Results, Setbacks

The losers are piling up after today’s big ASH abstract drop

Today is the big drop for ASH abstracts, and Spark $ONCE isn’t the only biotech company to be damaged in the response to some of the safety warnings or weak results to hit the biotech circuit on Twitter.

Trillium Therapeutics crushed

Trillium Therapeutics $TRIL gets the booby-trap prize after seeing its shares lose about half their value on a safety warning from their Phase I study of the CD47 drug TTI-621.

Here’s what caught investors’ attention in the abstract:

The 0.3 mg/kg dose was associated with reversible, dose-limiting toxicity (DLT) in 2 of 5 pts: one pt with G3 elevated ALT/AST and G4 platelet count, and a second pt with G4 platelet count who was transfused. Dosing at 0.2 mg/kg is now being explored.

Shares are down 48% in mid-morning trading.

Bluebird battered

Cambridge, MA-based bluebird bio $BLUE, meanwhile, didn’t impress anyone with its latest update on Lentiglobin. Its shares are down 16% on continued mixed results for its gene therapy for beta-thalassemia.

There is a super responder in the mix and the biotech continues to tout its successes, but a number of patients aren’t responding as well, and that has analysts worried that bluebird will have to do much better with a follow-on drug. Not what investors were hoping to see, but disappointment has become something of a habit for bluebird over the last year.

Achillion crashes

Then there’s Achillion Pharmaceuticals’ $ACHN oral factor D inhibitor ACH-4471. That drug is being investigated for conditions that include paroxysmal nocturnal hemoglobinuria (PNH). And it has a safety issue as well, which crushed its stock, now down 32%.

To date in the MAD study, ACH-4471 has been generally well tolerated across three dose cohorts (200 mg, 500 mg or 800 mg given every 12 hours) with no treatment-related SAEs reported. Two cases of self-limited, ALT elevations (Grade 3 and 4) were observed post-treatment in the mid- and high dose groups, respectively, with neither subject exhibiting signs or symptoms of hepatic decompensation. Both subjects’ ALT levels normalized without intervention during follow up. Further, no treatment-associated fever or infections were observed.

“Given the importance of the complement program to ACHN who otherwise is dependent on JNJ (OP) remaining committed to its HCV development program in a deteriorating marketplace, investors are understandably focused on the well being of this program, which we continue to view as high-risk/high-return,” noted Leerink’s Joseph Schwartz.

 


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