Merck’s enlicitide receives FDA approval as the first oral PCSK9 inhibitor for adults with hypercholesterolaemia, GSK’s zidesamtinib earns FDA approval as a next-generation ROS1 selective inhibitor for previously treated non-small cell lung cancer, GSK’s camlipixant delivers mixed Phase 3 results in refractory chronic cough prompting programme discontinuation, and Belite Bio’s tinlarebant reports additional positive Phase 3 data in what the company describes as the first-ever demonstration of clinical efficacy in Stargardt disease type 1: the most significant clinical trial results 24 July 2026 has to offer from across the pipeline.
This week delivered a diverse set of readouts spanning cardiovascular disease, thoracic oncology, respiratory medicine, and rare inherited ophthalmology. The headline result is an FDA approval that converts a well-established injectable drug class into a once-daily pill for the first time, an advance that could reshape how clinicians approach lipid management in millions of patients worldwide. The remaining results reflect both the promise and the persistent difficulty of late-stage drug development, with a precision oncology approval offering new hope in a rare lung cancer subtype alongside a high-profile Phase 3 failure that closes a multibillion-dollar programme and a rare disease readout that strengthens the first-ever efficacy case in a blinding childhood eye condition. Here, Life Science Daily News brings you the most significant clinical trial results 24 July 2026.
FDA Approves Merck’s Enlicitide as the First Oral PCSK9 Inhibitor for Adults with Hypercholesterolaemia
This story broke on 16 July and was not included in last week’s roundup. Given its clinical significance, we are covering it here.
The FDA approved Merck’s enlicitide (Lipfendra) on 16 July 2026 as the first and only oral PCSK9 inhibitor approved to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, including those with heterozygous familial hypercholesterolaemia (HeFH). Lipfendra is a novel macrocyclic peptide administered as a once-daily 20 mg tablet, offering an oral alternative to injectable PCSK9 inhibitors such as evolocumab (Repatha), alirocumab (Praluent), and inclisiran (Leqvio), which have until now been the only approved therapies in this class.
The approval is based on two pivotal Phase 3 trials from the CORALreef clinical programme. In the CORALreef Lipids trial (NCT05952856), which randomised 2,904 patients in a 2:1 ratio, Lipfendra achieved a placebo-adjusted 56 per cent reduction in LDL-C at week 24 (95 per cent CI: -61, -51; p<0.001). In the CORALreef HeFH trial (NCT05952869), which enrolled 303 patients with genetically or clinically confirmed HeFH, Lipfendra reduced LDL-C by 59 per cent compared with placebo at week 24 (95 per cent CI: -66, -53; p<0.001). Both trials also demonstrated statistically significant reductions in non-HDL cholesterol and apolipoprotein B. The safety profile in CORALreef Lipids was similar to placebo, while in CORALreef HeFH the most commonly reported adverse reactions at higher frequencies than placebo were diarrhoea (7 per cent versus 2 per cent) and dizziness (9 per cent versus 4 per cent). An ongoing cardiovascular outcomes trial, CORALreef Outcomes (NCT06008756), has completed enrolment with more than 14,500 participants and will determine whether the LDL-C reductions translate into reduced cardiovascular morbidity and mortality.
GSK’s Zidesamtinib Receives FDA Approval for Previously Treated ROS1-Positive Non-Small Cell Lung Cancer
GSK announced on 22 July 2026 that the FDA has approved Jideytro (zidesamtinib), a ROS1 selective tyrosine kinase inhibitor, for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who have received a prior ROS1 kinase inhibitor. The approval, which came ahead of the original PDUFA target action date of 18 September 2026, follows Breakthrough Therapy and Orphan Drug Designations from the FDA. Jideytro is GSK’s first approved medicine in lung cancer, acquired through the company’s recently completed $10.6 billion acquisition of Nuvalent, Inc.
The approval is based on efficacy data from the global Phase 1/2 ARROS-1 trial (NCT05118789), which evaluated zidesamtinib in 117 patients with ROS1-positive NSCLC previously treated with a ROS1 inhibitor. The objective response rate (ORR) was 44 per cent (95 per cent CI: 34-53 per cent), with duration of response rates of 82 per cent at six months and 69 per cent at 12 months. Zidesamtinib was designed as a next-generation ROS1 selective inhibitor with broad coverage of ROS1 resistance mutations, activity against brain metastases, and a tolerable profile. The most common adverse reactions (occurring in 15 per cent or more of patients in the pooled safety population of 446 patients) included oedema, peripheral neuropathy, constipation, fatigue, and dyspnoea. Approximately 50,000 people worldwide are diagnosed annually with ROS1-positive NSCLC, a subtype that often affects non-smokers in their 40s and 50s. GSK is also advancing neladalkib (NVL-655) for ALK-altered NSCLC, which is currently under FDA review with a target decision date of 27 November 2026.
