A Phase 2 Open Label Pilot Study to Evaluate the Safety and Efficacy of Subcutaneously Administered ELX-02 in Patients With Alport Syndrome With Col4A5 and Col4A3/4 Nonsense Mutation

This is a Phase 2 open label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with X-linked or autosomal recessive Alport Syndrome with Col4A5 and Col4A3/4 nonsense mutation.
In total, up to 8 participants, with a minimum of 3 adults, will be enrolled in the trial.
The study will be comprised of the following periods for each participant:
a Screening period of up to 6 weeks (42 days)
a total Treatment Period of 8 weeks (60 days)
a safety/efficacy Follow-up Period of 12 weeks (90 days) after the last treatment The Treatment Period will be a treatment of ELX-02 0.75 mg/kg SC QD for 8 weeks.

Start Date28 Nov 2022

Sponsor / Collaborator

Eloxx Pharmaceuticals, Inc.

ISRCTN12014920 / CompletedPhase 2

A Phase II open-label pilot study to evaluate the safety and efficacy of subcutaneously administered ELX-02 in patients with Alport syndrome with Col4A5 and Col4A3/4 nonsense mutation

Start Date31 Oct 2022

Sponsor / Collaborator

TMC Pharma Services Ltd.

NCT04135495 / CompletedPhase 2

A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X Allele

This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele.
In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation.
Each patient will receive up to 5 escalating doses as follows:
ELX-02 0.3 mg/kg per day SC
ELX-02 0.75 mg/kg per day SC
ELX-02 1.5 mg/kg per day SC
An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests.
ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid

Start Date25 Nov 2019

Sponsor / Collaborator

Eloxx Pharmaceuticals, Inc.

100 Clinical Results associated with Exaluren

100 Translational Medicine associated with Exaluren

100 Patents (Medical) associated with Exaluren

Literatures (Medical) associated with Exaluren

01 Dec 2023·Biological psychiatry

Prenatal and Postnatal Maternal Depressive Symptoms Are Associated With White Matter Integrity in 5-Year-Olds in a Sex-Specific Manner

Article

Author: Pulli, Elmo P ; Tuulari, Jetro J ; Karlsson, Hasse ; Kataja, Eeva-Leena ; Kumpulainen, Venla ; Merisaari, Harri ; Lewis, John D ; Silver, Eero ; Copeland, Anni ; Saukko, Ekaterina ; Karlsson, Linnea

BACKGROUND:

Prenatal and postnatal maternal psychological distress predicts various detrimental consequences on social, behavioral, and cognitive development of offspring, especially in girls. Maturation of white matter (WM) continues from prenatal development into adulthood and is thus susceptible to exposures both before and after birth.

METHODS:

WM microstructural features of 130 children (mean age, 5.36 years; range, 5.04-5.79 years; 63 girls) and their association with maternal prenatal and postnatal depressive and anxiety symptoms were investigated with diffusion tensor imaging, tract-based spatial statistics, and regression analyses. Maternal questionnaires were collected during first, second, and third trimesters and at 3, 6, and 12 months postpartum with the Edinburgh Postnatal Depression Scale (EPDS) for depressive symptoms and Symptom Checklist-90 for general anxiety. Covariates included child's sex; child's age; maternal prepregnancy body mass index; maternal age; socioeconomic status; and exposures to smoking, selective serotonin reuptake inhibitors, and synthetic glucocorticoids during pregnancy.

RESULTS:

Prenatal second-trimester EPDS scores were positively associated with fractional anisotropy in boys (p < .05, 5000 permutations) after controlling for EPDS scores 3 months postpartum. In contrast, postpartum EPDS scores at 3 months correlated negatively with fractional anisotropy (p < .01, 5000 permutations) in widespread areas only in girls after controlling for prenatal second-trimester EPDS scores. Perinatal anxiety was not associated with WM structure.

CONCLUSIONS:

These results suggest that prenatal and postnatal maternal psychological distress is associated with brain WM tract developmental alterations in a sex- and timing-dependent manner. Future studies including behavioral data are required to consolidate associative outcomes for these alterations.

