OBI-902 has been granted by US FDA for Orphan Drug Designation for the Treatment of Cholangiocarcinoma

  • OBI-902 is the first ADC utilizing OBI’s proprietary GlycOBI glycan-based ADC enabling technology for evaluation of safety and efficacy in patients with Cancer.

OBI Pharma, a clinical-stage oncology company (4174.TWO) received notification from the US FDA stating that the request for Orphan Drug Designation of OBI-902 TROP2 ADC for the treatment of Cholangiocarcinoma has been granted. OBI-902 is the first OBI-developed ADC that incorporates our proprietary site-specific glycan-conjugated ADC enabling technology.

Cholangiocarcinoma is a rare and lethal malignancy with fewer than 50,000 patients in the United States and a 5-year survival rate ranging from 2% and 23% depending on disease stage, histological subtype, and localization[1]. At present, there are no FDA approved ADC therapies for cholangiocarcinoma.

To encourage the industry to develop new treatment options for rare diseases, the US FDA grants Orphan Drug Designation to experimental therapies that have the potential to treat these diseases. In the United States, a rare disease is defined as any condition that affects fewer than 200,000 patients. After granting Orphan Drug Designation, the US FDA qualifies companies or drug developers incentives such as tax credits for clinical trials, exemption from user fees, and marketing exclusivity[2].

In August 2025, OBI launched a phase I/II clinical trial in the United States and Taiwan, recruiting patients with advanced solid tumors. The objectives of the trial are to study the safety, pharmacokinetics, and preliminary efficacy profile of OBI-902 in these patient populations.

Heidi Wang, Ph.D, OBI Pharma’s Chief Executive Officer noted, “Based on our preclinical data, OBI-902 has several important advantages over other TROP2 ADCs either approved or in development; including high stability in blood circulation, excellent bystander effect that extends the killing to neighboring cancer cells lacking TROP2 expression, potential ability to overcome drug resistance, and outstanding activity in animal and organoid models of cancer. Importantly, this marks the first time an ADC that incorporates OBI’s proprietary GlycOBI® ADC technology is being evaluated in patients, including those diagnosed with cholangiocarcinoma. We look forward to investigating this potential best-in-class TROP2 ADC in the clinic.”

About OBI-902
OBI-902 is a TROP2-targeted antibody-drug conjugate (ADC) that carries a potent topoisomerase I inhibitor payload to kill tumor cells and with a drug-antibody ratio (DAR) of 4. TROP2 is highly expressed in a variety of solid tumors such as breast, lung, biliary, bile duct (cholangiocarcinoma), ovarian, gastric, and many other cancer types, rendering it an ideal target for cancer therapy.

OBI-902 is a novel site-specific glycan-conjugated ADC using OBI’s proprietary GlycOBI platform, which provides improved stability and enhanced hydrophilicity. OBI-902 demonstrated remarkable antitumor efficacy, improved pharmacokinetic characteristics, and a favorable safety profile in various animal models. The IND of OBI-902 was cleared by the US FDA on April 30, 2025.

Since December 2021, OBI has been granted by Biosion, Inc. (www.biosion.com) an exclusive, worldwide (except in China) license to a TROP2 targeting antibody amino acid sequence. Biosion holds exclusive rights to that antibody sequence in China. OBI holds worldwide commercial rights to OBI-902, except for the rights pertaining to the antibody in China.

About GlycOBI®
OBI has developed a unique clinical stage, glycan-based site-specific ADC technology (GlycOBI®), which is in a ‘Plug and Play’ format and compatible with any antibodies, linkers, and payloads in drug-antibody ratio (DAR) up to 16. Utilizing OBI’s proprietary dual-function enzyme (EndoSymeOBI®) and linker technology (HYPrOBI®), homogenous ADCs are manufactured with an efficient and scalable process under GMP conditions. The conjugation process of GlycOBI® avoids disrupting the antibody structure and ensures the ADC has similar biophysical characteristics to the native antibody. Furthermore, OBI’s linker technology has improved conjugation efficiency of the payload, reduced aggregation propensity, which provides advantages on manufacturing ADC products. GlycOBI® conjugated ADCs have overcome the limitations of traditional ADCs and achieved better antitumor activity and stability in various in vivo animal studies. GlycOBI®, EndoSymeOBI®, and HYPrOBI® are part of the armamentarium of OBI’s Obrion™ ADC Enabling Technologies that also include ThiOBI® and GlycOBI DUO™. OBI-902 is the first ADC utilizing OBI’s Obrion™ ADC enabling technology for evaluation of safety and efficacy in Cancer, currently under Phase I/II clinical trial in the US and Taiwan.

About OBI Pharma
OBI is a clinical stage global oncology company that is headquartered in Taiwan and established in 2002. Its mission, together with its wholly owned subsidiary OBI Pharma USA, Inc., is to develop novel therapeutic agents for patients with high unmet medical needs.

OBI’s primary focus is the development of novel ADCs, including the first-generation cysteine-based TROP2 ADC, OBI-992. Using the company’s proprietary ADC enabling technology, GlycOBI®, powered by EndoSymeOBI® and HYPrOBI®; OBI has created its next-generation novel ADC pipeline, including monospecific: OBI-902 (TROP2), OBI-904 (Nectin-4), bispecific single payload (HER2 x TROP2), and bispecific, dual payload (cMET x HER3) ADCs. To broaden the applicability of linker technology, HYPrOBI®, OBI further developed a novel ThiOBI® platform to enable irreversible cysteine-based conjugation. Additionally, OBI’s pipeline includes the first-in-class AKR1C3-targeted small-molecule prodrug OBI-3424, which selectively releases a potent DNA-alkylating antitumor agent in the presence of the aldo-keto reductase 1C3 (AKR1C3) enzyme that is highly expressed in tumors. Additional information can be found at www.obipharma.com.

GlycOBI®, EndoSymeOBI®, ThiOBI® and HYPrOBI®are registered trademarks of OBI Pharma. Obrion™ and GlycOBI DUO™ are trademarks under registration.

1 National Institute of Health for Rare Diseases. Sept. 2025
https://rarediseases.info.nih.gov/diseases/9304/cholangiocarcinoma

2 US FDA website. Designating an Orphan Product: Drugs and Biological Products Sept.25 https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

Forward-Looking Statements
Statements included in this press release that are not a description of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about future clinical trials, results and the timing of such trials and results. Such risk factors are identified and discussed from time to time in OBI Pharma’s reports and presentations, including OBI Pharma’s filings with the Taiwan Securities and Futures Bureau.

COMPANY CONTACT:
Kevin Poulos, Chief Business Officer
OBI Pharma USA, Inc.
+1 (619) 537 7698, ext. 102
kpoulos@obipharma.com

SOURCE: OBI Pharma USA, Inc.

Innovative Vaccine Engine Continues to Drive Growth: CanSinoBIO Enters a New Growth Cycle

In the first half of 2025, the structural transformation of the pharmaceutical industry continued to deepen. Driven by favorable policies, product upgrades, and technological innovation, the vaccine industry maintained a stable and positive growth momentum. As a leading enterprise in China’s innovative vaccine sector, CanSino Biologics Inc.(Stock Code: 688185.SH, 6185.HK, “CanSinoBIO” or “the Company”) once again delivered a solid performance.

According to its 2025 interim report, CanSinoBIO achieved revenue of RMB 382 million in the first half, up 26% from a year earlier, extending its growth streak. Although the company has yet to turn profitable, losses have narrowed significantly, with the reduction exceeding 94% compared to the same period last year. This reflects improved operational quality and lays a solid foundation for steady full-year growth. With core products selling strongly and multiple pipeline programs advancing, CanSinoBIO’s long-term growth story is steadily taking shape.

Meningococcal Vaccine Portfolio Continues to Scale Up, Unlocking Growth Drivers
CanSinoBIO’s two currently marketed meningococcal conjugate vaccines—the quadrivalent meningococcal conjugate vaccine (MCV4, Menhycia) and the bivalent meningococcal conjugate vaccine (MCV2, Menphecia)—remain the company’s key revenue drivers. In particular, the sustained scaling-up of MCV4 demonstrates strong market competitiveness and is central to CanSinoBIO’s growth narrative.

In 2024, Menhycia and Menphecia together generated nearly RMB 800 million in sales revenue, representing year-on-year growth of over 40%. In the first half of 2025, driven by deeper channel penetration and higher terminal market coverage, the sales of both vaccines continued to grow steadily. During the same period, the company’s meningococcal vaccine portfolio generated sales of over RMB 364 million, representing more than 38% year-on-year growth and providing a solid foundation for earnings.

