Nutropin AQ (somatropin) / Ipsen, Roche - LARVOL DELTA

Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children (clinicaltrials.gov) - P3 | N=50 | Recruiting | Sponsor: Northwell Health

New P3 trial • Endocrine Disorders • Growth Hormone Deficiency • IGF1 • IGFBP3

STARFISH: Study of Testosterone and rHGH in FSHD (clinicaltrials.gov) - P1 | N=20 | Completed | Sponsor: University of Rochester | Active, not recruiting ➔ Completed

Trial completion • Muscular Dystrophy • Solid Tumor • IGF1

Lonapegsomatropin (Skytrofa) for growth hormone deficiency. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Endocrine Disorders • Growth Hormone Deficiency

STARFISH: Study of Testosterone and rHGH in FSHD (clinicaltrials.gov) - P1; N=20; Active, not recruiting; Sponsor: University of Rochester; Recruiting ➔ Active, not recruiting

Enrollment closed • Muscular Dystrophy • IGF1

Effectiveness and Overall Safety of NutropinAq for Growth Hormone Deficiency and Other Paediatric Growth Hormone Disorders: Completion of the International Cooperative Growth Study, NutropinAq European Registry (iNCGS). (PubMed, Front Endocrinol (Lausanne)) - P=N/A | "There was an improvement in mean height SDS in all aetiology subgroups after rhGH treatment. No new safety concerns were identified."

Clinical • Journal • Chronic Kidney Disease • Endocrine Disorders • Genetic Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Nephrology • Ophthalmology • Pediatrics • Renal Disease • Strabismus • Turners Syndrome

STARFISH: Study of Testosterone and rHGH in FSHD (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: University of Rochester; Trial completion date: Apr 2021 ➔ Apr 2022; Trial primary completion date: Apr 2021 ➔ Apr 2022

Trial completion date • Trial primary completion date • Muscular Dystrophy • IGF1

Growth Hormone Use in Adults With Prader-Willi Syndrome (clinicaltrials.gov) - P3; N=11; Completed; Sponsor: Children's Mercy Hospital Kansas City; Recruiting ➔ Completed

Clinical • Trial completion • Prader–Willi syndrome • IGF1

COMPLIA: A 12-MONTH PROSPECTIVE, MULTICENTRE, NON-INTERVENTIONAL STUDY TO EVALUATE TREATMENT ADHERENCE AND TREATMENT SATISFACTION IN A GROWTH HORMONE DEFICIENT PAEDIATRIC POPULATION TREATED WITH NUTROPINAQ A SOMATROPIN ANALOGUE. (PubMed, Acta Endocrinol (Buchar)) - "Overall treatment adherence to NutropinAq was high in the Romanian GHD paediatric population, and a high level of treatment satisfaction was reported by patients/caregivers. This suggests reliable treatment outcomes can be anticipated in this population."

Clinical • Journal • Observational data • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

PLGA Microspheres of hGH of Preserved Native State Prepared Using a Self-Regulated Process. (PubMed, Pharmaceutics) - "Reduced initial burst enabled reduced protein loading to 9% (from 22% of the once commercialized Nutropin depot), which in turn reduced the dosage form index from 80 to 8.7 and eased the initial burst. A series of physical chemical characterizations as well as biologic and pharmacokinetic assays confirmed that the present method is practically feasible for preparing microspheres of proteins."

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Human Growth Hormone Pre-treatment for 6 Weeks Prior to Ovulation Induction for IVF (clinicaltrials.gov) - P1/2; N=30; Not yet recruiting; Sponsor: Center for Human Reproduction

New P1/2 trial • Biosimilar • Growth Hormone • Oncology

Europe - First to market - 22 February 2011: J.P. Morgan Cazenove FTM Europe 22 Feb 11: Millennium & Copthorne (OW): intro PT 774p; Abertis (N): upside pot’l on CVC : TomTom (OW): buying opp; Global IBs: OW Europeans; Prudential (UW); Swiss Re (OW); & MOR (J.P. Morgan) -

Accelerated recognition of Roche milestones (€48M) & the impairments around Increlex (€98M) to be added back to Adjusted Operating Income

None

Ipsen: 2013 half-year results and financial objectives (Ipsen Press Release) - "In the second quarter 2013, sales reached €15.1 million, up 12.6% year-on-year. In the first half 2013, sales of NutropinAq reached €29.2 million, up 10.1%, driven by a solid performance in Germany, France, Kazakhstan and the Netherlands."

