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AC Immune Unveils Novel Therapeutic Antibody Drug Conjugate (ADC) Technology for Improved Efficacy in Neurodegenerative Diseases at AAIC 2024

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AC Immune SA (NASDAQ: ACIU) unveiled a novel class of neurodegenerative disease-fighting drug-candidates called morADC (Morphomer® Antibody Drug Conjugate) at AAIC 2024. This technology combines brain-penetrant Morphomer® small molecules with SupraAntigen® monoclonal antibodies to target toxic proteins in the central nervous system. Key findings include:

1. Significant synergies in blood-brain barrier penetration and protein aggregation inhibition
2. Capability for single or dual-targeting strategies
3. Enhanced anti-aggregation effects compared to parent molecules
4. Multiplied antibody brain exposure

The morADC platform aims to overcome challenges faced by existing modalities and improve clinical outcomes for patients with neurodegenerative diseases.

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Positive

  • Development of a novel therapeutic technology (morADC) potentially disruptive in the neurodegenerative disease treatment industry
  • Significant synergies observed in blood-brain barrier penetration and protein aggregation inhibition
  • Capability for single or dual-targeting strategies, allowing for combination therapy in a single agent
  • Enhanced anti-aggregation effects compared to parent molecules
  • Potential to deliver first- and best-in-class efficacy against multiple high-value therapeutic targets

Negative

  • None.

Insights

AC Immune's unveiling of the morADC (Morphomer® Antibody Drug Conjugate) technology at AAIC 2024 represents a potentially significant advancement in the field of neurodegenerative disease treatment. This novel approach combines two proprietary technologies - brain-penetrant Morphomers® and SupraAntigen® monoclonal antibodies - to create a new class of drug candidates targeting toxic proteins in the central nervous system (CNS).

Key highlights of the morADC technology include:

  • Ability to address important targets such as Abeta, Tau and a-synuclein
  • Potential for single or dual-targeting strategies, enabling combination therapy in a single agent
  • Significant synergistic anti-aggregation effects compared to individual components
  • Enhanced brain exposure of antibodies when conjugated with brain-penetrant Morphomers

The morADC technology's potential to improve blood-brain barrier penetration and increase potency in inhibiting protein aggregation is particularly noteworthy. This could address a major challenge in treating neurodegenerative diseases, where drug delivery to the brain has been a significant hurdle.

However, it's important to note that while these in vitro results are promising, the technology is still in its early stages. The true potential of morADC will only be realized through successful in vivo studies and clinical trials, which will be important in determining its efficacy and safety in humans.

AC Immune's morADC technology represents an innovative approach in the biotechnology sector, particularly in the field of neurodegenerative disease therapeutics. By combining two established platforms - SupraAntigen® and Morphomer® - AC Immune has created a potentially disruptive technology that could address several limitations of current treatment modalities.

The key advantages of this technology include:

  • Improved blood-brain barrier penetration, a critical factor in CNS drug delivery
  • Enhanced potency through synergistic effects of combined modalities
  • Flexibility in targeting single or multiple pathological proteins
  • Potential for combination therapy in a single agent, which could simplify treatment regimens

From a competitive standpoint, this technology could position AC Immune as a leader in the neurodegenerative disease space. The company's track record of developing valuable therapeutic candidates through its existing platforms lends credibility to this new approach.

However, investors should be aware that the path from preclinical studies to approved therapies is long and fraught with challenges. While the initial data is promising, the true value of this technology will only be realized after successful clinical trials and regulatory approvals.

Furthermore, the company's ability to protect this intellectual property and potentially license it to larger pharmaceutical companies could be a significant value driver in the future. As such, investors should closely monitor AC Immune's patent strategy and any potential partnership announcements in the coming months.

The unveiling of AC Immune's morADC technology at AAIC 2024 represents a potentially significant development for the company's future prospects. While it's too early to quantify the financial impact, this innovation could have several implications for AC Immune's market position and valuation:

  • Expanded pipeline potential: The morADC technology could lead to multiple new drug candidates, diversifying and strengthening AC Immune's product pipeline.
  • Increased partnering opportunities: Given the novelty of the technology, it may attract interest from larger pharmaceutical companies, potentially leading to lucrative licensing or collaboration agreements.
  • Enhanced competitive position: If successful, this technology could give AC Immune a significant edge in the highly competitive neurodegenerative disease market.
  • Long-term revenue potential: While commercialization is likely years away, successful development of morADC-based therapies could translate into substantial future revenues.

