Phase 2 cystic fibrosis clinical trial IND is open in the U.S., protocol endorsed by the
Initiated a Phase 2 clinical trial for ELX-02 in cystinosis in
On track to report top line data from Phase 2 clinical trials for ELX-02 in cystic fibrosis and cystinosis in the U.S.,
Three abstracts for ELX-02 were accepted for presentation at the
Company to host webcast and conference call on
“We are pleased to be rapidly advancing our Phase 2 programs for ELX-02. Our IND for a Phase 2 clinical trial in cystic fibrosis is open in the U.S. and the protocol has been endorsed by the
“We are gratified to have two leading CF experts leading our Phase 2 clinical trial of ELX-02 in cystic fibrosis patients: Dr.
Cystic Fibrosis Program Updates
- Our IND in the U.S. is open and the CFF has endorsed the protocol for our Phase 2 clinical in cystic fibrosis. We are on track to report top line data from a Phase 2 clinical trial in cystic fibrosis patients with the G542X CFTR mutation in the U.S. and
Europe this year. - Dr.
Ahmet Uluer , Director of the Adult Cystic Fibrosis Program at the Boston Children’s Hospital/Brigham and Women’s Hospital CF Center, will be the lead study investigator in the U.S., and Dr.Eitan Kerem , Head of theDivision of Pediatrics , Children’s Hospital,Hadassah Medical Center , will serve as the Global Lead Investigator. - Our Phase 2 program will include up to 24 patients in the U.S.,
Europe andIsrael . The protocol calls for 4 increasing doses of ELX-02 ranging from 0.3 up to 3.0 mg/kg/day in order to identify an optimal dose to carry into further development. While patient safety is the primary endpoint in Phase 2, we will be evaluating changes in sweat chloride at multiple ascending doses of ELX-02 as the primary biomarker, which is consistent with other successful Phase 2 programs for approved drugs to measure CFTR activity. We will also be evaluating changes in FEV1. - We are pleased with our participation in the European HIT-CF program and the progress being made. The program is now in the screening phase and has already identified around 40 cystic fibrosis patients with nonsense mutations who are participating in organoid development and further testing.
- Three abstracts have been accepted for presentation at the
North American Cystic Fibrosis Conference onOctober 31 st throughNovember 2 nd, 2019 inNashville, Tennessee :
- “Pharmacokinetics, Safety, and Tolerability of Single Ascending Doses of ELX-02 in
Healthy Volunteers , a Potential Treatment for Cystic Fibrosis Caused by Nonsense Mutations”Thursday, October 31, 2019 11:15 a.m. –1:45 p.m. CDT , Poster Session 1 - “Pharmacokinetics, Safety, and Tolerability of Multiple Ascending Doses of ELX-02 in
Healthy Volunteers , a Potential Treatment for Cystic Fibrosis Caused by Nonsense Mutations”Thursday, October 31, 2019 11:15 a.m. –1:45 p.m. CDT , Poster Session 1 - “Investigational Drug ELX-02 Mediates CFTR Nonsense Mutation Read-through to Increase CFTR MRNA, CFTR Protein Translation and CFTR Function”
Friday, November 1, 2019 2:15 p.m. –3:50 p.m. CDT ,Oral Workshop “New & Emerging Therapies to Correct the Basic Defect” Workshop
- “Pharmacokinetics, Safety, and Tolerability of Single Ascending Doses of ELX-02 in
Cystinosis Program Updates
- We have initiated a Phase 2 clinical trial in cystinosis in
Canada . Dr.Paul Goodyer , a Professor of Pediatrics atMcGill University and recognized leader in hereditary renal disease, is the principal investigator. The Phase 2 clinical trial is a single arm, open label study designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) ELX-02 in 6 patients with nephropathic cystinosis with at least 1 nonsense mutation in the cystinosis gene. The study will measure the dose-dependent effect of ELX-02 on cysteine levels in white blood cells, the biomarker used in the development of the most recently approved drugs for cystinosis. The study will include three nominal doses ranging from 0.5 to 2.0 mg/kg/day in order to identify an optimal dose to carry into further development.
- Genome
Quebec and Genome Canada are providing non-dilutive funding for the Phase 2 clinical trial of ELX-02 in cystinosis.The Cystinosis Research Foundation provided non-dilutive funding for the preclinical phase of the program.
