Clinical Trial News

COVID-19 accelerated mRNA vaccine development, leading to record profits and FDA approval of mRNA vaccines. Moderna and Pfizer/BioNTech are expanding their mRNA pipelines into other diseases, with Moderna's RSV vaccine recently approved. The mRNA therapeutics market is projected to reach $68 billion by 2030. mRNA technology is favored for its rapid development potential compared to traditional vaccines. Five mRNA vaccine candidates, including Pfizer/BioNTech's influenza vaccine and Moderna's combination COVID-19/influenza vaccine, are highlighted as near-future prospects.

The FDA faces several key decisions by September, including Vanda's tradipitant for gastroparesis, Zevra's arimoclomol for Niemann-Pick disease type C, Heron's extended-release needle for Zynrelef, Merck's Keytruda for pleural mesothelioma, and Bristol Myers Squibb's KarXT for schizophrenia. Sanofi and Regeneron seek to expand Dupixent's use for COPD.

Despite challenges, the HEALEY ALS Platform Trial continues with optimism, as Clene Nanomedicine and Prilenia Therapeutics progress their ALS treatments based on positive secondary endpoint data. The trial, initiated by a $40 million donation from Sean Healey and AMG, aims to efficiently test multiple therapies simultaneously. While primary endpoints have not been met, the trial's design allows for quick identification of non-viable treatments, and positive outcomes in subgroups have supported further development for some candidates.

Dimerix collaborates with UK’s National Registry of Rare Kidney Diseases to identify FSGS patients for ACTION3 trial, aiming to evaluate DMX-200 efficacy and safety. RaDaR, the largest kidney registry, holds records of 34,500 patients. The trial, currently recruiting across 16 countries, targets 286 patients over two years, with interim analysis showing DMX-200 outperforming placebo in reducing proteinuria.

Peter Macallum Cancer Centre's UpFrontPSMA study shows LuPSMA therapy significantly improves outcomes for newly diagnosed prostate cancer patients, with 41% achieving undetectable PSA at 48 weeks compared to 16% with standard-of-care. The results were published in *Lancet Oncology* and presented at ESMO 2024.

Peter Macallum Cancer Centre's UpFrontPSMA study shows LuPSMA therapy significantly improves outcomes in newly diagnosed metastatic prostate cancer patients, outperforming standard chemotherapy alone. The trial, funded by Prostate Cancer Research Alliance, involved 130 patients from 11 Australian hospitals and was presented at ESMO 2024. ANSTO supplies the Lutetium-177 isotope used in the therapy.

U.S. Senators introduced the FDA Modernization Act 3.0 to mandate FDA update regulations within six months, aligning with FDAMA 2.0, which removed mandatory animal testing for investigational new drugs in 2022.

Trastuzumab deruxtecan (T-DXd) improved physical/role functioning and pain in HR+, HER2-low/-ultralow metastatic breast cancer patients compared to treatment of physician’s choice (TPC), with significant reductions in pain deterioration (22.0 months vs 6.3 months) and other symptoms, while maintaining global health/QOL over 31 weeks.

The APOLLO trial at ESMO 2024 showed Chia Tai Tianqing's Focus V and Akeso's penpulimab significantly improved PFS and OS in advanced hepatocellular carcinoma (aHCC) compared to sorafenib, with a manageable safety profile. This contrasts with the failure of Merck & Co's Keytruda in the LEAP-002 trial. Competitive IO-TKIs like Elevar Therapeutics' camrelizumab and rivoceranib also showed benefits but with more TRAEs. Roche's Tecentriq with bevacizumab remains the first-line standard-of-care in aHCC, posing a challenge to new IO-TKI combinations.

ESMO 2024 session on rare genitourinary cancers featured updates on penile cancer by Prof. Andrea Necchi, highlighting geographic variation in incidence, surgical indications, and treatment trends. Key updates included neoadjuvant chemotherapy recommendations and ongoing challenges in patient selection for perioperative therapy. Clinical trials like InPACT and HERCULES evaluated neoadjuvant therapies, while systemic therapy options for metastatic cases were also discussed. HPV status and tumor mutational burden (TMB) were noted to influence treatment outcomes. Ongoing research emphasizes the need for tailored approaches and clinical trial participation.