VX-522 / Vertex, Moderna - LARVOL DELTA

Vertex Reports Fourth Quarter and Full Year 2023 Financial Results (Businesswire) - "Vertex on track to submit new drug applications (NDAs) to the FDA by mid-2024 for...Vanzacaftor Triple in CF....Product revenue increased 11% to $9.87 billion compared to 2022, primarily driven by the continued strong uptake of TRIKAFTA/KAFTRIO in ex-U.S. markets...Net product revenue in 2023 increased 6% to $6.04 billion in the U.S. and increased 18% to $3.83 billion outside the U.S., compared to 2022....Vertex completed dosing in the single ascending dose (SAD) portion of the Phase 1/2 study of VX-522 in people with CF. Vertex initiated the multiple ascending dose (MAD) portion of the study; screening, enrollment and dosing are underway. Vertex expects to share data from this study in late 2024 or early 2025."

FDA filing • P1/2 data • Sales • Cystic Fibrosis

Vertex Reports Second Quarter 2024 Financial Results (Businesswire) - "FDA accepted NDA for vanzacaftor triple in CF with Priority Review and PDUFA target action date of January 2, 2025; additionally, MAA submissions validated in EU and U.K....Product revenue increased 6% to $2.65 billion compared to the second quarter of 2023, primarily driven by the continued strong performance of TRIKAFTA/KAFTRIO, including in younger age groups. Net product revenue in the second quarter of 2024 increased 7% to $1.61 billion in the U.S. and increased 5% to $1.03 billion outside the U.S., compared to the second quarter of 2023....Vertex completed the single ascending dose (SAD) portion of the Phase 1/2 study of VX-522 in people with CF late last year, and the multiple ascending dose (MAD) portion of the study is ongoing. Vertex expects to complete the trial and share data from this study in the first half of 2025."

P1/2 data • PDUFA • Priority review • Sales • Cystic Fibrosis

Vertex Reports First Quarter 2024 Financial Results (Businesswire) - "Product revenue increased 13% to $2.69 billion compared to the first quarter of 2023, primarily driven in ex-U.S. markets by the continued strong uptake of TRIKAFTA/KAFTRIO...Net product revenue in the first quarter of 2024 increased 8% to $1.52 billion in the U.S. and increased 21% to $1.17 billion outside the U.S., compared to the first quarter of 2023....Vanzacaftor/tezacaftor/deutivacaftor, the next-in-class triple oral small molecule combination, in cystic fibrosis: Vertex plans to initiate a new cohort in the Phase 3 study, RIDGELINE, in the second half of 2024 in children with cystic fibrosis ages 2 to 5 years who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators....Vertex continues to enroll and dose patients in the multiple ascending dose (MAD) portion of the Phase 1/2 study of VX-522 in people with CF."

Enrollment status • Sales • Trial status • Cystic Fibrosis

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Phase classification: P1 ➔ P1/2 | Trial completion date: Jan 2024 ➔ Mar 2025 | Trial primary completion date: Jan 2024 ➔ Mar 2025

Phase classification • Trial completion date • Trial primary completion date • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Vertex Reports Fourth Quarter and Full Year Financial 2022 Results (Businesswire) - "Vertex completed enrollment in the pivotal SKYLINE 102 and SKYLINE 103 trials....Vertex expects to complete the SKYLINE studies by the end of 2023. In parallel, Vertex has also initiated a study of vanzacaftor/tezacaftor/deutivacaftor in children with CF 6 to 11 years of age, known as the RIDGELINE study....Vertex expects to complete the SAD and initiate the multiple ascending dose (MAD) study in 2023. In the U.S., the FDA has granted Fast Track designation for VX-522....Vertex has initiated a single ascending dose (SAD) clinical trial for VX-522 in people with CF, which is active and enrolling patients."

