Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies fo...


          
		  
          
            
            
            Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval
,  /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, today reaffirmed its strong market position around its Duchenne Muscular Dystrophy (DMD) program following  yesterday's congressional announcement that it had passed a five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher ("PRV") Program. The program extends the Company's ability to be eligible to receive a future PRV upon the U.S. Food and Drug Administration's ("FDA") approval following the FDA granting Rare Pediatric Disease ("RPD") designation to (Z)-endoxifen for the treatment of DMD late last year.
RPD designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect individuals from birth to 18 years of age. Upon approval of a qualifying marketing application, drugs with RPD designation may be eligible for a PRV, which can be used to obtain priority review for a future application or may be sold or transferred to another sponsor. In the last 18 months, disclosed PRV sales have ranged from $150–$200 million.
"The renewal of the PRV program represents an important signal from Congress that it understands the complexities and financial burden on the drug development industry. The program's continuation, along with the RPD designation, underscores the validation of the science supporting the potential of (Z)-endoxifen as a treatment for Duchenne Muscular Dystrophy," said Steven Quay, M.D., Ph.D., Atossa Therapeutics President and Chief Executive Officer. "DMD is one of the most devastating childhood diseases. Families urgently need better options beyond steroids and gene-targeted approaches. While oncology remains our core focus, this milestone highlights (Z)-endoxifen's potential as a platform therapy in both cancer and rare diseases, opening the door to potential non-dilutive value creation through the Rare Pediatric Disease program."
"The passage the PRV reauthorization is a win for all children with serious and rare diseases," said Janet Rea, MSPH, Senior Vice President of Research and Development at Atossa. "We are very encouraged by emerging preclinical data with (Z)-endoxifen and its potential in DMD, and we look forward to advancing this program to the clinic for boys living with DMD. Unlike more recent therapeutic approaches, (Z)-endoxifen does not target specific exon defects, thus potentially offering a broader and more accessible treatment approach for this patient population. Having previously secured IND clearance for what is now the DMD treatment, EXONDYS 51® (eteplirsen), I am excited to further Atossa's DMD (Z)-endoxifen program."
About Rare Pediatric Disease Designation
The FDA's Rare Pediatric Disease designation is reserved for serious or life-threatening diseases that primarily affect individuals from birth to 18 years old and that meet the definition of a rare disease or condition within the meaning of Section 526 of the Federal Food, Drug & Cosmetic Act ("FD&C Act"). Drugs granted RPD designation may be eligible for a PRV upon FDA approval of a qualifying New Drug Application or Biologics License Application, provided it meets all statutory criteria under Section 529(a)(4) of the FD&C Act. A PRV may be used by the sponsor or sold or transferred to another company.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy is a rare, progressive, X-linked neuromuscular disorder caused by one or more mutation in the dystrophin gene. Symptoms typically emerge in early childhood and include progressive muscle weakness, loss of ambulation, respiratory compromise, and cardiomyopathy. DMD is uniformly fatal, often in early adulthood, and despite recent therapeutic advances, there remains a substantial unmet medical need for safe, effective, and accessible treatments.
About (Z)-Endoxifen
(Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/D) with demonstrated activity across multiple mechanisms of interest. Atossa is evaluating its potential applications in oncology and rare diseases. The Company's proprietary oral formulation has shown a favorable safety profile and pharmacology distinct from tamoxifen, including ER-targeted effects and PKC inhibition. Atossa's (Z)-endoxifen is not approved for any indication.
Atossa's (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including multiple recently issued U.S. patents and numerous pending applications worldwide.
About Atossa Therapeutics
Atossa Therapeutics, Inc. (Nasdaq: ATOS) is a clinical-stage biopharmaceutical company developing innovative medicines in oncology and other areas of significant unmet need. The Company's lead product candidate, (Z)-endoxifen, is currently in development across several clinical settings. More information is available at https://atossatherapeutics.com.
Forward-Looking Statements
This press release contains certain "forward-looking statements" within the meaning of applicable securities laws, including but not limited to, our expectations regarding the Company's development and regulatory strategy and related milestones, the potential indications that the Company may pursue for (Z)-endoxifen, the potential for (Z)-endoxifen to receive regulatory approval and the timing thereof, the potential extension of the RPD Priority Review Voucher program and the Company's potential eligibility for and value of a future Priority Review Voucher, and the potential market and growth opportunities for the Company. Words such as "expect," "potential," "continue," "may," "will," "should," "could," "would," "seek," "intend," "plan," "estimate," "anticipate," "believe," "design," "predict," "future," or other similar expressions or statements regarding intent, belief or current expectations, are forward-looking statements.
Forward-looking statements in this press release are subject to risks and uncertainties that may cause actual results, outcomes, or the timing of actual results or outcomes to differ materially from those projected or anticipated, including, without limitation, risks and uncertainties associated with: our ability to successfully execute our strategy to shorten our clinical development timelines and pursue a metastatic breast cancer indication, DMD indication or other indications for our lead program, (Z)-endoxifen; expected timing, completion and results of our preclinical studies, clinical trials and research and development programs; the unpredictable relationship between preclinical study results and clinical study results; the timing or likelihood of regulatory filings and approvals; the outcome or timing of necessary regulatory approvals; our ability to regain and maintain compliance with Nasdaq listing requirements; our ability to establish and maintain intellectual property rights covering our products; the impact of general macroeconomic conditions on our business; our ability to raise capital; and other risks and uncertainties detailed from time to time in Atossa's filings with the SEC, including, without limitation, its Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q.
The market value of a Priority Review Voucher is variable and subject to a number of factors beyond our control and reported past PRV sale amounts are not necessarily indicative of PRV sale amounts in the future.
Forward-looking statements are presented as of the date of this press release. Except as required by law, we do not intend to update any forward-looking statements.
SOURCE Atossa Therapeutics Inc

