Conditions: Borderline Personality Disorder (BPD)
Sponsors: University Hospital Heidelberg
Recruiting
Sponsors: University Hospital Heidelberg
Recruiting
STAT+: Pharmalittle: We’re reading about another FDA reversal, pharma’s M&A spree and much more
Good morning, everyone, and welcome to another working week. We hope the weekend respite — longer than usual thanks to a holiday on this side of the pond — was relaxing and invigorating. Now, though, that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on a cuppa stimulation. Our choice today is English breakfast. Feel free to join us. Remember, no prescription is required. Meanwhile, here are a few items of interest. Best of luck accomplishing your goals and we hope you conquer the world. And, of course, do keep in touch …
The U.S. Food and Drug Administration will reconsider approving an experimental gene therapy for a deadly and rare childhood brain disorder that it rejected just four months ago, STAT tells us. The sudden turnaround is the latest in a series of apparent FDA reversals in the past two months, after leaders installed by the Trump administration resigned or were fired. Just last week, UniQure announced it was cleared to submit an application for a Huntington’s disease gene therapy that the agency had previously spurned and that former commissioner Marty Makary appeared to disparage on national television.
The U.S. launched a trade investigation into a German plan to lower its spending on pharmaceutical products, to see whether it is unreasonable or discriminatory, Reuters notes. The probe by the U.S. Trade Representative comes under Section 301 of the Trade Act of 1974 and follows a move by the German Ministry of Health unveiled plans in April for a wide-ranging overhaul of the country’s statutory healthcare system to reduce a looming funding gap by $23 billion. The plan, which would have introduced variable discounts on pharmaceuticals, is being replaced after the pharmaceutical industry expressed opposition to it.
Beyond the Genome: Five Emerging Leaders in Epigenetics Diagnostics
Epigenetics is increasingly powering cancer diagnostics and liquid biopsies. These emerging private companies are spurring the market with structural DNA, fragmentomics, and DNA methylation tests.
Our understanding of the role of epigenetics in disease is growing rapidly, driven by rapid advances in sequencing technology and computing.
Epigenetic processes such as DNA methylation, histone modifications, and non-coding RNA expression can interact with genomic changes to cause cancer. Therefore, diagnostics can detect early signs of disease by screening for these epigenetic signals.
Players including Illumina, Agilent Technologies, and Roche Diagnostics are leading the global market for epigenetic diagnostics, which is expected to swell by 15.5% per year from $17 billion in 2024 to $39 billion by 2030.
Growth is being driven by the increasing affordability of genome sequencing; the integration of AI tools in data analysis; growing investments; and soaring demand for liquid biopsies—noninvasive cancer tests based on blood and urine samples.
One of the first diagnostics with an epigenetic component to be approved by the U.S. Food and Drug Administration (FDA) was Exact Sciences’ (now part of Abbott Laboratories) ColoGuard® noninvasive stool test for colorectal cancer in 2014.
Since then, epigenetics diagnostics have already been generating M&A activity, with deals in the space including Cardio Diagnostics of the U.S. going public via a merger with special purpose acquisition company Mana Capital in 2022; the takeover of Ireland’s EpiCapture by compatriot Trinity Biotech in 2024; the 2023 acquisition of Germany’s Epigenomics AG by U.S.-based New Day Diagnostics; and U.S. Agilent’s acquisition of Avida Biomed, also in 2023.
There is also corporate venture interest with giants like Illumina Ventures, the Labcorp Venture Fund, and Lilly Asia Ventures making investments in small startups.
Check out below for our take on the most promising privately-owned players in the epigenetics diagnostics space, based on their investor attraction and market potential.
1. Arima Genomics
Founded: 2015 | Headquarters: Carlsbad, California
Arima Genomics was spun out of UC San Diego and developed research tools to pinpoint the 3D structure of DNA in cells.
However, the company pivoted to cancer diagnostics after its assay discovered vital clues on how to treat a teenage girl with glioblastoma in 2022.
