ELOX Eloxx Pharmaceuticals Inc.

3.32
-0.01  -0%
Previous Close 3.33
Open 3.28
52 Week Low 1.44
52 Week High 8.1614
Market Cap $133,257,141
Shares 40,137,693
Float 28,256,118
Enterprise Value $111,074,517
Volume 35,844
Av. Daily Volume 149,473
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Drug Pipeline

Drug Stage Notes
ELX-02
Cystic fibrosis
Phase 2
Phase 2
Phase 2 enrollment has resumed following pause due to the COVID-19 pandemic.
ELX-02
Cystinosis
Phase 2
Phase 2
Phase 2 data released January 14, 2020.

Latest News

  1. WALTHAM, Mass., Aug. 12, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that enrollment in its Phase 2 clinical trial for ELX-02 in cystic fibrosis has resumed at certain clinical sites in the U.S. after being temporarily paused in response to the COVID-19 pandemic. On June 17, 2020, we announced that enrollment had been resumed in the Phase 2 clinical trial in cystic fibrosis in Europe and Israel after a temporary pause due to the COVID-19 pandemic.

    "We are pleased that enrollment…

    WALTHAM, Mass., Aug. 12, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that enrollment in its Phase 2 clinical trial for ELX-02 in cystic fibrosis has resumed at certain clinical sites in the U.S. after being temporarily paused in response to the COVID-19 pandemic. On June 17, 2020, we announced that enrollment had been resumed in the Phase 2 clinical trial in cystic fibrosis in Europe and Israel after a temporary pause due to the COVID-19 pandemic.

    "We are pleased that enrollment in our Phase 2 clinical trial program in cystic fibrosis has now resumed in the U.S., Israel and Europe, following temporary pauses due to the COVID-19 pandemic. Our highest priority is to complete the Phase 2 proof of concept clinical trial program and report top line data as soon as possible," said Dr. Gregory Williams, Chief Executive Officer of Eloxx. "We are grateful for the continued support and guidance from leading cystic fibrosis clinical investigators and our Cystic Fibrosis Medical Advisory Board members throughout the development, conduct and resumption of our Phase 2 clinical trial program."

    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. 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 also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with operations in Rehovot, Israel and Morristown, NJ.  For more information, please visit www.eloxxpharma.com.

    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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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:

    Barbara Ryan

    203-274-2825

    SOURCE: Eloxx Pharmaceuticals, Inc.

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  2. Enrollment in our Phase 2 clinical trial program for ELX-02 in cystic fibrosis has been resumed in Europe and Israel after being temporarily paused due to the COVID-19 pandemic

    U.S. FDA has granted orphan drug designation for ELX-02 for the treatment of cystic fibrosis

    Completing enrollment in our Phase 2 cystic fibrosis clinical trials and reporting top line data remain our highest priorities

    Preclinical studies continuing to advance in autosomal dominant polycystic kidney disease (ADPKD) and inherited retinal disorders

    Cash and cash equivalents of $37.1 million as of June 30, 2020 provides cash runway through the end of 2021

    Company to host webcast and conference call today, Thursday,

    Enrollment in our Phase 2 clinical trial program for ELX-02 in cystic fibrosis has been resumed in Europe and Israel after being temporarily paused due to the COVID-19 pandemic

    U.S. FDA has granted orphan drug designation for ELX-02 for the treatment of cystic fibrosis

    Completing enrollment in our Phase 2 cystic fibrosis clinical trials and reporting top line data remain our highest priorities

    Preclinical studies continuing to advance in autosomal dominant polycystic kidney disease (ADPKD) and inherited retinal disorders

    Cash and cash equivalents of $37.1 million as of June 30, 2020 provides cash runway through the end of 2021

    Company to host webcast and conference call today, Thursday, August 6, 2020, at 2:00 pm ET

    WALTHAM, Mass., Aug. 06, 2020 (GLOBE NEWSWIRE) -- August 6, 2020 – Eloxx Pharmaceuticals, Inc., (NASDAQ:ELOX) a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis and other diseases caused by nonsense mutations limiting production of functional proteins, today reported its financial results for the three and six months ended June 30, 2020 and provided a business update.

