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A potential fast-track to market for NTI's drug candidate


Published 09-JUL-2024 14:47 P.M.


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Today, our biotech Investment, Neurotech International (ASX: NTI) has filed for Orphan Drug Designation with the US FDA for its drug candidate NTI164 to treat the rare neurological disorders PANDAS and PANS in children and adults.

Key points from today’s NTI announcement

  • Orphan Drug Designation provides incentives like tax credits, fee waivers, and 7 years of market exclusivity if approved
  • PANDAS/PANS is a rare autoimmune disorder with no approved treatments, estimated $1.2B annual market
  • Importantly, NTI expects FDA response on Orphan Drug status within 3 months

🎓Learn more: Orphan Drugs Explained

Why This Matters

Securing Orphan Drug Designation would be a major milestone for NTI and NTI164. It would validate NTI164's potential in treating PANDAS/PANS and put NTI on a fast-track to market with valuable incentives.

We think the combination of positive clinical data (which NTI already has in hand) and an Orphan Drug approval in this indication could drive a substantial re-rate for NTI, as we've seen with other companies that successfully brought treatments for rare paediatric neurological disorders to market (notably Neuren Pharmaceuticals).

Read more about the successful PANDAS/PANS clinical trial below:

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NTI’s clinical trial meets primary endpoints for neurological disease

How this news impact our NTI Investment Memo

NTI is targeting “orphan” diseases - treatments that are highly valuable
NTI is currently targeting PANDAS/PANS and Rett Syndrome, which are potentially eligible for orphan drug designation. The average price for an orphan drug is US$32,000 per year per patient and government approvals to market are fast tracked.

Source: NTI Investment Memo 18 September 2023

Today’s announcement is further evidence that NTI is delivering, and aligns well with one of the key reasons we Invested in NTI.

Competition risk
NTI will need to move quickly to establish its presence in the market. If progress is slow, alternative treatments could emerge hurting NTI’s prospects.

Source: NTI Investment Memo 18 September 2023

With the market exclusivity offered by Orphan Drug Designation, NTI could reduce competition risk - importantly there are no approved treatments in the market for PANDAS/PANS.

What's Next for NTI?

  • FDA response on Orphan Drug Designation expected in ~3 months
  • Advancing NTI164 through clinical pipeline across multiple rare neurological disorder indications (including Cerebral Palsy)