Scaffold Therapeutics is developing oral therapies for ALS, a fatal disease with no approved treatments that change its course. The biology behind our approach also points the way to other diseases driven by the same protein, TDP‑43.
In nearly all ALS patients, a protein called TDP‑43 becomes overactive. Our drugs act on a chemical signal that controls how TDP‑43 reads its instructions, restoring its normal activity level rather than chasing symptoms. That same overactivity is seen in other neurodegenerative diseases, which is why a win in ALS opens doors elsewhere.
Our prototype is taken by mouth and crosses into the brain, a challenge that has stopped many drug discovery programs before they reach patients. Early animal studies show strong brain exposure and a clean tolerability profile.
A simple test on spinal fluid identifies the patients most likely to benefit and lets us measure the drug's effect directly. So even a small human trial can produce meaningful data about how well the therapy works and who will respond best to treatment.
ALS is our focus. There are roughly 30,000 patients in the US, with median survival of 2–5 years from symptom onset, and not a single approved drug that addresses the cause of the disease. In more than 97% of those patients, TDP‑43 is not properly regulated. That's the problem we're solving.
The same TDP‑43 pathology manifests in other neurodegenerative diseases, so a drug that succeeds in ALS has a credible path to a much larger patient population. Scaffold is building the first phase of a platform for treating TDP‑43 driven neurodegenerative diseases.
An oral small molecule with confirmed brain exposure, demonstrated target engagement and clean early tolerability in animal studies.
Developing a clinical medicine from our advanced set of development candidate molecules.
The preclinical work required to file with the FDA, followed by an early‑stage trial in ALS patients.
TDP‑43 is the next frontier in brain disease. The biology is established, the patients are waiting, and the chemistry is finally within reach.