• Octapharma to present new clinical and scientific findings for wilate^® and Nuwiq^® at the upcoming ISTH 2024 Congress
• New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia

LACHEN, Switzerland, June 10, 2024 /PRNewswire/ — The most recent developments from Octapharma’s Haematology portfolio will be presented at the 32^nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care.

Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients’ quality of life and mental health.

The presentations at ISTH 2024 reflect Octapharma’s long-term commitment to improving patient care and addressing unmet clinical needs.

“Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024.” said Olaf Walter, Board Member at Octapharma. “Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients’ lives.”

Oral Presentations

Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate^®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population.

MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study’s coordinating investigators, will present an interim analysis of the study. 

Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial.

Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq^® can bind to endothelial cell surfaces, improving their functionality.

FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq^® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A.

The oral presentations are scheduled at the following times:

Sunday June 23

• OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality.
  Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D

Monday June 24

• OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability.
  Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C
• OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A.
  Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C
• OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability.
  Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C

Tuesday June 25

• OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study.
  Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3
• OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study.
  Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1

Wednesday June 26

• OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study.
  Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4

Poster Presentations 

Poster presentations are scheduled at the following times:

Sunday June 23

• PB 0251 – Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States.
  Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall

Tuesday June 25

• PB 1102 – Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function.
  Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall

Supported Symposia 

Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD.  

Octapharma’s Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A.

This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced.

Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress.  An opportunity to ask questions will be available at both symposia. 

Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented:

“We are excited to present new insights from the WIL-31 data, and  will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A.”

About Nuwiq®

Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein¹. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor¹.

Nuwiq® treatment has been assessed in nine^{1 2 3} completed clinical trials which included 201 previously treated patients (190 individuals)¹ and 108 previously untreated patients² with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations⁴. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups⁴.

About wilate®

wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production¹.

No albumin is added as a stabiliser⁵. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma¹. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma¹. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres⁶. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)⁶.

About Octapharma

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care.

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US.

“Octapharma press releases are specifically for health specialist/medical media and are not for consumer press.“

[1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471.
[2] Liesner RJ et al. Thromb Haemost 2021; 121:1400–8.
[3] Octapharma AG, data on file.
[4] Nuwiq® Summary of Product Characteristics
[5] Stadler M et al. Biologicals 2006; 34:281-8.
[6] wilate® Summary of Product Characteristics.

Source

• FDA’s Fast Track designation speeds up the development of new biologic drugs for rare diseases

YONGIN, South Korea, June 10, 2024 /PRNewswire/ — GC Biopharma (006280. KS) and Novel Pharma have announced that the U.S. FDA has granted Fast Track Designation for their jointly developed MPSIIIA (Sanfilippo syndrome Type A) treatment, GC1130A.

This designation follows the FDA’s clearance of the investigational new drug (IND) application for GC1130A last month. With Fast Track Designation, the development process for GC1130A is set to be expedited.

About Sanfilippo Syndrome Type A

Sanfilippo syndrome Type A is a rare genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients face life-threatening complications by the age of 15. GC1130A represents a potential breakthrough as the first and only treatment option specifically targeting MPSIIIA.

GC1130A

GC1130A is a biological drug that is being developed using high-concentration protein formulation technology, designed for administration to the central nervous system. It is delivered directly into the brain’s ventricles through intracerebroventricular (ICV) injection, a method first applied globally by GC Biopharma’s Hunter syndrome treatment, ‘Hunterase’, which has received market approval in Japan.

The potential of GC1130A to meet the unmet medical needs of Sanfilippo syndrome has been recognized by major drug regulatory agencies; in 2023, the FDA granted GC1130A Rare Pediatric Disease Designation (RPDD) and Orphan Drug Disease (ODD), and earlier this year, the European Medicines Agency (EMA) also granted GC1130A ODD status.

Currently, GC Biopharma and Novel Pharma are preparing to initiate a multinational first-in-human clinical trial to evaluate the safety and tolerability of GC1130A in Korea, the US, and Japan.

