EyeDNA Therapeutics Announces Positive 24-month Data from Ongoing Phase I/II Trial of HORA-PDE6b Gene Therapy in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

Clinically meaningful benefit in visual functions and good safety profile of HORA-PDE6b is confirmed at 24-month follow-up

Phase I/II results will be discussed with US and European health authorities to define optimal path to making HORA-PDE6b available to patients with PDE6b Retinitis Pigmentosa

Paris, France, May 7, 2024 – eyeDNA Therapeutics (‘eyeDNA’), a newly created subsidiary of Coave Therapeutics (‘Coave’), a genetic medicine company focused on developing life-changing therapies, today announces positive 24-month follow-up results from its Phase I/II study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b, its investigational gene therapy for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6b gene (PDE6b RP). These data were reported during an oral presentation* on May 6, at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, US.

The positive 24-month follow-up data presented confirm results from the previous interim analysis of the trial conducted at the 12-month follow-up point and support preparation for a registrational trial for HORA-PDE6b in PDE6b RP patients. Further discussions with health authorities in the US and Europe are planned to define the optimal path to making HORA-PDE6b available to PDE6b RP patients.

To date, HORA-PDE6b has been administered in 17 evaluable patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses in four consecutive cohorts. The treatment was administered in the more affected eye while the other eye served as an untreated control.

In a subgroup of clinical interest of six patients receiving the high dose, with less advanced disease (Best Corrected Visual Acuity (BCVA) score ≤75 ETDRS letters [ ≤ 20/32 Snellen equivalent], Goldmann Visual Field (GVF) ≥ 10 degrees), positive efficacy results were reported at 24 months on the BCVA and the GVF outcomes. The BCVA mean change from baseline increased by +0.09 LogMar in the untreated eye, while acuity in the treated eye was stabilized (+0.02 LogMar). From baseline, the mean reduction of the GVF was over 300 deg² superior in the untreated eyes compared to the treated eye.

Interestingly, long-term data available from patients from the low dose group (n=7) followed up over a five-year period found that the BCVA of the untreated eyes consistently declined (increase of 0.05 to 0.08 LogMAR/year from the second year), which is in line with the natural history of the disease. Meanwhile, the mean change from baseline of BCVA of the treated eyes in the same group is stabilized (between +0.03 and +0.06 LogMAR over the same follow-up period). The difference of BCVA mean change from baseline between the treated eyes and the untreated eyes at five years is 0.25 LogMAR (12 Letters).

Furthermore, the full-field stimulation test (FFST) in blue light assessing rod function continues to show an improvement of the light perception threshold in favor of the treated eyes, which is considered clinically meaningful (improvement of almost six decibels). The interesting positive trend on the retinal anatomical evaluation by Optical Coherence Tomography (OCT; Ellipsoid Zone horizontal length) observed at 12-month follow-up on the subgroup of clinical interest is maintained after 24 months.

Following the 24-month study period, both doses were well tolerated (n=17). Five ocular Serious Adverse Events (SAEs) occurred including two resolved SAEs possibly related to HORA-PDE6b (one chorioretinitis and one reduced visual acuity). Patients did not receive preventive oral corticosteroids.

An additional cohort of four to six younger patients aged 13-17 years old with a GVF at baseline ≥ 20 degrees in each meridian and at an earlier disease stage is ongoing with three patients enrolled.

The highly encouraging safety and efficacy data observed in patients two years after treatment with HORA-PDE6b continue to support our view that this novel gene therapy could provide an important clinical benefit for PDE6b RP patients. These data will support our discussions with regulators to determine the optimal route for getting HORA-PDE6b to patients,” said Rodolphe Clerval, Chief Executive Officer. “At the same time, we continue to evaluate HORA-PDE6 in an expansion cohort of younger patients with less advanced disease, for whom treatment with HORA-PDE6b could have an even greater therapeutic impact.”

PDE6b retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness. These two-year safety and promising efficacy results are of great medical interest and could represent a significant step towards providing an effective treatment for patients with this devastating disease,” commented Dr. Jean-Baptiste Ducloyer, MD, Nantes University Department of Ophthalmology.

