Facebook-f X-twitter Linkedin-in

EAP

Clene Nanomedicine, Inc. Expanded/Early Access Policy

PURPOSE AND SCOPE

Clene is focused on the discovery and development of innovative, life-changing medicines for people with serious and life-threatening diseases. To do this, Clene conducts clinical studies to assess the safety and effectiveness of its investigational medicines. Clinical studies are controlled research studies in people designed to understand if an investigational medicine is safe and effective. Data generated by these studies provide health regulatory authorities with essential information needed to evaluate an investigational medicine. Only upon review of this information will regulators approve a new medicine, allowing it to become widely available.

Importantly, Clene believes that participating in clinical trials is the best way for patients to access investigational medicines prior to regulatory approval. In certain cases, when it is not possible for patients to participate in a clinical trial, a physician, hospital, or university may seek special access to a Clene investigational medicine outside of a clinical study. These requests are typically called ‘expanded access’, ‘early access’, ‘compassionate use’, ‘named patient use’, or ‘temporary authorization for use’. These requests are different from a clinical trial, where more comprehensive safety and efficacy data are collected.

TYPES OF EXPANDED/EARLY ACCESS PROGRAMS OFFERED

On a limited basis, Clene offers two different types of expanded/early access programs (collectively, hereinafter “EAP”) based on specific criteria described below:

(1) Multiple Patient EAP
If, in cooperation with select academic collaborators, Clene believes there is sufficient scientific information to identify a population of patients for whom Expanded Access may be appropriate and aligned with the eligibility criteria below, Clene may work with select academic sites to establish an EAP program for a group of patients who meet specific eligibility criteria. An EAP granted on this basis will follow a specific protocol, developed in cooperation with a university or hospital physician investigator and submitted to an institutional review board and health regulatory agency for review. These multiple-patient Expanded Access programs are managed either by Clene or a select hospital, institution, or university.

(2) Individual Named Patient EAP
In rare cases, Clene may provide investigational medicine access to an individual patient for treatment, where it is allowed by local regulations. An individual patient EAP is managed by the patient’s physician and is not generally governed by a protocol.

ELIGIBILITY CRITERIA FOR AN EAP REQUEST

Making decisions about requests for an EAP is complex. Clene works with cooperating clinicians in thorough, individualized reviews of each request for an EAP on a case-by-case basis. Clene strives to make these decisions as ethically and equitably as possible, in cooperation with participating clinicians, taking into consideration the patient’s need, the available medical and scientific information about the investigational medicine, the seriousness of the disease, and the potential risk and benefit to the patient. Foremost, Clene is committed to patient safety and well-being; our top priority when evaluating requests for an EAP is to ensure that we are not putting patients at unreasonable risk of harm given the context of the disease or condition being treated.

Clene will consider an EAP for an investigational medicine only if all of the following criteria are met:

  • The disease must be serious or life-threatening;
  • There are no comparable or satisfactory alternatives available to treat the disease or condition;
  • The patient is ineligible or unable to participate in a clinical trial for the investigational medicine;
  • The EAP is permitted under applicable law;
  • The investigational medicine has established safety data from a controlled clinical trial;
  • In the judgment of Clene and the requesting physician, there is sufficient scientific evidence to warrant that the potential benefit of the investigational medicine would likely outweigh the potential risks to the patient;
  • There are available clinical data to identify an appropriate dose;
  • There is adequate manufacturing capacity to support both the EAP as well as ongoing and planned clinical trials without compromising compliance with applicable manufacturing requirements and standards;
  • Granting the EAP is unlikely interfere with or delay current clinical trials or regulatory filings;
  • A licensed physician, hospital, or university has communicated an unsolicited request for an EAP for the investigational medicine to Clene;
  • The treating physician(s) has expertise with the specific disease and ability to monitor the patient’s safety, and will obtain appropriate institutional approval(s) and informed consent or assent from the individual patient for treatment use of the investigational medicine, as necessary;
  • An EAP will only be granted in a country where Clene expects to file for regulatory approval for the investigational medicine;
  • The treating physician(s) will collect patient data, and maintain and release to regulatory authorities, and to Clene, all treatment records and data, as applicable;
  • The patient, or the patient’s legal representative, is able to sign an informed consent document.

