SCD-1 inhibitor (Pfizer Inc)

Initial data from the clinical trial is expected in 2024 PORTLAND, Ore.--(BUSINESS WIRE)-- Sparrow Pharmaceuticals, an emerging, clinical-stage biopharmaceutical company developing novel, targeted therapies to address unmet needs in both rheumatology and endocrinology, today announced that the first patient has been dosed in the Phase 2 ACSpire study (NCT number: NCT05436639) of SPI-62, a potent and selective HSD-1 inhibitor, for the treatment of autonomous cortisol secretion (ACS), a prevalent yet serious condition caused by the overproduction of cortisol from a benign tumor of the adrenal gland. This trial will complement the ongoing Phase 2 RESCUE study (NCT number: NCT05307328) for the treatment of ACTH-dependent Cushing’s syndrome. “ACS is a dangerous condition with high morbidity and risk of death, with patients often having unexplained metabolic disease, cardiovascular disease, or osteopenia, and experiencing a 35% higher rate of mortality over a five-year span,” said Frank Czerwiec, chief medical officer of Sparrow. “However, most patients don’t even know they have ACS and are only diagnosed after an appropriate endocrine workup following the discovery of an adrenal tumor on a CT or MRI scan for unrelated reasons. SPI-62 could represent the first treatment to be approved for this large yet underappreciated and underserved patient population.” The pilot, long-term, open-label study is evaluating the efficacy, safety, and pharmacological effect of SPI-62 in participants with hypercortisolism related to a benign adrenal tumor. The study will examine SPI-62's effect on morbidities of hypercortisolism including diabetes or impaired glucose tolerance, hyperlipidemia, hypertension, and osteopenia. Sparrow is actively enrolling up to 30 participants in ACSpire at sites in the United States, soon to be joined by sites in Romania, France, and the United Kingdom. Criteria for participation include adults with documented benign adrenal lesions with proven ACS, and documentation of treatment for, or evidence of, ongoing metabolic consequences for at least one of the following: hyperglycemia, hypertension, hyperlipidemia, or osteopenia, attributable to clinically significant hypercortisolism. Surgery as first-line therapy should be discussed with all eligible participants, who will be included only if they have failed or rejected available surgical therapy. To learn more about Sparrow Pharmaceuticals and its clinical trials, visit the website at . About Autonomous Cortisol Secretion Autonomous cortisol secretion (ACS) is caused by the overproduction of cortisol from a tumor of the adrenal gland. It is a somewhat milder form of hypercortisolism than Cushing’s syndrome, which is differentiated by a characteristic constellation of signs and symptoms. As patients with ACS lack these outward clinical features of Cushing’s but present with common, chronic conditions (diabetes, hypertension, high cholesterol, weakened bones), it is rarely diagnosed or addressed specifically. ACS is currently only discovered when the tumor shows up on a radiology scan for an unrelated health condition and the patient subsequently receives an adequate endocrine workup. As this infrequently occurs, most patients with ACS are dangerously exposed to excess cortisol for long periods, leading them to face major health complications, significant healthcare costs, and increased risk of early death. The signs and symptoms of ACS vary across a wide range of mental and physical effects – including diabetes, hypertension, bone fractures, and weight gain, as well as mood, cognition, and sleep disorders. About Sparrow Pharmaceuticals Sparrow Pharmaceuticals was founded to spare patients the ravages of steroids. Leveraging underappreciated scientific insights into glucocorticoid biology, the company is working to provide better treatment options for serious disorders of hypercortisolism, and to revolutionize the treatment of autoimmune and inflammatory conditions. Its lead product, SPI-62, is an oral, small molecule, novel therapeutic treatment designed to target the source of active intracellular glucocorticoids in key tissues.

