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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. Traditional methods like diet and exercise often fail to provide long-term success, and newer pharmacological options like hormone-based GLP-1 agonists, while effective, can be expensive and carry side effects.
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the lifesciences. Here is a look at some lifescience trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.
Cardiac biomarkers can not only serve important roles in the diagnosis, management and prognosis of cardiac diseases, but they can also be significant tools in evaluating treatments in cardiovascular (CV) clinicaltrials. In CV clinicaltrials, cardiac biomarkers are most commonly included in efficacy and safety endpoints.
Sling Therapeutics has positive topline results from its Phase IIb/III LIDS clinicaltrial evaluating its oral small-molecule therapy linsitinib for the treatment of thyroid eye disease (TED). It is a complication of Graves disease, an immune system condition that causes the thyroid gland to make excess amounts of thyroid hormone.
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 in its Phase Ib trial. Metsera, Inc.,
Incytes recent announcement on the topline results from two Phase III clinicaltrials of povorcitinib in patients with hidradenitis suppurativa has stirred both hope and caution in the market. Janus kinase one (JAK1) plays a central role in the signaling pathways of several inflammatory cytokines.
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
VK2809 is a novel, oral small molecule thyroid hormone receptor beta agonist that has selectivity for liver tissue. It is now seen as an epidemic within chronic liver disease, particularly as its prevalence has been increasing and paralleling that of obesity, making it a major health concern worldwide.
Pancreatic cancer occurs when cells in the pancreas, a vital organ responsible for producing enzymes and hormones, begin to grow uncontrollably. Targeted therapies and clinicaltrials also offer potential treatment avenues for those diagnosed with the disease.
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
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.
These gut hormones are released in response to nutrient intake and play a crucial role in the incretin effect, which enhances insulin secretion. It’s the first and only approved dual agonist targeting both the GIP and GLP-1 receptors.
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.
Petrelintide, administered once a week under the skin, targets obesity through its action as an amylin analog, a hormone co-secreted with insulin from the pancreas. By mimicking this hormone, petrelintide may offer an alternative or complement to incretin-based therapies, known for their role in weight management.
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?
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.
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.
David Cameron, Chair of the Breast International Group (BIG) , we delve into the landscape of breast cancer research today, clinicaltrials and the incorporation of the patient voice in breast cancer research. Breakthroughs in Treatment BIG has been at the forefront of numerous clinicaltrials.
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.
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.
In addition to innovative new antibody drugs and mRNA vaccines, researchers have found that the sleep-regulating hormone melatonin may also offer protection against COVID-19. The results still require further validation, including experimental and clinical evaluation. The list also included melatonin.
GLP-1 receptor agonists mimic the action of GLP-1, an intestinal hormone that stimulates the secretion of insulin when blood glucose levels rise. Semaglutide acts by inhibiting glucagon, which is a hormone that increases blood sugar. It combines the action of both glucose-lowering metabolic hormones into a single molecule.
Amgen announced positive results from a Phase II clinicaltrial 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. percent in the 68-week STEP-1 clinicaltrial.
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.
This result came from the promising Phase III EMERALD global clinicaltrial ( NCT03778931 ). Recent studies in mouse models implanted with human tumor cells (xenografts) have shown that elacestrant exhibits significant anti-tumor activity , even in ESR1 mutated models resistant to hormone therapy. This oral drug binds to ER? —
Erythropoietin is a hormone that is produced by the kidneys and signals the body to make red blood cells. ClinicalTrial 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?
Amgen announced positive results from a Phase II clinicaltrial 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. percent in the 68-week STEP-1 clinicaltrial.
GLP-1 receptor agonists, or incretin (metabolic hormones) mimetics, are analogs of the GLP-1 peptide hormone that binds to the GLP-1 receptor to regulate blood sugar levels by boosting insulin secretion. GIP is a metabolic hormone that promotes insulin secretion and also weakly inhibits gastric acid secretion to suppress appetite.
Notably, the EMPA-KIDNEY Phase III clinicaltrial results published in The New England Journal of Medicine revealed that Jardiance (empagliflozin), an SGLT2 inhibitor, significantly reduced the risk of kidney disease progression or death from cardiovascular causes compared to placebo in patients with pre-existing CKD.
Aditya Bardia, MD, MPH, director of Breast Cancer Research at Mass General Cancer Center, associate professor at the Medicine Department at Harvard Medical School and principal investigator for the EMERALD trial, in the same press release statement. Mutations in the ESR1 , however, have contributed to the rise of endocrine resistance.
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.
Although still in early clinicaltrials, this immunotherapy approach could represent a significant advance for patients with limited treatment options. Make an Impact on Prostate Cancer Awareness Month 2024 Ongoing research and clinicaltrials are bringing the medical community closer to more effective treatments.
In the ASCEND trials, Daprodustat was comparable to Amgen’s Aranesp (darbepoetin alfa), an erythropoiesis-stimulating agent (ESA), with respect to heart safety. Darbepoetin alfa is a synthetic version of the hormone erythropoietin, which is made in the kidneys and stimulates the production of red blood cells (RBC), or erythrocytes.
Eli Lilly announced that its obesity drug tirzepatide has scored favorably in a late-stage clinicaltrial, 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.
Symptoms and Etiology: Caused by an overproduction of growth hormone, acromegaly primarily affects adults. By reducing the amount of growth hormone in the blood, progressive enlargement of bones should be slowed. By reducing the amount of growth hormone in the blood, progressive enlargement of bones should be slowed.
This funding infusion is poised to support Septerna’s expanding pipeline and ongoing clinicaltrials 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.
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 clinicaltrial. Diamyd Medical is a clinical-stage biopharmaceutical company that develops therapies for type 1 diabetes.
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.
Tirzepatide’s mechanism of action involves activating two naturally produced hormones: glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP). ClinicalTrials for MASH Treatment The tirzepatide trial involved approximately 190 adults with MASH at different stages.
Current treatments for HR+ breast cancer patients include hormonal therapies, chemotherapies for those that do not respond to hormonal therapy alone and targeted therapies, either stand-alone or in combination with hormonal therapies. What does this Approval Mean for Breast Cancer Patients?
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.
AstraZeneca has won its first-in-class approval for its AKT inhibitor Truqap (capivasertib) in combination with Faslodex (fulvestrant) for treating patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer.
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