Thank you to Lumos Pharma for the following update on the Vigilan Observational Study. We are excited for the progress that has been made in making this study a viable option for a larger portion of our community.
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Thank you to Lumos Pharma for providing the following information to ACD and our community. If you have any specific questions or would like to inquire about how you may be able to participate in the Vigilan study, please contact: firstname.lastname@example.org or email@example.com.
Lumos Pharma is hosting a webinar on February 8th @ 11am CT to help CTD families better understand:
To register for the webinar and/or to submit questions in advance, visit: Lumos Pharma Webinar Series
Lumos Pharma is sponsoring a multi-year observational clinical study of males with Creatine Transporter Deficiency (CTD) that is currently recruiting individuals to participate. ClinicalTrials.gov Listing:
This CTD observational study is an international, multiple site, study that intends to enroll 50 males with CTD over the next 12-16 months. The study’s initial clinical site is actively enrolling patients, and many additional clinical sites will start to enroll in the coming months.
To: Association for Creatine Deficiencies (ACD)
From : Carol Dutch, Senior Director, Patient Engagement
Date: Aug 2, 2016
Re: Q & A Statement regarding “cyclocreatine products” and explanation of pharmaceutical drug names
Thank you very much for your email, dated Aug 1st, posing the questions you have received from both your community along with the French advocacy group, Xtrodinaire. Below please find the two Q&A statements that you may share with your CTD community.
ACD Question: There are products containg cyclocreatine on the global market. Are they different from what Lumos Pharma is developing?
Lumos Pharma is aware that there are products being marketed that purportedly contain cyclocreatine. These products might be called “dietary supplements”. Lumos has tested, via reputable laboratories, some of these “cyclocreatine products” and found that some of the products do not contain cyclocreatine, or the amounts of cyclocreatine were negligible.
In the United States, dietary supplements are not subject to the same degree of regulatory scrutiny as pharmaceutical products. Importantly, dietary supplements may not necessarily contain what they claim to contain, and the purity of the substance is nearly always far inferior to that of a pharmaceutical product.
Dietary supplements: According to the FDA, dietary supplement manufacturers and distributors are not required to obtain approval from the FDA before marketing dietary supplements. Dietary supplement companies are responsible to ensure that the product is safe and that any claims made about the products are not false or misleading.
Pharmaceutical drugs: According to the FDA, pharmaceutical drugs are required to undergo regulated clinical development designed to meet safety and efficacy standards. They are also regulated by the FDA to prove that the drug contains the active principle under very high standards of quality assurance and consistency.
ACD Question: Can you explain the difference between the name cyclocreatine and the name LUM-001, the new drug that Lumos is developing?
In the course of developing a new pharmaceutical therapy, there might be four different types of names assigned: a chemical name, a company designated name, a generic name, and a brand name.
When a novel chemical or biological therapy is developed, a chemical name is given.Based on the compound’s chemical structure, the chemical name is a scientific name and is almost never used to identify the drug in a clinical setting. In our case, cyclocreatine’s proper chemical name is: (2-Amino-4,5-dihydro-1H-imidazol-1-yl) acetic acid. Cyclocreatine is a commonly used synonym to make it easier to communicate (2-Amino-4,5-dihydro-1H-imidazol-1-yl) acetic acid.
2. Company designated name
A company that is developing a new drug will give it a company codename to identify it within the company.For example, LUM-001, is Lumos Pharma’s company codename for the drug we are developing that contains cyclocreatine (2-Amino-4,5-dihydro-1H-imidazol-1-yl) acetic acid.
3. Generic name
The generic name, or International Non-proprietary Name (INN), is a commonly used, formal means to identify a drug during its useful clinical lifetime.INNs facilitate the identification of pharmaceutical substances or ingredients. Each INN is a unique name that is globally recognized and is public property. An INN is designated by the World Health Organization. LUM-001 does not yet have a generic name.
4. Brand name As the clinical development of LUM-001 progresses, Lumos will choose a brand name, trade name or trademark. Under trademark law this name is owned by the company which has exclusive rights to use it. Note that a drug may have more than one brand name. LUM-001 does not yet have a brand name.
Dear Friends of Lumos Pharma,
I’m taking this opportunity to bring you up-to-date on the progress of the development of our potential therapeutic for CTD patients.
For many of you in the CTD community, I know it is difficult to understand the time that it requires to satisfy regulatory bodies around the world so that our compound LUM-001 can safely enter the clinic.
