Latest news with #clinical trials
Yahoo
9 hours ago
- Business
- Yahoo
Leerink Partners Affirms Outperform Rating on AbCellera Biologics (ABCL) Following Pivot
AbCellera Biologics Inc. (NASDAQ:ABCL) is one of the top high-return penny stocks to buy now. On July 7, Leerink Partners resumed coverage of the stock with an 'Outperform' rating and a $5 price target. The research firm remains confident about the company's long-term prospects following its pivot from an antibody discovery platform to one that develops its internal pipeline. A close-up shot of various types of medicines on a table, illustrating the specialty and generic products offered by the pharmaceutical company. Leerink Partners is especially bullish about the company's upcoming programs ABCL575, an OX40L antagonist targeting atopic dermatitis, and ABCL635, an NK3R antagonist for vasomotor symptoms. The two programs are scheduled to enter Phase 1 clinical trials in the third quarter of 2025. The research firm also pointed out that AbCellera's existing antibody discovery collaborations are well-positioned to deliver long-term value. It expects the company to benefit from milestone payments and royalties as it leverages antibody capabilities towards a pipeline focused on validated targets. AbCellera Biologics Inc. (NASDAQ:ABCL) is a biotechnology company that focuses on discovering and developing antibody-based medicines. It leverages a proprietary, AI-powered technology platform to search, decode, and analyze natural immune systems, identifying antibodies that can be developed into potential drugs. While we acknowledge the potential of ABCL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 Best Chemical Stocks to Buy According to Billionaires and 7 Most Undervalued Pot Stocks To Buy According To Analysts. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Medscape
2 days ago
- Health
- Medscape
TNT Shows Similar Efficacy Across Regimens in Rectal Cancer
TOPLINE: In a multicentre study of patients with locally advanced rectal cancer, substantial variation existed in the choice of total neoadjuvant therapy (TNT), but efficacy was comparable across different regimens and consistent with that reported in clinical trials. METHODOLOGY: Researchers conducted an international, multicentre study in 21 countries and included 1585 patients (median age, 61 years; 37.1% women) with stage II/III rectal adenocarcinoma from September 2012 to December 2023. The primary objective focused on the type of TNT administered depending on the regimen, timing and type of chemotherapy, and type of radiotherapy. Secondary objectives encompassed safety and efficacy overall and on the basis of the type of TNT after propensity vector matching. Efficacy endpoints included pathologic complete response, complete response, local or distant progression at the time of treatment failure, event-free survival (EFS), and overall survival (OS). Overall, 17.7%, 33.4%, 12%, and 16.2% of patients were treated according to PRODIGE 23-like, RAPIDO-like, OPRA induction-like, and OPRA consolidation-like regimens, respectively. TAKEAWAY: Chemotherapy was given as induction, consolidation, and sandwich for 34.5%, 51.0%, and 14.5% of patients, respectively; regimens were single agent (1.1%), doublet (78.8%), and triplet (20.1%). Radiotherapy was delivered as short-course radiotherapy in 37.2% and long-course chemoradiotherapy in 62.8% of cases. The pathologic complete response rate was 21.3%, and the complete response rate was 23.2%; local and distant progression at the time of treatment failure were 7% and 16.2%, respectively. Three-year EFS reached 68%, and 5-year OS was 79%. In the overall population, PRODIGE 23-like regimens showed better survival outcomes than RAPIDO-like regimens (EFS: hazard ratio [HR], 0.68; P = .03; OS: HR, 0.51; P = .04), OPRA induction-like regimens (EFS: HR, 0.66; P = .04; OS: HR, 0.35; P = .003), and OPRA consolidation-like regimens (EFS: HR, 0.64; P = .02; OS: HR, 0.50; P = .05). After the propensity vector matching analysis of 928 patients (58.5%), no significant differences in survival outcomes were observed between TNT regimens. IN PRACTICE: "This case series study illuminates the applicability of TNT to clinical practice," the authors of the study wrote. "TNT decisions should be made based on the individual risk profile and following an accurate discussion about the positives and negatives of each option while considering patient preferences and expectations," they added. SOURCE: This study was led by Alessandro Audisio, MD, Université libre de Bruxelles, Institut Jules Bordet-Hôpital