EVALUATION OF REQUESTS

Clene’s willingness to consider requests for an EAP for an investigational medicine is based on its review of the patient information, in cooperation with the requesting physician and/or institution, supporting early access as submitted by the requesting physician and/or institution, as well as the provision to Clene of satisfactory evidence that the physician responsible for treating the patient is appropriately qualified in accordance with local laws and regulations.

To ensure a fair review of the EAP requests, decisions will be based on the above criteria. Requests will be considered on a first come, first served basis.

In considering and administering requests for an EAP, our patient-focused principles are based on a commitment to be fair, impartial, and transparent. Our decisions are guided by the best clinical and scientific evidence currently available for any given investigational medicine.

Of note, Clene cannot guarantee that the investigational medicine will be available for a particular patient under an EAP. This policy does not provide a guarantee of access to any specific investigational medicine for any patient. If an EAP program is granted, access will be granted only for a designated period of time, following which an additional request must be submitted and reviewed to continue the program. An EAP will be discontinued as soon as feasible once the investigational medicine is approved by the health regulatory authority for the relevant use. In accordance with the 21st Century Cures Act, Clene Nanomedicine, Inc. may revise this policy at any time. This policy will be updated with a hyperlink or other reference to the EAP on clinicaltrials.gov once posted.

TREATING PHYSICIAN CRITERIA AND RESPONSIBILITIES

Any physician treating a patient receiving an investigational medicine through EAP must be properly licensed and fully qualified to administer the product. Such physician will be responsible for collecting data throughout the treatment and for submitting those data to Clene in accordance with local laws and regulations. In addition, the physician will be responsible for ensuring all local legal, regulatory and Health Authority requirements are met, including Investigational Review Board or Ethics Committee approval for the EAP, and reporting of Adverse Events.

The physician must agree beforehand in writing to comply with the following terms:

  1. To use the product only for the patient(s) specified.
  2. To follow any applicable country-specific legal and regulatory requirements related to providing an investigational medicine under Expanded Access, Early Access, or comparable program.
  3. To comply with any Clene requirements applicable to patient confidentiality and data privacy, medical criteria, safety reporting, drug supply handling and use, and protection of intellectual property and confidential information.
  4. To serve as the Sponsor-Investigator for the EAP with respect to the FDA and file all necessary and required forms, reports and other submissions with FDA.
  5. To obtain an informed consent form (ICF) from the patient(s), which has been approved by an Institutional Review Board, Human Ethics Research Committee, or comparable regulatory review body. The ICF must include a statement that the investigational medicine maybe provided under the EAP only until such time as regulatory approval has been received for the investigational medicine. Clene will provide the investigational medicine to be administered under the EAP. However, any associated costs of treatment, including but not limited to drug administration costs, costs associated with laboratory or radiological monitoring, travel, physician or facility fees will be the responsibility of the insurer, health care system, and/or patient.

REQUEST AND APPROVAL PROCESS

Requests for an EAP for a Clene investigational medicine must be made by the patient’s treating physician. Physicians should submit requests to: EAP@clene.com.

If an Individual Named Patient EAP is approved, Clene will send to the treating physician:

  • a Letter of Acceptance (including a request for confirmation of applicable institutional approvals and the patient’s informed consent);
  • a Letter of Authorization, if applicable, authorizing the physician to reference Clene’s existing IND in submissions to the FDA;
  • Instructions for Use for the investigational medicine; and
    Adverse Event Report Form.
  • By signing a Letter of Acceptance, the treating physician agrees to fulfill all the responsibilities outlined in such letter.
  • If a Multiple Patient EAP is approved, Clene will partner with the requesting physician and/or institution to develop an EAP protocol, a formal contract to support or supply the EAP, and will file the protocol with the appropriate Institutional Review Board, Human Ethics Research Committee, or comparable regulatory review body, as applicable.

