IRVINE, Calif.—Novus Therapeutics, Inc., a company focused on developing products for disorders of the ear, nose, and throat (ENT), has announced results from two OP0201 single-dose Phase 1 clinical trials. The primary objective of establishing safety and tolerability was achieved in both healthy adults (study C-001), and in adults with acute otitis media (study C-004).
OP0201 is being developed as a potential first-in-class treatment option for otitis media (OM), often caused by Eustachian tube dysfunction (ETD). OP0201 is comprised of a novel formulation of a surfactant (dipalmitoylphosphatidylcholine) and a spreading agent (cholesteryl palmitate) suspended in propellant. The product is administered intranasally via a pressurized metered-dose inhaler.
OP0201 is intended to restore the normal physiologic activity of the Eustachian tube (ET). Together, the active ingredients in OP0201 effectively absorb to the air-liquid interface of the mucosa and reduce the interfacial surface tension of the ET, which reduces the passive pressure required for the ET to open, promoting “de-sticking” of the ET so that ventilation of the middle ear may occur.
“We are delighted that results from single-dose Phase 1 studies met the primary objectives and demonstrated a favorable safety and tolerability profile,” said Dr. Catherine Turkel, president of Novus Therapeutics. “The safety profile of OP0201 was similar to placebo, and also similar when administered to healthy adults or adults with acute otitis media.”
Study C-001 was a randomized double-blind, cross-over, single-dose Phase 1 trial in healthy adults designed to evaluate safety, tolerability and explore Eustachian tube function within a 150-minute period following a single 20 mg intranasal dose of OP0201 or placebo. Participants were exposed to changes in pressure induced by a 6-minute hyperbaric/hypobaric protocol in an atmospheric pressure chamber.
The mean age of the 17 participants was 25.8 years; 52.9% of patients were female. In total, 76.5% (13/17) of participants who received OP0201 and 81.3% (13/16) of participants who received placebo experienced one or more adverse events. The most common OP0201 adverse events were tympanic membrane hyperemia and tympanic membrane disorder. These events were attributed by the ENT physician investigator to be related to the pressure chamber, and not deemed related to OP0201 or placebo. No serious adverse events (SAEs) occurred during the study.
“Study participants did not report any adverse effect as it relates to taste or smell after exposure to OP0201. In study C-001, we did not observe any adverse effect on Eustachian tube function,” Turkel continued. “In study C-004, the majority of the participants in both treatment groups reported relief of ear pain, although no treatment difference was observed between the treatment groups.”
Study C-004 was a randomized double-blind, placebo-controlled, parallel-group Phase 1 trial in adults with acute otitis media and moderate or worse ear pain designed to evaluate safety, tolerability and to explore relief of ear pain within a 60-minute period following a single 20 mg intranasal dose of OP0201 or placebo. The mean age of the 24 participants was 49.5 years; 66.7% were female. 16.7% (2/12) of participants who received OP0201 and 50.0% (6/12) of participants who received placebo experienced one or more adverse events. The two adverse events reported in the two participants who received OP0201 were mild nasal discomfort and mild lacrimation increase. Both adverse events were mild and resolved. No SAEs occurred during the study.
“We have also recently completed our multiple-dose Phase 1 safety trial, and in the coming months we will report data from the 14-day dose-escalation safety and tolerability study in healthy adults,” Turkel mentioned.
Study C-002 is a Phase 1 clinical trial designed to evaluate safety and tolerability of twice daily intranasal administration of OP0201 over 14 consecutive days in 30 healthy adults. The trial includes a 30 mg/day dose (Cohort A) and a 60 mg/day dose (Cohort B) of OP0201.
Turkel also noted that “this past February we initiated our Phase 2a pediatric otitis media development program which is ongoing, and by year end we will have our first look at safety, tolerability and efficacy with 10 days of OP0201 treatment compared to placebo in infants and children with acute otitis media.”
Study C-006 is an exploratory Phase 2a clinical trial designed to evaluate safety, tolerability and efficacy of twice daily intranasal administration of OP0201 over 10 consecutive days in infants and children 6 to 24 months of age with acute OM. The trial explores the effects of a 20 mg per day dose of OP0201 as an adjunct to oral antibiotics. Patients will be followed for up to 30 days, during which multiple efficacy endpoints will be explored.