    "We are pleased that enrollment in our Phase 2 cystic fibrosis clinical trial has been resumed in Europe and Israel after having been temporarily paused in response to the COVID-19 pandemic in support of global healthcare providers and our shared commitment to ensure patient safety. In the U.S., we are gratified to have received orphan drug designation for ELX-02 for cystic fibrosis from the FDA which confers substantial benefits to the program including seven years of exclusivity upon marketing approval and a potential waiver of the PDUFA application fee," said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. "We are working very closely with our clinical investigators and study sites and evaluating additional sites where patient enrollment may be feasible. Our highest priority is to complete our Phase 2 proof of concept clinical trial program for ELX-02 in cystic fibrosis as soon as possible, as we believe the data from these trials will represent a substantial value inflection point for the Company."

    Company Updates

    • On August 4, 2020, we announced that the U.S. FDA had granted orphan drug designation for ELX-02 for the treatment of cystic fibrosis. The orphan drug designation confers several important benefits to support development for medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation qualifies Eloxx for certain benefits, including eligibility for marketing exclusivity for seven years post approval, tax credits on qualified U.S. clinical trial expenses, potential grant funding opportunities that can be used for clinical trials and a potential waiver of the PDUFA application fee, which is currently set at just under $3 million dollars. ELX-02 had previously been granted orphan medicinal product designation by the European Medicines Agency for the treatment of cystic fibrosis.

       
    • Our scientific manuscript titled: "ELX-02 generates protein via premature stop codon read-through without inducing native stop codon read-through protein" has been published in the August 2020 edition of by the Journal of Pharmacology and Experimental Therapeutics. This manuscript demonstrates that while ELX-02 mediates read-through of premature stop codons, the fidelity of stop codons found at the end of healthy transcripts is maintained. This indicates that translation integrity is preserved with target-therapeutic exposure of ELX-02, consistent with the favorable tolerability profile across our preclinical and clinical datasets.

       
    • On June 17, 2020, we announced that enrollment in our cystic fibrosis Phase 2 clinical trial in Europe and Israel had been resumed, while the trial in the U.S. remains paused due to the COVID-19 pandemic.

       
    • On May 6, 2020, we presented new preclinical data in a scientific presentation at the virtual Association for Research in Vision 2020 (ARVO 2020) Annual Meeting in a presentation entitled "Intravitreal administration of small molecule read-through agents demonstrate functional activity in a nonsense mutation mouse model". The studies demonstrated restoration of melanin production in a nonsense mouse model and helped validate the potential to promote read-through activity in ocular tissues via intravitreal injection.

       
    • In April 2020, we applied for and received a loan of approximately $800,000 through the U.S. SBA's "Paycheck Protection Program", which was a component of the CARES Act, signed into law in late March. PPP loans are eligible for partial forgiveness, which we will apply for, based on using the proceeds for payroll, maintaining headcount, and other specified costs. The remaining balance of the loan bears interest at the rate of 1% and is to be repaid commencing at the end of 2020.

    Cystic Fibrosis Phase 2 Program

    • Our Phase 2 program consists of two trials, one currently enrolling patients at sites in Europe and Israel and the second in the U.S., where enrollment remains temporarily paused due to the COVID-19 pandemic.



      • In the U.S., partial funding is being provided by the Cystic Fibrosis Foundation (CFF) for a portion of the trial and our protocol has been sanctioned by the Cystic Fibrosis Therapeutics Development Network (TDN).



      • In Europe, the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) has given our trial a "high priority" ranking.



    • Professor Eitan Kerem, M.D., Head of the Division of Pediatrics, Children's Hospital, Hadassah Medical Center, is the Global Lead Investigator and Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program at the Boston Children's Hospital/Brigham and Women's Hospital CF Center, is the lead study investigator in the U.S.



    • We are participating in the European HIT-CF consortium to support the collection of cystic fibrosis patient-derived organoids and the initiative to conduct a prospective clinical trial to confirm the translational potential of the organoid model. The intent of the program is to use these positive results to enroll patients with responsive organoids in a prospective trial with ELX-02. We believe this program will continue to expand the application of organoid technology from drug discovery through drug approval, and also offers possible label expansion opportunities.