GSK Discontinues Camlipixant Programme in Refractory Chronic Cough After Mixed Phase 3 Results
This story broke on 17 July and was not included in last week’s roundup. Given its clinical significance, we are covering it here.
GSK announced on 17 July 2026 that it is discontinuing the development of camlipixant in refractory chronic cough (RCC) following mixed results across its two Phase 3 clinical trials. The CALM-1 trial met its primary endpoint, with the 50 mg twice-daily dose demonstrating statistically significant reductions in 24-hour cough frequency versus placebo at week 12. However, the CALM-2 trial did not reach statistical significance on the same primary endpoint with the same dose at week 24. The lower 25 mg twice-daily dose did not achieve statistical significance in either study, and key secondary endpoints, including a Chronic Cough Diary measure, did not meet target thresholds in either trial.
GSK stated that the limited efficacy demonstrated by camlipixant “is unlikely to transform patient care” and confirmed it will no longer progress the drug in refractory chronic cough. The safety profile across both trials was favourable, with the overall incidence and severity of adverse events similar between camlipixant and placebo groups. Camlipixant is a P2X3 receptor antagonist that GSK acquired through its approximately $2 billion purchase of Bellus Health in 2023. The discontinuation adds to the difficult track record of the P2X3 class in the United States, where Bayer’s eliapixant was discontinued during Phase 2 development and Merck’s gefapixant (Lyfnua) has been rejected twice by the FDA despite approvals in Japan, the EU, and other markets. There are currently no approved medicines for refractory chronic cough in the United States. GSK is continuing to evaluate camlipixant in a Phase 2b BALANCE trial in irritable bowel syndrome.
Belite Bio’s Tinlarebant Reports Additional Positive Phase 3 Data in Stargardt Disease Type 1 at ASRS 2026
Belite Bio announced on 20 July 2026 additional positive secondary endpoint data from its Phase 3 DRAGON trial of tinlarebant in Stargardt disease type 1 (STGD1), presented in an oral presentation at the American Society of Retina Specialists (ASRS) 2026 Annual Meeting in Montreal on 18 July 2026. Tinlarebant is described as the first therapeutic candidate to demonstrate clinical efficacy in STGD1, a rare inherited retinal disease that causes progressive vision loss, predominantly in children and young adults.
The DRAGON trial, which randomised 104 patients in a 2:1 ratio, had previously reported meeting its primary endpoint with a statistically significant 35.7 per cent reduction in retinal lesion growth compared with placebo. The newly presented data showed a marked divergence in quantitative autofluorescence (qAF), a biomarker of toxic bisretinoid accumulation: at month 25, tinlarebant-treated patients showed stable to slightly decreased qAF values (approximately 2 per cent reduction from baseline), compared with an approximately 20 per cent increase in the placebo group. Tinlarebant is a novel oral therapy designed to reduce the accumulation of vitamin A-based toxins (bisretinoids) that drive retinal degeneration in STGD1. It works by reducing serum retinol binding protein 4 levels, thereby limiting the supply of retinol to the eye. Tinlarebant was well tolerated in the trial, and Belite Bio has filed a New Drug Application with the FDA. The drug has received Breakthrough Therapy and Orphan Drug designations.
Looking Ahead
This week’s clinical trial results 24 July 2026 span cardiovascular medicine, precision oncology, respiratory drug development, and rare inherited eye disease. Enlicitide’s approval as the first oral PCSK9 inhibitor represents a significant shift in lipid management, potentially making a highly effective class of cholesterol-lowering therapy accessible to millions of patients who were unable or unwilling to use injectable options. Zidesamtinib’s approval gives patients with ROS1-positive lung cancer a next-generation treatment option designed to overcome resistance and address brain metastases, both persistent clinical challenges in this molecularly defined cancer subtype. Camlipixant’s failure is a sobering reminder that the P2X3 receptor pathway continues to resist translation into consistent clinical benefit in chronic cough, despite the clear unmet need in a condition affecting millions globally. And tinlarebant’s strengthening data package in Stargardt disease is an important development for a patient community that has never before had evidence-based pharmacological options. We will continue to track these programmes as further data emerge and regulatory decisions unfold.
