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Aeterna Zentaris focused on accelerating recruitment for ongoing DETECT trial and efforts to companion rights to Macrilen™ in the U.S. and Canada
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Firm continues to construct rising physique of information throughout its growth pipeline, advancing in the direction of go/no–go resolution milestones at every stage to ascertain prioritization and drive momentum
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Strategic resolution to streamline growth packages by discontinuing the Firm’s early–stage vaccine packages presents Aeterna Zentaris with an anticipated prolonged cash runway into 2025
TORONTO, ONTARIO, Jan. 17, 2023 (GLOBE NEWSWIRE) — Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the “Firm”), a specialty biopharmaceutical firm growing and commercializing a diversified portfolio of pharmaceutical and diagnostic merchandise, at this time offered a enterprise replace and outlined upcoming key milestones.
“Now we have made important growth progress since we initiated six pre-clinical growth packages in 2021,” mentioned Dr. Klaus Paulini, Chief Govt Officer of Aeterna. “Now we have established a rising physique of information in the direction of our pre-defined go/no-go resolution factors with the purpose of enabling us to determine and choose from these packages probably the most compelling alternatives that warrant continued growth. We consider that we’ve got constructed the muse for continued momentum and are very inspired with the disciplined progress we’ve got made up to now.”
Diagnostics Growth and Commercialization Replace
Macimorelin Diagnostic: Accredited and commercialized as take a look at for grownup progress hormone deficiency and in scientific section 3 growth for childhood-onset progress hormone deficiency (“CGHD”) testing
Aeterna is presently conducting its pivotal Part 3 security and efficacy research AEZS-130-P02 (the “DETECT-trial”) evaluating macimorelin for the analysis of Childhood Onset Progress Hormone Deficiency (“CGHD”). Most scientific websites within the U.S. in addition to European nations are open for affected person recruitment.
Current Highlights
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Part 3 Study: Bolstered enrollment anticipated by the substitute of inactive nations/websites and engagement of an extra Scientific Analysis Group (CRO). Presently 4 new nations (Armenia, Slovakia, Greece, and Turkey), have ongoing DETECT scientific trial software actions.
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Macimorelin Commercialization: Having introduced that Aeterna Zentaris will regain full rights to Macrilen™ for the U.S. and Canada from Novo Nordisk in Could 2023, the Firm is progressing its efforts to hunt an alternate growth and commercialization companion for Macrilen™ in these nations for continued commercialization of Macrilen™ in grownup progress hormone deficiency (“AGHD”) and growth and commercialization of Macrilen™ in CGHD.
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Achieved regulatory approval for Macrilen™ as AGHD diagnostic product in Israel.
Subsequent Steps and Expectations
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Obtain approvals to conduct the DETECT trial within the new nations within the first half of 2023.
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Full DETECT enrollment by the top of 2023.
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Proceed efforts to safe a U.S. and Canadian growth and commercialization companion for Macrilen™ in AGHD and CGHD.
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Obtain regulatory approval for Macrilen™ as AGHD-diagnostic in South Korea, Turkey and a few non-EU Balkan nations.
Resolution to Discontinue Our Early–Stage Vaccine Growth Pipeline
Orally lively, live-attenuated bacterial vaccine platform with potential software in opposition to coronavirus, together with COVID-19 (SARS-CoV-2) and Chlamydia
The Firm labored on evaluating administration routes, dosing and immunization scheme, and performed in-vivo immunology experiments with candidate strains expressing varied antigens in related mice fashions. Up to now, the Firm has not been capable of choose a scientific growth candidate for a COVID-19 vaccine.
