On October 29, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the third quarter ended September 30, 2020 and revised upward its full-year 2020 financial guidance for product revenue (Press release, Vertex Pharmaceuticals, OCT 29, 2020, View Source [SID1234569362]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"This has been another very strong quarter for Vertex with execution across our CF business and continued earnings and revenue growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are pleased with our progress toward treating all people with cystic fibrosis highlighted by several recent milestones: the early approval and encouraging start of the launch of KAFTRIO in the EU, positive TRIKAFTA data in children ages 6-11, and continued expansion of our medicines’ labels as with our recent approval of KALYDECO for infants as young as 4 months of age."
"As we extend our leadership in CF, we are also advancing a broad pipeline of innovative therapies," continued Dr. Kewalramani. "Our R&D strategy contemplates the high-risk nature of drug development and therefore includes a portfolio approach to each of our disease areas of interest. In AATD, while disappointed by the VX-814 outcome, we look forward to the VX-864 Phase 2 proof-of-concept data in the first half of 2021. Our pipeline spans multiple diseases, and multiple important clinical readouts are expected from now through the end of 2021, each of which we expect will hold transformative potential for patients and further growth for Vertex."
Product revenues increased 62% compared to the third quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S. and the uptake of our medicines outside the U.S. following the completion of several significant reimbursement agreements.
GAAP and non-GAAP net income increased compared to the third quarter of 2019, largely driven by strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of September 30, 2020 were $6.2 billion, an increase of approximately $2.3 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.
Combined GAAP R&D and SG&A expenses decreased compared to the third quarter of 2019 due to a decrease in collaboration payments.
Combined Non-GAAP R&D and SG&A expenses increased compared to the third quarter of 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other disease areas.
GAAP and Non-GAAP income taxes increased compared to the third quarter of 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.
Full-Year 2020 Financial Guidance
Vertex today revised upward its guidance for full-year 2020 product revenues. The company also adjusted its expectation for combined GAAP R&D and SG&A expenses and non-GAAP effective tax rate. Vertex’s guidance is summarized below:
Key Business Highlights:
Cystic Fibrosis (CF) R&D pipeline:
Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in supporting the extension of the eligible patient population and expansion to additional geographies and age groups.
TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)
•The European Commission granted marketing authorization for KAFTRIO to treat people with CF ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•The European Medicines Agency (EMA) validated a Type II Variation Marketing Authorization Application (MAA) for KAFTRIO that will support future indication expansion of the EU label to people with CF who have one copy of the F508del mutation.
•Vertex reported positive Phase 3 data for the elexacaftor/tezacaftor/ivacaftor triple combination in children with CF ages 6-11 who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Vertex expects to file a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020.
•The FDA accepted three sNDAs for TRIKAFTA, SYMDEKO and KALYDECO. These regulatory submissions are intended to expand the labels of these drugs to include additional people with CF who have rare CFTR mutations.
•Vertex is initiating a Phase 3 study for the elexacaftor/tezacaftor/ivacaftor triple combination in children with CF ages 2-5 who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.
SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)
•Vertex announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of SYMKEVI for the treatment of children with CF ages 6-11 with two F508del mutations or one F508del mutation and certain residual function mutations.
KALYDECO (ivacaftor)
•The FDA approved KALYDECO for use in infants with CF ages four months to less than six months old who have at least one mutation that is responsive to KALYDECO.
•Vertex announced that the EMA’s CHMP adopted a positive opinion for the label extension of KALYDECO for the treatment of infants with CF ages 4 months to less than 6 months who have at least one mutation that is responsive to KALYDECO.
Genetic therapies
•Vertex and Moderna established a new collaboration aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs that can deliver gene-editing therapies to cells in the lung for the treatment of CF.
R&D pipeline outside of CF:
Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:
Beta Thalassemia and Sickle Cell Disease:
•Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex-vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease.
•Vertex and CRISPR Therapeutics previously announced that, as of June, seven patients had been dosed across its two Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 and presented data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress from two TDT patients and one SCD patient. Additional patients have been enrolled and dosed in both TDT and
SCD studies and the company expects to report clinical data from more patients treated with CTX001 in addition to data from patients with longer follow-up in the fourth quarter.
•The EMA granted Priority Medicines (PRIME) designation to CTX001 for the treatment of severe SCD. CTX001 has also been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA and Orphan Drug Designation from the European Commission for both TDT and SCD.
Alpha-1 Antitrypsin (AAT) Deficiency:
•Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
•Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.
•In October, Vertex discontinued development of VX-814 based on the safety and pharmacokinetic profile of VX-814 observed to date in the Phase 2 clinical study.
APOL1-mediated Kidney Diseases:
•Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
•Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.
Type 1 Diabetes (T1D):
•Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
•Vertex has completed the required enabling nonclinical studies and manufacturing work to support the submission of an Investigational New Drug (IND) application to the U.S. FDA for the islet cells alone program in the fourth quarter of 2020.