Kiora Pharmaceuticals, Inc. announced its second quarter 2023 financial results, reported on the progress of its clinical development pipeline, and provided an update on the company's sharpened strategic focus.
The most notable milestone of the second quarter was the reported preliminary data from the ABACUS phase 1b clinical trial in patients with retinitis pigmentosa. The data clearly demonstrated that the lowest dose of KIO-301 is safe and tolerable while driving positive objective and subjective impact on vision for the initial trial participants.
This reinforced the company's plans to complete enrollment using the higher doses of KIO-301. The company plans to complete all dosing in Q3 and expects to announce topline results across all patients and doses at an upcoming ophthalmic medical conference in the fourth quarter.
The ABACUS study's preliminary results, along with subsequent internal analysis of additional data, were a major contributor to the Company's decision to further sharpen its clinical development focus exclusively on rare retinal diseases. Specifically, the Company plans to expand clinical development of KIO-301 to include choroideremia and Stargardt's disease. These diseases, like RP, result in the degeneration of retinal photoreceptors but preserve the downstream retinal ganglion cells, which are the target of KIO-301's mechanism-of-action.
All three indications represent rare, orphan diseases for which there are no available treatments. As a result, the Company may pursue cost-effective and expedited paths to market.
As part of this new focus, Kiora will shift clinical development resources allocated for KIO-101 to treat the ocular presentation of rheumatoid arthritis (OPRA) to KIO-104, for the treatment of posterior non-infectious uveitis, a rare T-cell-mediated, intraocular inflammatory disease. KIO-104, which uses the same active compound in KIO-101 but formulated for intravitreal delivery, is ideally suited to potentially suppress overactive T-cell activity to treat the underlying condition.
Data from a previous phase 1b/2a study, reported in October 2022, showed that a single injection of KIO-104 decreased intraocular inflammation in a dose-dependent fashion and improved visual acuity significantly during the duration of the study. Further, there is evidence of reduced Cystoid Macular Edema from baseline.
Continued development of the anterior segment applications for KIO-101 and KIO-201 will be dependent on potential strategic partnerships. This will allow Kiora to retain the potential upside while working with a partner who has the resources to dedicate to developing and ultimately commercializing these promising therapies.
"Our sharpened focus is due in large part to the preliminary results we've observed in the ABACUS study, which reaffirm we're on the right path," said Brian M. Strem, PhD, president and CEO of Kiora. "There's a tremendous need for new treatments for RP, choroideremia, and Stargardt's disease and KIO-301 has the potential to treat all three in a similar fashion. This includes our newly planned extension of the ABACUS study to include additional RP and Choroideremia patients. By pursuing all three with the same therapeutic, we plan to realize efficiencies that can get a new therapy to patients as quickly as possible."
Achieved and upcoming milestones:
The milestones that Kiora achieved in the first half of 2023 include the following:
- KIO-301: Reported initial results from ABACUS at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in Q2 2023
- Entered into a partnership with the Choroideremia Research Foundation to support strategic clinical development of KIO-301 in Choroideremia
- KIO-201: Reported results from a Phase 2 Persistent Corneal Epithelial Defect study at the ARVO Annual Meeting in Q2 2023
- Business: Raised $6.3 million in funding to extend runway into May 2024
- Business: Received $1.2 million of research tax incentives from Australia and Austria
- Business: Appointed Carmine Stengone to the Board of Directors
The company anticipates achieving the following clinical and regulatory milestones:
- KIO-301: Complete dosing of high-dose cohort of ABACUS in Q3 2023
- KIO-301: Report topline results in RP from the ABACUS study in Q4 2023
- KIO-301: Discuss the development path for KIO-301 in RP with the FDA in a type 2 pIND meeting
- KIO-301: Initiate phase 2 trials for KIO-301 in RP, Choroideremia and Stargardt's patients
- KIO-301: Pursue Orphan Drug Designations for KIO-301 for Choroideremia and Stargardt's Disease
- KIO-104: Pursue Orphan Drug Designation for KIO-104 for Posterior Non-Infectious Uveitis
- KIO-104: Discuss the development path for KIO-104 in Posterior Non-Infectious Uveitis with the FDA in a type 2 pIND meeting
Financial results
In the first half of 2023, research and development expenses were $1.8 million, compared to $1.3 million for the first half of 2022. Research and development expenses for the second quarter of 2023 were $1.4 million compared to $0.6 million for the second quarter of 2022. The increase was primarily due to clinical trial-related expenses.
General and administrative expenses for the first half of 2023 were $2.4 million compared to $3.5 million for the first half of 2022. General and administrative expenses for the second quarter of 2023 were $1.1 million, compared to $1.8 million in the second quarter of 2022. The reduction is attributable to reduced consulting fees, lower facilities costs due to the consolidation of offices, and lower personnel costs due to staffing optimization and benefit cost savings.
Net loss was $4.5 million for the first half of 2023 compared to $6.0 million for the first half of 2022. Net loss was $2.6 million for the second quarter of 2023 compared to $2.4 million for the second quarter of 2022. This decrease was primarily due to lower personnel and facilities expenses offset by an increased investment in clinical trial activities to support the advancement of KIO-301.
Kiora ended the quarter with $8.0 million in cash and cash equivalents. In June 2023, the Company received an additional $1.2 million in research tax credits related to development research that was performed in Australia and Austria. Additionally, Kiora may in the future draw on a $10 million equity line of credit (subject to certain limitations), of which there is up to approximately $9.6 million available.