Multicenter, Open Label Trial to Investigate the Efficacy and Safety of a Single Oral Dose of 1.0 mg/kg Macimorelin Acetate as Growth Hormone Stimulation Test (GHST) in Pediatric Patients With Suspected Growth Hormone Deficiency (GHD)

This research study will find out if a new growth hormone stimulation test is safe and works as well as other tests to diagnose growth hormone deficiency (GHD) in children. The stimulation test will use a new growth hormone stimulating substance called macimorelin. By now, only adults in the USA can get this new stimulation test. The results of this study are expected to help children and teenagers with suspected GHD to get the macimorelin stimulation test.
The macimorelin test will be compared to a clonidine and an arginine test. Both are known standard stimulation tests. Altogether two macimorelin tests are planned to be performed in the study, to show how repeatable macimorelin tests results are (under a set of similar conditions).

Start Date16 Nov 2021

Sponsor / Collaborator

Æterna Zentaris, Inc.

EUCTR2018-001988-23-PL / Not yet recruitingPhase 2

Open label, group comparison, dose escalation trial to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of macimorelin acetate after single oral dosing of 0.25 mg/kg, 0.5 mg/kg, and 1 mg/kg in pediatric patients with suspected growth hormone deficiency (GHD)

Start Date19 Apr 2019

Sponsor / Collaborator

AEterna Zentaris GmbH

NCT02558829 / CompletedPhase 3

Confirmatory Validation of Oral Macimorelin as a Growth Hormone (GH) Stimulation Test (ST) for the Diagnosis of Adult Growth Hormone Deficiency (AGHD) in Comparison With the Insulin Tolerance Test (ITT)

The Macimorelin Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, 2-way crossover Trial. The trial will include subjects suspected to have adult growth hormone deficiency (AGHD) and a group of healthy control subjects.

Start Date03 Dec 2015

Sponsor / Collaborator

Æterna Zentaris, Inc.

100 Clinical Results associated with Macimorelin Acetate

100 Translational Medicine associated with Macimorelin Acetate

100 Patents (Medical) associated with Macimorelin Acetate

Literatures (Medical) associated with Macimorelin Acetate

04 Jul 2023·Diagnostics (Basel, Switzerland)

Contribution of Dynamic and Genetic Tests for Short Stature Diagnosing: A Case Report.

Author: Biagetti, Betina ; Díaz-Troyano, Noelia ; Iniesta-Serrano, Emilio ; Campos-Martorell, Ariadna ; Valenzuela, Irene ; Giralt, Marina ; Hernandez, Sara ; Campos, Berta ; Simó, Rafael ; Yeste, Diego

Genetic tests have led to the discovery of many novel genetic variants related to growth failure, but the clinical significance of some results is not always easy to establish. The aim of this report is to describe both clinical phenotype and genetic characteristics in an adult patient with short stature associated with a homozygous variant in disintegrin and metalloproteinase with thrombospondin motifs type 17 gene (ADAMTS17) combined with a homozygous variant in the GH secretagogue receptor (GHS-R). The index case had severe short stature (SS) (-3.0 SD), small hands and feet, associated with eye disturbances. Genetic tests revealed homozygous compounds for ADAMTS17 responsible for Weill-Marchesani-like syndrome but a homozygous variant in GHS-R was also detected. Dynamic stimulation with an insulin tolerance test showed a normal elevation of GH, while the GH response to macimorelin stimulus was totally flattened. We show the implication of the GHS-R variant and review the molecular mechanisms of both entities. These results allowed us to better interpret the phenotypic spectrum, associated co-morbidities, its implications in dynamic tests, genetic counselling and treatment options not only to the index case but also for her relatives.

01 Apr 2023·Journal of cachexia, sarcopenia and muscle

Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia

Article

Author: Herodes, Megan ; Shober, Samuel ; Marcelli, Marco ; Ammer, Nicola ; Graf, Solomon A ; Schur, Ellen A ; Anderson, Lindsey J ; Garcia, Jose M ; Salas, Ramiro

Abstract:

Background:

Cancer cachexia is associated with reduced body weight, appetite and quality of life (QOL) with no approved treatments. Growth hormone secretagogues like macimorelin have potential to mitigate these effects.