Arima’s Hi-C technology involves locking the DNA structure in place via crosslinking. DNA strands are then cut with enzymes and labeled with a marker called biotin. Arima uses a process called proximity ligation to connect DNA strands that were physically close together into a single strand, and then sequences the resulting molecule.
Last year, Arima launched a lymphoma test that is delivered via the firm’s laboratory testing service, certified by the U.S. Clinical Laboratory Improvement Amendments (CLIA) program. The test is designed to be used to help patient management by discovering gene fusions and rearrangements for 417 genes in different types of lymphoma.
The test helps to fill in the gaps left by the gold standard, fluorescent in situ hybridization, which can be time- and resource-intensive and lead to conflicting results.
Arima raised $22 million in a Series C round led by Illumina Ventures in 2025 and appointed a former venture partner from Illumina Ventures as CEO. The firm is using the proceeds to launch a pipeline of clinical assays in cancer.
Arima also closed a partnership with Fox Chase Cancer Center earlier this year to co-develop diagnostic tests for lymphoma and sarcoma.
2. DELFI Diagnostics
Founded: 2019 | Headquarters: Baltimore, Maryland
DELFI Diagnostics was founded on an “aha” moment at Johns Hopkins University School of Medicine when a group of researchers aimed to overcome the high costs and low sensitivity of traditional liquid biopsies.
The breakthrough involved hunting for the certain way cell-free DNA fragments appear in the blood. Healthy cells and cancer cells package their DNA in different patterns, reflecting changes in the cell’s genomic and epigenomic machinery.
Using this method of “fragmentomics,” DELFI’s technology can tap into orders of magnitude more data than traditional methods.
DELFI’s product FirstLook Lung uses artificial intelligence (AI) and fragmentomics technology to screen a blood sample for signs of lung cancer. It is designed as an adjunct tool to check whether patients are eligible for lung cancer screening, and is regulated under the CLIA program.
The startup’s other product, DELFI-Tumor Fraction (DELFI-TF), allows pharmaceutical companies to track the effectiveness of a cancer therapy based on a sample of less than one milliliter of plasma.
Delfi raised $5.5 million in a seed round when it was founded, with investors including Menlo Ventures and Illumina Ventures.
The startup subsequently raised a $100 million Series A round led by OrbiMed in 2021, a $225 million Series B round led by DFJ Growth in 2022, and a $34 million debt round last year.
3. Element Biosciences
Founded: 2017 | Headquarters: San Diego, California
Element Biosciences was co-founded by three former Illumina employees who dreamed of democratizing access to genomic sequencing.
The company markets devices designed to sequence genetic information at a lower cost and higher performance than traditional next-generation sequencing. These include AVITI
—its flagship benchtop sequencer—and AVITI24, which can simultaneously analyze DNA, RNA, proteins, and phosphorylated proteins.
The company generated $25 million in revenue in 2023, partly driven by orders of AVITI.
The technology, based on a process called Avidite Base Chemistry (ABC), uses a dye-labeled polymer to bind genetic material and produce sequencing data with the need for fewer reagents than traditional sequencing.
Element is working with epigenetics specialists to boost their research offerings, including Dovetail Genomics and biomodal.
The company has also formed collaborations with diagnostics providers to enhance their offerings, including Revvity’s neonatal genetic tests and Medicover Genetics’ tests for hereditary cancers, metabolic and cardiovascular disorders, infertility, and neonatal diseases.
The company plans to market a clinical diagnostics-focused sequencing product called AVITI Dx, with EU approval expected this year in the form of a CE In Vitro Diagnostic (IVD) mark.
Element Biosciences has raised more than $680 million since it was founded, including a $277 million Series D round in 2024. The asset manager, Wellington Management, led the oversubscribed round, with participation from Samsung Electronics, Fidelity, and more.
This year, Element plans to commercialize a benchtop device, called VITARI, that can sequence a whole genome at high quality for just $100.
4. Nucleix
Founded: 2008 | Headquarters: Rehovot, Israel
Nucleix was initially founded to use epigenetics to trace falsified DNA in forensic investigations. Although the technology worked well, the management team decided to pivot to cancer screening.
