Phase 1 clinical trial in patients with renal impairment shows that balixafortide is safe and well tolerated
Safety results support the possibility of using much higher doses of balixafortide than those previously investigated
Further observations show the potential to induce long-term stem cell mobilization, an advantage that may be important in the implementation of hematopoietic stem cell transplantation.
ALLSCHWIL, Switzerland, Sept. 22, 2022 (GLOBE NEWSWIRE) — Spexis AG (SIX: SPEX ), a clinical-stage biopharmaceutical company focused on rare diseases and oncology, announced the results of a Phase 1 clinical trial with balixafortide (“BLX” ), a potent, specific and highly selective macrocycle inhibitor of CXCR4.This study was conducted to investigate whether BLX dosing needs to be adjusted in patients with renal impairment as part of any future market approval submission.
A Phase 1 trial was designed to investigate the pharmacokinetics, safety, and tolerability of BLX in subjects with mild (n=8), moderate (n=8), or severe (n=7) renal impairment compared to a control group (n =). 8) with normal renal function. All subjects received a single 2-hour intravenous infusion at the previously studied clinical dose of 5.5 mg/kg BLX.
In this trial, BLX was shown to be safe and well tolerated in all subjects, and management of transient and mild-to-moderate events such as hypersensitivity during initial drug infusion with CXCR4 inhibitors was independent of impairment grade. renal With increasing grades of renal impairment, BLX administration resulted in greater and longer circulating white blood cell levels, including neutrophils. Previous clinical trials with BLX at lower doses have demonstrated the ability to stimulate stem cell mobilization in healthy volunteers and patients with advanced hematologic malignancies. The data from this renal impairment trial show that doses of BLX significantly higher than 5.5 mg/kg, the highest dose previously tested, can be administered safely, induce higher levels of stem cell mobilization and for longer periods of time. much longer than 24 hours.
“The safety and pharmacokinetic results in the renal impairment trial with balixafortide and data from previous non-clinical toxicity studies support potential dose levels of up to 16.5 mg/kg, which is three times higher than previously studied dose levels. Additionally, we are encouraged by the increases we observed in white blood cells and neutrophils, which support further clinical testing of balixafortide at higher dose levels to assess its potential in generating stem cell mobilization. Based on the results observed to date, we believe that balixafortide may have the ability to generate a more potent stem cell mobilization than previously observed with CXCR4 inhibitors,” said Juergen Froehlich, MD, Chief Medical Officer of Spexis. “Patients with hematologic malignancies worldwide need additional highly effective treatment options when preparing for hematopoietic stem cell transplantation after treatment with high-dose chemotherapy. We look forward to further investigating the potential role of Balixafortide in this important area of cancer treatment.”
An increase in functional stem cells, also known as mobilization, is an important prerequisite for the collection and transplantation of bone marrow cells in patients with hematologic malignancies. CXCR4 inhibition has been shown to be a viable approach to increase the outcome of stem cell collection from peripheral blood.1 Haematological malignancies are caused by the uncontrolled growth of blood cells and lymphoid tissues and account for 6.5% of cancers worldwide and about 9% in Europe and the United States.2
About Balixafortide (“BLX”)
Discovered from the Spexis macrocycle platform, BLX is a potent, specific and highly selective macrocycle inhibitor of CXCR4 that has a role in several tumor types as well as non-oncology indications. BLX has been studied in 8 clinical trials to date involving more than 500 subjects and in multiple therapeutic indications such as solid tumors, hematological malignancies and stem cell mobilization. Spexis has partnered the Chinese rights to BLX with Fosun Pharma, a leading pharmaceutical company in China. In addition, Spexis is analyzing BLX’s existing clinical and preclinical data to determine the appropriate development path forward, including potential new indications. In line with this ongoing review, Spexis recently published data showing synergistic efficacy in combination with docetaxel in metastatic prostate cancer. These data, combined with the renal impairment clinical trial data announced today and other ongoing studies and analyses, will inform decisions on the potential future development of BLX and related macromolecules within the Spexis portfolio.
About the Spexis Macrocycle Platform:
Macrocyclines are a chemically proven class of pharmaceutical therapeutics that occupy the chemical space between small molecules and biologics. With a unique conformational balance of flexibility and rigidity, macromolecules can act on extracellular and intracellular targets not easily accessible by other chemical classes. In fact, as of 2014, around 19 distinct macrocyclic compounds have been approved by various drug regulatory authorities worldwide, covering a wide range of diseases and indications. More recently, with the emergence of the field of molecular adhesives and protein-protein interactions/degradants, it is increasingly recognized that macrostimulators have significant potential to target unique molecular targets and combine them with a variety of complementary technologies. Spexis has two separate, very diverse and well-characterized macrocycle libraries with in-depth data: PEMfinder®consisting of peptomimetic macromolecules, and MacroFinder®, composed of small molecule macrocrystals. Each library has specific applications depending on the goals in question. In addition, Spexis has a number of preclinical leads for various indications that already have significant characteristics. Spexis is looking to partner with these preclinical leads or the entire platform itself, possibly in combination with complementary technologies.
Spexis (SÉ: SPEX) is a clinical-stage biopharmaceutical company based in Allschwil, Switzerland, focused on rare diseases and oncology. For more information please visit: www.specisbio.com.
This press release contains forward-looking statements that are based on current assumptions and forecasts regarding Spexis management. Known and unknown risks, uncertainties and other factors could cause material differences between the forward-looking statements made herein and actual development, particularly Spexis’ results, financial position and performance. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date of this communication. Spexis disclaims any intention or obligation to update and revise any forward-looking statements, whether as a result of new information, future events or otherwise.