Tiny Scottish biotech TauRx lobs bomb into Alzheimer’s fracas with Phase 3 success.

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The News:

After countless failures, the race for a treatment saw obscure TauRx Therapeutics Ltd. (Aberdeen Scotland) sprint ahead as its Alzheimer’s drug candidate prevented the formation of Tau tangles that are implicated in the disease.

The company saw positive results during a second Phase 3 trial investigating its anti-tau aggregation candidate, LMTX: a dose of just 4mg twice-daily significantly reduced the loss of neurons in the brain. Following the recent failures by Eli Lilly & Co., Merck & Co. and Bermuda-based Axovant Ltd., this is a welcome piece of good news for the field, LaBiotech reports.

TauRx will be eager to build on this, with the Alzheimer’s disease treatment market set to almost triple from $4.9B (€4B) in 2013 to an estimated $13.3B (€11B) by 2023. LMTX reduces the levels of aggregated or misfolded tau proteins, a major characteristic of Alzheimer’s. The drug shares its active ingredient, methylthioninium, and mode of action with Rember, TauRx’s first generation tau aggregation inhibitor.

Phase 2 trials of Rember demonstrated that targeting protein aggregation could slow disease progression. TauRx comes up against strong competition in the field, including AC Immune SA (Lausanne CHE), which has covered all bases, developing both anti-Tau and anti-amyloid-beta therapies, and tiny Araclon Biotech SL (Barcelona) is entering Phase 2 with its vaccine. The study reporting the LMTX results was published online last week in the Journal of Alzheimer’s Disease.

Steve’s Take:

There’s good news in the field of breakthrough disease treatments, and then there’s fantastic news.

Who would have thought an obscure biotech, incorporated in Singapore with operations and research facilities in Scotland, flying so far below the radar (it’s not covered by anyone here in the US as far as I could tell), would throw down the gauntlet in the race for a first, and likely blockbuster, treatment of dementia?

Steve's Take: @TauRx #Alzheimer breakthrough is fantastic news Click To Tweet

The fact that TauRx’s nonconformist drug may stop or slow down the disorder’s characteristic brain decay and memory loss when it is taken on its own, is a startling finding.

The twice-daily pill could reverse the worst symptoms of the condition in some patients, the data suggest. However, its effects appear to be blunted when it is administered at the same time as other conventional dementia medication.

Most pharmaceutical companies have tried and failed to treat Alzheimer’s by targeting its most obvious physical hallmark, corrosive clumps of a protein, amyloid-beta, The Times notes.

TauRx has gambled instead on a chemical that breaks down fibers that destroy brain cells from the inside out.

Key findings

In both the LMTX monotherapy and add-on therapy groups, whole brain atrophy (measured via MRI scans) initially progressed as expected for patients with mild Alzheimer’s disease, MedicalExpress reports.

However, after 9 months of treatment, the annualized rate of whole brain atrophy in monotherapy patients reduced significantly and became typical of that reported in normal elderly controls without Alzheimer’s disease. The comparable rate seen in the add-on therapy group progressed as reported for patients with mild Alzheimer’s disease.

Similarly, additional findings from FDG-PET scans in TRx-237-005 indicated that the decline in temporal lobe glucose uptake in those patients receiving LMTX monotherapy was significantly less than that typically reported for patients with mild Alzheimer’s disease.

When the various analyses were corrected for potential differences in severity or diagnosis at baseline between monotherapy and add-on therapy cohorts, the results remained robustly significant.

Known as LMTX or LMTM, the drug offered little benefit to most of the people with moderate Alzheimer’s who took part in two trials. But in the minority who took the pills without other dementia medicines it produced remarkable results even at low doses, according to an 800-patient study reported in the Journal of Alzheimer’s Disease, a gold standard among publications in the field.

David Reynolds, chief scientific officer at Alzheimer’s Research UK, said: “The results showed some potential benefits for LMTX as a monotherapy…but we can’t draw any firm conclusions without more research.”

“These highly significant results support further validation of tau-based therapy in Alzheimer’s disease,” said George Perry, Dean of Sciences, University of Texas at San Antonio and Editor-in-Chief of the Journal of Alzheimer’s Disease.

Bottom Line:

Indeed, by the time many people have dementia, so much damage has occurred that reversing it all–a cure, in effect–is a far-fetched goal at this time. That’s why a parallel focus must remain on prevention.

A balanced diet, exercise, blood pressure control, avoidance of obesity, good control of diabetes, possibly statins (see: Breakthrough study says blood thinners cut dementia risk by half. Message: If you start taking them, don’t quit.) and aspirin therapy; all have been studied and suggest dementia might be prevented in some cases.

Certainly, prevention is ideal. But Americans, for example, struggle with even avoiding obesity. Best also to have science and working toward a possible treatment–even a cure, someday–for those who have dementia already.

TauRx isn’t listed on any stock exchanges and is distinctly guarding against any forward-looking statements–even the slightest hint–if and when that might happen. But with this latest win in a study obtaining widespread outside validation from experts in the dementia/Alzheimer’s treatment field, that day is fast approaching. At a minimum it is now in the position to raise whatever capital it might need to take its candidate to the next clinical level.

I smell high-risk/high potential investment rewards down the road and will keep a close eye on this name. Stay tuned.

Steve Walker has no position in any stocks mentioned. MedContent Inc. has no position in any stocks mentioned. MondayMorning.com has a disclosure policy.