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Eloxx Pharmaceuticals

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

  • ZKN-013 is a phase I ready oral therapy designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP)

BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) — Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.

The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales.

We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform.”

This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases,” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall. “We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations.”

RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells.

ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene.

ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx’s TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). 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 Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations.

For more information, please visit www.eloxxpharma.com.

About Almirall  

Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others.

For more information, please visit almirall.com

Corporate Communications contact Almirall: Investors’ Relations contact
Corporate communications team Pablo Divasson del Fraile
corporate.communication@almirall.com investors@almirall.com
Phone: (+34) 659 614 173 Phone: (+34) 93 291 30 87
Eloxx Contacts:
Investors:
John Woolford
john.woolford@westwicke.com
443.213.0506
Media:
Laureen Cassidy
laureen@outcomescg.com

Eloxx Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Almirall Legal warning
This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are “forward-looking statements”. These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

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Eloxx Pharmaceuticals Announces FDA Clearance to Begin Single Ascending Dose Study of ZKN-013

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

May 2, 2023
ZKN-013 is in development for potential treatment of recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB) and Familial
Adenomatous Polyposis (FAP)
ZKN-013 is Eloxx’s first drug candidate from its TURBO-ZM™ platform to gain clearance for clinical testing
WATERTOWN, Mass., May 02, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the “Company”), a leader in
ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the
Company’s Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the
potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by
mutations in Collagen7 gene.
“FDA clearance to begin our planned single ascending dose trial is an important milestone towards providing a potential treatment option for patients
with RDEB and JEB,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “This update is also a momentous event for Eloxx, as
ZKN-013, our lead TURBO-ZM™ based molecule, is the first program developed from hit to lead and is now poised to enter clinical development.
Beyond ZKN-013, Eloxx has promising TURBO-ZM based discovery programs in cystic fibrosis (fully funded by the Cystic Fibrosis Foundation) and in
certain cMYC-overexpressing cancers.”
Further SAD and multiple ascending dose (MAD) testing is expected to be conducted following the completion of the planned dose cohorts in the SAD
study and discussion with the FDA. The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA.
ZKN-013 Preclinical Activity in RDEB, JEB and FAP
ZKN-013 has demonstrated robust functional preclinical activity in RDEB and JEB patient cells and in APCmin (multiple intestinal neoplasia) mice.
Eloxx also plans to develop ZKN-013 for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and
progression to colon cancer. In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in
intestinal polyps and adenomas, resulting in increased survival.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform
in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). 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 Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. Eloxx also has preclinical programs
focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant
polycystic kidney disease, as well as rare ocular genetic disorders.
For more information, please visit www.eloxxpharma.com.
Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other
than statements of present and historical facts contained in this press release, including without limitation, statements regarding clinical testing of
ZKN-013 for the treatment of RDEB and JEB and the expected timing of trials for our other product candidates and the potential of our product
candidates to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,”
“would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,”
“seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-
looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us.
Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ
materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to
progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical
studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research
and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic
on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing,
prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product
licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market;
general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and
the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the
fiscal year ended December 31, 2022, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s
website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to
update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances
or otherwise.
Contact
Investors
John Woolford
john.woolford@westwicke.com
443.213.0506
Media
Laureen Cassidy
laureen@outcomescg.com
SOURCE: Eloxx Pharmaceuticals, Inc.

Eloxx Pharmaceuticals Announces Fast Track Designation for ELX-02 for the Treatment of Cystic Fibrosis Patients with Nonsense Mutations

By Eloxx Pharmaceuticals, Press Release, Publicly Listed
Press Release.

 

September 9, 2021

WATERTOWN, Mass., Sept. 09, 2021 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02, a drug candidate intended to treat cystic fibrosis patients with nonsense mutations. ELX-02 is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene for whom there are no effective disease modifying therapies. The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity, and Eloxx expects to present data from the first four treatment arms in the fourth quarter of 2021.

“We are delighted to receive Fast Track Designation from the FDA for ELX-02 as the need for patients remains significant. The ability to have greater access to the FDA and their guidance on the regulatory pathway for ELX-02 can help provide the ability to work with the urgency needed on behalf of CF patients with nonsense mutations,”

said Sumit Aggarwal, President and CEO of Eloxx.

