On November 15, 2022 Apollomics Inc., an innovative biopharmaceutical company committed to the discovery and development of mono- and combination-oncology therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to vebreltinib (APL-101) for the treatment of non-small cell lung cancer (NSCLC) with MET genomic tumor aberrations. The FDA granted the Orphan Drug Designation in August (Press release, Apollomics, NOV 15, 2022, View Source [SID1234624133]).
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"While NSCLC is the most common type of lung cancer, a subset of patients will have MET genomic dysregulations in their tumors which make them more resistant to treatment, presenting an unmet medical need," said Guo-Liang Yu, PhD, co-founder, Chairman and Chief Executive Officer of Apollomics. "We are pleased to have received the Orphan Drug Designation for vebreltinib, as patients need new and better treatment options. Through genomic testing, we can identify patients who will benefit most from a targeted treatment like vebreltinib. Orphan Drug Designation brings significant developmental benefits to the vebreltinib program, most notably seven-ear market exclusivity upon its approval."
Apollomics’ ongoing global Phase 2 SPARTA study is evaluating vebreltinib in patients with NSCLC and other solid tumors with MET genomic dysregulation.
Dysregulation of the c-MET tyrosine kinase receptor is implicated in the development of tumor malignancy and can arise through several mechanisms, including gene fusion and amplification, overexpression of the receptor and/or its ligand hepatocyte growth factor (HGF), and the acquisition of activating mutations. One type of the activating mutations cause exon 14 to be skipped due to aberrant splicing of MET mRNA. MET exon 14 skipping occurs in approximately 3-4% of NSCLC and has been demonstrated to be an oncogenic driver. MET amplification, another potential oncogenic driver, occurs in ~3% of newly diagnosed NSCLC, as well as in some NSCLC patients treated with targeted TKI therapy, such as EGFR inhibitors, who become treatment resistant.
The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides certain benefits including assistance in the drug development process, tax credits for clinical costs, exemption from FDA Prescription Drug User Fee Act (PDUFA) fees, and seven years of post-approval exclusivity.
As previously announced on Sept. 14, 2022, Apollomics and Maxpro Capital Acquisition Corp. ("Maxpro") (Nasdaq: JMAC, JMACU, JMACW), announced a definitive agreement for a business combination (the "Transaction" or the "Business Combination") that would result in Apollomics becoming a publicly traded company on the Nasdaq Global Market ("Nasdaq"). The Business Combination is expected to close in the first quarter of 2023 and Apollomics is expected to be listed on Nasdaq under the ticker symbol "APLM."
About vebreltinib (APL-101)
Vebreltinib is a novel small molecule kinase inhibitor that targets c-MET. It is a Type 1b class highly selective c-MET inhibitor. Vebreltinib has demonstrated strong tumor inhibitory effect in a variety of preclinical c-MET dysregulated human gastric, hepatic, pancreatic and lung cancer xenograft animal models and patient-derived xenograft models (PDX). In Phase 1 clinical trials, vebreltinib (PLB1001) demonstrated a generally well-tolerated safety profile with preliminary evidence of clinical activity in NSCLC subjects harboring a mutation that leads to MET exon 14 skipping and in secondary glioblastoma multiforme (sGBM) patients harboring MET fusion and/or exon 14 skipping with evidence of brain penetration. In China, vebreltinib is referred to as PLB1001 where it is being developed by Apollomics’ partner Beijing Pearl Biotechnology Co. Ltd. Details on the Phase 1/2 SPARTA clinical trial can be found on clinicaltrials.gov: NCT03175224. Apollomics is actively assessing the potential of investigating vebreltinib in combination with novel therapies and in a variety of tumor types in addition to developing vebreltinib as single-agent cancer therapy. Vebreltinib is currently under clinical investigation and not approved for any use anywhere in the world.
About the Phase 2 SPARTA Study
SPARTA is the Phase 2 portion of an ongoing Phase 1/2 international multicenter, open-label study evaluating the safety, pharmacokinetics, and preliminary efficacy of vebreltinib. SPARTA is assessing activity in NSCLC with a mutation that leads to MET exon 14 skipping, and across tumor types (pan-cancer) with MET amplification or fusions. SPARTA is enrolling patients into multiple cohorts. In NSCLC, the trial will evaluate both c-MET inhibitor naïve and experienced patients with mutations that lead to MET exon 14 skipping. Two cohorts will enroll patients with solid tumors with MET amplifications and fusions, including glioblastoma multiforme, the most aggressive form of brain cancer. The primary objective of SPARTA is to assess efficacy by overall response rate (ORR) and duration of response (DOR) per Response Evaluation Criteria in Solid Tumors (RECIST) or relevant evaluation criteria per tumor type. Secondary objectives include the incidence and severity of adverse events and additional efficacy measurements including time to progression, progression free survival, and overall survival.
About Non-Small Cell Lung Cancer (NSCLC)
Lung cancer is a disease in which malignant cancer cells form in the tissues of the lung. In 2017, there were an estimated 558,000 people living with lung and bronchus cancer in the United States, with new cases estimated to be over 228,000 in 2020. Lung cancer is also one of the more deadly cancers with a relative 5-year survival rate of around 20%. NSCLC is the most common type of lung cancer with about 80% to 85% of lung cancers falling into this category.