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MONTREAL, Oct. 1, 2024 /PRNewswire/ - Thryv Therapeutics, a clinical stage biotech company dedicated to developing treatments for rare and life-threatening cardiovascular indications, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to LQT-1213 for the treatment of Long QT Syndrome (LQTS). LQT-1213 is a novel, first-in-class SGK1 inhibitor specifically designed to treat congenital LQTS. Thryv has developed a series of SGK1 inhibitors for the treatment of cardiometabolic stress associated with various arrhythmic diseases including LQTS, atrial fibrillation, and heart failure. "We are pleased with the FDA's Orphan Drug Designation for LQT-1213 based partly on emerging positive clinical data from our Wave 1 clinical study in patients with Long QT Syndrome" said Debra Odink, PhD, President and Chief Development Officer of Thryv Therapeutics. "People with Long QT Syndrome deserve a properly studied and FDA-approved therapy to help in their battle against this potentially lethal genetic disease. There is currently no FDA-approved therapy for people with Long QT Syndrome. This designation reinforces the potential of LQT-1213 to fulfill this unmet need and provides critical incentives for Thryv to accelerate our efforts to deploy prospectively designed, pivotal efficacy studies in people with congenital Long QT Syndrome. We remain focused on bringing innovative treatments to adults and children with rare diseases who have limited options." Following a request from the FDA, clinical data from the ongoing Wave I clinical study in people with congenital LQTS was provided to the Office of Orphan Products Designation. Shortly thereafter, the company received notification that its request for Orphan Drug Designation was approved. The FDA Orphan Drug Designation is a special status granted to drugs and biologics targeting rare diseases, defined as those affecting fewer than 200,000 people in the United States. This designation offers numerous benefits to support the development of treatments for rare conditions, including tax credits for clinical trial costs, exemption from certain FDA fees, and up to seven years of market exclusivity following FDA approval. About LQT-1213 LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Type 1, 2, and 3. By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in people with LQTS, thereby decreasing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating congenital LQTS. Additional information about the on-going Wave I clinical study is available at using identifier: NCT05906732. About Thryv Therapeutics Inc. Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit . SOURCE Thryv Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Topline results anticipated in Q4 2024 for Phase 2 trial evaluating the safety and tolerability of OCS-05 in patients with Acute Optic Neuritis (AON) Oculis also aims to complete an IND submission for OCS-05 in the U.S. in 2024 OCS-05 has been granted orphan drug designation in the United States and Europe for AON, an indication for which there are no approved therapies May 08, 2024 -- Oculis Holding AG (Nasdaq: OCS; XICE: OCS) (“Oculis” or the “Company”), a global biopharmaceutical company purposefully driven to save sight and improve eye care, today announced the completion of enrollment in its multi-center, randomized, double-blind, placebo-controlled Phase 2 ACUITY trial evaluating the safety and tolerability of OCS-05 in patients with Acute Optic Neuritis (AON). Topline results are expected in Q4 2024. The Phase 2 ACUITY study is evaluating once-daily OCS-05 intravenous infusion in patients with AON. The study is ongoing across four (4) sites in France. At present, there are no approved therapies for AON, leaving a significant gap in medical care for treatments that offer neuroprotection and can prevent vision loss. OCS-05 has been granted orphan drug designation in both the United States and Europe, highlighting the importance of this unmet medical need. Riad Sherif, M.D., Chief Executive Officer of Oculis, commented: “We continue to execute on our strategic programs and are pleased to complete enrollment in the ACUITY trial, an important milestone for OCS-05 clinical program that keeps us on track for an anticipated topline readout in Q4 this year. In the meantime, we keep on working towards an IND submission for OCS-05 in the U.S. in 2024. We anticipate that a positive readout in this trial would support further development of OCS-05 as a potential first-in-class neuroprotective treatment with wide applicability in neuro-ophthalmic diseases such as AON, glaucoma, geographic atrophy, diabetic retinopathy and other diseases where protecting retinal neurons is key to preserving patients’ sight.” Sophie Bonnin M.D., Ph.D., Deputy Head of the Ophthalmology Department, at Rothschild Foundation Hospital, in Paris, and Scientific Advisor for the ACUITY trial, said: “AON is a rare disease characterized by acute inflammation and demyelination of the optic nerve. While corticosteroids are used to shorten the attack, there is no approved therapy for AON and unmet needs remain for therapies that can prevent vision loss after an acute episode of optic neuritis. The completed enrollment brings us a step forward in the development of OCS-05 as a potential neuroprotective