World's First Gene Therapy Candidate Completes Clinical Trial
Osaka University spin-off AnGes, Inc. (TSE:4563) has completed its U.S. clinical trial for the gene therapy drug Collategene, targeting comprehensive chronic limb-threatening ischemia (CLTI) a severe condition where blood flow is critically impaired, often leading to ulcers, gangrene, and ultimately amputation.
Based on these results, AnGes is now preparing a Biologics License Application (BLA) to the U.S. FDA. If approved, Collategene would become the world's first gene therapy drug, marking a historic milestone.
The company has already signed a manufacturing contract with Boehringer Ingelheim, a move seen in the industry as evidence that approval and commercialization are effectively on track.
Stock Price Stagnant: Market Cap Only ¥34 Billion
Despite this breakthrough, the market reaction has been muted. Following the August 9 announcement, the share price rose only briefly. As of August 26, the stock traded at ¥94, with a market capitalization of roughly ¥34 billion (US$230 million).
One reason is the company's past Collategene once received conditional approval in Japan, but the subsequent full approval application was withdrawn. This history has left investors wary.
Yet analysts note that the real battleground is no longer Japan but the U.S. and Europe. With FDA Breakthrough Therapy Designation already granted, many argue the current valuation grossly underestimates the potential.
M&A Precedents Point to Far Higher Valuations
The gene therapy space has seen a string of high-profile acquisitions:
- AveXis (Zolgensma): Acquired by Novartis for US$8.7 billion (approx. ¥900 billion) in 2018
- Spark Therapeutics (Luxturna): Acquired by Roche for US$4.8 billion (approx. ¥500 billion) in 2019
- uniQure (Hemgenix related): Entered into a licensing deal with CSL Behring valued at over US$2 billion
Each deal occurred just before or immediately after regulatory approval. By comparison, if Collategene secures FDA approval, AnGes' acquisition value could range from ¥300–500 billion, and in a bullish scenario, exceed ¥1 trillion (US$7–10 billion).
Overseas Investors Already Moving
Overseas investors have wasted no time. On August 12, Hong Kong-based hedge fund Athos Capital Limited acquired more than 5% of AnGes shares through off-market transactions.
Reports also suggest that investment firms from the UAE and the U.S. have begun quietly accumulating positions.
While Japanese investors remain cautious due to past setbacks, foreign funds appear ready to bet on the upside of a "world's first" gene therapy drug.
Could This Be Japan's First Major Biotech Takeover?
No Japanese biotech has ever been acquired by a global pharmaceutical giant. If Collategene gains FDA approval, that precedent could finally be broken.
The world's first approved therapy in its class would be an exceptionally attractive buyout target.
Some observers are already predicting that by 2026, AnGes could become the centerpiece of the most talked-about M&A deal in global biotech.
Investor Takeaway
At today's ¥34 billion market cap, AnGes trades at a fraction of its potential value. Past M&A precedents suggest multi-hundred-billion-yen to trillion-yen buyout levels.
For investors, the gap between current market perception and potential valuation represents a rare opportunity.
A true Hidden Gem Stock in the Land of Gold AnGes may soon shine on the global stage.