Methods:

This pilot study assessed the safety and efficacy of macimorelin for 1 week. Efficacy was defined a priori as 1‐week change in body weight (≥0.8 kg), plasma insulin‐like growth factor (IGF)‐1 (≥50 ng/mL) or QOL (≥15%). Secondary outcomes included food intake, appetite, functional performance, energy expenditure and safety laboratory parameters. Patients with cancer cachexia were randomized to 0.5 or 1.0 mg/kg macimorelin or placebo; outcomes were assessed non‐parametrically.

Results:

Participants receiving at least one of either macimorelin dose were combined (N = 10; 100% male; median age = 65.50 ± 2.12) and compared with placebo (N = 5; 80% male; median age = 68.00 ± 6.19). Efficacy criteria achieved: body weight (macimorelin N = 2; placebo N = 0; P = 0.92); IGF‐1 (macimorelin N = 0; placebo N = 0); QOL by Anderson Symptom Assessment Scale (macimorelin N = 4; placebo N = 1; P = 1.00) or Functional Assessment of Chronic Illness Therapy‐Fatigue (FACIT‐F; macimorelin N = 3; placebo N = 0; P = 0.50). No related serious or non‐serious adverse events were reported. In macimorelin recipients, change in FACIT‐F was directly associated with change in body weight (r = 0.92, P = 0.001), IGF‐1 (r = 0.80, P = 0.01), and caloric intake (r = 0.83, P = 0.005), and inversely associated with change in energy expenditure (r = −0.67, P = 0.05).

Conclusions:

Daily oral macimorelin for 1 week was safe and numerically improved body weight and QOL in patients with cancer cachexia compared with placebo. Longer term administration should be evaluated for mitigation of cancer‐induced reductions in body weight, appetite and QOL in larger studies.

01 Aug 2022·Pituitary

The effect of glucose dynamics on plasma copeptin levels upon glucagon, arginine, and macimorelin stimulation in healthy adults

Article

Author: Atila, Cihan ; Refardt, Julie ; Monnerat, Sophie ; Christ-Crain, Mirjam ; Urwyler, Sandrine Andrea ; Winzeler, Bettina

Abstract:

Purpose:

Non-osmotic stimulation tests using glucagon, arginine, or macimorelin were recently evaluated for their ability to assess posterior pituitary function. Glucagon and arginine, but not macimorelin, stimulated copeptin secretion (a surrogate marker of vasopressin) and, therefore, provide novel tests to assess the posterior pituitary. The exact underlying mechanism behind their stimulatory effect remains elusive.

Methods:

This analysis combined data from three diagnostic studies conducted at the University Hospital Basel, Switzerland. In total, 80 healthy adults underwent the glucagon (n = 22), arginine (n = 30), or macimorelin (n = 28) stimulation tests. The primary objective was to investigate glucose course upon glucagon, arginine, and macimorelin stimulation tests and its effect on plasma copeptin release.

Results:

Upon glucagon stimulation, the median [IQR] glucose level at baseline was 5.0 [4.6, 5.2] mmol/l, peaked at 8.1 [7.2, 9.4] mmol/l after 30 min and decreased to a minimum of 3.8 [3.5, 4.5] mmol/l after 120 min. The median copeptin increase upon glucagon stimulation was 7.7 [2.6, 28.0] pmol/l. Upon arginine, the glucose level at baseline was 4.9 [4.8, 5.5] mmol/l, peaked at 6.0 [5.2, 6.4] mmol/l after 30 min and decreased to a minimum of 4.3 [3.8, 4.8] mmol/l after 60 min. The median copeptin increase upon arginine stimulation was 4.5 [2.9, 7.5] pmol/l. Upon macimorelin, glucose levels showed no notable dynamics over the 120 min, and no major change in copeptin was observed.In the pooled dataset, a decrease in glucose levels was significantly correlated with copeptin increase (ρ = 0.53, p < 0.01).

Conclusion:

A similar course in plasma glucose was observed in the copeptin-stimulating test, i.e., after glucagon and arginine, while macimorelin had no effect on glucose and copeptin levels. We hypothesize that a drop in glucose levels observed upon glucagon and arginine might stimulate copeptin.

