Pfizer and Repligen have ended their partnership focused on developing therapeutic options for the treatment of spinal muscular atrophy (SMA), according to a news report from GEN and other news outlets. The collaboration was worth up to $70 million, but was ended by Pfizer two years later after investing $7 million into the collaboration, and a month after the company granted Repligen a $1 million payment for the fulfilling of an undisclosed milestone.
Pfizer has presented a notice of termination to Repligen on January 26, and since the end of the partnership will take effect officially on April 26, Repligen has already filled a Form 8-K, which will be submitted to the U.S. Securities and Exchange Commission. Pfizer had granted a $1 million milestone payment a month ago to Repligen as part of its $7 million in upfront and milestone payments.
Even though Repligen was supposed to receive more $63 million in milestone payments, as well as royalties related to sales of compounds developed within the collaborative work, Pfizer is now giving back the SMA compound called RG3039 or PF-06687859 meant to treat SMA to Repligen, as well as several other compounds that were only in the preclinical phase of development. However, Pfizer will continue to work on studying treatments for rare diseases with unmet medical needs.
“Our portfolio currently consists of 22 marketed medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology, and our investigational programs in rare disease include seven programs from Phase I through registration,” said a representative of Pfizer on an interview to GEN. “Pfizer will continue to leverage our legacy expertise, technology platforms and leading capabilities in collaboration so that we can advance the research and development in rare diseases to ultimately lead to potential treatments for those we serve.”
The two companies had announced the partnership in 2013, which was established on December 28, 2012 and included supervision by Repligen of the phase I studies of RG3039 in healthy participants. The inhibitor of the enzyme DcpS RG3039 had been granted Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration, and the results of the phase I trial, presented at the 2014 SMA Conference, revealed encouraging outcomes.
In addition, under the agreement Repligen was granting technology transfer services, while Pfizer was responsible for the SMA program, with tasks such as the registrations needed for the approval of the compound. The preclinical studies were being financially supported and directed with a $13 million grant from the patient organization Families of SMA.