- The stock price of Curis, Inc. (NASDAQ: CRIS) increased by 354.86% as it went from a previous close of $1.44 to $6.55 per share today. This is why it happened.
The stock price of Curis, Inc. (NASDAQ: CRIS) increased by 354.86% as it went from a previous close of $1.44 to $6.55 per share today. And the main trigger for the stock price increase is an announcement about positive preliminary data from the ongoing phase 1 study of CA-4948 monotherapy in patients with relapsed or refractory acute myeloid leukemia and myelodysplastic syndromes.
Curis said that it saw a reduction of marrow blasts observed in all evaluable patients. And the company is expecting to report additional phase 1 data in H2 2021.
CA-4948 is a novel small molecule IRAK4 kinase inhibitor in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). And IRAK4 plays an essential role in the toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) signaling pathways And these pathways are frequently dysregulated in patients with AML and MDS.
Third-parties have recently discovered that the long form of IRAK4 (IRAK4-L) is oncogenic and preferentially expressed in over half of patients with AML and MDS. And a variety of drivers are believed to cause this, including specific spliceosome mutations.
The reported preliminary data are from Curis’s ongoing open-label, single arm Phase 1 dose escalation 3+3 study of orally administered CA-4948 monotherapy in adult patients with AML or high-risk MDS. And a minimum of 3 patients will be enrolled in each cohort of the two-part study, starting with 200 mg BID — which was demonstrated to be well-tolerated, capable of achieving relevant levels of drug exposure and has demonstrated signs of biologic activity in Curis’s ongoing Phase 1 study of CA-4948 for the treatment of patients with relapsed or refractory non-Hodgkin’s lymphoma.
The main objective of the study is to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) for CA-4948 based on safety and tolerability, dose-limiting toxicities (DLT), and any biologic activity, pharmacokinetic and pharmacodynamic findings from the study trial population.
The additional objectives include characterization of CA-4948’s pharmacokinetic parameters, and biomarker correlations. As of November 23, 2020, 4 AML patients and 3 high-risk MDS patients were enrolled in the first 2 study cohorts and no DLTs had been observed. And the data being reported from this ongoing trial are preliminary and subject to change.
The key findings include: marrow blast reductions observed in all evaluable patients (6 patients); 6 of 7 patients enrolled remain on study; patients enrolled experienced a median of 3 prior lines of treatment (range 1-4); 2 patients experienced a marrow complete response, one with blast count going from 23% pretreatment to 1% on treatment and the other going from 11% pretreatment to 2% on treatment; No DLTs observed in 7 DLT-evaluable patients in the 200 mg BID and 300 mg BID cohorts. And enrollment has begun in the 400 mg BID cohort.
“We are highly encouraged by the breadth of clinical activity with CA-4948 seen with this early data, especially as this study is both monotherapy and in a late line, relapsed/refractory population. Historically, monotherapy studies in AML and MDS have proven underwhelming; monotherapy studies in a relapsed/refractory setting have been especially challenging. We also have been pleased by the pace at which our trial partners have been able to enroll patients. We look forward to continuing to advance CA-4948 and reporting additional Phase 1 data in the second half of 2021.”
— James Dentzer, President and Chief Executive Officer of Curis
“As a clinician intimately familiar with the treatment challenges faced by patients with AML or high-risk MDS, I am very encouraged by the early data coming out of this study. While there have been important advancements in the development of new therapeutics for patients with previously untreated AML or MDS in recent years, relapsed and refractory patients are still in need of better treatment options. These preliminary data suggest, for the first time in a clinical setting, that successfully targeting the long isoform of IRAK4, which we know to be preferentially expressed in over half of AML and MDS patients, could be the first major breakthrough in over a decade for patients with these diseases.”
— Dr. Guillermo Garcia-Manero, Chief of the Section of Myelodysplastic Syndromes within the Department of Leukemia at The University of Texas MD Anderson Cancer Center
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