News (Medical) associated with Macimorelin Acetate

13 Jun 2024

·www.globenewswire.com

Aeterna Zentaris Announces Last Patient Last Visit in Pivotal DETECT-Trial for the Diagnosis of Childhood-Onset Growth Hormone Deficiency

Company expects to report top-line data and complete study results in Q3 2024 TORONTO, ONTARIO, June 13, 2024 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products and active ingredients for healthcare and cosmetics industries, today announced the clinical completion of its Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD"). The Company confirms that the last patient conducted the last study visit successfully in Europe on June 13, 2024. The DETECT-trial is a multicenter, open-label trial to investigate the efficacy and safety of a single oral dose of 1.0 mg/kg macimorelin acetate as growth hormone stimulation test (“GHST”) in pediatric patients with suspected growth hormone deficiency ("GHD"). The study enrolled 100 subjects in Europe and North America. The Company remains on track to report top-line data for the DETECT-trial in Q3 2024.Gilles Gagnon, Chief Executive Officer of the Company commented, “The completion of the DETECT-trial represents a major milestone for the Company. In addition to the already approved indication for adults, the successful development of macimorelin as a unique diagnostic tool for the detection of growth hormone deficiency in children would represent a major benefit for patients while significantly expanding this market in endocrinology. On behalf of the Company, I would like to thank the patients and clinical staff for their continued efforts to bring this study across the finish line as well as our shareholders for their continued support. Looking ahead, we are eagerly anticipating the presentation of the top-line data as well as the full study results in the third quarter of this year.” The DETECT-trial is the second and final study needed evaluating the potential use of macimorelin as diagnostic test in children as agreed upon with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the Company's Pediatric Investigation Plan. For more information about the DETECT-trial, please visit EU Clinical Trials Register and reference EudraCT #2018-001988-23 and clinicaltrials.gov identifier NCT04786873.About Macimorelin (Macrilen®; GHRYVELIN™) Macimorelin, an oral drug used for the diagnosis of adult growth hormone deficiency (AGHD) is approved for marketing under the brand name GHRYVELIN™ in the European Economic Area and Macrilen® in the United States, South Korea and Israel. Macimorelin (Macrilen®; GHRYVELIN™), a ghrelin receptor agonist, is an orally administered peptidomimetic molecule that stimulates the secretion of growth hormone from the pituitary gland. Stimulated growth hormone levels are measured in blood samples taken after oral administration of macimorelin for the assessment of AGHD. Approval of macimorelin for use in adult was granted by the FDA in 2017 and by the EMEA in 2019, based on Phase III data showing that oral macimorelin provides accuracy comparable to that of standard insulin tolerance testing (ITT), but has a more favorable safety profile compared to ITT. Oral macimorelin also reduces false positive test results, helping to avoid unnecessary treatment of patients. About Aeterna Zentaris Inc. Aeterna is a specialty biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of pharmaceutical and diagnostic products, including those focused on areas of significant unmet medical need. One of Aeterna’s lead products is macimorelin (Macrilen; Ghryvelin), the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Aeterna is also engaged in the development of therapeutic assets and proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt’s Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol “AEZS”. For more information, please visit Aeterna’s website at www.zentaris.com. Forward-Looking Statements The information in this news release has been prepared as of June 13, 2024. Certain statements in this news release, referred to herein as "forward-looking statements", constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended, and "forward-looking information" under the provisions of Canadian securities laws. All statements, other than statements of historical fact, that address circumstances, events, activities, or developments that could or may or will occur are forward-looking statements. When used in this news release, words such as "anticipate", "assume", "believe", "could", "expect", "forecast", "future", "goal", "guidance", "intend", "likely", "may", "would" or the negative or comparable terminology as well as terms usually used in the future and the conditional are generally intended to identify forward-looking statements, although not all forward-looking statements include such words. Forward-looking statements in this news release include, but are not limited to, statements relating to: the timing of the DETECT-trial, including timing of top-line data and full study results; and the development of macimorelin as a diagnostic test for CGHD. Forward-looking statements are necessarily based upon a number of factors and assumptions that, while considered reasonable by the Company as of the date of such statements, are inherently subject to significant business, economic, operational and other risks, uncertainties, contingencies and other factors, including those described below, which could cause actual results, performance or achievements of the combined Company to be materially different from results, performance or achievements expressed or implied by such forward-looking statements and, as such, undue reliance must not be placed on them. Forward-looking statements involve known and unknown risks and uncertainties which include, among others: the combined Company’s present and future business strategies; operations and performance within expected ranges; anticipated future cash flows; local and global economic conditions and the environment in which the combined Company operates; anticipated capital and operating costs; uncertainty in product development and related clinical trials and validation studies, including our reliance on the success of the pediatric clinical trial in the European Union and U.S. for Macrilen™ (macimorelin); the commencement of the DETECT-trial may be delayed or we may not obtain regulatory approval to initiate that study; we may be unable to enroll the expected number of subjects in the DETECT-trial and the result of the DETECT-trial may not support receipt of regulatory approval in child-onset growth hormone deficiency; results from ongoing or planned pre-clinical studies of macimorelin by the University of Queensland or for our other products under development may not be successful or may not support advancing the product to human clinical trials; our ability to raise capital and obtain financing to continue our currently planned operations; 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; the ability to secure strategic partners for late stage development, marketing, and distribution of our products, including our ability to enter into a new license agreement or similar arrangement following the termination of the license agreement with Novo Nordisk AG; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to protect and enforce our patent portfolio and intellectual property; and our ability to continue to list our common shares on the NASDAQ Capital Market. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those discussed in our Annual Report on Form 20-F and MD&A filed under the Company’s profile on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov. We disclaim any obligation to update any such risks or uncertainties 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. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this news release.Investor Contact:Jenene ThomasJTC TeamT (US): +1 (833) 475-8247E: aezs@jtcir.com