Fast Track Designation is granted to drugs being developed for the treatment of serious or life-threatening diseases or conditions where there is an unmet medical need. The purpose of the provision is to help facilitate development and expedite the review of drugs to treat serious or life-threatening conditions so that an approved product can reach the market expeditiously. Sponsors of drugs that receive Fast Track Designation have the opportunity for more frequent interactions with the FDA review team throughout the development program.

ELX-02 has previously been granted orphan drug designation by the FDA and orphan medicinal product designation by the European Medicines Agency.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging both its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). 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 clinical development focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx also has preclinical programs focused on select rare diseases including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit www.eloxxpharma.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation, statements regarding the expected timing of trials and results from clinical studies of our product candidate and the potential of our product candidate to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words.

Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2021, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financial-information/sec-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contact

Investors
John Woolford
john.woolford@westwicke.com
443.213.0506

Media
Laureen Cassidy
laureen@outcomescg.com

Source: Eloxx Pharmaceuticals, Inc.

Eloxx Pharmaceuticals Acquires Zikani Therapeutics

By Eloxx Pharmaceuticals, Press Release, Publicly Listed
Press Release.

 

Combined Company to be Leader in Ribosomal RNA-Targeted Genetic Therapy Bringing Together Complementary Platforms

Maximizes Potential for ELX-02 for Cystic Fibrosis in Phase 2 Development

Adds Preclinical Stage Pipeline in Rare Diseases and Oncology Targeting RNA
and Ribosomal Mutations

Expect to File IND for First Oral Drug to Treat Patients with Recessive Dystrophic
and Junctional Epidermolysis Bullosa (RDEB and JEB)

Sumit Aggarwal, Zikani President and CEO, to Lead the Combined Company

Eloxx to Issue Approximately 7.6 Million Shares to Zikani Stockholders

Company to Host Investor Call at 8:30 a.m. ET, April 1

WALTHAM, MA and WATERTOWN, MA – APRIL 1, 2021 – Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today announced it has acquired Zikani Therapeutics, Inc. in an all-stock transaction, with the potential to create a leader in ribosomal RNA-targeted therapies for treatment of rare diseases and oncology. Sumit Aggarwal, previously the President and Chief Executive Officer of Zikani, has been named President and Chief Executive Officer of Eloxx, and Vijay Modur, M.D., Ph.D., who was Zikani’s Chief Scientific and Medical Officer, has been named Eloxx’s Head of Research and Development.

“With the strength of our ELX-02 program for cystic fibrosis, this acquisition provides us with the opportunity to amplify the potential of our innovative science by developing a new class of therapies to treat diseases with limited to no treatment options under the stewardship of leaders with a proven ability to translate technology into treatments for patients,”

said Tomer Kariv, Eloxx Chairman.

“We are excited about the potential of ELX-02 and combining the companies opens the door to build a leadership position in genetic therapy by rapidly developing treatments that can restore functional proteins in patients with nonsense mutations in their RNA,”

said Aggarwal.

“The combined capabilities of Eloxx and Zikani in chemistry, biology, regulatory and drug development, including Zikani’s TURBO-ZMTM synthetic chemistry platform for designing macrolide-based Ribosome Modulating Agents (RMAs), along with a committed leadership team and talented employees, will further accelerate our ability to impact the lives of those who have rare diseases with the type of urgency and novel thinking that they deserve,”

added Aggarwal.

ELX-02 is currently in Phase 2 clinical trials in Cystic Fibrosis (CF) patients affected by nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The investigational therapy has shown strong activity across a full range of mutations in CF preclinical models. In Phase 1 testing, ELX-02 was generally well- tolerated and demonstrated high bioavailability with consistent pharamacokinetics across both single and multiple-dose studies.

“The Phase 2 trials are designed to validate the safety of ELX-02 and assess its biological activity. We look forward to completing enrollment in the first four treatment arms by mid-year and reporting data from these treatment arms in the second half of this year,”

said Dr. Modur.

In addition to CF, the company plans to file an IND in 2022 for what could potentially become the first oral therapy for protein restoration for patients with nonsense mutations in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB). RDEB is an incurable, extremely painful and often fatal skin blistering condition caused by a lack of collagen type VII that is estimated to affect more than 3,000 people worldwide. JEB is the most severe form of EB, with most patients dying in infancy.

