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.

J&J announced that it is in-licensing global rights to Japan’s Kaken Pharmaceutical’s STAT6 program for autoimmune and allergic diseases, including atopic dermatitis (AD). Kaken’s STAT6 program includes lead candidate KP-723, which is expected to enter phase I studies in AD in 2025.

The NYSE ARCA Pharmaceutical Index declined 1.01% in the past five trading sessions.

Large Cap Pharmaceuticals Industry 5YR % Return

Large Cap Pharmaceuticals Industry 5YR % Return

Here’s how the eight major stocks performed in the previous five trading sessions.

Image Source: Zacks Investment Research

In the last five trading sessions, Novo Nordisk rose the most (0.2%), while Lilly declined the most (2.2%).

In the past six months too, while AbbVie rose the most (9.7%), Novo Nordisk declined the most (37.0%).

(See the last pharma stock roundup here: NVO Obesity Study Failure, FDA Nods for PFE, LLY)

What's Next in the Pharma World?

Watch this space for regular pipeline and regulatory updates next week.

J&J has a Zacks Rank #2 (Buy), while Pfizer, Novartis and Sangamo have a Zacks Rank #3 (Hold) each. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

Novartis AG (NVS) : Free Stock Analysis Report

Johnson & Johnson (JNJ) : Free Stock Analysis Report

Pfizer Inc. (PFE) : Free Stock Analysis Report

Sangamo Therapeutics, Inc. (SGMO) : Free Stock Analysis Report

To read this article on Zacks.com click here.

Zacks Investment Research