Novel Drug & Emergent Therapeutics | Ophthalmology | Retinitis Pigmentosa
Beacon Therapeutics presents positive 24-month results from Phase 2 SKYLINE Trial at AAO Annual Meeting
Time to read: 01:02 minutes
Originally published: 15 October 2024
Source: PR Newswire
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDC009
Category: Ophthalmology
Crossreference: Retinitis Pigmentosa, Rare Diseases
Keywords: XLRP, genetics, retinitis pigmentosa, gene therapy
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| Phase 2 Trial |
15 October 2024, PR Newswire Boston
The 24-month data from the Phase 2 SKYLINE trial showed encouraging results for AGTC-501, a gene therapy developed for X-linked retinitis pigmentosa (XLRP). Beacon Therapeutics, a leading ophthalmic gene therapy company, presented the results at the American Academy of Ophthalmology’s Annual Meeting in Chicago, Illinois, this week.
XLRP is a severe, aggressive condition affecting approximately three in every 100,000 people in South Africa1. Currently, no treatment exists for the disease.
AGTC-501, which expresses the full-length RPGR protein and targets both rod and cone photoreceptor damage, shows potential to be a breakthrough treatment.
The 24-month data showed a response rate of 57% (4/7) in study eyes treated with a high dose (6.8 E+11 vg/eye) of AGTC-501, defined as a patient who has an improvement in retinal sensitivity as assessed by microperimetry of at least 7 decibels (dB) in at least 5 loci.
Patients in the high-dose cohort also showed a robust improvement in mean retinal sensitivity.
In contrast, study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 showed a response rate of 25% (1/4).
Importantly, no serious safety concerns were noted. Any adverse events were mild to moderate, reinforcing AGTC-501's favourable safety profile.
The company is advancing its pivotal Phase 2/3 VISTA and Phase 2 DAWN trials to evaluate further this gene therapy’s potential.
| Clinical Trial | Clinicaltrial.gov Registration | Status |
|---|---|---|
| Phase 2 Complete |
Access Original Press Release
15 October 2024 | PR Newswire | Beacon Therapeutics Announces Positive 24-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa
Additional References
1. Retina South Africa: Clinical Trials. Retrieved 14 October 2024
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FDA Approves New Antibiotic for Three Different Uses
Originally published: 04 April 2024
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDN008
Category: Paediatrics & Neonatology
Crossreference: Infectious Diseases, Antimicrobials
Category tags: Antimicrobials, Antibiotics, CAP, pneumonia, URTI, LRTI
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| Completed |
The U.S. Food and Drug Administration has approved Zevtera (ceftobiprole medocaril sodium for injection) for the treatment of Staphylococcus aureus bloodstream infections (SAB), acute bacterial skin and skin structure infections (ABSSSI), and community-acquired bacterial pneumonia (CABP) in adults and pediatric patients aged three months to less than 18 years with CABP.
Zevtera’s efficacy in treating SAB was assessed in a randomized, controlled, double-blind trial involving 390 subjects. A total of 69.8% of subjects who received Zevtera achieved overall success compared to 68.7% of subjects who received the comparator.
For ABSSSI, Zevtera's efficacy was evaluated in a multinational trial with 679 subjects. Of those who received Zevtera, 91.3% achieved an early clinical response within 48-72 hours compared to 88.1% of subjects who received the comparator.
In a double-blind, multicenter trial involving 638 adults with CABP, 76.4% of subjects who received Zevtera achieved clinical cure compared to 79.3% of subjects who received the comparator.
Evidence from trials in adults and pediatric subjects aged three months to less than 18 years with pneumonia supported the approval for pediatric patients with CABP.
Common side effects of Zevtera for adults with SAB include anaemia, nausea, hypokalemia, vomiting, diarrhoea, increased hepatic enzymes, increased blood creatinine, high blood pressure, leukopenia, fever, abdominal pain, fungal infection, headache, and dyspnea.
Clinical Trials & Supporting Information
Access Original Press Release
03 April 2024, FDA Approves New Antibiotic for Three Different Uses
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Disclaimer
This article is compiled from a variety of resources. Every effort has been made to correctly attribute quotes and content. Where possible all information has been independently verified. The Medical Education Network bears no responsibility for any inaccuracies which may occur from the use of third-party sources. If you have any queries regarding this article contact us
Fact-checking Policy
The Medical Education Network makes every effort to review and fact-check the articles used as source material in our summaries and original material. We have strict guidelines in relation to the publications we use as our source data, favouring peer-reviewed research wherever possible. Every effort is made to ensure that the information contained here is an accurate reflection of the original material. Should you find inaccuracies, out of date content or have any additional issues with our articles, please make use of the contact us form to notify us.
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| Completed |
