In This Article:
Novartis’ NVS phase III study showed a positive risk-benefit profile of an investigational gene therapy candidate, intrathecal onasemnogene abeparvovec (OAV101 IT), for treating spinal muscular atrophy (SMA). Sangamo Therapeutics SGMO announced that its partner Pfizer PFE has decided to end their global collaboration and license agreement for giroctocogene fitelparvovec, a one-time gene therapy candidate for treating moderately severe to severe hemophilia A. The European Commission granted approval for the expanded use of J&J’s JNJ drug, Rybrevant, as a combination therapy in non-small cell lung cancer (NSCLC).
Here's a recap of the week’s most important stories.
PFE Returns Rights to Hemophilia A Candidate to Partner SGMO
Sangamo Therapeutics regained development and commercialization rights for its hemophilia A candidate, giroctocogene fitelparvovec, after partner Pfizer conveyed its decision to terminate their collaboration. SGMO’s stock crashed on the news. Sangamo is considering all options to continue the program, which includes looking for a new collaboration partner for the candidate. In July, the companies announced positive data from the pivotal phase III AFFINE study on giroctocogene fitelparvovec. The study met its primary as well as key secondary endpoints.
Pfizer had in-licensed global rights to giroctocogene fitelparvovec from Sangamo in 2019 and was responsible for pivotal studies as well as regulatory and potential commercialization activities for the candidate. Sangamo said that Pfizer had informed the company of its intention to file regulatory applications for giroctocogene fitelparvovec in the United States and EU in early 2025. Sangamo was eligible to earn up to $220 million in milestone payments from Pfizer upon achieving certain regulatory and commercial milestones for giroctocogene fitelparvovec.
NVS’ Study on SMA Candidate Meets Primary Goal
Novartis’ phase III study evaluating OAV101 IT in treatment-naïve patients with SMA type 2, rare, genetic neuromuscular disease, aged two and older, met its primary endpoint. Data from the STEER study showed that treatment with OAV101 IT led to an increase from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) total score. HFMSE is a scale that measures physical abilities in patients with SMA types 2 and 3. Novartis plans to share data from the SHEER study with the FDA and other regulatory agencies in 2025.
EU Nod for J&J’s Rybrevant-Lazcluze Combo for NSCLC
J&J announced that the European Commission has granted approval to a combination of its intravenously administered EGFR/MET inhibitor Rybrevant (amivantamab) and oral EGFR-TKI inhibitor, Lazcluze (lazertinib), for use in certain patients with EGFR-mutated non-small cell lung cancer (NSCLC). The approval is for the Rybrevant-Lazcluze combination as a first-line treatment for adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions (ex19del) or exon 21 L858R substitution (L858R) mutations. The FDA approved this combination in August for a similar indication. This approval is based on data from the phase III MARIPOSA study.