Blueprint takes a long shot at a small niche

Blueprint Medicines' ability to make a success of LNG-451, a preclinical EGFR exon 20 inhibitor it obtained yesterday via its $250m purchase of Lengo Therapeutics, depends on a number of other projects failing. Aside from the two bispecifics already approved for this particular lung cancer niche there are half a dozen in mid-stage trials and a few preclinical assets that might be ahead of '451. Stifel analysts suggest that that the approved drugs for this setting “have clear deficiencies”, but add that the EGFR exon 20 non-small cell lung cancer market is worth less than $500m annually. Leerink analysts were vastly more optimistic, forecasting peak sales of $600m in the exon 20 NSCLC niche and an NPV of $1bn, if the drug makes it all the way to market. There might be an advantage for ’451 in that it is said to be brain-penetrant – none of the approved or clinical-stage projects are known to have demonstrated meaningful CNS responses. But for Blueprint to get more than a small fraction of a tiny market LNG-451 needs to show remarkable benefits over those already available, when it does eventually yield clinical data.

NSCLC therapy landscape for EGFR+ exon 20 ins
Drug Company Status
Exkivity (mobocertinib) Takeda/Ariad Approved in US for 2L treatment of pts w EGFR+ exon 20 ins
Rybrevant J&J Approved in US for 2L treatment of pts w EGFR+ exon 20 ins
Poziotinib Spectrum Ph2 Zenith20 trial failed in EGFR+ exon 20 ins cohorts
JMT101 Shanghai JMT-Bio Ph2 data due late 2023 
Tarlox (tarloxotinib) Rain Therapeutics Ph2 trial enrolment paused
CLN-081 Cullinan/Otsuka Ph1/2 trial data due mid-2022
BDTX-189 Black Diamond Therapeutics Ph1/2 trial (MasterKey-01) data due 2023
DZD9008 Dizal Pharmaceuticals Ph1/2 trial (Wu-Kong1) data due 2023
LNG-451 Blueprint Medicines Preclinical; first clinical data could come 2023
STX-EGFR-Exon20 Scorpion Therapeutics  Preclinical; could reach clinic in 2023
Source: Evaluate Pharma,, company websites.

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