We have been diligently working to validate and document our manufacturing process including establishing an easier, safer route of synthesis of LUM-001. We have transferred this process to a commercial contract manufacturer, and LUM-001 is now being produced on a much larger scale than we have previously made. This will provide enough compound to complete our non-clinical stage of testing and our clinical testing program. Importantly, a new dry-milled powder formulation has been developed that we anticipate will substantially reduce the amount of liquid required to be taken on a daily basis.
The US and European regulatory requirements for documentation of our manufacturing methods, reproducibility, analytical chemistries, control and other important prerequisites have been well-documented and prepared for our Pre-IND (Investigational New Drug) meeting with the FDA at the end of the first quarter next year.
Prior to administering any product to humans, toxicology studies are required. We have completed several of these studies and will be conducting additional non-clinical studies that will be ongoing with our clinical development plan. We are hopeful that by mid-year 2016, we will obtain regulatory approval to proceed into the clinic.
I know that for many of our stakeholders, most notably, our CTD families, this probably seems like an inordinate, protracted delay. However, the drug development process requires careful, thoughtful advancement with safety of patients first and foremost in our minds.
Lumos has added many part-time sub-contractors and expert advisors along the way including consultants with expertise in formulation, synthesis, analytical chemistry, scale-up manufacturing and clinical/regulatory drug development in the US, EU and Japan. We are attempting to build a breadth of important advisors to ensure our success to regulatory approvals around the world.
Sadly, Liza Squires, MD Lumos’ Chief Medical Officer has taken another position with a pharmaceutical company near her home in Philadelphia. She will be working with former colleagues at Shire within a larger, publicly-traded company while eliminating the commute to our headquarters in Austin. We wish her well in her new responsibilities. Nevertheless, Liza will continue as a consultant in order to maintain continuity in our plan.
In addition to having Liza continue as a consultant, we are working with other experienced, clinical consultants to help ensure that our clinical program continues as planned until we can find a full-time Chief Medical Officer.
I would also like to introduce you to Alex Bruchey, PhD who has joined us in Austin. She is a neurobiologist with a decade of drug development experience. As the Senior Project Manager, she will be overseeing both the Observational/CTD Natural History Study as well as the pivotal interventional study.
Chris Bemben has also joined Lumos. Chris is a chemist and seasoned project manager with an MBA background. He is managing the manufacturing portion of our plan including the scale-up production for our trials next year. In addition, Chris has oversight of our toxicology studies among other important non-clinical studies.
Stephen Ceresia is a relatively new member of the team. As office Manger, he is responsible for managing many administrative aspects of our day to day operations.
There is much work to be done. We look forward to interacting with you again soon. Your collective help and support is essential toward Lumos’ goal of providing a useful therapeutic for CTD patients around the world. We hope we can continue to count on your for your sage advice!
April 6th, 2015
Dear Association for Creatine Deficiencies Board Members and Friends,
As the CEO of Lumos Pharma, Allow me to personally congratulate the ACD for their important work in advocating for patients with Creatine Deficiencies.
The ACD’s efforts to raise awareness and organize the community are critical steps toward improving the lives of patients and families affected by these disorders. Additionally, the Association’s Patient Registry is a significant undertaking, which will provide critical information about Creatine Deficiencies to the community at large.
I would also like to share with you some information about Lumos Pharma, which was founded in 2011 with the sole mission of developing a therapeutic for Creatine Transporter Deficiency (CTD). Last January, we achieved a significant milestone when we completed our Series A financing round. These funds are being used to advance our development program through the preclinical stage (developing the pharmaceutical product, and the required safety testing in animals) and into the early clinical stage (safety and phamacokinetic testing healthy volunteers). Once these critical steps have been achieved, studies in CTD patients can begin.
We are fortunate to be partnered with the National Institutes of Health’s (NIH) Therapeutics for Rare and Neglected Diseases (TRND) program, which provides critical scientific support to accelerate early stage drug development. As part of the TRND program, the NIH is planning a Natural History Study of CTD in 2015. If you are interested in more information about the upcoming NIH study, please discuss with your physician. Lumos’ program has also been granted Orphan Drug Designation, which will streamline and accelerate the standard regulatory pathway and timelines.
The process of drug development is an arduous task that can be fraught with many challenges, therefore, we ask for your support and patience as we advance our development program. Lumos Pharma will endeavor to keep the Creatine Deficiency Community informed of our progress through the Association and communication with physicians involved in the care of patients with Creatine Transporter Deficiency.
I have put together a dedicated team of experienced professionals at Lumos Pharma and we all share your sense of urgency in bringing forward meaningful therapeutic options for those affected by CTD.
Thank you and best wishes,
Richard J. Hawkins
President and CEO
Lumos Pharma, Inc