Percheron: Phase 2 Readout + Additional Catalysts to Drive Re-Rate

Given that the SPP will close tomorrow, and thus the lid on the share price lifted, I thought it would be an opportune time to start a new thread focusing on the rapidly approaching, and incredibly exciting (company-making) catalyst, due some time in December. There is a lot of bickering in some of the other threads, but I am hoping this thread would be focused on discussing this readout, amongst the numerous other fast approaching catalysts, that will hopefully send the price to 30c and beyond. DXB last year is one of many excellent examples of how quickly sentiment can change to incredibly pessimistic, to incredibly optimistic, with simply a single announcement. Not to mention we no longer have the election uncertainty weighing on the price...


I will get this thread started:

Percheron Therapeutics (ASX: PER) is on the brink of a potentially transformative moment with the upcoming December 2024 readout of its Phase IIb clinical trial for ATL1102. This drug, an antisense oligonucleotide targeting CD49d, is being tested in non-ambulant boys with Duchenne Muscular Dystrophy (DMD). This report provides an in-depth analysis of the trial, existing supporting data, expectations for the readout, and future catalysts that position Percheron as an exciting prospect for investors.


The December 2024 Catalyst: Phase IIb Trial Readout
Background on ATL1102 and Its Significance

ATL1102T is designed to modulate immune responses through its action on CD49d, a component involved in leukocyte adhesion and migration. The drug’s anti-inflammatory properties have been demonstrated across several conditions, showing potential for broader therapeutic applications.

The ongoing Phase IIb trial represents a critical inflection point for Percheron. This double-blind, randomised, placebo-controlled trial aims to provide robust data by comparing two doses of ATL1102 against a placebo in 48 non-ambulant boys aged 10 to 18. The primary endpoint is the change in the Performance of the Upper Limb (PUL2.0) score at six months. Secondary endpoints include assessments like Myopinch and Myogrip, along with exploratory biomarkers, which may further underline the drug’s efficacy.

Why This Readout Matters The readout is crucial as it could establish ATL1102’s clinical and commercial viability, opening doors to potential expedited regulatory pathways or strategic partnerships. Positive results could position Percheron as a major player in the rare disease segment, with ATL1102 addressing a substantial unmet need in DMD.


Existing Data Supporting Optimism
Phase IIa Results and Preclinical EvidenceThe optimism surrounding the Phase IIb results is rooted in the success of a prior Phase IIa study involving nine non-ambulant boys with DMD, which showed promising activity and was published in a peer-reviewed journal. This earlier study provided preliminary but encouraging data that ATL1102 could mitigate inflammation and improve clinical outcomes in DMD patients.

In addition, preclinical studies have demonstrated ATL1102’s efficacy in other inflammatory diseases. For example, a preclinical trial on autoimmune epilepsy confirmed its anti-inflammatory activity by significantly reducing seizure frequency. These results contribute to the hypothesis that ATL1102’s mechanism may yield beneficial results in DMD as well.

Toxicology Data: A Path to Regulatory ApprovalThe completion of a nine-month Good Laboratory Practice (GLP) toxicology study in non-human primates, with no unexpected toxicity findings, is another key piece of supportive evidence. This study satisfies an FDA prerequisite for trials involving extended dosing, strengthening ATL1102’s profile for future regulatory discussions. These data, combined with the anticipated Phase IIb results, could lay the groundwork for Percheron’s discussions with the FDA in early 2025.



What to Expect in DecemberThe December readout is expected to provide:

• Primary Endpoint Results: A comparison of the PUL2.0 scores at six months for patients on ATL1102 versus those on placebo.
• Secondary Endpoints: Data on Myopinch and Myogrip scores, which will help assess the broader impact of ATL1102 on muscle strength.
• Safety and Tolerability: Preliminary safety data to reinforce findings from the toxicology study.

A positive readout could validate ATL1102’s efficacy and safety profile, potentially enabling accelerated regulatory pathways or securing strategic partnerships to support further development.


Looking Ahead: Key Catalysts for 2025
Twelve-Month and Final Data
While the six-month data is pivotal, additional data will be reported in 2025:

• Twelve-Month Data (Mid-2025): Expected to provide longer-term insights into ATL1102’s sustained efficacy and safety.
• Final Data (Q4 2025): Will include the four-month off-treatment period, highlighting potential long-term benefits and durability of response.

Regulatory Discussions and Potential Approval PathwaysArmed with comprehensive data, Percheron plans to engage with the FDA and other regulatory agencies in early 2025. These discussions could determine the feasibility of seeking marketing authorisation based on existing data or pursuing a Phase III registration study. The outcome of these engagements will be pivotal in shaping ATL1102’s commercial trajectory.

Strategic Partnerships and Market Expansion
Percheron’s management has indicated strong interest from potential partners. Successful Phase IIb results could accelerate these discussions, positioning ATL1102 for broader development, possibly including combination therapies for ambulant DMD patients and other muscular dystrophies.

The Opportunity Ahead
Market Potential DMD affects approximately 1 in 10,000 males, creating a substantial market for effective treatments. Existing therapies can cost up to USD $750,000 per year, and the overall market is projected to reach around USD $10 billion by 2030. ATL1102’s broad applicability, being mutation-agnostic, enhances its potential market share significantly.

Broader Pipeline and Future Indications Beyond DMD, ATL1102 has shown promise in other conditions such as limb girdle muscular dystrophy and autoimmune diseases. This versatility could lead to expanded indications and additional revenue streams.

Financial Position and Strategic Moves Percheron has demonstrated fiscal prudence, securing funds through strategic placements and maintaining a strong cash position. The company’s lean, virtual model allows for efficient resource allocation, supporting ongoing R&D and potential partnerships.


ConclusionThe December 2024 readout for ATL1102 represents a high-stakes event that could catalyse a significant revaluation of Percheron’s share price and growth prospects. The anticipated results, underpinned by strong Phase IIa and preclinical data, as well as comprehensive toxicology studies, support a positive outlook. With further data readouts in 2025 and potential regulatory discussions, Percheron is positioned for a series of value-driving events. Investors should monitor these milestones closely, as success in December could set off a chain reaction leading to partnerships, regulatory submissions, and market entry for ATL1102.This confluence of upcoming events, robust supporting data, and strategic foresight make Percheron an attractive, albeit speculative, opportunity in the biotech sector.