Deliver Locally. Measure in Letters. Answer for Two Eyes.
Regulatory strategy for ophthalmology — where the endpoint is visual acuity in ETDRS letters and durability is the whole competition.
Local Delivery, a Functional Endpoint, and Two Eyes Per Patient.
Ophthalmology is unlike almost anything else. The drug is delivered straight into the eye, so systemic exposure is low but ocular safety — infection, pressure, inflammation — is paramount. The endpoint is best-corrected visual acuity, a functional measure read in ETDRS letters, usually paired with anatomic OCT data.
And there are two eyes: which is the study eye, how the fellow eye is handled, and how bilateral disease is measured all become design questions. We build ophthalmology programs around the eye's particular rules, from first injection to durability claim.
Delivery into the eye keeps systemic exposure low — and makes ocular safety and the injection itself central to the file.
In the Eye, Benefit Is Measured One Letter at a Time.
Best-corrected visual acuity, read on a standardized ETDRS chart, is the currency of ophthalmology trials. The number of letters gained — and the share of patients crossing a meaningful threshold — is what the division weighs.
From Anti-VEGF Injections to Retinal Gene Therapy.
Ophthalmology runs from the largest injectable market in medicine to the first approved gene therapy — each with its own endpoint and delivery challenge.
Wet AMD
The anti-VEGF space, where visual-acuity maintenance is table stakes and reducing injection frequency is the entire competitive edge.
Diabetic Eye Disease
Diabetic macular edema and retinopathy, where acuity, the diabetic retinopathy severity scale, and OCT anatomy define the pathway.
Geographic Atrophy
The newest frontier, where complement therapies won approval on slowing atrophic lesion growth — an anatomic, not acuity, endpoint.
Glaucoma
Where intraocular pressure is an accepted surrogate, and sustained-delivery implants are reshaping a historically drop-based field.
Retinal Gene Therapy
Inherited retinal disease, where the first FDA gene therapy was approved — subretinal delivery, rare-disease flexibility, and durability.
Dry Eye & Ocular Surface
Where a co-primary sign and symptom endpoint, and a strong vehicle response, make trial design its own discipline.
When the standard of care is a monthly injection, the winning claim is fewer injections for the same vision.
In the Retina, the Competition Is About How Long the Effect Lasts.
Anti-VEGF therapy transformed retinal disease — and turned frequent intravitreal injections into the treatment burden every new entrant tries to reduce. Durability, dosing interval, and sustained-delivery implants are now the battleground, and the FDA scrutinizes non-inferiority-on-vision plus a real extension of the interval.
We build the durability claim the division will accept — the right non-inferiority margin, the extension endpoint, and the ocular-safety database — and manage the combination-product questions that sustained-delivery implants and injectors raise.
Local Delivery Rewrites the Safety and Evidence Questions.
Delivering a drug into the eye lowers some risks and raises others — and creates evidence and design questions that don't exist for systemic therapies.
One patient, two eyes — the study-eye designation, fellow-eye handling, and bilateral-disease measurement are core design decisions, not details.
Endophthalmitis, intraocular pressure, and inflammation define the ocular-safety database that local delivery makes central to the file.
Sustained-delivery implants and injectors are combination products — the device and the drug are reviewed and defended together.
Six Failure Modes We Are Brought In to Prevent.
Most trace to the eye's peculiar rules being treated as ordinary ones.
A durability claim the data can't hold
Claiming a longer dosing interval without the non-inferiority margin and extension endpoint the division requires to accept it.
Study-eye and fellow-eye confusion
A design that never cleanly resolved which eye is analyzed and how the fellow eye is handled, muddying the primary read.
An anatomic endpoint over-trusted
Leaning on OCT or lesion-growth data as if it were a validated surrogate for vision where the precedent doesn't support it.
The ocular-safety database undersized
Underbuilding the endophthalmitis, IOP, and inflammation data that local intraocular delivery makes central to approval.
Implant treated as a formulation
Missing that a sustained-delivery implant or injector is a combination product with its own device and human-factors expectations.
Dry-eye vehicle effect ignored
A sign-and-symptom dry-eye program that didn't design for the large vehicle response that has sunk many before it.
Ophthalmology Regulatory Leadership Built Around the Eye's Rules.
Our ophthalmology leads have taken anti-VEGF, complement, and gene-therapy programs through visual-acuity and anatomic endpoints, durability claims, and the ocular-safety database.
"Ophthalmology plays by its own rules — local delivery, letters on a chart, two eyes, and a race on durability. The programs that win are designed around those rules from the first protocol."
The discipline we bring across wet AMD, diabetic eye disease, geographic atrophy, glaucoma, and retinal gene therapy.
Developing an Ophthalmic Therapy? Build for the Eye's Rules From the First Protocol.
Bring senior ophthalmology regulatory leadership in early — while the endpoints, durability claim, and delivery strategy are still yours to shape.
Senior-led. Embedded in your team. No junior hand-offs.