Dr. Klaus Paulini reports
AETERNA ZENTARIS PROVIDES CORPORATE UPDATE
Aeterna Zentaris Inc. has provided a corporate update.
Recently secured financing bolsters cash runway to finance operations and provides significant optionality for growth.
- Pivotal phase 3 safety and efficacy study AEZS-130-P02 (study P02) expected to commence in Q1 2021.
- Continuing evaluations to expand pipeline seeking to transform into company with multiple assets in development.
- Efforts to secure a commercialization partner for macimorelin in Europe and other key global markets continuing.
"Over the course of the last year since my appointment as CEO, a tremendous amount has been achieved. With the set intention to first assess and determine how best to support our current development partnership and how to maximize its success, followed by pursuing the expansion of macimorelin into pediatric use, I believe we are now well positioned to advance Aeterna into its next phase of growth," commented
Dr. Klaus Paulini, chief executive officer of Aeterna. "A catalyst that has significantly powered the company with the momentum we have today was successfully raising a total of $23.5-million since the beginning of this year. By strategically and opportunistically securing this capital, we believe we have both a solid foundation to support optionality moving forward and equipped the company with necessary resources to enhance our pipeline in a meaningful way.
"Our team is first and foremost focused on continuing our efforts to bring the expanded use of macimorelin over the finish line for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need and, if approved, has the potential to significantly increase the available patient population for macimorelin. We are actively preparing, and hope to be able to commence, our pivotal phase 3 safety and efficacy study AEZS-130-P02 (study P02) in the first quarter of 2021," added Dr. Paulini.
Macimorelin clinical program update
The company's lead product,
macimorelin, is the only United States Food and Drug Administration-(FDA)-approved oral drug indicated for the diagnosis of AGHD and is currently marketed in the United States under the trade name Macrilen by Novo Nordisk. Aeterna is currently developing macimorelin for the diagnosis of CGHD, an area of significant unmet need, in collaboration with Novo Nordisk.
Results from the AEZS-130-P01 (study P01), the first of two studies as agreed with the European Medicines Agency (EMA) in the company's pediatric investigation plan (PIP) for macimorelin, were announced in April, 2020. The positive dose-finding results from study P01 provide the clinical framework for study P02 and establish a dose that could both be safely administered to pediatric patients and cause a clear rise in growth hormone concentration in subjects ultimately diagnosed as not having GHD.
Study P02 is planned as open-label, single dose, multicentre and multinational study expected to enroll about 100 subjects worldwide, with at least 40 prepubertal and 40 pubertal subjects, and a minimum of 25 subjects expected to be enrolled in the U.S. Enrolled subjects in the study will be children and adolescents from two years of age to less than 18 years of age with suspected GHD. Enrolled subjects in study P02 will have macimorelin GHST performed twice (for repeatability data) in comparison with two standard GHSTs as controls, arginine (IV) and clonidine (PO). The study design is suitable to support a claim for potential stand-alone testing, if successful.
Aeterna has also begun exploring the potential therapeutic use of macimorelin in various other indications. The company has continuing plans to evaluate the development of alternative formulations or administration routes with the goal of ensuring sufficient bioavailability and expects to be providing updates on its progress as results become available over the course of the next several months.
In addition to advancing its pediatric program, Aeterna is intensifying its business development activities with the goal of securing marketing partners for macimorelin for the diagnosis of GHD in Europe and other key markets.
Pipeline expansion opportunities
Aeterna Zentaris intends to balance risks and secure growth opportunities by re-establishing a diversified, yet focused, development pipeline to which the company can best leverage its expertise and experience. The company is focused on opportunistically utilizing its well-established, extensive network with universities in Europe and the U.S. This strong network of strategic collaborations with universities, provides what the company believes will be vital access to innovative development candidates in different indications, with a focus on rare or orphan indications and potential for pediatric use.
Dr. Paulini concluded: "Aeterna Zentaris was established with strong expertise in research and development, which I believe is an incredible asset to the company and one that we plan to leverage moving forward. Apart from investigating opportunities for new therapeutic usages of macimorelin, we are assessing whether development candidates from our previous programs may be repurposed for alternative indications based on prior key findings from data already available to us. With our capabilities and specific know-how, we believe we are well positioned as we explore these opportunities and consider potential co-development strategies that could be synergistic for the company moving forward."
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests. The company's lead product, Macrilen (macimorelin), is the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Macrilen is currently marketed in the United States through a licence agreement with Novo Nordisk and Aeterna Zentaris receives double-digit royalties on sales. Aeterna Zentaris owns all rights to macimorelin outside of the U.S. and Canada.
Aeterna Zentaris is also leveraging the clinical success and compelling safety profile of macimorelin to develop it for the diagnosis of child-onset growth hormone deficiency (CGHD), an area of significant unmet need.
The company is actively pursuing business development opportunities for the commercialization of macimorelin in Europe and the rest of the world, in addition to other non-strategic assets to monetize their value.
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