Loading, Please Wait...
-Presented Positive Phase 1 Data from SAD and MAD Studies at North American Cystic Fibrosis Conference
-On track to report top line Phase 2 data in nephropathic cystinosis this quarter, preliminary results show cystine reduction with 1mg/kg dose
-Actively enrolling Phase 2 cystic fibrosis trial in IL with additional sites in EU and US to open this quarter. Expect fully enrolled Phase 2 cystic fibrosis trials in the first quarter of 2020
-Company to present Phase 1 Renal Impairment Study results and additional preclinical data at Kidney Week
-Maintained Strong Balance Sheet with $64.9 Million in Cash and Equivalents as of September 30, 2019
-Company to host webcast and conference call today, Tuesday, November 5, 2019, at 4:30 pm ET
WALTHAM, Mass., Nov. 05, 2019 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc., (NASDAQ: ELOX) a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis, inherited retinal disorders, and other diseases caused by nonsense mutations limiting production of functional proteins, today reported its financial results for the three and nine months ended September 30, 2019 and provided a business update.
“We are very pleased to have presented the results of our MAD study at NACFC which reinforced the overall favorable tolerability profile and pharmacokinetics of ELX-02, with no serious adverse events or nephrotoxity. To date, ELX-02 safety and tolerability has been evaluated in 105 healthy volunteers,” said Dr. Gregory Williams, Chief Operating Officer of Eloxx Pharmaceuticals. “We are encouraged by the preliminary results of our phase 2 clinical trial in nephropathic cystinosis, as ELX-02 has demonstrated a statistically significant reduction in white blood cell cystine levels at the second dose level of 1.0 mg/kg. We are on track to complete this cohort and report top line data this quarter. We believe that achievement of proof of concept for ELX-02 in cystinosis will provide a basis for expansion to studies of additional kidney diseases, as well as increasing our probability of success in other clinical uses across this dose range.”
“This week the Eloxx team will be presenting the results from our completed Phase 1 Renal Impairment Study at Kidney Week. These data enable dose adjustment based on individual patient’s kidney function,” said Robert Ward, Chairman and CEO of Eloxx Pharmaceuticals. “We are pleased to be actively enrolling patients in our Cystic Fibrosis program, and we expect additional clinical trial sites to open in the US and EU this quarter and full enrollment to be achieved during the first quarter of 2020. With this change, we expect to report top line data from these trials in the first half of 2020.”
During the quarter ended September 30, 2019, Eloxx appointed Dr. Thomas Haverty as our Chief Medical Officer. Dr. Haverty has over 30 years of pharmaceutical and biotechnology experience leading clinical research and operations teams with responsibility for large development portfolios in virtually all classes of molecules and indications. Prior to joining us in his current capacity, Dr. Haverty, a Board-certified Nephrologist, served as a consultant to us following a long tenured career at Johnson & Johnson, Schering Plough and Merck Research Labs. Dr. Haverty has successfully led the development and approval of over 20 leading drugs.
Cystic Fibrosis Program Updates
Cystinosis Program Updates
Additional Development Programs
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use which is currently in Phase 2 clinical trials in cystic fibrosis and cystinosis.
Third Quarter 2019 Financial Results
As of September 30, 2019, we had cash, cash equivalents and marketable securities of $64.9 million, which we expect will be sufficient to fund our operations through top line data from our Phase 2 clinical trials in cystic fibrosis and cystinosis and our current and planned operations into the first quarter of 2021.
For the three months ended September 30, 2019, we incurred a loss of $12.9 million or $0.32 per share, which includes $3.0 million non-cash expense related to stock-based compensation. For the same period in the prior year, we incurred a net loss of $11.2 million, or $0.32 per share.
Our research and development expenses were $6.8 million for the three months ended September 30, 2019 which includes $0.7 million non-cash expense related to stock-based compensation. For the same period in the prior year, R&D expenses were $5.4 million. Quarter to quarter increases in R&D expenditures were largely driven by preparations for our multiple Phase 2 clinical trials, along with pre-clinical and CMC operations.
