VERTEX PHARMACEUTICALS INC annual report, FY2013

Vertex Pharmaceuticals — 2013 Form 10-K

Reporting period: Fiscal year ended December 31, 2013. This is an annual filing, not a quarterly report; fourth-quarter figures are noted where relevant. Amounts are in U.S. dollars.

Business context

Vertex discovers, develops, manufactures and commercializes specialty small-molecule medicines. Its marketed products were KALYDECO (ivacaftor) for certain cystic fibrosis (CF) patients and INCIVEK (telaprevir) for adults with genotype 1 hepatitis C (HCV). Management was shifting investment toward CF and research programs as HCV competition rapidly eroded INCIVEK sales.

Financial results and liquidity

Metric20132012Change / context
Total revenue$1.212 billion$1.527 billionDown 21%
Net product revenue$837.6 million$1.333 billionDown 37%
INCIVEK revenue$466.4 million$1.162 billionDown $695.5 million
KALYDECO revenue$371.3 million$171.6 millionUp $199.6 million
Royalty revenue$156.6 million$141.5 millionJanssen INCIVO royalties end after Q4 2013 under amendment
Collaborative revenue$217.7 million$52.1 millionIncludes Janssen amendment-related revenue
Operating loss$903.4 millionOperating income of $2.3 millionIncludes $663.5 million in intangible-asset impairments
Net loss attributable to Vertex$445.0 million$107.0 millionLoss per diluted share: $1.98 vs. $0.50
Operating cash flow$(51.6) million$267.8 millionCash used in operations in 2013

2013 total costs and expenses were $2.115 billion, including $918.8 million of R&D expense, $362.3 million of sales, general and administrative expense, $40.5 million of restructuring expense, and $663.5 million of impairment charges. Product revenue fell sharply despite KALYDECO growth, primarily because of INCIVEK’s decline. The filing does not provide a single consolidated gross-margin percentage.

Cash, cash equivalents and marketable securities totaled $1.465 billion at year-end, up $143.9 million from 2012. Current assets were $1.589 billion and current liabilities $397.8 million. Cash and cash equivalents alone were $569.3 million. Investing cash flow was $(54.1) million; financing cash flow was $180.9 million, including $265.9 million from employee-plan stock issuances, offset by lease-related payments and other items. Cash and cash equivalents increased $79.9 million during the year.

Vertex had no convertible notes outstanding at year-end: $400 million principal of 3.35% notes due 2015 was converted into stock or redeemed in 2013. The balance sheet included a $440.9 million construction-financing lease obligation and $65.6 million of capital lease obligations. Total contractual commitments were $1.468 billion, substantially including long-term headquarters and facility leases.

Material changes and unusual items

  • In November 2013, Janssen paid Vertex $152 million for a fully paid license to market INCIVO in its territories; Vertex’s royalty rights ended after Q4 2013. The amendment generated $182.4 million of Q4 collaborative revenue, including accelerated recognition of prior deferred revenue. Vertex expects no significant future Janssen collaborative revenue.
  • Vertex recorded impairment charges of $412.9 million for VX-222 and $250.6 million for its VX-135 HCV nucleotide-analogue program. The company concluded both assets had zero fair value amid clinical, competitive and development concerns.
  • Vertex deconsolidated Alios BioPharma as of December 31, 2013 after the VX-135 impairment; the deconsolidation produced a $68.2 million gain attributable to Vertex. Alios-related consolidation and noncontrolling-interest accounting materially affected reported results.
  • In October 2013, Vertex announced a workforce reduction of about 370 positions, approximately 15% of its workforce, and recorded $39.0 million of related restructuring charges. Year-end headcount was approximately 1,800, down 18% from 2012.
  • Vertex converted approximately $400 million of notes into 8.3 million common shares, reducing future principal and interest payments but contributing to dilution. Shares outstanding rose to 233.8 million at year-end from 217.3 million.
  • Q4 revenue was $351.2 million, including $185.4 million of collaborative revenue, while net loss attributable to Vertex was $44.3 million of income. That result reflected unusual Alios noncontrolling-interest accounting and should not be treated as a recurring operating trend.

Outlook, management commentary and key risks

  • Management expected a substantial operating loss in 2014, lower total revenue versus 2013, and only a small portion of revenue from INCIVEK. It expected KALYDECO growth but did not expect it to offset the HCV-related decline in the near term.
  • Management said existing cash, cash equivalents and marketable securities plus product cash flows should fund operations for at least the next 12 months; future liquidity depends on product revenue, clinical outcomes and spending needs.
  • Vertex expected mid-2014 data from the TRAFFIC and TRANSPORT Phase 3 trials of lumacaftor plus ivacaftor in F508del-homozygous CF patients. If successful, it planned U.S. and EU filings in the second half of 2014. These were expectations, not guaranteed outcomes.
  • An ivacaftor label-expansion submission for specified CF mutations was under FDA review, with a March 27, 2014 target date. The R117H Phase 3 trial did not meet its primary endpoint; management planned to discuss adult subgroup findings with the FDA. Pediatric and residual-function trial data were expected in Q2 2014.
  • The FDA had placed VX-135 on partial clinical hold in the United States after reversible liver-enzyme elevations at a 400 mg dose with ribavirin in a European Phase 2 trial. The hold, weak competitive positioning and need for collaborators put further development at risk.
  • Management highlighted competition from newer HCV treatments, reimbursement and pricing uncertainty for KALYDECO, clinical and regulatory execution risk, reliance on third-party and sole-source manufacturers, and uncertainty around the novel continuous-manufacturing process planned for lumacaftor/ivacaftor. Failure or delay in validating that process could delay a launch.
  • Other disclosed matters include a shareholder securities class action seeking unspecified damages; the company said it would defend the case and had recorded no reserve. No material contingent liabilities were accrued at year-end. The financial statements received an unqualified audit opinion, and management and the auditor reported effective internal control over financial reporting.

Important facts for investors to verify

  • Whether lumacaftor/ivacaftor Phase 3 results, regulatory decisions, manufacturing validation and reimbursement support the expected launch opportunity.
  • How quickly INCIVEK revenue declines and whether KALYDECO growth, including international reimbursement and label expansion, can compensate.
  • Whether the VX-135 clinical hold is lifted and whether any viable partner or development plan emerges.
  • The recurring cash burn and the effects of headquarters leases, restructuring costs and other contractual commitments on liquidity.
  • How much future reported performance depends on one-time collaboration, impairment, tax and Alios-related accounting items rather than product sales.
  • The status and potential financial exposure of the securities class action, and any changes in manufacturing or supply-chain risks.