A long-watched Parkinson’s therapy got a jumpstart last week with a major deal that puts more resources behind the program.
In 2012, funded by The Michael J. Fox Foundation (MJFF), Austrian biotech company AFFiRiS brought the first therapy targeting the Parkinson’s protein alpha-synuclein into clinical trials. Its PD01 vaccine prompts the body to produce antibodies against alpha-synuclein, clearing out the clumps of the protein believed to harm brain cells.
Read below for the latest on the full pipeline of alpha-synuclein therapies.
This week another MJFF-funded company, AC Immune in Switzerland, acquired the AFFiRiS alpha-synuclein therapeutics portfolio in an all-stock transition valued at $58.7 million.
“This partnership and the resulting advancement of our anti-alpha-synuclein vaccine candidates is a great achievement for AFFiRiS and good news for the entire PD community. It would not have been possible without the support, financial and intellectual, of The Michael J. Fox Foundation,” Günther Staffler, PhD, AFFiRiS Chief Technology Officer.
MJFF granted AFFiRiS nearly $3.5 million between 2010 and 2015 for pre-clinical development of its vaccine, a first-in-human Phase I trial, and follow-up trials of boost vaccines. Early trials showed positive results around safety and alpha-synuclein antibody response. AC Immune says it is ready to move an optimized version of PD01, called ACI-7104, into a Phase II trial.
This hand-off from a smaller company to a larger one with deeper pockets and more experience with drug development is a textbook example of our de-risking strategy. MJFF supports early, risky projects to help them build data that will attract partners who can usher them through larger, more expensive later-stage trials.
We also de-risk investments like AC Immune’s by supporting development of tools that will ease and speed testing of all therapies. AC Immune is a leader in the pursuit of a tool to image alpha-synuclein in the living brain. MJFF has granted the biotech more than $5 million since 2015 to develop an alpha-synuclein tracer, including $3.25 million as part of our 2020 Ken Griffin Alpha-synuclein Imaging Competition. Such a tool would speed development and testing of new treatments by allowing more nuanced selection of study participants and faster assessment of therapeutic impact on underlying disease biology.
“Any major deal to advance Parkinson’s therapies is heartening as they spur momentum toward patient hands. This AFFiRiS and AC Immune partnership is especially rewarding as we have supported each company and as an alignment of therapy and assessment tool may speed development of both,” said Jamie Eberling, PhD, MJFF Vice President of Research Programs.
Since AFFiRiS brought PD01 to trials in 2012, a dozen other therapies with varied approaches targeting alpha-synuclein have entered human studies:
Like ACI-7104, this treatment prompts the body to produce alpha-synuclein antibodies.
- UB-312 (Vaxxinity): active but not recruiting (following participants, analyzing data)
Instead of prompting the body to produce antibodies, these therapies introduce alpha-synuclein antibodies made in the lab.
- ABBV-0805 (Abbvie): active but not recruiting
These medicines aim to break up or prevent clumps of alpha-synuclein to keep cells healthy.
- Anle-138b (MODAG): recruiting in the United Kingdom
- YTX-7739 (Yumanity): active but not recruiting
- NPT088 (Proclara): planning next trial
- ATH434 (Alterity Therapeutics): planning next trial
A handful have been funded by MJFF, and all will benefit from the creation of assessment tools such as an alpha-synuclein imaging tracer. A diverse portfolio of approaches and programs is heightening the likelihood of success in bringing an alpha-synuclein therapy to patients.