Erasme, Brussels, Belgium. It was published online on July 10, 2025, in JAMA Oncology. LIMITATIONS: The retrospective design of the study introduced potential data collection errors and biases, which were only partially addressed through remote monitoring and data imputation. The relatively short follow-up period may have prevented the detection of differences in long-term outcomes between TNT regimens. Additionally, variations in treatment delivery, staging methods, and supportive care across institutions complicated direct comparisons. Despite involving multiple countries, the predominant European patient population limited the generalisability of the results. DISCLOSURES: This study was sponsored by the Institut Jules Bordet and endorsed by the Oncodistinct Network. Several authors reported receiving personal fees and grants and having other ties with various sources. Additional disclosures are noted in the original article. This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
Reuters
4 days ago
- Health
- Reuters
Exclusive: Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys
July 18 (Reuters) - U.S. regulators asked Sarepta Therapeutics (SRPT.O), opens new tab on Friday to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died, but the company said it would not do so. The Food and Drug Administration announced the move, confirming an earlier Reuters report, after making the request at a meeting with Sarepta on Friday. After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy to ambulatory people but maintain a halt it implemented June 15 for non-ambulatory patients after reporting to the FDA a case of acute liver failure in a patient who could not walk. Sarepta said it made the decision "based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population." While the 51-year-old man with limb girdle muscular dystrophy who died most recently was not taking Elevidys, his experimental therapy and Elevidys are based on similar gene technology, the FDA said. The FDA said it was putting clinical trials for limb girdle muscular dystrophy on hold due to safety concerns. Elevidys received traditional approval in 2024 for patients age 4 and older with the Duchenne muscular dystrophy gene mutation who can walk, as well as accelerated, conditional approval for those with the muscle-wasting disease who cannot, even though the therapy failed to meet the main goal in a late-stage trial. In another setback, the regulator also revoked the platform technology designation for Sarepta's gene therapy, a status that can streamline regulatory review and that is given when a technology has promise across multiple indications. Sarepta shares ended down 36% at $14.08. At one point on Friday, the stock tumbled over 40% to a more than nine-year low after the company disclosed the third patient death. The agency increased scrutiny of Sarepta when two teenage boys receiving Elevidys died this year. All three of the deaths were caused by acute liver failure and occurred in non-ambulatory patients. The FDA said it is continuing to investigate the risk of acute liver failure with serious outcomes, including hospitalization and death, with gene therapies using Sarepta's AAVrh74 platform technology. Wall Street analysts have said the third death could make patients more hesitant to use Elevidys. Patient groups said the developments around Elevidys have been concerning. "Families with Duchenne muscular dystrophy are grappling with a mix of disappointment, uncertainty about choices they are making for their own children or themselves," said Debra Miller, founder of the non-profit CureDuchenne. On Wednesday, Sarepta said it was working with the FDA to add a warning label about liver toxicity risks to Elevidys' packaging. On an investor call on Friday, analysts asked Sarepta why it had not disclosed the latest patient death on Wednesday, when it announced 500 layoffs and cuts to its limb-girdle muscular dystrophy program, citing financial reasons. CEO Doug Ingram said the matter was "neither material, nor central" to Wednesday's update and the decision to end the limb-girdle muscular dystrophy gene therapy study was made independently of the patient death. The company also said liver issues were not a new safety signal in the study. However, some analysts, including those at BMO Capital Markets, warned that Sarepta's handling of the disclosure could damage management credibility. At least two analysts asked whether other deaths had occurred in Sarepta's gene therapy programs. The company said it was not aware of any beyond the three that were disclosed.
