Eloxx announces the hiring of Dr.
Abstract accepted for presentation at
ELX-03 abstract accepted for presentation at the
On track to report top line data from a Phase 2 clinical trial for ELX-02 in cystic fibrosis in the U.S. and
Company to host webcast and conference call on
“We are very pleased to be advancing ELX-02 in cystic fibrosis and look forward to reporting top line data from our Phase 2 clinical trial in the U.S. and
Cystic Fibrosis Program Updates
- An abstract reporting on new data demonstrating the ability of ELX-02 to restore production of the CFTR protein has been accepted for presentation at the ECFS Basic Science Meeting on
May 25 th-27th 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”.
- Previously, Eloxx presented positive data for ELX-02 at the
North American Cystic Fibrosis Conference onOctober 18 th, 2018 inDenver, Colorado in a poster presentation titled: “Measuring mRNA levels in cystic fibrosis organoids with nonsense mutations following treatment with ELX-02,” which demonstrated ELX-02 mediated dose responsive increases in CFTR function, as measured by FIS swelling activity, which was found to correlate with increased in CFTR mRNA, as measured by nanoString™ technology, with elevations above wild-type. ELX-02 appears to increase the steady state concentrations of CFTR mRNA suggesting that ELX-02 may be modulating nonsense mediated decay. These data demonstrate that ELX-02 promotes translation of functional CFTR and we believe, de-risks our planned Phase 2 clinical trial of ELX-02 in cystic fibrosis patients with the G542X CFTR mutation on one or both alleles, which is the second most common mutation globally and accounts for about 5% of the cystic fibrosis population.
- 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
- The results from our Phase 1a single-ascending dose (SAD) study for ELX-02 have been published in the
January 2019 edition of the Journal of Clinical Pharmacology and Drug Development titled: “Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations. In Healthy Volunteers” - We have initiated the sixth cohort of our multiple-ascending dose (MAD) study for ELX-02 and expect to complete the final cohort in the U.S. in the first half of 2019.
- 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 date from this trial in 2019. Our Clinical Trial Application has been approved and granted orphan drug designation by theEuropean Medicines Agency .
- 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.
- Eloxx announced that is has hired Dr.
Kristie Kapinas to its Patient Advocacy team. Dr. Kapinas has deep cystic fibrosis experience and previously worked as a Medical Science Liaison and Disease Educator.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use.
Inherited Retinal Disease Program Updates
- Eloxx today announced that it has hired a highly experienced ophthalmology executive, Dr.
Susan Schneider , as Senior Vice President Ophthalmology to lead the clinical development team and will be responsible for strategic clinical oversight and the advancement of our inherited retinal disease programs across our library of eukaryotic ribosomal selective glycosides (ERSG). Dr. Schneider has extensive experience leading the clinical development and strategic planning efforts across a range of ocular indications at leading companies including most recently as Chief Medical Officer at ThromboGenics, Vice President and Therapeutic Head, Glaucoma & Retina, Glaucoma and wet AMD atAllergan , as well as leadership roles in Ophthalmology at GlaxoSmithKline, Bausch & Lomb, and Genentech.
- 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 have demonstrated positive activity on nonsense mutations across different inherited retinal disorders as well as a favorable tolerability profile. The preservation of the electroretinogram wave function and retinal histology are important safety considerations, and the preclinical data to date show that our investigational agent has an acceptable safety profile. We believe that these data are supportive of the use of these compounds for intravitreal injection with initial development focused on Usher’s Syndrome. There are an estimated 4,000 individuals in the U.S. alone with nonsense mutations underlying Usher’s Syndrome.
- An abstract has been accepted for presentation at the ARVO Meeting
April 28-May 2, 2019 , inVancouver titled: “ELX-03, a translational nonsense mutation read-through agent demonstrates tolerability and activity for use in inherited retinal disorders”.
Fourth Quarter 2018 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
Full Year 2018 Financial Results
We incurred a loss for the twelve 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: 7877535
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/hcyzfmto
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
ELOXX PHARMACEUTICALS, INC. | |||||||||
UNAUDITED CONSOLIDATED BALANCE SHEETS | |||||||||
(Amounts in thousands, except share and per share data) | |||||||||
December 31, | |||||||||
2018 | 2017 | ||||||||
ASSETS | |||||||||
Current assets: | |||||||||
Cash and cash equivalents | $ | 48,606 | $ | 24,049 | |||||
Restricted bank deposit | 45 | 102 | |||||||
Prepaid expenses and other current assets | 1,690 | 355 | |||||||
Total current assets | 50,341 | 24,506 | |||||||
Property and equipment, net | 248 | 278 | |||||||
Other long-term assets | 129 | — | |||||||
Total | $ | 50,718 | $ | 24,784 | |||||
LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||||
Current liabilities: | |||||||||
Accounts payable | $ | 747 | $ | 1,530 | |||||
Accrued expenses | 6,938 | 1,893 | |||||||
Taxes payable | 122 | — | |||||||
Total current liabilities | 7,807 | 3,423 | |||||||
Stockholders’ equity: | |||||||||
Common stock, $0.01 par value 500,000,000 and 500,000,000 shares authorized as of December 31, 2018 and 2017, respectively; 35,860,114 and 27,527,738 shares issued and outstanding as of December 31, 2018 and 2017, respectively |
360 | 274 | |||||||
Common stock in treasury stock, at cost, 91,423 and 0 shares at December 31, 2018 and 2017, respectively |
(1,129 | ) | — | ||||||
Additional paid in capital | 129,825 | 60,047 | |||||||
Accumulated deficit | (86,145 | ) | (38,960 | ) | |||||
Total stockholders’ equity | 42,911 | 21,361 | |||||||
Total | $ | 50,718 | $ | 24,784 | |||||
ELOXX PHARMACEUTICALS, INC. | |||||||||||||
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||||||||
(Amounts in thousands, except share and per share data) | |||||||||||||
Year ended December 31, | |||||||||||||
2018 | 2017 | 2016 | |||||||||||
Operating expenses: | |||||||||||||
Research and development | $ | 20,489 | $ | 16,398 | $ | 8,986 | |||||||
General and administrative | 26,482 | 3,992 | 854 | ||||||||||
Reverse merger related expenses | 594 | — | — | ||||||||||
Total operating expenses | 47,565 | 20,390 | 9,840 | ||||||||||
Loss from operations | (47,565 | ) | (20,390 | ) | (9,840 | ) | |||||||
Other (income) expense, net | (502 | ) | 824 | 7 | |||||||||
Loss before income taxes | (47,063 | ) | (21,214 | ) | (9,847 | ) | |||||||
Provision for income taxes | 122 | — | — | ||||||||||
Net loss | $ | (47,185 | ) | $ | (21,214 | ) | $ | (9,847 | ) | ||||
Less: Dividends accumulated for the period | — | 2,404 | 1,100 | ||||||||||
Net loss available to common stockholders | $ | (47,185 | ) | $ | (23,618 | ) | $ | (10,947 | ) | ||||
Basic and diluted net loss per share | $ | (1.45 | ) | $ | (4.75 | ) | $ | (2.60 | ) | ||||
Weighted average number of Common Stock used in computing basic and diluted net loss per share |
32,436,506 | 4,976,377 | 4,205,277 |
Source: Eloxx Pharmaceuticals