Amylyx is starting to recruit for a phase III clinical trial

For PSP Patients and Care Partners: Amylyx is starting to recruit for a phase III clinical trial to evaluate the clinical safety, tolerability, and efficacy of a potential treatment for adults with PSP. We encourage you to learn more about this effort. Below, you will find more information on the trial and a link to a resources that Amylyx created about the trial.

The ORION trial will evaluate to see if a study drug is safe and effective as a treatment for adults living with PSP.  The trial will compare the safety and effectiveness of drug AMX0035 to a placebo that looks and tastes the same as the investigational drug and is given the same way. However, the placebo does not contain the investigational drug. Eligible participants will be chosen at random to receive AMX0035 or placebo. Participants will have a 60% chance of receiving AMX0035 and 40% chance of receiving a placebo in the randomized phase.

Who can participate?

An individual may be able to take part in the study if they meet the following qualifications:

• Diagnosis of possible or probable PSP also known as PSP-Richardson Syndrome (based on MDS 2017 criteria)
• Experiencing PSP symptoms for less than 5 years
• Age 40-80 years
• Able to walk independently at least 5 steps with minimal assistance
• Live at home or in an assisted living facility, not in a skilled nursing or memory care facility
• Have a reliable study partner to accompany on study visits
• Willing/able to undergo a brain MRI and lumbar puncture twice during the randomized phase, and 1 additional time during the OLE phase
• Participants must NOT require the use of a feeding tube
• If participants are taking medications such as coenzyme Q10, levodopa/carbidopa, a dopamine agonist, etc., then the dosing of these medications must be stable.

Visit clinicaltrials.gov for the full ORION trial eligibility criteria.

Study Sites:

• The ORION trial will be conducted at numerous centers in North America, Europe, and Japan. The latest information is available at clinicaltrials.gov
• Participants and their trial partner can receive travel reimbursement for required in-clinic study visits.
• View planned sites here. https://www.amylyxpsptrial.com/

What can a participant expect?

• You will be in the ORION Study for up to 2 years and 4 months. After the screening visit at the study clinic, participants will have up to 8 more visits to the study clinic and up to 12 remote visits by phone or video call.
• Expect to undergo a brain MRI and lumbar puncture twice during the randomized phase.
• Participants will complete questionnaires at regular intervals throughout the trial.
• Participants (with the help of their study doctor or study coordinator) will document all the medications they take, medical aids they use, and falls they experience during the trial.

Additional Resources:

• Questions: clinicaltrials@amylyx.com
• Study website: www.amylyxpsptrial.com/
• Clinicaltrials.gov: clinicaltrials.gov/study/NCT06122662?term=ORION&rank=1

Overview of Study Timing:

• Screening Phase: First, participants will be screened for eligibility.
• Randomized Phase (fifty-two weeks): Eligible participants will be chosen at random to receive AMX0035 or placebo. Participants will have a 60% chance of receiving AMX0035 and 40% chance of receiving a placebo in the randomized phase.
  • Including screening period and follow-up after the treatment period, participants will be in the randomized phase of the ORION trial for a maximum of about 14 months (about 62 weeks).
• Open Label Extension Phase: Participants who complete the randomized phase will be given the option to enroll in the OLE phase where all participants receive AMX0035.