01 Feb 2024·Journal of thrombosis and haemostasis : JTH

In vitro and ex vivo rescue of a nonsense mutation responsible for severe coagulation factor V deficiency

Article

Author: Toffanin, Serena ; Radu, Claudia M ; Hackeng, Tilman M ; Campello, Elena ; Cuneo, Antonio ; Serino, M Luisa ; Simioni, Paolo ; Castoldi, Elisabetta ; Lunghi, Barbara ; Gemmati, Donato ; Bulato, Cristiana ; Ciccone, Maria ; Todaro, Alice M ; Bernardi, Francesco

BACKGROUND:

Coagulation factor V (FV) deficiency is a rare bleeding disorder that is usually managed with fresh-frozen plasma. Patients with nonsense mutations may respond to treatment with readthrough agents.

OBJECTIVES:

To investigate whether the F5 p.Arg1161Ter mutation, causing severe FV deficiency in several patients, would be amenable to readthrough therapy.

METHODS:

F5 mRNA and protein expression were evaluated in a F5 p.Arg1161Ter-homozygous patient. Five readthrough agents with different mechanisms of action, i.e. G418, ELX-02, PTC-124, 2,6-diaminopurine (2,6-DAP), and Amlexanox, were tested in in vitro and ex vivo models of the mutation.

RESULTS:

The F5 p.Arg1161Ter-homozygous patient showed residual F5 mRNA and functional platelet FV, indicating detectable levels of natural readthrough. COS-1 cells transfected with the FV-Arg1161Ter cDNA expressed 0.7% FV activity compared to wild-type. Treatment with 0-500 μM G418, ELX-02, and 2,6-DAP dose-dependently increased FV activity up to 7.0-fold, 3.1-fold, and 10.8-fold, respectively, whereas PTC-124 and Amlexanox (alone or in combination) were ineffective. These findings were confirmed by thrombin generation assays in FV-depleted plasma reconstituted with conditioned media of treated cells. All compounds except ELX-02 showed some degree of cytotoxicity. Ex vivo differentiated megakaryocytes of the F5 p.Arg1161Ter-homozygous patient, which were negative at FV immunostaining, turned positive after treatment with all 5 readthrough agents. Notably, they were also able to internalize mutant FV rescued with G418 or 2,6-DAP, which would be required to maintain the crucial platelet FV pool in vivo.

CONCLUSION:

These findings provide in vitro and ex vivo proof-of-principle for readthrough-mediated rescue of the F5 p.Arg1161Ter mutation.

01 Jun 2023·American journal of physiology. Lung cellular and molecular physiology

The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators.

Article

Author: Keeling, Kim M ; Bedwell, David M ; Rowe, Steven M ; Chen, Jianguo ; Aghamohammadzadeh, Soheil ; Tang, Liping ; Wang, Wei ; Thrasher, Kari ; Fu, Lianwu ; Falk Libby, Emily ; Wen, Hui

Ten percent of cystic fibrosis (CF) patients carry a premature termination codon (PTC); no mutation-specific therapies exist for these individuals. ELX-02, a synthetic aminoglycoside, suppresses translation termination at PTCs (i.e., readthrough) by promoting the insertion of an amino acid at the PTC and restoring expression of full-length CFTR protein. The identity of amino acids inserted at PTCs affects the processing and function of the resulting full-length CFTR protein. We examined readthrough of the rare G550X-CFTR nonsense mutation due to its unique properties. We found that forskolin-induced swelling in G550X patient-derived intestinal organoids (PDOs) was significantly higher than in G542X PDOs (both UGA PTCs) with ELX-02 treatment, indicating greater CFTR function from the G550X allele. Using mass spectrometry, we identified tryptophan as the sole amino acid inserted in the G550X position during ELX-02- or G418-mediated readthrough, which differs from the three amino acids (cysteine, arginine, and tryptophan) inserted in the G542X position after treatment with G418. Compared with wild-type CFTR, Fischer rat thyroid (FRT) cells expressing the G550W-CFTR variant protein exhibited significantly increased forskolin-activated Cl^- conductance, and G550W-CFTR channels showed increased PKA sensitivity and open probability. After treatment with ELX-02 and CFTR correctors, CFTR function rescued from the G550X allele in FRTs reached 20-40% of the wild-type level. These results suggest that readthrough of G550X produces greater CFTR function because of gain-of-function properties of the CFTR readthrough product that stem from its location in the signature LSGGQ motif found in ATP-binding cassette (ABC) transporters. G550X may be a particularly sensitive target for translational readthrough therapy.NEW & NOTEWORTHY We found that forskolin-induced swelling in G550X-CFTR patient-derived intestinal organoids (PDOs) was significantly higher than in G542X-CFTR PDOs after treatment with ELX-02. Tryptophan (W) was the sole amino acid inserted in the G550X position after readthrough. Resulting G550W-CFTR protein exhibited supernormal CFTR activity, PKA sensitivity, and open probability. These results show that aminoglycoside-induced readthrough of G550X produces greater CFTR function because of the gain-of-function properties of the CFTR readthrough product.