Sales • Growth Hormone

Q1 2012 Sales (Ipsen) - Increlex / Ipsen; Roche; Products manufactured for the US in Hopkinton facility are currently on hold, Anticipated FDA inspection of US Hopkinton facility and results before the end of H1 2012

Anticipated FDA decision • Growth Hormone

Three Times Weekly (TIW) Growth Hormone Therapy in Children on Hemodialysis (clinicaltrials.gov) - P3; N=3; Completed; Sponsor: Nationwide Children's Hospital; N=20 -> 3

Enrollment change • Biosimilar • Chronic Kidney Disease • Renal Disease

Principles of growth hormone and insulin-like growth factor-I treatment in children with idiopathic short stature (Horm Res Paediatr.) - IGF-I is now approved in U.S & European Union for treatment of short stature in children with severe primary IGF-I deficiency, a condition characterized by unresponsiveness to GH in IGF-I-producing tissues; This has increased the focus on growth response to GH therapy in short children treated according to current recommendations; In particular, children with idiopathic short stature may have some degree of GH insensitivity that decreases their response to GH treatment

Review • Growth Hormone

Drug Approval Reports (WCG’s FDAnews) - Manufacturing Change or Addition of NUTROPIN AQ on 05/15/2014.

FDA event • Growth Hormone

Increlex-treated children enrolled in the increlex growth forum database (IGFD) in Europe: 2 years interim results on safety and effectiveness (ESPE 2012) - Presentation time: Saturday, Sep 22, 2012, 13:00 - 14:00; P=Obs, N=128; EU-IGFD; 2-year interim results from EU-IGFD registry did not show any new safety signals; After 1 year of treatment height velocity increased & results of 2nd year HV should be interpreted with caution due to limited number of data & large inter-individual variability observed in response to treatment

Observational data • Growth Hormone

Drug patent expirations this week: UCB, Roche's Genentech (SeekingAlpha) - Neutropin depot patent No. 5,654,010 expired on Aug 5 2014.

Patent expiry • Growth Hormone

Safety results from a phase II, randomized, open-label, active-controlled trial of rhGH/rhIGF-1 co-administration therapy in children with short stature, low IGF-1 and GH sufficiency (ENDO 2013) - Presentation time: Sunday, June 16, 2013: 10:45 AM-11:15 AM; P2, N=106; Sponsor: Ipsen; NCT00572156; "Additional safety concerns were not evident with co-administration of rhGH/rhIGF-1 therapy in this trial compared with previously reported rhGH and rhIGF-1 safety profiles. rhGH/rhIGF-1 therapy resulted in a first-year height velocity exceeding that of rhGH alone."

P2 data • Growth Hormone

STARFISH: Study of Testosterone and rHGH in FSHD (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: University of Rochester; Trial completion date: Jul 2020 ➔ Apr 2021; Trial primary completion date: Jul 2020 ➔ Apr 2021

Trial completion date • Trial primary completion date • Genetic Disorders • Muscular Dystrophy • Oncology • Solid Tumor • IGF1

Human Growth Hormone Pre-treatment for 6 Weeks Prior to Ovulation Induction for IVF (clinicaltrials.gov) - P1/2; N=30; Enrolling by invitation; Sponsor: Center for Human Reproduction; Trial completion date: Jun 2019 ➔ Jun 2022; Trial primary completion date: Jun 2018 ➔ Jun 2021

Trial completion date • Trial primary completion date

"Nutropin" (@marlomazz)

Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency (clinicaltrialsregister.eu) - P2; N=106; Sponsor: Ipsen Pharma

Clinical • Combination therapy • New P2 trial

Long-Acting Growth Hormone Preparations in the Treatment of Children. (PubMed, Pediatr Endocrinol Rev) - "Nutropin Depot is the only long acting rhGH ever approved by the U.S. Food and Drug Administration, and it was removed from the market in 2004. Of the approximately seventeen candidate drugs, only a handful remain under active clinical investigation or are commercially available."

Clinical • Journal

Structural Stability of Recombinant Human Growth Hormone (r-hGH) as a Function of Polymer Surface Properties. (PubMed, Pharm Res) - "Overall, the nature and magnitude of structural changes observed has a strong dependence on the pH and differences and degree of hydrophobicity of PLGA."

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