Pinned straw:
US Patent – Pitt Hopkins Syndrome (24/2/25)
NEU has been awarded a US patent for NNZ-2591 to treat Pitt Hopkins syndrome (PTHS) with an expiry date of April 2040.
There are no other FDA approved treatments for PTHS and NEU has both Orphan Drug designation and Fast Track designation for it’s PTHS program which has had positive Phase 2 results.
Good news, market has rewarded it so far +11%, but I have to say that it should probably have already been priced in.
Disc: I own RL+SM
Neuren Pharmaceuticals adds another feather to their cap for NNZ-2591 for hypoxic-ischemic encephalopathy (HIE) a type of brain injury caused after birth when the brain does not receive enough oxygen or blood flow.
Announcement: 06h1pvfr55c99y.pdf
It would qualify for Orphan Drug and Rare Pediatric disease designation with the FDA
A pre-IND meeting with FDA is targeted in Q4 2025.
Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins syndrome
NEU announced at 1pm today they have fast track approval for their Pitt Hopkins syndrome trials with the FDA. Given the untreated nature of this condition it was likely this would happen, but it's now official.
The fast track effectively puts them at the front of the que for FDA reviews when they get to them - which can save considerable time when dealing with what is already an under resourced FDA that is likely to be less resources with Trump/Musk/RFK. Below is the detail:
A drug that receives Fast Track designation is eligible for some or all of the following:
• More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval • More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
• Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
• Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA
Disc: I own RL+SM
@mikebrisy , it is a bit surprising as the impression i had previously was that they were just working through the fine print but everyone was more or less on the same level for the design endpoints. I don't see this development as a negative per se, more just part of the process.
I asked chatgpt about what a type C meeting was and it looks like its really just a more formal version of email correspondence. It looks like it is designed to work through any problem areas in person to save time and misunderstandings. I would think that NEU would have a pretty good understanding of what areas are currently ambiguous to them and this should give them the chance to move through these quicker than continued email correspondance.
In general, Type C meetings are a tool that helps the drug sponsor navigate the complexities of regulatory requirements and ensures alignment with the FDA's expectations throughout the drug development process.