Dec 11, 2023

Théa Completes Acquisition of ProQR’s LCA10 and USH2A Treatment Programs, Plans to Continue Clinical Development

Eye On the Cure Research News

Known as antisense oligonucleotides, the treatments performed encouragingly in ProQR’s clinical trials

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Laboratoires Théa, the leading European developer of eye care products, has acquired two emerging antisense oligonucleotide (AON) treatments for inherited retinal diseases from ProQR Therapeutics. Both AON therapies — sepofarsen (LCA10) and ultevursen (USH2A) — previously demonstrated vision improvements in ProQR’s clinical trials.

In October 2023, Théa had announced termination of its agreement to acquire ProQR’s LCA10 and USH2A assets, but the companies have since moved forward to ultimately complete the acquisition.

Under the terms of the agreement, ProQR has received an initial payment of €8M and may be eligible for up to €165M in further development, regulatory, and commercial earn-out payments upon related achieved milestones, as well as double-digit royalties based on commercial sales in the US and EU.

The RD Fund, the venture philanthropy arm of the Foundation Fighting Blindness, invested in the development of ultevursen and supported ProQR’s effort to find a buyer for both of its ophthalmic assets.

“We are delighted that Théa’s acquisition of sepofarsen and ultevursen has been completed and to see these promising treatments move forward in clinical development,” says Jason Menzo, chief executive officer, Foundation Fighting Blindness. “Both emerging therapies have demonstrated encouraging results in human studies and provide hope for preserving and restoring vision for patients.”

Sepofarsen was developed for people with Leber congenital amaurosis 10 (LCA10) caused by the IVS26 mutation in the gene CEP290. Some patients in ProQR’s Phase 2/3 Illuminate clinical trial for sepofarsen had meaningful vision improvements. However, the treatment did not meet its primary trial endpoint, best-corrected visual acuity (BCVA), nor did it meet its secondary endpoint, navigation of a mobility course.

Ultevursen was developed for people with mutations in exon 13 of the USH2A gene which leads to Usher syndrome 2A or non-syndromic retinitis pigmentosa. In ProQR’s Phase 1/2 Stellar clinical trial, ultevursen demonstrated benefits in BCVA, static perimetry (retinal sensitivity), and retinal structure as measured by optical coherence tomography (OCT).

Both sepofarsen and ultevursen are comprised of tiny pieces of genetic material that are injected into the vitreous, the soft gel in the middle of the eye. The genetic material masks the disease mutation in RNA, the genetic messages that cells read to make proteins which are critical for the cells’ health and function. Masking the mutation enables cells to make the correct protein.

AONs can be advantageous when large retinal disease genes — such as CEP290 and USH2A — exceed the capacity of viral gene replacement delivery systems thereby making gene therapy development for these genes more challenging.