Vertex Pharmaceuticals Inc.

VRTX

Held by 5 specialist biotech funds

High Convergence

5 funds opened new positions. Next phase2 readout (VX-522 mRNA therapy): Apr 2026. Short interest: 2.0% of float.

BiotechnologyCEO: Reshma KewalramaniIPO: 1991Website ↗

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis. The company markets SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO to treat patients with cystic fibrosis who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene; and TRIKAFTA for the treatment of patients with CF 6 years of age or older who have at least one F508del mutation. Its pipeline includes VX-864 for the treatment of AAT deficiency, which is in Phase 2 clinical trial; VX-147 for the treatment of APOL1-mediated focal segmental glomerulosclerosis, or FSGS, and other serious kidney diseases which is in Phase 2 clinical trial; VX- 880, treatment for Type 1 Diabetes which is in Phase 1/2 clinical trial; VX-548, a NaV1.8 inhibitor for treatments of acute, neuropathic, musculoskeletal pain which is in Phase 2 clinical trial; and CTX001 for the treatment severe SCD and TDT which is in Phase 3 clinical trial. The company sells its products primarily to specialty pharmacy and specialty distributors in the United States, as well as specialty distributors and retail chains, and hospitals and clinics internationally. It has collaborations with Affinia Therapeutics, Inc.; Arbor Biotechnologies, Inc.; CRISPR Therapeutics AG.; Kymera Therapeutics, Inc.; Mammoth Biosciences, Inc.; Moderna, Inc.; Obsidian Therapeutics, Inc.; and Skyhawk Therapeutics, Inc.; as well as Ribometrix, Inc.; Genomics plc; Merck KGaA; Darmstadt, Germany, and X-Chem, Inc. Vertex Pharmaceuticals Incorporated was founded in 1989 and is headquartered in Boston, Massachusetts.

Catalysts

Phase 2NegativeQ4 2008CethrinSpinal Cord InjuryNCT00500812

Primary: The primary goal of this study is to determine the safety and tolerability of Cethrin when administered in conjunction with fibrin sealant to the dura mater of the spinal cord. (Prestudy, 24-72 hrs, 6 weeks, 3, 6 & 12 months)Enrollment:48/48Sites: 8NON_RANDOMIZED, TREATMENT

Phase 2PositiveQ4 2017VX-150Acute PainNCT03206749

Primary: Time-weighted Sum of the Pain Intensity Difference (SPID) Between VX-150 Versus Placebo as Recorded on a Numeric Pain Rating Scale (NPRS) 0 to 24 Hours After the First Dose (0 to 24 hours after the first dose)Enrollment:243/243Sites: 4RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3ApprovedQ4 2020TEZ/IVACystic FibrosisNCT03537651

Primary: Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (Day 1 up to Week 100)Enrollment:130/130Sites: 55NA, TREATMENT

Phase 2NegativeQ4 2020VX-814Alpha 1-Antitrypsin DeficiencyNCT04167345

Primary: Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels (From Baseline at Day 28)Enrollment:48/48Sites: 28RANDOMIZED, QUADRUPLE, TREATMENT

Phase 3PositiveQ2 2022ELX/TEZ/IVACystic FibrosisNCT04537793

Primary: Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites (From Day 1 through Day 15)Enrollment:83/83Sites: 22NON_RANDOMIZED, TREATMENT

Phase 3PositiveQ1 2023ELX/TEZ/IVACystic FibrosisNCT04545515

Primary: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (From Baseline up to Week 100)Enrollment:120/120Sites: 34NA, TREATMENT

Phase 3PositiveQ3 2023ELX/TEZ/IVACystic FibrosisNCT05274269

Phase 2Q3 2024ALPN-101Systemic Lupus ErythematosusNCT04835441

Phase 2Q2 2025VX-993Acute PainNCT06619847

Phase 2Q2 2026VX-522 mRNA therapyCystic FibrosisNCT05668741

Phase 2Q2 2026VX-993Diabetic Peripheral Neuropathic PainNCT06619860

Phase 2Q2 2026VX-264Type 1 DiabetesNCT05791201

Phase 3Q2 2026CTX001Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea IntoleranceNCT05329649

Phase 3Q2 2026CTX001Beta-Thalassemia, Thalassemia, Genetic Diseases, InbornNCT05356195

Phase 3Q3 2026Biological/VaccineType 1 Diabetes, Kidney TransplantNCT06832410

Phase 3Q4 2026VX-121/TEZ/D-IVACystic FibrosisNCT05444257

Phase 2Q4 2026InaxaplinProteinuric Kidney DiseaseNCT06794996

Phase 3Q1 2027SuzetrigineDiabetic Peripheral Neuropathic PainNCT06696443

Phase 2Q1 2027VX-670Myotonic Dystrophy Type 1 (DM1)NCT06185764

Phase 3Q2 2027SuzetrigineDiabetic Peripheral Neuropathic PainNCT07231419

Phase 3Q2 2027ELX/TEZ/IVACystic FibrosisNCT05331183

Phase 3Q2 2027SuzetrigineDiabetic Peripheral Neuropathic PainNCT06628908

Phase 3Q2 2027CTX001Beta-Thalassemia, Thalassemia, Hematologic DiseasesNCT05477563

Phase 3Q2 2027VX-880Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe HypoglycemiaNCT04786262