As the only MCV4 in China, Menhycia effectively covers four meningococcal serogroups—ACYW135—and leverages the high-safety CRM197 carrier protein technology. With outstanding clinical immunogenicity and safety, it has become the preferred choice for many parents seeking meningococcal vaccination for their children.

Furthermore, Menhycia’s market potential is still being unlocked. The vaccine has been submitted to the National Medical Products Administration (NMPA) for an expanded age indication—from the current “children aged 3 months to 3 years (47 months)” to “children aged 3 months to 6 years (83 months).” If approved, this will directly broaden its coverage, strengthen penetration in the non-national immunization program market, and inject greater certainty into CanSinoBIO’s growth over the next two to three years, further consolidating the company’s leadership in the meningococcal vaccine field.

iPneucia Officially Launched, Setting a New Benchmark for Differentiated Pneumococcal Vaccine
In June, CanSinoBIO’s self-developed product—the 13-valent pneumococcal conjugate vaccine  (PCV13, iPneucia)—was officially approved for market launch, marking the company’s entry into the RMB 10 billion-plus pneumococcal vaccine market and the beginning of a new growth cycle.

Pneumococcal diseases remain a serious global public health concern, with particularly high morbidity and mortality rates among children under five years old. Addressing the remaining immunization gaps in China, the launch of iPneucia fills the domestic technological void in high-end 13-valent pneumococcal conjugate vaccines, demonstrating CanSinoBIO’s leadership in differentiated innovation.

Compared with existing competing products in the market, iPneucia achieves breakthroughs in three major dimensions. First, it offers more targeted protection, focusing on four high-risk serotypes—19F, 19A, 7F, and 3—that together account for over 60% of pneumococcal disease cases among Chinese children. Clinical trial data show that its Geometric Mean Concentration (GMC) of antibodies is significantly higher than that of competing products. Second, in carrier protein technology, it adopts the globally pioneering CRM197+TT dual-carrier technology, overcoming the immune interference limitations of single-carrier vaccines. This both reduces the risk of immune suppression when co-administered with other vaccines and significantly enhances immunogenicity. Finally, in manufacturing safety, iPneucia uses an animal component–free fermentation process, reducing risks associated with animal-derived biological factors. It also employs no formaldehyde detoxification and adds no phenol during production, significantly improving vaccine safety.

With these three advantages, iPneucia could potentially become another “ace” for CanSinoBIO in the infant bacterial vaccine market, following Menhycia. Notably, iPneucia shares a highly overlapping target population with Menhycia, creating strong channel synergies that will accelerate commercialization and enable the company to quickly capture market share in the RMB 10 billion-plus pneumococcal vaccine segment.

Combination Vaccine Strategy Progresses Steadily
Beyond this, CanSinoBIO’s long-term potential lies in its continued build-out of a “Combination Vaccine” portfolio. Compared with traditional monovalent vaccines, multi-conjugate vaccine and polyvalent vaccine can prevent multiple diseases with a single injection, significantly improving vaccination efficiency and willingness—representing a key global direction in vaccine technology development.

Following the quadrivalent vaccine Menhycia and multi-valent vaccine iPneucia, CanSinoBIO’s DTcP-Hib-MCV4 pentavalent vaccine—a representative combination vaccine—has attracted strong market attention. In February this year, it received clinical trial approval, marking a “zero-to-one” breakthrough for the company in the high-end combination vaccine sector.

The DTcP-Hib-MCV4 pentavalent vaccine can simultaneously prevent pertussis, diphtheria, tetanus, Haemophilus influenzae type b (Hib) disease, and infection by the four meningococcal serogroups (ACYW135), achieving “protection against five diseases with a single shot”. This significantly reduces the number of injections and improves compliance among infants. More importantly, its core component Menhycia has already undergone commercial validation in China and enjoys a strong reputation, providing robust support for the combination vaccine’s development. This “point-to-surface” product upgrade logic enables CanSinoBIO to advance in the combination vaccine market with greater efficiency and confidence. With the government placing high importance on combination vaccine R&D and offering policy support, CanSinoBIO is well-positioned to capture this high-value market.

R&D-Driven Growth with a Clear Product Pipeline and Strong Reserves
While consolidating its existing market advantages, CanSinoBIO continues to strengthen its mid-to-long-term growth momentum through robust R&D capabilities and differentiated pipeline planning.

Its DTcP for infants and young children has entered priority review and is expected to fill a domestic market gap. The Tdcp for people aged 6 years old and above has completed Phase III subject enrollment, enabling full life-cycle immunization coverage. The tetanus vaccine’s registration application has been accepted, and it is expected to offer superior safety and immunogenicity data compared with existing products. The Recombinant Poliomyelitis Vaccine, funded by Gates Foundation, has initiated clinical trials in Indonesia and has also received domestic clinical trial approval. Globally innovative Protein Based Pneumococcal Vaccine (PBPV) and inhaled tuberculosis vaccines have entered clinical research and proof-of-concept validation.

The company has now built a rich pipeline covering multiple technology platforms and full life-cycle segments, including meningococcal vaccines, pneumococcal vaccines, diphtheria tetanus pertussis vaccine, polio vaccines, tuberculosis vaccines, and zoster vaccine. Several major products are entering critical stages of registration review or clinical trials. Meanwhile, CanSinoBIO’s continuous breakthroughs in key technology platforms support a strategic shift from reliance on single products to sustained pipeline output.

Overall, in the first half of 2025, CanSinoBIO maintained the strong growth momentum seen since 2024, proving that its closed-loop capabilities from product R&D to commercialization have become increasingly mature. Against the backdrop of ongoing national policy support for innovative vaccines and domestic high-end vaccine substitution, CanSinoBIO—leveraging its solid R&D foundation, differentiated pipeline layout, and efficient commercialization execution—is accelerating the realization of its long-term growth potential and moving steadily toward becoming a global leader in innovative vaccines.

NMPA Accepted Essex’s Biologics License Application for EB12-20145P (HLX04-O) for the Treatment of Wet Age-Related Macular Degeneration

Essex Bio-Technology Limited (Essex or the Group, HKG: 1061) is pleased to announce that a Biologics License Application (BLA) for EB12-20145P (HLX04-O), a recombinant anti-VEGF humanized monoclonal antibody injection, has recently been accepted by the Centre for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. The product is jointly developed by the Group and Shanghai Henlius Biotech, Inc. (Henlius, HKG: 2696) for the treatment of wet age-related macular degeneration (wet-AMD) in China.

The phase 3 clinical trial of EB12-20145P (HLX04-O) among Chinese patients (“AURA-1”) has successfully reached the primary endpoint in April this year. AURA-1 is a multi-centre, randomised, double-blind, active-controlled, and non-inferiority phase 3 clinical trial which aimed to compare the efficacy and safety of EB12-20145P (HLX04-O) with that of ranibizumab administered by intravitreal injection (“IVT”) in newly diagnosed wet-AMD patients.

In addition to AURA-1, the BLA of which has been validated by the NMPA, an international, multi-centre phase 3 clinical study of EB12-20145P (HLX04-O) in patients with wet-AMD is ongoing successively in several European countries, Australia, the United States, and China (“AURA-2”) with last patient last visit completed by January 2025. Moving forward, Essex will continue to strive for excellence by embracing innovation to develop first-in-class and best-in-class products, providing solutions for Tomorrow’s healthcare problems, Today.

About wet-AMD
Age-related macular degeneration (“AMD”) is one of the leading causes of visual impairment and blindness in the elderly worldwide [1]. According to the World Health Organization (WHO), about 30 million people have suffered from AMD globally, and about half a million people become blind due to AMD each year [2]. Wet age-related macular degeneration (“wet-AMD”) is characterised by the formation of subretinal choroidal neovascularization (CNV) and is responsible for approximately 90% of cases of AMD-related blindness. Due to an aging population, wet-AMD has become a serious social medical problem and indicated a huge burden of unmet need [3]. With the development of treatment for fundus diseases, anti-VEGF drugs are becoming the first-line therapy for the management of wet-AMD [4], and the efficacy and safety of vitreous injection of bevacizumab for wet-AMD have been verified in multiple clinical studies [5-11].

About Essex
Essex is a bio-pharmaceutical company that develops, manufactures, and commercialises genetically engineered therapeutic b-bFGF, with six commercialised biologics currently marketed in China. Additionally, the Company has a diverse portfolio of commercialised preservative-free unit-dose eye drops, Shilishun (Iodized Lecithin Capsules) and others, which are principally prescribed for wound healing and diseases in Ophthalmology and Dermatology.

