Kiora Pharmaceuticals Reports 2023 Results; Continues to Advance Pipeline of Treatments for Rare Retinal Diseases
- Strategic partnership with Théa Open Innovation and private placement expected to fund operations through 2026
- Focus on advancing KIO-301 and KIO-104 for rare retinal diseases
- KIO-301 could be the first vision-restoring option for RP patients
- Achievements in 2023 include positive results from ABACUS-1 for KIO-301
- Financially strengthened with cash and cash equivalents exceeding $30 million
- Plans for clinical and regulatory milestones for KIO-301 and KIO-104 in 2024
- None.
Insights
Examining Kiora Pharmaceuticals' latest strategic moves reveals a company poised for growth, leveraging both innovation in rare retinal disease treatments and shrewd financial management. The partnership with Théa Open Innovation and the subsequent private placement not only infuse Kiora with immediate capital but also lay a foundation for sustained funding through 2026. This positions the company favorably against competitors who may face funding challenges.
From a financial perspective, the tiered royalty structure up to the low 20s percentage on net sales for KIO-301 is a notable detail. It suggests confidence in the product's market potential and provides a clear revenue stream upon commercialization. Furthermore, the eligibility for up to $285 million in milestones offers significant upside potential. Investors should note the strategic allocation of R&D expenses, with TOI's reimbursement mechanism effectively mitigating financial risk associated with the development of KIO-301.
The reduction in G&A expenses is an encouraging sign of operational efficiency. However, investors should monitor the projected increase in R&D spend for 2024, ensuring it correlates with value-creating activities. Kiora's strengthened cash position, with over $30 million as of March 2024, alleviates near-term liquidity concerns and supports the company's strategic R&D initiatives.
The clinical development of KIO-301 and KIO-104 is a testament to Kiora's commitment to addressing unmet needs in the treatment of rare retinal diseases. KIO-301, as a potential first-in-class vision-restoring treatment for retinitis pigmentosa, represents a significant breakthrough for patients with inherited retinal degenerative diseases. The safety and tolerability demonstrated in the ABACUS-1 study, along with improvements in multiple visual measures, underscore the drug's promise.
For KIO-104, targeting non-infectious uveitis, the direct ocular delivery system could be a game-changer, potentially reducing systemic side effects associated with current treatments. The drug's mechanism of action, already proven in systemic autoimmune diseases, could be transformative if successfully adapted to ocular applications. The expansion into other retinal conditions like proliferative vitreoretinopathy further illustrates the broad applicability of Kiora's research.
With the FDA's feedback aligning with Kiora's clinical development plans, the path to market for KIO-301 appears straightforward, potentially expediting the delivery of this innovative treatment to patients. Stakeholders should watch for progress in the upcoming Phase 2 trials and any additional data that will shed light on the drugs' efficacy and safety profiles.
The strategic partnership with Théa Open Innovation is a calculated move by Kiora to cement its presence in the eye care market. TOI's established leadership in this space can enhance Kiora's market penetration and brand credibility. Additionally, the focus on orphan indications, known for their efficient paths to market, could result in a quicker return on investment and lower marketing costs.
Market demand for new treatments in the space of rare retinal diseases is high, as current options are limited. Kiora's pipeline, especially with KIO-301's potential as a first vision-restoring option, could disrupt the market and capture significant share if clinical outcomes prove positive. The emphasis on non-infectious uveitis with KIO-104 taps into a niche yet critical need for safer, more effective treatments.
The financial stability provided by the recent funding and strategic partnership allows Kiora to focus on its R&D without the immediate pressure of fundraising, which could be beneficial in a market where investor sentiment can quickly shift based on clinical trial outcomes. The company's ability to maintain a strong cash position while advancing its pipeline will likely be viewed favorably by the market.
Recent Strategic Partnership and Financing Expected to Fund Operations Through 2026
Encinitas, California--(Newsfile Corp. - March 25, 2024) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced its 2023 financial results and provided an update on its retinal disease development pipeline. The Company's major initiatives planned for 2024 are to initiate Phase 2 clinical development of KIO-301, a small molecule photoswitch, for the treatment of inherited retinal diseases, starting with retinitis pigmentosa (RP); and further development of KIO-104, an intravitreal, anti-inflammatory for treatment of non-infectious uveitis. The
"Our greatest priorities this year are to advance KIO-301 and KIO-104 to assess their further potential to benefit patients," said Brian M. Strem, Ph.D., chief executive officer of Kiora. "Both products are clinically validated drug candidates based on proprietary and innovative small molecules targeting rare retinal diseases with large unmet needs.
Kiore’s pipeline of drug candidates against rare retinal diseases.
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"KIO-301 could potentially become the first vision restoring option for patients with inherited retinal degenerative diseases like RP. Ongoing development will be collaboratively guided and fully funded by our partner, TOI. Should KIO-301 gain marketing authorization, it would provide Kiora with meaningful commercial milestones and royalties up to the low 20s (%) with a partner who has a proven track-record of leadership in the eye space. Working with TOI, we are finalizing details of the randomized, double-masked, controlled, dose-ascending Phase 2 trial in RP and look forward to providing more specifics on expected enrollment and results timelines.
