Please Note: Discussion of any FDA approvals for this issue are limited to new chemical entities approved to treat cancer since the previous CICR Newsletter issue (November 2019).
Merck buys ArQule: Keyed by the successes with ARQ 531, a BTK inhibitor in Phase II, Merck has acquired ArQule for $2.7 billion to strengthen its oncology pipeline. Source: Merck
E3 ligase companies proliferate: Emerging biotech company Plexium is using a proprietary DEL screening platform to take on E3 ligases, joining companies such as Arvinas, Kymera, C4, Cedilla, and Nurix in using these proteins to discover new therapies by manipulating protein degradation.
A “friendly” FDA in 2019: The FDA approved 45 new drugs in 2019, reflecting a general uptick in approvals since 2016. Cancer leads all indications, with 11 new approvals.
Sources: FierceBiotech, C&E News
FDA approval of avapritinib (Ayvakit): On January 9, 2020, Blueprint received the first FDA approval of the New Year, for the kinase inhibitor avapritinib (Ayvakit), to treat adults with unresectable or metastatic gastrointestinal stromal tumor (GIST). The compound is an inhibitor of platelet-derived growth factor receptor alpha (PDGFRA) and targets the PDGFRA exon 18 mutation, but also inhibits c-KIT kinase. The approval marks the first approval for Blueprint Medicines and provides an effective targeted therapy towards a subset of GIST patients who do not respond to the current standard of care.
Sources: FDA, BioPharmaDive, FiercePharma
FDA approval of trastuzumab deruxtecan (Enhertu): On Dec 20, 2019 Daiichi Sankyo received approval for the antibody drug conjugate fam-trastuzumab deruxtecan-nxki (Enhertu) for the treatment of metastatic breast cancer. This ADC consists of the approved HER2-directed antibody trastuzumab appended to the cytotoxic topoisomerase I inhibitor deruxtecan. ADCs are a therapeutic modality that has exploded in recent years with many new approvals and advances and the topic has recently been highlighted in the May 2019 CICR Newsletter. See related blogpost.
FDA approval of enfortumab vedotin (Padcev): On December 18, 2019, Astellas Pharma received approval for the antibody drug conjugate (ADC) enfortumab vedotin-ejfv (Padcev) to treat refractory bladder cancer. ADCs are a therapeutic modality that has exploded in recent years with many new approvals and advances and the topic has recently been highlighted in the May 2019 CICR Newsletter. This antibody-drug conjugate comprises the antimitotic drug monomethyl auristatin E (MMAE, vedotin) attached to the monoclonal antibody enfortumab.
FDA approval of zanubrutinib (Brukinsa): The BTK inhibitor zanubrutinib (Brukinsa, BeiGene) was approved on Nov 14, 2019 to treat certain patients with mantle cell lymphoma (MCL), a form of blood cancer. This is the third approval of a BTK inhibitor following ibrutinib (Pharmacyclics/Johnson & Johnson, 2013) and acalabrutinib (Acerta Pharma/Astra Zeneca, 2017). Like other approved BTK inhibitors, the molecule is an irreversible drug which contains a Michael acceptor as the covalent warhead.