Nucleix’s kits involve screening for specific cancer-linked DNA methylation patterns using polymerase chain reaction (PCR) tests.
The company also uses machine learning to construct biomarker panels best suited to the application of interest.
Nucleix’s Bladder EpiCheck® urine test is designed to detect the recurrence of bladder tumors based on changes in DNA methylation. It can also be used to support standard diagnostics when detecting bladder cancer in cases where malignancy is suspected.
The test has a CE mark in Europe and FDA 510(k) clearance in the U.S. for bladder cancer recurrence, meaning it can be marketed as substantially equivalent to another device in the U.S. market.
Nucleix is also developing a blood test for detecting lung tumors based on their DNA methylation signatures.
The company raised a $55 million funding round led by RA Capital Management in 2021, with participation from investors including BlackRock and corporate venture firm Lilly Asia Ventures. It followed up with a $22 million extension round in 2022.
In 2024, Nucleix sealed a strategic partnership with A. Menarini Diagnostics, part of the Italian Menarini Group, to bring its Bladder EpiCheck test to the European market.
5. Precede Biosciences
Founded: 2021 | Headquarters: Boston, Massachusetts
Precede was established by a team comprising Dana Farber Cancer Institute researchers and the venture capital firm 5AM Ventures.
Precede is developing blood tests that measure signals of disease based on the genomic and epigenomic characteristics of cell-free DNA shed into the blood by tumors.
For example, the company tracks gene transcription activity and DNA methylation based on as little as one milliliter of plasma. It can then use machine learning to interpret the results and predict the optimal treatment for each patient.
Precede collaborates with drugmakers to harness its technology to inform the development of next-generation radioligand therapies and antibody-drug conjugates, which depend on the knowledge of target expression and pathway activity rather than single genomic alterations.
The research-focused product Precede Bio Insight is designed to track the progress of cancer, with data spanning breast and prostate cancer.
The second product, Precede Bio Dx, also allows clinicians to select patients for clinical trials based on the blood test results.
The company emerged from stealth mode with $57 million in 2023, and followed up with a Series B round worth $83.5 million in January this year to fund the scaling of its technology as it gains commercial traction.
Among the B round’s syndicate were corporate venture investors Labcorp Venture Fund and Lilly Asia Ventures, and existing investor Illumina Ventures.
Jonathan Smith, PhD, is a freelance science journalist based in the U.K. and Spain. He previously worked in Berlin as a reporter and news editor at Labiotech, a website covering the biotech industry. Prior to this, he completed a PhD in behavioral neurobiology at the University of Leicester and freelanced for the U.K. organizations Research Media and Society of Experimental Biology. He has also written for medwireNews, Biopharma Reporter, and Outsourcing Pharma.
The post Beyond the Genome: Five Emerging Leaders in Epigenetics Diagnostics appeared first on Inside Precision Medicine.
In Ebola outbreak, a number of Americans in the Congo believed to have had exposure to suspected cases
A number of Americans who are in the Democratic Republic of the Congo are believed to have had exposure to suspected cases in the country’s latest Ebola outbreak, with several deemed to have had high-risk exposures, sources have told STAT. At least one of these individuals may have developed symptoms.
One source said that there are not yet test results for any of the individuals, but the U.S. government is reportedly trying to arrange to transport them out of the DRC to somewhere they can be safely quarantined, and cared for, if they prove to have been infected. It’s not clear if that would be in the United States; there is some discussion of perhaps taking the individuals to an American military base in Germany, a source said.
