- Category: Pipeline
- Tags: retina
Pipeline
4DMT reports new phase 2b gene therapy data for wet AMD
New 2-year data demonstrates an overall 78% treatment reduction in patients treated with 4D-150 compared to bimonthly 2 mg aflibercept.
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Belite Bio presents updated phase 3 data on oral tinlarebant for Stargardt
Findings released at ASRS highlight an 80% reduction in key protein implicated in Stargardt disease after 2 years.
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RegenxBio doses first patient in phase 2b/3 trial for DR
One-time gene therapy continues to move down the pipeline, with presentations on efficacy and safety data expected at ASRS.
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Alkeus begins dosing in global phase 3 Stargardt trial on oral gildeuretinol
Phase 3 NORTHSTAR study seeks to build on growing evidence that ALK-001 benefits low luminance visual acuity.
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Oculis's phase 3 DME eye drop trial fails primary goals
While OCS-01 was well-tolerated, the DIAMOND program did not meet either outcome measures; company will not seek an FDA NDA filing.
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Atsena releases positive XLRS interim data from LIGHTHOUSE study
Enrollment for Part C of the study is underway, with the company targeting a 2028 BLA submission for ATSN-201, an investigational gene therapy.
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MeiraGTx acquires J&J's XLRP investigational therapy
Deal includes all interests in botaretigene sparoparvovec (bota-vec); company plans to immediately file for global regulatory approval.Pipeline
Belite Bio begins rolling NDA submission of oral tinlarebant for Stargardt
Full submission planned for Q2 2026, once-daily tablet has potential to become the first-ever treatment approved for this patient base.
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Ocular Therapeutix releases additional phase 3 AXPAXLI data for wet AMD
Data from SOL-1 phase 3 data will become the basis of a planned NDA submission in near future.
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FDA accepts Outlook Therapeutics' request over rejected wet AMD BLA
Company challenges decision on failed third submission package of ONS-5010 / LYTENAVA (bevacizumab-vikg), with a decision expected in May.
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FDA grants Orphan Drug Designation to SignaBlok's ROP therapy
TREM-1 inhibitor is designed to selectively target inflammation via a noninvasive delivery; company plans to report preclinical data at ARVO annual meeting.
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EyePoint begins dosing in phase 3 DME trials for DURAVYU
COMO and CAPRI pivotal studies are evaluating vorolanib insert when intravitreally-injected every 6 months; topline data expected H2 2027.
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FDA grants Orphan Drug designation to BlueRock's RP cell therapy
Marks second designation for OpCT-001; includes tax credits for qualified clinical testing and, if approved, 7 years of U.S. market exclusivity.
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BioAge Labs expands into DME clinical program for oral inhibitor asset
Preclinical evidence on NLRP3 inhibitor BGE-102 shows potential for therapeutic retinal exposure, with an initial POC trial to kick off later this year.
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Complement Therapeutics secures Fast Track Designation for lead GA therapy
AAV-based CTx001 modulates multiple pathways of the complement system when delivered as a one-time, subretinal injection.
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Aviceda releases phase 2b topline data on AVD-104 for GA
Latest SIGLEC trial results show both dosing arms with 31% to 38% less lesion growth than prior trials; phase 3 plans are underway.
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FDA rejects Outlook Therapeutics' third BLA submission of bevacizumab for wet AMD
Agency cites need for confirmatory evidence of efficacy as reason for latest CRL; ONS-5010 is already approved in the EU and UK as LYTENAVA.
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Ocular Therapeutix plans for NDA submission of AXPAXLI for wet AMD
Company expects to submit package in Q1 2026, following the anticipated release of positive 1-year data from the phase 3 SOL-1 trial.
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SeaBeLife and Unither to formulate and develop retinal degenerative disorder formulation
Collaboration focuses on accelerating development of SBL03, formulated for topical delivery to target retinal cell necrosis in GA and dry AMD.
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