- We expect to report top line data in cystinosis early in the fourth quarter of 2019.
- In support of the cystinosis program where many patients have impaired renal function, we have successfully completed a renal impairment study with ELX-02 in subjects with mild, moderate, and severe renal impairment. To date, the preliminary results support continuing our existing clinical programs and expanding our research in other kidney disorders such as autosomal dominant polycystic kidney disease. We expect to present the results from the completed renal impairment study at a scientific meeting later this year.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use.
Second Quarter 2019 Financial Results
As of
For the three months ended
Our research and development expenses were
Our general and administrative expenses were
First Half 2019 Financial Results
For the six months ended
Our research and development expenses were
Our general and administrative expenses were
Conference Call Information:
Date:
Time:
International Dial-in Number: (210) 874-7715
Conference ID: 1754316
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/9axiqnvt
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
SOURCE:
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES | |||||||
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
(Amounts in thousands, except share and per share data) | |||||||
June 30, 2019 |
December 31, 2018 |
||||||
ASSETS | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 49,326 | $ | 48,606 | |||
Marketable securities | 26,973 | — | |||||
Restricted bank deposit | 45 | 45 | |||||
Prepaid expenses and other current assets | 1,392 | 1,690 | |||||
Total current assets | 77,736 | 50,341 | |||||
Property and equipment, net | 214 | 248 | |||||
Operating lease right-of-use asset | 989 | — | |||||
Other long-term assets | 100 | 129 | |||||
Total assets | $ | 79,039 | $ | 50,718 | |||
LIABILITIES AND STOCKHOLDERS’ EQUITY | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 2,473 | $ | 747 | |||
Accrued expenses | 5,773 | 6,938 | |||||
Current portion of long-term debt | 2,231 | — | |||||
Current portion of operating lease liability | 473 | — | |||||
Taxes payable | 43 | 122 | |||||
Total current liabilities | 10,993 | 7,807 | |||||
Long-term debt | 12,306 | — | |||||
Operating lease liability | 516 | — | |||||
Stockholders’ equity: | |||||||
Common stock, $0.01 par value per share, 500,000,000 shares authorized, 40,043,365 and 35,951,537 shares issued and 39,914,668 and 35,860,114 shares outstanding as of June 30, 2019 and December 31, 2018, respectively | 402 | 360 | |||||
Common stock in treasury, at cost, 128,697 and 91,423 shares at June 30, 2019 and December 31, 2018, respectively | (1,539 | ) | (1,129 | ) | |||
Additional paid in capital | 168,848 | 129,825 | |||||
Accumulated other comprehensive income | 24 | — | |||||
Accumulated deficit | (112,511 | ) | (86,145 | ) | |||
Total stockholders’ equity | 55,224 | 42,911 | |||||
Total liabilities and stockholders' equity | $ | 79,039 | $ | 50,718 | |||
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES | |||||||||||||||
UNAUDITED CONSOLIDATED INCOME STATEMENTS | |||||||||||||||
(Amounts in thousands, except share and per share data) | |||||||||||||||
Three Months Ended June 30, |
Six Months Ended June 30, |
||||||||||||||
2019 | 2018 | 2019 | 2018 | ||||||||||||
Operating expenses: | |||||||||||||||
Research and development | $ | 7,340 | $ | 4,150 | $ | 13,359 | $ | 8,544 | |||||||
General and administrative | 6,971 | 9,560 | 12,929 | 12,953 | |||||||||||
Reverse merger related expenses | — | (167 | ) | — | 594 | ||||||||||
Total operating expenses | 14,311 | 13,543 | 26,288 | 22,091 | |||||||||||
Loss from operations | (14,311 | ) | (13,543 | ) | (26,288 | ) | (22,091 | ) | |||||||
Other expense (income), net | 138 | (137 | ) | 78 | (94 | ) | |||||||||
Net loss | $ | (14,449 | ) | $ | (13,406 | ) | $ | (26,366 | ) | $ | (21,997 | ) | |||
Basic and diluted net loss per share | $ | (0.40 | ) | $ | (0.42 | ) | $ | (0.73 | ) | $ | (0.74 | ) | |||
Weighted average number of Common Shares used in computing basic and diluted net loss per share | 36,278,567 | 31,839,303 | 36,098,171 | 29,695,430 |
Source: Eloxx Pharmaceuticals