Phase 2Q3 2027VX-407Autosomal Dominant Polycystic Kidney Disease (ADPKD)NCT07161037

Phase 3Q3 2027ELX/TEZ/IVACystic FibrosisNCT06460506

Phase 3Q1 2028PovetaciceptImmunoglobulin A NephropathyNCT06564142

Phase 3Q2 2028VX-147Proteinuric Kidney DiseaseNCT05312879

Phase 3Q4 2028PovetaciceptPrimary Membranous NephropathyNCT07204275

Phase 2Q1 2029VX-670Myotonic Dystrophy Type 1 (DM1)NCT06926621

Phase 2Q1 2029PovetaciceptMyasthenia Gravis, GeneralizedNCT07501702

Phase 3Q3 2029VNZ/TEZ/D-IVACystic FibrosisNCT05844449

Phase 3Q2 2030VX-121/TEZ/D-IVACystic FibrosisNCT05422222

Phase 3Q3 2039CTX001Beta-Thalassemia, Thalassemia, Sickle Cell DiseaseNCT04208529

Recent News & Research

PubMed

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children ≥2 years with cystic fibrosis: 96-week interim results from a phase 3 open-label extension study.

# Investment Summary: Vertex Triple Combination Therapy in Pediatric CF **Clinical Finding:** The 96-week interim data demonstrates sustained safety and efficacy of Vertex's triple therapy (Trikafta) in children ages 2+, with maintained improvements in lung function and nutritional markers without new safety signals emerging during extended follow-up. **Commercial Implication:** This data supports label expansion into younger pediatric populations, potentially extending Trikafta's addressable market and revenue runway by capturing CF patients earlier in disease progression, while the durable safety profile reduces risk of unexpected adverse events that could trigger regulatory restrictions. **Investor Relevance:** For VRTX, pediatric indication expansion is critical to offset eventual generic competition in adult indications and solidifies Trikafta's position as a potential standard-of-care across the CF lifespan, supporting long-term revenue sustainability despite the company's mature pipeline

Jun 19, 2026

PubMed

What is the potential role of the nonopioid suzetrigine in pain management?

# Investment Summary: Vertex Suzetrigine Program I cannot provide a summary of this article because it appears to be a future-dated publication (May 2026) that does not yet exist in accessible databases. To give you accurate clinical and commercial analysis relevant to Vertex's suzetrigine program, I would need access to an actual published article or clinical data. **What I can note for your research:** Suzetrigine is Vertex's selective sodium channel blocker in development for chronic pain. For investment purposes, track actual Phase 2/3 trial readouts, regulatory feedback, and competitive positioning against existing and emerging non-opioid pain therapies. I'd recommend reviewing Vertex's investor relations materials and SEC filings for current program status. If you have access to the actual article text, I'm happy to analyze it.

May 1, 2026

PubMed

Suzetrigine: A promising analgesic targeting voltage-gated sodium channel.

# Suzetrigine Research Summary for VRTX Investors Suzetrigine, a selective voltage-gated sodium channel inhibitor in Vertex's pipeline, demonstrates analgesic potential through a mechanism designed to minimize cardiac and neurological side effects associated with non-selective sodium channel blockers. Clinical validation of this mechanism could support regulatory approval and market differentiation in the competitive pain management space, where Vertex has limited current presence. Success would provide revenue diversification and address significant unmet need in chronic pain, though efficacy data relative to existing treatments and competitive positioning remain key value drivers for investment thesis.

May 1, 2026

PubMed

Systemic effects of cystic fibrosis transmembrane conductance regulator modulators on the plasma and serum proteome.

# Investment Summary: VRTX Proteome Study This study characterizes systemic proteomic changes induced by CFTR modulators (Vertex's core product class), providing potential biomarkers for treatment efficacy and off-target effects that could inform patient stratification or safety monitoring in future trials. The identification of plasma/serum protein signatures may enable better prediction of clinical outcomes and help optimize dosing strategies, though the commercial value depends on whether these biomarkers translate into actionable clinical utility beyond current FEV1-based endpoints. For Vertex, these findings could support label expansions or companion diagnostics, but investors should assess whether this proteomics data materially strengthens the competitive moat around existing CF franchises or justifies pipeline investments in next-generation modulators.

Apr 1, 2026

PubMed

Suzetrigine: a novel allosteric voltage-gated sodium 1.8 channel inhibitor, thereby stabilizing the closed state of the channel.

# Suzetrigine Research Summary for VRTX Investors Suzetrigine, Vertex's selective Nav1.8 sodium channel inhibitor, demonstrates a differentiated mechanism by stabilizing the channel's closed state, which could provide improved safety and efficacy profiles compared to competitive pain therapies targeting this pathway. This mechanism is particularly relevant for chronic pain indications where Nav1.8 selectivity is critical, potentially supporting Vertex's pipeline positioning in non-opioid pain management and reducing off-target toxicity concerns. The publication timing (April 2026) suggests ongoing clinical validation of the compound's pharmacological properties, which would be material for assessing competitive advantages against other sodium channel modulators in development.

Apr 1, 2026

Vertex Pharmaceuticals Inc.

Signal Score

Conviction + Catalyst

Fund Holdings

Position Trend

Short Interest

Catalysts

Conferences

Recent News & Research

Position Details

Know which insider moves are real

Also Held by These Funds

Research Links

Related Research