These products are marketed and sold through approximately 14,000 hospitals, supported by the Company’s 44 regional offices in China. Leveraging its in-house R&D platform in growth factor and antibody technology, Essex maintains a robust pipeline of projects in various clinical stages, covering a wide range of fields and indications.

Reference
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[3] Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health. 2014;2(2): e106-116.
[4] Li X R, Liu J P. Recognition of anti-VEGF therapy base on the mechanism of VEGF in wet age-related macular degeneration[J]. Zhonghua Shiyan Yanke Zazhi/Chinese Journal of Experimental Ophthalmology, 2012, 30(4):289-292.
[5] Tufail A, Patel PJ, Egan C, Hykin P, da Cruz L, Gregor Z, Dowler J, Majid MA, Bailey C, Mohamed Q, Johnston R, Bunce C, Xing W; ABC Trial Investigators. Bevacizumab for neovascular age related macular degeneration (ABC Trial): multi-centre randomized double masked study. BMJ. 2010 Jun 9;340:c2459.
[6] Martin DF, Maguire MG, Ying GS, Grunwald JE, Fine SL, Jaffe GJ. Ranibizumab and bevacizumab for neovascular age-related macular degeneration. N Engl J Med. 2011 May 19;364(20):1897-908.
[7] Chakravarthy U, Harding SP, Rogers CA, Downes SM, Lotery AJ, Wordsworth S, Reeves BC. Ranibizumab versus bevacizumab to treat neovascular age-related macular degeneration: one-year findings from the IVAN randomized trial. Ophthalmology. 2012 Jul;119(7):1399-411.
[8] Kodjikian L, Souied EH, Mimoun G, Mauget-Faÿsse M, Behar -Cohen F, Decullier E, Huot L, Aulagner G; GEFAL Study Group. Ranibizumab versus Bevacizumab for Neovascular Age-related Macular Degeneration: Results from the GEFAL Noninferiority Randomized Trial. Ophthalmology. 2013 Nov;120(11):2300-9.
[9] Krebs I, Schmetterer L, Boltz A, Told R, Vécsei-Marlovits V, Egger S, Schönherr U, Haas A, Ansari-Shahrezaei S, Binder S; MANTA Research Group. A randomized double-masked trial comparing the visual outcome after treatment with ranibizumab or bevacizumab in patients with neovascular age-related macular degeneration. Br J Ophthalmol. 2013 Mar;97(3):266-71.
[10] Berg K, Pedersen TR, Sandvik L, Bragadóttir R. Comparison of ranibizumab and bevacizumab for neovascular age-related macular degeneration according to LUCAS treat-and-extend protocol. Ophthalmology. 2015 Jan;122(1):146-52.
[11] Schauwvlieghe AM, Dijkman G, Hooymans JM, Verbraak FD, Hoyng CB, Dijkgraaf MG, Peto T, Vingerling JR, Schlingemann RO. Comparing the Effectiveness of Bevacizumab to Ranibizumab in Patients with Exudative Age-Related Macular Degeneration. The BRAMD Study. PLoS One. 2016 May 20;11(5): e0153052.

CMS Collaborates with SGX to Explore New Paradigms for Industrial Globalization and Pharmaceutical Expansion Overseas across Emerging Markets

On July 15 2025, to mark the successful secondary listing of China Medical System Holdings Limited (“CMS” or the “Group”) on the Main Board of the Singapore Exchange (“SGX”), SGX and CMS co-hosted the “Singapore and Emerging Markets Pharmaceutical Industry Growth Forum & CMS SGX Secondary Listing Appreciation Dinner”. Held in a grand fashion, the event was held at the Group’s CDMO manufacturing facility, PharmaGend, which is located in Tuas, Singapore.

The event brought together about 150 representatives from local government agencies, multinational pharmaceutical companies, innovative biotech companies, leading investment institutions, and the KOLs in the pharmaceutical industry. Through a series of insightful keynote speeches and panel discussions, guests engaged in in-depth exchanges and shared ideas on various topics, such as the pharmaceutical industry’s development in Singapore and emerging markets across the Asia-Pacific region, the breakthroughs and overseas expansion of Chinese innovative drugs, the globalization strategies, commercialization pathways, as well as ecosystem collaboration of innovative pharmaceutical companies.

The forum began with opening remarks by Ms. Caihan Chia, Head of Greater China Capital Markets and Chief Representative of Beijing Representative Office at SGX, and Ms. Louise Ho, Assistant Vice President of Healthcare Division and China Desk at the Singapore Economic Development Board. These were followed by keynote addresses from Mr. Siang Sheng Foo, Head of Investment Banking at Singapore CGS International Securities, Mr. Shriharsha Sarkar, Partner for Asia Healthcare at L.E.K. Consulting, and Ms. Kah Yean Neo, Senior Director at Singapore’s Agency for Science, Technology and Research (A*STAR).

Ms. Caihan Chia stated that in recent years, SGX has become increasingly attractive to Chinese enterprises through policy refinements, including tax incentives, capital support from the secondary market, and streamlined regulatory procedures. The successful listing of CMS showcases the growing interest among Chinese companies in the Singapore market. As one of leading healthcare companies, CMS’s listing highlights the growing demand for medical innovation and medical service accessibility across Asia. With CMS seeking to expand its business in Southeast Asia, its listing on SGX will serve as a strategic springboard to connect with international investors and further reinforce Singapore’s role as a vital capital hub.

Emerging Markets: A New Growth Engine for the Global Pharmaceutical Industry

Emerging markets, such as Southeast Asia and the Middle East, are becoming new growth drivers for the global pharmaceutical industry. A combination of factors, including large populations, early signs of ageing, the rise of the middle class, growing health awareness, and the increasing burden of chronic diseases, is driving higher demand for medicines and improved accessibility. According to IQVIA, by 2028, the combined pharmaceutical market size of four major emerging regions – Asia-Pacific, India, Africa & the Middle East, and Latin America – is expected to reach USD 336 – 384 billion, comparable to the USD 410 billion market size projected for Western Europe.

Singapore possesses geographical and institutional advantages for accessing Southeast Asia, the Middle East, and other emerging markets. With its robust financial system, open and inclusive policy environment, and thriving pharmaceutical industry, Singapore is increasingly becoming a global hub for capital and innovation. It has also become the preferred location for regional headquarters for many Chinese enterprises expanding into Southeast Asia.

Seizing Opportunity: Strategic Pathways for Chinese Innovative Pharma to Expand into Emerging Markets

In Southeast Asia’s six major economies (SEA6), limited healthcare coverage means that out-of-pocket payments constitute the primary source of drug expenditure. While generics dominate, branded originator drugs continue to hold significant market share in private hospitals, retail pharmacies, and clinics. Patient demand for biologics and biosimilars continues to grow.

In terms of commercialization models, traditional distribution model, which relies on third-party logistics (3PL), is gradually giving way to models with stronger commercial capabilities and strategic licensing partnerships. To achieve sustainable success in Southeast Asia, pharmaceutical companies must build competitive product portfolios, leverage experienced local sales teams, and consider establishing localized manufacturing capabilities, widely seen as key strategic advantages.

The CMS’s Approach: Building Dual Hubs in China and Singapore to Drive End-to-End Innovation

With over 30 years of experience in the Chinese market, CMS has accumulated a differentiated product portfolio and mature commercialization capabilities. Today, the Group is expanding its strategic vision across the Asia-Pacific region, using China as a foundation and Singapore as its regional hub. Through an end-to-end value chain of “R&D–manufacturing–commercialization–investment”, CMS is driving innovation to deliver high-quality pharmaceutical products and services to patients worldwide.

Mr. Lam Kong, Chairman, Chief Executive and President of CMS, delivered a keynote speech titled “New CMS, New Ascent: Three Strategies to Drive the Second Growth Curve.” He shared that since launching its “New CMS” transformation strategy in 2018, the Group has propelled growth through three engines — product innovation, commercial transformation, and international expansion. This has enabled the Group’s transition from “China’s largest CSO” to “a pharmaceutical company in transformation,” and finally, to “an end-to-end innovative pharmaceutical enterprise”, with a sustainable second growth curve.