FDA’s Fast Track Program

U.S. FDA’s Fast Track program is designed to expedite the development and review of drugs intended to treat serious or unmet medical needs. Fast Track designation provides extensive support, including frequent meetings with the FDA throughout the drug development, clinical, and approval stages.

GC Biopharma and Novel Pharma stated, “We are pleased with the FDA’s decision to grant Fast Track designation for GC1130A, especially given the lack of approved treatments for Sanfilippo syndrome.” The company added, “This designation will enable us to accelerate the development of this new drug, bringing hope to patients and families affected by Sanfilippo syndrome.”

[Attachment] GC Biopharma’s Lysosomal Storage Disease research and development status

About GC Biopharma

GC Biopharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers lifesaving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century

About Novel Pharma Inc.

Novel Pharma is an emerging biotechnology company that focuses on developing first-in-class treatments for rare pediatric diseases (MPS/LSD) utilizing ICV (intracerebral ventricular) administration.  Headquartered in Seoul, South Korea, it is currently engaged in the development of (i) MPSIIIA, (ii) GM1, (iii) MPSIVB and (iv) Krabbe disease.    

This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma’s management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.

GC Corp. Contacts (Media)

Sohee Kim
shkim20@gccorp.com

Yelin Jun
yelin@gccorp.com

Rachel Kim
rachel.kim@gccorp.com 

Source

TAITUNG, June 10, 2024 /PRNewswire/ — Imagine yourself gazing at a breathtaking tapestry of stars, unmarred by light pollution. Welcome to Taitung, Taiwan’s ‘starry city,’ where nights come alive with celestial wonders. Nestled in the southeastern corner of Taiwan, Taitung is a treasure trove of natural wonders, rich landscapes, and diverse ecological habitats. The local Indigenous cultures add a layer of historical depth to its charm. With its commitment to maintaining low development and preserving its pristine air quality, Taitung has been dubbed “Taiwan’s most beautiful stargazing city.”

Since 2018, the Taitung County Government has been promoting the Starry Taitung series, which includes the Starry Taitung Night Concert and other events, all free to attend, nurturing sustainable tourism through expertly curated tours and awareness programs that showcase the magnificent night skies.

This year, from June to August, the Starry Taitung Night Concert will be the highlight of the series. The concerts offer a chance for visitors from around the globe to enjoy the star-filled skies above the region, whilst appreciating the diverse musical talents we have to offer. In 2021, the Starry Taitung initiative received ISO 20121 certification, recognizing its sustainable event management practices. The campaign is noted for its commitment to creating a tranquil atmosphere, minimizing light pollution, reducing single-use waste, and promoting low-carbon emissions. The series will take place in six locations across Taitung: Guanshan, Dawu, Donghe, Beinan, Lanyu, and Ludao townships, from 6 pm to 9 pm. Each event will feature a vibrant starlit bazaar, live music performances, and guided night tours, all offered free of charge.

Travelers who appreciate the tranquillity of rural landscapes should consider cycling through Chishang Township to Dapo Pond or Bolang Avenue, or exploring Guanshan Waterfront Park in Guanshan Township. Those who prefer panoramic sea views can visit the Jialulan Recreation Area along the coastal highway just north of Taitung City, the Sanxiantai Scenic Area in Chenggong Township, or the 9420 Coastal Park in Dawu Township, all which overlook the Pacific Ocean. Each of these sites also doubles as a perfect location for stargazing, offering clear views of the night sky.

For a more guided experience, Starry Taitung provides tours led by knowledgeable locals, focusing on the region’s evening skies. Visitors can find stargazing maps and event guides on Starry Taitung’s official website or Facebook page. Adding “Taitung County” and “Starry Taitung” to your Taiwan itinerary promises an enriching experience that combines the Taitung’s unique countryside, valleys, and serene coastal areas with the awe-inspiring beauty of its night skies.

