University of California, Davis

A panel of experts today voted against Capricor’s cell therapy for Duchenne muscular dystrophy (DMD), capping off an advisory committee meeting marked by statistical confusion. Capricor’s therapy, deramiocel, is meant to restore heart and skeletal muscle function in DMD patients. Capricor’s approval application is specifically for DMD-associated cardiomyopathy, a common complication of the rare genetic disease, and the panelists were asked to vote only on deramiocel’s effectiveness in cardiomyopathy. Speaking to Fierce the day before the committee met, Capricor co-founder and CEO Linda Marbán, Ph.D., cautioned that she doesn’t have a crystal ball but “cannot imagine why somebody would vote against this.” Nine experts ultimately did, with only three voting in support of deramiocel’s efficacy. While advisory committees provide recommendations, the FDA is still the ultimate arbiter of approvals and can choose to go against the experts’ consensus. A decision on deramiocel is expected from the agency by August 22. Patient representatives Buddy Cassidy, Ph.D., and Debra Dunn voted in favor of the therapy’s efficacy, and were joined by the adcomm’s acting chair Evan Snyder, M.D., Ph.D., a neurologist at Sanford Burnham Prebys Medical Discovery Institute in San Diego. “As a scientist I would have voted no,” Snyder said when explaining his decision. As a clinician, however, “I voted a highly qualified yes, because I don't want methodologic[al] issues to close the door on something that could have something there.” The adcomm centered on Capricor’s resubmitted biologics license application (BLA) for deramiocel, which was based on new data from the phase 3 HOPE-3 trial . Capricor’s original rejected submission used data from the earlier Hope-2 trial and its open-label extension, the results of which the company and regulator firmly disagree on. Capricor put forth its revised BLA following the announced departure of Vinay Prasad, M.D., the former director of the FDA's Center for Biologics Evaluation and Research, who oversaw the previous rejection of deramiocel and numerous other rare disease therapies during his turbulent tenure . Deramiocel consists of specialized heart cells, called cardiosphere-derived cells, sourced from donor cadavers. The treatment is given via IV infusion every three months. Overall, experts considered the results of Capricor’s Hope-3 trial too “fragile,” meaning that they were overly dependent on the type of analysis performed and on the exclusion of certain patients. Like the Hope-2 trial before it, the FDA and Capricor fundamentally disagree on whether or not the Hope-3 study of deramiocel hit its primary endpoints. Capricor announced the trial succeeded last December, but the FDA maintains that the trial in fact failed, and that Capricor’s claims of efficacy are due to numerous changes the company made to its statistical analysis plan. In Capricor’s presentation at the adcomm, Marbán condemned the FDA’s use of statistical analysis plan (SAP) 1.1, “which was never formalized to evaluate this data," and which she said "is simply not appropriate and conceals a true treatment effect.” “For reasons we do not understand why—the FDA has seized upon that unfinished, unsigned draft SAP and performed numerous analyses based on this incomplete draft,” Marbán said. “This would be like your professor grading your term paper on an early draft you have never even submitted.” Results from Hope-3, using Capricor’s preferred SAP version 3.0, were published in The Lancet during the adcomm. During its own presentation later in the day, the agency defended this decision. "The models specified in SAP 1.1 are consistent with those specified in SAP 1.2 through 1.5,” Tingting Zhou, Ph.D., an FDA statistician, said during the agency’s presentation. "Notably, the analytical models and missing data handling are similar across all SAP versions and protocol versions that preceded the interim futility analysis.” Through the meeting, committee members largely sought to clarify details of the different analysis plans, with several expressing concerns over the extent of Capricor’s changes and others taking issue with the FDA’s failure to provide feedback on SAP 2.0, which was ultimately supplanted by SAP 3.0. Though not the trial’s primary endpoint, deramiocel’s approval in DMD-associated cardiomyopathy hinges on left ventricular ejection fraction (LVEF) data, a measure of the heart’s ability to pump blood. Capricor’s original plan was to analyze the change in LVEF in patients after treatment, but the biotech pivoted the day before data unblinding to instead analyze ranked change in LVEF using a different type of statistical test. Even with Capricor’s last-minute analysis change, the final p-value for the LVEF data presented to the committee still was not statistically significant, sitting at 0.09. "In some ways, this discussion is sort of moot, inasmuch as the sponsors' analysis, as presented in The Lancet and presented to us, show that they did not meet this endpoint statistically,” committee member John Teerlink, M.D., a cardiologist at the San Francisco Veterans Affairs Medical Center, said during the discussion. Teerlink was also concerned by data suggesting patients given deramiocel experienced increases in left ventricular volume. “Whenever I see increases in volumes, that's a no-go area,” he said. “In other areas, drugs that increase ventricular volumes actually end up hurting people.” Though deramiocel is under consideration only for approval in DMD-associated cardiomyopathy, Marbán urged the experts at the adcomm to consider deramiocel’s full potential in the disease. “We respectfully ask that your vote reflect the potential of deramiocel to address the disease as a whole, and whether these boys deserve a chance to have deramiocel in their arsenal of opportunities to treat DMD,” Marbán said. During the adcomm’s public comment section, multiple patients, patient advocates and physicians urged the FDA to add a voting question for the skeletal