Mechelen, Belgium; 24 August 2026, 22.01 CET; regulated information – Lakefront Biotherapeutics NV ((Euronext &amp;, NASDAQ:LKFT) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug

LKFT

U.S. FDA Grants Orphan Drug Designation to Gamgertamig for the Treatment of Pemphigus

Lakefront's gamgertamig earns FDA Orphan Drug designation for pemphigus

<section class="insight" data-version="1">
  <section data-block="information">
    <h3>Information</h3>
    <p>Mechelen, Belgium; 24 August 2026, 22.01 CET; regulated information – Lakefront Biotherapeutics NV ((Euronext &amp;amp;, NASDAQ:LKFT) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug</p>
  </section>

  <section data-block="ai-summary-paragraph">
    <h3>AI Summary</h3>
    <p>Lakefront Biotherapeutics disclosed that the FDA awarded Orphan Drug Designation to gamgertamig for pemphigus, complementing existing Fast Track and Orphan status for AIHA and ITP. The Gilead collaboration and ongoing Phase 1b trials with planned 2027 proof-of-concept and registrational studies outline a clearer development path and potential upside, likely supporting a near-term positive reaction in LKFT shares.</p>
  </section>

  <section data-block="ai-summary-bullets">
    <h3>Key Points</h3>
    <ul><li>FDA grants Orphan Drug Designation for pemphigus to gamgertamig.</li><li>Gamgertamig already has Fast Track and Orphan for AIHA/ITP.</li><li>Lakefront collaborates with Gilead; 2027 registrational plans expected.</li><li>Phase 1b trials active in AU/US and other regions; PoC basket studies slated for 2027.</li></ul>
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  <section data-block="companies-mentioned">
    <h3>Companies Mentioned</h3>
    <ul><li><strong>Lakefront Biotherapeutics NV (LKFT)</strong>: Main beneficiary of the announcement; development path and partnering potential could drive equity value.</li><li><strong>Gilead Sciences (GILD)</strong>: Collaborator on gamgertamig; potential for co-development funding and broader upside exposure.</li><li><strong>Keymed Biosciences (Unlisted)</strong>: Licensee for Greater China rights; strategic importance but no public ticker.</li></ul>
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  <section data-block="category">
    <h3>Corporate Developments</h3>
    <p>Category: Corporate Developments. The FDA designation is a regulatory milestone that informs LKFT&#039;s pipeline trajectory and potential value inflection, particularly given the Gilead collaboration and upcoming 2027 readouts.</p>
  </section>

  <section data-block="market-moving">
    <h3>Market-Moving</h3>
    <ul><li>FDA Orphan Drug Designation for pemphigus strengthens LKFT&#039;s regulatory positioning.</li><li>Gilead collaboration enhances development upside and potential funding support.</li><li>Phase 1b programs across AU, US, NZ, Czech Republic, and Japan with 2027 PoC plans.</li></ul>
  </section>

  <section data-block="key-facts">
    <h3>Key Facts</h3>
    <ul><li>Date of release: 24 August 2026.</li><li>Orphan Drug Designation granted for gamgertamig in pemphigus by FDA.</li><li>Gamgertamig already holds Fast Track and Orphan Designations for AIHA and ITP.</li><li>Phase 1b trial NCT07083960 active in Australia and the United States (autoimmune cytopenias).</li><li>Phase 1b trial NCT07229144 active in Australia, New Zealand, Czech Republic, and Japan (seropositive autoimmune diseases).</li><li>Expansion of the clinical program and PoC basket studies anticipated in 2027.</li><li>Gamgertamig is in-licensed from Keymed Biosciences for Greater China.</li></ul>
  </section>

  <section data-block="trading-thesis">
    <h3>Trading Thesis</h3>
    <p>Bullish: Orphan designation plus ongoing trials could re-rate LKFT toward a 2027 registrational pathway.</p>
  </section>
</section>


