Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad group of patients aged 2 to under 18 years with spinal muscular atrophy (SMA). OAV101 IT is an investigational, one-time gene therapy for patients with SMA.

In the registrational phase III STEER study, OAV101 IT treatment resulted in a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE) compared to a 0.51-point increase in the sham control group. HFMSE is a key measure of motor function and disease progression in SMA.

Additionally, in the phase IIIb STRENGTH study, treatment with OAV101 IT demonstrated stabilization of motor function over 52 weeks of follow-up in patients who had previously discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY Evrysdi (risdiplam).

SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, results in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

Year to date, shares of Novartis have gained 14.6% compared with the industry’s 7.2% growth.

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Novartis presented these data at a recent medical conference held in Dallas, Texas. Per NVS, OAV101 IT is the first investigational gene replacement therapy to show clinical benefits in both children and young adults with SMA while maintaining a favorable safety profile, highlighting its potential to help patients avoid the need for repeated treatments.

More on NVS’ STEER Study

The STEER study is a phase III sham-controlled study evaluating the clinical efficacy, safety and tolerability of a one-time dose of intrathecal OAV101 IT in treatment-naïve patients with SMA type 2, aged two to less than 18 years who were able to sit, but never walked independently.

Last year, Novartis reported meeting the primary endpoint in the STEER study for SMA, showing improved HFMSE scores from baseline in patients treated with OAV101 IT, which demonstrated better motor function compared to sham controls. Sham control is a procedure designed to mimic the administration of an investigational drug without delivering any active treatment. Additionally, the safety profile of OAV101 IT was favorable as the overall adverse and serious adverse events were similar between treatment arms.

Apart from the STEER and STRENGTH studies, OAV101 IT was also evaluated in a phase I/II STRONG study. The early to mid-stage dose-ranging study evaluated the safety and efficacy of the candidate in patients with SMA with three copies of SMN2 aged 6 months to less than 60 months. The OAV101 IT clinical development program was studied in a broad population of approximately 170 patients with SMA and follow-up was conducted for up to 6.4 years.

Based on the success of these studies, Novartis plans to file applications seeking approval of OAV101 IT for SMA with regulatory agencies in multiple geographies in the first half of 2025.

Roche’s Evrysdi is approved for SMA in adults, children and infants from birth. Evrysdi was developed by RHHBY in collaboration with PTC Therapeutics and the SMA Foundation.

Novartis AG Price and Consensus

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NVS’ Zacks Rank & Stocks to Consider

Novartis currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the sector are Gilead Sciences GILD and Dynavax Technologies Corporation DVAX, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 30 days, Gilead Sciences’ earnings estimate for 2025 has improved from $7.80 to $7.87 per share. During the same timeframe, the estimate for earnings per share for 2026 has improved from $8.12 to $8.27. Year to date, shares of Gilead Sciences have gained 16.4%.

GILD’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 19.47%.

In the past 30 days, estimates for Dynavax’s earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have gained 6.8%.

DVAX’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%.

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