|
|
[Definitie:] "An ergogenic aid is any substance or phenomenon that enhances performance."
(Wilmore and Costill) 
|
|
|
||
|
||
|
0 3 - 0 4 - 2 0 0 3 Verbeterd IGF-1-preparaat SomatoKine bijna klaar voor de markt
Als alles meezit komt het nieuwe IGF-1-preparaat van het biotechbedrijf Insmed medio 2004 op de markt. Dat heeft Geofrey Allan, bestuurder van Insmed, in een brief aan de aandeelhouders bekend gemaakt. Het nieuwe preparaat heet SomatoKine en bevat verknutselde IGF-1-moleculen die het lichaam minder goed kan neutraliseren.
Het probleem met de klassieke IGF-1 is dat het hormoon zich vastmaakt aan het eiwitten IGF-BP-1, waardoor het zijn werking verliest. IGF-BP-1 doet met IGF-1 ongeveer wat SHBG met testosteron doet. Een eiwit dat ook aan IGF bindt, maar de werking van IGF-1 intact laat, is IGF-BP-3. Insmed heeft van die wetenschap handig gebruik gemaakt en zijn IGF-1 al van tevoren gekoppeld aan IGF-BP3.
Bestuurder Allan schijft de aandeelhouders dat de fase 3 trials op het punt staan te beginnen. Als die positief uitvallen, begint het bedrijf meteen de procedure om wettelijke goedkeuring voor SomatoKine in Europa en de VS te krijgen. Insmed heeft al contacten met grote farmaceuten, die geïnteresseerd zijn in het middel.
Eén daarvan is een maker van de oude IGF-1, Pharmacia, die de productie van IGF-1 inmiddels heeft gestaakt. Insmed mag gebruik maken van alle papieren en kennis waarmee Pharmacia destijds zijn IGF-1 op de markt heeft gekregen.
In eerste instantie gaat Insmed SomatoKine lanceren als medicijn voor de kleine groep patiënten met groeistoornissen die ongevoelig is voor groeihormoon en niet reageert niet op somatotropine. De FDA heeft, om de ontwikkeling van een medicijn voor die mensen te stimuleren, het IGF1-IGFBP3-complex alvast uitgeroepen tot een Orphan Drug. Daardoor kan Insmed allerlei ondersteuning van de overheid krijgen bij het produceren en vermarkten van zijn middel.
Op termijn hoopt Insmed dat artsen SomatoKine ook voor andere doeleinden gaan gebruiken. Het bedrijf denkt daarbij vooral aan suikerziekte. IGF-1 verhoogt de gevoeligheid van de receptoren voor insuline. Veel diabetici worden na verloop van tijd ongevoelig voor insuline en hebben aanvullende medicijnen nodig. Komt SomatoKine op die multi billion dollar markt, dan wordt het medicijn misschien een bockbuster. De kans dat er ampullen in het dopingmilieu terecht zullen komen is dan levensgroot.
Insmed heeft een financieel succes hard nodig. In 2002 moest het bedrijf ingrijpende bezuinigingen doorvoeren, en stopte het noodgedwongen de ontwikkeling van een ander experimenteel medicijn, INS-1. INS-1 moet, net als IGF-1, de receptoren voor insuline gevoeliger maken. Het middel bleek in trials de achteruitgang van de bètacellen in de pancreas van suikerpatiënten te remmen.
1. Geoffrey Allan.
Insmed Chairman And CEO Provides Positive Outlook in Special Letter to Shareholders.
BioWire2k via Businesswire.com, 1-4-2003.
1 9 - 0 9 - 2 0 0 5 SomatoKine(R) Pivotal Data to Be Featured in a Podium Presentation at the ESPE/LWPES 7th Joint Meeting of Pediatric Endocriniology in Lyon, France
9/19/2005 Insmed Incorporated (NASDAQ: INSM) today announced that data from the Company's New Drug Application for Somatokine (rhIGF-I/rhIGFBP-3) (mecasermin rinfibate) for the treatment of Growth Hormone Insensitivity Syndrome (GHIS), will be featured in a podium presentation on Friday, September 23 at the European Society Pediatric Endocrinology/ Lawson Wilkins Pediatric Endocrine Society 7th Joint Meeting, a global meeting of pediatric endocrinologists occurring every four years. During the Conference Symposium titled, "Update on Growth and Anabolic Therapies", Cecelia Camacho-Hubner, M.D., of St. Bartholemew's Hospital, London, United Kingdom will give the lecture featuring long-term safety and efficacy of the once-daily IGF-I replacement therapy, SomatoKine, in children with GHIS. About GHIS GHIS encompasses a variety of genetic and acquired conditions in which the action of growth hormone (GH) is absent or severely attenuated, resulting in low serum levels of IGF-I. Because IGF-I is the primary mediator of the growth-promoting actions of GH, SomatoKine replacement therapy in children with GHIS is intended to bypass the blocked actions of GH by replacing the deficient IGF-I, resulting in improved growth. More on SomatoKine Insmed's SomatoKine is a proprietary drug product for the delivery of recombinant insulin-like growth factor I (IGF-I). It is administered as a preformed complex with a recombinant form of its natural binding protein, insulin-like growth factor binding protein 3 (rhIGFBP-3). The novel compound is administered as a once-daily subcutaneous injection, which can restore and maintain IGF-I levels to physiologically relevant levels. The binding protein (rhIGFBP-3) extends the residence time of IGF-I in the blood, conferring a superior pharmacokinetic profile as compared with rhIGF-I alone. In the bound state, the IGF-I is inactive, and remains so until delivered to target tissues in the body where it is released and becomes biologically active. This reduces the risk of short- and long-term safety concerns that have been associated with unrestrained levels of free IGF-I. SomatoKine has been investigated in a number of other indications in addition to growth disorders. In patients with Type 1 and Type 2 