muscle data. Many spoke of deramiocel’s positive effects on preserving muscle function and helping patients maintain independence. The adcomm’s acting chairperson, Snyder, ultimately said FDA guidance does not allow for adding a voting question but encouraged the committee to share their full views on the PUL 2.0 data. Like the secondary ejection fraction endpoint, Capricor and the FDA also disagree on whether the primary skeletal muscle endpoint—improvement in a measure of upper limb function called PUL 2.0 —was met. While initially meant to be analyzed as an absolute change in PUL 2.0 score, Capricor updated the analysis while the trial was ongoing—but again before unblinding—to assess percent change from baseline instead. Capricor contends this change was scientifically warranted, based on a 2025 paper from experts not affiliated with the company, and that the PUL 2.0 endpoint was met however one looks at the data. “Hope-3 met the primary endpoint,” Craig McDonald, M.D., a pediatrician and neuromuscular specialist at the University of California, Davis and national principal investigator for the Hope-2 and Hope-3 trials, said during Capricor’s presentation. “Primary analysis gives a mean difference of 4.55% in favor of deramiocel, with a p-value of 0.029, which corresponds to a 1.2-point absolute change in total PUL 2.0.” A change in PUL 2.0 score of one point or more is generally considered clinically meaningful. A pre-specified sensitivity analysis of the absolute change in PUL 2.0 produced a p-value of 0.05, McDonald added, right on the cutoff for statistical significance. “The conclusion regarding the primary endpoint does not depend on the choice of scale,” McDonald said. Per the FDA, however, the opposite is true. "The results are unambiguous,” said Prateek Shukla, M.D., an acting branch chief in CBER’s general medicine division. “The primary endpoint, mean change from baseline in PUL 2.0 total score at month 12, showed a mean difference of 0.66 points favoring deramiocel with a p-value of 0.24. This is not a near miss. The study did not meet its primary endpoint.” While the experts saw more encouraging signs in the PUL 2.0 data and felt compelled by the patient testimony asserting deramiocel’s effectiveness in upper limb function, they still found the data unconvincing and too dependent on the kind of analysis used. But for future research, the panel highlighted upper limb function as an area for Capricor to pursue further. “There may be something in the upper limbs, but it's really not proven here, either quantitatively or qualitatively,” committee member Steven Pavlakis, M.D., a pediatric neurologist at SUNY Downstate Health Sciences University in Brooklyn, said. “But if there was anything to look at going forward, that would be it.”

New Pilot Aims to Build a Scalable Model for Integrating At-Home HPV Screening Into Routine Care SAN FRANCISCO, July 23, 2026 /PRNewswire/ -- Teal Health, creators of the first and most accurate FDA-authorized self-collection device for at-home cervical cancer screening, today announced a pilot study with UC Davis Health to evaluate how at-home HPV self-collection integrates directly within a health system setting and reaches women who are due or overdue for care. The study, funded through Teal Health's NIH SBIR grant, represents an important step toward enabling health systems to offer at-home cervical cancer screening at scale. The program assesses how Teal Health's at-home HPV self-collection pathway can help close preventive care gaps, improve screening completion, and reduce burden on clinics and care teams, while establishing a repeatable model for broader health system adoption. Through the pilot, women ages 30–65 who are due or overdue for cervical cancer screening will receive outreach through MyChart, email, text message, or their care teams, directing them to Teal's at-home screening pathway. Patients who participate will complete an eligibility screening before receiving a kit mailed directly to their homes. Completed kits will be returned by mail and processed through the UC Davis in-house laboratory, with results integrated directly into the health system's electronic health record. "This is an important milestone in making at-home cervical cancer screening more accessible through the health systems women already trust," said Kara Egan, CEO and Co-Founder of Teal Health. "UC Davis Health was looking for a partner that matched their in-clinic accuracy, delivering results that providers and patients could trust. By embedding Teal's FDA-authorized Teal Wand and telehealth platform into existing health system workflows, we're working toward a future where preventative care is accurate, easier to access, and easier for health systems to deliver at scale." A core focus of the pilot is understanding how at-home screening works within real-world clinical operations. Teal Health and UC Davis Health have integrated ordering, lab processing, and results reporting through Epic and HL7 workflows, helping reduce manual work and laying the foundation for scalable implementation across additional health systems. "The future of preventive care lies in delivering evidence-based services in ways that are more accessible, equitable, innovative, and responsive to patients' lives," said Heather Leisy, medical director of Population Health Quality at UC Davis Health. "By expanding screening options we can help more individuals receive the screenings that prevent disease and improve long-term health." At the conclusion of the pilot, researchers will assess how the at-home screening pathway compares with standard in-clinic cervical cancer screening approaches by evaluating screening completion rates, patient engagement, operational feasibility, and the program's ability to reach women overdue for care. "This collaboration represents an exciting opportunity to explore how at-home screening can expand access to preventive care and offer a more convenient, accessible, and patient-centered option for staying up to date on recommended