Sarepta Reports New Elevidys Data from Two Separate Studies in Duchenne Muscular Dystrophy
Sarepta Therapeutics (SRPT) said Friday that new data from two separate studies of its gene therapy
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35 minutes ago
- Yahoo
Circle Pharma Receives FDA Orphan Drug Designation for CID-078 for the Treatment of Small Cell Lung Cancer
SOUTH SAN FRANCISCO, Calif., June 16, 2025--(BUSINESS WIRE)--Circle Pharma, a clinical-stage biopharmaceutical company advancing macrocycle therapeutics for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CID-078 for the treatment of small cell lung cancer (SCLC). Small-cell lung cancer is a highly aggressive form of lung cancer that accounts for approximately 13–15% of all lung cancer cases1 and is strongly linked to tobacco exposure. Despite existing treatments, SCLC has a high recurrence rate and is associated with poor overall prognosis. While improvements in overall survival are occurring with newer therapies, most patients experience rapid disease progression2. "The Orphan Drug Designation from the FDA underscores both the seriousness of small cell lung cancer and the lack of effective treatment options," said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development Circle Pharma. "We are committed to accelerating the clinical development of CID-078 to offer new hope for patients who face limited therapeutic choices." The FDA's Orphan Drug Designation program is intended to promote the development of drugs for rare diseases or conditions affecting fewer than 200,000 people in the United States3. This designation provides several development incentives, including seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trial costs, and eligibility to apply for FDA-administered research grants4. Circle Pharma has initiated a Phase 1 clinical trial (NCT06577987) of CID-078 to evaluate its safety, tolerability, pharmacokinetics, and early signs of anti-tumor activity in patients with advanced solid tumors, including SCLC. About CID-078, Circle Pharma's Cyclin A/B RxL Inhibitor Program CID-078 is an orally bioavailable macrocycle with dual cyclin A and B RxL inhibitory activity that selectively targets tumor cells with oncogenic alterations that cause cell cycle dysregulation. In biochemical and cellular studies, Circle Pharma's cyclin A/B RxL inhibitors have been shown to potently and selectively disrupt the protein-to-protein interaction between cyclins A and B and their key substrates and modulators, including E2F (a substrate of cyclin A) and Myt1 (a modulator of cyclin B). Preclinical studies have demonstrated the ability of these cyclin A/B RxL inhibitors to cause single-agent tumor regressions in multiple in vivo models. A multi-center phase 1 clinical trial (NCT06577987) is currently enrolling patients. About Circle Pharma, Inc. South San Francisco-based Circle Pharma is a clinical-stage biopharmaceutical company harnessing the power of macrocycles to develop therapies for cancer and other serious illnesses. The company's proprietary MXMO™ platform overcomes key challenges in macrocycle drug development, enabling the creation of intrinsically cell-permeable and orally bioavailable therapies for historically undruggable targets. Circle Pharma's pipeline is focused on targeting cyclins, key regulators of the cell cycle that drive many cancers. Its lead program, CID-078, a cyclin A/B-RxL inhibitor, is in a Phase 1 clinical trial (NCT06577987) for patients with advanced solid tumors. To learn more about Circle Pharma, please visit American Cancer Society. What is Small Cell Lung Cancer? National Cancer Institute. Small Cell Lung Cancer Treatment (PDQ®)–Patient Version. U.S. Food and Drug Administration. Developing Products for Rare Diseases & Conditions. U.S. Food and Drug Administration. Benefits of Orphan Drug Designation. View source version on Contacts Media Contact: Roslyn PattersonPhone: 650.825.4099Email:
Yahoo
35 minutes ago
- Yahoo
Sleep loss rewires the brain for cravings and weight gain – a neurologist explains the science behind the cycle
You stayed up too late scrolling through your phone, answering emails or watching just one more episode. The next morning, you feel groggy and irritable. That sugary pastry or greasy breakfast sandwich suddenly looks more appealing than your usual yogurt and berries. By the afternoon, chips or candy from the break room call your name. This isn't just about willpower. Your brain, short on rest, is nudging you toward quick, high-calorie fixes. There is a reason why this cycle repeats itself so predictably. Research shows that insufficient sleep disrupts hunger signals, weakens self-control, impairs glucose metabolism and increases your risk of weight gain. These changes can occur rapidly, even after a single night of poor sleep, and can become more harmful over time if left unaddressed. I am a neurologist specializing in sleep science and its impact on health. Sleep deprivation affects millions. According to the Centers for Disease Control and Prevention, more than one-third of U.S. adults regularly get less than seven hours of sleep per night. Nearly three-quarters of adolescents fall short of the recommended 8-10 hours sleep during the school week. While anyone can suffer from sleep loss, essential workers and first responders, including nurses, firefighters and emergency personnel, are especially vulnerable due to night shifts and rotating schedules. These patterns disrupt the body's internal clock and are linked to increased cravings, poor eating habits and elevated risks for obesity and metabolic disease. Fortunately, even a few nights of consistent, high-quality sleep can help rebalance key systems and start to reverse some of these effects. Your body regulates hunger through a hormonal feedback loop involving two key hormones. Ghrelin, produced primarily in the stomach, signals that you are hungry, while leptin, which is produced in the fat cells, tells your brain that you are full. Even one night of restricted sleep increases the release of ghrelin and