<link type="text/css" rel="stylesheet" href="https://www.globenewswire.com/styles/gnw_nitf.css"></link><p>Mechelen, Belgium; 24 August 2026, 22.01 CET; regulated information – Lakefront Biotherapeutics NV ((Euronext &amp, NASDAQ:<a class="ticker" href="https://www.benzinga.com/quote/LKFT">LKFT</a>) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to gamgertamig for the treatment of pemphigus. Gamgertamig was previously granted both Fast Track and Orphan Drug Designation by the U.S. FDA for the treatment of autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). </p>  <p>Gamgertamig is being developed by Lakefront in collaboration with Gilead Sciences. Ongoing clinical trials in autoimmune diseases are actively enrolling, and expansion of the clinical trials program is anticipated in 2027.</p>  <ul type="circle"><li>Phase 1b study with gamgertamig in patients with autoimmune cytopenias (including ITP and AIHA) (NCT07083960), active in Australia and the United States.</li><li>Phase 1b study with gamgertamig in seropositive autoimmune diseases (including idiopathic inflammatory myositis, Sjögren’s disease, pemphigus vulgaris (PV), and pemphigus foliaceus (PF)) (NCT07229144) active in Australia, New Zealand, Czech Republic, and Japan. </li><li>Expect to initiate proof-of-concept basket studies assessing gamgertamig in additional autoimmune indications.<br></br><br></br></li></ul>  <p align="justify">"We’re excited by the U.S. FDA’s award of Orphan Drug Designation for gamgertamig for the treatment of pemphigus as we continue to advance it for patients suffering with this debilitating and potentially life-threatening autoantibody-driven disease," said Eric Hedrick MD, Chief Medical Officer of Lakefront. "I continue to believe gamgertamig has the potential to represent a transformative medicine in autoimmune diseases." </p>  <p align="justify"><b>About Gamgertamig</b><br></br>Gamgertamig is an investigational BCMAxCD3 bispecific T cell engager for the treatment of autoantibodies driven immune-mediated disease. Gamgertamig has been granted Orphan Drug Designation and Fast Track Designation by the U.S. FDA for certain autoimmune diseases. Gamgertamig is currently in Phase 2 studies and is expected to enter registrational studies in 2027. Gamgertamig is in-licensed from Keymed Biosciences, which owns the rights to develop the program in Greater China.</p>  <p align="justify"><b>About Lakefront Biotherapeutics</b><br></br>Lakefront Biotherapeutics is a biotechnology company dedicated to building a differentiated pipeline of medicines for patients with serious diseases in areas of high unmet need. The Company has established a clinical‑stage portfolio in immunology and inflammation, anchored by gamgertamig, a potential first‑in‑class and best-in-class BCMAxCD3 T‑cell engager for autoimmune diseases. Backed by deep deal‑making expertise, operational flexibility, and a strong capital position, Lakefront identifies, acquires, and advances high‑quality assets with clear potential to deliver meaningful patient impact and long‑term shareholder value. For more information, visit <a href="https://www.globenewswire.com/Tracker?data=Bp4U8VUKbC9ZOITPx9ZhM_vdMT_-dVkQasbXbw2RLBQDOzf3KJY09eEdmiPq1KGHdFTef2EeEDdnaPlPuLdckR2HvWP7ONazKfaKN_mx2wxCOsAvNX5iKFE8xdoNKoII" rel="nofollow" target="_blank">https://www.lakefrontbio.com</a> or follow us on <a href="https://www.globenewswire.com/Tracker?data=rpghkh35gUTVlp1-eDEMDAVUo0Ub8Ka0PsgetHg_a3OzKENjoq1ounChZ84Vgk4Ham_YMOF_pALhnUXKGZB95UXk_4I4zrNJbS0h36JX5K2pqkYSyQ0eGiidmERJ_zGd" rel="nofollow" target="_blank">LinkedIn</a> or <a href="https://www.globenewswire.com/Tracker?data=AHS1_-wXRLDHMx2FSWFnE24qwyhuJz78jKUPdek1RlIbNoHtWm1z8ncDyVlcWg3VcqfhzvXg2dsco43B80GrCw==" rel="nofollow" target="_blank">X</a>.</p>  <p align="justify"><b>For further information, contact Lakefront Biotherapeutics:</b><br></br><b>Investor Relations </b><br></br>Sherri Spear<br></br>+1 412 522 6418<br></br><a href="https://www.globenewswire.com/Tracker?data=CATYXvioN56mywjpKlo9gmwz1nY3hRvbkrohHqNw79bep9-YcBnltJYTXa31z_d1kqljz-b9sslCClg1BqYfUaUonAiI3_98GaH_RO2JDT2-LhdwHaMxE3cFtn5Mdcnv" rel="nofollow" target="_blank">sherri.spear@lakefrontbio.com</a></p>  <p align="justify"><b>Forward-looking statements</b><br></br><i>This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements are often, but are not always, made through the use of words or phrases such as "anticipate," "expect," "plan," "estimate," "will," "continue," "aim," "intend," "future," "potential," "could," "indicate," "forward," as well as similar expressions. Forward-looking statements contained in this release include, but are not limited to, statements regarding gamgertamig’s phase 1b and proof-of-concept basket studies, statements regarding the expected timing, design and readouts of the gamgertamig studies, and statements regarding the potential benefits of gamgertamig. Forward-looking statements involve known and unknown risks, uncertainties and other factors which might cause Lakefront&#39;s actual results to be materially different from those expressed or implied by such forward-looking statements. These risks, uncertainties and other factors include, without limitation, the risk that preliminary or interim clinical results may not be replicated in ongoing or subsequent clinical trials, the risk that ongoing and future clinical studies with gamgertamig may not be completed in the currently envisaged timelines or at all, the inherent uncertainties associated with competitive developments, clinical trial and product development activities and regulatory approval requirements (including that data from the ongoing and planned clinical development program may not support registration or further development of gamgertamig due to safety, efficacy, or other reasons), Lakefront&#39;s reliance on collaborations with third parties, and that Lakefront&#39;s estimations regarding its gamgertamig clinical program and regarding the commercial potential of gamgertamig may be incorrect, as well as those risks and uncertainties identified in Lakefront&#39;s Annual Report on Form 20-F for the year ended December 31, 2025 filed with the U.S. Securities and Exchange Commission (SEC) and its subsequent filings with the SEC. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The forward-looking statements contained herein are based on management’s current expectations and beliefs and speak only as of the date hereof, and Lakefront makes no commitment to update or publicly release any revisions to forward-looking statements in order to reflect new information or subsequent events, circumstances, or changes in expectations, unless specifically required by law or regulation.</i></p> <p id="gnw_attachments_section-header">
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        <a target="_blank" href="https://ml-eu.globenewswire.com/Resource/Download/87905420-f406-43a7-ae61-fe4d319799ab">gamgertamig ODD</a>
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Lakefront Biotherapeutics disclosed that the FDA awarded Orphan Drug Designation to gamgertamig for pemphigus, complementing existing Fast Track and Orphan status for AIHA and ITP. The Gilead collaboration and ongoing Phase 1b trials with planned 2027 proof-of-concept and registrational studies outline a clearer development path and potential upside, likely supporting a near-term positive reaction in LKFT shares.

News insight

U.S. FDA Grants Orphan Drug Designation to Gamgertamig for the Treatment of Pemphigus

AI summary

Sentiment rationale

Key facts

Corporate Developments