For the latest updates on the event, please visit our official Facebook page (https://www.facebook.com/StarryTaitung).

Source

• Hotel de Crillon, A Rosewood Hotel, voted as the top luxury hotel in the world
• Asian attractions chosen as best things to do
• Hot springs and ski resorts globally are top-of-mind for travellers
• Singapore dominates the restaurant scene in Asia, with three-Michelin-star Odette as the best fine-dining restaurant in Asia

SHANGHAI, June 8, 2024 /PRNewswire/ — Leading online travel service provider Trip.com has released its 2024 Trip.Best Global and Asia 100 lists, a collection of curated travel rankings based on real user reviews and year-round sales popularity. These Trip.Best lists enable global travellers to select the best experiences a destination has to offer, including hotels and attractions, as well as lifestyle choices such as restaurants and nightlife.

Top Hotels, Attractions, Nightlife, Restaurants and Destinations Make the List

The Global and Asia lists comprise different categories:

• Hotels: 100 Luxury Hotels, 100 Family Hotels, 100 Cultural Hotels, 100 Scenic Hotels, 100 Villas, 100 Ski Hotels, 100 Instagrammable Hotels, 100 Hot Spring Hotels (Asia only)
• Attractions: 100 Best Things to Do, 100 Family-friendly Attractions, 50 Hot Springs, 50 Ski Resorts
• Nightlife: 100 Best Nightlife
• Destinations: 100 Best Destinations
• Restaurants (Asia only): 100 Fine Dining, 100 Local Restaurants, 100 Restaurants for Views & Experiences.

Paris took centre stage for the 2024 GLOBAL 100 Luxury Hotels list, with Hotel de Crillon, a Rosewood Hotel, taking the top spot. Le Bristol Paris – an Oetker Collection Hotel came in third while La Reserve Paris Hotel and Spa came in fifth. Raffles Singapore and Aman Tokyo came in second and fourth, respectively, to make up the top five luxury hotels globally.

In both lists, Asia offers a wealth of hotel recommendations for all types of travelers. Indonesia boasts luxury hotel Buahan, a Banyan Tree Escape, as well as AYANA Villas Bali, one of the best villas in the region. Families will love child-friendly hotels such as Hard Rock Hotel Desaru Coast in Malaysia and Vinpearl Resort & Spa Nha Trang Bay in Vietnam. Travellers seeking cultural experiences can consider Sawasdee Village in Thailand, while those looking for a more scenic holiday can choose to stay at The Bellevue Resort in the Philippines known for its white beach views, or Karuizawa Prince Hotel West with some skiing in the mix.

With social media as a means of travel inspiration these days, some of the top Instagrammable hotels include Mai House Saigon Hotel in Vietnam and Good Nature Hotel Kyoto in Japan.

Asian attractions also took all top five spots on the 2024 GLOBAL 100 Best Things to Do list, with a strong showing from Universal Studios and Disneyland theme parks – Universal Beijing Resort, Shanghai Disney Resort, Hong Kong Disneyland, Universal Studios Japan, Universal Studio Singapore and Universal Studios Hollywood. In addition to theme parks being a key attraction in these destinations, museums also made it into the top 10, specifically The Palace Museum Hong Kong, the Louvre Museum in Paris and The British Museum in London.

The 2024 GLOBAL 50 Hot Springs list features the best hot springs from around the world, including Iceland’s Blue Lagoon, Japan’s Arima Onsen and Yufuin Onsen, the Travertines of Pamukkale in Turkey, as well as Queenstown Angson Barrel Hot Spring in New Zealand.

Travellers seeking a night out have declared the Empire State Building as the quintessential spot to immerse in the dazzling lights of New York City after dark. Paris and London offer plenty of popular nightlife options, including the Bateaux Mouches Paris Seine river cruise, the Eiffel Tower, the London Eye and Tower Bridge, all of which were voted in the top 10 of the 2024 GLOBAL 100 Nightlife list. Dubai’s Burj Khalife and The View at the Palm topped the 2024 ASIA 100 Nightlife list, with Xi’an’s “The Song of Everlasting” show, the Xi’an City Wall and Tang Paradise capping off the top five.

