As discussed at the 2016 MSUD Symposium and in the previous issue of this newsletter, Acer Therapeutics is following up on the research initiated by Dr. Lee on phenylbutyrate and its potential to aid in the control of branched chain amino acid levels in those with MSUD. I was asked to provide a quick update on ACER-001 – the taste-masked, immediate release formulation of sodium phenylbutyrate – that we at Acer are developing for the treatment of MSUD.
We’ve spent the better part of the past year optimizing
the formulation, ensuring that it is completely
taste-masked, yet behaves like regular sodium phenylbutyrate
once it’s in the body. It’s really important
that we get this right, so that patients taking ACER-
001 get the maximum benefit of the drug, while not
detecting the horrific taste of phenylbutyrate! While
this has been an incredibly challenging project, we
believe we now have the best formulation developed
to take into the clinic.
Before we begin the phase 2 clinical trial for MSUD,
we need to run a quick study in healthy volunteers
to make sure the drug is completely taste-masked,
safe, and behaves similarly to regular phenylbutyrate
in the body. This is a very quick study that should be
completed in the fall. Assuming everything goes well
with this study, we will begin the MSUD trial shortly
As a reminder, the MSUD phase 2 trial will enroll
approximately 60 patients at 10-15 clinics across
the US. Once a patient begins the study, they will be
required to maintain a very tightly-controlled diet
for about a month. ACER-001 will be added on top of
their diet for about two weeks, to determine if ACER-
001 + diet can lower levels of leucine by a meaningful
amount over diet alone.
We invite you to visit our recently updated website,
where you can access patient resources, subscribe to
our mailing list to receive program updates and clinical
trial information, or ask questions directly to the
company. Please visit:
We look forward to hearing from you, and keeping you updated on our progress!
Acer Therapeutics: working diligently to advance ACER-001