Aldeyra Therapeutics Announces Statistically and Clinically Significant Improvement from Baseline in Phase 2 Clinical Trial of ADX‑629 in Patients with Atopic Dermatitis
- Statistically significant improvement in EASI, IGA, HAM-D, and POEM scores
- Complete resolution of affected body surface area in one patient
- Encouraging results supportive of advancing ADX-246 to Phase 1/2 clinical testing
- None.
Insights
The recent Phase 2 clinical trial results from Aldeyra Therapeutics for ADX-629, a RASP modulator for treating atopic dermatitis, indicate a promising therapeutic potential. The drug's ability to achieve statistically significant improvements in both physician-assessed and patient-reported outcomes is noteworthy. Specifically, the data showing improvement in EASI scores and patient-reported itching, as well as a positive impact on depression scores measured by HAM-D, suggest a multifaceted benefit that extends beyond skin symptoms.
From an investment perspective, these results could be a positive catalyst for Aldeyra's stock, as they not only support the progression of ADX-629 but also validate the company's RASP modulator platform. The absence of serious adverse events and the lack of discontinuations due to adverse events underscore the drug's safety profile, an essential factor in drug development success. Moreover, the planned advancement of ADX-246, a next-generation RASP modulator, into Phase 1/2 trials, could further bolster investor confidence in the pipeline's depth and the company's long-term prospects.
The clinical trial outcomes for ADX-629 indicate a significant therapeutic advance in the treatment of mild to moderate atopic dermatitis. Achieving a 75% improvement in EASI scores in 38% of patients is particularly impressive, considering the chronic and often treatment-resistant nature of the condition. The complete clearance of affected body surface area in one patient may represent a breakthrough for individuals with persistent skin lesions.
As a dermatologist, the potential for a new oral therapy that is both efficacious and well-tolerated could change the current treatment landscape, which heavily relies on topical agents and injectables. The improvement in psychosocial measures such as depression and anxiety scores also highlights the profound impact atopic dermatitis has on quality of life and the importance of developing treatments that address these comorbidities.
The progression of ADX-629 through clinical trials exemplifies the intricate process of drug development, especially in the field of immune-mediated diseases. The statistical significance achieved in various efficacy endpoints is promising and suggests a strong rationale for proceeding to later-stage trials. It is important to note that the trial was conducted in a single-center with a small sample size, which may limit the generalizability of the results.
The positive safety profile observed is as important as efficacy in determining a drug's potential for FDA approval and market success. The transition from ADX-629 to the next-generation compound ADX-246 should be watched closely, as it may offer improved efficacy or safety profiles. Investors should monitor the upcoming multicenter Phase 1/2 trials for ADX-246, as these will provide more robust data on the drug's potential.
- Statistically significant improvement from baseline observed in investigator-assessed Eczema Area and Severity Index (EASI, p=0.0006) and Investigator Global Assessment (IGA, p<0.0001)
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EASI
75% improvement (EASI-75) threshold observed in three patients (38% ), and affected body surface area was completely cleared in one patient (13% ) -
Patient-reported itching eliminated in two patients (
25% ) and clinically relevant threshold achieved in patient-reported eczema score (POEM) in six patients (75% ) - Statistically significant improvement from baseline observed in Hamilton Rating Scale for Depression (HAM-D, p=0.02)
- Results supportive of advancing ADX-246, an analog investigational drug of ADX‑629, to Phase 1/2 placebo-controlled clinical trial in healthy volunteers and atopic dermatitis patients
- Company to present top-line results in conference call and webcast at 8:00 a.m. ET today
“The demand for safe, tolerable, and orally administered atopic dermatitis therapies, particularly for mild to moderate patients, is substantial,” stated Dr. Matthew Zirwas, founder of the Bexley Dermatology Research clinic and Board-certified dermatologist who served as Principal Investigator of the clinical trial. “The data announced today offer a glimpse into what may be possible for many patients who today are not adequately treated.”
An open-label, single-center Phase 2 clinical trial of ADX-629 was conducted in eight mild to moderate atopic dermatitis patients. Over three months of treatment, patients received 250mg ADX-629, administered orally twice daily. The primary endpoint of the clinical trial was safety and tolerability. Secondary endpoints included Eczema Area and Severity Index (EASI), Investigator Global Assessment (IGA), Patient-Oriented Eczema Measure (POEM), Peak Pruritus Numerical Rating Scale, time to flare, Hamilton Depression Rating Scale (HAM-D), and Beck Anxiety Inventory (BAI).
Relative to baseline, over three months of treatment, improvement was observed in all patients. Statistical significance was achieved for improvement in EASI (p=0.0006). EASI thresholds for
All enrolled patients completed the trial per protocol. No patients experienced flare requiring rescue therapy. Only two adverse events deemed to be at least possibly related to ADX-629 were reported, and both events were mild. There were no observed serious adverse events or discontinuations due to adverse events.
“The results from the clinical trial of ADX‑629 in atopic dermatitis are consistent with activity demonstrated in previously disclosed clinical trials of ADX‑629, including Phase 2 clinical trials in psoriasis, asthma, and chronic cough, adding to a growing body of evidence that we believe is supportive of the activity of RASP modulators in systemic diseases associated with inflammation,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “Based on the signal-finding activity of ADX‑629, we enthusiastically plan to advance our next-generation investigational RASP modulator ADX‑246 to Phase 1/2 clinical testing in healthy volunteers and patients with atopic dermatitis.”
