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FDMT Positive Sentiment

In race to make gene therapy for age-related blindness, 4D Molecular announces positive results

Wednesday, October 2, 2024 BiopharmaWatch Research 2 min read
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Key Takeaway: 4D Molecular Therapeutics has reported promising results from its Phase 2 study on gene therapy for wet age-related macular degeneration. The therapy led to an 89% reduction in the need for standard injections, with 73% of patients not requiring further treatment for at least 32 weeks. The incidence of ocular inflammation was notably low among high-dose recipients, although the field has faced challenges in the past.
Price reaction · baseline $10.41 (2024-10-01 close) · hit pre-market · 2 other FDMT headline(s) in the window, move may be shared
day 0 close · peak
-1.3%

Market Sentiment Analysis

POSITIVE FACTORS

  • Significant reduction in need for standard injections (89%)
  • Majority of patients (73%) did not require additional shots for 32 weeks
  • Low incidence of ocular inflammation among high-dose patients

CONCERNS & RISKS

  • Previous setbacks in the field, such as Adverum's patient going blind
  • Limited sample size in the Phase 2 study (30 patients)

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+128%
120-day peak, hindsight
Typical move
11.2%
average across 9 past catalysts
Cash runway
~18 mo
Low dilution risk
Lead asset
4D-150 IVT
Phase 3 · Macular Neovascularization Secondary to Age-Related Macular Degeneration

Full Press Release Details

Wet age-related macular degeneration affects hundreds of thousands a year

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By Jason Mast
Sept. 18, 2024
General Assignment Reporter
Jason Mast
Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.
A long-running race to develop a gene therapy for the most common cause of age-related blindness is heating up.
On Wednesday, 4D Molecular Therapeutics announced new data from its program for the disease, known as wet age-related macular degeneration, or wet-AMD. In one 30-person Phase 2 study, patients’ need for standard-of-care injections fell by 89% after receiving gene therapy, and 73% did not need another standard-of-care shot for at least 32 weeks.
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Notably, only two of 71 patients who received a high dose of therapy have shown signs of ocular inflammation, 4D said. In 2021, another leading contender, Adverum, was set back after a patient with a related disease went blind in one eye.
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• Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.
Jason Mast
General Assignment Reporter
Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77.

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Frequently Asked Questions

What is wet age-related macular degeneration?

Wet age-related macular degeneration (wet AMD) is a leading cause of vision loss in older adults.

What were the results of the Phase 2 study?

The Phase 2 study showed an 89% reduction in the need for standard injections after gene therapy.

How many patients were involved in the study?

The study involved 30 patients receiving the gene therapy.

What is the incidence of ocular inflammation?

Only two out of 71 high-dose patients showed signs of ocular inflammation.

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Tags: FDMT Viatris Investor Conferences Healthcare 4D Molecular gene therapy wet AMD ocular inflammation Phase 2 study
Last updated: Oct 2, 2024

Biotech snapshot

Cash runway
$431M liquidity · $23M/mo burn
~18 mo
Low dilution risk
Lead asset
4D-150 IVTPhase 3
Macular Neovascularization Secondary to Age-Related Macular Degeneration →

Catalyst track record

+128%
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Peak hindsight return over a 120-day window, across 9 tracked FDA/PDUFA catalysts for FDMT.

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