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From diabetes to DMD
by Kelsey Kaustinen  |  Email the author


NEW YORK—N-Gene Research Laboratories Inc. (N- Gene), a diabetes research biopharmaceutical company based in New York, recently announced a new discovery centered on BGP-15, the company's lead compound. Australian scientists working in close cooperation with N-Gene researchers discovered that the compound, which is currently being developed as a diabetes treatment, could have potential as a novel therapy for treating Duchenne muscular dystrophy (DMD). The results were recently published in Nature.  
BGP-15 is a heat shock protein inducer and a co-inducer of heat shock protein 72 (Hsp72) that causes an increase in insulin sensitivity by blocking JNK phosphorylation. The compound is currently being tested in an international Phase IIb study in patients with type 2 diabetes, with results expected by the end of this year.
As a diabetes therapy, BGP-15 exhibits a variety of advantages over existing approaches, says Gabor K. Kalman, CEO of N-Gene. It offers a better safety profile, he says, without the cardiovascular issues, water retention or liver toxicity often seen in some diabetes drugs. In addition, Kalman adds that as the compound is "basically a cytoprotective agent," it could have been developed independently from diabetes as a cardioprotective drug as well.
DMD is a form of muscular dystrophy that gets worse rapidly. Caused by a defective gene for dystrophin, a protein found in the muscles, it manifests itself in muscle weakness, which begins in the legs and pelvis. The condition occurs in roughly 1 out of every 3,500 male infants. There is no cure or any effective therapies for DMD. In the recently published work, BGP-15 was tested in animal models of DMD and was found to not only make a pivotal protein functional again, reducing muscle damage, but to also increase the strength, endurance and lifespan of the animal models.  
"We believe this publication reinforces the expectation that N-Gene's platform technology, based on stress-response regulation, will eventually result in the emergence of a novel drug class with diverse therapeutic directions," Dr. Peter Literati, co-founder and chief scientific officer of N-Gene, said in a press release.  
BGP-15's potential in treating DMD came as a surprise, says Kalman. The company's co-chief scientific officer, Dr. Mark Febbraio, is also the head of the Cellular and Molecular Metabolism Laboratory and director of Basic Science in the Division of Metabolism and Obesity at the Baker IDI Heart and Diabetes Research Institute. Febbraio has a working relationship with Prof. Gordon Lynch, head of the Department of Physiology at the University of Melbourne, says Kalman, and the two discussed BGP-15's potential in treating muscular dystrophy, which then led to the animal studies. Lynch led the research, which was conducted by Dr. Stefan Gehrig of the Basic and Clinical Myology Laboratory in the University of Melbourne's Department of Physiology in collaboration with Febbraio and his team at Baker IDI Heart and Diabetes Institute.  
It was noted in the paper that the results "provide evidence that increasing the expression of Hsp72 in muscle (through the administration of BGP-15) has significant therapeutic potential for DMD and related conditions, either as a self-contained therapy or as an adjuvant with other potential treatments, including gene, cell and pharmacological therapies." A news release from the Department of Medicine, Dentistry and Health Sciences on the University of Melbourne's website noted that "upregulating heat shock protein 72 in skeletal muscles of mice with severe muscular dystrophy, improved muscle strength, slowed the disease progression and increased lifespan by more than 20 percent."  
Kalman says the company believes heat shock proteins could have potential in several other indications as well.  
"We believe that there may be a part in Parkinsonism, in Alzheimer's and other neurodegenerative and metabolic diseases, in polycystic ovarian syndrome and muscle dystrophy," says Kalman. "So quite a wide range of therapies may benefit from heat shock protein science."  
He adds that "it is yet a dilemma" whether they will pursue the muscular dystrophy avenue with the compound, as the diabetes indication is both clearer and more advanced in development. According to Literati, the company will remain focused on advancing the compound within the diabetes indication, "but we look forward to seeking partners with which to advance this technology and realize its potential in the treatment of DMD as well as many other disease indications."  
"I believe that quite a lot of companies are looking for indications such as DMD, and we already had one tentative interest from a Big Pharma partner, and I believe that because this is such a unique result, what we could demonstrate, that it's really gearing interest or it will gear interest from Big Pharma," says Kalman. 

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