Phase 3Drug Approval

03 Jun 2024

·www.globenewswire.com

Aeterna Zentaris and Ceapro Complete Merger Transaction

TORONTO and EDMONTON, Alberta, June 03, 2024 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”) and Ceapro Inc. (TSX-V: CZO) (OTCQX: CRPOF) (“Ceapro”), two innovative biopharmaceutical development companies, are pleased to announce the successful completion and closing of their all-stock merger of equals transaction (the “Transaction”), which was previously announced by Aeterna and Ceapro in their joint press release of December 14, 2023. “This is an important day for shareholders of both companies as Aeterna and Ceapro have now officially come together to create a diversified business that is expected to create value for many years to come,” said Ronald W. Miller, Chair of the Company. “With the successful completion of this merger, we are now optimized to bring value-driving, transformational products to the market.” Gilles Gagnon, Chief Executive Officer of the Company, said: “with the shared benefits, additional competencies and resources resulting from this merger, we are now poised to push forward exciting development programs in selected areas while continuing to grow our revenue generating base business and constantly looking for strategic growth opportunities.” “We would like to thank Aeterna shareholders for their support for this transaction,” said Carolyn Egbert, former Chair of the Company. “We also look forward to working with the Ceapro team to build a long-term, sustainable business.” Greater potential for stable cash flow to support R&D of potentially higher return pharmaceutical products. The Company currently generates revenues from two main active ingredients, oat beta glucan and avenanthramides, extracted and purified using its proprietary technology. Cash from these products is planned to be used along with the Company’s revenue from the commercialization or licensing of its macimorelin product to support the development of exciting, high potential-return products, ideally creating growing and sustainable revenue for the Company and investors.Greater diversification of commercial and development product pipeline lowers risk. The Company is expected to benefit from an extensive and diversified pipeline of innovative products in development, including quicker-to-market biotechnology products and exciting potentially higher return, but longer-horizon, products. With this pipeline rejuvenation, the Company is anticipated to boast: more products in the pipeline that are closer to potential commercialization;an enhanced ability to strategically focus financial and company resources in a manner that provides the most value to the company and shareholders; anda more compelling value proposition and lower risk profile. Expanded pharmaceutical research and development capabilities. The Company’s talented team brings deep expertise and knowledge that are expected to play a key role in advancing the Company and its development pipeline. The Company has the infrastructure to support development activities and potentially offer improved efficiencies, in addition to cost savings. It now also has an expanded development pipeline of products which its leadership is committed to prioritizing as they evaluate what will provide the best overall potential for the Company, shareholders, and consumers.Compelling North American + European combination. The Company now has an operational presence in North America and Europe. While the Company expects to continue to maintain some presence in Europe, its leadership believes that it needs to re-focus operations within the North American biotechnology market, to provide optimal exposure to potential new investors, business development opportunities and talent.Expertise and efficiencies. The Company can now leverage the combined experience and expertise of its predecessor companies, including navigating the conduct of human clinical trials and the crucial regulatory approval process required to bring pharmaceutical products to market. The Company plans to leverage this expertise with the higher value pharmaceutical opportunities being advanced for its active ingredients and technologies. Following the closing of the Transaction, Aeterna’s board of directors now consists of eight directors: Ronald W. Miller (Chair), Carolyn Egbert, Gilles Gagnon, Ulrich Kosciessa, Geneviève Foster, William Li, Dennis Turpin and Peter Edwards. The executive leadership team now consists of Gilles Gagnon, President and Chief Executive Officer, and Giuliano La Fratta, Senior Vice President and Chief Financial Officer. A new name for the combined company is expected to be announced in the coming weeks and will be put forward for shareholder approval at the upcoming annual meeting of shareholders, details of which are expected to be announced shortly. Full details of the Transaction and certain other matters are set out in the respective information circulars filed by Aeterna and Ceapro which are available under each company’s profile on SEDAR+ at www.sedarplus.ca or, as regards Aeterna, its reports on Form 6-K and other filings on EDGAR at www.sec.gov. Aeterna is an “Eligible Interlisted Issuer” as such term is defined in the TSX Company Manual. As an Eligible Interlisted Issuer, the Company has relied on an exemption pursuant to Section 602.1 of the TSX Company Manual, the effect of which is that the Company was not required to comply with certain requirements relating to the issuance of securities in connection with the Transaction. Information for Ceapro Shareholders The shares of Ceapro are expected to be delisted from the TSX Venture Exchange within five business days. Ceapro is also in the process of applying to cease to be a reporting issuer under applicable Canadian securities