Positive new preclinical data for Eloxx ERSG molecules presented at the
- dose dependent restoration of missing protein of Usher Syndrome nonsense mutations,
- encouraging pharmacokinetics in the retina by intravitreal injection
- favorable tolerability profile
On track to report top line data from Phase 2 clinical trials for ELX-02 in cystic fibrosis and cystinosis in the U.S. and
Company to host webcast and conference call on
“We are very pleased to be completing the final cohort of our multiple ascending dose study in the U.S. and rapidly advancing ELX-02 into Phase 2 clinical trials in cystic fibrosis and cystinosis. We look forward to reporting top line data in the U.S. and
Cystic Fibrosis Program Updates
- We initiated the seventh and final cohort of our multiple-ascending dose (MAD) study for ELX-02 and expect to complete the study in the U.S. in the next two weeks.
- We are on track to initiate a Phase 2 clinical trial this year in the U.S. and
Europe in cystic fibrosis patients with the G542X CFTR mutation. We expect to report top line data from this trial in 2019. Our Clinical Trial Application has been approved and granted orphan drug designation by theEuropean Medicines Agency .
- Three abstracts have been accepted for presentation at the 42nd
European Cystic Fibrosis Society (ECFS) Conference June 5-8, 2019 titled:
- “ELX-02 increases full length CFTR mRNA through nonsense mediated decay interruption”
June 6 th 15:00-16:30, ORAL, Hall 2E, Workshop: Abnormalities in cystic fibrosis cells and strategies to fix them - “ELX-02 Pharmacokinetic profile appropriate for CF patient use”
June 7 th 14:00-15:00, POSTER, Clinical Trials and New Therapies - “Administration of ELX-02 to Healthy Volunteers Demonstrates Dose-linearity and Proportionality as Well as Low Inter-subject Variability”
June 6 th 17:00-18:30, ORAL, Hall 1A, Workshop: Hot off the press; new data from drug trials
- “ELX-02 increases full length CFTR mRNA through nonsense mediated decay interruption”
- On
March 27, 2019 , Eloxx presented new positive data for ELX-02 at theEuropean Cystic Fibrosis Basic Science Conference inCroatia . In a poster titled: “CFTR protein detection in organoids from healthy and CF patients with nonsense mutations support using organoid model to test ELX-02 mediated CFTR read-through restoration”, Dr.Shira Landskroner-Eiger, Sr. Principal Scientist, Translational Sciences at Eloxx, reported that:
- Consistent with increased CFTR activity observed in the organoid swelling assay, ELX-02 mediates a significant restoration of CFTR protein expression as measured via a capillary-based immunoassay approach in multiple G542X or W1282X nonsense carrying organoids.
- G542X organoids treated with ELX-02 demonstrate proper cell surface CFTR localization on the apical surface, which is consistent with increased CFTR, mRNA, and CFTR function in the swelling assay.
- Consistent with increased CFTR activity observed in the organoid swelling assay, ELX-02 mediates a significant restoration of CFTR protein expression as measured via a capillary-based immunoassay approach in multiple G542X or W1282X nonsense carrying organoids.
While ELX-02 mediated protein increases have been previously demonstrated, this is the first demonstration reported in cystic fibrosis patient organoids. Within this translational CF organoid model, ELX-02 dose-dependent increases in CFTR mRNA stability and function can now be extended to the demonstration of accompanying increases of CFTR protein.
Previously, Eloxx presented positive data for ELX-02 at the
- Eloxx has continued to generate new data for ELX-02 activity from a growing number of patient-derived organoids which represent multiple nonsense mutations across a variety of genotypes representing the top 5 nonsense mutations in the cystic fibrosis population, which cover over 75% of the nonsense bearing cystic fibrosis patients.
- On
February 26, 2019 , Eloxx announced it joined the consortium agreement of the European HIT-CF project, aEuropean Union funded preclinical and clinical research program evaluating the efficacy and safety of several disease modifying drug candidates in Cystic Fibrosis (CF) patients with rare genetic mutations. Eloxx’s lead investigational drug candidate, ELX-02, a small molecule eukaryotic ribosomal selective glycoside (ERSG), will be evaluated in cystic fibrosis patients with nonsense mutations for whom there are few available treatment options.
- The goal of the European HIT-CF project is to investigate whether a positive response to therapies in a patient derived organoid can be predictive of clinical response in a controlled trial. The project represents a new era in CF treatment and personalized medicine, as it has the potential to shift therapeutic trials from patients to the laboratory. The organoid model could be extended to all patients with CF and other rare genetic diseases to identify appropriate therapeutic options. The EU has granted
EUR 6.7 million from the Horizon 2020 research program to HIT-CF.
- The goal of the European HIT-CF project is to investigate whether a positive response to therapies in a patient derived organoid can be predictive of clinical response in a controlled trial. The project represents a new era in CF treatment and personalized medicine, as it has the potential to shift therapeutic trials from patients to the laboratory. The organoid model could be extended to all patients with CF and other rare genetic diseases to identify appropriate therapeutic options. The EU has granted
- To support the Phase 2 clinical trial program for ELX-02, Eloxx has completed the manufacturing of a lyophilized clinical drug product. Eloxx has also identified a commercial manufacturer and is engaged in the process development work to scale up activities required to support Phase 3 clinical development.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use.
