According to the Alzheimer’s Association, Alzheimer’s disease is the sixth leading cause of death in the US, and is the only one among the top 10 that cannot be prevented, cured or slowed. This year AD (afflicting 5.3 million Americans) and other dementias will cost the US $226 billion.

The great unmet need for AD treatments and the failure of numerous drug candidates against the disease has led scientists to search for alternate targets. Tau-targeting candidates, though historically rejected in favor of amyloid-beta targeting ones, have gained increasing interest from investors and larger pharma companies.

Over the past five years a crop of closely held biopharmas with Tau-focused candidates have raised healthy sums of money, and some have also gained large pharma partners:

AC Immune SA: To date AC Immune has raised about $88 million and has an impressive portfolio of partnerships, potentially worth at least $1.2 billion. The most recent financing for the Swiss biotech was its $22 million Series D in January 2014 from returning shareholders, including the company’s primary investor German billionaire Dietmar Hopp. [See Deal] Three months later it licensed Piramal Enterprises Ltd.’s Piramal Imaging SA exclusive global rights to its Tau protein positron emission tomography radiopharmaceuticals to diagnose AD and other Tau-related disorders. [See Deal] In January 2015 J&J’s Janssen R&D LLC licensed AC Immune’s anti-Tau therapeutic Alzheimer’s vaccine, ACI-35, in Phase Ib. The deal provided up-front money, up to $509 million in milestones, plus tiered royalties. [See Deal] AC Immune also has deals with Roche’s Genentech Inc., the first in 2006 for anti-beta monoclonal antibody crenezumab (which missed its primary endpoints from the Phase II data in 2014), worth an undisclosed sum of up-front money, over $300 million in milestones and royalties. [See Deal] In 2012 Genentech in-licensed exclusive global rights to AC Immune’s preclinical anti-Tau antibodies for AD and other neurodegenerative disorders in a deal potentially worth over $418 million. [See Deal] This July AC Immune achieved a milestone under that deal when Genentech selected a specific antibody to bring to the clinic.
NeuroPhage Pharmaceuticals Inc.: Launched in 2008, the company has raised over $50 million to date from Merieux Developpement, Shire LLC and US and European private investors. Its most recent financing was a two-tranche Series D (concluded January 2015) worth $27 million. [See Deal] The company’s lead candidate NPT088 targets multiple toxic misfolded proteins including Tau and amyloid beta, and is in preclinical trials for AD and PD.
iPierian Inc.: Raised $22 million in a Series B round in 2010 to Google Ventures, Mitsubishi UFJ Capital, ATEL Ventures, Kleiner Perkins Caufield & Byers, Highland Capital Partners, MPM Capital and FinTech Global Capital. [See Deal] In 2013 it raised another $30 million from all existing investors in a C round, using some proceeds for the Series A funding of its spin-off company True North Therapeutics. [See Deal] [See Deal] Founded in 2007, Ipierian’s lead candidate, IPN-007, is a preclinical antibody against AD and Tau pathologies including progressive supranuclear palsy and frontotemporal dementia. The company was acquired by Bristol-Myers Squibb Co. in April 2014 for $175 million up front, up to $550 million more in earn-outs, plus net sales royalties. [See Deal]
QR Pharma Inc.: Closed a $5.7 million A round in May 2015 to Robin Hood Ventures, returning angel backers and angel investors from Delaware Crossing and Keiretsu Forum. [See Deal] Founded in 2008, QR’s lead candidate Posiphen (in-licensed from TorreyPines Therapeutics) inhibits amyloid-precursor protein synthesis and reduces levels of Tau and alpha-synuclein. Posiphen is in Phase I for AD, and in preclinical trials for Parkinson’s and traumatic brain injury.
Oligomerix Inc.: Has raised $5.5 million to date; $2 million from its 2012 A round, $2.8 million from its Series B in 2013 and $700,000 in seed money. [See Deal] [See Deal] Backers are Wheatley MedTech Partners, Wheatley New York Partners and Durand Venture Associates. Founded in 2006, the company has grant money from the Alzheimer’s Drug Discovery Foundation and the NIH and its National Institute of Aging. It has five programs aimed at inhibiting the Tau protein, specifically targeting extracellular forms of Tau oligomers. The company plans to select candidates from its program to move into animal testing and IND-enabling studies.

Investors Find Opportunity In Tau-Targeting Biopharmas

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