    ADPKD Kidney Program Update

    • ADPKD is a relatively common inherited genetic kidney disease, which in the U.S. affects between 300,000 and 600,000 individuals and is the leading cause of end stage renal disease.

       
    • In our preclinical studies in ADPKD, we have observed dose-dependent read-through with our ERSG compounds across the most common PKD1 alleles and have expanded our studies to include PKD2. Using a patient-derived organoid with the most common PKD2 nonsense allele, we have repeated encouraging results of reduced cystogenesis and also observe a reduction in cyst size. These results demonstrate that a read-through approach potentially can have a direct impact on meaningful metrics of ADPKD progression, cyst number and size.

       
    • We continue our effort in establishing and evaluating functional models of ADPKD in order to confirm that the read-through we observe has an impact on cyst formation and growth. We are working on this program with Dr. Benjamin Freedman, a Professor in the Division of Nephrology, Department of Medicine, University of Washington, and a pioneer in ADPKD organoid technology.

    Ocular Program Update

    • In our ocular program focusing on inherited retinal disorders, our library of compounds has demonstrated dose-dependent read-through using our in vitro assay platform, acceptable intravitreal tolerability and restored protein production in an animal model via ERSG intravitreal injection. Our intravitreal read-through approach provides the opportunity to reach the totality of the retina. We achieved an important preclinical milestone which we reported on at this year's virtual ARVO Meeting by showing an increase in pigment, an indication of functional restoration of OCA2, after a single intravitreal injection of Eloxx ERSG compounds. This outcome demonstrates that ERSG compounds can reach inherited retinal disorder-relevant tissue layers beyond the photoreceptors.

       
    • While we continue in our ocular program efforts, we are gratified to observe that other researchers are also reporting on the exciting potential of Eloxx compounds in this area. For example, the group of Bikash Pattnaik at the University of Wisconsin-Madison recently published in the American Journal of Human Genetics that ELX-03 (also known as NB84) was sufficient to rescue Kir7.1 channel function in a nonsense allele cellular model derived from an individual with a form of pediatric blindness, Lebers congenital amaurosis. These promising results represent another example of how intravitreal delivery of an ERSG may have broad application across nonsense-related inherited retinal disorders through restoring production of essential proteins.

       
    • We are actively working to develop a sustained release formulation for intravitreal injection and are exploring several biodegradable, controlled release technologies. We are encouraged by the in vitro release rates achieved to date which are consistent with our target release profile of one to three months. When our tissue exposure data is coupled with our ongoing sustained release formulation efforts and the read-through potential we observe, we are encouraged that the intravitreal ERSG approach could provide restoration of critical proteins to preserve or restore visual function across nonsense-related inherited retinal disorders.

    ELX-02 is an investigational agent not approved by any regulatory authority for therapeutic use, which is currently in Phase 2 clinical trials in cystic fibrosis.

    Second Quarter 2020 Financial Results

    As of June 30, 2020, we had cash, cash equivalents and marketable securities of $37.1 million, which we expect will be sufficient to fund our operations through the end of 2021.

    For the three months ended June 30, 2020, we incurred a loss of $7.9 million or $0.20 per share, which includes $2.0 million in non-cash stock-based compensation. For the same period in the prior year, we incurred a net loss of $14.4 million, or $0.40 per share.

    Our research and development expenses were $3.5 million for the three months ended June 30, 2020, which includes $0.3 million in non-cash expense related to stock-based compensation. For the same period in the prior year, R&D expenses were $7.3 million. The quarter to quarter decrease in R&D expenses of $3.8 million was driven by reduced professional service fees largely due to the pause in our clinical trials due to COVID-19, and reduced headcount and related salaries for a portion of the 2020 period.

    Our general and administrative expenses were $4.1 million for the three months ended June 30, 2020, which includes $1.7 million in non-cash expense related to stock-based compensation. For the same period in the prior year, G&A expenses were $7.0 million. The decrease was primarily driven by lower headcount and professional services costs.

    First Half 2020 Financial Results

    For the six months ended June 30, 2020, we incurred a loss of $21.8 million or $0.54 per share, which includes a one-time restructuring charge of $4.0 million associated with our realignment in the first quarter (comprised of $2.1 in non-cash stock based compensation and $1.9 million in cash severance) and $3.8 million in non-cash stock-based compensation associated with ongoing operations. For the same period in the prior year, we incurred a net loss of $26.4 million, or $0.73 per share.

