Rockville’s RegenxBio Inc. (NASDAQ: RGNX) just scored an important regulatory clearance to advance its eye disease treatment.
The Montgomery County biotech said Tuesday the Food and Drug Administration has given the green light to its investigational new drug application for RGX-314, its current gene therapy candidate for wet age-related macular degeneration, as a one-time treatment for patients with diabetic retinopathy. That means the company can start dosing patients in a phase 2 trial, which it plans to do this year in hopes of reporting initial data next year.
That trial will evaluate the safety and effectiveness of the candidate, delivered through the eye, to 40 patients with diabetic retinopathy. That same candidate is already being primed for a phase 3 trial for patients with wet AMD that it hopes to start this year. It's also being tested in yet another clinical trial for a different delivery method, through a device at the doctor’s office — data from that second phase of trials could come by year’s end as well.
All three studies of RGX-314 set out to provide patients a one-time alternative to the current standard of care for wet AMD and diabetic retinopathy, which involves monthly or bimonthly anti-vascular endothelial growth factor injections, or anti-VEGF as they’re called, to manage the condition.
Diabetic retinopathy, which affects more than 8 million people in the U.S., “is often asymptomatic until it progresses or patients develop vision-threatening complications which require intervention,” said Dr. Steve Pakola, chief medical officer for RegenxBio, in an email to the Washington Business Journal. “We believe that one-time treatment with RGX-314 could provide sustainable, long-term anti-VEGF delivery to potentially reduce severity of DR and prevent vision-threatening complications.”
The company’s work has not stalled because of the pandemic, Pakola said. “We do not anticipate that Covid will affect our plans through the remainder of the year.”
Outside of ophthalmology, RegenxBio is separately advancing several other gene therapy programs through clinical trials, those focused on rare diseases such as Mucopolysaccharidosis Type II — or Hunter syndrome, a debilitating disease that affects young children — which kicked off a phase 1/2 study this year. The company is also developing gene therapy treatments for Hereditary Angioedema, a rare genetic condition involving painful swelling episodes, and neuromuscular indications.
RegenxBio has also made some changes to its pipeline this year. It added RGX-381, a treatment candidate for visual changes from CLN2, or Batten, disease, alongside its existing RGX-181 program to treat the disease in the central nervous system. But it has opted to end development of a candidate to treat Homozygous Familial Hypercholesterolemia, or HoFH, which can cause heart disease in teenagers and young adults.
That decision came as the landscape of treatment options has shifted “dramatically” since the company started the program, “with the emergence of several new therapies,” RegenxBio CEO Ken Mills said on a recent second-quarter earnings call with analysts.
“The target product profile has changed, so while our gene therapy approach supported signs of biological activity at the second dose level, we acknowledge that further pharmacology work is needed,” he said on the call of its HoFH candidate. “We will look for opportunities to support continued advancement of this program through business development, and certainly focus on ways to share clinical trial data that benefits patients and foster scientific discovery in cooperation with the investigators in the future.”
This all comes as the business is looking to grow, preparing to move to its new 132,000-square-foot headquarters and manufacturing facility in 2021. That site, still in Rockville at 9800 Medical Center Drive, grows and centralizes the biotech’s 80,000-square-foot footprint that’s now distributed across five locations, including its main home at 9600 Blackwell Road.
That expansion follows a deal RegenxBio struck with Alpharetta, Georgia-based Clearside Biomedical Inc. (NASDAQ: CLSD) last September, for rights to its platform to treat several eye diseases. That agreement, which followed others, gave the local company Clearside’s in-office injection that it’s now studying as an alternative to surgery.
RegenxBio closed out the first half of 2020 with $339.2 million in cash and equivalents, according to its second-quarter disclosures. The company generated $34.2 million in revenue in the first two quarters of this year, up from $8.8 million during the same period of 2019, per Securities and Exchange Commission filings. That came from an increase in royalty revenue from sales of Zolgensma, a gene therapy for spinal muscular atrophy in young children that relies on RegenxBio’s gene delivery platform.
That drug was launched last year by Novartis (NYSE: NVS), which inherited it when it acquired Chicago-based AveXis Inc. (NASDAQ: AVXS). RegenxBio is also eligible for $80 million in milestone payments from Novartis when it reaches $1 billion in cumulative net sales of the product — which, per SEC filings, has raked in more than $735 million since May 2019.
