Astrogen

ENG

• KOR
• ENG

• 022026.01 Astrogen and Cigalah Sign Exclusive MENA Partnership for AST-001 in a Deal Valued at up to USD 209 Million Plus Royalties - AST-001 is currently under review by the Korea MFDS for the treatment of core symptoms of Autism Spectrum Disorder (ASD), and has been granted Orphan Drug Designation - Agreement includes technology transfer and commercialization rights for AST-001 across the MENA region - Cigalah to pay Astrogen up to USD 77.5 million in regulatory and commercial milestones, in addition to tiered royalties of up to the double digit - Partnership accelerates the commercialization of a first-in-class therapy targeting core symptoms of ASD DAEGU, South Korea – January 2, 2026 – Astrogen, inc. (“Astrogen”) today announced that it has entered into an exclusive agreement for the technology transfer and commercialization of AST-001 with Cigalah Medpharm, part of Cigalah Group. AST-001 is Astrogen’s lead therapeutic candidate specifically developed to treat the core symptoms of Autism Spectrum Disorder (ASD). The partnership spans 16 countries across the Middle East and North Africa (MENA) region, including key markets such as the Kingdom of Saudi Arabia (KSA), the United Arab Emirates (UAE), and Egypt. Under the terms of the agreement, Astrogen will supply AST-001 valued at USD 131 million, and is eligible to receive up to USD 77.5 million in regulatory and commercial milestone payments, in addition to double digits tiered royalties on net sales of AST-001. Initial sales to begin through named patient basis in select markets to enable early access for patients, followed by a full commercial launch upon completion of technology transfer, regulatory approvals, and pricing agreements. A key component of the partnership is the commitment to localizing pharmaceutical manufacturing within the region. Astrogen will proceed with technology transfer to Alpha Pharma, Cigalah’s manufacturing arm located in King Abdullah Economic City, KSA. This initiative aligns with Saudi Vision 2030, which emphasizes strengthening domestic pharmaceutical production, improving patient access, and enhancing supply-chain resilience for high-value CNS therapies. “We are thrilled to partner with Cigalah, a proven leader in the MENA pharmaceutical market with deep regulatory expertise and robust commercial infrastructure,” said Dr. Su-Kyeong Hwang, CEO of Astrogen, inc. “This agreement is a transformative milestone in Astrogen’s global expansion strategy. By leveraging Cigalah’s regional capabilities and expertise in local manufacturing, we aim to accelerate the availability of AST-001 to families in the MENA region who urgently need effective treatment options for ASD.” Mr. Yaser Naghi, Chairman and CEO of Cigalah Group, commented: “Cigalah is committed to bringing innovative, life-changing therapies to patients across the MENA region. ASD remains an area with significant unmet medical need, and Astrogen’s AST-001 represents a promising advancement in this field. This partnership not only enables us to deliver an important new therapeutic option, but also further strengthens our local manufacturing ecosystem through Alpha Pharma. We look forward to a long and impactful collaboration.” About AST-001 AST-001 is an investigational drug candidate currently under NDA review by South Korea MFDS for the treatment of core symptoms of ASD in pediatric patients. The drug utilizes a proprietary oral syrup formulation designed to cross the blood–brain barrier and distributes to the brain. Its mechanism of action targets small conductance calcium-activated potassium (SK) channels, modulating dopaminergic signaling and restoring the excitation/inhibition (E/I) balance—one of the key pathophysiological features of ASD. AST-001 has demonstrated favorable tolerability, with clinical exposure in more than 400 subjects across clinical trials conducted in South Korea. Additionally, AST-001 has been granted Orphan Drug Designation (ODD) at the development stage in South Korea and becoming the 51st substance to receive this designation. Astrogen is actively preparing for regulatory interactions with the U.S. FDA, the EMA, and China’s NMPA to advance global development. About Autism Spectrum Disorder (ASD) ASD is a complex neurodevelopmental disorder characterized by persistent deficits in social communication and interaction, accompanied by restricted and repetitive behaviors. Its etiology is multifactorial, involving genetic, environmental, and prenatal factors. Once considered rare, ASD prevalence has risen significantly over recent decades due to improved diagnostic awareness and potential true increases in incidence. Today, more than 61 million individuals worldwide are estimated to live with ASD. The disorder affects adaptive functioning, communication, daily living, and social interaction, placing a significant burden on families and healthcare systems. Effective therapeutic options remain limited, underscoring the substantial unmet need. South Korea, in particular, is facing a severe ASD crisis, with data showing the shortest average life expectancy for individuals with ASD among developed nations. Average age at death for persons with ASD in South Korea is only 16.6–28.2 years, significantly below peer countries such as the U.S. (36.2–39.4), Denmark (33.3), Australia (35.3), Canada (46.2), and Sweden (53.8). Currently, there is no approved pharmacological treatment for children under five, despite early childhood being a critical period for brain plasticity. The treatment gap—with currently approved therapies limited to addressing irritability or aggression but not the core symptoms of ASD—has been associated with long-term disability, premature mortality, and in extreme cases, parent–child tragedy. About Astrogen, inc. Astrogen is a clinical-stage pharmaceutical company dedicated to developing innovative therapies for rare diseases and neurological disorders. In addition to AST-001, the company