In product innovation, driven by a three-dimensional approach of “Licensing, Strategic partnerships, and in-house R&D”, the Group has built a pipeline of nearly 40 FIC/BIC innovative drugs, five of which have been approved in China and are in large-scale clinical use. In the area of commercialization, CMS remains focused on cardio-cerebrovascular, gastroenterology, ophthalmology, and skin health specialties, while enhancing anti-cyclical resilience through a diversified ecosystem of “New retail, E-commerce, and Consumer healthcare”. Its skin health subsidiary, Dermavon, has become a niche market leader in China and is now progressing toward a spin-off for an independent listing on the Hong Kong Stock Exchange. In the area of globalization, CMS is creating a dual-track model centred in China and Singapore, using a strategy of “bringing in” to accelerate overseas product launches in China, and a strategy of “moving outward” to establish an end-to-end presence in emerging markets. The successful listing on SGX will enhance its regional synergy and close the loop in the “R&D – Manufacturing – Commercialization – Investment” global value chain, unlocking growth from emerging markets and creating a multi-regional growth framework.

CMS formally launched its industrial globalization strategy in 2022. At this event, CMS’s international business clusters made their debut, showcasing its forward-looking, full industry chain layout and leadership in setting a new paradigm for Chinese pharmaceutical companies expanding overseas.

PharmaGend

Established in 2023, PharmaGend aims to become Southeast Asia’s largest and most reliable CMO/CDMO. It has a site spanning 30,000 square meters and is capable of manufacturing dosage forms such as tablets and capsules, which has been certified by the FDA and HSA, demonstrating its high-standard pharmaceutical manufacturing capabilities for global export. It has future plans to expand production lines for injections, ointments, and nasal sprays. 

Rxilient

Established in 2021, Rxilient operates by a professional and experienced localized team, and has fully established BD, registration, marketing, andcommercialization capabilities. Leveraging its unique local expertise and advantages, Rxilient can bring innovative drugs to emerging markets led by Southeast Asia and the Middle East. It has submitted marketing applications for nearly 20 drugs and medical devices across Southeast Asia, the Middle East, and regions such as Hong Kong, Macao, and Taiwan, covering the therapeutic areas of dermatology, ophthalmology, oncology, autoimmune, and central nervous system. As more drugs receive regulatory approval in these countries, Rxilient anticipates sustained and significant revenue growth.

CMS R&D

Established in Singapore in 2024, CMS R&D has been working on more than 10 early-stage innovative drug projects. Leveraging China’s mature early-stage R&D and clinical resources, it aims to synchronize China speed with global standards to advance more Chinese innovative drugs toward globalization.

HiGend

Established in 2025, HiGend is a global early-stage bio-pharma incubation platform which uses a “hub-and-spoke” model, integrating China’s innovation capabilities to accelerate global R&D and commercialization.

Subsequently, three panel discussions were held in succession, which facilitated an in-depth exchange between industry and capital, driving the forum to its climax. Distinguished guests from various parties freely shared insights on the continuous development and diversification of the pharmaceutical ecosystem, and jointly explored the future of pharmaceutical expansion into emerging markets.

Panel Discussion 1 – Challenges and Breakthroughs: The Enduring Power of Organizational and Strategic Long-Term Vision

The emerging markets of today share numerous similarities with China’s pharmaceutical landscape twenty years ago, which are currently experiencing a critical period of accelerated demand release for pharmaceuticals, constituting medium- to long-term structural opportunities. Undoubtedly, these markets are diverse and complex — each country has its own unique characteristics in terms of drug regulation, healthcare insurance mechanisms, and market acceptance. However, CMS’s core strength lies in its systematic commercialization capabilities, which it is now extending to emerging markets. CMS’s senior management team, alongside its business partners, jointly reviewed and discussed the key factors contributing to its commercial excellence, as well as the pathways driving the Group’s second growth curve.

The first panel discussion was moderated by Mr. Brian Yang, Vice President for Business Development at Rxilient. Participants included Mr. Karl Luschmann, Managing Director of Pharma Stulln GmbH, and Ms. Linlang Wang (formerly the first product manager of the Augentropfen Stulln Mono Eye Drops (“Stulln”) in China), General Manager of CMS’s ophthalmology business, CMS Vision, among others. Collectively, they reviewed the core strategies behind the rapid, year-on-year growth of Stulln in the Chinese market- a focus on clinical value and continuous innovation in commercialization models. CMS adhered to prioritizing clinical efficacy, amassing substantial evidence to demonstrate the clinical value of Stulln in treating asthenopia, and leveraging medical advancements to drive product commercialization. Meanwhile, CMS also continuously revamped its commercialization model by establishing a full-channel retail system that integrates in-hospital and out-of-hospital sales, developing an “online + offline” omnichannel marketing system, and adopting a diversified product portfolio strategy in consumer attributes. These concerted efforts facilitated the sustained, rapid growth and wide recognition of Stulln within the Chinese market.

Mr. Victor Yin, Country Manager of Incyte Bioscience China, Mr. Huang Anjun, CEO of Dermavon (CMS’s skin health business), and Mr. Lawrence He, CEO of Rxilient, jointly retraced the entire journey of launching ruxolitinib cream — a blockbuster prescription drug with consumer attributes. From the signing of the collaboration agreement and pilot launch in Hainan Boao Lecheng pilot zone, to marketing approvals in Macao, followed by Hong Kong, introduction into designated hospitals in the Greater Bay Area in China, and the NDA has been submitted in China, Singapore, and other countries or regions. Leveraging mature clinical development experience and capabilities, proven commercialization competence, a compliant operational system, and efficient execution, the group earned high recognition and trust from Mr. Victor Yin.

Together, the parties have helped bring new hope to vitiligo patients in both China and Southeast Asia simultaneously.

These successful commercialization experiences not only provide valuable business model references for international pharmaceutical companies entering the Chinese market, but also offer significant insights for Chinese pharmaceutical companies looking to expand into emerging markets overseas.

Panel Discussion 2 – Breaking Through: Diverse Explorations for Chinese Innovative Pharmaceutical Companies to Expand into Emerging Markets

Over the past three years, Chinese pharmaceutical companies have secured over USD 10 billion in upfront payments through license-out deals. However, the majority of these transactions remain concentrated in mature markets led by Europe and the United States. Looking ahead, the next engine of growth may shift toward emerging markets such as Southeast Asia, the Middle East, and Latin America — regions with a combined population of approximately 1.8 billion and per capita healthcare spending is merely one-fifth that of Western markets. While pharmaceutical demand is accelerating in these areas, challenges persist, including limited payment capacity and significant differences in regulatory systems. Whether Chinese pharmaceutical companies can effectively replicate and localize their domestic development and innovation models in these blue ocean markets, will determine the scale and sustainability of their second growth curve.

The second panel discussion, moderated by Mr. Brian Yang, featured esteemed representatives from leading Chinese innovative pharmaceutical companies that are closely collaborating with CMS, including Tibet Nordicon Pharma, NeuroDawn Pharmaceutical (Ningdan Pharmaceutical), Mabgeek Biotech, and Jingze BioPharmaceutical. Using examples such as XinHuoSu (for acute decompensated heart failure), Y-3 for Injection (under development for stroke treatment), ABP-671 (under development for gout) etc., participants held in-depth discussions on topics, including “What constitutes truly clinically valuable innovation” and “How to implement commercialization pathways within emerging markets”

True innovation value stems from professionalism and dedicated focus. It requires researchers to remain committed to a specific field over the long term, and to validate new targets and drug structures through reverse translational research, thereby identifying their potential clinical value. Since China officially joined the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) in 2017 and became a member of its steering committee, the quality of Chinese innovative pharmaceutical products has improved rapidly and has gradually gained global recognition. In addition to entering mature markets led by Europe and the U.S., Chinese innovators are increasingly turning their attention to emerging blue ocean markets such as Southeast Asia, actively exploring parallel regulatory filings in both emerging and mature markets. For innovative pharmaceutical companies, globalization strategies should be integrated into the early stages of pipeline development cycles and macro-level strategy. It is also crucial to seek out a business partner that possesses the entire value-chain capabilities — including experienced local clinical registration teams, manufacturing capacity, and commercialization operations—in order to drive efficient product launches and expand access to high-quality medical solutions across a broader range of geographies.

Panel Discussion 3 – Setting Sail: Pharmaceutical Expansion into Emerging Markets

The third panel discussion was moderated by Mr. Frank Hong, Managing Director of Legend Capital, engaging multiple leading pharmaceutical analysts from renowned investment banks and representatives from investment institutions in an insightful sharing and in-depth discussion on the international expansion of innovative drug products and pharmaceutical industry globalization. Participants pointed out that China’s pharmaceutical industry is currently at a strategic inflection point for global expansion. The Intellectual Property (IP) licensing model has preliminarily demonstrated the global competitiveness of Chinese innovation. However, for most domestic pharmaceutical companies, this process remains in the “isolatedbreakthrough” phase. To achieve the transition from a practice of “one-time licensing” to “sustained global engagement,” Chinese pharmaceutical companies must look to multinational pharmaceutical giants as a benchmark — internationalizing their commercialization, manufacturing, and R&D capabilities to build a replicable, scalable, and sustainable global industrial ecosystem.