The spotlight is on Singapore when it comes to Asia’s best restaurants, with its entries taking first place across all three categories. Three-Michelin-star Odette leads the ASIA 100 Fine Dining list, along with Palm Beach Seafood as the number one local restaurant and Spago Dining Room by Wolfgang Puck as the best restaurant for views and experiences. The lists are presented by Trip.Gourmet, which aims to showcase the best dining options and local food culture globally for travellers and food lovers.

“Adhering to the principle of selecting the best from the best, Trip.Gourmet aims to create themed rankings that cater to different travel scenarios with ingenuity, especially as food becomes an increasingly important part of travel plans,” shared Sunny Sun, Vice President of Trip.com Group and CEO of Trip.Gourmet.

Looking ahead to the winter season, Switzerland remains the top destination for skiing, with Zermatt Ski Resort crowned the most popular ski resort globally. Japan dominated the 2024 Asia 50 Ski Resorts, with Sapporo Kokusai Ski Resort, GALA Yuzawa Snow Resort, Furano Ski Resort and Niseko Tokyu Grand Hirafu taking up four of the top five spots.

The 2024 Trip.Best Global and Asia 100 lists are curated by examining various travel products on Trip.com and scoring them based on an algorithm. The rankings are based on real user reviews, year-round sales popularity, continuous quality assurance, and distinctive themes, covering worldwide and Asian hotels, attractions, night tours, food, and destinations. Trip.com selects hundreds of millions of data points and verifies word-of-mouth from people around the world, aiming to create fair, comprehensive, professional, and trustworthy travel rankings to solve users’ travel choice problems.

The full lists of rankings are available here.

Promoting Tourism with AI

The 2024 Trip.Best Global and Asia 100 lists highlight the best in various travel categories, which builds upon the AI-powered “Trip.Best” list on the app. “Trip.Best” is one example of the Group’s innovative use of AI, alongside other lists such as “Trip.Trends” and “Trip.Deals.”

• Trip.Best showcases top global destinations.
• Trip.Trends reveals the latest travel trends based on user searches across Trip.com, search engines, and social media.
• Trip.Deals brings the best promotions to travellers.

The lists were unveiled at the 2024 Hunan International Cultural Tourism Festival Gala Dinner and Trip.Best Global Award Ceremony in Zhangjiajie on May 31. The event, attended by over 900 guests, including industry partners and tourism bureaus from more than 50 countries, celebrated the launch of the Trip.Best Global and Asia 100 lists, as well as the 2024 Trip.Trends 100 list.

Trip.Trends tracks the hottest travel topics and events, offering a glimpse into future trends. The 2024 Trip.Trends 100 list features newly opened hotels and attractions from 2023, giving travellers fresh and trendy options.

These curated lists are part of Trip.com’s vibrant content marketing strategy, designed to drive consumer awareness and provide relevant information for securing the best offerings and deals. For instance, Trip.Best inspires travellers to book their favourite properties, boosting traffic and bookings for hotel partners. This initiative propels Trip.com’s globalisation strategy, forging powerful alliances with global partners to deliver unparalleled, seamless travel experiences.

About Trip.com

Trip.com is an international one-stop travel service provider, available in 24 languages across 39 countries and regions in 35 local currencies. Trip.com has an extensive hotel and flight network consisting of more than 1.7 million hotels and flights from over 600 airlines covering 3,400 airports in 220 countries and regions around the globe. Trip.com‘s world-class 24/7 multilingual customer service, as well as additional centres in Edinburgh, Tokyo and Seoul, help to ‘create the best travel experience’ for its millions of customers worldwide. To book your next trip, visit trip.com.

Source