screenings," said Julie Dang, assistant director for Community Outreach and Engagement at UC Davis Comprehensive Cancer Center and a researcher who will be leading the study. The pilot marks continued momentum in Teal Health's broader strategy to partner with health systems, academic medical centers, employers, and public health organizations to expand access to cervical cancer screening nationwide. By demonstrating how at-home screening can integrate into existing clinical infrastructure, Teal Health is helping build the future of preventive care delivery. About Teal Health Teal Health is a woman-led company on a mission to improve women's healthcare experiences – starting with the first FDA-authorized vaginal self-collection device for at-home cervical cancer screening available nationwide. By creating the option for a woman to screen for cervical cancer from the comfort of home with virtual support and personalized follow-up care when needed, Teal can increase access to this life-saving cancer screening. Teal Health is a member of the Cervical Cancer Roundtable, a joint collaboration between the American Cancer Society and the Cancer Moonshot, a coalition of industry leaders with the goal of eliminating cervical cancer as a public health concern in the US. To learn more, visit . SOURCE Teal Health 21% more press release views with Request a Demo

Latent-Y, the first lab-validated drug design agent, is now open to researchers worldwide through the Latent Labs Platform Every approved researcher receives a free daily quota of 250 designs, with additional credits available on demand for larger campaigns In published results, Latent-Y reached a 67% target-level success rate across nine targets, with binding affinities in the single-digit nanomolar range Researchers at UC Davis, LMU University Hospital and the Translational Genomics Research Institute produced lab-validated designs on the Latent Labs Platform, including groups with no prior computational design experience Results include de novo designed functional inhibitors of human ion channels, CAR-T cells that killed tumour cells in vitro and are now in animal studies, and high-affinity binders against a conserved malarial target LONDON & SAN FRANCISCO--(BUSINESS WIRE)--Today, Latent Labs launches Latent-Y, its lab-validated drug design agent, to researchers worldwide through the Latent Labs Platform. Any approved researcher can now access Latent-Y for free, with a daily design quota included at no cost. For larger campaigns, researchers can purchase additional design credits on demand. Latent-Y is an AI agent for drug design. It takes a therapeutic goal, specified in written language or as a scientific publication, and reasons through the design process end to end, delivering lab-ready sequences. It works through the decisions a specialist structural biologist would make, from identifying the right site on the target to selecting and refining candidates against it. The agent can run without intervention, or pause at each stage for researcher review, giving researchers full control over every design decision. In published results, Latent-Y achieved a 67% target-level success rate across nine targets, with binding affinities reaching the single-digit nanomolar range, compressing weeks of expert workflow into hours, a 56x acceleration. Ahead of today's launch, the Latent Labs Platform was made available to a cohort of academic research groups, spanning ion channel structural biology, cancer immunotherapy and infectious disease, and including labs with no prior computational design experience. At UC Davis, Prof. Vladimir Yarov-Yarovoy's group designed nanobodies from scratch against a human ion channel. Of five taken into the lab, four blocked the channel's electrical current, and the most potent did so at very low concentrations (an IC50 of 30 nM), a potency usually reached only after rounds of laboratory refinement, but achieved here on the first attempt. At LMU University Hospital, Dr Daria Briukhovetska's group built designed binders into CAR-T cells that efficiently killed tumour cells in vitro and have since moved to animal studies. At the Translational Genomics Research Institute, Prof. John Altin's lab confirmed dozens of high-affinity binders against a conserved site on a malaria parasite protein, one the parasite cannot mutate away without losing its ability to cause severe disease. Latent-Y is the reasoning and natural-language interface to the Latent Labs Platform, the same models and tools that produced the results above. It runs entirely in the browser, with no infrastructure to set up and no pipelines to maintain, so researchers can start designing immediately and at scale. For scientific teams, this means a single researcher can pursue targets that would otherwise sit out of reach. To mark the launch, Latent Labs will host a live demonstration and scientific presentation in London, on July 15 2026. Attendees will see Latent-Y run a live design campaign and hear results from the beta program directly from the research teams. The evening will conclude with a panel discussion on “Frontier protein design, now in every researcher’s hands”, featuring the former CEO of Merck KgaA, Stefan Oschmann. Registration is open at www.luma.com/latent-y-launch. "We built Latent-Y to be a force multiplier for researchers. Just as coding agents are accelerating software development, Latent-Y accelerates drug development: one person running dozens of design campaigns in parallel, each progressing with expert-level biological reasoning. Researchers can now design antibodies, nanobodies and macrocycles at unprecedented scale, all from a natural language prompt," said Simon Kohl, CEO and founder of Latent Labs. Researchers own the sequences they generate, and Latent Labs does not train on user data or outputs. Access is available at platform.latentlabs.com; applicants provide their affiliation and research use case. Commercial partnerships are available through a separate enterprise tier at partnerships@latentlabs.com.