News (Medical) associated with Exaluren

16 Apr 2024

·www.biospace.com

Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates

ELX-02 Granted Orphan Drug Designation (ODD) from U.S. Food and Drug Administration (FDA) for Treatment of Alport Syndrome Pre-Investigational New Drug (IND) Meeting Requested with U.S. FDA for ELX-02 to Discuss Planned next Study In Nonsense Mutation Alport Syndrome Signed Global Licensing Partnership for ZKN-013 with Almirall Pharmaceuticals with $3M upfront and up to $470M in milestones and Tiered Royalties on Global Sales New Paper Published on Autosomal Dominant Polycystic Kidney Disease (ADPKD) shows that ELX-02 treatment prevents cyst formation in diseased PKD organoids with nonsense mutations and a single healthy gene copy WATERTOWN, Mass., April 16, 2024 (GLOBE NEWSWIRE) --Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today provided program updates for ELX-02 and ZKN-013, including Orphan Drug Designation (ODD) for ELX-02. Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS). Additionally, with the global licensing partnership with Almirall announced in March, the company has begun the development of ZKN-013 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDDEB) and Familial Adenomatous Polyposis (FAP) patients with nonsense mutations. The publication of a recent paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD), confirms the potential of ELX-02 for the treatment of all rare genetic kidney diseases with nonsense mutations in the disease causing genes. “The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “The designation further strengthens our belief that ELX-02 has the potential to be transformational in supporting the ultra-rare subset of Alport Syndrome patient population with nonsense mutations. We look forward to engaging the FDA on initiating the larger clinical trial to support the potential for regulatory marketing approval of ELX-02 in this patient population.” ELX-02: Regulatory and Clinical Updates for Alport Syndrome Nonsense Mutation Program ELX-02 Granted ODD from U.S. FDA: In April, the FDA Office of Orphan Products Development (OOPD) granted ODD for ELX-02 for the treatment of Alport Syndrome. The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023. FDA ODD is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States with a reasonable clinical or preclinical hypothesis for efficacy in the target population. Requested a Pre-Investigational New Drug (IND) meeting with U.S. FDA: The request for a pre-IND meeting formally initiates communications with the FDA regarding development of ELX-02 for the treatment of NMAS. The purpose of the pre-IND meeting will be to request feedback on the Eloxx’s planned clinical trial in nonsense mutation Alport syndrome patients. Presented Clinical Data Updates at Alport Workshop Meeting in March: Prof. Daniel Gale presented updated results from the Proof-of Concept Study in 3 NMAS patients treated with ELX-02 Meaningful reduction in Podocyte Foot Process Effacement after treatment measured as an average 60% increase in Filtration Slit Density in post treatment kidney biopsies in all 3 patients. Results confirm the expression of functional collagen 4 protein in-line with the mechanism of protein induction of ELX-02. Changes in UPCR noted to be consistent with biopsy results with reduction or stabilization of proteinuria during or up to 2 months post completion of dosing. ZKN-013: Update on Global Licensing Partnership with Almirall In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations. Received $3 million in upfront payment Additional payments of ELX-02 of up to $470 million and tiered royalties on global sales: Eloxx shall be eligible for additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million as well as tiered royalties on any potential global sales Initiated Phase 1 Single Ascending Dose (SAD) study: Initiated Phase 1 SAD study in Australia with initial results expected in 2nd half of 2024 Recently Published Paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD) may Have Other Rare Kidney Disease Applications A paper was published in April 2024, on ADPKD titled Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease, authored by Courtney E. Vishy, Chardai Thomas, Thomas Vincent, Daniel K. Crawford, Matthew M. Goddeeris, and Benjamin S. Freedman. Freedman and colleagues engineered base-edited human iPS cells to resolve the functional genetics of human PKD. They discovered that cyst formation in diseased organoids could be prevented with a single healthy gene copy. This insight enables the demonstration of both genetic and pharmacological therapeutic approaches for clinically relevant nonsense mutations and may have the potential to support other rare kidney diseases. About Nonsense Mutation Alport Syndrome Nonsense Mutation Alport syndrome (NMAS) is an ultra-rare Type IV Collagenopathy characterized by nonsense mutations in the genes (COL4A3, COL4A4, and COL4A5) that result in a less than full length (truncated) Type 4 Collagen. This disorder mostly affects children with a median age at diagnosis of 9 to 20 years. It is characterized by rapid and progressive damage to the kidneys, ear, and eyes, starting with worsening of kidney morphology to proteinuria and finally kidney failure, hearing loss and eye abnormalities. 90% NMAS patients progress to kidney failure and hearing loss before the age of 30. There are no approved therapies. It is estimated that there are approximately 11,000 NMAS patients in the US and >20,000 patients in US, Europe, Japan and China. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit . Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at and the “Financials & Filings” page of our website at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact Investors John Woolford john.woolford@westwicke.com 443.213.0506 Media Laureen Cassidy laureen@outcomescg.com Source: Eloxx Pharmaceuticals