Achieving this goal is no easy task. While many Chinese pharmaceutical companies have begun exploring emerging markets, they often face challenges such as fragmented distribution channels and difficulty in standardizing operational systems. Only by maintaining conviction and building a fully integrated, internationalized ecosystem across the entire value chain can companies transform one-off licensing revenues into long-term brand equity and control of the value chain, ultimately earning a sustained voice and competitive edge in the global arena.

Though the forum’s spotlight has dimmed, the consensus reached continues to shine like a beacon: Southeast Asia, the Middle East, and other emerging markets are quickly becoming the next major destinations for the global pharmaceutical industry. CMS’s fully localized framework covering “Research, Manufacturing, Commercialization, and Investment” has paved the way for industrial expansion overseas, constructing a bridge to globalization. With an open and win-win attitude, the Group welcomes innovators, regulators, and capital from around the world to work together in bringing more Chinese and global innovative drugs to emerging markets, fostering international growth. CMS sincerely invites partners across all sectors to seize the growth opportunities of the Asia-Pacific region and jointly promote innovation in the pharmaceutical industry across emerging markets, so that more innovative therapies may benefit patients around the world.

Media Contact:

Company: China Medical System Holdings Ltd.
Contact: CMS Investor Relations
Email: ir@cms.net.cn
Website: https://web.cms.net.cn/en/home/

Source: China Medical System Holdings Ltd.

Silo Pharma Enters Letter of Intent for JV with Hoth Therapeutics to Develop Obesity Treatment Targeting $16 Billion Global Market

Silo Pharma, Inc. (Nasdaq: SILO) (“Silo” or the “Company”), a developmental stage biopharmaceutical company focused on novel therapeutics and drug delivery systems, today announced that it has entered into a non-binding letter of intent to form a strategic 50:50 joint venture with Hoth Therapeutics, Inc. (Nasdaq: HOTH) to develop and commercialize a potential treatment for obesity and metabolic disease based on technology Hoth has exclusively licensed from the U.S. Department of Veterans Affairs (VA) that was co-developed by the VA and Emory University.

The novel therapeutic platform centers on glial cell line-derived neurotrophic factor (GDNF), a VA-invented biologic with demonstrated anti-obesity effects and metabolic regulation in preclinical models. The platform is protected under U.S. Patent No. 10,052,362 and targets multiple high-burden conditions, including non-alcoholic fatty liver disease (NAFLD), type 2 diabetes, and central obesity.

“With obesity at epidemic levels and no curative therapies available, we believe the VA’s biologic GDNF is potentially a game-changer,” said Eric Weisblum, CEO of Silo Pharma. “We believe that our potential JV collaboration with Hoth aligns with our mission to accelerate groundbreaking science into human trials.”

“This VA-originated obesity technology has the potential to disrupt a $16 billion market and deliver life-changing impact for millions, including veterans disproportionately affected by metabolic disorders,” added Robb Knie, CEO of Hoth Therapeutics. “We are proud to partner with Silo Pharma in an effort to bring this innovation to the public.”

Deal Highlights:

  • Exclusive U.S. License from the U.S. Department of Veterans Affairs covering all fields of use 
  • 50/50 joint venture structure with equal equity and governance participation 
  • Lead indication: Obesity and NAFLD — conditions with prevalence among both civilian and veteran populations

Unmet Need:

Obesity affects over 40% of U.S. adults and is a leading risk factor for diabetes, cardiovascular disease, and liver failure. Veterans are especially impacted due to chronic inflammation, PTSD-linked metabolic disruption, and access challenges to effective care. There are no approved curative biologics targeting the neuroinflammatory root of obesity, a gap that this VA-originated technology is uniquely positioned to fill.

Strategic Alignment:

The partnership leverages:

  • The clinical infrastructure and public mission of the VA
  • Hoth’s regulatory and development expertise
  • Silo’s translational capabilities and capital efficiency

About Silo Pharma

Silo Pharma Inc. (Nasdaq: SILO) is a developmental stage biopharmaceutical company focused on addressing underserved conditions, including stress-induced psychiatric disorders, chronic pain, and central nervous system (CNS) diseases. The Company’s portfolio includes innovative programs such as SPC-15 for PTSD, SP-26 for fibromyalgia and chronic pain, and preclinical assets targeting Alzheimer’s disease and multiple sclerosis. Silo’s research is conducted in collaboration with leading universities and laboratories. silopharma.com

Forward-Looking Statements
This news release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. These statements are identified using words “could”, “believe”, “anticipate”, “intend”, “estimate”, “expect”, “may”, “continue”, “predict”, “potential”, and similar expressions that are intended to identify forward-looking statements. Such statements involve known and unknown risks, uncertainties, and other factors that could cause the actual results of Silo Pharma, Inc. (“Silo” or “the Company”) to differ materially from the results expressed or implied by such statements, including changes to anticipated sources of revenues, future economic and competitive conditions, difficulties in developing the Company’s technology platforms, retaining and expanding the Company’s customer base, fluctuations in consumer spending on the Company’s products and other factors. Accordingly, although the Company believes that the expectations reflected in such forward-looking statements are reasonable, there can be no assurance that such expectations will prove to be correct. The Company disclaims any obligations to publicly update or release any revisions to the forward-looking information contained in this press release, whether as a result of new information, future events, or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events except as required by law.

Source: SILO Pharma Inc.
Contact: 800-705-0120
investors@silopharma.com

CanSinoBIO Launches Pneumococcal Vaccine: An Innovative Choice to Safeguard Children’s Health

Pneumonia is a form of acute respiratory infection, that is common in all age groups and can cause mild to life-threatening illness. It is also the single largest infectious cause of death in children worldwide.

According to the World Health Organization(WHO), pneumonia killed more than 808,000 children under the age of 5 in 2017, accounting for 15% of all deaths of children under 5 years. Notably, in China, non-invasive pneumococcal diseases(NIPD), result in approximately 550,000 cases annually, leading to around 6,000 deaths. Meanwhile, invasive pneumococcal diseases (IPD) are more severe, with over 7,200 cases of pneumococcal meningitis reported each year. The average treatment cost per case of IPD is approximately CNY 64,000, and the case-fatality rate exceeds 10%. Behind these stark numbers lie the heavy burdens on countless families and an urgent public health challenge.

Facing this challenge, CanSino Biologics Inc. (Stock Code: 688185.SH, 6185.HK, “CanSinoBIO” or “the Company”) has developed and launched its 13-valent pneumococcal conjugate vaccine, trade name “iPneucia®”, optimized for China’s local pneumococcal epidemiology, aiming to provide more precise and safer protection for Chinese children.

The main advantage of iPneucia® is its deep optimization based on China’s local epidemiological characteristics. Streptococcus pneumoniae is categorized into different serotypes based on polysaccharide structure and biochemical traits. However, not all serotypes pose the same threat; their pathogenicity and disease burden vary. Research indicates that serotypes 19F, 19A, 7F, and 3 are the four most burdensome for Chinese children, accounting for over 60% of cases. These serotypes are also characterized by high resistance and high risk of sequelae. Given these risks, iPneucia® is specifically designed to target these four high-risk serotypes, and clinical data show that the geometric mean concentration (GMC) of antibodies against these four serotypes in iPneucia® is significantly higher than in the control group. This targeted protection strategy greatly enhances the precision of prevention, reducing disease risks at the source.

The breakthrough of iPneucia® lies not only in its enhanced targeting serotypes protection effect, but also in its carrier technology. It pioneers a dual-carrier conjugate process that adopts CRM197 and tetanus toxoid(TT) as carrier proteins. This approach effectively avoids immune interference caused by excessive use of a single carrier and reduces the risk of immune suppression when co-administered with other vaccines, enhancing immunogenicity. Moreover, iPneucia® also adopts a more advanced carrier protein conjugation process, ensuring consistent polysaccharide antigen content across all serotypes, significantly reducing competition between components.

Notably, the process of using CRM197 carrier as carrier protein in iPneucia® does not require formaldehyde for detoxification, which improves vaccine safety. This is also supported by data from Menhycia, another CRM197-based vaccine developed by CanSinoBIO. Post-marketing surveillance data on adverse events following immunization (AEFI) for recipients of the Menhycia show a apparently decline in the incidence of AEFI, fever, and local induration..

