TG Therapeutics changed the design of the GENUINE study last October to eliminate progression-free survival (PFS) as a co-primary endpoint, leaving only overall response rate. The planned enrollment was also cut from 330 to 120 patients. The changes allowed TG Therapeutics to complete the GENUINE study faster, but it also nullified the Special Protocol Assessment (SPA) reached with the FDA spelling out the regulatory requirements for ublituximab's approval.
FDA is often displeased when companies renege on SPAs and fail to provide the previously agreed-upon clinical data necessary for approval.
While TG Therapeutics acknowledges the added risk of the lost SPA, the company still intends to meet with the FDA "to discuss the filing of the data for accelerated approval." The company's supporters argue the nearly doubling of the response rate in ublituximab's favor should be strong enough clinical evidence for FDA to approve.
The question of medical need for previously treated, high-risk CLL patients is the other, simmering issue. TG Therapeutics bears make the point these patients can be treated today with a combination of Rituxan, Roche's long-approved anti-CD20 monoclonal antibody, and Imbruvica. Therefore, ublituximab is not filling an unmet medical need and accelerated approval should be off the table.
Bulls counter with the argument that Rituxan is not specifically approved for use in previously treated, high-risk CLL patients. Doctors prescribe Rituxan/Imbruvica off label. From an FDA perspective, therefore, an unmet medical need still exists.
[TG Therapeutics is a member of the Gnarly Nine. Read more here.]
I had lunch Thursday with a healthcare fund manager and longtime TG Therapeutics shareholder. We talked about the ublituximab CLL-approval controversy. His reaction: Bemused. He owns TG Therapeutics for ublituximab but because he believes the drug will eventually be approved for multiple sclerosis (following the development plan used by Roche to win the approval of ocrelizumab -- another anti-CD20 monoclonal antibody.)
My TG Therapeutics shareholder/lunch date didn't participate in this week's $50 million stock offering, but he suspects (but doesn't know for sure) that the funds who did were also more interested in ublituximab-multiple sclerosis than ublituximab-CLL.
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