is advancing a pipeline that includes: AST-004 for Rett Syndrome, a rare neurodevelopment disorder with prevalence of about 1 in 10,000 to 15,000 live female births; AST-031, a selective norepinephrine reuptake inhibitor for Attention-Deficit/Hyperactivity Disorder (ADHD), which affects more than 300 million individuals globally; and AST-035, a small-molecule molecular glue degrader targeting glioma. For more information, visit the company’s website at www.astrogen.co.kr. About Cigalah Medpharm Trading LLC Founded in 2015 in the UAE, Cigalah Medpharm is Part of Cigalah Group, established in the late 1980s under Yousuf M.A. Naghi & Sons Group. Cigalah Group is one of the largest healthcare and consumer product distributors in the Middle East and is recognized as the No. 1 pharmaceutical distributor in Saudi Arabia in terms of registered SKUs with the SFDA. The Group represents multiple multinational pharmaceutical companies and maintains a strong presence across the GCC and broader MENA region. For more information, visit the company’s website at www.cigalah.com.   Details
• 172025.12 Astrogen Vows to Protect Developmental Golden Hour with ‘Safe Autism Treatment’. - Astrogen, a biotech startup, is developing 'Speragen (AST-001)', a new drug candidate targeting the core symptoms of ASD in young children, aiming to address the critical early intervention period when no globally approved treatments currently exist. - Clinical trials have shown that Speragen can improve social and communication skills in children as young as two, with the company seeking regulatory approval and planning for commercialization. - Beyond drug development, Astrogen is committed to supporting patients and caregivers through educational programs and social initiatives, striving to expand positive influence and improve quality of life for those affected by developmental disabilities. Autism Spectrum Disorder (ASD) is a neurodevelopmental condition characterized by difficulties in social interaction and communication, alongside restricted and repetitive behaviors. Symptoms typically emerge around two years of age. With the prevalence rate recently climbing to approximately 3%, ASD is increasingly recognized as a significant societal challenge, not just an individual one. ASD is caused by various factors, meaning treatment approaches can differ depending on the underlying cause. However, a lack of timely and appropriate intervention can limit long-term functional improvement. Crucially, no therapeutic agent for the core symptoms is currently approved for children under the age of five. Consequently, affected infants and toddlers have been forced to rely on limited specialty therapies or non-scientific treatments, despite high costs.     Su-kyeong Hwang, CEO of Astrogen / source=Astrogen Astrogen, a biotech startup, is developing 'Speragen (AST-001),' a drug candidate aimed at the core symptoms of ASD. Astrogen has validated Speragen's safety and efficacy, confirming its long-term safety and ability to improve core symptoms. The company completed its application for product approval with the Korean Ministry of Food and Drug Safety (MFDS) this year and aims for commercialization in the first half of next year. Beyond ASD, Astrogen is focused on developing new drugs for neurological disorders, including treatments for Attention Deficit/Hyperactivity Disorder (ADHD). The company also seeks to act as a motivator by supporting patients' social activities, aiming to care for their lives both before and after drug administration. The Limits of Current Treatment: A Gap in the 'Golden Hour' As a specialist in pediatric neurology, Astrogen CEO Su-kyeong Hwang has been on the front lines, treating patients with developmental and genetic disorders, and directly experiencing the reality of the treatment void. "Through personal experiences, such as a close family member suffering from a neurological condition, I deeply empathized with the pain of patients and caregivers, and felt a sense of powerlessness," said Hwang. "My desire was to personally create effective treatments that would genuinely help patients. I viewed the development of new drugs not merely as a business, but as a calling." According to Astrogen, there is currently no globally approved treatment that fundamentally improves the core symptoms of ASD (deficits in sociality and communication). Existing antipsychotic drugs, such as Risperidone or Aripiprazole, are limited to mitigating associated symptoms like anxiety or aggression. The problem centers on the therapeutic gap during the "golden hour." Early childhood is a critical period when neuroplasticity is highest. Intervention during this time can induce the formation and reorganization of neural circuits, critically influencing long-term functional improvement. Prognosis in autism treatment is decisively linked to early intervention. However, since existing antipsychotic drugs are prohibited for use in children under five, children diagnosed with ASD as early as two years old face a treatment void during this crucial phase. CEO Hwang explained, "Astrogen has focused on bridging this therapeutic gap, which has been a source of immense despair for parents and clinicians." 