Our general and administrative expenses were $6.0 million for the three months ended September 30, 2019 which includes $2.3 million in non-cash expense related to stock-based compensation. For the same period in the prior year, G&A expenses were $5.9 million. Our G&A expenses were relatively flat year over year with higher professional service fees offset by a decrease in salary related costs.
First Nine Months 2019 Financial Results
For the nine months ended September 30, 2019, we incurred a loss of $39.2 million or $1.05 per share, which includes $8.6 million in non-cash expense related to stock-based compensation. For the same period in the prior year, we incurred a net loss of $33.2 million, or $1.05 per share.
Our research and development expenses were $20.2 million for the nine months ended September 30, 2019 which includes $2.0 million in non-cash expense related to stock-based compensation. For the same period in the prior year, R&D expenses were $14.0 million. The year over year increase in R&D expenditures was driven primarily by growth in our clinical and preclinical operations.
Our general and administrative expenses were $18.9 million for the nine months ended September 30, 2019 which includes $6.6 million in non-cash stock-based compensation. For the same period in the prior year, G&A expenses were $18.9 million. Our G&A expenses were relatively flat year over year despite increases in salary related costs reflective of our year over year headcount growth and increases in professional service fees, due to lower non-cash expense related to stock-based compensation in the 2019 period.
Conference Call and Webcast Information
Date: Tuesday, November 5, 2019
Time: 4:30 p.m. ET
Domestic Dial-in Number: (866) 754-6374
International Dial-in Number: (210) 874-7715
Conference ID: 8339658
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/mmc/p/ni34pajh
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. 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 the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx’s preclinical candidate pool consists of a library of novel drug candidates designed to be eukaryotic ribosomal selective glycosides identified based on read-through potential. Eloxx recently announced a new program focused on rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with operations in Rehovot, Israel. For more information, please visit www.eloxxpharma.com.
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.
SOURCE: Eloxx Pharmaceuticals, Inc.
|ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES|
|UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS|
|(Amounts in thousands, except share and per share data)|
|Cash and cash equivalents||$||22,155||$||48,606|
|Restricted bank deposit||40||45|
|Prepaid expenses and other current assets||1,878||1,690|
|Total current assets||66,854||50,341|
|Property and equipment, net||220||248|
|Operating lease right-of-use asset||870||—|
|Other long-term assets||113||129|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Current portion of long-term debt||3,488||—|
|Advances from collaboration partners||403||—|
|Current portion of operating lease liability||461||—|
|Total current liabilities||11,227||7,807|
|Operating lease liability||409||—|
|Preferred stock, $0.01 par value per share, 5,000,000 shares authorized, no
shares issued and outstanding at September 30, 2019 and December 31,
|Common stock, $0.01 par value per share, 500,000,000 shares authorized,
40,117,186 and 35,951,537 shares issued and 39,977,654 and 35,860,114
shares outstanding as of September 30, 2019 and December 31, 2018,
|Common stock in treasury, at cost, 139,532 and 91,423 shares
at September 30, 2019 and December 31, 2018, respectively
|Additional paid-in capital||171,783||129,825|
|Accumulated other comprehensive income||12||—|
|Total stockholders’ equity||45,228||42,911|
|Total liabilities and stockholders' equity||$||68,057||$||50,718|
|ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES|
|UNAUDITED CONSOLIDATED INCOME STATEMENTS|
|(Amounts in thousands, except share and per share data)|
Three Months Ended
Nine Months Ended
|Research and development||$||6,801||$||5,415||$||20,160||$||13,959|
|General and administrative||5,978||5,945||18,907||18,898|
|Reverse merger related expenses||—||—||—||594|
|Total operating expenses||12,779||11,360||39,067||33,451|
|Loss from operations||(12,779||)||(11,360||)||(39,067||)||(33,451||)|
|Other expense (income), net||96||(199||)||174||(293||)|
|Basic and diluted net loss per share||$||(0.32||)||$||(0.32||)||$||(1.05||)||$||(1.05||)|
|Weighted average number of Common Shares used in
computing basic and diluted net loss per share