Opus Genetics | MedPath

Tango Therapeutics' TNG462 Receives Orphan Drug Status for Pancreatic Cancer as Company Advances PRMT5 Inhibitor Programs

3 months ago

• Tango Therapeutics' lead candidate TNG462 has been granted Orphan Drug Designation by the FDA for pancreatic cancer treatment, providing seven years of market exclusivity upon approval. • The company's second PRMT5 inhibitor, TNG456, received FDA clearance for its IND application and will begin Phase 1/2 trials in early 2025, including a collaboration with Eli Lilly combining it with Verzenio. • Key clinical data for TNG462 monotherapy focusing on pancreatic and lung cancers is expected later in 2025, with plans to initiate a registrational study in pancreatic cancer by 2026.

FDA Grants Fast Track Status to Opus Genetics' Eye Drop Treatment for Night Vision Impairment

3 months ago

• Opus Genetics receives FDA Fast Track designation for Phentolamine Ophthalmic Solution 0.75% to treat night driving impairment in post-keratorefractive surgery patients. • The company completes enrollment in VEGA-3 Phase 3 trial evaluating the same solution for presbyopia, with 545 participants across 39 U.S. sites. • LYNX-2 Phase 3 trial for post-surgical night vision impairment is 95% enrolled, with completion expected in first half of 2025.

Opus Genetics Advances LCA5 Gene Therapy Program with First Pediatric Patient Dosing and Promising Adult Data

3 months ago

• Opus Genetics has dosed the first pediatric patient in their Phase 1/2 trial of OPGx-LCA5 gene therapy for Leber congenital amaurosis, with initial data expected by Q3 2025. • New 12-month data from the first three adult patients treated with OPGx-LCA5 confirms durability of positive responses observed at 6 months, with results to be presented at a medical conference in Q2 2025. • The company has scheduled an FDA meeting in March 2025 to discuss Phase 3 trial design and registrational endpoints, marking a significant step toward potential therapeutic advancement.

Ashvattha Therapeutics' Migaldendranib Shows Promise in Reducing Treatment Burden for Wet AMD and DME

4 months ago

• Ashvattha Therapeutics announced positive Phase 2 data for Migaldendranib (MGB), a subcutaneous nanomedicine, in treating wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). • The study showed a significant reduction in the need for anti-VEGF intravitreal injections in patients treated with MGB, with a 69% and 76.5% reduction observed in wet AMD and DME, respectively. • MGB demonstrated maintenance of visual acuity and reduction of central subfield thickness, suggesting potential for convenient at-home treatment and reduced clinic visits for patients. • Ashvattha Therapeutics also secured $50 million in funding to advance the Phase 2 ophthalmology trial and Phase 1/2 neuroinflammation trial.

Opus Genetics Gains FDA Agreement for Phase 3 Trial of APX3330 in Diabetic Retinopathy

5 months ago

• Opus Genetics has reached an agreement with the FDA under a Special Protocol Assessment (SPA) for a Phase 3 trial of oral APX3330. • The Phase 3 trial will assess APX3330's efficacy in treating moderate to severe non-proliferative diabetic retinopathy (NPDR). • The primary endpoint is a reduction in 3-step or greater worsening on the binocular diabetic retinopathy severity scale (DRSS) score. • APX3330, a first-in-class Ref-1 inhibitor, aims to slow DR progression, with Opus seeking a partner to fund further development.

LENZ Therapeutics' LNZ100 NDA Accepted by FDA for Presbyopia Treatment

7 months ago

• The FDA has accepted the New Drug Application (NDA) for LENZ Therapeutics' LNZ100, a potential treatment for presbyopia. • The NDA is supported by Phase 3 CLARITY study data, which demonstrated significant near vision improvement in participants. • Participants in the CLARITY study achieved at least a 3-line improvement in Best Corrected Distance Visual Acuity at near without losing distance visual acuity.

Ocuphire Pharma Acquires Opus Genetics, Expanding Gene Therapy Pipeline for Inherited Retinal Diseases

7 months ago

• Ocuphire Pharma has acquired Opus Genetics in an all-stock transaction, with the combined company renamed Opus Genetics, Inc., focusing on inherited retinal diseases. • The acquisition expands the pipeline to include AAV-based gene therapies for IRDs and phentolamine ophthalmic solution for presbyopia, while seeking a partner for diabetic retinopathy program. • OPGx-LCA5, a gene therapy for LCA5, shows promising early Phase 1/2 data with visual improvements in patients, and pediatric enrollment is expected in early 2025. • The merger extends the company's cash runway into 2026, anticipating key clinical milestones, including data readouts from multiple Phase 1/2 and Phase 3 trials.

Positive Phase 3 Data and New Glaucoma Treatments Highlight Advancements in Ophthalmology

5 years ago

• A recent clinical trial achieved its primary visual acuity endpoints in both the US and EU/UK, demonstrating statistically significant near vision improvements over 8 hours. • Long-term analysis of a glaucoma treatment shows clinically and statistically significant reductions in intraocular pressure up to 36 months postoperatively. • Oxular initiated a Phase 2 trial in October 2024 for OXU-001, a therapeutic candidate targeting diabetic macular edema.