Innovative Therapies Changing Cancer Treatment Landscape
Blueprint Medicines (BPMC) is revolutionizing cancer treatment with groundbreaking drugs like avapritinib for GIST and SM, BLU-667 for RET-mutated cancers, showcasing superior efficacy and tolerability. The company's strategic approach and promising pipeline suggest BPMC is undervalued, offering potential blockbuster status in multiple indications.
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Presentation Transcript
THE FORMULA Blueprint Medicines (BPMC)
THE SUM OF BLUEPRINTS PIECES EXCEEDS CURRENT VALUATION Institutions include Perceptive, venBio, & Deerfield Blueprint plans to submit GIST-related NDAs this year Blueprint s avapritinib is a game-changer in GIST; BLU-667 provides therapeutic differentiation in MTC & NSCLC The sum of Blueprint s pieces exceeds current valuation PT: $115
AVAPRITINIB FOR GASTROINTESTINAL STROMA TUMORS (GIST) Avapritinib for 4th-line GIST: 20% ORR (vs. ORR < 5% for current treatments & 9% for relevant competition) Avapritinib for D842V mutant PDGFRA GIST: 84% ORR Avapritinib s safety profile is well-understood & tolerable Additionally, Avapritinib will likely be utilized, to a relevant extent, in 2nd& 3rd-line GIST
AVAPRITINIB FOR ADVANCED SYSTEMIC MASTOCYTOSIS ~95% of patients have a KIT D816V mutations, making avapritinib a prime candidate The current treatment landscape for SM lacks targeted therapies; current treatment algorithm suggests patients pursue a clinical trial (like avapritinib) over standard-of-care (Midostaurin). Avapritinib appears to have superior efficacy (69%-75% for midostaurin vs. 83%) and tolerability (22% discontinuation rate due to adverse events for midostaurin vs. 4%). Avapritinib for advanced SM achieved breakthrough therapy, suggesting FDA officials believe it provides substantial improvement over existing therapies
BLU-667 FOR RET-MUTATED CANCERS Cancers with RET-mutations are currently treated with multi- target therapies (which limits efficacy & tolerability) BLU-667 has shown superior efficacy & tolerability in RET- mutated NSCLC & MTC to current treatment and comparable data versus relevant clinical competition
SUM OF PARTS (667 & AVAPRITINIB) SUGGEST BPMC IS UNDERVALUED Avapritinib may achieve blockbuster status in GIST and SM BLU-667 may be a game-changer in RET-mutated cancers (NSCLC, MTC) Both drugs have preclinical + clinical profiles that de-risk upcoming clinical & regulatory readouts
~425 patients will have D842V mutant PDGFRA GIST $150k/treatment x 318 patients (75% penetration) = ~$50M in peak annual sales ~6,000 patients will be 4th-line GIST VALUING AVAPRITINIB FOR GIST $150k/treatment x 4,500 patients (75% penetration) = ~$675M in peak annual sales Note: the number of 4th-line GIST patients will probably be reduced now that drugs in clinical development are procuring better responses, to accommodate for this, I do not include any revenue for BPMC beyond PDGFRA-mutated & 4th-line GIST. $675M + $50M = $725M times a multiple of 4 = $2.9B ($65/share)
~2,500 advanced systemic mastocystosis patients VALUING AVAPRITINIB FOR ADVANCED SYSTEMIC MASTOCYTOSIS $150k/treatment x 1,250 patients (50% penetration) = ~$185M in peak annual sales $185M in peak annual sales times a multiple of 4 = $740M (~$15/share)
There are no FDA-approved drugs for RET-mutated NSCLC, which accounts for ~2% of the total NSCLC population While preliminary, 667 has procured superior efficacy, providing clear benefit over existing treatments and developing ones (50% ORR vs. ~25% for multi-targeted drugs like cabozantinib) VALUING BLU- 667 FOR RET- MUTATED NSCLC Loxo Oncology has procured an ORR of 65%. It is, however, too early to get a grasp on which drug is better . ~7,500 patients will have RET-mutated NSCLC $150k/treatment x 1,875 patients (25%) = ~$225M in peak annual sales $225M times a multiple of 4 = $900M (~$20/share)
MTC is a rare form of thyroid cancer RET-mutations account for ~60% of cases Targeted-oncology (BPMC, LOXO) provide obvious benefits over existing therapies ~750 patients will be appropriate candidates for RET-inhibitors 375 patients (50% penetration/split with Loxo) x $150k = ~$55M in peak annual sales $55M times a multiple of 4 = $220M ($5/share) VALUING BLU-667 FOR MEDULLARY THYROID CANCER
Cash per share: $10 GIST: $65/share ASM: $15/share NSCLC: $20/share MTC: $5/share = $115/share SUM OF PARTS VALUATION
LIMITATIONS The majority of risk involved with a Blueprint investment will involve clinical & commercial competition from the likes of Loxo and DCPH Any of BPMCs trials may result in failure, safety signals, etc. that may adversely impact its valuation The preceding sum of parts valuation is, admittedly, simplistic & may overestimate BPMCs potential
SUMMARY Blueprint is amongst the leaders in targeted-oncology & supported by three of the five top biotech funds we are following Starting this year, Blueprint will transition to a commercial biotechnology company Both avapritinib & BLU-667 provide obvious efficacy and tolerability benefits over existing therapies in specific mutations that de-risk upcoming clinical and regulatory catalysts I believe a sum of parts valuation in BPMC reveals an investment opportunity that is risk-averse & presents with upside compared to the average biotechnology investment (benchmarks: XBI, IBB)
The intention of this presentation is to provide insight, not investment advice. While the information provided in this presentation is intended to be factual, there is no guarantee and prospective investors are encouraged to do their own fact-checking and research before investing in a company. One must also consider one's own financial standings, risk tolerance, portfolio diversification, etc. before making a decision to buy shares in a company. The preceding information is abridged, purely supplemental, and should not be construed as investment advice. DISCLAIMERS