Presentations Will Feature Data From ZETA-1 Phase 2 Trial of APX3330 in Diabetic RetinopathyFARMINGTON HILLS, Mich., Oct. 06, 2023 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders, today announced presentations featuring results from the ZETA-1 Phase 2 Trial of APX3330 at the 23rd Euretina Congress to take place October 5-8, 2023 in Amsterdam, Netherlands and at the 56th Annual Retina Society Scientific Meeting to take place October 11-14, 2023 in New York, NY. 23rd Euretina Congress Title:Oral APX3330 Reduces the DRSS Worsening After 24-weeks of Daily Treatment: Efficacy and Safety Results of the ZETA-1 Phase 2 Trial in Diabetic RetinopathyPresenting Author:Veeral Sheth, MDSession:Free Paper Session 14: Mixed IIDate/Time:Saturday, October 7, 2023 at 3:55 PM CESTLocation:Room E103/104, RAI Amsterdam Convention Center 56th Annual Retina Society Scientific Meeting Title:ZETA-1 Phase 2 Trial Safety and Tolerability Results for of APX3330: A Novel, Oral Ref-1 Inhibitor for the Treatment of Diabetic RetinopathyPresenting Author:Anat Lowenstein, MDSession:Diabetic RetinopathyDate/Time:Friday, October 13, 2023 at 2:59 PM ETLocation:Grand Ballroom, Intercontinental New York Barclay About Ocuphire Pharma Ocuphire Pharma, Inc. is a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders. Ocuphire’s lead retinal product candidate, APX3330, is a first-in-class small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein). Ref-1 is a regulator of transcription factors such as HIF-1a and NF-kB. Inhibiting REF-1 reduces levels of vascular endothelial growth factor (“VEGF”) and inflammatory cytokines which are known to play key roles in ocular angiogenesis and inflammation. Through inhibition of Ref-1, APX3330 normalizes the levels of VEGF to physiologic levels, unlike biologics that abolish the VEGF levels required for normal function. APX3330 is an oral tablet administered twice per day for the treatment of diabetic retinopathy (“DR”) and diabetic macular edema (“DME”). A Phase 2 study in subjects with DR or DME has recently been completed, and an End-of-Phase 2 meeting is confirmed with the U.S. Food and Drug Administration (“FDA”) in Q4 2023. DR affects approximately 10 million people with diabetes and is projected to impact 14.6 million Americans by 2050. DR is classified as Non-Proliferative Diabetic Retinopathy (“NPDR”), the early stage of the disease in which symptoms may be mild or nonexistent or Proliferative Diabetic Retinopathy (“PDR”) which is the more advanced stage of diabetic eye disease that can be highly symptomatic with loss of vision. Approximately 80% of DR patients have NPDR that will progress to PDR if left untreated. Despite the risk for visual loss associated with this disease, over 90% of NPDR patients currently receive no course of treatment apart from observation by their eye care specialist until they develop sight-threatening complications. This is due to the burdensome and frequent eye injections currently required with currently approved therapies for this disease. APX3330 as an oral tablet has the potential to be an early, non-invasive treatment for the 8 million NPDR patients in the US. Treatment with APX3330 is expected to delay or prevent progression of NPDR, thereby reducing the need for expensive intravitreal injections with anti-VEGF therapies and reducing the likelihood of vision loss due to DR. Ocuphire has also in-licensed APX2009 and APX2014, which are second-generation analogs of APX3330. The unique dual mechanism of action of these Ref-1 inhibitors of reducing angiogenesis and inflammation could potentially be beneficial in treating other retinal diseases such as age-related macular degeneration (“AMD”), and geographic atrophy (“GA”). Ocuphire is currently evaluating local delivery routes in addition to the systemic (oral) route as part of its pipeline expansion in retinal therapies. Ocuphire has a partnership with Viatris, Inc. to develop and commercialize phentolamine ophthalmic solution. Phentolamine is a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size by uniquely blocking the alpha-1 receptors found on the iris dilator muscle without affecting the ciliary muscle. In September 2023, the FDA approved RYZUMVI™ (phentolamine ophthalmic solution) 0.75% to treat pharmacologically induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents. Phentolamine is also in Phase 3 clinical development for the treatment of presbyopia and dim light (night) vision disturbances. For more information, visit www.ocuphire.com Contacts CorporateInvestor RelationsRick Rodgers, MBAInterim President & CEOir@ocuphire.comCorey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com