Radically different
SAN DIEGO — Revolution Medicines is already cooking up the next iteration of RAS inhibiting drugs.
At the American Association of Cancer Research annual meeting here, the company is the talk of the town for the clinical trial success of daraxonrasib, its next generation targeted therapy, in advanced pancreatic cancer. And while the company presented more data on that drug Tuesday, showing promising first line and combination data on daraxonrasib, scientists also showed in another session intriguing preclinical data on a completely new compound that may represent what comes after the current lineup.
That drug, currently called RM-055, is what RevMed CEO Mark Goldsmith is calling an entirely “novel class of catalytic inhibitors.” These are targeted therapies that not only block the RAS signaling that drives cancer, but molecularly turn the cancer protein off.
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We’re nearing the end of a big AACR. We hope to see everyone at our live event on Tuesday night. Clearly, Revolution Medicines and KRAS have been the big topic of the meeting. Last year, AACR was dominated by big concerns over what cancer research funding would look like in the Trump administration. This year, the new head of the NCI tried to allay researchers’ fears. Read on!
Last week, researchers working with the biotechnology firm Revolution Medicines presented stunning news: the experimental drug daraxonrasib more than doubled survival in second-line pancreatic cancer compared to chemo — although that only meant increasing median survival in this terrible disease by six months.
At the 2026 annual meeting of the American Association for Cancer Research (AACR), researchers from Memorial Sloan Kettering Cancer Center (MSKCC) presented new evidence that a blood-based biomarker, combined with advanced imaging, could enable real-time adjustment of cancer treatment in patients with HPV-related throat cancer. Findings from this clinical study (NCT03323463) highlight a potentially important shift in care, particularly for HPV-associated oropharyngeal cancer, a disease with generally high cure rates but ongoing efforts to reduce treatment-related toxicity. Rather than waiting until therapy is complete, clinicians may be able to tailor treatment intensity based on early indicators of response.
Circulating tumor DNA (ctDNA) has already shown promise for detecting minimal residual disease (MRD), but its role in guiding treatment decisions during therapy remains largely unexplored. To address this gap, a research team led by Bill H. Diplas, MD, PhD, a radiation oncology fellow at MSKCC, investigated whether serial ctDNA measurements, paired with weekly MRI scans, could provide a more precise and dynamic view of tumor response. In collaboration with Labcorp and Biocartis, the MSKCC researchers developed a personalized ctDNA assay that combined two strategies: detection of patient-specific tumor mutations and quantification of DNA from high-risk HPV strains, particularly HPV-16 and HPV-18, using anchored multiplex PCR and high-throughput sequencing.
The study enrolled 158 patients with HPV-associated oropharyngeal cancer who had undergone primary tumor resection followed by risk-adapted chemoradiotherapy guided by hypoxia assessment. MRIs were performed pretreatment and weekly following treatment to determine tumor volume, and blood samples were collected before treatment and weekly during therapy, yielding nearly 1,000 samples from 119 patients (mean 8.2 samples/patient) up to 126 weeks.
At baseline, ctDNA was identified in 93.9% of patients—outperforming either mutation-based (89.4%) or HPV-based (80.3%) methods alone. ctDNA levels also correlated with tumor size and biological features such as cell death and viral load. Notably, ctDNA emerged as a faster and more sensitive indicator of treatment response than imaging. Changes in ctDNA levels appeared earlier and across a broader dynamic range than tumor size reductions observed on MRI. By the second week of therapy, ctDNA measurements could already distinguish patients likely to require more intensive treatment.
The identification of patients with high-risk disease was significantly improved by combining on-treatment ctDNA assessment with imaging in a multimodal model, outperforming any modality alone. These results underscore the complementary nature of molecular signals in blood and structural changes seen on imaging.
Similar multimodal strategies that integrate ctDNA with imaging have been explored in other cancers, including breast and lung, primarily in research settings. Studies suggest that combining these approaches can improve prediction of treatment response and enable earlier detection of resistance. Broader analyses across colorectal, lung, and breast cancers further support the value of integrating molecular and imaging data to refine models of response and survival. However, most of these approaches remain investigational, and the use of ctDNA to guide real-time treatment decisions is only beginning to be tested in prospective trials.
Although further validation is needed, this study establishes a framework for real-time, personalized treatment in oropharyngeal cancer. If translated into clinical practice, such an approach could accelerate the shift toward adaptive therapy—where decisions are guided not only by how tumors appear on imaging, but by how they respond at the molecular level throughout treatment.
The post AACR 2026: MRI-ctDNA Combo Informs HPV-Related Throat Cancer Treatment appeared first on Inside Precision Medicine.
