Genotropin (somatropin) / Pfizer - LARVOL DELTA

MISSION: Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature. (clinicaltrials.gov) - P3 | N=254 | Not yet recruiting | Sponsor: Rabin Medical Center

New P3 trial • Idiopathic Short Stature • Pediatrics • Small for Gestational Age • IGF1

Study of the efficacy and safety of somatropin in Japanese participants with PWS (clinicaltrialsregister.eu) - P3 | N=33 | Sponsor: Pfizer, Inc.

New P3 trial • Prader–Willi syndrome • IGF1

Pfizer's revenue in the second quarter of 2024 increased slightly by 2% year-on-year, and its oncology business revenue increased by 22% year-on-year. GLP-1 drugs returned [Google translation] (Eastmoney.com) - "In terms of sales growth, the company's third-generation ALK inhibitor Lorbrena (Borui Na), lung cancer drugs Braftovi/Mektovi, antibiotic Zithromax, anti-inflammatory drug Inflectra, recombinant human growth hormone Genotropin and other drugs all achieved revenue growth of more than 30% in the second quarter."

Sales • Infectious Disease • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS (clinicaltrials.gov) - P3 | N=33 | Completed | Sponsor: Pfizer | Active, not recruiting ➔ Completed

Trial completion • Prader–Willi syndrome • IGF1

Growth Hormone Therapy for Muscle Regeneration in Severely Burned Patients (clinicaltrials.gov) - P2/3 | N=13 | Completed | Sponsor: The University of Texas Medical Branch, Galveston | Active, not recruiting ➔ Completed | N=64 ➔ 13

Enrollment change • Trial completion • Thermal Injury

Growth Hormone Therapy for Muscle Regeneration in Severely Burned Patients (clinicaltrials.gov) - P2/3 | N=64 | Active, not recruiting | Sponsor: The University of Texas Medical Branch, Galveston | Recruiting ➔ Active, not recruiting

Enrollment closed • Thermal Injury

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS (clinicaltrials.gov) - P3 | N=32 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting

Enrollment closed • Prader–Willi syndrome • IGF1

Growth Hormone Therapy for Muscle Regeneration in Severely Burned Patients (clinicaltrials.gov) - P2/3; N=64; Recruiting; Sponsor: The University of Texas Medical Branch, Galveston; Active, not recruiting ➔ Recruiting; Trial completion date: Dec 2021 ➔ Dec 2023; Trial primary completion date: Aug 2021 ➔ Aug 2023

Enrollment open • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Thermal Injury

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS (clinicaltrials.gov) - P3; N=30; Recruiting; Sponsor: Pfizer; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Prader–Willi syndrome • IGF1

Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS (clinicaltrials.gov) - P3; N=30; Not yet recruiting; Sponsor: Pfizer

Clinical • New P3 trial • Prader–Willi syndrome • IGF1

Growth Hormone Therapy for Muscle Regeneration in Severely Burned Patients (clinicaltrials.gov) - P2/3; N=64; Active, not recruiting; Sponsor: The University of Texas Medical Branch, Galveston; Trial completion date: Oct 2020 ➔ Dec 2021; Trial primary completion date: Oct 2020 ➔ Aug 2021

Clinical • Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Thermal Injury

Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD (clinicaltrials.gov) - P3; N=87; Completed; Sponsor: Pfizer; Recruiting ➔ Completed

Clinical • Trial completion • Endocrine Disorders • Growth Hormone Deficiency • IGF1

GGAP: Global Growth Hormone Study in Adults With Prader-Willi Syndrome (clinicaltrials.gov) - P3; N=50; Not yet recruiting; Sponsor: Erasmus Medical Center

Clinical • New P3 trial • Prader–Willi syndrome

A four-year, open-label, multi-center, randomized, two-arm study of Genotropin in patients with idiopathic short stature: Comparison of an individualized, target-driven treatment regimen to standard dosing of Genotropin - Analysis of two-year data (Horm Res Paediatr.) - P=NA, N=316,89; PMID: 24021297; "The majority (83%) of subjects had Ht SDS within the normal range by 2 years. All subjects displayed catch-up growth consistent with other studies of GH treatment of ISS."

