RLAY (Best Idea) - BUY - Positive KOL Feedback On Vascular Anomalies Data, And Model Update; PT $26

Research evidence excerpt

RLAY (Best Idea) - BUY - Positive KOL Feedback On Vascular Anomalies Data, And Model Update; PT $26

of these patients

are symptomatic enough at any one time to get systemic treatment. For the 50% of his

macrocystic patients, his approach is a referal to interventional radiology for fluid draining

and sclerotherapy. However, he notes interventional radiologists are now advocating for 3 to

6 months of neoadjuvant systemic treatment to shrink the lesions and make sclerotherapy

easier, which opens up a new opportunity for RLAY’s drug. He doesn’t think systemic

therapies will replace sclerotherapy because the drugs have long-term toxicity and costs.

However, sclerotherapy requires some sedation and can leave a patient in pain for a week

or so. Plus, the disease is still making fluid after the procedure, which can create other

lesions that pop up down the road and need to be addressed. He says half of his macrocystic

patients require more than one procedure due to persistent symptoms, and those patients

could reach for systemic therapy alternatives over time.

Alpelisib is not a viable drug for VA: He thinks every one of the adverse events from

its VA trials is underreported, and his patients have much higher rates of toxicity in clinical

practice. His patients do not tolerate the higher dose of alpelisib 250mg. Patients don’t come

off because it’s not working, they generally come off because it’s not tolerable.

Alpelisib pediatric growth impairment is a concern: Novartis (NVS) suggests no

negative signal in growth velocity, but they only show absolute growth and it's not normalized

to expected growth curve. They claim no patient drops below two percentiles, but our KOL

doesn't want to drop a patient even one percentile. He had kids on alpelisib where he thought