Background: HIV testing is the gateway to the HIV prevention continuum and offers an important opportunity to provide HIV prevention services. TakeMeHome.org is an online program that enables state and local health departments to offer free in-home HIV and sexually transmitted infection self-testing. As few TakeMeHome users have used pre-exposure prophylaxis (PrEP), there is an opportunity to link TakeMeHome users to PrEP information and services. Objective: The aim of this study is to develop an implementation strategy to link HIV or sexually transmitted infection self-testers from online orders to PrEP services via direct digital linkage to a novel SMS text messaging navigation program. Methods: PrEPmate is an evidence-based bidirectional text-messaging platform that has demonstrated increased PrEP retention and adherence. We developed a novel program to link TakeMeHome testers to mobile SMS text messaging PrEP navigation via PrEPmate. We conducted focus groups among TakeMeHome users to elicit preferences for linkage from TakeMeHome to PrEPmate. Based on these focus groups, we revised the content and functionality of this linkage intervention. In October 2023, we launched a pilot implementation study in 2 US Ending the HIV Epidemic jurisdictions: Sacramento, California, and Tarrant, Texas. Results: Thirteen TakeMeHome users participated in 4 focus groups (mean age 31.5 years; n=4, 31% Latinx, n=2, 15% Black; n=9, 69% never used PrEP). When shown wireframes of the TakeMeHome or PrEPmate linkage, most thought they were easy to navigate and user-friendly. They liked the privacy of connecting with a PrEP navigator using SMS text messaging. Participants recommended providing a clear description of PrEP and PrEPmate services and indicating that PrEP is low or no cost on the TakeMeHome website. On the PrEPmate landing page, they recommended adding language on confidentiality and the partnership with TakeMeHome to show that both services are connected. Once enrolled, they recommended weekly or biweekly check-ins to assist with PrEP navigation. Overall, 92% (12/13) of focus group participants were likely to use PrEPmate to learn more about PrEP and/or link to PrEP services. From October 2023 to May 2024, among 537 individuals who ordered test kits and were not on PrEP, 169 (31%) were linked to the PrEPmate page, and 86 (16%) enrolled in PrEPmate. PrEP navigation was provided via SMS text messaging or phone, with 46 (53%) receiving PrEP education and 26 (30%) in various stages of starting PrEP. In exit interviews, participants found the intervention easy to use and appreciated being connected with an experienced PrEP navigator who helped them access PrEP. Conclusions: Through user-centered design, we successfully developed a program to link TakeMeHome testers to PrEP navigation via PrEPmate, with high feasibility and acceptability of the intervention and a substantial number of clients starting PrEP. The next steps will involve evaluating the effectiveness of this program on a larger scale and, if successful, expanding PrEPmate navigation to all Ending the HIV Epidemic jurisdictions using TakeMeHome.
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SAN DIEGO – A new AI model applied to routine pathology slides accurately predicts outcomes and response to immunotherapy in patients with metastatic non-small cell lung cancer (NSCLC). The study was study presented at the American Association for Cancer Research (AACR) Annual Meeting.
“Immunotherapy has transformed cancer treatment, but only a subset of patients benefit from it, and predicting who will respond remains challenging,” said Rukhmini Bandyopadhyay, PhD, a postdoctoral fellow at The University of Texas (UT) MD Anderson Cancer Center.
“This study represents, to our knowledge, the first deep learning-based pathomics biomarker rigorously validated across international real-world cohorts and a Phase III randomized clinical trial, directly addressing one of the most urgent unmet needs in precision oncology: reliable patient selection and stratification for immunotherapy,” he continued.
Pathomics applies computational and machine learning methods for high-throughput analysis of digital pathology images to extract large-scale data related to cell and tissue architecture linked to disease outcomes.
Bandyopadhyay and colleagues developed a deep learning survival prediction model called Pathology-driven Immunotherapy Optimization (Path-IO), which can study patterns across tissue to identify patients most likely to benefit from immunotherapy. The model then combines imaging and clinical data to estimate whether a patient may have a higher or lower risk of poor outcomes from immunotherapy.
The researchers tested the platform in a study that included 797 immune checkpoint inhibitor-treated NSCLC patients from UT MD Anderson, with external validation in 280 additional patients from Mayo Clinic, Gustave Roussy, and the Phase III Lung-MAP S1400I trial in which immunotherapy-naïve patients with lung squamous cell carcinoma, a subtype of NSCLC, were treated with immune checkpoint inhibitors.
The model reliably stratified patients into higher and lower risk groups. In the UT MD Anderson cohort, patients in the high‑risk group had more than double the risk of death or disease progression compared with patients in the low‑risk group.
Model performance was evaluated using the concordance index (C-index), which measures how well each biomarker distinguishes between patients with different outcomes. Notably, Path-IO consistently outperformed PD-L1, the U.S. Food and Drug Administration-validated standard-of-care biomarker for guiding immunotherapy use in NSCLC patients, across both discovery and test cohorts.