Clinical data • Growth Hormone

Aromatase inhibitors, alone and in combination with growth hormone in adolescent boys with idiopathic short stature (ThrasherAI) (clinicaltrials.gov) - P3, N=77; Active, not recruiting; Completion date: Mar 2015 -> Oct 2017.

Trial completion date • Growth Hormone

Cross over convenience and preference study of new mark VII compared to Genotropin pen in pediatric and adult subjects (CHOOSE) (clinicaltrials.gov) - P3, N=150; CHOOSE; Active, not recruiting -> Recruiting

Enrollment open • Growth Hormone

Pfizer achieves primary endpoint with phase 3b top-line results of Genotropin in very young children born small for gestational age (Pfizer Press Release) - P3, N=43; Sponsor: Pfizer; NCT00627523; "The primary endpoint of the study was achieved: there was a statistically significant difference at 24 months in change from baseline height between patients treated with GENOTROPIN and those in the untreated control group, as measured by height standard deviation score (SDS). The study results showed, after 24 months of treatment, the GENOTROPIN group had a statistically significantly greater gain in height SDS compared to the untreated control group."

P3 data: top line • Growth Hormone

Psychometric assessment of the Injection Pen Assessment Questionnaire (IPAQ): Measuring ease of use and preference with injection pens for human growth hormone (Health Qual Life Outcomes) - P3, N=132; Total IPAQ score is a useful measure for evaluating ease of use & preference for injection pens in clinical trials among pt dyads receiving hGH; The psychometric properties of individual subscales, mainly the lower internal consistency reliability of some of subscales & the predictive validity findings, do not support the use of subscale scores alone as a primary endpoint

P3 data • Growth Hormone

Results from an international multicenter trial evaluating the ease-of-use of and preference for a newly developed disposable injection pen for the treatment of growth hormone deficiency in treatment-naïve children and adults (Med Devices (Auckl)) - P=3, N=120; Sponsor: Pfizer; NCT01112865; "The primary endpoint was the proportion of participants who reported the new disposable pen to be no different from or easier to use than the current reusable pen. Safety was also assessed and reported according to local legal requirements. Of the 120 screened patients, 119 were included in the ease-of-use analysis and all were included in the safety analyses. In all, 67.2% found the new somatropin disposable pen to be no different from or easier to use than the reusable pen..."

P3 data • Growth Hormone

One-year data from a long-term phase IV study of recombinant human growth hormone in short children born small for gestational age (Biol Ther) - P4, N=278; Sponsor: Pfizer; NCT01859949; "Similarly, mean peak-centred HVSDS increased from -2.13 at baseline to +4.16 at Year 1. Mean IGF-I SDS and IGFBP-3 SDS also increased within the first year (by +1.80 and +0.41, respectively). 13 patients (4.7%) did not respond adequately to treatment (HVSDS <1); they were withdrawn from the study. In total, 192 children (69.3%) experienced treatment-emergent AEs; most (98.7%) were mild-to-moderate, and the majority (96.5%) were unrelated to study treatment."

P4 data • Growth Hormone

Patient perspectives on a new injection pen for growth hormone: Results of a multinational clinical study in children and adults (ESPE 2012) - Presentation time: Thursday, Sep 20, 2012, 14:15 - 15:15; P3, N=NA; NCT01112865; Both pens were considered easy to use with the majority finding the new pen is easier to use vs. current pen; Adult participants selected the new pen more often than child/parent dyads as did participants from the Eastern as opposed to Western parts of EU

P3 data • Growth Hormone

Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD (clinicaltrials.gov) - P3; N=90; Recruiting; Sponsor: Pfizer; Trial completion date: Mar 2020 ➔ Sep 2020; Trial primary completion date: Mar 2020 ➔ Sep 2020

Clinical • Trial completion date • Trial primary completion date

Study of Growth Hormone and Bone in Obesity (clinicaltrials.gov) - P=N/A; N=75; Completed; Sponsor: Massachusetts General Hospital; Active, not recruiting ➔ Completed

Trial completion

Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD (clinicaltrials.gov) - P3; N=90; Recruiting; Sponsor: Pfizer; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD (clinicaltrials.gov) - P3; N=90; Not yet recruiting; Sponsor: Pfizer

Clinical • New P3 trial