Savara has completed patient enrolment in the Phase IIa OPTIMA clinical trial to examine molgradex for the treatment of nontuberculous mycobacterial (NTM) lung infection.
Altogether, 30 patients with chronic mycobacterium abscessus or mycobacterium avium complex (MAC) infection were enroled in the open-label, non-controlled, multi-centre trial.
A 24-week treatment period and a 12-week follow-up stage are included in the trial, which comprises two subgroups of subjects.
Subgroup one comprises patients who are sputum culture positive and on a multidrug NTM guideline-based anti-mycobacterial regimen, which had been ongoing for a maximum of six months prior to the baseline.
The second subgroup includes patients who are sputum culture positive, but either stopped a multidrug NTM guideline-based anti-mycobacterial regimen at least 28 days prior to screening, or have never taken such treatment.
The trial's primary objective is sputum culture conversion, as measured by at least three consecutive sputum samples without the growth of NTM.
Its secondary objectives include the number of subjects with sputum smear conversion to negative, the number of subjects with durable sputum culture conversion, and the number of subjects with durable sputum smear conversion.
The trial is expected to provide interim results in the fourth quarter of this year and top-line results in the second quarter of next year.
Savara CEO Rob Neville said: “People with NTM lung infections urgently need more effective treatment options, and we believe the rapid enrollment completion in OPTIMA reflects a high interest in our innovative, anti-infective immunotherapy candidate.
“Antibiotics currently used with this patient population are poorly tolerated, frequently fail to eradicate the infection and are associated with a high recurrence rate.
“Molgradex has the potential to fundamentally change the treatment paradigm of NTM infections by stimulating the innate immune system in the lungs, instead of targeting bacteria directly, thereby avoiding problems of antibiotic resistance.”
Molgradex is an inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) being developed by Savara.