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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

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.

Genetics 116
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New ‘Mechanical’ Weight Loss Pill from Oxford Medical Products Shows Positive Safety Data

XTalks

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.

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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

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

Drugs 317
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Sling Therapeutics’s Linsitinib Meets Primary Endpoint in Thyroid Eye Disease Trial, Eyes Competition with Amgen’s Tepezza

XTalks

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). It is a complication of Graves disease, an immune system condition that causes the thyroid gland to make excess amounts of thyroid hormone.

Trials 59
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Obesity-Focused Metsera Launches IPO Amid Much Anticipation

XTalks

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.,

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Povorcitinib Phase III Results Stir Mixed Signals for Incyte and Investors

XTalks

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. Janus kinase one (JAK1) plays a central role in the signaling pathways of several inflammatory cytokines.

Trials 59
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Eneboparatide Advances in Hypoparathyroidism Trial: Key Phase III Update

XTalks

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

Trials 59