Scemblix Is First-in-Class STAMP Inhibitor for CML

by Angela Maas

On Oct. 29, the FDA approved Novartis Pharmaceuticals Corp.’s Scemblix (asciminib) for the treatment of chronic myeloid leukemia in two indications: (1) adults with Philadelphia chromosome-positive CML in chronic phase previously treated with at least two tyrosine kinase inhibitors (TKIs), which was given accelerated approval, and (2) adults with PH+ CML-CP with the T315I mutation, which was granted full approval. It is the first FDA approval of a CML therapy that is a Specifically Targeting the ABL Myristoyl Pocket (STAMP) inhibitor. The drug’s novel mechanism of action may offer an improvement over other therapies within the class, industry experts say. 

Payers, Providers Show Interest 

• For the Managed Care Oncology Index: Q2 2021, between May 18, 2021, and July 19, 2021, Zitter Insights polled 41 commercial payers with 128.1 million covered lives and 108 oncologists.
• Payers representing 82% of covered lives said they expected managing Scemblix to label within six months of its approval, while those with 10% of lives said they anticipated managing the drug more restrictively than its FDA-approved label based on its full body of clinical evidence and expert opinions. 
• In addition, 58% of oncologists said they had high or very high interest in the therapy upon approval and launch. 
• Reasons for oncologists’ interest included the drug’s good efficacy, its new innovative mechanism of action and clinical trials demonstrating safety and effectiveness. 

Scemblix Well-Positioned to Be a ‘Preferred Agent’ 

• According to Winston Wong, Pharm.D., president of W-Squared Group, “Scemblix is an example of how oncology research is becoming more targeted, showing activity against wild-type BCR-ABL1 and several mutant forms of the kinase, including the T315I mutation.” 
• Within the therapeutic class, Takeda Pharmaceuticals U.S.A., Inc.’s Iclusig (ponatinib) “may be the closest competitor from an indication perspective,” Wong continued.  
• “However, Scemblix does not contain the black box warning of Iclusig, thus will most likely be the preferred agent for this indication. History has shown us that when a product is first removed from the market and then relaunched with a black box warning, the product never fully achieves its previous utilization.” 
• Scemblix, adds Wong, “can be considered a first-in-class STAMP inhibitor, based upon its unique ability to target the ABL1 myristoyl pocket through its ability to inhibit the T315I mutation. Although Iclusig also inhibits T315I mutations, Scemblix also works very well against this and other mutations because it binds in this different domain. These more targeted TKIs bring a new dimension to the treatment of CML.”
• According to Todd Wills, managing director at CAS Custom Services, Scemblix’s “unique mechanism of action could potentially overcome resistance to treatment.” 

From Radar on Specialty Pharmacy