ABC News
5 days ago
- Health
- ABC News
Genetically modified bacteria tested in humans stayed in guts of some people
A study in the US trialling genetically modified gut bacteria in humans has had a potential escapee, after the microbe mutated. The team of researchers at Stanford University undertook early clinical trials of a strain of a common gut bacterium, which they genetically engineered to help prevent kidney stones. But the results of the trial, published today in Science, were mixed. While the therapy was mostly successful in healthy volunteers, it didn't treat the underlying cause of kidney stones in people who suffered from the condition. The bacterium also overstayed its welcome in two healthy volunteers, despite the scientists' best efforts to remove it. Weston Whitaker, a Stanford University microbiologist who led the study, said all the protocols were followed, and "there were no specific reasons to worry about the health of individuals where the bacterium persisted". "Many aspects of the technology worked surprisingly well, and we've clearly identified parts needing further development," Dr Whitaker said. "I think we showed that there is promise in continuing this approach." Sam Forster, a microbiologist at the Hudson Institute of Medical Research who was not involved with the research, said the findings showed both the hazards and potential of the technology. "There are risks associated with these approaches but it's also an amazingly powerful technology and these types of studies provide a key fundamental understanding," Dr Forster said. The researchers genetically modified a strain of bacterium called Phocaeicola vulgatus to carry the genes they were looking for. "We chose Phocaeicola vulgatus because it is one of the most prevalent and abundant bacteria in the gut," Dr Whitaker said. The team engineered the bacterium to have two new abilities: breaking up a compound called oxalate, which can cause kidney stones, and eating a compound called porphyran, which is found in seaweed. Humans and most other gut bacteria can't process porphyran, so this gave the modified microbe a reliable source of food — as long as the trial participants consumed a porphyran supplement. It also provided a handy way to get rid of bacteria once the experiment was over: trial participants could just stop taking porphyran. Or at least that was the theory. After testing the modified microbe on mice and rats, the researchers ran two human trials — one with 39 healthy volunteers, and another with 20 volunteers who had a condition called enteric hyperoxaluria, where the body absorbs too much oxalate, causing frequent kidney stones. The trial participants were either given a pill full of the bacterium or a placebo to swallow. The researchers found the modified microbe could safely colonise healthy participants' guts, and reduce their oxalate levels. And for most participants, the bacteria vanished after they stopped taking their porphyran doses. But it lingered in four healthy people even when they'd dropped the seaweed supplements. These study participants were treated with antibiotics, which successfully removed the modified microbe in two people — but it stayed put in the other two. While this is a novel situation, Dr Whitaker said there was no reason to be concerned. "The genes, bacteria, and activities we introduced are commonly found in a healthy gut, so we considered this a relatively safe initial application," he said. Engineered bacteria that stuck around in the gut in the healthy participants were successful because they mutated to eat foods other than porphyran. So when the porphyran was removed, the gut bug just began eating something else. The microbe also mutated to become less effective at degrading oxalate in some participants in the kidney stone group. Bacteria are able to swap genes with each other, which gives them an extremely quick way to evolve new characteristics. According to Dr Whitaker, the team knew it was possible the modified microbe could mutate but were "surprised it occurred" in the way it did, because it had been much less of an issue with lab studies or healthy volunteers. Dr Forster said this was a known issue of working with bacteria. "These bacteria exchange DNA all the time. And most of those exchanges are just as likely to be beneficial for us as detrimental to us." "[DNA exchange] is not a characteristic of this strain or this technology, it's a characteristic of bacteria." While this clinical trial was terminated by the team, both Dr Whitaker and Dr Forster were excited by the prospects this could provide in the future. The Stanford team has now designed a new bacterial strain that has three essential genes, which he said would provide a "triple safeguard" against mutation. The team is yet to test this new strain in patients, but when the researchers tested the bacteria in the lab, it was unable to bypass the protection provided by the addition of the genes. Dr Forster said the paper highlighted how bacteria could be used to treat inflammatory gut disorders, and even gut cancers. "In some cases there will already be species that can [be used as a therapy], and so there would be advantages to putting those natural species back in," he said. "But in some cases … genetically modifying them provides a much more efficient way of providing that therapy." "This paper is is a key step in that process."
