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CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.
Unlike drugs that alter metabolism or appetite by interacting with hormones, the pill functions entirely mechanically. In Oxford Medical’s first-in-human trials, conducted in a diverse patient group, data shows that the pill is well-tolerated, with no significant adverse events reported.
Eneboparatide (AZP-3601), an investigational parathyroid hormone 1 receptor (PTH1R) agonist, met its primary endpoint in the CALYPSO Phase III trial, offering new data for adults with chronic hypoparathyroidism. AstraZeneca received eneboparatide during its $1.05
Sling Therapeutics has positive topline results from its Phase IIb/III LIDS clinical trial evaluating its oral small-molecule therapy linsitinib for the treatment of thyroid eye disease (TED). Ninety patients in the trial were randomized to receive one of two doses of linsitinib or placebo.
The mutation leads to disordered thyroid hormone transport, which causes patients to experience intellectual and motor disabilities. Egetis is banking on tiratricol, an oral small molecule that restores dysfunctional thyroid hormone signalling, to be the first drug approved for this condition. In clinical trials, the drug showed a 3.8-point
a clinical-stage biotech company focused on developing next-gen injectable and oral nutrient-stimulated hormone (NuSH) analog peptides for the treatment of obesity, overweight and related conditions, has priced its initial public offering (IPO) at $18.00 If successful, Phase III trials are expected to follow shortly thereafter.
Dr. Neal-Perry spoke about some of the latest trial data for fezolinetant that was presented at the recent Annual Meeting of The Menopause Society. This includes the effect of the drug on moderate-to-severe vasomotor symptoms in subgroups based on hormone therapy history from pooled data from two randomized Phase III studies.
Incytes recent announcement on the topline results from two Phase III clinical trials of povorcitinib in patients with hidradenitis suppurativa has stirred both hope and caution in the market. These promising figures had set high expectations for subsequent trials. The other Phase III trial, STOP-HS2, showed a 42.3% for placebo.
In the gut, these receptors prompt the release of hormones such as cholecystokinin and GLP-1, which together signal satiety. In a Phase II trial involving patients with hyperphagia, ARD-101 was well tolerated and achieved an approximate eight-point reduction in hunger scores. The competitive landscape is equally dynamic.
billion, primarily linked to initiating Phase III trials for petrelintide as a stand-alone therapy, with sales-based milestones that could push the total value to $5.3 Amylin, a hormone co-secreted with insulin, plays a key role in regulating appetite by enhancing leptins effects the hormone that signals fullness after eating.
Since the FDA approved recombinant human growth hormone (rhGH) for PWS in 2000, multiple companies have tried and failed to develop a targeted treatment for hyperphagia. Vykay XR Clinical Trial Results Data from a randomized, placebo-controlled Phase III trial (Study 2-RWP or Study C602-RWP) support Vykat XRs approval.
Sweden-based biotech Camurus shared positive Phase III trial data for CAM2029, one of its lead candidates, in patients with the rare hormonal disorder acromegaly. Due to its ability to mimic somatostatin, octreotide is used to treat various conditions associated with excessive hormone production.
The thyroid plays a critical role in the body by producing hormones that regulate metabolism, heart rate and body temperature among others. Types of Thyroid Conditions There are several different thyroid conditions, with each affecting thyroid hormone production or the gland’s structure, leading to a range of health issues.
Amgen announced positive results from a Phase II clinical trial evaluating its investigational obesity drug MariTide (maridebart cafraglutide, formerly AMG 133) for the treatment of obesity and overweight in both diabetic and non-diabetic patients. The trial included 465 participants who were either obese or overweight.
VK2809 is a novel, oral small molecule thyroid hormone receptor beta agonist that has selectivity for liver tissue. VK2809 and the VOYAGE Trial. Results from the trial showed that patients that were administered VK2809 demonstrated a statistically significant 45.4 Related: Six of the Latest NASH/NAFLD Webinars. mg daily; 2.5
Zealand Pharma has commenced its global Phase IIb ZUPREME-1 trial, enrolling the first participant to evaluate petrelintide, a long-acting amylin analog, as a potential investigational treatment for people with overweight or obesity. And among its many obesity drugs in clinical trials, Eli Lillys amylin analog eloralintide is in Phase II.
In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Symptoms and Etiology: Caused by an overproduction of growth hormone, acromegaly primarily affects adults. Acromegaly. Prevalence: 50 to 70 people per 1 million.
Amgen announced positive results from a Phase II clinical trial evaluating its investigational obesity drug MariTide (maridebart cafraglutide, formerly AMG 133) for the treatment of obesity and overweight in both diabetic and non-diabetic patients. The trial included 465 participants who were either obese or overweight.
Novartis has spent three decades advancing breast cancer treatments, focusing on hormone receptor-positive (HR+), HER2-negative breast cancer, the most common subtype. Last year, the FDA approved Kisqali (ribociclib) to reduce the risk of recurrence in HR+/HER2- early breast cancer. Novartis dedicated approximately $0.47
Eli Lilly announced that its obesity drug tirzepatide has scored favorably in a late-stage clinical trial, with results showing that people who took the drug lost an average of 50 pounds, or 21 percent, of their body weight compared to placebo. percent and 22.5 percent, respectively, compared to placebo.
Related: Eli Lillys Zepbound Approved as First Treatment for Obstructive Sleep Apnea The commercial describes Zepbounds mechanism of action as a dual receptor against that works by activating two naturally occurring hormone receptors in [the] body [GLP-1 and GIP-1]. Its changing how much weight I lose, up to 48 lbs, he says.