However, investors should approach this news with cautious optimism. The development of CNS drugs is notoriously challenging, with high failure rates in clinical trials. Significant R&D investment will be required to advance morADC candidates through preclinical and clinical stages, which could impact the company's cash burn rate.

Moreover, as a clinical-stage biopharmaceutical company, AC Immune's valuation is largely based on the potential of its pipeline rather than current revenues. The market's reaction to this news will depend on how investors perceive the likelihood of long-term success for this new technology.

In the near term, investors should monitor for any updates on preclinical and early clinical development of morADC candidates, as well as any potential partnership announcements, which could serve as catalysts for the stock price.

AC Immune Unveils Novel Therapeutic Antibody Drug Conjugate (ADC) Technology for Improved Efficacy in Neurodegenerative Diseases at AAIC 2024

  • morADC combines proprietary brain-penetrant small molecule Morphomers® with SupraAntigen® monoclonal antibodies, forming a new class of drug-candidates to target toxic proteins in CNS
  • morADC demonstrated significant synergies, substantially increasing blood brain barrier penetration and potency to inhibit protein aggregation compared to the antibody or small molecule alone

Lausanne, Switzerland, July 31, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today unveiled a novel class of neurodegenerative disease-fighting drug-candidates called morADC (Morphomer® Antibody Drug Conjugate) in an oral presentation at the annual Alzheimer’s Association International Conference (AAIC 2024), taking place from July 28 – August 1, 2024, in Philadelphia, PA.

Dr. Madiha Derouazi, Chief Scientific Officer of AC Immune, presented the characterization and in vitro efficacy of morADC for the first time. The talk, entitled A new class of neurodegenerative disease-fighting drugs: morADC (Morphomer®- Antibody Drug Conjugates)”, showed that:

  • morADC are designed for CNS applications capable of addressing important targets including Abeta, Tau and a-synuclein
  • morADC enable single or dual-targeting strategies (e.g. an anti-Abeta antibody combined with an anti-Tau small molecule) to deliver combination therapy in a single therapeutic agent
  • single-targeting morADC (i.e. antibody and small molecule targeting the same protein) show significant synergistic anti-aggregation effects compared to the parental molecules
  • dual-targeting morADC (e.g. Abeta/Tau) show significantly enhanced anti-aggregation effects compared to the parental molecules
  • additionally, conjugation of brain-penetrant Morphomers with a monoclonal antibody can multiply antibody brain exposure in comparison to the parent antibody alone

While in oncology antibody-drug conjugates are designed to selectively kill tumor cells in a targeted manner, AC Immune’s morADC for neurodegenerative diseases combine the high brain penetrance of Morphomer small molecules with the target specificity of monoclonal antibodies, to reduce pathological, aggregating proteins in the central nervous system.

Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “Today we unveil our powerful new morADC technology, which synergistically combines the industry-leading capabilities of our SupraAntigen and Morphomer platforms. Based on the data we have produced to date, we are confident that this is a groundbreaking moment, showing that morADC offer unique functionality and are potentially disruptive in our industry. We look forward with great enthusiasm to developing these exciting first-in-class therapeutic candidates as part of our mission to pioneer precision prevention for neurodegenerative diseases.”

“Our clinically validated SupraAntigen and Morphormer platforms have been the engines driving our track record of discovering and developing highly valuable therapeutic candidates. Our partnering history has demonstrated that these platforms are robust and that they have reliably enabled us to develop multiple first- and best-in-class molecules for diagnostic and therapeutic development.”