By extending the application of ribosomal RNA modulation to the readthrough of nonsense mutations in tumor suppressor genes, the company is also rapidly advancing preclinical research for familial adenomatous polyposis (FAP), an inherited pre-cancerous colorectal disease frequently caused by nonsense mutations in the adenomatous polyposis coli (APC) gene.

Nonsense mutations cause approximately 10-12 percent of rare inherited diseases. ELX-02 along with the TURBO-ZMTM library of compounds are anticipated to significantly expand to include the treatment of many other rare diseases and certain cancers.

Acquisition Terms

Under the terms of the merger agreement, stockholders of Zikani received approximately 7.6 million Eloxx common shares and own approximately 16 percent of the combined company.

Board and Management Changes

In connection with the acquisition, Silvia Noiman, Ph.D., and Martijn Kleijwegt have stepped down from the Eloxx Board. Alan Walts, Ph.D., and Raj Parekh, Ph.D., who have both served as Zikani directors, were appointed to fulfill the vacancies and serve out the remaining terms of office.

“We’re pleased to welcome Sumit and Vijay to the Eloxx leadership team. They demonstrated their ability to transform Zikani by following the science and pursuing the creation of a new class of therapies on behalf of patients with unmet medical need. We want to extend our thanks and appreciation to Dr. Greg Williams for his stewardship of Eloxx and his commitment to advancing the critical work of the company. We are pleased that Greg will continue to advise Eloxx to facilitate a smooth transition” said Kariv.

Conference Call Information

Date: Thursday, April 1, 2021

Time: 8:30 a.m. ET

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

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

Conference ID: 8180169

Live Webcast: accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or by clicking here. A replay of this conference call will be available on the Eloxx and Zikani websites.

Leadership Profiles

Sumit Aggarwal

Sumit Aggarwal served as Zikani’s President and CEO. He has led the transformation of Zikani from an early-stage technology company to a development-stage rare disease and oncology focused organization. Under Aggarwal’s leadership, Zikani has concentrated its focus on demonstrating pre-clinical proof of efficacy across several disease states using its TURBO-ZM™ technology platform.

In his more than 20 years in pharmaceutical and biotechnology commercial operations, investment management and management consulting, Aggarwal has been successful in transforming companies by re-invigorating innovation, growth and profitability, and raising capital for promising technology companies.

Prior to joining Zikani, he reinvigorated growth and profitability at Progenity, raised $125 million in capital and built a novel drug delivery-based GI pipeline. He also held leadership roles in healthcare and biotechnology at Adage Capital and as an Associate Partner at McKinsey & Company in its healthcare practice.

Aggarwal has an MBA with distinction from the Johnson School, Cornell University, and a Bachelor of Technology with Honors in Chemical Engineering from the Indian Institute of Technology, Kharagpur.

Vijay Modur, M.D., Ph.D.

Vijay Modur, M.D., Ph.D., served as Zikani’s Chief Scientific and Medical Officer and has led the scientific efforts to transform medicines based on ribosomal modulation using Zikani’s proprietary TURBO-ZM™ technology platform.

In his more than 20 years in pharmaceutical and diagnostic roles in R&D, he has successfully translated research discovery efforts into products that have impacted medical practice.

Prior to joining Zikani, Dr. Modur led the venglustat rare disease program at Sanofi across multiple rare disease indications into Phase 2 and Phase 3 clinical development along with leading other early development programs. Prior to Sanofi, he held leadership roles in HTG Molecular, Novartis Oncology and Merck Research Labs.

Dr. Modur obtained his MBBS from Karnatak University and his Ph.D. from the University of Utah. He was a resident in Clinical Pathology at Washington University School of Medicine where he also completed his post-doctoral fellowship.

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 readthrough 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.

About Zikani Therapeutics

Zikani Therapeutics is an emerging leader in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform to develop novel Ribosome Modulating Agents (RMAs) as therapeutics for people with limited treatment options. Zikani’s TURBO-ZMTM platform allows rapid synthesis of novel compounds that can be optimized to modulate the ribosome in a disease specific manner. As the company evolves its focus from early-stage to clinical-stage research, Zikani is actively moving into pre-clinical development to target select rare diseases including inherited diseases and cancers caused by nonsense mutations. For more information, visit zikani.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 readthrough 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, including successfully integrating the combined companies; 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

Investors
John Woolford
john.woolford@westwicke.com
443.213.0506

Media
Laureen Cassidy
laureen@outcomescg.com