Mark Mortenson

Chief Science Officer

Mr. Mortenson is the co-inventor of the technology platform developed to produce Clene’s Clean-Surface Nanocrystal (CSN™) therapeutics, as well as the inventor/co-inventor for 30 other U.S. patents and hundreds of corresponding foreign patents.

Prior to joining Clene, Mr. Mortenson served as former Chief Operating Officer of Research, Development and Manufacturing for an advanced materials-based company of more than 300 employees. In addition to his professional experience, he is a former chief patent counsel responsible for approximately 5,500 patents and patent applications in the U.S. and 44 foreign countries. Mr. Mortenson holds both a Bachelor of Science in physics and a Bachelor of Science in ceramic engineering from Alfred University, a Master of Science in material science from Pennsylvania State University, and a Juris Doctor degree from George Washington University.

Rob Etherington

President & CEO

Mr. Etherington brings nearly 30 years of sales, marketing and leadership experience in the pharmaceutical industry to Clene. Prior to joining Clene as CEO, Mr. Etherington worked for Actelion Pharmaceuticals, which was the largest biopharmaceutical company based in the EU until Johnson & Johnson Pharmaceuticals acquired the company in 2017. He initially joined Actelion as founding Director of Marketing in 2000, the same year the company went public, and went on to hold various roles of increasing responsibility. Mr. Etherington served as the first U.S. commercial employee, leading U.S. commercial operations and serving as Chair of the U.S. Commercial Strategic team where he was responsible for five marketed drugs. Prior to Actelion, Mr. Etherington began his pharmaceutical career with a number of sales and marketing roles at Parke-Davis, a division of Pfizer, culminating in a Team Leader position that oversaw the drug Lipitor. As the founding CEO of Clene, Mr. Etherington has raised more than $150M in equity financings, and has driven Clene’s focus on neurodegenerative diseases.

Joanne Zhongyan Zhang M.D., PhD

Director of Micro and Biology

Joanne Zhongyan Zhang is the Director of Micro and Biology at Clene. Dr. Zhang has spent more than 20 years in medical research and is an experienced leader in quality control microbiology laboratory and scientific research in biology. Dr. Zhang helped design, and personally executed the initial and validated Cuprizone Studies of Clene’s asset, CNM Au8, suggesting that Au8 may enhance remyelination in MS. Dr. Zhang designed and directed the ALS pilot study proving that Au8 improved the motor coordination in congenic ALS SOD1 G93A mouse model. Both MS and ALS are now in Clinical Trial Phase II. Dr. Zhang brings to Clene both clinical experience and a successful medical research background. She spent over six years working as an attending physician and chief physician at Tianjin First Central Hospital. Dr. Zhang was a postdoctoral fellow in Cell Biology at Johns Hopkins University; she worked as research associate in the Cardiology Department at Duke University; and prior to Duke was awarded Special Fellow status by the Japan Society for the Promotion of Science while working at Osaka University. Dr. Zhang was awarded her medical degree from Tianjin Medical University and received her PhD from the Cardiology Division of Internal Medicine and Molecular Science at Osaka University.

D. Kyle Pierce

Vice President, Research & Development

D. Kyle Pierce is the Executive Director of Nano-Engineering at Clene. Mr. Pierce has been with Clene since its inception. Mr. Pierce oversees the R&D department, designed the production equipment and software which Clene uses in its manufacturing of their key asset, and developed production procedures. He started his carrier in an antecedent technology and development company, General Resonance, as a Senior Engineer developing the technology used for Clene’s production equipment. While attending the University of Maryland at College Park, where he received a BS in Electrical Engineering, he worked for the Army Material System Analysis Activity (AMSAA DoD). While at AMSAA Mr. Pierce debugged and maintained an active protection performance model software, he was issued a Secret Security Clearance from the Department of Defense. While at Clene, he has co-authored numerous peer reviewed publications.