    Our research and development expenses were $8.1 million for the six months ended June 30, 2020, which includes $0.5 million in non-cash expense related to stock-based compensation. For the same period in the prior year, R&D expenses were $13.4 million. The year over year decrease in R&D expenses of $5.3 million was driven by reduced professional service fees largely due to the pause in our clinical trials due to COVID-19, and reduced headcount and related salaries for a portion of the 2020 period.

    Our general and administrative expenses were $9.3 million for the six months ended June 30, 2020, which includes $3.4 million in non-cash expense related to stock-based compensation. For the same period in the prior year, G&A expenses were $12.9 million. The decrease of $3.6 million was primarily driven by lower headcount and professional services costs.

    Conference Call and Webcast Information:

    Date: Thursday, August 6, 2020

    Time: 2:00 p.m. ET

    Domestic Dial-in Number: (866) 913-8546

    International Dial-in Number: (210) 874-7715

    Conference ID: 2191126

    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/nxdhnad8.

    Eloxx Pharmaceuticals

    Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (each designed to be a eukaryotic ribosomal selective glycoside, or ERSG) 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, currently focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory authority. Eloxx's preclinical candidate pool consists of a library of novel ERSG drug candidates identified based on read-through potential. Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders. Eloxx is headquartered in Waltham, MA, with operations in Rehovot, Israel and Morristown, NJ. For more information, please visit www.eloxxpharma.com.

    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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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:

    Barbara Ryan

    203-274-2825





    SOURCE: Eloxx Pharmaceuticals, Inc.



    ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
    UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
    (Amounts in thousands, except share and per share data)
         
      June 30, December 31,
       2020   2019 
    ASSETS    
    Current assets:    
    Cash and cash equivalents $30,347  $22,493 
    Marketable securities  6,769   33,783 
    Restricted cash  52   43 
    Prepaid expenses and other current assets  1,800   1,390 
    Total current assets  38,968   57,709 
    Property and equipment, net  165   201 
    Operating lease right-of-use asset  678   924 
    Other long-term assets  110   113 
    Total assets $39,921  $58,947 
         
    LIABILITIES AND STOCKHOLDERS' EQUITY    
    Current liabilities:    
    Accounts payable $770  $1,871 
    Accrued expenses  3,485   4,655 
    Current portion of long-term debt  5,149   4,336 
    Advances from collaboration partners  805   403 
    Current portion of operating lease liability  524   499 
    Taxes payable  38   43 
    Total current liabilities  10,771   11,807 
    Long-term debt  8,704   10,502 
    Operating lease liability  155   425 
    Total liabilities  19,630   22,734 
    Stockholders' equity:    
    Preferred stock, $0.01 par value per share, 5,000,000 shares authorized, no

      shares issued or outstanding as of June 30, 2020 or December 31, 2019
          
    Common stock, $0.01 par value per share, 500,000,000 shares authorized,

      40,326,906 and 40,186,469 shares issued, and 40,135,290 and 40,030,763

      shares outstanding as of June 30, 2020 and December 31, 2019, respectively
      403   402 
    Common stock in treasury, at cost, 191,616 and 155,706 shares as of

      June 30, 2020 and December 31, 2019, respectively
      (1,822)  (1,703)
    Additional paid-in capital  180,549   174,515 
    Accumulated other comprehensive income  13   18 
    Accumulated deficit  (158,852)  (137,019)
    Total stockholders' equity  20,291   36,213 
    Total liabilities and stockholders' equity $39,921  $58,947 



    ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
    UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
    (Amounts in thousands, except share and per share data)
             
      Three Months Ended June 30, Six Months Ended June 30,
       2020   2019   2020   2019 
    Operating expenses:        
    Research and development $3,528  $7,340  $8,077  $13,359 
    General and administrative  4,058   6,971   9,282   12,929 
    Restructuring charges        3,994    
    Total operating expenses  7,586   14,311   21,353   26,288 
    Loss from operations  (7,586)  (14,311)  (21,353)  (26,288)
    Other expense, net  301   138   480   78 
    Net loss $(7,887) $(14,449) $(21,833) $(26,366)
             
    Net loss per share, basic and diluted $(0.20) $(0.40) $(0.54) $(0.73)
    Weighted average number of shares of common stock used in computing net loss per share, basic and diluted  40,129,304   36,278,567   40,101,789   36,098,171 

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  3. WALTHAM, Mass., Aug. 04, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for ELX-02 for the treatment of Cystic Fibrosis.

