Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) is taking a major step forward in the fight against Alport syndrome – a rare genetic disease – with the announcement of plans to move its experimental therapy, ELX-02, into a pivotal trial. In its Phase 2 clinical study, one patient achieved impressive remission after taking ELX-02, with the patient’s urine protein creatinine ratio (UPCR) plummeting from baseline.
Moreover, this remarkable result was achieved with the therapy being tolerated well by the patient. With ELX-02 potentially paving the way to a new therapy, those affected by Alport syndrome can look forward to a brighter future.
The promising success of ELX-02 in achieving remission in two of the first trial patients with Alport syndrome and nonsense mutations provides an exciting ray of hope to those living with this rare disease. Reversal of proteinuria in this patient population is rarely seen, making the results from the clinical tests of this potential new treatment an encouraging breakthrough. Professor Detlef Bockenhauer of University College London’s Paediatric Nephrology and Great Ormond Street Hospital NHS Foundation Trust in London believes further testing is warranted in this area.
Topline Results of ELX-02 Phase 2 Trial for Alport Syndrome
We are thrilled to report that one of our patients experienced a remission following dosing, as their average UPCR reading dropped an impressive 53% below the baseline! This is especially remarkable given that spontaneous reductions in proteinuria are rare in this population.
Eloxx has made exciting progress with its clinical trial of ELX-02, a potential breakthrough treatment for cystic fibrosis. Three patients have already been dosed and two of them have completed treatment. The third patient remains on the trial and results are eagerly awaited.
The patient experienced a remarkable outcome in their treatment, demonstrating a remarkable 49% decrease in UPCR from baseline to values during the treatment period, with a p-value of 0.009. Remission was achieved, which is an incredible accomplishment.
The UPCR value for week 6 was not included in this analysis due to a delay in processing, which risked protein degradation and thus an inaccurate result. To ensure reliability in the findings, it was prudent to exclude these data from the overall study.
No noteworthy alteration in UPCR was observed in the second patient who finished eight weeks of medication.
Both patients had the same rare Col4A4 mutation, but their treatments varied drastically to manage it. One thing was common between them: the use of renin-angiotensin-aldosterone system (RAAS) inhibitor drugs. However, the levels of RAAS inhibitors were quite different, suggesting that an individualized approach to treatment can be the most effective.
The results of the ongoing assessment of COL IV protein restoration for two patients with Col4A4 mutations is eagerly awaited. Through a kidney biopsy assay, the expression of COL IVA5 protein can be measured. It is hoped that this assessment will help to determine the efficacy of the treatments.
The results of the ongoing assessment of COL IV protein restoration for two patients with Col4A4 mutations is eagerly awaited. Through a kidney biopsy assay, the expression of COL IVA5 protein can be measured. It is hoped that this assessment will help to determine the efficacy of the treatments.
Patients have found ELX-02 to be a pleasant surprise, as its administration has yielded excellent results with no cases of early discontinuation due to side effects. This is in agreement with previous clinical studies that support its safety and effectiveness.
After 8 weeks of treatment, both patients underwent kidney biopsies, with results showing no signs of nephrotoxicity, confirming their excellent tolerance of the treatment.
Eloxx is thrilled with the promising preliminary results of its Phase 2 trial of ELX-02 for Alport Syndrome in patients with nonsense mutations. This devastating disease currently has no available disease modifying treatments and the positive results of this trial bring much needed hope for patients. Upon completion of the third patient, Eloxx plans to discuss the findings with the FDA with the aim of launching a pivotal trial, pending the necessary capital.
About the Phase 2 Clinical Study
This exciting Phase 2 trial for Alport Syndrome patients will explore the potential of ELX-02 to reduce proteinuria. Over two months, participants will take the therapy and have their progress monitored, measuring the primary endpoint of safety and the key secondary efficacy endpoint of proteinuria change from baseline every two weeks.
Results of how much COL4 is produced at the end of the two-month period will also be examined. With this data, we could be closer to understanding how ELX-02 could be a successful treatment for those suffering from Alport Syndrome.
About Alport syndrome
Alport Syndrome is a genetic disorder that affects an estimated 9,400 to 12,750 individuals each year. It is characterized by kidney disease, hearing loss and eye abnormalities, all caused by mutations in three genes: COL4A3, COL4A4 and COL4A5- genes needed to produce type 4 collagen. Those with a “nonsense mutation” tend to have far worse clinical outcomes, which, unfortunately, can not be treated or modified.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is revolutionizing the way we treat rare diseases with their innovative ribosomal modulation science and TURBO-ZMTM technology platform. Their landmark product candidate, ELX-02, is a small molecule drug designed to combat Alport Syndrome, a rare genetic condition that results in the inability to form full-length proteins. Currently being tested in Phase 2 clinical trials, ELX-02 has the potential to provide hope to those afflicted with this condition so that they can live their lives to the fullest.
Forward-looking Statements
Our vision of developing transformative treatments for individuals with genetic diseases involves taking a broad range of risks. But with that risk comes the potential for immense progress in reaching and treating those in need. By leveraging the latest research, clinical trials, and innovative therapies, we hope to deliver unprecedented breakthroughs and extend quality of life to those who suffer from nonsense mutations.
As we continue our research and development activities, we must also carefully evaluate our ability to secure capital, obtain the necessary resources, and remain compliant with our debt agreement. With dedication and innovation, we strive to make a real difference in individuals’ lives.
We are constantly striving to deliver the best possible outcomes for our customers and these forward-looking statements are a reflection of that commitment. However, as circumstances can change rapidly, we keep ourselves open to adjusting and revising these statements as needed and in line with applicable law. We look forward to providing you with up-to-date information on our progress and development.