View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
October 11, 2021updated 11 Jul 2022 2:22pm

4D Molecular’s therapy shows improvements in Phase I/II XLRP trial

In the trial, 4D-125 was well-tolerated with no treatment-associated serious adverse events reported.

4D Molecular Therapeutics has reported interim safety and clinical activity results from the Phase I/II clinical trial, where its intravitreal 4D-125 demonstrated functional improvements in patients suffering from advanced X-linked retinitis pigmentosa (XLRP).

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

4D-125 is a directed and advanced R100-based product candidate of the company.

The ongoing Phase I/II dose-escalation and dose-expansion trial is analysing intravitreal 4D-125 to treat XLRP.

As of 1 September, eight subjects with clinically advanced XLRP due to RPGR gene mutation were recruited.

Findings showed that 4D-125 was well-tolerated in all eight subjects, including five participants receiving the highest dose.

No dose-limiting toxicities or serious adverse events were reported in the trial.

No chronic inflammation was noted, and transient, grade 1+ anterior chamber and/or vitreous cells were seen in two subjects at assessment timepoint defined by the single protocol.

Interim results also showed that two subjects experienced anatomical retina preservation in the treated eye versus the untreated control eye of the same patient.

Anatomical retina preservation was assessed by ellipsoid zone Area progression, which is a measure of intact photoreceptors.

Furthermore, two subjects had functional improvements as assessed by a rise in the mean retinal sensitivity and number of loci gaining ≥ 7dB sensitivity in the treated eye as against the control eye which is not treated.

The company plans to progress to enrolment in the dose-expansion cohort to include subjects with less advanced disease.

4D Molecular Therapeutics co-founder and CEO David Kirn said: “These are the first clinical data reported with a product invented from our Therapeutic Vector Evolution platform at 4DMT, and these interim data demonstrate clinical proof-of-concept for safety, tolerability and clinical activity.

“These data support our belief that 4D-125 is well tolerated and has the potential to both slow the progressive loss of photoreceptors in patients with XLRP after a single intravitreal injection and to improve visual function.”

Related Companies

Free Case Study
img

Direct-to-Patient Trials: How IRT Plays an Important Role in Bellerophon's Direct-to-Patient Trials

As the industry strengthens its focus on patient centricity, Direct-to-Patient clinical trials have emerged as a popular trial design that have the potential to increase patient recruitment and retention. IRT plays a crucial role in the success of a Direct-to-Patient trial. Because drug supplies are being managed and shipped from distribution facilities directly to patients’ homes, a sponsor must have a high-quality system in place to accurately track the chain of custody, ensure patient-blinding and handle other logistical challenges. What You Will Learn Benefits and challenges associated with the Direct-to-Patient model Bellerophon's top considerations when implementing this trial design How IRT can equip study teams to successfully track chain of custody, ensure patient blinding, and handle logistical challenges
by Suvoda
Enter your details here to receive your free Case Study.

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena