June 24, 2015
By
Alex Keown
, BioSpace.com Breaking News Staff
TEL AVIV, Israel – Israel-based
Alcobra Ltd.
said its drug treating Fragile X, a genetic disorder that could lead to autism, failed to meet its primary endpoint in a mid-stage trial.
The company’s stock dropped this morning in response to the news. Shares of
Alcobra (ADHD)
were down about 13 percent, hitting a low of $6.76 per share.
The drug, MDX (Metadoxine Extended Release), did not achieve statistical significance from baseline to week 6 of the inattentive subscale of the Attention Deficit Hyperactivity Disorder Rating Scale. The difference between the MDX-treated group and placebo was not statistically significant (p=0.21), favoring placebo, the company said.
However, MDX did achieve statistical significance, in the Intent-to-Treat (ITT) population, on two secondary endpoints, including the Vineland Adaptive Behavior Scale (VABS) Daily Living Skills Domain (p=0.044), and the computerized cognitive Test of Attentional Performance for Children (KiTAP) Distractibility subscale, the company said.
Patients who received MDX during the trial showed improvements in adaptive behavior and cognition, which suggests a meaningful finding,
Elizabeth Berry Kravis
, the lead investigator and a professor of Neurology at Chicago-based
Rush University Medical Center
said in a
statement
.
The average IQ for participants in the study was 57 for subjects taking MDX and 52 for subjects taking placebo, with a wide overall range of 18 to 107. Autism spectrum disorder, as classified by the
Autism Diagnostic Observation Schedule
, was present in over 80 percent of the 40 subjects who participated in the study.
MDX received an Orphan Drug designation from the
U.S. Food and Drug Administration (FDA)
for the treatment of Fragile X Syndrome. There are currently no approved drugs to treat the genetic disorder.
Fragile X Syndrome is caused by changes in the fragile X mental retardation 1 (FMR1) gene, according to the
U.S. Centers for Disease Control
. Fragile X affects both males and females, however, females typically show milder symptoms than males. It is unknown how many people are afflicted with Fragile X Syndrome, but the
CDC
estimates that about one in 5,000 males are born with the disorder.
Alcobra
said it plans to discuss trial results with the
FDA
before finalizing the design of the next study of MDX for Fragile X Syndrome.
Yaron Daniely
, president and chief executive officer of
Alcobra
, said he was encouraged the drug met its secondary endpoint.
“FXS is a difficult disease to treat and study, as demonstrated by the lack of approved treatments. Our findings suggest a clinically meaningful advance for patients and caregivers affected by FXS and we plan to meet with the
FDA
to determine next steps in advancing our research,” Daniely said in a statement.
MDX was generally well tolerated and no safety concerns were identified. The most common side effects were an upper respiratory infection and irritability.
When used to treat adolescent patients with attention deficit disorder, MDX met primary endpoints in a Phase II study, the
company said in March
.
As Rumors Swirl About GlaxoSmithKline Bid, Who Could Suitors Be?
Rumors are swirling
that Swiss-based
Roche
and U.S.-based
Johnson & Johnson
are eying the U.K. company for approximately $143 billion. But
Roche
and
J&J
aren’t the only companies though who have been thought could go after the elephant that is Glaxo.
Last month there was buzz that
Pfizer Inc.
was considering acquiring
Glaxo
, a year after it failed to acquire
AstraZeneca PLC
. Just this month over a third of respondents in a poll conducted by
BioSpace
believe that
AstraZeneca PLC
could be in the running to acquire struggling
GlaxoSmithKline (GSK)
.
So
BioSpace
wants to ask our readers again what they predict for this new dealmaking bonanza. Will
Glaxo
go—and if so, to whom?
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