laws. Pursuant to the Transaction, former Ceapro shareholders are entitled to receive 0.02360 of an Aeterna Zentaris share for each Ceapro share held. In order to receive Aeterna Zentaris shares in exchange for Ceapro shares, Ceapro registered shareholders must complete, sign, date and return (together with the certificate or DRS statement representing their shares) the letter of transmittal that was mailed to them prior to closing of the Transaction. The letter of transmittal is also available under Ceapro’s profile on SEDAR+ at www.sedarplus.ca and by contacting Computershare Investor Services Inc., the depositary, by telephone at 1-514-982-7555 or toll-free in North America at 1-800-564-6253 or by email at corporateactions@computershare.com. For those shareholders of Ceapro whose shares are registered in the name of a broker, investment dealer, bank, trust company or other intermediary or nominee, they should contact such intermediary or nominee for assistance in depositing their Ceapro shares and should follow the instructions of such intermediary or nominee. About Aeterna Zentaris Inc. Aeterna is a specialty biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of pharmaceutical and diagnostic products, including those focused on areas of significant unmet medical need. One of Aeterna’s lead products is macimorelin (Macrilen; Ghryvelin), the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Aeterna is also engaged in the development of therapeutic assets and proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt’s Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol “AEZS”. For more information, please visit Aeterna’s website at www.zentaris.com. Forward-Looking Statements The information in this news release has been prepared as of June 3, 2024. Certain statements in this news release, referred to herein as "forward-looking statements", constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended, and "forward-looking information" under the provisions of Canadian securities laws. All statements, other than statements of historical fact, that address circumstances, events, activities, or developments that could or may or will occur are forward-looking statements. When used in this news release, words such as "anticipate", "assume", "believe", "could", "expect", "forecast", "future", "goal", "guidance", "intend", "likely", "may", "would" or the negative or comparable terminology as well as terms usually used in the future and the conditional are generally intended to identify forward-looking statements, although not all forward-looking statements include such words. Forward-looking statements in this news release include, but are not limited to, statements relating to: the future business and operations of the combined Company, including cash flow, research and development, and costs. Forward-looking statements are necessarily based upon a number of factors and assumptions that, while considered reasonable by the Company as of the date of such statements, are inherently subject to significant business, economic, operational and other risks, uncertainties, contingencies and other factors, including those described below, which could cause actual results, performance or achievements of the combined Company to be materially different from results, performance or achievements expressed or implied by such forward-looking statements and, as such, undue reliance must not be placed on them. Forward-looking statements involve known and unknown risks and uncertainties which include, among others: the combined Company’s present and future business strategies; operations and performance within expected ranges; anticipated future cash flows; local and global economic conditions and the environment in which the combined Company operates; anticipated capital and operating costs; uncertainty in product development and related clinical trials and validation studies, including our reliance on the success of the pediatric clinical trial in the European Union and U.S. for Macrilen™ (macimorelin); the commencement of the DETECT-trial may be delayed or we may not obtain regulatory approval to initiate that study; we may be unable to enroll the expected number of subjects in the DETECT-trial and the result of the DETECT-trial may not support receipt of regulatory approval in child-onset growth hormone deficiency; results from ongoing or planned pre-clinical studies of macimorelin by the University of Queensland or for our other products under development may not be successful or may not support advancing the product to human clinical trials; our ability to raise capital and obtain financing to continue our currently planned operations; 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; the ability to secure strategic partners for late stage development, marketing, and distribution of our products, including our ability to enter into a new license agreement or similar arrangement following the termination of the license agreement with Novo Nordisk AG; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to protect and enforce our patent portfolio and intellectual property; and our ability to continue to list our common shares on the NASDAQ Capital Market. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those discussed in our Annual Report on Form 20-F and MD&A filed under the Company’s profile on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov. We disclaim any obligation to update any such risks or uncertainties 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. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this news release. For Further Information Aeterna Investor Contact:Aeterna, Investor RelationsAZinfo@aezsinc.com +1 843-900-3223 Aeterna Media Contact:Joel ShafferFGS Longviewjoel.shaffer@fgslongview.com 416-670-6468