Medscape
11-07-2025
- Health
- Medscape
MCED Screening: What Patients Need to Know
With half a million people having already received multi-cancer early detection (MCED) screening, patients are and will continue to be asking about these blood tests during their visits, physicians say. During an Education Session at the American Society of Clinical Oncology (ASCO) 2025, several experts on these tests advised on how to approach these conversations with patients and described what they think is worth sharing about them. Front and center for the panelists of the session entitled, 'Multicancer Early Detection Testing: Are There Cures Without Costs?' Was that there are not yet any randomized clinical trial results showing that a positive MCED test result will actually decrease cancer mortality or even morbidity. 'We need to see trials that show something has changed for the better for the patient. Does it really move the needle for that patient, or are we just finding [the cancer] earlier?' said Jennifer Litton, MD, professor, vice president of clinical research, and interim chair of breast medical oncology at The University of Texas MD Anderson Cancer Center in Houston. 'Are we improving survival, improving treatment, or improving toxicity?' she asked, during her presentation in the session. Beyond the physical toxicity of treatments, there is also financial toxicity to consider. Although there are several MCED tests in the pipeline, GRAIL's Galleri test is the only one currently on the market. Studies of Galleri and other MCED tests still in development have so far shown that these blood-based screenings can, indeed, detect circulating tumor DNA (ctDNA) in patients who are asymptomatic, often suggesting cancer long before regular screening tests confirm it. But, thus far, there is no evidence that earlier detection saves lives, or can allow for less aggressive treatment. While Carmen Estela Guerra, MD, the chair of the session, acknowledged that clinicians are not obligated to initiate discussions about MCEDs until more is known about their effectiveness, as guidance from the American Cancer Society states, she provided starting points and resources for discussing these tests with patients. 'It's really important…to understand that patients may hear about Galleri tests, as will primary care physicians that might consult with us. And so we have to consider…that once people approach us, patients, primary care clinicians, that we really take this opportunity…to make sure that patients are up to date with evidence-based cancer screenings, whatever cancer screenings are appropriate for their age, sex, family history, tobacco history, and other risk factors,' said Guerra at the meeting. She added that if patients inquire about MCEDs, clinicians first want to determine if they are even eligible for testing. Guerra, who is professor of medicine at the University of Pennsylvania, and a general internist and cancer equity researcher at the Abramson Cancer Center, both in Philadelphia, also provided answers to several other questions patients may ask about the Galleri and other MCED tests during her presentation. Who is Eligible to Receive the Galleri Test? According to the Galleri website, anyone older than 50 years is eligible to receive these tests. 'Some experts have said that perhaps other people who might be eligible are those with family histories or personal histories of cancer in the past. And then, perhaps, even people with known genetic mutations, who are at risk for multiple cancers, may be eligible individuals,' Guerra said. 'Again, this is expert opinion. And you may read about these potential eligibility [criteria]. But the truth is, we just don't know yet.' Who Can't Receive This Screening? 'Some people have proposed pediatric populations, pregnant individuals, and patients who have had a cancer diagnosis within the past 3 years are not eligible for MCED testing,' Guerra said. 'In fact, they were excluded from the [ongoing] NHS-Galleri study.' An MCED consortium was created, which was a private, public partnership between the MCED companies, the American Cancer Society, and many other experts. This provided a list of additional risk factors 'that, perhaps in the future, may also help us identify who is a potential candidate for MCED testing,' she continued. That list, which was published in an article in JCO Precision Oncology in November 2021, includes: Using alcohol Having been exposed to cancer-causing substances (eg, fire smoke, tobacco smoke, radiation, and sunlight) Being immunosuppressed Having been exposed to infectious agents (eg, viruses and parasites) Having Overweight/Obesity Using Tobacco How to Approach Discussions With Patients and Clinicians One of the approaches that's being adapted to clinicians discussing MCEDs with patients and other clinicians is the shared decision framework, Guerra explained. That shared decision framework emphasizes that MCEDs are not replacements for evidence-based cancer screening, but that they could be additive in some cases, she said. The shared decision model is a talk model approach. 