Enrollment closed • Fast track designation • New trial • Trial completion date • Cystic Fibrosis

Vertex Reports Third Quarter 2023 Financial Results (Businesswire) - "Vertex remains on track to complete the pivotal SKYLINE 102 and SKYLINE 103 studies, which are evaluating the efficacy and safety of vanzacaftor/tezacaftor/deutivacaftor relative to TRIKAFTA in patients with CF 12 years of age and older, and the RIDGELINE study of vanzacaftor/tezacaftor/deutivacaftor in children with CF 6 to 11 years of age, by the end of 2023. Vertex expects to share the results of all three studies in early 2024....Vertex is enrolling patients in a single ascending dose (SAD) clinical trial for VX-522, the CFTR mRNA therapeutic that Vertex is developing in collaboration with Moderna and expects to complete the SAD and initiate a multiple ascending dose (MAD) study by the end of 2023....Product revenue increased 6% to $2.48 billion compared to the third quarter of 2022...Net product revenue in the third quarter of 2023 increased 7% to $1.55 billion in the U.S. and increased 6% to $929 million outside the U.S., compared to the third quarter of 2022."

New trial • P3 data • Sales • Trial completion date • Cystic Fibrosis

Moderna plots lung cancer trial for Merck-partnered vax, piggybacking on Keytruda's recent win (FierceBiotech) - "One program that does this is a Vertex Pharmaceuticals-partnered cystic fibrosis therapy called VX-522....The companies launched a phase 1 trial for VX-522 in February of this year, with an estimated enrollment of nine patients and a primary completion date set for January 2024."

Trial completion date • Cystic Fibrosis • Genetic Disorders

A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF) (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Not yet recruiting ➔ Recruiting

Enrollment open • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF) (clinicaltrials.gov) - P1 | N=9 | Not yet recruiting | Sponsor: Vertex Pharmaceuticals Incorporated

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Vertex (VRTX) to Start Clinical Study on mRNA-based CF Therapy (Nasdaq) - "Vertex Pharmaceuticals VRTX announced that the FDA cleared its investigational new drug (IND) application seeking approval to start clinical studies on VX-522....Shares of Vertex have gained 43.1% in the year so far against the industry’s 19.0% decline."

Stock price • Cystic Fibrosis • Genetic Disorders

"فيرتيكس @VertexPharma ستبدأ التجارب الإكلينيكية للدواء التجريبي 🧬VX-522 لعلاج التليف الحويصليCF (نسخة🧬CFTR🧬سليمة من الحمض النووي الرايبوزيmRNAلانتاج البروتين السليم لتصحيح المشكلة) الدواء مصمم ليؤخذ عن طريق الجهاز التنفسي https://t.co/Z1VRATQ1Vi 1️⃣4️⃣" (@YAYshamsaldeen)

Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA (Businesswire) - "Vertex Pharmaceuticals Incorporated...today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX-522...Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF in the coming weeks....'Clearance of the IND represents a pivotal turning point in reaching the remaining ~5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease.'..."

IND • New trial • Cystic Fibrosis

Vertex Reports Third Quarter 2022 Financial Results (Businesswire) - "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 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....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....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 collaboration with Moderna, Vertex is developing a CFTR mRNA therapeutic to treat the underlying cause of CF....IND-enabling studies have been completed, and Vertex is on track to submit an IND for this program in Q4 2022."

Commercial • Enrollment status • IND • Regulatory • Sales • Cystic Fibrosis • Genetic Disorders

MODERNA REPORTS FOURTH QUARTER AND FISCAL YEAR 2021 FINANCIAL RESULTS AND PROVIDES BUSINESS UPDATES (Moderna Press Release) - "VXc-522 is an mRNA therapeutic being designed in collaboration with Vertex Pharmaceuticals....IND-enabling studies are underway, and Vertex expects to submit an IND for this program in 2022."

IND • Cystic Fibrosis • Genetic Disorders

Moderna Reports Third Quarter Fiscal Year 2021 Financial Results and Provides Business Updates (Businesswire) - "Introducing inhaled pulmonary therapeutics modality; IND-enabling first-in-human studies of Vertex and Moderna mRNA cystic fibrosis (CF) therapeutic (VXc-522) are ongoing in new pulmonary modality....VXc-522 is an mRNA therapeutic being designed in collaboration with Vertex Pharmaceuticals....IND-enabling studies are underway, and Vertex expects to submit an IND for this program in 2022."

IND • Preclinical • Cystic Fibrosis • Genetic Disorders

COVID-19 vaccines likely won’t reach kids before the next school year starts (Family Health Centers of San Diego) - "'We know that children can be infected with COVID-19 and can transmit it to others. To reduce the spread of this virus and control the pandemic as well as for their own safety, it's crucial that children be included in the national vaccination program, and that vaccines are made available to children as soon as possible,' said Dr. Yvonne Maldonado...in another push last month."

Media quote • Video