License out/in

29 May 2024

·www.globenewswire.com

Aeterna Zentaris Provides Update on Timing for Annual Meeting of Shareholders and Due Bill Redemption Date

TORONTO, ONTARIO, May 29, 2024 (GLOBE NEWSWIRE) -- Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Company”), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, today announced that it is making an application for an order pursuant to section 133(3) of the Canada Business Corporations Act extending the time for the Company to call and hold its annual meeting of its shareholders to a date that is not later than July 31, 2024 (the “Application”). The Application is scheduled to be heard before a judge of the Ontario Superior Court of Justice, Commercial List (the “Court”) on June 3, 2024 at 11:00 a.m. via videoconference. There can be no assurance that the order sought by Aeterna will be granted. In support of the Application, the Company will file an application record, a factum and a draft order with the Court. These materials will also be available on the Company’s website at www.zentaris.com. Any shareholder that wishes to attend the hearing may request a link by emailing the Company at AZinfo@aezsinc.com. Aeterna is seeking the extension from the Court to provide sufficient time to complete the previously announced transaction (the “Transaction”) with Ceapro Inc. (“Ceapro”) in advance of the annual meeting so as to permit former Ceapro shareholders to attend and vote at the annual meeting. At the annual meeting, shareholders will be asked to vote on the new name and director nominees for the combined company. As previously disclosed, subject to obtaining all required approvals and satisfying all required conditions, the Transaction is expected to close on or about June 3, 2024. The Toronto Stock Exchange has also granted the Company an extension permitting the Company to hold its annual meeting on or before July 31, 2024. The Company would also like to clarify that the redemption date for the due bills attaching to the Company’s common shares as a result of the previously announced warrant issuance is June 3, 2024. About Aeterna Zentaris Inc. Aeterna is a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products focused on areas of significant unmet medical need. Aeterna's lead product, macimorelin (Macrilen; Ghryvelin), is the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Aeterna is leveraging the clinical success and compelling safety profile of macimorelin to develop it for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need. Aeterna is also dedicated to the development of its therapeutic assets and has established a pre-clinical development pipeline to potentially address unmet medical needs across a number of indications, including neuromyelitis optica spectrum disorder (NMOSD), Parkinson's disease (PD), hypoparathyroidism and amyotrophic lateral sclerosis (ALS; Lou Gehrig's disease). For more information, please visit www.zentaris.com and connect with the Company on Twitter, LinkedIn and Facebook. Forward-Looking Statements This press release contains statements that may constitute forward-looking statements within the meaning of U.S. and Canadian securities legislation and regulations, and such statements are made pursuant to the safe-harbor provision of the U.S. Securities Litigation Reform Act of 1995. Forward-looking statements are frequently, but not always, identified by words such as "expects," "aiming", "anticipates," "believes," "intends," "potential," "possible," and similar expressions. Such statements, based as they are on current expectations of management, inherently involve numerous risks, uncertainty and assumptions, known and unknown, many of which are beyond our control. Forward-looking statements in this press release include, but are not limited to, those relating to Aeterna's expectations regarding: the potential obtaining of an order from the Court via the Application; the timing for the Company’s annual meeting; the timing and location for the hearing of the Application; the ability of Aeterna and Ceapro to complete the Transaction on the terms described herein, or at all; the anticipated timeline for the completion of the Transaction; and receipt of final regulatory and stock exchange approvals with respect to the Transaction (including approval of the continued listing of the Common Shares on the Nasdaq and the TSX). Forward-looking statements involve known and unknown risks and uncertainties, and other factors which may cause the actual results, performance or achievements stated herein to be materially different from any future results, performance or achievements expressed or implied by the forward-looking information. Such risks and uncertainties include, among others, our reliance on the success of the DETECT clinical trial in the European Union and U.S. for Macrilen™ (macimorelin) in CGHD; results from our ongoing or planned pre-clinical studies and our DETECT clinical trial under development may not be successful or may not support advancing the product further in pre-clinical studies, to human clinical trials or regulatory approval; our ability to raise capital and obtain financing to continue our currently planned operations; 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; the global instability due to the global pandemic of COVID-19 and the war in the Ukraine, and their unknown potential effect on our planned operations; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to continue to list our common shares on the NASDAQ; and the availability and timing of required stock exchange, regulatory and other approvals for the completion of the Transaction with Ceapro. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those risks discussed in our Annual Report on Form 20-F under the caption "Risk Factors". Given the 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. For a more detailed discussion of such risks and other factors that may affect Aeterna’s and ability to achieve the expectations set forth in the forward-looking statements contained in this news release, see Aeterna’s Annual Report on Form 20-F and MD&A filed under Aeterna’s profile on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov, as well as Aeterna’s other filings with the Canadian securities regulators and the Securities and Exchange Commission. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this release. Investor Contact: Investor RelationsAZinfo@aezsinc.com +1 843-900-3223

