I expect it will not be long before we start to see revenues...

I expect it will not be long before we start to see revenues coming in from Off-Label Drug Use (OLDU). The lowest hanging fruit here is for the use of Ryoncil for Adults with aGvHD.

Off-label prescribing is a common and legal practice in medicine. This practice is justified when scientific evidence suggests the efficacy and safety of a medication for an indication for which it does not have FDA approval and when the practice is supported by expert consensus or practice guidelines. [Ref: Prescribing “Off-Label”: What Should a Physician Disclose? | Journal of Ethics | American Medical Association].

From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient. This should be quite straightforward for the above indication. Health Care Professionals (HCP) need to be very careful to not expose themselves to litigation, but for this particular indication they would likely be put at ease by the following article published in Transplantation and Cellular Therapy, Official Publication of the American Society for Transplantation and Cellular Therapy only 6 months ago (February 2025): "Ryoncil (Remestemcel-L) for Third-Line Treatment of SR-aGVHD in Adolescents and Adults by Joanne Kurtzberg, MD ∙ Elizabeth Burke, ANP ∙ Jack Hayes, M.A. ∙ Dr. Eric Rose, MD ∙ Silviu Itescu, MD Official Publication of the American Society for Transplantation and Cellular Therapy"

This case series comprises 25 adolescents and adults 12 years and older who received RYONCIL under expanded access for SR-aGVHD after failure of second line treatment, including ruxolitinib.

The 25 patients included 15 males (60%) and 10 females (40%), ranging in age from 12 to 63 years. Twenty one (84%) patients had Grade III-IV SR-aGVHD and 11 (44%) failed ruxolitinib prior to starting RYONCIL. Overall Response (OR), CR + Partial Response (PR), at Day 28 was 68%. Complete Response increased from 20% at Day 28 to 48% at Day 56. Overall survival through Day 100 was 72% for all 25 patients, and 73% for those aged 18 and older.

This study concluded that: "Adolescents and adults with SR-aGVHD who do not respond to ruxolitinib or other second line agents have survival of only 25-30% through 100 days. In this case series, treatment with RYONCIL as third line therapy in adolescents and adults with SR-aGVHD resulted in substantially improved survival outcomes. These results indicate that RYONCIL in these highest risk patients appears to favorably modify the course of disease preventing early mortality from SR-aGVHD."

Keep in mind that, without Ryoncil, 70-75% of these non-responding patients are likely to die within 100 days. Once their HCP's become aware of the results already achieved by Ryoncil in this recent case series, and the recent approval of Ryoncil for this disease in pediatric patients, they should be able to confidently prescribe Ryoncil for adult patients.

Please do not rely on the facts or opinions expressed in the above post when making an investment decision.