On October 27, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the third quarter ended September 30, 2022 and increased its full year 2022 revenue guidance (Press release, Vertex Pharmaceuticals, OCT 27, 2022, View Source [SID1234622489]).
"The third quarter marked another period of strong performance in the CF business and across the company," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "As we progress exa-cel towards global regulatory submission, initiate pivotal development of the acute pain program and continue the rapid advancement of multiple mid- and late-stage clinical programs, we are executing on our goal of serial innovation for patients, which will drive significant growth for the company for years to come."
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Product revenue increased 18% to $2.33 billion compared to the third quarter of 2021, primarily driven by the strong uptake of TRIKAFTA/KAFTRIO in multiple countries internationally and continued steady
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performance of TRIKAFTA in the U.S. Net product revenue in the third quarter of 2022 increased 5% to $1.46 billion in the U.S. and increased 46% to $879 million outside the U.S., compared to the third quarter of 2021.
GAAP and Non-GAAP net income increased by 9% and 14%, respectively, compared to the third quarter of 2021, primarily driven by strong revenue growth, partially offset by increased investment in our mid- and late-stage clinical pipeline.
Cash, cash equivalents and marketable securities as of September 30, 2022 were $9.8 billion, an increase of approximately $2.2 billion compared to December 31, 2021. The year-to-date increase was primarily driven by strong revenue growth and operating cash flow, partially offset by income tax payments and our acquisition of ViaCyte.
Combined GAAP and Non-GAAP R&D, Acquired IPR&D and SG&A expenses increased compared to the third quarter of 2021, due to increased investment in support of multiple programs that have advanced in mid- and late-stage clinical development and the costs to support launches of Vertex’s therapies globally.
GAAP and Non-GAAP income taxes increased compared to the third quarter of 2021, primarily due to Vertex’s increased pre-tax income.
Full Year 2022 Financial Guidance
Vertex today increased its full year 2022 product revenue guidance to reflect the uptake of KAFTRIO/TRIKAFTA in countries outside the U.S. and continued performance of TRIKAFTA in the U.S. Vertex’s guidance is summarized below:
Key Business Highlights
Cystic Fibrosis (CF) Marketed Products
Vertex anticipates the number of CF patients treated with our medicines will continue to grow, driven by the consistent performance of TRIKAFTA in the U.S., launches of KAFTRIO/TRIKAFTA outside the U.S., and additional reimbursement agreements and new approvals for the treatment of younger patients. Recent and anticipated progress includes:
•U.S. regulatory approval for ORKAMBI in children with CF 12 months to less than 24 months of age.
•Reimbursement of KAFTRIO in Italy in children with CF 6 to 11 years of age.
•Upcoming presentation of full results from the TRIKAFTA/KAFTRIO Phase 3 study in children with CF 2 to 5 years of age at the North American Cystic Fibrosis Conference (NACFC) in November 2022. Vertex is on track to submit global filings for TRIKAFTA/KAFTRIO in children with CF 2 to 5 years of age before the end of 2022.
•On track to submit global filings for KALYDECO in children with CF from 1 month to less than 4 months of age before the end of 2022.
R&D pipeline
Vertex is delivering on a diversified pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is summarized below.
Cystic Fibrosis
Vertex continues to pursue next-in-class, small molecule CFTR modulator therapies as well as new treatment options for the approximately 5,000 patients who cannot benefit from CFTR modulators.
•Vertex is conducting two Phase 3 global, randomized, double-blind, active-controlled clinical trials evaluating Vertex’s new once-daily investigational triple combination of vanzacaftor/tezacaftor/deutivacaftor, formerly known as VX-121/tezacaftor/VX-561, in patients with CF 12 years of age and older. The SKYLINE 102 and SKYLINE 103 trials will compare the efficacy and safety of vanzacaftor/tezacaftor/deutivacaftor to TRIKAFTA. Enrollment in both trials is on track to be completed before the end of 2022.
•In parallel to SKYLINE 102 and SKYLINE 103, Vertex has also initiated a study of vanzacaftor/tezacaftor/deutivacaftor in children with CF 6 to 11 years of age, which is ongoing.
•In collaboration with Moderna, Vertex is developing a CFTR mRNA therapeutic to treat the underlying cause of CF by programming cells in the lungs to produce functional CFTR protein for the treatment of the approximately 5,000 people with CF who do not produce any CFTR protein. IND-enabling studies have been completed, and Vertex is on track to submit an IND for this program in Q4 2022.
