– Recently secured funding bolsters cash runway to fund 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
– Ongoing 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
CHARLESTON, S.C., Sept. 16, 2020 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests, today provided a corporate update.
“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 (“U.S.”) under the tradename 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, multicenter and multinational study expected to enroll ~100 subjects worldwide, with at least 40 pre-pubertal and 40 pubertal subjects and a minimum of 25 subjects expected to be enrolled in the USA. Enrolled subjects in the study will be children and adolescents from 2 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 to two standard GHSTs as controls, arginine (i.v.) and clonidine (p.o.). 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 ongoing 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 US. 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 re-purposed 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 Inc. 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 license 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. For more information, please visit www.zentaris.com and connect with the Company on Twitter, LinkedIn and Facebook.
This press release contains forward-looking statements (as defined by applicable securities legislation) made pursuant to the safe-harbor provision of the U.S. Securities Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements include those relating to the Company obtaining approval of macimorelin for CGHD and the resulting potential to significantly increase the available patient population for macimorelin, the Company’s ability to to secure marketing partners for macimorelin for GHD in Europe and elsewhere, the commencement of the CGHD Study P02, the ability of the Company to identify and develop therapeutic uses for macimorelin in new indications and the ability of the Company to expand its pipeline of products, and may include, but are not limited to statements preceded by, followed by, or that include the words "will," "expects," "believes," "intends," "would," "could," "may," "anticipates," and similar terms that relate to future events, performance, or our results. Forward-looking statements involve known and unknown risks and uncertainties, including those discussed in this press release and in our Annual Report on Form 20-F, under the caption "Key Information - Risk Factors" filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form and with the U.S. Securities and Exchange Commission. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in forward-looking statements. Such risks and uncertainties include, among others, our ability to raise capital and obtain financing to continue our currently planned operations, our ability to continue to list our Common Shares on the NASDAQ, our now heavy dependence on the success of Macrilen™ (macimorelin) and related out-licensing arrangements and the continued availability of funds and resources to successfully commercialize the product, including our heavy reliance on the success of the License Agreement with Novo, the global instability due to the global pandemic of COVID-19, and its unknown potential effect on our planned operations, including studies, our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect, our reliance on third parties for the manufacturing and commercialization of Macrilen™ (macimorelin), potential disputes with third parties, leading to delays in or termination of the manufacturing, development, out-licensing or commercialization of our product candidates, or resulting in significant litigation or arbitration, uncertainties related to the regulatory process, unforeseen global instability, including the instability due to the global pandemic of the novel coronavirus, our ability to efficiently commercialize or out-license Macrilen™ (macimorelin), our reliance on the success of the pediatric clinical trial in the European Union (“E.U.”) and U.S. for Macrilen™ (macimorelin), the degree of market acceptance of Macrilen™ (macimorelin), our ability to obtain necessary approvals from the relevant regulatory authorities to enable us to use the desired brand names for our product, our ability to successfully negotiate pricing and reimbursement in key markets in the E.U. for Macrilen™ (macimorelin), any evaluation of potential strategic alternatives to maximize potential future growth and shareholder value may not result in any such alternative being pursued, and even if pursued, may not result in the anticipated benefits, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect our intellectual property, and the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law.