Ocuphire Pharma, Inc. announced it has acquired Opus Genetics, Inc. in an all-stock purchase, combining biotechnology and gene therapy into one merged company targeting inherited retinal diseases (IRDs).
Both companies in this deal have a hefty pipeline of clinical developments, so we’ll start with each and then move into this purchase.
Let’s start with these players.
As a late clinical-stage ophthalmic pharmaceutical company, the Farmington Hills, Michigan-headquartered Ocuphire has long directed its attention to developing and commercializing therapies for the treatment of refractive and retinal eye disorders.
Opus Genetics, meanwhile, is a clinical-stage gene therapy company headquartered in Raleigh, North Carolina, and funded by the Retinal Degeneration (RD) Fund, a venture arm of the Foundation Fighting Blindness (FFB).
- Its focus: developing treatments for IRDs by zeroing in on gene mutations that lead to vision loss
Now to this purchase.
As we mentioned earlier, this was an “all-stock acquisition” of Opus.
Per Ocuphire, the newly-combined company will be renamed Opus Genetics, Inc.—effective Oct. 23, 2024 (yes, that’s today)—and will trade on Nasdaq under the “IRD” ticker symbol starting one day later.
Go on …
As far as acquisition terms, Ocuphire “issued 5.2 million shares of its common stock and 14.1 thousand shares of its convertible preferred stock to existing stockholders of Opus Genetics.”
Also in terms of shares: Shares of convertible preferred stock will reportedly be convertible into shares of common stock (pending stockholder approval in the annual meeting in April 2025).
In regards to stockholders: Following the merger, Ocuphire’s original stockholders will own an estimated 58% of the newly-combined company’s “fully diluted capitalization,” whereas Opus Genetics’ original stockholders will own approximately 42%.
Now to its leadership.
Serving as CEO of the renamed Opus Genetics is Ocuphire’s President and CEO, George Magrath, MD, MBA, MS, who served as Ocuphire’s leader since his appointment in November 2023.
Also at the helm is the joint company’s new president Ben Yerxa, PhD, former president and CEO of Opus Genetics.
Take a look at its full leadership team and Board of Directors.
What did the leaders have to say about this merger?
Dr. Magrath noted the “promising early data” of Opus Genetic’s therapeutic IRD pipeline, saying that this acquisition “is an opportunity to advance these treatments quickly, with four major clinical milestones on the horizon in 2025.”
In line with this, Yerxa stated that Ocuphire’s late-state ophthalmic candidates and regulatory approval experience will be instrumental in accelerating the former Opus Genetics’ IRD pipeline.
Speaking of pipelines … give me a rundown on what each company is bringing to the table.
First up: Ocuphire.
The former company is bringing with it a product portfolio that includes (most notably):
- Phentolamine ophthalmic solution (POS) 0.75%, currently under clinical investigation for:
- Presbyopia
- Ongoing phase 3 LYNX-2 trial initiated In April 2024, with topline data expected by Q1 2025.
- Night vision disturbances (decreased visual activity [VA] under low [mesopic] light conditions post-keratorefractive surgery)
- First patient dosed in phase 3 VEGA-2 pivotal trial in September 2023
- Presbyopia
To note: Ocuphire previously partnered with Viatris, Inc. (via Viatris’s acquisition of Famy Life Sciences and Oyster Point Pharma) on the reversal of mydriasis (RM)-indicated RYZUMVI (phentolamine POS 0.75%) in January 2023, after which RYZUMVI was granted FDA approval in September 2023 and commercially launched in April 2024.
Gotcha. So what happened to Ocuphire’s retinal pipeline?
Out of the company's original three investigational candidates, two (APX2009 and APX2014) were most recently noted as in the preclinical stage for geographic atrophy (GA) and retina, respectively.
Ocuphire’s lead retinal candidate is APX3300, a twice-daily, small-molecule oral supplement intended for nonproliferative diabetic retinopathy (NPDR) treatment.
- Recent data on it: In May 2024, Ocuphire released post-hoc analysis data from the phase 2 ZETA-1 study.
Let’s stay on that APX3300 for a moment … what’s the plan for it?
Per the new company, Opus Genetics will be seeking a strategic partner to further advance this candidate’s clinical development due to its “capital requirements and developmental timelines.”
Specifically: Opus Genetics will “redirect its existing resources towards the acquired gene therapy programs.”
Alrighty, now to (the original) Opus Genetics pipeline.
This adeno-associated virus (AAV)-based gene therapy therapeutic portfolio leverages novel orphan drug manufacturing scale and efficiencies for the following indications:
- Leber congenital amaurosis (LCA)
- OPGx-LCA5
- OPGx-RDH12
- OPGx-NMNAT1
- Retinitis pigmentosa (RP)
- OPGx-RHO
- OPGx-MERTK
- OPGx-CNGB1
- Best vitelliform macular dystrophy (BVMD)
- OPGx-BEST1
And its candidates?
Most recently (August 2024), the FDA granted Rare Pediatric Disease designation (RPD) for its lead investigational ocular gene therapy candidate (OPGx-LCA5) for LCA resulting from biallelic mutations in the LCA5 gene (and previously received Orphan Drug designation (ODD).
- See here for details on that clinical data (NCT05616793).
- Reportedly, recent 6-month findings have demonstrated the candidate’s visual improvement in 100% of study participants so far.
To note: This followed the company receiving $1.7M from the FFB to support its IRD programs earlier in the summer.
What’s next for OPGx-LAC5?
Enrollment for pediatric participants in that same phase 1/2 trial is slated to begin Q1 2025—followed by the first data anticipated in Q3 2025, according to the company.
Most notably: With its RPD and OD designations, the candidate “will be eligible to receive a priority review voucher upon Biologics License Application (BLA) approval.”
Now, is this joint company already established?
Indeed it is. In fact, Ocuphire’s original website domain has already transferred over to the new company.
See here for a look at the joint company’s pipeline of therapeutics, which encompasses:
- IRD gene therapies (see above for specifics)
- Phentolamine POS 0.75% + its proposed indications
- APX330 oral for NPDR
Lastly, what’s next for clinical trial updates?
With the new company’s expected cash runway to extend into 2026, it anticipates clinical data readouts in the coming months for:
- Phase 1/2 trial of OPGx-LCA5 for pediatric patients
- Phase 1/2 trial of OPGx-BEST1 for initial patients
- Phase 3 LYNX-2 trial (NCT06349759)
- Phase 3 VEGA-3 trial (NCT06542497)