ProQR spins out CNS assets to form RNA rare disease biotech

Amylon Therapeutics CEO Thomas de Vlaam

ProQR has spun out its CNS assets to form Amylon Therapeutics. The offshoot starts life with seed funding and an RNA-based program targeting a rare genetic disease that causes strokes.

Amylon will further develop ProQR’s research into beta amyloid related disorders, initially by going after the stroke-causing disease hereditary cerebral hemorrhage with amyloidosis of the Dutch type (HCHWA-D). Further down the line, Amylon plans to target other beta amyloid related disorders, such as cerebral amyloid angiopathy (CAA).

That rough R&D strategy follows the well-trodden path of treating a rare genetic disease before expanding into a broader, related indication. HCHWA-D is a genetic subtype of CAA, a disorder that also causes strokes but isn’t confined to people with certain mutations. CAA affects anyone with buildups of beta amyloid in their brain’s vascular system. 

If Amylon’s science works in the well-defined HCHWA-D patient population, it may also help the many more seniors and Alzheimer’s disease patients who are at risk of stroke. 

Leiden, the Netherlands-based ProQR is facilitating this work by granting Amylon exclusive license to develop treatments for beta amyloid related disorders in return for milestones and royalties. ProQR has taken a majority stake in Amylon, but has also enlisted the support of institutional and private investors to put together seed funding for the startup.  

The spinout is the result of ProQR’s compartmentalized business structure and, more specifically, its decision to bring Thomas de Vlaam on board to step up its CNS activities in 2015.

“As part of our corporate strategy to bring as many of our programs to patients as possible, we built ProQR as a group of focused business units that enable us to attract external funding directly into the individual development programs or ultimately spin programs out as we have now done with Amylon,” ProQR CEO Daniel de Boer said in a statement.

Vlaam will serve as CEO of Amylon, capping off a fast rise through the ranks. ProQR originally hired Vlaam as a research intern in 2014. That was Vlaam’s first post in the industry. Six months later, ProQR named Vlaam as its CNS innovation lead, a position it said it created with a view to fostering a spinout. Vlaam held that position for about 30 months, in which time he invented Amylon’s lead program AT-010, before taking the helm at the startup. 

The newly minted CEO has the support of a three-person scientific advisory board that includes ProQR’s co-founder Gerard Platenburg and neurology professors working on both sides of the Atlantic. The chairman of ProQR’s supervisory board, Dinko Valerio, has taken up the same role at Amylon.