*Abstract 2134:

JB Ducloyer, et al. 12-month Safety and Efficacy Evaluation of HORA-PDE6b, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation


About eyeDNA Therapeutics and HORA-PDE6b

eyeDNA Therapeutics, a wholly owned subsidiary of Coave Therapeutics, is a clinical-stage gene therapy company, focused on developing life-changing therapeutics for inherited retinal disorders. Our lead program HORA-PDE6b, an AAV5-based gene replacement therapy, is being evaluated in a Phase I/II trial for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene (PDE6b RP) (NCT03328130).

eyeDNA and Théa Open Innovation (‘TOI’) are partners for the development and commercialization of HORA-PDE6b. eyeDNA is responsible for the global development of HORA-PDE6b and retains commercial rights to the product in the US, Japan, South Korea, China and other territories outside Europe. In Europe and certain other countries, HORA-PDE6b is being co-developed by Coave and TOI under a license and development agreement with exclusive rights granted to TOI to commercialize HORA-PDE6b in these territories.

About Coave Therapeutics

At Coave Therapeutics, we are leading the transition of genetic medicine from rare to prevalent conditions, starting with neurodegenerative and eye diseases. Our proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform introduces chemical modifications onto AAV capsids or Lipid Nanoparticles (LNPs) to overcome the limitations of current vectors on efficacy, safety, and manufacturability.

With low doses and optimized routes of administration, our conjugated vectors have demonstrated markedly improved transduction and biodistribution in the central nervous system and the eye across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent neurodegenerative and ocular diseases – including genetically and non-genetically defined indications.

Coave recently created its subsidiary eyeDNA Therapeutics to focus on the development – up to the marketing authorization application – of its unique gene therapy HORA-PDE6b for the treatment of inherited retinal diseases caused by mutations in the human PDE6b gene.

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit https://coavetx.com/ and follow us on LinkedIn.

eyeDNA Therapeutics and Coave Therapeutics
Rodolphe Clerval, CEO

Sylvie Berrebi, Leila Adlam, Mark Swallow

Qlaris Bio Completes $24 Million Series B Financing Round to Advance QLS‑111, a First-in-class IOP-lowering Drug Candidate for Glaucoma

Financing led by Canaan and New Leaf Venture Partners, and includes funds managed by abrdn Inc., Correlation Ventures, and Mayo Clinic Ventures

DEDHAM, Mass.–(BUSINESS WIRE)–#EVPQlaris Bio, Inc. (“Qlaris”), a clinical-stage biotechnology company targeting unmet needs in debilitating ophthalmic diseases, today announced that it has closed a $24 million Series B financing round. Co-led by Canaan and New Leaf Venture Partners, the financing also included new participation from funds managed by abrdn Inc., as well as existing investors Mayo Clinic Ventures and Correlation Ventures.

Proceeds from the financing will support the continued clinical development of QLS‑111, a first-in-class therapeutic being developed to lower intraocular pressure (IOP) by targeting episcleral venous pressure (EVP). Qlaris is currently conducting two U.S. Phase II clinical trials of QLS‑111 (Osprey and Apteryx). The Osprey and Apteryx studies are investigating the safety, tolerability, optimal dosing, and efficacy of QLS‑111 in patients with open angle glaucoma (OAG) and ocular hypertension (OHT). Additional clinical advancements, including the initiation of the Nightingale Phase II clinical trial in normal tension glaucoma (NTG), are anticipated by the end of 2024.

The science behind QLS‑111 originated in the lab of Dr. Michael Fautsch, Ph.D., a Mayo Clinic professor of ophthalmology, biochemistry, and molecular biology. The drug candidate aims to lower IOP by relaxing vessels of vascular and vascular-like tissues distal to the trabecular meshwork, thereby reducing distal outflow resistance and lowering EVP. As there are currently no approved medications that selectively target the reduction of EVP, there is a significant gap in the potential to maximally lower IOP, as EVP can be the largest determinant of overall IOP. Preliminary data suggest that QLS‑111 is well-tolerated with no significant hyperemia or safety concerns.