decreases leptin, which leads to greater hunger and reduced satisfaction after eating. This shift is driven by changes in how the body regulates hunger and stress. Your brain becomes less responsive to fullness signals, while at the same time ramping up stress hormones that can increase cravings and appetite. These changes are not subtle. In controlled lab studies, healthy adults reported increased hunger and stronger cravings for calorie-dense foods after sleeping only four to five hours. The effect worsens with ongoing sleep deficits, which can lead to a chronically elevated appetite. Sleep loss changes how your brain evaluates food. Imaging studies show that after just one night of sleep deprivation, the prefrontal cortex, which is responsible for decision-making and impulse control, has reduced activity. At the same time, reward-related areas such as the amygdala and the nucleus accumbens, a part of the brain that drives motivation and reward-seeking, become more reactive to tempting food cues. In simple terms, your brain becomes more tempted by junk food and less capable of resisting it. Participants in sleep deprivation studies not only rated high-calorie foods as more desirable but were also more likely to choose them, regardless of how hungry they actually felt. Sleep is also critical for blood sugar control. When you're well rested, your body efficiently uses insulin to move sugar out of your bloodstream and into your cells for energy. But even one night of partial sleep can reduce insulin sensitivity by up to 25%, leaving more sugar circulating in your blood. If your body can't process sugar effectively, it's more likely to convert it into fat. This contributes to weight gain, especially around the abdomen. Over time, poor sleep is associated with higher risk for Type 2 diabetes and metabolic syndrome, a group of health issues such as high blood pressure, belly fat and high blood sugar that raise the risk for heart disease and diabetes. On top of this, sleep loss raises cortisol, your body's main stress hormone. Elevated cortisol encourages fat storage, especially in the abdominal region, and can further disrupt appetite regulation. In a culture that glorifies hustle and late nights, sleep is often treated as optional. But your body doesn't see it that way. Sleep is not downtime. It is active, essential repair. It is when your brain recalibrates hunger and reward signals, your hormones reset and your metabolism stabilizes. Just one or two nights of quality sleep can begin to undo the damage from prior sleep loss and restore your body's natural balance. So the next time you find yourself reaching for junk food after a short night, recognize that your biology is not failing you. It is reacting to stress and fatigue. The most effective way to restore balance isn't a crash diet or caffeine. It's sleep. Sleep is not a luxury. It is your most powerful tool for appetite control, energy regulation and long-term health. This article is republished from The Conversation, a nonprofit, independent news organization bringing you facts and trustworthy analysis to help you make sense of our complex world. It was written by: Joanna Fong-Isariyawongse, University of Pittsburgh Read more: Late bedtimes and not enough sleep can harm developing brains – and poorer kids are more at risk Screen time is contributing to chronic sleep deprivation in tweens and teens – a pediatric sleep expert explains how critical sleep is to kids' mental health Daylight saving time and early school start times cost billions in lost productivity and health care expenses Joanna Fong-Isariyawongse does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.
Yahoo
38 minutes ago
- Yahoo
J&J reports results from antibody combo trial for MM patients
Johnson & Johnson (J&J) has reported new outcomes from the Phase II RedirecTT-1 trial of bispecific antibodies, Talvey (talquetamab-tgvs) and Tecvayli (teclistamab-cqyv) for relapsed/refractory multiple myeloma (r/r MM). The data showed a high overall response rate (ORR) with durability in those who have triple-class-exposed (TCE) RRMM with true extramedullary disease (EMD). In the trial, which enrolled 90 subjects, the investigational combo resulted in an ORR of 78.9%, with over half of the subjects achieving a complete response or better, representing a significant improvement over the average ORR of less than 40% for this patient group. Notably, responses were also found to be high among those previously treated with B-cell maturation antigen (BCMA) CAR-T or anti-FcRH5 bispecific antibodies. According to the company, subjects in the trial showed deep and durable responses, with 66.2% remaining in response at the data cutoff and a median follow-up of 13.4 months. At one year, 61% of subjects were progression-free and alive, and 74.5% were alive, with median overall survival not yet reached. The combo was found to be consistent with prior reports of them as single agents. Subjects had the option to switch to once-a-month dosing, which might have contributed to better tolerability. The reports of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were found to be mostly low grade. The study's findings were featured at the 2025 European Hematology Association Congress. EMD represents a severe form of MM, where myeloma cells form tumours in soft tissues and organs. Johnson & Johnson innovative medicine multiple myeloma disease area leader and vice-president Jordan Schecter said: 'Patients with extramedullary myeloma, especially those who have exhausted prior therapies, need more effective treatment options. 'Our first-in-class bispecific antibodies, Talvey and Tecvayli, have transformed treatment for relapsed or refractory multiple myeloma.' Recently, J&J reported that Tremfya decreased the symptoms and signs of active psoriatic arthritis (PsA) at 24 weeks in individuals against a placebo in the Phase IIIb APEX trial. "J&J reports results from antibody combo trial for MM patients" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. 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
