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SNY's Tolebrutinib Gets FDA Breakthrough Therapy Tag for nrSPMS

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Sanofi (SNY - Free Report) announced that the FDA has granted a Breakthrough Therapy designation to its investigational oral BTK inhibitor, tolebrutinib, for treating adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS).

The FDA grants breakthrough therapy designation to expedite the development and review of therapies for severe or life-threatening diseases where preliminary clinical evidence shows that the therapy may provide substantial improvements over available treatments.

There are no approved therapies to treat nrSPMS at present. Multiple sclerosis (MS) is a chronic neurodegenerative disease that causes disability accumulation over time, which is not properly addressed by the currently available therapies, and there remains a significant unmet need for MS treatment.

Year to date, shares of Sanofi have declined 5.5% against the industry’s increase of 7.4%.

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Breakthrough Therapy Tag Based on Data From SNY's HERCULES Study

The FDA’s Breakthrough Therapy designation for tolebrutinib was based on data from the phase III HERCULES study.

Data from the HERCULES study showed that treatment with tolebrutinib delayed the time to onset of six-month confirmed disability progression (CDP) by 31% versus placebo.

Also, further analysis of secondary endpoints showed that the number of participants who experienced confirmed disability improvement was nearly double when treated with tolebrutinib compared to those who received a placebo.

Tolebrutinib has now become the first and only brain-penetrant BTK inhibitor to receive the Breakthrough Therapy designation from the FDA for treating MS.

Regulatory submissions seeking approval for tolebrutinib in the United States are currently being finalized while the same is being arranged in the EU.

More on SNY's Tolebrutinib

Besides nrSPMS, the phase III PERSEUS study is currently evaluating tolebrutinib in primary progressive MS. Data from this study is expected to be released in the second half of 2025.

Tolebrutinib was added to Sanofi’s portfolio with the Principia acquisition in 2020.

In 2022, the FDA placed a partial clinical hold on Sanofi’s phase III studies on tolebrutinib in MS and myasthenia gravis (MG) indications after the regulatory agency identified cases of drug-induced liver injury in some study participants who were administered tolebrutinib.

The MG studies on tolebrutinib were eventually discontinued in 2022 after careful evaluation of the emerging competitive treatment landscape.

SNY's Zacks Rank & Key Picks

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

Some better-ranked stocks from the biotech sector are CytomX Therapeutics, Inc. (CTMX - Free Report) , Spero Therapeutics, Inc. (SPRO - Free Report) and Castle Biosciences, Inc. (CSTL - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Loss per share estimates for 2025 have narrowed from 56 cents to 35 cents during the same time. Year to date, shares of CTMX have declined 27.7%.

CTMX’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 115.70%.

In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. Loss per share estimates for 2025 have narrowed from $1.54 to 79 cents during the same time. Year to date, shares of SPRO have declined 25.2%.

SPRO’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 94.42%.

In the past 60 days, estimates for Castle Biosciences’ 2024 bottom line have moved from a loss of 58 cents per share to earnings of 34 cents per share. Loss per share estimates for 2025 have narrowed from $2.13 to $1.84 during the same time. Year to date, shares of CSTL have surged 34.9%.

CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.

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