The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for BRIM Biotechnology’s BRM424 in the treatment of neurotrophic keratitis (NK), a rare degenerative eye disease which causes very severe cornea damage and can lead to loss of sight.
Dr. Haishan Jang, chairwoman and CEO of BRIM Biotechnology, said: “Receiving FDA orphan drug designation allows us to proceed confidently with plans to advance the development of BRM424, a novel, first-in-class, potential treatment for NK. Clinically, the treatment of NK is very complicated, and there is a lack of effective and affordable treatment options.
“This is an important regulatory milestone that enables us to bring this potentially transformative treatment to patients sooner. Additionally, the designation validates our belief in the potential of our stem cell regenerative PEDF-Derived Short Peptide (PDSP), which underpins several products in our pipeline, and has the potential to be effective in multiple therapy areas and indications.”
Orphan Drug Designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, research and development tax credits, a waiver of prescription drug user fees, and the potential for a 7-year market exclusivity period upon FDA approval.
BRM424 uses the same active pharmaceutical ingredient (API) as BRM421, BRIM’s lead candidate for Dry Eye Disease which is about to begin Phase 3 clinical trials. The API is a synthetic peptide comprised of 29 amino acids derived from Pigment Epithelium-Derived Factor (PEDF) with neurotrophic and anti-inflammatory properties. This novel peptide activates limbal stem cells to regenerate and repair the cornea, meaning it can be used to treat various indications involving severe corneal damage.
NK is characterized by reduced corneal sensitivity, epithelium breakdown and impairment of corneal healing. Currently, there are very limited pharmacological treatment options available that involve either artificial tears for the passive relief of discomfort or aggressive surgery using invasive procedures. There is currently only one drug approved by the FDA for the treatment of NK which has a very high price point and requires a strict regimen of multiple daily doses.
BRIM plans to apply for a Phase 2 NK clinical trial of BRM424 in the US, with the intention of providing an affordable and effective new drug for treating NK patients. Using the NK rabbit model, BRM424 has demonstrated preclinical efficacy and safety in compliance with regulatory requirements.
Dr. Jang further commented: “The novel, regenerative effect of BRM424 could provide a new treatment option for patients that actively stimulates repair of corneal damage. Our ongoing studies indicate that the dosing regimen will be significantly less frequent than current treatment options and we estimate that the cost of treatment will be more affordable.
“Our focus now is to finalize our Phase 2 study protocol and accelerate the development toward commercialization, to bring this novel treatment to patients as early as possible. The ODD status of BRM424 may also help to speed up progress toward marketing approval and launch of BRM421. BRIM plans to harness its proprietary PDSP platform to address other diseases beyond ophthalmology and bring sustainable and affordable healthcare innovation to the world.”
