Somatrogon© COMPETITIVE ADVANTAGES
In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of Somatrogon©. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product.
- New molecular entity (NME) that maintains natural native sequence of growth hormone
- Once weekly injection vs. current products requiring daily injections
- Human growth hormone is used for:
- Growth hormone deficient children and adults
- SGA, PWS, ISS
- Final presentation:
- Refrigerated, liquid, non-viscous formulation
- Disposable easy to handle pen injection device with thin needle and small injection volume
- Orphan drug designation in the U.S. and the EU for children and adults
Somatrogon© PROGRAM STATUS
Phase 3 Pediatric Somatrogon©
- Phase 3 study in naive growth hormone deficiency pediatric population was completed.
The study was conducted in over 20 countries. This study enrolled and treated 224 pre-pubertal, treatment-naive children with growth hormone deficiency.
- OPKO and Pfizer Announce Positive Phase 3 Top-Line Results for Somatrogon© during Oct 2019.
- Achieved Primary Endpoint
- Somatrogon© was proven non-inferior to daily Genotropin® (somatropin) with respect to height velocity after 12 months
- Height velocity at 12 months of treatment was higher in the Somatrogon© group (10.12 cm/year) than in the somatropin group (9.78 cm/year)
- Secondary Endpoints Achieved
- Change in height standard deviation scores at six and 12 months were higher with Somatrogon© in comparison to somatropin
- At six months, change in height velocity was higher with Somatrogon© in comparison to somatropin
- Somatrogon© was generally well tolerated in the study and comparable to that of somatropin dosed once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms
- Children completing this study had the opportunity to enroll in a global, open-label, multicenter, long-term extension study, in which they were able to either continue receiving or switch to Somatrogon© Approximately 95% of the patients switched into the open-label extension study and received Somatrogon© treatment
Phase 3 adults Somatrogon© completed
- Primary endpoint of change in trunk fat mass from baseline to 26 weeks did not demonstrate a statistical significance between the Somatrogon© treated group and placebo
- Completed post hoc outlier analysis in June 2017 to assess the influence of outliers on the primary endpoint results
- Analyses which excluded outliers showed a statistically significant difference between Somatrogon© and placebo on the change in trunk fat mass: additional analyses that did not exclude outliers showed mixed results
- No safety concerns
- OPKO and Pfizer have agreed that OPKO may proceed with a pre-BLA meeting with FDA to discuss a submission plan
- OPKO plans to carry out an additional study in adults using a pen device
Pediatric Somatrogon© registration study in Japan- expected to be completed in Q1 2020
- 44 patients, comparison of weekly Somatrogon to daily growth hormone.
- Same pen device, dosage and formulation used in global study.
Somatrogon© Path to Approval
- BLA submission in US anticipated second half of 2020
- Completion of analysis of immunogenicity and safety data from pivotal Phase 3 study and open label extension study
- Two abstracts accepted for oral presentation of data set at the Endo Society’s Annual Meeting in March 2020
- “Somatrogon© Growth Hormone in the Treatment of Pediatric Growth Hormone Deficiency: Results of the Pivotal Phase 3”
- “Interpretation of Insulin-like Growth Factor (IGF-1) Levels Following Administration of Somatrogon© (a long acting Growth Hormone-hGH-CTP)”
- MAA submission in Europe to follow upon completion of open label study demonstrating benefit and compliance with reduced treatment burden
- Study expected to be completed in Q3 2020
Hershkovitz O, Bar-Ilan A, Guy R, et al. In vitro and in vivo characterization of MOD-4023, a long-acting carboxy-terminal peptide (CTP)-modified human growth hormone. Mol Pharm. 2016; 13:631–639 [PDF]
Strasburger CJ, Vanuga P, Payer J, et al. MOD-4023, a long-acting carboxy-terminal peptide-modified human growth hormone: results of a Phase 2 study in growth hormone-deficient adults. Eur J Endocrinol. 2017;176:283–294 [PDF]
Zelinska N, Iotova V, Skorodok J, et al. Long-acting CTP-modified hGH (MOD-4023): results of a safety and dose-finding study in GHD children. J Clin Endocrinol Metab. 2017;102:1578–1587 [PDF]
Fisher DM, Rosenfeld RG, Jaron-Mendelson M, et al. Pharmacokinetic and pharmacodynamic modeling of MOD-4023, a long-acting human growth hormone, in GHD Children. Horm Res Paediatr. 2017;87:324–332 [PDF]
Kramer W, Jaron-Mendelson M, Koren R, et al. Pharmacokinetics, Pharmacodynamics and Safety of a Long-Acting Human Growth Hormone (MOD-4023) in Healthy Japanese and Caucasian Adults. Clin Pharmacol Drug Dev. 2017 [in press]