SNY's Efdoralprin Alfa Meets Key Endpoints in Rare Disease Study

Sanofi SNY announced that the phase II ElevAATe study, which evaluated its pipeline candidate, efdoralprin alfa, for the treatment of alpha-1 antitrypsin deficiency (AATD) emphysema, met all primary and key secondary endpoints.

An inherited rare disease of the lungs and liver, AATD is marked by low levels of AAT protein, which mainly affects lung function. Efdoralprin alfa works by inhibiting neutrophil elastase, an enzyme responsible for lung tissue damage in AATD patients.

Data from the global ElevAATe study demonstrated that treatment with efdoralprin alfa administered every three weeks (Q3W) or four weeks (Q4W) resulted in a statistically significant greater mean increase in functional AAT levels — within the normal range — based on trough concentrations at steady state, compared to weekly plasma-derived augmentation therapy at week 32.

Treatment with efdoralprin alfa also led to a greater mean increase in average functional AAT (fAAT) concentrations and a higher percentage of days with levels above the lower limit of the normal range for both Q3W and Q4W dosing regimens, thereby meeting the key secondary endpoints of the study.

Additional safety follow-up of efdoralprin alfa will be evaluated in the phase II ElevAATe OLE study.

SNY's Price Performance

Year to date, shares of Sanofi have increased 3% compared with the industry’s rise of 6%.

More on Sanofi's Efdoralprin Alfa

Efdoralprin alfa (SAR447537, formerly known as INBRX-101) was added to Sanofi’s rare disease portfolio following the acquisition of Inhibrx in 2024.

The latest data from the ElevAATe study underlines the potential of efdoralprin alfa to be the first restorative recombinant therapy that normalizes and maintains functional AAT levels.

Also, the Q3W and Q4W dosing regimens for efdoralprin alfa offer a potentially significant improvement in convenience versus plasma-derived therapy, which is dosed weekly.

The company believes that the successful development of efdoralprin alfa could offer a significant improvement in treatment options and the quality of life for AATD patients, as the current standard of care allows patients to reach but not maintain normal protein levels between infusions.

The FDA has already granted a fast track designation and an orphan drug designation to efdoralprin alfa for the treatment of AATD emphysema.

SNY's Zacks Rank & Stocks to Consider

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

Some top-ranked stocks in the biotech sector are Chemomab Therapeutics CMMB, CorMedix CRMD and ANI Pharmaceuticals ANIP, 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 60 days, estimates for Chemomab Therapeutics’ 2025 loss per share have narrowed from $2.40 to 60 cents. Loss per share estimates for 2026 have narrowed from $2.80 to $1.00 during the same period. CMMB stock has plunged 53.2% year to date.

Chemomab Therapeutics’ earnings beat estimates in three of the trailing four quarters, while meeting the same on the remaining occasion, with an average surprise of 26.25%.

In the past 60 days, estimates for CorMedix’s 2025 earnings per share have increased from $1.22 to $1.85. Earnings per share estimates for 2026 have moved up from $2.12 to $2.49 during the same period. CRMD stock has rallied 43.8% year to date.

CorMedix’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 34.85%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share have risen from $7.25 to $7.29. During the same period, earnings per share for 2026 have increased from $7.74 to $7.81. Year to date, ANIP’s shares have surged 72.6%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.66%.

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