Inherited Retinal Disease Program Updates
- Eloxx has been advancing several new investigational product candidates from its library into IND enabling studies in ophthalmology. Currently available data for several of the molecules that have demonstrated positive activity on nonsense mutations across different inherited retinal disorders as well as a favorable tolerability profile.
- In a Poster presentation at the ARVO Annual Meeting on
May 2, 2019 titled: “Instituting a read-through therapeutic approach to nonsense-mutation based inherited retinal disorders, ELX-03, a translational nonsense mutation read-through agent demonstrates tolerability and activity for use in inherited retinal disorders”, presented by Dr.Matthew Goddeeris , Director of Research at Eloxx, it was reported that:
- Eloxx has screened multiple compounds from its ERSG library of read-through agents for potential use in the treatment of inherited retinal disorders with an initial focus on Usher Syndrome, beginning with USH2A.
- Multiple Eloxx compounds in preclinical studies have demonstrated:
- Dose-responsive activity against Usher mutations.
- Restoration of missing Usher Syndrome protein.
- Favorable tolerability profile at high doses in sensitive species; preserved electroretinogram (ERG) and no compound-related histopathological changes.
- Encouraging pharmacokinetics demonstrating retina exposure by intravitreal injection.
- IND-enabling studies are ongoing.
- Next steps include evaluation of patient-derived cell models and sustained release formulations.
- Eloxx has screened multiple compounds from its ERSG library of read-through agents for potential use in the treatment of inherited retinal disorders with an initial focus on Usher Syndrome, beginning with USH2A.
At the request of the Foundation Fighting Blindness, Dr.
First Quarter 2019 Financial Results
As of
We incurred a loss for the three months ended
Our research and development expenses were
Our general and administrative expenses were approximately
Conference Call Information:
Date:
Time:
International Dial-in Number: 210-874-7715
Conference ID: 5198713
Live Webcast: accessible from the Company's website at www.eloxxpharma.com under Events and Presentations or with this link: https://edge.media-server.com/m6/p/ve82h2hr
About
Forward-Looking Statements
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors,including: the development of the Company’s read-through technology; the approval of the Company’s patent applications; the Company’s ability to successfully defend its intellectual property or obtain necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the Company’s ability to obtain applicable regulatory approvals for its current and future product candidates; the acceptance by the market of the Company’s products should they receive regulatory approval; the timing and success of the Company’s preliminary studies, preclinical research, clinical trials, and related regulatory filings; the ability of the Company to consummate additional financings as needed; as well as those discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.
Contact:
203-274-2825
barbarar@eloxxpharma.com
ELOXX PHARMACEUTICALS, INC. | |||||||
UNAUDITED CONSOLIDATED BALANCE SHEETS | |||||||
(Amounts in thousands, except share and per share data) | |||||||
March 31, 2019 |
December 31, 2018 |
||||||
ASSETS | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 44,576 | $ | 48,606 | |||
Marketable securities | 8,928 | — | |||||
Restricted bank deposit | 45 | 45 | |||||
Prepaid expenses and other current assets | 1,911 | 1,690 | |||||
Total current assets | 55,460 | 50,341 | |||||
Property and equipment, net | 231 | 248 | |||||
Operating lease right-of-use asset | 1,106 | — | |||||
Other long-term assets | 94 | 129 | |||||
Total assets | $ | 56,891 | $ | 50,718 | |||
LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 2,240 | $ | 747 | |||
Accrued expenses | 4,597 | 6,938 | |||||
Current portion of long-term debt | 984 | — | |||||
Current portion of operating lease liability | 481 | — | |||||
Taxes payable | 122 | 122 | |||||
Total current liabilities | 8,424 | 7,807 | |||||
Long-term debt | 13,409 | — | |||||
Operating lease liability | 625 | — | |||||
Stockholders’ equity: | |||||||
Common stock, $0.01 par value per share, 500,000,000 shares authorized, 36,047,498 and 35,951,537 shares issued and 35,945,608 and 35,860,114 shares outstanding as of March 31, 2019 and December 31, 2018, respectively |
361 | 360 | |||||
Common stock in treasury, at cost, 101,890 and 91,423 shares at March 31, 2019 and December 31, 2018, respectively |
(1,250 | ) | (1,129 | ) | |||
Additional paid in capital | 133,383 | 129,825 | |||||
Accumulated other comprehensive income | 1 | — | |||||
Accumulated deficit | (98,062 | ) | (86,145 | ) | |||
Total stockholders’ equity | 34,433 | 42,911 | |||||
Total liabilities and stockholders' equity | $ | 56,891 | $ | 50,718 | |||
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES | |||||||
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||
(Amounts in thousands, except share and per share data) | |||||||
Three Months Ended March 31, |
|||||||
2019 | 2018 | ||||||
Operating expenses: | |||||||
Research and development | $ | 6,019 | $ | 4,394 | |||
General and administrative | 5,958 | 3,393 | |||||
Reverse merger related expenses | — | 761 | |||||
Total operating expenses | 11,977 | 8,548 | |||||
Loss from operations | (11,977 | ) | (8,548 | ) | |||
Other (income) expense, net | (60 | ) | 43 | ||||
Net loss | $ | (11,917 | ) | $ | (8,591 | ) | |
Basic and diluted net loss per share | $ | (0.33 | ) | $ | (0.31 | ) | |
Weighted average number of Common Shares used in computing basic and diluted net loss per share | 35,910,270 | 27,527,738 | |||||
SOURCE:
Source: Eloxx Pharmaceuticals