Aldeyra expects to initiate a multicenter, randomized, placebo-controlled Phase 1/2 clinical trial of ADX‑246 in healthy volunteers and patients with atopic dermatitis in the first half of 2024. Topline results from the trial are expected in the second half of 2024.
Conference Call & Webcast Information
Aldeyra will host a conference call at 8:00 a.m. ET today, December 19, 2023, to discuss the top-line results of the Phase 2 clinical trial of ADX-629 in atopic dermatitis. The dial-in numbers are (888) 415-4305 for domestic callers and (646) 960-0336 for international callers. The access code is 5858366. A live audio webcast of the conference call also will be accessible from the “Investors & Media” section of Aldeyra's website at ir.aldeyra.com. A live webcast of the conference call will be available on the Investor Relations page of the company’s website at https://ir.aldeyra.com. After the live webcast, the event will remain archived on the Aldeyra Therapeutics website for 90 days.
About Aldeyra
Aldeyra Therapeutics is a biotechnology company devoted to discovering innovative therapies designed to treat immune-mediated diseases. Our approach is to develop pharmaceuticals that modulate immunological systems, instead of directly inhibiting or activating single protein targets, with the goal of optimizing multiple pathways at once while minimizing toxicity. Our product candidates include RASP (reactive aldehyde species) modulators ADX‑629, ADX‑246, ADX‑248, and chemically related molecules for the potential treatment of systemic and retinal immune-mediated diseases. Our pre-commercial product candidates are reproxalap, a RASP modulator for the potential treatment of dry eye disease and allergic conjunctivitis, and ADX-2191, a novel formulation of intravitreal methotrexate for the potential treatment of proliferative vitreoretinopathy and retinitis pigmentosa.
About ADX-629 and ADX-246
ADX-629 is an orally administered RASP modulator currently in development as a signal-finding molecule for the treatment of mass-market immune-mediated diseases. ADX‑629 has demonstrated potential activity in clinical trials of patients with psoriasis, asthma, COVID, ethanol toxicity, chronic cough, and atopic dermatitis. In more than 100 healthy volunteers and patients, no consistent adverse events associated with ADX‑629 have been identified. An analog of ADX-629, ADX‑246 is an orally administered next-generation RASP modulator expected to initiate clinical testing in the first half of 2024 in a Phase 1/2 clinical trial in healthy volunteers and patients with atopic dermatitis. Top-line results from the Phase 1/2 clinical trial are expected in the second half of 2024.
Safe Harbor Statement
This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Aldeyra’s future expectations, plans, and prospects, including without limitation statements regarding: the goals, opportunity and potential for ADX-629 and ADX-246, and anticipated clinical or regulatory milestones for ADX-629 and ADX-246. Aldeyra intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as, but not limited to, “may,” “might,” “will,” “objective,” “intend,” “should,” "could," “can,” “would,” “expect,” “believe,” “anticipate,” “project,” “on track,” “scheduled,” “target,” “design,” “estimate,” “predict,” “contemplates,” “likely,” “potential,” “continue,” “ongoing,” “aim,” “plan,” or the negative of these terms, and similar expressions intended to identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions, and uncertainties. Aldeyra is at an early stage of development and may not ever have any products that generate significant revenue. All of Aldeyra's development timelines may be subject to adjustment depending on recruitment rate, regulatory review, preclinical and clinical results, funding, and other factors that could delay the initiation, enrollment, or completion of clinical trials. Important factors that could cause actual results to differ materially from those reflected in Aldeyra's forward-looking statements include, among others, the timing of enrollment, commencement and completion of Aldeyra's clinical trials, the timing and success of preclinical studies and clinical trials conducted by Aldeyra and its development partners; delay in or failure to obtain regulatory approval of Aldeyra's product candidates, including as a result of the FDA not accepting Aldeyra’s regulatory filings, or requiring additional clinical trials or data prior to review or approval of such filings; the ability to maintain regulatory approval of Aldeyra's product candidates, and the labeling for any approved products; the risk that prior results, such as signals of safety, activity, or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or clinical trials involving Aldeyra's product candidates in clinical trials focused on the same or different indications; the scope, progress, expansion, and costs of developing and commercializing Aldeyra's product candidates; uncertainty as to Aldeyra’s ability to commercialize (alone or with others) and obtain reimbursement for Aldeyra's product candidates following regulatory approval, if any; the size and growth of the potential markets and pricing for Aldeyra's product candidates and the ability to serve those markets; Aldeyra's expectations regarding Aldeyra's expenses and future revenue, the timing of future revenue, the sufficiency or use of Aldeyra's cash resources and needs for additional financing; the rate and degree of market acceptance of any of Aldeyra's product candidates; Aldeyra's expectations regarding competition; Aldeyra's anticipated growth strategies; Aldeyra's ability to attract or retain key personnel; Aldeyra’s commercialization, marketing and manufacturing capabilities and strategy; Aldeyra's ability to establish and maintain development partnerships; Aldeyra’s ability to successfully integrate acquisitions into its business; Aldeyra's expectations regarding federal, state, and foreign regulatory requirements; political, economic, legal, social, and health risks, public health measures, and war or other military actions, that may affect Aldeyra’s business or the global economy; regulatory developments in
In addition to the risks described above and in Aldeyra's other filings with the SEC, other unknown or unpredictable factors also could affect Aldeyra's results. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and Aldeyra undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
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Investor & Media:
David Burke
Tel: (917) 618-2651
investorrelations@aldeyra.com
Source: Aldeyra Therapeutics, Inc.
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