Novartis NVS announced positive top-line results from the late-stage STEER study on experimental candidate intrathecal onasemnogene abeparvovec (OAV101IT).

Intrathecal onasemnogene abeparvovec (OAV101IT) is an investigational, one-time gene therapy for patients with spinal muscular atrophy (SMA).

The STEER study is a phase III randomized, double-blind, sham-controlled study evaluating the clinical efficacy, safety and tolerability of a one-time dose of intrathecal onasemnogene abeparvovec (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.

Shares of Novartis have lost 3.6% in the past year aginst the industry’s growth of 1.8%.

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More on NVS’ STEER Study

The efficacy and safety results for OAV101IT in the STEER study were compared against a sham control, a procedure designed to mimic the administration of an investigational drug without delivering any active treatment.

Results showed that the study met its primary endpoint. Data from the study showed an increase from baseline across the study population in total Hammersmith Functional Motor Scale - Expanded (“HFMSE”) scores.

HFMSE is a standard for SMA-specific assessment of motor ability and disease progression.

The increase was primarily observed in patients treated with OAV101IT compared to sham controls, indicating better motor function in patients with SMA.

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.

In addition, the safety profile of OAV101IT was favorable as the overall adverse and serious adverse events were similar between arms.

Novartis plans to share STEER study results with regulatory agencies in 2025.

Apart from the STEER study, OAV101IT was evaluated in two clinical studies, including phase I/II STRONG and phase IIIb STRENGTH. The STRONG study was an open-label, dose ranging study evaluating the safety and efficacy of OAV101IT in patients with SMA with three copies of SMN2 aged 6 months to less than 60 months.  The STRENGTH study was an open-label, single arm, multi-center study evaluating the safety, tolerability and efficacy of OAV101IT in patients with SMA who had discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY Evrysdi (risdiplam). 

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.

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

NVS Looks to Strengthen Pipeline

NVS is a pure-play innovative medicine company with a focus on core therapeutic areas — cardiovascular, renal and metabolic, immunology, neuroscience and oncology.

The successful development of new drugs will boost NVS’ pharmaceutical business.

Novartis’ performance in the third quarter was impressive, with both earnings and sales beating estimates. The increase in annual guidance was another positive, indicating strong momentum across all key drugs in the upcoming quarters.

Novartis recently increased its sales guidance for the mid-term. The company now expects total sales to witness a compound annual growth rate (CAGR) of 6% during 2023-2028 compared with the previous estimate of 5%.

The rise in guidance was due to the strong momentum of NVS’ key drugs and its upcoming launches.

While organic growth continues to drive business, NVS is focused on strategic bolt-in acquisitions to strengthen its pipeline.

Novartis recently entered into a global license and collaboration agreement with PTC Therapeutics for the latter’s Huntington's disease candidate to strengthen NVS’ neuroscience pipeline.

Novartis will make an upfront payment of $1.0 billion to PTCT and the latter will also be eligible for up to $1.9 billion in development, regulatory and sales milestones. NVS will also share profits in the United States (40% PTC and 60% Novartis) and pay tiered royalties on ex-US sales.

NVS also announced a collaboration with Olema Pharmaceuticals, Inc. OLMA for a breast cancer drug.

Per the terms of the deal, Novartis will provide Olema with its breast cancer drug Kisqali (ribociclib) for the planned phase III OPERA-02 trial of Olema’s palazestrant in combination with ribociclib in ER+/HER2- frontline advanced or metastatic breast cancer.

NVS’ Zacks Rank

Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.


 

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