How The Curtain Finally Closed On Ferring/Blackstone’s Gene Therapy Venture FerGene
Back In Swiss Pharma’s Hands
Executive Summary
Having failed to live up to its promising beginnings, FerGene, the gene therapy firm founded with the backing of Ferring and Blackstone to advance the former’s candidate for bladder cancer is back under the Swiss pharma's wing. However, doubts about its future and that of nadofaragene firadonavec remain.
Just shy of three years after Ferring Pharmaceuticals and Blackstone formed FerGene to advance the late-stage bladder cancer gene therapy nadofaragene firadenovec, the pharma company and the private equity firm have formally ended the collaboration, which has not been smooth.
The partners teamed up to create FerGene to much fanfare at the end of November 2019, agreeing to pump up to $570m into the firm to advance nadofaragene firadenovec, a late-stage gene therapy candidate for patients with high-grade, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer. Blackstone was to invest the lion’s share, put in $400m while Ferring committed up to $170m to advance the gene therapy that the privately owned Swiss firm originally licensed from Finland's FKD Therapies Oy in May 2018.
Within a month, excitement around FerGene grew when the venture pulled off the coup of poaching Ipsen SA CEO David Meek to head it up. Meek was keen to jump onboard after FerGene unveiled Phase III data showing that nadofaragene firadenovec met its primary endpoint, with 53% of patients achieving a complete response (CR) at three months, and 24% continuing to show a CR at 12 months. (Also see "Fledgling Pharma FerGene Makes Quick Debut With Gene Therapy Candidate" - Scrip, 6 Dec, 2019.) (Also see "Blow To Ipsen As FerGene Nabs CEO Meek " - Scrip, 18 Dec, 2019.)
A biologics license application for the gene therapy submitted by FKD was accepted for filing and granted a priority review by the US Food and Drug Administration and FerGene began preparing its US commercialization plans; bringing Meek, with his 30 years of industry experience, on board was seen as a key step.
However, this was merely the prelude to a reversal of fortunes. In May 2020, the FDA issued a complete response letter for nadofaragene firadenovec, indicating that there were outstanding questions that FerGene's manufacturing partner needed to address further regarding its chemistry, manufacturing and controls processes. The company stressed that there were no outstanding questions regarding the clinical data for nadofaragene firadenovec, saying it remained confident in its potential approval.
That confidence did not seem to stretch to Meek who was ousted from the role in April 2021, and a number of lay offs ensued (Meek, for his part, swiftly returned to the sector, taking the top job at Mirati Therapeutics, Inc. in September last year). (Also see "Mirati’s New CEO Clears Out Execs Ahead Of Key Filing And Readout" - Scrip, 2 Nov, 2021.)
Fast-forward 18 months and Ferring and Blackstone say that they have reached an agreement to restructure their collaboration that will see the Saint-Prex-headquartered company assume "full and sole control of FerGene and nadofaragene firadonavec." The new arrangement allows for "a return on capital already invested by Blackstone" and also provides it with the option to make "a passive financial investment in nadofaragene firadonavec upon achievement of a specified regulatory event in exchange for a royalty interest."
Ferring gave no further financial details nor any update on the therapy, merely stating that it will continue developing nadofaragene firadonavec as an important treatment option for bladder cancer.
How much of a priority the gene therapy will be for Ferring in the short term, and whether or not FerGene continues as a separate entity, remains to be seen. Its focus in the next few months is likely to be on the fecal microbiota transplant Rebyota for recurrent Clostridioides difficile infection which received a favorable FDA advisory committee review last month. (Also see "Ferring’s Fecal Transplant Gets Through AdComm, Aided By Preference For Standardized Product" - Pink Sheet, 22 Sep, 2022.)
The panel voted 13 to 4 that the available data were adequate to support the effectiveness of Rebyota, also known as RBX2660 and developed by Ferring subsidiary Rebiotix, Inc., to reduce the recurrence of C diff in adults 18 years of age and older following antibiotic treatment for recurrent C diff infection. It also voted 12 to 4 with one abstention that the available data were adequate to support the safety.