          
        

ATOS

Atossa Therapeutics Maintains Strong Market Position for (Z)-Endoxifen for Duchenne Muscular Dystrophy as Congress Reauthorizes Priority Review Voucher Program

<section class="insight" data-version="1">
  <section data-block="information">
    <h3>Information</h3>
    <p>Atossa&#039;s Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies fo...</p>
  </section>

  <section data-block="ai-summary-paragraph">
    <h3>AI Summary</h3>
    <p>Atossa Therapeutics has attained Rare Pediatric Disease designation for (Z)-endoxifen, enhancing its potential for a future Priority Review Voucher (PRV) upon FDA approval. This milestone, coupled with renewed congressional support, showcases the drug’s promise in treating Duchenne Muscular Dystrophy and could create significant non-dilutive value for the company.</p>
  </section>

  <section data-block="ai-summary-bullets">
    <h3>Key Points</h3>
    <ul><li>Atossa receives Rare Pediatric Disease designation for (Z)-endoxifen.</li><li>Company eligible for future priority review voucher upon FDA approval.</li><li>Renewed PRV program highlights U.S. support for rare disease treatment development.</li><li>DMD program aims to offer broader treatment options beyond current therapies.</li><li>Emerging data supports (Z)-endoxifen&#039;s potential in both oncology and rare diseases.</li></ul>
  </section>

  <section data-block="companies-mentioned">
    <h3>Companies Mentioned</h3>
    <ul><li><strong>Exondys 51 (SRPT)</strong>: Atossa&#039;s DMD program aims to offer broader treatment options than Exondys 51.</li></ul>
  </section>

  <section data-block="category">
    <h3>Corporate Developments</h3>
    <p>This news falls under &#039;Corporate Developments&#039; due to the regulatory advancements impacting Atossa&#039;s drug candidate and its strategic positioning in the biopharmaceutical landscape. The PRV program introduces new avenues for funding and enhances the attractiveness of (Z)-endoxifen, which could bolster investor confidence and market presence.</p>
  </section>

  <section data-block="market-moving">
    <h3>Market-Moving</h3>
    <ul><li>Potential FDA approval could significantly enhance ATOS valuations through PRV sale.</li><li>Emerging clinical data could drive stock price volatility and investor sentiment.</li><li>Continued congressional support may increase funding opportunities for Atossa&#039;s programs.</li><li>Success in DMD could expand Atossa&#039;s pipeline and revenue streams.</li></ul>
  </section>

  <section data-block="key-facts">
    <h3>Key Facts</h3>
    <ul><li>Atossa received RPD designation for (Z)-endoxifen in DMD.</li><li>PRV sales in the last 18 months ranged from $150M to $200M.</li><li>Congress renewed the Rare Pediatric Disease PRV program for five years.</li><li>Duchenne Muscular Dystrophy primarily affects children under 18 years.</li><li>(Z)-endoxifen shows potential across multiple therapeutic areas.</li></ul>
  </section>

  <section data-block="trading-thesis">
    <h3>Trading Thesis</h3>
    <p>Consider buying ATOS shares in anticipation of increased investor interest and valuation due to PRV eligibility.</p>
  </section>
</section>


