closing in on getting a product to market after talks with regulators in the US laid an approval pathway for the French biotech's lead candidate eryaspase.
The Paris-headquartered firm is looking to submit eryaspase, which consists of the enzyme L-asparaginase (L-ASP) that is encapsulated inside donor-derived red blood cells, to the US Food and Drug Administration in the fourth quarter for hypersensitive acute lymphoblastic leukemia (ALL). The decision follows feedback from the agency in a pre-biologics license application (BLA) meeting and based on the discussion and the totality of the available information to the company to date, Erytech believes its regulatory package can potentially support an approval, "pending successful completion of remaining steps."
Erytech's confidence is based on data from a Phase II trial sponsored by the Nordic Society of Pediatric Hematology and Oncology (NOPHO) in ALL patients who had previously experienced hypersensitivity reactions to pegylated asparaginase therapy. The data showed that eryaspase in combination with chemotherapy, administered every two weeks, provided a sustained asparaginase enzyme activity level, and was generally well tolerated with few hypersensitivity reactions.
The ALL news is somewhat of a bonus for Erytech, given that three years ago, the firm pulled its European submission for eryaspase, deciding that solid tumors, particularly pancreatic cancer, represented the best way forward for the compound. Indeed, observers have been much more interested in TRYbeCA-1, a Phase III trial in second-line advanced pancreatic cancer, the top-line data for which are expected early in the fourth quarter of this year. (Also see "Erytech Pulls Graspa ALL Filing To Focus On Pancreatic Cancer" - Scrip, 25 Jun, 2018.)
Erytech CEO Gil Beyen said the company was "encouraged by the interactive dialog with the agency, and grateful for the feedback and guidance we received on a potential path to approval” for eryaspase. He added that "if all goes well, we could be in a position to make this product candidate available to ALL and pancreatic cancer patients in the course of next year.”
The update went down well with analysts at JMP Securities who issued a note saying that with the ALL filing, the TRYbeCA-1 data coming soon and preliminary results from a Phase II/III study of eryaspase in triple-negative breast cancer also slated for the fourth quarter, plus a cash position of €49.6m, "we believe Erytech represents a unique investment opportunity."
They also believe that the FDA is "positively inclined to review the eryaspase BLA," based on a discussion with Erytech's management and their own review of the NOPHO Phase II data. The analysts noted that the US agency had requested not just the clinical data from the NOPHO trial, "but also data from all the completed studies across a variety of indications where eryaspase was evaluated. By our count, the filing would include a safety database of around 500 patients."
The JMP team expects the company to receive a priority review from the FDA, resulting in a potential approval in the second or third quarter of 2022, a view shared by analysts at Jefferies. They are assuming an expedited review "given the unmet need with few options for the mostly pediatric patients that develop hypersensitivity."
They estimate that around 15%-20% of the 6,000 ALL patients diagnosed each year in the US go on to develop hypersensitivity to pegylated asparaginase, "hence this could represent a total available market of perhaps $100m."
Jefferies admitted that "we currently do not ascribe any value to use in ALL," arguing that the focus remains on the Phase III pancreatic cancer trial. The broker is forecasting $500m worldwide peak sales for eryaspase in second-line pancreatic cancer and expects an ex-US marketing partnership.