Spinal Muscular Atrophy Landscape Analysis June 2019
SMA Market SMA drug pipeline includes programs from both SMN-enhancing and non-SMN approaches BASIC RESEARCH GLOBAL SMA TREATMENT MARKET IS FDA TO CLINICAL DEVELOPMENT APPROVAL PATIENTS SEED IDEAS PROJECTED TO GROW AT CAGR PHASE 1 PHASE 2 PHASE 3 Biogen/Ionis-Spinraza (IT) OF AROUND 20.3% Novartis-Zolgensma (IV) Novartis-Zolgensma (IT) Roche-Risdiplam (Oral) Cytokinetics-Reldesemtiv (Oral) WORTH AROUND 4.66 BILLION USD Novartis-Branaplam (Oral) BY 2022 Scholar Rock – SRK-015 (IV) *Estimates achieved from BST data from Spinraza, Zolgensma, and Risdiplam only.
Zolgensma will face challenges DURABILITY NEONATAL SCREENING IV DELIVERY Long-term effects will take time to emerge. • Newborn screening would be essential to • Zolgensma uncertainties relates to the One-time IV delivery is a major differentiator to • • treat young SMA patients with Zolgensma. unknown duration of expression of the gene Spinraza. However, the small packaging SMA has been added to the RUSP for • therapy . If expression wanes over time, capacity of AAV vectors precludes the delivery newborns as the 35 th screened disease, but it subsequent treatment pathway is unclear. in patients. is up to each state to decide which diseases If antibodies to AAV form , patient would be If Zolgensma wants to expand treatment to • • to include in their screening. unable to receive another dose of Zolgensma. older patients, they would have to change Newborns can also acquired these antibodies intrathecal delivery (currently being tested in STRONG and expected to be tested in the from their mother in utero. REACH trial). PATIENT POPULATION ACCESS PRICING AND REIMBURSEMENT Because AAV is a naturally occurring virus, • Patients will still need to travel to either • Spinraza more cost-effective in • patients presenting antibodies against it will hospitals or centers of excellence to receive presymptomatic SMA, and Zolgensma more be ineligible for treatment. treatment with Zolgesma. cost-effective in symptomatic Type I SMA. Due to limitations of AAV9 delivery, • Unlikely that payers will reimburse Spinraza • There is a risk that Zolgensma may require • intrathecal delivery will be necessary for patients for Zolgensma treatment. maintenance , and there are questions of what older patients. Limited sample size raises patients’ would have to do, and how payers concerns about generalizability of results to would react. the wider population of infants with other How will U.S. payers handle a multi-million • types of SMA . dollar price tag?
Roche’s Risdiplam will change the market SYSTEMIC PRICING PATIENT ACCESS DELIVERY POPULATION ▪ SMA is a disease of ▪ COGS will be lower ▪ A very broad patient ▪ Oral delivery means ubiquitous depletion allowing for pricing population (ALL SMA patients will not need of SMN protein . flexibility. TYPES). to go to hospitals or centers of excellence ▪ Permitting Risdiplam to ▪ Ranging in functional Is paralysis derived solely from ▪ to benefit from dysfunction of motor neurons? be priced differently status from weak non If so, then restoring SMN to Risdiplam. in different parts of ambulant to strong CNS will be enough to treat SMA. But if it stems from a the world. ambulant, and with ▪ Accessible globally broader defect of the motor varying degrees of to the broad unit, there is reason to argue scoliosis from none to population the for a systemic severe. clinical trial is repletion of the covering. protein .
Route of administration, pricing, and reimbursement SPINRAZA ZOLGESMA RISDIPLAM INTRATHECAL: IV and INTRATHECAL: ORAL: Approved for all types and ages ▪ One-time injection, no maintenance ▪ Seeking approval for all types and ▪ (treatment of 6,000 patients). necessary (may require maintenance). ages. 6 injections (12mg) in first year. ▪ Small molecule, which given orally, ▪ Maintenance injection every 4 months ▪ PRICING: can cross BBB. (12mg). $2.125M for the one-time injection. ▪ Dosing regimen is the same ▪ Insurance companies will offer ▪ regardless of age, but changing it reimbursement with the risk that PRICING: may complicate pricing. patients may need maintenance. Pricing will rely on optimal dosing ▪ chosen in Part 1 of the clinical trial. PRICING: Flexibility to price it at a 10-15% ▪ $125k per injection. ▪ discount to take the market share, $4.1M over 10 years. ▪ especially outside of the U.S. Reimbursement for only Type I and II. ▪ ROUTE OF ADMINISTRATION: PRICING SOLUTIONS: Route of administration will have a great impact on patients/caregivers and FAIR VALUE RECOGNITION: Based on lifetime cost-time effectiveness ▪ ▪ could alleviate SMA patients’ healthcare burden. timeframe, based on patient QOL and life extension. It will also allow payers to look favorable on Risdiplam, since the oral SHARED RISK: Payment only if drug works, up to 80% of price is at risk. ▪ ▪ administration will save on hospital cost. AFFORDABILITY: Spread payment over up to 5 yrs., no increase over CPI. ▪ Neurologists administering Spinraza face difficulties especially in patients with CHANGE IN PAYER/PORTABILITY: Contract negotiation, mutual ▪ ▪ compromised spinal cords. recognition. When possible, physicians will most likely want to treat with gene therapy, and MEDICAID BEST PRACTICES RULES: Innovative federal/state pilot ▪ ▪ supplement with Spinraza/Risdiplam when needed, but it is unlikely payers will programs or waivers. approve of this treatment pathway. TRACKING OUTCOMES: Through claims and registry data. ▪
Why Choose in Thought as a Partner? SMA Our proprietary inVision Our Spinal Muscular Atrophy We are a life science Our senior principals have consulting firm made up of Wall Street analyst platform provides a capabilities include : MDs, PhDs, and MBAs with experience. customized environment. ✓ Competitive Intelligence Monitoring Reports deep therapeutic area ✓ State of the Art Landscape We answer questions quickly, inVision allows clients to have all of Reports expertise. always considering the impact to their critical intelligence in one ✓ Trial Design Analyses place, customized for their ✓ Deep Dive assessments of various stakeholders by putting the Our analysts leverage scientific and companies, drugs, and MOAs clinical data, medical opinion, and particular key questions and topics, medical knowledge as well as ✓ Licensing opportunity identification and due diligence company business maneuvers into and accessible on demand. business acumen to address ✓ Conference Coverage an actionable business context. strategic problems in healthcare. ✓ KOL interviews ✓ Revenue Forecast Modeling ✓ Probability of Approval Modeling ✓ Target Product Profiles
For More Information Michelle Rivera, PhD MBA Ben Weintraub, PhD Analyst (646) 331-9234 (917) 575-1372 bweintraub@inthought.com mrivera@inthought.com
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