Alnylam Opts Against Phase III Trial for Vutrisiran in Stargardt Disease - Updated

Alnylam CEO Yvonne Greenstreet_company courtesy

Alnylam CEO Yvonne Greenstreet, Courtesy of Alnylam Pharmaceuticals

Alnylam Pharmaceuticals released its report for Q3, and tucked between positive financial and drug development updates, the company stated it will no longer be initiating a Phase III trial for vutrisiran in Stargardt disease.

The trial was set to begin at the end of the year. Alnylam did not give a specific reason for the decision, stating only that it will not initiate the trial as it “continues to evaluate the impact of the Inflation Reduction Act.”

Passed in August, the Inflation Reduction Act has been criticized by many in the biopharma industry. As BioSpace previously reported, Merck CEO Robert Davis said the legislation on pricing would be “highly chilling on future innovation,” and Pharmaceutical Research and Manufacturers of America (PhRMA) CEO Stephen Ubl called the bill’s passage a “tragic loss for patients.”

Some of the provisions of the Inflation Reduction Act include:

  • An annual cap of $2,000 on out-of-pocket spending

  • Maximum “fair prices” for selected drugs covered under Medicare

  • A requirement for the federal government to negotiate prices for some drugs covered under Medicare Part B and Part D 

  • A requirement that drug companies pay rebates to Medicare if prices rise faster than inflation 

  • Expanding eligibility for full benefits under the Medicare Part D Low-Income Subsidy Program

Christine Lindenboom, senior vice president of investor relations and corporate communications at Alnylam, told BioSpace Alnylam's decision to end the trial is due to the drug's orphan drug designation.

"The IRA includes a narrow exemption from negotiations for drugs that have indications associated with only one orphan drug designation, which we believe applies to our two marketed therapies for transthyretin (ATTR) amyloidosis, as both products have orphan drug designation for ATTR amyloidosis," Lindenboom said.

So, if Alnylam were to continue developing vutrisiran for Stargardt Disease, it may no longer be exempt from these negotiations and risk losing a substantial amount of profit. 

In September, the EMA approved vutrisiran as AMVUTTRA for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy in Europe and the UK. It was also approved to treat transthyretin (TTR) type familial amyloidosis with polyneuropathy in Japan.

Alnylam has not released data from the Stargardt program that is now being halted, though other trials studying the drug have produced positive results.

"We will continue to evaluate the impact of the IRA and determine the best path toward advancing an RNAi therapeutic as a treatment option for patients with Stargardt Disease," Lindenboom added.

Stargardt disease is a rare hereditary illness presenting clinically as toxic levels of vitamin A accumulating in the patient’s retina and leading to blindness. There is currently no treatment available for the disease, although there has been some movement in the space. 

In May, the FDA granted Fast Track Designation to Belite Bio’s Stargardt therapeutic designed to slow disease progression and vision loss. Nanoscope Therapeutics and Alkeus are also developing therapeutics for the disease, with both companies currently conducting Phase II trials. 

As for the rest of its pipeline, Yvonne Greenstreet, CEO of Alnylam, said it is on track to reach its five-year goals, dubbed Alnylam P5x25.

“The rest of our pipeline continues to progress well...We are encouraged by our steady and continuous execution on all fronts, and believe we are on track to achieve our Alnylam P5x25 goals and become a top-tier biotech company," Greenstreet said. 

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