diabetes, administration of SomatoKine demonstrated a significant improvement in blood sugar control and a significant reduction in daily insulin use. In children and adults suffering severe burn injury, administration of SomatoKine demonstrated a significant improvement in muscle protein synthesis and a significant reduction in the inflammatory response associated with the trauma. In elderly individuals recovering from hip fractures, administration of SomatoKine demonstrated a significant improvement in functional recovery and bone mineral density. In addition to the GHIS program, SomatoKine is currently being studied in a Phase II clinical trial at the University of California, San Francisco in patients with HIV-Associated Lipodystrophy, and in a Phase II clinical trial at the University of Cambridge, U.K. in patients with Extreme Insulin Resistance. About Insmed Insmed is a biopharmaceutical company focused on the discovery and development of drug candidates for the treatment of metabolic diseases and endocrine disorders. For more information, please visit www.insmed.com. CONTACT:Insmed Incorporated Baxter Phillips, III, 804-565-3041 1 5 - 1 2 - 2 0 0 5 Insmed growth failure drug approved by FDA
Pharmaceutical Business Review The FDA has given its approval for Insmed Incorporated's drug Iplex for the treatment of growth failure in children. Iplex is specifically approved to treat children with severe primary IGF-1 deficiency or with growth hormone gene deletion who have developed neutralizing antibodies to growth hormones. The drug was previously given orphan drug designation which means that the company will enjoy seven years of marketing exclusivity for Iplex in its indication to treat primary IGF-1 deficiency. "Iplex (is) the only approved once-daily IGF-1 replacement therapy available to treat children with severe short stature," stated Dr Geoffrey Allan, president and CEO of Insmed. "Today marks the beginning of a new treatment paradigm for treating children with Primary IGFD." Iplex, mecasermin rinfabate, is the human recombinant of the naturally occurring protein complex of insulin-like growth factor-I (IGF-1), and insulin-like growth factor binding protein-3 (IGFBP-3). 2 1 - 0 5 - 2 0 0 6 IPLEX(TM) Demonstrates Significant, Dose-Dependent Increases in Growth Rate
Data Presented in Oral Presentation at Pediatric Academic Societies' 2006 Annual Meeting
Insmed Incorporated SAN FRANCISCO, May 1 /PRNewswire-FirstCall/ -- Insmed Incorporated (Nasdaq: INSM - News) today announced results from a prospective, multicenter clinical trial of rhIGF-I/rhIGFBP-3, or IPLEX(TM) (mecasermin rinfabate [rDNA origin] injection) administered once daily to children with severe primary insulin-like growth factor-I (IGF-I) deficiency. The study showed that treatment resulted in statistically significant, dose-dependent increases in height velocity (growth rate) with a favorable safety profile. The first-year results from the 24-month study were presented in an oral presentation at the Pediatric Academic Societies' (PAS) 2006 Annual Meeting at the Moscone Center in San Francisco, CA. In the scientific abstract presenting the safety and efficacy data for IPLEX in children with severe primary IGF-I deficiency (primary growth hormone insensitivity), the investigators reported that the mean height velocity for the dose group titrated with up to 2 mg/kg/day increased from 2.0 cm/year pre- treatment to 8.3 cm/year during treatment. Children with genetic and acquired forms of growth hormone (GH) insensitivity appeared to respond equally well to treatment. IPLEX is the only once daily IGF-I replacement therapy available for the treatment of severe primary IGFD. The once-daily dosing regimen used in the study was associated with a high compliance rate of 95% of injections taken. "We are very pleased with the growth rates and safety profile achieved with IPLEX in this patient population," remarked Dr. Kenneth Attie, Vice President, Medical Affairs at Insmed. "Once-daily administration of the complex provided physiologic replacement of IGF-I and was well-accepted by patients and their families in the study," he added. The safety profile of IPLEX proved favorable as instances of low blood sugar (hypoglycemia) were mostly mild and asymptomatic, with no hypoglycemic seizures reported. Patients with severe GH insensitivity have a predisposition to hypoglycemia without treatment. Of the subjects enrolled in the study, 28% reported a history of hypoglycemia prior to treatment. This was similar to the proportion of subjects (31%) who experienced at least one incidence of hypoglycemia during the first year of treatment with IPLEX, usually described as mild and asymptomatic. In the study, no patients discontinued IPLEX due to a related adverse event. Two patients had papilledema (one with a blocked pre-existing ventriculo-peritoneal shunt and one asymptomatic). No instances of facial nerve paralysis were reported. As is common with protein therapeutics, antibodies to the protein complex were detected in most patients, but were not associated with growth attenuation or adverse effects. IPLEX was approved as an orphan drug by the United States Food and Drug Administration in December 2005 for the treatment of growth failure in children with severe primary IGF-I deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.
|
|
|
