Last Updated 2/23/2023

Blueprint Medicines


Blueprint is focused on targeted therapies for oncology. They have their main franchise around Mast Cell disorders with Ayvakit and Elenestinib. They are already commercial for Ayvakit for Advanced SM. They should be commercial in 2023 for Indolent SM. Elenestinib should expand this franchise into more Mast Cells applications. Their second franchise is around Lung Cancer targeting EGFR with 3 drugs in development. They have BLU-945, BLU-525 and BLU-451 all targeting different variants of EGFR exon 19, 20 and 21. They just started a third franchise around Breast Cancer with a CDK2 inhibitor. All of these are large areas of oncology. Their main focus has been on kinases that drive cell growth in cancer. They use mostly small molecule inhibitors, but recently started looking at protein degraders.


Kate Haviland only took over last year as CEO, but she had been the COO under the management of Jeff Albers. Together, they have developed a winning commercial biotech company. I think she is doing a good job and moving the pipeline forward and bringing more drugs to commercial use.


Ayvakit is a PDGFRa/KIT inhibitor which is already commercial for D842V GIST and D816V Advanced SM. This is a fully mature market for them with 75% of patients on Ayvakit. They will expand this drug if they get indolent SM approval. There has been very strong data to support use of Ayvakit in both Advanced and Indolent SM.

Elenestinib is a pure KIT D816V inhibitor which is designed to be a pure target of Mast cell disorders. They had good safety data in phase 1 which showed similar side effects to placebo. They should be presenting the Phase 1 data in indolent SM in 2H of 2023

BLU-945 is an EGFR inhibitor which targets the L858R mutation of Exon 21 and the Exon 19 deletion. This is the most common mutation of EGFR with 41% of patients being Exon 19 deletion and 32% being L858R Exon 21 mutations. This drug targets the T790M and C797S mutations caused by Osimertinib. It's currently in phase 1.

BLU-451 is their Exon 20 insertion EGFR drug they went out and bought last year. Exon 20 insertions account for about 12% of EGFR mutations. We haven't seen any data from this program yet, but we should see it this year.

BLU-525 is their second L858 drug focused on Exon 21 mutations and Exon 19 deletions. This drug targets the C797X mutations. It is designed to be more balanced at hitting both variants than BLU-945. This is in IND enabling studies and should be ready for and IND this year.


Cash $1.078 billion

Ayvakit has about $200 million in annual sales right now with potential of upward of $750 million or more should they get approval in indolent SM which could have around 7,500 patients in the US. I would think they get 60% of those for 4,500. At a price of $100,000, that would reach $450 million sales. If you give them even 5x sales on their $200 million sales, this comes to $1 billion worth of value.

Elenestinib could expand the applications into Mast Cell disorders to include Advanced and Indolent SM along with other Mast Cell disorders. This could expand the population to another 10,000 patients. Even at a $75,000 price, it would be worth another $750 million sales for them in Mast Cells. I would only give this a 1x multiplier for being in phase 1. That makes it worth $750 million value.

BLU-945 affects the most common forms of resistance to Osimertinib which does upward of $5 billion a year in sales. That makes the potential of this drug very big. The whole EGFR space is large with upward of 30% of Lung Cancer patients having an EGFR mutation. I think this could do upward of $1 billion sales if the data is strong. I would give it a .1 multiplier since its still needs data. That makes it a value of $100 million.

BLU-451 affects the exon 20 insertions which is 12% of EGFR. That would be about 7,000 EGFR patients with this mutation in Lung Cancer. Even at a $250,000 price, that could be worth about $500 million or more. I would give it a .1 multiplier until we see the data. That makes it worth $50 million.

BLU-525 affects the Exon 21 L858R mutation and Exon 19 deletions. It is focused on the C797X mutation. I am not sure yet how this asset distinguishes itself from BLU-945, but we should find out as the data comes out. I think it could offer some additional treatment options. I would only give this about $250 million in sales until I see how they are developing this. With a .1 multiplier, that comes to $25 million value.

All in, that is a $3.003 billion market cap. Based on the 60 million shares outstanding, that comes to $50.05.


Roche has cancelled their partnership for Gavretto and will transition the asset back to Blueprint over the next year.

CStone has a partnership for China region on Ayvakit, Gavretto and Fisogatinib which they pay a royalty in the mid teens to low twenties to Blueprint on sales in China.

Zai Lab has a partnership for China region on BLU-945 and BLU-525 which they pay a royalty in the low to mid teens to Blueprint on sales in the China region.

Clementia has a license for BLU-782 worldwide on which they will pay a royalty in the low to mid teens to Blueprint on all sales.

IDRx has a worldwide license for a KIT13 inhibitor on which they will pay a royalty up to the low teens to Blueprint on all sales.

Royalty Pharma purchased the right to all the ex-US Gavretto royalties that Blueprint gets from Roche outside the US.

Sixth Street purchased the right to 9.75% of all Ayvakit and Elenestinib sales up to a max of $900 million sales per year.

Proteovant licensed to Blueprint technology for doing Targeted Protein Degraders. They are required to pay a royalty to Proteovant on commercial sales of in the range of mid to high single digits.


Ayvakit Indolent SM PDUFA date of May 22, 2023

Present Data at AAAAI on Feb 27 at 8am from Pioneer Trial

Elenestinib Phase 1 data in 2H of 2023

BLU-945 Phase 1 data in 2H of 2023

BLU-451 Dose Escalation data in 1H of 2023

BLU-525 Submit IND 1H of 2023

BLU-222 Clinical Hold due to SAE dose escalation data in 1H of 2023

Nominate the KIT Wildtype drug by mid 2023

Data Readouts

Ayvakit Advanced SM Long Term Data

Ayvakit Indolent SM phase 2 data

BLU-945 Early data

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* These are my Opinions and Estimates. They should not be considered financial advice.