Mesoblast gets FDA approval for new Ryoncil potency assay
The TIBA test joins two existing checks on new commercial lots as Mesoblast scales U.S. sales of its pediatric cell therapy.
Adrian Kessler
Commercial Ryoncil batches will be released with an added FDA-approved potency test, as Mesoblast Limited ASXMSB NASDAQMESO said in a Sept. 24 ASX announcement. The cell therapy generated US$115.2 million in net revenue in the year ended June 30, according to Mesoblast's annual results.
The T-cell Proliferation Inhibition BioAssay, or TIBA, is designed to detect shifts in potency when manufacturing processes change or production moves to new facilities, Mesoblast said. It joins two existing tests, one for IL-2Rα inhibition and another for cell adhesion and proliferation, in the matrix used to release each new commercial lot and monitor its stability. The announcement did not give a date for the first lot tested under the expanded matrix.
Mesoblast develops donor-derived cell medicines for severe inflammatory diseases and sells Ryoncil in the United States. The FDA's product page lists Ryoncil's approved use as treatment of steroid-refractory acute graft-versus-host disease in children aged 2 months or older.
The ASXMSB quote showed Mesoblast shares up at A$2.24 by 1:48 p.m. AEST on Thursday, Sept. 24. The NASDAQMESO quote showed US$15.29 at the 4 p.m. EDT close on Wednesday, Sept. 23.
The decision authorizes a new test in Ryoncil's commercial controls, rather than a new patient group or disease indication. Mesoblast gave no comparison of TIBA's performance against the existing assays and no estimate of how the added test will affect release times, batch yields or manufacturing costs. The company also did not say whether the assay replaces any testing steps outside the two existing potency checks.
"The TIBA assay will ensure sufficient sensitivity to detect any shifts in potency of the commercial product that could arise during changes in manufacturing processes or production in new facilities," Mesoblast said in its Sept. 24 announcement.
Read more: Lantheus Holdings wins FDA approval for Bravnetsa before Curium vote
Ryoncil Sales Put Batch Controls in Focus
Mesoblast reported Ryoncil net revenue of US$36 million for the quarter ended June 30, following its first full year of U.S. sales. Total company revenue for that fiscal year reached US$120.3 million, up from US$17.2 million a year earlier, according to its Aug. 27 results. The company reported a US$57.5 million net loss for the year.
Mesoblast had US$103 million in cash at June 30, after drawing US$50 million under a new credit facility to retire maturing debt. Its net operating cash spend was US$43.8 million for the year, according to the Aug. 27 results. The Sept. 24 assay notice did not announce a plant opening, additional capacity or a change to revenue guidance.
The company said in its Aug. 27 results that it had onboarded more than 50 U.S. pediatric transplant sites since launch. It is also enrolling adults with steroid-refractory acute graft-versus-host disease in a trial intended to support a separate label extension. TIBA approval does not authorize adult use, which would require its own regulatory decision.
For a listed cell-therapy comparison, Longeveron Inc. NASDAQLGVN said in its Aug. 12 update that its investigational laromestrocel was in a Phase 2b trial for a rare pediatric heart condition. Mesoblast's new assay instead concerns the release of a product with an existing FDA licence. The SPDR S&P Biotech ETF, a broad sector marker, stood at US$155.35 at 12:20 p.m. EDT on Sept. 23 on its NYSEARCAXBI quote page, before Thursday's Australian session.
Read more: Longeveron falls after stem cell therapy study failure
The next fixed reporting marker is Sept. 30, the end of Mesoblast's first fiscal quarter after the June 30 year-end. Its next quarterly report will show whether Ryoncil revenue advanced from the US$36 million June-quarter level and give the Sept. 30 cash balance; Mesoblast has not announced a report date.
