<div id="readability-page-1"><div>Drug Farm to Present Ophthalmologic and CNS Findings from Phase 1b Study of First-in-Class ALPK1 Inhibitor DF-003 in ROSAH Syndrome at ARVO 2026 Drug Farm today announced ophthalmologic and central nervous system (CNS) findings from its Phase 1b study of DF-003, a first-in-class oral ALPK1 inhibitor, will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2026 annual meeting in Denver, CO. The presentation will highlight early evidence of improvements in visual function, optic nerve pathology, and neurological symptoms in patients with ROSAH syndrome, a rare genetic autoinflammatory disease caused by activating mutations in ALPK1 and characterized by progressive retinal degeneration and systemic inflammation. Presentation Details The poster, titled “ROSAH Syndrome – an ALPK1-related autoinflammatory disease: Preliminary CNS and ophthalmic clinical outcomes from the first six patients in a Phase 1b study treated with DF-003,” will be presented during Poster Session 537: Retina/RPE: New drugs, mechanisms of action, metabolism, and toxicity. The session will take place on May 7, 2026, from 11:45 AM to 1:30 PM. Key Findings from Phase 1b Study In this open-label Phase 1b study (NCT06395285), six adult patients with genetically confirmed ROSAH syndrome completed 28 days of once-daily oral DF-003 treatment followed by post-treatment observation. Clinical improvements were observed across multiple disease manifestations during the treatment period, with all six patients demonstrating improvement in at least one clinical domain. Notably, these improvements reversed after stopping treatment, supporting a direct pharmacologic effect of DF-003. Ophthalmologic assessments demonstrated evidence of disease modulation at the level of the optic nerve and retina. Reductions in optic disc swelling and peripapillary retinal thickness were observed by optical coherence tomography (OCT), with improvements evident as early as Day 29. In one evaluable patient, measurable gains in visual function were observed, including improvements in best-corrected visual acuity of +12 letters in the left eye and +5 letters in the right eye after only eight days of treatment. In addition, improvements were observed in neurological and systemic manifestations of disease. Headache severity improved during the treatment period as measured by the HIT-6 validated questionnaire and worsened following treatment discontinuation. Reversal of anhidrosis and improvements in arthralgia were also observed, consistent with the systemic anti-inflammatory effects of ALPK1 inhibition. DF-003 was well tolerated, with no serious adverse events or treatment-emergent adverse events reported during the 28-day dosing period. No clinically significant abnormalities in liver, renal, hematologic, or coagulation parameters were observed. Adverse events were observed after treatment cessation and were consistent with recurrence of underlying ROSAH symptoms. Mechanistic Insights DF-003 is a potent and selective inhibitor of disease-causing mutants of ALPK1 (e.g. T237M) that can penetrate the blood-retina and blood -brain barriers and suppress phosphorylation of TIFA, a key mediator of innate immune signaling and NF-κB activation. The observed reversibility of clinical, ophthalmologic, and biomarker responses following DF-003 treatment discontinuation provides strong evidence that the therapeutic effects are mediated through on-target inhibition of the ALPK1 pathway. KOL Perspective “These early findings provide encouraging evidence that targeting ALPK1 may address both systemic and ocular manifestations of ROSAH syndrome,” said John Grigg, Professor of Clinical and Experimental Ophthalmology at the Save Sight Institute, University of Sydney. “The observed improvements in optic nerve pathology, visual function, and overall symptom burden, together with a favorable safety profile, support further clinical development of DF-003.” Company Statement “These data extend our clinical findings by demonstrating that selective inhibition of ALPK1 can translate into measurable improvements in ophthalmologic and neurological outcomes in patients with ROSAH syndrome,” said Henri Lichenstein, PhD, Chief Executive Officer of Drug Farm. “The consistency of response across patients, along with the reversibility of effects following treatment discontinuation, provides strong support for a direct pharmacologic mechanism and reinforces the potential of DF-003 as a disease-modifying therapy.” Next Steps Based on these findings, Drug Farm plans to advance DF-003 into a pivotal Phase 3 trial to further evaluate its efficacy, safety, and potential to address both systemic and ophthalmologic manifestations of ROSAH syndrome. About DF-003 DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and its disease-causing variants. It has therapeutic potential for ROSAH syndrome as well as heart and kidney diseases, based on efficacy observed in preclinical models. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in healthy volunteers and is currently enrolling patients with ROSAH syndrome in an ongoing Phase 1b study (NCT06395285). DF-003 has received Fast Track, Orphan Drug, Rare Pediatric Disease, Rare Disease Evidence Principles designations from the FDA. About ROSAH Syndrome ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease caused by activating mutations in the ALPK1 gene. It is characterized by progressive visual loss, optic nerve and retinal pathology, and systemic inflammatory manifestations, including elevated pro-inflammatory cytokines. Symptoms often begin in childhood or early adulthood, and there are currently no approved disease-modifying therapies. About Drug Farm Drug Farm is a private biotechnology company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH syndrome. Its proprietary IDInVivo platform integrates genetics and artificial intelligence to identify and validate novel drug targets directly in living systems. Drug Farm is advancing multiple first-in-class drug candidates into clinical development. For more information, please visit: https://www.drug-farm.com <img src="https://imageproxy.pbkrs.com/https://cts.businesswire.com/ct/CT/query-ZGlzdHJvPW54JmlkPWJ3bmV3cyZsYW5nPWVuJnNpZD1tc3RyMyZzdHk9MjAyNjA1MDc3NDgzMzFyMQ?x-oss-process=image/auto-orient,1/interlace,1/resize,w_1440,h_1440/quality,q_95/format,jpg" original-src="https://imageproxy.pbkrs.com/https://cts.businesswire.com/ct/CT/query-ZGlzdHJvPW54JmlkPWJ3bmV3cyZsYW5nPWVuJnNpZD1tc3RyMyZzdHk9MjAyNjA1MDc3NDgzMzFyMQ"/><div> United States: Henri Lichenstein, Ph.D. Chief Executive Officer Email: hlichens@drug-farm.com China: Tony Xu, Ph.D. Co-founder and Chief Operating Officer Email: tony.xu@drugfarminc.com</div>View source version on businesswire.com: https://www.businesswire.com/news/home/20260507748331/en/</div></div>

科济药业-B

招商中证生物科技主题ETF

天弘国证生物医药ETF

Drug Farm 宣布将于 2026 年 ARVO 会议上展示其 DF-003 的 1b 期研究结果，DF-003 是一种首创的 ALPK1 抑制剂。研究显示，患有 ROSAH 综合症的患者在视觉功能和神经症状方面有所改善。主要发现包括视盘肿胀的减少和视力的提高。DF-003 耐受性良好，没有严重不良事件。该公司计划将 DF-003 推进至 3 期临床试验，以进一步评估其疗效和安全性。该药物已获得多个 FDA 的认定，显示其在治疗 ROSAH 综合症及其他疾病方面的潜力

药物农场将在 ARVO 2026 会议上展示一类首创 ALPK1 抑制剂 DF-003 在 ROSAH 综合征中的 1b 期研究的眼科和中枢神经系统研究结果