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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.,
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
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.
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.
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.
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.
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? —
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.
Efruxifermin is a novel Fc-FGF21 fusion protein designed to emulate the activity of native FGF21, an endogenous hormone that regulates metabolism and alleviates cellular stress. This comprehensive action of efruxifermin addresses the multifaceted nature of MASH.
Tafinlar and Mekinist Combination ClinicalTrial Results. The safety and efficacy of the Tafinlar and Mekinist combination therapy was evaluated in three clinicaltrials. The safety profile of Tafinlar + Mekinist in these trials was consistent with data from its previous clinicaltrials.
It has been a year dominated by the pandemic and many lifesciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. The company’s menopause drug NT-814 is heading for phase 3 development in 2021 following a mid-stage trial readout.
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 ClinicalTrials 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 clinicaltrials across a range of potential therapeutic modalities and indications, with a focus on oncology, cardiovascular and women’s health. About Bayer Bayer is a global enterprise with core competencies in the lifescience fields of health care and nutrition.
Bayer will present the first clinical Phase 1 results on aryl hydrocarbon receptor (AhR) inhibitor BAY 2416964, the company’s most advanced Immuno-Oncology program.
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