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.

From ChatGPT

Key Characteristics:

• Flexibility: Type C meetings can cover a wide range of topics, providing flexibility in addressing specific challenges or concerns in the drug development process.
• Less Formal than Type A and B: Type A meetings are typically for more urgent issues (e.g., critical development decisions), while Type B meetings are for discussions of major clinical trial development plans (like end-of-phase 2). Type C meetings are the most common type and can address a broad range of issues.
• Outcome: The meeting will provide the drug sponsor with written feedback from the FDA on the discussed issues, helping to guide the development of the drug.

Examples of When a Type C Meeting Might Be Requested:

• Determining the appropriate clinical trial design or endpoints for a Phase 3 study.
• Clarifying statistical methodologies for analyzing clinical trial data.
• Seeking input on clinical development strategies or preclinical data before advancing to human trials.

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.