An eye for the eyes

CAMBRIDGE, Mass. & GAINESVILLE, Fla.—A new broad collaboration and license agreement was launched today as Biogen and AGTC paired up to develop gene-based therapies for a number of ophthalmic diseases. The deal's focus is on the development of a portfolio of AGTC's therapeutic programs, including a clinical-stage candidate and a preclinical candidate for orphan diseases of the retina that can cause blindness in children and adults.

 

The lead development programs in question for this collaboration include a clinical candidate for X-Linked Retinoschisis (XLRS) and a preclinical candidate for the treatment of X-Linked Retinitis Pigmentosa (XLRP), two conditions with significant unmet needs that could be treated by replacing the faulty gene that causes each disease.

 

"With this collaboration, we hope to advance gene therapies to open possibilities for patients who suffer from diseases that are well understood, but have no adequate treatment," Dr. Olivier Danos, senior vice president of cell & gene therapy at Biogen, noted in a statement. "AGTC is an exceptional partner to help us advance our gene therapy capabilities by targeting diseases of the eye—an organ that provides an ideal setting for the localized, selective delivery of gene-based therapies."

 

Per the terms of the collaboration, Biogen will pay AGTC $124 million up front, a sum that includes a $30-million equity investment in AGTC at a price equal to $20.63 per share and certain prepaid research and development expenditures. AGTC will grant Biogen a license to its XLRS and XLRP programs, as well as the option to license discovery programs for three further indications at the time of clinical candidate selection. AGTC is also eligible to receive upfront and milestone payments of more than $1 billion, which includes up to $472.5 million collectively for the two lead programs (which will also qualify for royalties in the high single-digit to mid-teen percentages of annual net sales). Biogen will also pay up to $592.5 million across the discovery programs, with royalties in the mid-single digits to low-teens.

 

Biogen will gain worldwide commercialization rights for the XLRS and XLRP programs, with AGTC holding an option to share development costs and profits after initial clinical trial data become available, and an option to co-promote the second of these products to gain approval in the United States. AGTC will spearhead the clinical development programs of XLRS through product approval and the clinical development programs for XLRP through the completion of first-in-human trials. For its part, Biogen will support clinical development costs, subject to certain conditions, following the first-in-human study for XLRS and IND-enabling studies for XLRP. Per the manufacturing license, Biogen gains an exclusive license to use AGTC's proprietary technology platform to develop AAV vectors for up to six genes, three of which are at AGTC's discretion, in exchange for milestone and royalty payments.

 

"We expect this collaboration will further validate our novel adeno-associated virus gene therapy platform and support the development of new therapies that may allow for transformative treatments for these rare inherited eye diseases and other clinical indications," commented Sue Washer, president and CEO of AGTC, in a press release. "Biogen's significant commitment to advancing gene therapies and demonstrated success in developing innovative therapies to treat complex diseases, combined with our proprietary manufacturing technology and extensive gene therapy experience, makes this an ideal partnership."

 

 

 

SOURCE: AGTC press release