Personalized Pharmaco-Lifestyle Interventions for Severe Mental Illnesses (LIFETRAIN)
Conditions: Severe Mental Illness; Depression / Major Depressive Disorder; Bipolar Disorder (BD); Schizophrenia
Interventions: Drug: Semaglutide (SEMA); Behavioral: Exercise module; Behavioral: Anti-inflammatory diet module; Behavioral: Sleep intervention module; Behavioral: Social prescribing module; Device: Closed-loop transcranial alternating current stimulation (CL-tACS); Behavioral: Structured lifestyle psychoeducation; Device: Sham CL-tACS
Sponsors: Ludwig-Maximilians – University of Munich
Not yet recruiting
Interventions: Drug: Semaglutide (SEMA); Behavioral: Exercise module; Behavioral: Anti-inflammatory diet module; Behavioral: Sleep intervention module; Behavioral: Social prescribing module; Device: Closed-loop transcranial alternating current stimulation (CL-tACS); Behavioral: Structured lifestyle psychoeducation; Device: Sham CL-tACS
Sponsors: Ludwig-Maximilians – University of Munich
Not yet recruiting
STAT+: Top U.S. officials pressured Germany to pay more for prescription drugs
WASHINGTON — Over a recent breakfast, U.S. officials had a message for the German ambassador: pay more for pharmaceuticals.
The meeting, between U.S. Trade Representative Jamieson Greer, chief health department adviser Chris Klomp, and German Ambassador Jens Hanefeld, was part of a larger push from the Trump administration to get other countries to pay more for medications as the U.S. pays less, according to a person familiar with the meeting.
The U.S. officials discussed the possible use of tariffs under Section 301 — which grants the government authority to combat trade practices considered “unfair.” The move would be similar to the tactic of threatening new tariffs to get other countries to pay more for drugs. The ambassador agreed to review the matter with officials in Germany, but no deal was made, the person said.
PSA screening for prostate cancer reduces disease-specific deaths, new review shows
Prostate-specific antigen (PSA) blood testing is likely to reduce the risk of death from prostate cancer, found a new review published on Thursday by an influential international science research organization — a shift in medical evidence that could encourage wider use.
The Cochrane review’s first author, Juan Franco from Heinrich Heine University Düsseldorf in Germany, said at a press conference that they have “moderate certainty” that the screening test, which identifies high levels of PSA as a potential marker of prostate cancer, leads to a reduction in disease-specific deaths. The benefits are marginal: the review analyzed results from six trials involving 800,000 participants conducted in Europe and North America, and found about two fewer prostate cancer deaths for every 1,000 men screened.
Usage of the Tablet-Based “Keep On Keep Up” Digital Program and Resulting Changes in Physical Capacity and Real-World Walking in Community-Dwelling Older Adults: Process Evaluation
Background: “Keep On Keep Up” (KOKU) is a tablet-based digital program based on the well-validated Otago and Fitness and Mobility Exercise programs for older adults to decrease the risk of falling. Objective: This substudy involved a process evaluation in order to analyze the usage patterns of the KOKU digital program, specifically training frequency, volume, and intensity among older adults over a 3-month self-managed training period. Pre-post changes in physical capacity and real-world walking were examined. Methods: This study is a nested cohort study within the three-armed randomized controlled SMART-AGE trial conducted in Germany (German Clinical Trials Register ID: DRKS00034316). Participants aged 67 years or older with basic digital literacy were included. KOKU provided guided but unsupervised progressive strength and balance training for 3 months. The data on training adherence, engagement, and progression were collected. Instrumented assessments included the Timed Up and Go Test, the 30-Second Chair Rise Test, and real-world walking monitoring using wearable sensors. Results: A total of 113 participants (n=63, 56% female; mean age 74.02, SD 5.36 y) were included in the analysis. During the 3-month period, participants used KOKU for 24 (SD 15) days, that is, 2 to 3 times per week. Over the entire study period, no falls or other adverse events were reported due to KOKU usage. The number of exercises performed per participant ranged from 2 to 213, with a median value of 70. The instrumented Timed Up and Go Test results revealed a prolonged total duration (=0.26; =.009). In the instrumented 30-Second Chair Rise Test, improvements were observed in the number of completed repetitions (=0.21; =.04) and frequency of repetitions (=0.23; =.03). This was mainly due to a reduction in inactive time (=−0.60; <.001). Real-world walking parameters remained unchanged, except for a slower walking speed during walking bouts of less than 30 seconds (=0.49; <.001). All changes did not meet the criteria for minimally important differences. Conclusions: KOKU is a novel digital intervention for older adults, promoting balance and strength exercises. Physical capacity improvements were small. However, the use of instrumented assessments provided further insights into participants’ capacity and mobility that would not have been identifiable with conventional assessments. Future improvements to the program should focus on incorporating more challenging exercises for individuals with varying levels of physical capacity. Trial Registration: German Clinical Trials Register DRKS00034316; https://drks.de/search/en/trial/DRKS00034316
Antibody Promotes Nerve Regeneration in Spinal Cord Injury Patients
Swiss research shows treatment with an antibody known as NG101 promotes regeneration of damaged spinal cord tissue in some people with spinal cord injury by blocking a protein called Nogo-A that normally suppresses nerve regrowth in the central nervous system.