In summary, iPneucia® offers precise protection by targeting key serotypes and innovative protein carrier technology, resulting in enhanced effectiveness while minimizing the risk of adverse reaction. It also utilize a mature production process that excludes phenol, further improving its safety profile. CanSinoBIO presents a superior option for preventing pneumococcal diseases in Chinese children.

As a leader in innovative vaccines, CanSinoBIO always dedicated to scientific research and exploration in vaccine development. The company strives to create innovative, high-quality, and affordable vaccines. CanSinoBIO is committed to tackling global health challenges and improving the health and well-being of children around the world.

CanSinoBIO, (688185.SH)(06185.HK), https://www.cansinobio.com

Southeast Asian stem cell leader Medeze launches voluntary conditional cash partial offer for 10% stake in SGX-listed Cordlife Group Limited

Thailand-listed Medeze Group Public Company Limited (“Medeze”, together with its subsidiaries, the “Medeze Group”), through its wholly owned subsidiary Medeze Treasury Pte. Ltd. (the “Offeror”), intends to make a voluntary conditional cash partial offer (the “Partial Offer”) to acquire 10.00% of the issued ordinary shares of Cordlife Group Limited (“Cordlife” or the “Offeree”), a Singapore-listed provider of cord blood banking services. The move signals Medeze’s first strategic step into the Singapore market, with the intention of exploring business opportunities with the Offeree to create long-term value and mutual benefit for both parties.

Terms of the Partial Offer

Under the terms of the Partial Offer, the Offeror is seeking to acquire 25,630,774 shares, equivalent to 10.00% of Cordlife’s total issued and paid-up ordinary shares (excluding treasury shares) (the “Shares”), at an offer price of S$0.25 per share (the “Offer Price”). This represents an attractive premium of approximately 61.3% to the last traded price of S$0.155 on 9 May 2025, and also the 12-month volume-weighted average price.

The Partial Offer will be subject to the minimum acceptance condition of the Offeror having received, by the close of the Partial Offer, valid acceptances and/or tenders in respect of not less than 25,630,774 Shares, representing 10.00% of the total number of Shares (excluding treasury shares) as at the Record Date. The Partial Offer will not become or be capable of being declared unconditional in all respects unless the above condition is satisfied.

The Partial Offer represents an attractive opportunity for Cordlife shareholders to realise part or potentially all of their investment. Based on the Offeror’s observation that the Shares of the Offeree has been thinly traded in recent years, the Offer Price reflects a healthy premium over recent trading levels.

Rationale of the Partial Offer

The Partial Offer, if successful, will allow the Offeror to increase its direct holdings (including the existing shares held by the Offeror) in the Offeree to approximately 10.68% of the total number of Shares (excluding treasury shares) as at the Record Date. Upon successful completion of the Partial Offer, the Offeror, together with Medeze, will communicate with and endeavour to explore business opportunities with the Offeree to create long-term value and mutual benefit for both parties.

The Offeror wishes to demonstrate its commitment to contribute positively to the Offeree as a long-term substantial shareholder. By acquiring a meaningful stake, the Offeror aims to collaborate with existing controlling shareholders and management to drive sustainable value creation for all stakeholders. The Offeror believes in engaging in constructive dialogue and partnership with existing shareholders and management to align on strategic priorities, governance, and operational enhancements that will benefit the Offeree’s long-term success.

If a collaboration eventuates, Medeze Group and Cordlife could provide services to each other’s customers which helps to strengthen the market position and network of Medeze Group and Cordlife.  As an example and for illustrative purposes only, Medeze Group could (where permissible), offer services like analyzing and storing NK cell and hair follicle banking services to Cordlife’s customers. Cordlife (where permissible) could on the other hand offer its chromosomal and genetic analysis and screening services to Medeze Group’s customers. This collaboration can further support the growth of both Medeze Group and Cordlife at the international level.

The Medeze Group recorded total revenue of THB 874.3 million and net profit of THB 338.7 million, representing growth of 23.6% and 41.4% year-on-year respectively. Medeze Group’s asset base more than tripled to over THB 3.4 billion, following its successful listing of the Stock Exchange of Thailand and strategic reinvestment into technology and capacity.

About Medeze

The Medeze Group has been in business for more than 14 years and is a leader in the Southeast Asia stem cell storage and services market.

Recognised as the Southeast Asia Stem Cell Banking Company of the Year by Frost & Sullivan for two consecutive years (2023 and 2024), the Medeze Group has a laboratory that is accredited by the Association for the Advancement of Blood and Biotherapies (AABB) from the United States. The laboratory is equipped with modern equipment and technology in the stem cell banking industry, such as AutoXpress and Quantum Machine.

The Medeze Group is founded and headed by the incumbent chief executive officer, Dr. Veerapol Khemarangsan, an experienced Obstetrics and Gynecology specialist, who brings deep clinical and commercial expertise. He continues to be personally involved in stem cell procedures, lending Medeze Group a rare combination of medical and operational leadership. He currently serves as the Director of Training Committee in Bangkok Metropolitan Endoscopic Center (BMEC).

For more information of the Medeze Group, please: https://www.medezegroup.com/th/.

About Cordlife

Cordlife was incorporated in Singapore on 2 May 2001 and has been listed on the Mainboard of the Singapore Exchange Securities Trading Limited (“SGX-ST”) since 29 March 2012. The Offeree is a group company in the business of providing cord blood banking services such as the collection, processing, testing, cryopreservation and storage of umbilical cord blood at birth in countries including Singapore, Hong Kong, Macau, Indonesia, Thailand, India and the Philippines.

For media queries, please contact:

Thailand
Investor Relations
Medeze Group
DID: +66 91 599 9999 (press 2)
Email: ir@medezegroup.com

Singapore
Tevinder Singh
Associate
Financial PR
DID: +65 6438 2990
Email: tevinder@financialpr.com.sg 

The directors of the Offeror (including any director who may have delegated detailed supervision of the preparation of this document) have taken all reasonable care to ensure that the facts stated and opinions expressed in this document are fair and accurate and that no material facts have been omitted from this document, and they jointly and severally accept responsibility accordingly.

Where any information has been extracted or reproduced from published or otherwise publicly available sources or obtained from the Offeree, the sole responsibility of the directors of the Offeror has been to ensure through reasonable enquiries that such information is accurately extracted from such sources or, as the case may be, reflected or reproduced in this document.

HighTide Therapeutics to Showcase New Analyses of Phase 2 MASH/T2DM Studies of Berberine Ursodeoxycholate (HTD1801) and Pre-Clinical Results of Rimtoregtide (HTD4010) in Presentations at EASL Congress 2025

HighTide Therapeutics, Inc. (HKG: 2511), a clinical stage biopharmaceutical company specializing in the development of multifunctional multi-targeted therapies for chronic liver and metabolic diseases, announced today that it will present at the EASL Congress 2025, taking place from May 7-10, 2025 in Amsterdam. The presentations include post-hoc analyses of two Phase 2 clinical studies of berberine ursodeoxycholate (HTD1801), a gut-liver anti-inflammatory metabolic modulator, being developed for treatment of metabolic dysfunction-associated steatohepatitis (MASH) and type 2 diabetes mellitus (T2DM). A third presentation will present preclinical results for rimtoregtide (HTD4010), a peptide derived from the Reg3a protein, in liver failure in mice.

“Effects of Berberine Ursodeoxycholate (HTD1801) in Patients with At-risk MASH and T2DM”
(Presentation SAT-440, Poster Presentation, May 10, 8:30 AM CET)

About the Abstract: Due to the ongoing unmet medical need, clinical development in MASH focuses on patients who are at a higher risk of disease progression and outcomes due to the presence of moderate to advanced fibrosis (defined as at-risk MASH). The purpose of this analysis was to assess the effects of HTD1801 in patients with at-risk MASH and T2DM as defined by baseline MRI cT1 >875 ms. Eighteen weeks of treatment with HTD1801 resulted in substantial improvements in key hepatic and cardiometabolic parameters in patients with at-risk MASH and compared to placebo, twice as many patients achieved a reduction in liver fat content (MRI-PDFF) or fibroinflammation (cT1) that have been associated with improvements in liver histology. These data are particularly insightful as HTD1801 continues to be evaluated in an ongoing paired biopsy study of patients with at-risk MASH and pre-diabetes or diabetes.

