Phase 2 Data Shows Cimdelirsen Benefits for Acromegaly

According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals (“Ionis”), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients with uncontrolled acromegaly. In particular, researchers evaluated cimdelirsen as an adjunctive therapy, to be used alongside somatostatin receptor ligands. Altogether, the treatment showed enough benefits that Ionis will continue developing this therapy moving forward.


So what exactly is cimdelirsen? According to Ionis, cimdelirsen is:

a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of the growth hormone receptor (GHr) to decrease the circulating level of insulin-like growth factor-1 (IGF-1). IGF-1 is a hormone primarily produced in the liver that plays an important role in childhood growth and has anabolic effects in adults.

Within this particular study, researchers explored the potential benefits of cimdelirsen for patients with uncontrolled acromegaly. Patients were either part of a low-dose cohort (from 60+80mg) or a high-dose cohort (120+160mg). Findings from the study show:

  • While patients in both the high and low-dose cohorts experienced some adverse reactions, such as headache and UTI, researchers found that cimdelirsen was relatively safe and well-tolerated overall. No deaths or serious adverse reactions occurred. Additionally, researchers determined that the headaches and UTIs were not associated with cimdelirsen treatment.
  • Cimdelirsen helped significantly reduce circulating growth hormone receptor levels, while also not increasing growth hormone levels. The treatment also reduced IGF-1 levels.
  • Altogether, cimdelirsen treatment helped improve quality of life (QOL) in patients with uncontrolled acromegaly.
  • GHBP reductions highlighted the study’s proof-of-mechanism and showed significant improvements in patients.

Moving forward, Ionis will continue to develop cimdelirsen for patients. Additional studies remain ongoing; data will be available from these studies at some point in 2022. Because patients with acromegaly, especially if uncontrolled, do not have a wealth of therapeutic options, cimdelirsen offers the potential to fill unmet needs within this community.


Normally, our pituitary gland produces growth hormone. However, in some cases, pituitary adenomas overproduce growth hormone, causing significant and abnormal growth. This can cause acromegaly, a rare hormonal disorder. An estimated 60 out of every 1 million people have acromegaly. The condition usually manifests in middle-aged individuals, or those between ages 30-50. It is important to note that acromegaly and gigantism are not the same condition; while gigantism occurs only in children, acromegaly can occur in those of all ages though, again, it often occurs in mid-life. The condition progresses slowly. However, as symptoms appear, they may include:

  • Swollen hands and feet that may appear disproportionately large compared to the rest of the body
  • Enlarged nasal bone
  • Protruding brow and lower jaw
  • Fatigue or difficulty sleeping
  • Blurred or reduced vision
  • Thick, hard nails
  • Course or oily skin that is prone to excessive sweating
  • Muscle and joint pain
  • Hyperpigmentation
  • High blood pressure
  • Enlarged facial features (nose, lips, tongue, vocal cords)
  • Organ enlargement

Learn more about acromegaly.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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