Repligen confirms licensing agreement with Pfizer for spinal muscular atrophy program

Repligen has announced a new licensing agreement with Pfizer in an effort to advance Repligen's spinal muscular atrophy (SMA) program.

The work focuses on RG3039, which is a small molecule drug candidate in clinical development for the condition, while backup compounds and technologies will also be investigated.

As part of the deal, Repligen is entitled to up to $70 million (£43 million) from Pfizer, arriving in the shape of a $5 million upfront payment and future instalments of up to $65 million, while royalties on any future sales of SMA compounds will also add extra money.

Repligen is also responsible for completing the first two cohorts of an active Phase 1 trial, which will review RG3039 in healthy volunteers using laboratory equipment. It is believed that this will take place in the first quarter of this year.

In addition, Pfizer will receive technology transfer services from Repligen.

Walter C. Herlihy, president and chief executive officer of Repligen, said: "This agreement is consistent with the strategic decision we announced in August 2012 to focus Repligen’s internal efforts on the growth of our bioprocessing business, while seeking external partners for our therapeutic development programs.

"We believe this collaboration with Pfizer, a leading pharmaceutical company with specialized efforts in orphan and genetic diseases, has the potential to accelerate the development of therapies for SMA."

Jose Carlos Gutierrez-Ramos, senior vice president of Pfizer BioTherapeutics research and development, also commented on the work, noting that there is a "critical need" to expedite potential treatment solutions for spinal muscular atrophy.

"This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen’s leading SMA program," he continued.

As well as working with Repligen, Pfizer has launched a new platform for its pharma customers.

Access Pfizer for Professionals provides a range of information and was developed using feedback from 200 pharmacists, giving web users 24/7 access to commercial and patient information.