Additionally, COVID-19 vaccine panorama has continued to evolve profoundly previously two years. There are extremely efficient vaccines obtainable, an rising variety of therapeutic choices are in the meantime authorized or in later stage growth and fewer deadly virus variants are spreading, all of which improve the monetary threat related to any early stage COVID-19 vaccine program. As a way to guarantee we’re prudent with the usage of assets, given the early stage of the Firm’s vaccine growth packages and the adjustments within the world scenario, Aeterna has determined that it’ll not pursue additional growth of the vaccine platform for both COVID-19 or Chlamydia (which was primarily based on the identical vaccine platform as used within the Firm’s COVID-19 program). Because of this, the Firm has additionally elected to terminate its current license agreements with the College of Wuerzburg for that vaccine platform expertise.
Therapeutics Growth Pipeline Replace
Autoimmunity Modifying (“AIM”) Biologicals: Focused, extremely particular autoimmunity modifying therapeutics for the potential remedy of neuromyelitis optica spectrum dysfunction (“NMOSD”) and Parkinson’s illness (“PD”)
Current Highlights
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Demonstrated constructive pre-clinical proof-of-concept in NMOSD and PD.
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NMOSD chosen as lead growth program.
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Entered into an R&D settlement with Massachusetts Normal Hospital (MGH) in Boston and Dr. Michael Levy, a worldwide main NMOSD knowledgeable.
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Additional optimization of candidates for potential use in scientific growth.
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Initiated ex-vivo efficacy testing to verify the AIM Biologicals mechanism of motion and efficacy in human blood samples from each NMOSD and PD sufferers.
Subsequent Steps and Expectations
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Following the completion of the pre-clinical research, Aeterna plans to compile a complete pre-clinical knowledge bundle for scientific recommendation conferences with regulatory authorities, anticipated to happen within the third quarter of 2023.
Delayed Clearance Parathyroid Hormone (“DC-PTH”, AEZS-150) Fusion Polypeptides: Potential remedy for power hypoparathyroidism
Current Highlights
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Profitable verification and replica of earlier in-vivo knowledge from College of Sheffield, in a rat mannequin of hypoparathyroidism, within the fourth quarter of 2022.
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Ongoing growth of the manufacturing course of for AEZS-150 with the Firm’s contract growth and manufacturing group, progressing towards institution of a grasp cell financial institution for a cell line expressing AEZS-150 and a course of appropriate for bigger scale GMP manufacturing.
Subsequent Steps and Expectations
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Proceed efforts to ascertain grasp cell financial institution for a cell line expressing AEZS-150 for use to conduct additional pre-clinical efficacy and security exams.
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Meet with regulatory authorities to find out the event path ahead; Firm expects assembly in mid-2023 and an abbreviated security and toxicology program because the lively precept ([1-34)-PTH fragment) is already approved and in use.
Macimorelin Therapeutic (AEZS-130): Ghrelin agonist in development for the treatment of
ALS (Lou Gehrig’s disease)
Recent Highlights
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Substantial progress in the development of a suitable, alternative formulation for use in ALS.
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Ongoing evaluation of AEZS-130 in transgenic mouse ALS models to demonstrate the therapeutic potential of macimorelin in this indication.
Next Steps
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Proof-of-concept results from ongoing pre-clinical studies expected in the first quarter of 2023.
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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.
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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 Giuliano La Fratta, Chief Financial Officer, Aeterna. “We continue to drive progress across a number of our programs and remain focused on advancing those programs to their next phase of development if we determine that the data and financial risk/reward of doing so is justified. The strategic decision to streamline our pipeline, by discontinuing our vaccine development, provides the Company with the ability to meet its currently anticipated cash needs into 2025. Revenues that may be achieved if we out-license any of those development programs are not considered in our budget and thus provides further upside potential.”
About Aeterna Zentaris Inc.
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 U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). The Company 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, in collaboration with Novo Nordisk.
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; 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: 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 U.S. and Canada and the ability to meet its currently anticipated cash needs into 2025.
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; and our ability to continue to list our common shares on the NASDAQ. 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 and annual information form, 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.
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:
Jenene Thomas
JTC Team
T (US): +1 (833) 475-8247
E: aezs@jtcir.com