“We are grateful to have the support of this outstanding syndicate of leading life science investors, which will enable the continued development of our lead product, QLS‑111,” said Thurein Htoo, MS, MBA, Chief Executive Officer of Qlaris. “Qlaris is dedicated to developing new mechanisms of action that target unaddressed parameters within IOP regulation, an area of critical need. QLS‑111 aims to achieve this goal by fundamentally targeting EVP to enable improved IOP control for patients with glaucoma, including those with NTG, a condition for which there are no currently approved therapeutics. This funding will provide Qlaris the means to execute on our clinical strategy to develop a novel solution to a critical unmet need.”

“Since the company’s inception, we have believed in Qlaris’ novel technology, talented leadership, and skilled team,” said Wende Hutton, General Partner at Canaan. “We invest in companies that partner an innovative approach with strong leadership and accomplished scientists to successfully reach critical milestones. Qlaris has the potential to demonstrate the importance of targeting EVP in the ongoing QLS‑111 Phase II trials and we look forward to the data from this innovative drug candidate.”

About Qlaris Bio, Inc.

Qlaris Bio, Inc. was founded in August 2019 with a singular focus: to develop novel, innovative therapies with first-in-class mechanisms of action to address serious and debilitating ophthalmic diseases. The company’s lead program, QLS‑111, uses ATP-sensitive potassium channel modulators that improve outflow through distal vascular tissues of the eye to reduce IOP. Qlaris Bio’s investors include Canaan and New Leaf Venture Partners, both of which were co-lead investors in the company’s $25 million Series A funding round in August 2019. Other investors include Correlation Ventures, Mayo Clinic, and funds managed by abrdn Inc. For more information, please visit qlaris.bio.


Michele Gray

(917) 449-9250

Biophytis – therapeutics to slow down the degenerative processes associated with aging

Biophytis is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratoy failure in patients suffering from COVID-19.

Biophytis - therapeutics to slow down the degenerative processes associated with aging

Biophytis SA, founded in 2006, develops drug candidates targeting diseases of aging. Using its technology and know-how, Biophytis has begun clinical development of innovative therapeutics to restore the muscular and visual functions in diseases with significant unmet medical needs. Specifically, the company is advancing two proprietary drug candidates into mid-stage clinical testing this year: Sarconeos (BIO101) to treat sarcopenic obesity and Macuneos (BIO201) to treat dry age-related macular degeneration (AMD). The business model of BIOPHYTIS is to ensure the conduct of the project until clinical activity in the patient is proven, then to license the technologies in order to continue the development in partnership with a pharmaceutical laboratory. The company was founded in partnership with researchers at the UPMC (Pierre and Marie Curie University) and collaborates with scientists at the Institute of Myology, and the Vision Institute

Its lead drug candidate, Sarconeos (BIO101), is an orally administered small molecule in development for the treatment of neuromuscular diseases, including sarcopenia and Duchenne muscular dystrophy (DMD). Our second drug candidate, Macuneos (BIO201), is an orally administered small molecule in development for the treatment of retinal diseases, including dry AMD and Stargardt disease.

Biophytis also has preclinical efficacy data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a rare neuromuscular disease. Spinal Muscular Atrophy is a rare progressive neurodegenerative disease that robs an individual of the ability to walk, eat and breathe. SMA is the leading genetic cause of death in infants. Symptoms may appear in the first 6 months of life (type 1, the most severe and common), in infancy (types 2 and 3) or in adulthood (type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and about 1 in 50 Americans is a genetic carrier.

More info : www.biophytis.com

Keywords for Biophytis :

neuromuscular diseases, dry age-related macular degeneration, AMD, ophthalmology, infectious diseases, COVID19, Asthma, COPD, small molecule, sarcopenia, Stargardt disease, Duchenne muscular dystrophy , DMD, preclinical stage pharma company, clinical stage pharma company , Spinal Muscular Atrophy , rare diseases , aging