CHA University

Mature results from Medivir's phase 1b / 2a open label trial of fostrox + Lenvima® confirm improved outcomes in second-line advanced liver cancer with a median time to progression (TTP) of 10.9 months1 (4.1 – 18.1). The results showed an objective response (ORR) of 24%, and a median duration of response of 7.0 months. Detailed safety update reinforces the ability to combine fostrox and Lenvima long-term, only 1 patient discontinuing fostrox due to adverse events. Medivir's fostrox (fostroxacitabine bralpamide) is the only orally-administered, liver-targeted inhibitor of DNA replication. Its unique mechanism for the treatment of liver cancer delivers the cell-killing compound selectively to tumor cells locally in the liver while minimizing harm to healthy cells. STOCKHOLM, Sept. 16, 2024 /PRNewswire/ -- Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, today presented positive, mature data from its ongoing phase 1b / 2a study of fostroxacitabine bralpamide (fostrox) + Lenvima® in advanced liver cancer (hepatocellular carcinoma/HCC) at the ESMO (European Society of Medical Oncology) Congress in Barcelona, Spain. Today's ESMO update, poster number 986P, presented by Dr Hong Jae Chon on Monday September 16, shows promising duration of benefit with 19% of patients continuing treatment for more than a year and the longest running patient remaining on treatment for over 2 years, with sustained partial response. The patients in the study had disease control on fostrox + Lenvima independent if they benefitted from previous line of therapy, showing potential for all second-line patients to benefit from the combination. The safety and tolerability profile continues to be encouraging with no unexpected adverse events. While hematological adverse events were common, they were temporary in nature. Decreases in neutrophil & platelet counts showed a cyclic pattern with recovery before next cycle of treatment, enabling patients to remain on treatment long-term. Importantly, no patient experienced febrile neutropenia or low platelet count with bleeding and there were no fostrox-related serious adverse events. Results come despite very poor prognosis for most second-line HCC patients today, with just 5–10% responding to current standard of care treatment, and a typical TTP of only 3–4 months. Dr. Pia Baumann, Chief Medical Officer at Medivir, said: - "With three patients still remaining on study treatment, all of whom treated for more than a year, this data-set is now quite mature. At a median follow-up of 10.5 months, fostrox + Lenvima have clearly shown promise of improved outcomes beyond current alternatives for second-line liver cancer patients. Fostrox is designed to only target tumor cells locally in the liver, without harming healthy cells. It is therefore reassuring to see the tolerability profile of fostrox enabling the combination of two highly potent treatments, fostrox + Lenvima, without compromising patient safety. Patients were able to stay on treatment long-term, which evidently contributes to the extended duration of benefit and a median time to progression of 10.9 months, substantially longer than previously seen in second-line liver cancer. It is with reinforced confidence we continue our preparations for the initiation of the planned phase 2b study comparing fostrox + Lenvima with Lenvima alone in a randomized setting to confirm the benefit of the combination." Dr Hong Jae Chon, Professor at CHA Bundang Hospital in Korea, and investigator in the fostrox + Lenvima study, commented: "Treatment outcomes have improved in first-line with the use of immunotherapy combinations, resulting in more patients fit enough to receive second-line treatment. But with no treatments approved in second-line after immunotherapy, there is a significant unmet medical need for new treatments options for these patients. The phase 1b/2a data for fostrox + Lenvima show highly encouraging clinical benefits for patients, indicating that when adding fostrox to Lenvima, efficacy is better than expected from Lenvima alone. It is especially encouraging that in addition to patients experiencing benefit for an extended period of time, patients also responded to the treatment independent of outcome in previous line of therapy. I look forward to evaluating the efficacy of fostrox plus Lenvima in a randomized, controlled trial." The data are from Medivir's ongoing phase 1b/2a open-label, multi-center, dose-escalation and dose-expansion study, evaluating the safety and efficacy of fostrox in combination with Lenvima in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable. HCC is the most common type of liver cancer, accounting for more than 80% of cases worldwide.2 There are approximately 660,000 patients diagnosed with HCC per year globally and current five-year survival is less than 20 percent3. Medivir will host a webcast where Dr Chon and Dr Pia Baumann will present the data and answer questions. The webcast will take place Today, September 16, at 13.45 CET, and will be streamed via a link on the website: . The poster and the presentation from the webcast will also be available on Medivir's website after the presentation. For additional information, please contact; Magnus Christensen, CFO, Medivir AB Telephone: +46 8 5468 3100 E-mail: [email protected] About fostrox Fostrox is a liver-targeted inhibitor of DNA replication that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. This design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver. With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing where it has shown encouraging anti-cancer efficacy with a good safety and tolerability profile. About primary liver cancer Primary liver cancer is the third leading cause of cancer-related deaths worldwide. Hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver and it is the fastest growing cancer in the USA. