Merck TIGIT drug fails trial; Intellia therapy succeeds in HAE study

we provide a brief update on recent developments involving Merck & Co., Cabaletta Bio, Intellia Therapeutics, Vertex Pharmaceuticals, and Novartis.

Merck & Co. has announced another setback for its experimental cancer immunotherapy drug targeting TIGIT. The company decided to halt a Phase 3 clinical trial involving a co-formulation of vibostolimab and its well-known drug Keytruda for advanced small-cell lung cancer. This decision came after independent trial monitors indicated that the combination was not likely to improve patient survival compared to an existing immunotherapy and chemotherapy regimen. Additionally, the study revealed higher rates of side effects with the new drug combination. This marks another failure for the vibostolimab-Keytruda duo, which previously did not meet expectations in trials for lung and skin cancer.

Cabaletta Bio faced a significant challenge when shares dropped by 34% following the disclosure of a severe adverse event in a clinical trial participant. The patient, who has lupus, experienced a life-threatening neurological side effect after receiving Cabaletta Bio's cell therapy for autoimmune diseases. The condition, known as ICANS, is a recognized complication of cell therapy but was resolved quickly with antiseizure medication. Despite the incident, a committee of investigators recommended that the trial continue at the current dosage level. Nonetheless, this event has sparked debate on the feasibility of using cell therapies for treating autoimmune conditions, as highlighted by Stifel analyst Benjamin Burnett.

Intellia Therapeutics reported promising results from a Phase 2 trial of its gene-editing therapy, NTLA-2002, aimed at hereditary angioedema (HAE). Although specific data were not disclosed, the company announced that both doses tested in the trial led to significant reductions in the swelling attacks characteristic of HAE. These findings build upon earlier Phase 1 results and have prompted Intellia to select a 50-milligram dose for an upcoming Phase 3 study scheduled to begin later this year. The full results of the Phase 2 trial will be shared in the fourth quarter.

Vertex Pharmaceuticals has reached an agreement with the National Health Service (NHS) in England regarding the reimbursement of its CRISPR therapy, Casgevy, for patients with transfusion-dependent beta thalassemia. The financial details were not disclosed, but funding will come from NHS England's Innovative Medicines Fund. Discussions are still ongoing about the reimbursement for Casgevy in treating sickle cell disease. In the U.S., Casgevy is priced at $2.2 million.

The Food and Drug Administration (FDA) has granted accelerated approval to Novartis' drug Fabhalta for treating primary IgA nephropathy (IgAN), a rare condition where the immune system attacks the kidneys. This marks Fabhalta's second U.S. approval following its initial clearance for paroxysmal nocturnal hemoglobinuria late last year. The approval in IgAN was based on data showing that the treatment lowered urine protein levels, serving as a surrogate marker for organ benefit. Novartis is currently investigating whether this reduction in urine protein levels correlates with a slower decline in kidney function.

These updates from Merck & Co., Cabaletta Bio, Intellia Therapeutics, Vertex Pharmaceuticals, and Novartis highlight both the challenges and advancements within the pharmaceutical and biotechnology sectors.

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