PD-L1 alone showed limited prognostic performance, with C-indices of 0.58 for overall survival (OS) and 0.57 for progression-free survival (PFS) in the discovery cohort, declining to 0.50 and 0.51, respectively, in the test cohort. In contrast, Path-IO demonstrated stronger discriminative ability, achieving C-indices of 0.69 for OS and 0.65 for PFS in the discovery cohort and 0.63 for OS and 0.58 for PFS in the test cohort.
Combining pathology-based predictions with radiomics and clinical data further improved the model’s performance, with the C-index increasing from 0.58 to 0.70 for PFS and from 0.63 to 0.75 for OS.
Given that the approach was designed to be applied to routine pathology slides, the platform can be incorporated into existing clinical workflows without significant expense compared to other emerging data-based technologies.
As the study is retrospective, further investigation is needed to go beyond the identification of patients who would benefit from immunotherapy and help predict what type of immunotherapy they can benefit from. Future directions include prospective validation and the integration of paired, more comprehensive molecular profiling to enhance predictive performance.
The post AACR 2026: Lung Cancer Immunotherapy Response Predicted by Pathomics AI Model appeared first on GEN – Genetic Engineering and Biotechnology News.
Cosmo Pharmaceuticals says it plans to file for FDA approval of its androgenetic alopecia (AGA) candidate clascoterone 5% topical solution early next year, after the androgen receptor inhibitor generated 12-month Phase III data showing statistically significant continued hair growth as well as positive safety for chronic use.
Data from Cosmo’s Phase III program for clascoterone confirmed the drug’s long-term safety profile was comparable to vehicle, supporting suitability for chronic use in a lifelong condition. Clascoterone also showed a novel mechanism designed to target the underlying biology of hair loss and continued efficacy with ongoing use, Cosmo said.
A total of 1,465 patients were enrolled in the Phase III program, the largest for any topical treatment candidate for male AGA, according to Cosmo. The program consists of the SCALP 1 (NCT05910450) and SCALP 2 (NCT05914805) trials, which evaluated patients across 51 study centers in the United States and Europe.
Patients who remained on continuous clascoterone treatment for the full 12 months achieved a statistically significant 239% improvement in Target Area Hair Count (TAHC) compared with patients who received clascoterone for six months and were then switched to vehicle from month 7 to month 12, according to Cosmo.
That’s down slightly from the 252% improvement in TAHC shown for clascoterone versus “vehicle” or placebo in Cosmo’s six-month results, released in December. Cosmo Pharma CEO Giovanni Di Napoli told GEN that the 6- and 12-month results were not directly comparable due to differences in Part 1 and Part 2 of the Phase III placebo-controlled program and the corresponding patient groups being compared.
Part 1 is a double-blind study assessing if clascoterone was effective and safe compared to placebo when applied twice daily for up to six months. Part 2 is a single blind study that measured clascoterone’s long-term safety and efficacy versus placebo for an additional six months in patients who had responded to the study drug in Part 1. During Part 2, participants were re-randomized to receive either clascoterone 5% solution or vehicle solution.
SCALP 1 enrolled 702 patients in the United States, while SCALP 2 enrolled 763 patients in the Unites States as well as Germany and Poland.
Change in vellus TAHC (hair of up to 30 micrometers in diameter) from baseline was the trials’ primary outcome measure, paired with a patient-reported outcome assessing participants’ perception of hair growth improvement.
Additional assessments of the trials included investigator-reviewed global scalp photography and secondary endpoints that included changes in non-vellus TAHC (thicker, pigmented hair >30-40 micrometers in diameter), and changes in subject’s assessment of satisfaction score.
Patients treated with clascoterone for 12 months reported a statistically significant +24.5% relative improvement in treatment satisfaction versus vehicle groups, according to Cosmo. The clascoterone users also reported positive ease of use and product acceptability at month 12—results that according to the company support positive real-world usability and long-term adherence potential for the drug.
Cosmo said it plans to submit its full Phase III dataset for publication in a leading peer-reviewed medical journal and present its findings at future major dermatology congresses.
“These 12-month Phase III results mark a defining moment for clascoterone and for the treatment of male hair loss,” Di Napoli stated. “We are now seeing the combination that matters most: positive long-term safety, statistically significant continued hair growth through one year, and clear evidence that ongoing treatment drives sustained benefit.”
Investors responded to the positive 12-month data by sending Cosmo shares traded on the SIX Swiss Exchange rising 6% on the day of the announcement, from CHF95.50 ($122.69) to CHF101.40 ($130.27) on April 15. Since then shares have fluctuated in the high CHF 90 range, closing Monday at CHF 98.50 ($126.55).
