We are committed to making a meaningful difference for patients with GHD
Growth hormone is vital for human growth and development. Receptors are found in virtually every tissue in the body, and it is necessary for overall endocrine health. Growth hormone contributes to development of healthy muscle, bone and adipose tissue. Yet some people – both children and adults – are unable to naturally produce enough growth hormone, which may lead to a medical condition known as growth hormone deficiency (GHD).
Endogenous growth hormone is key to development of healthy tissues
We believe it’s time for a new GHD treatment option
For more than 30 years, children and adults with GHD have been treated with daily injections of somatropin, also known as recombinant human growth hormone (rhGH or hGH). While daily treatment has been shown to be safe and effective, data that indicate that seven of ten patients do not adhere to their prescribed therapy. This means some patients treated for GHD – children who need growth hormone to achieve both their target height and support overall endocrine health – may not be getting the full benefits of treatment. We believe it’s time for a new treatment option for GHD, potentially with all the integrated, positive effects of growth hormone.
Lonapegsomatropin is an investigational, long-acting human growth hormone prodrug administered weekly that releases somatropin as a potential therapy for children and adults with GHD.
lonapegsomatropin
Lonapegsomatropin is an investigational prodrug of somatropin (human growth hormone) in phase 3 development as a once-weekly therapy for children and adults with GHD. We are also developing a proprietary auto-injector to administer lonapegsomatropin, stable at room temperature, in dual-chamber cartridges. Our intention is to develop a best-in-class therapy to treat GHD that provides all the integrated, positive effects of growth hormone.
Our global clinical development program supporting lonapegsomatropin includes eight clinical trials evaluating safety and efficacy in more than 400 participants with GHD – both treatment-naïve and treatment-experienced. Positive results have been reported from both the phase 3 heiGHt Trial, which compared lonapegsomatropin to a daily somatropin, and the phase 3 fliGHt (switch) Trial. Additionally, we are conducting the enliGHten Trial, a long-term extension in children with GHD, and we have recently initiated the global phase 3 foresiGHt Trial in adults with GHD.
Lonapegsomatropin has orphan designation for GHD in both the U.S. and Europe.
Auto-injector designed for a positive patient experience
Our investigational auto-injector and dual-chamber cartridge (DCC) system is designed to support patients and caregivers.
We conducted significant research and gathered insights from patients and caregivers throughout the development of our proprietary auto-injector – with the goal of providing a simple, positive user experience. We are also developing an integrated connectivity platform – reflecting our commitment to patients and caregivers, as well healthcare providers.
- Designed to deliver single, small volume once-weekly doses (< 0.60mL) of lonapegsomatropin for patients ≤ 60kg
- Each cartridge contains the medicine (lonapegsomatropin) in a powder form (lyophilized or freeze-dried powder) and preservative-free water (diluent) designed for one single dose
- Cartridges allow for room temperature storage after dispensing to patient
- Cartridges being developed in multiple potential dosing presentations
- Uses a 31-gauge and 4-millimeter needle
- Prepared for future Bluetooth® connectivity* to enable patient support and data capture
* Bluetooth Low Energy (BLE)
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