The jam-packed September biotech catalyst calendar is behind us, which means investors are now focused on the stock-moving binary events that will take us to the end of the year.
STAT is here to help. Introducing the Biotech Q4 Scorecard: Ten companies with important clinical trial and FDA milestones scheduled to read out over the next three months. We’ll use emojis to track the winners and losers as the outcomes are announced.
Here’s how the scorecard companies break down:
Axovant Sciences (AXON) and Sage Therapeutics (SAGE): Two holdovers from our September scorecard are seeking redemption after humbling failures of their lead drugs in pivotal clinical trials. Data are coming on a mid-stage study of Axovant’s intepirdine in patients with Lewy Body dementia. That’s the backup indication after the drug’s failure in Alzheimer’s. Sage is conducting a phase 3 study of brexanolone in postpartum depression following the drug’s failure to reduce seizures in patients with a severe form of epilepsy.
AbbVie (ABBV): Investors love AbbVie for the blockbuster potential of its R&D pipeline. This quarter will determine if the company’s aggressive, risk-forward approach pays off with pivotal study results from risankizumab in psoriasis and rova-t in small cell lung cancer.
Global Blood Therapeutics (GBT): Idiopathic pulmonary fibrosis, a progressive and fatal lung disease, is the next clinical target for the company’s lead drug, GBT-440, while a pivotal study in sickle cell disease continues to enroll patients.
DBV Technologies (DBVT): Results from a phase 3 clinical trial of the company’s peanut allergy vaccine, known as Viaskin Peanut, are due in October.
Cytokinetics (CYTK): In a phase 2 study, its lead drug, tirasemtiv, failed to improve fine or gross motor symptoms in ALS patients, but a positive efficacy signal in breathing ability kept the program alive for a phase 3 study.
Amicus Therapeutics (FOLD): Last month, Amicus announced the failure of its experimental therapy for a rare, skin-blistering disease and the stock actually traded up. Why? Because investors anticipated the setback and were focused more on a looming update from an early-stage study of a different Amicus drug called ATB200/AT2221 targeting Pompe disease, another orphan disorder.
Spark Therapeutics (ONCE): On Oct. 12, the FDA is convening an advisory panel to review the company’s first-of-its kind gene therapy aimed at curing a rare form of childhood blindness.
Bluebird Bio (BLUE): The American Society of Hematology annual meeting in early December has become the traditional venue for Bluebird to present stock-moving updates to its gene therapy programs in sickle cell disease and beta-thalassemia, and more recently, its CAR-T program in multiple myeloma. The company will do the same this year.
Revance Therapeutics (RVNC): RT002 is a long-acting injectable botulinum toxin being developed for the aesthetics market and possible rival to Allergan’s Botox blockbuster, depending on the readout of a phase 3 clinical trial in frown lines.