Phase 2Clinical ResultOrphan DrugLicense out/inPhase 1

10 Apr 2024

·www.sciencedaily.com

New insight into combating drug-resistant prostate cancer

New research sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the activity of coregulator proteins. New research from the University of Eastern Finland sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the activity of coregulator proteins. Glucocorticoids regulate vital biological processes by affecting gene encoding through a DNA-binding transcription factor, namely the glucocorticoid receptor. The activity of the glucocorticoid receptor is made extensive use of in medicine because glucocorticoids have a strong anti-inflammatory effect. For this reason, synthetic glucocorticoids are one of the most prescribed drugs in the world. They are used to treat inflammatory diseases, such as rheumatoid arthritis, and as adjuvant therapy for cancer patients to alleviate the side effects of cancer therapy. In blood cancer, glucocorticoids are important drugs that limit the growth of cancer cells. However, recent studies have shown that the glucocorticoid receptor also has an oncogenic, or cancer-promoting, effect in cancers like breast and prostate cancer. In prostate cancer, the glucocorticoid receptor can replace the activity of the androgen receptor, which is main oncogenic factor in this cancer, when its activity is inhibited by drug therapy. Thus, glucocorticoids help prostate cancer develop resistance to drug therapy. "Due to these drug resistance and cancer-promoting effects, it is important to study how the glucocorticoid receptor functions on the cellular and molecular level in cancer," Academy Research Fellow, Docent Ville Paakinaho of the University of Eastern Finland notes. The Paakinaho Lab has published two recent genome-wide deep sequencing studies on the subject. The first, published in Nucleic Acids Research, explored how the glucocorticoid receptor replaces the androgen receptor on the molecular level. "This study showed that the glucocorticoid receptor can only use regulatory regions that are already active in prostate cancer cells," says Doctoral Researcher Laura Helminen of the University of Eastern Finland. In other words, glucocorticoid receptor-mediated drug resistance emerges through these regulatory regions, and by affecting the activity of these areas, the harmful effects of glucocorticoids in prostate cancer could be prevented. Bioinformatics analyses indicated the pioneer transcription factor FOXA1 as one possible target. FOXA1 is known to have cancer-promoting properties, which is why the researchers assumed that inhibiting its activity would limit the development of glucocorticoid receptor-mediated drug-resistant prostate cancer. Surprisingly however, the effect was exactly the opposite: inhibiting the activity of FOXA1 further increased the activity of the glucocorticoid receptor -- and the development of drug resistance. This is because FOXA1 was found to be involved in the silencing of the glucocorticoid receptor gene, and this is what increased its activity when FOXA1 was inhibited. "Research often reveals the unexpected, and that's part of its charm," Paakinaho says. The activity of the glucocorticoid receptor in regulatory regions can, however, be influenced in drug-resistant prostate cancer through an alternative pathway. Coregulator proteins were identified as an alternative target through which the glucocorticoid receptor affects the regulation of gene expression. These proteins include EP300 and CREBBP. Several pharmaceutical companies are developing small-molecule inhibitors targeting these proteins, and some are already being studied in patients. In another study by the Paakinaho Lab, the researchers explored ways to inhibit glucocorticoid receptor-mediated effects by inhibiting coregulator proteins. These findings were reported in Cellular and Molecular Life Sciences. "Silencing the EP300 and CREBBP proteins with a small-molecule inhibitor clearly prevented the activity of the glucocorticoid receptor in prostate cancer cells," Project Researcher Jasmin Huttunen of the University of Eastern Finland says. This allowed the growth of drug-resistant prostate cancer cells to be inhibited. Furthermore, the researchers found that silencing EP300 and CREBBP also effectively inhibited the activity of the androgen receptor especially in prostate cancer cells that have an amplification of the androgen receptor gene. This amplification is found in up to half of patients with advanced prostate cancer. Surprisingly, the EP300 and CREBBP inhibitor also inhibited the activity of FOXA1, while still preserving its ability to silence the expression of the glucocorticoid receptor gene. By using the EP300 and CREBBP inhibitor, it was possible to block the activity of FOXA1 without the development of glucocorticoid receptor-mediated drug resistance. Ultimately, inhibiting the activity of both the androgen and the glucocorticoid receptor was found to be primarily due to the limitation of FOXA1 activity. The study suggests that treatment targeting coregulator proteins could also be effective in untreated prostate cancer. The studies were funded by the Research Council of Finland, the Sigrid Jusélius Foundation, and the Cancer Foundation Finland.