Austin Rynders

Vice President, Clinical Operations

Austin Rynders is the Vice President of Clinical Operation at Clene. Mr. Rynders is a multi-faceted professional with over 10 years of clinical experience heavily focused in CNS/pain methodology with an emphasis in investigational pain modeling, abuse potential, degenerative disorders, and unique early proof-of-concept study development across Phase I-III studies. With participation and oversight of over 200 clinical trials prior to joining Clene, Mr. Rynders’ exposure to complex and unique study designs has allowed for development of a robust clinical mindset and multiple key accomplishments. These accomplishments include the development and implementation of validated methodologies surrounding ventilatory responses to hypercapnia and quantitative sensory testing, development and beta testing of cognition-based assessment platforms, operational reorganization of multiple clinical operations teams, and the expansion and development of a purpose built 136 bed Phase 1 facility. Mr. Rynders has received a Bachelor of Nursing from the University of Utah and will soon obtain his Master of Science in Pharmacology and Toxicology from Michigan State University.

Adam Dorfman

Vice President, Quality & Technical Operations

Adam Dorfman is the Executive Director of Quality Control and Bioanalytical at Clene. Mr. Dorfman has over 13 years of experience in various roles including nanoparticle development and characterization, specimen bioanalysis, and quality control. Mr. Dorfman has been with Clene since 2013 and is responsible for ensuring that the finished pharmaceuticals meet all quality standards for Clene’s active clinical trials. In addition, he leads the bioanalytical group that analyzes all GLP/GCP tissue and pharmacokinetic specimens that are collected during pre-clinical and clinical activities. Mr. Dorfman was originally tasked with establishing a GMP/GLP compliant lab at Clene, and more recently has been responsible for developing and implementing Clene’s laboratory informatics system that will streamline all manufacturing and quality control processes. He has a BS in Chemical Engineering from The Pennsylvania State University and MS in Chemical and Biomolecular Engineering from Johns Hopkins University.

Mary Anne McNeil

Head of Human Resources

Mary Anne McNeil is the Head of Human Resources at Clene. Ms. McNeil is an accomplished professional with a diverse and successful career in human resources, management and marketing. Prior to joining Clene, Ms. McNeil served as the EVP of Human Resources, Marketing and Sales for a successful art business, where she executed a company culture of diversity, career development, and an outstanding sales force. While there, sales increased by 40%, the employee retention rate doubled and the signing of new artists to represent increased by over 12%. Ms. McNeil is creating a company culture which encourages diversity, growth and learning, open communication and accessibility, recognition for exemplary work, long term careers while striving to make Clene a leading and desirable place of employment. She has started a management training program by working with a local college, has implemented a review and raise process, has encouraged career mapping with all Clene colleagues which has led to many promotions within the company, assisted in developing a comprehensive training program and is working with workforce services to employ individuals from the local area. doubled. She is excited for the future of Clene and looks forward to its success and many upcoming milestones.

Jerry Miraglia, J.D.

General Counsel and Corporate Secretary

Mr. Miraglia has advised small private companies and large public companies, ranging from startups with only an idea to Fortune 125 companies. Most recently, he led the M&A legal team at Jabil Inc (NYSE: JBL), a contract manufacturer with 250,000+ employees operating in 30+ countries, including its medical solutions healthcare division. Mr. Miraglia spent the first 14 years of his legal career in Baltimore, joining Miles & Stockbridge out of law school and becoming a full equity partner in their corporate department, advising such clients as Black & Decker. Mr. Miraglia left Miles to serve as General Counsel for an interactive entertainment company, and subsequently entered the business world and served as EVP for Dover Motorsports (NYSE: DVD) and ultimately President of a media company. Mr. Miraglia’s diverse background navigating both business and legal aspects of corporate transactions, manufacturing, sales, marketing and distribution, providing Clene with a unique blend of insights as it leaves the private sector and enters the public.

