OHR Pharma Offers Positive Interim Top-Line Clinical Data from Phase II Study of Squalamine Eye Drops in Patients with WetAMD

Ohr Pharmaceutical, Inc.(Nasdaq: OHRP), an ophthalmology research and development company, todayannounced positive top-line interim results for its double-masked,placebo-controlled Phase II clinical trial of Squalamine eye drops in patientswith wet age-related macular degeneration (wet AMD). The data demonstrated apositive benefit in visual function across multiple clinically relevantendpoints, including a mean change in visual acuity at the end of study visitfor the interim analysis group of +10.4 letters with Squalamine eye drops plusLucentis^® PRN versus +6.3 letters in the placebo eye drops plus Lucentis PRNarm, a 65 percent additional relative benefit (p=0.18). The visual acuityimprovements were seen as early as four weeks and the relative difference invisual acuity between the two treatment arms continued to increase throughoutthe study.All patients in the study received an initial Lucentis injection followed byLucentis as needed (PRN) based on clinical response. The two treatment armswere Squalamine eye drops administered twice daily plus Lucentis PRN("Squalamine" arm or group) versus standard-of-care treatment: placebo eyedrops administered twice daily plus Lucentis PRN ("placebo" arm or group).This planned interim analysis was conducted on the first 62 patients (29treated in the Squalamine arm, 33 treated in the placebo arm), who completedthe entire nine months of the treatment protocol (representing approximately50 percent of the targeted study population). The Squalamine-treated groupdemonstrated improved best-corrected visual acuity (BCVA) gains relative tothe placebo group at all timepoints evaluated from four to 38 weeks. In theinterim analysis group, 48.3 percent of Squalamine-treated patients showedBCVA gains of ≥ 15 letters (≥ 3 lines) on a standard ETDRS eye-chart, comparedwith 21.2 percent in the placebo arm at the end of the study (p=0.025). Inaddition, patients receiving Squalamine drops were more than twice as likelyto gain ≥ 4 and ≥ 5 lines of vision compared with patients in the placebo eyedrop arm (≥ 4 lines p=0.022, ≥ 5 lines p=0.059). Importantly, the visualacuity gains for the placebo eye drop arm were consistent with those observedin previous clinical studies using Lucentis monotherapy treatment. Squalamineeye drops were well tolerated and had a comparable safety profile to placeboeye drops."The beneficial effects of Squalamine on visual acuity that we've seen thusfar, through its inhibition of multiple angiogenic growth factors andpathways, and in particular, the improvement in gains of three or more linesin vision compared with the placebo group, are truly remarkable," said Dr.Jason Slakter, Chief Medical Officer of Ohr and retina specialist atVitreous-Retina-Macula Consultants of NY. "Visual acuity is the mostclinically relevant endpoint for back-of-the-eye disorders. For wet-AMDpatients, such enhanced gains of visual acuity over standard-of-care anti-VEGFtreatments, and the restoration of vision lost to this devastating disease ofthe elderly using a convenient eye drop therapy is a very important clinicaloutcome."In the interim analysis, there were no significant differences in thefrequency of Lucentis PRN injections, which was the primary endpoint of thestudy. The mean number of Lucentis injections was 6.2 for the Squalamine armand 6.4 for the placebo arm, which included the baseline injection and anyinjections required up to and including the final study visit for the interimanalysis group."The interim results seen in this trial are encouraging," said Dr. Jeffrey S.Heier, Director of Vitreoretinal Service at Ophthalmic Consultants of Boston,member of Ohr's scientific advisory board, and study investigator. "Thepotential to treat patients with a non-invasive therapeutic option to provideadditional visual acuity benefit over the current standard-of-care, and do itwith a less than monthly injection frequency, would be a significant advancein the treatment of retinal neovascular disease and beneficial for ourpatients. We look forward to the results of the full data set and Phase IIItrials.""We are very excited by these promising interim results from this wet-AMDtrial," said Dr. Irach B. Taraporewala, President and Chief Executive Officerof Ohr. "The data further validate not only the clinical utility ofnon-invasive topical eye drop therapies for macular and retinal disorders, butalso the soundness of our company's drug development science, and ourproprietary formulation technologies that enable topical dosing to achievepositive therapeutic effects in back-of-the-eye disorders. These data give usa clear path for future registration studies, and we plan to discuss Phase IIIregistration study design and the path forward with the regulatory authoritiesin the coming months."The company plans to present the full data from this interim analysis at anophthalmology conference in the second half of this year, with final clinicaltrial data expected in the first calendar quarter of 2015.Study DesignThe ongoing clinical trial (Study OHR-002) is a randomized, double-masked,placebo-controlled Phase II study to evaluate the efficacy and safety ofSqualamine eye drops used in combination with Lucentis PRN for the treatmentof the advanced, exudative form of age-related macular degeneration (AMD),also known as "wet AMD." The trial has enrolled 142 newly diagnosed, treatmentnaïve patients, of which the first 62 had completed the treatment period atthe time of the interim analysis. The inclusion criteria allowed for patientswith visual acuity levels similar to previous Lucentis trials, varying lesionsizes up to 12 disc areas in size, and any lesion composition, includingclassic and occult only types of wet AMD. The trial also included diabeticswith no concomitant diabetic retinopathy.Patients received an initial intravitreal injection of Lucentis at studyentry, and then underwent a 1:1 randomization to receive a daily dose ofeither Squalamine eye drops or placebo eye drops administered twice daily fornine months. Patients had monthly follow-up clinic visits, where they wereevaluated and retreated as needed with Lucentis if pre-specified clinicalcriteria were met. The primary endpoint was the mean number of Lucentisinjections and secondary endpoints included visual acuity as well asdiagnostic imaging outcomes. The planned interim analysis was performed whenmore than 50 percent of the study population finished their final study visit.