candidate which could have a profound impact in ophthalmology, and we are eagerly awaiting the anticipated trial readout in Q4 of this year.” Pablo Villoslada M.D., Chair, Department of Neurology, Hospital del Mar, Barcelona, and Oculis Scientific Advisory Board member, said: “The completion of enrollment in the ACUITY trial in patients with an acute inflammatory-demyelinating disorder is very exciting. The novel and differentiated mechanism of action of OCS-05 triggers multiple beneficial effects on apoptosis, oxidation and inflammation which supports neuronal survival and repair. The results from this Phase 2 trial will provide an opportunity to further understand this mechanism of action in a clinical setting and explore its potential benefits for patients suffering from ophthalmic diseases where neuroprotection is needed.” OCS-05 is a serum-glucose corticoid kinase-2 (SGK-2) activator with the potential to become a neuroprotective therapy for acute optic neuritis and other neuro-ophthalmic diseases. In ophthalmology, this mechanism of action could potentially protect the nerve axons in conditions such as acute optic neuritis, to ultimately prevent vision loss. In animal models of neuroinflammation and neurodegeneration, OCS-05 has shown positive results in prevention of retinal ganglion cell damage and was associated with improvements in clinical function (disability). The Phase 2 ACUITY study in Acute Optic Neuritis (AON) is currently ongoing with results anticipated before the end of the year. OCS-05 is an investigational drug and has not received regulatory approval for commercial use in any country. The Phase 2 ACUITY is an ongoing randomized, double-blind, multi-center, two-arm, placebo-controlled study to evaluate the safety and tolerability of once daily OCS-05 intravenous infusion in patients with Acute Optic Neuritis (AON). Positive outcomes in this trial could support the development of the compound for potential application in the treatment of ophthalmic conditions where neuroprotection is needed. AON is a rare disease characterized by an acute inflammation of the optic nerve that can lead to permanent visual impairment. It affects up to 5 in 100,000 people worldwide each year and often represents the first sign of multiple sclerosis. It mainly occurs in adults between the age of 20 and 40 years and is more frequent in women (2:1). The acute inflammatory process of AON leads to the loss of myelin covering the optic nerve and the axons. At the onset, patients often suffer from ocular pain that increases with eye movement and vision loss. Once the inflammation recedes, remyelination often occurs but it is incomplete. Without the myelin sheath protecting the axon, neurons located in demyelinated segments become fragile and prone to death. Unfortunately, damaged axons cannot regrow, leading to permanent visual impairment. Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) purposefully driven to save sight and improve eye care. Oculis’ highly differentiated pipeline comprises multiple innovative product candidates in development. It includes OCS-01, a topical eye drop candidate for diabetic macular edema (DME) and for the treatment of inflammation and pain following cataract surgery; OCS-02, a topical biologic anti-TNFα eye drop candidate for dry eye disease (DED) and for non-infectious anterior uveitis; and OCS-05, a neuroprotective candidate for acute optic neuritis (AON). Headquartered in Switzerland and with operations in the U.S. and Iceland, Oculis’ goal is to improve the health and quality of life of patients worldwide. The company is led by an experienced management team with a successful track record and is supported by leading international healthcare investors. The content above comes from the network. if any infringement, please contact us to modify.

MONTREAL, May 7, 2024 /PRNewswire/ - Thryv Therapeutics Inc., a biotechnology company pioneering precision treatment to treat indications including Congenital Long QT Syndrome, atrial fibrillation, and heart failure, today announced a poster presentation of SGK1 inhibition in models of Drug-Induced QT prolongation at the 2024 Heart Rhythm Society meeting in Boston, MA. This presentation will take place on Friday, May 17th, from 10:30 a.m. to 12:30 p.m. ET. The meeting will commence with a presentation by Thryv co-founder and Principal Scientific Advisor, Dr. Saumya Das, at the Stanford Biodesign New Arrhythmia Technologies Retreat in Boston, where he will discuss Small Molecules for Arrhythmia Therapy. Heart Rhythm 2024 Poster Presentation Title: Attenuation of Drug-Induced QT Prolongation in Guinea Pig Isolated Heart and Anesthetized Dog Models by serum/glucocorticoid-regulated kinase 1 (SGK-1) Inhibitors. Date and Time: Friday, May 17th from 10:30 a.m. to 12:30 p.m. ET. 2024 Stanford BioDesign Arrhythmia Technologies Retreat Presentation Title: Small Molecules for Arrhythmia Therapy Presenter: Saumya Das, MD, PhD Date and Time: Wednesday, May 15th at 9:04 a.m. About Thryv Therapeutics Inc. Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to treat indications including Congenital Long QT Syndromes (LQTS), atrial fibrillation and heart failure, with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase (SGK1). For more information, please visit . SOURCE Thryv Therapeutics Inc.