Singapore-based TauRx Pharmaceuticals has pulled in $135 million in a fundraising program launched in March, setting the stage for ongoing Phase III trials for a unique Alzheimer's candidate focused on inhibiting the aggregation of tau tangles in the brain.
TauRx, with research offices in Aberdeen, Scotland, but a dedicated fund base in Singapore and Southeast Asia that includes some surprising names and scores of smaller individual investors, said in a press release the equity funding will help it complete and ongoing Phase III trials.
"The additional investment has been provided through a combination of new investors and existing shareholders and will support our Phase III clinical trials program," Claude Wischik, co-founder and executive chairman of TauRx, said in a statement. "This new investment was raised at successively higher share prices during the course of 2015, reflecting the confidence investors have in our tau aggregation inhibition technology and in the promise of our lead product, LMTX."
The company has been at work on the candidate for more than a decade, raising more than S$300 million from small investors mostly from Southeast Asia and medical backgrounds, but also from Singapore's Temasek and S$120 million from Malaysian casino and resort operator Genting Group.
Of course the stakes are high. A treatment for Alzheimer's disease, to delay its onset, has not only been elusive, but even understanding disease progression and diagnosing it correctly remains a hurdle as demand for acute care is surging as many countries face a steady increase in aging populations worldwide.
Companies such as Eli Lilly ($LLY) and Biogen Idec ($BIIB), among others, have focused on candidates to remove clusters of amyloid beta in the brain as the cause of the disease, sparking and dashing hopes on candidates after clinical results that were alternately parsed for solid signals, or dismissed as inconclusive.
Others, including Wischik, however point to tau tangles in the brain as the leading cause of the disease, though some researchers also suggest that any eventual successful treatment could focus on both areas.
For TauRx, as the name suggests, the candidate LMTX is "currently being investigated as the first tau-based disease-modifying agent for the effective treatment of Alzheimer's disease."
In March, the company published Phase II results of the first clinical trial of a tau aggregation inhibitor (TAI) for Alzheimer's disease in the Journal of Alzheimer's Disease, saying it was an important milestone as it moves to report top line results from a Phase III study in 2016.
That effort was followed up in June by 6 articles in journals on the candidate.
The journal publications were aimed at meeting some doubt about the candidate and the cause of the disease.
Wischik reported in 1996 that the chemical substance methylthioninium (MT), and commonly known as "methylene blue," used in medicine for the last 100 years, dissolves tangle filaments isolated from the human brain by selectively blocking a critical step in the process required to form the rogue filaments.
But there was a problem in the use of the drug in Phase II that the release says has been solved, he said previously, and that the Phase III work will show that outcome.
"TauRx scientists discovered that MTC suffers from dose-dependent impairment in absorption when taken with food," the company said in March, adding that LMTX is better absorbed and tolerated than MTC. "This is due to the fact that the oxidized MT+ form needs to be actively converted to the reduced LMT form in the gut before it can be absorbed as LMT. In other words, MTC is a pro-drug for LMT, and food interferes with the conversion and absorption process. Since MTC was given with food in the Phase II trial to maximize tolerability for patients, only 109 mg/day of the intended 228-mg dose was available for absorption. Therefore, the minimum effective dose of 138 mg/day identified in the trial was simply the highest available dose tested."
"This has enabled Phase III trials to test whether an even higher level of efficacy can be achieved without significant loss of tolerability and safety," the March release said.
Looking ahead, Wischik repeated in the most recent release that results are on track for 2016.
"We continue to stay on track to announce top-line results for all three Phase III studies in 2016 and expect this to be a defining year for both the development of LMTX and the future growth of TauRx."
- here's the release