Cisbio eBook - 22

Trends in Protein-Protein Interactions Research | Advantages May Place Peptides Ahead of Small-Molecule Drugs

"This is something that we have very convincingly
shown in our Phase II trial," asserts Dr. Francois. In
this trial, APL-2 was administered as an intravitreal
injection in the study eye monthly or every other
month for 12 months, followed by six months of
monitoring after the end of treatment.
"At 12 months, APL-2, administered monthly via
intravitreal injection, showed a 29% reduction in
the rate of GA lesion growth compared to sham,"
the company indicated in a press release. "With
every other month administration, a 20% reduction was observed. Additionally, in a post hoc
analysis, a greater effect was observed during the
second six months of the study: a reduction in
growth rate of 47% with monthly administration,
and a reduction of 33% with every other month
administration."
22 | GENengnews.com

According to Dr. Francois, this trial demonstrates
that complement immunotherapy can slow
photoreceptor loss in patients with geographic
atrophy, a disorder that affects 1 million people in
the United States. No treatments for this disorder
are currently available on the market, and all
other leads in clinical development have failed.
"We look forward to our Phase III clinical trials
to test whether we can repeat the data from the
Phase II trials," states Dr. Francois.

Preventing Amyloid Deposition
"Many of the approaches to reduce amyloid-β
(Aβ) peptide have failed not because scientists
were unable to reduce the levels of the peptide,
but because their approach-the targeting of the
enzymes that cleave Aβ from its precursor-can
lead to undersirable side effects," says Nazneen
Dewji, Ph.D., president and CEO of Cenna
An illustration of the amyloid precursor protein at a cell
membrane. The intact protein binds to many structural and
receptor proteins outside cells, causing aberrant internal
signaling pathways. Preventing the enzymatic cleavage of
this protein is of major interest to drug manufacturers.

Getty Images / JUAN GAERTNER / SCIENCE PHOTO LIBRARY

geographic atrophy, the FDA has allowed investigators to use not visual acuity but, instead, the
growth of atrophy as the primary endpoint," says
Dr. Francois. The registration endpoint involves
measuring the area of retinal atrophy, which is
indicative of the rate of photoreceptor loss


http://www.GENengnews.com

Cisbio eBook

Table of Contents for the Digital Edition of Cisbio eBook

Contents
Cisbio eBook - 1
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