Agios Bets on Mitapivat Expansion as Sickle Cell Decision Looms

Wells Fargo initiated coverage of Design Therapeutics with an overweight rating, citing the company's Friedreich ataxia candidate DT-216P2 as having potentially best-in-disease functional improvement based on results from the RESTORE-FA study released in May. The bank set a $26 price target, implying roughly 112% upside based on the October 1 close. Analyst TianQi Hang wrote that the May update showed pharmacokinetics look good, and that blood-FXN protein, muscle-mRNA data, plus an early mFARS signal further de-risk the platform. Hang estimates that the blood FXN protein increase seen after 6 weeks can translate to at least a 2-point mFARS change, and said that if the drug kinetics sustain for 12 weeks, which he believes they will, DT-216P2 could deliver best-in-disease functional benefits. He assigns DT-216P2 a 60% probability of success, with peak sales of approximately $600M in the US and approximately $900M outside it. If approved, DT-216P2 would compete against Biogen's Skyclarys, also known as omaveloxolone, and Hang sees it gaining a peak share of the FA treatment market of 30% in the US and 20% ex-US.

The US FDA has approved Bristol Myers Squibb's Camzyos, also known as mavacamten, for children with symptomatic obstructive hypertrophic cardiomyopathy, a rare genetic heart condition that can cause shortness of breath, fatigue, and abnormal heart rhythm. The approval was based on results of the phase 3 double-blind, placebo-controlled SCOUT-HCM trial, which enrolled children 12 to 18 years old with symptomatic oHCM; patients in the Camzyos arm had a significantly reduced Valsalva LVOT gradient, a measure of pressure in the left ventricular outflow tract while bearing down, compared to placebo. Camzyos carries a boxed warning for heart failure risk and is restricted through a Risk Evaluation and Mitigation Strategy program. The treatment was approved for adults in 2022.