Posts Tagged orphan drug
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on August 4, 2014
Rich Casey, President and CEO of Neuraltus (formerly Chairman and CEO of Scios), talked about treatment option for ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig’s Disease, a fatal neurodegenerative disorder of unknown etiology, at www.bio2devicegroup.org event.
Typically, the age of onset of ALS is anywhere between 40 and 70 years. Life expectancy after the diagnosis is between 2 and 5 years, although in very rare cases (e.g. in case of Stephen Hawking), it could be an extremely slow progression. No effective treatments for the disease exist. Motor neurons degenerate and early symptoms include muscle weakness, specifically involving arms and legs, lack of hand grip, deep tiredness and it progresses to difficulty in swallowing, breathing, and garbled speech.
Neuraltus’s lead product, NP001 is in clinical development, targeted for the treatment of ALS. While the exact cause of the disease is unknown, there is fair amount of evidence that points to increasing levels of inflammation in the macrophages. This increased inflammatory activity results in the release of factors in the central nervous system that leads to damage of motor neurons. Macrophages are cells produced by differentiation of monocytes in the tissues. Macrophages are highly specialized in removal of dead cells or debris. Additionally, macrophages also “present” antigen that plays a crucial role in initiating an immune response. There is an increasing evidence implicating neuroinflammation with the progression of ALS.
Neuraltus’s novel, proprietary drug, NP001 regulates macrophage activation and converts the activated inflammatory macrophages from activated, neurotoxic inflammatory state to a neuroprotective state. It thus normalizes the critical cellular environment. Phase I, single dose study indicated that that there are no safety concerns and the drug is well tolerated. Further, in a Phase II safety, tolerability and preliminary efficacy study, after administration of NP001, there occurred a close dependent reduction of inflammatory macrophages.
The dosing regimen is somewhat cumbersome, said Casey. First time, the patients come in the hospital and need to stay for 5 days and subsequently for 3 days. At low dose, phase II study results indicated positive trends in the ability of NP001 to slow the rate of disease progression but did not reach statistical significance. But at high dose, the drug freezes the disease in a 3rd of the patients. Additionally, the drug is found to be safe and well tolerated. This clearly looks like a very exciting potential treatment for a devastating disorder, ALS. The company is currently looking for funding. The talk was followed by Q&A.