Item 8.01 Other Events.
On
4D-125 Interim Clinical Data Summary
The data described are from the first-in-human, on-going Phase 1/2 dose
escalation and dose expansion clinical trial assessing intravitreal 4D-125,
4DMT's targeted and evolved R100-based product candidate for XLRP. As of the
data cutoff date (
Interim Safety Data Summary
• 4D-125 was well-tolerated in all eight XLRP patients, including in five patients at the top dose level of 1E12 vg/eye. • No dose-limiting toxicities or serious adverse events were observed. • No chronic inflammation was observed. • Transient, grade 1+ anterior chamber and/or vitreous cells were observed in two of the eight patients at a single protocol-defined assessment timepoint (SUN1 & NEI2 grading scales) • One patient had grade 1+ vitreous and anterior chamber cells • One additional patient had grade 1+ vitreous chamber cells
Interim Clinical Activity Summary
• In the two dose escalation patients who were evaluable for clinical activity in both the treated and untreated control eyes, interim data from both patients demonstrated anatomical retina preservation as measured by EZ Area progression, a measurement of intact photoreceptors, in the treated eye as compared to the untreated control eye in the same patient. In addition, interim data from both patients demonstrated functional improvements as measured by increases in the mean retinal sensitivity in the treated eye, and a greater number of loci gaining ³ 7 dB sensitivity in the treated eye, as compared to the untreated control eye in the same patient. • Patient 3 (3E11 vg/eye cohort - 9 months follow-up): Decreases from baseline EZ Area were -12.4% in the treated eye compared to -16.2% in the untreated control eye (~23% lower relative progression rate). An increase in mean retinal sensitivity was demonstrated, with an increase of +1.65 dB in the treated eye from baseline compared to +0.25 dB in the untreated control eye. The number of loci gaining greater than ³ 7 dB sensitivity were six in the treated eye compared to one in the untreated control eye. • Patient 5 (1E12 vg/eye cohort - 6 months follow-up): Decreases from baseline EZ Area were -20.2% in the treated eye compared to -28.7% in the untreated control eye (~30% lower relative progression rate). An increase in mean retinal sensitivity was demonstrated, with an increase of +0.90 dB in the
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treated eye from baseline compared to +0.10* dB in the untreated control eye. The number of loci gaining greater than ³ 7 dB sensitivity were three in the treated eye compared to zero in the untreated control eye. * note: microperimetry data for this patient's untreated eye were evaluable at 4 months but not available at month 6 due to an inability to fixate with the untreated control eye at that visit. 1. Standardization of Uveitis Nomenclature Grading Scheme -SUN Working Group 2005 (Jabs et al., 2005) 2. National Eye Institute Grading System for Vitreous Cells - (Mahendradas, Khanna, Kawali, & Shetty, 2014)
Forward-Looking Statements
This Current Report on Form 8-K contains forward-looking statements for purposes
of the safe harbor provisions of the Private Securities Litigation Reform Act of
1995. Forward-looking statements include statements regarding 4DMT's clinical
development plans for 4D-125. In some cases you can identify these statements by
forward-looking words such as "may," "will," "continue," "anticipate," "intend,"
"could," "project," "expect" or the negative or plural of these words or similar
expressions. Forward-looking statements are not guarantees of future performance
and are subject to risks and uncertainties that could cause actual results and
events to differ materially from those anticipated, including, but not limited
to: the impact of COVID-19 on countries or regions in which 4DMT has operations
or does business, as well as on the timing and anticipated results of 4DMT's
clinical trials, strategy and future operations; the delay of any current or
planned clinical trials for the development of 4DMT's drug candidates, the risk
that the results of its clinical trials, including any initial data therefrom,
may not be predictive of future clinical trial results, including those from
current and future clinical trials; 4DMT's ability to successfully demonstrate
the safety and efficacy of its drug candidates; the timing and outcome of 4DMT's
planned interactions with regulatory authorities; and obtaining, maintaining and
protecting its intellectual property. 4DMT discusses many of these risks in
greater detail under the heading "Risk Factors" contained in its quarterly
report on Form 10-Q for the quarter ended
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