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 afterward.

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!

Adjusting Protein In An Adult With MSUD

A little over a year ago, in January, Jeff was just not himself. He had limited energy, was not eating well, and often appeared slightly agitated.

Read More

Sweet Odyssey

Artur Santos was born on January 18, 2002, the second son of Soraya and Idario Santos. They never imagined how their lives were about change as the second baby arrived.

Read More

MSUD Awareness Day

Now more than ever before we are hearing about National Awareness Days which are set by organizations or the government to bring attention to issues of importance.

Read More

MSUD Advocacy Update

Healthcare and issues facing the rare disease community have been at the forefront of national conversations over the past 6 months.

Read More

I want to help out, but I don’t know how

Have you ever wanted to help out the MSUD Family Support Group but were unsure about how to contribute? The MSUD Family Support Group is actively looking for volunteers to assist with advocacy and fundraising efforts.

Read More

20th Annual White Horse Beach Charity Golf Tournament

On July 29, 2017 Charlie O’Rouke and his committee held the 20th Annual White Horse Beach Charity Golf Tournament.

Read More


A Child's Life

Subscribe to our mailing list

Signup To Our Newsletter Signup with your email address to receive news and updates