US Biotechnology: Grab our new top pick VRTX before your friends and neighbors - steal the opportunity while they are out east

Research evidence excerpt

US Biotechnology: Grab our new top pick VRTX before your friends and neighbors - steal the opportunity while they are out east

n

to translate to the registrational trial. Sionna’s pending Ph2a topline of SION-719 add-on to Trikafta is a key upcoming catalyst;

however we remain confident in the CF franchise as a backbone treatment for patients.

We rate ALNY Outperform with PT $429 (49% upside) based on the average of our DCF valuation, P/E and EV/R

multiples.

ALNY is our next best idea for investors. We rate ALNY Outperform with a $429PT and think that the market is overestimating

the potential readthrough of AZ/IONS CARDIO-TTRansform to nucresiran’s TRITON-CM. We assign 75% PTS for TRITON-CM

based on our outside-in statistical modeling that predicts target HR= 0.75 vs CV HR= 0.825. We project degradation of 1st

generation Amvuttra’s HR= 0.72 (HELIOS-B) due to a higher expected percentage of patients either on tafamidis at baseline or

to be commenced during the trial resulting in improved control arm performance. We mathematically adjust HELIOS-B efficacy

within a modified patient population reflected by CARDIO-TTRansform’s recent enrollment: 60% of patients on tafamidis at

baseline and another 25% of patients commencing a stabilizer during the trial. Now that AZN/IONS Ph3 is negative, ALNY leads

the competitive landscape: the future clinical decision between nucresiran vs Amvuttra will depend on TRITON-CM’s outcome.

We model global risk-adjusted sales of Amvuttra and nucresiran of $12.7Bn in 2035.

We rate BIIB Outperform with PT $236 (15% upside) based on the average of our DCF valuation, P/E and EV/R

We consider BIIB to be one of the more promising investments within our coverage group. Litifilimab looks like a potential leader