Di Napoli said clascoterone has the potential to emerge as a major new therapeutic option and a highly valuable growth platform for Cosmo by tackling the most common cause of hair loss in men. Androgenetic alopecia, also called male pattern hair loss, affects approximately 40% of men worldwide—including 39% of males in the United States (65 million men).
“We are moving with urgency toward regulatory submissions and commercialization discussions,” Di Napoli added.
Cosmo’s results “likely now enable more advanced partnership discussions, in our view, with detailed presentation of results the next step to fully de-risk the asset,” Benjamin Jackson, equity analyst with Jefferies, wrote April 15 in a research note.
Jackson predicted clascoterone could generate $4 billion in peak-year worldwide sales.
“Our $4 billion WW potential peak sales require just 4% penetration of treated and 6% penetration of untreated men at peak, assuming a capable commercial partner is successfully found,” Jackson added.
Cosmo said it is preparing to submit not only an NDA for clascoterone in the U.S., but a marketing authorization application to the European Medicines Agency.
Clascoterone’s 1% formulation is already FDA-approved and marketed as Winlevi® for topical treatment of acne vulgaris in patients ages 12 and older.
The post Cosmo Pharma Eyes 2027 NDA for Baldness Candidate After Positive Phase III 12-Month Data appeared first on GEN – Genetic Engineering and Biotechnology News.
Fast diagnostics could improves patient outcomes and save billions in healthcare costs if used systematically to identify sepsis in hospitalized adults at risk due to bloodstream infections, according to an independent report.
The research encompassing all G7 countries revealed multiple benefits from earlier intervention for time-critical infections through the extensive application of fast identification and antimicrobial susceptibility testing (Fast ID/AST).
The Value of Fast Diagnostics in Time-critical Infections report by the independent Office of Health Economics showed how systematic use of fast diagnostics would lead to far fewer sepsis-related deaths and significantly decrease long-term post-sepsis complications, thereby improving patients’ quality of life.
The multi-country economic evaluation, commissioned and funded by bioMérieux, also showed major savings in healthcare costs each year, with the magnitude dependent on country size, incidence, and cost structures.
“While the magnitude varies by country, the direction is consistent: the model demonstrates that early diagnostics reduce the likelihood that high-risk patients progress to sepsis,” said Julien Textoris, PhD, vice president of EMEA medical affairs at bioMérieux.
“Preventing cases of sepsis could therefore reduce the risk of long-term complications after hospital discharge, including recurrent infections, cognitive decline, psychological effects, and organ-specific complications.”
Sepsis is a life-threatening reaction to an infection responsible for 21 million deaths worldwide each year. The initial hours of management are crucial, with targeted antibiotic treatment a key predictor of survival.
However, conventional methods for diagnosis takes at least a couple of days deliver results and nearly one in five bloodstream infection patients receive an inappropriate initial treatment.
In a model-based health economic analysis, Shaheer Hassan and co-workers at the OHE examined what would happen if fast ID/AST were systematically used early in the care pathway before clinical deterioration occurs.
The study encompassed Canada, France, Germany, Italy, Japan, the United Kingdom, and the U.S. and used expert-validated clinical inputs, country-specific cost data, and conservative assumptions.
Results revealed consistent cost savings regardless of the structure or financing of the healthcare system.
More than half of all savings—between 53% and 83%—would occur during the initial hospitalization, when the clinical and economic consequences of deterioration are most evident.
This is because early diagnostic information would prevent the chances of patients progressing into one of the most resource intensive stages of sepsis care.
The savings per patient ranged from €500 in Canada to €3,800 in Japan. This was primarily driven by fewer admissions to the intensive care unit, shorter hospital stays, and educed management of severe complications.
Annual national savings ranged from €26 million in Canada to €2.5 billion in the U.S. and reflected both cost savings in the acute phase and from reduced long-term complications.
“To realize these benefits, hospitals must address structural and workflow barriers so fast results translate into faster therapy, alongside broader system reforms to correct the persistent undervaluation of diagnostics,” the report maintained.
“Overcoming the underutilization and undervaluation of diagnostics will require coordinated system-level action, with the G7 countries included in this analysis well-positioned to lead.”
The post Sepsis Fast Diagnostics Could Save Lives and Billions in Costs appeared first on Inside Precision Medicine.
The American Association for Cancer Research (AACR) meeting is off and running in San Diego. Julianna LeMieux, PhD, Deputy Editor in Chief at GEN, and Damian Doherty, Editor in Chief at Inside Precision Medicine, are on the ground—in the talks, expo hall, and press room, covering as much of the news as they can. Here, they take a moment to chat about the first few days at the meeting.
The post AACR 2026: A Video Update from San Diego appeared first on GEN – Genetic Engineering and Biotechnology News.