<article>
    <h2>Atossa Therapeutics Strengthens Its Position in Duchenne Muscular Dystrophy with (Z)-Endoxifen</h2>
    <p>Atossa Therapeutics, Inc. (Nasdaq: ATOS) has solidified its strategic foothold in the treatment of Duchenne Muscular Dystrophy (DMD) following a significant congressional announcement regarding the five-year reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program. This update enhances Atossa's eligibility for a future PRV upon the U.S. Food and Drug Administration’s (FDA) approval of its novel therapy, (Z)-endoxifen, which received Rare Pediatric Disease (RPD) designation last year.</p>

    <section>
        <h3>Implications of the PRV Program Reauthorization</h3>
        <p>The RPD designation is crucial as it is awarded to drug candidates targeting serious or life-threatening diseases primarily affecting individuals from birth to 18 years. The PRV allows drug companies to obtain priority review for future applications and may be sold or transferred, with past transactions revealing market prices ranging between <strong>$150 million and $200 million</strong>.</p>
        <p>Steven Quay, M.D., Ph.D., President and CEO of Atossa, remarked, <em>"The renewal of the PRV program represents an important signal from Congress that it understands the complexities and financial burden on the drug development industry."</em> Quay emphasized that the continuation of this program, coupled with the RPD designation, reinforces the scientific validation of (Z)-endoxifen as a potential treatment for DMD.</p>
    </section>

    <section>
        <h3>Advancing Treatments for Duchenne Muscular Dystrophy</h3>
        <p>Duchenne Muscular Dystrophy is a progressive, X-linked neuromuscular disorder, leading to severe muscle weakness and early mortality. With current treatments insufficient, Atossa aims to provide better options through (Z)-endoxifen. Janet Rea, Senior Vice President of Research and Development at Atossa, stated, <em>"We are very encouraged by emerging preclinical data with (Z)-endoxifen and its potential in DMD."</em> This compound is noted for its ability to offer a broader treatment approach compared to recent gene-targeting therapies.</p>

        <h3>About (Z)-Endoxifen</h3>
        <p>(Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/D) being explored for its applications not only in oncology but also in rare diseases such as DMD. This proprietary oral formulation stands out for its safety profile and pharmacological distinctions when compared to tamoxifen, including reduced side effects. However, it is important to note that (Z)-endoxifen is not yet approved for any indication.</p>
    </section>

    <section>
        <h3>Understanding Rare Pediatric Disease Designation</h3>
        <p>The FDA designates a drug as a Rare Pediatric Disease if it is intended to treat serious or life-threatening conditions primarily affecting individuals aged 0 to 18. Upon successful FDA approval, qualifying drugs under this designation may be eligible for a Priority Review Voucher, which provides faster access to the review process or allows sale to another company.</p>
    </section>
    
    <section>
        <h3>About Atossa Therapeutics</h3>
        <p>Atossa Therapeutics, Inc. (Nasdaq: ATOS) is committed to developing innovative therapies for significant unmet medical needs, focusing on both oncology and rare diseases. Its lead candidate, (Z)-endoxifen, is pivotal in its development pipeline, promising advancements in treatment options. More information can be found at <a href="https://atossatherapeutics.com">Atossa Therapeutics</a>.</p>
    </section>

    <section>
        <h3>Forward-Looking Statements</h3>
        <p>This article contains forward-looking statements regarding Atossa’s expectations concerning its development strategies and potential regulatory milestones for (Z)-endoxifen. Actual results may differ due to various risks and uncertainties, including the timing of regulatory filings and market reactions.</p>
    </section>
</article>

Atossa Therapeutics has attained Rare Pediatric Disease designation for (Z)-endoxifen, enhancing its potential for a future Priority Review Voucher (PRV) upon FDA approval. This milestone, coupled with renewed congressional support, showcases the drug’s promise in treating Duchenne Muscular Dystrophy and could create significant non-dilutive value for the company.

atossa-therapeutics-maintains-strong-market-position-for-z-endoxifen-for-duchenne-muscular-dystrophy-as-congress-reauthorizes-priority-review-voucher-program

Atossa Therapeutics Maintains Strong Market Position for (Z)-Endoxifen for Duchenne Muscular Dystrophy as Congress Reauthorizes Priority Review Voucher Program

AI Summary

Sentiment Rationale

Trading Thesis

Market-Moving

Key Facts

Companies Mentioned

Corporate Developments

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