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent2,3. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need. About Medivir Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a drug candidate designed to selectively treat cancer cells in the liver and to minimize side effects. Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. . Data cut-off 19 August, 2024 Rumgay et al.,European Journal of Cancer 2022 vol.161, 108-118. Yang, J.D., Hainaut, P., Gores, G.J. et al. A global view of hepatocellular carcinoma: trends, risk, prevention and management. Nat Rev Gastroenterol Hepatol 16, 589–604 (2019). 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Collaboration Aims to Harness HiFi Sequencing to Improve Diagnosis and Treatment of Subfertility and Recurrent Pregnancy LossSINGAPORE, Sept. 11, 2024 (GLOBE NEWSWIRE) -- The HiFi Solves Sub-fertility Consortium, an innovative collaboration involving five leading centers across the Asia-Pacific region, announces work aimed at redefining the landscape of fertility research. Utilizing PacBio HiFi long-read sequencing, the consortium, led by KK Women's and Children's Hospital (KKH) in Singapore, is pioneering the use of PacBio HiFi long-read sequencing to enhance the diagnosis and treatment of subfertility and recurrent miscarriages (RPL). The HiFi Solves Sub-fertility Consortium integrates DNAstack’s federated data platform, the world’s first solution to enable the creation and analysis of global federated networks of data in compliance with industry standards set by the Global Alliance for Genomics & Health. This platform allows for seamless global collaboration on complex datasets without the need to move the data, ensuring sensitive genomic information remains secure while supporting efficient analysis and compliance with regional data governance. Under the leadership of Associate Professor Saumya S. Jamuar, the consortium brings together esteemed experts: Professor Vorasuk Shotelersuk from the Center of Excellence for Medical Genomics, Chulalongkorn University, Thailand; Professor Ming Chen from Changhua Christian Hospital Medical Center, Taiwan; Assistant Professor Jee Soo Lee from Seoul National University College of Medicine (SNUCM) Laboratory Medicine, South Korea; and Associate Professor Sohyun Hwang, CHA Bundang Medical Center, CHA University School of Medicine, South Korea. Together, they are breaking new ground in fertility research with the goal of revolutionizing care for millions affected by subfertility and RPL globally. “The formation of the HiFi Solves Sub-fertility Consortium is a significant step forward in fertility research,” said Christian Henry, President and Chief Executive Officer of PacBio. “By bringing together leading experts and leveraging the power of HiFi sequencing along with DNAstack’s federated data platform, we are poised to make substantial advancements in the diagnosis and treatment of subfertility and recurrent pregnancy loss.” Subfertility affects approximately one in six people globally, while RPL impacts 1-2% of women, often resulting in considerable psychological and financial strain. The consortium is harnessing the capabilities of PacBio HiFi sequencing technology to tackle these challenges head-on. Unlike traditional methods—such as karyotyping, chromosomal microarray, and whole-exome sequencing—HiFi sequencing offers a comprehensive, high-resolution approach that identifies complex chromosomal rearrangements and sub-microscopic abnormalities that other technologies may miss. This holistic view allows for a more precise characterization of chromosomal changes, particularly beneficial in cases of subfertility and RPL where multiple genetic factors may be involved. The consortium’s research is poised to significantly reduce the often-frustrating 'diagnostic odyssey' that many patients endure, offering a more streamlined and effective pathway to diagnosis and care. This initiative is expected to greatly enhance the success of assisted reproductive technologies, such as preimplantation genetic testing for structural rearrangements, leading to improved conception rates and healthier pregnancies. Spanning through the end of 2025, the consortium’s phased approach includes patient recruitment, advanced sequencing, bioinformatics analysis, and rigorous validation of results. Beyond its immediate impact, the consortium aims to influence clinical practices globally, contributing to peer-reviewed publications and presenting breakthroughs at international conferences. This ambitious project represents a significant leap forward in the application of genomic medicine to reproductive health, with the potential to deliver profound benefits to individuals facing these challenging conditions, underscoring the transformative power of collaborative research and advanced genomic technologies in addressing complex health issues. For more information, please contact: Contacts (PacBio) Investors: Todd Friedmanir@pacificbiosciences.com Media:pr@pacificbiosciences.com Contacts (KKH) Charlotte Sammedia@kkh.com.sg About PacBio PacBio (NASDAQ: PACB) is a premier life science technology company that designs, develops, and manufactures advanced sequencing solutions to help scientists and clinical researchers resolve genetically complex problems. Our products and technologies stem from two highly differentiated core technologies focused on accuracy, quality and completeness which include our HiFi long-read sequencing and our SBB® short-read sequencing technologies. Our products address solutions across