SRRK Shares Pull Back After FDA Approval For Genetic Disease Drug — But Wall Street Hikes Price Targets

• COO Keith Woods said the launch began on Friday and that vials should be ready for infusion within days at clinics, infusion centers, or home.
• The U.S. Food and Drug Administration (FDA) cleared Isembyld on September 11 for adults and children two and older who already take certain SMA drugs.
• Citi, Truist, and Raymond James hiked their price targets on the stock on Monday.

Shares of Scholar Rock opened higher on Monday, then faded, as Wall Street lifted price targets after Friday’s early U.S. Food and Drug Administration (FDA) approval of the company’s first drug, Isembyld, for certain patients with spinal muscular atrophy (SMA).

SRRK closed Friday at $55.41, jumped double digits after-hours on the FDA approval, and opened Monday at $56.51. It closed down 6% on Monday as traders digested pricing announced on Monday and the shift from approval risk to launch execution.

On a call on Monday, the company set a list price of $11,659 per vial, or about $310,000 a year for a typical patient. COO Keith Woods said the launch began on Friday and that vials should be ready for infusion within days at clinics, infusion centers, or home, using a network of more than 10,000 nurses.

Second Time Through

The FDA cleared Isembyld on September 11 for adults and children two and older who already take certain SMA drugs. It was Scholar Rock’s second try. A 2025 rejection cited quality issues at a contract fill-finish plant in Indiana, not the trial data. The company switched to a backup plant and won approval weeks ahead of the FDA’s deadline.

Isembyld is given by infusion every four weeks. In a one-year study, the recommended 10 mg/kg dose improved motor scores by 2.2 points versus placebo. A gain of three points or more was seen in 34.2% of that group versus 13.5% on placebo.

SMA is a rare genetic disease that weakens the arms, legs, and muscles used to breathe and swallow. It affects about one in 10,000 births and around 10,000 people in the United States. Scholar Rock on Monday put the immediate U.S. pool at about 6,600 patients.

The firm ended June with $492 million in cash, can draw another $150 million of debt, and plans to sell a priority-review voucher. It said it does not intend to raise equity now.

Scholar Rock is also now working towards approvals in Europe and Japan for the drug.

Street Raises The Bar

Citi lifted its target to $74 from $60 and kept a ‘Buy’ rating ON SRRK. The firm said Isembyld’s approval removed the main overhang and arrived with a label at the broad end of expectations. Citi does not see the requirement that patients already be on an existing therapy as a constraint.

Truist raised its target to $76 from $55 and maintained a Buy rating. It noted the FDA nod came ahead of the Sept. 30 deadline, with a broad label in line with its model, and said it still expects wide use alongside other drugs such as Spinraza and Evrysdi.

Raymond James raised its target to $72 from $65 and kept Strong Buy. It sees a potentially fast U.S. start given demand and supply, and about $2.2 billion in 2035 sales, plus optionality abroad and in new uses — though it cautioned that much of a successful launch is already priced in. The three firms’ price targets imply a potential upside of 39% to 47% from the stock’s closing price on Friday.

How Did Retail Traders React?

On Stocktwits, retail sentiment around SRRK jumped from ‘bullish’ to ‘extremely bullish’ over the past 24 hours, while message volume increased from ‘high’ to ‘extremely high’ levels.

A Stocktwits user expressed surprise at Monday's selloff but noted they expect a rebound once retail investors take profits.

Another termed the pullback a good buying opportunity.

SRRK stock has gained 18% year-to-date. 

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