Receiving FDA orphan drug designation allows the company to proceed with plans to advance the development of BRM424, a novel, first-in-class, potential treatment for neurotrophic keratitis.
BRM424 works by stimulating proliferation and differentiation of corneal limbal stem cells to regenerate healthy limbus after extensive limbal layer removal and speed up the cornea repair process.
BRIM Biotechnology announced this week the FDA granted orphan drug designation to BRM424 (BRIM Biotechnology) to treat neurotrophic keratitis (NK), a rare degenerative eye disease that causes very severe corneal damage and destruction of vision.
“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,” said Dr. Haishan Jang, chairwoman and CEO of BRIM Biotechnology. “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.”
Jang explained that the orphan drug designation validates the potential of the company’s stem cell regenerative pigment epithelium-derived factor-derived short peptide, which underpins several of its products, and has the potential to be effective in multiple therapy areas and indications.
According to the company, BRM424 works by stimulating proliferation and differentiation of corneal limbal stem cells to regenerate healthy limbus after extensive limbal layer removal and speed up the cornea repair process. The stem cell activation results in faster and effective corneal healing.
This treatment fills a gap because of the limited pharmacologic treatment options available for NK, which is either artificial tears for passive relief of discomfort or aggressive surgery with invasive procedures, the company explained in a press release.
Jang noted in the news release that 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,” Jang said in the news release. “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.”
Jang added that 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.
One drug is currently approved by the FDA to treat NK, but it comes with a very high price and 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 patients with NK. The company is currently focused on finalizing the phase 2 study protocol.
Using the NK rabbit model, BRM424 has demonstrated preclinical efficacy and safety in compliance with regulatory requirements.