Dive Brief:
- TG Therapeutics took a major hit to its stock Thursday, after the company announced it had significantly altered the design of its Phase 3 GENUINE study testing the company’s lead candidate in relapsed/refractory chronic lympocytic leukemia (CLL).
- Enrollment challenges forced the New York-based biopharma to reduce the number of patients in the trial by two-thirds, eliminating a planned second stage aimed at evaluating ublituximab's effect on progression-free survival rates.
- With the changes, TG executives now expect enrollment to complete by year end, with topline data available as early as the first half of 2017. While the company still believes it is on track to file and win eventual approval, investors took a different view. Shares in TG fell by nearly 17% in Thursday trading.
Dive Insight:
Company CEO Michael Weiss framed the decision to pare down the trial's size as a chance to accelerate the timeline for filing and to allocate more resources to TG's other Phase 3 study — dubbed "UNITY" — evaluating ublituximab paired with another experimental TG compound.
"Given the GENIUNE enrollment challenges we’ve faced to do, we are very excited to accelerate the trial to a rapid conclusion, while also maintaining the ability to potentially file the data for accelerated approval," said Weiss said in the statement.
Yet as the stocks dipped yesterday, it was clear some investors didn’t have the same faith. Weiss said the company has been in talks with the Food and Drug Administration, which has given TG the go-ahead for the revised GENIUNE study.
Initially, TG meant to have two stages to the study. The company planned to evaluate the effect of ublituximab combined with AbbVie’s CLL drug Imbruvica (ibrutinib) on overall response rates in the first 200 patients, before moving on to measure progression free-survival in all study patients, initially targeted for 330.
Instead, the second stage will be eliminated and the number of patients to be enrolled will be cut down to 120. TG said the singular focus on ORR is consistent with other recent applications for accelerated approvals of CLL agents.
Trimming the GENUINE trial down will save $10 million over 2 years — money which Weiss said the company would reallocate to the UNITY study of its PI3K Delta inhibitor and ublituximab in CLL.
TG had $28.5 million in cash and equivalents, with another $25.6 million in short-term investments, on hand at the end of the second quarter. At the time, the company said it can fund operations for the next 18 months to 24 months.