Cinderella stories aren’t common in investing, but they do happen. Perhaps the best recent example is Axsome Therapeutics (NASDAQ:AXSM). The company’s market cap dipped to just $60 million in the final days of 2018, but promising results from a drug candidate aimed at treating depressive disorders propelled the stock onto Wall Street’s radar weeks later. After a string of positive regulatory developments and promising late-stage clinical results over the course of last year, the pharma company ended 2019 with a market cap of $3.8 billion.
Investors will find it difficult to replicate those returns, but there are still a handful of small-cap stocks with big-cap potential. Two biopharma stocks that come to mind are…
An overall response rate of 100%
The overall response rate (ORR) measures the percentage of patients in a clinical trial who respond to treatment at any point during the study. It says nothing about the duration of response (DOR) or if tumors disappeared completely, but ORR is the first metric researchers and investors seek out when trying to understand whether a treatment could be effective. By that measure, TG Therapeutics might have two very effective drug candidates in early stage development.
The small-cap biopharma presented data for two separate triple-combination therapies at the annual meeting of the American Society of Hematology (ASH) in early December. Both combinations included a proprietary duo of ublituximab, a monoclonal antibody, and umbralisib, a small-molecule inhibitor of cellular growth proteins. The pair is often just referred to as U2.
In a triple-combination of U2 and the approved cancer drug venetoclax, TG Therapeutics reported that all 23 individuals with relapsed/refractory chronic lymphocytic leukemia (CLL) responded to treatment. That gave the drug candidate an ORR of 100%, which is virtually unheard of. In fact, 20 of the 23 individuals responded to U2 alone (for an ORR of 87%) prior to the introduction of venetoclax later in the treatment regimen.
More impressive, all nine individuals who completed a full 12 cycles of treatment had undetectable minimal residual disease (MRD) in their blood, while seven of those individuals had undetectable MRD in their bone marrow. None of the 27 patients in the phase 1 trial (only 23 individuals were available for efficacy calculations in time for ASH) have progressed after treatment at a median follow-up of 6.4 months.
TG Therapeutics also reported promising data from a separate triple-combination comprising U2 and a proprietary BTK inhibitor called TG-1701. The therapy delivered an ORR of 86% (six of seven patients responded) at the lowest dose of TG-1701 tested.
As the biopharma sector goes all-in on complex cellular medicines for treating CLL, it’s refreshing to see the simpler, oral delivery approach of TG Therapeutics deliver such impressive outcomes. While the results collected to date are from small patient populations, they’re robust and certainly suggest a promising future is in store for the $1 billion company.
Could this help 75% of individuals without treatment options?
Shares of Forty Seven made an end-of-year movement that was more reminiscent of Axsome Therapeutics, soaring from a market valuation of $220 million in October to…
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