- Aeterna Zentaris focused on accelerating recruitment for ongoing DETECT trial and efforts to partner rights to Macrilen™ in the U.S. and
Canad a - Company continues to build growing body of data across its development pipeline, advancing towards go/no-go decision milestones at each stage to establish prioritization and drive momentum
- Strategic decision to streamline development programs by discontinuing the Company’s early-stage vaccine programs provides Aeterna Zentaris with an expected extended cash runway into 2025
“We have made significant development progress since we initiated six pre-clinical development programs in 2021,” said Dr.
Macimorelin Diagnostic: Approvedand commercialized as test for adult growth hormone deficiency and in clinical phase 3 development for childhood-onset growth hormone deficiency ("CGHD") testing
Aeterna is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency (“CGHD”). Most clinical sites in the
Recent Highlights
- Phase 3 Study: Bolstered enrollment expected by the replacement of inactive countries/sites and engagement of an additional Clinical Research Organization (CRO). Currently four new countries (
Armenia ,Slovakia ,Greece , andTurkey ), have ongoing DETECT clinical trial application activities. - Macimorelin Commercialization: Having announced that Aeterna Zentaris will regain full rights to Macrilen™ for the
U.S. andCanada from Novo Nordisk inMay 2023 , the Company is progressing its efforts to seek an alternate development and commercialization partner for Macrilen™ in those countries for continued commercialization of Macrilen™ in adult growth hormone deficiency (“AGHD”) and development and commercialization of Macrilen™ in CGHD. - Achieved regulatory approval for Macrilen™ as AGHD diagnostic product in
Israel .
Next Steps and Expectations
- Achieve approvals to conduct the DETECT trial in the new countries in the first half of 2023.
- Complete DETECT enrollment by the end of 2023.
- Continue efforts to secure a
U.S. and Canadian development and commercialization partner for Macrilen™ in AGHD and CGHD. - Achieve regulatory approval for Macrilen™ as AGHD-diagnostic in
South Korea ,Turkey and some non-EU Balkan countries.
Decision to Discontinue Our Early-Stage Vaccine Development Pipeline
Orally active, live-attenuated bacterial vaccine platform with potential application against coronavirus, including COVID-19 (SARS-CoV-2) and Chlamydia
The Company worked on evaluating administration routes, dosing and immunization scheme, and conducted in-vivo immunology experiments with candidate strains expressing various antigens in relevant mice models. To date, the Company has not been able to select a clinical development candidate for a COVID-19 vaccine.
Also, COVID-19 vaccine landscape has continued to evolve profoundly in the past two years. There are highly effective vaccines available, an increasing number of therapeutic options are meanwhile approved or in later stage development and less lethal virus variants are spreading, all of which increase the financial risk associated with any early stage COVID-19 vaccine program. In order to ensure we are prudent with the use of resources, given the early stage of the Company’s vaccine development programs and the changes in the global situation, Aeterna has decided that it will not pursue further development of the vaccine platform for either COVID-19 or Chlamydia (which was based on the same vaccine platform as used in the Company’s COVID-19 program). As a result, the Company has also elected to terminate its existing license agreements with the
Therapeutics Development Pipeline Update
Autoimmunity Modifying ("AIM") Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") and Parkinson's disease (“PD”)
Recent Highlights
- Demonstrated positive pre-clinical proof-of-concept in NMOSD and PD.
- NMOSD selected as lead development program.
- Entered into an R&D agreement with
Massachusetts General Hospital (MGH) inBoston and Dr.Michael Levy , a worldwide leading NMOSD expert. - Further optimization of candidates for potential use in clinical development.
- Initiated ex-vivo efficacy testing to confirm the AIM Biologicals mechanism of action and efficacy in human blood samples from both NMOSD and PD patients.
Next Steps and Expectations
- Following the completion of the pre-clinical studies, Aeterna plans to compile a comprehensive pre-clinical data package for scientific advice meetings with regulatory authorities, expected to take place in the third quarter of 2023.
Delayed Clearance Parathyroid Hormone ("DC-PTH", AEZS-150) Fusion Polypeptides: Potential treatment for chronic hypoparathyroidism
Recent Highlights
- Successful verification and reproduction of previous in-vivo data from
University of Sheffield , in a rat model of hypoparathyroidism, in the fourth quarter of 2022. - Ongoing development of the manufacturing process for AEZS-150 with the Company’s contract development and manufacturing organization, progressing toward establishment of a master cell bank for a cell line expressing AEZS-150 and a process suitable for larger scale GMP manufacturing.
Next Steps and Expectations
- Continue efforts to establish master cell bank for a cell line expressing AEZS-150 to be used to conduct further pre-clinical efficacy and safety tests.
- Meet with regulatory authorities to determine the development path forward; Company expects meeting in mid-2023 and an abbreviated safety and toxicology program as the active principle ([1-34)-PTH fragment) is already approved and in use.
Macimorelin Therapeutic (AEZS-130):Ghrelin agonist in development for the treatment of
ALS (
Recent Highlights
- Substantial progress in the development of a suitable, alternative formulation for use in ALS.
- Ongoing evaluation of AEZS-130 in transgenic mouse ALS models to demonstrate the therapeutic potential of macimorelin in this indication.
Next Steps
- Proof-of-concept results from ongoing pre-clinical studies expected in the first quarter of 2023.
- Following potential achievement of proof-of-concept, the Company will seek to have a scientific advice meeting with regulatory authorities to discuss program development next steps.
- Toxicology and safety studies to support treatment over prolonged periods are ongoing and based on the substantial body of pre-clinical and clinical data already available from the successful development of macimorelin (Macrilen™) as a diagnostic in AGHD.
Strategic Conclusion
“Our disciplined development strategy ensures that we establish progress in our projects within a defined timeframe, in order to optimize the Company’s capital and human resources,” said
About
Aeterna Zentaris is a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products focused on areas of significant unmet medical need. The Company's lead product, macimorelin (Macrilen™; Ghryvelin®), is the first and only
Aeterna Zentaris is dedicated to the development of therapeutic assets and has recently taken steps to establish a growing pre-clinical 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;
For more information, please visit www.zentaris.com and connect with the Company on
Forward-Looking Statements
This press release contains statements that may constitute forward-looking statements within the meaning of
Forward-looking statements in this press release include, but are not limited to, those relating to Aeterna's expectations regarding: its preclinical and clinical studies, its ability to secure regulatory approvals for Macrilen™, its efforts to obtain a development and commercialization partner for Macrilen™ in the
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
No securities regulatory authority has either approved or disapproved of the contents of this news release.
Investor Contact:
JTC Team
T (US): +1 (833) 475-8247
E: aezs@jtcir.com
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