While the exact physiology of vasomotor symptoms is not fully understood, it is believed that the significant fluctuations in reproductive hormones play a role in triggering these symptoms. Hormone replacement therapy is thought to be one of the most effective treatments for vasomotor symptoms. How Does Veozah Work?
GlaxoSmithKline (GSK) unveiled positive safety and efficacy data for its oral anemia drug daprodustat from five pivotal trials for the drug last week. Two of the five trials focused on non-dialysis (ASCEND-ND) and dialysis (ASCEND-D) patients, and detailed data from these trials was shared at the nephrology meeting.
Pediatric growth hormone deficiency (PGHD) is one such condition, affecting a child’s growth and development. This rare endocrine disorder, characterized by inadequate secretion of growth hormone from the pituitary gland, has profound impacts on physical and sometimes cognitive development.
Optimizing Sleep During Perimenopause and Beyond Sleep plays a crucial role in overall health, particularly for midlife women transitioning into menopause, a phase marked by perimenopause where hormonal changes can severely disrupt sleep due to symptoms like nocturnal hot flashes.
Insulin, a hormone produced by the pancreas, allows glucose to enter cells for energy. Merilogs approval is supported by a Phase III trial, which included 597 patients with type 1 and type 2 diabetes who received several shots of the insulin aspart biosimilar every day. Approximately 8.4
A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. In the trial, the vaccine was injected directly into the lymph nodes of type 1 diabetic individuals. The DIAGNODE-2 Trial.
David Cameron, Chair of the Breast International Group (BIG) , we delve into the landscape of breast cancer research today, clinical trials and the incorporation of the patient voice in breast cancer research. Breakthroughs in Treatment BIG has been at the forefront of numerous clinical trials.
Erythropoietin is a hormone that is produced by the kidneys and signals the body to make red blood cells. Clinical Trial Outcomes The FDA approval of Jesduvroq is based on results from the Phase III ASCEND-D trial , which were published in the New England Journal of Medicine. How Does Jesduvroq ( daprodustat) Work?
This funding infusion is poised to support Septerna’s expanding pipeline and ongoing clinical trials within its core therapeutic areas. SEP-786, an oral parathyroid hormone 1 receptor (PTH1R) agonist, aims to restore essential mineral balance. Phase I trials are underway, with data expected in mid-2025.
Mounjaro was approved this summer as a Type 2 diabetes treatment and is the first diabetes drug with dual action, targeting two hormones involved in insulin production and appetite (GIP and GLP-1). In contrast, Novo’s diabetes drugs Ozempic and Rybelsus (both semaglutide) are just GLP-1 agonists.
For example, pediatric growth hormone deficiency (PGHD) affects approximately one in 3,500 to one in 10,000 children. Existing therapeutic options for PGHD consist of injectable formulations of recombinant human growth hormone (rhGH), administered subcutaneously either on a daily or weekly basis.
Lutathera is used to treat adults and children aged 12 years and older with gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that are positive for the hormone receptor somatostatin, including GEP-NETs in the foregut, midgut and hindgut. If you want your company to be featured on Xtalks.com, please email ayeshar@xtalks.com.
Hypoparathyroidism arises when the parathyroid glands fail to produce enough parathyroid hormone (PTH) or when the PTH produced is ineffective. Yorvipath, developed using Ascendis Pharma’s TransCon technology, is the first and only approved treatment for hypoparathyroidism, marking this approval as a major milestone.
Currently, there is no scientific evidence to suggest that the vaccines induce any hormonal changes that would lead to irregular periods. This had led some to wonder whether period changes may be a potential side effect of the vaccines. Most physicians say there is nothing to worry about and that the changes are temporary.
Zilebesiran targets angiotensinogen (AGT), part of a hormone system that regulates blood pressure and fluid balance in the body, and according to Alnylam, could treat hypertension effectively when dosed either two or four times a year. The study is enrolling adults whose blood pressure is not adequately controlled by standard medicines.
Recently, Eli Lilly revealed promising results from a mid-stage trial, indicating that its popular drug, tirzepatide (marketed as Zepbound and Mounjaro for weight loss and diabetes, respectively), may be an effective treatment for the fatty liver disease metabolic dysfunction-associated steatohepatitis (MASH).
If successful, this trial could pave the way for a New Drug Application (NDA) submission. Autobahn is also planning a Phase II trial for bipolar disorder depression by late 2024, aiming to report topline data for MDD in the second half of 2025.
Norepinephrine is a hormone and neurotransmitter that helps in alertness and maintaining control in high-stress situations. Phase III trials have shown Qelbree’s effectiveness for children, adolescents and adults. SNRIs act by increasing the amount of available norepinephrine in the brain.
Tafinlar and Mekinist Combination Clinical Trial Results. The safety and efficacy of the Tafinlar and Mekinist combination therapy was evaluated in three clinical trials. The safety profile of Tafinlar + Mekinist in these trials was consistent with data from its previous clinical trials.
The exact cause of lupus is not entirely understood, but it is thought to be from a combination of genetic, environmental and hormonal factors. Investigational Drugs in Phase III Clinical Trials for Lupus There are a couple of investigational drugs that are new molecular entities (NME) in Phase III trials for lupus.
The company is advancing around 50 projects in ongoing clinical trials across a range of potential therapeutic modalities and indications, with a focus on oncology, cardiovascular and women’s health. Every year 47 million women enter menopause, a period where women are at the peak of their life’s activity, personally and professionally.
Based on these data, a first-in-human trial with BAY 2965501 in patients with advanced solid tumors was initiated and is currently enrolling patients. For the first time, Bayer will present the design of the clinical trial and the preclinical activity profile of the inhibitor in human epidermal growth factor receptor 2 (HER2) mutant NSCLC.
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