a broad set of research applications including human germline sequencing, plant and animal sciences, infectious disease and microbiology, oncology, and other emerging applications. For more information, please visit www.pacb.com and follow @PacBio. PacBio products are provided for Research Use Only. Not for use in diagnostic procedures. About KK Women's and Children's Hospital (KKH) in Singapore KK Women’s and Children’s Hospital (KKH) is Singapore’s largest tertiary referral centre for obstetrics, gynaecology, paediatrics and neonatology. The academic medical centre specialises in the management of high-risk conditions in women and children. Driven by a commitment to deliver compassionate, multidisciplinary care to patients, KKH leverages research and innovation to advance care. In 2021, the hospital launched the SingHealth Duke-NUS Maternal and Child Health Research Institute (MCHRI) to support the growth of every woman and child to their fullest potential, and transform national heath in the region. Some of the hospital’s breakthroughs include uSINE®, a landmark identification system for the administration of spinal epidural, the discovery of new genetic diseases like Jamuar Syndrome, and a series of guidelines for women and children to improve population health. The academic medical centre is also a major teaching hospital for Duke-NUS Medical School, Yong Loo Lin School of Medicine and Lee Kong Chian School of Medicine. In addition, KKH runs the largest specialist training programme for Obstetrics and Gynaecology, and Paediatrics in Singapore. KKH was founded in 1858 and celebrates its 100th year as a maternity hospital in 2024. For more information, visit www.kkh.com.sg Follow us onFacebook | Instagram | LinkedIn | TikTok | YouTube About DNAstack DNAstack is a Canadian company whose mission is to save and improve lives by unlocking the collective power of the world’s genomics and health data. Omics AI is a software suite by DNAstack that enables privacy-preserving federated insights across distributed data. DNAstack is a global leader in the development of open, interoperable standards as part of the Global Alliance for Genomics & Health (GA4GH). Forward-Looking Statements This press release may contain "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the U.S. Private Securities Litigation Reform Act of 1995. All statements other than statements of historical fact are forward-looking statements, including statements relating to the uses, coverage, advantages, quality or performance of, or benefits or expected benefits of using, PacBio products or technologies, including in connection with the HiFi Solves Sub-fertility Consortium research and its efforts to improve the research, diagnosis and treatment of female infertility; efforts to reduce the diagnostic odyssey and enhance the success of assisted reproductive technologies; and other future events. You should not place undue reliance on forward-looking statements because they are subject to assumptions, risks, and uncertainties and could cause actual outcomes and results to differ materially from currently anticipated results, including, challenges inherent in sequencing a large number of genomes, and the difficulty of generating discoveries across various areas of research; potential product performance and quality issues; third-party claims alleging infringement of patents and proprietary rights or seeking to invalidate PacBio's patents or proprietary rights; and other risks associated with international operations. Additional factors that could materially affect actual results can be found in PacBio's most recent filings with the Securities and Exchange Commission, including PacBio's most recent reports on Forms 8-K, 10-K, and 10-Q, and include those listed under the caption "Risk Factors." These forward-looking statements are based on current expectations and speak only as of the date hereof; except as required by law, PacBio disclaims any obligation to revise or update these forward-looking statements to reflect events or circumstances in the future, even if new information becomes available.

MONDAY, Aug. 5, 2024 -- For patients with gastroesophageal reflux disease (GERD), improvement in symptoms is seen with both antireflux mucosectomy (ARMS) and antireflux mucosal ablation (ARMA), but subjective and objective indicators of outcomes are better with ARMS than ARMA, according to a study published online June 21 in Digestive Endoscopy . Ah Young Lee, M.D., Ph.D., from Cha University in Seoul, South Korea, and colleagues compared the efficacy and safety of ARMS and ARMA for GERD in a multicenter, retrospective, observational study including 274 patients (96 underwent ARMA and 178 underwent ARMS). The primary outcome was subjective symptom improvement, which was based on the GERD questionnaire (GERDQ). The researchers found that compared with the ARMA group, baseline GERDQ scores were higher in the ARMS group (10.0 versus 8.0) and there was a greater median postprocedure improvement observed (4.0 versus 2.0), even after propensity score matching. There was significant improvement seen in reflux esophagitis in ARMS versus ARMA, with notable changes seen in Los Angeles grade and flap valve grade scores. Comparable improvement in Barrett esophagus was seen between the groups, with 94.7 and 77.8 percent resolution rates noted in the ARMS and ARMA groups, respectively. The ARMS group experienced higher bleeding rates than the ARMA group, along with comparable stricture rates and more proton pump withdrawals. "Both ARMA and ARMS are advantageous because they do not require specialized equipment, are relatively inexpensive, and can be easily performed anywhere," the authors write.