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SQNM (Sell Recommendation). (NIPD: Non-Invasive Prenatal Diagnostics) February, 2009. SQNM: non-invasive prenatal diagnostic test for Down’s Syndrome (T21). T21 lab test is not ready for launch Mgmt team is not ready for launch Data to support marketing is not mature Reimbursement
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SQNM (Sell Recommendation) (NIPD: Non-Invasive Prenatal Diagnostics) February, 2009
SQNM: non-invasive prenatal diagnostic test for Down’s Syndrome (T21) • T21 lab test is not ready for launch • Mgmt team is not ready for launch • Data to support marketing is not mature • Reimbursement • Competition • Royalty rates • Top holders under pressure • IP • ACOG/Physician adoption
SEQureDx for T21 is not ready for launch • SQNM has not announced if the T21 test will be based on mRNA and DNA or mRNA alone • Not commercially viable: 6-18% of samples are “no calls” • Because of legal climate, OBs and MFM specialists follow ACOG guidelines, which is years away from incorporating SQNM T21 test • OB/MFMs require tests to be published in peer-reviewed journals prior to widespread adoption. Current tests had 40,000 pt samples in published trials • The small amount of available data for SQNM T21 test was produced in-house • Independent trials will report 4Q09-1Q10, after the launch
Quantitative versus Qualitative • Qualitative: presence or absence of a dz. RhD +/-, sex determination, and x-linked dz. • Aneuploidy (T21, 18, 13): how many copies of Chr. 21 are present?
Not yet innovative • Without a SQNM test for T18, T13, and NT defects, the T21 test is additive to the prenatal work-up (ACOG guidelines), not a replacement. The current tests still need to be done (PAPP-A, beta-hCG, AFP, uE3, and inhibin A) • Goal is to replace invasive CVS/Amniocentesis with SQNM blood test; invasive tests have less fetal loss than SQNM says
Reimbursement • SQNM declined to give T21 revenue guidance and said it will likely defer 50% of 09 NIPD revenue because most payors will not have contracts at launch. This will lead to a large cash burn with no assurance of reimbursement • No sign that Blues will reimburse a test without large trials and ACOG backing, and a high “no call” rate
Competition 1. Lenetix – claims a DNA-based test using methylation-sensitive amplification of fetal nucleic acid markers2. Genzyme – nothing known about their test, but they confirmed an internal program and recently licensed EXAS IP3. PerkinElmer – implied they are working on it, but would not confirm4. Xenomics – working on a urine-based test5. Living Microsystems – working on a test to detect fetal cells in maternal blood6. BioCept – fetal cells, unknown source of cells7. Ikonisys – fetal cells, unknown source of cells8. Fluidigm – Digital PCR-based test9. Artemis – Digital PCR
IP • SQNM recently made a hostile bid for EXAS, likely to gain IP • EXAS licensed the relevant IP to GENZ and SQNM cancelled the tender offer • Many of the companies on the competition slide believe they have freedom to operate • Hard to protect a lab test when you do not yet know what it is (DNA, RNA, or both) • SQNM’s IP is untested in court
Royalties • SQNM must pay royalties of 2-4% each to 4 different parties. • This means 8-16% royalties
Trouble at the top of the holders list • Ridgeback and RA Capital are under considerable pressure based on over-exposure to SQNM. • RA: SQNM is > 40% of the fund. Cost basis $7/share. • Ridgeback has only 4 positions, including >15% of SQNM common stock
Physician adoption slow ahead of large trial data and ACOG support • Due to the litigious nature of OB/MFM, widespread adoption of prenatal testing requires incorporation into ACOG guidelines and large clinical trials published in peer-reviewed medical journals • These events are 2-3 years away, if they happen at all.
Current tests supported by large clinical trials • 2003 SURRUS: 47,000 pts • 2005 FASTER: 38,000 pts
Calendar June 2009: launch SEQureDx RNA T21 and DNAT21,T18, and T13 tests. Little data on the DNA tests. 3Q09: Publication of 1000 pt R&D study (T21 only). 4Q09: Publication of second trimester data from the RNA study in 10,000 pregnant women in high-prevalence pregnancies. (Brown University) 1Q10: Presentation of LDT validation study of 3,000-5,000 samples at SMFM 2010. Publication in a peer-reviewed journal in 2Q10 2Q10: Peer-reviewed publication of first trimester data from the RNA study and LDT study presented at SMFM.
Practical hurdles • Heterozygosity requirement at PLAC4 gene (mRNA). Add more mRNA targets? • Low “call rate” in some minority populations: African-Americans (85%) and Asians (65%) • Hemolyzed samples: blood must be drawn and stored on ice until it arrives at lab. This requirement has lead to many hemolyzed samples in the trial data
Mind the Gap: no SQNM test for T13, T18, or NT defects • The T21 testing is elegant, but SQNM has not presented data for T13, T18, or NT defects, meaning those tests still need to be done the old way (PAPP-A, beta-hCG, AFP, uE3, and inhibin A) • Until SQNM can offer a comprehensive solution (T21, T18, T13, and NT defect testing), significant market penetration for the SQNM T21 test alone is unlikely
Market Model • Total US mkt: 2.8M cases x $150 reimbursement = $420MM/year • After data publication and ACOG guidelines, SQNM will start to take a share of the current US market (PAPP-A, beta-hCG, AFP, uE3, and inhibin A) used to evaluate high-risk pts • Unlikely to gain premium pricing, per conversations with Blues (CA and MI)
Valuation • Diagnostic and genetic analysis peer group trades at EV/Sales ratio of 3x. • For SQNM, consensus 2009 sales $80MM, meaning EV $240MM versus SQNM current EV of $714MM • 60M shares out and EV of $240MM: $4/share • Take-out: 9x fwd sales (HOLX/Third Wave) would be $720MM ($12/share)
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