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Macimorelin Diagnostic

In Development for the Diagnosis of Childhood-Onset
Growth Hormone Deficiency

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Ongoing Pivotal Phase 3

DETECT-Trial

Our lead product, macimorelin, is the only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Macimorelin is currently marketed in the United States under the tradename Macrilen™, through a license agreement with Novo Nordisk. Through a license agreement with Consilient Health Ltd, macimorelin will be commercialized in Europe and the United Kingdom upon receipt of pricing and reimbursement approvals in the particular country. Additionally, we have entered into a distribution agreement with MegaPharm Ltd. under which MegaPharm will seek regulatory approval and then commercialize macimorelin in Israel and the Palestinian Authority.

 

We are also leveraging the clinical success and compelling safety profile of macimorelin to develop it for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need, in collaboration with Novo Nordisk.

Macimorelin, a ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone (GH) from the pituitary gland. Stimulated growth hormone levels are measured in blood samples after oral administration of macimorelin for the assessment of GHD.

“Because the macimorelin test is simple, well tolerated with minimal side effects, and of shorter duration with only 3 to 4 blood draws compared to other GH–stimulation tests, it is anticipated that its use will increase over time.”*

“Very promising test that is easy to conduct with high reproducibility, safety, and diagnostic accuracy comparable to the ITT and GHRH plus ARG test.”*

 

AACE 2019 Guidelines (https://journals.aace.com/doi/full/10.4158/GL-2019-0405)

*AACE 2019 Guidelines: American Association Of Clinical Endocrinologists And American College Of Endocrinology Guidelines For Management Of Growth Hormone Deficiency In Adults And Patients Transitioning From Pediatric To Adult Care, 2019

American Association of Clinical Endocrinologists 2019 Guidelines

Childhood-Onset Growth Hormone Deficiency (CGHD)

Growth Hormone is Critical to Child Development

Promotes Growth

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Reduction in auxological parameters:

• Short stature

• Low growth velocity (speed) for age

• Increased fat around the waist

• Delayed tooth development

Childhood-onset growth hormone deficiency (CGHD) is an endocrine condition associated with a broad range of health issues from childhood through to adulthood, which requires particular attention during the transition period from adolescence to young adulthood.

Number of Potential Tests Annually

27,000-28,800*

~25,000*

*Navigant study 2009, TVG study 2017, Huron Consulting study 2018

GHD in children can be congenital or acquired and either isolated or combined with other pituitary hormone defects. There are many well-defined causes of GHD in children, but the cause is often unknown (idiopathic GHD). The definitions of the various types of GHD and other causes of short stature are given in the International Classification of Pediatric Endocrine Diagnoses (ICPED).

Childhood-onset growth hormone deficiency and the transition to adulthood: current perspective