“Effects of Berberine Ursodeoxycholate (HTD1801) in Chinese Patients with T2DM and Presumed MASLD”
(Presentation SAT-432, Poster Presentation, May 10, 8:30 AM CET)

About the Abstract: T2DM typically coexists with other metabolic abnormalities such as hyperlipidemia, obesity, and MASH that can exacerbate T2DM and can lead to a worse prognosis with increased risk for mortality and cardiovascular outcomes. In a Phase 2 study in patients with T2DM, HTD1801 achieved the primary endpoint with a significant decrease in HbA1c. Based on the latest diagnostic criteria, it is likely that a substantial subgroup of the study may have had concurrent metabolic dysfunction-associated steatotic liver disease (MASLD). The purpose of this analysis was to evaluate the benefits of HTD1801 in patients with T2DM and MASLD identified by baseline controlled attenuation parameter values >288 dB/M (correlated to 5% liver fat content). HTD1801 treatment demonstrated both dose-dependent improvements in cardiometabolic and hepatic parameters in patients with T2DM and MASLD. These data suggest HTD1801 can comprehensively address metabolic and cardiovascular risk factors beyond glycemic control.

“A Comparison of the Protective Effects of Rimtoregtide (HTD4010) and DUR-928 on Acute Liver Failure in Mice”
(Presentation FRI-141, Poster Presentation, May 9, 8:30 AM CET)

About the Abstract: The purpose of this study was 1) to test the potential protective effects of HTD4010 in an LPS-induced model mimicking acute liver failure in mice and 2) compare these effects to DUR-928, which is currently in late-stage development for the treatment of alcohol-associated hepatitis. In an LPS-induced mouse model mimicking acute liver failure, HTD4010 resulted in significant improvement in survival rates (greater than 2-fold) compared to the model control.  These protective effects of HTD4010 were significantly greater than DUR-928.  These findings provide evidence that HTD4010 may have a beneficial effect on acute liver conditions including alcohol-associated hepatitis and other acute-inflammatory-related conditions.

About Berberine Ursodeoxycholate
Berberine ursodeoxycholate (HTD1801) is an orally delivered, gut-liver anti-inflammatory metabolic modulator being developed for the treatment of metabolic and digestive diseases. HTD1801, an ionic salt of berberine and ursodeoxycholate, is a new molecular entity with a unique dual mechanism of action: AMP kinase activation and NLRP3 inflammasome inhibition. These two key mechanistic pathways have been associated with improvements in insulin resistance, glucose metabolism, lipid metabolism, and hepatic inflammation, potentially providing a comprehensive treatment platform for the multifaceted nature of complex metabolic diseases. HTD1801 is being developed for multiple indications. HTD1801 met the primary endpoint in two Phase 3 clinical trials in patients with type 2 diabetes mellitus (T2DM), demonstrating a clinically meaningful effect on HbA1c. In both trials, key secondary endpoints were achieved, suggesting multiple advantages of HTD1801 including improvement in cardiometabolic risk indicators. In addition to T2DM, HTD1801 efficacy in treating metabolic dysfunction-associated steatohepatitis (MASH) has been demonstrated in a Phase 2a clinical trial, and a global multicenter Phase 2b trial assessing the histologic benefit of HTD1801 is currently ongoing, with topline results expected in 2025.

About Rimtoregtide
Rimtoregtide (HTD4010) is a clinical-stage compound in development for acute inflammatory-related indications including alcoholic hepatitis (AH). It is a peptide derived from the Reg3a protein with immunomodulatory, anti-inflammatory, and anti-apoptotic effects. HTD4010 has been evaluated in animal models of acute pancreatitis and acute liver failure, where protective effects were observed. A completed Phase 1 clinical trial of HTD4010 in healthy subjects demonstrated a favorable safety profile. AH is caused by chronic heavy alcohol abuse or a sudden, drastic increase in alcohol consumption. It is characterized by severe inflammation and, ultimately, liver failure and death. There is currently no approved treatment for AH and only a few drug candidates are in clinical development. The current standard of care focuses on symptom management, including abstinence, treating inflammation and providing nutrition.

About HighTide Therapeutics
HighTide Therapeutics, Inc. (HKG: 2511) is a globally integrated biopharmaceutical company focusing on the discovery and development of first-in-class multifunctional, multi-targeted therapies with poly-indication potential across metabolic diseases with significant unmet medical needs. HighTide is currently developing several clinical assets and associated global intellectual property rights, and advancing multiple mid-to-late-stage clinical trials including therapies for metabolic dysfunction-associated steatohepatitis (MASH), type 2 diabetes mellitus (T2DM), severe hypertriglyceridemia (SHTG) and primary sclerosing cholangitis (PSC). Berberine ursodeoxycholate (HTD1801), HighTide’s lead drug candidate, received Fast Track designation from the United States Food and Drug Administration for both MASH and PSC and Orphan Drug designation for PSC. HTD1801 has been included in the National Major New Drug Innovation Program under the 13th Five-Year Plan for Major Technology Project in China.

For more information, please visit www.hightidetx.com
Contact: pr@hightidetx.com

Berberine Ursodeoxycholate (HTD1801) Consistently Demonstrates Comprehensive Benefits for Patients with Type 2 Diabetes Mellitus, Approaching NDA

  • HighTide Therapeutics Announces HTD1801 Meets the Primary Endpoints in Two Phase 3 Clinical Trials in Patients with Type 2 Diabetes Mellitus

HighTide Therapeutics, Inc. (HKG: 2511), a clinical-stage biopharmaceutical company specializing in the development of multifunctional, multi-targeted therapies for chronic liver and metabolic diseases, today announced that two Phase 3 trials (SYMPHONY 1 and SYMPHONY 2) of berberine ursodeoxycholate (HTD1801) in Chinese patients with type 2 diabetes mellitus (T2DM) met their primary endpoints and gated secondary endpoints.

The results of these two Phase 3 clinical trials provide robust evidence that HTD1801 delivers comprehensive benefits for patients with T2DM. Based on these highly positive read-outs, HighTide plans to submit a new drug application (NDA) for HTD1801 as a treatment for T2DM to the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) later this year.

SYMPHONY 1 (NCT06350890) and SYMPHONY 2 (NCT06353347) are randomized, double-blind, placebo-controlled, Phase 3 clinical trials designed to evaluate the efficacy and safety of HTD1801 in adults with T2DM and inadequate glycemic control despite using diet and exercise (SYMPHONY 1; N=407) or Metformin (SYMPHONY 2; N=549). The primary endpoint in both studies was the change in HbA1c from baseline with HTD1801 compared to placebo after 24 weeks of treatment. Gated secondary endpoints included the percentage of subjects achieving HbA1c <7.0%, change in fasting plasma glucose (FPG), low-density lipoprotein cholesterol (LDL-C), glutamyl transpeptidase (GGT), and high-sensitivity C-reactive protein (hs-CRP).

The primary endpoint was achieved in both trials, showing a clinically meaningful, consistent glucose-lowering effect of HTD1801
SYMPHONY 1 (HTD1801 as monotherapy): At week 24, the reduction from baseline in HbA1c with HTD1801 (-1.3%) was superior to placebo. Further, those with more severe disease had a greater decrease with HTD1801: reduction in HbA1c was -1.5% for those with a baseline HbA1c ≥8.5%.

SYMPHONY 2 (HTD1801 as an add-on therapy to Metformin): At week 24, the reduction from baseline in HbA1c with HTD1801 (-1.2%) was superior to placebo. Further, those with more severe disease had a more significant decrease with HTD1801: reduction in HbA1c was -1.6% for those with a baseline HbA1c ≥8.5%.

In both Phase 3 trials, the efficacy on HbA1c reduction in patients treated with HTD1801 was sustained through week 24.

In both trials, gated secondary endpoints were achieved, suggesting multiple advantages of HTD1801 beyond glucose-lowering including improvement in cardiometabolic risk indicators
At week 24, in both studies, the proportion of patients who achieved HbA1c <7.0% was significantly higher in the HTD1801 treatment groups compared to placebo. Improvements in HbA1c with HTD1801 were parallelled with significant improvements in postprandial and fasting plasma glucose compared with placebo. In addition, HTD1801 demonstrated lipid-lowering effects, including significant reductions in low-density lipoprotein cholesterol (LDL-C) and non-high-density lipoprotein cholesterol (non-HDL-C). Moreover, HTD1801 treatment led to reductions in key inflammatory biomarkers – glutamyl transpeptidase (GGT) and high-sensitivity C-reactive protein (hs-CRP) – both of which are associated with cardiovascular risk in patients with T2DM.

Favorable safety and tolerability profile
Overall, safety and tolerability were favorable and consistent with previous clinical trials of HTD1801. The most commonly reported adverse events were gastrointestinal. In both studies <2% of patients discontinued early due to an adverse event. The incidence of hypoglycemia was low, with no severe hypoglycemia events reported.

