Published in Pipeline

Neurotech Pharma earns BLA priority review for MacTel cell therapy

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4 min read

The FDA has granted Neurotech Pharmaceuticals, Inc. priority review for the Biologics License Application (BLA) of NT-501, an encapsulated cell therapy (ECT) designed to treat macular telangiectasia type 2 (MacTel).

First: A quick refresh on MacTel.

MacTel is a rare, age-related neurodegenerative retinal disease that causes gradual (over 10 to 20 years) central vision deterioration—impacting both near and far vision—and, as a result, significantly impacts patients’ quality of life.

  • Patient demographic affected: Aged 40+

How does it develop?

  • Type 1 MacTel: Tiny blood vessels around the fovea dilate and form in aneurysms, leading to leakage and swelling (ie: macular edema)
    • Typically only manifests in one eye
  • Type 2 MacTel: Tiny blood vessels located around the fovea become abnormal and may dilate; new blood vessels may form under the retina (ie: macular neovascularization), leading to fluid leakage, swelling, or bleeding
    • Typically affects both eyes (in varying severity)

Next up: Neurotech.

Founded in 2000 and headquartered in Cumberland, Rhode Island, the private, clinical-stage biotechnology company is developing sustained drug deliveries for chronic retinal diseases—with a specific focus on MacTel.

  • Its core platform technology: The ECT platform

Tell me about this platform.

The ECT is designed as a versatile platform that continuously produces and delivers targeted therapies—in combination and singularly—to the back of the eye, for the long-term.

Its basis: The NTC-200 cell line, which operates under low oxygen and nutrient conditions and can be genetically manipulated under strict selection methods.

How does it work, exactly?

The platform features a small, semi-permeable capsule filled with proprietary allogeneic retinal epithelium cells (RPE) cells genetically modified to produce specific therapeutic proteins for targeted disease treatments.

  • The process: This capsule is inserted into a patient’s vitreous and sutured to the sclera during an outpatient procedure
  • Then: Once in place, the semipermeable membrane of the capsule enables critical nutrients to enter and therapeutic proteins to exit into the vitreous
    • The intended result: continuous and targeted treatment
      • Plus: During this process, the membrane provides protection to the encapsulated RPE cells from a patient’s human system, enabling both functionality and long-term survival

Alrighty, now what should we know about this cell therapy?

Constructed as an ocular implant, NT-501 relies on the ETC platform to deliver therapeutic doses of ciliary neurotrophic factor (CNTF) straight into the retina.

  • The intended purpose: Slow disease progression

And the clinical data on it?

According to Neurotech’s pipeline, NT-501 has completed six clinical trials, including safety, safety and efficacy, and extension studies.

Most recently: Two randomized, multicenter, sham-controlled phase 3 studies evaluating the safety and efficacy of NT-501 for MacTel Type 2:

  • Protocol A: NCT03316300
  • Protocol B: NCT03319849
    • Primary endpoint: Rate of change in ellipsoid zone (EZ) area loss from baseline to 24 months

And the findings?

Positive topline data on 200+ MacTel patients reported in November 2022 demonstrated statistical significance for the primary endpoint, with the implant determined to be safe, durable, and well-tolerated in both studies.

The numbers:

  • 56.4% rate of reduction in Protocol A (p<0.0001)
  • 29.2 % rate of reduction in Protocol B (p=0.021)

Sounds promising! So what do we know about this BLA?

The FDA’s priority review of NT-501’s BLA essentially means that the ocular implant has been deemed “sufficiently complete” to enable a substantive FDA review.

And what’s the timeline for this review?

Per the standard FDA review process: 6 months.

Which means …

A Prescription Drug User Fee Act (PDUFA) goal date has been set for: Dec. 17, 2024.

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