October 6, 2023 (Investorideas.com Newswire) The biopharma behind it plans to submit a new drug application, seeking approval of the compound, by year-end, noted an H.C. Wainwright & Co. report.
Syndax Pharmaceuticals Inc. (SNDX:NASDAQ) released topline data from the pivotal AUGMENT-101 trial of revumenib in relapsed/refractory KMT2A-rearranged acute leukemia in pediatrics and adults, and they were positive, reported H.C. Wainwright & Co. analyst Edward White in an October 2 research note.
Revumenib is Syndax’s selective menin inhibitor and is designated a breakthrough therapy in this indication and population, for which no U.S. Food and Drug Administration (FDA)-approved treatment exists, White explained. With the current standard of care treatment, the expected response rate among these patients is less than 10%, and the expected survival period is less than three months.
Higher Target, Compelling Return
Based on the newly reported results, H.C. Wainwright increased its estimated probability of success for revumenib to 75% from 55%, White reported, and this boosted the investment bank’s target price on Syndax to US$42 per share from US$38. In comparison, the current share price of this Massachusetts-based biopharma is about US$14.52 per share.
The difference between these two prices implies a significant potential return for investors of 189%.
Syndax remains a Buy.
About the Study Patients
The data cutoff for these AUGMENT-101 results was July 24, 2023, noted White. At the time, 94 patients had been treated, but only 57 were included in the efficacy evaluable population.
Patients in the study had a median of two prior lines of treatment.
Primary Endpoint Reached
White presented the new AUGMENT-101 results. The primary endpoint of the trial was complete response (CR) or complete response with partial hematologic recovery (CRh), and this was achieved.
Specifically, of the 57 evaluable patients with acute myeloid or acute lymphoid leukemia, 13 patients, or 23%, showed a statistically significant CR/CRh rate (p-value=0.0036). The overall response rate (ORR) was 63% or 36 patients. Of these 36 responders, 14, or 39%, proceeded to transplant.
Eight patients advanced to transplant without achieving CR/CRh, some of whom, White purported, likely would have reached CR/CRh. Those patients would have improved the reported overall CR/CRh rate.
In the acute lymphoid leukemia cohort of 49 patients, 12, or 24.5%, experienced a CR/CRh, and the ORR was 65%, or 32 patients.
The median duration of CR/CRh was 6.4 months. At the data cutoff date, 6 of 13 patients remained in response.
“We believe median duration of response could elongate over time,” White wrote.
The independent data monitoring committee recommended Syndax stop the KMT2A-rearranged cohorts for efficacy at the protocol-defined point of interim analysis, and the biopharma did.
Safety, Tolerability Results
White discussed the safety and tolerability findings. Trial results showed that revumenib was well tolerated, and the side effects that occurred were in line with previous data.
Of the patients who had treatment-associated adverse effects (TRAEs), 28% experienced nausea, 27% experienced differentiation syndrome, and 23% experienced QTc prolongation. However, all cases of QT prolongation were grade 3 or less. As for differentiation syndrome, there was one grade 4 case but no grade 5 cases.
TRAEs, excluding QTc prolongation and differentiation syndrome, caused 6% of patients to discontinue the trial.
“We have no concerns regarding differentiation syndrome or QTc prolongation, and we do not expect the FDA to have safety concerns for approval,” White commented.
Upside in Post-Transplant Use
Because relapsed/refractory KMT2A-rearranged acute leukemia patients may use and are using revumenib for maintenance following transplant, this additional use could boost commercial sales of the inhibitor, White pointed out.
According to Syndax, 50% of transplanted patients started revumenib as a post-transplant maintenance treatment, and 21% of transplanted patients were eligible for doing the same.
“We anticipate a revumenib launch in 2024 with sales of US$20 million (US$20M) that year, growing to US$591M in 2028,” indicated White.
Catalysts To Watch For
More data from AUGMENT-101 are expected at an upcoming medical meeting, likely that of the American Society of Hematology, according to White, to be held in December.
Syndax is working on a new drug application (NDA) for revumenib in adult and pediatric KMT2A-rearranged acute leukemia that encompasses all cohorts. Submission of the NDA to the FDA is expected to happen by year-end 2023.
In addition, White wrote, “There are multiple upcoming milestones for revumenib the company expects to achieve.” Among them is a data readout by year-end 2023 of the AUGMENT-102 trial, evaluating revumenib in combination with chemotherapy in patients with relapsed/refractory KMT2A-rearranged and mNPM1 acute leukemias.
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