    The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan-drug designation qualifies Eloxx for certain benefits…

    WALTHAM, Mass., Aug. 04, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted the Company orphan drug designation for ELX-02 for the treatment of Cystic Fibrosis.

    The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan-drug designation qualifies Eloxx for certain benefits, including seven years of market exclusivity upon regulatory approval if received, exemption of FDA application fees, tax credits on qualified U.S. clinical trials and eligibility for grant funding opportunities that can be used for clinical trial costs.

    "We are pleased to have received orphan drug designation from the FDA for ELX-02 in the U.S. Combined with our previous EMA orphan medicinal product designation in Europe, and upon approvals in the U.S. and the E.U., we will be able to obtain significant periods of marketing exclusivity in these important markets and provide treatment options to patients with high unmet medical needs, particularly in patients with nonsense mutations for whom there are few, if any, treatments available," said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. "As previously announced, we have resumed enrollment in our Phase 2 proof of concept clinical trials in Israel and Europe, with our trial in the U.S. continuing to be temporarily paused due to the COVID-19 pandemic. We look forward to reporting top line results as soon as feasible, and believe that this will be a major value inflection point for our Company."

    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.

    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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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:

    Barbara Ryan

    203-274-2825



    SOURCE: Eloxx Pharmaceuticals, Inc.

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  4. WALTHAM, Mass., July 28, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that Dr. Gregory Williams, Chief Executive Officer of Eloxx, will make a presentation and host one-on-one meetings with investors at the 40th Annual Canaccord Genuity Virtual Growth Conference on August 13th, 2020.

    The presentation will be webcast live and may be accessed by visiting the "Events & Presentations" page within the Investors section of the Eloxx website at http://investors.eloxxpharma.com or by using…

    WALTHAM, Mass., July 28, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that Dr. Gregory Williams, Chief Executive Officer of Eloxx, will make a presentation and host one-on-one meetings with investors at the 40th Annual Canaccord Genuity Virtual Growth Conference on August 13th, 2020.

    The presentation will be webcast live and may be accessed by visiting the "Events & Presentations" page within the Investors section of the Eloxx website at http://investors.eloxxpharma.com or by using the link below. A replay of the webcast will be available on the Eloxx website following the conference.

    Date: Thursday, August 13th, 2020

    Time: 4:00 P.M. ET

    Webcast:  http://wsw.com/webcast/canaccord42/elox/

    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.

    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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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:

    Barbara Ryan

    203-274-2825

    SOURCE: Eloxx Pharmaceuticals, Inc.

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  5. WALTHAM, Mass., July 21, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that it will release its second quarter 2020 financial and operational results and host a conference call and live webcast on Thursday, August 6, 2020.

    Conference Call Information:
    Date: Thursday, August 6, 2020
    Time: 2:00 p.m. ET
    Domestic Dial-in Number: (866) 913-8546
    International Dial-in Number: (210) 874-7715
    Conference ID: 2191126
    Live Webcast: accessible from the Company's website at www.eloxxpharma.com under…

    WALTHAM, Mass., July 21, 2020 (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 and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that it will release its second quarter 2020 financial and operational results and host a conference call and live webcast on Thursday, August 6, 2020.

    Conference Call Information:

    Date: Thursday, August 6, 2020

    Time: 2:00 p.m. ET

    Domestic Dial-in Number: (866) 913-8546

    International Dial-in Number: (210) 874-7715

    Conference ID: 2191126

    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/nxdhnad8.

    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.

    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; the impact of global health concerns, such as the COVID-19 global pandemic, on our ability to continue our clinical and preclinical programs and otherwise operate our business effectively; 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:

    Barbara Ryan



    (203) 274-2825

    SOURCE: Eloxx Pharmaceuticals, Inc.

    Primary Logo

    View Full Article Hide Full Article
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