The morADC technology platform combines AC Immune’s industry leading SupraAntigen® and Morphomer® technologies for discovery and development of biologics and small molecules to detect, prevent and/or treat neurodegenerative diseases. SupraAntigen is used to discover and develop both, active immunotherapies (vaccines) and passive immunotherapies (monoclonal antibodies). It uses small spherical liposomes, to present specific antigens in a format designed to generate conformation-specific, antigen-targeting antibodies. The Morphomer platform combines small molecule chemistry with proprietary biological assays enabling identification and development of small molecule Morphomers that target pathological protein aggregates in extra- and intracellular brain compartments.

By attaching Morphomer small molecules to SupraAntigen antibodies to form Morphomer Antibody Drug Conjugates (morADC), this new morADC technology combines the advantages of AC Immune’s clinically validated technology platforms to offer unique synergistic properties. These include increased blood brain barrier penetration and higher potency, highlighting their potential to deliver first- and best-in-class efficacy against multiple high value therapeutic targets, thereby overcoming challenges faced by existing modalities and improving clinical outcomes for patients.

About AC Immune SA
AC Immune SA is a clinical-stage biopharmaceutical company and a global leader in precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and NeuroOrphan indications driven by misfolded proteins. The Company’s two clinically validated technology platforms, SupraAntigen® and Morphomer®, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features sixteen therapeutic and diagnostic programs, five of which are currently in Phase 2 clinical trials and one of which is in Phase 3. AC Immune has a strong track record of securing strategic partnerships with leading global pharmaceutical companies, resulting in substantial non-dilutive funding in potential milestone payments plus royalties.

SupraAntigen® is a registered trademark of AC Immune SA in the following territories: AU, EU, CH, GB, JP, RU, SG and USA. Morphomer® is a registered trademark of AC Immune SA in CN, CH, GB, JP, KR, NO and RU.

The information on our website and any other websites referenced herein is expressly not incorporated by reference into, and does not constitute a part of, this press release.

For further information, please contact:

Head of Investor Relations & Corporate Communications
Gary Waanders, Ph.D., MBA
AC Immune
Phone: +41 21 345 91 91
Email: gary.waanders@acimmune.com



U.S. Investors
Corey Davis, Ph.D.
LifeSci Advisors
Phone: +1 212 915 2577
Email: cdavis@lifesciadvisors.com

International Media
Chris Maggos
Cohesion Bureau
Phone: +41 79 367 6254
Email: chris.maggos@cohesionbureau.com

 

Forward looking statements
This press release contains statements that constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements are statements other than historical fact and may include statements that address future operating, financial or business performance or AC Immune’s strategies or expectations. In some cases, you can identify these statements by forward-looking words such as “may,” “might,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “potential,” “outlook” or “continue,” and other comparable terminology. Forward-looking statements are based on management’s current expectations and beliefs and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. These risks and uncertainties include those described under the captions “Item 3. Key Information – Risk Factors” and “Item 5. Operating and Financial Review and Prospects” in AC Immune’s Annual Report on Form 20-F and other filings with the Securities and Exchange Commission. Forward- looking statements speak only as of the date they are made, and AC Immune does not undertake any obligation to update them in light of new information, future developments or otherwise, except as may be required under applicable law. All forward-looking statements are qualified in their entirety by this cautionary statement.

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FAQ

What is AC Immune's new morADC technology unveiled at AAIC 2024?

morADC (Morphomer® Antibody Drug Conjugate) is a novel class of neurodegenerative disease-fighting drug-candidates that combines brain-penetrant Morphomer® small molecules with SupraAntigen® monoclonal antibodies to target toxic proteins in the central nervous system.

How does the morADC technology improve upon existing treatments for neurodegenerative diseases?

morADC technology shows significant synergies in blood-brain barrier penetration and protein aggregation inhibition. It enables single or dual-targeting strategies, demonstrates enhanced anti-aggregation effects compared to parent molecules, and multiplies antibody brain exposure.

What are the potential targets for AC Immune's morADC technology (ACIU)?

AC Immune's morADC technology (ACIU) is designed to address important targets in neurodegenerative diseases, including Abeta, Tau, and a-synuclein proteins.

When and where did AC Immune present its morADC technology findings?

AC Immune presented its morADC technology findings at the Alzheimer's Association International Conference (AAIC 2024), which took place from July 28 to August 1, 2024, in Philadelphia, PA.
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