100 Deals associated with Macimorelin Acetate

External Link

KEGG	Wiki	ATC	Drug Bank
D10562	Macimorelin Acetate	V04CD06	DB13074

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Growth hormone deficiency	US	Novo Nordisk, Inc.	20 Dec 2017

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	US	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	AT	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	FR	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	DE	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	IT	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	PL	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	RS	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	ES	Æterna Zentaris, Inc.	03 Dec 2015
Growth Hormone Deficiency With Pituitary Anomalies	Phase 3	GB	Æterna Zentaris, Inc.	03 Dec 2015
Cachexia	Phase 2	US	Æterna Zentaris, Inc.	01 May 2012

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01614990 (CTgov)	Phase 2	Cachexia	15	Macimorelin (Macimorelin)	otxoawepvh(aizknvfwrb) = cubojtyzuv hjqkaltfza (epimgbbemj, nbxsrylpvv - jwutoguqto) View more	-	19 Mar 2024
				Placebo (Placebo)	otxoawepvh(aizknvfwrb) = jhxynvubli hjqkaltfza (epimgbbemj, scmzfokwmn - xkdrhfwqpl) View more
NCT03844217 (Pubmed \| Pituitary)	Not Applicable	-	28	Macimorelin 0.5 mg/kg	bhnwkpiogc(mblzfkzsme) = fdjuhgxuam lynezumrsd (dpsgxbafmk, 56.5; 110.96) View more	-	22 Feb 2021
NCT02558829 (Pubmed \| J Clin Endocrinol Metab)	Phase 3	Dwarfism, Pituitary	157	Macimorelin	wlpljscfoc(dhvoegfmhx) = yzjxhqleot qjnivuyexo (pxsjlvdhvx )	-	01 Aug 2018
				Macimorelin (Insulin tolerance test (ITT))	wlpljscfoc(dhvoegfmhx) = mnsoizigzi qjnivuyexo (pxsjlvdhvx )
Pubmed \| J Clin Endocrinol Metab	Not Applicable	Dwarfism, Pituitary	-	Macimorelin	vsnmjgjloa(iyebwwtbxc) = qhgiwaxtho bljgqbabta (jgczkldeot )	-	01 Jun 2013
				Arginine+GHRH	vsnmjgjloa(iyebwwtbxc) = xxwtkayfdv bljgqbabta (jgczkldeot )

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis