Damian O'Connell

Race Oncology is pleased to share that the US Food and Drug Administration (US FDA) has extended Rare Paediatric Disease Designation (RPDD) to RC220 bisantrene for the treatment of paediatric acute myeloid leukemia (#AML). #RPDD is granted by the US FDA to new treatments of serious or life-threatening diseases that affect fewer than 200,000 people in the US, primarily those who are under 18 years old. The RPDD may qualify Race to receive a Priority Review Voucher (PRV) at the time of marketing approval or authorisation for RC220 in paediatric AML. The PRV can be redeemed for an accelerated review of RC220 by the US FDA, or sold to other parties, on average for more than US$100 million on the current open market. Race is currently in talks with world-renowned opinion leader in the treatment of AML, Associate Professor Himalee Sabnis, as well as an international paediatric oncology cooperative group, to further develop the company’s paediatric AML program. Dr Daniel Tillett, Race Chief Executive Officer, says: “US FDA RPDD is incredibly valuable as not only does it offer eligibility for the award of a PRV, but the ability to work with passionate clinicians and regulators to bring help to children and adolescents facing an enormously challenging disease with few effective treatment options.” More details, including an interview with our Executive Director Dr Peter Smith, are available here: https://lnkd.in/gTwX5c4r #paediatricAML #childhoodcancer #oncologytreatment #bisantrene

57

1 Comment

See our latest issue for discussions on: • Therapies for Atopic Dermatitis: An updated consensus guidelines for managing moderate-to-severe atopic dermatitis in Singapore, including the use of biologics and Janus kinase inhibitors. • Bayesian Approach to Corticosteroids for Community-Acquired Pneumonia (CAP): This analysis investigates whether a Bayesian approach can offer clearer insights into the effectiveness of corticosteroids for severe CAP. • Gender Dysphoria in Children and Adolescents: This retrospective analysis provides valuable data and perspectives on gender dysphoria in Singapore. • Unplanned Hospitalisations Among Subsidised Nursing Home Residents: This data linkage study offers insights into improving care delivery and reducing hospital readmission rates for nursing home residents. Working on a systematic review, meta-analysis or clinical practice guideline? Submit your manuscript for peer review and publication: For instructions for authors, see: https://lnkd.in/gWj58mcV Stay up-to-date on the latest advancements in healthcare, subscribe to our mailing list: https://lnkd.in/gyEt_w2c #AnnalsSG  #healthcare  #research  #Singapore  #medicine

6

When it comes to #pharmaceutical and #biotech companies and their #reputation with doctors, fighting for #patientaccess to new medicines will improve their reputation, according to a survey of 540 physicians. The study, published by WE Communications titled 'Reputation Rules: Unlocking Physician Prescribing Power', shows almost two in three Australian physicians believe fighting for patient access can improve the reputation of a company. "The research emphasises the importance of ensuring the extensive work the industry does in supporting and advocating for access is communicated to the prescribing community," WE Communications Australia Head of Health Emma Hussey told MedNews Aust/NZ. "HCPs want #transparency from industry and, in return, are willing to partner to support efforts towards better access for patients.” Full analysis in today's MedNews Aust/NZ (paywall). #corporatereputation #pharmareputation #fightforpatients #medicinesaccess #patientaccessgap

17

5 Comments

[Stem cell therapy for kidney failure] Stem cell therapy has garnered significant attention and hope as a potential treatment for kidney failure, especially given the kidney's limited capacity for regeneration and repair. However, several limitations need to be acknowledged in evaluating the efficacy and safety of this therapy. 1. Unknown Long-Term Effects: Although stem cell therapy shows promise in preclinical studies and early clinical trials, the long-term effects of this treatment are still largely unknown. The case reported in the Journal of the American Society of Nephrology highlights a new and previously unrecognized complication where the patient developed blood vessel and bone marrow masses at the injection sites, leading to kidney removal and ultimately death. This underscores the need for further research to determine the long-term consequences of stem cell therapy for kidney failure. 2. Lack of Definitive Evidence: Despite the increasing number of studies, there is still a lack of definitive evidence demonstrating the superiority of stem cell therapy over conventional treatments for kidney failure. While stem cells may aid in the regeneration process by producing protective and regenerative factors, direct differentiation into renal tubular cells has been questioned in recent data. Thus, the mechanisms underlying the potential benefits of stem cell therapy are not fully elucidated. 3. Difficulties in Standardization: Standardization of stem cell therapy poses significant challenges. Factors such as the source of stem cells (autologous, allogeneic, or embryonic), the cell dose, and the route of administration can vary significantly across studies, making it difficult to compare results and draw definitive conclusions. Additionally, the presence of heterogeneous cell populations within stem cell preparations can also affect their efficacy and safety. 4. Risk of Complications: As with any medical intervention, stem cell therapy carries risks of complications. In addition to the aforementioned blood vessel and bone marrow masses, other potential complications include immune rejection, tumor formation, and unintended differentiation into undesired cell types. These risks must be carefully evaluated and monitored in clinical trials. 5. Limited Access and High Cost: Access to stem cell therapy for kidney failure is currently limited, especially in developing countries. Moreover, the cost of stem cell therapy can be prohibitively high for many patients, limiting its widespread use. As such, further research is needed to develop cost-effective and accessible stem cell therapies. 6. Ethical Considerations: Ethical concerns related to stem cell therapy, particularly those involving embryonic stem cells, must be addressed. There are ongoing debates about the moral and ethical implications of using embryos for research and therapeutic purposes.

1

https://lnkd.in/gC8QDtpw This issue’s cover is taken from the original research report “Mice hepatic organoids for modeling nonalcoholic fatty liver disease and drug response” submitted by Ruixi Li and Xianjie Shi of the Department of Hepatobiliary Pancreatic Surgery, The Eighth Affiliated Hospital of Sun Yat-sen University, Shenzhen, China. Using their model, the authors revealed a significant role of the NOD-like receptor (NLR) signaling pathway and the peroxisome proliferator-activated receptor (PPAR) pathway in free fatty acid (FFA) induced hepatic steatosis. The effect of pretreatment with the NOD pathway inhibitor JC2-11 and the PPAR agonist lanifibranor was assessed using (A) Oil red O staining and Nile red staining of lipid droplets; (B) Liver function indicators alanine aminotransferase (ALT), aspartate aminotransferase (AST), and total bilirubin (TBIL);  (C) Triglyceride (TG) levels; (D) inflammatory cytokines IL-6 and TNF-α and (F) Nitric oxide (NO); and finally, (E) fibrosis indicators COL1A1 and LAMA1.

22

2 Comments

Cholangiocarcinoma (CCA) is an aggressive cancer of the bile ducts that is usually diagnosed at an advanced stage. Incidence rates are increasing and approximately 20,000 people will be diagnosed with CCA in the next 12 months globally. ​ At Taiho Oncology, we have an unwavering commitment to improving the lives of patients with cancer through our daily pursuit of novel therapies across multiple cancer types, including our relentless dedication to advancing care in CCA.​ Find out more about how we are making the human connection through our work.​ #TaihoOncology #Oncology #TaihoCares

120

The UK Biobank Pharma Proteomics Project (UKB-PPP), backed by 13 biopharmaceutical companies, is among the largest and most important biomedical research initiatives run to date. This pioneering endeavor, utilizing the Olink® Explore proteomics platform, has yielded an extensive dataset encompassing nearly 3,000 proteins measured in over 54,000 individuals from the well-characterized UK Biobank. This openly accessible resource serves as a cornerstone for modern biomarker-driven research, promising to accelerate our understanding of human disease, expedite drug development, and catalyze breakthroughs in precision medicine. In late 2023, the UKB-PPP consortium reported the first outcomes from this project in two landmark articles published in the same issue of Nature (Volume 622 Issue 7982, 12 October 2023). These publications outline a unique, open-access data resource for scientists. Importantly, the articles also present initial findings from the data clearly demonstrating the power of combining genomic and proteomic analysis at this unprecedented scale. The first article outlines the outcomes of a GWAS-based proteogenomic analysis, identifying thousands of genetic associations with protein expression levels (known as protein quantitative trait loci, pQTLs). These give unique insights into the genetic regulation of proteins and bring genomics closer to phenotype. Over 14,000 pQTLs were identified, 81% of which were novel. Approximately 83% of measured proteins had at least one pQTL with around 67% of these cis-associations, where the genetic variant was closely located to the protein-coding gene. These can be used in Mendelian randomization (MR) analyses to assess causal associations of proteins to phenotypes, an immensely powerful tool to identify robust new drug targets. Moreover, the high number of cis-pQTLs identified provides genetic corroboration of the specificity of the Olink assays. Many trans-pQTLs were also discovered, identifying novel biological pathways and interactions that can accelerate drug discovery, exemplified by new data showing the effects of the ABO blood group on gastrointestinal protein expression and how it may be perturbed in gastrointestinal diseases. #proteogenomics #openaccess #drugtargets #drugdiscovery

2

The latest instalment of his industry news round-up, Sreeram Ramagopalan explores some of the latest developments regarding #RWE in the context of #HTA. Read at The Evidence Base® #heor #healtheconomics #outcomesresearch #healthoutcomes #rwd #realworldevidence #realworlddata #marketaccess #healthtechnologyassessment #valueassessment

13

CAR T cells can gain new vigor when engineered to have high levels of a particular protein - Cancer Research Institute (CRI) Junyun Lai Peter MacCallum Cancer Centre https://lnkd.in/gKcu9TMJ #oncodaily #oncology #cancer #CancerResearch #Immunotherapy #CARTcells #Bioengineering #StemCells #CancerTreatment #MedicalResearch

18

Breaking barriers in ADC development: What’s next? 🔎 Antibody-drug conjugates are no longer just a concept – they’re revolutionizing cancer therapy with precision-targeted treatments. However, their success hinges on robust development strategies. Allucent explores how improved analytical methods, scalable manufacturing, and compliance with global regulatory standards are key to unlocking the full potential of ADCs. 🚀 By understanding the complex interplay of science, strategy, and regulation, biotech companies can navigate the challenges of bringing ADCs from the lab to the clinic. Dive into Allucent’s roadmap for clinical success and learn how ADCs are evolving to meet the unmet needs in oncology. 🔬 Full article below ⬇️ https://lnkd.in/dmUUXcdB #ADCtherapies #oncologyinnovation #cancerresearch #targetedtherapies #precisionmedicine #bispecificADCs #pharmabreakthroughs #drugdevelopment 

9

BSI Immune Therapies 2024: Key Insights and Innovations Engaging Talks: Discussions across immune-oncology, autoimmunity, and inflammation sparked cross-disciplinary insights. Breakout Focus Sessions: Topics like innate immunity and checkpoint inhibitors fostered dynamic small-group debates on drug discovery, clinical trials, and patient care perspectives. Therapeutic Breakthroughs: From IL-23 pathway research to oncolytic viral immunotherapy for solid tumours, innovation continues to translate blue-sky research into real-world treatments. Biomarker Strategies: Emphasised their pivotal role in advancing from 'imprecision' medicine to tailored therapies for arthritis and IBD. The British Society for Immunology deserves praise for driving impactful discussions. Excited for future conferences! To read more, the link is in the first comment

25

1 Comment

We just published our latest update note on Dimerix Limited which is in the middle of a Phase III clinical trial for its DMX-200 drug to market for Focal Segmental Glomerulosclerosis (FSGS). Since our last report on Dimerix in August 2024, the company has taken several steps forward in its clinical trial. These include opening new sites for the trial, continuing to recruit patients and advancing in collaborations that could help recruit patients and also advance the company’s efforts towards obtaining regulatory approval (by conceiving alternative or supplementary endpoints for the study). DXB has also continued to expand its Scientific Advisory Board. And finally, it has enrolled the first patient in the open label extension part of the study that will continue to monitor patients that have completed the 2 year Phase III study. The next catalyst for the company will be the release of results from current stage of the study, which are due 35 weeks after the last patient is enrolled – an event expected around mid-CY25. This event means, if the data is compelling, it is possible the company could apply for accelerated regulatory approval. We think investors should also look out for licensing deals for the US and China, which will be far larger and lucrative markets than any the company has licensed out to date. We reiterate our previous valuation of DXB at A$0.84 and A$1.07 per share (base and optimistic cases). These assume successful commercialisation of DMX-200 and all that all promised milestones consequently eventuate. Please note that this report is General Advice only and one Pitt Street has been commissioned to prepare. Please refer to pages 7-8 for more details on our valuation and the key risks to our thesis, and to the final page for the full General Advice Warning, disclosure and disclaimers. https://lnkd.in/eyjWtZ8T

2

Come check out our latest blog https://lnkd.in/gDJMSdrj When outsourcing preclinical pharmaceutical development, choosing between a large CRO and a smaller, bespoke provider can be challenging. While large CROs offer scale and vast resources, institutions like the Institute of Cancer Therapeutics (ICT) provide a personalised and scientifically rigorous approach that larger organisations may lack. At ICT, we specialise in tailored solutions, adapting to each project’s unique needs with agility and deep expertise. Our multidisciplinary teams of experts ensure responsive communication and problem-solving to help projects thrive. In today’s fast-paced drug development landscape, a customised approach can make all the difference. #pharma #CRO #drugdevelopment #bespokeapproach

23

Earlier this week the FDA released draft guidance on the oversight of AI-enabled devices and the use of AI in the development of drugs and biological products. According to PitchBook, funding for AI and machine learning startups accounted for ~36% of global venture capital deal value in 2024. As AI-enabled tools and technologies continue to advance at an accelerated pace, we need to be thoughtful about the policies developed around them to unlock the opportunities (and potential challenges) created by the use cases for AI in healthcare and drug development (e.g., automation, cost reduction, and higher quality decision-making across the space). Comments on the guidance documents are due by April 7, 2025, so it will be interesting to see how the new administration handles next steps on what will become non-binding recommendations from the FDA on these topics. See below for a link to an excellent summary of the guidance documents from Covington & Burling LLP. https://lnkd.in/eTpNiBfH #healthcare #drugdevelopment #innovation #ai

8