'Speragen': A Candidate to Target Core Autism Symptoms   Astrogen is developing 'Speragen (AST-001),' a drug candidate for ASD / source=Astrogen Astrogen's core pipeline is the ASD treatment candidate, Speragen (AST-001). After investigator-initiated trials (IIT) from 2019 to 2021 verified Speragen's safety and efficacy, Astrogen completed four clinical trials (Phases 1, 2, 3, plus a Phase 2 extension study) between 2020 and 2024. In April 2024, Speragen was designated as an Orphan Drug in the Development Stage by the MFDS. Astrogen submitted its product approval application to the MFDS in June 2025 and is currently under review. Hwang noted, "This is considered a globally significant clinical trial case, as we confirmed long-term safety and core symptom improvement in the largest cohort of pediatric patients worldwide (320 patients in total)." A key factor enabling Astrogen's rapid progress in the challenging development of a core symptom treatment was Hwang's background in the clinical field. She stated, "I clearly understood what parts patients and caregivers struggled with the most." Notably, Astrogen focused on utilizing ingredients with proven safety profiles to minimize side effects. Hwang added, "We discovered a substance that could help improve sociality and communication. Since the substance's safety was already established, we were able to obtain approval from the Institutional Review Board (IRB) of university hospitals to conduct the investigator-initiated trial (IIT), which subsequently allowed us to proceed rapidly with formal clinical trials and shorten the approval timeline." Speragen differentiates itself by targeting the core symptoms of children with autism and being usable from the age of two. Hwang explained, "The brains of autistic children often suffer from inefficient Synaptic Pruning, where abnormally excessive synapses are not properly cleared. Speragen helps normalize this process by activating brain plasticity." She emphasized, "The pre-school age, when brain plasticity is most active, is the therapeutic golden hour where treatment effects are best. Clinical trials have confirmed cases where social skills recovered to within the normal range when the drug was administered during this period." The urgency of patients registering for the clinical trials, coupled with the observed effects of the new drug, is evident. Hwang shared, "A child who had never made eye contact finally looked at their parents for the first time, and a non-verbal child uttered their first words. Beyond core symptoms, patients' anxiety and sensitivity decreased, and overall daily life improved. Caregivers were so satisfied that some even submitted official statements to continue the drug's efficacy after the trial concluded." Beyond Drug Development: Extending Social Responsibility   Astrogen aims for the long-term goal of caring for the entire lives of patients, both before and after drug administration, not just focusing on new drug development / source=Astrogen Astrogen’s long-term vision extends beyond drug development, aiming to care for the patient’s entire life journey, pre- and post-drug administration. To this end, the company launched 'Stellar Steps' in April this year, providing a free self-help and independent living education program for adolescents with autism and developmental disabilities. Hwang stated, "What children with severe developmental disabilities need most is not cognitive enhancement, but motivation for life and assistance in performing daily living skills." Stellar Steps helps participants practice essential daily skills, such as bathing, dressing, and performing simple household chores without the aid of a caregiver. Furthermore, the plan is to support job placement and offer caregivers time for emotional respite. Hwang pointed to the insufficient support systems available in adulthood. "According to statistics from the Korean Ministry of Health and Welfare (MOHW), the average lifespan of individuals with autism is 23.8 years, the shortest among all disabilities. This is significantly lower than in other developed countries (Sweden 53.8 years, Canada 46.2 years) and is influenced by vulnerable support systems post-adulthood." She then introduced a case of directly employing an individual with an autism disability, asserting, "Their expressive abilities, social skills, and cognitive functions can improve through living and interacting with people. It is crucial to provide an environment that fosters social skills without setting limits." Driving Follow-Up Pipeline Development: The Goal of 'Expanding Positive Influence'   Speragen Development Journey / source=Astrogen Established in 2017, Astrogen is steadily growing, having been selected as a preliminary unicorn by the Korean Ministry of SMEs and Startups in September this year. Following the expected product approval of Speragen by the MFDS, the company aims for a domestic launch in the first half of 2026. Global expansion is also underway, including into the US, China, and the Middle East. Notably, the company is negotiating a large-scale contract with a major pharmaceutical distributor for entry into the Middle Eastern market, which is seeing active, government-led, large-scale health investments. Astrogen is systematically expanding its follow-up pipeline across central nervous system (CNS) disorders and is preparing for an Initial Public Offering (IPO). Recognizing that 80% of autism patients also have ADHD, the company is developing a non-stimulant ADHD treatment with fewer side effects, targeting clinical entry in 2026. It is also pursuing treatments for intractable neurological diseases such as Rett Syndrome and Parkinson’s disease. Astrogen’s growth has been supported by the Seoul Startup Hub under the Seoul Business Agency (SBA). Hwang acknowledged, "The Seoul Startup Hub provided practical workspace for our Seoul-based employees and laid the groundwork necessary for startup growth through acceleration, education, and consulting. It also offered opportunities for collaboration and growth with other resident companies." Finally, Hwang emphasized her vision to make growth not just a financial metric but an "expansion of positive influence." She concluded, "Astrogen seeks to restore ordinary daily life to patients and families not only through new drug development but also through social connection that supports adolescents and caregivers with developmental disabilities. We will be a company that consistently builds the connection between science and people, filling the gaps." By Ye-ji Kim (yj@itdonga.com) URL : https://www.donga.com/en/article/all/20251217/6015589/1 * This article was written with support from SBA and Seoul City. Details

맨처음1맨마지막