Michael Hotchkin

Chief Development Officer

Mr. Hotchkin brings more than 20 years of experience in pharmaceutical commercialization, planning, strategy development, and medical affairs to Clene, where he currently leads commercial planning and strategy development, preclinical translational medicine, and health regulatory authority filings. Prior to joining the company, Mr. Hotchkin was a founding team member at Actelion Pharmaceuticals US, Inc., where he helped launch multiple best-in-class products for orphan disorders. Having spent more than a decade at Actelion, Mr. Hotchkin led the commercialization efforts for the company’s Genetic Business Unit, including clinical development activities which resulted in the doubling of sales for two different pharmaceutical drugs. Among his contributions while at Actelion include the implementation of new market planning activities for the company globally, directing U.S. strategy development and leading marketing activities for multiple medicines. Mr. Hotchkin began his career at Parke-Davis, a subsidiary of Pfizer.

Morgan Brown

Chief Financial Officer

Mr. Morgan Brown has more than 29 years of finance and accounting experience with the last twenty-two years servicing biotechnology, pharmaceutical, medical device and clinical research companies.  Prior to joining Clene as CFO, Mr. Brown served in a similar capacity at Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company; at Innovus Pharmaceuticals, Inc. (OTCBB: INNV), an emerging pharmaceuticals company; at World Heart Corporation (NASDAQ: WHRT), a medical device company; at Lifetree Clinical Research, a clinical research organization; and, at NPS Pharmaceuticals Inc. (NASDAQ: NPSP), a biotechnology company.  Previously he worked for KPMG LLP, an international public accounting firm, in Salt Lake City where he served as a Senior Audit Manager. In his roles prior to Clene, Mr. Brown has participated in and led efforts to successfully evaluate, negotiate, and close transactions including equity and debt financings totaling over $1 billion and out-licensing of commercial stage asset transactions totaling $220 million. Mr. Brown has experience in sale-leaseback arrangements, facilities construction, and M&A activities.  Mr. Brown is a licensed certified public accountant in the state of Utah. Mr. Brown earned a Master of Science degree in Business Administration from the University of Utah and a Bachelor of Science degree in Accounting from Utah State University.

Benjamin Greenberg, MD, MHS, FAAN

Head of Medical

Dr. Benjamin Greenberg is an internationally recognized expert in disorders of the central nervous system. He joins Clene alongside his current role as professor of neurology and Vice Chair of Clinical and Translational Research in the Department of Neurology at UT Southwestern Medical Center in Dallas, TX.

His clinical and research expertise spans many areas of neurology including the diagnosis and treatment of MS and other rare neurological disorders. Dr. Greenberg received his Bachelor’s degree from Johns Hopkins University and his Master’s degree in Molecular Microbiology and Immunology from the Johns Hopkins School of Public Health. He graduated from Baylor College of Medicine and completed an internal medicine internship at Rush Presbyterian-St. Luke’s Medical Center in Chicago, before going on to residency in neurology at The Johns Hopkins Hospital in Baltimore.

Rowena Green

Executive Director, Quality Assurance

Rowena Green joined Clene Nanomedicine, Inc. in 2015, with experience in the (bio)pharmaceutical, nutraceutical, medical device and environmental industries. With over twenty-three years of GxP experience, eighteen years are as a quality assurance representative, which confirms her role as Sr. Director of Quality Assurance. Prior to Clene, Rowena spent three years as a consultant at Meridian BioGroup. There, she supported multiple clients to ensure compliance to the appropriate regulations (i.e., GMPs, GLPs). Prior to being a consultant, Rowena worked with Johns Hopkins University as the Quality Manager of the Cell Processing and Gene Therapy Department, which manufactured clinical trial material for multiple JHU Investigators. With a Bachelor of Science Degree in Chemistry, Rowena has worked as a Manufacturing Operator to produce brain cancer treatment, developed clinical trial projects to manufacture API as a Research Associate in Chemical Development, and performed the data review for one of the largest MRSA clinical studies ever conducted as a Sr. Quality Assurance representative. Rowena’s experience in the various industries has led her to Clene, where she and her direct reports provide regulatory support and quality assurance oversight for at least seven on-going clinical studies.