“The encouraging results from these studies suggest that HTD1801 may offer a novel and improved therapeutic option for patients with T2DM,” said Dr. Linong Ji, the leading principal investigator for the two Phase 3 clinical trials and former Vice President of the International Diabetes Federation (IDF), Director of the Peking University Diabetes Center and Director of the Department of Endocrinology and Metabolism, Peking University People’s Hospital. “As an innovative drug candidate, HTD1801 exhibits a unique dual mechanism of action – AMP kinase activation and NLRP3 inflammasome inhibition – that is distinct from any existing T2DM drugs on the market. It is an oral therapy designed to deliver comprehensive clinical benefits – not only lowering blood glucose but also improving lipid metabolism and exerting anti-inflammatory effects – thereby potentially reducing diabetes-related complications and addressing significant unmet clinical needs. With continued clinical exploration, HTD1801 is expected to further expand its application to benefit patients globally. In today’s pharmaceutical landscape, investment has become heavily concentrated on GLP-1-based drug development. While GLP-1 therapies hold significant clinical value, they do not adequately address the full spectrum of pathophysiological mechanisms underlying T2DM. To truly meet the multifaceted needs of T2DM management, continued innovation across a diverse range of therapeutic targets remains essential.”

“We extend our deepest gratitude to the patients who participated in these pivotal trials,” said Dr. Liping Liu, HighTide’s founder and CEO. “HTD1801’s innovative dual-action approach – targeting both metabolic regulation and inhibiting inflammation – represents a potential breakthrough in diabetes treatment. We look forward to sharing data from the 28-week open-label extension of these studies and a Phase 3 head-to-head comparison with the dapagliflozin; we will continue to explore the clinical potential of HTD1801 to provide patients with chronic metabolic diseases a comprehensive treatment solution.”

About Type 2 Diabetes Mellitus (T2DM)
According to the International Diabetes Federation (IDF), 537 million adults (ages 20-79) were living with diabetes in 2021, and this number is projected to grow to 783 million (representing 1 in 8 adults) by 2045, of these, around 90% are T2DM cases. China has the largest population of diabetes patients worldwide, estimated to be 141 million in 2021, and projected to grow to 174 million in 2045. Diabetes is a global societal burden leading to over 6 million deaths per year. To address this urgent challenge, there is a critical need for innovative therapies that can deliver comprehensive clinical benefits for patients worldwide.

About Berberine Ursodeoxycholate (HTD1801)
Berberine ursodeoxycholate (HTD1801) is an orally delivered, gut-liver anti-inflammatory metabolic modulator being developed for the treatment of metabolic and digestive diseases. HTD1801, an ionic salt of berberine and ursodeoxycholate, is a new molecular entity with a unique dual mechanism of action: AMP kinase activation and NLRP3 inflammasome inhibition. These two key mechanistic pathways have been associated with improvements in insulin resistance, glucose metabolism, lipid metabolism, and hepatic inflammation, potentially providing a comprehensive treatment platform for the multifaceted nature of complex metabolic diseases.

HTD1801 is being developed for multiple indications. In addition to T2DM, its efficacy in treating metabolic dysfunction-associated steatohepatitis (MASH) has been demonstrated in a Phase 2a clinical trial and a global multicenter Phase 2b trial assessing the histologic benefit of HTD1801 is currently ongoing, with topline results expected in 2025.

About HighTide Therapeutics
HighTide Therapeutics, Inc. (HKG: 2511) is a globally integrated biopharmaceutical company focusing on the discovery and development of first-in-class multifunctional, multi-targeted therapies with poly-indication potential across chronic liver and metabolic diseases with significant unmet medical needs. HighTide is currently developing several clinical assets and associated global intellectual property rights, and advancing multiple mid-to-late-stage clinical trials including therapies for metabolic dysfunction-associated steatohepatitis (MASH), type 2 diabetes mellitus (T2DM), severe hypertriglyceridemia (SHTG) and primary sclerosing cholangitis (PSC). Berberine ursodeoxycholate (HTD1801), HighTide’s lead drug candidate, received Fast Track designation from the United States Food and Drug Administration for both MASH and PSC and Orphan Drug designation for PSC. HTD1801 has been included in the National Major New Drug Innovation Program under the 13th Five-Year Plan for Major Technology Project in China.

For more information, please visit www.hightidetx.com.
Contact: pr@hightidetx.com

HighTide Therapeutics Announces Publication of Phase 2 Study of Berberine Ursodeoxycholate for the Treatment of Type 2 Diabetes Mellitus in JAMA Network Open

– Data from the proof-of-concept study showed berberine ursodeoxycholate was generally well tolerated and had beneficial therapeutic effect in improving glycemic, hepatic and cardiometabolic parameters
– The multifaceted effects demonstrated by berberine ursodeoxycholate support this new molecular entity as a unique oral treatment option for T2DM and its comorbidities
– T2DM phase 3 data readout is expected to be announced in the first half of 2025

HighTide Therapeutics, Inc. (HKG: 2511), a clinical stage biopharmaceutical company specializing in the development of multifunctional multi-targeted therapies for chronic liver and metabolic diseases, today announced that data from the Phase 2 proof-of-concept study evaluating berberine ursodeoxycholate (HTD1801) for the treatment of type 2 diabetes mellitus (T2DM) was published online in JAMA Network Open. 

The publication reports results of the randomized, placebo-controlled 12-week study to assess efficacy, safety and tolerability of HTD1801 compared to placebo in patients with T2DM. The trial achieved its primary endpoint by demonstrating a statistically significant and superior reduction in HbA1c at week 12 with HTD1801 versus placebo. A majority of patients treated with HTD1801 achieved target HbA1c <7%. Further, results show therapeutic benefit of HTD1801 on LDL and total cholesterol, markers of liver injury and systemic inflammation. In this study, HTD1801 was well tolerated with no treatment-related serious adverse events observed. These findings are being confirmed in ongoing Phase 3 studies.

“We are encouraged by the results of this Phase 2 study. The study provides evidence that HTD1801 can treat the core aspects of metabolic syndrome, with potential to provide a spectrum of therapeutic effects that appear to address comorbid conditions that exacerbate disease and worsen prognosis of patients with T2DM,” said Dr. Linong Ji, Professor of Medicine at Peking University, Director of Peking University Diabetes Center and Director of the Department of Endocrinology and Metabolism, Peking University People’s Hospital, in Beijing, China.

“Based on these positive findings, we are continuing to advance the clinical development of HTD1801 in our ongoing Phase 3 T2DM program with topline results expected in the first half of this year,” said Dr. Liping Liu, CEO of HighTide Therapeutics.

About JAMA Network Open Journal
JAMA Network Open is an international, peer-reviewed, open access, general medical journal that publishes research on clinical care, innovation in health care, health policy, and global health across all health disciplines and countries for clinicians, investigators, and policy makers. JAMA Network Open is a member of the JAMA Network, a consortium of peer-reviewed, general medical and specialty publications.

About Berberine Ursodeoxycholate (HTD1801)
Berberine ursodeoxycholate (HTD1801) is an orally delivered, gut-liver anti-inflammatory metabolic modulator being developed for the treatment of metabolic and digestive diseases. HTD1801, an ionic salt of berberine and ursodeoxycholate, is a new molecular entity with unique dual mechanisms of action, including AMP kinase activation and NLRP3 inflammasome inhibition. These two key mechanistic pathways have been associated with improvements in glucose metabolism, insulin resistance, lipid metabolism, and hepatic inflammation, potentially providing a comprehensive treatment platform for the multifaceted nature of complex metabolic diseases such as T2DM.

About HighTide Therapeutics
HighTide Therapeutics, Inc. (HKG: 2511) is a globally integrated biopharmaceutical company focusing on the discovery and development of first-in-class multifunctional multi-targeted therapies with poly-indication potential across multiple metabolic and digestive diseases with significant unmet medical needs. The Company is currently developing several clinical assets and holding global intellectual property rights, advancing multiple mid-to-late-stage clinical trials including therapy for metabolic dysfunction-associated steatohepatitis (MASH), type 2 diabetes mellitus (T2DM), severe hypertriglyceridemia (SHTG) and primary sclerosing cholangitis (PSC). Berberine ursodeoxycholate (HTD1801), the Company’s lead drug candidate, received Fast Track designation from the United States Food and Drug Administration for both MASH and PSC and Orphan Drug designation for PSC. HTD1801 has been included in the National Major New Drug Innovation Program under the 13th Five-Year Plan for Major Technology Project in China.

For more information, please visit www.hightidetx.com

Contact: pr@hightidetx.com