On October 30, 2024 Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, and Advancium Health Network ("Advancium"), a public charity founded by Deerfield Management and the Deerfield Foundation to meet the needs of underserved patient populations, reported entry into an exclusive option agreement for the acquisition of ALRN-6924, a clinical-stage oncology agent developed by Aileron prior to its 2023 merger with Lung Therapeutics, Inc (Press release, Aileron Therapeutics, OCT 31, 2024, View Source [SID1234647580]).
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During the option period, Advancium intends to evaluate ALRN-6924 as a potential therapy for retinoblastoma (RB), a rare but devastating cancer of the eye, with around 300 cases diagnosed in the United States and 9,000 cases worldwide each year, nearly all in children [1]. With early intervention the disease is rarely fatal; however, non-selective chemotherapy with its attendant side effects remains the usual treatment strategy, and removal of the affected eye(s) is a frequent outcome. In contrast to chemotherapy, ALRN-6924 selectively targets MDM2 and MDMX, the endogenous inhibitors of the regulatory protein p53, to activate p53-mediated tumor suppression in cancer cells [2]. Previous studies have implicated the MDMX protein as a key driver of RB tumorigenesis [3], and ALRN-6924 is the first and only clinical-stage drug that acts on MDMX. ALRN-6924 has previously been studied in preclinical models of RB, where it showed potent, on-mechanism anti-proliferative activity in RB cell lines and was found to be highly soluble and compatible with intraocular injection [4].
Under the terms of the option agreement, Advancium paid Aileron a non-refundable fee for the exclusive option to acquire ALRN-6924 and related assets. If Advancium exercises its option, Aileron will receive an exercise payment with potential for additional development, regulatory and commercial milestone payments and sales royalties.
"This agreement is a significant landmark for our work at Advancium, as it is the first drug opportunity we are pursuing in our mission as a public charity to develop much-needed therapies for children with cancer," said Mark Veich, Chief Executive Officer of Advancium and Vice President of Philanthropy and Executive Director of the Deerfield Foundation. "We look forward to evaluating ALRN-6924 as a potential treatment for children with retinoblastoma who currently have limited care options."
"Aileron is a strong supporter of Advancium’s cause," said Brian Windsor, Ph.D., President and Chief Executive Officer of Aileron. "Although we remain focused on developing novel therapies for the treatment of orphan pulmonary and fibrosis indications, we are honored that they have chosen to partner with us in this important step towards their goal of delivering therapies to pediatric patients with high unmet need."