By: Reportable
June 24, 2025
LEKSUM: New Study Indicates Efficacy of Multiple Doses of Extracellular Vesicles to Address Muscle Myopathy
BOSTON, Massachusetts, June 24, 2025; A new study by Leksum LLC (Leksum) successfully demonstrates that multiple doses of extracellular vesicles (EVs) could mitigate the risk of lung injury for patients placed on mechanical ventilation (MV) while in intensive care units (ICU).
Early results show significant improvement in both size and function of diaphragm muscle fibers after one dose of EVs. Although the new data is preliminary, additional improvement has been achieved from two doses. The study was conducted at Leksum's new one-of-a-kind laboratory located at SLU, Uppsala, Sweden, which uniquely replicates the effects of MV on muscle tissue in ICU conditions.
The positive results following a second dose of EVs support the hypothesis of Professor Lars Larsson, Leksum’s Chief Scientific Officer, regarding the efficacy of EVs derived from bone marrow mesenchymal stromal cells (BM-MSCs) to combat lung injury and diaphragm muscle dysfunction associated with immobilized patients subject to MV. Dr. Larrson stated, "If these preliminary results hold true, they indicate an almost complete preservation of both diaphragm muscle fiber size and function." Leksum’s scientific team will follow-up with detailed single muscle fiber contractile and muscle fiber size measurements as it prepares for Phase I trials in Europe.
Findings at day five after multiple doses of extracllular vesicles (EV’s):
- Fiber cross-sectional area (CSA) and specific force (SF) measurements for specimens receiving two doses of EVs were 93% and 94%, respectively, compared with control fibers (0-day). For specimens not receiving treatment, previous studies showed CSA and SF measurements of 57% and 52%, respectively, after five days.
- Great stability of hemodynamic parameters, respiratory peak pressure, and peripheral oxygen saturation (SpO2 = 97-99%).
- No accumulation of mucus in the mouth around the tracheal tube - accumulation of mucus is typically observed on the first or second day of MV in control subjects and on the fourth and fifth day with a single dose of EVs.
- At the dissection: the lungs appeared in very good condition according to visual inspection (i.e., they looked like controls); the blood also looked highly oxygenated.
- The diaphragm muscle, at dissection microscope, appeared in very good condition compared with samples after five days without intervention; in particular, the thickness appeared preserved.
This latest research is the fifth study investigating the effect of EVs on single muscle fiber size and force generation capacity (specific force, maximum force normalized to fiber cross-sectional area). The team's previous studies successfully demonstrated the efficacy of EVs derived from BM-MSCs in combatting lung injury associated with MV, i.e., targeting a potential pathophysiological mechanism underlying Ventilator Induced Diaphragm Dysfunction (VIDD).
MV is a life-saving intervention. However, it often results in a delayed and complicated weaning process due to diaphragm muscle dysfunction, which is associated with higher mortality and morbidity rates. Mitigating the negative effects of MV promises to enhance the quality of life for millions of patients suffering from muscle myopathy and related conditions while reducing the expense of the weaning process, totals for which are estimated to be upwards of $100 billion a year (US & EUR).
About Leksum
Leksum LLC, based in Boston, Massachusetts with its primary laboratory at SLU, Uppsala, Sweden, is an early-stage life sciences company developing advanced biologic therapies to address acquired muscle myopathies. The company is preparing to enter into Phase I/IIa studies related to its LK-ICU program, which encompasses therapies addressing muscle myopathies resulting from the use of mechanical ventilation in ICUs, in particular with immobilized patients. Leksum's scientific team is also researching additional skeletal muscle conditions including sarcopenia, cancer cachexia, and muscle-loss due to the use of weight-loss drugs (i.e., GLP-1s).
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