13 Mar 2024

·www.businesswire.com

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to License ZKN-013 for Rare Dermatological Diseases

BARCELONA, Spain & WATERTOWN, Mass.--( BUSINESS WIRE )-- Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013 . Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases. The drug candidate is expected to shortly enter into Phase I development in healthy volunteers. As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales. " We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases ,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx . “ In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform .” “ This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases ” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall . “ We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations .” RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells. ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene. ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx's TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com . About Almirall Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients' world by helping them realize their hopes and dreams for a healthy life . We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs. Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others. For more information, please visit almirall.com Eloxx Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings . All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Almirall Legal warning This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are "forward-looking statements". These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

License out/inPhase 1Phase 2

100 Deals associated with Exaluren

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Nephritis, Hereditary	Phase 2	AU	Eloxx Pharmaceuticals, Inc.	28 Nov 2022
Nephritis, Hereditary	Phase 2	GB	Eloxx Pharmaceuticals, Inc.	28 Nov 2022
Cystic Fibrosis	Phase 2	AU	Eloxx Pharmaceuticals, Inc.	05 Nov 2019
Cystic Fibrosis	Phase 2	DE	Eloxx Pharmaceuticals, Inc.	05 Nov 2019
Cystic Fibrosis	Phase 2	IL	Eloxx Pharmaceuticals, Inc.	05 Nov 2019
Cystinosis	Phase 2	CA	Eloxx Pharmaceuticals, Inc.	02 Aug 2019
Genetic Diseases, Inborn	Phase 1	-	Eloxx Pharmaceuticals, Inc.	01 Jul 2016
Mucopolysaccharidosis I	Phase 1	IL	Eloxx Pharmaceuticals, Inc.	-
Muscular Dystrophy, Duchenne	Phase 1	IL	Eloxx Pharmaceuticals, Inc.	-
Rett Syndrome	Phase 1	IL	Eloxx Pharmaceuticals, Inc.	-

Clinical Result

Indication

Phase

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


not available (ASN2022)	Not Applicable	Nephritis, Hereditary COL4A3 \| COL4A4 \| COL4A5	-	ELX-02	lcmijlafgt(puvqzgxzwi) = pniwjntssp nqrjysflsz (oiqvwjghhd ) View more	Positive	04 Nov 2022
NCT04126473 \| NCT04135495 (Corporate Publications) Manual	Phase 2	Cystic Fibrosis	31	ELX-02	bafsxzqlvo(gektdfsqoq) = No ELX-02 related serious adverse events fnznufzgcx (tqtynfbygi )	Negative	14 Sep 2022
NCT04126473 \| NCT04135495 (Corporate Publications) Manual	Phase 2	Cystic Fibrosis	31	Ivacaftor+ELX-02		Negative	14 Sep 2022
NCT04126473 \| NCT04135495 (Corporate Publications) Manual	Phase 2	Cystic Fibrosis	-	ELX-02	hdtjzteolc(aosiucewvq) = qmwioifteg rhykoctbeh (roqxfijczi ) View more	Positive	17 Nov 2021
NCT03309605 (Pubmed) Manual	Phase 1	-	62	ELX-02	vgvdxdiqjp(zkioqftmmk) = At the top dose of 5.0 mg/kg, 1 of 6 subjects experienced auditory threshold changes in which ototoxicity could not be clearly ruled out, and 2 of 6 had hearing threshold changes consistent with possible ototoxicity. Two of 3 subjects receiving placebo in the 5.0 mg/kg group also had significant hearing threshold changes. cwjmuhggat (loyxbgxxbt ) View more	Positive	01 Aug 2021

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