Beta Thalassemia and Sickle Cell Disease
Exagamglogene autotemcel (exa-cel), formerly known as CTX001, is a non-viral ex vivo CRISPR gene-editing therapy, which is being developed as a potential functional cure for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). Vertex is developing exa-cel in collaboration with CRISPR Therapeutics.
•Vertex concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exa-cel a rolling review, for which Vertex will submit its biologics licensing application (BLA) beginning in November 2022. Vertex expects to complete the submission by the end of Q1 2023. In the U.S., exa-cel has been granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease and Orphan Drug designations.
•European/U.K. (EMA and MHRA) submissions remain on track for Q4 2022, and exa-cel has been granted EMA Priority Medicines (PRIME) designation in Europe and Orphan Drug designation in Europe and the U.K.
•Two additional Phase 3 studies of exa-cel in pediatric patients with TDT and SCD are ongoing.
Pain (NaV1.8)
Vertex has discovered multiple selective small molecule inhibitors of NaV1.8 with the objective of creating a new class of pain medicines that have the potential to provide effective pain relief, without the limitations of opioids and other standard-of-care pain medicines.
•Vertex reached agreement with the FDA on the Phase 3 pivotal program design for its lead compound, VX-548, for moderate to severe acute pain. The pivotal program has been initiated and will enroll a total of 2,000 patients with moderate to severe acute pain across two randomized controlled trials with treatment for 48 hours following bunionectomy or abdominoplasty surgery. An additional 250-patient single-arm study will evaluate the safety and effectiveness of VX-548 in multiple other types of moderate to severe acute pain with a treatment period of up to 14 days.
•VX-548 has been granted Fast Track and Breakthrough Therapy Designation in the U.S. for moderate to severe acute pain.
•Vertex also remains on track to initiate a Phase 2 study of VX-548 in neuropathic pain by year-end 2022.
APOL1-Mediated Kidney Disease (AMKD)
Vertex has discovered multiple oral, small molecule inhibitors of APOL1, pioneering a new class of medicines that target an underlying genetic driver of kidney disease.
•Vertex continues to enroll the pivotal program for its lead compound, inaxaplin, formerly known as VX-147, in a single Phase 2/3 clinical trial in patients with AMKD.
•Inaxaplin was granted breakthrough therapy designation by the FDA for APOL1-mediated focal segmental glomerulosclerosis (FSGS), as well as Orphan Drug and PRIME designations by the EMA for AMKD.
Type 1 Diabetes (T1D)
Vertex is evaluating cell therapies using stem cell-derived islets to replace the endogenous insulin-producing islet cells that are destroyed in people with T1D, with the goal of developing a potential functional cure for this disease.
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•Proof of concept has been achieved in the VX-880 program, with the first two patients treated with half the targeted dose.
•Vertex continues to enroll patients in Part B of the Phase 1/2 study.
•Vertex remains on track to submit an IND for the cells plus device program in Q4 2022.
Alpha-1 Antitrypsin (AAT) Deficiency
Vertex is working to address the underlying genetic cause of alpha-1 antitrypsin (AAT) deficiency by developing novel small molecule correctors of Z-AAT protein folding, with the goal of increasing the secretion of functional AAT into the blood and addressing both the lung and the liver aspects of AAT deficiency.
•Vertex recently initiated a first-in-human clinical trial for VX-634, a small molecule AAT corrector in healthy volunteers. VX-634 is the first in a series of next-wave investigational molecules with significantly improved potency and drug-like properties compared to previous Vertex AAT correctors, allowing potential exploration of the full dose response.
•Additionally, Vertex will soon initiate a 48-week Phase 2 study of VX-864, a first-generation AAT corrector, to assess the impact of longer-term treatment on polymer clearance from the liver, as well as the levels of functional AAT (fAAT) in the plasma.
Duchenne Muscular Dystrophy (DMD)
Vertex is investigating a novel approach to treating DMD by delivering CRISPR/Cas9 gene-editing technology to muscle cells, with the goal of restoring near-full length dystrophin protein expression by targeting specific mutations in the dystrophin gene that cause the disease.
•IND-enabling studies for the first in vivo gene editing therapy for DMD are underway. Vertex anticipates submitting an IND in 2023.
Consistent with its overall strategy, Vertex takes a portfolio approach to all of its programs, with additional assets in CF, SCD, Beta Thalassemia, Pain, AMKD, T1D and AATD in earlier stages of development.
Investments in External Innovation
Consistent with its strategy to develop transformative medicines for serious diseases, on September 27, 2022, Vertex closed the acquisition of ViaCyte, a regenerative medicine company focused on delivering novel stem cell-derived cell replacement therapies as a potential functional cure for type 1 diabetes.