Compared to placebo, participants treated with the antibody showed faster lesion volume shrinkage and a significantly slower loss of spinal cord tissue, both in terms of size and myelin content, above the injury site.
This study, published in Nature Communications, is an additional analysis of a subset of 106 patients of a Phase IIb study published last year in The Lancet Neurology and carried out by Swiss and German academic researchers as well as the Swiss biotech NovaGo Therapeutics, which is developing the antibody. The original trial population included 126 people. It did not robustly meet its primary endpoint across the full patient population but showed promising signals in some motor-incomplete patients.
The current study specifically looked at people for whom magnetic resonance imaging (MRI) data were available. It investigated whether NG101 produced measurable structural changes in the spinal cord over time, whether MRI could detect those changes even in patients without obvious clinical improvement, also whether combining imaging with electrophysiology could better identify likely responders to inform more efficient future trial designs.
In the Phase IIb study the antibody was delivered directly into the spinal canal to 106 participants with acute cervical spinal cord injury within weeks of injury. Over a six month follow up period, the team measured lesion volume, spinal cord cross-sectional area, and myelin integrity.
The patients treated with NG101 showed a slowing of damage and faster injury improvement than those in the placebo group. The researchers believe this suggests the drug either slows post-injury neurodegeneration or actively promotes nerve fiber sprouting.
“Our data suggest that NG101 antibody treatment demonstrates both focal and remote structural preservation following cervical spinal cord injury, consistent with regenerative mechanisms seen in preclinical models,” write lead author Patrick Freund, MD, PhD, a professor and researcher at the University of Zurich and Balgrist University Hospital, and colleagues.
“These changes are detectable even in participants without overt functional improvement, underscoring the value of quantitative MRI in revealing treatment effects that may be missed by clinical scores alone.”
The post Antibody Promotes Nerve Regeneration in Spinal Cord Injury Patients appeared first on Inside Precision Medicine.
RESCUE- expected usefulness and willingness to participate in a trauma-informed group intervention for coping with traumatic work experiences in the emergency medical services
Emergency Medical Services personnel (EMS) are confronted with potentially highly stressful and traumatic occupational experiences, placing them at high-risk for mental disorders. Avoidant coping mechanisms, internalized and occupational stigma not only impede disclosure of and processing the incident-related stress but also weaken the overall resilience of the EMS. Effective interventions are needed that operate both at the individual and group level. The aim of this study was to investigate the expected usefulness and willingness to participate in a trauma-informed intervention (Facts derived from Narrative Exposure Therapy, NETfacts). A total of 256 German EMS (67.19% men, 32.81% women) participated in the online survey. We assessed the expected usefulness and willingness to participate in NETfacts, critical incident-related stress (EMS Critical Incident Inventory EMS-CII), burnout symptoms (Professional Quality of Life ProQOL), age and work experience. Overall, about half of the EMS expected NETfacts to be at least somewhat useful, and reported a generally willingness to participate. Expected usefulness was neither associated with critical incident-related stress nor burnout symptoms. Younger participants (<35 years) showed a generally higher willingness to participate than older participants. However, the willingness is across both age groups positively associated with higher levels of critical incident-related stress. Nevertheless, burnout symptoms and the willingness are negatively associated among participants age 35 and older, while remaining stable among their younger colleagues. Early, trauma-informed and age-sensitive prevention programs are needed to mitigate the adverse effects of critical incidents among EMS. Our study presents EMS preferred circumstances to enhance employees’ uptake of such a program.