By Deena Beasley
LOS ANGELES, Dec 9 (Reuters) - Nearly a third of amyotrophic lateral sclerosis (ALS) patients treated with an experimental drug from Sangamo BioSciences Inc had muscle improvement, according to preliminary mid-stage study results announced on Tuesday.
The small Phase 2 study showed that six out of 19, or 32 percent, of patients injected on two separate occasions with the drug, called SB-509, had improved muscle function after about four months, based on manual testing.
Historical records for similar untreated patients showed that 17 percent had improvement in muscle manual testing (MMT), the company said at a medical meeting in Berlin.
The data, presented in Berlin, covered patients who had completed the first 120 days of the 180-day trial.
'The data so far ... show an approximate doubling of the patients who responded,' said Sangamo Chief Executive Edward Lanphier.
He said he was not aware of the magnitude of the muscle function improvement seen in the trial, which is expected to conclude in the second quarter of next year.
Forty-five patients were enrolled: five were not available for follow-up, one died, one withdrew after the first dose and another went into hospice care, according to Sangamo.
The drug was well-tolerated and there were no adverse side effects associated with treatment, researchers said.
Lanphier said the early data appears to indicate 'that we want to treat more frequently.'
The company said that of the patients so far shown to have muscle improvement, five showed parallel improvement in one or more additional trial goals, such as the ALS-FRS-R scale, which also measures respiratory function, or forced vital capacity, a measure of lung function.
SB-509, which is also being studied as a treatment for nerve damage caused by diabetes, is a 'zinc finger' activator of vascular endothelial growth factor, which has been shown to have a significant role in ALS.
Sanofi Aventis SA's Rilutek, known generically as riluzole, is the only approved treatment for ALS, also known as Lou Gehrig's disease -- a fatal neuro-degenerative disease afflicting about two out of 100,000 people a year.
Lanphier said he had no comment on when or if Sangamo would launch a pivotal-stage trial of SB-509 in ALS patients, but did say that such a trial would compare the drug to Rilutek, which is designed to protect nerve cells from exposure to a chemical called glutamate.
'One of the challenges in ALS is to find different drugs that act in different areas of the nervous system,' said Ely Benaim, vice president of clinical affairs at Sangamo. 'There is the potential for combination therapies.'
He said the medical community has come to realize that there is no 'magic pill' out there for ALS and drugs that can improve quality of life -- allowing patients to feed themselves or continue to push a wheelchair -- could have tremendous value.
(Reporting by Deena Beasley; Editing by Steve Orlofsky) Keywords: SANGAMO ALS (deena.beasley@thomsonreuters.com; 1-213-955-6746) COPYRIGHT Copyright Thomson Reuters 2009. All rights reserved. The copying, republication or redistribution of Reuters News Content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters.
LOS ANGELES, Dec 9 (Reuters) - Nearly a third of amyotrophic lateral sclerosis (ALS) patients treated with an experimental drug from Sangamo BioSciences Inc had muscle improvement, according to preliminary mid-stage study results announced on Tuesday.
The small Phase 2 study showed that six out of 19, or 32 percent, of patients injected on two separate occasions with the drug, called SB-509, had improved muscle function after about four months, based on manual testing.
Historical records for similar untreated patients showed that 17 percent had improvement in muscle manual testing (MMT), the company said at a medical meeting in Berlin.
The data, presented in Berlin, covered patients who had completed the first 120 days of the 180-day trial.
'The data so far ... show an approximate doubling of the patients who responded,' said Sangamo Chief Executive Edward Lanphier.
He said he was not aware of the magnitude of the muscle function improvement seen in the trial, which is expected to conclude in the second quarter of next year.
Forty-five patients were enrolled: five were not available for follow-up, one died, one withdrew after the first dose and another went into hospice care, according to Sangamo.
The drug was well-tolerated and there were no adverse side effects associated with treatment, researchers said.
Lanphier said the early data appears to indicate 'that we want to treat more frequently.'
The company said that of the patients so far shown to have muscle improvement, five showed parallel improvement in one or more additional trial goals, such as the ALS-FRS-R scale, which also measures respiratory function, or forced vital capacity, a measure of lung function.
SB-509, which is also being studied as a treatment for nerve damage caused by diabetes, is a 'zinc finger' activator of vascular endothelial growth factor, which has been shown to have a significant role in ALS.
Sanofi Aventis SA's Rilutek, known generically as riluzole, is the only approved treatment for ALS, also known as Lou Gehrig's disease -- a fatal neuro-degenerative disease afflicting about two out of 100,000 people a year.
Lanphier said he had no comment on when or if Sangamo would launch a pivotal-stage trial of SB-509 in ALS patients, but did say that such a trial would compare the drug to Rilutek, which is designed to protect nerve cells from exposure to a chemical called glutamate.
'One of the challenges in ALS is to find different drugs that act in different areas of the nervous system,' said Ely Benaim, vice president of clinical affairs at Sangamo. 'There is the potential for combination therapies.'
He said the medical community has come to realize that there is no 'magic pill' out there for ALS and drugs that can improve quality of life -- allowing patients to feed themselves or continue to push a wheelchair -- could have tremendous value.
(Reporting by Deena Beasley; Editing by Steve Orlofsky) Keywords: SANGAMO ALS (deena.beasley@thomsonreuters.com; 1-213-955-6746) COPYRIGHT Copyright Thomson Reuters 2009. All rights reserved. The copying, republication or redistribution of Reuters News Content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters.
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