"The balance sheet provides us the financial ability to fund further development of KIO-104 for the treatment of posterior non-infectious uveitis. Because non-infectious uveitis can be sight threatening, there is a strong market need for new, steroid sparing therapeutic approaches. The active compound in KIO-104 is a highly potent, disease modifying anti-inflammatory agent belonging to a class of drugs helping hundreds of thousands of patients with systemic autoimmune diseases including multiple sclerosis and rheumatoid arthritis. By delivering KIO-104 directly to the eye, we believe we can reduce the negative effects of retinal inflammation without the associated potential side effects of systemic anti-inflammatory drugs or chronic steroid exposure. Beyond non-infectious uveitis, the mechanism of action of KIO-104 could apply to other retinal conditions, such as proliferative vitreoretinopathy (PVR), a complication following retinal detachment repair, where nonclinical work is ongoing."
"Orphan indications involve efficient, cost-effective paths to market," added Melissa Tosca, EVP Finance. "For 2024, we expect to increase our R&D spend, with expenses for KIO-301 offset by quarterly reimbursement from TOI. Further, we anticipate G&A expenses to remain relatively flat for the year. We believe this balances our desire to achieve meaningful development while maintaining a strong cash position expected to fund operations through 2026."
Kiora has strengthened its fundamentals by advancing its pipeline, entering a strategic partnership, and strengthening its balance sheet.
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Achieved and Upcoming Milestones:
Notable milestones that Kiora achieved in 2023 and year-to-date 2024 include the following:
KIO-301
- Entered a strategic partnership granting TOI exclusive worldwide development and commercialization rights, excluding Asia, to KIO-301 for the treatment of retinal degenerative diseases. Under the terms of the deal, Kiora received an upfront payment of
$16 million and is eligible to receive up to an additional$285 million in development, regulatory, and commercial milestones; tiered royalties of up to low 20s on net sales; and reimbursement of KIO-301 research and development expenses.
- Completed enrollment and reported topline results from ABACUS-1. Results demonstrated KIO-301 was safe and tolerable with improvements consistent across multiple measures, including improved visual field, visual acuity, and functional vision (navigating in real-world simulations). In addition, functional MRI measures showed reactivation of the specific regions of the brain responsible for vision.
- Held a Type B, pre-IND meeting with the US FDA regarding Kiora's proposed clinical development plans and received feedback on trial design, including approvable endpoints, details for the control group, and outstanding non-clinical requirements, all consistent with Kiora's current plans.
KIO-100 Family (KIO-104, KIO-101)
- The publication of results from a Phase 1 double-masked study of KIO-101 in the medical journal Pharmaceutics, documenting a 12-day treatment of KIO-101 topically at multiple doses was well tolerated in healthy volunteers and patients with inflammation of the eye. There was a significant decrease in conjunctival hyperemia in the treatment group compared to the placebo group.
- Granted US and European patents covering local ocular delivery of the KIO-100 family of non-steroidal, anti-inflammatory small molecules.
Kiora anticipates achieving the following clinical and regulatory milestones:
KIO-301
- Report additional ABACUS-1 data, including quantitative functional MRI measures showing reactivated brain activity in the visual cortex in a time-dependent manner.
- Complete comprehensive design, under the joint Kiora-TOI steering committee, of a randomized, controlled, multicenter, double-masked, dose-ascending Phase 2 study of KIO-301 in retinitis pigmentosa with initiation expected as early as the fourth quarter of 2024.
- Pursue Orphan Drug Designations for KIO-301 for choroideremia and Stargardt disease in the US and EU.
KIO-104
- Initiate supportive non-clinical research to inform and optimize Phase 2 and registration studies in 2024.
- Plan a multicenter, randomized, double-masked, dose-ascending Phase 2 study of KIO-104 for the treatment of non-infectious uveitis.
- Perform additional pre-clinical proof-of-concept studies of KIO-104 in proliferative vitreoretinopathy, a rare but serious complication of retinal reattachment procedures beginning in 2024.
Financial Results
Kiora ended the year with
In 2023, research and development expenses were
General and administrative expenses for 2023 were
Net loss was
About Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing and commercializing products for the treatment of orphan retinal diseases. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of posterior non-infectious uveitis. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase. In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on Twitter and LinkedIn as well as to visit our website and/or subscribe to email alerts.
Forward-Looking Statements
Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, Kiora's ability to execute on development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104, KIO-301, KIO-201 and KIO-101, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, the sufficiency of existing cash on hand to fund operations for specific periods, the ability to timely complete planned initiatives for 2024, including phase 2 clinical development of KIO-301 and KIO-104, the potential for KIO-301 to be the first treatment options for patients with inherited degenerative diseases like RP, Kiora's plans to further fund development of KIO-104, the potential for KIO-104 to reduce inflammation, the timing of topline results from a Phase 2b trial of KIO-104, the potential for KIO-104 to apply to other retinal inflammatory diseases, and expected trends for research and development and general and administrative spending in 2024. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to satisfy the closing conditions related to the offering ,the ability to conduct clinical trials on a timely basis, market and other conditions and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2024 or described in Kiora's other public filings. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law.
Contacts:
Investors
Investors@kiorapharma.com
Media
Kiora@crowepr.com
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