'It starts with the option or choice talk. And that's about basic education about MCEDs, what they detect, who is eligible, which tests are available, and how much does it cost,' she said. The option talk involves addressing what the potential benefits and harms are, the likelihood of harm, interpretation of the tests, test uncertainties, and the option of not testing at all, Guerra continued. Finally, to follow this model, clinicians help patients decide whether to have the testing. Part of this includes recognizing that 'some patients, after hearing all this, may not want to have the test. And that's OK, too,' she said. Guerrera also pointed out that other option- and decision-related talk points for approaching these conversations were published in April in the American Society of Clinical Oncology Education Book . She and her colleagues coauthored this article. One example of a 'decision talk' point in the article aimed at answering how to decide whether to have these tests is that 'MCED tests are not a recommended part of your routine cancer screening at this time.' Another is: 'If you are unsure about getting tested, it may be best to wait. We can always revisit your decisions as more information becomes available.' What are the Advantages and Disadvantages of the Tests? During her presentation, Guerra also described some of the advantages and disadvantages to MCED screening that clinicians could speak about with their patients. 'The advantages are, obviously, that screening can occur for multiple cancers at the same time, and that MCEDs can detect cancers [for which we currently don't have a modality], and that MCEDs have increased positive predictive value compared to single cancer screening tests,' she said. 'But you can see there's a lot of potential disadvantages, at the moment. Much of this is because we don't have information to inform our patients, including that patients should know that MCEDs are not currently covered by insurance,' Guerra continued. 'In addition, there's no consensus or guidelines for who should be tested and what the best testing pathway is for a positive MCED. That even if they have a 'no cancer signal' detected, it does not rule out future cancer diagnoses.' With these tests, 'consequential cancers could be found early, but the patient may not live longer because of overdiagnosis,' she said. More potential disadvantages to these tests, which Guerra included in a slide for her presentation, included: Possible harm from unnecessary diagnostic procedures due to false positives or missed diagnoses due to false negatives Overdiagnosis and overtreatment of cancers that would have otherwise never bothered a patient Increased inequities if tests are not widely available, affordable, and acceptable to minority groups What's Missing and What to Offer Patients Who Want an MCED Test? 'One of the things that's really missing to guide clinicians right now is what to do with those positive cancer signals. And there is no document that has some guidance for any of us, or even our oncologists because many clinicians, primary care clinicians, will refer those patients to oncologists,' Guerra said. 'Given the uncertainties about MCEDs, one of the options that we might offer our patients that want to have an MCED is a recommendation to enroll [in one of the MCED studies], where they will be tracked as part of the clinical trial, and where outcomes will be evaluated and inform future practice.' Among these trials is the Vanguard study, a National Cancer Institute study that is being designed across nine geographical areas, that will enroll 24,000 participants in the US. The first two tests that will be used in this trial are Shield and Avantect, and others may be used as well, according to Guerra. The objective of the Vanguard study is to determine if patients will be willing to even be randomized to this study and determine adherence and feasibility questions about the study, that will later inform a larger study, she said. Another trial patients can enroll in is the REACH initiative, which is a collaboration between Grail and Medicare that will prospectively look at 50,000 Medicare beneficiaries to compare patients receiving usual care with those receiving the Galleri test. The investigators for this study 'will try to answer questions about whether there are reduced diagnoses of late stage, stage IV, and the safety and healthcare utilization,' Guerra said. This is a 3-year study across 50 sites that was initiated about 2 years ago. More Information About Galleri The Galleri test has a list price of $949 and is available only by prescription. It's designed to flag the possibility of up to 50 different cancers by detecting ctDNA in a blood sample. Because Galleri is not approved by the FDA, most insurance would not cover it or any follow-ups prompted by a positive result, according to the American Cancer Society. GRAIL has applied for Breakthrough Device Designation and Pre-Market Approval for Galleri. The company is currently authorized to perform the test at its laboratory, which is certified by the Centers for Medicare & Medicaid Services under the Clinical Laboratory Improvement Amendments of 1988. Litton disclosed financial relationships with UpToDate, Physicans' Education Resource, Merck, Pfizer, and Certis. Guerra reported financial relationships with BEAM Therapeutics, Crispr Therapeutics, Editas Medicine, Intellia Therapeutics, National Comprehensive Cancer Network, GlaxoSmithKline, Guardant Health, Impulse, Natera, Roche, Janssen, and Genentech. Kate Johnson contributed to this report.
