The Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences (CRMH), in collaboration with research teams from the City University of Hong Kong (CityU), has successfully developed Neu-001, a first-in-class small-molecule drug candidate for the treatment of amblyopia. At a press conference held on December 17 at the Hong Kong Science Park, the breakthrough was officially announced, with Neu-001 receiving Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA). This marks the first approval for an innovative chemical drug under InnoHK initiatives set by the Hong Kong SAR Government.
The press conference was hosted by Professor Guangjin Pan, Director of the Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences. VIP guests at the event included Ms. Lillian Cheong Man-lei, JP, Under Secretary for Innovation, Technology and Industry, HKSAR Government; Professor Anderson Shum Ho-Cheung, Vice-President (Research) of the City University of Hong Kong; Ms. Yan Wu, Deputy Director-General of the Bureau of International Cooperation, Chinese Academy of Sciences; and Mr. Albert Wong, Chief Executive Officer of the Hong Kong Science and Technology Parks Corporation (HKSTP).
Ms. Lillian Cheong Man-lei, JP, Under Secretary for Innovation, Technology and Industry, HKSAR Government, highlighted the significance of the IND approval, describing it as a testament to Hong Kong’s significant position in the global biotechnology sector and a clear indicator of its pivotal role in advancing the biomedical industry. She further underscored the government’s unwavering commitment to supporting the development of life and health technologies.
Professor Anderson Shum Ho-Cheung, Vice-President (Research) of the City University of Hong Kong, remarked that CityU is deeply committed to academic excellence and translational application. He noted, “The approval of this IND application affirms the University’s dedication to driving innovation and accelerating the commercialization of breakthrough technologies while strengthening Hong Kong’s status as a global center for biomedical research and development.”
Ms. Yan Wu, Deputy Director-General of the Bureau of International Cooperation, Chinese Academy of Sciences, pointed out that the Centre for Regenerative Medicine and Health (CRMH) is the Chinese Academy of Sciences’ first biomedical research centre established in Hong Kong. She mentioned that this collaboration work demonstrates exceptional scientific innovation and coordinated development between the two regions. CRMH, through enhanced partnerships with universities, research institutions in Hong Kong, and global collaborators, aims to make significant contributions to scientific progress and public health across the Greater Bay Area, China, and beyond.
In the opening remarks, Professor Guangjin Pan, Director of the Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences, expressed profound gratitude to the Innovation, Technology, and Industry Bureau of the Hong Kong SAR Government for advancing the InnoHK Research Platform and acknowledged the strong support from the Innovation and Technology Commission. He stated that CRMH is committed to building a globally connected platform. He added, “Looking ahead, CRMH will actively promote international cooperation to tackle major health challenges such as amblyopia and cancer.”
Addressing Unmet Needs in Amblyopia Treatment
Amblyopia, commonly known as “lazy eye,” is a developmental vision disorder that reduces visual acuity and may disrupt stereoscopic vision, significantly impacting patients’ daily lives. According to the World Health Organization, about 150 million people worldwide are afflicted with amblyopia. In Hong Kong, more than 170,000 individuals are estimated to be affected, representing 2.4% of the population. Due to its origin in underdeveloped optic nerves, effective pharmacological treatments for amblyopia are currently lacking. Mainstream therapies like occlusion therapy (covering of the healthy eye), corrective glasses, and vision training are utilized, but their success rates vary. Furthermore, these approaches lose efficacy significantly in severe cases and in patients beyond the critical developmental period (usually between ages 6 and 8).
Due to the constraints of current treatment options, there is a pressing need to develop novel therapies independent of the critical developmental period. Such breakthroughs could offer effective treatments for older children and adults alike. Moreover, these new approaches should aim to reduce treatment time, improve therapeutic outcomes, and accommodate patients with amblyopia across different age groups and severity levels.
Co-developed by Prof. Micky Tortorella’s team in CRMH and Prof. He Jufang’s team in CityU, Neu-001 stands out as a pioneering solution for amblyopia treatment. With an innovative chemical composition, this first-in-class small-molecule drug is distinguished by its novel mechanism that activates neural plasticity within the visual cortex. This unique feature unlocks the unprecedented potential to cure amblyopia in both children and adults, even beyond the critical period of visual development. Preclinical studies indicate that Neu-001 can revolutionize amblyopia treatment with shorter therapy durations and sustained effectiveness, thus overcoming the limitations associated with traditional occlusion therapy.
A Milestone for InnoHK’s Innovation Platform
Neu-001 is the first chemical drug candidate to achieve the FDA IND approval under InnoHK initiative. It marks a significant achievement for the InnoHK platform, Hong Kong’s flagship innovation initiative.
Prof. Micky Tortorella, Senior Principal Investigator of CRMH, stated: “Securing IND approval for Neu-001 is an exciting milestone that recognizes the dedication of our team and the innovation behind the project.“
Prof. He Jufang, Head and Chair Professor of the Department of Neuroscience at CityU, added: “We plan to initiate a Phase I clinical trial by mid-2025 to evaluate Neu-001’s safety profile and preliminary efficacy. This multi-centre trial will occur across locations, including Hong Kong and Guangzhou. Our next goal is to obtain FDA approval for Neu-001’s market launch, offering a transformative solution for amblyopia patients worldwide.“
